[House Hearing, 113 Congress]
[From the U.S. Government Publishing Office]



 
            ADDRESSING THE NEGLECTED DISEASES TREATMENT GAP
=======================================================================


                                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

                               __________

        Printed for the use of the Committee on Foreign Affairs


Available via the World Wide Web: http://www.foreignaffairs.house.gov/ 
                                  or 
                       http://www.gpo.gov/fdsys/

                                 ______




                  U.S. GOVERNMENT PRINTING OFFICE
81-698                    WASHINGTON : 2013
-----------------------------------------------------------------------
For sale by the Superintendent of Documents, U.S. Government Printing 
Office Internet: bookstore.gpo.gov Phone: toll free (866) 512-1800; DC 
area (202) 512-1800 Fax: (202) 512-2104  Mail: Stop IDCC, Washington, DC 
20402-0001



                      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
                                 ------                                

    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

                              ----------                              
                                                                   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

                              ----------                              


                        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:]
    
    
    
    
    
    
    
    
    
    
    
    
    
    
    
    
    
    
    
    
    
    
    
    
    
    
                              ----------                              

    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.]
                                     

                                     

                            A P P E N D I X

                              ----------                              


     Material Submitted for the Hearing RecordNotice deg.




               \\ts\



        \s
                  t\


                                 
