[House Hearing, 115 Congress]
[From the U.S. Government Publishing Office]
EXAMINING MEDICAL PRODUCT MANUFACTURER COMMUNICATIONS
=======================================================================
HEARING
BEFORE THE
SUBCOMMITTEE ON HEALTH
OF THE
COMMITTEE ON ENERGY AND COMMERCE
HOUSE OF REPRESENTATIVES
ONE HUNDRED FIFTEENTH CONGRESS
FIRST SESSION
__________
JULY 12, 2017
__________
Serial No. 115-44
[GRAPHIC NOT AVAILABLE IN TIFF FORMAT]
Printed for the use of the Committee on Energy and Commerce
energycommerce.house.gov
__________
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26-958 PDF WASHINGTON : 2018
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COMMITTEE ON ENERGY AND COMMERCE
GREG WALDEN, Oregon
Chairman
JOE BARTON, Texas FRANK PALLONE, Jr., New Jersey
Vice Chairman Ranking Member
FRED UPTON, Michigan BOBBY L. RUSH, Illinois
JOHN SHIMKUS, Illinois ANNA G. ESHOO, California
TIM MURPHY, Pennsylvania ELIOT L. ENGEL, New York
MICHAEL C. BURGESS, Texas GENE GREEN, Texas
MARSHA BLACKBURN, Tennessee DIANA DeGETTE, Colorado
STEVE SCALISE, Louisiana MICHAEL F. DOYLE, Pennsylvania
ROBERT E. LATTA, Ohio JANICE D. SCHAKOWSKY, Illinois
CATHY McMORRIS RODGERS, Washington G.K. BUTTERFIELD, North Carolina
GREGG HARPER, Mississippi DORIS O. MATSUI, California
LEONARD LANCE, New Jersey KATHY CASTOR, Florida
BRETT GUTHRIE, Kentucky JOHN P. SARBANES, Maryland
PETE OLSON, Texas JERRY McNERNEY, California
DAVID B. McKINLEY, West Virginia PETER WELCH, Vermont
ADAM KINZINGER, Illinois BEN RAY LUJAN, New Mexico
H. MORGAN GRIFFITH, Virginia PAUL TONKO, New York
GUS M. BILIRAKIS, Florida YVETTE D. CLARKE, New York
BILL JOHNSON, Ohio DAVID LOEBSACK, Iowa
BILLY LONG, Missouri KURT SCHRADER, Oregon
LARRY BUCSHON, Indiana JOSEPH P. KENNEDY, III,
BILL FLORES, Texas Massachusetts
SUSAN W. BROOKS, Indiana TONY CARDENAS, California
MARKWAYNE MULLIN, Oklahoma RAUL RUIZ, California
RICHARD HUDSON, North Carolina SCOTT H. PETERS, California
CHRIS COLLINS, New York DEBBIE DINGELL, Michigan
KEVIN CRAMER, North Dakota
TIM WALBERG, Michigan
MIMI WALTERS, California
RYAN A. COSTELLO, Pennsylvania
EARL L. ``BUDDY'' CARTER, Georgia
Subcommittee on Health
MICHAEL C. BURGESS, Texas
Chairman
BRETT GUTHRIE, Kentucky GENE GREEN, Texas
Vice Chairman Ranking Member
JOE BARTON, Texas ELIOT L. ENGEL, New York
FRED UPTON, Michigan JANICE D. SCHAKOWSKY, Illinois
JOHN SHIMKUS, Illinois G.K. BUTTERFIELD, North Carolina
TIM MURPHY, Pennsylvania DORIS O. MATSUI, California
MARSHA BLACKBURN, Tennessee KATHY CASTOR, Florida
CATHY McMORRIS RODGERS, Washington JOHN P. SARBANES, Maryland
LEONARD LANCE, New Jersey BEN RAY LUJAN, New Mexico
H. MORGAN GRIFFITH, Virginia KURT SCHRADER, Oregon
GUS M. BILIRAKIS, Florida JOSEPH P. KENNEDY, III,
BILLY LONG, Missouri Massachusetts
LARRY BUCSHON, Indiana TONY CARDENAS, California
SUSAN W. BROOKS, Indiana ANNA G. ESHOO, California
MARKWAYNE MULLIN, Oklahoma DIANA DeGETTE, Colorado
RICHARD HUDSON, North Carolina FRANK PALLONE, Jr., New Jersey (ex
CHRIS COLLINS, New York officio)
EARL L. ``BUDDY'' CARTER, Georgia
GREG WALDEN, Oregon (ex officio)
(ii)
C O N T E N T S
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Page
Hon. Michael C. Burgess, a Representative in Congress from the
State of Texas, opening statement.............................. 1
Prepared statement........................................... 2
Hon. Gene Green, a Representative in Congress from the State of
Texas, opening statement....................................... 4
Hon. Greg Walden, a Representative in Congress from the State of
Oregon, opening statement...................................... 5
Prepared statement........................................... 6
Hon. Frank Pallone, Jr., a Representative in Congress from the
State of New Jersey, opening statement......................... 7
Prepared statement........................................... 9
Witnesses
Coleen Klasmeier, Partner, Sidley Austin, LLP.................... 10
Prepared statement........................................... 13
Answers to submitted questions............................... 154
R. Alta Charo, Warren P. Knowles Professor of Law, University of
Wisconsin...................................................... 23
Prepared statement........................................... 26
Answers to submitted questions............................... 164
George Van Hare, M.D., President, Heart Rhythm Society........... 28
Prepared statement........................................... 30
Answers to submitted questions............................... 169
Aaron S. Kesselheim, Associate Professor of Medicine, Harvard
Medical School and Brigham and Women's Hospital................ 36
Prepared statement........................................... 38
Answers to submitted questions \1\........................... 174
Linda House, President, Cancer Support Community................. 46
Prepared statement........................................... 49
Answers to submitted questions............................... 178
Katherine Wolf Khachatourian, Vice President, Delegation
Oversight, Pharmacy Services, and Strategy, Qualchoice Health
Plan Services, Inc............................................. 55
Prepared statement........................................... 57
Answers to submitted questions \1\........................... 183
Submitted Material
Discussion Draft, H.R. ___, Facilitating exchange of information
prior to approval.............................................. 108
Discussion Draft, H.R. ___, Communications regarding intended
uses of drugs and devices; scientific exchange................. 112
Letter of July 10, 2017, from Academy of Managed Care Pharmacy,
et al., to Mr. Guthrie, submitted by Mr. Guthrie............... 115
Letter of May 2, 2017, from the Alliance of Specialty Medicine,
to Mr. Griffith, submitted by Mr. Griffith..................... 117
Letter of May 16, 2017, from Dystrophic Epidermolysis Bullosa
Research Association of America, Inc., et al., to Mr. Griffith,
submitted by Mr. Griffith...................................... 118
Letter of May 5, 2017, from Mary R. Grealy, President, Healthcare
Leadership Council, to subcommittee leadership, submitted by
Mr. Griffith................................................... 120
----------
\1\ Dr. Kesselheim and Ms. Khachatourian did not answer submitted
questions for the record by the time of printing.
Letter of July 11, 2017, from Michael Carome, M.D., Director,
Public Citizen's Health Research Group, to Mr. Burgess and Mr.
Green, submitted by Mr. Green.................................. 122
Statement of John Rother, Executive Director, The Campaign for
Sustainable Rx Pricing, July 12, 2017, submitted by Mr. Green.. 143
Statement of America's Health Insurance Plans, July 12, 2017,
submitted by Mr. Green......................................... 147
EXAMINING MEDICAL PRODUCT MANUFACTURER COMMUNICATIONS
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WEDNESDAY, JULY 12, 2017
House of Representatives,
Subcommittee on Health,
Committee on Energy and Commerce,
Washington, DC.
The subcommittee met, pursuant to call, at 10:18 a.m., in
Room 2322, Rayburn House Office Building, Hon. Michael C.
Burgess (chairman of the subcommittee) presiding.
Members present: Representatives Burgess, Guthrie, Barton,
Upton, Shimkus, Blackburn, Lance, Griffith, Bilirakis, Bucshon,
Brooks, Mullin, Hudson, Collins, Carter, Walden (ex officio),
Green, Engel, Schakowsky, Butterfield, Matsui, Castor,
Sarbanes, Kennedy, Cardenas, Eshoo, and Pallone (ex officio).
Staff present: Adam Buckalew, Professional Staff Member,
Health; Daryll Dykes, Health Fellow; Paul Edattel, Chief
Counsel, Health; Adam Fromm, Director of Outreach and
Coalitions; Jay Gulshen, Legislative Clerk, Health; Alex
Miller, Video Production Aide and Press Assistant; Jennifer
Sherman, Press Secretary; Danielle Steele, Policy Coordinator,
Health; John Stone, Senior Counsel, Health; Hamlin Wade,
Special Advisor for External Affairs; Jeff Carroll, Minority
Staff Director; Samantha Satchell, Minority Policy Analyst;
Andrew Souvall, Minority Director of Communications, Member
Services, and Outreach; Kimberlee Trzeciak, Minority Senior
Health Policy Advisor; and C.J. Young, Minority Press
Secretary.
Mr. Burgess. The Subcommittee on Health will now come to
order. I will recognize myself for 5 minutes for the purpose of
an opening statement.
OPENING STATEMENT OF HON. MICHAEL C. BURGESS, A REPRESENTATIVE
IN CONGRESS FROM THE STATE OF TEXAS
From last year's 21st Century Cures Act to this year's Food
and Drug Administration reauthorization, this subcommittee has
been committed to bringing Federal regulation into the modern
era of medicine. Today, we continue that work by examining
legislation to update the regulatory framework affecting the
dissemination of truthful and nonmisleading information about
products approved by the Food and Drug Administration.
I practiced medicine for several decades. I know firsthand
how challenging it is it and how challenging it can be for
providers to stay up to the minute with cutting-edge
information in both medicine and science. Following the Food
and Drug Administration's approval of a product, the use of
that product rapidly evolves based on patient and provider
experience. Frequently, the standard of care for a condition is
outside of the Food and Drug Administration-approved labeling.
Ensuring that healthcare providers have access to new
information generated by real-world evidence is critical to
optimizing patient care and outcomes. Particularly in medicine,
the old adage holds true, knowledge is power.
Our legal framework for the regulation of manufacturer
communications sometimes prevents healthcare professionals from
receiving the most current scientific information available
about the benefits and risks of FDA-approved medicines. A lack
of relevant information can lead to physicians making patient
care decisions with incomplete information. This is both unfair
to the physician and unsafe for the patient.
We owe it to the patient and medical communities to ensure
that there is free and full dissemination of truthful and
nonmisleading scientific and medical information for healthcare
professionals.
I certainly want to thank two of our committee members, the
vice chairman of the committee, Brett Guthrie, and
Representative Morgan Griffith from Virginia for offering the
bills that will be under discussion today. I feel they offer a
targeted approached to addressing the problems presented by our
regulatory framework for medical product communication. And, if
he would like time, I am prepared to yield to the gentleman
from Kentucky, if he would like time for an opening statement.
[The prepared statement of Mr. Burgess follows:]
Prepared statement of Hon. Michael C. Burgess
From last year's 21st Century Cures Act, to this year's FDA
Reauthorization, this subcommittee has been steadfast in its
commitment to bring Federal regulation into the modern era of
medicine. Today we will continue that work by examining
legislation to update the regulatory framework affecting
dissemination of truthful and nonmisleading information about
FDA-approved products.
I practiced as a physician for several decades, and so I
know firsthand how challenging it can be for providers to stay
abreast of cutting-edge information in medicine and science.
Following FDA-approval of a product, the use of that product
rapidly evolves based on patient and provider experience.
Frequently the standard of care for a condition is outside of
the FDA-approved labeling. Ensuring that healthcare providers
are able to access new information generated by real-world
evidence is critical to optimizing patient care and outcomes.
Particularly in medicine, the old adage holds true-knowledge is
power.
Unfortunately, our legal framework for the regulation of
manufacturer communication prevents healthcare professionals
from receiving the most current scientific information
available about the benefits and risks of FDA-approved
medicines. A lack of relevant information can lead to
physicians making patient care decisions with incomplete
information. This is both unfair to the physician and unsafe
for the patient.
We owe it to the patient and medical communities to ensure
that there is free and full dissemination of truthful and
nonmisleading scientific and medical information to healthcare
professionals.
I would like to yield the balance of my time to Vice
Chairman Guthrie and Representative Griffith to discuss their
bills--each of which is a targeted approach to addressing the
problems presented by our outdated regulatory framework for
medical product communication.
Mr. Guthrie. Thank you, Mr. Chairman. There is another very
important hearing on opioids going on downstairs, and we have
our Kentucky Justice Secretary there.
Mr. Chairman, I want to thank you for holding this hearing
today to examine communications between manufacturers and
healthcare payers which I addressed in my bill, H.R. 2026, the
Pharmaceutical Information Exchange Act. My bill will enable
greater information exchange in order to guide health plans,
pharmacy benefit managers, and others who develop prescription
drug formularies and medical devices to make well-informed
decisions about the benefits and costs of medications and
medical devices for the populations they cover.
Patients benefit when these formulary decisions are
informed by the most recent and reliable scientific evidence on
drugs and devices beyond just what we learn from the clinical
trials conducted by FDA approval. Our committee has addressed
post-approval information exchange. We should take the next
logical step by addressing what information can and should be
exchanged preapproval by considering the updated discussion
draft we are examining today.
I would like to submit for the record a letter of support
for my bill by a number of organizations, including the Academy
of Managed Care Pharmacy, Humana, Sanofi, and Mayo Clinic.
Mr. Burgess. Without objection, so ordered.
[The information appears at the conclusion of the hearing.]
Mr. Guthrie. Thank you, Mr. Chairman. I yield back.
Mr. Burgess. The Chair thanks the gentleman. The Chair
would like to recognize the gentleman from Virginia, Mr.
Griffith, if he would seek time for an opening statement.
Mr. Griffith. Thank you very much, Mr. Chairman, I do
appreciate it. Mr. Guthrie and I were both downstairs
introducing former colleagues from the House of Delegates, so
we apologize that we came rushing in, but we got that done.
The draft version of my bill that we are discussing today
will responsibly set the rules of the road so that
manufacturers have the most accurate and up-to-date information
about their products that can provide doctors and researchers
with that information, and in the appropriate context, to
improve patient care and facilitate additional research.
Not only does the lack of clear rules have a public health
ramification, but also it has legal consequences. There have
been a number of court decisions that raise significant First
Amendment questions about the FDA's authority to restrict a
drug or device manufacturer from communicating truthful and
nonmisleading off-label information about their products.
The Judiciary Branch should not be turned into de facto
policy makers because of FDA's misunderstanding of the law or
our inaction here in Congress.
I remain open to any and all suggestions from both sides of
the aisle and from stakeholders as to how this legislation may
be improved, but I am glad we are continuing the dialogue.
Also, I also forward to hear from witnesses today about how the
FDA's vague policies hinder the facilitation of information to
healthcare providers and how this legislation could be a first
step in addressing some of the challenges that we will hear
about. Thank you. I yield back.
Mr. Burgess. The gentleman yields back, and the Chair yield
back. The Chair recognizes the ranking member of the
subcommittee, Mr. Green, 5 minutes for an opening statement,
please.
OPENING STATEMENT OF HON. GENE GREEN, A REPRESENTATIVE IN
CONGRESS FROM THE STATE OF TEXAS
Mr. Green. Thank you, Mr. Chairman. Today, we are
considering two draft bills addressing pharmaceutical
manufacturer communications on medical products. The Medical
Product Communications Act and the Pharmaceutical Information
Exchange Act suggest the changes of the rules surrounding the
communications from medical product manufacturers will likely
have far-reaching implications for decisions made by healthcare
providers about which therapies are appropriate for their
patients. It is critically important for us to fully consider
and appreciate the impact those proposed changes could have on
patient safety, health outcomes, and the promotion of value in
our healthcare system.
My concern with the two bills we are considering today is
that as drafted they would undermine public health, discourage
pharmaceutical research, and undermine the FDA's central
capacity to ensure medical products used on patients have
demonstrated safety and efficiency. Opening the floodgates for
off-label communication puts patients at risk, puts a dent in
the armor that ensures patients get effective therapies, and
not snake oil.
Broadening off-label communications could erode FDA's
approval standard as it would enable the uses of products never
found to be safe or effective in patients and weaken consumer
confidence in the FDA approval process. FDA's approval standard
of safety and efficiency is considered to be the gold standard
globally. As the FDA Commissioner Dr. Scott Gottlieb has said,
the most important incentive to developing useful information
remains the ability for companies to market drugs based on what
has been proven scientifically. There is an incentive currently
for companies to seek FDA's approval for all uses of a drug
product if they wish to market the product for those uses and
gain coverage for these uses.
Allowing manufacturers to communicate about unproven uses
of their products reduces the incentive to go through the FDA's
approval process as clinical trials are the most expensive part
of the development. Thus, it is not hard to imagine a scenario
where a company seeks the narrowest indication for their
product, gets on the market, and forgoes on continuing large,
well controlled, randomized clinical trials that would prove a
product is both safe and effective for broader populations or
indications. Patients and doctors should fully be empowered to
make joint decisions about their care. This includes the
efficiency, risk, and cost of their options.
Information is key, however, and the best decisions are
based on accurate, evidence-based information, not just for
information that may be incomplete, inconclusive, or at worst
inaccurate. The discussion draft of the Medical Product
Communications Act would not provide or ensure that patients
and care providers have access to better research and evidence.
Rather, it would allow drug manufacturers to communicate
information about prescription drugs that have not been
approved by the FDA. The lack of approval may be due to
contradictory evidence or the lack of any evidence at all, or
the need for additional research.
While I have concerns with both discussion drafts as
written, I do appreciate that our audience matters. The
discussion draft of the Pharmaceutical Information Exchange Act
would expand the ability of drug and device manufacturers to
communicate healthcare economic information, and scientific
information to payers, formularies, technology review
committees, or other entities about unapproved uses of
products. These audiences are sophisticated and have an
inherent interest in being skeptical of claims made outside a
product's label. Therefore, it is less problematic in its
premise than the other bill we are considering.
While I am willing to work with my colleagues on the
proposal, it is critical that these communications promote
patient safety, public health, and the appropriate safeguards
are in place to avoid damaging unintended consequences. As we
consider the issue of off-label communication, we must always
keep in mind that the way to truly help patients get the most
effective treatments is to maintain the highest standards of
safety and evidence and appropriate risk of benefit balance.
Scientifically validated safety and efficiency and the
incentives for manufacturers to seek FDA approval are clear and
should be preserved. I look forward to hearing from our
witnesses and if anybody wants time, I will yield my 45 seconds
back. Thank you, Mr. Chairman.
Mr. Burgess. The gentleman yields back. The Chair thanks
the gentleman. The Chair recognizes the gentleman from Oregon,
the chairman of the full committee, Mr. Walden, 5 minutes for
an opening statement, please.
OPENING STATEMENT OF HON. GREG WALDEN, A REPRESENTATIVE IN
CONGRESS FROM THE STATE OF OREGON
Mr. Walden. I thank the subcommittee chairman, Chairman
Burgess. Thanks for this holding this hearing. It is a really
important topic, and it is a topic that has been important for
our Members for some time.
Approximately 40 percent, 40 percent of prescriptions in
the United States are for indications or uses not included in
the FDA-approved product labeling. Although off-label uses of
drugs and devices are often the recognized standard for care
for treating many conditions, the lack of clarity in the
statute and implementing regulations has stifled important
information about such uses for being communicated in a
responsible and nonpromotional manner by manufacturers.
The FDA has attempted to address this issue, but it has
been in a piecemeal fashion or the last 2 decades with various
nonbinding guidance documents and policy statements that
frankly fall woefully short, particularly given the criminal
penalties in play.
As the Supreme Court affirmed in 2011 that First Amendment
commercial speech protections extend to medical product
manufacturers, every subsequent judicial decision, every
decision, has raised significant questions about the extent of
FDA's authority to restrict truthful and nonmisleading off-
label communications.
So where are we today? The regulators and the courts have
spoken. Everyone is left with a vast amount of uncertainty that
does nothing to protect or benefit patients. So it is time for
Congress to act. And as FDA's authorizing committee, it is our
job to clarify this statute and get it right which brings us to
this hearing. Neither of these bills are new to my fellow
committee members. We discussed an earlier version of both
bills during a markup in this subcommittee back in May and we
reviewed these updated versions of the full committee markup of
the FDA Reauthorization Act last month. Both bills were
ultimately withdrawn as amendments to FDARA with a commitment
from our colleagues on the other side of the aisle to work with
us together to iron out a compromise so we could move these
important policies forward and speak as the Congress and not
leave this up to a mishmash of court decisions.
So I look forward to continuing that work today.
I believe Morgan Griffith's bill, H.R. 1703, is a serious,
well-thought-out policy proposal that responsibly sets the
rules of the road in a constitutionally sound manner. I greatly
appreciate his willingness to continue to address concerns. He
has heard about the legislative language.
I also appreciate Ranking Member Pallone's commitment at
the user fee markup to work with us in good faith on this issue
through regular order which starts with this important hearing.
In addition. Representative Guthrie's amended version of
H.R. 2026 would clarify how drug and medical device companies
can share healthcare, economic, or scientific information
related to investigational uses of their products with payers
and similar entities. These bills do not provide manufacturers
with free reign to communicate any and all information about
their products. They establish targeted, statutory boundaries
within which manufacturers may responsibly disseminate accurate
and up-to-date information about medical products. These
clarifications will lead to a better informed healthcare
system. They will ensure that patients receive high-quality
care based on current sound, scientific, and clinical
information.
Today, we continue the dialogue. I look forward to a
productive discussion and I appreciate the input of our
witnesses who are before us today, and with that, unless there
are other Members who would like to use the balance of my time,
I will yield back the balance of my time.
Mr. Burgess. The Chair thanks the gentleman. The gentleman
yields back. The Chair recognizes the gentleman from New
Jersey, the ranking member of the full committee, 5 minutes for
an opening statement, please.
[The prepared statement of Mr. Walden follows:]
Prepared statement of Hon. Greg Walden
Thank you, Chairman Burgess, for holding today's hearing on
an increasingly important topic--one that has been a topic
amongst our Members for some time.
Approximately 40 percent of prescriptions in this country
are for indications or uses not included in the FDA-approved
product labeling. Although ``off-label'' uses of drugs and
devices are often the recognized standard of care for treating
many conditions, the lack of clarity in the statute and
implementing regulations has stifled important information
about such uses from being communicated in a responsible,
nonpromotional manner by manufacturers.
FDA has attempted to address this issue in a piecemeal
fashion over the last two decades with various nonbinding
guidance documents and policy statements that fall woefully
short, particularly given the criminal penalties in play.
Since the Supreme Court affirmed in 2011 that First
Amendment commercial speech protections extend to medical
product manufacturers, every subsequent judicial decision has
raised significant questions about the extent of FDA's
authority to restrict truthful and nonmisleading off-label
communications.
So, where are we today? The regulators and the courts have
spoken. Everyone is left with a vast amount of uncertainty that
does nothing to protect or benefit patients.
It is past time for Congress to act, and as FDA's
authorizing committee it is our job to clarify the statute.
Which brings us to this hearing. Neither of these bills are
new to my fellow committee members. We discussed an earlier
version of both bills during a markup in this subcommittee back
in May, and we reviewed these updated versions at the full
committee markup of the FDA Reauthorization Act (FDARA) last
month.
Both bills were ultimately withdrawn as amendments to
FDARA, with a commitment from our Democrat colleagues to
continue to work together to iron out a compromise on moving
these important policies forward. I look forward to continuing
that work today.
I believe Morgan Griffith's bill, H.R. 1703, is a serious,
well-thought-out policy proposal that responsibly sets the
rules of the road in a constitutionally sound manner. I greatly
appreciate his willingness to continue to address concerns he
has heard about the legislative language. I am also
appreciative of Ranking Member Pallone's commitment at the user
fee markup to work with us on this issue through regular order,
which starts with this important hearing.
In addition, Rep. Guthrie's amended version of H.R. 2026
would clarify how drug and medical device companies can share
healthcare, economic, or scientific information related to
investigational uses of their products with payers and similar
entities.
These bills do not provide manufacturers with free rein to
communicate any and all information about their products. They
establish targeted statutory boundaries within which
manufacturers may responsibly disseminate accurate, and up-to-
date information about medical products. These clarifications
will lead to a betterinformed health care system and will
ensure that patients receive high-quality care based on
current, sound scientific and clinical information.
Today we continue the dialogue, and I look forward to a
productive discussion.
OPENING STATEMENT OF HON. FRANK PALLONE, JR., A REPRESENTATIVE
IN CONGRESS FROM THE STATE OF NEW JERSEY
Mr. Pallone. Thank you, Mr. Chairman. I want to thank you
for holding today's hearing. The issue before us today is an
important one and I hope that our discussion today will help to
inform whether or not it would be appropriate for this
committee to take further action.
Today, under current law, medical product manufacturers are
required to demonstrate the safety and effectiveness of each
intended use of their medical product. This review process has
been critical to protecting and promoting public health by
ensuring that the benefits of medical products that are
prescribed to patients outweigh the risk. It also is common
sense. Just because a medical product approved for one use may
be found to be safe and effective for that use, doesn't
necessarily mean that it will be safe and effective for another
use or for another population.
Recognizing that physicians may prescribe treatments off-
label in response to individual patient needs, FDA allows the
communication of truthful and nonmisleading scientific or
medical information regarding unapproved uses of medical
products that may assist physicians in making treatment
decisions. In those instances, FDA has allowed for
manufacturers to respond to requests from physicians about
unapproved uses and provide peer reviewed journal articles,
scientific or medical texts, and clinical practice guidelines.
Following 21st Century Cures, manufacturers are also now
able to share healthcare economic information with payers to
help them better understand the economic benefits of an
approved treatment.
These are commonsense approaches that allow doctors to
address the individual needs of a patient, but also ensure that
patients are not unnecessarily exposed to unproven or harmful
medical products.
Now today, we are here to examine discussion drafts from
Representatives Griffith and Guthrie that would greatly expand
the types of scientific information that manufacturers could
share without any FDA oversight. While I understand that
medical product manufacturers have voiced concerns about their
ability to communicate with doctors about their products, I am
concerned that these drafts would severely undermine the
current protections against marketing unsafe and ineffective
medical products.
During this hearing, I hope to hear what materials
manufacturers want to share with healthcare professionals and
payers that they feel they can't under current law.
The scientific exchange discussion draft would severely
restrict the types of evidence the FDA has always relied on to
determine the intended use of a medical product. It would also
hamstring the Agency from holding bad actors who distribute
dangerous drugs or medical devices accountable.
The preapproval communication discussion draft will blow a
hole in the current approval process by allowing the
communication of any scientific evidence or healthcare economic
information to payers or formularies without any recourse to
the FDA to prevent bad actors from communicating false or
misleading information. Allowing manufacturers to communicate
about unapproved products and unapproved uses of their products
reduces the incentive of those through FDA's approval process
and that is grossly irresponsible in my opinion.
For example, the proposed discussion draft would allow for
a manufacturer to publish a biased, scientific study in any
medium to constitute scientific exchange. This could simply
include posting results of a nonpeer-reviewed study on a
company's website, and there is no requirement that this
information be truthful.
I am also concerned that these two discussion drafts could
expose more patients to medical products that have never been
proven to be safe or effective. One study found that 81 percent
of medications prescribed for off-label purposes had poor or no
scientific support, while another found that patients who
received off-label prescriptions were 54 percent more likely to
experience an adverse event, as compared to on-label use. And
these are risks that we simply cannot ignore.
So Mr. Chairman, if there is a need for greater certainty
and clarity on the types of communications that manufacturers
are permitted to use under current law, I am willing to have
that discussion. However, broadening communication in the way
it is proposed under these discussion drafts would, in my
opinion, undermine FDA's regulatory review process and the
safety and effectiveness approval standard.
I have about a minute. I don't know if anybody wants it. If
not, I yield back, Mr. Chairman.
[The prepared statement of Mr. Pallone follows:]
Prepared statement of Hon. Frank Pallone, Jr.
Mr. Chairman, I want to thank you for holding today's
hearing. The issue before us today is an important one, and I
hope that our discussion will help to inform whether or not it
would be appropriate for this committee to take further action
at this time.
Today, under current law, medical product manufacturers are
required to demonstrate the safety and effectiveness of each
intended use of their medical product. This review process has
been critical to protecting and promoting public health by
ensuring that the benefits of medical products that are
prescribed to patients outweigh the risks. It is also
commonsense--just because a medical product approved for one
use may be found to be safe and effective for that use, does
not necessarily mean that it will be safe and effective for
another use or for another population.
Recognizing that physicians may prescribe treatments off-
label in response to individual patient needs, FDA allows the
communication of truthful and nonmisleading scientific or
medical information regarding unapproved uses of medical
products that may assist physicians in making treatment
decisions. In these instances, FDA has allowed for
manufacturers to respond to requests from physicians about
unapproved uses and provide peer-reviewed journal articles,
scientific or medical texts, and clinical practice guidelines.
Following 21st Century Cures, manufacturers are also now able
to share health care economic information with payors to help
them better understand the economic benefits of an approved
treatment.
These are commonsense approaches that allow doctors to
address the individual needs of a patient, but also ensure that
patients are not unnecessarily exposed to unproven or harmful
medical products.
Today, we are here to examine discussion drafts from
Representatives Griffith and Guthrie that would greatly expand
the types of scientific information that manufacturers could
share without any FDA oversight. While I understand that
medical product manufacturers have voiced concern about their
ability to communicate with doctors about their products, I am
concerned that these drafts would severely undermine the
current protections against marketing unsafe and ineffective
medical products. During this hearing, I hope to hear what
materials manufacturers want to share with health care
professionals and payors today that they feel they cannot under
current law.
The scientific exchange discussion draft would severely
restrict the types of evidence the FDA has always relied on to
determine the intended use of a medical product. It would also
hamstring the agency from holding bad-actors who distribute
dangerous drugs or medical devices accountable.
The preapproval communication discussion draft would blow a
hole in the current approval process by allowing the
communication of any scientific evidence or health care
economic information to payors or formularies without any
recourse for the FDA to prevent bad actors from communicating
false or misleading information. Allowing manufacturers to
communicate about unapproved products and unapproved uses of
their products, reduces the incentive to go through FDA's
approval process. This is grossly irresponsible.
For example, the proposed discussion draft would allow for
a manufacturer to publish a biased scientific study in any
medium to constitute ``scientific exchange.'' This could
include simply posting results of a nonpeer reviewed study on a
company's own website, and there is no requirement that this
information be truthful.
I am concerned these two discussion drafts could expose
more patients to medical products that have never been proven
to be safe or effective. One study found that 81 percent of
medications prescribed for off-label purposes had poor or no
scientific support, while another found that patients who
received off-label prescriptions were 54 percent more likely to
experience an adverse event as compared to on-label use. These
are risks that we simply cannot ignore.
If there is a need for greater certainty and clarity on the
types of communications that manufacturers are permitted to use
under current law, I am willing to have that discussion.
However, broadening communication in the ways proposed under
these discussion drafts would undermine FDA's regulatory review
process and the safety and effectiveness approval standard.
Thank you.
Mr. Burgess. The gentleman yields back. The Chair thanks
the gentleman. This concludes Member opening statements, and I
would like to remind Members that, pursuant to committee rules,
all Members' opening statements will be made part of the
record.
And we want to thank our witnesses for being here with us
this morning, for taking time to testify before the
subcommittee. Each witness will have the opportunity to give a
summary of their opening statement, followed by questions from
Members.
This morning, we are going to hear from Coleen Klasmeier, a
partner of Sidley Austin, LLP; Alta Charo, the Warren Knowles
Professor of Law at the University of Wisconsin; Dr. George Van
Hare, the Division Chief, Pediatric Cardiology; Louis Larrick
Ward, Professor of Pediatrics at Washington University School
of Pediatrics; and Co-Director of the St. Louis Children's and
Washington University Heart Center; Aaron Kesselheim, Associate
Professor of Medicine, Harvard Medical School, Director of
Program on Regulation, Therapeutics and Law from the Division
of Pharmacoepidemiology and Pharmacoeconomics at the Brigham
and Women's Hospital; Linda House, President of the Cancer
Support Community; and Katherine Wolf Khachatourian, Vice
President, Delegation Oversight, Pharmacy Services of
QualchoiceHealth Plan Services.
We appreciate all of you being here today and Ms.
Klasmeier, you are recognized for 5 minutes for the purpose of
an opening statement. Thank you for being here.
STATEMENTS OF COLEEN KLASMEIER, PARTNER, SIDLEY AUSTIN, LLP; R.
ALTA CHARO, WARREN P. KNOWLES PROFESSOR OF LAW, UNIVERSITY OF
WISCONSIN; GEORGE VAN HARE, M.D., PRESIDENT, HEART RHYTHM
SOCIETY; AARON S. KESSELHEIM, ASSOCIATE PROFESSOR OF MEDICINE,
HARVARD MEDICAL SCHOOL AND BRIGHAM AND WOMEN'S HOSPITAL; LINDA
HOUSE, PRESIDENT, CANCER SUPPORT COMMUNITY; AND KATHERINE WOLF
KHACHATOURIAN, VICE PRESIDENT, DELEGATION OVERSIGHT, PHARMACY
SERVICES, AND STRATEGY, QUALCHOICE HEALTH PLAN SERVICES, INC.
STATEMENT OF COLEEN KLASMEIER
Ms. Klasmeier. Thank you, Mr. Chairman. Chairman Burgess,
Vice Chairman Guthrie, Ranking Member Green, Chairman Walden,
members of the subcommittee, my name is Coleen Klasmeier. I am
a partner and the head of the FDA Regulatory Practice at Sidley
Austin in Washington, DC. I am appearing today on behalf of the
Medical Information Working Group.
Today, I would like to make three points. First, FDA's
rules governing manufacturer communications are neither clear
nor precise. Decisions to prescribe and use lawfully marketed
drugs and medical devices in ways that differ from the FDA
authorized labeling, so-called off-label use, are a constituent
part of medical and surgical practice and can also be the
standard of care. FDA has long recognized the need for
prescribers to receive and for manufacturers to have some
ability to provide information outside of product labeling to
help support clinical decision making. As a result, although a
manufacturer is prohibited from promoting its product for new
uses, it can lawfully provide information about off-label uses
within defined circumstances.
Currently, there are four safe harbors. Only one is set
forth in a binding regulation. The others are in nonbinding
documents. They therefore lack the force of law. Moreover, two
of the four safe harbors have been the subject of ongoing FDA
proceedings since 2011. Under these policies, a manufacturer
can provide off-label information ostensibly without fear of
enforcement in four scenarios involving scientific exchange,
responses to unsolicited requests, continuing education, and
reprints of journal articles, reference texts, and clinical
practice guidelines. Each safe harbor is subject to a number of
qualifying criteria and additional requirements which are
unclear in many key respects.
Moreover, FDA has been unable to complete its process of
revising the safe harbor policies, so questions frequently
arise regarding the relationship between the old policies and
the new policies.
In addition, there is a lack of symmetry between the safe
harbors that apply to drugs and those that apply to medical
devices. In short, the safe harbors are a mess. As a result,
manufacturers cannot confidently rely on the safe harbors and
that has public health consequences. For example, it is common
for the Advisory Committee on Immunization Practices, a Federal
statutory advisory committee to the CDC, to make
recommendations for vaccines that are arguably off-label. ACIP
recommendations might vary the dosing schedule or recommend use
of a vaccine in a new patient population. The vaccine
manufacturer would reasonably fear that communicating about the
ACIP recommendation to physicians or payers could be
characterized by Government as unlawful, off-label promotion.
Ultimately, the public health would not be advanced because
physicians would not receive manufacturer communications
reinforcing that recommendation.
The regulatory scheme also has legal consequences. The
First Amendment case law makes clear that FDA is limited in its
power to prohibit drug and device manufacturers from engaging
in accurate communications about their product. FDA's
regulatory scheme also implicates the due process laws of the
Fifth Amendment which requires Government agencies to establish
rules that are clear and to give fair notice of what is
prohibited, particularly in the context of free expression.
Second, the existing FDA regulatory scheme for manufacturer
communication is highly unstable. The lack of clear rules to
allow manufacturers an appropriate measure of latitude to
communicate about their products is only a part of the problem.
FDA and the Justice Department impose aggressive restraints on
manufacturers' speech. Although manufacturers have indeed
settled many cases involving off-label promotion allegations in
recent years, in some instances individuals and firms have
raised First Amendment arguments in court and those arguments
have succeeded. FDA's regulatory scheme continues to burden
constitutionally protected speech and is therefore at risk from
additional lawsuits.
The Medical Information Working Group has for more than 10
years and across more than 20 submissions, requested targeted
clarifications to the existing FDA safe harbors and to key
statutory terms such as labeling and intended use. We have not
asked for and we do not want a healthcare system in which
manufacturers can market their product based on spurious or
unsubstantiated claims of safety or efficacy.
Third, legislation could dramatically improve the
regulatory scheme. Although the MIWG has been dedicated to
direct engagement with FDA on manufacturer communication issues
since 2006, we also recognize the paramount role of Congress
and we believe that legislation may be necessary for several
reasons.
For one thing, FDA action has been slow and ineffectual. It
has been almost 6 years, for example, since FDA published a
notice in the Federal Register asking for comment on scientific
exchange and responses to unsolicited requests. Where FDA has
taken action, the policy has tacked in the wrong direction
becoming less clear and even more speech restrictive. For these
reasons, it would be helpful for Congress to step in and set
the overall policy direction for FDA to implement.
Legislation is also more durable than unilateral FDA
action. Statutory law is not subject to the same variability as
agency pronouncements and cannot be undone in a future
administration. Legislation would be less susceptible to legal
challenge than a regulation or an FDA guidance document.
Regulations have the force of law, but the Administrative
Procedure Act creates a vehicle for challenge in court, whereas
a statutory change could only be challenge successfully in
court on constitutional grounds.
Legislation may also be necessary given the likelihood of
continued judicial involvement in this area. Although we value
the contributions that recent judicial decisions have made to
the body of relevant law, we also believe that litigation is
not the best way to make law on important public health issues
where there is little room for error. We are especially
concerned that some future lawsuit might eviscerate the FDA
regulatory scheme.
We see great value in congressional engagement with FDA on
manufacturer communication issues to help assure the regulatory
scheme is put on to a more stable and sustainable footing.
Thank you very much for the opportunity to testify today and I
look forward to your questions.
[The prepared statement of Ms. Klasmeier follows:]
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Mr. Burgess. I thank the gentlelady for her testimony.
Ms. Charo, you are recognized for 5 minutes, please.
STATEMENT OF R. ALTA CHARO
Ms. Charo. Chairman Burgess, Vice Chairman Guthrie,
Congressman Green, and members of the committee, thank you for
the opportunity to address you on issues surrounding
communication and marketing of off-label uses.
My name is Alta Charo. I am the Warren P. Knowles Professor
of Law at the University of Wisconsin. I am an elected member
of National Academy of Medicine, formerly known as the
Institute of Medicine, where I have served on a number of
committees including the one that produced a report on ensuring
the safety of the U.S. drug system. I also served as an advisor
in the Office of the Commissioner at FDA from 2009 to 2011, but
I would like to note for the record that I speak for myself
only and not for FDA and not for the National Academies.
There are two possible reasons to expand communication
about off-label uses. One is to ensure that the law is
consistent with the requirements of the First Amendment. The
other is to protect public health by increasing patient access
to safe and effective drugs. And I share those two goals. I
don't, however, believe that the two amendments under
discussion are necessary to achieve those goals. Indeed, I fear
the unintended consequence of adopting the language in these
amendments would be to undermine public health, to discourage
pharmaceutical research, and to set pharmaceutical regulation
back by more than 100 years.
As noted in an article I co-authored with Josh Sharfstein,
formerly the principal deputy at FDA, our drug regulation
system has prohibited false or misleading advertising since
1906. And in 1962, broad marketing for secondary uses of
thalidomide caused thousands of severe birth defects worldwide,
and Congress then recognized that a product can be ``safe and
effective'' for one intended use where the benefits exceed the
risks, but not ``safe and effective'' for another which why
approval of a drug for a labeled indication does not mean it
will be safe and effective for off-label uses and precisely why
additional studies are needed.
This requirement to demonstrate safety and effectiveness
for an intended use applies both to the first approval of a new
compound or a new drug, as well as to any supplemental
indication. And while it is true there have been a handful of
cases narrowing constraints on commercial speech regarding
unapproved ``off-label'' uses, the courts have consistently
upheld commercial speech restriction with respect to the first
approval of a new product. If the First Amendment means that
off-label promotion must be permitted, then promotion of
entirely untested, never-approved drugs should also garner the
same protection. In both cases, the majority of drugs will fail
to show that they are safe and effective when the testing has
been completed and the substantial public interest in achieving
that certainty is the same regardless of whether it is an
entirely new drug or a supplemental indication for an existing
drug.
If we were to eliminate the restrictions on commercial
speech for entirely unapproved drugs, it would return us to the
1906 law where prosecution for false and misleading marketing
took place only after people had been harmed.
Scientific journals and conferences are already allowed to
present information about off-label uses. Sponsors can answer
questions from physicians and provide reprints of peer-reviewed
articles, even if related to off-label uses. And in April 2017,
the FDA further clarified these rules and used guidances as a
more flexible mechanism to provide that information.
Legislation, regulation, and court decisions have not the kind
of flexibility that guidances have. We have entered an era in
which communication takes on many new forms ranging from tweets
to Facebook to any number of internet sources and it is
important to maintain flexibility in how we regard
communication and its influence and its intended purpose,
rather than solidifying it in legislation which can be
difficult to change over time.
Now the proposed amendment of Section 201 muddies the
exceptions that FDA has outlined and I fear it risks
eviscerating the general rule against off-label promotion even
if that is not its intent. It also has the effect of immunizing
sponsors from responsibility even if they know and take
advantage of the now blurry line between legitimate scientific
exchange and illegal marketing.
The proposed amendment of Section 502, I fear, will
exacerbate this problem, by allowing premature information to
be delivered to formularies and payers with the probable effect
of increasing patient use of unproven and unsafe therapies. And
as has been noted already by Members here on the committee,
studies have repeatedly shown that even products that look
promising in early trials will usually be shown to be unsafe or
ineffective when larger trials are completed. And indeed,
overall only about one in five compounds, only one in five,
will successfully move from Phase 2 to Phase 3 trial, with lack
of efficacy as the most common reason for failure.
In a series of articles recently produced by Professor
Christopher Robinson at the University of Arizona, we can also
see that multiple studies show that the majority of off-label
uses also will turn out to be either unsafe or ineffective.
Encouraging coverage before approval is to encourage expanded
use before approval of treatments that we now know empirically
are likely to fail. And I fear that the effect would be to
increase use that will harm more patients than it helps.
History amply demonstrates there is a compelling public
interest in unbiased evaluation of evidence; in clear, accurate
communication; in maintaining incentives for research. The
combined effect of these amendments is to expand promotion and
payment for unproven uses of drugs. It undercuts the marketing
advantages that the law now uses as an incentive for sponsors
to complete the research needed to see which uses are, in fact,
safe and effective. And in turn, it leaves physicians,
patients, formularies, and payers without independently
verified information. For complex products like drugs, the
marketplace of ideas cannot work properly with unvetted
information from necessarily self-interested sources. And when
using the wrong drug can injure patients or cause them to miss
out on effective treatment, it is an invitation to another
tragedy when we prevent FDA from doing its job to protect the
public.
Thank you very much.
[The prepared statement of Ms. Charo follows:]
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Mr. Burgess. The Chair thanks the gentlelady.
The Chair recognizes Dr. Van Hare, 5 minutes for your
opening statement, please.
STATEMENT OF GEORGE VAN HARE
Dr. Van Hare. Good morning, Chairman Burgess, Ranking
Member Green, and members of the subcommittee. Thank you for
holding this hearing and for inviting me to testify on this
important topic. My name is George Van Hare. I am Chief of
Pediatric Cardiology at St. Louis Children's Hospital in St.
Louis, Missouri. My clinical practice is focused on caring for
children with heart rhythm disorders. This year I have the
honor of serving as the president of the Heart Rhythm Society.
The Heart Rhythm Society is the international leader in
science, education, and advocacy for cardiac arrhythmia
professionals. Its members include 6,100 physicians,
scientists, nurses, and other allied health professionals in
more than 90 countries.
Sharing comprehensive, scientifically valid data is
critical to the practice of medicine generally, and it is even
more critical for particular specialties. It is sometimes
claimed that the use of drugs or devices off-label is the
result of a choice by physicians. Sometimes this is
true. However, for pediatric sub-specialists, this is
usually not the case. This is due to the fact that very few of
the medications for arrhythmias that are on the market are
formally approved for use in children. Thus, using treatments
off-label is often our main method of
treatment of children. Similarly, catheters that we use for
catheter ablation procedures are labeled for a limited number
of specific arrhythmias, but are used for treating and curing
all types of arrhythmias in adults and children.
By way of example, I would like to cite the specific drug,
amiodarone, brand name Cordarone. This is one of our most
important medications for the treatment of potentially life-
threatening arrhythmias, particularly in patients who have
undergone successful surgical repair of complex
congenital heart defects. The FDA-approved label simply
states ``The safety and efficacy of Cordarone Tablets in
pediatric patients have not been established.'' This means that
the manufacturer is not allowed to share prospectively any data
that they may have concerning experience with this drug in
children.
Another example, not specific to children, is a labeling of
ablation catheters. These devices are used in performing
catheterization procedures to cure arrhythmias. In the last 25
years, these procedures have essentially replaced open heart
surgery as the best option for a curative procedure. Their
labeling is limited to only certain arrhythmias. For example,
the Cryocath, a cryoablation catheter manufactured by
Medtronic, is only labeled for treating one common arrhythmia,
AVNRT, despite the fact that it is ideal for treating other,
more dangerous arrhythmias. It would be absurd to use a
different catheter for these other arrhythmias on the basis of
the labeling, and even more absurd if you consider open heart
surgery. However, because of the labeling, technical support
representatives of the manufacturer are not allowed to discuss
other indications directions and prospectively, despite the
fact that the use of this catheter for these other indication
sis widely agreed to be the standard of care.
There is an important way in which information sharing
among physicians may also be adversely affected. When a medical
conference is directly sponsored by a manufacturer, these
conferences do not qualify as official continuing medical
education events. Consequently, physician speakers are
considered to be ``agents'' of the manufacturer sponsoring the
event, and they are also limited to discussing only the labeled
indications. Any discussion between physicians regarding
experiences with drugs or devices that are off-label at such
events must occur informally, rather than as part of the
program, and thus these discussions do not benefit from the
great potential for information sharing among physician
attendees.
The good news is that it doesn't have to be this way. It is
likely that there is a large amount of data maintained by
manufacturers, which under the current rules they are not
allowed to proactively share with clinicians. I urge the
committee to explore ways to define acceptable types of real-
world evidence that manufacturers might proactively share with
medical decision makers. These types of data might include
observational studies, pharmacokinetic studies, and information
on particular sub-populations. The data must be truthful,
presented in context, and scientifically valid.
There is some concern that manufacturers might overwhelm
physicians with data taken out of context or data that are
misleading and skewed to present a more favorable picture than
is realistic. However, physicians are trained to analyze data.
We know how to evaluate the validity of studies. If regulatory
restrictions provide guard rails to ensure that data are
truthful and presented in context, physicians are fully capable
of analyzing such data effectively.
In my opinion, a reasonable regulatory paradigm lies
somewhere between no communication and completely unrestricted
communication. The current structure is not optimal for
fostering the advancement of medical knowledge, and it leaves
many patients and their physicians at an unnecessary
disadvantage. Additionally, it seems incongruous to me that the
manufacturer, the entity with the most robust data related to a
product, cannot share information they hold proactively while
any lay person with an internet connection can freely
disseminate whatever information they like about that same
product however biased and unreliable.
In closing, I hope that my testimony has provided the
committee with a real-world perspective on how the current
rules often prevent physicians from receiving valuable,
clinical information in a timely fashion. I respectfully
suggest that Congress should establish ways to unlock
data maintained by manufacturers related to off-label use
of drugs and devices. I thank the committee for its time. The
Heart Rhythm Society would welcome the opportunity to work with
you on policy proposals related to this topic. Thank you.
[The prepared statement of Dr. Van Hare follows:]
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Mr. Burgess. The Chair thanks the gentleman for his
testimony.
Dr. Kesselheim, you are recognized for 5 minutes for your
statement, please.
STATEMENT OF AARON S. KESSELHEIM
Dr. Kesselheim. Good morning, Chairman Burgess, Ranking
Member Green, and other members of the committee, thank you for
the opportunity to join you today. In my time I want to make
four main points.
First, the current restrictions on manufacturers' ability
to market their drugs for non-FDA-approved indications is not a
bureaucratic or paternalistic effort to prevent manufacturers
from communicating. These rules were developed in response to
major public health problems caused by the lack of such
regulation. Evidence of the public health dangers that arise
from widespread off-label marketing can be seen in the drug
paroxetine or Paxil, an antidepressant that was promoted off-
label for use in children leading to at its peak over two
million prescriptions per year for use in children until it was
ultimately linked to self-injury and suicide in that
population. Or, the off-label promotion of anti-psychotic
medications to control behavioral symptoms in elderly patients
with dementia, uses that are not only generally ineffective,
but that also increase the risk of death by 60 to 70 percent.
At one point, due to off-label promotion approximately one
in seven elderly nursing home residents reportedly received
these drugs.
Over and over again, these episodes show us, as former
Chief Justice William Rehnquist originally put it that ``there
are sufficient dangers attending [the] widespread use [of
pharmaceuticals] that they simply may not be promoted in the
same manner as hair creams, deodorants, and toothpaste.''
Second, the dangers from off-label promotion do not come
simply from the spread of false information about these
products, although that does happen on occasion of course.
Rather, in one study that I led, we found that off-label
promotion most commonly involved presenting reports of
individual cases or poorly designed studies as definitive
evidence supporting an off-label use, while de-emphasizing data
that didn't fit the narrative the manufacturers were creating.
In each of these particular cases, the words themselves may not
have been false or strictly misleading, but the benefits of the
drug overstated and the risks down played in ways that the
physicians might have needed advanced training in epidemiology
or access to the underlying clinical trial data to understand
which they simply do not have. This is why we need the
diligent, independent assessment of safety and efficacy
provided by the FDA. The complexity of the assessment that is
required, along with the high stakes of getting that assessment
wrong provides the rationale for having a formal drug approval
process in the first place.
Third, the Griffith and Guthrie discussion drafts directly
risk these outcomes. The Guthrie discussion draft, for example,
defines scientific information that could support an off-label
marketing claim as including preclinical data in petri dishes
or in mice, and all it requires is a study that was conducted
that the manufacturer anticipates could be sufficient to
support FDA approval.
The Griffith draft, in creating a so-called safe harbor for
scientific exchange, purports to require manufacturers to
disclose appropriate contextual information for their
statements, but it would be highly risky to give a manufacturer
with a strong financial and intellectual stake in the product's
success free reign to determine what is or isn't proper context
or what is or isn't contradictory for its product. At the same
time, it is unrealistic to expect each individual physician to
have the time and expertise to subject such claims to the same
kind of scrutiny that the FDA would exercise when it reviews a
drug application or a request for a new indication.
The drafts also purport to protect the public health by
attaching disclaimers to these off-label communications, but I
led a systematic review of the evidence about the impact of
such disclaimers, most of which currently come in the context
of promotional statements for herbal remedies and dietary
supplements for which Congress eliminated FDA oversight of
promotion more than 20 years ago. Many of these products
advertise health-enhancing effects despite no legitimate
evidence that they work with disclaimers that the FDA has not
evaluated the promotional claims, but the massive collective
evidence reveals that such disclaimers fail to adequately
inform or modify consumer behavior. So when anybody proposes a
disclaimer, I suggest that there be a disclaimer, that
disclaimers don't actually work.
Finally, I want to emphasize that the current system helps
protect patients from widespread promotion of drugs and devices
for potentially unsafe and ineffective off-label uses, while
still permitting off-label prescribing at the discretion of
physician and patients and providing well-circumscribed avenues
for manufacturer communication about these issues such as in
response to bona fide questions arising from physicians. By
contrast, the Griffith and Guthrie discussion drafts would
reduce manufacturers' incentives to conduct well-controlled
trials of potential off-label uses in the first place. Instead,
as Representative Green mentioned, manufacturers would be
incentivized to seek approval of drugs and devices for the
narrowest indication possible, and then conduct ``studies'' of
variable quality showing the utility of these products for
unapproved indications that would not meet current FDA
standards for scientific rigor.
I strongly recommend that the committee not pursue these
drafts and instead consider how we can give the FDA the proper
resources and authorities to continue to review emerging data
efficiently so that evidence that does support new uses of
drugs and devices can be incorporated into their labels and
clinical practice while uses that the totality of the data show
are unsafe can be identified for the benefits of patients.
Thank you very much.
[The prepared statement of Dr. Kesselheim follows:]
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Mr. Burgess. The Chair thanks the gentleman.
Ms. House, you are recognized for 5 minutes for an opening
statement, please.
STATEMENT OF LINDA HOUSE
Ms. House. Good morning. My name is Linda House, and I am
the president of the Cancer Support Community. I would like to
thank the committee for allowing us to be here and share this
testimony today.
The Cancer Support Community is an international nonprofit
organization whose mission is to ensure that all people
impacted by cancer are empowered by knowledge, strengthened by
action, and sustained by community. Our organization sees over
100,000 patients and families each year through a network of
affiliates around the world. We also have a Cancer Support
Helpline where we administer through both of those properties,
over $50 million of evidenced-based care and support each year
free of charge to patients and their families. Importantly, CSC
is also home to the only Research and Training Institute of its
kind whose mission is to collect and analyze information from
patients to elevate the voice of the patient and the caregiver
as it relates to their cancer experience.
I am here today to bring you what I feel is the most
important voice to this conversation and that is the voice of
the patient.
The last 20 years have delivered unprecedented growth in
innovation across all aspects of health care. Never before has
a patient had so many options for diagnosis, treatment, and
follow-up care as they do now. Patients are more educated. They
are more engaged. They are more empowered consumers of health
care than ever before. Yet, despite the emergence of patients
as important players, and even leaders of their care teams,
accessibility to comprehensive information continues to be
elusive.
We will be releasing data next week from our Cancer
Experience Registry where we have learned that 50 percent of
patients engage in shared treatment decision making with their
healthcare professionals. Only about eight percent report
allowing healthcare professionals to make decisions without
their input. Yet, only 25 percent indicate that they feel like
they are prepared to have those treatment decisions.
Importantly, our data reflects a growing concern about
inadequate collection, reporting, and label updating of
endpoints that are meaningful to patients. In our research, 93
percent of respondents considered quality of life as very
important when making treatment decisions. Quality of life
measured higher than length of life, and these are people with
cancer, yet product labels continue to focus very little on
fully measuring comprehensive quality of life metrics. Further,
product labels almost never reflect updates when there are
findings beyond the clinical trial setting including findings
about long-term effects which would be meaningful for patients.
A system that does not proactively collet, publish, and share
data poses a significant risk to patient care.
There are a few issues I would like to raise as current
limitations and we do support the work that the FDA does and we
do support the work of the clinical trial systems and we do
support accurate, meaningful, nonpromotional communication.
Preapproval information, as you know, is when clinical data
is available on a product prior to the product having an FDA
label. According to PhRMA, it takes an average of 10 to 15
years for a drug to make it to market. And during that time,
much is learned about the way in which the drug works in the
body, how the body works with the drug, what is the accurate
dose, what is the toxic dose, and what are the side effects
associated with that drug. Yet, this treasure trove of
information remains out of reach from individuals other than
the sponsor or potential trial investigators.
Number two, limiting communication of information to only
that which is reflected in the label poses a significant
challenge to patients. CSC appreciates the work of the FDA and
sponsors of phase IV studies, in particular, but recognizes
that these studies do not capture comprehensive data for the
use of the product as was mentioned in the real world. Also, it
is a rare occurrence for the label to be updated in a manner
that would allow for proactive communications of findings
outside of the controlled clinical trial setting. And as we
know, once trials go into broader, less controlled situations,
they perform differently in those patients.
Number three, data accumulated through Investigator
Initiated Trials on diseases that would never reach the
investment potential for registration in a label is extremely
important to clinical care. This information may never be
communicated to clinicians and will almost certainly not be
made available to patients who may benefit from the findings
and this is particularly important in patients with rare
disease.
Number four, information learned outside of the clinical
trial setting and not captured in the label can also have a
true impact on the patient experience. And as I submitted in my
written testimony, this could be things like burning at the
injection site, a reduction in fatigue by understanding how to
better supplement the treatment. That information is not in the
label and cannot be shared in a proactive way.
Number five, there are several elements in general clinical
practice that are continuing to contribute to the limitation
that patients have to access comprehensive medical information
through their healthcare team. And in particular, as there is
an active evolution of the care delivery systems from volume to
value, it has brought with it efficiency and cost containment
strategies that focus on limiting treatment decisions. And I am
talking about hospital-based formularies and clinical pathways
that are currently being used in physician practices.
Number six, there is an inconsistent practice and
reinforcement of publishing clinical trial data results in
scientific journals and other databases. This information has
to be published and as mentioned in my written comments, the
ratio of trials that have been opened, closed, and published,
the compliance rate with that abysmal and there must not only
be requirements, but also enforcement of the requirements to
ensure that all results of trials be posted whether those
results are positive or negative.
Finally, industry interpretation of the current regulations
is applied inconsistently across companies. This impacts the
way in which industry communicates with all stakeholders and
most certainly the way in which industry communicates with
patients and families forcing them only through the direct-to-
consumer marketing channel.
So in conclusion, while the comments that I have made have
simply scratched the surface on what is a much broader and
deeper issue, it is my hope that I have highlighted in your
mind the perspective of patients who are living with chronic
and life-threatening illness across the United States.
And to summarize in specific areas where we would like to
continue to work with the committee and the FDA, patients and
healthcare providers must have access to medical research
findings in a comprehensive and real-time manner. Product
labels should be updated in a timely manner and include data
from endpoints that matter most to patients and/or there must
be another mechanism by which to capture and proactively
communicate findings that are clinically meaningful and
relevant. Scientifically sound communications about safe and
effective uses of a product are essential and should be made
available to all stakeholders. Clinical trial results, positive
and negative, should be published by the trial sponsor in a
period of time that is reasonable to allow full and meaningful
data review while ensuring timely access to information. Data,
positive and negative, collected outside of the clinical trial
process, inclusive of real-world evidence that is collected and
analyzed with appropriate scientific rigor should be published
and made available to stakeholders. And finally, proactive
medical communication should be tailored to meet the needs and
literacy levels of specific stakeholders and should not, for
any stakeholder, be limited only to the product label which may
not yet exist or be outdated.
Thank you for allowing us to be here.
[The prepared statement of Ms. House follows:]
[GRAPHICS NOT AVAILABLE IN TIFF FORMAT]
Mr. Burgess. Thank you. Thank you for your testimony.
Ms. Khachatourian, you are recognized for 5 minutes,
please.
STATEMENT OF KATHERINE WOLF KHACHATOURIAN
Ms. Khachatourian. Thank you to Chairman Burgess, Ranking
Member Green, and members of the Subcommittee on Health for
providing me the opportunity to speak before you today.
I am Katherine Khachatourian, a pharmacist working in
Medicare health insurance and a member of the AMCP Professional
Practice Committee.
Imagine a world where you are required by Federal and State
laws to determine a budget and coverage criteria for all drugs
8 to 12 months in advance of the coverage year using limited
available information while knowing there is information that
could help you make more accurate and informed decisions. You
just don't have the key to unlock the consistent release of
that information. This is the world we live in as payers and
population health decision makers.
The limitations on information we are able to obtain
results in a hindrance to patience access to novel and emerging
therapies, limits our ability to accurately forecast, plan, and
budget for anticipated expenditures, and it precludes our
ability to contract on value rather than volume. This is the
reason I am here before you today, to demonstrate the need for
a legislative framework in support of House Bill 2026 which
will provide the key to unlock additional information needed
for us to make informed benefit decisions for better patient
access to treatment. These concepts have been discussed in
depth with a diverse group of stakeholders including payers,
manufacturers, clinicians, and patient advocacy groups who
provide consensus recommendations for how, who, and what
information should be exchanged prior to FDA approval. This
information should be limited to a narrow audience inclusive of
payers and population health decision makers. This scope does
not include manufacturer communications with patients or
prescribers prior to FDA approval.
Let me share a few personal examples where lack of
information has decreased patients' timely access to treatment.
In December of 2013 and October of 2014, the FDA-approved
breakthrough treatments for the treatment of hepatitis C. These
drugs had novel mechanisms of action which changed the
landscape for patients with this diagnosis. Note, these
approval dates were several months after we had already--one of
the payers had already analyzed costs and planned benefit. Had
we been able to discuss in advance of the approval of these
treatments, we would have had a better understanding of the
landscape, timing of approval of multiple products, the
relevant patients for each treatment, and any clinical
information that would help us to make better decisions and
ultimately been able to treat more patients in a more effective
manner without the subsequent criteria revisions that proceeded
after the approval of these products.
More importantly, the lack of needed information can impede
patient access as seen in the new treatments for Duchenne's
Muscular Dystrophy. In this instance, the level of evidence
required to deem products safe and effective met the
requirements for FDA approval. However, due to the inability of
payers and manufacturers to openly discuss the level of
evidence required for coverage, payers are not covering these
therapies at this time. This is why the bi-directional
information exchange is important to understand the level of
evidence available and necessary for coverage. This example has
left patients in a situation where they cannot access therapy.
Had payers been able to convey the level of evidence required
for coverage, could we have avoided this situation? Perhaps.
Another patient access issue was one I experienced in the
past year for a request for oncology. On September 21, 2016, we
received a coverage request for a treatment of a patient
diagnosed with inoperable lip cancer that had recently spread
to their tongue. The FDA granted accelerated approval to expand
the indications of an existing chemotherapy treatment on August
5, 2016 to include head and neck cancer. However, when we
received the request for coverage, the labeled indications and
data supporting the expanded indication were not publicly
available. In this situation, had I had the ability to discuss
the data in advance with the manufacturer, I could have been
better prepared to discuss the requested treatment with the
provider, rather than scrambling through clinicaltrials.gov and
requesting a copy of the clinical trial from the manufacturer
while the insured patient awaited my coverage decision.
Because we can only estimate when therapies will be
approved, if we receive a coverage request shortly after FDA
approval, the landscape still remains one of chaos and special
requests to manufacturers until the data is published,
compendia and guidelines are updated, and coverage criteria
reflect these new and novel treatments.
I have demonstrated in the previous examples each of these
breakthrough therapies represent innovations and the potential
to change a patient's life, if they are able to gain access to
treatment. The barrier to access to novel therapies is a
population health decision maker's ability to have sufficient
data and sophisticated discussions with those most informed
about the utility of the products in a timely enough fashion to
budget, plan and forecast it for the therapies coming to
market.
In conclusion, this is an issue of great importance for
patient access to emerging therapies where a diverse group of
stakeholders have come together to develop consensus
recommendations. This includes a very narrow audience and scope
of exchange between manufacturers and payers only. We need your
legislative support to better care for our patients. Thank you.
[The prepared statement of Ms. Khachatourian follows:]
[GRAPHICS NOT AVAILABLE IN TIFF FORMAT]
Mr. Burgess. I thank you for your testimony. I want to
thank all of our witnesses. It has certainly been compelling
testimony this morning. People will note that I allowed the
clock to run over because you had important information to
provide us. I guess we will underscore that I will not be so
generous with Members, so try to confine your time to the 5
minutes allotted to these products that have not been evaluated
by the FDA. This product is not intended to diagnose or prevent
any condition, just to get through the appropriate label
disclaimer.
Let me begin the questioning and I will recognize myself
for 5 minutes. And Ms. House and Ms. Khachatourian, thank you
so much for your testimony.
Ms. House, while you were talking and I actually wrote down
a note to myself about when you mentioned about clinical trials
and I was going to ask you about the utility of getting the
information off of clinicaltrials.gov and then Ms.
Khachatourian actually referenced that as well. So this is a
real-world phenomenon where payer decisions are unable to be
made, but the data is sort of accumulating on the data side of
the docket, but it is not coming up to the payer's side. So it
sounds like both of you have dealt with that.
And Ms. Khachatourian, I thank you for bringing up the
issue with the new hepatitis C drugs, because we were sitting
on these panels in 2012 and 2013. And I would suggest it is not
just an issue of commercial payers. Our State Medicaid
directors, our State prison directors, our Federal prison
directors were going to have to deal with this information in
very short order and they did not have it available to them.
And I would be happy to listen to what both of you have to
say, particularly on the clinicaltrials.gov. Are we doing a
good enough job getting that information out there in a usable
way so that you can actually begin the process of what are we
going to have to do as far as on the payer's side?
Ms. House, we will start with you, and then I would like to
hear Ms. Khachatourian's thoughts on that.
Ms. House. Thank you, but I didn't share my comments that I
have in my written testimony. I included two studies that were
done on the clinicaltrials.gov database where there was a
random sampling of 600 trials originally. And 50 percent of
those trials did not have a corresponding article. The second
study was even more alarming in that there was a look at 13,327
trials and 1-year post-data closure, only 13 percent of those
has posted clinical trials information. And even when they gave
a bit of a grace period and extended that for another couple of
years, only 38 percent had clinical trials posted there. So not
only is the system extremely difficult to sort of use and find
and especially as we are moving into the age of personalized
medicine to get to trials that are relevant for me, the data
results aren't there.
And I will give you an example that happened to me just
last week, is that a patient of ours reached out and he has a
certain type of lung cancer, ALK positive lung, in which there
are a number of solutions and options available for him. His
physician wanted to put him on a phase 2 trial with a new
product and he said, ``What do you think about this?'' And so I
went online to try to find information because I was trying to
decide why would they put him on a phase 2 trial instead of the
phase 3 trial, and I am an educated consumer and I have worked
in clinical trials for a long time. After about an hour and a
half, I could find two sources online to your point. One of
them was with a reputable medical society and the other was an
opinion piece on the way in which this product worked.
They are in a phase 3 setting already, so there is a lot of
evidence on this particular drug and not available to even
educated consumers.
Mr. Burgess. OK. Ms. Khachatourian.
Ms. Khachatourian. Thank you, Mr. Burgess. I actually
pulled some dates more relevant to some recently approved
therapies. In the hepatitis space, the products Zepatier and
Epclusa were approved January 28, 2016, and June 28, 2016, per
the FDA website. However, results on clinicaltrials.gov were
not published until September 27, 2016 and April 26, 2017
respectively. So just to give perspective regarding when data
is available and results are published, those are key dates
that I was able to glean. I have some oncology examples as
well, but I think that proves the point regarding the delay in
access to information that is necessary for coverage decisions.
Mr. Burgess. Dr. Van Hare, you referenced the rich data
sets that would be available by a drug or device manufacturer,
but that data is sort of locked away from the clinician. I
guess you have to go the bar to have those discussions? You
can't have those discussions in the hearing room or the
continuing education room? You have to go offsite?
Dr. Van Hare. On the stairwell.
Mr. Burgess. On the stairwell, OK. Very well. And you see
what we are talking about today as a way of unlocking those
data sets being available to the clinicians?
Dr. Van Hare. I think so. I think it is really pretty
simple for allowing off-label use. A physician who prescribes
something off-label is responsible for ensuring that they have
evaluated the most appropriate clinical data before they make a
decision about prescribing something off-label and some of that
data is actually held by the manufacturers.
They are allowed, as I understand it, to provide it to us
privately and in response to an unsolicited request, but you
know, there is 300 of me in the country, the pediatric
cardiologists who do what I do in the country. Every single one
of us has to independently call up the drug company to get the
information. It is not particularly efficient.
Mr. Burgess. No. I think my time is expired. I want to be
respectful of everyone's time.
Mr. Green, you are recognized for 5 minutes for questions,
please.
Mr. Green. Thank you, Mr. Chairman. Long ago, Congress
recognized the importance of requiring manufacturers to provide
evidence demonstrating the safety and efficiency of the
product. In marketing under current law, drug and medical
device manufacturers can disseminate certain medical and
scientific information about unapproved uses of approved or
cleared products to health care professionals and other
entities.
Recent court cases cited as a source of uncertainty around
the types of communication about these unapproved uses are
permissible.
Ms. Charo, in your written testimony, you said if the First
Amendment means that the off-label promotion must be permitted,
then the promotion of entirely untested, unproved drugs should
also garner the same protection. Is that true?
Ms. Charo. I fear that the logic would be the same in both
cases. Now it is true that for things that have been approved
at least once, one does have some, at least, early information
that the drug is not highly toxic because that is what we are
going to get from the early Phase 1 or 2 trials. But the
reality is over time, both the drugs that have never been
approved before or the off-label indications for things that
have been approved turn out to fail which means that one begins
with a presumption that any unapproved use or any unapproved
drug is probably not safe or not effective until it is proven
to be so.
Mr. Green. Well, this is an issue that this subcommittee
and our committee has wrestled with for a number of years. Can
you help us understand what restrictions the Constitution does
and does not allow? Does the First Amendment prohibit the FDA
from restricting promotion of unapproved uses?
Ms. Charo. No, there are a number of Federal cases that
have upheld the FDA's authority to do just that. There is
constitutional protection for commercial speech and there are
standards for that protection and in the area of commercial
speech it is a fair amount of protection although not the same
degree of protection as you would get for political speech or
other forms of speech. And those restrictions on commercial
speech are permitted when there is a substantial public
interest in doing so. In this case, by restricting off-label
promotion, one is able to create both a stick and a carrot that
drives the pharmaceutical industry toward the research needed
to actually figure out which things are safe and which things
are effective. If one is able to simply promote without
restriction and gets no market advantage by going in and
investing in the research, one loses that system entirely and
we really do risk having an absence of information for people
like Dr. Van Hare to solicit or to develop on his own, let
alone to share with his colleagues.
Mr. Green. Ms. House, I note in your focus on your
testimony the fact that so much clinical trial data is
unpublished. One thing that concerns me is the bias in what is
published. Multiple studies have shown that positive trial
results are more likely to be published than negative results.
And in particular, industry sponsorship has been demonstrated
to be a factor contributing to the biased publication. Industry
has no incentive to publish or promote negative findings.
My question is if industry is more likely to publish
positive than negative results, do you also worry that positive
results will be promoted more than negative results, even if
there is a particular research being communicated is truthful
and not misleading? Doesn't selected provocation create a
distorted view of the safety and effectiveness of the unproven
use?
Ms. House. I am going to answer this very carefully because
I have not seen the data that you re referencing that would
suggest that there is more positive data than negative data.
What I would say is that our position is is that both positive
and negative data needs to be published in an equal manner and
should be available for communication because we do know that
there are patient harms as well as benefits.
Mr. Green. And I think that is what we want to get to. If I
am a pharmaceutical or if I am advertising anything else, I am
going to talk about how great it is. If we are running for
office, I am not going to talk about our bad side. We are going
to talk about the good side. So we need to have it, but we need
some agency to be able to say this is what you are doing and
the FDA is what we have. That is my frustration, I guess.
Dr. Van Hare, in your testimony you note that Pediatric
Research Equity Act has not been sufficient in producing the
amount of shareable data we might like particularly in the
older drugs and clinical decisions are often made. I think you
raised an important point about the need for the robust data to
allow clinicians to make the best decisions they can. My
concern is there is nothing in this legislation we are talking
about today would actually encourage drug companies to conduct
those clinical trials that could answer important questions for
pediatric populations. And again, our subcommittee for decades
has wrestled around what may be appropriate for an adult is
just not appropriate for children and we need to do a lot more
work on that to make sure that we don't leave out the pediatric
population.
Mr. Chairman, I know I am over time, so I yield back my
time, unless you want to give it Dr. Van Hare?
Mr. Burgess. Dr. Van Hare, did you want to comment?
Dr. Van Hare. I think that legislation has actually helped
children in terms of getting a lot more information about
drugs. And certainly in the pediatric world, originally for
some companies or actually enticed some companies to actually
do some trials. For the most part though companies are not
really interested in the pediatric market. We are very, very
small market and sort of thinking about the carrot and stick
sort of approach, none of the carrots are really going to help
us in pediatrics because it is a fairly small market. So we are
left in a situation where no one is going to do the type of
clinical trial that was actually going to allow labeling for
pediatric application for a lot of the things that we actually
use.
Despite that, we are talking care of our children, and we
need the best available data to make those decisions.
Mr. Burgess. Thank you. The Chair recognizes the gentleman
from Illinois, Mr. Shimkus, 5 minutes for questions, please.
Mr. Shimkus. Well, thank you. I am going to follow up with
Dr. Van Hare first of all saying for my colleagues that the
Washington University School of Medicine is one of the
preeminent institutions in our country. And VJC which they are
affiliated with, that is the go-to for major deals. So welcome.
Dr. Van Hare. Thank you.
Mr. Shimkus. And I know that because--please extend my
hello to Dr. Braverman and Dr. Damiano, who I know personally
from personal medical stuff. I am a Homer for these folks and I
have great confidence in your testimony and your word. But I
would like to follow up on the question in that how often do
you assess the various information to try to treat kids? I mean
so we are talking about FDA approval, but you have given
testimony about outside information to make sure you can best
care for kids. How often do you go and search outside
information to try to bring the best medical care to the kids
in the cardiology aspects?
Dr. Van Hare. It really depends on what the condition is
that we are actually trying to treat. I would say that we do
have the process of developing consensus documents that
actually summarize the medical evidence, the clinical trials
and things like that that actually sort of express and
certainly our society, the Heart Rhythm Society does this all
the time to create these consensus documents to give physicians
guidance. But you know, I guess pediatrics and also really sub-
specialty medicine in general, we take care of a lot of very
unusual types of conditions that don't really fall under the
labels and the recommended uses. And so I guess for those less
common, more unusual types of situations, we are often looking
to our colleagues. We are calling around. We are finding what
has your experience been with this? What has your experience
been with that?
Interestingly, I am a real proponent of the concept of
partnership between industry and physicians. We often work
elbow to elbow when we put pacemakers in and when we do
different kinds of procedures. They have a lot of information
just from their experience and it is an important source for
us.
Mr. Shimkus. Great. Thank you. Let me go to Ms. Klasmeier.
In your testimony you talked about, and I quote, ``strict
scrutiny'' the test. What does that mean, strict scrutiny in a
test in court?
Ms. Klasmeier. As a practical matter, Congressman, it means
the goverment loses. So strict scrutiny is a bit of a legal
fiction that we indulgence. It reflects the notion that when
you examine Government regulation that affects core speech such
as political speech, it is very, very hard for the Government
to sustain its burden of justifying that speech regulatory
provision against First Amendment is solvent. So as a practical
matter, if the court concludes the applicable standard is
strict scrutiny, the Government loses.
Mr. Shimkus. Maybe my colleague, Mr. Griffith, will follow
up on that. He is our legal mind here on the committee and does
a good job.
Let me finish with Dr. Kesselheim. I am somewhat confused
in your testimony because you used numerous times the term
promotion over and over again in your testimony. But on page 2
of the Griffith draft, it explicitly excludes promotional
communications. Am I missing something?
Dr. Kesselheim. Well, no. I mean I think this is part of an
example of how the Griffith draft actually makes something that
is fairly clear a lot less clear because, you know, if the
pharmaceutical company defines something as promotion
determines whether or not they fall into this safe harbor.
Mr. Shimkus. What do you mean by promotion? You used it
numerous times.
Dr. Kesselheim. Sure. When a pharmaceutical company
promotes a drug, it goes out and it tells people about the use
of the----
Mr. Shimkus. For their ability to sell it?
Dr. Kesselheim. Yes. It goes out and it tells physicians
about how to use the product and it sort of promotes the use of
the drug through one of the various advertising----
Mr. Shimkus. I am reclaiming my time. I will let
Congressman Griffith kind of hash this out more, but again, on
page 2, it is pretty clear. It says communication is not
advertising or otherwise promotional in nature. So I just had a
concern with your statements in your opening statement because
you said it over and over again. I think it gives the wrong
indication of what my colleague is trying to do. With that, I
yield back my time.
Mr. Burgess. The Chair thanks the gentleman. The gentleman
yields back. The Chair recognizes the gentlelady from Illinois,
Ms. Schakowsky, 5 minutes for questions, please.
Ms. Schakowsky. Thank you. I think it is really important
that we step back and remember that the FDA approval process
really is the gold standard, the universal gold standard to
determine safety and efficacy. And efforts to undermine that
standard are very worrisome to me and I think that is what
happens in these drafts. I think that Ms. Charo put it best in
her testimony when she stated ``for complex products like
drugs, the marketplace of ideas cannot work properly with
unvetted information from a self-interested source.''
I mean I think that often this committee is inclined to say
whatever PhRMA wants, PhRMA gets. But I want to ask Dr.
Kesselheim, we have heard compelling testimony, I think, about
access for patients to drugs. And so it is very important, I
think, for you to explain what---does access trump safety or
does it have to by having these kind of off-label procedures?
It seems to me that safety ought to come first, but are there
ways to guarantee that safety without the process of approval
by the FDA?
Dr. Kesselheim. Well, I mean so sure and I think that part
of some of the testimony that we heard was a little bit
disingenuous because the access to the products was not defined
necessarily by the communications that occurred. The access in
the case of the hepatitis C drugs, the effectiveness of the
hepatitis C drugs is not a secret. Everybody knew how well they
worked. Access to them was determined by the high cost of the
product, not the evaluation, not whether or not there could
have been communication in the few months before the drug was
approved. So I mean I think the issue is really about getting
high quality evidence or high quality communications out to
help inform the market so that patients can make well-informed
decisions based on the highest quality information that is out
there possible. And the way to do that is to make sure that a
neutral, third party body of experts like the FDA is able to
vet the information. And I think what we should be doing is
talking about how to make sure that more information is
published, more trials are published, more trials are
available, open access, and that the FDA has more power and
more authority to review information so that they can make
those kinds of determinations so patients can benefit.
Ms. Schakowsky. Is there a way for the FDA to move more
quickly? We heard about 9, 10 years, or whatever?
Dr. Kesselheim. I think if the FDA had more resources, it
would be able to move more quickly. There are plenty of
examples where the FDA has gone out and has been concerned
about new safety issues that emerge, about off-label uses and
ultimately goes through the process of revising the label to
try to integrate those kinds of changes. If the FDA had more
resources added and more people doing that kind of post-market
surveillance, label updating kind of work, then I think we
would get that information out to patients and vetted
information out to patients more efficiently and more quickly.
Ms. Schakowsky. Ms. Charo, one of the most compelling
things I heard from you saying that, in fact, when you look at
these drugs, the majority of them, in fact, would probably not
meet the test. Am I hearing you right?
Ms. Charo. You are hearing me correctly, and I believe, in
fact, it was Ranking Member Green who referenced some of those
studies in his opening comments.
You know, scientific research is often somewhat equivocal
for a very long time. I think what we are discussing here is
really what to do in that interim period where the evidence is
shifting around. Do we presume everything is going to work and
therefore everything people want to say is likely to be true
and should be allowed or are we going to presume that it
probably isn't going to work out and we should restrain the
speech until we have actually proved it will.
From my perspective, given that the risk of incorrect
information is that people will actually be harmed, or they
won't go for the effective treatment, they will go for the
ineffective one, we need to err on the side of caution here and
protect the larger population.
That said, there are certainly going to be some occasions
in which it turns out that something does work and it would
have been wonderful if we could have seen it earlier and talked
about it earlier, but those incidents will be fewer than those
in which it would be damaging.
Ms. Schakowsky. In the last 30 seconds, Dr. Kesselheim,
what does history tell us about off-label promotion? Are there
some things we should be recognizing here?
Dr. Kesselheim. Sure, I mean over and over and over again
throughout history and you don't even have to go back to the
thalidomides 50 years ago, more recent history tells us that
off-label promotion drives physician practices in ways that
favor the drug being promoted, not in ways that favor the
overall state of the evidence and the overall state of
practice. I think that we need to be very wary about efforts to
try to expand that promotion when it covers nonevidenced
based--potentially nonevidenced based communications.
Ms. Schakowsky. I think we need to, when it comes to
patient access, discuss more about the cost. Thank you.
Mr. Burgess. The gentlelady yields back. The Chair thanks
the gentlelady. The Chair recognizes the gentleman from New
Jersey, Mr. Lance, 5 minutes for questions, please.
Mr. Lance. Thank you, Mr. Chairman. Let me state that I
don't believe any of the testimony has been disingenuous, in my
judgment. This is a very difficult issue, and we are trying to
balance the equities on this committee, and I am pleased that
every member of the panel is here, and I do not question the
integrity of any member of the panel.
Counselor Klasmeier, do you believe that the standard will
be strict scrutiny, or will it be rational basis, or will it be
some intermediate standard, based upon your professional
judgment as a distinguished member of the bar?
Ms. Klasmeier. Congressman, my judgment is that the
standard will be some variation of intermediate scrutiny.
Mr. Lance. Intermediate scrutiny, yes.
Ms. Klasmeier. And it will be most likely the Central
Hudson standard with a garnish of heightened scrutiny as a
result of the Supreme Court's decision in Sorrell in 2011.
Mr. Lance. Yes, that is my judgment as well, and I think
that there is a history of decisions in this regard that would
indicate that that is probably where we would be eventually as
a matter of legal analysis. Thank you.
Dr. Van Hare, we have all heard that some off-label uses
are well established in clinical practice, and supported by
high-quality evidence, and are the standard of cure for many
conditions. From your perspective, based upon your
distinguished history, how does the pieces of legislation
before this committee stand to improve care for patients?
Dr. Van Hare. Well, to the extent that the legislation
proposed by Congressman Griffith allows or improves the
efficiency of sharing data that the device companies and
pharmaceutical companies actually have, for physicians who are
prescribing off-label, I think it will actually help.
Mr. Lance. Thank you, and other members of the panel are
certainly welcome to comment.
Ds. Khachatourian, what are the odds that if we pass
legislation we are considering today, sophisticated population
health decision makers like payers, provider sponsored health
plans, pharmacy-benefit managers, and other organizations would
be misled by unscrupulous drug and device manufacturers who
make unfounded claims about their products?
Ms. Khachatourian. So first let me acknowledge my testimony
by no means disingenuous.
Mr. Lance. I am sure and that is why I raised it. And if I
might interrupt you, I try to lead by example in the Congress,
both on the floor and in committee, and I enjoy the testimony
of every witness who comes before us. Those who know me know
that disingenuous is not a word that I find attractive in
vocabulary here on Capitol Hill. Yes, please continue.
Ms. Khachatourian. Thank you. So population health decision
makers and clinicians that we are discussing here are well
trained to look at things with scrutiny and to determine what
level of evidence is acceptable. And during the multi-
stakeholder discussions that we have had, we did address the
need to determine a level of evidence and to have an agreement
on what is acceptable and nonmisleading. And as evidence
continues to evolve and as new therapies continue to emerge,
that is the goal, is to develop strict criteria that will be
used to apply to any level of evidence in order to ensure that
it is high level and with the patient's best interest in mind.
Mr. Lance. Certainly, and that is what we are attempting to
get to a place where we can make sure that always there is the
greatest standard of care. It is the jurisdiction of the
subcommittee and ultimately of the full committee to promote
the better health of the American Nation, and we recognize this
is a difficult issue and I certainly commend my colleagues,
including the gentleman to my immediate right, the
distinguished Member from Virginia, as we undertake an analysis
of how best to protect the American people recognizing that
that is the goal of this subcommittee in a bipartisan nature. I
yield back 22 seconds, Mr. Chairman.
Mr. Burgess. The Chair thanks the gentleman. The Chair
recognizes the gentlelady from California, 5 minutes for
questions, please, Ms. Matsui.
Ms. Matsui. Thank you very much, Mr. Chairman. This
committee recognizes the important role that FDA plays to
ensure public health and safety as evidenced by the bipartisan
User Fee Reauthorization that we intend to pass out of the
House this afternoon.
Now we can't tolerate efforts to jeopardize that role as
patients across America who take drugs to treat or cure
conditions rely upon the FDA to monitor the safety of these
drugs and devices.
I am really glad that we are holding this hearing today to
examine issues that arise around information sharing,
particularly for those so-called off-label use and what could
be done to alleviate those issues without detracting from FDA's
ability to regulate safety.
I am particularly interested in the situation that many
rare disease and cancer patients find themselves in. As many as
one in five prescriptions are written for drugs off-label,
meaning that they are prescribed for a condition or population
that has not been FDA-approved as safe and effective.
Oftentimes, off-label drugs are the only treatment available
and even the standard of care for rare disease patients with
limited options.
Ms. House, thank you very much for your advocacy on behalf
of cancer patients. Can you please discuss prevalence of off-
label use in cancer patients?
Ms. House. So there was a physician posted by the Friends
of Cancer Research just yesterday that indicated that the use
in cancer off-label was close to 80 percent. And part of--one
of the problems that I just wanted to raise is I was looking at
some other discussion is I am going to give you an example. It
is an older example, but it really talks about how the current
labels are out of date. There was a time around 2000 where this
is the time prior to personalized medicine, so it was still in
the era of poisons for cancer, that there was a combination
being used off-label as standard of care for the treatment of
lung cancer. That particular combination failed at that time 13
Phase 3 trials which is the gold standard for the evaluation
for the FDA, yet it continued to be used standard of care for
many, many, many years beyond that.
This morning, I went on the FDA website and pulled up the
label for the lead drug in that and today in 2017, still has
not been updated to reflect the use of that combination which
is a problem.
Ms. Matsui. It is a problem, right. Now, you know when a
family gets a cancer diagnosis, I think the world stops. And
you are sort of grasping at what can we do? And I think we all
go to the internet. That is where we go right now.
What types of information is generally available to
patients and their providers when a drug is used off-label and
even when you are an educated consumer, you really kind of hit
a brick wall. What kinds of solutions might you recommend to
address these challenges?
Ms. House. I think creating solutions that again are
tailored to the stakeholder, to their literacy level, to their
educational level. There is really no reason why we can't
create forums that would be peer reviewed, scientifically sound
analysis, and presentation of clinical data. What it does
prevent then is people going to the internet and getting into a
chat room that may be facilitated out of another country or by
somebody who has absolutely no medical background. And we see
that happening all the time. And furthermore, if a patient
calls a pharmaceutical company and says I am a patient, can you
give me information about XYZ, the response will almost
uniformly be, I cannot answer your question. You will have to
go speak with your doctor.
Ms. Matsui. Thank you very much. Ms. Charo, I know you have
concerns about the legislation that we are discussing today.
Are there ways that we can refine the legislation to reach our
shared goal of promoting public safety by increasing patient
access to safe and effective drugs? I think there is
information out there and you know, we are in a time now where
there is much more research and innovation and I would hate to
just have a hard and fast rule regarding this.
Ms. Charo. Thank you. I completely agree with you that
there are other avenues that need to be explored. For one
thing, it may make sense to try to distinguish those areas
where off-label use really is a necessary and important part of
medical care as we just heard in the area of cancer, and some
other areas there it really is not as prevalent and is not as
needed. And I would suggest that pediatrics may be another good
example.
And the Congress has made great strides in trying to create
new systems for both incentives and even possibly rewards for
continuing the necessary research to find what really is safe
and effective, for example, in the pediatric population.
Working on making sure that there is a proper incentive and
reward to fill in the gaps in those areas would be a good step
forward and might accomplish many of these goals without some
of the risks that are intended upon some of the ambiguities and
what constitutes promotional marketing or what constitutes
accurate information.
Ms. Matsui. Thank you. I have run out of time. I yield
back.
Mr. Burgess. The Chair thanks the gentlelady. The
gentlelady yields back. The Chair recognizes the gentleman from
Indiana, Dr. Bucshon, for 5 minutes for questions, please.
Mr. Bucshon. Thank you, Mr. Chairman. I was a practicing
cardiothoracic surgeon prior to coming to Congress and I just
have a comment, not a question, but the medical community is
relatively small and I think Dr. Van Hare said there is 300
pediatric cardiologists. There is about 4,500 to 5,000 cardiac
surgeons. Information travels quickly. Physicians are always
looking for better ways or effective ways to treat their
patients whether it is on label or off-label and information
passes quickly.
Frustration with labeling can be really high amongst
different physician communities because of the delay in
updating what may or may not be FDA-approved. Patients are
desperate and are getting information potentially from
incorrect sources including the internet as has been pointed
out and so I would suggest that we definitely need reform so
that patients have the opportunity to get more accurate
information.
With that, I am going to yield the remainder of my time to
Mr. Griffith.
Mr. Griffith. Thank you very much. I appreciate it greatly.
Let me first say that I appreciate everybody being here today
and appreciate all of your testimony. I am open to continue to
work on the language to make sure that we get it right. So that
is something that I would invite you all, if you have issues
with the language that we currently have, please get those
suggestions to us because we want to try to do this in the best
way that we can. We do believe that we need to do something on
a legislative side.
Also, Mr. Chairman, I have some letters in support of the
bill and a draft language, and, if I could have unanimous
consent to enter those into the record, I would appreciate it.
Mr. Burgess. If the gentleman will share those with us, I
will seek unanimous consent in a moment.
Mr. Griffith. I also want to make sure that we are all
working on the language that we currently have. And so what the
bill says is, when we are talking about communication, if you
look on page 2 it says, ``(a) the communication is not
advertising or otherwise promotional in nature; (b) the
communication is supported by competent and reliable scientific
evidence.'' And then (c) and this was to address some of the
concerns that have been raised here today, we put this language
in: ``The communication clearly discloses appropriate
contextual information about the data presented including
information about limitations.'' And I probably should put
numbers in front of these. ``(1) Limitations of the data; (2)
the scientific and analytical methodologies used; and (3)''--
and I think very importantly--``any contradictory data or
information known to the manufacturer or sponsor.''
We are never going to solve all of the problems if somebody
is not doing what they are supposed to do, but our intent is to
try to make sure that both sides are presented. I think
somebody mentioned that earlier in their testimony, that both
sides are presented and that the negative evidence is out there
as well.
And then we talk about situations related to the rare
diseases. Cancer has been mentioned today and the children
because one of the problems you have in those situations and
Dr. Van Hare, you touched on this is that there may not be a
sufficient number of patients to actually warrant doing a
clinical study. Nothing compared to what you deal with your
families Dr. Van Hare, but my son who is now 11 had \2/3\ of
his body covered with eczema when he was about 3 months old. I
kept telling my wife because of the history in the family we
have allergy problems, honey. We got him to an allergist.
Between the cream that worked for me that my pharmacist knew,
between the steroid creams, between the antihistamines that
they gave him, we were able to control that situation. We still
have issues there. But for a child under the age of 2, there
were no--some of that might have been on-label, but most of
that treatment was off-label, so I appreciated Ms. Charo saying
that we ought to take a look at that, because I think those are
the two hot-button areas. But that doesn't mean we should
exclude others.
I was very curious, too, about this whole agent concept
that is going on where you can't go and tell the 300 other
doctors, Dr. Van Hare. Could you speak on that briefly, and I
have only got a minute left of this time period.
Dr. Van Hare. Yes. It has to do with how CME or Continuing
Medical Education is defined. CME is actually a safe harbor. If
I am speaking at a conference that is sponsored by an
accredited CME provider, like the Heart Rhythm Society or the
American College of Cardiology or some other group, I can say
whatever I want and I can talk about off-label indications as
much as I want. If I am actually speaking at a conference that
is actually sponsored by the pharmaceutical company or the
manufacturer, then I basically am an agent, or considered an
agent.
Mr. Griffith. So if on the podium somebody asks you about a
catheter to be used in a child that might be off-label, you
could then be deemed and the company could be deemed that you
are their agent and then be in trouble under the current rules
of the FDA. Is that correct?
Dr. Van Hare. That is my understanding.
Mr. Griffith. That is my understanding also. All right, Ms.
Klasmeier, my friend and colleague from New Jersey, Mr. Lance,
did a great job of going through the intellectual. Let us
translate that into human regular English. That means that if
you bring that example to the courts, FDA is most likely going
to lose, wouldn't you agree?
Ms. Klasmeier. I would agree and I would go one further.
FDA did lose that case. That was the Washington Legal
Foundation decision in 1998 and the upshot of that is that the
court found it unconstitutional for the Government to purport
to restrict the identity of the speakers that could participate
in those kinds of continuing education events that Dr. Van Hare
described.
Mr. Griffith. Thank you very much. I yield back to my
colleague. Thank you.
Mr. Bucshon. I yield back.
Mr. Burgess. The gentleman had a unanimous consent request
and I sought counsel from the other side of the dais, so
without objection, so ordered if that unanimous consent request
still stands.
[The information appears at the conclusion of the hearing.]
Mr. Griffith. It does, and I apologize. I just saw my time
taken away.
Mr. Burgess. Very well. The Chair recognizes the gentlelady
from Florida, Ms. Castor, for 5 minutes for questions.
Ms. Castor. Well, thank you very much, Mr. Chairman, for
calling this hearing. I think allowing drug companies and
manufacturers to market their drugs and devices for unapproved
uses would be very dangerous for American families, American
consumers. It would reduce the incentive for them to go through
FDA's approval process and reduce the incentive to go through
clinical trials that really just test whether or not a product
is safe and it is effective. FDA's approval process right now
is the gold standard for safety and efficacy.
The FDA Commissioner, Dr. Gottlieb, has said the most
important incentive to developing useful information remains
the ability for companies to market drugs based on what can be
proven scientifically. Now this is not a hard and fast rule
because I have learned today and reviewing your testimony,
there are safe harbors, but nevertheless, Professor Charo, some
contend that we must revisit this regulation of off-label
promotion because the trend in the courts is that restrictions
on off-label promotion run afoul of the First Amendment. I
think this is a stretch. Does the First Amendment limit FDA's
responsibility for scientific review? Does it limit FDA from
restricting promotion of unapproved uses? If not, what avenues
do medical product manufacturers have to communicate about such
uses?
Ms. Charo. Well, we have seen some cases that have touched
on these things from the fringes, but you don't actually get
cases that touch on it directly. For example, in one case that
is frequently cited for the suggestion that the Constitution
prevents the FDA from restricting truthful speech, at issue at
the time was not truthful speech, but simply off-label speech
and the FDA premised its entire case on the fact that the
speaker had been discussing an off-label use and never really
talked to the issue about whether or not the speaker's comments
had been true.
The problem here has simply been that it is really and I
hope that Mr. Griffith's staff is still around for this, the
problem is that no company is going to have all the information
about all the studies that are being done at that time
including those that have negative results because of various
rules about confidentiality of information. The FDA may be in
possession of all the information, but not necessarily every
company. So even with the best of intentions to be conveying
what they believe to be truthful and contextualized
information, there is the risk that that actually is missing
large areas of data that would suggest that the studies they
are discussing are not, in fact, going to be indicative of a
truly safe and effective drug at the end of the day. This is
why there really is a substantial public interest which is one
of the key elements in the restriction of speech to the current
system.
And the alternatives that have been presented,
unfortunately, I believe offer risks to public health that
dwarf their benefits which is why the second rung, the second
prong of these tests which have to do with whether or not the
Government can find an alternative way of achieving its goals I
think show that really the current system is probably the best
way, tweaking, yes, but the removal of many of these
restrictions, I don't believe is necessary in order to meet the
Constitution test.
Ms. Castor. And there seems to be debate on whetherthe
Griffith proposal would restrict scientific exchange under the
safe harbor. What is your view of this and the Griffith
discussion draft?
Ms. Charo. You know, I think that the text does attempt
does attempt to isolate what is nonpromotional and protect that
while continuing the prohibit promotional language. I think
that the difficulty here is that the very notion of what is
promotional is actually somewhat ambiguous. We now know, for
example, that it is possible to tweak how various results come
up on the internet, whether or not it is the first, second, or
third thing you see on the page. If there is a tweaking
algorithm, does that constitute promotional if all it does is
raise your particular data to the front of the page? These are
the kinds of subtle questions that can both make the language
ambiguous despite our efforts and also from my perspective,
suggest that it is better to have the flexible tools of
guidances that can be negotiated over time with the constantly
changing nature of communication rather than the somewhat more
rigid tools of regulation and legislation, let alone having
courts do it 17 years after the fact and leave everybody
uncertain for that long period in between.
Ms. Castor. Dr. Kesselheim, do you have a comment on this
topic as well?
Dr. Kesselheim. I mean I also agree that the way that this
discussion draft is written provides substantial leeway for
companies to interpret these various provisions in ways that
are favorable to their particular advertising strategy.
Ms. Castor. And at the cost to public safety.
Dr. Kesselheim. And at the cost to public safety.
Ms. Castor. Thank you. I yield back. I am out of time.
Mr. Burgess. The gentleman yields back. The Chair thanks
the gentlelady. The Chair recognizes the gentleman from
Georgia, Mr. Carter, 5 minutes for questions, please.
Mr. Carter. Thank you, Mr. Chairman. And thank all of you
for being here. Certainly, an important subject.
Dr. Khachatourian, you are a pharmacist, as am I. And I can
tell you that after 30 years of practicing pharmacy, certainly
side effects are--we call them side effects. And you know, it
has always been interesting to me why we call them side effects
because essentially they are effects of the drug, but they are
not what we want it to do, so we kind of label them as side
effects.
I noticed in your statement, in your testimony, in your
written testimony that you feel like the Pharmaceutical
Information Exchange would be helpful and useful and there is
some debate on whether it should be evidenced based or whether
it should be information based. And I noticed that you said
that it should be based on information only, well, not only,
but basically. Can you kind of elaborate on that?
Ms. Khachatourian. Absolutely, thank you. So when we think
about evidence, there are established criteria for evidence as
far as what constitutes a clinical trial and the acceptable
level of evidence for FDA approval. When I talk about
information, information may include financial models, may
include other information that does not quite meet the level of
evidence that one might traditionally think. So when we talk
about information, if I am able to discuss with my clinical
colleagues at a manufacturer what models might be available,
what sub-populations were studied and what level of information
might be available that can help me to make more effective
decisions, that is what I mean by information.
And again, I will reference the multi-stakeholder forum
where we discuss developing criteria that will set the
foundation for what that information might entail and what
level of quality of information could be deemed acceptable.
Mr. Carter. You also mentioned in your testimony that a
very proactive pharmaceutical information exchange would lead
cost savings. It could lead to cost savings for patients. So in
that respect, how can we assure that the cost savings are going
to be passed on to the patients if we don't have transparency
within the prescription benefit managers and the other middle
men that are included so often in these scenarios?
Ms. Khachatourian. Sure. While cost is an aspect of
evolving and emerging therapies and treatments that are coming,
cost is an aspect that needs to be discussed. However, with the
exchange of information it makes us more effective in the use
of the funds that we have available to make benefit decisions.
So when we are structuring a benefit based on value, that is
what value will be conveyed to both us as the payer as well as
the patient. So ultimately from a cost discussion, that is, in
turn, outside of the transparency which is a little bit of a
different discussion.
Mr. Carter. I am not sure I understand how it can be a
little bit of a different discussion. Because I believe truly
that it can have cost savings to the patient if we have
transparency within the system and I don't see how it can be if
we don't have transparency.
Ms. Khachatourian. So I absolutely acknowledge transparency
is an important factor. However, the information exchange
between a payer, as well as the manufacturer, will help us to
make better decisions and with a limited pool of money that we
are able to allocate to benefit design. We try to make the most
cost-effective decisions on behalf of those patients that we
serve, so in turn, the cost savings are passed to the patient
as the ultimate user of our benefit design.
Mr. Carter. OK. I will move on. Dr. Van Hare--and thank you
very much for being here, Dr. Khachatourian.
Dr. Van Hare, I have seen in my practice over the years,
particularly with prescription drugs, a lot of off-label uses,
if you will, in pediatric patients. And I just want to get your
feeling on the value of that? Because I have seen it first hand
that it has been very valuable.
Dr. Van Hare. Yes, well, so I would say it is essential, in
fact, for most of what we do, particularly in the pediatric
cardiology area. But I mean I do think we have reservations
about it. When people make decisions based on information they
get from like one other colleague who used it once on some
patient, that is very, very sort of limited. But I would say
that certainly we have to do it. We have no choice but to do
off-label prescribing in a lot of situations. And we would
prefer to have the best possible information.
We also use what is known about the use of these
medications in other age groups, particularly adults, or other
particular conditions and basically extend to these particular
populations. That may or may not be valid as some other members
of the panel here have talked about. But absent better data, it
is all we actually have.
Mr. Carter. Great. Thank you all very much for your
participation here today. A very important subject I can tell
you. Many years of practice in pharmacy, we have used many
drugs that were not indicated or at least not approved for
certain therapies that have been very, very beneficial to
patients.
Thank you, Mr. Chairman. I yield back.
Mr. Burgess. The Chair thanks the gentleman, the gentleman
yields back. The chair recognizes the gentlelady from
California, Ms. Eshoo, 5 minutes for questions, please.
Ms. Eshoo. Thank you, Mr. Chairman. And thank you to all of
the witnesses. I also want to thank our colleagues who are
offering the drafts and to Mr. Griffith, I especially
appreciate your openness to suggestions and I think that that
is very important.
Over all the years I have been in Congress, this is my 25th
year, and have worked with medical device manufacturers, worked
with the biotechnology industry, done legislation that has
reformed how medical devices are approved, passed legislation
signed into law, but I can't remember which President relative
to pediatric medications, and improved that system for
children. This issue, the issues that are being discussed here
today, no one has ever raised with me. So this is the first
time I am hearing about it. But it is good. It is a discussion,
but it still says something to me that no one has contacted me
about this. So I don't think it is exactly a burning issue.
Number two, it is my understanding that what is being
offered by our two colleagues today were supposed to be a part
of the overall approval for the FDA, but were pulled because
they were controversial. I can hear today where the controversy
is coming from. That is legitimate and I am glad that it wasn't
in the larger bill, because they really didn't belong there.
This cake has not been baked yet.
Now it is my understanding that in one of the discussion
drafts, that there is no clear list of what qualifies as
scientific information. Now that is foundational to me,
scientific information. Not who is gabbing and saying what from
a given industry. That is always interesting and those
discussions take place. But we are dealing with over 200
million people in our country and these words are going to walk
into their life. This is a huge responsibility. They don't know
that we are here today. They don't know any of our names, but
we have the public interest in the safety and the efficacy of
what takes place on their behalf.
To Ms. House, I am not sure, are you in favor of the two
discussion drafts? Yes or no?
Ms. House. We have not taken a formal position on either.
Ms. Eshoo. That is fine.
Ms. House. Neither of them are perfect.
Ms. Eshoo. Yes, well, but I couldn't tell from your
testimony whether you were for or against or where you were.
Ms. Charo, thank you for your testimony. I think that you
have set down the importance of where the information comes
from and that it can't be haphazard. There has to be a final
kind of resting place that has all of the information for
people in our country that can be used.
I don't think anyone has really made the case here to take
it outside of the FDA. Maybe I am missing something, but I
haven't heard that.
To Ms. Khachatourian--I love the I-A 09N; I share either
your husband's heritage or yours--when you spoke about hep C,
how many patients were excluded from treatment?
Ms. Khachatourian. So while I can't speak for all payers
and all----
Ms. Eshoo. No, but you used that as an example, hep C. So
we know, it is a company I am very familiar with in my
district. I have worked with them. They have presented a cure
which we are not accustomed to. It is expensive. But who was
left out, according to your testimony?
Ms. Khachatourian. Sure. So in the initial approval, we
approved treatments according to the label. So for the first
time in hepatitis C, we saw the criteria, the approval criteria
change multiple times. So initially it excluded patients that
might have cirrhosis. It initially excluded patients that
according to the FDA label----
Ms. Eshoo. How do these drafts fix that?
Ms. Khachatourian. So with the drafts, we could understand
that there would be evidence published that would add
additional clinical evidence to indicate effectiveness of
treatment in those sub-populations although at the time of the
initial approval, that evidence was not available for decision
making.
So in my medical space----
Ms. Eshoo. You are saying people were excluded, but you
don't know how many?
Ms. Khachatourian. I can't speak to the exact number
globally. However, within our population, Medicare is who
defines our coverage criteria. So when we submit our criteria
to CMS for approval, it has to be according to the Part D
coverage, what is listed in the FDA-approved label. So we
cannot cover off-label unless it is within the oncology space.
When we are talking about a Part D indication.
Ms. Eshoo. I still don't know who has been injured in this
according to your testimony. That is why I am asking you, and I
still don't know. But I appreciate your trying.
Thank you, Mr. Chairman.
Ms. Khachatourian. If we expand the discussion to
commercial payer outside of Part D, the additional patients
that were denied treatment.
Ms. Eshoo. But you don't know how many.
Ms. Khachatourian. I don't coverage commercial insurance,
however, that is something I would be happy to look into for
you.
Ms. Eshoo. Thank you.
Mr. Burgess. The gentlelady yields back. The Chair thanks
the gentleman. The Chair recognizes the gentleman from
Virginia, Mr. Griffith, 5 minutes for questions, please.
Mr. Griffith. Thank you very much. I appreciate it. Ms.
Klasmeier, we have had some discussions and I know this is not
the Judiciary Committee, but this is where the law touches
everything. And so as we consider legislation in this area,
just so the committee knows as a whole and that I am better
educated, what points should we be taking away from the various
judicial cases in considering First Amendment challenges to the
FDA's regulations? And what should we be looking out for? So
that is Part A and Part B. What should we be looking out for to
make sure that we get it right and that we do it where it is
constitutional as we draft this?
Ms. Klasmeier. Thank you very much for the question,
Congressman. I think a very important take away from the case
law is the need for clarity and that point arises out of the
intersection of the Fifth Amendment case law and the First
Amendment case law. I think there is a lot of discussion about
the First Amendment, but the due process laws requires clarity
and precision, requires rules that give regulated entities
clear notice on an a priori basis of what conduct is prohibited
versus permitted.
Mr. Griffith. And let me, I don't want to cut the rest of
the answer off, but let me interrupt up there because that is
one of my pet peeves. So many times people think that means we
have to define every word in the bill, but if there is no
definition in the bill, then the courts use the normal usage of
the English language or if it is a term of art, the term of art
in this case from the medical community. Is that not correct?
Ms. Klasmeier. It is absolutely correct, sir. And just to
augment your observation, there was a conversation earlier this
morning about the definition of claim and promotion and where
do we draw the line. And I understand why there may be some
misunderstanding around that, but I have to say as a
practitioner in this area--and I also have to say I suffer from
a little bit of an existential crisis because the news that
this is not a hot-button issue or something that needs to be
resolved makes me question what I have spent the last 20 years
of my life doing. But that is an aside.
Mr. Griffith. No worry, her phones will be lit up before
the day is done, I am sure.
Ms. Khachatourian. But there is among those of us who
practice in this area day in and day out a very well-understood
line between promotional speech and nonpromotional speech. So I
think the legislative measures that we have been talking about
this morning would just under foundational interpretive
principles be examined against those background legal norms. So
there is a very rich body of administrative precedent from FDA
in addition to case law and the statutory foundation of the
measures that you are talking about. We know what these words
mean. So I agree to the extent that you are saying we ought not
to feel overly anxious about those two or three words. I think
folks who are battle tested in this area know the difference
between promotional speech and nonpromotional speech and can
advise clients accordingly.
Mr. Griffith. And I kind of got you off track there for a
second. You were talking about the First and the Fifth. I am
going to let you go back to is there anything else on that you
wanted to touch base on that I distracted----
Ms. Khachatourian. Many things, but I will try to limit it
to a big-ticket item, which is it is increasingly obvious from
the case law, which goes back to at least to 1976, that it is
very hard for the Government to defend any speech regulation
that affects accurate communication regarding lawful activity.
I think we tend to get hung up on the kind of Central Hudson
test and prongs and that sort of thing. But just to sort of
bring it down to its essence, if the Government wants to
restrain accurate speech about conduct that is permitted--and
off-label use is not only permitted in almost all cases, it is
by Federal law, it is also the standard of care in many
instances--it has really got an uphill battle.
I think there is probably a way for all of these very
challenging and complex policy considerations to be balanced in
a smart way that takes account of the First Amendment backdrop,
and I think the measures that we are talking about today have
done an admirable job of strengthening that balance. But there
is a little bit of a thumb on the scale, if you like, as a
result of years and years of case law going back to at least
1976 against anything that would purport to prohibit accurate
speech about lawful activity.
Mr. Griffith. And while I wasn't as concerned about the
freedom of speech, per se, although it is very important to me,
when I put in that clause that they have to put in the
contradictory information, as well, and the contextual
information, that actually shores that up from a free speech
standpoint as well, because we are saying you have to present--
if you are going to present--you have to present both sides of
the data. Isn't that accurate?
Ms. Khachatourian. Absolutely accurate, yes, sir.
Mr. Griffith. I appreciate that. And it does make me worry
and I know it is not their field of expertise either, but you
indicated there was a late '90s case that clarified some of
this. I think the bill clarifies it more, but I am just curious
why the FDA keeps going down this pathway when they have lost a
number of cases over the years, if not in this circle of the
three-ring circus, in another circle of that same circus under
the same tent.
Ms. Khachatourian. Yes, well, it is concerning because you
have not only the cases that we have been talking about here,
Caronia and Amarin and Pacira, but also on the dietary
supplement side of the house, a great many cases from the DC
Circuit, a lot of other sources of precedent that draw into
question the constitutionality of the current scheme. That
said, I think there are a lot of undeveloped arguments that we
have been, in industry, waiting with bated breath for FDA to
articulate and there was a memoranda that FDA lodged in one of
its administrative dockets in January, right before the
inauguration that purported to explain for all the world to see
how the agency thought through these constitutional issues and
it was a little more than a defense of the status quo.
I think there is a lot of room for optimism in the coming
months, particularly with the involvement of this subcommittee
and the Congress, generally, that FDA will do a better job of
explaining and including stakeholders in a conversation about
the constitutionality and constitutional issues associated with
this current regulatory scheme.
Mr. Griffith. I appreciate it and yield back. Thank you,
Mr. Chairman.
Mr. Burgess. The Chair thanks the gentleman. The Chair
thanks Ms. Khachatourian for her optimism. We always welcome
optimism on this subcommittee.
The Chair now recognizes the gentleman from Maryland, Mr.
Sarbanes, 5 minutes for questions.
Mr. Sarbanes. Thank you, Mr. Chairman. I want to thank the
panel. This is a really complicated issue, I am finding. I sat
here through the entire testimony. And certainly the ability
and the internet is kind of at the center of this now for
people to get hold of information about beneficial off-label
use of drugs and medical devices much more readily than
obviously they ever could before, is creating some pressure to
figure out a way to make that opportunity more available to
people. The fast distribution of information can also allow for
the fast distribution of bad information and lead to poor
decision making. But I understand that Congressmen Griffith,
Guthrie, and others are trying to respond to pressure and often
it comes from patients that are seeking a solution.
What I am concerned about is that you could solve the way
they are proposing for this pressure, or you could solve
perhaps by building more capacity inside the FDA. So what I am
interested in hearing about, I don't want us to take a
shortcut. I don't want the reason we are reaching for the
proposed solution here to be that we have overlooked the
opportunity to build more capacity in FDA as a way of solving
for this, and perhaps solving for in a way that protects public
safety better than taking the alternative route.
So I wonder, Ms. Charo, maybe you could begin here. Speak
to that issue. How do we explore fully the opportunity to build
capacity in FDA to respond to the pressure we are talking
about? Can that be done? If so, what are the ways in which it
can be done, et cetera?
Ms. Charo. Well, first, I am going to second what has been
said by others here which is that FDA, just in terms of sure
personnel, would certainly benefit from having more people able
to act on data as it is coming in and everything would move
more rapidly with no question. But we shouldn't restrict
ourselves only to FDA. I mean one of the things we have been
struggling with here is that there are areas in which the
incentive systems that currently exist are inadequate for
driving the research that we all agree would be ideal to figure
out what really works and what does not. Pediatrics, rare
diseases are two very good examples.
Now we have some new tools. Congress have given things like
priority reviews and extended patent periods as incentives, but
we have yet to completely explore the full range of tools.
Antibiotics is another example where the Infectious Disease
Society of America has been pointing out for years we could use
rewards, milestone rewards. We have not talked about NIHI
funding for direction of studies that would look at things like
off-label uses that are hinted at already and that need to be
confirmed.
In other words, we need not restrict ourselves to only one
tool which is to pull the industry slowly to do the research
under the threat of not being able to market. But we could
bring to bear a combination of tools to get the information
developed more rapidly. And ideally, then everybody would
benefit because we would have a wider range of applications,
but we would have more confidence that they have been tested in
a way that is comprehensive and objective and has been vetted
by independent eyes.
Mr. Sarbanes. I appreciate that. I mean, I worry a little
bit that I don't completely trust the industries we are talking
about here to restrain themselves if they get--if there is an
avenue for aggressively pursuing a particular product's appeal
out there in ways that may compromise public safety, and I
worry about a bunch of camels starting to get their noses under
the tent. So I understand the desire to try to accommodate
people's interest in pursuing this, but if there are other ways
we can respond to that, without sacrificing some of these
concerns about public safety, then I think that we ought to
pursue those and explore some of the additional tools that you
have suggested, perhaps. With that, I yield back. Thank you.
Mr. Burgess. The gentleman yields back. The Chair thanks
the gentleman. The Chair recognizes the gentleman from Florida,
Mr. Bilirakis, 5 minutes for questions.
Mr. Bilirakis. Thank you, Mr. Chairman. I thank the panel
as well. I have a question for Ms. House. Again, thank you for
your testimony. Throughout my time on the Energy and Commerce
Committee, I have been involved with the rare disease
community. There are about 30 million Americans--and there are
7,000 rare diseases--30 million Americans have a rare disease,
which includes pediatric cancers. And I understand there are
about 500 FDA-approved treatments. Correct me if I am wrong.
Do you think that many of these 30 million Americans are
taking medications off-label? For Ms. House, please.
Ms. House. Yes. Yes, I do. I do. In my written comments, I
have referenced in particular lupus, and if you look at the FDA
site right now, there are only four drugs that are approved for
lupus. And the approvals of those go back into the mid-1900s.
So when you look at the drugs, aspirin was approved first in
1948, followed by steroids, and there was no drug listed. There
was an antimalarial that was approved in 1955. And finally, a
new drug approved in 2011. So if you are a patient living with
lupus, you are likely not getting aspirin as a therapeutic
option for your particular disease. And certainly when you look
at cancer, there is a reason why there is such a high rate of
pediatrics in cancer clinical trials, and it is because they
don't have a lot of other options available to them.
Mr. Bilirakis. Thank you, so there are other examples out
there. So a large percentage of the 30 million are taking
medication off-label.
Ms. House. Arthritis is another good sample. If you look at
the label of methotrexate, for example, you will see that the
label doesn't reflect the broad use of that particular product
and you can probably speak to that better than I could.
Mr. Bilirakis. Thank you. I am here with a young Floridian
from the Miami area who told me about how she came down with
ITP, a condition where her body destroyed her platelets. And I
have conversed with her over a long period of time on these
particular issues. I have sponsored the Open Act and we are
working together.
She had to become an expert. She became an expert on ITP,
and she really became her own doctor and found a treatment,
really extraordinary. She was able to find a drug that could
treat her condition. The drug was FDA-approved for non-
Hodgkin's lymphoma and rheumatoid arthritis, but not for ITP.
After a long conversation with her physician, we were able
to pursue that course, the off-label treatment, and it was very
successful. She comes to DC on a regular basis as an advocate
for cures and treatments for rare diseases.
Ms. House, does it make sense to withhold information from
physicians and not share truthful medical information that
could say a person's life? And who should be in charge of a
patient's treatment? The patient working with her physician or
again, a bureaucrat? If you could answer that question, I would
appreciate that.
Ms. House. Well, you know, we have spent 35 years trying
to assist patients to become equal participants and empower
participants in their care, so I am going to answer that as the
patient needs to be quarterback of their care, working with
their particular physician.
I will say that it is incredibly important though that the
information that is provided, both to patients and to
physicians, is fair balanced. I worked in the pharmaceutical
industry for a period of time, so I also understand the bright
white lines between what is promotional and what is
nonpromotional and we are not talking about shipping patients
or physicians glossy pieces of information on off-label uses or
other additional information, but we have to provide for them
and whether that is, I do agree that there are alternative
solutions, whether it is through the FDA, whether it is through
a professional society, whether it is through a third party
peer reviewed entity, we have to get to a point where we are
providing that data set to people who are making decisions,
including patients who are making more and more of their care
decisions as you have referenced.
Mr. Bilirakis. Thank you. Agreed. Dr. Van Hare, in your
practice, you deal with children and adults who suffer from a
heart condition such as the congenital heart and some are
congenital heart in nature. I sponsored a bill to reauthorize a
congenital heart program and it went through this committee and
hopefully on the floor as soon as possible.
If you have a child who comes to the hospital with a heart
condition, you might need to do a surgical procedure. How
common is it for medical devices to be approved for use in
children?
Mr. Bilirakis. Well, as I understand, most medical devices,
at least that I use in the cardiology sphere are not specific
to children or adults. They are more specific to actual
specific arrhythmias. And as I talked about in my oral
testimony, a lot of what we take care of, the devices, in fact,
are not labeled for those particular sort of conditions.
I will say that you sort of raise the issue of surgery for
congenital heart disease. We often think about surgery as
basically correcting a problem. But those patients need to have
a cardiologist for the rest of their life and one of the
biggest problems if they develop heart rhythm issues and those
heart rhythm issues are often very, very difficult to take care
of and so we are reaching for whatever we can find to treat
those things most effectively. And we use technology and we use
devices that have been approved for other indications for this
particular situation.
I just want to emphasize that we keep talking about
pediatrics as sort of being an important issue and I am a proud
pediatrician and I believe that. But I think pediatrics is a
special case of a larger issue which is there are a lot of
patients that devices and drugs have been developed for other
indications. We have to find a way to take care of our
patients. I think pediatric diseases, but also rare diseases,
and anything that is kind of on a cutting edge of what we are
doing medically to treat things are going to fall into this
discussion.
Mr. Bilirakis. Thank you very much. I yield back, Mr.
Chairman.
Mr. Burgess. The Chair thanks the gentleman. The gentleman
yields back. The Chair recognizes the gentleman from New York,
Mr. Engel, 5 minutes for questions, please.
Mr. Engel. Thank you very much, Mr. Chairman. I have long
been an advocate for those suffering from rare diseases. I was
an author of the ALS Registry Act and the two most recent
Muscular Dystrophy Care Act reauthorizations and I know how
much relief and encouragement new therapies can bring to rare
disease patients. And I think I speak for everyone on this
subcommittee when I say that all of us want to do what we can
to bring effective and potentially life-saving treatments to
patients as quickly as possible, but it is absolutely critical
that we ensure our actions do not compromise patient safety.
Efficiency is a worthwhile goal that we all share, but as
we strive to hasten the delivery of new treatments, safety and
effectiveness must always be paramount and that is why this
hearing is so important. Any action by this committee needs to
take into account the input of expert witnesses who can speak
to the potential implications of our actions. And that is what
we have, Mr. Chairman, in our panel. And so I want to thank
today's witnesses for being here and sharing your insights.
Let me start with Ms. Charo. During your testimony, you
noted that ``approval of a drug for labeled''--I am quoting
you--``indication does not mean it will be safe and effective
for off-label uses.'' And that ``additional studies are needed
to explore them.''
Now it would seem to me that if a manufacturer wished to
communicate about an off-label use for a product that
manufacturer must already have reason to believe that this
product is safe and effective for the given off-label use. So
if there is already evidence supporting an off-label use, can
you explain why additional studies would be necessary?
Ms. Charo. Of course. And I think other people on this
panel are even more expert than I in research trial design, but
the reality is that evidence comes in many forms and often it
is based on small numbers of people with very homogenous kinds
of situations. But in the real world, you need larger numbers
of people with a wider variety of background conditions and
complexities in order to detect both the areas in which it will
or will not be effective. It might depend upon co-morbidity,
and also to detect some of the less common kinds of side
effects or adverse events.
And those things are relevant to deciding whether or not
the benefit that some people get will be sufficient to outweigh
the kinds of risks or failures to work for other people.
So initial evidence often can look extremely promising.
Preclinical evidence, particularly we have cured cancer in mice
countless times, but also early human evidence is often very,
very promising and then when we move into larger trials with
more complicated and more diverse populations we discover that,
unfortunately, it was misleading. And it is just a matter of
basic statistics as well as medicine. That is why there is such
an emphasis on properly controlled trials of sufficient size
and statistical power and the ability, too, to look at the
possibility of inherent biases and how you structure the
trials. It is very easy to structure trials in a way that
subtly lead to one conclusion or another without even intending
to do so. That is the value of the independent expert eyes that
the agency brings.
Mr. Engel. Thank you very much. Dr. Kesselheim, you also
touched on the need for additional studies in your testimony.
So I would like to ask you the same question. If there is
already evidence supporting an off-label use, can you explain
why additional studies would be necessary?
Dr. Kesselheim. Sure. I mean if there is evidence
supporting an off-label use and there are certainly plenty of
ways that that evidence can already be communicated under the
current rules. I think the rules are fairly clear about what
types of communications are, where there are opportunities to
communicate that information. And if there are additional
studies and again, I think the importance is what is the nature
of that evidence. How is that evidence defined? What are the
statistical methods that were used in testing? How is the
population defined? And these are details that, you know,
average physicians don't know a lot, don't have a lot of
training in and don't know a lot about it and these are the
details that the FDA has expertise in. And so if there are
nuances that might not be caught in initial examination of the
information, additional studies that are necessary, then the
numerous dozens of experts at the FDA with training in various
different fields can identify that and pick up on that and
determine whether or not what might initially be seen in the
data, turns out to be legitimate.
Mr. Engel. Thank you. Ms. Charo, I have one final question
for you. It is my understanding that in January the FDA
released draft guidance regarding which manufacturer
communications are consistent with the FDA required labeling in
which are not. And I understand also that this guidance has not
yet been finalized.
So do you feel that draft guidance strikes the right
balance between enabling potentially helpful communications to
take place and protecting patient safety and why shouldn't we
legislate in this space to provide even greater clarity for
manufacturers?
Ms. Charo. I do think the FDA is moving in the right
direction. I agree that draft guidances would be better off if
they were finalized guidances, although it is worth noting that
a tremendous amount is already done through draft guidances at
the FDA without any Fifth Amendment due process questions being
raised about it.
The thing that I think is most important about what the FDA
has been doing is its insistence that actual knowledge about
how your product is being used can be in some instances
considered to be evidence that you actually intended for the
product to be used that way. I think a lot of the debate has
been around that phenomenon. But we have seen that phenomenon
in other contexts. We have seen it in areas having to do with
constructive knowledge in tort law where if you know something
is about to happen and you actually go ahead and do all the
things that are necessary for it to come about, you are
actually going to be considered to have intended that to happen
in many cases.
On the other hand, we have seen in the area of gun law, a
lot of resistance to the idea that actual knowledge constitute
intent. I do think that is an area where we have to have some
more discussion to clarify, but I also think that it is risky
to simply allow for an expansion of communication while
simultaneously saying but now that I have communicated more,
the fact that I know that it is having an effect doesn't mean
that I intended that particular outcome. I think to have both
of those things at once I think is particularly risky. Choosing
one or the other at least would be the right direction.
Mr. Engel. Thank you very much. Thank you, Mr. Chairman.
Mr. Burgess. The gentleman yields back. The Chair thanks
the gentleman. Does the gentleman from Texas have a unanimous
consent request?
Mr. Green. Yes, Mr. Chairman, I have a consent request.
Mr. Burgess. I will yield for a unanimous consent request.
Mr. Green. I move that we have statements in the record
from the American Health Insurance Plans, the Campaign for
Sustainable Drug Pricing, and also Public Citizen Action be
placed into the record.
Mr. Burgess. Without objection, so ordered. Seeing no other
Members wishing to ask questions, I once again want to thank
our witnesses for being here today.
[The information appears at the conclusion of the hearing.]
Mr. Burgess. Pursuant to committee rules, I remind Members
they have 10 business days to submit additional questions for
the record. I ask the witnesses to submit their responses
within 10 business days upon receipt of those questions. And
without objection, the subcommittee stands adjourned.
[Whereupon, at 12:37 p.m., the subcommittee was adjourned.]
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