[House Hearing, 113 Congress]
[From the U.S. Government Publishing Office]
ADDRESSING THE NEGLECTED DISEASES TREATMENT GAP
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HEARING
BEFORE THE
SUBCOMMITTEE ON AFRICA, GLOBAL HEALTH,
GLOBAL HUMAN RIGHTS, AND
INTERNATIONAL ORGANIZATIONS
OF THE
COMMITTEE ON FOREIGN AFFAIRS
HOUSE OF REPRESENTATIVES
ONE HUNDRED THIRTEENTH CONGRESS
FIRST SESSION
__________
JUNE 27, 2013
__________
Serial No. 113-76
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Printed for the use of the Committee on Foreign Affairs
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COMMITTEE ON FOREIGN AFFAIRS
EDWARD R. ROYCE, California, Chairman
CHRISTOPHER H. SMITH, New Jersey ELIOT L. ENGEL, New York
ILEANA ROS-LEHTINEN, Florida ENI F.H. FALEOMAVAEGA, American
DANA ROHRABACHER, California Samoa
STEVE CHABOT, Ohio BRAD SHERMAN, California
JOE WILSON, South Carolina GREGORY W. MEEKS, New York
MICHAEL T. McCAUL, Texas ALBIO SIRES, New Jersey
TED POE, Texas GERALD E. CONNOLLY, Virginia
MATT SALMON, Arizona THEODORE E. DEUTCH, Florida
TOM MARINO, Pennsylvania BRIAN HIGGINS, New York
JEFF DUNCAN, South Carolina KAREN BASS, California
ADAM KINZINGER, Illinois WILLIAM KEATING, Massachusetts
MO BROOKS, Alabama DAVID CICILLINE, Rhode Island
TOM COTTON, Arkansas ALAN GRAYSON, Florida
PAUL COOK, California JUAN VARGAS, California
GEORGE HOLDING, North Carolina BRADLEY S. SCHNEIDER, Illinois
RANDY K. WEBER SR., Texas JOSEPH P. KENNEDY III,
SCOTT PERRY, Pennsylvania Massachusetts
STEVE STOCKMAN, Texas AMI BERA, California
RON DeSANTIS, Florida ALAN S. LOWENTHAL, California
TREY RADEL, Florida GRACE MENG, New York
DOUG COLLINS, Georgia LOIS FRANKEL, Florida
MARK MEADOWS, North Carolina TULSI GABBARD, Hawaii
TED S. YOHO, Florida JOAQUIN CASTRO, Texas
LUKE MESSER, Indiana
Amy Porter, Chief of Staff Thomas Sheehy, Staff Director
Jason Steinbaum, Democratic Staff Director
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Subcommittee on Africa, Global Health, Global Human Rights, and
International Organizations
CHRISTOPHER H. SMITH, New Jersey, Chairman
TOM MARINO, Pennsylvania KAREN BASS, California
RANDY K. WEBER SR., Texas DAVID CICILLINE, Rhode Island
STEVE STOCKMAN, Texas AMI BERA, California
MARK MEADOWS, North Carolina
C O N T E N T S
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Page
WITNESSES
Lee Hall, M.D., Ph.D., chief, Parasitology and International
Programs Branch, Division of Microbiology and Infectious
Diseases, National Institute of Allergy and Infectious
Diseases, National Institutes of Health, U.S. Department of
Health and Human Services...................................... 5
Jesse Goodman, M.D., chief scientist, Food and Drug
Administration, U.S. Department of Health and Human Services... 21
Peter J. Hotez, M.D., Ph.D., president, Sabin Vaccine Institute.. 52
Jay Siegel, M.D., chief biotechnology officer and head of
scientific strategy and policy, Johnson & Johnson.............. 69
Alix Zwane, Ph.D., executive director, Evidence Action........... 84
LETTERS, STATEMENTS, ETC., SUBMITTED FOR THE HEARING
Lee Hall, M.D., Ph.D.: Prepared statement........................ 8
Jesse Goodman, M.D.: Prepared statement.......................... 24
Peter J. Hotez, M.D., Ph.D.: Prepared statement.................. 58
Jay Siegel, M.D.: Prepared statement............................. 72
Alix Zwane, Ph.D.: Prepared statement............................ 87
APPENDIX
Hearing notice................................................... 100
Hearing minutes.................................................. 101
Written response from NIAID to question submitted for the record
by the Honorable Christopher H. Smith, a Representative in
Congress from the State of New Jersey, and chairman,
Subcommittee on Africa, Global Health, Global Human Rights, and
International Organizations.................................... 102
ADDRESSING THE NEGLECTED DISEASES TREATMENT GAP
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THURSDAY, JUNE 27, 2013
House of Representatives,
Subcommittee on Africa, Global Health,
Global Human Rights, and International Organizations,
Committee on Foreign Affairs,
Washington, DC.
The subcommittee met, pursuant to notice, at 2:06 p.m., in
room 2200, Rayburn House Office Building, Hon. Christopher H.
Smith (chairman of the subcommittee) presiding.
Mr. Smith. Good afternoon.
Today's hearing will examine the neglected diseases that
affect a relatively small but a significant number of children
around the world. These diseases are not only debilitating for
their victims but far too often fatal when untreated. Such
diseases largely impact poor people in poor countries.
They are not only small in numbers but those folks who are
unable to pay market prices for treatments and are unlikely to
lead the social movements to force action on their respective
diseases. That means that research on detection, vaccines, and
drug treatment for their ailments do not receive the priority
that diseases such as HIV/AIDS, often seen in pandemic levels,
are given.
The World Health Organization has identified 17 neglected
tropical diseases, or NTDs. The list ranges from Chagas to
rabies to leprosy to dengue fever. However, there are others
not on the list of 17 diseases that also receive less
attention. These include such diseases as polio and smallpox,
which have largely been eliminated from the planet, and fatal,
fortunately rare NTDs such as kuru and Ebola.
I would note parenthetically, back in the early 1980s, when
I authored what was known as the Child Survival Reauthorization
Amendment for some $50 million, I traveled to El Salvador, and
the polio vaccine was given to upwards of 200,000 children,
along with other immunizations to guard against pertussis,
diphtheria, and other leading killers of children. Thankfully,
some of those diseases are largely gone, but some, sadly, are
making a comeback.
I would also point out that we have had hearings in this
subcommittee and I have introduced legislation to focus on
another problem, particularly in Africa, of the infection-based
hydrocephalic condition that is hurting so many children in
places like Uganda.
I actually went to CURE International's clinic in Uganda
and saw children who got the treatment. Dr. Benjamin Warf has
developed it out of Harvard. There are no shunts involved, and
these kids went from very large painful conditions in their
head, water on the brain, to very healthy children. And at
least 5,000 kids have been saved there. We have been asking,
urging, begging practically, that USAID do something
administratively to try to address that issue.
This hearing will consider the current U.S. Government's
handling of these and other neglected diseases to determine
what more can be done or should be done to address this
situation. Current U.S. law favors research on those diseases
threatening the American homeland, but in today's world
diseases can cross borders as easily as those affected by them
or the products imported from the United States.
For example, Chagas is most prevalent in Latin America, but
it has been identified in patients in Texas. And cases of
dengue fever have recently been reported in Florida. We cannot
afford to assume that what may have seemed to be an exotic
disease only happens to people in other countries.
Ten years ago, West Nile virus, another NTD, was not seen
in the United States or anywhere else outside of the East
African nation of Uganda, but in less than a decade it has
spread across the country and much of the rest of the world.
Last year, 286 people died from West Nile virus in the United
States alone. As recently as the mid-1990s, this disease was
seen only sporadically and was considered a minor risk for
human beings.
Generally, NTDs affect the health of the poor in developing
countries where access to clean water, sanitation, and health
care is limited. Roughly 2 billion people are being treated for
at least one NTD, although most individuals are infected with
several NTDs at once.
Several NTDs are difficult to control by drug treatment
alone because of their complicated transition cycles that
involve nonhuman carriers, such as insects. Furthermore, some
of the drugs have significant side effects, including death,
and cannot be used by young children or pregnant women.
A study done in 2001 found that research and development of
drugs to treat infectious diseases had ground to a near
standstill. From 1975 to 1999, the report stated 1,393 new
drugs were brought to the market globally, but only 16, or 1.1
percent, were for tropical diseases, including malaria and even
tuberculosis, although these diseases represented 12 percent of
the global disease burden.
A 2012 update of that study found that the gap between the
percentage of research and development of NTDs and their
percentage of the global disease burden had narrowed, but there
is still a long way to go to reach an adequate balance.
Of the 756 new drugs approved between 2000 and 2011, 29, or
3.8 percent, were for neglected diseases, although the global
burden of such diseases was estimated at 10.5 percent. Of
these, only four were new chemical creations, three of which
were for malaria, but none for tuberculosis or neglected
tropical diseases.
It is unprofitable for companies to create treatments for
diseases with few victims, and especially for few paying
victims, and no certain way to recover research and development
costs. Our heart goes out to those who suffer from these
neglected diseases, and we want our Government to speed up
research and development in cooperation with universities and
private companies.
However, research and development takes time and effort and
costs money that private companies perhaps cannot easily
justify to their stockholders, including many of us, without
incentives. We are here today to consider such incentives and
to look at the system in place to forge successful efforts to
deal with NTDs.
We have with us representatives from the National
Institutes of Health, which was established to understand,
treat, and ultimately prevent the many infectious immunologic
and allergic diseases that threaten millions of human lives.
Their government partner in the system for developing solutions
to the problem of NTDs and other diseases, of course, is the
Food and Drug Administration, which, among other
responsibilities, is charged with protecting and promoting
public health through the regulation and supervision of
prescription and over-the-counter pharmaceutical medications,
vaccines, and biopharmaceuticals.
Also joining us today are representatives from a network
specializing in providing medicines at the lowest possible
price to those suffering from NTDs, a major pharmaceutical
company that develops new drugs for the treatment of diseases,
rare and otherwise, and a new organization seeking to extend
the benefits of proven interventions to improve the lives of
the poor in developing countries.
If a solution to the gap between existing research and
development and successful strategies to meet the challenges of
NTDs is to be found, it will take the collaboration of
organizations represented here today, who are all leaders in
their field.
I would like to yield to Dr. Bera and then to my friend,
Mr. Meadows.
Mr. Bera. Thank you, Chairman.
And I want to applaud you for having a series of hearings
on the importance of emerging diseases and the importance of
developing new therapeutics and so forth. Return on investment
can't just be measured in dollars returned and dividends.
Return on investment also is on relief of suffering, lives
saved, and so forth, and those are clearly very important
measures. And I applaud you for championing that and having
these hearings.
You know, I look at this issue as a physician and have to
look at it from that perspective of global health. I am excited
about hearing the testimony. It is not going to be an easy
challenge, elevating the consciousness. And, you know, when I
look at a number of the neglected diseases--you know, the last
I discussed this was when I took parasitology in medical
school.
But having recently, you know, a few years ago, traveled to
Nicaragua with our medical students from UC-Davis to work out
there, it is very, you know--we were dealing with a dengue
fever epidemic. And, you know, I am going to be curious and
certainly ask some questions about developing better
surveillance mechanisms, as well, so we are actually better
capturing the burden of disease, especially with emerging
diseases.
And, you know, I am excited about this testimony. So,
again, that is the doctor in me, and that is the public health
side of me.
So, with that, I will yield back. And, again, I am looking
forward to the testimony.
Mr. Smith. Thank you.
Mr. Meadows?
Mr. Meadows. Thank you, Mr. Chairman.
And thank each of you for coming today.
I want to echo what my colleague, Dr. Bera, just said. The
chairman has been a strong, unflinching voice for those that,
many times, never have a voice here on Capitol Hill.
And it is an honor to serve with you.
And I certainly look forward to hearing your testimony as
we look at this. They have called votes, so I am going to keep
my opening remarks brief and hear from the experts here, but
thank you so much for being willing to highlight this and help
us prioritize and help identify what we can do legislatively
and certainly from an appropriations standpoint to make your
job easier. Thank you.
With that, I will yield back, Mr. Chairman.
Mr. Smith. Thank you very much, Mr. Meadows.
I would like to introduce our very distinguished panelists.
We do have a vote under way, and I deeply apologize for the
inconvenience. I thought I would introduce you and then we
would be in brief recess. And hopefully several other members
will come back to hear your testimony so there is no rush.
So let me introduce our very distinguished panel, beginning
with Dr. Lee Hall, who is chief of Parasitology and
International Programs Branch, Division of Microbiology and
Infectious Diseases at the National Institute of Allergy and
Infectious Diseases, part of the National Institutes of Health.
He oversees multiple programs that support basic translational
and clinical research in development in parasites and vectors
responsible for transmission of parasites.
He has developed and provided oversight for a range of
research programs and has served on numerous committees for
Federal international activities, including vaccine R&D and
global health. Dr. Hall has also chaired and participated in
numerous scientific symposia and national and international
meetings.
And our next witness will then be Dr. Jesse Goodman, who is
the chief scientist of the FDA. He has a broad responsibility
for and engagement in strategic leadership and coordination of
FDA's crosscutting scientific and public health efforts,
including for public health preparedness and medical
countermeasures. He led the 2009 H1N1 pandemic response and the
medical countermeasure review for the Food and Drug
Administration.
Prior to joining FDA, he was a professor of medicine and
chief of infectious diseases at the University of Minnesota,
where he directed multihospital infectious disease research. He
has authored numerous scientific papers and has been elected to
the American Society for Clinical Investigation and to the
Institute of Medicine.
We will stand in brief recess and then continue with the
hearing.
[Recess.]
Mr. Smith. The committee will resume its meeting.
And I would like to start with Dr. Hall, if you could, and
then go to Dr. Goodman. Thank you.
I do apologize for that very long delay with the votes.
Let me just say, vice chairman of the committee, Mr. Weber,
do you have anything to add?
Mr. Weber. I am good to go.
Mr. Smith. Okay.
STATEMENT OF LEE HALL, M.D., PH.D., CHIEF, PARASITOLOGY AND
INTERNATIONAL PROGRAMS BRANCH, DIVISION OF MICROBIOLOGY AND
INFECTIOUS DISEASES, NATIONAL INSTITUTE OF ALLERGY AND
INFECTIOUS DISEASES, NATIONAL INSTITUTES OF HEALTH, U.S.
DEPARTMENT OF HEALTH AND HUMAN SERVICES
Dr. Hall. Mr. Chairman and members of the committee, thank
you for the opportunity to discuss the global and domestic
public health threat of neglected tropical diseases, or NTDs,
which affect millions of people worldwide and significant
numbers in the United States.
I am chief of the Parasitology and International Programs
Branch of the National Institute of Allergy and Infectious
Diseases, or NIAID, at the National Institutes of Health. I
will now present a summary of my written testimony, which has
been submitted for the record.
NIAID supports research to better understand, treat, and
ultimately prevent infectious, immunologic, and allergic
diseases, including the great global killers HIV/AIDS,
tuberculosis, and malaria; emerging infectious pathogens, such
as West Nile virus, Ebola virus, and the novel coronavirus
emerging in the Middle East; and reemerging infectious
diseases, such as dengue fever.
NIAID is committed to improving public health through
support of basic research, identification of drug and vaccine
targets, preclinical testing, and clinical trials. NIAID also
offers a broad array of preclinical and clinical resources for
researchers to help generate the evidence necessary for the
review and eventual approval and licensure of diagnostics,
therapeutics, and vaccines by the Food and Drug Administration.
The World Health Organization has identified 17 infectious
diseases as NTDs. These NTDs are concentrated in impoverished
populations in the developing world. Among them are several
well-known diseases such as dengue fever, African sleeping
sickness, and Hansen's disease, or leprosy.
The global burden of these diseases is high. Over 1 billion
people suffer from one or more NTDs, which exact an
extraordinary human and economic cost. NTDs both result from
and contribute to poverty. And they are often co-endemic with
other infectious diseases, such as HIV/AIDS, tuberculosis, and
malaria.
Treatment and prevention options for NTDs are currently
limited. NIAID is working to strengthen the research and
development pipeline for NTDs by leveraging existing clinical
research infrastructure and resources. For example, the NIAID
Tropical Medicine Research Centers are designed to facilitate
research on the cause, diagnosis, prevention, and treatment of
NTDs in countries where they are endemic.
NIAID also facilitates research on NTDs by providing access
to resources, including repositories of genomic sequences and
samples of parasites, transmission vectors, and hosts, as well
as services for early-stage development of NTD countermeasures.
NIAID enters into public-private partnerships with a variety of
organizations to share the cost and risk of developing new and
improved vaccines, treatments, diagnostics, and vector control
strategies.
But time does not permit me to describe all of the work we
are doing in this area. I will now highlight recent NIAID-
supported scientific discoveries on several important NTDs.
Dengue fever affects millions of people every year and is
reemerging as a disease of public health importance in the
Americas, including endemic transmission in Puerto Rico and
locally acquired cases in Florida. The disease is caused by
mosquito-borne viruses that produce high fever, joint and
muscle pain, and, in severe cases, death.
NIAID is funding studies on dengue fever, including
development of rapid diagnostic tests, therapies, and vaccines.
Recently, NIAID scientists identified TetraVax, a promising
vaccine candidate that has been licensed by manufacturers in
Brazil, India, and Vietnam. Phase two clinical trials to
further evaluate TetraVax will begin soon in Brazil and
Thailand. If successful, this vaccine could be instrumental in
limiting the spread of dengue fever worldwide.
Seven million to eight million people have Chagas disease.
As many as 300,000 people in the United States may have Chagas
disease, most having been infected in endemic countries and
then traveling to the United States. The growing pipeline of
treatments for Chagas disease includes a promising drug
candidate, K777. NIAID-funded basic research identified this
drug. NIAID also supported preclinical studies and will
continue clinical development of K777 to determine its safety
and efficacy for treating Chagas disease. NIAID-supported
researchers also are exploring innovative designs for Chagas
disease vaccines. Such interventions are showing great promise
in limiting Chagas disease worldwide.
Schistosomiasis is a chronic disease caused by parasitic
worms. Though its mortality is relatively low, tens of millions
of people worldwide suffer chronic and debilitating
consequences. NIAID supported the genome sequencing of multiple
species of worms responsible for different forms of
schistosomiasis, including one that causes a form associated
with bladder cancer. This genomic information has helped to
identify potential cancer-causing genes in this organism as
well as targets for new antiparasitic therapies.
NIAID also pursues the development of schistosomiasis
vaccines. Recently, NIAID-supported researchers identified a
promising vaccine candidate, and NIAID is partnering with a
small business under NIH's Small Business Innovation Research
Program to further preclinical development of this vaccine.
Together with the Bill and Melinda Gates Foundation, NIAID
sponsored a meeting this year to assess schistosomiasis vaccine
candidates and provide guidance for future vaccine research.
Hansen's disease, also known as leprosy, is a chronic
bacterial disease that often leads to lifelong disability.
Hansen's disease has long been associated with social stigma
and discrimination. Nearly 230,000 new cases of Hansen's
disease were identified globally in 2010. In the United States,
213 cases were reported in 2009, including some thought to
result from transmission to humans from armadillos.
NIAID research efforts on Hansen's disease are focused on
early detection, prevention of nerve damage, and discovery of
emerging drug resistance. Armadillos, the only animal known to
be susceptible to Hansen's disease, are an important tool for
research on this disease. NIAID has supported the only globally
available research reagents from Hansen's disease propagated in
armadillos and also supported the development of the armadillo
as an animal model to evaluate the efficacy of new drugs and
vaccines.
In conclusion, NIAID will continue its longstanding
investment in improved diagnostics, therapeutics, and vaccines
to control the global and domestic threat of NTDs by
capitalizing on public-private partnerships and fruitful
collaborations with academia, nonprofit organizations, and
industry.
Thank you for the opportunity to testify about this
important issue. I would be pleased to answer the committee's
questions.
Mr. Smith. Thank you very much, Dr. Hall.
[The prepared statement of Dr. Hall follows:]
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Mr. Smith. Dr. Goodman?
STATEMENT OF JESSE GOODMAN, M.D., CHIEF SCIENTIST, FOOD AND
DRUG ADMINISTRATION, U.S. DEPARTMENT OF HEALTH AND HUMAN
SERVICES
Dr. Goodman. Mr. Chairman and members of the subcommittee,
I am Jesse Goodman, chief scientist at the Food and Drug
Administration. I, too, want to thank you for this opportunity
to discuss rare and neglected diseases and the progress that is
being made to accelerate development of products needed to
diagnose, prevent, and treat them.
Rare and neglected diseases taken together are a major
public health concern. For example, in the United States, rare
diseases together affect 30 million Americans. Around the
world, most often in impoverished areas, over 1 billion people
are affected by at least one neglected disease. As a practicing
infectious disease physician, much like you have mentioned, Dr.
Bera, I have personally witnessed the devastating toll of these
diseases on humans infected with them.
This hearing is also a great opportunity to remind our
Nation, as the chairman did, that infectious diseases know no
boundaries and that threats to health anywhere are threats to
everyone. For example, in recent months, we have seen outbreaks
of avian flu in China and of coronavirus, a SARS-like
coronavirus in the Middle East that have alarmed the world and
spurred preparedness efforts here in the United States.
As you also noted, in 2010 our colleagues at CDC reported
dengue, a mosquito-borne infection common throughout the world,
for the very first time in United States residents who had not
traveled abroad. So this is what we would consider a tropical
disease now occurring in our country. Dengue is also a
potential threat to blood safety, as also is Chagas disease. So
there are both compelling humanitarian reasons but also U.S.
national security and health reasons to work together to
protect against these diseases.
For all of these reasons, we engage in close collaborations
to accelerate development and availability of needed products.
I also want to recognize the innovative efforts of many in
industry and nongovernmental organizations such as those that
Drs. Siegel, Zwane, and Hotez are going to talk about here
today.
Our efforts at FDA depend on strong science and a highly
interactive review process and utilize a variety of novel tools
and incentives. While the need remains great, these approaches
have led to important successes, particularly in rare diseases,
and I am optimistic that many of them can be applied to
neglected diseases.
The Orphan Drug Act provides important incentives to
encourage development of drugs to treat rare diseases.
Neglected diseases almost always will qualify for orphan drug
designation. When drug sponsors apply for and obtain orphan
drug designation, they get a number of benefits: Tax credits
and grant opportunities for clinical trials, waivers of their
user fees, and a period of market exclusivity if the drug is
approved.
The FDA Safety and Innovation Act, passed by Congress just
last year, provides additional new incentives for certain drugs
intended to treat serious or life-threatening infections,
including drug-resistant infections. So we are very interested
in that.
Just this week, we issued new draft guidance for industry
that puts together all of our different programs for expediting
development of products for serious and life-threatening
conditions. They are discussed in detail in my written
testimony, but one program I wanted to highlight is what we
call accelerated approval, which allows approval of a drug or
vaccine based on what we call a surrogate endpoint, such as a
lab finding or a clinical finding, that is scientifically shown
to be likely to predict an ultimate benefit on a serious
outcome. This can allow much earlier assessment and approval of
likely effectiveness, particularly for chronic diseases.
FDA also recognizes the importance of global collaboration
and engagement. While it is a resource challenge, FDA, NIH, and
CDC are very involved in global health. We played an important
role working together in developing what is called the Global
Vaccine Action Plan, which was recently adopted by the World
Health Assembly. Lee and I both worked on that.
FDA is also a key partner with the WHO in what we call
diagnostics and vaccine prequalification programs. We help them
evaluate products for global use, which is particularly
important in collaborative efforts to build regulatory capacity
in other parts of the world. We work, for example, at something
called the African Vaccine Regulatory Forum. The idea here is
to help our colleagues in regulatory agencies around the world
and potentially get access to countries that need products
sooner.
It is also important to make sure, though, that for the
products we have now that people have access to them and that
they are safe. And there is a big problem with the substandard
and counterfeit medicines that are common in many parts of the
world. For example, malaria kills more than 600,000 people
globally every year, mostly children, but compromised or fake
antimalarial medicines have been found to make up 10-50 percent
of the drug supply in a number of countries. Such medicines
will not cure patients. Sometimes their use results in death or
serious injury, and they can lead to resistant strains.
This April, FDA announced a unique public-private
partnership to identify counterfeit or substandard antimalarial
medications with the development and testing of a device
developed in our laboratories called CD-3, or the Counterfeit
Detection Device. This partnership includes the Skoll Global
Threats Fund, the United States Pharmacopeia, our colleagues at
NIH and CDC, the President's Malaria Initiative, as well as the
Corning company.
To further promote development or treatments for rare and
neglected diseases, we also strive to bridge the gap between
basic scientific research and getting to products that people
can use. This gap, in fact, can be filled through enhanced
regulatory science, for example, the development of those
surrogate endpoints I mentioned that can speed product
development, or through disease models. One example, just to
mention one of many, is a new model developed at FDA to develop
drugs and vaccines against leishmaniasis, which infects 12
million people around the world.
Another remarkable accomplishment is the development of a
new vaccine to protect people against serogroup A meningitis,
which causes devastating epidemics in Africa. Working through a
unique public-private partnership, the Meningitis Vaccine
Project, FDA scientists developed and made available an
innovation that allowed a safe and effective meningitis vaccine
to be manufactured efficiently at greatly reduced cost. As a
result, over 100 million people in Africa have now been
vaccinated, and that disease has been beaten back dramatically.
While much remains to be done, recent FDA approvals
highlight the success and promise of these kinds of proactive
approaches. For example, Sirturo, or bedaquiline, was granted
accelerated approval as an orphan drug just this last December
to be used as part of combination therapy for multidrug-
resistant pulmonary tuberculosis when there are no other
treatment options. Similarly, FDA used expedited approaches to
approve Kalydeco to treat a rare form of cystic fibrosis. This
drug was approved within 3 months, a near record time.
So, in conclusion, thank you for this opportunity to
testify about our work with others in combating rare and
neglected diseases. Your engagement and support, including the
strong science at FDA, has been and will remain important in
helping to solve problems in developing these needed products.
I am frankly optimistic that, with so many people working
together so well, we will continue to see progress.
I look forward to working with you and welcome your
questions.
Mr. Smith. Dr. Goodman, thank you so very much.
[The prepared statement of Dr. Goodman follows:]
----------
Mr. Smith. And Dr. Bera, I know, has to leave.
But thank you so much for both of you for your full
statements, which will be made a part of the record. And I
think it will provide some very useful guidance for this
committee. And we look forward to collaborating with you going
forward on how we might be helpful as a subcommittee on these
issues.
Just a couple of questions. Again, I will submit many more,
but because this committee was in recess for almost an hour, I
won't keep you much longer.
But you mentioned the counterfeiting issue. We have heard a
number of reports that artemisinin, that there are concerns
that its efficacy, particularly in some parts of Southeast
Asia, is becoming questionable because of wrongful use but also
maybe because of some of the bogus medicines that are being
proffered out there.
How big of a counterfeiting problem is it? Who are making
the counterfeit drugs? Is it coming out of China? Is it coming
from people in other countries, from the U.S.? Where are these
drugs coming from?
Dr. Goodman. Well, you know, I would like to get back to
you with more detailed data on issues like what we found in
terms of sources of counterfeit drugs. It may, in fact, not
always be apparent, but what I can tell you is it is a big
problem.
As I mentioned, most of the studies were in Africa, but
there are studies also in Southeast Asia that show substantial
percentages of counterfeit or what are often substandard
antimalarial medicines. They may contain lower amounts; they
may contain other contaminants that are potentially harmful to
health. Sometimes they are an antimalarial medicine but they
are the wrong one, so that they may not work in an infection
that would be resistant to the medicine the doctor doesn't know
they are actually administering.
So this is a big problem. And our device is one approach.
It can be used, you know, remotely in the field; it is a
handheld device. It appears to be pretty effective, although it
is undergoing field testing now.
You know, in addition, FDA has recently been working with
INTERPOL and others to really crack down on Internet sales
through fake Internet pharmacies and outfits. I suspect some of
those fuel a lot of the bad product that is out there.
But we are really gratified that people have recognized the
importance of this as a public health problem. As an infectious
disease person, I will say misuse of antibiotics or use of
antibiotics that are substandard or subpotent is very
concerning, both because it hurts patients and it does
contribute to this problem of increasing drug resistance that
we are seeing.
Mr. Smith. You note in your testimony the--or Dr. Hotez, I
should say, notes in his testimony that, when you talk about
stunting, that tropical diseases is also a major contributor to
stunting.
And I know, you know, Congress and the administration is
working very hard, as is the world, on that first 1,000 days,
making sure that from conception to about the second year,
children get the right kind of nutritional aid.
But how do you see tropical diseases as contributing to the
stunting problem that we have seen all around the world,
particularly in places like Africa and North Korea?
Dr. Hall?
Dr. Hall. Well, thank you. That is a very interesting
question but a rather complex one.
I think the answer is that these tropical infectious
diseases in many cases contribute to malnutrition in a variety
of different ways. And, obviously, relieving or treating the
infectious diseases or preventing them in the first place would
be a great way to try to avoid this problem altogether. And
that is really one of the things that we would want to focus on
with the development of new interventions and other approaches
to try to minimize the impact of these diseases.
Dr. Goodman. Dr. Hotez could probably shed even more light
on it, but we know simple things like, you know, even improving
anemia, which is common in young children who have parasites,
can improve their performance in school and their learning.
There is both very obvious malnutrition and infection that
occurs, but there are also much more subtle effects, you know,
where, if these were our children, we would be deeply, deeply
concerned, and we should be concerned more generally.
Mr. Smith. You cite, Dr. Hall, in your testimony that the
U.S. is the largest public funder of neglected tropical disease
research. Who would be second? Third?
And what are we doing to try to--I mean, you mentioned, Dr.
Goodman, you know, the importance of global collaboration. I
will, for example, be in Istanbul on Saturday working as co-
chairman of the Helsinki Commission with 300 parliamentarians
from 57 countries. And while we usually work on issues related
to human rights and democracy, rule of law, I have brought the
autism issue there, have brought some other issues, you know,
where we need to be working across--well, that is lawmakers;
you obviously work with the experts.
Who are your best collaborators? And how can we be helpful,
Members in the House and Senate, in promoting that?
Dr. Hall. So, again, it is very important, because of the
multidimensional nature of this problem, to engage multiple
stakeholders.
With respect to the actual rank ordering of who is first,
second, and third, I would have to get back to you. I can tell
you that, certainly, the U.S. Government is the largest funder
for neglected tropical diseases. Certainly, other foundations,
such as the Gates Foundation, have had a major role to play.
And, certainly, USAID and other groups have also contributed in
this area, as well as overseas agencies.
Mr. Smith. It would be helpful if you could get back to us
with that.
Dr. Zwane from Evidence Action points out that there are
some 400 million children at risk, schoolchildren, without
treatment for intestinal worms. How has that improved over the
last--I mean, how do we reach those? I know the Carter Center
works on, you know, certain parasites. Are we doing enough to
rid the world of parasites and especially intestinal worms?
Dr. Goodman. Well, you know, I think they have, from
reading the testimony, some very innovative programs and are
delivering medicine and hopefully having a real impact on that.
You know, I think one of the things we are seeing
increasingly in making a difference in global public health is
also integrating these different efforts across multiple
diseases. You know, it really becomes about people getting the
care they need. I know WHO and others and Gates are really
focusing on, you know, how do we deliver these interventions,
of course, and how do you keep people from getting reinfected
with some of these pathogens. So I think you need a holistic
approach, but, as some of these efforts point out, you can make
a tremendous amount of difference often with inexpensive
medicines and interventions.
Mr. Smith. Let me just ask a few final questions.
Are there gaps with USAID funding, funding that Congress
has provided? You know, I know that OMB goes through
everything, goes through your agencies to scrub and make final
recommendations. But if you had a wish list, how much more do
you think would need to be added in order to really even push
the envelope even further?
And I would say parenthetically, 20 years ago I contacted
WHO and asked their tropical disease side, what would it take,
and actually got an amendment passed that tried to get us on a
glide slope to meeting some of those figures.
What would it take? I mean, you know, we can work in a
bipartisan way. I believe Dr. Bera and I have talked, you know,
previously about filling in those gaps. How do we do it?
Dr. Goodman. Well, you know, I think for USAID and some----
Mr. Smith. That is not to take anything away from the great
work you are doing. Please don't----
Dr. Goodman. Right, right.
Mr. Smith. I underscore that with exclamation points. It is
a resourcing problem.
Dr. Goodman. You know, I was going to say, I think for the
agencies, you know, delivering some of these medicines and
care, they would be the best to answer, you know, how they feel
they are doing. You know, I know the whole Federal budget is
tight, and we all have a lot of obligations and
responsibilities.
From the FDA point of view, there are certainly areas that,
were there resources available, we could potentially build out.
You know, those would include enriching our continuing
collaborations with regulatory agencies around the world. It is
sort of like, if you help people do something, then they can
take that on themselves.
I think also in this applied science space, where we could
develop better ways to develop and test these drugs more
efficiently and work very interactively with industry, you
know, those are things that are, to some extent, resource-
dependent.
I will say we feel this is very important. And a lot of our
staff work on these issues, you know, because it means a lot to
them, even if they may not quite have the bandwidth.
Dr. Hall. So, again, this is a very important issue. And I
think that having hearings like this that highlight this issue
are important, because it is really crucial to try to engage
multiple stakeholders because the solution is not going to come
necessarily from one group or one agency. It is going to be a
very collaborative process, working with a wide variety of
groups. And there are many different aspects to this problem.
So it is very difficult to say in a short statement what
would be required. I think that this is something that would
require the engagement of many groups, and it would require a
considerable amount of thought.
Mr. Smith. If you could give it some thought.
Again, I will give you the example of the way we do
business. When I was chairman of the Veterans' Affairs
Committee, I became very, very aware that homeless veterans
were not getting the kind of help they need, and it was a
resourcing problem. I met with the VSOs, they gave some great
ideas--American Legion, all the others.
But I sat down with the people at the VA who really do the
homeless veterans programs, and I said, what works? Tell us
what we can do. And I wrote a major landmark law called the
Homeless Veterans Assistance Act of 2001, which, while it
hasn't solved the problem, it has certainly made a major dent
in it.
We would love to be helpful, you know, on this subcommittee
if we just had a better roadmap as to how to proceed. So
consider this an engraved invitation. I can't guarantee
success, but we can build on your already very considerable
success.
Dr. Goodman. I am sure, you know, we would be eager to work
with you or comment on any ideas that you develop. You know,
there are some roadmaps out there that may be of interest to
you, like the Global Vaccine Action Plan we discussed in that
area.
Mr. Smith. Thank you.
One last question. You know, you mentioned new tools and
incentives, and I think that is what we want to build on, as
well.
You know, we have been admonished by the experts for some
time now, you, of how important it is not to overuse antibiotic
treatment, how resistance is growing. We have had hearings in
our subcommittee on the tuberculosis extreme as well as
multidrug-resistant.
Is there anything you might say about probiotic use and the
lack of probiotics in people's gut, where the immunity is so
important? Are we emphasizing that enough?
Dr. Goodman. Well, there has been incredibly exciting
science in the last 5 years, I would say, particularly, as we
have been able to now just take the contents of any environment
or any part of the human and we find this entire world of
microbes there. So we have realized that we are not at war with
microbes. Some of the ones you discuss, you know, clearly they
infect a person, it is a bad situation. But much of our health
seems to depend on a normal balance of microbes.
For example, recently this has affected treatment of an
antibiotic-associated diarrhea called clostridium difficile,
which is a complication of antibiotic treatment. And it turns
out that restoring the normal bacteria to the intestines can
help in the majority of cases of severe disease.
So what we are learning is, many things humans do,
including antibiotic treatment, which may have a very good
purpose, may have other effects that are more complex. And so
we are just getting the tools to study this. I think it is an
area that we are going to find, as we understand better how to
restore or nurture our normal microbiome, we may find
innovations that really help in treatment of disease.
I think a lot of the reports and, to some degree, you know,
products that are out there are not as well-substantiated yet,
so we don't know yet what the role is. But I would be surprised
if in some diseases, just like antibiotic-associated diarrhea,
there isn't going to be a really important role for that.
Dr. Hall. Yes. And I would just add to what Dr. Goodman
said, that we do have this large program at the NIH, the Human
Microbiome Project, that is providing incredible insights into
how the microbiome works and that, as we move forward with
this, this will hopefully open up many new opportunities to not
only better understand what is going on but to develop novel
interventions that would allow us to better intervene in a
number of these diseases and in some cases to do things--for
example, convert vaccines that are delivered intramuscularly to
perhaps an oral formulation, things like that--as we understand
better how to capitalize on this new understanding.
Mr. Smith. Thank you so much.
Mr. Weber?
Mr. Weber. Thank you, Mr. Chairman. You actually asked one
of the questions I was going to ask.
I think it is interesting--I will kind of back into my
questioning, if I may. Dr. Goodman, you said the last 5 years
we have discovered an entire world of microbes, I think you
said. I guess it is kind of like what science fiction used to
say, we are not alone in this world, and you all are
discovering that.
You made a couple of statements early on, Dr. Hall. You
said--or let me ask you a question from your testimony,
actually. You talked about the rise of diseases. Is there any
way to track or do we have guesstimates of how much of that
disease is coming into our country through the immigration
road? Is there any thought or discussion given to that?
Dr. Hall. That is something that I don't really feel I
could comment on. It is really something that perhaps the CDC
would be in a better position to comment on.
Mr. Weber. Okay. Are you aware, do they track that in any
form, do you know?
Dr. Hall. They do track--I don't know the extent to which
they track this in particular. But they do have an interest in
a variety of diseases, and they are tracking a variety of
different ones.
Mr. Weber. Okay.
Do you have knowledge of that, Dr. Goodman?
Dr. Goodman. For certain diseases, you know, which don't
normally occur here, there is very good reporting--you know,
for example, malaria. Typically, it is acquired in another
country, and then a traveller comes here not knowing he has it
and develops illness. For other diseases, you know, there is a
focus on the health of people who may have moved here. And,
certainly, when people come from other countries, they may have
some of these infections.
Also, as Dr. Hotez may tell you, many of these infections
can be acquired here, as I mentioned, even with dengue now. It
is not really communicable from person to person, but now there
are mosquitos in our southern climates carrying these diseases
so that the diseases are coming to us, not even necessarily
through people.
Mr. Weber. Okay. And you mentioned that the dengue and the
Chagas disease, I think is what you call it--I am a blood
donor. I try to be. Like the Congress, I can't do it every 8
weeks for some reason. But I have AB-blood, which less than \1/
2\ of 1 percent of the American population has. Now you know
what is wrong with me.
Dr. Goodman. That is what is right.
Mr. Weber. You made the comment that dengue and Chagas
disease is spreading to blood safety?
Dr. Goodman. Well, there is a potential. You know,
actually, there has been very little documented spread for
Chagas----
Mr. Weber. Okay.
Dr. Goodman [continuing]. And virtually none for dengue.
But we are aware that people--dengue, in certain ways, is like
West Nile virus was, and so we want to be prepared. You know,
so there may be a period where the virus is in the blood and
could be transmitted, so we want to be prepared if that becomes
a problem.
With respect to Chagas disease, people can be infected for
years without being aware of it, and that is the reason for
that concern.
But right now I don't think there is an active problem with
the blood supply.
Mr. Weber. Well, I get asked that that question every time
I give blood, you know, have I ever been diagnosed with this
and----
Dr. Goodman. That is why. So it is really in this stage of
trying to be vigilant, do surveillance, and prevent there from
being problems, develop tests in case they are needed.
Mr. Weber. Okay. And then you also said the Orphan Drug
Act--and, as a new Member of Congress, I am not really familiar
with that--you said that neglected diseases almost always
qualify. Elaborate on that.
Dr. Goodman. Well, the major cutoff point for the Orphan
Drug Act, as I understand it, is that, to be eligible, a
disease has to affect less than 200,000 people in the United
States.
So when you think about things like Chagas disease or,
certainly, rabies or many of the things on that list, while
they may be extremely common if you were in Asia or Africa,
they are rare here. So a sponsor developing a drug for one of
those neglected diseases would usually be able to take
advantage of the benefits of the Organ Drug Act, which include,
you know, grants for research, as I mentioned, additional
exclusivity, which can be financially rewarding to companies.
Mr. Weber. Right. And so if it is less than, say, 200,000--
that was one of the questions I had.
How often--you track the number of infections to the best
of our ability.
Dr. Goodman. Yeah.
Mr. Weber. How often are those numbers updated? Is it every
month? Every 6 months? Every quarter?
Dr. Goodman. It is a question that depends, to some degree,
on the resources available to track them and how they are
viewed in terms of as a public health threat in the United
States. CDC has overall responsibility for that, and, you know,
I am sure we could ask them to get back to you with some of
that information.
But our country does have probably one of the strongest
infectious disease surveillance systems in the world, and it
really helps us know. But there is a balance between requiring
reporting and not being too burdensome. So, for some diseases,
we probably have extremely good idea of numbers, like
tuberculosis. For others, we may have less certainty,
particularly if they often have no symptoms, like Chagas
disease, for example.
Mr. Weber. Let's take that question and let's extrapolate
it overseas to follow up on kind of what the chairman was
saying, who is number two, who is number three. The United
States has a great track record, and you mentioned the Gates
Foundation helping, and you mentioned some others, USAID I
think.
But when we are tracking in other countries and we see an
outbreak, how often are those numbers updated?
Dr. Goodman. Well, I think a very positive thing that, you
know, I have seen even in my time in the government and in the
last 10 years as we faced various influenza threats, for
example, is the world has become much more aware of the
importance of surveillance of infectious disease, there is much
more international collaboration----
Mr. Weber. Okay. Is there an FDA counterpart in China or
Japan or Russia?
Dr. Goodman. Well, in this case, it would be more, you
know, the CDC counterpart for disease surveillance. But, yes,
there are in many of these countries. And----
Mr. Weber. They communicate back and forth?
Dr. Goodman. Yeah. In many cases, there are scientific
exchanges between our governments and theirs. There is also
something called the Global Health Security Initiative, which
involves some of the U.S.'s major allies, that shares
information on these diseases.
So there is much, much more than there used to be. But
there also is a concern, as you can imagine, in many countries
of the world that are very resource-poor; they may not have
either the resources or, in many cases, the expertise to devote
to disease surveillance. So there is a lot of recognition of
the importance of trying to, wherever we can, improve
surveillance in those areas too.
Mr. Weber. And that was one of my questions. Would you
agree that developing countries that maybe don't have an
infrastructure, maybe not as much clean water, maybe not as
good health care, that part of helping to stabilize them, if
you will, in some fashion, is going to be training and
equipping them even at that basic level?
Dr. Goodman. Absolutely. Absolutely. You know, knowing what
is going on--even in the United States, if you think about
health of our communities, if you don't understand fully and
you don't have good data about the diseases people have, you
know, how do you track changes, how do you make people
healthier?
So that disease surveillance is very important all over the
world, and the same with the other kinds of expertise. You
know, there are many countries throughout the world that have
made tremendous progress, and there are a lot of exciting
models for how to improve things in those areas.
Mr. Weber. Well, I am just wondering how much of our
focus--it is kind of like the old saying, you give a man a
fish, you feed him for a day, you teach him to fish--we can
give them vaccines, but can we help with their infrastructure
and their training so that the underlying--I won't say
problem--the underlying situations that keeps them from being
maybe as healthy and as knowledgeable as they could--might
help.
Let me shift gears for just a minute. You made the comment
you are trying to build regulatory capacity around the world
and you are trying to get, I think, access to the products
sooner. What is that time frame, when a product is begun and
studied and put out? Is that typically 3 months? Three years?
Dr. Goodman. You mean for other countries?
Mr. Weber. Well, for us to get a product--well, your
comment was you were trying to build regulatory capacity around
the world and get access to the products sooner.
Dr. Goodman. Well, so what I am thinking about, for
example, is, what if there is a lifesaving intervention that
could help people, you know, who are primarily in other places?
You know, there are many factors in having access to that.
There is cost; there is their ability to deliver it.
But one of them also is that, you know, really, FDA or the
European medicines agencies, you know, which are relatively
well-resourced and experienced, you know, in reviewing
products, we don't approve products for use in other countries,
rightly so. You know, countries have their autonomy and need to
be sure that their assessment of a product for use in their
country is science-based and satisfies their needs.
So one of the things we have tried to do through WHO is
support both countries and regions who are trying to develop
that regulatory capacity.
Mr. Weber. Do you find generally that most of those
countries--how do you say it--they rely on us, rely on the
FDA's assessment? Or do they pretty much want to do their own
testing?
Dr. Goodman. You know, as you can imagine, there is
incredible diversity. A lot of countries look to FDA and also
to our European colleagues, you know, as having expertise and
really may not rely on but use our reviews as part of the basis
for their decisions.
But many of them are coming together also in regions, for
example, in Africa and in the pan-American region, to build
their own regulatory capacity and to cooperate with us, you
know, almost as partners and equals. We have had situations
where we can learn from work they are doing--for example, in
Latin America, the monitoring of the 2009 influenza outbreak.
Studies of vaccine safety for other vaccines have been
performed there.
So it is really a win-win solution when we build that
capacity. You know, of course, people have to want that and
invite us, and we have to provide it in a way that is useful to
them, which may be different than what we do.
Mr. Weber. Right.
Okay. That is all my questions. Thank you, Mr. Chairman.
Mr. Smith. Thank you very much.
Just one final question. And, again, thank you for your
patience.
Dr. Peter Hotez, as you know, the president of the Sabin
Vaccine Institute, in his testimony will say that NTDs are the
most important diseases you have never heard of. He talks about
how well over 1 billion people suffer from it.
But he has a very important part in his testimony where he
talks about a problem that is coming to light with regards to
female genital schistosomiasis that malaffects some 100 million
girls and women in sub-Saharan Africa, and makes a point that
one thing very concretely that we might do--you might do, we
might do collectively--is to establish a center of excellence
for NTDs, that the time has come for such a center.
Again, I would note parenthetically, back in 1998, I am the
author of the Combating Autism Act. It took me 3 years to get
it passed. We put it as Title I of the Children's Health Act in
2000. And then, just most recently, I was the author of the
reauthorization for 3 years.
And there was pushback initially from some within the
agencies about a center of excellence for NIH and for CDC. I
noted at the time that we were spending $287,000 per year
straight line for 5 years at CDC on autism, even though there
was a huge seeming epidemic, developmental epidemic, arising.
And I am wondering if perhaps it might be time for a center
of excellence for NTDs. What are your thought?
Dr. Hall?
Dr. Hall. Well, again, I think that product development is
a very complex area. It requires a multidisciplinary approach.
And it is really important to bring together groups that have a
variety of different expertise. And that is one of the things
that really makes for successful product development.
At the NIH, we have tried a variety of different methods,
and what we have found works is that you have to have a
spectrum of activities that run from basic research through to
preclinical target validation, preclinical research,
translational research, and then on to clinical and field
evaluation, and that when these parts are working together
well, then you really are able to accelerate the development of
products.
Our current mechanisms are actually quite flexible,
responsive, and timely. And just to give you an example, about
a third of the global antimalarial drug pipeline came out of
NIH-supported research and has been handed off to partners. We
have supported multiple products for NTDs, including vaccines,
drugs, and diagnostics. And we worked with a wide variety of
different technologies, different companies, and different
entities to try to accelerate that.
And just to build on this question about screening donated
blood that came up earlier, in fact there are at least two
blood tests for screening the blood bank, the blood supply, and
at least one of those came out of NIAID-supported research.
So our feeling is that a strong multidisciplinary approach
with a variety of mechanisms and technologies is really very
helpful in terms of bringing these things forward.
Mr. Smith. Thank you. I do have a lot of questions but, in
the interest of time, will submit them.
Thank you for your tremendous leadership, and we look
forward to working with you.
Dr. Hall. Thank you.
Dr. Goodman. Thank you very much.
Mr. Smith. Now we would like to invite our second panel to
the witness table, beginning with Dr. Peter Hotez, who is the
president of the Sabin Vaccine Institute and leads the Texas
Children's Hospital Center for Vaccine Development based at the
Baylor College of Medicine in Houston, Texas. He is also the
founding dean of the new National School of Tropical Medicine
at Baylor College of Medicine.
His academic research focuses on vaccine development for a
wide range of neglected tropical diseases around the globe as
well as studies to increase awareness about the neglected
tropical diseases in developing countries and in the United
States. Dr. Hotez created the Sabin Vaccine Institute Product
Development Partnership and was instrumental in creating the
Global Network for Neglected Tropical Diseases.
We will then hear from Dr. Jay Siegel, who is the chief
biotechnology officer and head of scientific policy at Johnson
& Johnson. He is actively engaged in R&D leadership and in
policy development at the national as well as international
levels with regard to regulatory and scientific issues.
Dr. Siegel joined J&J in 2003, and prior to that he worked
at the FDA's Center for Biologics Evaluation and Research,
where he worked to regulate the biotechnology industry. He has
authored numerous publications in the area of clinical trials,
design, biotechnology, immunology, and drug development policy.
J&J submitted written testimony on April 20th at one of our
hearings back on drug-resistant disease, and we are very
grateful for that but also even more to have you here today.
We will then hear from Dr. Alix Zwane, who is executive
director of Evidence Action, a new organization working to
scale proven interventions to improve the lives of the poor in
Africa and Asia, and the Deworm the World Initiative, which
supports the scaleup of school-based deworming programs
worldwide to improve children's health, education, and long-
term development.
She previously worked for the Bill and Melinda Gates
Foundation, where she led the Water, Sanitation, and Hygiene
Initiative strategic planning process and their measurement and
evaluation plan. Dr. Zwane worked at Google.org to develop and
manage health and water issues, as well.
Dr. Hotez?
STATEMENT OF PETER J. HOTEZ, M.D., PH.D., PRESIDENT, SABIN
VACCINE INSTITUTE
Dr. Hotez. Thank you very much, Chairman Smith. I
appreciate the opportunity. Congressman Weber, thank you so
much. It is a pleasure to come before you today to discuss the
importance of U.S. Investments in neglected tropical diseases.
But what I also want to do today is make you aware of a very
troubling and disturbing problem of neglected tropical diseases
in Texas and in the southern United States that we have not
been aggressively addressing.
A copy of my written testimony has been submitted for the
record.
As you pointed out, I serve as president of the Sabin
Vaccine Institute, which has just celebrated its 20th
anniversary, and also founding dean of the new National School
of Tropical Medicine at Baylor College of Medicine in Houston
which was launched in 2011. I am a pediatrician and a scientist
who has devoted my entire life to developing innovations to
combat neglected tropical diseases (NTDs) including new
vaccines and drug packages.
You are absolutely right; these are the most important
diseases you have never heard of. Today, virtually every person
on our planet who lives in extreme poverty, lives below the
World Bank poverty figure of $1.25 a day, the bottom billion,
as they are sometimes called, and suffers from one or more of
those neglected tropical diseases.
A couple of people brought up the problem of intestinal
worms, such as hookworms, which feed on blood and actually rob
children of nutrients. The problem with stunting is that
instead of feeding the kid, you are feeding the worms. These
are the leading causes of growth stunting on our planet.
What is even worse is intestinal worms like hookworms have
been shown to reduce childhood intelligence and cognition. And
now the economists have come in from the University of Chicago
to show that chronic hookworm infection can reduce future wage
earning by 40 percent.
So this is why these diseases are so devastating: Not
necessarily because they are killers; they are trapping people
in poverty. They actually thwart future wage earnings. So
getting rid of hookworms, it turns out, may be one of the most
cost-effective ways to lift the bottom billion out of poverty.
And it is not just children. More than a quarter of
pregnant women in sub-Saharan Africa have hookworms and go into
labor and delivery with profound anemia and risk for severe
morbidity or even death. As my obstetrician colleagues point
out, it is not that African women bleed more during childbirth,
but it is that they begin the delivery process with two strikes
against them because they start out with so little blood
because they have hookworms. These are not rare diseases. One-
quarter to one-third of pregnant African women have this
condition.
But an even more or equally concerning problem that I also
want to tell you about in Africa today before I move to the
U.S. is what is coming to light is a manifestation of snail
fever known as female genital schistosomiasis, a parasitic
infection that produces bleeding ulcers on the cervix, the
uterus, the lower genital tract. It causes bleeding, pain,
terrible shame, terrible stigma.
This is not a rare disease--it impacts 100 million girls
and women and this may be the most common gynecologic condition
of girls and women on the African continent. Now, can you
imagine if we had 100 million girls and women in the U.S. With
female genital schistosomiasis? As a society, we would never
tolerate it. But because it affects girls and women who live in
abject poverty, most often in remote parts of rural Africa,
they go untreated.
Now, what is very equally concerning is the fact that this
female genital schistosomiasis is associated with a three- to
four-fold increase in horizontal transmission of HIV/AIDS. It
may be Africa's most important co-factor in the AIDS epidemic,
and you have never heard of it. So we need to change that.
Just a couple of other final examples to wrap up the global
situation is the stigmatizing effects of cutaneous
leishmaniasis, also known as Aleppo ulcer or Aleppo evil. It is
now affecting more than 100,000 people living in Syria who have
fled to refugee camps. It has also been a problem among our
U.S. troops in Iraq and Afghanistan. But in the Middle East,
again, it is girls and women who are permanently scarred and
rendered unmarriageable or not allowed to hold their children
as a result of this neglected tropical disease.
And the point I keep wanting to emphasize is the magnitude.
Everything I say is multiplied times 100,000 or, in some cases,
100 million--it is just an enormous amount of suffering.
So every year we are learning about these new tragedies
from neglected tropical diseases, many of which are
counterintuitive. Our recent study found that these diseases,
for instance, hit Catholic-majority countries particularly
hard, such as Chagas disease in Honduras or hookworm and
schistosomiasis in Angola and the Philippines. It is no
accident.
You know, these diseases are--you all know about emerging
infections. This is what gets the newspapers' attention, right?
Avian flu or H7N9. These diseases are just the opposite. These
have been around forever. These have plagued humankind for
centuries. I like to call them the biblical diseases, because
you can find detailed descriptions of these diseases in the
Bible, and also in the Talmud, the Vedas, the writings of
Hippocrates, and Egyptian papyri. That is how long they have
been around.
Now, the good news--and there is some good news to this
story--is that we can do something about seven of the most
common neglected tropical diseases for a ridiculously low cost,
approximately 50 cents per person per year.
So what happened was, in 2005 and 2006, with colleagues
from the United Kingdom, we put forward this concept of a rapid
impact package in 2006, which is now being scaled up through
the support of the United States Agency for International
Development, USAID, and with advocacy from our Global Network
for Neglected Tropical Diseases, which is a Sabin initiative.
And thanks to generous drug donations from leading
pharmaceutical companies like Johnson & Johnson, a unique and
innovative public-private partnership has been created to
efficiently and cost-effectively address neglected tropical
disease control.
And the cost is, again, ridiculously modest, partly because
we are leveraging more than $4 billion worth of drugs that have
been donated from the pharmaceutical industry. And more than
250 million people have been treated. This may be one of the
world's largest public health interventions.
So, I don't want to leave today without saying that it is
extremely important that you continue support for USAID's
Neglected Tropical Disease Program. It is very low-cost. It is
important that we at least match 2013 levels to deliver these
medicines to people who need them the most so they can benefit
from these treatments.
While these diseases are incredibly potent, the U.S.
Government has actually been the lead in supporting these
neglected tropical disease packages; the U.K. has now also
provided support. We also think the private sector needs to
step up, as well as some of the other G-20 countries. We
established the Global Network for Neglected Tropical Diseases
and our END7 campaign to raise awareness about these diseases.
And, if I may, I would like to show a 1-minute video about
these conditions.
[Video shown.]
Dr. Hotez. This is lymphatic filariasis, or elephantiasis.
That is 120 million people with lymphatic filariasis, 800
million with roundworm, 700 million people with hookworm.
Notice the numbers add up to more than 1 billion. People are
polyparasitized. They have multiple diseases at the same time.
Now, given the recently revealed links between snail fever
and female genital schistosomiasis and AIDS, as well as malaria
and hookworm. You've asked, where do we go from here? Well, I
think version 2.0 includes looking at better links between the
USAID Neglected Tropical Disease Program and other U.S. global
health investments, such as PEPFAR or the Global Fund. So, for
instance, PEPFAR doesn't actively have a program to link it up
with female genital schistosomiasis, even though it may be the
most important co-factor in the AIDS epidemic. So we need to do
a better job linking.
But all of them are extraordinary programs. The Global Fund
to Fight AIDS, TB, and Malaria needs to do the same thing. And
the few times they have done it, the results have been
extraordinary. Because Global Fund supported lymphatic
filariasis, one of those diseases in Togo, it has now been
eliminated in Togo. So there is incredible power that could be
brought to bear by bringing PEPFAR and the Global Fund into the
equation.
We also need to encourage governments beyond the U.S. I
think it is unfair that we put all of the burden on U.S.
taxpayers. Our country has been more generous than any other,
but we need to bring in some of the other G-20 countries. What
is Indonesia doing about this? What is India doing about this?
What is China doing about this? China is investing billions of
dollars in sub-Saharan Africa, and, so far, not a penny is
going to neglected tropical diseases.
So action item number two is we have an exciting new Office
of Global Health Diplomacy in the State Department. What is
going to be the role for them? Well, I think one of them would
be putting diplomatic pressure on all the G-20 countries to
step up support for NTD control and elimination.
So we are finding a lot of these NTDs among G-20 countries.
One of the surprising findings that we have made in the last 2
years is that, while we think of these diseases exclusively as
sub-Saharan African diseases, in fact, the lion's share of most
neglected tropical diseases are in the G-20 countries, the 20
wealthiest economies. It is the extreme poor living in the
wealthiest economies.
And that includes the United States. We have 20 million
people now in the U.S. that live in what is called extreme
poverty; that is a standard deviation below the poverty line.
It also includes now--and this is an amazing fact--1.46 million
families, with nearly 3 million children, who live on less than
$2 a day. So, for the first time to my knowledge, we are taking
that same global benchmark for global poverty and applying it
to the United States, especially in the southern United States.
At our National School of Tropical Medicine based in
Houston, what we have done now is to take that global health
lens and turn it inward. Beginning in 2008, I identified a
group of neglected tropical diseases in the U.S., mostly in the
southern U.S. and particularly in Texas, that is affecting 5
million Americans.
So 5 million Americans have one or more neglected tropical
diseases. It includes 2.8 million African-Americans with
toxocariasis, a larval worm infection of the lungs and the
brain that has been now linked to pulmonary dysfunction and
asthma, developmental delays, and seizures; millions of
African-American women with trichomoniasis that has been linked
with HIV/AIDS; hundreds of thousand of Hispanics with Chagas
disease, a debilitating heart condition.
To get to your question, Congressman, we have found now in
Texas that 1 in 3,500 blood donors are seropositive for
trypanosoma cruzi, or have Chagas disease.
Now, for the most part, extremely poor people and people
who live under extreme conditions don't give blood. So this is
an extreme underestimate of the number of true cases of Chagas
disease. Some estimates say 200,000 people with Chagas disease
are in the State of Texas. That is probably a little high. We
are finding it now possibly among the homeless in Houston. We
are finding it widespread.
Another key point to mention is, one, they are not rare
diseases; two, everyone thinks this is a problem of
immigration. Immigration may be a part of it, but we believe
there is actual transmission of these diseases going on in the
U.S.
The link is poverty. There is something about people living
in extreme poverty, whether it is in the Third or Fifth Ward of
Houston, whether it is in south Texas. This is what seems to be
predisposing it.
We have a dengue problem in Texas. We know it is widespread
in south Texas. We believe now that it might have emerged in
Houston, as well. We have had the Aedes aegypti mosquito here
for a long time. It is occurring primarily in the poorest parts
of Houston.
Why the link to poverty? Why is dengue linked to poverty?
We don't really know. You know, when you drive through some of
these areas, you see people without window screens, without air
conditioning, or they will have those box-like air conditioners
that are very porous. Maybe that has something to do with it.
Our scientists at the National School of Tropical Medicine
have identified a new syndrome linked with West Nile virus.
Everyone knows about the very rare condition of neurologic
involvement. We are finding maybe 10, 20 percent of people with
West Nile are getting chronic renal disease, something that we
didn't know about before.
So this is a problem that we really need to take on. What
do we do about it? We are not doing programs of active
surveillance looking for these diseases. These problems are
being largely ignored. We are not really doing anything to
investigate how these diseases are transmitted. Partly because
the CDC has been terribly underfunded, they haven't had the
resources to really take this on. So we are trying to fill in
the gaps and are doing this at the National School of Tropical
Medicine.
And, this gets to this whole idea of creating a ``Center of
Excellence.'' I think the Center of Excellence concept has to
take on neglected tropical diseases abroad among the bottom
billion, taking on the biblical diseases, but we also need to
have that ``Center of Excellence,'' address the at least 5
million Americans living in extreme poverty in the United
States with these NTDs.
A fair amount of effort is going to be needed for research
and development. That is one of the things that we are doing at
our Sabin Vaccine Institute, which is a nonprofit product
development partnership, a PDP, that will make the products
that the drug companies can't make or won't make. Not that the
drug companies are bad guys. They have been amazing, incredibly
generous in donating medicines. But it is another leap of faith
to ask them to go back to their shareholders and ask them to
invest tens of millions of dollars for R&D.
That is what we are doing at Sabin Vaccine Institute and
our National School of Tropical Medicine. We have a low-cost
hookworm vaccine that is in phase one trials; a schistosomiasis
vaccine that is moving into phase one trials through support
from Dr. Lee Hall, through the National Institute of Allergy
and Infectious Diseases.
And now we are developing new vaccines for Chagas disease
and leishmaniasis through support from the Carlos Slim Health
Institute and a man named Len Benckenstein, through the
Southwest Electric Energy Medical Research Institute. So we are
very excited about being able to advance these products into
clinical development.
So we do urge you to vigorously support private-public
partnerships, product development partnerships, that will
develop not only the developing world but will ultimately lift
people out of poverty.
Mr. Chairman, Congressman Weber, Congressman Stockman, this
concludes my testimony. Again, I thank you for your interest in
furthering U.S. engagement in neglected diseases and the
opportunity to address you this afternoon. And, of course, I am
always happy to answer questions. Thank you.
[The prepared statement of Dr. Hotez follows:]
----------
Mr. Smith. We have been joined by Congressman Stockman.
Mr. Stockman. I have a question for Dr. Hotez.
Congress Weber and myself are both in the area in which
they spray for dengue fever. And I know in our neighborhood in
the evenings as the sun is setting they are spraying. I think
it is for dengue fever.
Dr. Hotez. Actually, you are spraying more for culex
mosquitos that transmit West Nile virus. So the spraying could
be extended to daytime spraying to get the Aedes aegypti
mosquitos, as well.
Mr. Stockman. So those are the ones that carry the dengue,
the daytime mosquitos?
Dr. Hotez. That is right.
Mr. Stockman. I understand, too, it is mostly the female
mosquitos, right?
Dr. Hotez. Yeah.
Mr. Stockman. Do you know Edward Rensimer? He is a travel
doctor in Houston. Do you know him too?
Dr. Hotez. I don't.
At our National School of Tropical Medicine now, we have
actually created what we think is the first tropical medicine
clinic in the United States at Baylor College of Medicine with
Ben Taub General Hospital--it is a partnership--where we now
see patients with all of these diseases coming in every Friday.
Mr. Stockman. So you are at the medical center?
Dr. Hotez. I am at the medical center, right, Baylor
College of Medicine at the Texas Medical Center.
Mr. Stockman. Yeah.
Dr. Hotez. So it is amazing, every Friday we have patients
coming in with Chagas disease, with cysticercosis, a brain
parasitic infection. And we are seeing amazing stories of
cutaneous leishmaniasis coming in. So it is pretty impressive.
Mr. Stockman. But there was a prominent scholar--and
correct me if I am wrong, but is some of this or a lot of it,
can it be delegated or attributed to immigration?
Dr. Hotez. So, I am not sure when you walked in. I think
immigration may be a part of it, but I don't think it is a
major part of it.
My point is it is linked with poverty, so that we have
transmission of these diseases right now going on within the
borders of Texas, more south Texas, in Houston. It is also
along the Gulf Coast. It is in Florida.
There is something about this pernicious combination of
extreme poverty and warm, moist climate, just like it is
elsewhere. I mean, after all, Houston is on the 30th parallel,
right?
Mr. Stockman. Yeah.
Dr. Hotez. It is on the same parallel as New Delhi and
Cairo and----
Mr. Stockman. And in reference to our own city, I think the
mosquitos bite anybody. I don't think they just bite poor
people.
Dr. Hotez. Yeah, so, as I said, we are trying to get our
arms around what is it about poverty that seems to make people
particularly susceptible.
Mr. Stockman. I mean, they are pretty smart mosquitos if
they know the difference.
Dr. Hotez. No, I will tell you what it is. Here is what I
think it is. I think it is, people living in extreme poverty
have more exposure because they don't have the adequate
housing, hence they are getting exposed more to mosquitos. We
are seeing very high rates of West Nile virus among the
homeless. One of our faculty members has found the same with
Chagas disease.
So I think--and we haven't proven it; this is only a
hypothesis--that the link between poverty and these diseases is
levels of exposure to the vectors.
Mr. Stockman. I was in DRC, and I was telling the chairman
about how I experienced firsthand some of the tropical
diseases. And their inability to deal with it is pretty sad,
actually. I think the basic stuff they didn't have in their--in
fact, it was supposed to be their advanced hospital. I was in
Kinshasa, and it was horrible.
Dr. Hotez. Yeah.
Mr. Stockman. It was absolutely horrible. So I appreciate
what you are doing and the research you are doing. It is really
needed. And for someone that got sick, pretty sick there, I
very much appreciate what you are doing.
Dr. Hotez. We actually have a paper coming out at the end
of July on the Democratic Republic of the Congo (DRC), and
NTDs. And I call them ``Neglected Tropical Diseases in the
Heart of Darkness,'' referring to Joseph Conrad's book. So you
are absolutely right, the DRC is a brutal place for these
diseases.
Mr. Stockman. Yeah. And I know some of our people from WHO
are over there dressed up in the suits, and they thought they
were ghosts, and they murdered them, which----
Dr. Hotez. Oh, my God, that is terrible.
Mr. Stockman [continuing]. Was not good. They were there
for, what is it, the bleeding of the pores----
Mr. Smith. Ebola.
Dr. Hotez. The Ebola virus, right, right.
Mr. Stockman. So we have to do something to educate them
before we put people in that kind of situation.
Dr. Hotez. Yes, we have this epidemic now of healthcare
workers getting killed while doing noble pursuits. We see this
now in the delivery of polio vaccines with healthcare workers
in Pakistan----
Mr. Stockman. Yeah, they spread the rumor that it was----
Dr. Hotez. Yeah. It is just so profoundly sad, yeah.
Mr. Stockman. Well, Mr. Chairman, I would just--go ahead.
Mr. Smith. Oh, I was going to go to Dr. Siegel.
Mr. Stockman. Oh, okay.
Mr. Smith. Unless you want to----
Mr. Stockman. No, I would just yield back. And thank you. I
appreciate it.
Mr. Smith. Dr. Siegel, please proceed.
STATEMENT OF JAY SIEGEL, M.D., CHIEF BIOTECHNOLOGY OFFICER AND
HEAD OF SCIENTIFIC STRATEGY AND POLICY, JOHNSON & JOHNSON
Dr. Siegel. Thank you.
Chairman Smith, members of this esteemed committee, thank
you for inviting me to testify. My name is Jay Siegel. I am
chief biotechnology officer and head of scientific strategy and
policy at Johnson & Johnson.
On behalf of the Johnson & Johnson family of companies, I
applaud you for organizing this hearing on the important
subject of treatments for neglected diseases. As an infectious
disease physician, a retired United States Public Health
Service officer, and a former senior FDA official, as well as a
pharmaceutical R&D executive, I greatly appreciate both the
importance and challenge of this issue.
There is a deep and lasting commitment at Johnson & Johnson
to global public health. In 2012, we extended that commitment
as we signed the London Declaration on Neglected Tropical
Diseases. And, earlier this week, we created Johnson Global
Public Health, an internal organization to coordinate and
advance our efforts in developing and providing access to
therapies with important global public health impact.
Johnson & Johnson has several active efforts in this area.
We are collaborating on development of a new bioavailable
formulation of flubendazole with potential to eradicate the
parasites that cause lymphatic filariasis and onchocerciasis,
debilitating diseases affecting millions of people in tropical
counties.
We donate more than 200 million doses of mebendazole yearly
through the Children Without Worms partnership as part of a
comprehensive strategy to reduce the burden of intestinal
parasites. And we are now developing a chewable formulation of
mebendazole to enable its use in younger children.
We discovered and developed Sirturo, the first new medicine
for tuberculosis, or the first medicine with a new mechanism of
action, in more than 40 years. Recently approved for use in
treatment of adults with pulmonary multidrug-resistant
tuberculosis, Sirturo will help address this important and
growing public health challenge. Our 10 years' investment in
Sirturo discovery and development will continue and grow to
address product introduction, to ensure appropriate use and
access, and to continue clinical research. Cost recovery from
sales of Sirturo is expected to be relatively small, elusive,
and incomplete.
Of course, our investments in these projects are not
motivated by the potential profits, but rather by the
opportunity to make a contribution to global public health.
Notwithstanding our efforts and the laudable efforts of many
other public- and private-sector organizations, as you have
been hearing, much, much more remains to be done.
In the U.S., we ought to address these issues with a sense
of urgency. Given the realities of climate change and
increasing international travel, it may be more prudent for us
to conceive of several diseases that occur predominately in
developing countries but rarely in the U.S. not as orphan
diseases but rather as morphing diseases--morphing in terms of
size and extent of their reach. Americans at home are at
increasing risk, as you have heard from several before me, for
example, with regard to dengue fever.
Even diseases that are more tightly constrained to
resource-limited settings often take their toll not only in
terms of tremendous human suffering but also in terms of social
and economic impact that can reverberate across our
increasingly interconnected world.
We greatly need new and improved therapies, and Congress
can accelerate progress toward that goal. U.S. Government
agencies and departments, including FDA, NIH, CDC, and the DoD,
play important roles in addressing neglected diseases and can
be and have been valuable partners.
These efforts and partnerships can be facilitated. A
reworking of R&D models, regulatory models, incentive models,
and partnership models could enable greater investments and
greater progress. In my written testimony, I describe several
existing models that might stimulate more investment and more
progress in this area.
Drawing on our experiences, we encourage Congress to
reflect upon three considerations in the design of policies to
stimulate investment and accelerate progress in this area.
First, such policies should take into consideration a
holistic view of the costs and risks across all stages of
developing and supporting treatments.
Second, the menu of options available to stimulate
investment should be varied and extensive. This approach would
help spark the level of investment needed, would address all
stages of development, and would expedite assessment of which
options work best.
Third, policies should encourage partnerships that bring
broad expertise to bear and help to diffuse the risk of drug
development and delivery across multiple actors.
In conclusion, the challenge of addressing neglected
diseases is large and complex, but the U.S. Congress has
opportunities to facilitate, coordinate, and incentivize the
needed efforts of many interested and capable parties, thereby
accelerating important advances against these devastating
diseases.
Thank you, Chairman Smith and members of this committee,
for your leadership on this issue. I look forward to answering
any questions you may have.
Mr. Smith. Dr. Siegel, thank you very much for your
testimony and for your very concrete recommendations.
[The prepared statement of Dr. Siegel follows:]
----------
Mr. Smith. Dr. Zwane?
STATEMENT OF ALIX ZWANE, PH.D., EXECUTIVE DIRECTOR, EVIDENCE
ACTION
Ms. Zwane. Thank you. Thank you very much.
Mr. Chairman and members of the committee, thanks for the
opportunity to speak with you today.
My name is Alix Zwane, and I am the executive director of
the Deworm the World Initiative. That is a consortium of
organizations which actively supports the scaleup of national
or, in the case of India, state-level school-based deworming
programs.
I come to my leadership role in the Deworm the World
Initiative because my full-time job is the executive director
of Evidence Action, a new nongovernment organization working to
scale proven interventions to improve the lives of the poor in
Africa and Asia. We are the leaders of the Deworm the World
consortium.
School-based deworming is now recognized as one of the
smartest and most cost-effective global health investments by
leading academic centers, including our partner, Innovations
for Poverty Action. This year, Deworm the World has helped our
Government partners provide deworming treatment to over 35
million children in Kenya and in India.
My testimony today offers some practical recommendations on
how to bridge the treatment gap in mass drug administration for
the treatment of soil-transmitted helminths, schistosomiasis,
and other intestinal parasites.
I am not going to focus primarily on a plea for a large
infusion of donor resources. Thanks to generous drug donation
programs by our pharmaceutical company partners like Johnson &
Johnson, the quantity of available drugs currently outstrips
programmatic demand. Our experience suggests that targeted,
catalytic investments can be hugely beneficial.
I also do not focus on calling for additional political
will from developing-country governments. We recognize that our
partner governments have many competing priorities and limited
budgets. They have to make hard choices, even with the very
best of intentions.
In this summary of my submitted testimony, I highlight
instead how the gap between service need and service delivery
can be bridged by bringing useful but nonintuitive information
and evidence to the attention of policymakers; how sharing
targeted and practical lessons about best practices and
implementation can leverage and unlock developing-country
government money; and reducing the NTD knowledge gap. I will
discuss each of these very briefly in turn.
First, on the question of evidence, as has been discussed
earlier in this session, the magnitude of the benefits from
deworming primary-school-age children is truly striking. A 2007
study that measured the impact of an early 20th-century
hookworm eradication effort in the southern United States found
treatment to increase school enrollment, school attendance, and
literacy. A randomized control trial in Kenya of a school-based
deworming program found similar results.
And by tracking those children over time, it has been shown
that the benefits translate into more hours worked and higher
wages for adults when they enter the formal labor market. This
suggests a clear and identifiable link between global health
interventions that reduce the suffering of children now and
economic growth over the longer run.
Sharing these kinds of results with policymakers as part of
a larger package of services or a structured engagement on
doing more evidence-based policymaking more generally can help
the decision-makers prioritize investments so that their scarce
resources stretch further.
Increasing the capacity of governments to implement school-
based deworming can make a path to sustainable, scalable
services less daunting and more politically palatable. It is a
challenge to convince a ministry of health official to take on
a new national program, but by demonstrating how this can be
done, by showing what kind of support can be provided that is
targeted and straightforward, it can make it easier to take on
a new program.
The kind of support needed includes mapping which areas of
the country have the highest level of worm infection, creating
plans and budgets that actually target those areas carefully,
designing program processes and materials for training teachers
and coordinating implementation logistics, and developing
monitoring and evaluation systems to reliably assess program
performance.
Joint ownership across ministries of education and health
is crucial to school-based deworming. At Deworm the World, we
help to cultivate that partnership. We also identify
opportunities to integrate with other synergistic programs,
such as micronutrient initiatives or vitamin A programs that
also target schoolchildren. By leveraging other programs and
piecing together budgets from different pots, we can unlock
government resources and point the way to success.
Donor support for technical assistance in school-based
deworming can be a highly leveraged form of support for child
health. For example, we estimate that for every dollar of donor
resources that we bring to bear for our work in India, we
leverage something like $17 of Indian Government money. That
has helped us to treat some 30 million Indian children across
several states in the past year.
Finally, reducing the knowledge gap. Thanks to investments
by USAID and The Gates Foundation and others, we understand
increasingly well the benefits of deworming and how to achieve
scale with quality. However, we continue to face uncertainty
about the necessary duration of mass treatment.
As an implementing organization, I can tell you it is
difficult to work with a government counterpart to bring a new
program on board when you can't tell them with certainty if
they are making a 5-year commitment or a 10-year commitment.
Those are very different things from a budget perspective and a
political perspective.
But right now there is a lot of uncertainty just from a
science perspective about how long to do mass treatments. This
uncertainty makes risk-averse officials hesitant to act.
Increased effort to understand how treatment strategies should
change over time would be enormously beneficial to achieving
our collective goals around coverage.
In summary, our experience in the Deworm the World
Initiative suggests that supporting the activities I have
discussed here can address the political, technical, and
managerial challenges to expanding deworming programs at a cost
that is less than 50 cents per child per year. This is one of
the most cost-effective means of improving educational outcomes
for children in developing countries that we know, and
expanding its reach is well within our grasp.
Thank you for your attention and the opportunity.
Mr. Smith. Dr. Zwane, thank you very much for your
testimony and for your leadership.
[The prepared statement of Ms. Zwane follows:]
----------
Mr. Smith. And I can say, I am practically in awe with the
information conveyed to this committee. I have followed this,
but, as you said, Dr. Hotez, this is the disease that you never
heard of, most people have not heard of, and yet it is having
such a debilitating impact on countries, including our own.
Dr. Zwane, you mentioned the 1-to-17 ratio in India. Are
the occurrences of worms, the prevalence, is it mostly with the
Dalits? Is it, like Dr. Hotez talked about, disproportionally
poor people who suffer from it?
And when you mentioned the Kenya study, that also comports
with what you were saying, Dr. Hotez, about the hookworms have
actually been shown to reduce childhood intelligence and
cognition and earnings by 40 percent or more. If we want to see
economic growth, people reaching their objectives and
maximizing their potential, it seems to me that getting rid of
the worms has to be a very significant priority for us here and
people all over the world.
Dr. Hotez. As they say, it is the worm, stupid. Right?
Mr. Smith. You said, Dr. Hotez, that more than one-quarter
of pregnant women in sub-Saharan Africa have hookworms, go into
labor, and deliver profoundly anemic infants. If you could
speak to the impact it is having on the women themselves in
terms of morbidity and mortality for those women.
You did say--and the number is astonishing--schistosomiasis
malaffects 100 million girls in sub-Saharan Africa and young
women.
And you also mentioned, I think all of you perhaps
referenced it, but you did especially, the Global Fund. We had
Mark Dybul, the executive director, the former AIDS czar for
George W. Bush, before us. I wish we had this hearing first
because I would have asked him, but we will follow up with him.
What is the Global Fund doing on this?
You know, you mentioned the 3-4 percent increase in
acquiring HIV/AIDS. It may be Africa's most important co-factor
in the HIV/AIDS epidemic. How many times have we had PEPFAR
hearings, and we don't hear that, but you have brought it to
this committee.
Dr. Hotez. Yes, indeed. I mean, there are a couple of
issues.
So, first of all, these are the most common diseases of
girls and women on the planet. And, I think we often don't
think about neglected tropical diseases as a girls' and women's
issue; everyone talks about how we can address the plight of
girls and women living in poverty and how we are going do it.
This is how you do it. This is the cheapest way to do it and
have the greatest bang for your buck, point number one.
Point number two, Mark Dybul is a wonderful man. When we
worked with the Bush administration on getting those funds
appropriated through USAID, it was Mark Dybul who was providing
a lot of the conduits. So he gets it, he understands the
importance of neglected tropical diseases.
That is not the issue. The problem is these big
organizations, whether it is PEPFAR or whether it is the Global
Fund to Fight AIDS, TB, and Malaria, tend to get very siloed
because it is everything we can do to keep up with their
current agreements. So, when you go to them and you say, guys,
you have to take on neglected tropical diseases, they just look
and sigh and say, ``We know, Peter, but, I mean, we are so
buried right now, how we going do it?''
And, so I don't know how we enable PEPFAR and the Global
Fund to take it on. You know, they are exhausted, amazing
public servants and really smart people. We have good people
that work in PEPFAR, good people that work in USAID, good
people that work in the Global Fund to Fight AIDS, TB, and
Malaria. And I think this could be an important role for
Congress, is to help that, help facilitate that process. But it
has to be done.
Mr. Smith. Dr. Siegel, you mentioned three very specific
recommendations. Is that something that would lend itself to
putting into a bill, or could it just be done by admonishing
the current administration and other governments and NGOs do
more?
Dr. Siegel. I think a lot of what can be done and needs to
be done would not require legislation, although I won't say
that I have a lot of expertise in this area.
So one of the areas, of course, in my testimony,
particularly the written testimony goes into significant length
about, is establishment of different mechanisms, systems,
partnerships that would create either incentives or facilitate
investment from not just industry but private foundations,
other parties, private investors, to help address this problem.
A lot of that work, I think, does not require legislation.
I think congressional support, exhortation, increased
involvement, and perhaps increased funding for involvement from
public health agencies and the Department of Defense can all
play an important role in making that happen. And I think
Congress can really, you know, stimulate those sorts of
actions. There might be some areas and some types of investment
scenarios that would require legislation, though.
Mr. Smith. Yes, Dr. Hotez? And, Dr. Hotez, do you think
that a centerpiece of a neglected diseases bill, tropical
diseases, would be these ``Centers for Excellence''?
Dr. Hotez. So the ``Centers of Excellence'' would be a key
component, you know, both to focus on neglected tropical
diseases abroad as well as here.
So what we are seeing is a blurring; it is wherever poverty
exists. In fact, I have this new provocative map that was just
published in Foreign Policy about where the neglected tropical
diseases are, and it is those pockets of intense poverty in G-
20 countries--northeastern Brazil, southern Mexico. And right
there is the southern United States, with Texas and elsewhere
and the Gulf Coast. So that is point one.
I think point two is--and I have written about this--we
need to set aside a portion of the President's Global Health
Initiative, it could be just 1-2 percent, for new product R&D.
I think that would be a real game-changer. That would put a lot
of support into the system and to make that happen. So that is
a key component.
And I think, lastly, we need to really look at these
diseases as girls' and women's issues and think about, when we
talk of addressing women's health, ask, what are we doing about
the neglected tropical diseases? I think that is extremely
important.
And, finally, I will just say, NIH, you know, the
leadership of NIH, including Roger Glass at Fogarty, Tony Fauci
at NIAID, have really bent over backwards to keep NTDs on the
radar screen, oftentimes against political pressure. We really
have to congratulate them for their leadership.
The other thing we are not doing, though, is we are not
supporting our military. We are not supporting the Walter Reed
Army Institute of Research. We are not supporting the Navy
equivalent.
We often forget, many of these products that everyone talks
about for scaling up were actually invented by the U.S.
military. One of the reasons why we initially set up in
Washington, DC, is, how do you learn how to make a vaccine in a
nonprofit sector? Well, we do it through learning from--people
from Walter Reed Army Institute of Research have been our
teachers.
They are dying on the vine right now because they are not
being provided any funding, even though they have one of the
greatest track records in leadership. We have an article
talking about the role of military leadership in tropical
disease coming out in the fall.
Mr. Smith. We had a representative from Walter Reed testify
on malaria just a few weeks. But true to form, because, you
know, he is a good--he knows the border----
Dr. Hotez. Yeah, they can't say it. That is why I have to
say it.
Mr. Smith. I asked him, what do you need? How can we be
helpful? And he was very creative in how he answered; well, we
are doing the best we can with what we have.
Dr. Hotez. You know, Walter Reed is one of our greatest
national treasures. And we are not doing either the military or
our U.S. citizens a service by cutting them short.
Dr. Siegel. Chairman Smith?
Mr. Smith. Yes?
Dr. Siegel. I just want to get back to your earlier
question and say that I would like to take the opportunity to
consult with a broader range of experts, because there is a lot
that can be done without further legislation, but there is also
a lot, potentially, where legislation would be helpful. And we
would be pleased to----
Mr. Smith. If you could help us craft a bill. I find
sometimes when you get a bill passed in the House, even if it
doesn't pass the Senate, it does have an impact.
Some years ago, I introduced a bill to deal with obstetric
fistula. And I visited a hospital which many people have
visited in Ethiopia that has worked miracles on women who
suffer from that horrible, disfiguring, and debilitating
outcome from obstructed delivery and other reasons.
And we got the bill passed in the House. The Senate never
took it up. I asked USAID if they would just follow the
parameters. They had the authority to do what the bill had
recommended. And Kent Hill, who was then the USAID health
official, the top guy, he did it. And 20,000 obstetric fistula
surgeries so far have been performed via USAID dollars,
augmenting what the Africans are doing, what everyone else is
doing, including that great hospital in Ethiopia.
So, you know, any ideas you have, we will work hard to get
it enacted into law when we get a, you know, tropical disease--
--
Dr. Hotez. That is so reassuring. And we are not talking
billions. We are not even talking hundreds of millions. We are
talking very modest amounts that can be transformational.
Mr. Smith. Please give us your best ideas on what it should
look like. And we work very collaboratively, obviously, with
our folks over at the State Department, USAID, NIH, and CDC.
But best ideas, please, fork them over.
Dr. Zwane, did you want to comment, particularly on the
Dalits issue? Are they mostly----
Ms. Zwane. Oh, yes. On the issue of who has worms in India,
the first thing I would say is there is a lot we don't know
about that. Unlike in Africa, where quite a bit of mapping has
been done to understand where the worms are, we know relatively
less about that in South Asia.
A highly catalytic investment would be to systematically
map India for the likelihood of soil-transmitted helminth
infections, which would cost something on the order of $8
million to $10 million and wouldn't be, you know, from the--
maybe I am an ex-Gates Foundation person, but that is not a lot
of money in the grand scheme of things.
But what we do know even without that systematic map of
India is that the worms are a problem of rural poverty. So
there are fewer worms even amongst the poor in urban settings.
And where there is lack of sanitation is where there are a lot
of worms. So, you know, without saying I have the perfect maps
that would satisfy Dr. Hotez or CDC, the primary maps are
concentrated amongst the poor in the Hindi Belt, so Bihar,
Rajasthan, that area of India.
Mr. Smith. The two Millennium Development Goals, one
dealing with reducing maternal mortality and also infant
mortality--again, getting back to your statement, Dr. Hotez,
about the anemic infants, is there any number of how many of
those children, because they are born prematurely and anemic,
actually die or suffer from other diseases that would reduce
their quality of life?
Dr. Hotez. Well, again, this is one of the reasons why
neglected tropical diseases have been neglected. With some
exceptions, most of these are not killer diseases. So none of
the children are dying from the diseases that Dr. Zwane talked
about or I talked about. But they are no longer wage-earning,
productive individuals.
And, so what needs to be done is to aggressively scale up
things like deworming, but we are going to need new
interventions. So you talk about the fact that you don't know
how long we are going to have to treat for; it depends. So for
that horrific limb-disfiguring disease, four or five yearly
treatments can actually eliminate the disease as a public
health problem. We are talking pennies a year. So incredible.
For some of the intestinal worms, like hookworm, they keep
coming back. So what we are doing at Sabin Vaccine Institute
through Texas Children's and Baylor is we are making a hookworm
vaccine. The idea is that you fold this in, so after you
vaccinate, they don't come back.
Now, the key is, it has to be a cheap vaccine, right? So
our economists tell us that we have to make these vaccines
under $2 a dose. So it is a very fascinating process of how we
make vaccines. We use the cheapest inexpensive expression
vectors, column resins that have been off-patent for years.
Because we know we can make a vaccine for hundreds of dollars;
the trick is doing it for $2. And I think we are succeeding in
that, and now it is in clinical trials.
Mr. Smith. Do they require a cold chain?
Dr. Hotez. Well, right now our vaccines require a cold
chain. It would be nice if we can factor that out, as well. But
for version 1.0 of our vaccines, we still need a cold chain.
Ms. Zwane. Just to follow up on that with respect to very
young children, the drug donation program and the program--
please correct me if I say anything incorrect--as overseen by
WHO focuses on primary-school-age children. But we know that,
in fact, children who are 1 year old or certainly 2 years old
also may very well often have worms. But the drug donation
program does not provide drugs for those children.
So if we want to work with a country government to expand
treatment down, getting into that 1,000-days period, then they
themselves have to purchase the drugs, which changes the cost,
the calculus of the program and the complexity of the program
significantly. Expanding our conversation around how to handle
children under primary-school age could also be something quite
valuable.
Dr. Siegel. That is true, and that is one of the--we are
actually investing some number millions of the dollars in the
development of chewable, as I mention in my testimony, a
chewable version of mebendazole. And one of the main reasons is
in order to be able to bring that down and make it more
accessible to younger children.
Dr. Hotez Yes, one of the things that has been found now
which is very curious is that we are seeing high rates of
hookworm infection in young neonates, as well. There is some
suggestion that the neonates are acquiring it vertically
through breast milk, that the women are infected, the larvae
get into breast milk, and it is being transmitted vertically
from mother to child.
So we are finding all these new mother-to-child NTDs. So,
for instance, maternal-child Chagas disease, we are finding it
is transmitted from mother to baby. And it is, we estimate,
300,000 pregnant women in Latin America with Chagas disease.
Researchers at Tulane have estimated 40,000 pregnant women in
North America alone with Chagas disease, transmitting it from
mother to baby around 5-10 percent of the time.
We have nothing to offer those pregnant women, because the
medicines that are used to treat Chagas disease, like many of
the NTDs, they are basically poisons. And you hope you can
poison the parasite before you poison the person who is getting
the medicine. These medicines were developed in the Pleistocene
era, right? They were developed a long time ago, and we need to
do better.
So organizations like ours, Sabin Vaccine Institute, and
Texas Children's and Baylor College of Medicine, their National
School of Tropical Medicine, are making vaccines. And then you
have very exciting drug product development partnerships like
DNDI, the Drugs for Neglected Disease Initiative, Institute for
OneWorld Health working on the small molecule. And then,
together, we think we want to create a whole portfolio of these
products that can be made.
Mr. Smith. Real quick, two final questions. How many total
people are malaffected by worms, you know, hookworm, every
other worm?
And, secondly, in treating that one-quarter of pregnant
women in sub-Saharan Africa who have hookworms, is the
treatment injurious to the mother and baby?
Dr. Hotez. So what we do is we treat--we now recommend
treating in the second or third trimester. Nobody likes to give
the medicine in the first trimester of pregnancy. And it has
been shown to be extremely beneficial, both to mother and the
baby in terms of infant survival downstream.
Total number of people infected, well, wherever you find
poverty, you find a worm. So we know they are ubiquitous among
the 1.3 billion people who live on less than $1.25 a day. A
significant percentage of people live on less than $2 a day. So
it would not be an exaggeration to say 2 billion people on our
planet with worms.
Mr. Smith. Do the worms go through the placenta, or is it
at birth?
Dr. Hotez. Worms, generally speaking, do not go through the
placenta, but the Chagas disease parasite can go through the
placenta. The malaria parasite can go through the placenta.
These are single-celled organisms that have the ability to do
that.
Mr. Smith. We are joined by Congressman Meadows, who I know
had a meeting with the Ambassador from Turkmenistan that
prevented him from being here.
But thank you for joining us.
Mr. Meadows. Thank you, Mr. Chairman.
And thank each of you. And I came back to show the
importance of it. Actually, I have a meeting in a few minutes
with a head of the cabinet from Japan. So I am going to run
out.
Dr. Hotez. You can congratulate the Ambassador to Japan if
you like. They just stepped up and provided the first major
contribution for neglected disease product----
Mr. Meadows. I spoke to him----
Dr. Hotez. It is a partnership with Japanese industry.
Mr. Meadows [continuing]. And it is a wonderful
partnership, and we have a great relationship. So I will pass
that on to him.
But I want to just say thank you for highlighting this. And
the chairman is correct; when we highlight things, it may not
pass in legislation, but we do see a difference, truly, in
terms of other programs that are discretionary in terms of
where those dollars go. And so your testimony today is very key
and very apparent in terms of making real changes.
I would ask each of you, but specifically Dr. Siegel, if
you will get back to us in terms of legislative tweaks or
legislative initiatives that we can look at. And I will work
with the chairman in terms of putting forth and working hard to
make that a reality.
The other thing that I would ask, not necessarily for you
to comment, unless you have strong comments on that today, is
with regards to this 40 percent, where we are talking about a
40 percent reduction in terms of mental capacity or economic
benefit, that is huge because there are those that will sign on
to a piece of legislation based on the humanitarian aspect of
it, but there is another group that will sign on based on the
economic benefit.
And what we are seeing is we are having to spend major
economic dollars to go into these countries in Africa and
Central Asia and other places, that we could hopefully reduce
our long-term economic support if we increase that ability.
And so I would really like, if you would, to focus on that,
where we can put together a model that----
Dr. Hotez. We can pull together all the numbers from the
different counties--India loses $1 billion a year in economic
losses from lymphatic filariasis. We have now collected all
those numbers in one repository. We can provide that. When we
provide information on the legislation, we will be happy to
provide this data--and it is the same in the U.S. These
diseases are trapping people in poverty in the U.S.
Mr. Meadows. Well, and it is not the whole story. I mean--
--
Dr. Hotez. The humanitarian piece is important, but you are
absolutely right, this is enlightened self-interest.
Mr. Meadows. Well, I appreciate it. And I am going to have
to run, but I wanted to come and say thank each of you.
Thank you, Mr. Chairman, for once again being a great
voice. I yield back.
Mr. Smith. Thank you, Mr. Meadows.
Is there anything you would like to add before we conclude?
Dr. Hotez. Just to thank you for your leadership. And, you
know, as we often say, we need leaders in Congress to take the
``N'' out of ``NTD.'' And I am profoundly appreciative.
Dr. Siegel. Again, I also want to thank you.
And, you know, it was interesting that we heard earlier
from perhaps some younger members, but I lived through seeing
the impact the Orphan Drug Act had, and so you don't need to
sell me on the importance that legislation but also--and the
reason for my comment earlier was, just congressional attention
to an issue can make a big difference, as well.
So thank you very much, and we will get back with further
thoughts.
Ms. Zwane. Thank you very much.
Mr. Smith. Thank you.
We will use this hearing and your tremendous contributions
to combating these horrible diseases to launch what I hope will
be a significant bill. We will look to make it totally
bipartisan and look for some friends over on the Senate side to
do likewise, but we need your input.
And I would say, Dr. Hotez, in the early 1980s, I traveled
with Dr. Sabin to El Salvador when they had a ``day of
tranquility.'' The FMLN and President Napoleon Duarte's
government, at the behest of Jim Grant from UNICEF, actually
had a day or days of tranquility. They vaccinated about 200,000
kids. Nobody knows the exact number, but it was incredible. And
everywhere we went to the vaccination sites, kids got the
vaccinations for pertussis, diphtheria, and other diseases, but
they also got the drops for polio. And it was amazing to see.
I was back there at a conference on trafficking, combating
human trafficking, about 4 years ago. And I looked around, and
everybody in the audience seemed to be the age that they were
little tikes, little children when I was there before. And I
said, I bet you I met some of you 25 years ago, in a different
setting of course.
Dr. Hotez. And we shouldn't underestimate American
technology and its role as Ambassadors. And how do we project
power? Well, I think a lot of it has to do with what we are
doing for these infectious and neglected diseases.
Mr. Smith. I couldn't agree more.
Thank you so much.
The hearing is adjourned.
[Whereupon, at 5:10 p.m., the subcommittee was adjourned.]
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