[Senate Hearing 115-341]
[From the U.S. Government Publishing Office]
S. Hrg. 115-341
NOMINATION OF SCOTT GOTTLIEB, M.D., TO SERVE AS COMMISSIONER OF FOOD
AND DRUGS
=======================================================================
HEARING
OF THE
COMMITTEE ON HEALTH, EDUCATION,
LABOR, AND PENSIONS
UNITED STATES SENATE
ONE HUNDRED FIFTEENTH CONGRESS
FIRST SESSION
ON
EXAMINING THE NOMINATION OF SCOTT GOTTLIEB, M.D., TO SERVE AS
COMMISSIONER OF FOOD AND DRUGS
__________
APRIL 5, 2017
__________
Printed for the use of the Committee on Health, Education, Labor, and Pensions
[GRAPHIC(S) NOT AVAILABLE IN TIFF FORMAT]
Available via the World Wide Web: http://www.gpo.gov/fdsys/
U.S. GOVERNMENT PUBLISHING OFFICE
25-027 PDF WASHINGTON : 2018
COMMITTEE ON HEALTH, EDUCATION, LABOR, AND PENSIONS
LAMAR ALEXANDER, Tennessee, Chairman
MICHAEL B. ENZI, Wyoming PATTY MURRAY, Washington
RICHARD BURR, North Carolina BERNARD SANDERS (I), Vermont
JOHNNY ISAKSON, Georgia ROBERT P. CASEY, JR., Pennsylvania
RAND PAUL, Kentucky AL FRANKEN, Minnesota
SUSAN M. COLLINS, Maine MICHAEL F. BENNET, Colorado
BILL CASSIDY, M.D., Louisiana SHELDON WHITEHOUSE, Rhode Island
TODD YOUNG, Indiana TAMMY BALDWIN, Wisconsin
ORRIN G. HATCH, Utah CHRISTOPHER S. MURPHY, Connecticut
PAT ROBERTS, Kansas ELIZABETH WARREN, Massachusetts
LISA MURKOWSKI, Alaska TIM KAINE, Virginia
TIM SCOTT, South Carolina MAGGIE HASSAN, New Hampshire
David P. Cleary, Republican Staff Director
Lindsey Ward Seidman, Republican Deputy Staff Director
Evan Schatz, Minority Staff Director
John Righter, Minority Deputy Staff Director
(ii)
C O N T E N T S
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STATEMENTS
WEDNESDAY, APRIL 5, 2017
Page
Committee Members
Alexander, Hon. Lamar, Chairman, Committee on Health, Education,
Labor, and Pensions, opening statement......................... 1
Murray, Hon. Patty, a U.S. Senator from the State of Washington,
opening statement.............................................. 3
Murphy, Hon. Christopher, a U.S. Senator from the State of
Connecticut.................................................... 6
Enzi, Hon. Michael B., a U.S. Senator from the State of Wyoming.. 15
Sanders, Hon. Bernard, a U.S. Senator from the State of Vermont.. 17
Young, Hon. Todd, a U.S. Senator from the State of Indiana....... 19
Kaine, Hon. Tim, a U.S. Senator from the State of Virginia....... 20
Scott, Hon. Tim, a U.S. Senator from the State of South Carolina. 22
Warren, Hon. Elizabeth, a U.S. Senator from the State of
Massachusetts.................................................. 24
Burr, Hon. Richard, a U.S. Senator from the State of North
Carolina....................................................... 26
Casey, Hon. Robert P., Jr., a U.S. Senator from the State of
Pennsylvania................................................... 28
Paul, Hon. Rand, a U.S. Senator from the State of Kentucky....... 30
Hatch, Hon. Orrin G., a U.S. Senator from the State of Utah...... 33
Bennet, Hon. Michael F., a U.S. Senator from the State of
Colorado....................................................... 35
Cassidy, Hon. Bill, a U.S. Senator from the State of Louisiana... 36
Franken, Hon. Al, a U.S. Senator from the State of Minnesota..... 38
Roberts, Hon. Pat, a U.S. Senator from the State of Kansas....... 40
Baldwin, Hon. Tammy, a U.S. Senator from the State of Wisconsin.. 41
Murkowski, Hon. Lisa, a U.S. Senator from the State of Alaska.... 43
Whitehouse, Hon. Sheldon, a U.S. Senator from the State of Rhode
Island......................................................... 45
Hassan, Hon. Margaret Wood, a U.S. Senator from the State of New
Hampshire...................................................... 47
Witness
Gottlieb, Scott, M.D., Nominee to Serve as Commissioner of Food
and Drugs,..................................................... 7
Prepared statement........................................... 10
ADDITIONAL MATERIAL
Statements, articles, publications, letters, etc.
Letters of Support........................................... 54
Response by Scott Gottlieb to questions of:
Senator Alexander........................................ 59
Senator Murray........................................... 60
Senator Enzi............................................. 83
Senator Sanders.......................................... 84
Senator Burr............................................. 86
Senator Casey............................................ 87
Senator Isakson.......................................... 90
Senator Franken.......................................... 92
Senator Paul............................................. 94
Senator Bennet........................................... 96
(III)
Senator Collins.......................................... 97
Senator Whitehouse....................................... 100
Senator Cassidy.......................................... 105
Senator Baldwin.......................................... 106
Senator Hatch............................................ 107
Senator Murkowski........................................ 109
Senator Murphy........................................... 110
Senator Roberts.......................................... 113
Senator Warren........................................... 114
Senator Kaine............................................ 125
Senator Hassan........................................... 127
NOMINATION OF SCOTT GOTTLIEB, M.D., TO SERVE AS COMMISSIONER OF FOOD
AND DRUGS
----------
WEDNESDAY, APRIL 5, 2017
U.S. Senate,
Committee on Health, Education, Labor, and Pensions,
Washington, DC.
The committee met, pursuant to notice, at 10:02 a.m. in
room SD-430, Dirksen Senate Office Building, Hon. Lamar
Alexander, chairman of the committee, presiding.
Present: Senators Alexander, Enzi, Burr, Paul, Cassidy,
Young, Hatch, Roberts, Murkowski, Scott, Murray, Sanders,
Casey, Franken, Bennet, Whitehouse, Baldwin, Murphy, Warren,
Kaine, and Hassan.
Opening Statement of Senator Alexander
The Chairman. The Senate Committee on Health, Education,
Labor, and Pensions will please come to order.
This morning, we are holding a hearing on the nomination of
Dr. Scott Gottlieb to be the next Commissioner of Food and
Drugs.
Senator Murray and I will each have an opening statement
then we will introduce Dr. Gottlieb. After his testimony,
Senators will each have two rounds of 5-minute questions, if
they wish to.
Last year, the most important legislation that Congress
enacted was the 21st Century Cures law. Those are not my words;
they are Majority Leader McConnell's words.
The reason it was such an important bill is that it will
drive forward research and the extraordinary medical miracles
that are in the works and that have the potential to affect
every American family.
Dr. Francis Collins, at the National Institutes of Health,
has talked about some of the discoveries that he predicts are
possible in the next decade: Non-addictive painkillers; hearts
rebuilt from our own stem cells; a universal flu vaccine; an
HIV/AIDS vaccine; an artificial pancreas for diabetes patients
who have spent decades injecting themselves with insulin.
The key to making these miracles a reality is not just
investment in research, but a regulatory process that is
efficient and effective enough to bring safe discoveries to
patients in a timely way.
The Food and Drug Administration has always been important,
but there has never been a more important time to capitalize on
the significant funding Congress has given to medical research,
and to realize the promise of 21st Century Cures.
Dr. Gottlieb, congratulations to you on your nomination.
Welcome to you and to your family members who are here. I hope
you will introduce them at the appropriate time. We have
enjoyed having the opportunity to visit with you in my office.
If confirmed to lead the Food and Drug Administration as
its commissioner, you will be in charge of steering the agency
responsible for assuring the safety and effectiveness of our
Nation's medical products and protecting our country's food
supply. My hope is you will help move the agency forward so
that America's patients benefit from the remarkable discoveries
our Nation's researchers are working on.
The FDA affects nearly every single American. It regulates
a quarter of all consumer spending in the United States, over
$4 trillion annually.
It is responsible for prescription drugs for humans and
animals, medical devices, biologics, dietary supplements,
cosmetics, over the counter medications, food, and tobacco
products. It is a vital mission, and we all want to make sure
the right person is leading it.
The President has nominated you to do that job, and like
every full-time nominee, you have been through an exhaustive
process to make sure you do not have conflicts of interest or
other problems in your background.
The President announced your nomination on March 10, after
an extensive vetting process by the White House and the FBI.
Your official nomination was received on March 27 by the
Senate. Eight days ago on March 28, this committee received a
letter from the Office of Government Ethics, which carefully
reviewed your financial information and found that, with
several recusals which you have committed to do, you are, ``In
compliance with applicable laws and regulations governing
conflicts of interest.''
In accordance with our committee rules, you have submitted
your committee paperwork to Senators on March 31, 5 days before
this hearing. You have offered to meet with every Senator on
this committee. You have met with every Democratic Senator and
all but two Republican Senators.
That brings us to today. You come here with impressive
qualifications.
You were a practicing physician and a hospitalist for many
years receiving your medical degree at Mount Sinai and your
residency at the Mount Sinai Hospital.
We will hear more from Senator Murphy about your other
credentials, so there is no need for me to repeat them at this
time, including those in the Health and Human Services, and
your time as a Resident Fellow at the American Enterprise
Institute.
You are a prolific writer and speaker, and no stranger to
testifying to Congress. You have testified here 18 times on a
variety of issues. You are also a cancer survivor. You know
firsthand how medical treatments affect patients and their
families.
I am eager to hear your views today on both the User Fee
reauthorizations and 21st Century Cures. Your first order of
business will be to work with us on the reauthorization of the
User Fee Agreements. We have had over 15 bipartisan briefings
on the User Fees going back to late 2015.
Senator Murray and I have held two bipartisan hearings on
the reauthorization, our second one yesterday. I support
quickly moving the reauthorization recommendations sent to us
in January, and I am committed to working with the
Administration, and all members of this committee, to authorize
the User Fees before August 1.
In addition to drugs and medical devices, you are
responsible for protecting our Nation's food supply and working
to reduce the number of people who get sick from foodborne
illness. Technology is improving and changing the way we
improve food safety. It holds the potential to reduce foodborne
illnesses and deaths.
FDA is a large and diverse organization that faces
management challenges. When I asked Dr. Califf, your
predecessor, his top priority while we were working on 21st
Century Cures, he said it was to give the FDA the authority to
hire and to pay people to do what the agency needs to do. We
included that authority in 21st Century Cures.
I am concerned, as are other members of this committee,
about the Administration's hiring freeze, and how it will
affect the FDA, and how you plan to deal with that, if
confirmed.
Thank you for being here. I look forward to hearing your
testimony on these important issues.
Senator Murray.
Opening Statement of Senator Murray
Senator Murray. Thank you very much, Chairman Alexander.
Dr. Gottlieb, I want to welcome you and your family. Thank
you for being here and for your willingness to serve.
I do want to start by expressing my disappointment about
the limited time we have had to review Dr. Gottlieb's committee
paperwork.
We have the full paperwork on Friday, meaning we have had
just a handful of days to fully understand the extent of Dr.
Gottlieb's unprecedented financial entanglements with the
industries he would regulate as FDA Commissioner, find and
review the more than 800 publications Dr. Gottlieb has listed,
and delve into the wealth of companies and products that raise
concerns about potential conflicts of interest.
Chairman Alexander, as you know, I have repeatedly
stressed--privately and publicly--the importance of a thorough
and complete vetting process for each of President Trump's
nominees. Fully vetting the Administration's nominees should
not be a priority for Democrats alone. Both parties deserve to
make fully informed decisions about the potential leaders of
these critical departments and agencies that we oversee.
Unfortunately, the inexplicable rush to advance this
nomination falls far short of that basic standard. I will
continue to push for a thorough review of Dr. Gottlieb's
nomination. Dr. Gottlieb, I hope you will give clear and
thorough responses to any followup questions after today as
well.
I do appreciate that you responded to my letter requesting
additional information on your clients. I hope you are
committed to leading an agency that responds to and works with
Congress.
I have to say I have been disheartened by the unprecedented
lack of responsiveness by this Administration. Throughout
several nomination processes, Democrats have requested
documents and additional information to assist in our vetting.
Disappointingly, Secretary Price failed to respond to a
single one of the questions this committee asked him following
his confirmation hearing. We have yet to receive a response to
even one of the inquiries we have sent since his confirmation.
Across the Administration, this seems to be the new normal.
No responsiveness. No transparency. No accountability. It is
really frightening and frustrating. It cannot go on. I hope,
given the importance of the work of this agency, that if
confirmed, you will not follow that trend.
Our constituents rely on the work of the FDA every single
day. They trust the food they buy from the grocery store is
safe. That when they go to the emergency room, the drugs and
medical devices used in their care have been held to the
highest standards of approval, and that the FDA's decisions are
based upon science--not politics or ideology--the gold
standard.
As you well know, Dr. Gottlieb, I have a long history of
holding very firm on that particular point. To me, it is
critical the FDA have strong, independent leadership,
especially now in light of President Trump's apparent disregard
for public health.
Dr. Gottlieb, in the limited time we have had to review
your professional history and background, I have grown
increasingly concerned about whether you can withstand
political pressure pushing you to ignore science by upholding
the gold standard, and if you can lead the FDA in an unbiased
way, given your unprecedented industry ties. I will ask you to
address those concerns here today.
I am very interested in how you would ensure independent,
science-based decisionmaking at the FDA if you are confirmed.
During your time at the FDA under the Bush administration,
then-Senator Clinton and I fought long and hard to ensure that
emergency contraception, known as Plan B, would be sold over
the counter to all age groups, consistent with expert
recommendations.
The Administration then ignored the science and made a
decision based on purely ideological grounds, a choice that a
GAO report later called unprecedented.
Dr. Gottlieb, you defended the Administration's ideological
position on behind the counter options for Plan B, allowing
politics to interfere directly with women's access to the
health services they need.
Given the Trump administration's clear willingness to skirt
ethics' rules and pressure Federal employees to jam their
policies through, not to mention their commitment to
undermining women's access to birth control and other health
services, I find that aspect of your professional history
especially troubling.
As I mentioned, I am also very concerned about your
unprecedented financial entanglements, especially given this
Administration's record on this issue from President Trump on
down.
One example of my concerns is in 2012, you were quoted in
the ``Washington Post'' stating,
``If consumers can track their blood sugar levels
using pen and paper, why should the Government have to
clear an application that does the same thing more
reliably?''
You are an investor in Glytec, a medical technology company
that developed software that allows patients and doctors to
manage and adjust insulin therapy using smart phones and
devices, and received an FDA approval for that software in
2012. Not surprisingly, you have also served on Glytec's board
since 2013.
Another example, FDA and Congress have both been engaged in
an ongoing debate about the regulation of medical tests. In a
column related to embattled lab company Theranos, you argued
the regulation of these tests is best left outside of FDA's
jurisdiction, all the while serving on the board of two medical
lab companies that would be directly impacted by your preferred
regulatory scheme.
Dr. Gottlieb, it does trouble me greatly that you appear to
be investing in and advising a company, and then using your
public platform to promote policies that actually benefit that
company in the future.
Meanwhile, reports continue to surface about Secretary
Price's questionable actions on behalf of companies in which he
invested. In fact, this committee should know that just last
Friday, a ProPublica story indicated Secretary Price had
lobbied the Department of Health and Human Services on behalf
of companies on the very same day his broker invested in them
on his behalf.
When the Trump administration released its financial
disclosures just a few days ago, we learned of more financial
entanglements in the health sector.
President Trump's top economic advisor, Gary Cohn, has
millions of dollars in financial investments in a number of
medical technology, drug, and tobacco companies.
President Trump's pick to lead the Justice Department's
Anti-Trust Division--which decides whether to approve the
proposed $54 billion Anthem-Cigna merger--received hundreds of
thousands of dollars as a lobbyist on behalf of Anthem.
You can see there is a concerning pattern here, and we do
not need more of it. I know that, if confirmed, you have agreed
to recuse yourself for 1 year from decisions involving some
companies in which you have invested. I do struggle to see how
this will make sure your views and decisions will not be shaped
by your investments. I will ask for your response on that
today.
Our HELP committee research shows that companies that you
invested in have more than 60 drugs that could come before the
FDA for approval, and companies you worked for have interests
in over 120 drugs that are currently being tested, and that is
unprecedented.
Finally, Dr. Gottlieb, the vast majority of your
professional work is focused on drugs and medical devices, and
I have some concerns with your published positions on a number
of important issues.
On marketing and communications by drug companies for off-
label or unapproved uses of their products, you were quoted in
2006 saying,
``Efforts to limit prescription and scientific
exchange to indications only specified on a label could
slow the most important advances in 21st century
medicine.''
I would argue that over the last 11 years we have seen
incredible advances in medicine, while also ensuring that only
truthful and non-misleading information is given to doctors and
patients.
I am concerned about what you describe as regulatory
overreach by FDA, including your opposition to the regulation
of medical applications, and your rejection of Risk Evaluation
and Mitigation Strategies, or REMS, meant to protect patients:
FDA's central mission.
I am also eager to hear how you will implement recent
legislation passed by this committee, including 21st Century
Cures and the Drug Quality and Security Act, passed to regulate
compounding pharmacies and build a modern supply chain.
Your plans to encourage a more robust market for generics
and biosimilars to help reduce the astronomically high cost of
prescription drugs.
The FDA also does far more than drugs and devices. I hope
in this hearing you will directly address priorities like
keeping tobacco out of the hands of children, ensuring a safe
and nutritious food supply, and other efforts to protect public
health. These are all core responsibilities at the FDA, and I
would be very concerned if you simply aligned with President
Trump's extreme vision and take orders from his Administration.
I will ask about those today.
Again, I really appreciate you and your family who are
doing really well behind you right now, joining us today. I
look forward to hearing from you about whether and how you will
provide strong, independent, and science-based leadership that
families in my State and across the country expect from this
agency.
Thank you very much.
The Chairman. Thank you, Senator Murray.
We will now welcome the nominee Dr. Scott Gottlieb. We
welcome your wife and your daughters, your parents, your
sisters-in-law, as well as your other guests whom you should
feel free to introduce.
Dr. Gottlieb will be introduced by a member of this
committee, Senator Murphy. I will turn it over to him and then
to Dr. Gottlieb for his opening statement.
Statement of Senator Murphy
Senator Murphy. Thank you very much, Chairman Alexander,
for the opportunity to introduce Dr. Scott Gottlieb to this
committee.
I am indeed eager to hear from him today. I have disclosed
to Dr. Gottlieb that I have not made up my mind as to his
nomination as will be clear. We have some serious policy
disagreements between us, but I am very eager to take the
opportunity to welcome an important Connecticut resident to the
HELP committee.
While Dr. Gottlieb was born in New Jersey, he does have
long ties to my State of Connecticut. He has lived in Westport
since 2010. Dr. Gottlieb is joined today by his family
including his wife Allison and their three daughters; I will
let Dr. Gottlieb do the formal introductions.
I would note that Dr. Gottlieb's twin daughters' first
grade class in Westport will be watching part of today's
hearing to learn about our work in Washington. As the father of
an 8-year-old and a 5-year-old, I know that first graders
really want nothing more than to hear about the FDA and watch
the HELP committee in action.
Senator Murphy. I am really glad that they are here with us
today.
Dr. Gottlieb and Allison are active members in their town
and their surrounding community, especially through their
synagogue Temple Israel. Dr. Gottlieb serves on the board of
directors at the temple and Allison helps run a local homeless
shelter in Westport and volunteers in the Bridgeport school
system.
Dr. Gottlieb's connection to our State started when he
attended Wesleyan University in Middletown. He was the editor
of the school newspaper there and a student member of the board
of trustees.
After graduating from Wesleyan with a bachelor's degree in
economics, he went on to receive his medical degree from Mount
Sinai School of Medicine in New York. After completing his
residency at Mount Sinai Medical Center, Dr. Gottlieb then
practiced medicine for 6 years at Stanford Hospital, again in
Connecticut.
If confirmed, Dr. Gottlieb will be returning to the FDA for
the third time. He first served as senior advisor to
Commissioner Mark McClellan for medical technology and then as
director of the medical policy development. During this time,
he would travel back to Connecticut to work at Stanford
Hospital on the weekends.
Dr. Gottlieb also brings a unique perspective to this
committee because he has both been a practitioner and a
patient. He is a survivor of Hodgkin's lymphoma and has served
as a policy board member with the Leukemia and Lymphoma Society
from 2012 to 2014.
Since leaving the FDA, Dr. Gottlieb has primarily worked as
a consultant and advisor to a number of companies including as
a venture partner at New Enterprise Associates. NEA is one of
the largest venture capital firms in the country. It has
investments in many firms in the broader healthcare space. He
also has his own consulting firm. He has been a managing
director of T.R. Winston, a merchant corporate investment
banking firm which has a focus in healthcare.
He has come before various committees in Congress over the
years to offer his opinions as a resident fellow at the
American Enterprise Institute. She is no stranger to the U.S.
Congress. We are proud of him in Connecticut and his commitment
to Stanford Hospital and his community in Westport, and I look
forward to his testimony, and I welcome him to the committee
today.
The Chairman. Thank you, Senator Murphy.
Dr. Gottlieb, we now invite you to give your opening
remarks. Your written statement will be entered into the record
in its entirety.
STATEMENT OF SCOTT GOTTLIEB, M.D., WESTPORT, CT, NOMINEE TO
SERVE AS COMMISSIONER OF FOOD AND DRUGS
Dr. Gottlieb. I would like to just take a moment to
introduce my family.
My wife Allison is here with my three daughters, Alex, Em,
and Dillon. Do you want to say hi?
My mother and father are here. My mother is a school
teacher in New Jersey. My father is a physician and a veteran
of the Vietnam War. He was stationed in Cam Ranh Bay and
wounded in that theater.
Chairman Alexander, Ranking Member Murray, members of the
committee, thank you for the invitation to testify this
morning. I am honored to appear before you today as the
President's nominee to be the next Commissioner of Food and
Drugs.
I come before you today humbled by the realization that the
lives and futures of families like mine are affected by the
decisions made by the FDA.
Should you choose to confirm me, I will make it my mission
to fight for those families every single day, and ensure that
the FDA puts their interests first in everything we do.
I have seen the importance of FDA's work as both a doctor
and a patient.
I graduated from Wesleyan University in Middletown, CT and
went on to graduate from the Mount Sinai School of Medicine,
where I also completed a residency in Internal Medicine.
I have had the honor to serve in senior roles at both CMS
and FDA.
I have practiced medicine as a hospitalist physician,
taking care of hospitalized patients. I have tried to ease
suffering and illness as a physician, and I have had both
visited upon me. I am a cancer survivor. I was treated for
cancer during my last tour at FDA, so I know the importance of
what American medicine does and what the FDA does for every one
of us.
For the last 10 years, I have been a policy analyst and an
entrepreneur starting and building businesses. I have advised
and invested in early stage medical technology and healthcare
services companies with the hope that some of these innovations
could improve the medical technology that we use and the
systems through which we deliver care.
Some of these endeavors were successful. Some were not. For
many others, it is still too early to tell. That is the
unpredictable nature of innovation in this dynamic sector. It
is a dynamism that I have come to know well from working on the
regulatory, policy, clinical, and business aspects of these
enterprises.
I am proud of the projects I have worked on, and what I
have learned in the process. The things I have done--my
accomplishments, my failures, and everything in between--have
shaped who I am today. Collectively, they have helped inform my
values and my perspectives. But among other things, they have
taught me the need for an absolutely objective regulatory
watchdog over this field.
If confirmed, I will lead the FDA as an impartial and
passionate advocate for the public health. I know what is at
stake here.
People's lives are literally on the line when it comes to
the decisions that the FDA makes in its oversight and its
enforcement of Congress' laws. The American people deserve to
trust that the agency is led in an impartial manner, guided
only by the science that informs its work and an abiding faith
to the public health. That is the mandate by which I would lead
this agency, if I were fortunate enough to win your approval.
I will respect the intent of Congress. I will make sure the
laws you passed are implemented in a timely fashion and in the
way you intended. Every decision I make will be guided by the
advice of career experts. I will be guided by the scientific
rigor that the public deserves and the rigor that the hard
challenges before this agency demands. It is to take on these
challenges that I seek this role.
We are at an inflection point in biomedical science. New
technologies give us a fundamental chance to cure many
intractable diseases. We have more opportunities to improve our
diets and our health with the foods we eat.
In areas where there is an inherent, obvious, and seemingly
unavoidable risk related to certain consumer products--whether
it is combustible tobacco or dangerously addictive opioid
drugs--we have the opportunity to help consumers move to less
risky alternatives.
This owes to the foresight of Congress in envisioning paths
to reduced harm as an animating principle in FDA regulation. I
want to build on these opportunities and achievements.
I want to use the authorities Congress recently included in
the 21st Century Cures Act to develop a template to lean
forward in these areas. We need to make sure we are getting the
most bang for our regulatory buck. That means being cognizant
of the risks and being sure that we are not adding to consumer
costs without improving consumer safety.
We must constantly ask ourselves are we doing everything we
possibly can? Does the FDA have the policies and processes in
place to play its part in tackling the important public health
issues of our day?
We should be reminded always that we save lives by allowing
good things to happen, but we also save lives when we keep bad
things from happening. FDA's enforcement tools are a bedrock of
its mission. We should reject the false dichotomy that it all
boils down to a choice between speed and safety.
If the FDA is leaning forward in these areas of new
technology--if it is investing in good tools for doing its own
work, and better science for evaluating regulatory questions,
in other words, if we are doing our jobs and leveraging the
authorities you have given us in new congressional mandate--we
could have better efficiency and better safety, and also remain
faithful to FDA's gold standard for regulatory conduct.
I have seen FDA's positive impact in my prior roles at the
agency. I am seeking this new role because I am drawn to FDA's
unique spirit of public health protection that inspires its
work and its workforce.
I am drawn to the opportunities we have to leverage FDA's
platform and its new authorities and resources to enable
advances in medicine and science to safely reach consumers.
I am drawn by the challenges the agency confronts as it
tries to address and enable Americans to make the most of this
unique moment in science.
I hope to earn your confidence and support in delivering on
these opportunities.
Thank you again for the opportunity to appear before you
this morning and I look forward to answering your questions.
[The prepared statement of Dr. Gottlieb follows:]
Prepared Statement of Scott Gottlieb, M.D.
Chairman Alexander, Ranking Member Murray, members of the
committee: Thank you for the invitation to testify this morning.
I am honored to appear before you today as the President's nominee
to be the next Commissioner of Food and Drugs.
I come before you today humbled by the realization that the lives
and futures of families like mine are affected by the decisions made by
FDA.
Should you choose to confirm me, I will make it my mission to fight
for those families every single day, and ensure that FDA puts their
interest first in everything we do.
I have seen the importance of FDA's work as both a doctor and a
patient.
I graduated from Wesleyan University, in Middletown, CT and went on
to graduate from the Mount Sinai School of Medicine, where I also
completed a residency in Internal Medicine.
I have had the honor to serve in senior roles at both CMS and FDA.
I practiced medicine as a hospitalist physician, taking care of
hospitalized patients.
I have tried to ease suffering and illness as a physician, and I
have had both visited upon me--I am a cancer survivor, I was treated
for cancer during my last tour at FDA, so I know the importance of what
American medicine does--and what the FDA does--for every one of us.
For the last 10 years, I have been a policy analyst and an
entrepreneur, starting and building businesses.
I have advised and invested on very early stage medical technology
and healthcare services companies with the hope that some of these
innovations could improve the medical technology that we use, and the
systems through which we deliver care.
Some of these endeavors were successful. Some were not. For many
others, it is still too early to tell.
That is the unpredictable nature of innovation in this dynamic
sector.
It is a dynamism that I have come to know well from working on the
regulatory, policy, clinical, and business aspects of these
enterprises.
I am proud of the projects I have worked on, and what I have
learned in the process. The things I have done--my accomplishments, my
failures, and everything in between--have shaped who I am today.
Collectively, they have helped inform my values and my
perspectives.
Among other things, they have taught me the need for an absolutely
objective regulatory watchdog over this field.
If confirmed, I will lead the FDA as an impartial and passionate
advocate for public health.
I know what is at stake here. People's lives are literally on the
line when it comes to the decisions FDA makes, its oversight, and its
enforcement of Congress' laws.
The American people deserve to trust that the agency is led in an
impartial manner--guided only by the science that informs its work--and
an abiding faith to the public health.
That is the mandate by which I would lead this agency, if I were
fortunate enough to win your approval.
I will respect the intent of Congress.
I will make sure the laws you passed are implemented in a timely
fashion and in the way you intended.
Every decision I make will be guided by the advice of career
experts.
I will be guided by the scientific rigor that the public deserves,
and the rigor that the hard challenges before this agency demand.
It is to take on these challenges that I seek this role. We are at
an inflection point in biomedical science.
New technologies give us a fundamental chance to cure many
intractable diseases.
We have more opportunities to improve our diets and our health with
the foods we eat.
In areas where there is an inherent, obvious, and seemingly
unavoidable risk related to certain consumer products--whether its
combustible tobacco or dangerously addictive opioid drugs--we have the
opportunity to help consumers move to less risky alternatives.
This owes to the foresight of Congress, in envisioning paths to
reduced harm as an animating principle in FDA regulation.
I want to build on these opportunities and achievements.
I want to use the authorities Congress recently included in the
21st Century Cures Act to develop a template to lean forward in these
areas.
We need to make sure we are getting the most bang for our
regulatory buck. That means being cognizant of risks and being sure
that we are not adding to consumer costs without improving consumer
safety.
We must constantly ask ourselves, are we doing everything we
possibly can? Does FDA have the policies and processes in place to play
its part in tackling the important public issues of our day?
We should be reminded always, that we save lives by allowing good
things to happen, but we also save lives when we keep bad things from
happening. FDA's enforcement tools are a bedrock of its mission.
We should reject a false dichotomy that it all boils down to a
choice between speed and safety.
If FDA is leaning forward in areas of new technology, if it is
investing in good tools for doing its own work, and better science for
evaluating regulatory questions--in other words, if we are doing our
jobs and leveraging the authorities you have given us in new
congressional mandate--we can have better efficiency, and better
safety, and also remain faithful to FDA's gold standard for regulatory
conduct.
I have seen FDA's positive impact in my prior roles at the agency.
I am seeking this new role because I am drawn to FDA's unique
spirit of public health protection that inspires its work and its
workforce.
I am drawn to the opportunities we have to leverage FDA's
platform--and its new authorities and resources--to enable advances in
medicine and science to safely reach consumers.
I am drawn by the challenges the agency confronts as it tries to
enable Americans to make the most of this unique moment in science.
I hope to earn your confidence and support in delivering on these
opportunities.
Thank you again for the opportunity to appear before you this
morning. I would be happy to answer any questions.
The Chairman. Thank you, Dr. Gottlieb.
We will now begin two rounds of 5-minute questions for
Senators who wish to ask those.
Dr. Gottlieb, you have written a lot and said a lot. If one
were looking for your articles, where would one find them?
Dr. Gottlieb. I sent you most of the URL's, I believe.
The Chairman. Are they on the Web?
Dr. Gottlieb. They are. Most of them are on the Web except
for the ones that are very old. I have put together, we have
put together, a binder of all of them. I would be happy to make
it available to the committee.
The Chairman. Thank you.
As far as the opportunity to review, Dr. Gottlieb, I would
go back through what I said in my statement. He has visited
with every Senator who wanted to visit with him on the
committee. He has complied with all the requirements of the
Office of Government Ethics who have agreed that with the
recusals he has agreed to do, he will not have a conflict of
interest.
All of his papers are in, in a timely way according to the
rules of our committee. We will have a chance to question him
today through two rounds of questions, if members would like to
do that. It will be at least 2 weeks before we have a chance to
have a markup. Members will have a chance to thoroughly
consider him and his record before casting a vote as to whether
to report him to the full Senate.
As to his work with companies that have to do with drugs
and food, that is not so unusual for someone who is going to be
head of the FDA. In my view, it helps to have somebody who
knows something about the subject.
An example would be Dr. Califf, his predecessor, who
supports Dr. Gottlieb's nomination. Dr. Califf from Duke
University was previously employed by over 20 drug and device
companies between 2010 and 2014, before he became Commissioner
of the FDA. That did not disqualify him from serving. I
supported him. He was approved by the Senate 89 to 4.
I am glad to know that you have a background and experience
in the issues before you.
As far as Dr. Califf goes, let me say a question to you
that I asked him. When we were working on 21st Century Cures, I
asked him his No. 1 priority for the Food and Drug
Administration. He said it was to be able to hire and pay
personnel at the FDA so they could deal with these lifesaving
drugs and devices that are coming the way of the FDA. The
Administration has placed a hiring freeze that seems to be
interfering with that.
Should you be confirmed, what would you do about that?
Dr. Gottlieb. Senator, thanks for the question.
I understand how important a strong workforce is to FDA and
helping FDA maintain its gold standard. I think FDA is unique
among Federal agencies in that it does not pass through most of
its money. Most of the money that Congress allocates to the FDA
gets spent at the FDA on the work.
It is incumbent upon us to have a world class workforce
that we are providing the proper tools to and the proper
training to, to maintain that very high standard.
I have, through my career and my time at FDA, been
committed to making sure we have a very strong workforce at
FDA. I have spoken out about that. I will continue to make my
views known on that issue.
The Chairman. I hope that you will because this was not a
minor issue with us. It was his top priority and it was a top
priority of ours, both Democrats and Republicans. We want 21st
Century Cures to be a reality and that is an important part of
it.
Dr. Collins, the head of the National Institutes of Health,
has predicted a number of medical miracles over the next 10
years. One of the most important would be the possibility of
the discovery of non-addictive pain medicines, which would make
the opioid epidemic much less of a problem by providing a
substitute. The President has indicated a similar priority.
Many of us have on both sides of the aisle.
What could you do as Commissioner of the FDA working with
Dr. Collins, the President, and others in the Administration,
and with us to be forward leaning on accelerating and finding a
discovery of non-addictive pain medicines, which might be more
than anything else to relieve the opioid epidemic?
Dr. Gottlieb. Thank you, Senator.
The opioid epidemic in this country is having staggering
human consequences. I think that this is the biggest crisis
facing the agency, and is going to require dramatic action on
the part of whoever steps into the agency, and I hope the
Senate confirms me to take on this challenge.
It is going to require an all-of-the-above approach. There
are some things we are going to have to do to really push the
boundaries of the policy framework in this area, and that does
include re-evaluating the framework for how we can develop
alternatives to opioid drugs.
It also includes looking at device alternatives to opioid
drugs and looking at devices in the context of drugs. I would
also add to that looking at medically assisted therapy to help
people live a life of sobriety after they have become addicted.
There is going to be a need--
The Chairman. Thank you.
Dr. Gottlieb. Thank you.
The Chairman. My time is running out. It is out.
I will submit for the record a question about a persistent
issue with the FDA not properly clearing medical device
shipments, and I hope you will look into it.
Dr. Gottlieb. Absolutely.
The Chairman. Thank you.
Senator Murray.
Senator Murray. Dr. Gottlieb, you do wear an extraordinary
number of hats. You are a partner in an investment bank, a
venture partner in a large venture capital firm, a CEO or co-
CEO of two health companies, and an individual investor in more
than 20 health companies. You have sat on various types of
boards for 16 companies including two of the world's largest
pharmaceutical companies. You also publish regularly, make
speeches, consult for a number of large drug companies, and
practice medicine.
My question for you is about one of those hats, your role
as a venture partner for New Enterprise Associates, the largest
venture capital firm in the world. You revealed in your ethics
agreement that you hold a direct financial interest in six
client companies including a laboratory and pathology test
company, two health insurance companies, a medical equipment
and supply company, a rapidly growing company that buys
radiology practices, and the third largest dialysis firm in the
country. You have also received hundreds of thousands of
dollars from New Enterprise Associates as a consulting
retainer.
If confirmed, you are committed to making the required
steps to divest and recuse yourself from a select number of
companies. Those were really just the tip of the iceberg.
Until recently, you were also a consultant and an investor
in two large firms operated by NEA. Those funds respectively
have investments in an additional 75 health-related companies
who have dozens of new drugs that could potentially come before
FDA for approval.
Those companies include Cerecor, which is a
biopharmaceutical company with eight drugs in its pipeline;
Galera, a biotechnology company with cancer drugs in
preclinical and clinical trials; CRSPR, a company developing
novel therapeutics based on the new gene editing technology;
and Intact Vascular, it is a medical device company focusing on
treating vascular disease.
Your involvement in so many companies likely to have
business before the FDA--including key decisions by the agency
on the safety and effectiveness of the company's drugs,
devices, and products--is unprecedented.
Will you recuse yourself for the 2 years laid out in the
Trump Ethics Pledge from all companies in which NEA is an
investor?
Dr. Gottlieb. Thank you, Senator. Thank you for the
question.
I recognize the importance to maintain my impartiality in
this role and make sure I am taking the proper steps. I have
taken the proper steps through the OGE process and I look
forward to working with the ethics officials at HHS and FDA to
have continued discussions about what additional steps I should
take to make sure I am fully compliant with the law if I am
confirmed into this role.
As you noted, all of the investments I have made in NEA are
healthcare services companies and a lot of my work at NEA was
related to healthcare services, not to their life sciences
portfolio.
Senator Murray. I am aware that you have met all your
minimum legal obligations, but that does not mean you are
recused from involvement in decisions that affect all of those
companies.
What I am concerned about is how your involvement with so
many companies shapes your priorities. Tell me how you answer
that.
Dr. Gottlieb. Senator, I am going to work hard to make sure
I preserve my integrity in this role and the integrity of the
FDA. I get it. I understand how important the impartiality of
this agency is so that people can continue to have trust in the
decisions that FDA makes.
I am going to make sure that I have a process in place, if
I am confirmed into this role, in my front office for helping
me to manage whatever recusals I do have to put into place, and
I will consult with ethics officials. This is exceedingly
important to me. I want to earn and keep the public's trust.
Senator Murray. OK. The FDA is considered the gold standard
for approval around the world because the FDA reviews raw data
to determine if a new drug is both safe and effective, and that
scientific and nonpolitical process is critical to the trust of
patients and families across this country.
Do you commit to upholding that standard?
Dr. Gottlieb. Yes, I do, Senator.
Senator Murray. That is good to hear, but the Trump
administration you will be working under has been clear about
their disregard for our country's leadership in this respect.
Trump claims the FDA approval process is slow and
burdensome. Secretary Price has said, ``One of my passions is
to get Government out of the way of innovation.''
Do you commit to making decisions on safety and efficacy of
new products based on the standards in the current law and the
science, and not bow to political pressure from the
Administration?
Dr. Gottlieb. Senator, thank you for the question.
I am going to be guided by the science, I am going to be
guided by the expertise of the career staff, and I am going to
be guided by impartiality and what is good for patients as a
physician.
Senator Murray. OK. I have an additional question on that,
but to be clear here, you are willing to stand up to the
Administration if they put political pressure on you?
Dr. Gottlieb. Senator, thanks for the question.
For those who have worked with me, I have not been shy
about offering my unvarnished advice. We mentioned 866 articles
I have written where I offered very clear thoughts, and I am
going to continue to offer people my very clear thoughts on
whatever issues I am asked to apply it on, including my bosses.
Senator Murray. Thank you very much.
The Chairman. Thank you, Senator Murray.
Senator Enzi.
Statement of Senator Enzi
Senator Enzi. Thank you, Mr. Chairman.
Thank you Dr. Gottlieb for being willing to take this on
and obviously to leave all those things behind that were very
profitable. Working in Government is not that profitable and
your expertise is greatly needed.
One of the most common themes that we talk about in medical
innovation is precision medicine and advances in science that
put formerly inconceivable cures within reach. The FDA has the
authority and, in some cases, does work with manufacturers to
do adaptive trial designs that meet the needs of a particular
disease population more appropriately.
I also hear about frustrations--that it can be difficult to
work out with the FDA an approach to clinical trial design for
small or more complicated populations.
When I hear this, I think about the difficulty that
manufacturers with their innovative products can face in the
drug approval process. They have to fight it out with the FDA,
and that can be a delicate balance for these companies, and can
mean significant delays in moving forward through the approval
process. You have written extensively about this sort of thing.
Can you tell me what you view as the biggest challenge to
addressing this problem?
Dr. Gottlieb. Senator, thanks for the question.
We have a real opportunity to try to improve the efficiency
of the development process and give us better tools for
ensuring the safety and effectiveness of drugs at reduced costs
with more efficiency. What was done in the Cures Act gives us a
great template for doing that.
If I am confirmed into this role, I am going to look
forward to trying to make efficient implementation of the Cures
Act.
I do not want some future Commissioner to be sitting at
this table 10 years from now discussing that they have not
fully implemented Cures the way--when I was at FDA--we were
still implementing provisions of FDAMA 10 years after it had
passed.
I am going to be focused on doing that, and you provided
those provisions in that bill.
Senator Enzi. Thank you.
Congress did enact the Food and Drug Administration Safety
and Innovation Act in 2012 in which it reinforced its support
for the use of accelerated approval to speed up access to novel
treatments.
The FDA has been very clear that drugs approved this way do
not have a different set of requirements to meet or a lesser
standard. These are drugs reviewed for the same safety and
effectiveness requirements as any drug that receives FDA
approval.
However, there is some sense outside of the FDA that
accelerated approval does mean a lesser standard. This is
concerning as it can impact patients and their access to these
drugs.
Will you commit to working to clarify that accelerated
approval of drugs are not investigational? What do you see as
any specific actions that the FDA can take to dispel the notion
that these products are investigational in nature, and to
clarify that these drugs are fully approved?
Dr. Gottlieb. Thank you for the question, Senator.
There is one standard for safety and effectiveness, and no
commissioner can change that standard. It has been enshrined in
law many times. I will continue to affirm that there is a
single standard for safety and effectiveness.
The FDA does have the ability to make certain
accommodations owing to congressional statute in the kinds of
requirements it can pose to demonstrate that standard; but the
standard is a single standard.
Senator Enzi. Thank you.
Changing gears again. For years, we have talked in this
committee about biosimilars. Since the launch of the Biosimilar
User Fee Agreement in 2012, there have been four approvals.
However, only two made it onto the market. By 2021, there will
be more than 70 biological agents coming off of the patent
protection and there could be a significant amount of activity
in the biosimilar space.
What kind of progress do you hope to see in the review of
biosimilars? Knowing that the FDA has been slow in implementing
the biosimilar pathway, what do you see as improvements that
can be made?
Dr. Gottlieb. Thank you for the question, Senator.
Many of us have been disappointed by the economic savings
we have seen from biosimilars so far. I do think that there is
a lot of opportunity for these to have a meaningful impact on
consumers and spending going forward.
I was at FDA when we first started to contemplate a pathway
for biosimilars and started to do it through the 505(b)(2)
process.
If I have the opportunity to be confirmed into this role, I
am going to want to make sure that we are implementing guidance
in a timely fashion to try to create the kinds of opportunities
for competition. Issues like can biosimilars be used
interchangeably in the market, could have a meaningful impact
on the potential to get economic savings.
Congress recently directed the agency to put out that
guidance. I want to make sure that we are putting out those
documents in a timely fashion in evaluating these questions.
Senator Enzi. Thank you for the conciseness of your
answers.
I have a couple of other questions that I will just submit.
Thank you, Mr. Chairman.
The Chairman. Thank you, Senator Enzi.
Senator Sanders.
Statement of Senator Sanders
Senator Sanders. Thank you, Mr. Chairman.
Welcome, Dr. Gottlieb. I enjoyed our conversation in the
office, and welcome to your family, and all the first graders
in Connecticut.
Dr. Gottlieb. Thank you.
Senator Sanders. Let me begin, Mr. Chairman, by expressing
my amazement, I think, shared by millions of Americans that we
have a President who ran for President, and perhaps won the
election, by saying that he was a ``champeen'' of the working
class of America.
He said that he would not cut Social Security, and
Medicare, and Medicaid. You know what? He appointed his key
advisors, people like Representative Mulvaney and
Representative Price, who spent their entire careers trying to
do exactly that.
He said that he would, ``Drain the Swamp.'' He now has more
billionaires in his administration than any President in
American history.
He said he would provide, ``Health insurance for
everybody,'' but he just supported an embarrassing, disastrous
healthcare proposal that would have thrown 24 million people
off of health insurance.
He said he was, ``Going to stop Wall Street from getting
away with murder,'' but he has drained half of Goldman Sachs
into his administration.
In other words, he ran for President saying one thing, and
he ended up doing something exactly the opposite, which brings
us to Dr. Gottlieb.
Candidate Trump and, in fact, President Trump after he was
elected, made some very important and, in my view, correct
statements about the outrageously high prices that we pay for
prescription drugs in this country.
I must say, Mr. Chairman, that if you go out to the
American people and you ask them the most important issue that
concerns them in healthcare, do you know what it will be? It is
the fact that we pay, by far, the highest prices in the world
for prescription drugs, an issue which has not yet been even
mentioned in this hearing.
The fact that almost one out of five adults in this
country, who get a prescription from their doctor, cannot
afford to fill that prescription.
The fact that in my State, and I expect in Tennessee, you
have senior citizens who are cutting their medicine in half.
We have heard from oncologists and other doctors that
people are dying. Their patients are dying because they cannot
afford the outrageously high prices of prescription drugs.
You know what? Trump talked about that and he was right. As
Senator Murray indicated, he nominates somebody who has
received millions of dollars from the pharmaceutical industry.
According to the ``New York Times,'' he has received more than
$150,000 in compensation from Vertex Pharmaceuticals, a company
that is charging more than $250,000 a year for drugs to treat
cystic fibrosis.
Even more interestingly, Trump runs for office and he says,
``Look. There are, among other things, two ways that
we can deal with the high cost of prescription drugs.
We should be able to re-import lower cost prescription
drugs from Canada and other countries. We should be
able to negotiate prices with Medicare.''
Yet, Dr. Gottlieb writes an Op Ed for ``Forbes,'' in which
he directly contradicts what Trump has to say about re-
importation. That was the thrust of his Op Ed.
I ask Dr. Gottlieb, why would President Trump appoint
somebody to the very important position of head of the FDA
whose views run diametrically opposite to what he said during
the campaign?
Do you support, as Trump does, the re-importation of low
cost medicine from Canada and from other countries, allowing
Americans to save significant sums of money on the medicine
they need?
Dr. Gottlieb. Thank you, Senator, for the question.
I obviously cannot speak to why the President of the United
States nominated me for this role. I can tell you, as we
discussed in your office, I have a lot of ideas that I want to
work on right away for how I think we can get more product
competition onto the market.
You and I talked about the fact that many complex drugs
have monopolies effectively in perpetuity because of--
Senator Sanders. I apologize for having interrupted you. I
have very little time left.
A quote from your article in ``Forbes,''
``The problem is that drug importation does not
address any of these core challenges. In fact, the
imported drugs may end up being quite expensive.''
That is fine. That is your opinion.
It happens not to be the opinion of the guy you will be
working with, and I just find it amazing that Trump says
something during the campaign, and then appoints people who
have radically different ideas.
What is your view on the need for Medicare to negotiate
prescription drug prices with the pharmaceutical industry?
Dr. Gottlieb. Senator, it is true. I have written a lot of
things on a lot of different subjects, including issues around
Medicare.
I am coming before you for the position at the FDA and I am
going to get asked my position on a lot of different subjects
that fall outside of FDA's purview. I would do the agency, that
I hope to lead, no favors by wading into other territory.
Senator Sanders. Dr. Gottlieb, as you are more than aware
of, part of the FDA's mission is to make medicines--this is the
FDA mission not Bernie Sanders' idea--``More effective, safer,
and more affordable.''
That is within the jurisdiction of the FDA.
Dr. Gottlieb. that is my goal, Senator.
Senator Sanders. Your goal is, from what I am hearing, you
oppose the ideas that President Trump said he was going to do
to the American, for the American people.
Sounds a little bit strange to me, Mr. Chairman.
The Chairman. Thank you, Senator Sanders.
Senator Young.
Statement of Senator Young
Senator Young. Dr. Gottlieb, thanks so much for being here
today.
First to my colleagues who are concerned about the price of
prescription drugs, there are a number of ways we can tackle
that issue. I look forward to engaging with them for, perhaps,
some alternative paths to get where I know others want to go
that I think could be palatable in a bipartisan way. With that
said, we will discuss that offline, as opposed to negotiating
publicly here.
On the issue of safety and efficacy, which has already been
addressed in a fulsome way, you have responded to some of the
questions. Thank you.
I just want to give you the opportunity to discuss the
existing standards. You have indicated you support the existing
standards, Doctor, with respect to the drug approval process.
Do those standards, however, provide enough flexibility for
rare and common diseases, chronic and acute diseases, and the
varying levels of knowledge we have about different diseases?
Dr. Gottlieb. Thank you for the question, Senator.
There are parts of the agency that have leaned forward to
try to use some of the new authorities that Congress has given
to the FDA to contemplate how to incorporate better scientific
principles into the evaluation of products, particularly
targeted to unmet medical needs.
I would commend, in particular, the Oncology Division and
what they have done, as an outside observer, to try to lean
forward in new areas, to find new metrics for the approval of
products that incorporate new science.
I think there are other parts of the agency that have not
focused as much on the adoption of some of these new
opportunities. And I would hope in leading the agency, if I am
confirmed into this role, to bring more consistency to how
different parts of the FDA look at the authorities that
Congress has given to the agency to achieve the kinds of things
you talk about.
Senator Young. As a follow up, some of FDA's review
divisions perform incredibly well, as I understand it. The
Oncology Division being perhaps the most notable positive
example. They collaborate with patients, manufacturers, and
other stakeholders to proactively facilitate innovation.
However, I understand not all review divisions are as forward
leaning or as flexible as others.
As FDA Commissioner, specifically, how will you work to
raise the performance or consistency of all review divisions
regardless of the therapeutic areas they focus on?
Dr. Gottlieb. Thank you for the question, Senator.
One of the things that I tried to do last time I was at the
agency was to compel the agency to look at how it was
performing. By doing that, we sometimes were able to help
people who were working in the divisions reveal these things
for themselves, and that was a healthy exercise.
I would hope to do the same thing, if I am confirmed into
the role, again to allow review staff and leadership in the
Centers the opportunity to evaluate how different parts of
their Centers are working, and to find those places where we
might be able to adopt some best practices and broaden them.
Senator Young. Sounds as though it is a bottom-up approach,
which strikes me as good management.
Dr. Gottlieb. Everything is a bottom-up approach at the
FDA. The ideas really need to come from the career staff in the
Centers.
What leadership can do is help facilitate the opportunities
to recognize places where there could be underperforming parts
of the agency.
Senator Young. Doctor, you have spoken of biosimilars and
some changes that might be made to improve the approval process
and expedite the ability of bringing them to market.
What about in the area of generics? Are there certain
regulations or guidances you think are particularly burdensome,
or obsolete that should be withdrawn, or significantly revised?
In generics, you could touch on medical devices or pharma as
well, if you like.
Dr. Gottlieb. Thank you for the question, Senator.
One of the issues I have looked at recently is the issue of
so-called high-value generics or complex generics where the FDA
struggles to put certain drugs through the ANDA process because
it is difficult to demonstrate substantial equivalents using
just the traditional tools, which is bioequivalence and
bioavailability studies.
Congress did not envision with Hatch-Waxman that certain
drugs would have monopolies in perpetuity long after their
intellectual property has expired but for the inability of the
FDA to have a scientific process that can prove
interchangeability for those drugs.
This is an area where we can make a lot of progress. We
might need to come back to Congress, to talk to Congress about
what additional steps we need to take. There are things the FDA
could contemplate administratively.
This is an area I want to work on. There is literally
billions of dollars worth of drugs each year that are sold as
branded drugs at high prices, but should be subject to generic
competition.
Senator Young. Very encouraged that you intend to be
forward leaning in this area of delivering to this committee,
and perhaps others, some solutions that we might work with you
on, and I would like to play an active role in that effort.
Thank you.
Dr. Gottlieb. Thank you, Senator.
The Chairman. Thank you, Senator Young.
Senator Kaine.
Statement of Senator Kaine
Senator Kaine. Thank you, Mr. Chair.
Thank you, Dr. Gottlieb for your testimony. I want to talk
to you about opioids, which you addressed in your opening
comment.
Two million Americans today have a substance abuse disorder
with prescription pain relievers and in the most recent year,
more than 20,000 died of overdoses on prescription drugs,
prescription opioids.
Another 600,000 Americans have a substance abuse disorder
because of addiction to heroin. Four-fifths of new heroin users
began by using prescription opioids. An additional 12,000
Americans died last year from heroin overdoses.
In 2012, the last year for which we have very good
statistics, 259 million opioid prescriptions were written in
this country--prescriptions with multiple doses of prescription
opioids.
There are many at fault for this scourge. There was bogus
research that was published and perpetrated suggesting that
these drugs do not have addictive qualities, which they do.
There was unscrupulous advertising, a drug developed that
provided pain relief in emergency situations. There was a
realization that there was not a big enough patient base, and
so there was a decision to market it more broadly to people
suffering from chronic pain conditions.
There are dishonest providers who have been caught, pill
mills and there might be doctors, there might be allied health
professionals or pharmacies that were being caught.
There has been inadequate training about pain management,
often in medical schools and other places. Without any malice,
the absence of appropriate training about how to manage pain
has contributed to this.
The FDA has played a role in it too. The FDA approved
Zohydro, despite the fact that the advisory committee at the
FDA recommended that that not be the case.
The FDA approved use of Oxycontin by adolescents, despite
the fact that the advisory committee expressed grave
reservations about that.
You and I chatted a little bit about this yesterday. I
would like to hear you talk about in your leadership of the
FDA, should you be confirmed, what would be your strategy for
taking on this challenge that the FDA has been somewhat
complicit in, even if unwittingly, in the past?
Dr. Gottlieb. Thank you for the question, Senator.
I enjoyed our discussion yesterday.
This is a staggering human tragedy that, as I said at the
outset, is going to require dramatic action on the part of the
agency. It is going to an all-of-the-above approach. There are
a number of things we can do.
I feel I have a bipartisan mandate through my discussions
with this committee to try to push the agency to look harder at
what the right framework would be.
To give you some examples, FDA does take on as part of its
review process the mandate for looking at the potential for
abuse and diversion as a component of the approval process. I
think we need to ask hard questions whether or not the agency
has the adequate authorities, resources, and policy framework
for doing that, to make sure it is being done appropriately.
We need to look at opioid drugs in the context of medical
devices that could provide alternatives. The safety and benefit
of a systemic opioid drug might look a lot different when it is
juxtaposed against a device alternative that is delivering
localized therapy. That we might need to look at a different
framework.
Congress has given the FDA some authority to look at drugs
and devices in conjunction. We have done that with the Oncology
Division.
We have to look harder about how to create generic
standards for the drugs that have abuse deterrents, so that we
feel more confident clearing the market of the older drugs that
do not have abuse deterrent features. We need good guidelines
for how we are going to genericize those drugs so that we are
not just forcing people onto high-cost drugs when we take the
older, generic drugs off the market.
We need to do all these things and we need to look at
alternatives to opioids, and do we have the right framework in
place, and the guidance in place to accelerate the development
of non-opioid alternatives for the treatment of pain. We are
going to need to push harder on all these things.
It might be the case that when we look at these questions
in a hard way, I might have to come back to Congress, if I am
fortunate enough to be confirmed into this role, and have
another conversation with all of you about how to make these
things happen.
Senator Kaine. Dr. Gottlieb, if I could just follow up on
that.
As I think about the FDA, I am not an expert, but as I
think about it, I think of the work of this agency as largely
around applications and then the testing to determine--when
whether it is a device or a drug--is it available, and ready,
and proven efficacious, and safe for use by the public. An
application, we are going to look at this particular
application to see when it is ready for prime time.
What I would hope that the FDA would be able to do is look
more broadly at the issue of pain management. Do we have the
right set of tools and strategies, devices and pharmaceuticals
that can effectively manage pain in a way that is safe? To the
extent that we do not, I would hope the FDA could look in a
proactive way about, what do we not have? What do we still
need?
If you need to come back to us and tell Congress that the
FDA's mission is to fine tune narrowly just around the approval
process rather than being proactive to deal with an issue that
is as massive as the management of pain, I hope you will come
back to us and tell us that because I would think we would need
to. You would probably find some bipartisan support for
altering and expanding the mission of the FDA so that we could
take a proactive approach to this public health scourge.
Dr. Gottlieb. Senator, this will be my highest immediate
priority and I am committed to do that.
Senator Kaine. Thank you, Mr. Chair.
The Chairman. Thank you, Senator Kaine.
Senator Scott.
Statement of Senator Scott
Senator Scott. Thank you, Mr. Chairman.
Dr. Gottlieb, thanks for being here this morning.
You bring a healthy balance, or equilibrium, to this
opportunity. You are a doctor. You have also been a patient.
Not just a patient, but a cancer survivor. You appreciate the
necessity of new drugs, and sound quality, and safe drugs.
You have worked for the FDA on a couple of occasions, but
you have also worked with industry for a long time. You also
have the support of Dr. Hamburg and Dr. Califf, the
Commissioners of the FDA. So I like the equilibrium that we see
here.
One of the comments that you made during your opening
statement, or at least the comments that I read in your opening
statement, was that you see a false dichotomy that it boils
down to either speed or safety. I would appreciate you
expounding on that because there has been much conversation
around the pricing. Price, oftentimes, is a component of how
long it takes to get to market.
There is a successful venture from Clemson University, in
my home State of South Carolina, of tissue bioprinting. I would
imagine that, given the current investment in capital and time
to get a drug to market, we should encourage every tool
possible that helps us get there faster and safer.
I would love to hear your comments on tools like
bioprinting, as well as your comments about the dichotomy that
may be a false paradigm.
Dr. Gottlieb. Thank you for the question, Senator.
It is the case that drugs do get priced to some measure of
the cost of capital to create those drugs. The longer the
timeline--the more costs, and the more uncertainty of that
process--the higher the cost of capital to fund an endeavor to
try to find a new innovation.
Anything we can do to try to make that process more
predictable, to create bright lines, to use better tools to
evaluate safety and effectiveness that could bring down the
cost--while not doing anything to sacrifice our ability to
ferret out the safety of a product--are things we should be
looking at.
I come back to the Cures Act because I think that there is
a lot of opportunity for that to be a template for policymaking
in this regard, not just what Congress directed the agency to
do with respect to adaptive clinical trial designs. Also what
Congress directed the agency to do with respect to modeling and
simulation as a tool to helping better evaluate safety and
effectiveness.
All of these things can make the process more efficient and
perhaps less costly while still allowing us to fully evaluate
safety and effectiveness.
This is one place, if we are doing our jobs right, we can
have our cake and eat it too.
Senator Scott. That is awesome. Thank you.
Another question for you, I spend a lot of time every
Valentine's Day, I enjoy going to the Medical University of
South Carolina and visiting the pediatric unit, specifically
the oncology center because you have an opportunity to see
children who are going through incredible situations,
challenging times, but they are optimistic; they are positive.
The care that they receive at the Medical University of
South Carolina is outstanding care. But there are times when
doctors do not have all the tools necessary. As you are well
aware, cancer affects children and adults differently and often
the type of cancer that develops in a child is very different
than what might develop in an adult.
Given this, and the fact that pediatric cancer is so rare,
what can the FDA do to continue to enable innovation in this
space?
Dr. Gottlieb. Thank you for the question, Senator.
Congress has given the agency certain authorities to try to
encourage the development of clinical data in the pediatric
setting. As you noted, there are still obstacles to getting
drugs studied in these ways.
This is something I would be committed to trying to work
with you and others on to see if there are additional steps we
could be taking to try to create incentives for the development
of information around the use of products in the pediatric
population, and also finding ways to conduct more feasible
clinical trials in these settings. Sometimes it is hard to run
the same kinds of perspective, large, randomized trials in very
small populations where children are involved, and I think we
need to look at different clinical trial constructs as well.
I, again, come back to the issue of adaptive designs and
alternative clinical trial designs as being something that
could be particularly applicable in this area. I would be
committed to working on this if I am confirmed.
Senator Scott. Last question and probably, perhaps, a quick
answer because we have little time left.
A lot of the smaller biotech companies are working on
cutting edge research and development. They may have one drug
in the pipeline and none on the market. Working with small
staffs and, frankly, private capital.
What are some of the ways that the FDA can support these
smaller companies and help more drugs come to market at the
lowest cost?
Dr. Gottlieb. Thank you, Senator.
As it was noted at the outset, I have helped create some of
those smaller companies.
Senator Scott. I did note that.
Dr. Gottlieb. I have seen this experience from both sides.
The most important thing when you are trying to raise
capital to develop a new innovation is understanding exactly
what your costs are going to be. Obviously, there is an
inherent unpredictability to the scientific process, and you
can never fully anticipate that.
Understanding what the benchmarks are, and what the
clinical requirements are going to be, is very important. It
becomes important for the FDA to have very clear guidance,
especially in areas of unmet medical needs so that people who
are trying to find capital for these endeavors know what their
costs are going to be.
The Chairman. Thank you, Senator Scott.
Senator Warren.
Statement of Senator Warren
Senator Warren. Thank you, Mr. Chairman.
In the 1950s, a German company began selling a drug called
Thalidomide all over Europe, a sleep aid that was also marketed
for nausea during pregnancy. Pregnant women, who took
Thalidomide, starting having babies with severe birth defects
most notably deformed flipper-like arms and legs.
More than 10,000 children were born with severe
disabilities caused by Thalidomide, but fewer than 100 of those
were born in the United States and that is because an FDA
reviewer kept the drug off the market over concerns about the
shoddy data to support the drug's safety.
Following that episode, public outrage led Congress to
strengthen the FDA so that drug company profits would never be
put ahead of the safety of our children.
I was surprised, Dr. Gottlieb, to read in a 2012 article
that you asserted that the Thalidomide episode had a harmful
effect on the FDA. You said the incident,
``Fostered an idealization of FDA reviewers as
`championing' an issue of safety against the prevailing
orthodoxies especially when it meant taking on
corporate interests.''
You were critical.
Dr. Gottlieb, do you think the FDA puts too high a priority
on championing safety and protecting unborn babies and other
consumers?
Dr. Gottlieb. Absolutely not, Senator.
The thrust of that article was about a much different
point, but I absolutely believe the FDA needs to be answering
these questions in full. In fact, the modern FDA does a very
good job ferreting out risks of teratogenicity preclinically,
actually because of the modern tools that we use in the drug
development process.
Senator Warren. I am not quite sure that I am following
because you talk about,
``In so heavily prioritizing the protection of
consumers, the FDA has `subordinated and neglected' its
obligation to `guide new medical innovations to
market.' ''
In other words, it sounds like to me you are saying in this
piece that you think that the FDA places too much emphasis on
consumer protection.
Dr. Gottlieb. Senator, I remember the piece well and the
thrust of the piece was focused on my concern that the agency
was losing confidence in physicians and felt that it needed to
step into the regulation and the practice of medicine to try to
supplant its judgement for the judgement of doctors in certain
situations. Moreover, I was concerned that the agency did not
have the enforcement tools to follow up on what it was doing.
One thing I learned when I was at FDA was you have had to
be careful about imposing a requirement that you are not
capable of enforcing, and that was the thrust of the article as
I remember it.
Senator Warren. I have to say that is not how I read the
article, and I worry about your language, in that you
criticized FDA reviewers saying that they, ``Believe it is
appropriate to prioritize safety over speed.''
I know there are always judgments to be made, but
belittling reviewers who are concerned about safety makes me
very uneasy. I want to get innovative products to market as
fast as possible, and I support more resources for the FDA to
be able to do that. I support better science for the FDA. I
have written bills to do both of those.
Your view of the FDA's response to the Thalidomide problem
is deeply disturbing and it raises, for me, real questions
about your commitment to the FDA's basic safety mission.
As my colleagues have pointed out, you have spent your life
entrenched in the companies that would benefit from looser
regulations. I think it raises the very real question of
whether someone who seems to oppose the FDA's basic safety
mission should be running the agency. I just have real concerns
here.
I also have concerns about off-label marketing. I see that
I am running out of time and it is a long series of questions.
In deference to the Chair, I will do those as questions for
the record.
I just want to say the position of the head of the FDA is
one in which all of America and, indeed, all of the world
places its trust.
For me, that means it is profoundly important that you be
committed to safety and that you be committed to the role of
the FDA; not the drug companies, not others outside who stand
to profit, but to the FDA itself to watch out for that safety.
Dr. Gottlieb. I agree, Senator.
Senator Warren. Thank you, Mr. Chairman. Thank you.
The Chairman. Thank you, Senator Warren, for respecting the
time. There will be an opportunity for another round of
questions if you wish.
Senator Burr.
Statement of Senator Burr
Senator Burr. Thank you, Mr. Chairman.
Dr. Gottlieb, to your parents, congratulations; you did a
great job. I hope you are proud of him. All the criticism is
about his successes.
To your wife and to your children, thank you for the
sacrifice that you are making. It is a job I have always
wondered as people get nominated why they do it, and especially
somebody with as many irons in the fire as Dr. Gottlieb has.
Let me ask you, Dr. Gottlieb, do you think the FDA follows
the 1997 FDAMA statutes today?
Dr. Gottlieb. It follows important elements of the FDAMA
statutes in part because of the foresight of this committee in
recodifying some of those and reinvigorating them for the
Breakthrough Therapies.
Senator Burr. We went through a period of time where the
FDA did not even follow the statute of law. Right?
Dr. Gottlieb. I have been on the record of being critical
of that. Yes.
Senator Burr. As the author of it, that was discouraging to
me. Yet, I hear you having to be held to a standard of
following the letter of a statute. Yet, you are inheriting an
agency that had not done that.
Let me ask you. In 2017, should there still be double-blind
studies where the doctor does not even know whether the patient
is getting a placebo or not?
Dr. Gottlieb. Senator, thank you for the question.
I believe that there are opportunities to modernize how we
do clinical trials in ways that are not going to sacrifice on
the gold standard of safety and effectiveness, but perhaps
could think of clinical trial constructs that do not require
the tight randomization that current clinical trials do.
Congress believes this as well because it has directed the
agency to look at alternative clinical trial design as the
component of the review process.
To your point about FDAMA, I believe that getting
sufficient implementation of Cures and following Congress'
intent in that regard is going to be an essential part of what
I hope to do if I am confirmed into this role.
Senator Burr. In fact, Congress in 2012 passed Breakthrough
Therapy legislation on drugs. We followed it up in the Cures
Act with a Breakthrough Therapy Pathway for devices. That was
an acknowledgement by Congress that there was a risk aversion
within the culture of the FDA.
If confirmed, what are you going to do to change the
culture of the FDA; not the standard, the culture?
Dr. Gottlieb. Thank you for the question, Senator.
I want to find ways to look at what is working well within
the agency, if I am confirmed into this position, and try to
bring those best practices to the parts of FDA that might not
be adopting the tools, especially the tools that Congress has
instructed FDA to start to incorporate.
We have seen, for example, I think to your point, uneven
adoption of the principles embedded in the Breakthrough Therapy
Pathway, and I think there are ways to try to inspire more
consistency and more broad adoption of that ethos.
That would be something I hope to work on. That is
something we were able to do when I was at FDA the last time in
part by challenging the agency to examine its own processes to
see how it was performing against these kinds of benchmarks and
against the prerogatives of Congress.
I would hope to bring back those same tools, those same
techniques, to try to do that again with respect to these new
sets of authorities that Congress has given the FDA.
Senator Burr. When Andrew von Eschenbach was the
commissioner of the FDA, Dr. von Eschenbach recognized at the
time that the success of the FDA was in his ability to have the
talent that he needed in the future to be able to address
technology changes that they were going to be presented with
from the standpoint of reviewers.
If you are confirmed as the next FDA Commissioner, how will
you ensure that the FDA is ready to regulate cutting edge
products coming before the agency today and in the future? I
might add, that Dr. von Eschenbach set up a whole mechanism to
identify future employees, recruit future employees, train
future employees that was dismantled after Dr. von Eschenbach
left as commissioner.
What are your plans?
Dr. Gottlieb. Thank you for the question, Senator.
This is one of the most critical issues facing the agency
right now. I think it is critical, in part, because the
complexity of the science coming before the agency is going to
require the FDA to have more and more specialized talent.
It is also critical insofar as we have a workforce at the
FDA that is aging to the point where you are going to have, a
spate of retirements. When you look at the demographics of the
agency, you are going to lose a lot of very senior
professionals over a short period of time. Replacing that kind
of expertise and institutional knowledge is going to be a real
significant challenge.
This is something, if I am confirmed into this role, that
is going to be something I need to work very hard on and a
significant challenge to the job.
I do believe what Congress has done in Cures--in giving the
agency certain hiring authorities and certain abilities to go
out and target people with specialized knowledge--does provide
the basis for trying to address this challenge. Getting that
implemented as Congress intended is something I hope to do
working with my administration, if I am confirmed into this
role.
Senator Burr. I thank you for that commitment.
Thank you, Mr. Chairman.
The Chairman. Thank you, Senator Burr.
If I may editorialize, that is a commitment that almost all
of us share.
Senator Casey.
Statement of Senator Casey
Senator Casey. Thank you, Mr. Chairman.
Doctor, good to be with you. Thank you for coming to visit
our office to talk about some issues. I will raise some other
issues with you now that we did not have a chance to review in
my office.
The first is the Over-the-Counter Monograph System, which I
think many would argue is ineffective and in need of
improvement. I have been working with Senator Isakson on
legislation to reform the Over-the-Counter Monograph process,
to modernize it. In particular, to modernize drug regulations,
and ensure that both safety and efficacy information is
communicated to consumers in a timely fashion.
I would ask you first about that system, your view of it.
Second, ask you if you would commit to work with us in this
process to create a better, safer, and more efficient
regulatory structure for over the counter drugs.
Dr. Gottlieb. Thank you for the question, Senator.
Anytime a problem persists from when I was there 10 years
ago to today is an indication that it requires immediate
action.
I am familiar with what Congress is working on. I have the
sense from what I read outside of the agency--I do not have the
benefit of being inside the agency--that there is support in
the agency for Congress' ideas. I also believe, I think like
you, that this is a system that is in need of modernization.
This is something I would be very committed to work with
you on, if I had the opportunity to be confirmed into this
role.
Senator Casey. I wanted to ask you as well about a rather
recent development.
A January 2017 white paper from the FDA analyzed 22 cases
of drugs, vaccines and devices where promising Phase 2 results
were not supported by subsequent trials in Phase 3. Alarmingly,
seven of these products had safety problems that were not
detected until Phase 3 trials were completed.
Based upon that report, and the concerns that emanate from
that, I would ask you about the Phase 3 process in particular.
You have been critical of FDA's reliance on data from Phase 3
clinical trials for drug approvals.
Do you continue to hold that view?
Dr. Gottlieb. Thank you for the question, Senator.
I am not sure insofar as I am critical of Phase 3 trials. I
have articulated a point of view that with more modern clinical
trial designs, you could compress the Phase 2 and Phase 3
clinical trials into one, big adaptive design. That is a view
that the FDA has had and FDA officials in terms of trying to
design modern clinical trial constructs.
That a proper clinical program is an essential feature of
ensuring the safety and effectiveness of products, and I am
familiar with the study you talk about. Having proper post-
market enforcement tools is an essential part of the overall
paradigm of regulatory approval.
Senator Casey. Do you believe in allowing market forces to
be the judge of efficacy in Phase 3? Do you believe that?
Dr. Gottlieb. I believe in the gold standard for safety and
effectiveness, and I believe Congress has delineated a single
standard for demonstrating that, Senator. That is something
that needs to be demonstrated through a regulatory process
guided by science and the public health.
Senator Casey. Do you support, or I should say, do you
continue to support the requirement that all new drug
applications must include Phase 3 trial data for a drug to be
approved for marketing?
Dr. Gottlieb. Congress has directed the FDA to allow
certain drugs in certain situations to be approved on the basis
of Phase 2 data under the Breakthroughs Pathway under some of
the provisions in FDAMA. Congress has given FDA the tools to
make certain accommodations in areas of unmet medical need.
I support following the directive of Congress in making
sure your laws are faithfully implemented.
Senator Casey. I wanted to ask you as well something we
raised initially yesterday with regard to preparedness for all
kinds of threats to public health. We have had so many lately
that we are reminded all the time of the emerging--not just the
threats we have seen--but emerging diseases and bioterrorism
concerns.
Senator Burr and I have worked for years on medical
countermeasures, policy, and legislation. The development of
these new countermeasures to combat these threats is, of
course, a huge priority for everyone here. The FDA plays an
important role in that, but here we are now facing a hiring
freeze and potentially facing adverse budget impacts.
I would hope, and I will ask you the question, it is a
simple yes or no question. Would you advocate within the
Administration against the views of the Administration
apparently? Would you advocate in the Congress for
appropriations needed to meet FDA's mission as a public health
agency including FDA's obligation with respect to medical
countermeasure development?
Dr. Gottlieb. I am going to be committed, Senator, to
advocating for a strong FDA.
Senator Casey. I would hope that means that you would
advocate against proposals with regard to a hiring freeze or
the budget.
Would you or not?
Dr. Gottlieb. I am going to be committed to advocating for
proper resources at the FDA and a strong user fee program,
making sure that the mandates that you have given FDA are
properly resourced, so we could fulfill our mission.
Senator Casey. That was not the answer I was waiting for,
but I know my time is over.
The Chairman. Thank you, Senator Casey.
Senator Paul.
Statement of Senator Paul
Senator Paul. Dr. Gottlieb, congratulations on your
nomination.
It is my considered and biased opinion that we have plenty
of lawyers in Government, so I am always happy to see a fellow
medical doctor being appointed to something.
It is important just to reiterate maybe and tell us your
approach in general and motives. Would you ever let drug
profits--drug company profits or medical device company
profits--would you ever let that obscure your duty to safety
and efficacy?
Dr. Gottlieb. Absolutely not, Senator.
Senator Paul. That is important, because that goes to
character. To me it is sort of your honor is on the line when
you say that. We can argue back and forth, but you only can
tell us that, and I think that is your stated opinion, and I
accept that.
While your primary duty is safety and efficacy, there are
sort of market forces that ultimately determine prices of
drugs, availability of drugs, whether there are monopolies or
not. The FDA gets in the middle of that.
My hope is that we can get to a point where generics are,
obviously knowing that safety and efficacy is our primary duty,
but that we can maybe get them approved quicker so we can have
more competition. That we cannot have markets where we only
have one generic, that we are doing things to allow more
competition, understanding that competition will bring prices
down.
I guess what I would like to hear from you is just in
general your approach to how we balance that, patented drugs
versus generics. What are the things you think we can do better
in the generic market?
The one you and I have talked about, the EpiPen, where the
generic, applied for approval like in 2009, and still does not
have approval. The FDA will say, ``Oh, we are doing a lot
better and we are only doing them in a year and a half now. We
used to do them in 25 years,'' or whatever.
It still has to be even better, and I hope you will see
that as something that we can work on, and that you will give
us feedback on how you are going to make that better. I would
just appreciate your general comments.
Dr. Gottlieb. Thanks for the question, Senator.
This is something I would hope to work on early if I am
fortunate enough to be confirmed into this role.
That when it comes to generics, there are really two
problems. The first is just the ordinary generic approval
process where we have seen situations where certain drug
markets have fallen to one or two drugs, prices have been
raised, and the market is not self-correcting because trying to
get in an application and get it approved could take up to 4
years.
You have people who have been able to take advantage of
regulatory arbitrage by buying a product that might not face
generic competition, even though it is a generic drug, jack up
the price, and it takes a long time for other people to come
into the market even though the high price should be attracting
competition.
That is a solvable problem and something I would hope to
work on.
The other problem, which is more complex, are the generic
drugs that we have talked about here today where the agency
does not have good standards for demonstrating substantial
equivalence because the drug is a complex formulation.
Simply looking at the blood levels of the drug, which is a
standard by which we approve generics under Hatch-Waxman, is
not sufficient for demonstrating substantial equivalence.
This might be a drug that acts topically where measuring
the blood levels does not approximate the effect. It might be a
drug that acts inside the lungs, like a metered dose inhaler.
It might be a drug that acts inside the gut, a pill you swallow
that acts inside the gut.
In all these cases, you cannot simply look at blood levels
as a proxy for how the drug is going to have its therapeutic
effect.
In those cases, we need to develop better scientific
principles for doing that. There is opportunity to do that
within the framework of Hatch-Waxman. I would want to challenge
the agency to do that. But this might be an area where we need
to come back to Congress and have a broader discussion around
what that should look like.
This is a situation where you have drugs that Congress
intended for them to be subject to vigorous competition, but we
did not envision it when we passed Hatch-Waxman because the
drugs themselves have had 10 more complex.
Senator Paul. Thank you.
The Chairman. Thank you, Senator Paul.
Senator Murphy.
Senator Murphy. Thank you very much, Mr. Chairman.
Dr. Gottlieb, I think you are hearing from this side a
level of discomfort with your nomination, certainly connected
to your private sector history. It is also fairly unprecedented
to have a nominee before this committee for this position that
has such an extensive political history as well.
It is very well known you were one of the most outspoken
opponents of the Affordable Care Act. There is virtually no
piece of that legislation that you did not have a strong
opinion on that was very strongly worded. You have been a
political advisor to Republican candidates running for
President.
The worry about impartiality is certainly connected to the
private sector experience, but it is also to your very deep
political involvement as well.
I may want to ask a question about where today politics and
science are intersecting, in a way that make a lot of us on
this committee uncomfortable, and that is on the issue of
vaccines.
I am encouraged that you have commented and published on
multiple occasions in support of vaccinations based on
scientific data.
However, you are going to be working for a President who
has been a frequent critic of vaccinations. He has also
suggested that he might convene a political commission that
will look into the connection between vaccines and autism, for
instance.
Let me just ask you these two questions. No. 1, is there
any medical evidence that you know of to support the idea that
vaccines cause autism or that vaccines administered in the
current recommended vaccination schedule cause autism?
If this political commission is convened, if there is an
attack on vaccinations from this Administration, will you
commit to publicly opposing that effort?
Dr. Gottlieb. Thank you for the question, Senator.
As a physician, I share the concerns that parents have
about putting any product in an otherwise healthy child. I
understand that this issue elicits a lot of emotion.
That this has been one of the most exhaustively studied
questions in scientific history.
You had the 540,000 patient Danish study. You have had
exhaustive studies by the IOM and other esteemed bodies. I
think we need to come to the point where we can accept no for
an answer around this question, and come to a conclusion that
there is no causal link between vaccination and autism.
At some point, we have to accept no for the answer after we
have invested, and Congress has invested, enormous resources in
studying this question.
I have a history of not being shy. I think to your original
premise of your question, when you talked about some of my
writings, not being shy about speaking truth to power, and
making my views known sometimes in an unvarnished way on the
editorial pages of America's newspapers.
I will bring the same operating platform to this position.
I will give people my direct advice, my unvarnished opinion, my
science-based judgement, and the science-based judgement of the
people of the agency I hope to lead. I will make that known in
the proper venues.
Senator Murphy. The worry is that there will be industry-
supported reforms that will find a voice inside the agency
because of your connection to the industry.
Let me talk about a piece that you wrote, I believe, in the
``New England Journal of Medicine'' that discussed your
``National Affairs'' article in 2012, where you proposed a
major shift in the FDA drug approval process where you
suggested that there should be a politically accountable board
that ultimately makes decisions on drugs, which is a
revolutionary change from the way that we do things now.
It seems like it would be a big gift to the drug industry
being able to use their political donations in order to
ultimately put a group of ``friendlies'' on a process or a
commission that decides approval rather than having that
process sheltered from the political process.
Can you talk a little bit more about that proposal and
explain to this committee why that ultimately would not lead to
the politicization of drug approvals if you put that process
into the political realm outside of the protected area of the
FDA?
Dr. Gottlieb. I appreciate the question and a chance to
clarify what the article said.
In that article, if I remember correctly--and it has been a
while, I have written a lot of things on a lot of different
topics as you noted--I lamented the fact that the advisory
committee process itself, in my view, was already becoming
politicized.
My point was, and it was sort of a rhetorical point,
perhaps we should be more transparent about what is happening
and just allow Congress to appoint the members to the boards or
the GAO as we do with other advisory committees in Congress.
Because it might be a more transparent and deliberate way to
achieve what seems to have been the effort underway, which was
to influence the appointment of members to its board.
It was in some respects a rhetorical exercise, but in some
respects an acknowledgement of what I thought was a trend that
was underway with respect to the appointment of those members.
Senator Murphy. I hope that you will see that it is your
role to depoliticize that process to the extent that you think
it is politicized rather than to elevate the politicized
process.
Dr. Gottlieb. I agree with you, Senator.
Senator Murphy. Thank you. Thank you, Mr. Chairman.
The Chairman. Thank you, Senator Murphy.
Senator Hatch.
Statement of Senator Hatch
Senator Hatch. Thank you, Mr. Chairman.
I just want to compliment you for the experiences you have
had in this life. You have had a wealth of experience. Yes? A
lot of it in business, and a lot of it in industry, but on the
other hand, there is nothing that should discourage you from
being head of the FDA because you have had extensive industry
experience as well.
I am going to personally compliment you because I am
wondering, why are doing this? Why go through all this pain?
You do not have to give a long answer.
Dr. Gottlieb. Senator, I could think of no better time to
serve the public health in this capacity than right now.
Given the opportunities we have with science and technology
to find fundamental cures for many diseases because of the new
scientific platforms that we are seeing. The authorities that
Congress has recently passed, I think will give the agency a
fundamentally different way or the opportunity to enact a
fundamentally different way of looking at these new
technologies.
We have within our grasp the ability to cure pediatric
inherited diseases. We have within our grasp the ability to
cure many cancers with immunotherapy and other applications,
regenerative medicine, cell therapy, gene therapy. These are
enormous scientific opportunities, and I think the FDA stands
at a tremendous opportunity to try to make these come to the
market.
Senator Hatch. I want to commend you for your attitude
because there should be no question about your being selected
here. In all honesty, you bring a tremendous amount of
experience and ability to the agency. You know where business
is right and where business is wrong too.
I am absolutely convinced that you will stop some of the
things that are wrong in the agency and outside of the agency.
I have great confidence in you.
Take Hatch-Waxman, can we improve Hatch-Waxman?
Dr. Gottlieb. There are opportunities to make sure that
Hatch-Waxman is having its intended effect on the market,
Senator.
Sitting here today without the benefit being briefed by the
staff at FDA and better understanding these issues--because
issues always look different from the inside than they do
outside--I think that there are opportunities to make sure the
law is having its intended impact.
If that requires us to look at certain aspects of the
statute, I would certainly come back to Congress and have that
discussion.
Senator Hatch. I would like you to do that because as the
author of Hatch-Waxman, I want it to be perfect for the agency
and for our country, and not just what I thought at the time
was perfect. I appreciate your answer on that particular
subject.
We are very fortunate to have you willing to do that with
the vast experience that you have. Having worked with the FDA
for, really, years, and years, and years, this is a pleasant
experience as far as I am concerned.
Let me just ask you this. The independent Office of
Government Ethics, in consultation with the Department of
Health and Human Services' Ethics Division, approved your
public financial disclosure form and your ethics agreement.
Is that correct?
Dr. Gottlieb. They did, Senator.
Senator Hatch. They went into that thoroughly?
Dr. Gottlieb. They did, Senator.
Senator Hatch. OK. Based on information the OGE--the Office
of Government Ethics--concluded, Dr. Gottlieb is in compliance
with applicable laws and the regulations governing conflicts of
interest. According to your ethics agreement, upon
confirmation, you will resign from 13 positions.
Is that correct?
Dr. Gottlieb. I have not counted, Senator, but it sounds
about right.
Senator Hatch. Within 3 months of confirmation, you will
divest yourself from 30 financial interests?
Dr. Gottlieb. That could be right, Senator. That sounds
about right. Yes.
Senator Hatch. How stupid can you be?
The reason I am asking that rhetorical question is because
I do not think people appreciate what it means for somebody
like you, with the tremendous expertise that you have, to walk
out of the private sector into this Government job, with all
the sacrifices that are going to be required and the huge
commitments that are required.
I do not think they appreciate what you are doing here.
They should.
I want to personally thank you for your willingness to
serve this country in this position, with your vast background,
and with your reputation as an honest, decent, very scientific
person.
There should not even be any question about your ability to
do this job and to do it well. I personally want to
congratulate you for being willing to do it and to tell you how
much I appreciate it.
Dr. Gottlieb. Thank you, Senator.
The Chairman. Thank you, Senator Hatch.
Senator Bennet.
Statement of Senator Bennet
Senator Bennet. Thank you, Mr. Chairman.
Dr. Gottlieb, congratulations on your nomination and
welcome to the committee.
I wanted to go back to the opioid and heroin epidemic that
is happening in this country. I have, like my colleagues on
this panel, spent a lot of time having town hall meetings
throughout my State. I have noticed over time that families are
ripped apart and communities are having profound difficulties
coping with this problem, which, by the way, we do not have an
answer for right now in terms of addiction treatment especially
in rural areas of this country. I am going to park that
observation and ask you the question.
How did we get here in your view? Not just from the FDA
point of view, because the problem of very addictive opioid
prescription drugs being approved without a sense of how
addictive they were, if you look at the history, and then cheap
heroin coming in behind those drugs that have perpetuated the
addictions that we face.
I wonder if you could give us a perspective, not just as
the potential Commissioner of the FDA, but also as a physician
in thinking about the way these drugs have been prescribed and
used to manage pain.
What do we need to learn from this experience? So that we
not just do not make the same mistakes, but we can dig
ourselves out of this horrible epidemic across our country.
Dr. Gottlieb. Thank you for the question, Senator.
I have stated here a number of times, I think this is a
public health emergency on the order of Ebola and Zika. We need
to treat it that way. We need to treat this as a public health
crisis that is going to require dramatic action.
For a long period of time, we did not fully recognize the
scope of this evolving problem and our actions to try to
address it might have been too incremental in nature. I do not
say this to try to pass judgement on people who have preceded
me at the FDA. I was at the FDA for part of that time as well.
I do not think we fully recognized the scope of the
emerging problem, the true addictive nature of these products.
Clinical medicine and physicians, and I am a physician who has
prescribed these drugs, are partly responsible for that.
To your point, this is now a problem that is big enough
that whereas at one time it might have been within the scope of
FDA to address this problem in a more robust way--I do not want
to say solve the problem--but address it in a more fulsome way.
It has now grown so large that it has grown outside of FDA's
ability to address by itself.
This is a public health challenge that we are going to need
to address through the full gamut of our public health
resources. That does not mean that the FDA does not have an
important role to play, but I will say from the standpoint of
FDA, I think in order to address it now, the types of actions
we are going to need to take are going to be far more dramatic,
perhaps, than the types of actions we would have needed to take
if we had done more 10 years ago to get ahead of this.
Again to your point, we now have the problem that people
move to the lowest cost alternatives. It is no longer just
opioids. Having become addicted on opioid, people are moving to
heroin.
Senator Bennet. I am delighted to hear you say that and the
Chairman's very first question was about opioids today. You now
probably have a sense of the priority this is for this
committee.
I agree that this is beyond the purview of just the FDA. We
are going to have to find a way to get agencies to collaborate
to deal with these issues, and you calling it an Ebola-like
crisis, is very helpful to the cause.
I want to shift gears just for the remainder of my time. Am
I out of time, Mr. Chairman?
The Chairman. No.
Senator Bennet. For the remainder of my time just to talk a
little bit about the Breakthrough Therapy legislation Richard
Burr and I worked on.
That is, as I said the other day in the committee, we have
seen great breakthroughs as a result of that in oncology, not
so much in neurology.
I wonder if you have some views of why that has been?
Whether in the progress that you are seeing or predicting,
whether neurological diseases will be part of that as well?
Dr. Gottlieb. Thank you for the question, Senator.
We have seen some parts of the agency work exceedingly well
with respect to implementing the spirit and the practicality of
the Breakthrough Pathway.
We have seen uneven application of it, but I think that
that is resolving inside the FDA. You are starting to see more
uniform application of both the spirit and the letter of the
law in that regard.
I would hope if I am confirmed into this role to find ways
to try to install a broader embrace of this Pathway. It is an
exceedingly important Pathway. It has provided the opportunity
for patients who have unmet needs to get therapy that might not
have been available as efficiently without this in place. It
has had a real positive impact.
As a physician, I can say I think this has had a real
positive impact on the ability of patients with unmet medical
needs to get safe and effective therapeutics in a timely
fashion.
Senator Bennet. Thank you for that.
Thank you, Mr. Chairman.
The Chairman. Thank you, Senator Bennet.
Senator Cassidy.
Statement of Senator Cassidy
Senator Cassidy. Dr. Gottlieb, enjoyed our conversation.
Dr. Gottlieb. Thank you.
Senator Cassidy. One thing I remember is that when I almost
tempted you to criticize the staff, you said no. You thought
you must first look at the systems. That oftentimes you can
improve the performance because you just put in better systems.
That was obviously the insider's perspective, but also very
generous. Some who have suggested, that you are not going to be
supportive, and I just thought you were incredibly supportive.
That said, there is a GAO report that I am looking at from
May 2016 in which--just a comment on this briefly because this
will lead to the next question--in which the GAO was somewhat
critical of the FDA saying it lacks measurable goals to assess
its progress in advancing regulatory science. The science
supporting its effort to assess the products it regulates.
It goes on further to say there are lots of resources that
have been given to the FDA, but that indeed they have not
systematized how they account for the spending.
Any thoughts on that?
Dr. Gottlieb. Thank you for the question, Senator.
I am familiar with the report. I have reviewed it in the
past. That this is both a challenge and an opportunity trying
to make sure that we not only have the best workforce possible
in order to ensure the FDA gold standard. That they have the
best training, the best tools, and are being forward leaning in
trying to adopt the best science into the principles that they
use to govern the review process.
This is something--when I was at the FDA with Dr.
McClellan, we initiated a critical path initiative to try to
democratize an effort to try to improve the quality of the
tools that were being used. Not just by reviewers, the tools on
their desk, but also the tools that they were enabling sponsors
to use as part of the requirements by which they judged drugs.
This would be a high priority of mine if I was fortunate
enough to be confirmed into this role to continue to push on
these opportunities.
Ultimately, this is how we are going to make the review
process more efficient without sacrificing on the gold
standard. I think it is ultimately how we are going to make the
development process less costly, while still meeting FDA's high
bar.
Senator Cassidy. You said it is a false dichotomy, science
versus safety or speed versus safety. This would be one way you
could achieve both.
Dr. Gottlieb. This is the way you can achieve both.
Absolutely.
Senator Cassidy. Let me ask you something else. I am going
to hit a tangent and then come back.
One of my favorite historical figures is William
Wilberforce, many reasons why, but among them is that he
realized there were a lot of problems with drunkenness related
to gin. The way he mitigated that was he had pushed the sale of
beer. The idea is you would get so bloated, you have to
urinate, et cetera, etc., that you could only drink so much
beer as opposed to drinking a lot of gin. It was really
successful.
About that because tobacco is a scourge in terms of health,
but I sometimes think maybe the way to address tobacco is by
tobacco mitigation. If you look at the Center for Tobacco
Products, it seems as if there is less than openness about
looking for products that would be an alternative to a
cigarette, although still a nicotine delivery system, that
would have less of the untoward effects associated with
cigarettes.
It does seem like the Center for Tobacco Products does not
really use the traditional notice and rulemaking process. You
know more about this than I, but it seems to not follow a
process that would allow some of these mitigating products to
be released.
Any thoughts on that?
Dr. Gottlieb. Thank you for the question, Senator.
If I am confirmed into this position, I am committed to the
goals of the TCA and making the TCA work.
Congress had great foresight in envisioning the opportunity
for reduced harm products to transition smokers off of
combustible tobacco onto reduced harm products that posed less
of a risk as an animating principle as part of a comprehensive
regulatory scheme for nicotine containing products. I am
committed to trying to make that component of the TCA work
along with all of the other components of the TCA.
These are ultimately questions that can be adjudicated in a
proper regulatory context. An e-cigarette, for example, or a
vaping product might be a good smoking cessation tool and an e-
cigarette flavored like chocolate chip cookie dough might not
be.
That in the proper regulatory context, we have the tools,
thanks to Congress, to adjudicate these questions.
I do think that, to your point, I think there is an
opportunity to make this framework much more comprehensive and
much more viable.
Senator Cassidy. I have 16 other questions, but I have 15
seconds, so I will yield back and thank you.
The Chairman. Thank you, Senator Cassidy. We will have a
second round if you want one.
Senator Franken.
Statement of Senator Franken
Senator Franken. Thank you. I am struck by the gin for beer
because I would just like to throw it open to anybody here.
That does not actually work.
Senator Cassidy. It worked in England. That was actually
seen as a milestone. I do not want to use your time, but it is
used as a milestone in the cessation of alcoholism.
Senator Franken. I am skeptical.
Sorry, it just threw me.
Let us talk a little bit about, first, conflicts of
interest. You are taking yourself out of this for a year and
you have recused yourself from decisions that would impact 20
or so healthcare companies that you have been involved in for a
period of a year.
President Trump pledged that all his appointees would
recuse themselves for 2 years. Why do you not just do that?
Dr. Gottlieb. Senator, I appreciate the question.
That was the discussion that I had with the Office of
Government Ethics and what they required me to do. I am going
to have a separate discussion, if I am fortunate enough to be
confirmed into this role, with the ethics officials at FDA and
HHS. They might very well impose additional requirements, and I
will follow whatever advice and counsel I get from those
officials.
Senator Franken. OK. I would prefer it, too.
You mentioned on opioids, you said we need an all-of-the-
above strategy, but I did not hear you mention some of the
obvious all-of-the-above. One is treatment. Treatment now in
ACA is an essential health benefit, but there was an attempt to
take that away in the House bill.
I just want to know how you feel about taking that
essential health benefit away?
Dr. Gottlieb. I thank you for the question, Senator.
I did mention MAT, Medically Assisted Therapy, and I think
within the context of my responsibilities at FDA, if I were to
be confirmed--
Senator Franken. What did you say?
Dr. Gottlieb. I said within the context of my
responsibilities--if I was going to be confirmed into this
role--would be, in the context of this problem, to look at what
the principles are for evaluating Medically Assisted Therapy,
MAT.
Senator Franken. OK.
Dr. Gottlieb. Medically Assisted Therapy as a component of
treatment.
Senator Franken. I thought you said medications.
Dr. Gottlieb. Yes it is medication, but--
Senator Franken. OK. That is what you mean.
Dr. Gottlieb. Right. Medication for people who are
addicted, yes.
Senator Franken. That is different than in-house treatment.
I am sorry. That is different than rehab.
Dr. Gottlieb. You are talking about a service. I am talking
about the medication that is used in the context of a service.
My responsibility as FDA Commissioner would be to approve--
Senator Franken. I am talking about the essential health
benefits in the ACA. That was my question.
Dr. Gottlieb. Right. I appreciate the question.
Senator Franken. You can answer the question without it
having to be directly related to your duties at FDA, please.
Dr. Gottlieb. Senator, I appreciate the question. If I am
confirmed into this job, I am going to have a lot of people
asking me to opine on issues that fall outside of the scope of
FDA. I think I would do--
Senator Franken. OK. Never mind. I am just saying that
residential treatment for opioid addiction is a really good
thing. That is what I wanted to say, and if you are talking
about all-of-the-above, if you are talking about all of the
above that is really important to people.
If you are talking about a crisis that is the scope of
Ebola, and you do not keep residential treatment as part of the
essential benefits package of insurance, I do not think you are
taking it as seriously as you would an Ebola-level crisis.
I want to talk about something more maybe in your purview
which is Naloxone, which you have not mentioned either. If you
are taking an all-of-the-above approach, I think you would talk
about Naloxone.
I spent a minute on beer, Mr. Chairman, so I want 30
seconds.
A two pack dose of the auto-injector for Naloxone rose from
$690 in 2014 to $4,500 today. This goes to the prices of drugs,
which everyone in Minnesota talks about when I go around the
State.
You did not mention Naloxone which saves a tremendous
number of lives. It is part of the all-of-the-above, believe
me.
What are you going to do to keep down the cost of drugs?
Dr. Gottlieb. Thank you, Senator, for the question.
I talked about the importance of MAT generally, which all
of these things fall under. I share your concerns.
With respect to Naloxone, that falls into the scope of
complex drugs that I have talked about today. It is a drug-
device combination that is hard to ANDA those processes. It is
hard to put alternatives through the generic drug approval
process.
I do think that there are ways that the FDA can
administratively, perhaps, and it might require a statute,
allow a pathway to make it easier to put generic alternatives
to some of these drugs through the generic drug approval
process so we can create more competition.
Some of these issues relate to instructions for us under
the current guidelines. The instructions for use for drug-
device combinations needs to be precise or the same for the
branded drug and the copy drug in order to go through the ANDA
process. There might be opportunities to relook at that
framework.
This is something I have spoken about a number of times
here today that I am committed to working on with you.
Senator Franken. OK. I hope you address that.
Thank you, Mr. Chairman, for indulging.
The Chairman. Thank you, Senator Franken.
Senator Roberts.
Statement of Senator Roberts
Senator Roberts. Doctor, congratulations on your nomination
and thank you for being here today.
I want to associate myself with the very pertinent remarks
by Senator Hatch regarding your decision for public service.
Your background, as a physician and prior service at the FDA
make you an excellent choice to lead the agency.
We talked in my office, or to be more accurate, I talked
and you listened. I am confident in your commitment to public
health and in putting patients first in decisionmaking. I am
going to vote for you and I am not going to ask you a yes or no
question.
I am chairman of the Agriculture Committee, as well as a
member of this committee. I am particularly interested in your
thoughts on the FDA's role in ensuring food safety.
Under the previous administration, it is my opinion, that
we saw increased activity and regulation actions on nutrition
policies such as issuing voluntary guidance. Yet, at the same
time, requesting additional money to comply with statutory
requirements under the Food Safety Modernization Act, the
acronym for that, by the way, is FSMA, which I find somewhat
unique.
I have concerns that the agency has not prioritized the
FDA's mission to protect our Nation's food supply and instead
focused on nutrition policies.
How will you focus on core FDA duties such as implementing
the law that Congress passed rather than agenda-driven
nutrition policy guidance as we saw with sodium and added
sugars?
Dr. Gottlieb. Thanks for the question, Senator.
This is an agency that has a statute that has been amended
over 100 times since 1938 and has a vast scope of
responsibilities. We do need to focus on core mission and make
sure we are achieving what Congress intended in terms of
protecting and promoting the public health.
FSMA was a significant advance in terms of giving the
agency authorities it needed and the resources it needed to
ensure the food supply is safe. My mandate is going to be to
make sure FSMA is implemented in a proper way and that we are
striking the right balance with respect to that implementation.
Senator Roberts. Last year in the Agriculture Committee, we
worked the whole year and we are finally successful in passing
the Biotech Labeling bill of which the Department will be
working to implement in the next couple of years.
At the same time, FDA has been working to update the
Nutrition Facts Panel, which is set to go into effect next
summer.
There is a great deal of concern that the FDA has not
provided the guidance necessary for compliance. This includes
guidance for dietary fibers, which we discussed previously when
you were in my office.
In addition, hundreds of millions of dollars could be lost
due to lack of coordination between the Department of
Agriculture and the Food and Drug Administration on these label
change timelines.
Will you please work to ensure proper guidance is available
and consider postponing the current deadline for the Nutrition
Facts Panel to help reduce regulatory burdens?
Dr. Gottlieb. Senator, this is something I would certainly
be delighted to work with you on if I was confirmed into this
role.
I am philosophically in favor of trying to make sure that
we do these things efficiently. Not only because it imposes
undue costs on the manufacturers if they are constantly
updating their labels, but we also have to keep in mind it does
create confusion for consumers if the labels are constantly
changing.
You want to try to consolidate the label changes when you
are making changes as a matter of public health so the
information is conveyed accurately and efficiently to the
consumers.
This is something that I do care about, and I would look
forward to working on it if I am confirmed.
Senator Roberts. I appreciate that.
Mr. Chairman, I have further questions. I will submit them
for the record.
Thank you very much.
The Chairman. Thank you, Senator Roberts.
Senator Baldwin.
Statement of Senator Baldwin
Senator Baldwin. Thank you, Mr. Chairman.
Welcome to the committee.
Dr. Gottlieb. Thank you.
Senator Baldwin. I had to step out to attend another
committee meeting and missed some of the questioning, but
hopefully I will not be covering all the same ground.
I want to start by saying that I share some of my
colleagues' concerns about both the optics and the potential
for conflict of extensive financial ties and relationships with
companies that you would directly regulate at the FDA.
In addition to your financial holdings in multiple medical
companies and board memberships, you have also long served as
an advisor to and invested in New Enterprise Associates, a
venture capital firm.
During our meeting, and I appreciate you taking the time to
visit with me, you noted that the FDA should focus on a more
holistic approach to chronic pain to address, in particular,
the opioid epidemic that we talked at some length about.
One of the things you talked about was looking to medical
devices as therapy options, but it turns out that New
Enterprise Associates has invested in one such company, Nevro
Corporation, which markets devices for chronic pain.
I would like to ask you beyond what is already in your
ethics agreement, what assurances can you give us that those
significant business and financial relationships will in no way
influence or bias your work directly regulating these companies
at FDA, should you be confirmed?
Dr. Gottlieb. I appreciate the question and I appreciate
the opportunity to answer it, Senator.
Just by way of background, and I am proud of my
relationship with NEA. It has been one of the premiere venture
capital firms in the country starting a lot of innovative
ventures.
Over the time period that I was there, they invested about
$14 billion in 500 different companies. Per my ethics
agreement, I disclosed that I had investments in five of those,
all healthcare services companies.
I was not actively involved on the medical device
portfolio. A lot of my time was spent on the healthcare
services portfolio, and that is not to say that it was
exclusively spent on the healthcare services portfolio, but a
lot of my time was spent working on healthcare services
companies. That is just sort of a general understanding of my
role at FDA [sic].
I recognize the importance of bringing impartiality to this
position. I recognize that someone could look at my background
and have these questions. I appreciate the opportunity to
answer them.
I am going to be cognizant of trying to make sure I
preserve the integrity of my role and do nothing in exercising
my obligations, if I am confirmed into this role that would
besmirch the agency and reduce people's confidence in the
agency's mission.
This is exceedingly important to me. I get it. I know why
people care. The FDA's decisions are literally matters of life
and death and I do not want to do anything in my conduct to
reduce people's confidence in the agency's mission.
Senator Baldwin. Thank you.
You have also stated that the FDA should approve drugs
faster and that too much regulation is the main barrier to
lower prices, and these are certainly positions supported by
the drug industry.
To me it is about much more than just speeding more drugs
to market. The FDA should address this through robust oversight
of industry tactics especially when they are meant to game the
system; simply to boost their bottom line. For example, by
seeking to add or prolong market exclusivity periods or
creating misleading direct-to-consumer advertisements.
Will you commit to working with Congress to advance
policies, which may be unpopular with drug companies, but yet
give the FDA more authority to crackdown on abuses that lead to
higher prices?
Dr. Gottlieb. Thank you for the question, Senator.
I agree with your concerns here. I think the FDA is
constantly getting drawn into commercial disputes, which puts
the agency in a very difficult position because it is a public
health agency. It is not the FTC.
It is hard for the FDA to simultaneously design policies
intended to protect and promote the public health, and design
policies intended to try to prevent companies from trying to
use the regulatory process for commercial advantage.
This is a problem that I am uniquely suited to try to look
at and solve because of my background. Because I understand how
companies have tried to game the process in the past. It is not
illegal to try to use the regulatory process to gain commercial
advantage.
We should try to design policies that prevent those abuses
because we do not want to be playing whack-a-mole with
companies and going after them one by one.
What I want is a framework in place that prevents those
kinds of things from happening so people cannot use the
regulatory process as a commercial arbitrage to gain unfair
advantages. I could think of other places where I think that
goes on.
These are things I want to look at and frankly, I think
these are things I am uniquely positioned to look at,
hopefully, because of my background. That is where my work does
inform some of these issues.
Senator Baldwin. Thank you.
The Chairman. Thank you, Senator Baldwin.
Senator Murkowski.
Statement of Senator Murkowski
Senator Murkowski. Thank you, Mr. Chairman.
Dr. Gottlieb, welcome. I look forward to our visit this
week.
I want, as a kind of follow-on to what Senator Baldwin has
raised in your response here, but I want to speak about it in
the context of a specific example, and that relates to
genetically engineered salmon.
As you know, there were millions of Americans that wrote to
the FDA opposing the approval of genetically engineered salmon.
A whole number of grocery stores have announced that they will
not sell it.
Despite all of this immense opposition, in November 2015,
the FDA approved AquaBounty Technologies' application for this
new animal drug process. It was approved through that process
despite the application being for Genetically Engineered
AquAdvantage salmon for human consumption. It was the first
G.E. animal approved for human consumption through this
process.
The FDA did not have any mandatory labeling requirement.
Instead it said, well, it can be labeled voluntarily. We refer
to this G.E. salmon as ``frankenfish,'' nobody is going to
voluntarily label it as such.
The valid question and concern is whether or not this fish
should even be called a salmon, and there is great fear that
lies with these mutated fish that have not gone through a
proper EIS.
The question to you this morning is whether or not you
believe that genetically engineered fish and other animals for
human consumption should be approved through the animal drug
process?
I am looking for your commitment to work with me to ensure
that we have a better process, and hopefully a better solution,
to what many of us, particularly in Alaska, are quite concerned
about as a big problem.
Dr. Gottlieb. Senator, I am familiar with the issue.
I was not at the FDA when these issues were adjudicated as
part of the approval process. The FDA has recently issued
guidance on this matter that does not address all the issues
that you have raised here.
You have my commitment, if I am confirmed into this role,
to work with you on it after I have had the opportunity to
study the issue more closely once I am at the agency, if I am
confirmed.
Senator Murkowski. Good. Well another, keeping in that vein
of seafood or fish, let me continue with an issue that is very,
very concerning to us. This stems from FDA published advice on
January 19 of this year, and it was advice for pregnant and
nursing women on seafood consumption. This advice that came out
purports to update the prior version of the same advice that
was published back in 2004.
When you look back at that scientific authority that speaks
to regular seafood consumption by pregnant and nursing women,
and the benefits that it provides, it is important information.
The FDA's peer-reviewed study on maternal seafood
consumption for fetal brain development, this is the Net
Effects report, concludes that regular seafood consumption by
pregnant and nursing mothers adds 2.63 IQ points to a child's
growing brain. You have the additions for the cardiovascular
and general health benefits of eating fish by the mothers
themselves.
What we then see is we have this guidance that comes out,
or this advice that comes out, on January 19 that is based not
on the Net Effects report. Instead on EPA's mercury reference
dose, which is a toxicological standard that ignores the
benefits of seafood and measures risks in isolation.
What we have seen from this is that this advice is
confusing. It does not give clear guidance to pregnant or
nursing women. It widens the gap between the seafood advice and
the dietary guidelines for Americans. It gives the EPA a more
prominent role in developing a better nutrition policy.
We have a great deal of concern about this particularly
with regards to the latest information relating to halibut and
sable fish.
I would like your commitment to me that, if confirmed, you
will revisit this seafood advice and consider modifications to
what was issued in January of this year.
Dr. Gottlieb. Senator, you have my commitment to making
sure that the FDA guidance is fully science-based and providing
proper information to consumers.
I am familiar with the issue, but again, have not had the
benefit of studying it and being fully briefed on it. You have
my commitment that I will take a look at it and work with you
on it.
Senator Murkowski. It is something that we will have an
opportunity to discuss further, but I will tell you this is one
of those issues that I am really running out of patience on.
We need to make sure that full consideration is given to
the FDA science that was the basis for updating the advice in
the first place, instead of this EPA mercury data that has
nothing to do with ocean-caught fish. I want to work with you
on that as well.
Mr. Chairman, my time has expired.
I thank you and I look forward to further conversation.
The Chairman. Thank you, Senator Murkowski.
Senator Whitehouse.
Statement of Senator Whitehouse
Senator Whitehouse. Thank you, Mr. Chairman.
Thank you, Dr. Gottlieb, for being here. A-plus to your
girls for the super-good behavior they have had. It is probably
an exceedingly boring morning for them.
Senator Whitehouse. Back to the question of New Enterprise,
I have been told that there are 82 companies that New
Enterprise has investments in that might have products, or do
have products that are before the FDA.
Because of the nature of your relationship with New
Enterprise, you were paid directly by New Enterprise. You were
cut out from having a direct, financial relationship with those
companies, which would have triggered more recusal
requirements.
I am not an investment person, but I do believe that very
often people have pretty close working relationships on the
investment banking side with the companies that they are
invested in and are often de facto managers.
When those--assume I am right about it being 82 companies--
when those 82 companies crop up with applications at the FDA,
assuming that you are confirmed, who will take a look at that
to make sure that the relationship is not one that you should
recuse from?
Dr. Gottlieb. Thank you for the question, Senator.
Senator Whitehouse. By the way, this is not your fault.
This is a question that has to do with the way the Government
demands things of you. Your compliance is fine with what you
have been asked to do.
A little bit like Miss DeVos, not having to answer
questions about her dark money operation or Administrator
Pruitt not having to answer questions about his dark money
operation. We have these places where our disclosure rules have
not caught up with, what you might call, the new technology.
I do not want to blame you for this, but I do think to what
you said very clearly before about not impugning the integrity
of the FDA. How would you manage that, or how would the FDA
manage that for these exact companies?
Dr. Gottlieb. Right. Thank you, Senator.
I do not fully know what the phrase ``dark money'' refers
to, but we have been very transparent with the NEA portfolio
including putting the entire portfolio of my Form 278. The
companies are all listed there with brief descriptions.
Just to table set with respect to my relationship to New
Enterprise Associates, and since it has come up a couple of
times today.
I was effectively a consultant to NEA and paid as a
consultant to NEA, and advised them on a selected number of
companies in their portfolio. Those companies are not entirely,
but largely, reflected by the list of companies I recused from
because a lot of my time was spent on the healthcare services
portfolio at NEA, not on the life sciences and medical deice
portfolio.
That is not to say that I did not occasionally get drawn
into meetings related to other portfolio companies, but it is a
circumscribed set of companies that I might have touched in
that relationship.
This is another issue where I am looking forward, if I have
the opportunity to be confirmed into this position, to talking
to the ethics officials at the FDA and HHA about how to manage
any potential conflicts that could arise with my relationship
to New Enterprise Associates.
You and I spoke in your office about the importance of
personal integrity in this position, and I took that to heart.
That means a lot to me. I am going to make sure that I follow
the rules and do what I am told to with respect to this
relationship.
Senator Whitehouse. We have talked a little bit in this
hearing about pricing of pharmaceuticals and talked a lot about
opioids. I associate myself with the questions and concerns of
FDA's role in the opioid epidemic.
I do not think that we have talked much about antibiotics.
I worry a bit that you described a ``brave new world'' in which
new discoveries were going to lead to a whole new era of health
and prosperity. At the same time, we are looking at potentially
an end to a great era of health and prosperity brought on by
antibiotics that were effective against most of the dangerous
bugs that come after us.
The more those bugs adapt to our antibiotics, if we do not
keep up, I understand that the Centers for Disease Control
estimates that 2 million people develop antibiotic resistant
infections in the United States every year, and it kills 23,000
of them. Antibiotic resistance is described by the World Health
Organization as, ``One of the biggest threats to global
health.''
In all of these areas, we have concerns that really impact
the public health that do not relate narrowly to the product
being safe when used as prescribed. Right? The opioids were
going to be likely safe when used as prescribed. It was being
overprescribed that was the problem. The antibiotics,
obviously, you have that problem of resistance developing. And
with respect to price, if you cannot afford it, you have a real
problem. There is an access issue that affects the public
health.
To what extent do you think the FDA should be venturing
outside of that narrow question and into the broader questions
of public health around those three points? Sorry, a long
question.
Dr. Gottlieb. As a hospital-based physician who has lost
patients to resistant infections in the ICU, I recognize the
gravity of the problem that you describe.
That the agency has taken steps in recent years largely
owing to congressional statute to try to address the problem in
different ways, both on the development side with the
provisions embedded in Cures, as well as what they have done on
the animal feed side with respect to the use of anti-infectives
in animal feed.
What you describe is the broader stewardship question and
whether or not it is within the scope of FDA's current
authority to address that more societal question as a component
of the review process.
My short answer is: I do not know. My instinct is to think
that this is something Congress needs to contemplate, and I
would certainly be happy to work with you on this question.
Senator Whitehouse. Fair enough.
The Chairman. Thank you, Senator Whitehouse.
Senator Hassan.
Statement of Senator Hassan
Senator Hassan. Thank you, Mr. Chair.
I will add my compliments to your girls. Senators have a
hard time sitting still through these hearings, so you are
doing really, really well. Thank you.
Dr. Gottlieb, I appreciated the visit we had in my office
as well. I just do want to go on the record echoing the
concerns that many of my colleagues have raised about the issue
of conflict of interest.
To Senator Whitehouse's point, you are following the rules.
That is good. You have extensive ties to the drug and biotech
industry. If confirmed, you are going to be tasked with
regulating the same companies.
You take the issue of personal integrity very seriously. I
appreciate that. I will just say that part of this issue is
about self-awareness, and perspective, and who you identify
with. If you are confirmed, I hope you will take that to heart
as well.
I wanted to spend a little bit more time on the issue of
opioids. We spoke about it in my office and New Hampshire is
one of the States which has just been devastated. We had about
500 deaths in New Hampshire in the last year; 70 percent of
those deaths involved the drug Fentanyl, which is becoming even
more of an issue around the country.
At a hearing that the FDA was at before this committee a
few weeks ago, I asked about what are known as abuse-deterrent
formulations, and you referred to them a little while ago in
one of your exchanges.
The opioids in the abuse deterrent formulations are just as
addictive as they are in other products without the so-called
abuse deterrent properties. Experts have done surveys that show
that 46 percent of primary care providers think that these
abuse deterrent products are less addictive than other opioids.
That statistic is very high, but it does not surprise me
because I can see how the term ``abuse deterrent'' could be
confusing or misleading in this regard. What we are learning is
that the opioid at issue may be packaged in a way that makes it
hard to use in a traditional way for somebody suffering with
addiction, but they just find a different way to use it.
Do you think the FDA has a role to play here in ensuring
that there is a common understanding among providers that
products called ``abuse deterrents'' are just as addictive as
other opioids and are not abuse proof? Because they can still
be abused easily by, for example, just swallowing them.
Dr. Gottlieb. Right. I echo your sentiments in many
regards, Senator. I appreciate the question.
The primary impediment to these abuse deterrent
formulations becoming abused is that they are expensive right
now. Once they go generic and become cheap, they are just as
easily abused if you just take more of them.
Senator Hassan. Right.
Dr. Gottlieb. What we are talking about are drugs that are
harder to divert or tamper with in ways that might make them
more attractive abusable items.
The underlying issue that you surface as to whether or not
we have the right nomenclature to describe these drugs--and
whether or not the nomenclature that we are using in how we
describe these drugs is conveying the right message, not only
to patients but in particular to providers--is a reasonable
question.
Within the scope of what I want to do to try to push the
agency around looking at a different framework for how we
address this problem, I would include that question.
You and I talked about it. I saw the press release that
went out from one of the groups, and when I read it, it struck
me as a reasonable question to be asking. I want to ask that
question.
Senator Hassan. Thank you.
One of the other tools that we know we can use to combat
this public health crisis, and one of the ones that you have
that the FDA has at its disposal, is the Risk Evaluation and
Mitigation Strategies, otherwise known as REMS, which the
agency uses to try to stem the risks associated with certain
medications. As you know, that includes things like prescriber
training.
You have been public in the past about your opposition to
REMS saying that they put burdens on providers. Because we are
in the middle of a public health crisis, as you have
acknowledged with this addiction epidemic, and because I know
that many providers in my State welcome guidance from the FDA
or anybody else who has the expertise about how to curb this
public health crisis.
Do you think physicians should be able to look to the FDA
for guidance about how to prescribe drugs like opioids?
Dr. Gottlieb. I do, Senator.
The article you reference--which was a ``Health Affairs''
article, I think in 2007, 10 years ago--spoke to a different
issue, which was the use of REMS to start to address drugs
where there was a known risk. The FDA wanted to attenuate the
off-label prescribing of the drugs across the range of purposes
for which a physician might want to use a drug under his
discretion; very different than this issue.
I actually affirmed in that article--I largely affirmed the
historical context in which the REMS had been used, which
included the use of trying to prevent diversion and abuse of
opioid drugs. That had been the historical use of REMS and, in
fact, the early genesis of REMS under a different name--it was
not called REMS back then--was created under Subpart H for the
purpose, largely, of trying to address the opioid problem back
at FDA.
I do fully support the use of that tool in this context. In
fact, I support the use of the tool across a lot of contexts.
In many respects, the agency has gone on to use the program in
ways that are far more judicious than what I was initially
worried about when I wrote that article.
Senator Hassan. Thank you.
I see I am over time.
I will submit for the record a question about some women's
health issues because I am concerned about your involvement in
what I thought, and many have thought, was a politically
motivated delay of the emergency contraceptive Plan B when you
were at the FDA. I will submit that in writing.
Thank you.
The Chairman. Thank you, Senator Hassan.
Senator Murray.
Senator Murray. I will actually follow up on that because I
am concerned about political pressure at the agency, and I
personally am outraged by this Administration's continued
efforts to undermine and rollback women's rights and access to
healthcare.
This is not the first time I have had to fight a President
and his administration to protect healthcare for women.
In fact, I fought back the last time you were actually at
the FDA, and the Bush administration ignored overwhelming
scientific consensus and recommendations of two advisory
committees and FDA career staff by deciding not to sell Plan B
emergency birth control over the counter.
I want to know if you will stand up for women and fight
against any political attempts by President Trump and Vice
President Pence to politicize decisions about women's health.
Let me ask you specifically. Do you think the Bush
administration made the wrong decision to deny women access to
Plan B over the counter?
Dr. Gottlieb. Senator, I am fully supportive of efforts to
promote women's health.
Insofar as there are concerns, I am not in my capacity, if
I am confirmed into this role, going to re-litigate settled
science and settled approval decisions in the absence of some
compelling safety information that drives the agency to do that
and a very clear recommendation from the career staff.
Senator Murray. Do you think they were wrong in denying
Plan B?
Dr. Gottlieb. Senator, I was not involved in adjudicating
that decision. That decision was made at the time based on the
science that was available and the judgement of the people who
were involved in doing that.
I do not want to supercede the judgement of people
retrospectively who made a decision at a certain point in time
who were looking at certain data and certain issues, when I was
not party to that evaluation.
Senator Murray. You will be under immense pressure from
this President and Secretary Price who continue to put politics
ahead of women.
Can you commit to me today that you will not allow them to
use the FDA to further a political agenda against women's
health?
Dr. Gottlieb. In this regard, Senator, I appreciate the
question.
I am not going to relitigate settled approval decisions.
Senator Murray. I am not talking about the past. I am
talking about the future.
We have watched the agenda of this Administration, and I am
very fearful that they will put political pressure on you to
make decisions not based on science and not based on evidence-
based treatment, and ask you to make decisions. What will you
do?
Dr. Gottlieb. As a physician and someone who cares about
women's health issues, I am going to be guided by the science,
and the public health, and the judgement of the career staff in
how the FDA makes decisions under my leadership.
Senator Murray. OK. This is very important because I asked
Secretary Price during his confirmation hearing before this
committee for his commitment that all 18 FDA-approved methods
of contraception would continue to be covered, and he refused
to make that commitment.
Your commitment to make sure that any decision is science-
based, evidence-based is really important on this issue to me.
Dr. Gottlieb. Thank you.
Senator Murray. I also want to ask you about tobacco, which
we touched briefly on before. When we met, you said you would
prioritize continued implementation of the Tobacco Control Act.
I have to tell you, I am concerned about your commitment to
do everything you can to protect health under this really
important landmark law in looking at your record in terms of
this. Because while the Tobacco Control Act prohibited fruit
and candy-flavored cigarettes, flavored e-cigarettes, and
cigars have flooded the market in recent years. E-cigarettes
are now available in thousands of flavors. You mentioned one a
moment ago about Cookies and Cream. There is Pop Rocks.
This should come as no surprise to you. I know that you had
a financial stake and then served on the board of directors of
the interestingly named KURE, a company that sells liquid for
e-cigarettes in huge numbers of flavors including Gummy Bear,
and bases its business model on attracting Millennials. You
also had a financial stake in a separate company that markets
those products, KURE's products.
Children's use of these flavored tobacco products is a
serious public health concern. Research supported, actually, by
the FDA itself says 81 percent of children who have ever used
tobacco products started with a flavored product. You cite
flavors as the major reason for their current use of non-
cigarette tobacco products.
If you are confirmed, do you commit to wholeheartedly
addressing the clear, public health risk posed by flavored e-
cigarettes and cigars by resisting industry pressure to weaken
the so-called ``deeming rule'' which brought e-cigarettes and
cigars under FDA's authority?
Dr. Gottlieb. Senator, I appreciate the question.
I am committed to proper implementation of the TCA. As a
physician and a cancer survivor, I am not going to countenance
a rise in adolescent smoking rates in this country under my
watch. I am going to make sure that we appropriately implement
the law and fulfill Congress' intent in this regard.
These are empirical questions, in my view, that you are
raising about when a reduced harm product can be a useful tool
for transitioning people off of combustible cigarettes onto a
reduced harm product, and when they might be a gateway towards
adolescent smoking to the point, you are making.
In a properly constructed, properly overseen regulatory
process, we should have the capacity under the authority the
Congress gave to the agency to make these determinations. I am
committed to trying to make that work.
Senator Murray. What about banning flavored tobacco
products and marketing practices that actually target our
children?
Dr. Gottlieb. Again, certainly marketing practices that
target children, as I understand, are already illegal under the
scope of the law.
The issues of the flavoring and all these other issues that
deal with the specific qualities of the vaping products are
those kinds of empiric questions that I think career staff
should be adjudicating in the Center.
I want to provide the proper support to make these
judgments. Make sure that we are finding a way to fulfill
Congress' intent here, that there should be reduced harm
products available to consumers to transition them off of
combustible cigarettes.
I do not want to supercede my judgement sitting here today
without having the expertise of the career staff and certainly
the ability to talk to them for what might or might not be a
proper product. Other than to say that I recognize that a
vaping product or an e-cigarette manufactured and flavored in a
certain way might be inappropriate in one context, appropriate
in another.
Senator Murray. Gummy Bears? Cookies and Cream?
Dr. Gottlieb. That I used the example of Cookies and Cream
in my own comment, so I recognize that there is a line here
somewhere, and I do not know where that line gets drawn. I
think that that line needs to get drawn by people who are
expert in evaluating that science, and I want to support them.
Senator Murray. My time has run out. I have gone over. I
appreciate that, Mr. Chairman.
I do have a number of other questions and I appreciate your
willingness to be here to answer them that I will submit for
the record. I know a number of my committee members on this
side do as well.
Your thorough and straightforward responses will be much
appreciated.
Dr. Gottlieb. Absolutely.
The Chairman. Thank you, Senator Murray.
Thanks, Dr. Gottlieb, for your testimony this morning and
let me join several of the Senators who commented on your
children.
As a father and a grandfather, I do not think I have seen
5- and 7-year-olds sit so still for so long in a long period of
time better than some I know pretty well.
We compliment you and your family on that.
Listening to the comments today, most of the criticism of
you, to the extent that there has been any, has been because of
your work with industries, and companies, and agencies that
have something to do with the job you are about to do.
We put public servants like you in an odd position. We ask
someone to come in and run the Agriculture Department, and then
we criticize them if they were a farmer.
We expect someone to come in and take jobs in the Pentagon
and deal with very complex issues of war and peace, and assume
most of the time it would help if they knew something about
military strategy before they come.
The Secretary of Education, Miss DeVos, was heavily
criticized because she had not worked for a public school or
one of the public education agencies that she would be in
charge of regulating as the Secretary.
I remember when Secretary Tillerson was nominated, some
even on my side of the aisle raised great questions because he
knew Vladimir Putin so well.
My own view is I like to have a Secretary of State who
knows Vladimir Putin very, very well. I do not want someone in
his pocket, but I want someone who does not spend 4 years
getting to know him, trying to evaluate him, understanding his
strengths and weaknesses, and what he might do right, and what
he might do wrong.
I agree with those Senators who said we are fortunate that
you do have this broad experience. You are asked to be
responsible for product areas as diverse as prescription drugs
for humans, prescription drugs for animals, medical devices,
biologics, dietary supplements, cosmetics, over the counter
medication, food, and tobacco products.
You are asked to implement a law we call 21st Century Cures
that we worked on for more than 2 years and it is filled with
nuance, and opportunity, conflicts, and differences of opinion.
I like the idea of having someone in your position who is
experienced, who recognizes those nuances, who sees the
conflicts, who knows what a company may be able to do to create
a new drug and how a company may be trying to game the system.
Who might understand more rapidly than someone who does not
have your background, how to look at a market that does not
have competition and speed up competition so that it is, as you
mentioned, less than 4 years and brings the price down so more
families can afford the drug.
I welcome your experiences and your background. I am glad
that you are willing to serve.
I would note that we had a very good experience with Dr.
Califf, who had a distinguished career at Duke, who worked with
20 drug and device companies before he came. I think that made
him a much better Commissioner of the Food and Drug
Administration. He did what you have done. He signed an
agreement with the Office of Government Ethics and he recused
himself from any activity that he had that presented a conflict
of interest.
On March 28, we received a letter from the Office of
Government Ethics, which carefully reviewed your financial
transactions, found that with several recusals--which you have
committed to do--you are in ``Compliance with the applicable
laws and regulations governing conflicts of interest.''
You have said here that while you are in your position, you
will continue to consult, as all agency heads must do, with the
appropriate ethics personnel. If there are other recusals or
decisions that you need to make, you will make those decisions
at that time.
Am I correct about that?
Dr. Gottlieb. Yes, Sir.
The Chairman. All these questions are appropriate about
your past background, but in my view, I am delighted you have
that background. I welcome your coming.
This is a committee that, in the Senate, it does not take a
genius to notice that we are in a little bit of a disagreeable
patch right now in the U.S. Senate on some issues and that this
is a committee with a broad diversity of opinions.
We usually find an opportunity to work together on some big
issues that benefit the American people.
You heard one mentioned several times today and that was to
see whether you and other parts of the Government could be
forward leaning on opioids and particularly the idea of non-
addictive pain medicines, which Dr. Collins has talked about.
You have heard us emphasize the importance of the new
hiring authority that you have and our hope that you will work
with the Administration with our support to quickly move to
fill those spots in the agency, so that you will be able to
consider and approve rapidly those applications that deserve to
be approved.
Senator Murray and I are committed to working together on
the User Fees, which provide a lot of the funding, and it is
important that we do that in a timely way.
Senator Bennet asked you about the Breakthrough Pathways
that he and Senator Burr authored. The new legislation has
Breakthrough Device Pathways. It allows regenerative medicine
to be a part of the existing, accelerated pathway.
We hope that you will take advantage of all of those ideas
and those authorities.
Thank you for your testimony today.
I ask consent to put into the record seven letters of
support representing 29 groups including physician,
pharmaceutical, and patient advocacy groups.
[The information referred to may be found in Additional
Material.]
The Chairman. If Senators wish to ask additional questions
of our nominee, questions for the record are due by the close
of business Friday, April 7.
For all other matters, the hearing record will remain open
for 10 days. Members may submit additional information for the
record within that time.
Thank you for being here today.
The committee will stand adjourned.
[Additional Material follows.]
ADDITIONAL MATERIAL
Letters of Support
Alliance of Specialty Medicine,
March 31, 2017.
Hon. Lamar Alexander, Chair,
Senate HELP Committee,
U.S. Senate,
Washington, DC 20510.
Hon. Patty Murray, Ranking Member,
Senate HELP Committee,
U.S. Senate,
Washington, DC 20510.
Dear Chairman Alexander and Ranking Member Murray: The undersigned
members of the Alliance of Specialty Medicine write to express our
support for the recent appointment of Scott Gottlieb, M.D. to
Commissioner of the Food and Drug Administration (FDA).
As a practicing physician and hospitalist for many years, Dr.
Gottlieb has a deep understanding of the effect healthcare policy can
have on patients as well as physicians. We believe he will bring a
wealth of knowledge to the position and be an important advocate for
the health care community.
He has also served in government in various capacities, including
as senior adviser for medical technology, director of medical policy
development, and deputy commissioner for medical and scientific affairs
at the FDA. Given that wealth of policy experience, we believe he will
provide a steady hand at the FDA to ensure that our patients receive
products that are both safe and effective. In addition, his broad-based
policy experience will hopefully help to guide a more collaborative
effort between the FDA and other entities, especially the Centers for
Medicare and Medicaid Services (CMS).
Like Dr. Gottlieb, our organization is dedicated to fostering
patient access to the highest quality care. We look forward to working
with him to improve health care policy for specialty physicians and
their many patients.
Sincerely,
American Academy of Facial Plastic & Reconstructive Surgery; American
Association of Neurological Surgeons; American Society of Cataract
and Refractive Surgery; American Society of Echocardiography;
American Society of Plastic Surgeons; Coalition of State
Rheumatology Organizations; Congress of Neurological Surgeons.
Association for Accessible Medicines (AAM),
Washington, DC 20001,
April 4, 2017.
Hon. Lamar Alexander, Chair,
U.S. Senate,
455 Dirksen Senate Office Building,
Washington, DC 20510.
Hon. Patty Murray, Ranking Member,
U.S. Senate,
154 Russell Senate Office Building,
Washington, DC 20510.
Dear Chairman Alexander and Ranking Member Murray: On behalf of the
Association for Accessible Medicines (AAM), formerly the Generic
Pharmaceutical Association (GPhA), I am writing in support of the
nomination of Dr. Scott Gottlieb, M.D. to serve as the next
Commissioner of Food and Drugs. AAM represents the manufacturers and
distributors of finished generic pharmaceuticals and biosimilars,
manufacturers and distributors of bulk pharmaceutical chemicals, and
suppliers of other goods and services to the generic industry. I urge
the committee to conduct its business promptly and approve the
President's nominee without delay.
Dr. Gottlieb is one of the most formidable thought leaders in the
medical field and makes an excellent choice to lead the agency
responsible for ensuring the health of the millions of patients FDA
serves. I believe his firm grasp on the science and his understanding
of the pharmaceutical market, and specifically the generic and emerging
biosimilar markets, is unmatched. Dr. Gottlieb has advocated for
effectively addressing the backlog of generic drug applications pending
at FDA and ensuring that safe, effective and quality products are
approved at their earliest possible date. This not only has the benefit
of increasing competition to lower costs of drugs and biologics for
millions of Americans, the Federal Government and other payors, but
also improves public health by expanding access to quality treatments.
We stand ready to work with Congress, the President, Dr. Gottlieb
and his entire team to ensure that generic medicines continue to keep
lifesaving treatments within reach of all Americans. We strongly urge
the Senate to move forward with his confirmation in order to begin
addressing the important task of lowering prescription drug costs for
Americans.
Sincerely,
Chester ``Chip'' Davis, Jr.,
President and C.E.O.
Every Life Foundation for Rare Diseases,
Novato, CA, 94949,
April 3, 2017.
Hon. Lamar Alexander, Chair,
455 Dirksen Senate Office Building,
Washington, DC 20510.
Hon. Patty Murray, Ranking Member,
154 Russell Senate Office Building,
Washington, DC 20510.
Dear Chairman Alexander and Ranking Member Murray: On behalf of the
EveryLife Foundation for Rare Diseases, we write today to strongly
endorse Dr. Scott Gottlieb for the next Commissioner of the Food and
Drug Administration (FDA) and urge his speedy confirmation. The FDA is
critical for advancing treatments for patients affected by rare
diseases and we believe Dr. Gottlieb has the necessary skills,
experience, and knowledge to lead the Agency.
In particular, Dr. Gottlieb's prior experience at FDA means that he
has the institutional knowledge that will allow him to begin working
immediately on enhancing the agency. He has spoken out about the
workforce issues that continue to impact the FDA, as well as the need
for flexibility in review of rare disease therapies and the importance
of improving specialization and expertise among FDA reviewers.
Improving specialization will help ensure potential rare disease
therapies receive a thorough review by the agency staff and will
improve health outcomes for rare disease patients.
Dr. Gottlieb has highlighted the importance of biomarkers in the
use of development of novel treatments for rare diseases, which we view
as a vital tool for helping to improve the efficiency and speed of drug
development, while maintaining FDA's core and critical standards for
safety and efficacy. If we are to successfully close the innovation gap
that currently exists for the 95 percent of rare diseases without an
FDA-approved treatment, new models and approaches are needed to help
the 30 million Americans affected by a rare disease.
Dr. Gottlieb has a strong track record as a supporter of biomedical
research and innovation for rare diseases and understands how to
improve and enhance drug review at the FDA. We believe he is well-
suited to be the next Commissioner.
The EveryLife Foundation for Rare Diseases is a 501(c)(3) nonprofit
dedicated to accelerating biotech innovation for rare disease
treatments through science-driven public policy. We can do more with
the science we already have and bring life-saving treatments to
millions of people suffering from rare diseases.
Sincerely,
Max G. Bronstein,
Chief Advocacy & Science Policy Officer.
Julia Jenkin,
Executive Director.
HealthCare Leadership Council,
April 3, 2017.
Hon. Lamar Alexander, Chairman,
U.S. Senate,
Health, Education, Labor, and Pensions Committee,
Washington, DC 20510.
Hon. Patty Murray, Ranking Member,
U.S. Senate,
Health, Education, Labor, and Pensions Committee,
Washington, DC 20510.
Dear Chairman Alexander and Ranking Member Murray: The Healthcare
Leadership Council (HLC), comprised of leaders from all sectors of
American healthcare, enthusiastically endorses the nomination of Scott
Gottlieb, M.D., to serve as Commissioner of the Food and Drug
Administration (FDA) and encourages your committee and the full Senate
to support his confirmation.
Dr. Gottlieb's qualifications to lead the FDA are extensive and
indisputable. In his previous roles at the FDA, the Centers for
Medicare and Medicaid Services (CMS), and the Department of Health and
Human Services, he has been centrally involved in some of the most
important healthcare policy advances in recent years, including the
implementation of the Medicare Prescription Drug, Improvement and
Modernization Act, the generic drug user fee program, and expanding CMS
coverage parameters to provide beneficiaries greater access to new
medical innovations.
Additionally, he has acted as a health policy thought leader,
helping to shape the Nation's discussion on key issues through his work
at the American Enterprise Institute, the American Medical
Association's ``Pulse'' journal, and op-ed pieces in many major
publications. In his writings, Dr. Gottlieb has consistently
demonstrated his vision for accelerated medical innovation in this
country and greater patient access to the drugs and devices that extend
and improve lives.
We need strong, active leadership at the FDA. In the ongoing effort
to address concerns over healthcare pricing, enabling more robust
competition in the biopharmaceutical marketplace is one of the most
effective actions government can take. This can occur by expediting the
processes through which generic drugs are approved for patient use. We
believe this should be one of Dr. Gottlieb's highest priorities, should
he be confirmed to lead the agency. As well, it is essential that the
FDA take decisive administrative and regulatory actions in implementing
the 21st Century Cures Act, enacted by Congress last year, and remove
barriers that slow the movement of new, beneficial medical technologies
to patients and healthcare providers.
In coming years, the FDA will be instrumental in supporting
profound advances in healthcare and victories against cancer, heart
disease, diabetes and other severe illnesses. We believe Scott Gottlieb
is the right person to lead the agency at this critical time and,
again, we urge his confirmation.
Sincerely,
Mary R. Grealy,
President.
Sarcoma Foundation of America (SFA),
Damascus, MD 20872,
March 31, 2017.
Hon. Lamar Alexander, Chairman,
Committee on Health, Education, Labor, and Pensions,
U.S. Senate,
466 Dirksen Senate Office Building,
Washington, DC 20510.
Hon. Patty Murray, Ranking Member,
Committee on Health, Education, Labor, and Pensions,
U.S. Senate,
154 Russell Senate Office Building,
Washington, DC 20510.
Dear Senators Alexander and Murray: The Sarcoma Foundation of
America (SFA), the leading national patient advocacy organization
representing the needs of sarcoma patients and their families, would
like to express our enthusiastic support for the nomination of Scott
Gottlieb, M.D., as Commissioner of the Food and Drug Administration
(FDA).
The SFA, a 501(c)(3) nonprofit charitable organization, advocates
for increased research to find new and better therapies with which to
treat patients with sarcoma. Sarcoma is a rare cancer of the connective
tissue (bone, muscle, nerve, blood vessel, tendon, fat) with about
16,000 new cases and 6,000 deaths each year in the United States. At
any one time, more than 50,000 patients and their families are
struggling with Sarcoma. It is rather prevalent in children, accounting
for about 20 percent of all childhood cancers.
The sarcoma community understands the importance of having strong
leadership at the FDA. As a doctor and a cancer survivor, Dr. Gottlieb
has proven through his distinguished career that he has the experience
and knowledge to ensure that the agency successfully complete its
important work, particularly in its efforts in drug review.
Dr. Gottlieb has an exemplary track record of supporting and
spearheading efforts to ensure that safe and effective treatments are
moved through the review process in an expedient manner. During his
tenure as the Deputy Commissioner for Medical and Scientific Affairs at
the FDA, Dr. Gottlieb championed the need for the FDA to be efficient
and patient-focused in its job of evaluating new therapies. This prior
experience at the FDA and his efforts to increase efficiency through
the Critical Path Initiative demonstrate his ability to improve the FDA
through thoughtful, science-driven reforms. For these efforts, the SFA
honored Dr. Gottlieb in 2007 with our Visionary in Medicine Award.
As you complete your work to confirm the net Commissioner of the
FDA, we ask that you take into consideration the needs of the sarcoma
community and confirm Dr. Scott Gottlieb. The Sarcoma Foundation of
America looks forward to working with Dr. Gottlieb upon his
confirmation on the important issues that directly impact the lives of
sarcoma patients.
Sincerely,
Bert E. Thomas IV, Ph.D., MBA,
Chief Executive Officer.
Society of Hospital Medicine (SHM),
Philadelphia, PA 19130,
March 17, 2017.
Hon. Lamar Alexander, Chairman,
Committee on Health, Education, Labor, and Pensions,
428 Senate Dirksen Office Building,
Washington, DC 20510.
Hon. Patty Murray, Ranking Member,
Committee on Health, Education, Labor, and Pensions,
428 Senate Dirksen Office Building,
Washington, DC 20510.
Dear Chairman Alexander and Ranking Member Murray: On behalf of
more than 50,000 practicing hospitalist physicians nationwide, the
Society of Hospital Medicine (SHM) strongly supports the nomination of
Scott Gottlieb, M.D., as Commissioner of the Food and Drug
Administration (FDA).
Practitioners of hospital medicine include physicians
(``hospitalists'') and non-physician providers who engage in clinical
care, teaching, research, or leadership in the field of general
hospital medicine. In addition to their core expertise managing the
clinical problems of acutely ill, hospitalized patients, hospital
medicine practitioners work to enhance the performance of hospitals and
healthcare systems.
As a hospitalist and active member of SHM, Dr. Gottlieb has served
on SHM's Public Policy Committee since 2011. In this period, Dr.
Gottlieb has been an invaluable asset to SHM in understanding and
addressing the most pressing healthcare issues of the day. During his
time with SHM, Scott has been very involved with our efforts around
empowering hospitalists and the overall healthcare system to push
forward in delivering quality, patient-centered care at lower cost. His
expertise has been helpful to both hospitalists and the patients we
care for.
Throughout his service to SHM, Dr. Gottlieb has shown his deep
understanding of the many public health issues, delivery system
reforms, and quality of care issues that are important to hospitalists
and our patients. And he has been an invaluable resource and partner in
developing solutions to these issues. His public service to the
government in various healthcare-related roles, including his tenure at
the Centers for Medicare and Medicaid Services (CMS) as Senior Policy
Adviser to the Administrator, Senior Adviser for Medical Technology,
Director of Medical Policy Development, and Deputy Commissioner for
Medical and Scientific Affairs at the FDA will provide him with the
deep and diverse experience that the role of FDA Commissioner demands.
His insight into both the CMS and the FDA make him a rare and
invaluable asset linking approval of much-needed treatments with access
to such care.
As a practicing hospitalist, and in his work with SHM, Dr. Gottlieb
has shown an unwavering respect for physician autonomy and is a
champion for the preservation of the patient-physician relationship. He
is committed to putting the patient first in the delivery of high
quality care.
Dr. Gottlieb has a track record of leadership and dedication to
building a patient-centered, quality-focused and efficient health care
system. As FDA Commissioner, Dr. Gottlieb will bring a demonstrated
commitment to public service coupled with the quality-driven, results-
oriented mindset of a hospitalist physician.
We urge you to vote in favor of his nomination.
Sincerely,
Brian Harte, M.D., SFHM,
President, Society of Hospital Medicine.
April 4, 2017.
Hon. Mitch McConnell, Majority Leader,
U.S. Senate,
SR-317 Russell Senate Office Building,
Washington, DC 20510.
Hon. Charles Schumer, Democratic Leader,
U.S. Senate,
SH-522 Hart Senate Office Building,
Washington, DC 20510-6300.
Hon. Lamar Alexander, Chairman,
Senate HELP Committee,
SD-428 Dirksen Senate Office Building,
Washington, DC 20510-6300.
Hon. Patty Murray, Ranking Member,
Senate HELP Committee,
SD-428 Dirksen Senate Office Building,
Washington, DC 20510-6300.
Dear Majority Leader McConnell, Democratic Leader Schumer, Chairman
Alexander, and Ranking Member Murray: The undersigned organizations,
representing millions of patients, advocates, caregivers, and health
care providers would like to reaffirm our support for President Trump's
nomination of Dr. Scott Gottlieb as Commissioner of the Food and Drug
Administration (FDA). We ask that Senators in the Republican and
Democratic caucuses, and the Senate HELP committee, vote to confirm Dr.
Gottlieb.
The United States is at a pivotal moment in terms of public health.
The FDA and patients need the leadership and experience that Dr.
Gottlieb will bring as soon as possible.
Dr. Scott Gottlieb is well-qualified and has received broad-based
support. Equally important, as a physician who has treated patients, he
knows the value of having the best available treatments based on the
best science. As a survivor himself, he knows what it's like to fight
cancer and understands the challenges that patients face every day.
Currently a resident fellow at the American Enterprise Institute
focusing on the FDA and CMS, Dr. Gottlieb is also a member of the
Federal Health IT Policy Committee. His previous experience includes
public service from 2005 to 2007, as FDA deputy commissioner under
President George W. Bush.
Dr. Gottlieb has not only the experience that will be critical to
expand upon the agency's important work, but also firsthand expertise
as a physician who has treated patients, understanding the breadth of
work that needs to be achieved on their behalf.
Dr. Gottlieb's strong scientific base and in-depth knowledge of key
regulatory processes will be key to his success in this position. Due
to his knowledge and experience, Dr. Gottlieb is the right person to
ensure the FDA keeps pace with science and innovation without
sacrificing the safety and efficacy gold standard established by the
agency.
Additionally, we know that Dr. Gottlieb can maximize value for
patients through the FDA. Congress must ensure that FDA continues its
important mission to provide patients with safe and effective
treatments. We ask the Senate to do what is right for patients and
immediately confirm Dr. Scott Gottlieb as FDA commissioner.
Sincerely,
Alliance for Aging Research; American Association for Cancer Research;
Association of American Cancer Institutes (AACI); CancerCare; CEO
Roundtable on Cancer; Coalition of Cancer Cooperative Groups;
FasterCures, a Center for the Milken Institute; Friedreich's Ataxia
Research Alliance (FARA); Friends of Cancer Research; Global
Healthy Living Foundation; Grandparents in Action; Lung Cancer
Alliance; LUNGevity; Lupus and Allied Diseases Association, Inc.;
Men's Health Network; National Coalition for Cancer Research
(NCCR); National Health Council; National Infusion Center
Association (NICA); National Kidney Foundation; National Patient
Advocate Foundation (NPAF); The Nicholas Conor Institute; Prevent
Cancer Foundation; Swifty Foundation.
______
Response to Questions of Senator Alexander, Senator Murray, Senator
Enzi, Senator Sanders, Senator Burr, Senator Casey, Senator Isakson,
Senator Franken, Senator Paul, Senator Bennet, Senator Collins, Senator
Whitehouse, Senator Cassidy, Senator Baldwin, Senator Hatch, Senator
Murkowski, Senator Murphy, Senator Roberts, Senator Warren, Senator
Kaine, and Senator Hassan
senator alexander
Question 1. I have heard that the FDA import clearance process does
not allow critical medical shipments to be cleared in time to be
delivered as required. Based on the FDA's data, 75 percent of the
imports are subject to some type of review at the border, which can
take hours, days, or even weeks to resolve. This problem will only get
worse as new data requirements went into effect February 9.
As more FDA-regulated products are imported, people who expedite
shipment of those products need to have confidence that the products
are legitimate and that they will get to patients on time.
Under your leadership, what will the FDA do to improve?
Answer 1. As a physician, I am sensitive to the needs of patients
who are seeking critical medical shipments. I am familiar with this
clearance process and the new data requirements that go into effect in
February. But I cannot speak to the specific reasons as to why the
timelines for the clearance process are unpredictable. If confirmed by
the U.S. Senate, I will commit to working with you and your staff to
identify and address the underlying issues related to this process to
ensure that patients are not waiting for critical medical shipments any
longer than the necessary time it takes the agency staff to assure the
authenticity of the medical product and the integrity of supply chain
through which it is delivered.
Question 2. I have heard that the Center for Tobacco Products needs
significant improvement and reconsideration with regard to the agency's
regulatory path for newly deemed tobacco products, particularly premium
cigars. Preventing long-term health effects of addiction to tobacco,
particularly among youth, is an important aspect of FDA's public health
mission. It is important that the tobacco center recognize the
significant differences in newly deemed tobacco products, such as
premium cigars, from traditional products covered under the Tobacco
Control Act, and that the level of risk may be different depending on
the product category. If confirmed, how could the FDA reconsider the
regulation of premium cigars?
Answer 2. Through the Tobacco Control Act (TCA), Congress gave FDA
regulatory responsibility over tobacco products. If confirmed, I will
be committed to implementing the TCA, as intended by Congress,
including section 911 related to modified risk products, which I
recognize can provide helpful tools for current tobacco-users to
transition off combustible tobacco. I will also commit to better
understanding the decision FDA made with respect to premium cigars, and
any changes that were made in regard to premium cigars between the
proposed and the final rule. As I was not at FDA during the agency's
initial TCA implementation activities, I am not fully acquainted with
internal processes or specific decisions to-date. If confirmed, I will
work with the staff to quickly get up-to-speed on this issue, and I
will review current FDA policies, including the deeming rule, to ensure
FDA treats products appropriately, implements provisions in a timely
fashion, and in a manner that is consistent with congressional intent
under the TCA. I believe responsibly implementing the TCA is an
integral part of FDA's core mission to protect and promote public
health.
senator murray
Question 1. The Food and Drug Administration (FDA) assures that
drugs meet the gold standard of being both safe and effective based on
a scientific, non-political review of raw data before they are marketed
to consumers. Do you support upholding this gold standard?
Answer 1. Maintaining the Gold Standard of safety and efficacy for
medical products is fundamental to FDA's mission to protect and promote
public health. If confirmed, I will uphold the Gold Standard by
ensuring FDA makes regulatory decisions based on sound science, good
regulatory practices, and the support of a strong staff. FDA should
thoroughly consider regulatory approaches that could improve the
efficiency of drug and device discovery, development, and regulation.
But FDA should only adopt those sound scientific approaches that
reliably improve on its gold standard for being both safe and
effective.
Question 2. The field of regenerative medicine has immense promise
for patients--from cell and gene therapies to stem cells. I am proud
that some of this innovative research is taking place in my home State
of Washington within both cutting edge companies and our leading
research institutions. As part of 21st Century Cures, Congress
established the regenerative medicine advanced therapy designation for
cell-based therapies that have the potential to address unmet medical
needs. There have been repeated reports of patients being harmed by
experimental regenerative medicine treatments that are being sold both
here in America and abroad--for example, last month the New York Times
reported that a clinic blinded three patients by injecting their eyes
with stem cells. As Commissioner, what steps will you take to ensure
FDA takes action against unscrupulous providers and ensures continuing
innovation, the success of the RMAT designation pathway, and patient
and provider confidence in this emerging field?
Answer 2. Regenerative medicine is one of the most innovative and
promising emerging advancements in our scientific approaches to the
treatment of human disease. Regenerative medicine appears to hold great
promise for new therapeutic options for patients and physicians,
particularly in areas of unmet or underserved medical need. However, as
with all products FDA regulates, the agency must have the appropriate
policies and processes in place to assess and ensure the safety and
efficacy of regenerative medical products before they are approved for
use by American patients. FDA must also ensure patients and providers
are appropriately educated about the potential risks and benefits of
regenerative medicine therapies that fall within the scope of FDA's
oversight, and that these products meet the agency's standard for
safety and effectiveness. If confirmed, I will embrace the
responsibility to facilitate important medical innovation in the
regenerative medicine space, and will seek timely implementation of the
new pathway created as part of 21st Century Cures, while maintaining
the agency's Gold Standard of safety and efficacy.
Question 3. Antibiotic resistance is growing public health threat.
CDC estimates that there are at least 2 million drug-resistant
infections each year in the United States resulting in approximately
23,000 deaths. All antibiotic use increases the risk of development of
antibiotic resistance. In order to minimize the growth of antibiotic
resistance, antibiotics should be prescribed judiciously, but the CDC
estimates that up to 50 percent of antibiotics in human healthcare
settings in the United States are inappropriately prescribed.
What do you feel is the appropriate role for FDA in ensuring
judicious use of antibiotics?
21st Century Cures established the Limited Population Approval
pathway for antibiotics. Are you committed to following the strong
labeling requirements for these products in the law and educating
prescribers about appropriate use?
How will you work to prevent drug shortages of antibiotics, which
can lead to overprescribing of broad spectrum antibiotics?
Answer 3. The availability and appropriate prescribing of
antibiotics are vital to our Nation's public health. Additionally,
antibiotic resistance is a significant and growing public health
challenge facing our Nation. Within its statutory authorities, FDA
should encourage the development of new antibiotics and ensure proper
labeling to help address the issue of inappropriate prescribing and/or
use. If confirmed, I would commit to following the law, as it relates
to the Limited Population Approval pathway for antibiotics. Further, I
would prioritize effectively preventing and decisively alleviating drug
shortages, particularly those of antibiotics, many of which are
parenteral drugs used in hospitalized settings.
Question 4. I believe that precision medicine relies on effective
and meaningful lab tests to inform treatments, and that this area of
innovation should be fostered. However, there are many recent examples
of lab tests being marketed to physicians and patients that have no
real clinical meaning, have led to patients being over- or undertreated
for diseases, or exposed patients to inappropriate therapies or stopped
them from receiving effective therapies. On October 3, 2014 the FDA
issued a draft oversight framework for LDTs based on risk to patients,
rather than whether they were an LDT of IVD. Since that time, Congress
and stakeholders have been actively engaged in the discussion about how
best to modernize lab oversight in the era of precision medicine. In
January of this year, FDA released a white paper that updated the
agency's position based on stakeholder feedback to the 2014 framework.
You have argued that the regulation of these tests is best left outside
of FDA's jurisdiction, and instead, CLIA should be updated to be more
robust.
Do you support the framework in the January 2017 white paper?
What FDA role in oversight of LDTs do you believe is necessary to
address the Agency's longstanding and well-publicized concerns about
those tests?
Do you believe that FDA has the legal authority to regulate lab
rests?
How will you work with Congress and CMS to ensure that lab tests
are both accurate and clinically meaningful?
Are you committed to supporting a regulatory framework that would
give patients and their provider's confidence in the quality and
veracity of their tests, and level the playing field for innovators?
Answer 4. Defining an appropriate regulatory framework for
Laboratory Developed Tests (LDTs) is important to FDA's mission to
protect and promote public health. In order to both protect patient
safety and encourage innovation and patient access, I believe we must
strike the right balance between Clinical Laboratory Improvement
Amendments (CLIA) and FDA regulation and regulatory requirements. If
confirmed, I would commit to working with Congress and stakeholders to
develop appropriate LDT regulatory policies that strike the right
balance between encouraging innovation while making sure that patients
and providers can be confident in the clinical validity of the results
that they receive from LDTs.
Question 5. In your hearing, you were asked whether there should
still be double-blind trials in 2017. While I agree that the FDA should
consider all appropriate trial designs to assess the safety and
efficacy of drugs, I am concerned that your response did not provide
any examples in which you believed that a double-blind study would be
appropriate. Are there any diseases or drug classes that you believe
double-blind studies are the best way to determine efficacy or safety?
If so please include examples. Or do you believe that there are no
circumstances that warrant such a trial?
Answer 5. There are many clinical settings where double-blind,
placebo controlled trials remain the gold standard for properly
determining safety and efficacy and define the appropriate standard for
FDA's approval requirements. While it might not be appropriate for me
to opine on specific approval standards with respect to specific
clinical circumstances and drug trials--as these determinations are
best left to the expert judgment of FDA's career staff--I fully
recognize that there is a continuum of clinical trial designs and
evidentiary standards that FDA must require based on the clinical
setting, the clinical appropriateness, the feasibility, and the
underlying patient need. These requirements are exercised within the
scope of the discretion that is afforded to agency staff by statute and
regulation. This ability of FDA's staff to use judgment in how they
apply the single gold standard for determining safety and effectiveness
is a reflection of the careful consideration that FDA's staff must
strike in order to properly balance access, innovation, and safety. If
confirmed, I will support a strong FDA, with staff making these kinds
of judgments through a rigorous, science-based process.
Question 6. I understand the urgency that patients and their
families feel when they are desperate for new treatments. I believe
that we must ensure therapies are available to those who need them
most. I have concerns about so-called ``right-to-try legislation,''
which could hurt those it is designed to help. I believe that access to
investigational drugs should be done in a way that maintains and
bolsters the drug development process that brings us lifesaving cures,
and prevents a two-tiered system where those who can afford to buy an
investigational treatment can have access and those that cannot have to
wait. In your confirmation hearing I asked if you were willing to stand
up to the Administration if they put political pressure on you, and you
stated that you would give ``unvarnished advice'' and ``clear
thoughts'' on issues you are asked to opine on. Given the Trump
administration's support of right to try laws, please give your
perspective on both the impact on patients, and drug development
process, if the current Federal legislative proposal was enacted.
Answer 6. Access to off-label and investigational products for
patients facing serious and terminal illness is not an abstract issue
to me. As a cancer survivor who used a commercially available
combination therapy in an off-label manner, I understand, at a very
personal level, that patients who are fighting serious or life-
threatening diseases want the flexibility to try new therapeutic
approaches, including access to investigational medical products,
particularly when there is no other FDA-approved treatment option. I
also believe that the clinical trial process is crucial to the
development of innovative new medical products that can improve or save
patients' lives. If confirmed, I would commit to ensuring FDA has the
right policies and processes in place to appropriately balance
individual patients' needs for access to investigational therapies
while recognizing the importance of maintaining a rigorous clinical
trial paradigm for testing investigational products and demonstrating
safety and efficacy. I would be happy to work with Congress as it
considers Right-to-Try legislative proposals.
Question 7. According to a New England Journal of Medicine study
published earlier this week,\1\ the FDA in recent years has approved
more products, and approved them more quickly, than the European
Medicines Agency. In a 2012 article published in National Affairs, you
stated that the FDA puts up too many barriers to approval, and
prioritized safety over speed.
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\1\ Downing, Nicholas S., et al. ``Correspondence: Regulatory
Review of New Therapeutic Agents--FDA versus EMA, 2011-2015.'' The New
England Journal of Medicine, vol. 376, no. 14, 2017, pp. 1386-1837,
http://www.nejm.org/doi/pdf/10.1056/NEJMc1700103. Accessed 7 Apr. 2017.
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Given the implementation of FDASIA and 21st Century Cures, do you
still hold that view?
Can you give examples of products approved in recent years, that
have since demonstrated a clear benefit to patients, and that you
believe the FDA moved too slowly in approving?
Answer 7. FDA has made significant progress in recent years to
ensure that patients in the United States have access to new,
innovative therapies thanks to new legislative pathways like
Breakthrough Therapy designation. The adoption of the Breakthrough
pathway addressed many of the concerns I raised in that 2012 article. I
believe we should continue to look for ways to improve the efficiency
of FDA's medical product review program, modernize the scientific
standards used in drug regulation, and seek more uniform adoption of
pathways created by Congress like the Breakthrough Designation, and
build on these opportunities through adoption of the new provisions in
Congress in 21st Century Cures. We need to do all these things while
continuing to ensure that new products meet FDA's Gold Standard for
safety and efficacy.
Question 8. In my meeting with Tom Price, he stated that he
believed in the gold standard of safety and efficacy for, but that it
needed to be made ``evergreen'' so that it's easier for drug developers
to innovate. Section 505(b) of the Food Drug and Cosmetic Act states
that for a drug to be approved, a person must file an application which
includes ``full reports of investigations which have been made to show
whether or not such drug is safe for use and whether such drug is
effective in use.'' \2\ Please describe if you think this language is
``evergreen'' and applicable to today's technology. What specific
current regulatory authorities and flexibilities does FDA currently
have that makes the current standard ``evergreen?''
---------------------------------------------------------------------------
\2\ 21 U.S.C. Sec. 355(b). 2012. LexisNexis Academic. Web. 7 Apr.
2017.
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Answer 8. Maintaining the Gold Standard of safety and efficacy for
medical products is fundamental to FDA's mission to protect and promote
public health. But owing to opportunities created by evolving modern
science as well as new policies created by Congress--including
provisions in 21st Century Cures--there are now more opportunities to
make sure that FDA is adopting the best science and most efficient and
modern regulatory tools for demonstrating that standard. This creates
opportunities to make the development process more efficient, less
costly, and at times faster, while also improving upon our ability to
demonstrate safety and efficacy. These basic principles have formed the
basis of our collective public health goals with respect to drug
review. I recognize that FDA must reject policies or processes that
would in any way undermine the safety and efficacy of our Nation's drug
and medical technology supply because, for example, we were
inappropriately prioritizing speed over the gold standard for safety
and efficacy. If we are adopting modern science and investing in a
strong FDA workforce, we can strive to achieve greater efficiency while
improving on our gold standard for safety and efficacy. Getting better
efficiency, and taking a risk-based approach to FDA's work, means FDA
needs the appropriate policies, resources, and processes to
consistently utilize 21st century regulatory science. FDA should
thoroughly consider regulatory approaches that could improve the
efficiency of drug and device discovery, development, and regulation,
but only adopt approaches that reliably improve the ability to
determine the safety and efficacy of medical products that Americans
use.
Question 9. While the FDA gold standard of approval helps to ensure
the safety and efficacy of new products, we know that many new drugs
and devices have not been studied on adequate numbers of women, people
of different races and ethnicities, nor all age ranges. By implementing
provisions of FDASIA, the FDA has taken a number of steps to improve
the data required to be submitted for a new drug, and is reporting
through Drug Snapshots the summaries of who was included in trials and
whether there are safety or efficacy differences among subpopulations.
The Drug Snapshots released this year from FDA shows many drugs are
still being tested predominately in white men--even those intended to
treat conditions that can have a disproportionate impact on women or
minorities.
Do you support requiring inclusion of women and minorities in
clinical trial data submitted to the FDA?
Are you committed to supporting additional work by the FDA to
improve the diversity of clinical trials used to support medical
product approvals?
How will you ensure that doctors have information they need about
how a new medication works in these subpopulations?
Answer 9. I think it is very important that clinical trials capture
the diversity of the population who will likely use the medical product
once it is marketed and becomes available. If confirmed, I will work to
ensure that FDA policies support the conduct of clinical trials that
represent the clinical diversity of the intended patient population,
including through the implementation of Section 907 of the Food and
Drug Administration Safety and Innovation Act (FDASIA).
Question 10. This year, Congress needs to reauthorize FDA's
authority to collect user fees from the drug, medical device,
biosimilar, and generic drug industries. It is the first time that
Congress has been charged with this task in the first year of a new
administration--and the Trump administration has proposed to both
``recalibrate'' user fees and replace FDA budget authority with
additional fees. In addition the President has issued a ``Hiring
Freeze'' across the Federal Government.
Do you support reauthorization of the PDUFA, MDUFA, GDUFA, and
BsUFA agreements as finalized and submitted to Congress by the Obama
administration?
How does the FDA's appropriated budget contribute to the Agency's
overall health and independence?
Do you believe that user fees are an effective replacement for
congressional appropriations?
How will you ensure the FDA progresses toward its hiring and
retention goals in the agreements and in the spirit of 21st Century
Cures? In January, Senator Warren and I sent a letter to FDA Acting
Commissioner Ostroff inquiring about the impact and implementation of
the hiring freeze. When can we expect an answer to this correspondence?
Answer 10. The reauthorization proposals for PDUFA, MDUFA, GDUFA,
and BsUFA were developed and submitted to Congress prior to the end of
the previous Administration. I was not involved with the FDA-industry
technical negotiations on any of these proposals. I was also not
involved in the development of the President's Blueprint Budget. I
recognize these user fee programs are critically important to FDA, and
the patients the agency serves, as they provide significant resources
to support FDA's regulatory activities related to innovative and
generic medicines, biosimilars, and medical technologies. In order to
ensure FDA is adequately resourced to facilitate the discovery,
development, and regulatory review of safe and effective medical
products to help American patients, if confirmed, I will work with my
colleagues in the Administration, Congress, industry, and stakeholders
to reauthorize these critical user fee programs in a timely manner. I
will also commit to making sure that congressional correspondence is
answered in a timely fashion.
Question 11. Biologic drugs are extremely important to patients,
but the historic absence of competition has limited access and
affordability to these important medicines while driving up health care
costs. One of the best opportunities to help address this is support
for timely review and approval of biosimilars. The biosimilar drug user
fee agreement supports the review of these products.
Do you support the biosimilars pathway?
How will you work both within FDA and in collaboration with CMS to
encourage and speed their development and availability to patients?
How do you plan to help educate physicians and the public about
biosimilars to increase their uptake in the market?
Answer 11. Yes, I fully support the biosimilars pathway. This
pathway is a critical part of the careful balance Congress prescribed
between access and innovation, and the ability for consumers to get
more value when it comes to the medical products they use. If
confirmed, I intend to work closely with FDA staff and CMS to ensure
the availability of biosimilar products. I recognize the importance of
educating physicians and the public about the availability of, and
FDA's confidence in, approved biosimilar products. I look forward to
working with FDA staff, if confirmed, to increase education and build
on current methods of outreach at FDA.
Question 12. In 2013, a fungal meningitis outbreak took the lives
of 64 people and sickened 751 in more than 20 States, including
Georgia, Tennessee, North Carolina, Illinois, Rhode Island, Minnesota,
and Pennsylvania. Congress responded by passing the bipartisan Drug
Quality and Security Act, which clarified and enhanced FDA's authority
to regulate drug compounding. Are you committed to protecting patients
who need compounded drugs through implementation and enforcement of
this important public health law as written, including the limitations
under 503A?
Answer 12. The practice of pharmacy compounding can serve an
important role, allowing providers to develop individualized
formulations of certain medicines for specific patients with unique
needs. However, I know that there are examples of actors operating as
manufacturers of unapproved new drugs under the guise of a pharmacy
license, violating the careful framework created by Congress,
circumventing the FDA oversight that Congress intended for certain
products, and putting patient safety at significant risk. Congress
clarified FDA's regulatory authorities related to compounding by
passing the Drug Quality and Security Act (DQSA). If confirmed, I am
committed to implementing DQSA, as intended by Congress, to both
protect patient safety, and allow the safe and appropriate practice of
pharmacy compounding to occur in the way that Congress intended.
Question 13. Generic drugs now comprise the vast majority of the
pharmaceutical market, and in many cases are the only products
available for patients after a brand name product is discontinued.
However, generic drug manufacturers are not able to update their labels
efficiently with safety information without prior FDA approval and
without the brand drug's label being updated as well. The FDA has
recognized that this is a public health problem, and issued a proposed
rule to allow such updates, known as CBE-0, but the release of the
final rule has been delayed repeatedly. You have spoken out against
this rule in multiple publications. Will you support issuing
regulations allowing generic drug companies to amend their labels in
order for patients and physicians to have ready access to the most up-
to-date information about their products?
Answer 13. I believe it is important that generic drug labels be
kept up-to-date and generic firms engage in appropriate post-market
safety surveillance. FDA's proposed rule would alter the legal
responsibilities of generic firms. If confirmed, I will work with FDA
staff as we consider future regulatory actions to best achieve the
underlying public health goals.
Question 14. In 2013 in my home State of Washington, 32 patients
were sickened with antibiotic resistant infections that were traced
back to contaminated medical devices known as duodenoscopes. An
investigation by my staff found that duodenoscopes around the country
were harboring these bacteria, and that the cleaning protocols issued
by the manufacturers were not sufficient. Since then, it has come to
light that other reusable medical scopes have harbored potentially
harmful bacteria--putting patients at risk. Section 3059 of the
recently enacted 21st Century Cures Act requires the Secretary to
publish a list of reusable devices, like medical scopes, that are
required to have validated cleaning, disinfecting, and sterilization
information for 510(k) clearance, and publish a guidance to clarify
when device modifications require the submission notification under
510(k). But just 2 weeks ago, another outbreak of antibiotic resistant
infections was traced back to the same devices that the company said
were fixed after the outbreak in Washington.
Are you committed to meeting the deadlines in 21st Century Cures
statue for these publications?
What additional actions will you take to ensure reprocessed devices
are safe for patients?
Answer 14. If confirmed, I will continue to emphasize the
importance of maximizing patient benefit and reducing safety risks
related to duodenoscopes and will work to implement the related
provisions of the 21st Century Cures Act. I am committed to working
with FDA staff and Congress, where necessary, to ensure that reusable
devices are safe for patients.
Question 15. My investigation of contaminated duadenoscopes
revealed that for medical devices, ``FDA's reliance on self-reporting
of adverse events by manufacturers and hospitals is unworkable and
outdated, particularly when contrasted with the active post-market
surveillance system for drugs.'' \3\ I believe that the FDA needs to do
more to improve post-market surveillance for medical devices. The FDA
has recently engaged with external stakeholders to establish the
National Evaluation System for health Technology, which acts as a hub
for electronic data sources for medical device outcome and safety
information. The NEST is also supported by the medical device industry
in the MDUFA IV agreement, however, only for uses to improve pre-market
review and approval of devices. Do you support the utilization of NEST
for post-market surveillance activities? If so, do you commit to
requesting funds in your budget for these activities?
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\3\ ``Preventable Tragedies: super bugs and How Ineffective
Monitoring of Medical Device Safety Fails Patients.''United States
Senate Committee on Health, Education, Labor, and Pensions, 13 Jan.
2016, https://www.help.senate.gov/imo/media/doc/Apples.pdf.
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Answer 15. The reauthorization proposals for PDUFA, MDUFA, GDUFA,
and BsUFA were developed and submitted to Congress prior to the end of
the previous Administration. I was not involved with the FDA-industry
technical negotiations on any of these proposals.
However, I am generally supportive of data transparency and
recognize the importance of collecting valid pre- and post-market data
that can be used to bring new medical devices to market, expand
indications for approved medical devices, and enhance the agency's
ability to collect important patient safety information. If confirmed,
I will commit to working with the FDA professional staff to quickly get
up to speed on this issue and help the agency evaluate whether NEST
could be an appropriate tool for post-market surveillance.
Question 16. In 2016, CMS and FDA wrote a joint letter in support
of including unique device identifiers (UDI) in medical claims during
the next update of the electronic form, the process for which is
ongoing. Both agencies recognize that our health care system takes too
long to recognize problems with devices and then take appropriate
actions, harming patients and resulting in billions in preventable
costs to Medicare. As pointed out by clinical societies (including the
Society of Thoracic Surgeons, American College of Cardiology, and
American Academy of Orthopedic Surgeons), health plans, hospitals,
Democrats, Republicans, and registries such as the American Joint
Replacement Registry and many others, adding medical device identifiers
to health insurance claims would generate better data to detect these
problems sooner.
Will you continue to support the process of adding device
identifiers to claims as a critical tool to better understand the
performance of these products after approval?
How will you engage with stakeholders, including CMS and the X12
Committee, to facilitate the adoption of a field for the DI in
electronic insurance claims following the February recommendation of
X12 to move forward such a field.
Answer 16. I am committed to reviewing the work done to date by
staff at CMS and FDA on this issue. Appropriate policies that could
enhance our ability to capture valid post-market data should be
thoughtfully considered. This also includes achieving interoperable
electronic health records with UDIs--a goal that is fully consistent
with the health information technology provisions in 21st Century
Cures. If confirmed, I look forward to working with my colleagues at
CMS and the X12 Committee to explore all of these policies.
Question 17. Medical devices, including imaging equipment, can be
used for many years and can undergo maintenance and repair. This
medical device servicing, when done by the original equipment
manufacturer, is subject to FDA regulation. However, if servicing is
done by a 3d party, it is not subject to FDA oversight. This regulatory
gray area causes uncertainty for doctors and patients who trust that
medical devices are held to FDA standards, and for equipment
manufacturers who are liable for the safe and effective performance of
the devices. I was pleased that last year, the FDA opened a public
docket for comments and held a public workshop on medical device
servicing. What do you believe FDAs role is in ensuring that servicing
of medical devices by original equipment manufacturers, hospitals, and
3d parties is held to the same standards?
Answer 17. An important part of FDA's responsibility to protect and
promote public health is upholding the Gold Standard of safety and
efficacy for medical products American patients use. With regard to the
issue of medical devices that are serviced by 3d parties, if confirmed,
I will commit to quickly engaging with FDA's staff to get up to speed
on this issue, including a review of the public comments received by
the agency. I look forward to working with FDA's staff, Congress, and
stakeholders to ensure that the agency has in place the right policies
and processes to ensure the safety and efficacy of medical devices.
Question 18. While some blood donor deferral criteria are based on
an individual's risk of a transfusion transmissible infection, others
are not. Non-risk-based criteria prevent many healthy people from
donating blood, while still allowing some high risk donors to donate.
In December 2015, the FDA published final guidance that overturned the
non-risk-based criteria that banned on blood donations from men who
have sex with men (MSM). The 2015 guidance replaced the lifetime ban
with a 1-year deferral, but the 1-year deferral remains an arbitrary
time-based deferral, not a risk-based deferral. Since the guidance was
released, the FDA, in collaboration with other HHS agencies, has been
working to collect the data necessary to implement a true risk-based
deferral system for all donors, which will lead to a more robust and
safer blood supply for American patients.
Do you commit to continuing the studies and data collection
necessary, including monitoring of behavioral risk factors of viral
infections through the Transfusion-Transmissible Infections Monitoring
System (TTIMS), to support the goal of transitioning to a risk-based
blood donation deferral system for all blood donors?
Answer 18. Ensuring the safety and adequacy of our Nation's donated
blood supply is critically important to public health. If confirmed, I
will work with FDA staff to closely develop, implement, and monitor the
impact of policies to promote blood safety. I will also commit to
continuing to work with FDA staff to review its donor deferral policies
to ensure they reflect the most up-to-date scientific knowledge.
Question 19. I am fully supportive of the bipartisan Food Safety
and modernization Act, which has since helped to protect the public's
health, strengthen consumer confidence in American food products, and
level America's playing field with foreign competitors. FDA oversees 80
percent of the food supply and its oversight prevents countless
incidents of foodborne illness every year. Critical to implementation
are the major rules that are due to go into effect this year and
funding for the States, which will take most of the responsibilities
for on-farm inspections and other day-to-day work under the produce
rule. Are you committed to requesting the funds in your budget
necessary to support FDAs implementation of FSMA to ensure that
consumers remain confident in our food supply?
Answer 19. The Food Safety Modernization Act (FSMA) provides FDA
with important tools and authorities to support its responsibility to
ensure the safety of our Nation's food supply. If confirmed, I will
work to ensure the agency has the appropriate policies, processes, and
resources in place to implement FSMA, as intended by Congress. FDA
should implement FSMA in a way that protects and promotes public health
by enhancing food safety, while also collaborating with the U.S.
Department of Agriculture, State officials, and other government
agencies to conduct regulatory activities in a manner that takes into
account the unique challenges faced by small farmers and small
businesses.
Question 20. According to the CDC, two million Americans develop
antibiotic-
resistant infections each year, costing 23,0000 lives and $20 billion
annually. The World Health Organization's global assessment of
antibiotic resistance concluded that antibiotic resistance ``is now a
major threat to human health.'' I am particularly concerned by the
connection between the use of antibiotics in agriculture and increasing
antibiotic resistance among foodborne pathogen, which CDC estimates
cause nearly half a million Americans to become sick each year. Public
health authorities, including CDC and the FDA, and the production
agriculture sector itself have made important strides to begin to
address these challenges. I was pleased the FDA finalized Guidance for
Industry 209 and 213 which established judicious use principles and
removed production indications for medically important antimicrobials.
FDA's publication of the Veterinary Feed Directive (VFD) final rule
provided veterinarians with clear direction for how to authorize the
use of antibiotics that are important to human health in animal feed
when they are needed to protect animal health; this will further
reinforce the critical role of veterinarians in animal-health
decisionmaking.
How do you plan to ensure that antibiotics are indicated for
``disease prevention'' at the same doses and duration as now removed
production indications--meaning they could still be used non-
judiciously--are being used for legitimate prevention uses?
How do you plan to collaborate with USDA to collect the on-farm
data required to truly assess antibiotic use?
Answer 20. Antibiotic resistance is a significant and growing
public health challenge facing our Nation. In addition to measures FDA
should take to address this issue within the context of human use, the
agency must effectively collaborate with other government agencies and
public health authorities to develop policies and processes to address
the issue of antibiotic use in animals intended for human consumption.
If confirmed, I will ensure FDA remains engaged on this important
public health issue, making sure that animal drug labeling reflects the
most up-to-date science, and working closely with the U.S. Department
of Agriculture, the Centers for Disease Control, the U.S. Department of
Defense, and other appropriate Federal and State agencies. FDA should
also consider input from other important stakeholders, such as the
farmers, the agriculture industry, and veterinarians. FDA's
implementation of a voluntary plan with industry to phaseout the use of
certain antibiotics is an important step in the direction of more
appropriate stewardship and use of antibiotics.
Question 21. Drug companies are only allowed to market their
products for the uses approved by the FDA. Proponents of relaxing this
FDA standard to allow more so called ``off-label communication'' argue
that such communication is critical for physicians in this age of rapid
innovation. Opponents of off-label communication believe it will lead
us back to an era where companies could peddle unproven products and
reduce the incentive to invest in truly innovative research and
development. In a Forbes column, you agreed with this point, writing,
``[t]he most important incentive to developing useful information
remains the ability for companies to market drugs based on what can be
proven scientifically.'' \4\ Can I have your commitment that, if
confirmed, you will ensure that FDA regulation of communications will
preserve physician and public trust in approved medical products and
the important incentive of FDA approval?
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\4\ Gottlieb, Scott. ``Merck's Pain is Medicine's Gain.'' Forbes, 4
Oct. 2014, https://www.
forbes.com/2004/10/04/cz_sg_1004soapbox.html.
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Answer 21. Medical product labeling is one of the primary tools FDA
uses to promote the appropriate use of medicines and technologies and
communicate risk information. It is important that information on
product labeling is accurate, clear, and scientifically based; and be
the result of a sound regulatory process. Further, it is crucial that
manufacturers continue to develop and submit to the agency clinical
data demonstrating the safety and efficacy of medical products for new
indications they seek to include on labeling and in their marketing
communications with patients, payers, and providers. I also believe
that patients and physicians make the best decisions when they have
access to as much truthful, non-misleading, scientifically based
information as possible. FDA has long recognized that there is public
health benefits of allowing certain non-promotional communication about
truthful, non-misleading, clinical information that is not previously
incorporated into FDA-approved product labeling. If confirmed, I will
commit to working with FDA's professional staff to get up to speed on
the agency's latest thinking and actions on these matters, and
providing clarity to manufacturers, payers, providers, and patients
about acceptable truthful and non-misleading communications related to
clinical data not already incorporated in a label.
Question 22. As a physician, I am sure you are aware that tobacco
use is still the leading preventable cause of death and disease in the
United States.\5\ Nonetheless, when Congress was on the verge of
approving the legislation which allowed FDA to regulate tobacco
products in 2009, you were quoted as saying that, instead of addressing
the public health threats posed by tobacco products, this legislation
would ``gut'' the FDA's resources--and ``distract it from its core
mission.'' \6\ Nearly 8 years have passed since Congress enacted the
Family Smoking Prevention and Tobacco Control Act, and the law has not
gutted FDA's resources or distracted it from its core mission. As FDA
Commissioner, you would oversee the Center for Tobacco Products, which
is funded by user fees from the tobacco companies. Have your views on
FDA oversight of tobacco products changed? What initiatives do you
envision the Center for Tobacco Products take to reduce the death and
disease caused by tobacco?
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\5\ ``Burden of Tobacco Use in the U.S.--Current Cigarette Smoking
Among U.S. Adults Aged 18 Years and Older.'' Centers for Disease
Control and Prevention, https://www.cdc.gov/tobacco/campaign/tips/
resources/data/cigarette-smoking-in-united-states.html. Accessed 7 Apr.
2017.
\6\ Associated Press. ``Lawmakers Renew Efforts for Government
Regulation of Cigarettes.'' Fox News, 2 Mar. 2017, http://
www.foxnews.com/politics/2009/03/02/lawmakers-renew-efforts-
government-regulation-cigarettes.html.
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Answer 22. If confirmed, I will be committed to implementing the
TCA, as intended by Congress. As I noted during my confirmation hearing
before the committee, as a physician and cancer survivor, if confirmed,
I will be fully committed to the TCA's public health goal of reducing
morbidity associated with tobacco use in this country. I believe
responsibly implementing the TCA is an integral part of FDA's core
mission to protect and promote public health.
Question 23. Last year, FDA issued a final rule under the
authorities of the Family Smoking Prevention and Tobacco Control Act
(TCA), which enabled the agency to begin to oversee e-cigarettes and
other tobacco products. In the years before FDA completed this rule, e-
cigarette manufacturers introduced thousands of nicotine-
delivering devices and liquids to the marketplace, many with fruit and
candy flavors, without having to meet any independent standards to
protect consumers' and the public's health. During this absence of FDA
oversight, youth use of e-cigarettes soared, eclipsing youth use of
regular cigarettes. E-cigarette proponents argue that they are less
harmful than regular combustible cigarettes and can help adult
cigarette smokers to quit. However, many e-cigarette manufacturers do
not want to develop the data to support these claims. They instead want
to exempt e-cigarettes that were on the market before the effective
date of the deeming rule from a scientific review by FDA. Under the
product review requirement in the TCA, tobacco product manufacturers
must provide information about their products so that FDA can assess
the toxicity, addictiveness, and appeal to youth of a new product. This
independent scientific review will help answer important questions
about these products, like whether manufacturers--use of flavors is
increasing youth use of e-cigarettes, whether adults are using e-
cigarettes to quit regular cigarettes or do they continue to use
regular cigarettes, and what the risks are of using e-cigarettes. It
will enable FDA to block more harmful or addictive products from the
market and incentivize manufacturers to develop products that pose less
risk to public health.
Do you support the deeming rule, including the product review
requirement?
Can I count on you to ensure that this rule is fully implemented
and not weakened?
What will you do to help prevent new users from getting hooked on
nicotine through electronic cigarettes?
Answer 23. If confirmed, I will be committed to implementing the
TCA, as intended by Congress, including Section 911 related to modified
risk products, which I recognize can provide helpful tools for current
tobacco-users to transition off combustible tobacco. As I was not at
FDA during the agency's initial TCA implementation activities, I am not
fully acquainted with internal processes or specific decisions to-date.
If confirmed, I will work with the professional staff to quickly get
up-to-speed on this issue, and I will review current FDA policies,
including the deeming rule, to ensure FDA treats products
appropriately, implements provisions in a timely fashion, and in a
manner that is consistent with congressional intent under the TCA.
Clearly, manufacturers should not be allowed to target minors through
indefensible marketing options of any tobacco related products,
including e-cigarettes. I believe responsibly implementing the TCA is
an integral part of FDA's core mission to protect and promote public
health.
Question 24. The FDA was instructed by Congress to look into the
addition of menthol to cigarettes and its impact on public health. In a
2011 report, the FDA found that menthol is a problem--it is more likely
to initiate smokers and keep them hooked.\7\ However, the FDA has not
promulgated regulations to reflect this threat. What would you do to
address the threat of menthol cigarettes?
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\7\ ``Preliminary Scientific Evaluation of the Possible Public
Health Effects of Menthol Versus Nonmenthol Cigarettes.'' Food and Drug
Administration, 23 Aug. 2013, https://www.fda.gov/downloads/
UCM361598.pdf.
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Answer 24. I have not reviewed the scientific evidence related to
the addition of menthol in cigarettes. If confirmed, I will commit to
engaging with FDA's staff to quickly get up to speed on the regulatory
history of this issue, and the agency's latest information, thinking,
and actions. I would welcome the opportunity to work with Congress on
this issue moving forward.
Question 25. In 2012, you wrote an op-ed in the New York Post on
FDA oversight of premium cigars. In that article, you suggest that
expanding FDA's oversight to premium cigars was ``never envisioned by
Congress'' and that doing so could divert the agency's attention from
other important duties. In 2014, FDA issued a proposed rule that sought
comments on whether FDA should oversee all cigars or exempt premium
cigars. In 2016, it issued a final rule that--based on its review of
comments and the scientific evidence--concluded ``there is no
appropriate public health justification to exclude premium cigars from
the scope of the final deeming rule.'' \8\ It concluded that all cigars
pose serious health risks, all cigars are potentially addictive, and
premium cigars are not exclusively used by adults.
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\8\ Deeming Tobacco Products To Be Subject to the Federal Food,
Drug, and Cosmetic Act, as Amended by the Family Smoking Prevention and
Tobacco Control Act; Restrictions on the Sale and Distribution of
Tobacco Products and Required Warning Statements for Tobacco Products,
81 Fed. Reg. 90 (May 10, 2016). Federal Register: The Daily Journal of
the United States. Web. 10 May 2016. https://www.federalregister.gov/
documents/2016/05/10/2016-10685/deeming-
tobacco-products-to-be-subject-to-the-federal-food-drug-and-cosmetic-
act-as-amended-by-the.
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As FDA Commissioner, would you accept the assessment of FDA's
scientists about the health risks of cigars? Would you continue to
implement and enforce the deeming rule as it applies to premium cigars?
I am concerned about the use of these products by children,
particularly high school boys, who now smoke cigars at a higher rate
than regular cigarettes. Are you concerned about efforts to exclude
from FDA regulation a product that is so frequently being used by high
school boys?
Answer 25. If confirmed, I will work with FDA's professional staff
to quickly get up-to-speed on this issue, and I will review current FDA
policies, including the deeming rule, to ensure FDA treats products
appropriately, implements provisions in a timely fashion, and in a
manner that is consistent with congressional intent under the TCA. I
will also commit to better understanding the decision FDA made with
respect to premium cigars--and any considerations that were made in
respect to premium cigars between the proposed and the final rule--and
would be happy to work with Congress on this issue.
Question 26. Today, Americans consume sodium mostly through
processed foods that they purchase from a grocery store or at a
restaurant.\9\ Researchers estimate that reducing current sodium
intakes by 1,200 milligrams a day would prevent 60,000 to 120,000 cases
of coronary heart disease, 32,000 to 60,000 cases of stroke, and 54,000
to 99,000 heart attacks annually. This reduction would also save an
estimated $10 billion to $24 billion in health-care costs and 44,000 to
92,000 lives annually.\10\ Last June, FDA proposed draft voluntary
guidance to industry to reduce sodium in processed and restaurant foods
and has received comments from industry, public health groups and
consumers. Will you commit to the Agency's finalizing this voluntary
guidance in 2017?
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\9\ ``Salt--Sodium and Food Sources.'' Centers for Disease Control
and Prevention, https://
www.cdc.gov/salt/food.htm. Accessed 7 Apr. 2017.
\10\ Bibbins-Domingo, Kirsten, et al. ``Projected Effect of Dietary
Salt Reductions on Future Cardiovascular Disease.'' The New England
Journal of Medicine, vol. 362, no. 7, 2010, pp. 590-99, http://
www.nejm.org/doi/full/10.1056/NEJMoa0907355#t=article. Accessed 7 Apr.
2017.
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Answer 26. We need to ensure that everything FDA does is science-
based and try to encourage science-based, voluntary action to reduce
sodium levels in foods. I know some companies have already taken
voluntary steps to reduce sodium levels, and I support these public
health goals. In some cases sodium plays an important food safety role,
but many companies are already reducing sodium levels, and we want to
find ways to continue to encourage those actions in a risk-based and
science-based manner. If confirmed, I will review the comments received
from stakeholders and the scientific evidence related to salt intake as
well as consult agency staff before proceeding on this issue. I am
committed to taking science-based steps, within the scope of FDA's
authority and mandate, to reduce the burden of heart disease.
Question 27. On January 19, 2017, FDA and EPA published a guidance
document concerning seafood consumption by pregnant and nursing women,
which was a significant departure from the draft advice on the same
topic. While I have long urged FDA to finalize this advice, I also have
emphasized that the advice must reflect the latest science and be
presented to consumers clearly so they can make the best possible
decisions about the nutritional value of seafood during pregnancy and
nursing. I am concerned that the final advice appears not to meet that
standard. If confirmed, will you consider revisions to this document to
ensure it is in line with the latest science and provides clear advice
to consumers?
Answer 27. If confirmed, I will ensure FDA's advice concerning
seafood consumption by pregnant and nursing women is based on the most
current and relevant nutritional science and appropriately takes into
account both the nutritional benefits, and any toxicological risks
associated with seafood consumption. I will also work to ensure
effective collaboration between FDA and the U.S. Environmental
Protection Agency (EPA) on this issue, and a range of other public
health matters over which both agencies share regulatory authority.
Question 28. After many delays, the compliance date for menu
labeling requirements is May 5, 2017. Calorie labeling at chain
restaurants, supermarkets, convenience stores, and other food service
establishments allows consumers to make their own choices about what to
eat and feed their families. This information is more important than
ever because people are eating out more than ever before; in 2015, food
sales at restaurants surpassed spending at grocery stores for the first
time.\11\ Furthermore, more than 70 percent of Americans support menu
labeling.\12\ Researchers have concluded that menu labeling could
prevent up to 41,000 cases of childhood obesity and save over $4.6
billion in healthcare costs over 10 years.\13\ The restaurant industry
and over 100 nutrition and public health organizations and
professionals supported the law, which was the result of a bipartisan
compromise. Many food establishments including Starbucks, McDonald's,
Panera, Publix Super Markets, Wegmans Food Markets, and many others
recognize the importance of this public health measure and are already
labeling or working toward the May 5 compliance date. Unfortunately,
efforts to weaken the menu labeling law continue. If confirmed, how
will you ensure that the FDA implements menu labeling beginning May 5,
2017, as planned, and does not penalize food establishments that have
followed the law and prepared to meet the compliance date over food
establishments that have not? How will you ensure that all Americans
have access to basic nutrition information to allow them to make up
their own minds about what to eat?
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\11\ Jamrisko, Michelle. ``Americans' Spending on Dining Out Just
Overtook Grocery Sales for the First Time Ever.'' Bloomberg Markets, 14
Apr. 2015, https://www.bloomberg.com/news/articles/2015-04-14/
americans-spending-on-dining-out-just-overtook-grocery-sales-for-the-
first-time-ever.
\12\ ``Poll Data re: Support Caloric Labeling in Supermarkets,
Vending Machines, Movie Theaters.''Center for Science in the Public
Interest, 27 May 2012, https://cspinet.org/sites/default/files/
attachment/restaurant-calorie-content.pdf.
\13\ Gortmaker, Steven L. ``Three Interventions that Reduce
Childhood Obesity Are Projected to Save More than they Cost to
Implement.'' Health Affairs, vol. 34, no. 11, 2015, pp. 1932-39, http:/
/content.healthaffairs.org/content/34/11/
1932.full?ijkey=lnFXpx4AIM506&keytype=ref&
siteid=healthaff. Accessed 7 Apr. 2017.
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Answer 28. While I am broadly aware of the menu labeling issue,
this is not a matter on which I am familiar with the specific technical
details. As a general matter, I support providing clear, accurate, and
understandable information to American consumers to help inform healthy
dietary choices. I believe information about caloric content can be a
useful tool.
However, I am mindful of the unique challenges that developing and
communicating such information can pose, particularly on small,
independent businesses. If confirmed, I will commit to working with
FDA's professional staff to quickly get up to speed on the regulatory
history related to menu labeling, as well as FDA's latest thinking and
actions. I would welcome the opportunity to work with Congress and
stakeholders to ensure any regulatory requirements would promote public
health by providing helpful information to consumers, while not placing
excessive compliance burden on businesses, particularly small,
independent ones.
Question 29. In your November 2005 speech to the Grocery
Manufacturers of America (GMA) as Deputy Commissioner for Medical and
Scientific Affairs, you made a number of important statements about the
importance of nutrition in public health:
``Clearly, there is a correlation between food, diet and
disease.''
``FDA has also emphasized that our policies need to be
solidly based on the latest science, and must emphasize protecting and
helping consumers.''
``. . . (A)ll of these efforts represent a significant
update to the food label based on science that has been developed in
recent years, and it represents a major opportunity to re-educate
consumers about the food label, and the impact of diet on their
health.''
``As people shop for food, they should have at their
fingertips accurate, helpful, and understandable information about the
most important nutritional implications of the products on the shelves,
and they should be able to easily fit individual food products into
overall healthy diets. People should not need a calculator or an
advanced degree in math or nutrition to calculate the components that
comprise a healthy meal.''
Following up on these statements of yours, I have a few questions:
Will you commit to prioritizing giving industry the guidance they need
to move forward with updated packages and maintaining a compliance date
no later than July 2019 for the revised Nutrition Facts label, which
would be a 1-year delay from the current compliance date? Other
countries, like the United Kingdom, France, and Chile, have implemented
front-of-package labeling where a consumer does not ``need a
calculator'' as you so aptly put it, and major international companies
with strong market presence in the United States--Mars, Nestle
Mondelez, Coca-Cola, Unilever, and Pepsi--have already taken major
strides toward uniform labeling across Europe. As commissioner, would
you move ahead with a uniform and useful version of front-of-pack
labels?
Answer 29. I agree that providing industry sufficient information
to comply with this rule, as with any rule, is imperative. I also agree
that FDA should be open to considering and evaluating additional
approaches, especially those that could promote better consumer
awareness and understanding of nutritional information about the foods
they eat.
Question 30. Currently, there is little knowledge around the safety
and efficacy of medications used by pregnant and lactating women, due
in large part to a lack of inclusion in clinical trials for medications
and treatments. The 21st Century Cures Act created a Federal task force
to examine some of the issues involved in doing so but much more
remains to be done. What steps would you take to increase knowledge
regarding safe treatments for pregnant and lactating women?
Answer 30. If confirmed, I would look forward to FDA's
participation in the Federal task force to examine the inclusion of
pregnant and lactating women in clinical trials. As always, my
commitment is to seek advances in treatments that are useful to
individuals in all stages of life. Understanding the impact of
treatments on women who are pregnant and lactating is crucially
important. I commit to working with scientific experts at the various
agencies and participants of the task force to develop our body of
knowledge on safe treatments for this population and help to make sure
FDA policies reflect this science.
Question 31. We know that you would like to see FDA partner with
industry to bring innovative products more quickly to market for the
benefit of patients, who are the ultimate beneficiaries of
breakthroughs and improved drugs and devices. However, you disapprove
of ACA provisions intended to make preventive care more widely
available to patients, especially women. What is the appropriate role
of government in ensuring that all women can benefit from newly
approved preventive care like contraception and pre-exposure
prophylaxis?
Answer 31. As the nominee to be the next Commissioner of Food and
Drugs, I do not believe it would be appropriate to comment on questions
about issues that are outside the jurisdiction of FDA.
Question 32. Dr. Gottlieb, you are aware of the history of the
Thalidomide disaster in the 1960s, which was fortunately averted in the
United States because of our rigorous drug approval standards. Pregnant
women in other countries were not so fortunate. They were prescribed
Thalidomide, a treatment for morning sickness that was not thoroughly
tested, and which resulted in severe birth defects and complications.
You cited this case in a lengthy article criticizing the ``culture'' of
the FDA. You grudgingly concede that FDA's rejection of Thalidomide was
a success story, avoiding the disastrous results women experienced in
other countries. Yet you conclude that the Thalidomide episode led FDA
to become overcautious, and too concerned with product safety. Do you
believe, when it comes to any experimental product for women's
reproductive health, that today's FDA should prioritize speedy approval
over thorough study and understanding of long-term effects?
Answer 32. Maintaining the Gold Standard of safety and efficacy for
medical products is fundamental to FDA's mission to protect and promote
public health. There are also unique risks and challenges when it comes
to studying drugs used in pregnancy, and for this reason, we have not
seen the sort of innovation and investment in drugs to treat conditions
of pregnancy as we have achieved in other areas of clinical medicine.
Because of the unique safety issues related to drugs used during
pregnancy, we need to make sure we are investing in, and using, the
best science to fully evaluate the benefits and potential long-term
effects of any drug used in this setting. Making sure we maintain the
gold standard for safety and effectiveness in this clinical setting,
while finding ways to help facilitate investment and innovation in
medicines used to support pregnant women, should be one of our highest
public health priorities.
Question 33. FDA's own guidance states that advisory committees
should be empaneled when the matter before the agency is one of
significant public interest. However, through the years, the FDA has
been inconsistent in convening external advisory committees for opioid
approval decisions, often bypassing this step all together. The
Comprehensive Addiction and Recovery Act (CARA), passed in July 2016,
requires FDA to convene an Advisory Committee for any new drug that is
an opioid, except if the agency finds that that such referral is not
necessary for public health and notifies Congress.\14\ But in the FDA's
``Opioid Action Plan,'' released September 2016, the agency only
committed to empaneling an external advisory committee for every opioid
under consideration for approval without abuse-deterrent properties.
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\14\ Public Law 114-198, Section 106.
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In 2011, FDA approved a reformulated, abuse deterrent version of
Opana ER without the benefit of review from an advisory committee. Just
last month, a post-market review of Opana ER found that the risks of
Opana ER substantially outweighed the benefits. Given examples like
this and the goal of CARA, do you believe opioids with claims of abuse-
deterrent properties should also go through an advisory committee, as
appropriate?
How will you determine whether a new opioid drug should go to an
advisory committee?
Do you commit to ensuring that those who serve on advisory
committees for the FDA are held to its current high standard of
impartiality to avoid conflicts of interest?
Answer 14. I believe FDA should have the benefit of independent
advice from outside experts and convene Advisory Committees, when
appropriate. I understand that this advice is often critical to FDA as
they consider challenging regulatory decisions. I believe that FDA
should develop a comprehensive and consistent policy with respect to
the types of opioids that should be reviewed by an Advisory Committee.
Whether an Advisory Committee is convened should not be made only on a
product-by-product basis. There should also be clear guidelines used to
make these determinations. I believe it is important that potential
Advisory Committee members are screened for conflicts of interest.
Question 34. In 2015, more than 650,000 opioid prescriptions were
filled in the United States.\15\ One-in-five individuals experiencing
non-cancer pain or living with a pain-related diagnosis received an
opioid prescription from an office-based setting.\16\ And a survey of
primary care physicians found that 46 percent of doctors mistakenly
believed that abuse deterrent formulations are less addictive.\17\
These statistics indicate that many in the provider community are
dangerously unaware of the risks of prescribed opioids. An advisory
committee to the FDA suggested that the agency put in place mandatory
training for prescribers. Currently, this opioid prescriber education
is voluntary and fewer than 15 percent of prescribers have availed
themselves of this training. The FDA has determined that it does not
have authority to mandate prescriber training, but the agency and
others inside and outside of the government have called on Congress and
the DEA to institute a mandatory training/education requirement for
DEA-controlled substances license, with input on the content of
training coming from HHS.
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\15\ ``The Opioid Epidemic: By the Numbers.'' U.S. Department of
Health and Human Services, https://www.hhs.gov/sites/default/files/
Factsheet-opioids-061516.pdf. Accessed 7 Apr. 2017.
\16\ ``Opioid Overdose--Prescribing Data.'' Centers for Disease
Control and Prevention, https://www.cdc.gov/drugoverdose/data/
prescribing.html. Accessed 7 Apr. 2017.
\17\ Hwang, Catherine S., et al. ``Primary Care Physicians
Knowledge and Attitudes Regarding Prescription Opioid Abuse and
Diversion.'' The Clinical Journal of Pain, vol. 32, no. 4, 2016, pp.
278-84, http://journals.lww.com/clinicalpain/Citation/2016/04000/
Primary_Care_Physi-
cians Knowledge_And_Attitudes.1. aspx. Accessed 7 Apr. 2017.
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Do you agree that all prescribers should have some minimal
education and familiarity with the dangers of opioid addiction and
overdose as well as best practices for prescribing?
How will the FDA engage with the CDC, the CARA-established task
force on best practices, and the Trump administration's taskforce on
opioids to ensure appropriate use of prescription painkillers?
What steps need to be taken to ensure the FDA can help facilitate
this type of prescriber education?
Answer 34. Opioid abuse, misuse, and addiction constitute one of
the most urgent and immediate public health threats facing our Nation.
It is also the biggest public health crisis facing the FDA. The human
and economic toll of this crisis is staggering. If confirmed, this will
be my highest immediate priority. I will make sure FDA is aggressive,
forward leaning, and fully engaged in combating this epidemic. I will
work with FDA's professional staff to ensure FDA has the right policies
and processes in place to:
Facilitate the developments of new approaches and
technologies to reduce the abuse/addictive potential of painkillers
American patients use;
Support the development of non-opioid analgesic
alternatives for physicians and patients;
Assess whether FDA's current approach to opioid regulatory
decisions, including labeling, REMS, and physician/patient education
are appropriate, robust, and fully effective;
Encourage the development of new pharmacological tools for
physicians and patients to both prevent opioid misuse and abuse, and
support treatment and recovery for patients struggling to overcome
opioid addiction;
Enhance physician and patient educational materials to
strengthen public awareness of the risks of opioids, as well as the
FDA-approved resources available to them, using the full range of FDA's
risk communication tools to better target this information;
Taking steps to make sure that providers are appropriately
educated on identifying, and helping to property intervene with, abuse-
prone patients;
Re-assess whether FDA has the appropriate framework and
authorities for evaluating the risk of abuse and diversion as a
component of its review and approval process for opioids;
Undertake a comprehensive effort to evaluate the full
scope of the sources and threats from foreign imported narcotics;
Evaluate whether FDA should bring more alignment between
the review and approval of different medical product platforms used in
the treatment of pain to make sure the agency is adopting the best
public health standard in assessing these products; and
Collaborate effectively with other government agencies and
external stakeholders to develop and execute comprehensive and
effective strategies to win the battle against opioid abuse, misuse,
and addiction. This includes steps for FDA to more closely collaborate
and coordinate with DEA around the two agencies' shared goals.
Question 35. In 2007, you openly criticized the REMS program in
writing \18\ and in speeches.\19\ During your confirmation hearing,
however, you indicated that your original concerns about REMS did not
manifest because FDA used the REMS process more judiciously than
initially proposed. Recognizing the overuse and addiction potential of
all opioid painkillers, in 2012, the FDA approved a post-market shared
REMS for a subset of highly potent prescription opioid painkillers
known as extended-release (ER) and long-acting (LA) formulations, of
which OxyContin is included. In developing the class-wide ER/LA REMS
established in 2012, FDA convened an advisory committee that
overwhelmingly indicated that the REMS proposed was not sufficient to
address the risks associated with use of opioid painkillers.\20\
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\18\ Gottlieb, Scott. ``Drug Safety Proposals and the Intrusion of
Federal Regulation Into Patient Freedom and Medical Practice.'' Health
Affairs, vol. 26, no. 3, 2007, pp. 664-77, http://
content.healthaffairs.org/content/26/3/664. Accessed 7 Apr. 2017.
\19\ Gottlieb, Scott. ``Remarks by Scott Gottlieb, M.D., Deputy
Commissioner for Medical and
Scientific Affairs, Food and Drug Administration.'' American Medical
Association, 12 June 2006, https://www.fda.gov/NewsEvents/Speeches/
ucm051908.htm.
\20\ ``Summary Minutes of the Joint Meeting of the Anesthetic and
Life Support Drugs Advisory Committee (ALSDAC) and the Drug Safety and
Risk Management Advisory Committee.'' Food and Drug Administration,
http://www.supportprop.org/wp-content/uploads/2014/12/Minutes_20100722-
23-ALSDAC-DSaRM-M1-Minutes.pdf. Accessed 7 Apr. 2017.
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Do you believe the REMS process has sufficiently mitigated the
risks of opioids as originally intended?
Do you think REMS are a good tool to manage public health risks
associated with opioids?
How would you strengthen this process?
Answer 35. REMS are an important tool for helping to address the
risk of diversion, misuse and addiction related to opioids. But they
are only one tool. Clearly FDA needs to be taking many more, and more
aggressive steps, to address this staggering human catastrophe of
addiction and abuse. To date, no tool or policy, or combination of
approaches, has sufficiently mitigated the risk of opioids. If
confirmed, I will immediately work closely with staff at FDA to see
what additional steps we can take across the full range of FDA's
authorities and responsibilities, including through the use of the
agency's authorities under the REMS provisions, to more aggressively
address this crisis.
Question 36. During your confirmation hearing, you referred to the
opioid crisis as an important public health emergency on the same scale
as Ebola. For Ebola, in a matter of a few months, the U.S. Government
increased funding and resources and stood up centers of excellence to
better understand and combat this epidemic. The FDA responded by doing
the following:\21\
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\21\ Hamburg, Margaret A. ``FDA as Part of a Coordinated Global
Response on Ebola.'' Food and Drug Administration, 28 Oct. 2014,
https://blogs.fda.gov/fdavoice/index.php/2014/10/fda-as-part-of-a-
coordinated-global-response-on-ebola/.
Created an Ebola Task Force to help accelerate the
development of medical products, which included direct engagement with
industry and academic experts.
Deployed staff to the heart of the epidemic.
Collaborated with other agencies and regulators to share
insight and information.
Used the agency's Emergency Use Authorization to test
unapproved products or uses of products to help stymie the spread of
the disease.
Monitored fraudulent or misleading claims from companies
about the efficacy of their products.
If confirmed, will you advocate for similar initiatives to address
the opioid epidemic to happen immediately?
What could the FDA have done differently to help prevent, or at
least stem, the opioid crisis?
If confirmed, how will you incorporate these lessons into your
leadership agenda?
Answer 36. If confirmed, I will take immediate and aggressive steps
to try and get ahead of this crisis. In my opinion, one of the many
lessons learned from our Nation's inability to effectively combat the
opioid epidemic to-date is that we didn't take aggressive action early
enough in the throes of this crisis to stem its tide, and to get ahead
of its evolution from a problem of prescription drugs to one that now
also involves illicit street drugs. That will be one of among other
lessons that I take to this task if confirmed--that to adequately
address the opioid crisis, it will require us to take perhaps even more
aggressive measures than we might have originally contemplated, since
we have not been accurate in measuring the full scope of this growing
crisis, or in effectively calibrating our regulatory steps to confront
the epidemic. I also believe, among other things, effectively combating
this crisis is going to require much closer collaboration between
different Federal and State agencies, and it would be my immediate goal
to seek even closer partnerships with agencies such as DEA and CMS and
other Federal and State entities that play an important role in
confronting aspects of this crisis.
Question 37. In 1995, the FDA approved the original formulation of
OxyContin, which FDA considered abuse-deterrent based on premise that
its extended-release properties would make it less likely to be abused.
However, we know from OxyContin that abuse-deterrent formulations do
not make opioids less addictive. Unfortunately, many prescribers appear
to be unaware of this pivotal implication. In fact, a survey of primary
care physicians found that nearly half of all primary care doctors
incorrectly believed that abuse-deterrent formulations are less
addictive.\22\
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\22\ Hwang, Catherine S., et al. ``Primary Care Physicians
Knowledge and Attitudes Regarding Prescription Opioid Abuse and
Diversion.'' The Clinical Journal of Pain, vol. 32, no. 4, 2016, pp.
278-84, http://journals.lww.com/clinicalpain/Citation/2016/04000/
Primary_Care_Physi-
cians Knowledge_And_Attitudes.1.aspx. Accessed 7 Apr. 2017.
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Do you think ``abuse deterrent formulation'' is good terminology
that the agency should continue to use?
What steps would the FDA need to take to make a labeling change
that better characterizes the fact that abuse deterrent does not mean
addiction proof?
Answer 37. We need to make sure we are using the appropriate
terminology to describe these technologies and not creating
misperceptions with respect to how we label these products. If
confirmed into this role, I would be committed to working with the
professional staff at FDA to make sure we are asking the appropriate
questions about how we describe these features in labeling, and what
perceptions are conferred to providers by those descriptions.
Question 38. Last year, the CDC issued guidelines on the use of
opioid pain medication for chronic pain that reflects the rise in
opioid addiction and overdoses.\23\ These guidelines recommended, among
other things, that opioids should be prescribed at the lowest effective
dose, and that an upper limit of 90 milligrams/day should not be
exceeded. The CDC has made clear that a dose above 90 milligrams/day is
dangerously high. Yet opioid formulations come in dosage units that are
so high, just one pill twice a day can exceed 90 mg of morphine. For
example, a patient taking Opana ER 40 mg twice a day is taking the
equivalent of 240 mg of morphine. That is more than 2.5 times the CDC's
upper dose limit. Yet the patient and prescriber may be unaware that
this is a dangerously high dose, because it is only one pill taken
twice a day. If confirmed, how can FDA incorporate some of the CDC
guidelines on opioid medication into FDA policies related to approvals
and labeling?
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\23\ Dowell, Deborah. ``CDC Guideline for Prescribing Opioids for
Chronic Pain--United States, 2016.'' Morbidity and Mortality Weekly
Report, vol. 65, no. 1, 2016, pp. 1-49, https://www.cdc.gov/mmwr/
volumes/65/rr/rr6501e1.htm. Accessed 7 Apr. 2017.
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Answer 38. If confirmed, I would be committed to working with staff
at FDA to fully evaluate the CDC guidelines as one part of a
comprehensive effort to try and address this crisis, and to making sure
we are properly and fully leveraging the expertise at sister agencies
like CDC and other government partners in addressing these challenges.
Question 39. You said during your confirmation hearing that the
opioid epidemic is larger than the FDA, and that we must use an ``all-
of-the-above approach'' to tackling this epidemic. In addition to
spending several years at the FDA, you were also a senior adviser to
the Centers for Medicare and Medicaid Services (CMS). CMS also has
levers to pull to help combat the opioid crisis, including coverage for
and availability of treatment options for mental health and substance
use disorders. Given your knowledge of both CMS and FDA, and your
experience working on behalf of pharmaceutical companies navigating
these agencies, how would you ensure these agencies work together and
coordinate on policies to address the opioid epidemic?
Answer 39. I believe there are many opportunities for agencies to
more closely collaborate to address this crisis. Indeed, properly
addressing it is going to require much closer coordination between law
enforcement and public health agencies at both the local and Federal
level.
Among other things, CMS can help FDA better understand patterns of
use and abuse that can help inform the drug review process, both pre-
and post-market, as well as assist the FDA in its post-market
surveillance. If confirmed, it would be one of my immediate goals, in
an all-of-the-above approach, to seek new ways to collaborate more
closely with other local and Federal agencies, including CMS, to see
where we can gain more alignment and leverage in combating this public
health crisis.
Question 40. In a 2015 op-ed published in the Wall Street Journal,
you argued that the ACA permits a ``government takeover of drug
pricing.'' Specifically, you cite the Centers for Medicare and Medicaid
Innovation (CMMI) as a mechanism for this government control. Even
private sector payers and drug manufacturers agree that value-based
models of drug pricing could help ensure patients have access to
affordable prescription drugs. What types of drug pricing
demonstrations do you think would be reasonable for CMMI to test?
Answer 40. As the nominee to be the next Commissioner of Food and
Drugs, I do not believe it would be appropriate to comment on questions
about issues that are outside the jurisdiction of FDA.
Question 41. In your OGE Form 278, you stated that Venture Partner
at New Enterprise Associates (NEA) was simply a working title. It is
unclear what that means and to what extent you were involved in the
company's broader healthcare portfolio. Collectively, NEA's client
companies have at least 40 drug products in the product pipeline for
which they may seek approval from the FDA. In your ethics agreement,
you committed to recuse yourself from matters involving NEA and two
client companies--American Pathology Partners and Collective Health--
for 1 year.
Will you recuse yourself for the 2 years laid out in the Trump
ethics pledge, from matters involving all of NEA's clients? If not, why
will you not take this extra step promised by the President who
nominated you?
You have committed to recusing yourself for 1 year from the date of
your resignation for matters involving NEA, American Pathology
Partners, and Collective Health. Will you recuse yourself for the 2
years laid out in the Trump ethics pledge for those companies? If not,
why will you not take this extra step promised by the President who
nominated you?
Answer 41. If confirmed, I will abide by all of the commitments set
forth in my ethics agreement and will comply with all applicable laws
and regulations. I will also be bound by the requirements and
restrictions set forth in this Administration's Ethics Pledge (EO
13770). If confirmed, I will meet with the career ethics officials at
the Department of Health and Human Services during the first week of my
service to ensure compliance with all of these ethics standards.
Throughout my public service I will consult with appropriate ethics
officials for guidance on these matters, including with respect to any
recusal obligations not already set forth in my ethics agreement. I am
committed to leading the FDA in an impartial manner that gives the
American public confidence in the integrity of the FDA's decisionmaking
process.
Question 42. In your OGE Form 278e, you disclose that you were an
investor in NEA 14 Limited Partnership (``NEA 14''). You further
disclose that NEA 14 holds an investment in Cerecor, Inc., a
biopharmaceutical company with two compounds--CERC-501 and CERC-611--
currently being tested in clinical trials. While I understand that you
have divested yourself from NEA 14 pursuant to your ethics obligations,
will you recuse yourself personally and substantially in any particular
matter in which Cerecor Inc. is a party, including, but not limited to,
proceedings concerning CERC-501 and CERC-611?
Answer 42. If confirmed, I will abide by all of the commitments set
forth in my ethics agreement and will comply with all applicable laws
and regulations. I will also be bound by the requirements and
restrictions set forth in this Administration's Ethics Pledge (EO
13770). If confirmed, I will meet with the career ethics officials at
the Department of Health and Human Services during the first week of my
service to ensure compliance with all of these ethics standards.
Throughout my public service I will consult with appropriate ethics
officials for guidance on these matters, including with respect to any
recusal obligations not already set forth in my ethics agreement. I am
committed to leading the FDA in an impartial manner that gives the
American public confidence in the integrity of the FDA's decisionmaking
process.
Question 43. In your OGE Form 278e, you disclose that you were an
investor in NEA 14 Limited Partnership (``NEA 14''). You further
disclose that NEA 14 holds an investment in Loxo Oncology, Inc., a
biopharmaceutical company with three compounds--LOXO-101, LOXO-195, and
LOXO-292--currently being tested in clinical trials or slated to begin
Phase I trials imminently. While I understand that you have divested
yourself from NEA 14 pursuant to your ethics obligations, will you
recuse yourself personally and substantially in any particular matter
in which Loxo Oncology, Inc. is a party, including, but not limited to,
proceedings concerning LOXO-101, LOXO-195, LOXO-292?
Answer 43. If confirmed, I will abide by all of the commitments set
forth in my ethics agreement and will comply with all applicable laws
and regulations. I will also be bound by the requirements and
restrictions set forth in this Administration's Ethics Pledge (EO
13770). If confirmed, I will meet with the career ethics officials at
the Department of Health and Human Services during the first week of my
service to ensure compliance with all of these ethics standards.
Throughout my public service I will consult with appropriate ethics
officials for guidance on these matters, including with respect to any
recusal obligations not already set forth in my ethics agreement. I am
committed to leading the FDA in an impartial manner that gives the
American public confidence in the integrity of the FDA's decisionmaking
process.
Question 44. In your OGE Form 278e, you disclose that you were an
investor in NEA 14 Limited Partnership (``NEA 14''). You further
disclose that NEA 14 holds an investment in Lumena Pharmaceuticals,
Inc. a biopharmaceutical company with two compounds--LUM-001 and LUM-
002--currently being tested in clinical trials. While I understand that
you have divested yourself from NEA 14 pursuant to your ethics
obligations, will you recuse yourself personally and substantially in
any particular matter in which Lumena Pharmaceuticals, Inc. is a party,
including, but not limited to, proceedings concerning LUM-001 and LUM-
002?
Answer 44. If confirmed, I will abide by all of the commitments set
forth in my ethics agreement and will comply with all applicable laws
and regulations. I will also be bound by the requirements and
restrictions set forth in this Administration's Ethics Pledge (EO
13770). If confirmed, I will meet with the career ethics officials at
the Department of Health and Human Services during the first week of my
service to ensure compliance with all of these ethics standards.
Throughout my public service I will consult with appropriate ethics
officials for guidance on these matters, including with respect to any
recusal obligations not already set forth in my ethics agreement. I am
committed to leading the FDA in an impartial manner that gives the
American public confidence in the integrity of the FDA's decisionmaking
process.
Question 45. In your OGE Form 278e, you disclose that you were an
investor in NEA 14 Limited Partnership (``NEA 14''). You further
disclose that NEA 14 holds an investment in TRACON Pharmaceuticals,
Inc., a biopharmaceutical company with three compounds--TRC-105, TRC-
102, and DE-122--currently being tested in clinical trials. While I
understand that you have divested yourself from NEA 14 pursuant to your
ethics obligations, will you recuse yourself personally and
substantially in any particular matter in which TRACON Pharmaceuticals,
Inc. is a party, including, but not limited to, proceedings concerning
TRC-105, TRC-102, and DE-122?
Answer 45. If confirmed, I will abide by all of the commitments set
forth in my ethics agreement and will comply with all applicable laws
and regulations. I will also be bound by the requirements and
restrictions set forth in this Administration's Ethics Pledge (EO
13770). If confirmed, I will meet with the career ethics officials at
the Department of Health and Human Services during the first week of my
service to ensure compliance with all of these ethics standards.
Throughout my public service I will consult with appropriate ethics
officials for guidance on these matters, including with respect to any
recusal obligations not already set forth in my ethics agreement. I am
committed to leading the FDA in an impartial manner that gives the
American public confidence in the integrity of the FDA's decisionmaking
process.
Question 46. In your OGE Form 278e, you disclose that you were an
investor in NEA 15 Limited Partnership (``NEA 15''). You further
disclose that NEA 15 holds an investment in Ardelyx, Inc., a
biopharmaceutical company with three compounds--Tenapanor, RDX-8940,
and RDX-7675--currently being tested in clinical trials. While I
understand that you have divested yourself from NEA 15 pursuant to your
ethics obligations, will you recuse yourself personally and
substantially in any particular matter in which Ardelyx, Inc. is a
party, including, but not limited to, proceedings concerning Tenapanor,
RDX-8940, and RDX-7675?
Answer 46. If confirmed, I will abide by all of the commitments set
forth in my ethics agreement and will comply with all applicable laws
and regulations. I will also be bound by the requirements and
restrictions set forth in this Administration's Ethics Pledge (EO
13770). If confirmed, I will meet with the career ethics officials at
the Department of Health and Human Services during the first week of my
service to ensure compliance with all of these ethics standards.
Throughout my public service I will consult with appropriate ethics
officials for guidance on these matters, including with respect to any
recusal obligations not already set forth in my ethics agreement. I am
committed to leading the FDA in an impartial manner that gives the
American public confidence in the integrity of the FDA's decisionmaking
process.
Question 47. In your OGE Form 278e, you disclose that you were an
investor in NEA 15 Limited Partnership (``NEA 15''). You further
disclose that NEA 15 holds an investment in Millendo Therapeutics,
Inc., a biopharmaceutical company with two compounds--ATR-101 and MLE-
4901--currently in clinical trials. While I understand that you have
divested yourself from NEA 15 pursuant to your ethics obligations, will
you recuse yourself personally and substantially in any particular
matter in which Millendo Therapeutics, Inc. is a party, including, but
not limited to, proceedings concerning ATR-101 and MLE-4901?
Answer 47. If confirmed, I will abide by all of the commitments set
forth in my ethics agreement and will comply with all applicable laws
and regulations. I will also be bound by the requirements and
restrictions set forth in this Administration's Ethics Pledge (EO
13770). If confirmed, I will meet with the career ethics officials at
the Department of Health and Human Services during the first week of my
service to ensure compliance with all of these ethics standards.
Throughout my public service I will consult with appropriate ethics
officials for guidance on these matters, including with respect to any
recusal obligations not already set forth in my ethics agreement. I am
committed to leading the FDA in an impartial manner that gives the
American public confidence in the integrity of the FDA's decisionmaking
process.
Question 48. In your OGE Form 278e, you disclose that you were an
investor in NEA 15 Limited Partnership (``NEA 15''). You further
disclose that NEA 15 holds an investment in ObsEva SA, Ltd., a
biopharmaceutical company with three compounds--OBE-2109, OBE-001, and
OBE-022--currently in clinical trials. While I understand that you have
divested yourself from NEA 15 pursuant to your ethics obligations, will
you recuse yourself personally and substantially in any particular
matter in which ObsEva SA, Ltd. is a party, including, but not limited
to, proceedings concerning OBE-2109, OBE-001, and OBE-022?
Answer 48. If confirmed, I will abide by all of the commitments set
forth in my ethics agreement and will comply with all applicable laws
and regulations. I will also be bound by the requirements and
restrictions set forth in this Administration's Ethics Pledge (EO
13770). If confirmed, I will meet with the career ethics officials at
the Department of Health and Human Services during the first week of my
service to ensure compliance with all of these ethics standards.
Throughout my public service I will consult with appropriate ethics
officials for guidance on these matters, including with respect to any
recusal obligations not already set forth in my ethics agreement. I am
committed to leading the FDA in an impartial manner that gives the
American public confidence in the integrity of the FDA's decisionmaking
process.
Question 49. In your OGE Form 278e, you disclose that you were an
investor in NEA 14 Limited Partnership (``NEA 14'') and NEA 15 Limited
Partnership (``NEA 15''). You further disclose that NEA 14 and NEA 15
hold investment in the following biopharmaceutical companies: Amplyx
Pharmaceuticals, Inc. (NEA 15); Cleave Biosciences, Inc. (NEA 14);
Clementia Pharmaceuticals Inc. (NEA 15); Envisia Therapeutics Inc. (NEA
14); Galera Therapeutics, Inc. (NEA 14); Lumos Pharma, Inc. (NEA 14);
Mersana Therapeutics, Inc. (NEA 14); Mirna Therapeutics, Inc. (NEA 14);
NightstaRx Ltd. (NEA 15); SetPoint Medical Corp. (NEA 15); Vtesse Inc.
(NEA 14); and Ziarco Group Ltd. (NEA 14). While I understand that you
have divested yourself from NEA 14 and NEA 15 pursuant to your ethics
obligations, will you recuse yourself personally and substantially in
any particular matter in which any of the companies listed above is a
party?
Answer 49. If confirmed, I will abide by all of the commitments set
forth in my ethics agreement and will comply with all applicable laws
and regulations. I will also be bound by the requirements and
restrictions set forth in this Administration's Ethics Pledge (EO
13770). If confirmed, I will meet with the career ethics officials at
the Department of Health and Human Services during the first week of my
service to ensure compliance with all of these ethics standards.
Throughout my public service I will consult with appropriate ethics
officials for guidance on these matters, including with respect to any
recusal obligations not already set forth in my ethics agreement. I am
committed to leading the FDA in an impartial manner that gives the
American public confidence in the integrity of the FDA's decisionmaking
process.
Question 50. In your OGE Form 278, you stated that managing
director of investment banking at T.R. Winston & Company was simply a
working title.\24\ It is unclear what that means and to what extent you
oversaw transactions specifically as part of the healthcare banking
team. Since you started in April 2013, T.R. Winston was involved in 12
large healthcare transactions involving 9 healthcare companies.\25\ You
have financial interests in five of those nine companies.\26\
Additionally, you have financial interests in two other T.R. Winston
healthcare client companies that were not involved in transactions
since the beginning of your tenure. Collectively, T.R. Winston's client
companies have at least 77 drug products in the products pipeline for
which they may seek approval from the FDA.
---------------------------------------------------------------------------
\24\ Executive Branch Personnel Public Disclosure Report (OGE Form
278e), Scott Gottlieb (U.S. Office of Government Ethics Certification
Mar. 28, 2017).
\25\ T.R. Winston & Company, Transactions (online at http://
www.trwinston.com/trans-
actions/) (accessed Apr. 7, 2017).
\26\ Letter from Scott Gottlieb, M.D., to Elizabeth J. Fischmann,
Esq., Associate General
Counsel for Ethics/Designated Agency Ethics Official, U.S. Department
of Health and Human Services (Mar. 28, 2017).
---------------------------------------------------------------------------
Did you oversee the 12 healthcare transactions as managing director
of investment banking? If not, what was your involvement in those
transactions? Will you recuse yourself for the 2 years laid out in the
Trump ethics pledge, from matters involving all of T.R. Winston's
clients? If not, why will you not take this extra step promised by the
President who nominated you?
You have committed to recusing yourself for 1 year from the date of
your resignation for matters involving T.R. Winston, Cell BioTherapy,
Tivorsan Pharmaceuticals, and Kure.
Will you recuse yourself for the 2 years laid out in the Trump
ethics pledge for those companies? If not, why will you not take this
extra step promised by the President who nominated you? Will you recuse
yourself from involvement in decisions that affect all of T.R.
Winston's investment banking healthcare clients?
Answer 50. As a general matter, my role at T.R. Winston was to
provide clinical and healthcare policy support to the transaction team.
I did not oversee transactions. If confirmed, I will abide by all of
the commitments set forth in my ethics agreement and will comply with
all applicable laws and regulations. I will also be bound by the
requirements and restrictions set forth in this Administration's Ethics
Pledge (EO 13770). If confirmed, I will meet with the career ethics
officials at the Department of Health and Human Services during the
first week of my service to ensure compliance with all of these ethics
standards. Throughout my public service I will consult with appropriate
ethics officials for guidance on these matters, including with respect
to any recusal obligations not already set forth in my ethics
agreement. I am committed to leading the FDA in an impartial manner
that gives the American public confidence in the integrity of the FDA's
decisionmaking process.
Question 51. In your ethics agreement, you noted that you do not
``hold any financial interest in T.R. Winston & Company;'' however, you
disclosed that you do hold a financial interest in 13 of T.R. Winston's
client companies--one of which is Inspyr Therapeutics, Inc. (previously
known as GenSpera).\27\ You have not committed to recuse yourself from
matters involving Inspyr. This company is currently developing a
platform technology to deliver its active ingredient to tumors in a way
that is less toxic to the body. Inspyr's lead investigational agent,
mipsagargin (G-202), is a prodrug in human clinical trials for patients
with hepatocellular carcinoma, glioblastoma, and prostate cancer.
Inspyr has four different products in ongoing or completed Phase II
clinical trials.\28\ The decision whether to approve these products may
occur during your tenure at FDA if you are confirmed.
---------------------------------------------------------------------------
\27\ Letter from Scott Gottlieb, M.D., to Elizabeth J. Fischmann,
Esq., Associate General
Counsel for Ethics/Designated Agency Ethics Official, U.S. Department
of Health and Human Services (Mar. 28, 2017).
\28\ Inspyr Therapeutics, Products Pipeline (online at http://
www.inspyrtx.com/product-pipeline) (accessed Apr. 7, 2017)
---------------------------------------------------------------------------
Though you will have divested your financial interest in the
company, do you think you can truly be impartial and independent as the
agency makes approval decisions for these products?
To avoid any appearance of impropriety, do you think it would be
better to recuse yourself from these decisions?
Answer 51. If confirmed I will undertake and perform the duties of
FDA commissioner impartially, as a passionate advocate for public
health and in the best interests of the American people, guided by the
science that informs the FDA's work. I will abide by all applicable
ethics laws and regulations, including those that govern recusals, and
am committed to performing my official duties in a manner that gives
the public confidence in the integrity of the FDA's decisionmaking
process.
Question 52. Neuralstem is a biotechnology company that specializes
in developing commercial-scale production of multiple types of central
nervous system stem cells. While you were managing director of T.R.
Winston's Investment Bank, T.R. Winston oversaw a $4,556,000 capital
markets transaction in September 2014 and a $20,000,000 capital markets
transaction in January 2014.\29\ Additionally, you have stated that you
hold financial interests in this company. According to your OGE Form
278, you have 22,308 warrants to purchase shares at $39 a share.\30\
You have not committed to recuse yourself from matters involving
Neuralstem. Neuralstem has at least six products in clinical trials
that will require FDA approval.\31\ For example, Neuralstem is
expecting to initiate a Phase II trial evaluating NSI-189 a treatment
for major depressive disorder (MDD). Do you think your previous
business and financial ties to the company have compromised your
ability to be impartial when approval decisions come before the agency?
---------------------------------------------------------------------------
\29\ T.R. Winston & Company, Transactions (online at http://
www.trwinston.com/trans-
actions/) (accessed Apr. 7, 2017).
\30\ Executive Branch Personnel Public Disclosure Report (OGE Form
278e), Scott Gottlieb (U.S. Office of Government Ethics Certification
Mar. 28, 2017).
\31\ NeuralStem, Cell Therapy Treatments in Development (online at
http://www.neural
stem.com/patient-info-treatments-in-development#celltherapy) (accessed
Apr. 7, 2017)
---------------------------------------------------------------------------
Answer 52. If confirmed I will undertake and perform the duties of
FDA commissioner impartially, as a passionate advocate for public
health and in the best interests of the American people, guided by the
science that informs the FDA's work. I will abide by all applicable
ethics laws and regulations, including those that govern recusals, and
am committed to performing my official duties in a manner that gives
the public confidence in the integrity of the FDA's decisionmaking
process.
Question 53. Celgene is a global biotechnology company that is
currently sponsoring more than 100 clinical trials examining on at
least 25 compounds that Celgene may seek FDA approval.\32\ Celgene is a
T.R. Winston client company, but your ethics agreement does not
indicate whether you will not be recused from matters involving
Celgene.\33\ Will you be recused from working on matters involving
Celgene?
---------------------------------------------------------------------------
\32\ Celgene, Product Pipeline (online at http://www.celgene.com/
content/uploads/product-pipeline.pdf) (accessed Apr. 7, 2017).
\33\ Letter from Scott Gottlieb, M.D., to Elizabeth J. Fischmann,
Esq., Associate General
Counsel for Ethics/Designated Agency Ethics Official, U.S. Department
of Health and Human Services (Mar. 28, 2017).
---------------------------------------------------------------------------
Answer 53. If confirmed, I will abide by all of the commitments set
forth in my ethics agreement and will comply with all applicable laws
and regulations. I will also be bound by the requirements and
restrictions set forth in this Administration's Ethics Pledge (EO
13770). If confirmed, I will meet with the career ethics officials at
the Department of Health and Human Services during the first week of my
service to ensure compliance with all of these ethics standards.
Throughout my public service I will consult with appropriate ethics
officials for guidance on these matters, including with respect to any
recusal obligations not already set forth in my ethics agreement. I am
committed to leading the FDA in an impartial manner that gives the
American public confidence in the integrity of the FDA's decisionmaking
process.
Question 54. Two T.R. Winston companies in which you have a
financial interest--Angion Biomedica Corp. and Emmaus Life Sciences--
have products in clinical trials. While I understand that you will
divest yourself from these two companies pursuant to your ethics
obligations, will you recuse yourself personally and substantially in
any particular matter in which any of the companies listed above is a
party?
Answer 54. If confirmed, I will abide by all of the commitments set
forth in my ethics agreement and will comply with all applicable laws
and regulations. I will also be bound by the requirements and
restrictions set forth in this Administration's Ethics Pledge (EO
13770). If confirmed, I will meet with the career ethics officials at
the Department of Health and Human Services during the first week of my
service to ensure compliance with all of these ethics standards.
Throughout my public service I will consult with appropriate ethics
officials for guidance on these matters, including with respect to any
recusal obligations not already set forth in my ethics agreement. I am
committed to leading the FDA in an impartial manner that gives the
American public confidence in the integrity of the FDA's decisionmaking
process.
Question 55. AMAG Pharmaceuticals--a T.R. Winston client company in
which you do not have a financial interest--has at least two products
in clinical trials. Your ethics agreement does not address whether you
will be recused from matters involving AMAG Pharmaceuticals. Will you
recuse yourself personally and substantially in any particular matter
in which AMAG Pharmaceuticals is a party?
Answer 55. If confirmed, I will abide by all of the commitments set
forth in my ethics agreement and will comply with all applicable laws
and regulations. I will also be bound by the requirements and
restrictions set forth in this Administration's Ethics Pledge (EO
13770). If confirmed, I will meet with the career ethics officials at
the Department of Health and Human Services during the first week of my
service to ensure compliance with all of these ethics standards.
Throughout my public service I will consult with appropriate ethics
officials for guidance on these matters, including with respect to any
recusal obligations not already set forth in my ethics agreement. I am
committed to leading the FDA in an impartial manner that gives the
American public confidence in the integrity of the FDA's decisionmaking
process.
Question 56. You have served as an independent member of the board
of directors for Gradalis, a position for which you earned
approximately $65,000 last year.\34\ In addition, you have stated that
you have a financial interest in the company--25,000 stock options to
purchase shares at $3.16 a share and 25,000 options at $3.57 a share.
You have noted that these are vested stock options and you intend to
divest within 90 days of your confirmation.\35\ Further you have noted
that you will recuse yourself from 1 year after your resignation, which
should expire March 2018 since you resigned your position in March
2017. Gradalis is a late-stage biopharmaceutical company developing a
platform technology which may have multiple cancer indications, and
currently have at least five products in clinical trials that may
require FDA approval.\36\ Do you think your 1-year recusal sufficiently
removes any bias posed by your investment and ties to this company?
---------------------------------------------------------------------------
\34\ Executive Branch Personnel Public Disclosure Report (OGE Form
278e), Scott Gottlieb (U.S. Office of Government Ethics Certification
Mar. 28, 2017).
\35\ Letter from Scott Gottlieb, M.D., to Elizabeth J. Fischmann,
Esq., Associate General
Counsel for Ethics/Designated Agency Ethics Official, U.S. Department
of Health and Human Services (Mar. 28, 2017).
\36\ Gradalis, Platform & Pipeline (online at http://
www.gradalisinc.com/index.php/pipeline/pipeline.html) (accessed Apr. 7,
2017).
---------------------------------------------------------------------------
Will you recuse yourself for the 2 years laid out in the Trump
ethics pledge for Gradalis? If not, why will you not take this extra
step promised by the President who nominated you?
Do you think it would be better and more likely to avoid conflicts
if your recusal were longer?
Answer 56. I have previously resigned from the Gradalis board and
surrendered my stock options. If confirmed, I will abide by all of the
commitments set forth in my ethics agreement and will comply with all
applicable laws and regulations. I will also be bound by the
requirements and restrictions set forth in this Administration's Ethics
Pledge (EO 13770). If confirmed, I will meet with the career ethics
officials at the Department of Health and Human Services during the
first week of my service to ensure compliance with all of these ethics
standards. Throughout my public service I will consult with appropriate
ethics officials for guidance on these matters, including with respect
to any recusal obligations not already set forth in my ethics
agreement. I am committed to leading the FDA in an impartial manner
that gives the American public confidence in the integrity of the FDA's
decisionmaking process.
Question 57. Daiichi Sankyo is a global pharmaceutical company; you
have served as an independent member of the board of directors since
April 2015 and you have agreed to resign upon your confirmation. You
have agreed to recuse yourself for 1 year after your resignation from
matters affecting Daiichi Sankyo, which has at least 28 products being
tested in clinical trials that may seek FDA approval.\37\
---------------------------------------------------------------------------
\37\ Daiichi-Sankyo, Pipeline Chart (Jan. 2017) (online at http://
www.daiichisankyo.com/rd/pipeline/development_pipeline/index.html).
---------------------------------------------------------------------------
Do you think your 1-year recusal sufficiently removes any bias
posed by your investment and ties to this company?
Will you recuse yourself for the 2 years laid out in the Trump
ethics pledge for Daiichi Sankyo? If not, why will you not take this
extra step promised by the President who nominated you?
Do you think it would be better and more likely to avoid conflicts
if your recusal were longer?
Answer 57. I have previously resigned from the Daiichi board of
directors. If confirmed, I will abide by all of the commitments set
forth in my ethics agreement and will comply with all applicable laws
and regulations. I will also be bound by the requirements and
restrictions set forth in this Administration's Ethics Pledge (EO
13770). If confirmed, I will meet with the career ethics officials at
the Department of Health and Human Services during the first week of my
service to ensure compliance with all of these ethics standards.
Throughout my public service I will consult with appropriate ethics
officials for guidance on these matters, including with respect to any
recusal obligations not already set forth in my ethics agreement. I am
committed to leading the FDA in an impartial manner that gives the
American public confidence in the integrity of the FDA's decisionmaking
process.
Question 58. GlaxoSmithKline is a global pharmaceutical company
that you have served as member of the Product Investment Board (PIB),
and you have agreed to resign upon your confirmation. You have agreed
to recuse yourself for 1 year after your resignation from matters
affecting GlaxoSmithKline, which has nearly 100 products being tested
in clinical trials, for which it may seek FDA approval.
Do you think your 1-year recusal sufficiently removes any bias
posed by your investment and ties to this company?
Will you recuse yourself for the 2 years laid out in the Trump
ethics pledge for GlaxoSmithKline? If not, why will you not take this
extra step promised by the President who nominated you?
Do you think it would be better and more likely to avoid conflicts
if your recusal were longer?
Answer 58. If confirmed, I will abide by all of the commitments set
forth in my ethics agreement and will comply with all applicable laws
and regulations. I will also be bound by the requirements and
restrictions set forth in this Administration's Ethics Pledge (EO
13770). If confirmed, I will meet with the career ethics officials at
the Department of Health and Human Services during the first week of my
service to ensure compliance with all of these ethics standards.
Throughout my public service I will consult with appropriate ethics
officials for guidance on these matters, including with respect to any
recusal obligations not already set forth in my ethics agreement. I am
committed to leading the FDA in an impartial manner that gives the
American public confidence in the integrity of the FDA's decisionmaking
process.
Question 59. In your ethics agreement, you explained that you
established Innovating Healthcare LLC for the purposes of a single
consulting project with Bristol-Myers Squibb. You have agreed to recuse
yourself for matters involving Bristol-Myers Squibb for 1 year from the
date you last provided service to the company. Bristol-Myers Squibb has
at least 30 products being tested in clinical trials, for which it may
seek FDA approval.
Do you think your 1-year recusal sufficiently removes any bias
posed by your investment and ties to this company?
Will you recuse yourself for the 2 years laid out in the Trump
ethics pledge for Bristol-Myers Squibb? If not, why will you not take
this extra step promised by the President who nominated you?
Do you think it would be better and more likely to avoid conflicts
if your recusal were longer?
Answer 59. If confirmed, I will abide by all of the commitments set
forth in my ethics agreement and will comply with all applicable laws
and regulations. I will also be bound by the requirements and
restrictions set forth in this Administration's Ethics Pledge (EO
13770). If confirmed, I will meet with the career ethics officials at
the Department of Health and Human Services during the first week of my
service to ensure compliance with all of these ethics standards.
Throughout my public service I will consult with appropriate ethics
officials for guidance on these matters, including with respect to any
recusal obligations not already set forth in my ethics agreement. I am
committed to leading the FDA in an impartial manner that gives the
American public confidence in the integrity of the FDA's decisionmaking
process.
Question 60. You served as member of the board of director of
Tolero Pharmaceuticals until December 2016. You have agreed to recuse
yourself for 1 year after your resignation from matters affecting
Tolero Pharmaceuticals, which has two products being tested in clinical
trials, for which it may seek FDA approval.
Do you think your 1-year recusal sufficiently removes any bias
posed by your investment and ties to this company?
Will you recuse yourself for the 2 years laid out in the Trump
ethics pledge for Tolero Pharmaceuticals? If not, why will you not take
this extra step promised by the President who nominated you?
Do you think it would be better and more likely to avoid conflicts
if your recusal were longer?
Answer 60. If confirmed, I will abide by all of the commitments set
forth in my ethics agreement and will comply with all applicable laws
and regulations. I will also be bound by the requirements and
restrictions set forth in this Administration's Ethics Pledge (EO
13770). If confirmed, I will meet with the career ethics officials at
the Department of Health and Human Services during the first week of my
service to ensure compliance with all of these ethics standards.
Throughout my public service I will consult with appropriate ethics
officials for guidance on these matters, including with respect to any
recusal obligations not already set forth in my ethics agreement. I am
committed to leading the FDA in an impartial manner that gives the
American public confidence in the integrity of the FDA's decisionmaking
process.
Question 61. In your ethics agreement, you explained that you
provided consulting services for Vertex Pharmaceuticals through
YourEncore. You have agreed to recuse yourself for matters involving
Vertex Pharmaceuticals for 1 year from the date you last provided
service to the company. Vertex Pharmaceuticals has at least seven
products being tested in clinical trials, for which it may seek FDA
approval.
Do you think your 1-year recusal sufficiently removes any bias
posed by your investment and ties to this company?
Will you recuse yourself for the 2 years laid out in the Trump
ethics pledge for Vertex Pharmaceuticals? If not, why will you not take
this extra step promised by the President who nominated you?
Do you think it would be better and more likely to avoid conflicts
if your recusal were longer?
Answer 61. If confirmed, I will abide by all of the commitments set
forth in my ethics agreement and will comply with all applicable laws
and regulations. I will also be bound by the requirements and
restrictions set forth in this Administration's Ethics Pledge (EO
13770). If confirmed, I will meet with the career ethics officials at
the Department of Health and Human Services during the first week of my
service to ensure compliance with all of these ethics standards.
Throughout my public service I will consult with appropriate ethics
officials for guidance on these matters, including with respect to any
recusal obligations not already set forth in my ethics agreement. I am
committed to leading the FDA in an impartial manner that gives the
American public confidence in the integrity of the FDA's decisionmaking
process.
Question 62. The FDA Commissioner will need to be deeply engaged
when we face another public health crisis. Given the scope of your
financial entanglements, I am concerned that you may be unable to
effectively lead the agency in this scenario. You are, unfortunately,
familiar with being recused when FDA responds to an emerging threat.
When you were the agency's deputy commissioner in 2005, you could not
participate in many decisions about how to combat the avian flu because
you had worked for two drug companies--Roche and Sanofi--who worked to
develop products to fight back against the deadly flu.\38\ Now, in
order to comply with Federal conflict-of-interest and ethics laws, you
have committed to recuse yourself from decisions related to more than
20 companies where you hold, or previously held, positions. While this
represents a small fraction of the medical product companies in which
you have a financial stake, this includes seven pharmaceutical
companies and two clinical lab companies. Of particular concern is your
recusal from matters involving GlaxoSmithKline. GSK is one of the top
vaccine makers in the world, and they have been involved in developing
vaccines in response to the Ebola and Zika outbreaks.\39\ You received
hundreds of thousands of dollars in compensation from the company in
the last several years.\40\ It seems possible--if not likely--that they
would be involved in a response to the next disease outbreak. Any of
these 20 companies could be called on to assist in the response to a
public health crisis. Would the American people be better served in a
time of crisis by an FDA Commissioner who was not recused from matters
related to such a large number of companies and whose focus was
guaranteed to be on the crisis and not on the sidelines?
---------------------------------------------------------------------------
\38\ http://archive.boston.com/business/healthcare/articles/2005/
11/12/fda_official_recused
_in_flu_fight/.
\39\ https://www.bloomberg.com/news/articles/2016-09-08/glaxo-
proposes-global-body-to-
tackle-outbreaks-like-zika-ebola.
\40\ https://www.wired.com/2017/03/trumps-fda-pick-friends-big-
pharma-doesnt/.
---------------------------------------------------------------------------
Answer 62. I was compensated by GSK, for my services on the product
investment board, through a retainer in the amount of $60,000 annually.
I was also reimbursed for out-of-pocket travel costs to attend
meetings. If confirmed, I intend to lead the FDA as an impartial and
passionate advocate for the public health and am confident that I can
fully perform the duties of the FDA commissioner. I do not believe that
the recusals set forth in my ethics agreement will impair my ability to
discharge the responsibilities of this office. All nominees come to
their positions with a range of experiences, which necessitate some
recusals to ensure compliance with relevant ethics standards. It is
routine in those circumstances for other senior agency officials to be
involved in the matter and I have every confidence that if I am
recused, my team at FDA will ensure that Congress's laws will be
properly implemented and the FDA's mission fulfilled.
senator enzi
Question 1. FDA's menu-labeling rule, even after an initial stay,
will take effect 1 month from today. Grocery stores and other food
retailers across America continue to be frustrated with FDA's handling
of things, including for local and seasonal food items. Fresh and local
food items may be sold at a few stores, under the same name, but the
ingredients or recipe can vary, yet they would be considered ``standard
menu items'' and subject to enforcement. The irony is that this will
cause stores and restaurants to move away from fresh, local, and
seasonal offerings. With just a month before the compliance date, we
need FDA to act quickly to further delay, withdraw, or stay the rule so
it can be rewritten to give businesses the flexibility to comply. Would
you be willing to explore ways to encourage FDA to act before the
compliance date to provide this much-needed flexibility for businesses?
Answer 1. While I am broadly aware of the menu labeling issue, this
is not a matter on which I am familiar with the technical specifics. As
a general rule, I support providing clear, accurate, and understandable
information to American consumers to help inform healthful dietary
choices. I believe information about caloric content can be a useful
tool. However, I am mindful of the unique challenges that developing
and communicating such information can pose, particularly on small,
independent businesses. If confirmed, I will commit to working with the
agency's staff to quickly get up to speed on the regulatory history
related to menu labeling, as well as FDA's latest thinking and actions.
I would welcome the opportunity to work with Congress and stakeholders
to ensure any regulatory requirements would promote public health by
providing helpful information to consumers, while not placing
unnecessary compliance burden on businesses, particularly small,
independent ones.
senator sanders
Question 1. The FDA's mission statement includes the directive to
make medicines ``more effective, safer, and more affordable.'' Setting
aside the ongoing debate over generic drug approvals, please discuss
how you think FDA can make medicines more affordable.
Answer 1. While drug pricing does not fall directly within FDA's
purview, I believe the agency can play an important role on this
important issue by taking steps to improve product competition. If
confirmed, I will work to ensure FDA has the appropriate policies and
processes in place to effectively facilitate product competition,
especially for complex drugs that sometimes do not face effective
generic competition even long after the patent expires.
Reforming the regulatory pathway for complex generic products would
address one key policy deficiency that results in unnecessary barriers
to the development and review of generic competitors for some innovator
products for which traditional bioequivalence and bioavailability
testing alone are sometimes insufficient for proving sameness. FDA
should also explore options to improve the efficiency and consistency
of ANDA review processes and timelines, so that financial speculators
cannot engage in a regulatory arbitrage, by dramatically hiking the
price of some very old generic drugs because they know it can take
years for new generic competitors to enter the market.
Question 2. It has been reported that you have received millions of
dollars from pharmaceutical companies. Please explain how you would
address your myriad conflicts of interest.
Do you believe you can be an effective Commissioner when you will
need to recuse yourself so often? Do you think there is a tipping point
where the conflicts are too great to overcome?
Answer 2. If confirmed, I intend to lead the FDA as an impartial
and passionate advocate for the public health and am confident that I
can fully perform the duties of the FDA commissioner. I will abide by
all of the commitments set forth in my ethics agreement and will comply
with all applicable laws and regulations. I will also be bound by the
requirements and restrictions set forth in this Administration's Ethics
Pledge (EO 13770). If confirmed, I will meet with the career ethics
officials at the Department of Health and Human Services during the
first week of my service to ensure compliance with all of these ethics
standards. Throughout my public service I will consult with appropriate
ethics officials for guidance on these matters, including with respect
to any recusal obligations not already set forth in my ethics
agreement.
I am committed to leading the FDA in an impartial manner that gives
the American public confidence in the integrity of the FDA's
decisionmaking process. I do not believe that the recusals set forth in
my ethics agreement will impair my ability to discharge the
responsibilities of this office. All nominees come to their positions
with a range of experiences, which necessitate some recusals to ensure
compliance with relevant ethics standards. It is routine in those
circumstances for other senior agency officials to be involved in the
matter and I have every confidence that, if I am recused, my team at
FDA will ensure that Congress's laws will be properly implemented and
the FDA's mission fulfilled.
Question 3. During our meeting in my office, you stated that the
Nation's opioid crisis would be among your top priorities. As I am sure
you know, it has hit my State of Vermont very hard. You also noted that
the FDA took incremental actions that never managed to get ahead of the
opioid crisis.
What role do you think FDA should play today in more aggressively
addressing the opioid epidemic while also ensuring that Americans
living with both acute and chronic pain do not suffer from poor pain
management?
How do we balance the need for patient access to effective pain
relief medications while also preventing opioid addiction?
Answer 3. Opioid abuse, misuse, and addiction constitute one of the
most urgent and immediate public health threats facing our Nation. It
is also the biggest public health crisis facing the FDA. The human and
economic toll of this crisis is staggering. If confirmed, this will be
my highest immediate priority. I will make sure FDA is aggressive,
forward leaning, and fully engaged in combating this epidemic. I will
work with FDA's staff to ensure FDA has the right policies and
processes in place to:
Facilitate the developments of new approaches and
technologies to reduce the abuse/addictive potential of painkillers
American patients use;
Support the development of non-opioid analgesic
alternatives for physicians and patients;
Assess whether FDA's current approach to opioid regulatory
decisions, including labeling, REMS, and physician/patient education
are appropriate, robust, and fully effective;
Encourage the development of new pharmacological tools for
physicians and patients to both prevent opioid misuse and abuse, and
support treatment and recovery for patients struggling to overcome
opioid addiction;
Enhance physician and patient educational materials to
strengthen public awareness of the risks of opioids, as well as the
FDA-approved resources available to them, using the full range of FDA's
risk communication tools to better target this information;
Taking steps to make sure that providers are appropriately
educated on identifying, and helping to property intervene with, abuse-
prone patients;
Re-assess whether FDA has the appropriate framework and
authorities for evaluating the risk of abuse and diversion as a
component of its review and approval process for opioids;
Undertake a comprehensive effort to evaluate the full
scope of the sources and threats from foreign-imported narcotics;
Evaluate whether FDA should bring more alignment between
the review and approval of different medical product platforms used in
the treatment of pain to make sure the agency is adopting the best
public health standard in assessing these products; and
Collaborate effectively with other government agencies and
external stakeholders to develop and execute comprehensive and
effective strategies to win the battle against opioid abuse, misuse,
and addiction. This includes steps for FDA to more closely collaborate
and coordinate with DEA on the two agencies shared goals.
Question 4. Tobacco use is the leading cause of preventable death
and disability in the United States. Cigarette smoking claims nearly
half a million lives every year and more than 8.5 million people suffer
from tobacco-related chronic diseases. In 2006, you recognized that
cigarette smoking is ``the single most preventable cause of death in
the United States and is responsible for a growing list of cancers, as
well as chronic diseases.'' But since then, you have made disturbing
comments about where you stand on tobacco regulation. In 2009, you
opposed FDA regulating tobacco because you said it would ``gut the
agency's resources and distract it from our core mission.''
Why should we confirm someone to be the head of the FDA who does
not believe the FDA should regulate tobacco?
Could you or any of the firms you have worked for financially
benefit if the FDA weakens or eliminates regulations on tobacco or
nicotine products?
Answer 4. Through the Tobacco Control Act (TCA), Congress gave FDA
regulatory responsibility over tobacco products. If confirmed, I will
be committed to implementing the TCA, as intended by Congress. As I was
not at FDA during the agency's initial TCA implementation activities, I
am not fully acquainted with internal processes or specific decisions
to-date. If confirmed, I will work with staff to quickly get up-to-
speed on this issue, and I will review current FDA policies, to ensure
FDA treats products appropriately, implements provisions in a timely
fashion, and in a manner that is consistent with congressional intent
under the TCA. I believe responsibly implementing the TCA is an
integral part of FDA's core mission to protect and promote public
health. In pursuing these objectives, I will be guided only by the
public health and the mandate of Congress.
Question 5. While the law bans companies from using flavors like
cherry, vanilla and cinnamon in cigarettes, there was one exemption
from that list of flavors: Menthol. Today, nearly one in every three
cigarettes sold in the United States are menthol cigarettes and there
are an estimated 20 million people who smoke menthol cigarettes.
Tobacco companies have long used aggressive marketing of menthol
cigarettes to target our Nation's most vulnerable populations: Young
people, women, LGBTQ populations and people of color. It is not a
surprise that nearly 50 percent of all teenagers--many of whom are in
middle school--who are addicted to cigarette smoking started by smoking
menthol cigarettes because the minty flavor tastes better to first-time
smokers.
Women are 1.6 times more likely than men to smoke menthol
cigarettes. Additionally, 8 in 10 African Americans, more than half (53
percent) of Native Hawaiian and Pacific Islanders, and one-third of
Latinos and Asians who smoke choose menthol cigarettes.
One of the things that the 2009 law did was give the FDA the
authority to ban menthol cigarettes. The 2011 TPSAC report concluded
that the ``removal of menthol cigarettes from the marketplace would
benefit public health in the United States.'' A separate study
conducted by the FDA in 2011 and reported out in 2013 found that
``menthol use is likely associated with smoking initiation by youth and
young adults.'' This same report also found that ``menthol in
cigarettes is likely associated with greater addiction'' and that
menthol cigarettes post a greater risk to public health than non-
menthol cigarettes.
Following these report findings, cigarette companies sued the FDA
in 2011 in an attempt to prevent the FDA from acting on the TPSAC
findings and recommendations. A tobacco-sympathizing judge (Judge Leon)
initially directed the FDA to overlook and disregard the TPSAC report
and findings. The FDA appealed the ruling and in January 2016, Judge
Leon's decision was reversed, opening the door for the FDA to take
action to regulate and even ban menthol cigarettes. Yet, menthol
cigarettes remain on the shelves of stores across the country today.
Given all that we know about menthol cigarettes--none of which is
good--do you support banning menthol cigarettes from the U.S. market
and, if so, would banning menthol cigarettes be an action you would
take during your first year as Commissioner?
Answer 5. I have not reviewed the scientific evidence related to
the addition of menthol in cigarettes. If confirmed, I will commit to
engaging with FDA's staff to quickly get up to speed on the regulatory
history of this issue, and the agency's latest information, thinking,
and actions. I would welcome the opportunity to work with Congress on
this issue moving forward.
Question 6. Marijuana is currently listed as a Schedule I substance
(``drugs with no currently accepted medical use and a high potential
for abuse'') under the Controlled Substances Act (CSA)--meaning under
Federal law, marijuana is considered to be as dangerous as heroin, and
more dangerous than opioids. The Drug Enforcement Agency could
potentially take action to reschedule marijuana, pending evaluation
from the FDA. If marijuana were rescheduled, the FDA would likely wind
up with the authority to regulate marijuana and marijuana-derived
drugs.
Do you believe that marijuana is properly classified under the CSA?
Do you think FDA should have the authority to regulate marijuana?
Answer 6. I am aware that in July 2016, DEA determined, in
consultation with HHS, that marijuana continues to meet the criteria
for Schedule I control under the Controlled Substances Act. I cannot
speak to decisions that the DEA might take in the future or the
likelihood of FDA regulating marijuana. I do know that FDA is currently
involved in supporting scientific research related to medicinal uses of
marijuana and its constituents.
senator burr
Question 1. The 21st Century Cures Act requires FDA to update
guidance and regulations for regenerative therapeutic products and to
hold a public meeting to encourage innovation. This is a great first
step, and we are hopeful that you will prioritize the potential of
these products as the new Commissioner of the FDA. If you are
confirmed, how do you envision FDA's regulatory framework for these
cutting edge treatments and therapies now and in the future?
Answer 1. Regenerative medicine is one of the most innovative and
promising emerging advancements in our scientific approaches to the
treatment of human disease. Regenerative medicine appears to hold great
promise for new therapeutic options for patients and physicians,
particularly in areas of unmet or underserved medical need. However, as
with all products FDA regulates, the agency must have the appropriate
policies and processes in place to assess and ensure the safety and
efficacy of regenerative medical products before they are approved for
use by American patients. FDA must ensure patients and providers are
appropriately educated about the potential risks and benefits of
regenerative medicine therapies that fall within the scope of FDA's
oversight, and that these products meet the agency's standard for
safety and effectiveness. If confirmed, I will embrace the
responsibility to facilitate important medical innovation in the
regenerative medicine space, while maintaining the Gold Standard of
safety and efficacy.
Question 2. The Tobacco Control Act gave FDA the authority to
regulate tobacco products. The tobacco industry has seen an evolution
in the products available to consumers, and the FDA has deemed new and
novel technologies to be regulated the same way as traditional
combustible products. These products vary based on risk, and because of
these new technologies, consumers have the opportunity to choose a less
harmful product. If you are confirmed, how do you envision new
technologies being reviewed by the Center for Tobacco Products at the
FDA?
Answer 2. If confirmed, I will be committed to implementing the
TCA, as intended by Congress, including Section 911 related to modified
risk products, which I recognize can provide helpful tools for current
tobacco-users to transition off combustible tobacco. As I was not at
FDA during the agency's initial TCA implementation activities, I am not
fully acquainted with internal processes or specific decisions to-date.
If confirmed, I will work with staff to quickly get up-to-speed on this
issue, and I will review current FDA policies, including the deeming
rule, to ensure FDA treats products appropriately, implements
provisions in a timely fashion, and in a manner that is consistent with
congressional intent under the TCA. I believe responsibly implementing
the TCA is an integral part of FDA's core mission to protect and
promote public health.
senator casey
Question 1. The FDA plays an important role in responding to real
and potential biological threats. Under the Pandemic and All-Hazards
Preparedness Reauthorization Act of 2013 (PAHPRA), the FDA has
important authorities such as the Emergency Use Authorization and the
so-called ``Animal Rule'' regulation that outlines how FDA will
consider animal data when it would be impossible or unethical to test a
medical countermeasure. Another important consideration is the need for
robust medical countermeasure development. Instead of a piecemeal
approach that tackles one threat at a time--the so-called ``one bug,
one drug'' approach--there is great potential to develop products so
that one product can tackle multiple threats. Will you commit to
considering the potential scale and scope of potential medical
countermeasures during the FDA review process?
Answer 1. I recognize the important role FDA plays in supporting
the development of medical countermeasures. I commit to considering the
potential scale and scope of potential medical countermeasures during
the FDA review process.
Question 2. Back in 2009, you wrote quite extensively about the
Federal Government's response to the H1N1 pandemic flu, \41\ both
praising the speed of the Federal Government's decision to order
vaccine and criticizing the speed at which the vaccines were produced
and distributed. Since then, we have made significant improvements
toward greater preparedness for influenza, including bringing new
vaccine production capacity online and approving a new quadrivalent
vaccine that protects against four strains of flu.
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\41\ American Enterprise Institute Health Policy Outlook, November
2009, and the Wall Street Journal, https://www.wsj.com/articles/
SB10001424052748704335904574497324151841690.
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Do you agree that vaccines are an important part of our public
health preparedness, and that we must continue to develop, produce and
distribute vaccines (such as, but not limited to, flu vaccine) to
improve the Nation's public health, preparedness and response
capabilities for emergencies?
Do you think we are better prepared to approve and make available
vaccines for vaccine-preventable illnesses? What further steps would
you take as Commissioner to promote preparedness?
Answer 2. Yes, I strongly agree that vaccines are an important
component of our public health preparedness. I believe that FDA has
taken significant steps to support the development and approval of new
vaccines. If confirmed, I will commit to ensuring that FDA has the
appropriate policies and processes in place to protect and promote
public health and working with FDA's partners to pursue a comprehensive
strategy to increase our public health preparedness.
Question 3. Dr. Gottlieb, during our meeting you raised the
possibility of steps that the FDA can take to increase what you termed
``product competition,'' or the availability of generics. Yet of the
roughly 2,000 Abbreviated New Drug Applications pending as a part of
the generic backlog, only 15 are for first-in-class generics or for
drugs that currently have a single manufacturer on the market.
I am not sure I understand how adding the tenth or twelfth generic
of a common drug will address the high costs facing patients with
branded drugs. What other steps would you take as Commissioner to
increase generic competition?
Additionally, 8 of the 10 drugs that experienced the greatest drug
price increases in 2014 were generic medications, with multiple
manufacturers. What will you do to address these price hikes?
Answer 3. While drug pricing does not fall directly within FDA's
purview, I believe the agency can play an important role on this
important issue by taking steps to improve product competition.
If confirmed, I will work to ensure FDA has the appropriate
policies and processes in place to effectively facilitate generic
market entry and competition, especially for complex drugs that
sometimes do not face effective generic competition even long after the
patent expires. Reforming the regulatory pathway for complex generic
products would address one key policy deficiency that results in
unnecessary barriers to the development and review of generic
competitors for some innovator products for which traditional
bioequivalence and bioavailability testing alone are sometimes
insufficient for proving sameness. FDA should also explore options to
improve the efficiency and consistency of ANDA review processes and
timelines, so that financial speculators cannot engage in a regulatory
arbitrage, by dramatically hiking the price of some very old generic
drugs because they know it can take years for new generic competitors
to enter the market.
Question 4. If confirmed as FDA Commissioner, what strategies do
you support to share meaningful data on the safety of medical devices
with the public, so that doctors and patients can make informed
decisions?
Answer 4. I am a strong proponent of greater data transparency--for
patients, physicians, and manufacturers. If confirmed, I will be
committed to working with Congress, patients, industry, and
stakeholders on the issue of data transparency and other ways FDA could
potentially make important information available to the public.
Question 5. You have been a proponent of loosened restrictions on
direct-to-consumer advertising of prescription drugs. If confirmed,
what principles will guide you in revising industry guidance on direct-
to-consumer advertising of prescription drugs to assure that patients
have accurate information on the safety and efficacy?
Answer 5. I believe that providing consumers and providers with
truthful, non-misleading, science-based information can promote public
health by empowering Americans to make informed decisions about their
health. This is especially true when it comes to truthful, non-
misleading clinical information provided in a non-promotional context,
which is the context for many of my prior statements on this topic. If
confirmed, I will work with FDA staff to review FDA's current policies
related to these issues. I would approach any policy considerations
related to the sharing of truthful, non-misleading information mindful
of the existing statute and regulations, and FDA's mission to protect
and promote public health.
Question 6. In April 2015, the FDA published Guidance for Industry
on abuse-
deterrent opioids, which stated that the Agency considers the
development of opioids that are formulated to deter abuse as a ``high
public health priority.'' In its 2016 action plan on opioids and in a
fact sheet posted on Monday January 9, 2017, the FDA stated its strong
support for transitioning from the use of non-abuse-deterrent opioids
to opioids with meaningful abuse-deterrent formulations and stated,
``The FDA looks forward to a future in which most or all
opioid medications are available in formulations that are less
susceptible to abuse than the formulations that lack abuse-
deterrent properties.''
What further actions can and should the FDA take to transition the
market to one in which patients receive abuse-deterrent opioids?
Answer 6. Opioid abuse, misuse, and addiction constitute one of the
most urgent and immediate public health threats facing our Nation. It
is also the biggest public health crisis facing the FDA. The human and
economic toll of this crisis is staggering. If confirmed, this will be
my highest immediate priority. I will make sure FDA is aggressive,
forward leaning, and fully engaged in combating this epidemic. I will
work with FDA's staff to ensure FDA has the right policies and
processes in place to:
Facilitate the developments of new approaches and
technologies to reduce the abuse/addictive potential of painkillers
American patients use;
Support the development of non-opioid analgesic
alternatives for physicians and patients;
Assess whether FDA's current approach to opioid regulatory
decisions, including labeling, REMS, and physician/patient education
are appropriate, robust, and fully effective;
Encourage the development of new pharmacological tools for
physicians and patients to both prevent opioid misuse and abuse, and
support treatment and recovery for patients struggling to overcome
opioid addiction;
Enhance physician and patient educational materials to
strengthen public awareness of the risks of opioids, as well as the
FDA-approved resources available to them, using the full range of FDA's
risk communication tools to better target this information;
Taking steps to make sure that providers are appropriately
educated on identifying, and helping to property intervene with, abuse-
prone patients;
Re-assess whether FDA has the appropriate framework and
authorities for evaluating the risk of abuse and diversion as a
component of its review and approval process for opioids;
Undertake a comprehensive effort to evaluate the full
scope of the sources and threats from foreign imported narcotics;
Evaluate whether FDA should bring more alignment between
the review and approval of different medical product platforms used in
the treatment of pain to make sure the agency is adopting the best
public health standard in assessing these products; and
Collaborate effectively with other government agencies and
external stakeholders to develop and execute comprehensive and
effective strategies to win the battle against opioid abuse, misuse,
and addiction. This includes steps for FDA to more closely collaborate
and coordinate with DEA on the two agencies' shared goals.
Question 7. The Food and Drug Administration has moved forward with
Food Safety Modernization Act implementation by finalizing seven rules
required under the act. However, there are several required rules that
remain to be addressed, including enhanced recordkeeping requirements
for high-risk foods, in-store consumer notification of reportable foods
and accreditation standards for food laboratories. There are also
multiple guidance documents meant to help producers and food processors
comply with FSMA that have yet to be developed.
Will you commit to developing these important rules and guidance
documents by the end of 2018?
Answer 7. The Food Safety Modernization Act (FSMA) provides FDA
with important tools and authorities to support its responsibility to
ensure the safety of our Nation's food supply. If confirmed, I will
work to ensure the agency has the appropriate policies, processes, and
resources in place to implement FSMA, as intended by Congress. I am
committed to timely implementation of the provisions of FSMA,
consistent with congressional intent. FDA should implement FSMA in a
way that protects and promotes public health by enhancing food safety,
while also collaborating with the U.S. Department of Agriculture, State
officials, and other government agencies to conduct regulatory
activities in a manner that takes into account the unique challenges
faced by small farmers and small businesses.
Question 8. The Food and Drug Administration is responsible for the
safety of about 80 percent of the U.S. food supply. The Food Safety
Modernization Act enhanced FDA's ability to do more to prevent
foodborne illness, rather than responding to foodborne illness
outbreaks. This goal will only be realized if FDA is given the funding
necessary to help train growers and food processors, update IT
infrastructure and develop its own staff of skilled professionals. Will
you commit to advocating for the funding necessary to successfully
implement FSMA and protect our Nation's food supply?
Answer 8. The Food Safety Modernization Act (FSMA) provides FDA
with important tools and authorities to support its responsibility to
ensure the safety of our Nation's food supply. If confirmed, I will
work to ensure FDA has the appropriate policies, processes, and
resources in place to implement FSMA, as intended by Congress.
Question 9. According to the U.S. Department of Agriculture
National Agriculture Statistics Service, PA is home to 17,000 bee
colonies, producing 901,000 lbs. of honey for a value of $3.26 million
in 2016. Countries like China continue to smuggle potentially unsafe
honey into the United States, which has a serious impact on jobs and
economic growth in Pennsylvania. Will you work to establish a Federal
standard of identity for honey to ensure that consumers and producers
are protected from substandard or falsely labeled honey?
Answer 9. This is not an issue that I have previously considered,
but I will look forward to working with you to better understand and
advance policy in this area, if I am confirmed. I have always been a
proponent of accurate labeling.
Question 10. In May 2016, FDA published its final Nutrition and
Supplement Facts Label rule to update the nutrient fact label. This
rule required the declaration of the amount of added sugar in a
product. While I support the declaration of added sugars so that
consumers can make healthy choices for themselves and their family, I
am concerned that this designation could cause confusion for consumers
with regards to products like a jar of honey or maple syrup when the
sugar content is naturally occurring.
According to FDA's final rule on the nutrient fact label, a jar of
honey would be required to label the sugar content in the honey as an
``Added Sugar,'' rather than solely the ``Total Sugars'' in the
product. An ``Added Sugar'' declaration for a jar of honey or maple
syrup could imply to consumers that the sugar in honey is added, rather
than naturally occurring. This could be misleading for consumers and
not accurately convey the sugar content of the product.
When crafting the final guidance for industry on added sugars will
you provide clarity to the honey and maple syrup industry on the
labeling of the sugar content of their packaged product (ex. a jar of
honey)?
How do you believe that the sugar content of a jar of honey or
maple syrup should be declared? Should packaged honey and maple syrup
be subject to the ``Total Sugars'' declaration, rather than the ``Added
Sugars'' declaration?
Answer 10. This is not a discussion that I have been privy to
although I am familiar with the Nutrition and Supplement Facts Label
rule. I believe in transparency for consumers, and that information
must be understandable and clear and science-based. If confirmed, I
commit to looking into this issue related to the labeling of sugars in
honey and maple syrup.
Question 11. Nutrition during pregnancy is critical for the health
of the mother and developing fetus. It is vital that pregnant and
nursing women have access to nutrition information that is rooted in
science to ensure that they can make healthy decisions for their
family. In 2014, FDA and EPA issued draft-updated advice about seafood
consumption for pregnant and nursing women. That advice recommended
that pregnant women, nursing women, women who may become pregnant and
young children should eat more fish that is lower in mercury in order
to benefit from important nutrients that can aid growth and
development. These recommendations were consistent with the 2010
Dietary Guidelines for Americans. In January 2017, FDA and EPA
published revised advice for seafood consumption for these target
groups of women and children.
Will you ensure that future nutrition guidance related to seafood
consumption for pregnant women, nursing women, women who may become
pregnant and young children be based on the most current and relevant
nutrition science?
How do you intend to work with EPA to accomplish this?
Answer 11. If confirmed, I will ensure FDA's advice concerning
seafood consumption by pregnant and nursing women is based on the most
current and relevant nutritional science and appropriately takes into
account both the nutritional benefits, and any toxicological risks
associated with seafood consumption. I will also work to ensure
effective collaboration between FDA and the U.S. Environmental
Protection Agency (EPA) on this issue, and a range of other public
health matters over which both agencies share regulatory authority.
senator isakson
Question 1. A GAO report in September 2015, requested by this
committee, noted that it is illegal under current law to compound
animal drugs using bulk active ingredients. While the report noted that
a limited amount of compounding from bulk is needed to meet unmet
medical needs, some pharmacies are endangering animal health by
producing large quantities of near copies of approved animal drugs and
mass marketing them essentially acting as manufacturers while skirting
the safety and efficacy protections of the FDA approval process.
The GAO strongly urged the Agency to provide clear final guidance
outlining conditions under which FDA will generally not take
enforcement actions for violations of specific provisions of the Food,
Drug and Cosmetic Act with respect to animal drug compounding. FDA
released draft guidance to this effect in 2015 and the comment period
for that draft ended in November 2015. When will the agency move to
protect animal health and issue this final guidance for animal drug
compounding?
Answer 1. Ensuring the safety of compounded animal drugs is an
important part of FDA's mission to protect and promote public health.
If confirmed, I will commit to engaging with FDA's staff to quickly get
up to speed on the regulatory history of this issue, and the agency's
latest information, thinking, and actions. I would welcome the
opportunity to work with Congress on this issue moving forward to make
sure the agency is taking timely steps on this matter and implementing
the laws and regulations consistent with the intent of Congress.
Question 2. Dr. Gottlieb, as you know, the majority of OTC
medicines are regulated under the OTC Monograph system, which is built
around notice and comment rulemaking. These burdensome regulations make
changes, such as label warnings or new dosage forms, cumbersome and
slow. Shifting these decisions to the drug center within FDA will add
efficiency to scientific and medical determinations by removing
decisionmaking layers, while continuing to include due process
controls. Monograph reform will also allow for greater innovation and
offerings for consumers. It is also my hope that this will address the
15-year backlog sunscreen ingredient applications, as well as currently
marketed products in the stayed final monograph.
Will you commit to working with Senator Casey and myself on
Monograph reform?
Answer 2. OTC products play an important role in our Nation's
healthcare system. I believe that the current monograph system for
regulating OTC medicines should be evaluated to determine whether
certain improvements may benefit public health. I am aware of some of
the current proposals for improving on the monograph process. If
confirmed, I will commit to working with you, Senator Casey, and
members of your staffs on this important issue.
Question 3. I am interested in acceleration of translating
scientific research into more effective treatments for cancer and other
diseases. The emergence of several notable efforts to conduct
observational research of cancer patients and collect large amounts of
data--including clinical outcomes, demographic information, and
molecular profiles--to create ``learning systems'' to accelerate
research and improve the quality of care.
A notable example of this is a partnership called the Oncology
Research Information Exchange Network--or ORIEN for short. ORIEN is a
national partnership in which 15 of the Nation's top cancer centers
have agreed to use the same protocol to enroll patients and collect and
share data on patient outcomes and molecular profiles. As I understand
it ORIEN is the largest big-data effort of its kind, with more than
170,000 patients consented. In addition to using this system to work
together to tackle major cancer research projects, ORIEN is also
partnering with pharmaceutical companies who can use the data to speed
up the process of finding the right patients for clinical trials to get
new drugs to the market faster.
Another example of a ``big data'' approach to observational
research in cancer is the American Society for Clinical Oncology's
``CancerLinQ'' program, which is also now aggregating and analyzing
large amounts of patient data to support clinical decisions in cancer
care.
To what extent could these new health learning systems be of use to
the Food and Drug Administration in the evaluation of emerging cancer
treatments or related tasks, such as looking at the efficacy of
sequences of treatments involving drugs that have already been
approved? Would you see any impediment to working in partnership with
ORIEN, ASCO and similar organizations to expand the FDA's current
capacity for drug evaluation and research in oncology?
Answer 3. As the delivery of cancer care becomes more personalized,
and more closely tied to information about the molecular signatures of
individual patients, approaches that enable the collection of better,
patient-specific data are going to become increasingly important not
only in informing the regulatory process, but also the delivery of care
and the practice of oncology medicine and personalized medicine. I am
committed to finding ways that FDA can more closely partner with, and
leverage these opportunities to inform its own work and help fulfill
the public health mission of these efforts advance opportunities for
improved patient care.
Question 4. Dr. Gottlieb, in 1978 FDA committed to address the
overregulation of medical gases, like Oxygen, by creating separate
regulations for medical gases as a unique class of drug products.
Thirty-nine years later, FDA has not followed through on its commitment
to create an appropriate framework specific to medical gases. In 2012,
Congress enacted historic and bipartisan reforms at the request of the
medical gas industry and pharmacists to require FDA to follow through
on its 1978 commitment to address the overregulation of medical gases
in the Food and Drug Administration Safety and Innovation Act (FDASIA).
In November 2016, FDA issued a final rulemaking that addressed some
medical gas labeling issues, however FDA did not, as intended by FDASIA
and reiterated to FDA in the fiscal year 2016 Appropriations report
language, modify current regulations to address the overregulation
regulation of medical gas, such as the medical air labeling, adverse
event reporting, expiration dating, calculation of yield and a host of
other safety and enforcement issues identified by the medical gas
industry as necessary to appropriately regulate medical gases. If
confirmed as Commissioner of FDA, would you ensure that FDA fully
implements Section 1112 of FDASIA by working with stakeholders to
either incorporate by reference industry consensus standards or issue
new final rulemakings on medical gas to address these unique medical
gas regulatory issues?
Answer 4. If confirmed, I am committed to implementing all
congressional laws, including Section 1112 of FDASIA. I look forward to
working with you on this issue.
Question 5. I am interested in bringing more predictability and
consistency to the device inspections process. For routine inspections,
FDA should be able to give companies advance notice that they will be
inspecting, as well as providing regular communications throughout the
inspection process. Additionally, should FDA find an issue that needs
to be addressed during an inspection, companies have 15 days to submit
a remediation plan to FDA but there's no such timeline for FDA to
respond to companies to communicate whether the remediation plan meets
FDA expectations. I believe it makes sense for FDA inspections to be
done in a risk-based system, meaning that FDA should focus its
resources on those inspections that will have the most meaningful
impact on patient safety. Will you commit to working with me and
Senator Bennet on this issue?
Answer 5. I agree that predictability and consistency are very
important aspects of the FDA device inspections process. I also agree
that across its regulatory portfolio, FDA should be taking a risk-based
approach to its work in order to make sure the agency is maximizing its
resources in pursuit of its important public health mission to protect
and promote public health. If confirmed, I commit to working with you,
Senator Bennet, and members of your staff on this issue.
senator franken
Question 1. The MDUFA agreement includes funding for implementation
of NEST and requires a pilot project to test the NEST system to
facilitate pre-market approval of medical devices. Will the pilot
projects be designed to support the generation and collection of data
on devices, even after they come to market? How will this data be used
to develop a more active post-market surveillance system?
Answer 1. The reauthorization proposals for PDUFA, MDUFA, GDUFA,
and BsUFA were developed and submitted to Congress prior to the end of
the previous Administration. I was not involved with the FDA-industry
technical negotiations on any of these proposals.
However, I am supportive of data transparency and recognize the
importance of collecting valid data that can be used to bring new
medical devices to market, expand indications for approved medical
devices, and enhance the agency's ability to collect important patient
safety information. If confirmed, I will commit to working with staff
to quickly get up to speed on this issue and help the agency evaluate
whether NEST could be an appropriate tool for post-market surveillance.
Question 2. When I met with you in my office prior to your
nomination hearing, you explained that while the industry funding will
help develop NEST, the FDA will need to update its tools to fully
realize the potential of NEST and other initiatives. What is the FDA's
role in this process and does the FDA have the resources it needs to
make these updates?
Answer 2. FDA will play an important role in developing NEST but
the system will be owned and operated by multiple stakeholders. I
support a properly resourced FDA that also has the modern tools it
needs in order to use data in pursuing its public health goals and, if
confirmed, will work to ensure that FDA is well-positioned to carry out
its mission.
Question 3. Rising drug prices continues to be problem in the
United States and over 70 percent of Americans think that Congress
should address the issue. If appointed as commissioner, will you commit
to working with the HELP committee to address skyrocketing drug prices
through actions under the FDA's purview? What proposals do you think
have the biggest potential to lower costs?
Answer 3. While drug pricing does not fall directly within FDA's
purview, I believe the agency can play an important role on this
important issue by taking steps to improve product competition. If
confirmed, I will work to ensure FDA has the appropriate policies and
processes in place to effectively facilitate generic market entry and
competition, especially for complex drugs that sometimes do not face
effective generic competition even long after the patent expires.
Reforming the regulatory pathway for complex generic products would
address one key policy deficiency that results in unnecessary barriers
to the development and review of generic competitors for some innovator
products for which traditional bioequivalence and bioavailability
testing alone are sometimes insufficient for proving sameness. FDA
should also explore options to improve the efficiency and consistency
of ANDA review processes and timelines, so that financial speculators
cannot engage in a regulatory arbitrage, by dramatically hiking the
price of some very old generic drugs because they know it can take
years for new generic competitors to enter the market.
Question 4. In 2001, the FDA withdrew Avastin, a drug approved for
breast cancer through an accelerated approval pathway with surrogate
endpoint data, due to findings that the drug was not as effective as
was indicated by the surrogate endpoint. What did you take away from
FDA's experience with Avastin and had you been commissioner in 2011,
would you have made a different decision? Would you have overruled your
top cancer scientists and drug experts on Avastin?
Answer 4. Insofar as I am not privy to all of the details related
to the 2011 withdrawal from the market of Avastin, I do not believe it
would be appropriate to opine on all of the things that I
hypothetically might, or might not have done differently in that
situation. Maintaining the Gold Standard of safety and efficacy for
medical products is fundamental to FDA's mission to protect and promote
public health. If confirmed, I will uphold the Gold Standard by
ensuring FDA makes regulatory decisions based on sound science, good
regulatory practices, and the support of a strong scientific staff.
Question 5. Based upon the outcome in the case of FDA's withdrawal
of Avastin to treat breast cancer and your published criticism of the
FDA's decision, what assurances can you give that indicate that you
will be willing to take action to protect public safety by removing a
product from the market if and when later stage studies show drugs to
be ineffective or unsafe?
Answer 5. FDA's ability and obligations to remove a drug from the
market, or in this case rescind a specific indication from drug
labeling, if a drug fails to meet the required standard in a
confirmatory trial, is an important component of the accelerated
approval process, as established by Congress. If confirmed, I will
follow the science and support the staff in upholding congressional
intent in the conduct of the drug review program.
Question 6. Congress passed the Pediatric Device Consortia Program
in 2007 to help address the slow rate of innovation in pediatric
medical devices but pre-market approvals for pediatric devices still
only comprise 5 percent of pre-market approval submissions. What more
can the FDA do to foster the development of new pediatric medical
devices and improve the number of approvals?
Answer 6. I agree that FDA should support the development of safe
and effective pediatric medical devices. I am aware of a number of
efforts FDA is undertaking in this area, including data collection on
barriers to the development of and the unmet needs for pediatric
medical devices. If confirmed, I will continue FDA's work and consider
additional efforts in this area.
Question 7. The passage of the Biologics Price Competition and
Innovation Act (BPCIA) created a pathway for biosimilar entry. Yet, the
FDA is still in the developing stages of developing and implementing
this pathway. There are a number of outstanding issues that make it
harder for FDA to implement the biosimilar pathway and increase
competition to lower prices. What is FDA's role in encouraging more
manufacturers to develop biosimilars for the U.S. market?
Answer 7. I fully support the BPCIA. FDA can most effectively
support a robust biosimilar marketplace by providing clarity about a
consistent, transparent, science-based regulatory process for
biosimilars. If confirmed, I will work to ensure the agency has the
appropriate policies and processes in place to fully implement the law,
as intended by Congress.
Question 8. There are currently 182 drugs on the market that no
longer have patent protection but also do not have any generic
competition. Further, there are more than 500 drugs with only one
marketed generic. In these situations, brand companies are able to
price their product however they choose without market competition to
drive down prices. What is the FDA's role in monitoring, identifying,
and remedying market situations that lead to access barriers for
patients or price gouging by manufacturers?
Answer 8. While drug pricing does not fall directly within FDA's
purview, I believe the agency can play an important role on this issue
by taking steps to improve product competition. If confirmed, I will
work to ensure FDA has the appropriate policies and processes in place
to effectively facilitate generic market entry and competition,
especially for complex drugs that sometimes don't face effective
generic competition even long after the patent expires.
Reforming the regulatory pathway for complex generic products would
address one key policy deficiency that results in unnecessary barriers
to the development and review of generic competitors for some branded
drugs for which traditional bioequivalence and bioavailability testing
alone are sometimes insufficient for proving sameness. FDA should also
explore options to improve the efficiency and consistency of ANDA
review processes and timelines, so that financial speculators cannot
engage in a regulatory arbitrage, by dramatically hiking the price of
some very old generic drugs because they know it can take years for new
generic competitors to enter the market.
senator paul
Question 1. Since passage of the Drug Quality Security Act in 2013,
the FDA has implemented and enforced the law in a manner inconsistent
with clear congressional intent. The agency has used guidance documents
to assert regulatory authority over State-licensed compounding
pharmacies and to treat them like drug manufacturers, and overly
restricted the geographic limits for compounding in hospital systems.
If confirmed as Commissioner, can you can you commit to taking a fresh
look at the FDA's interpretation of the DQSA to assure that any
regulations or guidance documents issued or related enforcement actions
undertaken against pharmacies are consistent with the DQSA as written
and in a way that better balances public safety with patient access to
critical medications?
Answer 1. If confirmed, I am committed to implementing DQSA, as
intended by Congress, to both protect patient safety, and allow the
safe and appropriate practice of pharmacy compounding. I will commit to
working with your office to make sure we are appropriately pursuing the
goals of DQSA, including protecting the role for the safe practice of
pharmacy medicine.
Question 2. Currently, FDA has a number of tools at its disposal to
allow for innovative clinical trial design and for the consideration of
foreign drug data in the drug approval process. Will you consider using
or expanding the use of FDA's current authorities to expedite
consideration of drugs approved in other countries?
Answer 2. FDA has taken certain steps to bring more harmonization
between international regulatory authorities in various aspects of its
review programs, and make better use of regulatory data generated from
oversees regulatory processes. If confirmed, I would commit to
continuing to work with the FDA staff to look at additional ways that
the agency can leverage the expertise and experience of its foreign
counterparts.
Question 3. OTC medicines are a cost-effective first-line therapy
for many conditions, and the Rx-to-OTC switch often has a positive
impact on the healthcare system and drives down costs. Since 1976, 106
ingredients have made the Rx-to-OTC switch since 1976. Furthermore,
FDA's OTC monograph approval work is far from complete.
What do you believe is the appropriate role of FDA in the Rx-to-OTC
process and what can the agency do to improve the process, and
ultimately provide greater access to OTC drugs?
There are two older types of insulin available in OTC form.
Understanding that that appropriate guidance from a health care
professional is important, will FDA continue to consider whether
additional formulations of drugs such as insulin can be safely
available to patients over-the-counter?
Answer 3. I believe that there may be additional policy steps we
can take to leverage opportunities to facilitate more patient access to
OTC medicines and that this is an important public health goal. For
example, I believe that the current monograph system for regulating
over-the-counter drugs should be evaluated to see if improvements can
be made. I recognize the potential public health benefit inherent in
the increased availability of OTC drugs and, if confirmed, will work to
improve this important regulatory pathway.
Question 4. Recently, there have been a number of older, off-patent
drugs, such as insulin and tetracycline, that have seen large price
increases. Understanding that FDA does not have direct control over
drug prices, what role do you believe the agency can play in working to
address this problem?
Answer 4. While drug pricing does not fall directly within FDA's
purview, I believe the agency can play an important role on this
important issue by taking steps to improve product competition. If
confirmed, I will work to ensure FDA has the appropriate policies and
processes in place to effectively facilitate generic market entry and
competition, especially for complex drugs, including potentially some
insulin products, that sometimes don't face effective generic
competition even long after the patent expires. Reforming the
regulatory pathway for complex generic products would address one key
policy deficiency that results in unnecessary barriers to the
development and review of generic competitors for some branded products
for which traditional bioequivalence and bioavailability testing alone
are sometimes insufficient for proving sameness. FDA should also
explore options to improve the efficiency and consistency of ANDA
review processes and timelines, so that financial speculators cannot
engage in a regulatory arbitrage, by dramatically hiking the price of
some very old generic drugs because they know it can take years for new
generic competitors to enter the market.
Question 5. The FDA's tobacco ``deeming rule'' threatens to upend
the tobacco industry subject to this regulation. In particular, I am
concerned that the regulation puts vapor, cigars, and other deemed
products at a significant disadvantage to combustible cigarettes, which
were allowed to remain on the market when Congress subjected them to
FDA review. Furthermore, some of these products may present harm
reduction opportunities for tobacco users. Will you commit to working
to make this regulation more reasonable, so that it is workable for
companies trying to keep products on the market?
Answer 5. If confirmed, I will be committed to implementing the
TCA, as intended by Congress, including Section 911 related to modified
risk products, which I recognize can provide helpful tools for current
tobacco-users to transition off combustible tobacco. As I was not at
FDA during the agency's initial TCA implementation activities, I am not
fully acquainted with internal processes or specific decisions to-date.
If confirmed, I will work with the professional staff to quickly get
up-to-speed on this issue, and I will review current FDA policies,
including the deeming rule, to ensure FDA treats products
appropriately, implements provisions in a timely fashion, and in a
manner that is fully consistent with congressional intent under the
TCA. I believe that responsibly implementing the TCA is an integral
part of FDA's core mission to protect and promote public health.
Question 6. A midnight rule proposed by FDA under the Obama
administration intends to limit a specific chemical compound's (NNN)
presence in smokeless tobacco to presently unachievable levels.
Approximately 1,200 Kentucky farm jobs and an additional 600 Kentucky
manufacturing jobs are at risk from this proposed rule because nearly
all of this dark tobacco is grown within a 50-mile radius of
Hopkinsville, KY. If this rule were to be finalized, these jobs would
either be eliminated or moved overseas. As commissioner, how will you
work to balance regulating chemicals like NNN with the importance of
these farming and manufacturing jobs?
Answer 6. I have not been privy to the development of this proposed
rule although I understand the comment period has been extended. If
confirmed, I will commit to reviewing the scientific evidence and
working with you on this issue.
Question 7. Last year, DEA, with FDA's input, decided not to change
the controlled substance schedule for cannabis. Also, last year, FDA
approved the second cannabis-based drug. I believe continued research
in this area is critical to understand the potential medical benefits
of cannabis and cannabis-based products. What role can FDA play in
continuing to consider the therapeutic benefits and provide access to
patients where appropriate?
Answer 7. I am aware that in July 2016, DEA determined, in
consultation with HHS, that marijuana continues to meet the criteria
for Schedule I control under the Controlled Substances Act. I cannot
speak to decisions that the DEA might take in the future, but I do know
that FDA is currently involved in supporting scientific research
related to medicinal uses of marijuana and its constituents. If
confirmed, I am committed to continuing this research.
Question 8. FDA, along with USDA and DEA, published guidance last
August on industrial hemp. This guidance went far beyond what Congress
had explicitly constructed in statute by narrowing the definition of
hemp, restricting commerce from hemp pilot programs, and prohibiting
transportation of hemp plants and seeds across State lines. As
commissioner, how would FDA work with the industrial hemp industry to
ensure it is able to continue thriving?
Answer 8. I have not been involved in this issue so I cannot
comment directly. However, I will note that I am committed to
implementing congressional laws as they are intended. If confirmed, I
will commit to working with you to address this issue.
Question 9. Obamacare authorized the creation of a Federal menu
labeling standard. On May 5, 2017, businesses will have to comply with
FDA's Federal menu labeling rule. With this rule, businesses could face
criminal penalties for violation. To provide context for how difficult
this could be, a pizza company based in Kentucky determined there are
over 30 million pizza combinations an individual could order at their
restaurants. This rule would require them to post calorie counts for
all those possible combinations on a menu in their restaurants, even
though it already publicly provide all this information on its Web
site. If an employee accidentally puts an extra handful of cheese on
the pizza, those calories will inevitably be off--and now they will be
subject to criminal penalties. As commissioner, how would you move
forward with implementation of this rule and provide flexibility to
business owners and their employees?
Answer 9. While I am broadly aware of the menu labeling issue, this
is not a matter on which I am familiar with the technical specifics. As
a general rule, I support providing clear, accurate, and understandable
information to American consumers to help inform healthful dietary
choices. I believe information about caloric content can be a useful
tool. However, I am mindful of the unique challenges that developing
and communicating such information can pose, particularly on small,
independent businesses. If confirmed, I will commit to working with the
agency's professional staff to quickly get up to speed on the
regulatory history related to menu labeling, as well as FDA's latest
thinking and actions. I would welcome the opportunity to work with
Congress and stakeholders to ensure any regulatory requirements would
promote public health by providing helpful information to consumers,
while not placing unnecessary compliance burden on businesses,
particularly small, independent ones.
senator bennet
Question 1. Antibiotic resistance is a real and growing public
health crisis. The Centers for Disease Control and Prevention (CDC)
estimates that at least 23,000 people die every year in the United
States from resistant infections.
Last year I worked with Senator Hatch on the PATH Act, which
creates a new pathway for antibiotics to treat potentially deadly
infections for which there are no other treatment options. The
legislation was passed in the 21st Century Cures Act. This law allows
FDA to approve these drugs on the basis of limited data sets, and also
puts important protections in place to ensure these drugs are used only
by patients for whom the benefit would outweigh the risk.
If you are confirmed, will you commit to fully implementing this
law including the provisions designed to ensure that these drugs go to
the patients who actually need them?
Answer 1. The availability and appropriate prescribing of
antibiotics are vital to our Nation's public health. Additionally,
antibiotic resistance is a significant and growing public health
challenge facing our Nation. Within its statutory authorities, FDA
should encourage the development of new antibiotics and ensure proper
labeling to help address the issue of inappropriate prescribing and/or
use. If confirmed, I would commit to fully implementing, in a timely
fashion and consistent with congressional intent, the PATH Act for
antibiotics.
Question 2. Clinical trials are a necessity to understand the
safety and efficacy of new medicine, but participation in trials is
remarkably low. One reason for this is the eligibility criteria for
potential patients can be quite restrictive. This can result in trials
conducted with patients that are not reflective of the people that will
ultimately use the drug.
What would be needed for FDA to play a more active role in
determining clinical trial eligibility criteria?
What can the FDA do to help trials be more representative of the
overall population?
Would efforts to expand eligibility criteria help more patients
with rare diseases enroll in clinical trials?
Answer 2. I think it is very important that clinical trials capture
the diversity of the population who will likely use the medical product
once it is marketed and becomes available. If confirmed, I will work to
ensure that FDA policies support the conduct of clinical trials that
represent the clinical diversity of the intended patient population,
including through the implementation of Section 907 of the Food and
Drug Administration Safety and Innovation Act (FDASIA). With respect to
rare diseases in particular, I would be committed to working with your
office and other stakeholders to address ways that FDA can encourage
more patients with rare diseases to enroll in clinical trials.
Question 3. Currently, we hear from Coloradans who have a loved one
suffering from a severe terminal illness and have no choices in terms
of drugs already approved. For those who have no treatment options and
are unable to participate in a clinical trial, they understandably seek
access to unapproved experimental treatments.
When you previously spoke as the Deputy Commissioner for Medical
and Scientific Affairs you stated that ``participation in clinical
trials is the best way for patients to get access to unapproved
drugs.''
What else can the FDA do to ensure that these patients can explore
all of their options?
Is there a way that FDA can collect safety and efficacy data from
expanded access cases in a way that gives companies regulatory clarity
while providing another option for patients?
Answer 3. FDA works to inform potential participants on a wide
range of issues related to clinical trials. For example, FDA created
the Office of Health and Constituent Affairs to serve as a contact for
patients. NIH also plays a key role in maintaining ClinicalTrials.gov,
a data base which anyone, including providers, patients, and family
members, can use to search for information about current, ongoing
clinical research studies. Nothing prohibits FDA from collecting safety
and efficacy data during expanded access cases. However, the conditions
and circumstances of expanded access cases do not generally lend
themselves to determining the efficacy of a drug. If confirmed, I
commit to looking into this issue and working with you on it. I am
committed to exploring ways that we might improve expanded access
programs to provide patients with terminal diseases more options to get
access to promising experimental medicines, and perhaps learn more from
these access programs without creating any additional burdens or
disincentives for sponsors who are seeking to make drugs available to
terminal patients within the scope of the laws and regulations
governing these programs.
senator collins
Question 1. FDA's menu-labeling rule, even after an initial stay,
will take effect 1 month from today. Grocery stores and other food
retailers across America continue to be frustrated with FDA's handling
of things, including for local and seasonal food items. Fresh and local
food items may be sold at a few stores, under the same name, but the
ingredients or recipe can vary, yet they would be considered ``standard
menu items'' and subject to enforcement. The irony is that this will
cause stores and restaurants to move away from fresh, local, and
seasonal offerings. With just a month before the compliance date, we
need FDA to act quickly to further delay, withdraw, or stay the rule so
it can be rewritten to give businesses the flexibility to comply. Would
you be willing to explore ways to encourage FDA to act before the
compliance date to provide this much-needed flexibility for businesses?
Answer 1. While I am broadly aware of the menu labeling issue, this
is not a matter on which I am familiar with the technical specifics. As
a general rule, I support providing clear, accurate, and understandable
information to American consumers to help inform healthful dietary
choices. I believe information about caloric content can be a useful
tool. However, I am mindful of the unique challenges that developing
and communicating such information can pose, particularly on small,
independent businesses. If confirmed, I will commit to working with the
agency's staff to quickly get up to speed on the regulatory history
related to menu labeling, as well as FDA's latest thinking and actions.
I would welcome the opportunity to work with Congress and stakeholders
to ensure any regulatory requirements would promote public health by
providing helpful information to consumers, while not placing
unnecessary compliance burden on businesses, particularly small,
independent ones.
Question 2. The safety of personal care products is an issue of
interest to many Americans. The average consumer uses 10 personal care
products every day, yet the laws governing the cosmetics and personal
care products industry have not been updated since 1938, and States
have been acting on their own in the absence of a national safety
standard. There is growing support in Congress for modernizing cosmetic
safety laws and providing greater transparency for consumers and
regulatory certainty for manufacturers. Can you commit to working with
us and other Senators to modernize FDA's authority to regulate cosmetic
products to better serve public health?
Answer 2. If confirmed, I commit to working with you and others in
Congress to explore options to potentially modernize FDA's authorities
related to the regulation of cosmetic products.
Question 3. Dr. Gottlieb, in our meeting we discussed the Aging
Committee's investigation into the sudden price-spikes of decades old
drugs and the report we issued outlining a number of potential
bipartisan solutions to help improve access to affordable medications.
You spoke about a number of regulatory obstacles to making drugs
generic. Would you share with the committee any ideas you have, related
to REMS or easing other regulatory obstacles, that could increase
generic entry?
We discussed unintended impacts of the FDA risk-evaluation and
mitigation strategies (REMS) on generic competition. Some drug
companies have used REMS to prevent potential generic competitors from
getting access to the drug for the FDA required bioequivalence studies.
Two weeks ago, Dr. Janet Woodcock testified that the FDA had reported
150 cases of REMS abuse to the FTC. What can the FDA do to ensure the
safe handling of these drugs while still promoting generic entry?
The need for more affordable, complex drugs is also great. Last
summer, for example, we saw the Mylan EpiPen price increase by 500
percent. For complex products, such as auto-injectors and metered-dose
inhalers, what role can the FDA play in helping sponsors get generic
competition approved?
Answer 3. My understanding is FDA has already taken action to
instruct manufacturers that they may make samples of products subject
to a REMS available to prospective generic competitors for the purpose
of bioequivalence studies without running afoul of their legal
requirements. It is important to note though, that appropriate safety
precautions must be in place under specific circumstances, given the
unique patient safety and public health risks associated with certain
REMS products. But we need to make sure our policies are striking the
right balance between safety and access, and evaluate carefully if
regulations meant to improve safety are also becoming an unintended
barrier to access and competition. If manufacturers inappropriately
refuse to provide their product to prospective generic competitors,
this would be a concern to FDA and become a matter for potential
enforcement action by the Federal Trade Commission. While drug pricing
does not fall directly within FDA's purview, I believe the agency can
play a key role on this important issue by taking steps to improve
product competition. If confirmed, I will work to ensure FDA has the
appropriate policies and processes in place to effectively facilitate
generic market entry and competition, especially for complex drugs that
sometimes do not face effective generic competition even long after the
patent expires and other places where there are specific issues that
make it hard for generic manufacturers to copy certain products and
demonstrate sameness under FDA's existing guidance. Reforming the
regulatory pathway for complex generic products would address one key
policy deficiency that results in unnecessary barriers to the
development and review of generic competitors for some branded products
for which traditional bioequivalence and bioavailability testing alone
are sometimes insufficient for proving sameness. FDA should also
explore options to improve the efficiency and consistency of ANDA
review processes and timelines, so that financial speculators cannot
engage in a regulatory arbitrage, by dramatically hiking the price of
some very old generic drugs because they know it can take years for new
generic competitors to enter the market.
Question 4. Alzheimer's disease is one of the greatest public
health threats of our time. More than five million Americans are living
with Alzheimer's and other dementias. It is the sixth leading cause of
death, and yet there is no cure, treatment, or means of prevention.
Researchers are making major advancements in understanding the
pathology of Alzheimer's beyond the amyloid beta and tau proteins.
Today scientists are finding new genetic links, neuroprotective
factors, and brain wide connectivity patterns associated with dementia
risk. With the advancements of these biomarkers, we are poised to make
progress in cures. How can the FDA support drug development using these
biomarkers?
Answer 4. Alzheimer's disease is, indeed, a major public health
challenge today, and absent the discovery and development of new
medicines that can slow, reverse, and perhaps even prevent the onset of
the disease, the future impact Alzheimer's disease is staggering, in
terms of human suffering and societal cost. If confirmed, making
certain FDA has the right policies and processes in place to
effectively encourage the development of safe and effective new medical
products for Alzheimer's disease--to prevent its onset, to slow its
progression, and one day to cure it--will be one of my highest
priorities. I will work to ensure the agency consistently uses sound,
rigorous, 21st century regulatory science, tools and approaches to
facilitate the discovery and development of drugs for Alzheimer's
disease. The use of well-validated biomarkers could be an important
component of the future of Alzheimer's research and development. In
short, patients with Alzheimer's disease, their families, and our
Nation as a whole will benefit from the development of new Alzheimer's
treatment options. If confirmed, I look forward to working with
Congress, other government agencies like the NIH, industry, academia,
and the patient and stakeholder communities to ensure FDA does
everything it can to play its important role in tackling this
challenging public health issue.
Question 5. This is an exciting time for biologic products and
regenerative medicine. In laboratories across America, every day we are
learning something new. We are on the verge of real breakthroughs that
may change disease trajectories fundamentally. In this era of discovery
in biomedical research, we must promote and incentivize success.
Developing biologic products and regenerative therapies is unique
compared to traditional targeted drugs. In some cases, different
endpoints may be needed. What role do you see for the FDA in
facilitating new endpoints?
Answer 5. New areas of biologics and regenerative medicine are some
of the most innovative and promising emerging advancements in our
scientific approaches to the treatment of human disease. Regenerative
medicine, in particular, appears to hold great promise for new
therapeutic options for patients and physicians, particularly in areas
of unmet or underserved medical need. I believe more generally, FDA
should lean forward in defining the regulatory principles on which the
safety and effectiveness of these new areas of medicine are going to be
judged by the agency, in order to help facilitate their development.
This includes the development of appropriate endpoints for evaluating
the safety and effectiveness of new areas of biological products and
regenerative medicine. If confirmed, I will embrace the responsibility
to facilitate important medical innovation, while maintaining the
agency's Gold Standard of safety and efficacy.
Question 5. In 2011, FDA issued an Advisory on glass lamellae
formation in certain injectable drugs. This action was taken out of
concern for the impact of glass contamination on drug quality and
patient safety. The Advisory noted at the time that ``there is the
potential for drugs administered intravenously that contain these
fragments to cause embolic, thrombotic and other vascular events.'' I
understand that it has been discovered recently that glass issues, in
addition to glass lamellae formations, can increase the presence of
glass in injectable drugs beyond the level previously known when the
Advisory first was issued. This potential to cause harm to patients
presents a public health risk. If confirmed, what action would you plan
to take to address these glass issues and when?
Answer 5. I recognize the potential safety concern related to the
use of glass vials and, if confirmed, will ensure that FDA is taking
every necessary step to address this issue.
Question 6. I mentioned during our meeting the FDA's proposed rule
on electronic prescribing information and its implication for rural
pharmacists. I continue to be concerned that it would have an adverse
effect on patient safety, particularly for Americans who live in areas
with limited Internet access, and have serious implications for
patients and pharmacists during a power outage or in the wake of a
natural disaster or terrorist attack. Given that 96 percent of the
public comments were in opposition to the proposal, are you willing to
evaluate carefully the concerns and consider withdrawing this ill-
conceived proposal?
Answer 6. Ensuring that accurate, science-based information about
medical products is readily accessible to patients and providers is an
issue that is critically important to public health. Drug labeling is
FDA's primary vehicle for communicating risk information to consumers
and providers and it needs to be readily accessible to patients. If
confirmed, I will commit to engaging with the agency's professional
staff to quickly get up to speed on this issue. I would welcome the
opportunity to work with you on this issue moving forward.
Question 7. Another area of increasing concern is the overuse of
antibiotics in agriculture and the rise of antibiotic-resistant
bacteria. Preserving the effectiveness of medically important
antibiotics used to treat human and animal bacterial diseases is
critical and has been described as the single most important challenge
in infectious disease. The CDC estimates that at least 2 million
illnesses and 23,000 deaths caused by antibiotic-resistance occur every
year in the United States. I have introduced legislation with Senator
Feinstein aimed at reducing the overuse of antibiotics in agriculture.
Will you continue to support ways that the FDA, in cooperation with
other Federal agencies, can address the growing problem of antibiotic-
resistant bacteria and the overuse of antibiotics in agriculture?
Answer 7. Antibiotic resistance is a significant and growing public
health challenge facing our Nation. In addition to measures FDA should
take to address this issue within the context of human use, the agency
must effectively collaborate with other government agencies and public
health authorities to develop policies and processes to address the
issue of antibiotic use in animals intended for human consumption. If
confirmed, I will ensure FDA remains engaged on this important public
health issue. This includes making sure that animal drug labeling
reflects the most up-to-date science, and working closely with the U.S.
Department of Agriculture, the Centers for Disease Control, the U.S.
Department of Defense, and other appropriate government agencies. FDA
should also consider input from other important stakeholders, such as
the farmers, the agriculture industry, and veterinarians. FDA's
implementation of a voluntary plan with industry to phaseout the use of
certain antibiotics is an important step in the right direction.
senator whitehouse
Question 1. The FDA is currently implementing the 21st Century
Cures Act, including its provisions on improving coordination between
the relevant centers during the review of a combination product. Though
FDA seems to be making progress on improving this review process, I
think it is important that we continue to monitor this issue and be
ready to make bigger changes to the combination products approval
process if they are needed. As Commissioner, will you commit to keeping
the HELP Committee updated on FDA's work to improve the combination
products review process, and to propose any recommended legislative
updates in a timely manner?
Answer 1. I recognize the importance of FDA's combination product
review program and, if confirmed, will commit to implementing 21st
Century Cures and working with you and the other Senators on the HELP
Committee on any related legislative proposals.
Question 2. You have written extensively on your views regarding
the regulation of medical software and apps. In a 2014 Wall Street
Journal commentary titled, ``Why Your Phone Isn't as Smart as It Could
Be,'' you wrote ``The FDA should exempt the majority of mobile-health
apps from premarket review.'' Last Congress, members of this committee
led by Senators Hatch and Bennet worked to define FDA's authority over
medical software as part of the 21st Century Cures Act. In that law, we
exempted many forms of low-risk software, such as software intended to
encourage a healthy lifestyle, from FDA oversight.
Do you believe that there are additional types of medical software
that should be exempted from FDA oversight? If so, which ones and why?
Answer 2. I support tools that can help provide consumers with more
information about their health. FDA must clearly define when an app is
a medical device subject to its regulation, and when it falls outside
FDA's framework or is low risk, and therefore appropriate to be exempt
from the full scope of FDA's pre-market requirements. For products
subject to FDA's pre-market requirements, the agency should use a risk-
based approach in regulating consumer apps and other medical software,
and, if confirmed, I look forward to helping further refine FDA's
approach and implement the related provisions of 21st Century Cures in
a timely fashion and consistent with congressional intent.
Question 3. In the 21st Century Cures Act, Congress established an
administrative process that FDA can use to bring medical software that
is exempted from regulation back under FDA oversight if it is
reasonably likely to have ``serious adverse health consequences.''
How would you determine whether a piece of medical software or an
app poses an adverse health threat?
Under what circumstances would you conclude that this
administrative process should not be used if medical software is
determined to have a risk of serious adverse health consequences?
Answer 3. If confirmed, I look forward to working with the FDA's
professional staff to properly implement this framework, consistent
with congressional intent. I would also commit to working with you and
your colleagues in Congress, to keep you informed about FDA's
implementation efforts.
Question 4. In the same Wall Street Journal commentary referenced
in question 2, you suggested that FDA should employ ``post-market
controls'' to make sure mobile health apps are meeting expectations.
What types of post-market controls should FDA use to make sure such
software--as you wrote--``meets expectations''?
Answer 4. Controls should be used to ensure, among other things,
that mobile health apps are providing consumers with data that is
accurate and reliable. If confirmed, I would work with Agency staff to
develop appropriate post-market controls that ensure the safety of
consumers but continue to allow for innovation in product development.
Question 5. The Centers for Disease Control and Prevention
estimates that 2 million people develop antibiotic-resistant infections
in the United States each year, resulting in at least 23,000 deaths.
The World Health Organization has concluded that antibiotic resistance
. . . ``is one of the biggest threats to global health'' . . . One area
in which CDC, FDA, and the agriculture sector has made strides in
recent years is the judicious use of medically important antimicrobial
drugs in food-producing animals. Do you believe FDA should have a role
in improving antimicrobial stewardship? What is that role?
Answer 5. Antibiotic resistance is a significant and growing public
health challenge facing our Nation. In addition to measures FDA should
take to address this issue within the context of human use, the agency
must effectively collaborate with other government agencies and public
health authorities to develop policies and processes to address the
issue of antibiotic use in animals intended for human consumption. If
confirmed, I will ensure FDA remains engaged on this important public
health issue, making sure that animal drug labeling reflects the most
up-to-date science, and working closely with the U.S. Department of
Agriculture, the Centers for Disease Control, the U.S. Department of
Defense, and other appropriate government agencies, among other steps.
I believe it is a priority for FDA to continue to play an important
role in improving antimicrobial stewardship.
Question 6. Stakeholders within the animal health sector
acknowledge the need for better data and more timely information about
the use of antibiotics in production agriculture and its role in
antibiotic resistance. As you may know, the National Antimicrobial
Resistance Monitoring System (NARMS), jointly coordinated by CDC, FDA,
and the USDA, analyzes trends in antibiotic resistance by collecting
bacteria samples from animal and meat products as well as cases of
human foodborne illness. This allows the aforementioned Federal
agencies to accelerate their response to emerging public health
threats. Do you support the NARMS initiative and intra-agency efforts
to conduct surveillance of antibiotic resistance across the food chain?
Answer 6. I fully support efforts that will help facilitate the
collection of more and better information about foodborne illness and
emerging and resistant infections.
Question 7. As you know, earlier this year President Trump signed
an Executive order instituting a hiring freeze for the executive
branch. The order did not state how the freeze would affect many
agencies, including the FDA. The 21st Century Cures Act recently
provided the FDA with new hiring authorities, and a significant portion
of user fee dollars goes toward supporting the hiring of FDA review
staff in the drug and device centers.
Do you agree that FDA's approval process would benefit if it could
promise higher salaries to attract scientific expertise? If so, what
steps will you take to advocate for an exemption for FDA from the
hiring freeze?
Do you believe the FDA will be able to fulfill its mission if the
freeze prevents you from hiring new staff?
Answer 7. I was not involved with the development of the
Administration's Federal workforce proposals, including the hiring
freeze Executive order. FDA's ability to fulfill its mission to protect
and promote public health depends on its world-class workforce of
talented and dedicated public servants. If confirmed, I will commit to
working with my colleagues in the Administration, as well as Congress,
to ensure FDA is appropriately resourced and staffed.
Question 8. Regulatory capture has been a concern within regulated
industries since Woodrow Wilson. In the case of FDA, industry is not
only regulated by the agency, but is also its funder.
According to the Congressional Research Service, when the
prescription drug user fee agreement was first authorized, user fees
funded 10 percent of the Human Drug Program's activities. In 2016, it
was nearly 65 percent. The medical device user fees covered 16 percent
of the device center's costs in 2006, compared with 35 percent in 2015.
As Commissioner, what specific steps will you take to ensure the FDA is
able to maintain its independence so it can continue to put public
health and safety first when reviewing drug and device applications?
Answer 8. As you note, user fees have grown to provide a
significant portion of FDA's funding. At the same time, I have
confidence that these fees are structured in a way that does not allow
regulated industry to exert inappropriate influence on the agency.
Maintaining the Gold Standard of safety and efficacy for medical
products is fundamental to FDA's mission to protect and promote public
health. If confirmed, I will uphold the Gold Standard and the
independence of FDA's review process by ensuring FDA makes regulatory
decisions based on sound science, good regulatory practices, and the
support of a strong scientific staff.
Question 9. Many of my Senate colleagues and I have called on the
FDA to issue advice to pregnant women about the safe consumption of
seafood that is based on the latest nutrition science. FDA research
shows that maternal seafood consumption offers significant
neurodevelopmental benefits for children. FDA committed to completing
the seafood advice in 2011, but only did so on January 18, 2017. While
I was pleased to see the advice finalized, it is now inconsistent with
the Dietary Guidelines issued by the Department of Health and Human
Services and the Department of Agriculture.
Have you reviewed FDA's advice on safe seafood consumption for
pregnant women?
Do you believe Federal guidance in this area should be based on the
best available science?
If you are confirmed, will you work with the relevant Federal
agencies to ensure the guidance about seafood consumption for pregnant
women is consistent?
Answer 9. If confirmed, I will ensure FDA's advice concerning
seafood consumption by pregnant and nursing women is based on the most
current and relevant nutritional science and appropriately takes into
account both the nutritional benefits, and any toxicological risks
associated with seafood consumption. I will also work to ensure
effective collaboration between FDA and the U.S. Environmental
Protection Agency (EPA) on this issue, and a range of other public
health matters over which both agencies share regulatory authority.
Question 10. According to the Centers for Disease Control and
Prevention, tobacco use is the leading cause of preventable death in
the United States, responsible for 1,300 deaths every day. I think
tobacco use needs more regulation, not less, which is why last Congress
I cosponsored the Tobacco to 21 Act, which would have raised the
minimum legal age for the sale of tobacco from 18 to 21. A number of
States and localities across the country have already recognized the
benefits of this policy and taken action to raise the age to 21. As FDA
Commissioner, you would be charged with working to enhance the health
and well-being of Americans, and there is clear evidence that access to
tobacco products does nothing but detract from it.
Do you believe FDA should have a role in regulating tobacco
products?
As Commissioner, will you support efforts to restrict the sale of
tobacco to young Americans?
Answer 10. Through the Tobacco Control Act (TCA), Congress gave FDA
regulatory responsibility over tobacco products. If confirmed, I will
be committed to implementing the TCA, as intended by Congress. I fully
support efforts to restrict the illegal sale of tobacco products to
minors and reduce smoking rates in this country. I believe responsibly
implementing the TCA is an integral part of FDA's core mission to
protect and promote public health.
Question 11. Will you support the ongoing efforts of the Center for
Tobacco Products to monitor retailer, manufacturer, importer, and
distributor regulatory compliance, as well as educate the public about
the health effects of tobacco use?
Answer 11. Yes. Ensuring compliance and educating the public are
important public health responsibilities for FDA, and an important
component of the goals of the TCA.
Question 12. In May 2016, the FDA announced changes to the
Nutrition Facts label for packaged foods in an effort to make it easier
for consumers to understand and make informed food choices. One
significant change is that the label will now include ``added sugars,''
including the grams of sugar and the percentage of a ``Daily Value''
that is included in a product. Evidence shows that while the sugars
found in fruits and other natural foods can be part of a healthy diet,
consuming large amounts of added sugar can make it difficult to meet
your body's nutritional needs. Will you ensure that the Nutrition Facts
label update, including the ``added sugar'' line, is implemented by the
current compliance date of July 2018?
Answer 12. I believe consumers should have access to standard,
understandable, and accurate information that they can use to make
educated decisions about a healthful diet. If confirmed, I will commit
to engaging with the agency's professional staff to quickly get up to
speed on this issue. I would welcome the opportunity to work with you
on this issue moving forward.
Question 13. It has been reported that President Trump, who has
publicly questioned the safety of vaccines and perpetuated the myth
that vaccines cause autism, will convene a Commission on Vaccine
Safety. The scientific community has overwhelmingly concluded vaccines
are safe and have saved countless lives. As FDA Commissioner under
President Trump, you may be charged with implementing an anti-vaccine
agenda.
Do you commit to protecting access to life-saving vaccinations?
Do you commit to ensuring that information about vaccine safety and
efficacy that is disseminated by the Trump administration reflects the
best available scientific evidence?
Answer 13. As I stated during my confirmation hearing before the
committee, this scientific question pertaining to vaccine safety and
autism is perhaps one of the most rigorously and exhaustively studied
public health questions in modern times. I believe the science with
respect to the question of there being any causal link between
childhood vaccines and autism is clear and settled. Vaccines are safe
and effective, and they are among the most impactful tools we have at
our disposal to protect and promote public health. If confirmed, I will
advise my colleagues and superiors within the Administration about the
scientific validity and public health value of vaccines and will
promote sound vaccination public policies.
Question 14. I have heard from medical device companies in Rhode
Island who are pleased with the medical device user fee agreement that
has been negotiated between FDA and industry, and would like to see it
enacted by Congress as negotiated. Yet, with only a few months left
before the agreements expire, President Trump has proposed doubling the
amount of user fees collected by the FDA. Have you reviewed the MDUFA
IV agreement, and do you support its enactment as negotiated?
Answer 14. The reauthorization proposals for PDUFA, MDUFA, GDUFA,
and BsUFA were developed and submitted to Congress prior to the end of
the previous Administration. I was not involved with the FDA-industry
technical negotiations on any of these proposals. I was also not
involved in the development of the President's Blueprint Budget. I
recognize these user fee programs are critically important to FDA, and
the patients the agency serves, as they provide significant resources
to support FDA's regulatory activities related to innovative and
generic medicines, biosimilars, and medical technologies. In order to
ensure FDA is adequately resourced to facilitate the discovery,
development, and regulatory review of safe and effective medical
products to help American patients, if confirmed, I will work with my
colleagues in the Administration, Congress, industry, and stakeholders
to reauthorize these critical user fee programs in a timely manner.
Question 15. In addition to challenges obtaining FDA approval,
innovative technologies can face additional hurdles getting authorized
for reimbursement once they are approved. There have been efforts to
make these two processes move on a parallel path, instead of one after
the other. In your role as FDA Commissioner, would you support efforts
to make these two approval processes work in tandem instead of
separately?
Answer 15. I have been supportive of the concept of parallel
review. If undertaken appropriately, it could facilitate more timely
patient access to safe and effective new FDA-approved medical products.
If confirmed, I would look forward to working with my colleagues at CMS
on this issue.
Question 16. Our prescription drug market relies on competition to
keep costs down for consumers. Generic competitors help give patients
more options and drive down drug prices across the board. However, some
brand-name drug manufacturers have engaged in behavior that makes it
more difficult for potential generic competitors to obtain samples of
their drugs--samples they need to be able to prove substantial
equivalence and get approval from the FDA. Or they refuse to agree on a
shared safety protocol for the drug, another FDA requirement. Last
Congress, I cosponsored the CREATES Act, which would have allowed a
generic company facing one of these delay tactics to bring action in
Federal court to obtain the sample it needs, or enter into court-
supervised negotiations for a shared safety protocol. Will you support
efforts to remove the incentives for companies to engage in these delay
tactics and improve opportunities to create generic competition?
Answer 16. My understanding is FDA has already taken action to
instruct manufacturers that they may make samples of products that are
subject to REMS available to prospective generic competitors for the
purpose of bioequivalence studies without running afoul of their legal
requirements. It is important to note though, that appropriate safety
precautions must be in place under specific circumstances, given the
unique patient safety and public health risks associated with certain
REMS products. We need to make sure our policies are striking the right
balance between safety and access, and evaluate carefully if
regulations meant to improve safety are also becoming an unintended
barrier to access and competition. If manufacturers inappropriately
refuse to provide their product to prospective generic competitors,
this would be a concern to FDA and become a matter for potential
enforcement action by the Federal Trade Commission. While drug pricing
does not fall directly within FDA's purview, I believe the agency can
play a key role on this important issue by taking steps to improve
product competition. If confirmed, I will work to ensure FDA has the
appropriate policies and processes in place to effectively facilitate
generic market entry and competition, especially for complex drugs that
sometimes do not face effective generic competition even long after the
patent expires and other places where there are specific issues that
make it hard for generic manufacturers to copy certain products and
demonstrate sameness under FDA's existing guidance. Reforming the
regulatory pathway for complex generic products would address one key
policy deficiency that results in unnecessary barriers to the
development and review of generic competitors for some branded products
for which traditional BE/BA testing alone are sometimes insufficient
for proving sameness.
Question 17. Increasing generic competition is one way the FDA can
support efforts to lower prescription drug prices. One issue in the
generic drug program has been deficient applications, requiring FDA and
the sponsor to go through multiple review cycles before a product can
be approved. The GDUFA II agreement includes changes that would give
sponsors more opportunities to address deficiencies in their
applications before FDA decides it does not have enough information to
proceed with its review.
Do you support efforts like those in GDUFA II to reduce the number
of review cycles for generic drug applications?
How else can the FDA work with sponsors to reduce the number of
review cycles needed and get products to market faster?
Answer 17. The reauthorization proposals for PDUFA, MDUFA, GDUFA,
and BsUFA were developed and submitted to Congress prior to the end of
the previous Administration. I was not involved with the FDA-industry
technical negotiations on any of these proposals. While drug pricing
does not fall directly within FDA's purview, I believe the agency can
play an important role on this important issue by taking steps to
improve product competition. If confirmed, I will work to ensure FDA
has the appropriate policies and processes in place to effectively
facilitate generic market entry and competition, especially for complex
drugs that sometimes do not face effective generic competition even
long after the patent expires. FDA should also explore options to
improve the efficiency and consistency of ANDA review processes and
timelines, so that financial speculators cannot engage in a regulatory
arbitrage, by dramatically hiking the price of some very old generic
drugs because they know it can take years for new generic competitors
to enter the market.
Question 18. While much of the conversation about high drug prices
has focused on innovative blockbuster drugs, like treatments for
hepatitis C, the prices of many older drugs are rising too. I heard
from the medical director of a health center in Providence that some of
her patients can no longer afford insulin, a drug that has been around
in some form for nearly a century. An insulin biosimilar was finally
brought to market at the end of 2016, and more products are in
development now, but Americans should not have to wait nearly 100 years
to be able to purchase a lower cost version of a drug.
What will you do to ensure drug companies can not abuse their
patent protections to keep generic competitors out of the market?
What steps will you take to incentivize potential competitors to
use the generic and biosimilar approval pathways to improve
competition, even for decades-old drugs?
Answer 18. If confirmed, I will work to ensure FDA has the
appropriate policies and processes in place to effectively facilitate
generic market entry and competition, especially for complex drugs that
sometimes do not face effective generic competition even long after the
patent expires. This could include certain insulin products. I am also
committed to working with the FDA professional staff to make sure the
agency has appropriate standards and guidance in place that help
balance the mandate to ensure with the safety and effectiveness of
biosimilars while creating efficient pathways for inclusion of the sort
of labeling claims on these products that will help facilitate their
broader adoption and product competition that can enhance patient
access.
Question 19. Last year, the FDA announced it would prioritize the
review of generic products that would compete with ``sole-source''
products, or drugs that have only one manufacturer. However, other
uncompetitive markets that have supply shortages or limited competition
could also be improved by approving competitors more quickly. Should
the FDA prioritize generic applications for market deficiencies caused
by factors other than there being a single manufacturer?
Answer 19. If confirmed, I am fully committed to looking at
additional ways that FDA can take steps, under the agency's existing
legal authorities, to facilitate more product competition and generic
market entry as a way to enhance patient access to medicines.
Question 20. Many agencies responsible for the approval of
prescription drugs in other countries take cost-benefit analyses and
comparative-effectiveness research into account during the approval
process. Although the FDA does not make decisions about the prices of
drugs or whether health insurance programs will cover them, could the
consideration of comparative-effectiveness research help inform the
FDA's decisionmaking?
Answer 20. The legal standard for FDA drug approval is whether a
drug is safe and effective for its intended use. I am committed to
working with Congress to consider additional ways that FDA could
potentially improve the quality of data used to inform the agency's
decisionmaking while maintaining its Gold Standard of safety and
efficacy.
Question 21. Both the PDUFA and GDUFA fee structures include
mechanisms to reduce the financial burden for small companies. Do you
support efforts like these to make the new drug and abbreviated new
drug application processes more accessible to small, innovator
companies? What else would you do to ensure small companies are not
priced out of the FDA review process?
Answer 21. I support policies that appropriately take into
consideration the unique challenges posed by small companies. Among the
many steps that we can take to ensure small and sometimes
undercapitalized companies are not priced out of the FDA review process
is to make sure the regulatory requirements are consistent,
transparent, efficient and based on the most modern science. FDA must
also take a risk-based approach to regulation, so that we are not
imposing unnecessary regulatory costs that are not achieving their
intended public health purpose.
senator cassidy
Question 1. The literature often speaks of the activated patient--
one engaged in their own health care. Ideally, each patient would be
engaged in their own health care decisions, and would choose the most
efficient expenditure of their health care dollar. However, in health
care, a given patient may have a plethora of options from which to
choose--this can be very true in the device space.
Patients are seeing an increasingly crowded field of devices.
However, it is very difficult for a typical patient to find the device
that would produce the best value for somebody with similar health
status. As a provider for 30 years, I can tell you that providers are
in the same boat--data on outcomes for medical products and services
certainly exist, but are not readily consumable.
Currently, the FDA regulatory structure incentivizes device makers
to make their products as similar to each other as possible, in order
to avoid lengthy and expensive premarket approvals. Dr. Gottlieb, do we
reduce incentives for patient-focused innovation in the device industry
this way?
For drugs, the FDA uses a rating system to differentiate between
drugs that provide a substantial improvement over current standards of
care--priority-rated drugs--and those that bat par with existing
options. While there is little reward for those more substantive
improvements today, this priority rating system has been well-developed
between patient groups, clinical experts, industry, and the FDA--and
serves as a clear signal to the health care system and to patients.
Dr. Gottlieb, could a parallel effort by the FDA in the device
space help us identify those devices which show big steps forward in
patient outcomes? Could this be done some time after market
introduction, based upon real world evidence?
Answer 1. If confirmed, I would be committed to working with you to
explore additional ways we might modernize the medical device review
process to ensure a proper balance between the need to ensure the
safety and effectiveness of these products, and appropriate steps to
facilitate innovation. Medical devices are important tools in the hands
of physicians, and much of the impactful innovation in devices have
been through successive improvements made over periods of time, often
based on the real world experience of providers. From a public health
perspective, we should support a regulatory process that helps
facilitate this sort of continuous innovation that can improve the
safety and effectiveness of products.
Question 2. It seems as though some drugs attain orphan drug status
for treating a subset of a condition, but go on to gain additional
approvals for treating the condition more broadly. Orphan products may
face a different set of standards for approval, and earn a different
amount of market exclusivity, compared with non-
orphan products. Dr. Gottlieb, what are your thoughts on our incentive
structure for orphan drugs, and how these incentives interact with
those drugs indicated for populations larger than orphan size?
Answer 2. The Orphan Drug Act has played an important role in
incentivizing manufacturers to develop treatments for rare diseases.
Given the small patient populations and inherent challenges associated
with clinical trials for rare diseases, I believe we must protect these
incentives and assess whether additional incentives may be necessary to
spur the development of safe and effective new treatments for patients
with rare diseases. We must also make sure that the Orphan Drug Act
isn't being used in ways that Congress did not intend. If confirmed, I
pledge to work with Congress, patients, industry, and other
stakeholders to make sure the Orphan Drug Act continues to achieve its
important public health goals.
senator baldwin
Question 1. Wisconsin has been a leader in advancing new
technologies to better diagnose and screen for disease. An innovative
company in Madison was the first to receive FDA approval for a new
stool-based DNA test for colorectal cancer. But, in recent years, we
have seen significant growth of the laboratory-developed test (LDT)
industry, which are not approved by FDA. I am concerned with this
uneven oversight of critical diagnostics, and believe that FDA
oversight of LDTs must be appropriately updated and modernized. You
have stated that ``the regulation of lab tests is best left to a more
robust CLIA [or framework through CMS], and not FDA.'' \42\ If
confirmed, would you support FDA as the primary regulator of
diagnostics, including LDTs?
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\42\ http://www.aei.org/publication/theranos-woes-offer-lesson-in-
how-labs-should-be-regula-
ted/.
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Answer 1. Defining an appropriate regulatory framework for
Laboratory Developed Tests (LDTs) is important to FDA's mission to
protect and promote public health. In order to both protect patient
safety and encourage innovation and patient access, I believe we must
strike the right balance between Clinical Laboratory Improvement
Amendments (CLIA) and FDA regulation and regulatory requirements. If
confirmed, I would commit to working with Congress and stakeholders to
develop appropriate LDT regulatory policies.
Question 2. As we discussed when we met, dairy farmers in Wisconsin
pride themselves on producing high quality milk that meets very
specific requirements. In fact, many of those requirements are set by
the FDA's standards of identity regulations. Despite the constant work
farmers do to meet these standards, there are many imitation products
on the market today that get away with using dairy terms without
meeting the standard of identity for that product. This is unfair and
I, along with thousands of farmers across America, am very frustrated
that FDA has not enforced its own regulations. If confirmed, how will
you enforce FDA regulations against all plant-based imitation products
that use dairy terms?
Answer 2. While I am not personally familiar with the current
discussion inside FDA related to this specific issue, I strongly
support accurate product labeling and consistent and effective
implementation of laws Congress passes and the rules FDA promulgates.
If confirmed, I will commit to engaging with the agency's professional
staff to quickly get up to speed on this issue. I would welcome the
opportunity to work with you on this issue moving forward.
Question 3. Dairy farmers have been waiting for FDA to address this
for years without action, and they are tired of waiting. That is why I
introduced the DAIRY PRIDE Act earlier this year--my bill would require
FDA to enforce its own regulations within 60 days. But the agency
should be doing this already. If confirmed, will you commit to
beginning enforcement within 60 days?
Answer 3. If confirmed, I commit to implementing congressional laws
according to the timelines prescribed by the law and to working with
your office on this issue.
Question 4. According to the Institute of Medicine, nearly 50
percent of all cancer patients experience distress. Further, studies
suggest that distress in cancer patients leads to higher healthcare
costs, less compliance with treatment pathways and poorer health
outcomes. While we have seen significant advancements in biomedical
treatments, there remain barriers to more effective development of
therapies. I am encouraged by FDA's enhanced focus on considering
patient experience data when assessing treatments. I believe that FDA
should explore mechanisms to capture data beyond just disease symptoms
and physical functioning to include psychosocial health measures,
including distress screening (e.g., concerns related to disruption of
work/family life [due to the regimen], concerns related to nutrition,
financial impact and others). This would provide meaningful patient
feedback about issues that may not be identified through the current
measures being used in the clinical trial process. How would you work
to enhance FDA's patient-reported outcomes work to include
comprehensive and reliable measures on patient experiences that capture
physical and psychosocial symptoms?
Answer 4. I strongly agree that patient experiences, preferences,
and perspectives should play an important and appropriate role in FDA's
regulatory policymaking and decisionmaking. Among other approaches, I
have advocated that we continue to advance well-validated, scientific
tools for incorporating Patient Reported Outcomes (PROs) as endpoints
in clinical trials. I support these and other measures that would allow
meaningful patient feedback to be incorporated into regulatory
decisionmaking to better define patient-experience data that may not be
identified through the current measures being used in the clinical
trial process. If confirmed, I look forward to working with you on this
issue.
Question 5. You have argued that the FDA should allow drug
companies to promote uses for drugs that have not yet been FDA
approved--a position the drug industry shares--because FDA labels, are
``slow to incorporate important medical results about the effectiveness
of medical products.'' \43\ The agency has been working to improve the
accuracy of labels. For instance, FDA proposed a rule to ensure that
labels for generics drugs were rapidly updated to include the latest
safety information. However you, along with the generic drug industry,
opposed FDA finalizing this rule. You have said this is because it
would ``expose the generic-drug industry to the same kind of costly
product liability suits that plague branded-drug makers'' \44\ I am
concerned with these seemingly contradictory positions that suggest--at
least when it comes to drug labels--a potential bias in favor of
positions supported by the biggest industry stakeholder. As FDA
Commissioner, responsible for guaranteeing that patients have the most
up-to-date and accurate information about drugs, what principles would
you use to determine the best communication and labeling standards?
---------------------------------------------------------------------------
\43\ http://healthaffairs.org/blog/2008/04/23/from-fda-a-good-
framework-for-distributing-in-formation-on-off-label-uses/.
\44\ https://www.aei.org/publication/how-obamas-fda-keeps-generic-
drugs-off-the-market/.
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Answer 5. I believe it is important that generic drug labels be
kept up-to-date and generic manufacturers engage in appropriate post-
market safety surveillance. The FDA proposed rule would alter the legal
responsibilities of generic firms. If confirmed, I will work with
agency's staff as we consider future regulatory actions. As a general
matter, I am philosophically in favor of approaches to enable providers
to get more and timelier truthful, non-misleading information about the
medical products they prescribe, in a non-promotional context.
Question 6. Among its many roles, FDA plays a key part in helping
ensure that U.S. companies that export foods such as dairy products are
able to keep tapping into those foreign markets and thereby support
American jobs. Can you assure me FDA will prioritize working with its
interagency partners to swiftly resolve export certification issues in
order to keep those foreign markets open to U.S. products?
Answer 6. I will commit to working with our interagency partners on
this issue. I recognize the importance of maintaining access to these
foreign markets for our U.S. companies.
senator hatch
Question 1. Dr. Gottlieb, one issue that was not really addressed
by the 21st Century Cures law as enacted is how reimbursement and
coding issues can affect the availability of needed treatments. While
we recognize that reimbursement is not directly within the purview of
FDA, your mission in speeding safe and effective treatments and cures
to patients has a direct relationship to how quickly patients can
benefit from medical products FDA approves. This is particularly
important for new and innovative products, both drugs and devices. It
strikes us that your background at both CMS and FDA puts you in a
unique position to make a valuable contribution toward addressing this
issue. Our question is simple: When confirmed, will you take steps to
partner with CMS to work on ways to make certain that new therapies in
the critical areas you mentioned at your hearing are made available to
patients as early as possible?
Answer 1. I believe my prior experiences at both FDA and CMS will
help guide my work as Commissioner, if confirmed. As you mentioned,
reimbursement and coding issues are outside the purview of FDA, but I
agree that there are areas where CMS and FDA could work more closely
together to increase the availability of innovative therapies. I commit
to working with my colleagues at CMS and you on this issue.
Question 2. Since its inception, the Orphan Drug Act has fueled
treatment discoveries, and even cures, for rare diseases that would
probably remain untreated to this day without the Orphan Drug Act's
incentives. We still have about 95 percent of the over 7,000 identified
rare diseases remaining with no FDA-approved treatment option. Many of
these disorders have prevalence rates of several hundred to several
thousand patients, leaving little commercial incentive for investment.
As such, what do you anticipate doing to ensure that patients with
these extremely rare conditions, and innovators, see FDA as an
effective partner toward approving new treatments and cures?
Answer 2. The Orphan Drug Act has played an important role in
incentivizing innovators to develop treatments for rare diseases. Given
the small patient populations and inherent challenges associated with
clinical trials for rare diseases, I believe we must protect these
incentives and assess whether additional incentives may be necessary to
spur the development of safe and effective new treatments for patients
with rare diseases. If confirmed, I pledge to work Congress, patients,
industry, and other stakeholders to make sure the Orphan Drug Act
continues to achieve its important public health goals. Given the
unique challenges to developing drugs for extremely rare conditions, we
also must continue to make sure that we are using the most modern
regulatory science in order to make the process for developing safe and
effective drugs targeted to extremely rare diseases as efficient as
possible.
Question 3. Every day, millions of Americans safely take one or
more dietary supplements. As consumers continue to take greater control
of their health, it is important that they have access to safe products
that fit their needs. The FDA currently has the oversight authority to
remove any product it finds is unsafe and to take enforcement action to
remove the product from the market. Dr. Gottlieb, do you believe the
FDA's current regulatory framework under DSHEA provides for adequate
enforcement tools to remove unsafe dietary supplements from the market?
Answer 3. As someone who uses dietary supplements every day, I
believe they serve an important role in health promotion for millions
of Americans and I support consumer access to these products. I believe
the regulatory framework established under DSHEA is the right one, and
if confirmed, I would commit to enforcing DSHEA, as intended by
Congress.
Question 4. One of the world's most pressing health problems is the
emergence of bacterial infections that are resistant to antibiotics.
According to the Centers for Disease Control and Prevention (CDC),
every year at least 2 million Americans get sick with antibiotic-
resistant infections. Sadly, an estimated 23,000 people die as a
result. As bacterial strains consistently evolve and adapt to current
drug therapies, providers struggle to improve upon documented over-
prescription of antibiotics. Moreover, the Nation faces a lack of new
antibiotics in the development pipeline, leading researchers to
estimate that 10 million people annually could die from drug resistant
infections by the year 2050.
While reducing the inappropriate and unnecessary use of antibiotics
can slow how quickly bacteria become resistant to currently utilized
drug therapies, this alone is unlikely to solve the problem.
Manufacturers must develop new antibiotics. Yet reduced antibiotic
discovery, gaps in scientific research, and poor return on investment
pose significant barriers to developing new, novel antibiotics.
Congress has responded with a number of initiatives to respond to this
crisis. Specifically, in 2012, legislation I authored called the
Generating Antibiotic Incentives Now (GAIN) Act was signed into law.
This law gave companies enhanced tools that encourage development of
new antibiotics and allowed an expedited FDA approval process for
antibiotics that treat life-threatening infections. Last Congress, I
also authored legislation that was included in the 21st Century Cures
Act to speed the research and development of new antibiotics that would
address an unmet medical need by establishing a limited population
antibiotic approval pathway that allowed smaller, faster clinical
trials. These laws have had an impact, but more action is needed to
increase development of novel therapies.
Dr. Gottlieb, in 2013, you co-authored an article printed in The
RPM Report titled ``Paying for New Drugs for New Bugs: Regulation is
Only One Side of the Coin''. In that article you assert that, while the
FDA and Congress are making headway to improve the regulatory
environment for antibiotic development, that these changes are not
sufficient without adjustments to reimbursement. What do you believe
Congress should be doing, in consultation with the Administration, to
spur innovation in the antibiotic class of prescription drugs?
Answer 4. As the nominee to be the next Commissioner of Food and
Drugs, I do not believe it would be appropriate to comment on questions
about issues that are outside the jurisdiction of FDA. With regard to
FDA's role in this important issue, I believe the availability and
appropriate prescribing of antibiotics are vital to our Nation's public
health. Additionally, antibiotic resistance is a significant and
growing public health challenge facing our Nation. Within its statutory
authorities, FDA should encourage the development of new antibiotics
and ensure proper labeling to help address the issue of inappropriate
prescribing and/or use. If confirmed, I would commit to fully
implementing the law as it relates to these issues, and in particular,
the Limited Population Approval Pathway for antibiotics.
Question 5. The 21st Century Cures Act requires FDA to update
guidance and regulations for regenerative therapeutic products and to
hold a public meeting to encourage innovation. This is a great first
step, and we are hopeful that you will prioritize the potential of
these products as the new Commissioner of the FDA. If you are
confirmed, how do you envision FDA's regulatory framework for these
cutting-edge treatments and therapies now and in the future?
Answer 5. Regenerative medicine is one of the most innovative and
promising emerging advancements in our scientific approaches to the
treatment of human disease. Regenerative medicine appears to hold great
promise for new therapeutic options for patients and physicians,
particularly in areas of unmet or underserved medical need. However, as
with all products FDA regulates, the agency must have the appropriate
policies and processes in place to assess and ensure the safety and
efficacy of regenerative medical products before they are approved for
use by American patients. FDA must ensure patients and providers are
appropriately educated about the potential risks and benefits of
regenerative medicine therapies that fall within the scope of FDA's
oversight, and that these products meet the agency's standard for
safety and effectiveness. If confirmed, I will embrace the
responsibility to facilitate important medical innovation in the
regenerative medicine space, while maintaining the Gold Standard of
safety and efficacy.
Question 6. There has been much discussion on the use of biomarkers
in the context of rare disease drug development and the FDA has a
formal procedure to qualify such biomarkers. To your knowledge, has the
FDA ever qualified a biomarker as a surrogate efficacy outcome measure
to expedite the full approval of therapeutics for rare diseases? How
would you seek to balance discussions on the utility of biomarkers
between fast and slow adopters at the agency?
Answer 6. I am not aware of whether the FDA has ever qualified a
biomarker as a surrogate efficacy outcome measure to expedite the full
approval of therapeutics for rare diseases. If confirmed, I will ensure
that FDA is using the most current science to maximize the potential of
biomarkers to advance drug development and that we bring consistency
across the agency with respect to the application of modern and well-
validated scientific and regulatory principles.
Question 7. Dr. Gottlieb, 21st Century Cures contained provisions
that specifically exempt certain low-risk software functions from FDA
pre- and post-market regulatory requirements. However, the law provides
a mechanism for the FDA to pull a product back under FDA jurisdiction
if there is a determination that a product ``would be reasonably likely
to have serious adverse health consequences.'' How do you plan to
operationalize this provision in a way that provides as much clarity to
innovators as possible?
Answer 7. I support tools that can help provide consumers with more
information about their health. FDA must clearly define when an app is
a medical device subject to its regulation, and when it falls outside
FDA's framework or is low risk, and therefore appropriate to be exempt
from the full scope of FDA's pre-market requirements. For products
subject to FDA's pre-market requirements, the agency should use a risk-
based approach in regulating consumer apps and other medical software,
and, if confirmed, I look forward to helping further refine FDA's
approach and implement the related provisions of 21st Century Cures.
senator murkowski
Question 1. FDA's menu-labeling rule, even after an initial stay,
will take effect 1 month from today. Grocery stores and other food
retailers across America continue to be frustrated with FDA's handling
of things, including for local and seasonal food items. Fresh and local
food items may be sold at a few stores, under the same name, but the
ingredients or recipe can vary, yet they would be considered ``standard
menu items'' and subject to enforcement. The irony is that this will
cause stores and restaurants to move away from fresh, local, and
seasonal offerings. With just a month before the compliance date, we
need FDA to act quickly to further delay, withdraw, or stay the rule so
it can be rewritten to give businesses the flexibility to comply. Would
you be willing to explore ways to encourage FDA to act before the
compliance date to provide this much-needed flexibility for businesses?
Answer 1. While I am broadly aware of the menu labeling issue, this
is not a matter on which I am familiar with the technical specifics. As
a general rule, I support providing clear, accurate, and understandable
information to American consumers to help inform healthful dietary
choices. I believe information about caloric content can be a useful
tool. However, I am mindful of the unique challenges that developing
and communicating such information can pose, particularly on small,
independent businesses. If confirmed, I will commit to working with the
agency's staff to quickly get up to speed on the regulatory history
related to menu labeling, as well as FDA's latest thinking and actions.
I would welcome the opportunity to work with Congress and stakeholders
to ensure any regulatory requirements would promote public health by
providing helpful information to consumers, while not placing
unnecessary compliance burden on businesses, particularly small,
independent ones.
Question 2. Dr. Gottlieb, one issue that was not really addressed
by the 21st Century Cures law as enacted is how reimbursement and
coding issues can affect the availability of needed treatments. While
we recognize that reimbursement is not directly within the purview of
FDA, your mission in speeding safe and effective treatments and cures
to patients has a direct relationship to how quickly patients can
benefit from medical products FDA approves. This is particularly
important for new and innovative products, both drugs and devices. It
strikes us that your background at both CMS and FDA puts you in a
unique position to make a valuable contribution toward addressing this
issue. Our question is simple: When confirmed, will you take steps to
partner with CMS to work on ways to make certain that new therapies in
the critical areas you mentioned at your hearing are made available to
patients as early as possible?
Answer 2. I believe my prior experiences at both FDA and CMS will
help guide my work as Commissioner, if confirmed. As you mentioned,
reimbursement and coding issues are outside the purview of FDA, but I
agree that there are areas where CMS and FDA could work more closely
together to increase the availability of innovative therapies. I commit
to working with my colleagues at CMS and you on this issue.
senator murphy
Question 1. Dr. Gottlieb, in 1978 FDA committed to address the
overregulation of medical gases, like Oxygen, by creating separate
regulations for medical gases as a unique class of drug products.
Thirty-nine years later, FDA has not followed through on its commitment
to create an appropriate framework specific to medical gases. In 2012,
Congress enacted historic and bipartisan reforms at the request of the
medical gas industry and pharmacists to require FDA to follow through
on its 1978 commitment to address the overregulation of medical gases
in the Food and Drug Administration Safety and Innovation Act (FDASIA).
In November 2016, FDA issued a final rulemaking that addressed some
medical gas labeling issues, however FDA did not, as intended by FDASIA
and reiterated to FDA in the fiscal year 2016 Appropriations report
language, modify current regulations to address other aspects that are
unique to medical gases. For example, the November 2016 rule did not
address medical air labeling, adverse event reporting, expiration
dating, calculation of yield and a host of other safety and enforcement
issues identified by the industry as necessary to appropriately
regulate medical gases.
If confirmed as Commissioner of FDA, would you ensure that FDA
fully implements Section 1112 of FDASIA by working with stakeholders to
either incorporate by reference industry consensus standards or issue
new final rulemakings on medical gas to address these unique medical
gas regulatory issues?
Answer 1. If confirmed, I am committed to implementing all
congressional laws, including Section 1112 of FDASIA. I look forward to
working with you on this issue.
Question 2. In April 2016, the FDA proposed to ban electrical
stimulation devices (ESDs) used for self-injurious or aggressive
behavior because they present an unreasonable and substantial risk to
public health and they have been used on children with intellectual and
developmental disabilities as young as 9 years old. This step was the
result of deliberative discussion and consideration about the harmful
nature of these devices. The proposed rule stated that the FDA
determined that ESDs presented a number of psychological and physical
risks, including depression, fear, panic, aggression, pain, burns and
errant shocks from device misapplication or failure. Moreover, ESDs
have been associated with additional risks such as suicidality, chronic
stress, acute stress disorder and hypervigilance. It is not surprising
that this is the case because as Dr. Margaret Nygren from the American
Association of Intellectual and Developmental Disabilities noted during
the April 2014 advisory committee meeting,
``These devices are explicitly intended to inflict pain. The pain
is not an unfortunate risk or byproduct of the intervention. With these
devices, the pain is the intervention.''
I, along with a number of my colleagues, wrote to then Commissioner
Califf to support this proposed ban. In that letter, we stated, ``The
use of these electric shock devices as aversive therapy for individuals
with developmental disabilities is inhumane, especially since many of
these individuals have difficulty communicating and alternative
effective treatment options are available. Put simply, it is outrageous
that this practice is allowed in the United States for this vulnerable
population and it should be stopped immediately. As such, we urge you
to finalize the proposed rule as quickly as possible.''
Leading disability organizations and advocates also support
finalizing the proposed ban. A letter from two dozen of these groups
from July 25, 2016, stated,
``We applaud the FDA for taking this critically important
step, which we believe is long overdue. For too many decades,
children with disabilities have been subjected to physical and
psychological abuse through the use of these devices, and have
experienced pain, trauma, suffering and long-term harms.''
If confirmed, would you ensure that the FDA moves forward with the
proposed ban of electrical stimulation devices used to treat self-
injurious or aggressive behavior?
Answer 2. While I am not personally familiar with the specific
details of this issue, if confirmed, I commit to engaging with the
agency's professional staff to get up to speed on this issue and
reviewing the agency's scientific opinions and input from stakeholders,
including Congress.
Question 3. The CDC estimates that 23,000 Americans die each year
from drug-resistant infections. The World Health Organization predicts
that without urgent action, we are headed toward a ``post-antibiotic''
future where common infections and minor injuries will once again kill.
The over-use of broad-spectrum antibiotics is a major contributor
to the rise of these super bugs. Antibiotic stewardship--more judicious
use of antibiotics in medical practice--is therefore an important
public health initiative. One way we can reduce the prescribing of
broad-spectrum antibiotics is to make better use of penicillins, which
are less costly, have fewer side effects and are not known to give rise
to some of the most worrying resistant bugs, like C. difficile.
More than 30 million people in the United States self-report as
penicillin allergic. Most of these patients are prescribed a broad-
spectrum antibiotic in place of penicillin or amoxicillin. Yet, the
American Academy of Allergy, Asthma and Immunology has said that less
than 10 percent of those people actually have the allergy, and with
improved penicillin allergy testing, we could make much better use of
penicillins.
The CDC and other public health organizations have been educating
the community on the need for penicillin allergy testing to confirm
current allergy, before resorting to non-penicillin broad spectrum
antibiotics.
Do you agree that penicillin allergy testing could help us be
better stewards of the antibiotic drug supply and do you agree that it
is important for public health that FDA work with sponsors to approve
tests for penicillin allergy testing?
Answer 3. Yes, I believe penicillin allergy testing could be an
important piece of addressing antibiotic drug resistance, and that we
should advance technologies that can help widen such testing and make
sure that the testing that is done is safe and reliable. I look forward
to working with CDC and other public health organizations on this
issue.
Question 4. How can the FDA promote research transparency and
clinical data sharing so that patients, healthcare providers, and
independent researchers have access to information about the risks and
benefits of medical products? How can the FDA be more transparent with
key decisions in its own review process for medical products?
Answer 4. I am a strong proponent of data transparency--for
patients, physicians, and manufacturers. I have long advocated that the
FDA release more information related to its own review process that
could be used to better inform consumers and product developers alike.
If confirmed, I will be committed to working with Congress, patients,
industry, and stakeholders on the issue of data transparency and new
ways that FDA could potentially make the importance of its own
information and deliberations more readily available to the public.
Question 5. New innovations in wearable wellness technology are
empowering consumers by providing access to health data, including
steps taken, hours slept, and heart rate. It is my understanding the
FDA's current policy is that these devices do not require FDA approval
as long as the information is not used by a medical professional to
make a diagnosis.
As wearable technology continues to advance, such as warning
consumers about an abnormal heart rate reading, where do you see the
line between a medical device and a consumer application?
Answer 5. I support tools that can help provide consumers with more
information about their health. FDA must clearly define when an app is
a medical device subject to its regulation, and when it falls outside
FDA's framework or is low risk, and therefore appropriate to be exempt
from the full scope of FDA's pre-market requirements. For products
subject to FDA's pre-market requirements, the agency should use a risk-
based approach in regulating consumer apps and other medical software,
and, if confirmed, I look forward to helping further refine FDA's
approach and fully implement the related provisions of 21st Century
Cures.
Question 6. Connecticut is a State with a growing number of small
farms. These small farmers face unique challenges when adapting to new
food safety regulations. I have led my colleagues in letters urging the
FDA to expedite implementation of Sec. 209 of the Food Safety
Modernization Act (FSMA), which includes a provision that provides
technical assistance and training for small farmers, small producers,
and fruit and vegetable merchant wholesalers in order to reduce
confusion surrounding new regulations and promote effective
implementation.
Can you commit to ensuring FSMA implementation will continue to
have a focus on the unique challenges faced by small farmers?
Answer 6. The Food Safety Modernization Act (FSMA) provides FDA
with important tools and authorities to support its responsibility to
ensure the safety of our Nation's food supply. If confirmed, I will
work to ensure FDA has the appropriate policies, processes, and
resources in place to implement FSMA, as intended by Congress. I agree
that FDA should implement FSMA in a way that protects and promotes
public health by enhancing food safety, while also collaborating with
the U.S. Department of Agriculture, State officials, and other Federal
and State Government agencies to conduct regulatory activities in a
manner that takes into account the unique challenges faced by small
farmers and small businesses.
Question 7. Sesame is now one of the most prevalent, and most
dangerous, food allergies in the United States. I have heard from many
Connecticut constituents about the need for the FDA to require that
sesame seeds and sesame products be labeled as such, in a manner
similar to the other eight major allergens. Sesame allergies are
particularly severe and, for some, sesame exposure can trigger fatal
anaphylaxis. Allergists estimated 300,000 to 500,000 people in the
United States have sesame allergies.
Given the severity and number of sesame allergies, what is the FDA
doing to address this problem? What more can the FDA do under its
current authority in the Food Allergen Labeling and Consumer Protection
Action to require sesame labeling?
Answer 7. I am not aware of what FDA has reviewed or done to date
with respect to sesame and labeling. If confirmed, I look forward to
reviewing the Food Allergen Labeling and Consumer Protection Act to
understand what authority FDA may have under the current law with
regard to this issue.
Question 8. Nationally, the production value of farmed oysters and
clams is growing at a rate of 8 percent a year, with some areas of the
country like the Northeast growing at a much higher rate of 31 percent.
With this surge in production, producers in my home State of
Connecticut are anxiously looking to expand to new markets. The EU and
United States have been in trade negotiations to open up trade between
two U.S. States--Massachusetts and Washington--and two EU countries--
Spain and the Netherlands. Negotiators finally arrived at a compromise
that is designed to resolve the issue and technical audits were
performed in 2015 that satisfied the health concerns of the respective
health agencies. Connecticut producers are anxious for this agreement
to be finalized so trade can begin and agencies can start discussions
to further expand trade to new U.S. States and EU countries.
What progress has the FDA made toward finalizing an equivalence
determination with the European Union? What more can the FDA do to
accelerate the process of finalizing this agreement and publish a
notice in the Federal Register? What can we do to ensure that the other
interested States gain access in a timely fashion?
Answer 8. I am not aware of where FDA is in the process of
finalizing an equivalence determination with the EU. I commit to
reviewing this issue, and working with your office on this matter, if
confirmed. If FDA is able to finalize the agreement, then I would work
to ensure that other States interested in participating do not face
lengthy waits.
Question 9. Connecticut shade tobacco, which is used as a wrapper
for premium cigars, has been grown in Connecticut for centuries and is
sought after for its unique flavor profile. I recently wrote to the FDA
about an issue Connecticut tobacco farmers face regarding potential
misbranding of foreign tobacco products.
Will you commit to working with me going forward to address this
issue and assist Connecticut's small tobacco farmers?
Answer 9. Yes, I commit to working with you on this issue, if
confirmed.
senator roberts
Question 1. FDA's menu-labeling rule, even after an initial stay,
will take effect 1 month from today. Grocery stores and other food
retailers across America continue to be frustrated with FDA's handling
of things, including for local and seasonal food items. Fresh and local
food items may be sold at a few stores, under the same name, but the
ingredients or recipe can vary, yet they would be considered ``standard
menu items'' and subject to enforcement. The irony is that this will
cause stores and restaurants to move away from fresh, local, and
seasonal offerings. With just a month before the compliance date, we
need FDA to act quickly to further delay, withdraw, or stay the rule so
it can be rewritten to give businesses the flexibility to comply. Would
you be willing to explore ways to encourage FDA to act before the
compliance date to provide this much-needed flexibility for businesses?
Answer 1. While I am broadly aware of the menu labeling issue, this
is not a matter on which I am familiar with the technical specifics. As
a general rule, I support providing clear, accurate, and understandable
information to American consumers to help inform healthful dietary
choices. I believe information about caloric content can be a useful
tool. However, I am mindful of the unique challenges that developing
and communicating such information can pose, particularly on small,
independent businesses. If confirmed, I will commit to working with the
agency's staff to quickly get up to speed on the regulatory history
related to menu labeling, as well as FDA's latest thinking and actions.
I would welcome the opportunity to work with Congress and stakeholders
to ensure any regulatory requirements would promote public health by
providing helpful information to consumers, while not placing
unnecessary compliance burden on businesses, particularly small,
independent ones.
Question 2. There has been much discussion recently about how FDA
restricts what drug makers and medical device manufacturers can say
about their products. Legislation has been introduced in the House of
Representatives and there have been high profile lawsuits surrounding
FDA's actions and the First Amendment. There are many treatments that
have additional uses that are medically accepted, that doctors can
prescribe for their patients, and that the Federal Government will even
pay for--but FDA prohibits the manufacturers, who know the most about
their products, from discussing those uses under most circumstances.
Do you have ideas on how to modernize or improve how FDA regulates
communication between doctors, patients and payers, while making sure
information is truthful and not misleading? How do we strike the right
balance to allow more communication while maintaining incentives to get
additional indications for therapies approved?
Answer 2. Medical product labels are one of the primary tools FDA
uses to promote the appropriate use medicines and technologies and
communicate risk information. It is important that information on
product labels is accurate, clear, and scientifically based; and be the
result of a sound regulatory process. Further, it is crucial that
manufacturers continue to develop and submit to the agency clinical
data demonstrating the safety and efficacy of medical products for new
indications they seek to include on labeling and in their marketing
communications with patients, payers, and providers. I also believe
that patients and physicians make the best decisions when they have
access to as much truthful, non-misleading, scientifically based
information as possible. So, I believe and FDA has similarly maintained
that there is some public health benefit in certain contexts of
allowing non-promotional communication about truthful, non-misleading,
clinical data that is not already incorporated into FDA-approved
product labeling. If confirmed, I will commit to working with FDA's
staff to get up to speed on the agency's latest thinking and actions on
these matters, and providing clarity to manufacturers, payers,
providers, and patients about acceptable, truthful and non-misleading
communications related to clinical data not already incorporated in a
label.
Question 3. Various U.S. agencies, including the FDA, are tasked
with implementing laws and furthering policies that are science-based
regarding food and beverages. We expect the same from other countries
and our partners abroad. Decisions based on science provide
predictability and certainty for the value chain, as well as a level
playing field for all countries. In late 2015, the World Health
Organization International Agency for Research on Cancer (IARC)
classified the consumption of red and processed meat as probably
carcinogenic and carcinogenic to humans, respectively. While then USDA
Secretary Tom Vilsack did publically state that U.S. dietary guidelines
rely on science regarding healthful meat consumption, in December 2016,
the Food Safety and Inspection Service was petitioned to inform the
public about the ``risk'' of cancer from processed meat. This request
relied strongly upon the IARC classification. In addition, when issuing
draft guidance on sodium reduction in June 2016, FDA relied in part on
a recommendation of the Dietary Guidelines Advisory Committee--which
has specifically come under intense criticism for cherry-picking its
science.
Under your leadership, what efforts would the FDA undertake to
ensure that the U.S. Government inquires and responds to these types of
findings and data in a science-based manner? Individual countries also
pass food and beverage safety labeling laws that are not grounded in
sound science. How will the FDA support a strong U.S. Government signal
to such countries regarding the need for science-based laws and
regulations for food and beverages? How will FDA ensure that its own
guidance and other decisions will be thoroughly science-based?
Answer 3. I agree that FDA should base its regulatory decisions on
sound and rigorous science. If confirmed, I will work to ensure that
FDA possesses the scientific expertise necessary to inform these
decisions.
Question 4. Recently, FDA has been pursuing considerable regulatory
change as it relates to the use of antibiotics in agriculture,
specifically their use in animals. You will certainly get pressure to
``do more'' and to limit certain uses of antibiotics, something the FDA
has requested comment on regarding how to apply judicious use policies.
Much scientific evidence has proven that healthy animals produce
healthy food. Keeping animals from getting sick and preventing disease
outbreaks should be a top priority. Allowing veterinarians and others
in agriculture the flexibility to prevent disease outbreaks is critical
for FDA to consider.
Will you work with, and be responsive to, the concerns of the
agriculture and veterinary industries on this issue?
Answer 4. Antibiotic resistance is a significant and growing public
health challenge facing our Nation. In addition to measures FDA should
take to address this issue within the context of human use, the agency
must effectively collaborate with other government agencies and public
health authorities to develop policies and processes to address the
issue of antibiotic use in animals intended for human consumption. If
confirmed, I will ensure FDA remains engaged on this important public
health issue, making sure that animal drug labeling reflects the most
up-to-date science, and working closely with the U.S. Department of
Agriculture, the Centers for Disease Control, the U.S. Department of
Defense, and other appropriate government agencies. FDA should also
consider input from, and be responsive to, other important
stakeholders, such as the farmers, the agriculture industry, and
veterinarians. If confirmed, I would commit to working closely with
these important stakeholders.
senator warren
Question 1. Approximately 48 million Americans experience age-
related hearing loss, including over half of adults between the ages
70-79.\45\ Yet only a small share of Americans with hearing loss--
around 14 percent--use assistive hearing technologies, primarily
because they cannot afford to buy costly hearing aids.\46\ Hearing aids
are not covered by Medicare or most private insurance plans, and out-
of-pocket costs for a single hearing aid average $2,400--far out of
reach for many consumers.\47\ Senators Grassley, Isakson, Hassan, and I
recently introduced bipartisan legislation that would make certain
types of hearing aids available over the counter (OTC).\48\ In December
2016, the FDA announced a commitment to examining OTC hearing aids and
said it will no longer enforce the medical waiver requirement
associated with hearing aids. In making this announcement, FDA
Commissioner Califf stated that the guidance would support consumer
access, ``while the FDA takes the steps necessary to propose to modify
our regulations to create a category of OTC hearing aids that could
help many Americans improve their quality of life through better
hearing.'' \49\
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\45\ Frank R. Lin, John K. Niparko, and Luigi Ferrucci. 2011.
``Hearing Loss Prevalence in the United States,'' Archives of Internal
Medicine 171: 1851-53 (online at: http://www.ncbi
.nlm.nih.gov/pmc/articles/PMC3564588/).
\46\ National Academies of Sciences, Engineering, and Medicine.
2016. Hearing Health Care for Adults: Priorities for Improving Access
and Affordability. Washington, DC: The National
Academies Press (online at: http://www.nationalacademies.org/hmd/
Reports/2016/Hearing-Health-Care-for-Adults.aspx).
\47\ President's Council of Advisors on Science and Technology,
Aging America and Hearing Loss: Imperative of Improved Hearing
Technologies (October 2015) (online at: https://www.whitehouse.gov/
sites/default/files/microsites/ostp/PCAST/pcast_hearing_tech_lette_
report_final.pdf), p.1. National Academies of Sciences, Engineering,
and Medicine. 2016.
Hearing Health Care for Adults: Priorities for Improving Access and
Affordability.
Washington, DC: The National Academies Press (online at: http://
www.nationalacademies.org/hmd/Reports/2016/Hearing-Health-Care-for-
Adults.aspx), p. 21-2. Sergei Kochkin. 2007.
``MarkeTrak VII: Obstacles to Adult Non-User Adoption of Hearing
Aids,'' The Hearing Journal
60: 24-50 (online at: http://www.betterhearing.org/sites/default/files/
hearingpediaresources/
MarkeTrak%20VII%20bstacles%20to%20adult%20nonuser%20adoption%20of%20hear
ing%20aids
.pdf). Karl E. Strom. 2014. ``HR 2013 Hearing Aid Dispenser Survey:
Dispensing in the Age of Internet and Big Box Retailers,'' The Hearing
Review 21 (4): 22-28 (online at: http://www.hearingreview.com/2014/04/
hr-2013-hearing-aid-dispenser-survey-dispensing-age-internet-big-
boxretailers-comparison-present-past-key-business-indicators-
dispensing-offices/).
\48\ S. 670 (115th Congress)--Over the Counter Hearing Aid Act of
2017 (online at:
https://www.warren.senate.gov/files/documents/
3_21_17_Hearing_Aids_Bill_Text.pdf).
\49\ FDA, ``Immediately in Effect Guidance Document: Conditions for
sale for air-conduction hearing aids--Guidance for industry and FDA
staff '' (Dec. 12, 2016) (online at: http://www.fda.gov/downloads/
MedicalDevices/DeviceRegulationandGuidance/GuidanceDocuments/
UCM531995.pdf); FDA News Release, ``FDA takes steps to improve hearing
aid accessibility'' (online at: http://www.fda.gov/NewsEvents/Newsroom/
PressAnnouncements/ucm532005.htm).
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Do you agree that over the counter hearing aids would expand access
to hearing aids to millions of Americans with hearing loss who struggle
to afford these devices?
If confirmed, will you commit to work toward making safe and
innovative hearing aids intended to be used by adults to compensate for
mild to moderate hearing impairment available over the counter?
If confirmed, what steps will you take to implement the FDA's
stated commitment to creating a category of OTC hearing aids?
Do you agree that a key step in improving access to OTC hearing
aids will be pre-empting the many State requirements that now regulate
access to hearing aids?
If confirmed, what steps will you take to develop a clear OTC
access standard by pre-empting these State requirements?
Answer 1. I have not been privy to the discussions that FDA has had
related to this matter, although I am aware the agency announced a
commitment to examining OTC hearing aids. If confirmed, I commit to
reviewing the scientific evidence and to working with you, Senators
Grassley, Isakson, and Hassan, and other Members of Congress on this
issue.
Question 2. The Department of Health and Human Services (HHS)
Office of Inspector General (OIG) recently released preliminary results
of an ongoing review, finding that procedures associated with fixing
seven faulty cardiac implants cost Medicare $1.5 billion and resulted
in an additional $140 million in out-of-pocket costs to Medicare
beneficiaries.\50\ The OIG recommended that the Center for Medicare and
Medicaid Services (CMS) collaborate with the Accredited Standards
Committee X12 (ASC X12) to include medical devices' unique device
identifier (UDI) on health insurance claim forms. In October 2016, ASC
X12 voted in favor of a technical solution to this issue.\51\
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\50\ Letter from HHS Inspector General, Daniel Levinson, to Andrew
Slavitt, Acting
Administrator, Centers for Medicare and Medicaid--``Early Alert:
Incorporating Medical Device-Specific Information on Claim Forms (A-01-
16-00510)'' (September 30, 2016) (online at: https://oig.hhs.gov/oas/
reports/region1/11600510.pdf).
\51\ American Hospital Association, ``Standards organization
approves UDI changes'' AHA News Now (September 20, 2016) (online at:
http://news.aha.org/article/160920-standards-organiza-
tion-approves-udi-changes).
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In a July 2016 joint letter to X12, the FDA and CMS identified
several benefits to collecting device identifiers on medical claims
form, including: Strengthening capacities to evaluate product
performance and safety concerns for specific models of medical devices,
improving surveillance efforts, helping support device innovation,
allowing providers and payers to compare costs and outcomes by device
model, and supporting program integrity efforts.\52\ Inclusion of UDIs
in claims is also supported by clinical societies like the American
Academy of Orthopedic Surgeons (AAOS),\53\ and by members of both
parties in Congress.\54\ FDA has agreed to develop a list of specific,
high-risk implantable devices for which reporting on claims will be
recommended.\55\
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\52\ Letter from CMS Acting Administrator Andrew M. Slavitt and FDA
Commissioner Robert M. Califf to Gary Beatty, Chair, Accredited
Standards Committee X12 (July 13, 2016) (online at: http://
pascrell.house.gov/sites/pascrell.house.gov/files/wysiwyg_uploaded/
LETTER_FDA
%20CMS%20Beatty%20Letter%20on%20UDI%20in%20Claims%207.13.16.pdf).
\53\ American Academy of Orthopedic Surgeons letter to FDA
Commissioner Margaret A.
Hamburg (Nov. 7, 2012) (online at: http://www.aaos.org/uploadedFiles/
PreProduction/Advocacy/Federal/FDA/
UDI%20Proposed%20Rule%20Comment%20Draft_final.pdf); Multi-organization
letter to CMS (December 15, 2015) (online at: https://s3.amazonaws.com/
assets.fierce
markets.net/public/004Healthcare/external/
Public+health%2C+clinician+and+stakeholder+com
ments+in+support+of+UDI+in+EHRs+12-15+FINAL.pdf).
\54\ Letter from Senator Warren and Senator Grassley to Gary
Beatty, Chair of ASC X12 (Aug. 29, 2016) (online at: http://
www.warren.senate.gov/files/documents/2016-8-29_UDI_letter_
to_ASC_X12.pdf).
\55\ American Hospital Association, ``Standards organization
approves UDI changes'' AHA News Now (September 20, 2016) (online at:
http://news.aha.org/article/160920-standards-organi
zation-approves-udi-changes).
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The AAOS stated in a 2015 letter to then-Acting FDA Commissioner
Stephen Ostroff that UDIs ``will significantly enhance post-market
surveillance activities by providing a standard and unambiguous way to
document device use in electronic health records, clinical information
systems, claims data sources, and registries.'' \56\ Do you agree that
including device identifier information in medical claims could support
the evaluation of medical devices after approval?
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\56\ American Academy of Orthopedic Surgeons letter to Acting FDA
Commissioner Stephen Ostroff (Oct. 26, 2015) (http://www.aaos.org/
uploadedFiles/PreProduction/Advocacy/Federal/FDA/
AAOS_FDA%20Comment%20Letter%20on%20MDEpiNet%20Report_FINAL%20%20.pdf).
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If confirmed, will you continue to support the process of adding
device identifiers to claims as a critical tool to better understand
the performance of these products after approval?
How will you direct FDA to work with CMS to ensure that device
identifiers can be effectively used to monitor threats to Medicare
program integrity, as well as to patient health?
What steps will you take to develop a list of high-risk implantable
devices and support the inclusion of device identifiers for these
devices on claims forms? More specifically:
What criteria should be used to determine whether a
device should be included on the list?
What factors should FDA consider in deciding what these
criteria should be?
How will FDA support providers and insurers who wish to
exchange device identifier information, even for devices not included
on the FDA's high-risk list?
Answer 2. I am committed to reviewing the work done to date by
staff at CMS and FDA on this issue. Policies that can help us identify
problems with devices are important, and if confirmed, I look forward
to working with my colleagues at CMS and the X12 Committee to explore
these policies. These considerations should also include achieving
interoperable electronic health records with UDIs--a goal that is
consistent with the health information technology provisions in 21st
Century Cures.
Question 3. Once relatively simple, well-understood pathology tests
used for diagnostic purposes, lab-developed tests (LDTs) have become
more advanced and a staple of clinical decisionmaking. They are often
now used to diagnose high-risk, but relatively common, diseases.
Increased understanding of genetics and the role particular genes play
in disease has led to the creation of new, more complex, medical
diagnostic technology. LDTs hold great promise to customize healthcare
to be more efficient and targeted for an individual patient. However,
because of their increasing prevalence in the clinic, it is imperative
that they perform reliably and accurately. Incorrect results mean that
patients either will not seek out the care and therapy that is needed,
or will be subject to treatments that do not work or are harmful.\57\
FDA, which has authority under the Food, Drug and Cosmetic Act to
regulate LDTs, released draft guidance in October 2014 after years of
delay.\58\ However, in November 2016, FDA stated that it would not
release final guidance during the Obama administration. If you are
confirmed Commissioner--
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\57\ FDA, ``The Public Health Evidence for FDA Oversight of
Laboratory Developed Tests: 20 Case Studies'' (November 16, 2015)
(online at: http://www.fda.gov/downloads/AboutFDA/ReportsManualsForms/
Reports/UCM472777.pdf).
\58\FDA, ``Draft Guidance for Industry, Food and Drug
Administration Staff, and Clinical
Laboratories'' (October 3, 2014) (online at: http://www.fda.gov/
downloads/medicaldevices/deviceregulationandguidance/guidancedocuments/
ucm416685.pdf).
What role do you believe that FDA should play in ensuring
that LDTs provide clinically relevant information to the physicians and
patients who rely on them for making decisions impacting patient health
and well-being?
Do you agree that high-risk LDTs that inform clinical
diagnoses should be clinically validated?
How will you ensure patient safety by implementing risk-
based oversight and regulation of LDTs?
How will you direct FDA to ensure reliability of LDTs,
particularly those that are moderate- or high-risk?
What steps should FDA take to improve reporting by
clinicians and patients of faulty or unreliable LDTs?
What steps should be taken when such LDTs are identified?
Answer 3. Defining an appropriate regulatory framework for
Laboratory Developed Tests (LDTs) is important to FDA's mission to
protect and promote public health. In order to both protect patient
safety and encourage innovation and patient access, I believe we must
strike the right balance between Clinical Laboratory Improvement
Amendments (CLIA) and FDA regulation and regulatory requirements. If
confirmed, I would commit to working with Congress and stakeholders to
develop appropriate LDT regulatory policies.
Question 4. Ensuring a safe and adequate blood supply is a critical
aspect of our public health system. The Food and Drug Administration
(FDA) develops blood donation policy for the Nation's blood banks--a
task that is even more important as we respond to emerging diseases
such as the Zika virus that threaten the safety of our blood supply.
Evidence indicates that moving to a risk-based referral policy could
increase the U.S. blood supply by up to 4 percent, helping to address
the Nation's blood shortage.\59\ In June 2016, FDA started collecting
public input on scientifically sound solutions to risk-based screening,
and the information collection period closed in November 2016.\60\
Building on these steps will require leadership from the next FDA
Commissioner.
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\59\ Ayako Miyashita and Gary J. Gates, ``Update: Effects of
Lifting Blood Donation Bans on Men Who Have Sex with Men,'' The
Williams Institute (September 2014) (online at: http://
williamsinstitute.law.ucla.edu/wp-content/uploads/Blood-Ban-update-Jan-
2015.pdf). See for example: ``American Red Cross reports severe winter
blood shortage,'' WTHITV.com (January 4, 2017) (online at http://
wthitv.com/2017/01/04/american-red-cross-reports-severe-winter-blood-
shortage/) and ``San Antonio in extreme blood shortage,'' KHOU.com
(January 10, 2017) (online at http://www.khou.com/news/local/texas/san-
antonio-in-extreme-blood-shortage/384692455).
\60\ Federal Register Notice 81 FR 49673 ``Blood Donor Deferral
Policy for Reducing the Risk of Human Immunodeficiency Virus
Transmission by Blood and Blood Products; Establishment of a Public
Docket; Request for Comments'' (https://www.federalregister.gov/
documents/2016/07/28/2016-17804/blood-donor-deferral-policy-for-
reducing-the-risk-of-human-immunodeficiency
-virus-transmission-by).
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If you are confirmed as Commissioner--
Are you committed to implementing a risk-based blood donation
deferral policy for all donors?
As FDA Commissioner, how would you support the FDA's efforts to
move to a risk-based referral policy for all blood donors?
How do you anticipate using public comments received during the
comment period for the FDA's recent request for information to
implement a risk-based deferral system for all donors? More
specifically:
Will you commit to developing a risk-based onsite
questionnaire to be used at blood donation clinics?
When can we expect FDA to release a draft of a risk-
based questionnaire?
Will you commit to gathering stakeholder input on the
questionnaire?
Over what time period will you test the questionnaire and
gather input?
Will you commit to integrating stakeholder input into the
questionnaire? How will you perform that integration?
What specific steps will you take to engage with impacted groups,
which may be newly eligible for blood donation, to encourage blood
donation in line with new policies?
Answer 4. Ensuring the safety and adequacy of our Nation's donated
blood supply is critically important to public health. If confirmed, I
will work with FDA staff to closely develop, implement, and monitor the
impact of policies to promote blood safety. I will also commit to
continuing to work with FDA staff to review its donor deferral policies
to ensure they reflect the most up-to-date scientific knowledge.
Question 5. There is strong and growing evidence that antibiotic
use in food animals can lead to antibiotic resistance in humans, yet
the use of medically important drugs in food animals continues to grow.
According to the FDA, ``Domestic sales and distribution of medically
important antimicrobials approved for use in food producing animals
increased by 26 percent from 2009 through 2015, and increased by 2
percent from 2014 through 2015.'' \61\
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\61\ Food and Drug Administration, ``2015 Summary Report on
Antimicrobials Sold or
Distributed for Use in Food-Producing Animals'' (December 2016) (online
at: http://www.fda
.gov/downloads/ForIndustry/UserFees/AnimalDrugUserFeeActADUFA/
UCM534243.pdf).
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The 2014 National Strategy for combating Antibiotic-Resistant
Bacteria brought together the Secretaries of Health and Human Services,
Agriculture, and Defense to declare that,
``the misuse and over-use of antibiotics in health care and
food production continue to hasten the development of bacterial
drug resistance, leading to the loss of efficacy of existing
antibiotics.'' \62\
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\62\ ``National Strategy for Combating Antibiotic-Resistant
Bacteria,'' The White House (September 2014) (online at: https://
www.whitehouse.gov/sites/default/files/docs/carb_national_
strategy.pdf), p.4.
This initiative has enabled significant progress in establishing
policies that better protect lifesaving antibiotics. FDA policies
(Guidance for Industry (GFI) #209 and #213 and the Veterinary Feed
Directive Final Rule) now make the use of antibiotics to promote animal
growth illegal and subject all remaining uses of antibiotics to
veterinary oversight. However, more work could be done to strengthen
FDA policies aimed at preventing bacterial drug resistance.
As FDA Commissioner, will you commit to continuing this important
collaborative work with the Secretaries of Agriculture and Defense to
combat antibiotic-resistant bacteria?
Will you ensure that FDA staff time is dedicated to continuing the
relationship with USDA and DOD in service of the important goal of
reducing antibiotic overuse and misuse in animals?
Using low doses of antibiotics for long periods of time--as called
for by many growth promotion indications--can lead to resistance, yet
many commonly used antibiotics do not come with instructions regarding
treatment time limits. Without instructions on the label, there is no
mechanism for enforcement. The FDA recently sought comments on a plan
to establish treatment time limits for medically important
antimicrobial drugs when administered to animals.\63\ This comment
period closed on March 13, 2017.
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\63\ Food and Drug Administration, ``The Judicious Use of Medically
Important Antimicrobial Drugs in Food-Producing Animals; Establishing
Appropriate Durations of Therapeutic
Administration; Extension of Comment Period'' (Nov. 29, 2016) (online
at: https://www.
federalregister.gov/documents/2016/11/29/2016-28660/the-judicious-use-
of-medically-import
ant-antimicrobial-drugs-in-food-producing-animals-establishing).
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Will you commit to ensuring that this process for establishing
treatment time limits continues in a timely manner once the comment
period closes? When can we expect a proposed regulation?
GFI #213 describes principles that veterinarians should consider
when determining the appropriateness of antibiotic use for disease
prevention. While the FDA ``intends to work with veterinary and animal
producer organizations to reinforce the importance of these
principals,'' \64\ not all stakeholders agree on the need to reduce
antibiotic use or on the impact that the FDA's policies will have on
the amount of drugs used. Given the documented disagreements among
stakeholders, and given that veterinary adherence to appropriate
antibiotic prescribing guidelines is a critical part of FDA's policies,
FDA oversight is particularly important. Will you commit to putting in
place a protocol to monitor and report on veterinary compliance with
GFI#213's appropriate antibiotic prescribing guidelines?
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\64\ Kraus, Thomas A., Associate Commissioner for Legislation, FDA
to Senators Warren,
Feinstein and Gillibrand, Sept. 8, 2014; FDA, Guidance for Industry
#213: New Animal Drugs and New Animal Drug Combination Products
Administered in or on Medicated Feed or Drinking Water of Food
Producing Animals: Recommendations for Drug Sponsors for Voluntarily
Aligning Product Use Conditions with GFI #209 (December 2013), p. 7.
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GFI #213 was released in 2012, but only just fully implemented on
January 3, 2017. If confirmed, your agency will have the responsibility
for measuring whether or not this policy successfully reduces
antibiotic resistance in humans and taking additional action if it does
not. What metrics would you use to evaluate whether the FDA's policies
(Guidance for Industry (GFI) #209 and #213 and the Veterinary Feed
Directive Final Rule) have been successful or unsuccessful at reducing
the misuse and over-use of antibiotics in animal agriculture?
If FDA's guidance documents succeed in reducing the use of
dangerous antibiotics, what changes should we expect to see in sales
data and in the Veterinary Feed Directive? When do you expect to see
those changes?
The FDA and USDA have both acknowledged that collecting data that
shows how antibiotics are used on farms is vital to enhanced
monitoring, however they have not yet enacted a joint plan. The
Antimicrobial Resistance Action Plan, released by the USDA's Animal and
Plant Health Inspection Service (APHIS) National Animal Health
Monitoring System (NAHMS), proposed initiatives include on-farm
studies.\65\ Will you commit to working with USDA to prioritize the
collection of on-farm data on antibiotic use?
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\65\ ``Proposed Initiatives from the USDA Antimicrobial Resistance
Action Plan,'' APHIS, April 2015 (Online at: https://
www.aphis.usda.gov/animal_health/nahms/amr/downloads/Pro-
posedInitiatives.pdf).
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A report published in October 2016 by the European Medicines Agency
shows that sales of antimicrobials used in animals in Europe fell
between 2011 and 2014.\66\
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\66\ European Medicines Agency, ``Sales of veterinary antimicrobial
agents in 29 European countries in 2014,'' Oct. 14, 2016. (Online at:
http://www.ema.europa.eu/docs/en_GB/document_
library/Report/2016/10/WC500214217.pdf).
What policies in selected countries in the European Union
do you believe have led to that reduction?
As FDA Commissioner, which of these policies will you
look to as an example of how we can better manage the use of
antibiotics in food animals in the United States?
Answer 5. Antibiotic resistance is a significant and growing public
health challenge facing our Nation. In addition to measures FDA should
take to address this issue within the context of human use, the agency
must effectively collaborate with other Federal and State government
agencies and public health authorities to develop policies and
processes to address the issue of antibiotic use in animals intended
for human consumption. If confirmed, I will ensure FDA remains engaged
on this important public health issue, making sure that animal drug
labeling reflects the most up-to-date science, and working closely with
the U.S. Department of Agriculture, the Centers for Disease Control,
the U.S. Department of Defense, and other appropriate government
agencies. While I am familiar with the EMA report, I have not reviewed
it in detail. If confirmed, I would commit to reviewing it further and
sharing my views on any lessons we could learn from that experience.
FDA should also consider input from other important stakeholders, such
as the farmers, the agriculture industry, and veterinarians. FDA's
implementation of a voluntary plan with industry to phaseout the use of
certain antibiotics is an important step in the right direction.
Question 6. Compounding pharmacies serve individual patients who
need specialized drugs. Without these customized products, some of the
most vulnerable patients would not be able to get the precisely
formulated medications they need. While intending to provide special
services for individual patients, the lack of regulation and oversight
of compounding pharmacies led to tragedy in 2012, when a widespread
fungal meningitis outbreak caused by contaminated compounded drugs from
New England Compounding Center (NECC) impacted over 20 States. The
outbreak resulted in over 750 people falling ill, including 64
deaths.\67\ The following year, Congress passed the bipartisan Drug
Quality and Security Act (DQSA), which clarified and enhanced FDA's
authority to regulate drug compounding.\68\ Since then, the FDA has
issued a number of draft and final guidances and rules, performed over
300 inspections of drug compounders, and found numerous violations,
causing some facilities to close and recall their products.\69\ The FDA
Commissioner oversees the agency's activities regarding implementation
of DQSA and ensures the safety and health of the American people by
regulating the safety and efficacy of domestic and imported
pharmaceutical products, including compounded drugs. If you are
confirmed Commissioner--
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\67\ Centers for Disease Control and Prevention, ``Multistate
outbreak of fungal meningitis and other infections--Case count'' (Oct.
30, 2015) (https://www.cdc.gov/hai/outbreaks/meningitis-map-
large.html). Accessed Jan. 17, 2017.
\68\ H.R. 3204, Drug Quality and Security Act (113th Congress)
(https://www.congress.gov/bill/113th-congress/house-bill/3204).
\69\ See for example, Christine Blank, Drug Topics, ``FDA
temporarily shutters `filthy' compounding pharmacy'' (May 12, 2016)
(http://drugtopics.modernmedicine.com/drug-topics/news/fda-temporarily-
shutters-filthy-compounding-pharmacy); Food and Drug Administration,
``Well Care Compounding Pharmacy issues voluntary statewide recall of
all sterile compounded products due to lack of assurance of sterility
concerns'' (May 17, 2016) (http://www.fda.gov/Safety/Recalls/
ucm501543.htm); Mintz Levin Cohn Ferris Glovsky and Popeo PC, Lexology,
``Health care enforcement review and 2017 outlook: FDA's wide-ranging
activities'' (Jan. 4, 2017) (http://www.lexology.com/library/
detail.aspx?g=efefdb42-b573-4193-990a-2f903cb866f0). Accessed Jan. 17,
2017. ``Drug Compounding: FDA has Taken Steps to Implement Compounding
Law, but Some States and Stakeholders Reported Challenges,'' GAO
(November 2016) (online at: http://www.gao.gov/assets/690/681096.pdf ).
Accessed April 5, 2017.
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Will you agree that effective Federal oversight of compounding
facilities is essential in preventing disease outbreak and danger to
patients?
Will you commit to protect patients through strong enforcement of
the DQSA?
Will you commit to supporting FDA's efforts in carrying out
inspections of compounding facilities?
What specific steps will you take to ensure patient health and
safety for patients who need compounded drugs?
Answer 6. Congress clarified FDA's regulatory authorities related
to compounding by passing the Drug Quality and Security Act (DQSA). If
confirmed, I am committed to implementing DQSA, as intended by
Congress, to both protect patient safety, and allow for safe and
appropriate practice of pharmacy compounding. The practice of pharmacy
compounding can serve an important role, allowing providers to develop
individualized formulations of certain medicines for specific patients
with unique needs. However, I know that there are examples of actors
operating as manufacturers of unapproved new drugs under the guise of a
pharmacy license, violating the careful framework created by Congress,
circumventing the FDA oversight that Congress intended for certain
products, and putting patient safety at risk.
Question 7. On January 18, 2017, the FDA and the Environmental
Protection Agency (EPA) released final advice on fish consumption,
clarifying appropriate amounts of fish consumption for pregnant and
breast feeding women, and parents with young children.\70\ Many
pregnant and breast feeding women rely on Federal nutrition advice, and
so over the past few years, my colleagues and I have pushed for the FDA
to finalize updated advice on fish consumption for pregnant women that
reflects the most up-to-date, scientific information.
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\70\ FDA News Release, ``FDA and EPA issue final fish consumption
advice'' (Jan. 18, 2017) (http://www.fda.gov/NewsEvents/Newsroom/
PressAnnouncements/ucm537362.htm).
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As a doctor, do you agree that pregnant women should have access to
the latest science-based nutrition advice so that they can make healthy
nutrition decisions before and after pregnancy?
If confirmed Commissioner, what additional steps will you take to
ensure that this final advice is consistent with the latest nutritional
science?
If this advice is confirmed to be consistent with the latest
nutritional science, what steps will you take to work with appropriate
stakeholders to ensure that this final advice is clearly communicated
to pregnant women?
Answer 7. As a doctor, I do agree that pregnant women should have
access to the latest science-based nutrition advice so that they can
make healthy nutrition decisions before and after pregnancy. If
confirmed, I will ensure FDA's advice concerning seafood consumption by
pregnant and nursing women is based on the most current and relevant
nutritional science and appropriately takes into account both the
nutritional benefits, and any toxicological risks associated with
seafood consumption. I will also work to ensure effective collaboration
between FDA and the U.S. Environmental Protection Agency (EPA) on this
issue, and a range of other public health matters over which both
agencies share regulatory authority.
Question 8. In March 2016, the FDA issued a request for comment
entitled, ``Refurbishing, Reconditioning, Rebuilding, Remarketing,
Remanufacturing, and Servicing of Medical Devices Performed by Third
Party Entities and Original Equipment Manufacturers.'' \71\ In October
2016, the agency held a public workshop, in which they heard from a
variety of stakeholders, including OEMs, third party vendors,
healthcare technology management professionals, and trade associations.
This action was taken because of concerns over ``quality, safety, and
continued effectiveness of medical devices'' that have been subject to
third-party repair and servicing.\72\ As you know, determining industry
best practices and appropriate regulation of third-party repair of
medical devices is an important part of ensuring patient safety.
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\71\ ``Refurbishing, Reconditioning, Rebuilding, Remarketing,
Remanufacturing, and Servicing
of Medical Devices Performed by Third Party Entities and Original
Equipment Manufacturers; Request for Comments,'' [Docket No. FDA-2016-
N-0436] Federal Register (March 4, 2016) (online at: https://
www.gpo.gov/fdsys/pkg/FR-2016-03-04/pdf/2016-04700.pdf).
\72\ ``Public Workshop--Refurbishing, Reconditioning, Rebuilding,
Remarketing, Remanufacturing, and Servicing of Medical Devices
Performed by Third-Party Entities and Original Equipment
Manufacturers,'' FDA (October 27-28, 2016) (online at: https://
www.fda.gov/downloads/MedicalDevices/NewsEvents/WorkshopsConferences/
UCM525760.pdf).
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If confirmed FDA Commissioner, do you agree that it is important to
ensure the safety and efficacy of medical devices that are repaired and
serviced by third-party entities?
How will you continue to engage with stakeholders, such as patient
groups, healthcare technology managers, and the medical device
industry, in responding to the comments received through the FDA's
Request for Comment and public workshop?
What specific steps would you recommend the FDA take to address the
comments received by the agency?
What would the timeframe for those actions be?
How would the actions you propose be impacted by the
regulatory freeze initiated by President Trump?
Answer 8. An important part of FDA's responsibility to protect and
promote public health is upholding the Gold Standard of safety and
efficacy for medical products American patients use. With regard to the
issue of medical devices that are serviced by 3d parties, if confirmed,
I will commit to quickly engaging with FDA's professional staff to get
up to speed on this issue, including a review of the public comments
received by the agency. I look forward to working with FDA's staff,
Congress, and stakeholders to ensure that the agency has in place the
right policies and processes to ensure the safety and efficacy of
medical devices.
Question 9. The Food, Drug, and Cosmetic Act prohibits the
marketing of medical products for uses not approved by the FDA. In
January 2017, FDA issued two draft guidance documents on off-label
communications to promote medical products, and the 21st Century Cures
Act made changes to the safe harbor requirements for the communication
of health care economic information (HCEI) between medical product
companies and payers.\73\ I am concerned that, should you be confirmed
FDA Commissioner, you would aim to loosen restrictions on off-label
promotion even further. Even though physicians can already use their
expert judgment to prescribe drugs for off-label use, you have
advocated allowing companies to actively promote their products for
non-FDA-approved indications.\74\
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\73\ See for example, David C. Gibbons, Dara Katcher Levy, ``Slower
than Molasses in January, FDA Moves to Provide Guidance on Product
Communications by Pharmaceutical and Device Manufacturers,'' FDA Law
Blog (March 2, 2017) (online at: http://www.fdalawblog.net/
fda_law_blog_hyman_phelps/2017/03/slower-than-molasses-in-january-fda-
moves-to-provide-guidance-on-product-communications-by-pharmaceu.html).
\74\ See for example, Bronwyn Mixter, ``Trump's FDA Nominee Spurs
Concerns About Drug Approvals, Off-Label Promotion,'' Bloomberg BNA
(March 14, 2017) (online at: https://www.bna.com/trumps-fda-nominee-
n57982085167/).
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Do you believe that medical product companies should be given
greater latitude to promote their products for non-FDA-approved
indications?
If confirmed Commissioner, would you commit to not loosening
restrictions on off-label communications to physicians and payers?
Answer 9. Medical product labels are one of the primary tools FDA
uses to promote the appropriate use of medicines and technologies and
communicate risk information. It is important that information on
product labels is accurate, clear, and scientifically based; and be the
result of a sound regulatory process. Further, it is crucial that
manufacturers continue to develop and submit to the agency clinical
data demonstrating the safety and efficacy of medical products for new
indications they seek to include on labeling and in their marketing
communications with patients, payers, and providers. I also believe
that patients and physicians make the best decisions when they have
access to as much truthful, non-misleading, scientifically based
information as possible. Toward these same ends, FDA has consistently
acknowledged that there is some public health benefit of allowing
certain non-
promotional communication about truthful, non-misleading, clinical data
that is not already incorporated into FDA-approved product labeling.
Indeed, FDA has carved out certain ``safe harbors'' for such
communications. If confirmed, I will commit to working with FDA's staff
to get up to speed on the agency's latest thinking and actions on these
matters.
Question 10. America is in the midst of an opioid epidemic, which
is devastating communities in every State. According to the Centers for
Disease Control, 33,000 Americans died of an opioid overdose in 2015--
that's an average of 91 people every day. Nearly half of all opioid
overdose deaths involved a prescription opioid and three out of four
new heroin users abused prescription opioids before moving to heroin.
We must take action to address this epidemic.
What role do you believe the FDA has in combating this epidemic?
If you are confirmed as Commissioner, what FDA authorities could
you use to help address the opioid crisis?
Both the FDA's Opioid Action Plan and the Comprehensive Addiction
and Recovery Act emphasize the role that the FDA's advisory committees
should play in the decision to approve opioid medications. If you are
confirmed as Commissioner, would you pledge to respect the advice of
the FDA's advisory committees in their recommendations with regard to
the safety and public health risks of dangerous and addictive opioids?
If you are confirmed, what will you do to improve physician
education on the safe prescribing of opioid medications?
If you are confirmed, what will you do to improve physician
education on the safe prescribing of benzodiazepines to patients who
may already be prescribed opioid medications?
Answer 10. Opioid abuse, misuse, and addiction constitute one of
the most urgent and immediate public health threats facing our Nation.
It is the biggest public health crisis facing the FDA. The human and
economic toll of this crisis is staggering. If confirmed, this will be
my highest immediate priority. I will make sure FDA is aggressive,
forward leaning, and fully engaged in combating this epidemic. I will
work with FDA's staff to ensure FDA has the right policies and
processes in place to:
Facilitate the developments of new approaches and
technologies to reduce the abuse/addictive potential of painkillers
American patients use;
Support the development of non-opioid analgesic
alternatives for physicians and patients;
Assess whether FDA's current approach to opioid regulatory
decisions, including labeling, REMS, and physician/patient education
are appropriate, robust, and fully effective;
Encourage the development of new pharmacological tools for
physicians and patients to both prevent opioid misuse and abuse, and
support treatment and recovery for patients struggling to overcome
opioid addiction;
Enhance physician and patient educational materials to
strengthen public awareness of the risks of opioids, as well as the
FDA-approved resources available to them, using the full range of FDA's
risk communication tools to better target this information;
Taking steps to make sure that providers are appropriately
educated on identifying, and helping to property intervene with, abuse-
prone patients;
Re-assess whether FDA has the appropriate framework and
authorities for evaluating the risk of abuse and diversion as a
component of its review and approval process for opioids;
Undertake a comprehensive effort to evaluate the full
scope of the sources and threats from foreign imported narcotics;
Evaluate whether FDA should bring more alignment between
the review and approval of different medical product platforms used in
the treatment of pain to make sure the agency is adopting the best
public health standard in assessing these products; and
Collaborate effectively with other government agencies and
external stakeholders to develop and execute comprehensive and
effective strategies to win the battle against opioid abuse, misuse,
and addiction. This includes steps for FDA to more closely collaborate
and coordinate with DEA on the two agencies shared goals.
Question 11. Nearly 2,000 opioid-related deaths occurred in
Massachusetts during 2016.\75\ HHS data also shows that the State had
the highest rate of opioid-related emergency room visits among the 30
States analyzed in a recent Federal report.\76\ Access to the
prescription drug naloxone, a medication that can arrest or reverse an
opioid overdose, saves lives in Massachusetts. However, more could be
done to expand access to naloxone. In August 2016, the FDA outlined the
steps it was taking to ensure greater access to naloxone, including
``helping manufacturers pursue approval of an OTC naloxone product,
including helping to develop the package label that would be required
for such a product.'' \77\ The FDA indicated that it had created a
model Drug Facts Label and accompanying pictogram that could provide
consumers with necessary information about how to use naloxone safely,
and was engaged in label comprehension testing of this model label.
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\75\ Massachusetts Department of Public Health, ``Data Brief:
Opioid-related Overdose Deaths Among Massachusetts Residents''
(February 2017) (online at: www.mass.gov/eohhs/docs/dph/stop-addiction/
current-statistics/data-brief-overdose-deaths-february-2017.pdf).
\76\ Matt Rocheleau, ``Mass. Had Highest Rate of Opioid-Related ER
Visits,'' The Boston Globe
(April 3, 2017) (online at: https://www.bostonglobe.com/metro/2017/04/
02/mass-had-highest-rate-opioid-related-visits/
6vJ4kwtO1dvQGf7TGuXueN/story.html).
\77\ Karen Mahoney, ``FDA Supports Greater Access to Naloxone to
Help Reduce Opioid Overdose Deaths,'' FDA Voice (August 10, 2016)
(online at: https://blogs.fda/gov/fdavoice/index.php/2016/08/fda-
supports-greater-access-to-naloxone-to-help-reduce-opioid-overdose-
deaths/).
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Do you agree that expanding access to naloxone, including by making
it safely available over the counter, is an important part of FDA
efforts to address the opioid epidemic?
What is the current status of FDA efforts to develop and test a
package label for an OTC naloxone product?
What efforts does the FDA have underway to encourage physicians to
co-prescribe naloxone with opioid medications?
What additional steps could the FDA take to safely facilitate
increased rates of co-prescribing of naloxone with opioid medications?
What additional steps can the FDA take to work with interested
manufacturers to continue expanding access to naloxone?
Answer 11. I support increased access to drugs like naloxone, which
can arrest or reverse opioid overdoses. I am not aware of the current
status of FDA efforts to develop and test a package label for an OTC
naloxone product or closely familiar with the FDA efforts currently
underway to encourage physicians to co-prescribe naloxone with opioid
medications. If confirmed, I will commit to working with FDA staff to
quickly get up to speed on this specific issue. More broadly, opioid
abuse, misuse, and addiction constitute the most urgent and immediate
public health threat facing our Nation. It is the biggest public health
crisis facing our Nation, and it will be my highest immediate priority
if confirmed to lead FDA.
Question 12. Increased sharing of clinical trial data could
strengthen academic research, improve the practice of medicine, and
protect the integrity of the clinical trials system.\78\ I have
supported the proposal advanced by the International Committee of
Medical Journal Editors to require that researchers share data as a
condition of publication in major medical journals,\79\ but there are a
variety of approaches to expanding data sharing and transparency that
could improve medical research.
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\78\ Elizabeth Warren, ``Strengthening Research through Data
Sharing,'' New England Journal of Medicine 2016; 375:401-3 (online at:
http://www.nejm.org/doi/full/10.1056/NEJMp160
7282).
\79\ Taichman DB, Backus J, Baethge C, et al. Sharing clinical
trial data--a proposal from the International Committee of Medical
Journal Editors. N Engl J Med 2016;374:384-6.
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Unfortunately, some efforts to improve data sharing have been
hampered by incomplete compliance with Federal requirements. A 2015
study published in the British Medical Journal, found that several
major drug companies have not met the standards for clinical trial
results reporting under the Food and Drug Administration Amendments Act
(FDAAA) of 2007.\80\ FDAAA established civil monetary penalties of up
to $10,000 per day for non-compliance, and yet the FDA has never
imposed such penalties. In September 2016, the FDA removed a major
barrier to enforcement of the FDAA penalties by issuing a final rule
detailing the requirements for submitting clinical trial results to
Clinicaltrials.gov.\81\
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\80\ Jennifer E. Miller, David Korn, and Joseph S. Ross, ``Clinical
trial registration, reporting, publication and FDAAA compliance: A
cross-sectional analysis and ranking of new drugs approved by the FDA
in 2012,'' BMJ Open 2015;5:e009758. doi: 10.1136/bmjopen-2015-009758.
\81\ Department of Health and Human Services, ``Clinical Trials
Registration and Results Information Submission: Final Rule,'' 42 CFR
Part 11, Federal Register 81:183 (online at: https://www.gpo.gov/fdsys/
pkg/FR-2016-09-21/pdf/2016-22129.pdf).
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What do you believe the impact of greater transparency of clinical
trial data and results would be on--
1. Clinical trial efficiency
2. The cost of drug development
3. Drug safety
4. Biomedical innovation
If you are confirmed Commission, what specific steps would you take
to increase sharing of clinical trial data?
You have supported making FDA's complete response letters publicly
available to improve information about why the agency has rejected a
company's application.
What would be the impact of making complete response
letters publicly available on:
1. Clinical trial efficiency
2. The cost of drug development
3. Drug safety
4. Biomedical innovation
If you are confirmed as Commissioner, will you commit to
making complete response letters publicly available?
What specific steps would you take to make complete
response letters publicly available?
If you are confirmed Commissioner--
How will you ensure compliance to the disclosure policy
implemented by FDAAA?
Will you enforce the law using civil monetary penalties
or by other means?
Answer 12. I am a strong proponent of data transparency--for
patients, physicians, and manufacturers. I have long advocated that the
FDA release more information related to its review process that could
be used to better inform consumers and product developers alike. This
includes the complete response letters, after proper redaction of
commercial confidential information. If confirmed, I will be committed
to working with Congress, patients, industry, and stakeholders on the
issue of data transparency and new ways that FDA could potentially make
important information more readily available to the public.
Question 13. Recent news reports have discussed,
``a surge in human infections of a deadly bird flu in China
[that] is prompting increasing concern among health officials
around the world . . . [and that] poses the greatest risk of a
pandemic threat if it evolves to spread readily from human to
human.'' \82\ I recently sent a letter with Senator Patty
Murray and Representatives Pallone, DeGette, and Green
expressing my concern over the impact of a series of actions
taken by President Trump on the Nation's preparedness for a
pandemic flu outbreak.\83\
---------------------------------------------------------------------------
\82\ Lena H. Sun, ``Surge in Human Cases of Deadly Bird Flu is
Prompting Alarm,'' Washington Post (March 3, 2017) (online at: https://
www.washingtonpost.com/news/to-your-health/wp/2017/03/03/surge-in-
human-cases-of-deadly bird-flu-is-prompting-alarm/?postshare=847148855
6990075&tid=ss_tw&utm_term=.40b2cf15e3cb).
\83\ Letter from Senators Warren and Murray and Representatives
Pallone, DeGette, and Green to HHS Secretary Tom Price and Acting CDC
Director Anne Schuchat (March 17, 2017) (online at: https://
www.warren.senate.gov/files/documents/2017_3_13_Letter_to_CDC.pdf).
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I am also concerned that, during your previous tenure at the FDA,
you had to recuse yourself from pandemic planning efforts inside the
agency, due to your conflicts of interest with companies that
manufactured flu vaccines.\84\ You have indicated to the Office of
Government Ethics that--if confirmed as Commissioner--you will recuse
yourself from participating personally or substantially in any
particular matter involving specific parties in which New Enterprise
Associates, American Pathology Partners, or Collective Health is a
party or represents a party, or in which a former client is a party or
represents a party.
---------------------------------------------------------------------------
\84\ Diedtra Henderson, ``FDA Official Recused in Flu Fight,''
Boston Globe (November 12, 2002) (online at: http://archive.boston.com/
business/healthcare/articles/2005/11/12/fda_official_
recused_in_flu_fight/).
---------------------------------------------------------------------------
What role does the FDA play in preparing for a pandemic flu
outbreak?
Are New Enterprise Associates, American Pathology Partners,
Collective Health parties, or do they represent any parties, that has
any involvement with flu vaccines, flu preparedness, or flu response?
Are any of your former clients a party, or do they represent any
party, that has any involvement with flu vaccines, flu preparedness, or
flu response?
Will this commitment to recuse yourself from participating
personally or substantially in any particular matter involving these
specific parties affect your ability to engage in pandemic planning
efforts as FDA Commissioner, should you be confirmed in this role?
Answer 13. The FDA's medical product centers play an important role
in preparing for pandemics by assisting in and fostering the
development and approval of safe and effective medical products, such
as vaccines, drugs, diagnostic tests, masks and gloves, to help respond
to emerging public health threats. Career officials, without the
involvement of the Commissioner, typically handle this work. The
Commissioner has broad leadership, policy and advocacy roles but is
rarely involved in individual product development and approval matters.
To my knowledge, New Enterprise Associates is not invested in entities
that have a direct interest in flu vaccines, flu preparedness, or flu
response. I do not believe Collective Health and American Pathology
Partners are engaged in such efforts as they are healthcare services
companies. I believe GlaxoSmithKline, a former client, develops flu
vaccines and other products for flu response but I do not know whether
it or any other entity may become involved with flu response efforts
during my public service. For all of these reasons, I do not believe
that the recusals set forth in my ethics agreement would impair my
ability to fully perform my duties with respect to pandemic response.
Moreover, should a circumstance arise that requires my recusal, my
deputy or another senior FDA official will handle that matter. I am and
will remain committed to pandemic preparedness and have every
confidence that my team at FDA will ensure that the FDA's mission with
respect to this important public health concern is fulfilled. Finally,
I would like to clarify that contrary to certain erroneous press
reports, I was not recused from pandemic planning efforts during my
prior tenure at the FDA. Rather, I was only recused from certain
matters related to particular individual companies that manufactured
vaccines.
Question 14. FDA's work is supported by highly skilled,
professional employees who uphold the agency's mission and protect
public health in the United States.
If you are confirmed as Commissioner, will you work cooperatively
with employees and employees' representatives, including unions?
Should you be confirmed, will you meet with national leadership of
employees' union representatives soon after you begin your duties?
Answer 14. FDA's ability to fulfill its mission to protect and
promote public health depends almost entirely on its world-class
workforce of talented, dedicated public servants. If confirmed, I look
forward to building strong and trusting relationships with the agency's
career employees, and their representatives.
senator kaine
Question 1. During the hearing, I appreciated your interest in
addressing the opioid epidemic. According to the CDC, opioid-related
overdose deaths in the United States have quadrupled in the last
decade. During this same time period, prescriptions for opioids have
also increased. Do you agree voluntary opioid prescribing guidelines
can be a useful tool to help inform physicians in treating pain and in
opioid prescribing?
Answer 1. I agree that better information about appropriate
prescribing can be an important tool in combating this epidemic by
helping ensure physicians are properly informed about the risks and
benefits of opioid prescribing, and in particular, in educating
providers about identifying and prescribing opioids in patients at risk
for abuse. We need to enhance physician and patient educational
materials to strengthen public awareness of the risks of opioids, as
well as the FDA-approved resources available to them, using the full
range of FDA's risk communication tools.
Question 2. Do you agree that a focus on abuse deterrent
formulations by FDA, while important, are not the only steps the Agency
can help reduce opioid addiction?
Answer 2. Given the tragic scope and urgency of this crisis, FDA
has to take an all of the above approach to addressing this epidemic.
The opioid crisis is a human tragedy of enormous scope and should be
the FDA's highest public health priority. It is the biggest crisis
facing the agency. ADFs are just one tool in addressing this crisis.
Among other steps that, I believe, FDA can take to address the opioid
epidemic, I am committed to helping:
Facilitate the developments of new approaches and
technologies to reduce the abuse/addictive potential of painkillers
American patients use;
Support the development of non-opioid analgesic
alternatives for physicians and patients;
Assess whether FDA's current approach to opioid regulatory
decisions, including labeling, REMS, and physician/patient education
are appropriate, robust, and fully effective;
Encourage the development of new pharmacological tools for
physicians and patients to both prevent opioid misuse and abuse, and
support treatment and recovery for patients struggling to overcome
opioid addiction;
Enhance physician and patient educational materials to
strengthen public awareness of the risks of opioids, as well as the
FDA-approved resources available to them, using the full range of FDA's
risk communication tools to better target this information;
Taking steps to make sure that providers are appropriately
educated on identifying, and helping to property intervene with, abuse-
prone patients;
Re-assess whether FDA has the appropriate framework and
authorities for evaluating the risk of abuse and diversion as a
component of its review and approval process for opioids;
Undertake a comprehensive effort to evaluate the full
scope of the sources and threats from foreign imported narcotics;
Evaluate whether FDA should bring more alignment between
the review and approval of different medical product platforms used in
the treatment of pain to make sure the agency is adopting the best
public health standard in assessing these products; and
Collaborate effectively with other government agencies and
external stakeholders to develop and execute comprehensive and
effective strategies to win the battle against opioid abuse, misuse,
and addiction. This includes steps for FDA to more closely collaborate
and coordinate with DEA on the two agencies shared goals.
Question 3. As we discussed when we met, I was extremely concerned
about the action the Agency took with regards to Zohydro and the
approval of Oxycontin for children despite the recommendations from an
Advisory Committee. Can you address under what circumstances the Agency
should override recommendations from an advisory committee?
Answer 3. I believe FDA should have the benefit of independent
advice from the outside experts who serve on Advisory Committees. I
understand that this advice is often critical to FDA as they consider
challenging regulatory decisions. However, I also recognize that FDA
retains the ultimate responsibility to consider the totality of
evidence in making a final agency determination.
Question 4. You also discussed abuse-deterrent formulations for
opioids. Do you agree that a focus on abuse deterrent formulations by
FDA, while needed, fall well short of how the Agency can help reduce
opioid dependence?
Answer 4. Please see my response to this question in your question
No. 2.
Question 5. You have argued that drug manufacturing standards to
assure safe products are a cause of price spikes and drug shortages.
Specifically, you said in Forbes In August 2016 when discussing the
application of regulations to generic manufacturers:
``In a push to reduce the risk of contamination, the agency
in 2009 forced generic-drug makers to retool their sterile
manufacturing plants and make production lines less intricate.
The abruptness of the change caused many facilities to be shut
down, creating drug shortages and driving up prices.''
Do you believe that the regulation is not the only cause of spiking
drug prices? What are other causes of drug price spikes and how would
you use your role as Commissioner to address this issue?
Answer 5. Regulatory factors relating to manufacturing are only one
factor causing price spikes for certain drugs. In many cases, the
issues causing specific drugs to experience sharp increases in price
are different. While drug pricing does not fall directly within FDA's
purview, I believe the agency can play an important role on this
important issue by taking steps to improve product competition. If
confirmed, I will work to ensure FDA has the appropriate policies and
processes in place to effectively facilitate generic market entry and
competition. Reforming the regulatory pathway for complex generic
products would address one key policy deficiency that results in
unnecessary barriers to the development and review of generic
competitors for some innovator products for which traditional
bioequivalence and bioavailability testing alone are sometimes
insufficient for proving sameness. FDA should also explore options to
improve the efficiency and consistency of ANDA review processes and
timelines, so that financial speculators cannot engage in a regulatory
arbitrage, by dramatically hiking the price of some very old generic
drugs because they know it can take years for new generic competitors
to enter the market.
Question 6. You have acknowledged that high drug prices are a
problem for consumers, and said in an FDA speech on 9/20/2005,
``Many people are rightly concerned about the high prices on
many drugs, especially people who can least afford to pay for
medicines because they lack good health insurance, or have no
health insurance at all.''
But you are also an opponent of the Affordable Care Act. You have
referred to the ACA's Essential Health Benefits as ``politically
crafted.'' [Forbes, 2/19/2016] Do you consider the inclusion of
prescription drug coverage as an essential health benefit to be
politically crafted? Do you think more people will have access to
prescription drugs if we repeal the ACA?
Answer 6. As the nominee to be the next Commissioner of Food and
Drugs, I do not believe it would be appropriate to comment on questions
about issues that are outside the jurisdiction of FDA.
senator hassan
Question 1. As you know, the recommendations of FDA physicians and
scientist reviewers about safety and efficacy, approval, and labeling
of products--including those related to women's reproductive health--
should be based solely on scientific evidence. Do you commit to
allowing FDA physicians and scientist reviewers to make decisions on
safety and efficacy, approval, and labeling of products related to
women's reproductive health, including new and existing drugs and
devices, without political interference or interference from you,
should you be confirmed as FDA Commissioner?
Answer 1. Maintaining the Gold Standard of safety and efficacy for
medical products is fundamental to FDA's mission to protect and promote
public health. If confirmed, I will uphold the Gold Standard by
ensuring FDA makes independent regulatory decisions based on sound
science, good regulatory practices, and the support of a strong
scientific staff. This applies to all clinical areas, including
products related to women's reproductive health.
Question 2. In a 2012 op-ed published in the Wall Street Journal,
you questioned the role of the Drug Enforcement Agency (DEA) in
regulating opioid use and abuse. You said that the DEA may be the
``wrong enforcer'' and that their tactics are ``imprudent.''
Since you penned that piece, the number of opioid deaths in the
country has exploded, surpassing 33,000 in 2015. We know that
prescription opioids have contributed to our current epidemic. Further,
we know that the DEA is authorized through the Controlled Substances
Act to play a law enforcement role in the opioid crisis and to set
limits on overall active ingredient allowed in the marketplace.
In light of our current opioid epidemic, have your views on the
DEA's regulation of opioids changed?
At the time that you wrote this article, were you being paid by or
representing pharmaceutical distributors or any entity in the opioid
industry? If so, which ones?
Did any individual or organization connected to or hired by an
opioid manufacturer or distributor assist in the drafting of this op-
ed? If so, who helped you and in what capacity?
Answer 2. I believe now, as I did at the time that I wrote the op
ed, that there needs to be closer coordination and collaboration
between the law enforcement and public health entities charged with
combating this tragic human crisis. This is especially true when it
comes to DEA and FDA. I believe the two agencies need to be working
closely together to combat this crisis, and such coordination would be
a top priority of mine if I were confirmed into this role. I also
believe that the tools and approaches for achieving that purpose have
evolved as the crises has grown larger and more intractable since the
time I wrote that op ed. I was the author of the op ed article and was
representing my personal views on this policy matter. As is customary,
I do research and communicate with sources in advance of writing
articles. I was paid by the Wall Street Journal in connection with this
op-ed.
Question 3. In the aforementioned article, you advocate for having
some of DEA's current authorities transferred to the Department of
Health and Human Services (HHS), including having HHS take on the
``responsibility for apportioning active ingredients to manufacturers
of narcotics,'' commonly referred to as ``quotas.''
In your capacity as an FDA official, did you ever advocate for the
DEA to increase, or directly or indirectly ask the DEA to increase, any
opioid quota, including any quotas for active opioid ingredients? If
so, why?
If the above answer is yes, were your activities advocating for the
DEA to increase, or asking the DEA to increase, any opioid quota,
including any quotas for active opioid ingredients, undertaken on
behalf of or intended to benefit a particular person or company? If so,
please identify that person or company and explain the rationale for
your involvement.
Answer 3. At various times, DEA has advocated that the quota for
active pharmaceutical ingredients used to manufacture opioid drugs be
limited, while FDA has, at the same time, maintained that such limits
could contribute to a drug shortage for appropriate patients. This
engagement is a matter of public record. These discussions are a
reflection of the careful balancing that must occur between the need to
maintain access to important medicines for appropriate patients, while
taking the necessary steps to address the tragic and rampant abuse and
diversion of opioid drugs. Drug shortages are an issue of critical
concern for FDA. I recall the issue of quotas being raised to me by FDA
career staff and some interactions between FDA and DEA on this subject.
My involvement in these matters was to support the science-based
positions of the FDA's professional staff. None of my actions were
undertaken on behalf of, or intended to benefit, a particular person or
company. Rather, my role was to represent the agency's public health
positions. FDA's positions in these matters were taken in consideration
of issues related to abuse and diversion, consistent with the FDA's
regulatory and public health mandates. However, since my tenure at FDA
more than a decade ago, the scientific and public health consensus
about the proper ways to combat the opioid health crisis have evolved
sharply as this epidemic has grown in scope and severity and become our
Nation's most urgent public health crisis and a tragedy of enormous
proportion. I also believe, based on this collected experience, that it
is even more critically important that FDA and DEA collaborate very
closely in order to properly confront this human tragedy. Seeking such
collaboration would be one of my highest priorities, if confirmed.
Question 4. In your article, you said that other public health
agencies within HHS would be better able to judge ``distinctions
between illicit diversion and the legitimate practice of medicine.''
Effectively, you suggest transferring the regulatory and
enforcement components from an agency equipped for those roles to an
agency that has little capacity to execute them. With its current
authority and resources, it is unlikely HHS would be able to
effectively take on this role.
Describe how agencies within HHS--which President Trump wants to
cut by $15 billion in 2018--would be able to effectively absorb the
DEA's roles in regulating overprescribing from pharmacies and
providers?
Answer 4. Given the tragic scope and urgency of this crisis, FDA
has to take an all-of-the-above approach to addressing this epidemic.
The opioid crisis is a human tragedy of enormous scope and should be
the FDA's highest public health priority. It is the biggest crisis
facing the agency. Among other steps that, I believe, FDA can take to
address the opioid epidemic, I am committed to helping:
Facilitate the developments of new approaches and
technologies to reduce the abuse/addictive potential of painkillers
American patients use;
Support the development of non-opioid analgesic
alternatives for physicians and patients;
Assess whether FDA's current approach to opioid regulatory
decisions, including labeling, REMS, and physician/patient education
are appropriate, robust, and fully effective;
Encourage the development of new pharmacological tools for
physicians and patients to both prevent opioid misuse and abuse, and
support treatment and recovery for patients struggling to overcome
opioid addiction;
Enhance physician and patient educational materials to
strengthen public awareness of the risks of opioids, as well as the
FDA-approved resources available to them, using the full range of FDA's
risk communication tools to better target this information;
Taking steps to make sure that providers are appropriately
educated on identifying, and helping to properly intervene with, abuse-
prone patients;
Re-assess whether FDA has the appropriate framework and
authorities for evaluating the risk of abuse and diversion as a
component of its review and approval process for opioids;
Undertake a comprehensive effort to evaluate the full
scope of the sources and threats from foreign-imported narcotics;
Evaluate whether FDA should bring more alignment between
the review and approval of different medical product platforms used in
the treatment of pain to make sure the agency is adopting the best
public health standard in assessing these products; and
Collaborate effectively with other government agencies and
external stakeholders to develop and execute comprehensive and
effective strategies to win the battle against opioid abuse, misuse,
and addiction. This includes steps for FDA to more closely collaborate
and coordinate with DEA on the two agencies' shared goals.
[Whereupon, at 12:37 p.m., the hearing was adjourned.]