[Senate Hearing 115-844]
[From the U.S. Government Publishing Office]


                                                      S. Hrg. 115-844

                          PRIORITIZING CURES:
                       SCIENCE AND STEWARDSHIP AT
                   THE NATIONAL INSTITUTES OF HEALTH

=======================================================================

                                HEARING

                                OF THE

                    COMMITTEE ON HEALTH, EDUCATION,
                          LABOR, AND PENSIONS

                          UNITED STATES SENATE

                     ONE HUNDRED FIFTEENTH CONGRESS

                             SECOND SESSION

                                   ON

 EXAMINING PRIORITIZING CURES, FOCUSING ON SCIENCE AND STEWARDSHIP AT 
                   THE NATIONAL INSTITUTES OF HEALTH

                               __________

                            AUGUST 23, 2018

                               __________

 Printed for the use of the Committee on Health, Education, Labor, and 
                                Pensions


        Available via the World Wide Web: http://www.govinfo.gov
        
                              __________
                               

                    U.S. GOVERNMENT PUBLISHING OFFICE                    
31-330 PDF                  WASHINGTON : 2020                     
          
--------------------------------------------------------------------------------------
        
        
          COMMITTEE ON HEALTH, EDUCATION, LABOR, AND PENSIONS

                  LAMAR ALEXANDER, Tennessee, Chairman
                  
MICHAEL B. ENZI, Wyoming		PATTY MURRAY, Washington
RICHARD BURR, North Carolina		BERNARD SANDERS (I), Vermont
JOHNNY ISAKSON, Georgia			ROBERT P. CASEY, JR., Pennsylvania
RAND PAUL, Kentucky			MICHAEL F. BENNET, Colorado
SUSAN M. COLLINS, Maine			TAMMY BALDWIN, Wisconsin
BILL CASSIDY, M.D., Louisiana		CHRISTOPHER S. MURPHY, Connecticut
TODD YOUNG, Indiana			ELIZABETH WARREN, Massachusetts
ORRIN G. HATCH, Utah			TIM KAINE, Virginia
PAT ROBERTS, Kansas			MAGGIE HASSAN, New Hampshire
LISA MURKOWSKI, Alaska			TINA SMITH, Minnesota
TIM SCOTT, South Carolina		DOUG JONES, Alabama                          
                  
                                     
               David P. Cleary, Republican Staff Director
         Lindsey Ward Seidman, Republican Deputy Staff Director
                 Evan Schatz, Democratic Staff Director
             John Righter, Democratic Deputy Staff Director
                            
                            
                            C O N T E N T S

                              ----------                              

                               STATEMENTS

                       THURSDAY, AUGUST 23, 2018

                                                                   Page

                           Committee Members

Alexander, Hon. Lamar, Chairman, Committee on Health, Education, 
  Labor, and Pensions, Opening statement.........................     1
Bennet, Hon. Michael F., a U.S. Senator from the State of 
  Colorado, Opening statement....................................     4

                               Witnesses

Collins, Francis, M.D., Ph.D., Director, National Institutes of 
  Health, Bethesda, MD...........................................     5
    Prepared statement...........................................     8
Bianchi, Diana, M.D., Director, National Institute of Child 
  Health and Human Development, Bethesda, MD.....................    14
Fauci, Anthony, M.D., Director, National Institute of Allergy and 
  Infectious Diseases, Bethesda, MD..............................    31
Hodes, Richard, M.D., Director, National Institute on Aging, 
  Bethesda, MD...................................................    23
Sharpless, Ned, M.D., Director, National Cancer Institute, 
  Bethesda, MD...................................................    29

                         QUESTIONS AND ANSWERS

Response by Francis Collins to questions of:
    Senator Alexander............................................    34
    Senator Roberts..............................................    37
    Senator Young................................................    42
    Senator Enzi.................................................    44
    Senator Collins..............................................    45
    Senator Burr.................................................    49
    Senator Murray...............................................    52
    Senator Casey................................................    73
    Senator Baldwin..............................................    76
    Senator Warren...............................................    77
    Senator Kaine................................................    79
    Senator Smith................................................    80
    Senator Jones................................................    81

 
                          PRIORITIZING CURES:
                       SCIENCE AND STEWARDSHIP AT
                   THE NATIONAL INSTITUTES OF HEALTH

                              ----------                              


                       Thursday, August 23, 2018

                                       U.S. Senate,
       Committee on Health, Education, Labor, and Pensions,
                                                    Washington, DC.
    The Committee met, pursuant to notice, at 10:02 a.m. in 
room SD-430, Dirksen Senate Office Building, Hon. Lamar 
Alexander, Chairman of the Committee, presiding.
    Present: Senators Alexander [presiding], Isakson, Collins, 
Cassidy, Scott, Murray, Casey, Bennet, Murphy, Warren, Kaine, 
Hassan, Smith, and Jones.

                 OPENING STATEMENT OF SENATOR ALEXANDER

    The Chairman. The Senate Committee on Health, Education, 
Labor, and Pensions will please come to order.
    Senator Bennet and I will each have an opening statement, 
and then I will introduce our witness, National Institutes of 
Health Director, Francis Collins. Then we will hear from Dr. 
Collins, and Senators will each have 5 minutes to ask 
questions.
    We have a vote at 10:30, not in the Committee but on the 
floor, and so we will continue straight through with the 
questioning. Senator Bennet, and I, and other Senators will 
share the presiding today so that we can continue the 
discussion.
    Not long ago, I ran into a friend from Vanderbilt 
University who is perhaps our largest contributor to cancer 
research there. This is what he said to me, ``Is it not a shame 
that the Congress is not doing anything to fund biomedical 
research?''
    [Laughter.]
    The Chairman. This is how I replied to him. I said, ``In 
December 2016, Congress passed what Senator McConnell called, 
`The most important legislation of the year,' the 21st Century 
Cures Act. That Act gave the National Institutes of Health $4.8 
billion for the Precision Medicine Initiative, the BRAIN 
Initiative, the Cancer Moonshot, regenerative medicine, as well 
as many new flexibilities and authorities to conduct the 
research that we hope will lead to breathtaking new medicines, 
treatments, and cures.''
    That was thanks to Senator Blunt, Senator Murray, Senator 
Durbin, Senator Moran, and many other Senators. The 
Appropriations Committee is on track to provide record funding 
for the fourth year in a row to the National Institutes of 
Health.
    First, Congress increased N.I.H. funding by $2 billion in 
2015; that is in addition to the Cures money. Then, we 
increased N.I.H. funding by $2 billion more in 2016. Then in 
2017, Congress increased funding for the National Institutes of 
Health by $3 billion, including $500 million to work on a non-
addictive painkiller. And today, we expect the full Senate to 
approve an additional $2 billion increase to N.I.H. funding for 
next year.
    This means, if the bill we hope the Senate approves today 
is signed into law, Congress will have increased funding for 
the National Institutes of Health by $9 billion since 2015, a 
30 percent increase.
    The way we do our budgets here, that usually builds into 
the budgets over a longer period of time, that money, as a 
base. So if you counted over ten years, a $2 billion increase 
in one year means over ten years $20 billion in new spending 
authority. These increases have included the funding we 
intended to deliver on Cures.
    The purpose of this hearing is to make sure that money is 
being spent wisely.
    The reason Congress has devoted so much funding to 
biomedical research is well-captured in testimony that Dr. 
Collins gave before the Appropriations Committee a couple of 
years ago, when he offered ten ``bold predictions,'' as you 
called them then, Dr. Collins, of what we might be able to 
achieve in the next ten years if we continued to invest in 
research as we now have.
    Some of these predictions that you made then were:
    Being able to identify Alzheimer's disease before symptoms 
appear;
    The possibility we could rebuild a patient's heart with 
their own cells;
    The creation of a safe and effective artificial pancreas, 
making life easier and healthier for the millions of Americans 
with diabetes;
    Development of new vaccines, including for Zika and HIV/
AIDS, and the universal flu;
    Development of a new, non-addictive pain medicine, which 
may be ``the Holy Grail'' to dealing with the opioid crisis;
    Significant progress on the Precision Medicine Initiative, 
which aims to map the genomes of one million volunteers so we 
can better tailor treatments to individual patients; and,
    New treatments for cancer patients.
    Those are all the bold predictions.
    The two things I hope we keep in mind when we look at these 
large increases in funding that Congress has given the National 
Institutes of Health in recent years is first, it is hard to 
think of a major scientific advancement since World War II that 
has not been supported by Federal research funding. But we are 
not the only country that has figured that out. Other countries 
have seen that investments in basic research can lead to 
breathtaking new discoveries.
    Since 2007, China has increased its spending on basic 
science by a factor of four and may surpass the United States 
in total spending on research and development this year, 
according to Norm Augustine, who, during the George W. Bush 
administration, chaired the Rising Above the Gathering Storm 
group, the bipartisan committee that was charged with making 
recommendations about how to keep America's competitive 
advantage.
    The second thing I hope we keep in mind is that these large 
increases in funding for biomedical research, and other 
increases for national laboratories and other basic research, 
are not the part of the Federal budget that creates the huge 
national deficit.
    This spending, the spending we are talking about here, is 
part of the so-called discretionary spending, which is now 
roughly 29 percent of all Federal spending and includes the 
national defense, the national parks, the national 
laboratories, the National Institutes of Health among other 
things.
    Over the last ten years, this is the part of the budget 
that has grown at about the rate of inflation. Over the next 
ten years, according to the Congressional Budget Office, it is 
expected to grow at only a little more than the rate of 
inflation. So funding for research has been carved out of these 
budget limitations and is not the reason for the increasing 
Federal debt.
    What causes the Federal debt to increase is spending on 
entitlements, which according to the CBO, is going to squeeze 
funding for research, our national labs, and our national 
security over the next ten years.
    I have one other topic, Dr. Collins, I want to give you an 
opportunity to discuss.
    You recently told Senator Murray and me about an ongoing 
investigation into federally funded research, including, in 
some cases, research conducted by foreign nationals. I would 
ask you to take a few extra minutes in your opening 
presentation to brief the Committee on this issue. It is 
important to protect the integrity of research funded by the 
Federal Government.
    It is also important to recognize the role that scientists 
from other countries have played in research funded by the U.S. 
Government.
    For example, the director of Oak Ridge National Laboratory 
came to this country from India, before he became a citizen. 
The incoming director of the Los Alamos Laboratory came from 
Canada, before he became a citizen. The director of the 
National Renewable Energy Laboratory came from Germany before 
he became a citizen.
    Many graduate students at American universities, who work 
on N.I.H. grants, are foreign nationals legally in our country. 
And since 2000, thirty-three Americans, who were born in other 
countries, have won Nobel Prizes in Chemistry, Medicine, and 
Physics.
    I want to acknowledge the great advantage to our country of 
attracting the brightest people from around the world to our 
universities and laboratories as long as they follow the rules 
and conduct their research in appropriate ways.
    This is an issue that impacts more than just the National 
Institutes of Health and more than just this Committee's 
jurisdiction. But if there are some bad actors who are 
attempting to influence N.I.H.-funded research, we want to know 
about it, and we want to know what authority you need, or 
others need, to deal with it.
    Thank you.
    Senator Bennet.

                  OPENING STATEMENT OF SENATOR BENNET

    Senator Bennet. Thank you, Chairman Alexander, for holding 
this bipartisan hearing on N.I.H.'s important work, including 
the agency's progress in implementing the 21st Century Cures 
Act.
    Dr. Collins, thank you for being here today and for your 
colleagues taking the time to be here to give us an update.
    In the last few decades, we have seen exponential 
advancements in medical research. The research community has 
developed cures and maintenance treatments for serious 
illnesses that used to be a death sentence.
    When I worked on the Breakthrough Therapies Act with 
Senators Burr and Hatch in 2012, we recognized the need to 
expedite treatments when early trials showed promises for 
conditions within an unmet need.
    We had no idea how successful the program would be. As of 
August 13, the FDA has approved 116 breakthrough therapy 
designated products. Many of these treatments show the promise 
of precision medicine.
    As N.I.H.-supported research has made clear, therapies that 
target specific genes or molecular pathways make it possible 
for providers to predict whether patients will respond to 
certain treatments.
    This Committee also recently worked to pass the RACE for 
Children bill to ensure that kids with cancer have the same 
access to targeted treatments that adults do. Pediatric 
oncologists at Children's Hospital Colorado are hopeful that 
they can launch as many as twenty-five new clinical trials 
because of the new law.
    These treatments will come from the research bench to the 
bedside, in large part, because of the great work happening at 
N.I.H. today.
    The 21st Century Cures Act included monumental policies to 
advance medical research. The hope of personalized medicine has 
already been a reality for some patients. I am looking forward 
to hearing more from Dr. Collins about the Precision Medicine 
Initiative and how we can reach even more Americans with 
therapies that maximize benefits and minimize toxic side 
effects.
    The 21st Century Cures Act also included the BRAIN 
Initiative, which will help researchers and the medical 
community grasp the intricacies of the human brain.
    Though we have gained a better understanding of how to 
treat different types of cancers or cystic fibrosis, the 
development of meaningful therapies for neurological diseases 
like Alzheimer's, Parkinson's, and ALS have lagged behind. I 
look forward to hearing about the progress on these 
initiatives.
    I am also interested to hear more about the work N.I.H. is 
doing to combat the opioid crisis, which continues to rip apart 
families and take lives in Colorado and across our country. 
This Committee has been active in working on an approach as a 
first step to respond to this epidemic, but there is so much 
more to do.
    With over 42,000 lives lost in 2016, and a preliminary 
estimate of almost 50,000 Americans in 2017, we still have much 
more to do.
    I want to thank the Chairman for raising the role of talent 
programs, and I am interested in hearing what you have to say 
on this subject, Dr. Collins. I would like to echo what the 
Chairman stated.
    Breakthroughs in medical research cannot happen in the silo 
of any one country, but we also want to ensure that we 
prioritize transparency and appropriately deal with bad actors 
who are taking steps that actually undermine the science and 
American efforts to do research.
    Thanks again to the Chairman, and the Ranking Member, and 
to Dr. Collins for being here today. I look forward to your 
testimony.
    The Chairman. Thank you, Senator Bennet.
    I am pleased to welcome Dr. Collins to today's hearing. 
Thanks to him for being here. He is overseeing the work of the 
largest supporter of biomedical research in the world. He has 
been the Director of N.I.H. since 2009.
    He is accompanied by Dr. Diana W. Bianchi, Director of the 
National Institute of Child Health and Human Development; Dr. 
Anthony S. Fauci, Director of the National Institute of Allergy 
and Infectious Diseases; Dr. Richard Hodes is Director of the 
National Institute on Aging; and Dr. Ned Sharpless, Director of 
the National Cancer Institute.
    We welcome Dr. Collins. Please give your testimony now.

    STATEMENT OF FRANCIS S. COLLINS, M.D., Ph.D., DIRECTOR, 
       NATIONAL INSTITUTES OF HEALTH, BETHESDA, MARYLAND

    Dr. Collins. Chairman Alexander, Senator Bennet, and 
Members of the Senate HELP Committee.
    Thank you for giving me a little extra time to speak on 
this issue of protecting the integrity of U.S. biomedical 
research from undue foreign influence, which both of you have 
raised.
    N.I.H. is built on the bedrock principles of scientific 
excellence, unassailable integrity, and fair competition. 
N.I.H.'s commitment to these principles is unwavering.
    We have long understood, however, that the robustness of 
the biomedical research enterprise is under constant threat by 
risks to the security of intellectual property and the 
integrity of peer review. This knowledge has shaped our 
existing policies and practices.
    But through our own investigations, conversations with law 
enforcement, and even just from watching the press, we can see 
that the magnitude of these risks is increasing.
    Yesterday, I wrote to the senior representatives of more 
than 10,000 N.I.H. grantee institutions to request that they 
review their records for evidence of malfeasance in three areas 
of concern.
    First, failure by some researchers at N.I.H.-funded 
institutions to disclose substantial contributions of resources 
from other organizations including foreign governments, which 
threatens to distort decisions about the appropriate use of 
N.I.H. funds.
    Second, diversion of intellectual property; in grant 
applications or produced by N.I.H. supported biomedical 
research to other entities, including other countries.
    Third, is failure by some peer reviewers to keep 
information on grant applications confidential including, in 
some instances, disclosure to foreign entities or other 
attempts to influence funding decisions.
    While we, at N.I.H., depend on the major security agencies, 
and the Department of Health and Human Services's broader 
national security efforts, to protect our interests, N.I.H. and 
the U.S. biomedical research community at large have a vested 
interest in mitigating these unacceptable breaches of trust and 
confidentiality that could undermine the integrity of U.S. 
biomedical research.
    To help address this challenge, I am today announcing the 
new Working Group of my Advisory Committee to the director 
whose charge will be to identify robust methods to, first, 
improve accurate reporting of all sources of research support, 
financial interests, and affiliations.
    Second, mitigate the risk to intellectual property 
security.
    Third, explore additional steps to protect the integrity of 
peer review.
    But fourth, and importantly, to carry out these actions in 
a way that reflects the long tradition of partnership between 
N.I.H. and grantee institutions, and that emphasizes the 
compelling value of ongoing honorable participation by foreign 
nationals in the American scientific enterprise, which both of 
you have already highlighted in your opening statements.
    President M. Roy Wilson of Wayne State University and Dr. 
Lawrence Tabak, my principal deputy, will co-chair this group. 
The other members include President Jeffrey Balser of 
Vanderbilt University, President Ana Mari Cauce of the 
University of Washington, President Michael Drake of Ohio State 
University, President Wallace Loh of the University of 
Maryland, President Samuel Stanley of Stony Brook University, 
and Dr. Maria Zuber, Vice President for Research at M.I.T.
    The U.S. biomedical research enterprise is the envy of the 
world for the excellence of our discovery and innovation. Our 
leadership is made possible because the overwhelming majority 
of researchers participating on N.I.H. grants, whether U.S. or 
foreign born, are honest, hardworking contributors to the 
advancement of knowledge that benefits us all.
    We must move effectively to root out examples where our 
system is being exploited, but make sure to preserve the 
vibrancy of a diverse workforce that has played a major role in 
the American biomedical research success story.
    But just like in sports, it takes more than a good defense 
to win at science. It also takes a strong and talented offense. 
So if you will allow me for the rest of my testimony, I would 
like to focus on the 21st Century Cures Act and many other 
proactive ways in which you and your colleagues are helping to 
bolster N.I.H.'s tradition of success.
    I spend a lot of time with early stage researchers. 
Wherever I go, I set aside time to hear directly from them 
about their dreams, their ideas and, yes, their concerns. I 
know you, too, have met many of them both in your home states 
and on your much appreciated visits to N.I.H.
    I think it is critical that we all ask ourselves, what are 
we doing to foster this next generation of discovery? And what 
can we do to help our Nation remain the world leader in 
biomedical innovation?
    I believe the answers could be said to lie in certain key 
areas that we could call the five keys to success in science 
today. They are: a stable trajectory of support; a vibrant 
workforce; computational power; new technologies and 
facilities; and most of all, scientific inspiration.
    The good news is that thanks to you--Mr. Chairman, you have 
outlined what has happened in the last three years and perhaps 
the fourth year about to happen--early stage researchers are 
now seeing a stable trajectory of support. That provides such 
an encouragement to tackle difficult, challenging, high risk 
projects.
    Your work over the last three years is helping us to begin 
to reverse a distressing decade long decline in N.I.H.'s 
purchasing power for research, which is carried out in every 
state of the Nation.
    This year, we expect at the end of Fiscal Year 2018 to fund 
more than 11,000 new and competing grants; the largest number 
in history. The 21st Century Cures with its total funding of 
$4.8 billion over ten years for four signature initiatives is a 
critical part of this.
    A second key to success is a vibrant workforce. Success 
cannot lie simply in boosting the number of grants made. It 
must also include increasing the number of creative minds that 
are receiving those grants. So have a look at a new metric that 
we are using to evaluate success.
    This shows the trend in the number of individual principal 
investigators supported by N.I.H. over the past fifteen years. 
As you can see, that number is once again growing nicely. Note 
the surge that occurs around 2016, a surge that reflects when 
Congress began to change the trajectory of N.I.H. support and 
shows how that investment is paying off.
    The third key to success is computational power. This 
probably would not have been on my short list in 2009 when I 
started as N.I.H. Director, but like so much else, biomedical 
research has been transformed by the recent explosion in 
computing power and all of the big data it is generating.
    For example, the BRAIN Initiative, which you supported 
through 21st Century Cures, has created new imaging tools that 
are turning out droves of amazing data. And there is also data 
generated by structural biology, and the microbiome and the All 
of Us Research Program are part of the Precision Medicine 
Initiative, also supported by the Cures Act.
    On May 6, all of us began enrolling one million people 
living in the United States. Today, we are going to hit the 
100,000 mark for volunteers. Nearly half of those are from 
communities historically underrepresented in medical research, 
providing a great opportunity to look at health disparity.
    To realize the full potential of these and other resources, 
we must also develop new technologies and facilities. Quite 
often, it is the technology itself that is driving the need for 
equally innovative facilities.
    Take the case of the new cell-based treatments, 
immunotherapy and gene therapy. Many involve removing cells 
from a patient's body using technology to reengineer those 
cells and then returning them to the patient.
    Many of our labs are not currently set up to handle these 
highly individualized processes, so it is crucial we make 
upgrades to keep pace.
    But now, onto my favorite: scientific inspiration. I can 
assure you that N.I.H.-funded researchers come to work every 
day full of innovative ideas and the wherewithal to see those 
ideas through, thanks to the Congress. Let me share just one 
example that really fits with the theme of this hearing, which 
is prioritizing cures.
    More than a decade ago, N.I.H. launched a special project 
on Spinal Muscular Atrophy, SMA, a tragic, inherited disease. 
As you see here, in its most severe form, it leaves babies 
floppy, unable to hold their heads up, feed well, and 
eventually even to breathe. Nearly all are deceased by fifteen 
months.
    Ten years ago, there was no treatment, but researchers had 
just discovered the DNA mutations that caused SMA. So N.I.H. 
supported more research, working closely with patient advocates 
and industry to move promising leads into therapeutic 
development.
    One of the most exciting comes from Jerry Mendell's team at 
nationwide Children's Hospital in Columbus, Ohio, which 
recently tested gene therapy for SMA in fifteen infants with 
severe disease. Again, these are infants not expected to 
survive more than fifteen months.
    They infused a viral vector designed to deliver the normal 
gene to the spinal cord, which is where the problem is and held 
their breath. Over the next few months, something truly 
dramatic happened.
    Like Evelyn Villarreal, who you see in this picture with 
her parents, 100 percent of the kids who got the highest dose 
of gene therapy were alive at twenty months. Nearly all could 
talk and feed themselves. And some, like Evelyn who is now 
three-and-a-half, not only can talk and walk, but she can even 
do pushups. Check out this video.
    [Video presentation.]
    Dr. Collins. I am very happy that Evelyn, her mom Elena, 
and her dad Milan, are here with us this morning. So please 
stand up, if you would, and say hello to the Members of the 
Committee.
    [Applause.]
    Dr. Collins. Evelyn, do you think you could do a twirl for 
us? I saw one earlier that looked pretty good; maybe a little 
too many witnesses. Well, does that not warm your heart?
    In closing, I am proud to lead N.I.H. at this time of 
unprecedented scientific opportunity and strong congressional 
support. The resources you have entrusted to us will be used to 
bring hope to untold numbers of patients and their families.
    We are the National Institutes of Health. But for many, 
like the Villarreal family, we are also the National Institutes 
of Hope.
    Thank you and we look forward to your questions.
    [The prepared statement of Dr. Collins follows:]
                prepared statement of francis s. collins
    Good morning, Chairman Alexander, Ranking Member Murray, and 
distinguished Members of the Committee. I am Francis S. Collins, M.D., 
Ph.D., and I have served as the Director of the National Institutes of 
Health (NIH) since 2009. It is an honor to appear before you today.
    Before I discuss NIH's diverse investments in biomedical research 
and some of the exciting scientific opportunities on the horizon, I 
want to thank this Committee for your sustained commitment to NIH to 
ensure that our Nation remains the global leader in biomedical research 
and advances in human health.
    As the Nation's premier biomedical research agency, NIH's mission 
is to seek fundamental knowledge about the nature and behavior of 
living systems and to apply that knowledge to enhance human health, 
lengthen life, and reduce illness and disability. As some of you have 
witnessed first-hand on your visits to NIH, our leadership and 
employees carry out our mission with passion and commitment. This 
extends equally to the hundreds of thousands of individuals whose 
research and training we support, located in every state of this great 
country, and where 81 percent of our budget is distributed.
    One of my personal priorities is developing the next generation of 
talented biomedical researchers. Last year, I shared with the Committee 
NIH's plans to build on our support for early stage investigators 
through a new initiative known as the Next Generation Researchers 
Initiative. NIH is developing evidence-based, data-driven strategies to 
assure that NIH investments are directed in ways that maximize 
scientific output. We are being aided in these efforts by an expert 
Working Group of the Advisory Committee to the Director, who will 
present recommendations in December 2018. But several important steps 
are already being taken: Institutes and Centers are placing greater 
emphasis on current NIH funding programs to identify, grow, and retain 
new-and early career investigators across these critical career stages. 
The Office of the Director is tracking progress across NIH in order to 
assess if these strategies are working. NIH remains committed to the 
development, support, and retention of our next generation of 
investigators.
    NIH is also committed to funding the highest priority scientific 
discoveries while also maintaining fiscal stewardship of Federal 
resources. Truly exciting, world class science is taking place. I would 
like to provide just a few examples of the depth and breadth of the 
amazing research NIH supports across the Institutes and Centers.
    The Brain Research through Advancing Innovative Neurotechnologies 
(BRAIN) Initiative is revolutionizing our understanding of the human 
brain, the most complex structure in the known universe. Launched in 
2013, this large-scale effort is pushing the boundaries of neuroscience 
research. Ultimately, these insights will have profound consequences 
for the prevention or treatment of a wide variety of brain disorders. 
By accelerating the development and application of innovative 
technologies, researchers are producing a revolutionary new dynamic 
picture of the brain that, for the first time, shows how individual 
cells and complex neural circuits interact in both time and space. This 
picture is filling major gaps in our current knowledge and providing 
unprecedented opportunities for exploring exactly how the brain enables 
the human body to record, process, utilize, store, and retrieve vast 
quantities of information, all at the speed of thought.
    This year, the BRAIN Initiative will support critical areas 
including data infrastructure and sharing, the BRAIN Initiative Cell 
Census Network (which is developing an atlas of brain cell types), the 
Team Research Brain Circuits Program, and human brain studies. In human 
studies, the BRAIN Initiative is advancing brain imaging and non-
invasive brain stimulation, and public private partnerships are 
investigating self-adjusting implanted brain stimulation therapies that 
are already showing promise. Ultimately, this will lead to an increased 
understanding of brain health, and a means of preventing brain 
disorders such as Alzheimer's disease, Parkinson's, schizophrenia, 
autism, and drug addiction.
    In April 2018, NIH launched the HEAL (Helping to End Addiction 
Long-term) Initiative, an aggressive, trans-agency effort to speed 
scientific solutions to stem the national opioid public health crisis. 
NIH has and will continue to support cutting-edge research on new 
treatments for the millions of Americans with opioid addiction, and for 
the millions more with daily chronic pain. Both pain and addiction are 
complex neurological conditions, driven by many different biological, 
environmental, social, and developmental contributors. To build on this 
understanding, NIH will: explore new formulations for overdose reversal 
medications capable of combatting powerful synthetic opioids; search 
for new options for treating addiction and maintaining sobriety; 
continue to research how best to treat babies born in withdrawal 
through our ACT NOW study; develop new non-addictive treatments for 
pain through the study of novel targets and biomarkers; and build a new 
clinical trials network focused on pain. NIH, in partnership with the 
Substance Abuse and Mental Health Services Administration (SAMHSA), 
will also study how effective strategies for opioid addiction and 
overdose reversal can be put into practice in places severely affected 
by the opioids crisis through the HEALing Communities study. Thanks to 
your support, all hands are on deck at NIH for this public health 
crisis.
    Another exciting area of continued investment is in cancer 
immunotherapy, in which a person's own immune system is taught to 
recognize and attack cancer cells. After years of research supported by 
NIH, immunotherapy is leading to cures of some cancers like leukemia, 
lymphoma, and melanoma.
    But other cancers, particularly solid tumors like colon, pancreas, 
breast, and prostate, have proven much less responsive. I am excited to 
tell you that some of those barriers may be ready to come down. Just 
last month, a team led by NIH's Dr. Steve Rosenberg announced a novel 
modification of an immunotherapy approach that led to a complete 
regression, most likely a cure, of widely metastatic breast cancer in a 
woman with this previously fatal form of the disease. As always, I must 
counsel patience--this immunotherapy success story for solid tumors 
involves very few cases right now, and must be replicated in further 
studies. But, without doubt, this woman's life-saving experience 
represents hope for millions more. As exciting as potential cures like 
this can be, NIH is focused on advancing not just cancer therapies, but 
also cancer care. I would like to tell you about an NIH-funded trial 
that beautifully illustrates the progress we are making in this area.
    Each year, as many as 135,000 American women who have undergone 
surgery for the most common form of early stage breast cancer face a 
difficult decision: whether or not to undergo chemotherapy to improve 
their odds. Now, thanks to a large, NIH-funded clinical trial, called 
TAILORx, we finally have some answers. It turns out about 70 percent of 
such women actually do not benefit from chemotherapy, and a genomic 
test of tumor tissue can identify them quite reliably. Clearly, it is 
best to spare women from the potentially toxic side effects of these 
drugs, if at all possible. Furthermore, the ability to limit the use of 
chemotherapy to the 30 percent of women who will really benefit can 
yield significant cost savings for our health-care system, as much as 
$1.5 billion a year.
    Indeed, figuring out what health approaches work best for each 
individual--and why--is the goal of another important NIH Initiative: 
the Precision Medicine Initiative (PMI). Precision medicine is a 
revolutionary approach for disease prevention and treatment that takes 
into account individual differences in lifestyle, environment, and 
biology. While some applications of precision medicine have found their 
way into practice over the years, this individualized approach is 
simply not available for most diseases. The All of Us Research Program, 
a key component of PMI, is building a national resource--one of the 
world's largest, most diverse biomedical data sets in history--to 
accelerate health research and medical breakthroughs, enabling 
individualized prevention, treatment, and care. All of Us will enroll 
one million or more U.S. volunteers from all life stages, health 
statuses, races/ethnicities, and geographic regions to reflect the 
country's diverse places and people to contribute their health data 
over many years to improve health outcomes, fuel the development of new 
treatments for disease, and catalyze a new era of evidence-based and 
more precise preventive care and medical treatment.
    Across the Nation, NIH has engaged ten large health provider 
organizations, six community health centers, and the Department of 
Veterans Affairs to be our partners in this ambitious study. The 
program has funded over thirty community partner organizations to 
motivate diverse communities to join and remain in the program, with a 
focus on those traditionally underrepresented in biomedical research.
    We began a robust, year-long beta testing phase in May 2017, during 
which each of our partners were able to test their systems and 
processes to ensure a good experience for participants and ensure that 
the security of the data systems was of the highest possible order. I 
am happy to tell you that All of Us launched nationally on May 6, 2018 
with events across the country to mark the program's open enrollment. 
As of August 15, 2018, almost 100,000 individuals have started the 
enrollment process, and over 50,000 have completed all the steps in the 
protocol. Of those almost 50 percent are from racial and ethnic groups 
who have been historically underrepresented in biomedical research.
    Following the national launch, we continue to improve and adjust 
the program based on participant feedback and emerging scientific 
opportunities and technological advances. We also are currently 
building the All of Us data resource, which is designed to be used by a 
broad range of researchers to study complex risk factors, support 
ancillary studies and clinical trials, and link to other large data 
sets. All of Us will be critical to realizing the promise of 
personalized medicine.
    We have never witnessed a time of greater promise for advances in 
medicine than right now. Your support has been critical, and will 
continue to be. Thank you again for inviting NIH to testify today. I 
look forward to answering your questions.
                                 ______
                                 
    The Chairman. Thank you.
    We will begin a round of five minute questions. As I 
mentioned earlier, we have a vote in a few minutes, but we will 
continue right through that, and pass the presiding 
responsibility around.
    First, to Evelyn and to her parents, thank you so much for 
coming. It is a wonderful story, and that is the reason we are 
so interested in the work that Dr. Collins and his associates 
do.
    Thanks to Dr. Collins's team for being here.
    Dr. Collins, let me ask you to talk a little more about 
some areas you mentioned. With all this new money, and it is a 
lot, a 30 percent increase in a short amount of time, there are 
three areas that, in my conversations with researchers around 
the country, they suggest that we could do a better job of, and 
maybe you already are and we just do not know about it. So let 
me tell you about those three areas and see what you say.
    Number one, support more young scientists. Now, you talked 
about it there. But the feeling is if whatever money, even if 
it is a lot of money is available only to the established 
figures, that it discourages the brightest of the youngest 
scientists who often do some of their best work of their lives 
in their early years.
    We have included that in our legislation that we passed. 
You have made a focus of it. So I would like to know, number 
one, about the progress you are making and what else you intend 
to do about making sure that a lot of this money is focused on 
young scientists.
    Number two, the peer review panels, some have said to me 
that the peer review panels are not as high quality as they 
once were. I do not know if that is true or not. The suggestion 
was made that anyone who receives an N.I.H. grant, and there 
are a lot of those, I think you said ten thousand?
    Dr Collins. Eleven thousand.
    The Chairman. Eleven thousand, has to sort of go into the 
jury pool and be eligible to be selected. They might not all be 
the very best, but be eligible to be selected for the peer 
review panel.
    The quality of the peer review panels would be my second 
question.
    The third question would be, I have heard some criticism 
that the proposals have become more conservative, and more 
bureaucratic, and longer. That at one time, proposals before 
the peer review panels were shorter, more succinct, and bolder.
    What about those three things? What are you doing about 
them? What is the validity of concern in those areas?
    Dr. Collins. Well, those are three wonderful questions and 
I am glad to respond because they resonate with things that we 
talk about and are doing things about at N.I.H.
    With regard to young scientists, totally agree with you 
that this is critical. This is the future and we have gone from 
2003 to 2015 through a tough time for those young scientists 
where N.I.H.'s purchasing power dropped way back and their 
likelihood of getting funded got to be to the point where many 
of them were really quite discouraged.
    We have benefited, of course, from congressional enthusiasm 
for N.I.H. over the last three years and that alone has helped, 
but we have actually prioritized the young investigators, what 
we call early stage investigators, to be the ones that we most 
want to be sure we are taking care of when they come forward 
with a new and wonderful idea.
    This year, in a program of next generation research 
initiative, which is actually part of 21st Century Cures, we 
expect to fund the largest number of early stage investigators 
ever; 1,100 of them who have never previously gotten a grant.
    We also have a very vigorous group, including some graduate 
students and post doctorates, and junior faculty, who are 
giving us additional ideas about how we could encourage those 
early stage folks. They will make a major set of 
recommendations to me in December, and I think that will add 
some additional new ideas about programs that we can do.
    We want to be sure that people not only see us as a place 
where they can bring their ideas, but they can bring bold ideas 
and we want to encourage that as well.
    Which is probably coming to your third, and I will come 
back to the second question, but the third question about 
conservatism in terms of applications, in terms of the kind of 
science that we fund. I also worry about that.
    We, at N.I.H., have been experimenting quite successfully 
in programs like the Pioneer Awards, which do not expect a lot 
of preliminary data, and a quite brief in the nature of the 
application, but need to propose something that is truly 
groundbreaking.
    With that program now having been in place for almost 10 
years, I can tell you that dollar for dollar, it pays off 
better than our traditional programs and many of the institutes 
are adopting a similar program. The General Medical Sciences 
Institute has moved almost all their portfolio into that kind 
of program, which is a different model and we think is very 
productive.
    Finally, I would say with regard to peer review, we agree 
that anybody who has a grant from N.I.H. ought to be willing to 
serve on peer review. We did a survey of that three years ago 
and discovered there were some exceptions.
    As of 2015, it is a condition of your grant award that if 
you are asked to serve in peer review, you are expected to say 
yes. And the numbers I looked at over the last couple of weeks, 
those who are receiving funding from N.I.H., about 80 percent 
of them are, in fact, now serving in that role.
    That includes some younger folks, who maybe the older 
emeritus folks do not recognize as being sort of the familiar 
faces they thought they would see on a peer review panel, but 
we need them to be there too.
    The Chairman. Thanks, Dr. Collins.
    Senator Bennet.
    Senator Bennet. Thank you.
    Dr. Collins, just along the lines of Chairman Alexander's 
first question, I remember you sitting at, I think, at this 
very table some years ago talking about the cost of the 
unpredictability of the funding that N.I.H. was getting at the 
time, and the difficulty of being able to recruit and sustain 
academic research if the funding was uncertain.
    Can you tell us today with more certain funding what 
difference that is making on the ground in these research 
institutions around the country?
    Dr. Collins. It has made an enormous difference. And again, 
I think the difficult period from 2003 to 2015 made it hard for 
investigators to be confident that they could tackle a program 
that was going to take several years to bear fruit. It made it 
hard for us at N.I.H., as project managers and as visionaries, 
trying to design something bold. Could we really be confident 
that was going to happen?
    Let me say that 21st Century Cures was a wonderful antidote 
to that providing a trajectory for funding for those four 
signature projects over ten years. We have almost never had 
that kind of confidence in the future, and that bill made that 
possible for us to see.
    But for the average investigator working in the laboratory 
to see the way in which this stability has crept into the 
circumstance, as opposed to the ups and downs, has given them--
and I talk to a lot of them every day--the confidence that they 
are in the right place, doing the right thing, and it is Okay 
to tackle something that is not going to get solved in a year 
or two.
    I might say the way in which this is happening is such a 
different landscape now than the world's worst moment for us, 
which was sequestration, where in March 2013, all of a sudden, 
we lost $1.5 billion on one very bad day. That sent ripples 
through the community that took a long time to recover from, 
but I think we are getting there.
    Now let me say, we are still, I am sorry to say, at the 
point where if you send a grant to N.I.H. your likelihood of 
getting funded is only about 20 percent. That is a lot better 
than the 15 or 16 percent it was, but we are looking forward to 
being able to see ways to continue to see that rise.
    Senator Bennet. Good. I think that is a real testament to 
Chairman Alexander and Ranking Member Murray's bipartisan 
support of this Committee at a moment when we are not getting 
much of that in the U.S. Congress demonstrates that you can 
actually get some things done.
    Dr. Collins. We are deeply grateful for that.
    Senator Bennet. Well, we are grateful to you.
    I sent you a letter with Senator Schatz and asked you a few 
questions about whether there is a consensus in the scientific 
community on whether our society is becoming addicted to 
technology and what the public health effects of social 
networking are.
    Just last week, the American Psychological Association 
released a study showing that in recent years, 20 percent of 
U.S. teens reported reading a book, magazine, or newspaper 
daily for pleasure, while more than 80 percent said they use 
social media every day.
    Additionally, it reported in 2017, it found that children 
eight years old and older spent 48 minutes a day on mobile 
devices, up from 15 minutes in 2013. Similarly, 42 percent of 
children eight years old and younger have their own tablets, a 
major increase from 7 percent in 2013.
    It seems to me clearly we need to prioritize some research 
here in these areas. Thank you for your response to the letter, 
but I wonder whether you could talk about what N.I.H. is doing 
to address these issues?
    Dr. Collins. Well, I will quickly tell you about a program 
that is funded by N.I.H, called ABCD, the Adolescent Brain and 
Cognitive Development Program.
    This has enrolled now more than 10,000 nine and ten year 
olds and is tracking them over the course of ten years to see 
what influences are happening to brain development, including 
screen time, including the use of social media, including drug 
access, and many other things, including brain images that will 
teach us something about what is happening to the wiring. That 
is going to be very useful in this regard.
    But let me ask, Dr. Bianchi, of the Child Health Institute, 
because they have recently held an important workshop on this 
very issue trying to design what the next research steps ought 
to be.
    Dr. Bianchi. Thank you for your question.
    There are really two issues. There are issues on early 
child development and then there is the issue of technology 
addiction later on and how it affects adolescents.
    NICHD has recently held a workshop in January that has 
examined some of the neuropsychiatric issues on technology and 
early brain development. We are particularly concerned about 
language development, reading comprehension, and also parent-
child interactions.
    We have come up with a number of recommendations to move 
forward with that and we are, of course, very interested in 
your legislation.
    Senator Bennet. Thank you.
    Thank you, Mr. Chairman.
    The Chairman. Thanks, Senator Bennet.
    I think the vote has started, so I am going to go vote and 
Senator Bennet, if you would chair the Committee. I will be 
back and we can swap the gavel.
    Senator Isakson.
    Senator Isakson. Thank you, Mr. Chairman.
    Dr. Collins, welcome. I want to add a comment, if I can, at 
the beginning rather than a question.
    My first engagement with you was at the National Prayer 
Breakfast when you demonstrated your gifted talent of playing 
classical music on the guitar, which to this day, was still one 
of the best performances I ever saw. But I knew then that you 
were a special person, and then with your success in the human 
genome, and all that you have done at N.I.H., we are blessed to 
have you.
    But I want to commend you on talking about the National 
Institutes of Hope. I have Parkinson's, and have had it, been 
diagnosed for six years. Evelyn, this child has a challenge and 
her family has a challenge. I am going to tell you about a 
challenge in our family in just a minute.
    But because you are the National Institutes of Hope, there 
are lots of people who have hope today that did not have it 
before primarily because you are changing attitudes in this 
country, both in the institution of medicine, as well as the 
patients who come in for help.
    I want to thank you for having such a positive, solution-
based favorable attitude toward research, toward cures, and 
toward the process that nothing is impossible if we just work 
at it. You do a great job and we appreciate it.
    Dr. Collins. Thank you, Senator.
    Senator Isakson. As far as Evelyn is concerned, my daughter 
Julie's best friend is named Julia Vitorello. She is a resident 
of, was a resident of Washington, DC. She is now a resident of 
Colorado.
    Her baby was born with Batten disease, which is a totally 
incurable childhood disease which terminates life somewhere 
around the age of ten or twelve. But it is a degenerative 
disease like some of the other diseases that have a lot of 
atrophy involved in them.
    She is now at Boston Children's Hospital undergoing a 
special treatment that has been designed by her doctors who 
have hope of using gene therapy as a way to transmit and I am 
out of my league now. I am a real estate salesman. I do not 
know about the human genome.
    But I know this. They are using that gene therapy through 
the spinal column to get the treatment to the place in the 
brain it needs to be and they are showing an amazing success. 
You referred to the gene therapy and some other things.
    Would you talk about the gene therapy for just a minute?
    Dr. Collins. I would love to, Senator. And thank you for 
your comments. That was most generous.
    My colleagues make this job for me the most amazing 
experience every day because of the talent that you see 
surrounding me and all the other folks who are not at the 
table.
    Batten disease is one of those incredibly tragic 
neurological conditions which is caused by genetic 
misspellings. And so, it is amenable to the idea of gene 
therapy, but to actually turn that into practice has been 
decades long and it is very exciting to see this is now 
starting to work in certain instances.
    You saw an example with Evelyn because the disorder that 
affects her, SMA, affects the spinal cord. For a long time, we 
thought that would be the hardest place you could possibly 
imagine getting your gene therapy to be delivered, but you have 
seen what has happened here; just an amazing experience for all 
of us to see how this is working.
    With Batten disease, likewise, you need to get the delivery 
into the brain and the spinal cord. Hence, in the protocol you 
are talking about, the delivery is into the spinal fluid, which 
then bathes the brain and provides that delivery. I do not know 
the precise status of that protocol.
    I was gratified, though, to see similar circumstances about 
Huntington's disease. Now, here is one of those incredibly 
troubling, dominantly inherited conditions. Woody Guthrie, one 
of my childhood heroes, had Huntington's disease.
    In the last few months, again with the gene therapy placed 
into the spinal fluid, there is clear evidence that they are 
able to reduce the amount of the toxic protein; an encouraging 
evidence that it is slowing or stopping the progression of the 
disease.
    Now, that was one of the ones that I thought might be the 
longest to ever yield up its secrets because of it being 
affected in the brain this way. But we are starting to see that 
happen.
    None of that happened without many, many years of hard 
fought progress and a lot of disappointments, but now I think 
gene therapy is really coming into its own.
    Senator Isakson. I agree and it is showing great promise, 
which we hope we will see one day, just like we are seeing in 
Evelyn right now.
    Evelyn, thank you for coming, by the way; my kids always 
got the shies just like Evelyn does.
    One last thing to talk about is what you talked about on 
the brain. The stimulation in the human brain is now being done 
to treat Parkinson's and other neurological diseases and making 
remarkable improvements and remarkable increases. The more we 
can continue to invest in that, the more we are going to invest 
in, not cures, but certainly ways to deal with some of the 
ramifications of neurological disease.
    I want to thank all my colleagues on the Committee who 
helped me working on the Neurological Disease Registry 
expansion under the 21st Century Cures bill to expand that 
registry, to expand our information for research.
    Thank you very much for being here today.
    Dr. Collins. We do appreciate that.
    Again, the BRAIN Initiative, one of the early results of 
this is going to be having a better wiring diagram of the brain 
so the deep brain stimulation, which right now works, but we 
are not exactly sure why. We will be able to do it much more 
precisely.
    Senator Isakson. Thank you very much.
    Senator Bennet [presiding]. Thank you, Senator Isakson.
    Senator Kaine.
    Senator Kaine. Thank you to Dr. Collins and to all. I 
especially want to thank you, Dr. Collins. You are a great 
Virginian and you highlighted a wonderful Virginia family when 
you talked about Evelyn Villarreal. She and her family are from 
Centreville, I believe. Is that correct? Very, very happy to 
have you here and to hear the story about the genetic therapy 
that has made such an advance with respect to children with 
SMA.
    It also highlights the importance of pediatric specific 
research. I came onto the Committee and I probably had an 
assumption that research into adult conditions could be just 
kind of scaled to pediatric conditions. And so often, they are 
very different.
    In 2014, I was proud to support the Gabriella Miller KIDS 
First Research Act, which increased funding for research on 
pediatric disease within the N.I.H. by taking a separate, non-
health related source of direct funding and putting it into 
pediatric research. And I think since that bill passed, it has 
directed about $55 to $60 million into pediatric conditions.
    There has also been improvements made for promoting such 
research in the 21st Century Cures Act to include the National 
Pediatric Research Network and the Global Pediatric Clinical 
Study Network.
    I would love it, Dr. Collins, if you could address this 
question.
    What promise do increasing research and the number of 
clinical trials in pediatric rare diseases or cancer hold for 
finding cures for diseases like SMA or like the childhood 
cancer that killed young Gabriella Miller when she was eleven 
years old?
    Dr. Collins. Well, I really appreciate the question. All of 
us at the table are deeply committed to advancing the cause of 
pediatric research.
    One of us happens to be a pediatrician and that is Dr. 
Dianna Bianchi. So I will ask her to address some of the points 
that you have raised, particularly about the Gabriella Miller 
KIDS First Research Act.
    Senator Kaine. Thank you.
    Dr. Bianchi. Thank you for your question. Always appreciate 
a focus on children.
    In fact, the N.I.H. funds $4.2 billion on pediatric 
research. Although we have child health in our institute name, 
research in pediatrics and pediatric conditions is done in 
virtually all of the institutes and we are all working together 
to make the best use of that $4.2 billion.
    We fulfilled a mandate of the recent Pediatric Research 
Network part that was in the Cures Act legislation by having 
four predominant networks that includes the IDeA States; the 
Pediatric Clinical Trials Network, which is focused on drugs, 
testing drugs in children; the Neonatal Research Network; and 
the Rare Disease Clinical Network, which is looking at over two 
hundred conditions.
    Those four networks are addressing many, if not most, of 
the conditions.
    Now the Gabriella Miller, we have had some successes in 
that area. I understand you knew Gabriella.
    Senator Kaine. I actually did not, but I know her parents 
very well. They were a great Loudoun County family.
    Dr. Collins. A wonderful family, I know them also quite 
well.
    Dr. Bianchi. The Gabriella Miller Network really creates an 
infrastructure so that researchers can collect large cohorts of 
biomaterials from children with conditions such as cancer and 
congenital anomalies.
    The infrastructure allows us to work at a very large scale 
and already has had successes. So we have a childhood cancer 
dataset that is already publicly available in pediatric Ewing's 
Sarcoma and we also have datasets that are available for 
congenital heart disease, cleft pallet, and diaphragmatic 
hernia. Researchers anywhere around the world can make use of 
that information.
    Senator Kaine. Thank you.
    Dr. Collins, one other question. You gave me an inspiring 
answer when you were before this Committee about a year ago--I 
used the analogy of President Kennedy saying we could be on the 
moon by the end of the decade, which seemed to many as science 
fiction, and yet it was doable and we did it--to ask you, could 
we, as a society make a pledge to be addiction free by 2030 and 
get there?
    You said not only could we, but we knew enough about 
addiction as long as we appropriately define what addiction 
free is, we should make such a commitment, and it was not a 
question about science or understanding. It was just really an 
issue of will and resources.
    I have continued to discuss that as I have traveled around 
the Commonwealth. Talk to me, if you can----
    Actually, I am right near the end of my time. This is 
probably going to be a long answer. I think what I will do is I 
will submit for the record, you did address it in your opening 
testimony. I would love to know some of the things that you are 
doing at the N.I.H. to really help us grapple with this 
problem.
    As you know, just last week, the statistics came out; 
72,000 Americans died of overdoses in 2017. Hundreds of 
thousands overdosed; 72,000 died. When I think that is more 
than the number of Americans that died in the entire Vietnam 
War, we are losing a war every year to despair and despondency, 
and your agency has a critical role in helping us figure out 
how to win that war.
    I will ask that question for the record to get status on 
current projects underway at the N.I.H.
    Dr. Collins. I would be happy to respond. We are very 
invested in this.
    The Congress gave us $500 million in the current fiscal 
year of additional funds to focus on the opioid crisis, and we 
are deeply engaged in that, and moving very quickly.
    Senator Kaine. Great, thank you so much.
    Thanks, Mr. Chairman.
    Senator Bennet. Thank you, Senator.
    Senator Cassidy.
    Senator Cassidy. Hello to you all. I would say gentlemen, 
but you too, Dr. Bianchi. Thank you all for being here.
    You probably know from previous kind of questioning, lines 
of questions, I have always been concerned about priorities in 
spending and so, just a couple of things as background.
    [Chart 1.]
    Senator Cassidy. The societal cost of disease here and you 
see that there is roughly, if this is disability life years 
adjusted and for my colleagues who may not be familiar with 
this, just an amalgamation. If somebody has an illness, how 
much do we lose in terms of productivity with an element of 
death. Then here is just the mortality. This is from 2015. The 
funding levels are from 2016.
    What we see as we look at societal costs of disease, there 
is roughly a correlation between how much it cost disease, how 
much it costs society, and the disability and the death rate it 
causes.
    I have two figures for obesity. One is how the CDC just 
says, ``These are the folks who die.'' And this is everybody 
for whom obesity is listed on the coroner's report knowing that 
obesity leads to a lot of other conditions that might be the 
primary cause of death, for example, heart disease.
    Can you hold up the other, please?
    [Chart 2.]
    Senator Cassidy. Here you see the N.I.H. funding and we see 
here is HIV, but obviously a lot for HIV. Here is diabetes. 
Societal cost. Although we spend a lot on diabetes, it is not 
as much. I am struck, though.
    What I want to emphasize is the obesity. Now, this scale 
cannot do justice to how much of a difference it costs society 
in terms of societal costs of obesity relative to funding. So 
there is the N.I.H. funding by disease where it is $965 million 
even though it costs us $190 billion.
    Again, it costs society, obesity, $190 billion, but we are 
spending $965 million. The size of the bubble represents how 
much money we are spending upon it.
    Can you hold up the racial disparity issue?
    [Chart 3.]
    Senator Cassidy. As some of you may know, I worked in a 
public hospital in Louisiana with the uninsured for thirty-five 
years and you cannot help but notice that there is a racial 
difference in obesity.
    If you look at race, any mention of obesity on a death 
certificate, African Americans have a much higher rate of 
obesity. American Indian or Alaskan Native, here is white, here 
is Asian Pacific. I think if we put Samoans, though, it would 
bend up like that. So there are some clear racial disparities 
associated with obesity.
    My question, is it just a function of how we appropriate 
money? Because it does seem that obesity as a primary illnesses 
is underfunded relative to the societal cost.
    Again, $190 billion societal cost, $965 million in contrast 
with some other diseases with far less societal cost, but far 
more N.I.H. funding, Dr. Collins.
    Dr. Collins. Well, Senator, it is nice to have another 
iteration of a conversation we have had over two or three 
years. I appreciate your perspective on obesity, which I 
totally agree, is an enormous public health challenge for our 
Nation.
    Senator Cassidy. By the way, can I just for those who may 
not know, obesity is implicated in Alzheimer's, implicated in 
heart disease, implicated in cancer. So although it may not be 
primary, it is the match that starts the fire for a lot of 
other diseases.
    I am sorry to interrupt.
    Dr. Collins. No, that is quite all right.
    I think your point is taken. The question that we, at 
N.I.H., are always wrestling with--and you have seen the way we 
have played this out in our strategic plan that we put forward 
a couple of years ago that tried to really articulate how we 
set priorities--is this balance between public health need and 
scientific opportunity.
    I think with obesity, we would all agree that the problem 
is a multi-factorial one. That there are many aspects of this 
that relate to things that N.I.H. probably cannot control in 
terms of diet, lifestyle, even the built environment, and so 
on. We are studying those things pretty intensively.
    In terms of interventions, though, to do something about 
this epidemic, which is a fairly recent one, it does not look 
as if a medical therapy is on the edge of happening. And so, it 
is a bit of a different circumstance than, say, HIV/AIDS where 
we have a vaccine.
    Senator Cassidy. If I may interrupt, Dr. Collins, in all 
due respect. In the past, you have told me, and I will not 
mention the institutes, but you have said, ``Well, we do not 
really fund that because we are really not on the cusp of great 
advances.''
    I go speak to the director of the same, without mentioning 
your name, and he says, ``You have got to be kidding. We have 
so much opportunity here.'' That was kind of repeated several 
times.
    If I spoke to obesity researchers, they may start speaking 
about microbiomes, and leptin, and all this other stuff that 
again, kind of quickly passes my level of knowledge.
    But it does seem to be self-filling that if you say, ``We 
are not going to fund it because we are not ready to go to 
primetime in our research,'' you never go to primetime in your 
research because you never have the requisite prefunding.
    Dr. Collins. I think we are ready to go to primetime in 
research with obesity. It is a question of where are the 
scientific opportunities.
    You mentioned the microbiome. That is certainly a very 
powerful one. Clearly, learning things there plays out both in 
terms of obesity and diabetes, for which a big investment is 
being made.
    Although, some of that research might not actually score as 
obesity; it might score as it is a diabetes project or it is a 
nutrition project. Some of this, therefore, is just the 
bookkeeping part. But I take your point.
    Again, I think this is something we worry about every day 
when we meet as institute directors around the table on 
Thursday morning. Are we setting our priorities properly?
    Your input has been very helpful in that regard.
    Senator Cassidy. I would just suggest that we begin to 
focus more upon obesity, which seems to be an outlier in terms 
of lack of funding relative to societal cost.
    I now defer to whichever of my colleagues on the other side 
of the aisle is due.
    Senator Collins [presiding]. Senator Warren.
    Senator Warren. Thank you.
    The National Institutes of Health funds this country's top 
researchers and doctors. N.I.H. grants fuel medical 
breakthroughs, help universities pursue cutting edge science. I 
want to talk about money, because I understand N.I.H. needs 
money to be able to do its work.
    The vast majority of the N.I.H.'s funding comes from 
taxpayers. But in 1990, Congress established the Foundation for 
the National Institutes of Health, a nonprofit foundation that 
solicits private donations to support N.I.H. research. That 
means that if a drug company, or a device company, or a big 
tech company, or a lobbying firm wants to fund N.I.H. research, 
they can do so by donating to the N.I.H. Foundation.
    Dr. Collins, according to the most recent list of donors, 
the top six largest contributors to the Foundation for the 
N.I.H. are all drug companies. Each of these drug companies has 
donated to the Foundation every year for at least the past 
fifteen years. Let me just ask this question.
    Do you agree that science should be setting the agenda at 
N.I.H., and not donors?
    Dr. Collins. Absolutely.
    Senator Warren. Good.
    I understand that is how it is supposed to work. The N.I.H. 
comes up with a plan based on science and the Foundation gets 
donations to fund it, but when you have your hand out for cash, 
it is sometimes possible that these lines get blurred.
    The N.I.H. recently canceled a study of the health effects 
of alcohol consumption following an internal investigation that 
revealed that the alcohol industry was not only funding the 
study, but that the study had been set up to deliver the 
results the industry wanted.
    This is not even the only case this year that has raised 
ethical questions.
    In April, you pulled the plug on a plan to take hundreds of 
millions of dollars from drug companies that make opioids, some 
of which are under investigation for causing the opioid crisis 
in the first place, and using that money to fund a study to 
treat addiction.
    Let me ask this question, Dr. Collins. If these donations 
from industry are raising so many ethical questions, why should 
N.I.H. accept them at all?
    Dr. Collins. Well, we are thinking a lot about this in the 
wake of the examples that you have just cited. But as N.I.H. 
director of the last nine years, I can also cite you some 
examples where this kind of partnership with industry has 
actually made science move faster than it otherwise would have.
    Take the Accelerating Medicines Partnership, a project 
which involves ten pharmaceutical companies working on 
diabetes, on Alzheimer's disease, on rheumatoid arthritis, and 
very recently adding Parkinson's disease to that.
    In those instances, this was all precompetitive research. 
The data was immediately accessible. It brings around the same 
table scientists from both public and private sectors who 
design together what the research ought to be, building on the 
strengths of both groups. And it advances the cause of science 
more rapidly than might otherwise happen.
    There are no strings attached to the money that is provided 
by the drug companies, basically, that goes to the Foundation 
for N.I.H. It is used to support this program that is totally 
public about what we are doing. I would defend that. It has 
been a very good thing.
    What we need to be careful about, and which has, I think, 
caused us to stub our toe here a couple of times, is a 
circumstance where the source of the funds has a vested 
interest in a particular outcome of the study.
    We have started a recent study on cancer immunotherapy that 
Dr. Sharpless is leading. Again, involving industry input, 
trying to identify what are the biomarkers that indicate 
whether immunotherapy is going to work. Everybody wants to know 
the answer to that. Nobody has a stake in what the answer is 
going to be. Only that we need the answer. This is a really 
good example of how to work together.
    We just have to be thoughtful about exactly what the design 
looks like.
    Senator Warren. I appreciate that and I am really glad you 
are working to address the ethical landmines in this area.
    I think the N.I.H. should be getting more funding, but I 
will be blunt. If drug companies and rich donors want to chip-
in for more N.I.H. research, they should do it through their 
taxes like everyone else. I would be happy to write the bill to 
bump up their contributions.
    But here is the bigger issue. Forcing an agency to beg for 
contributions for money just to carry out its essential mission 
is a glossy invitation for corruption.
    I believe it is time to end the influence of corporate 
money in Washington, and that means calling it out and shutting 
it down in whatever form it takes.
    Thank you very much. I appreciate the work all of you are 
doing.
    Senator Collins. Thank you.
    As luck would have it, I now not only get to be Chairman 
for a brief time, but I am up next for questions.
    [Laughter.]
    Senator Collins. Dr. Collins, it is always great to see 
you. I continue to claim you as my cousin and I hope you will 
not disabuse others.
    Dr. Collins. I am honored to be claimed.
    Senator Collins. The 21st Century Cures Act provided 
multiyear funding for the Regenerative Medicine Innovation 
Project.
    At MDI Biological Laboratory in Maine, researchers are 
working with a team from Jackson Labs in Maine and the Maine 
Medical Center Research Institute in an N.I.H.-led effort on 
kidney regeneration--Dr. Hodes may want to comment on this 
also--to address the high health care costs associated with 
treating chronic kidney disease.
    I visited the Maine Medical Center Research Institute, and 
it is absolutely fascinating the work that is going on.
    Could you tell us whether you are seeing any results yet 
from the Regenerative Medicine Initiative? I know it is early.
    Dr. Collins. I would love to talk about that and appreciate 
that this was included in 21st Century Cures as one of the four 
initiatives with specific call outs for extra funding.
    Certainly, this idea of being able to build whole organs 
from stem cells is one of the things that has really 
electrified a lot of the community. You could call this tissue 
engineering. What is happening with hearts and with kidneys is 
particularly of interest.
    If I had thought to put it in my briefcase today, I could 
have brought you a little kidney on a chip that has actually 
been synthesized by a different group, but very much working 
with the folks in Maine as well, because this is a very 
integrated community.
    The idea that we could figure out the appropriate kind of 
signals to send a stem cell that might have been derived from 
your skin and convince it that it should become your next 
kidney seems like science fiction, but maybe not so much.
    So far, these are pretty small renditions, but I have seen 
some of these that actually have a bit of a blood circulation. 
And even, if you will pardon me, can make a little bit of 
urine. So we are on the path here.
    Ultimately, what we hope is this could become an 
alternative to the need for a transplant for somebody whose 
kidneys have failed. And, of course, along the way, we learn a 
lot about normal kidney biology that maybe can keep peoples' 
kidneys from failing because we will have better signals about 
how to prevent that.
    Your group in Maine is a very important one in this effort. 
I am glad you have been by to see them.
    Senator Collins. It truly is miraculous work that they are 
doing and it is so exciting to me.
    As you are well aware, Dr. Collins, I have been the Founder 
and co-chair of the Senate Diabetes Caucus and the Alzheimer's 
Disease Task Force for many, many years.
    Dr. Collins. Yes.
    Senator Collins. As our population is growing older, we are 
seeing an increase of incidents in both those diseases.
    There is also some intriguing science that suggests that 
there may in some cases be a link between the two diseases as 
well as cardiovascular disease.
    Could you tell us what kinds of findings you are seeing in 
that area and what promising research is underway?
    Dr. Collins. That is a great question. I am going to ask 
Dr. Hodes----
    Senator Collins. That would be great.
    Dr. Collins.----Our international expert on Alzheimer's who 
also knows a lot about diabetes to respond.
    Senator Collins. Thank you.
    Dr. Hodes. Thank you for that question, Senator Collins.
    There has been extensive collaboration with investigators 
interested in diabetes and those in neurodegenerative diseases 
such as Alzheimer's and related dementias. It has taken several 
forms and areas.
    It has been known for some time, for example, that diabetes 
is a risk factor for Alzheimer's disease. There have been 
metabolic parallels and similarities between diabetes and what 
goes on in the brain. In fact, some have called Alzheimer's 
disease a Type 3 diabetes because of an inadequate effect of 
insulin.
    It is perhaps most graphically translated now into a 
clinical trial that is ongoing using an intranasal route for 
introducing insulin to the brain to look for its impact on 
progression of Alzheimer's and cognitive decline.
    At the basic science level and now translated into real 
clinical trails, very much aware of the commonalities and ways 
in which we have to borrow and form across disciplines and 
across silos in order to best accomplish our goals.
    Senator Collins. Thank you very much.
    Senator Hassan.
    Senator Hassan. Well, thank you very much, Madam Chairman.
    Good morning to this extraordinarily distinguished panel. 
Thank you all for being here and thank you for the work you do.
    As you know, Dr. Collins, the fentanyl, heroine, and opioid 
epidemic is ravaging my State of New Hampshire and communities 
across our country. I was very proud to work with the rest of 
the New Hampshire delegation to secure a truly significant 
increase in funds for the Granite State to use for prevention, 
treatment, and recovery through the Substance Abuse and Mental 
Health Services Administration's State Opioid Response Grants.
    Now, New Hampshire is receiving $23 million for Fiscal Year 
2018; before that, it was $3 million. So we think there is 
potential to really have an impact on the ground.
    I think it is really important that we stay focused on 
making sure that the hardest hit states, the states with the 
highest mortality rates, get the concentration of funds they 
need.
    But we also need to make sure that we are supporting 
science here because we need more and better ways to treat 
addiction and also to manage pain. It is a critical part of 
curbing the opioid crisis and I appreciate the conversations we 
have had about it.
    I also appreciate very much the work the N.I.H. is doing on 
the HEAL Initiative to advance this science. When you were 
before this Committee last, you explained that you needed more 
flexibility from Congress to allow the N.I.H. to fund research 
on the opioid epidemic more quickly and efficiently.
    Since that time, I have been really pleased to work with 
Chairman Alexander, with Ranking Member Murray, and Senator 
Young to introduce the Advancing Cutting Edge, ACE, Research 
Act to give the N.I.H. the flexibility it needs to quickly 
advance research on new treatments and non-addictive 
painkillers by providing them other transaction authority that 
we have talked about.
    Dr. Collins, how will the other transaction authority 
provided by the ACE Research Act help the N.I.H.'s work on the 
opioid epidemic including through the HEAL Initiative?
    Dr. Collins. Well, I appreciate the question and your 
support of this other transaction authority. Let me explain why 
it would be so useful and why the timing is really kind of 
urgent right now.
    Of the HEAL Initiative that you mentioned, HEAL standing 
for Help End Addiction Long-term. One of the projects that we 
are most excited about, which is truly ambitious, is to see if 
we could identify maybe three places in the Nation where a 
particularly hard hit circumstance is happening with opioids.
    Then bring together in a way that has not happened before, 
but as a research enterprise, all of the players in that--the 
primary care doctors, the emergency rooms, the police, the fire 
departments, the criminal justice system, all of the other 
support systems, the state health departments--and see what 
could we actually do if everybody worked together in a 
coordinated way to tackle this problem? No single one of those 
is going to be able to be successful in ending this terrible 
national crisis.
    To be able to do that, which has never really been 
attempted before, having the kind of flexibility where we could 
actually reach out and identify partners who maybe have never 
written an N.I.H. grant and say, ``We want you.''
    Senator Hassan. Right.
    Dr. Collins. Also have a very active role at N.I.H. 
managing this effort in a fashion which, with grants, sometimes 
we cannot do.
    It would allow us to go faster and more effectively. We are 
going to try to do this anyway, but if we had other transaction 
authority, maybe in the next month, it would make a big 
difference in our ability to carry out that part of the HEAL 
Initiative.
    Senator Hassan. Well, I thank you for that. I am glad to 
see the bill passed the House and I hope the Senate will act 
soon on this----
    Dr. Collins. I do too.
    Senator Hassan. ----Along with the entire opioid package 
that we passed out of this Committee.
    I want to go to one other New Hampshire issue, if I may, 
but again one that has applications all across the country.
    Families in my state continue to have questions about what 
PFAS contamination in drinking water means for their health and 
the health of their children. Once used for a variety of 
commercial and industrial applications, PFAS have seeped into 
water tables in many places, including New Hampshire.
    There is a critical need to better understand and address 
any potential adverse health effects the contaminants may have 
on our communities.
    Dr. Collins, what is the N.I.H. doing to study these 
chemical compounds and their potential health effects on 
Americans?
    Dr. Collins. Well, this is a significant environmental 
concern and I know in New Hampshire, there has been even a 
public discussion about it in Exeter that the E.P.A. came and 
led. Michigan is very much also caught up in this, particularly 
around Kalamazoo.
    Senator Hassan. Right.
    Dr. Collins. This is the kind of a substance that has a 
very long half-life. It is not naturally occurring, but has 
found its way into many groundwater and water supplies because 
of manufacturing of things such as carpet cleaners and so on.
    In terms of the environmental risks, we really do not know 
enough about the human risks to be very confident in saying 
whether this is really a big deal or whether actually we humans 
are able to handle it. We do know in animals, there is an 
association with immune consequences and maybe other things 
including, perhaps, cancer. But the human data is very 
uncertain.
    There is a big project which D.O.D. is funding which our 
NIEHS, National Institute of Environmental Health Sciences, is 
part of along with the C.D.C.'s ATSDR. That is going to, I 
think, provide the kind of data that we currently do not have, 
at least in terms of the epidemiology of what is the 
relationship of exposure and to human medical problems.
    We desperately need more information of that sort.
    Senator Hassan. I thank you and I agree with that. And I 
thank you for allowing me to go over, Madam Chairman.
    I am going to follow-up just to pinpoint any other gaps in 
research that you all might see, and I appreciate very much, 
again, all your work.
    Dr. Collins. Be glad to do it.
    Senator Collins. Thank you.
    Senator Smith.
    Senator Smith. Thank you, Madam Chairman.
    Thank you very much all of you for being here today. It is 
a very interesting panel. Though as is often the case, we are 
kind of coming and going from votes.
    If I have a moment, I would like to follow-up on the 
questions that Senator Hassan started. But I would like to 
start, actually, with something different.
    I want to start out by saying I really believe in the power 
of innovation in biomedical research. Coming from my home State 
of Minnesota, which is such a center of excellence both at the 
University of Minnesota and also Mayo Clinic.
    Senator Collins was talking about the power of regenerative 
medicine, which is also something that we have been working on 
intensely in Minnesota, especially through Mayo Clinic. So I 
believe very strongly in that.
    But I also believe that if people cannot afford the 
therapies and the medicines that we are imagining, that we are 
creating, then we have a real problem. I have to tell you that 
this is the No. 1 issue that I hear about from Minnesotans, 
whether it is figuring out how to pay for a therapy like 
insulin, which has been around for 100 years, to figuring out 
how to pay for the most recent cancer breakthrough medicines. 
It is a huge problem.
    A lot of these therapies, of course, have been created 
because of help from the National Institutes of Health. I am 
told that every one of the 210 new drugs approved by the FDA 
between 2010 and 2016, N.I.H. contributed to.
    What happens, of course, the cost of innovation is often 
the reason why medicines cost so much. Yet, in some ways, I 
think, taxpayers feel like they are paying twice. Once for the 
support to N.I.H. and then once again when they are asked to 
pay for these exorbitantly priced medicines when they show up 
at the pharmacy.
    Tell me a little bit about how you see the role of N.I.H. 
in helping to make sure that we do not only have innovation, 
but we also have innovation that people can afford.
    Dr. Collins. Obviously, this is a source of much discussion 
and much concern. I think you are echoing a lot of the views of 
the public about how this drug pricing issue is going to be 
wrestled to the ground and make it possible for people who need 
access to obtain that.
    We, at N.I.H., as you quoted this recent study, just 
published in the ``Proceedings of the National Academy of 
Sciences of the United States of America,'' where Fred Ledley 
and colleagues looked across a five-year or a six-year period 
and said every single one of the FDA-approved drugs in that 
timetable were based upon basic science discoveries that N.I.H. 
has supported.
    Some of those were basically to discover, ``Here is a drug 
target,'' and then a company went and made the drug that hit 
that target. So it is not as if we basically started making 
pills and somebody else----
    Senator Smith. There is a difference between 
commercialization and basic research, which I understand.
    Dr. Collins. I think you could say that the system in the 
United States, this ecosystem between basic science, much of it 
supported by N.I.H., and commercial application has been the 
reason that we have been so successful in making medical 
progress.
    But the prices are certainly a concern.
    We do not have a lot of levers to pull in terms of direct 
influence on how a price is set for a newly innovated kind of 
therapeutic. What we do, and what we can do more of now because 
science is going forward, is to make it possible for the 
successes to happen more often.
    One of the reasons drugs are so expensive is that the 
failure rate for a company trying to get something across the 
finish line is about 99 percent. And so, when you finally get 
something that works, you have all of that other stuff that you 
have spent money on that got you nothing; that has to be 
somehow accounted for.
    At the National Center for Advancing Translational 
Sciences, which is part of N.I.H., we are identifying the areas 
that lead to that high failure rate systematically in coming up 
with new technologies to make that less likely to happen.
    If the success rate was just 5 percent instead of 1 
percent, it would make a huge difference in the overall 
financial circumstances that companies face. We are pushing as 
hard as we can on that. That is probably our best contribution.
    Senator Smith. Well, I think that is an important issue for 
us all to work on together. It is basic access to these 
incredible therapies that are being created is fundamental to 
whether our health care system works at all.
    For those of us who watch this and try to understand it, 
and we understand what you are saying, but we also see that 
these big companies are making a ton of money, and yet, we are 
all paying. That is, I think, the fundamental issue that I am 
grappling with and trying to find solutions to.
    I would like to be able to--because innovation is so 
important and affordable drugs are so important--I would like 
to be able to work together on that.
    Mr. Chairman, I am out of time, but I would like to submit 
to the record and for follow-up a question having to do with 
what Senator Hassan was talking about.
    In Minnesota, we call it ``diseases of despair''. The 
significant uptick, 40 percent increase in suicide, and other 
diseases related to behavioral health, and opioids, and 
addiction. What we can do and how we can work with N.I.H. on 
that.
    Dr. Collins. Glad to.
    Senator Smith. Thank you.
    The Chairman [presiding]. Thank you, Senator Smith.
    Senator Jones.
    Senator Jones. Thank you, Mr. Chairman.
    Thank you, Dr. Collins, and the whole team for being here 
and for the incredible work you do that touches on every family 
in America. I really appreciate that.
    A couple of weeks ago, I had the privilege of meeting with 
some of the leaders at the University of Alabama in Birmingham, 
which I consider to also be one of the leading institutions of 
not only higher learning, but research in the country.
    Specifically not only have I met with them with a 
comprehensive cancer institute, and all the work that they are 
doing there, but I had a chance to talk about their precision 
medicine program.
    I know that everyone is excited about the All of Us 
Research Program because precision medicine truly has potential 
to be a game changer for delivering the right treatment to the 
right person at the right time. I am so happy that Alabama is 
playing a role.
    Dr. Collins, just a very general question, what is it 
Congress can do other than just continuing to try to fund at 
the levels--and I also commend Chairman Alexander and Ranking 
Member Murray about the work on this--is there something else 
specifically that we, as Members of Congress, can do to really 
help promote and accelerate the use of precision medicine in 
this country?
    Dr. Collins. I appreciate you raising this issue and 
mentioning the All of Us programs.
    In response to your ``what could we do?'' maybe it would be 
useful, in fact, for Congress to become an ally with N.I.H. in 
encouraging people to take part in this unprecedented national 
experiment where we are asking 1 million people to sign up. I 
think we mentioned, we just hit 100,000 today. So we have a 
little ways to go, but it is a really wonderful start. I 
appreciate the way in which UAB is a critical part of this 
partnership in the south.
    We can have people sign up either by direct volunteer, 
where they basically get online, JoinAllOfUs.org and sign up. 
Or, if they are nearby to one of the health provider 
organizations that is a partner with us and get their care 
there, they can sign up in that fashion.
    We are hoping to see this really go forward quite quickly. 
And any kind of assistance we could have in terms of local 
events to raise the enthusiasm for this.
    This is taking what we have learned from a program like 
Framingham, which taught us an awful lot about cardiovascular 
disease, and extrapolating it by about a factor of 40 in terms 
of the size, in covering all diseases, not just cardiovascular. 
Everybody sitting at the table has a stake in all of us turning 
out. We will enroll children starting next year as well.
    Senator Jones. I appreciate that and I will tell you, even 
before you said that, one of the things that I discussed with 
them at UAB was that at some point in the very near future that 
my wife and I will go, and we will sign up, and we will try to 
make an event of that. I will encourage all of my colleagues to 
do the same.
    Let me move on to one other question that I had and you 
touched on this earlier in your testimony in response to a 
question. That is about developing the next generation of 
talented biomedical researchers, which is an extraordinary 
effort, and I applaud that effort.
    But one of the things that I am concerned about is trying 
to reach into underserved communities. It seems that we are 
missing such talent that is out there whether they are 
researchers, or whether they are doctors, or lawyers.
    What can we do as part of the programs that we have got now 
to specifically reach into underserved areas to try to grab 
that talent out and give them that extra boost that they need? 
Because they do not always have the same chances as some of the 
kids in the more urban areas and schools that have a lot more 
money.
    Dr. Collins. Well, I really appreciate that point because 
this is an area of great interest and concern.
    N.I.H. has been working for decades in trying to increase 
the participation in our research workforce by people from all 
different backgrounds. And frankly, we have not been that 
successful in many of those decades in terms of making this 
happen. Our workforce is still underrepresented when it comes 
to African Americans, and Latinos, and Native Americans.
    But we have a couple of new programs that are now underway 
for about three or four years that are starting to show 
promise. One of them is to recognize that a lot of that talent 
does not necessarily end up in a research intensive four year 
college environment, but has the interest in getting involved 
in research.
    The thing that really makes that interest turn into a 
reality is the chance to take part in a real research project. 
Not hearing about it in a lecture hall, but actually doing 
research yourself.
    The program called BUILD, which we have started three years 
ago, is a partnership between universities that have a lot of 
underrepresented groups in their student body, but do not have 
the research opportunities that would really benefit. They 
partner up, with some funds from us, with institutions that do 
have those research capabilities to give those talented folks a 
chance to see what that is like.
    The other thing that is often missing is mentoring. If you 
do not see anybody who looks like you who is a role model, it 
is a lot harder when you hit a bump to imagine that this is 
your future.
    We set up a whole National Research Mentoring Network to 
connect people up. If you do not have somebody down the hall 
from you, well, maybe there is somebody in your town, or even 
in your state, or even just somebody you can talk to on the 
phone who has lived the life that you are trying to live. That 
seems to be a big encouragement too.
    We are evaluating this at every step along the way. I know 
this is a hard problem. I am not going to declare victory yet, 
but I am seeing real progress.
    Senator Jones. Well, thank you very much and thank you for 
your efforts. Thanks to all the Committee. I see my time is up, 
Mr. Chairman. I will probably have a couple of questions 
particularly about infant mortality and maternal mortality, 
which I think is something that is going underreported today.
    Thank you, Mr. Chairman.
    The Chairman. Thank you, Senator Jones.
    Senator Bennet.
    Senator Bennet. Thank you, Mr. Chairman. I just had a 
couple of remaining questions.
    Dr. Collins, after we passed 21st Century Cures, we worked 
on and were able to pass, thanks to the Chairman and the 
Ranking Member, the RACE for Children Act as part of the FDA 
User Fee package. I know that NCI has been collaborating with 
the FDA on the implementation.
    As you know, the bill directs pharmaceutical companies to 
study some of the most innovative cancer drugs for children 
when treatments are effective for adults and that may be a 
benefit for children. Some of the treatments maybe 
immunotherapies that use the body's own immune system to fight 
cancer. I understand that some of these therapies have been 
successful in treating certain pediatric cancers, yet other 
approaches have not been as effective.
    I wonder whether you could talk a little bit, Dr. Sharpless 
actually, about what NCI is doing to ensure children will 
benefit from promising advances in cancer immunotherapy.
    Dr. Sharpless. Thank you. This is an exciting area.
    As you alluded, there is a lot of progress going on in 
cancer research. A lot of new therapies have become available; 
a lot of excitement, a lot of new targets.
    But because of the structure of the commercialization of 
novel therapies, there is sometimes a disincentive, actually, 
to test these therapies in children.
    I think the RACE Act was laudably intended to encourage 
pharmaceutical companies to develop their drugs for pediatric 
use, in addition to adult use, when the target was relevant in 
children. I think it is a smart way to do it.
    I think it is not onerous on the drug companies. It does 
not hurt innovation, but it still provides a real emphasis on 
childhood cancer, which is an area where we had seen a lot of 
progress, but we still need a lot more.
    The RACE Act directed the NCI and the FDA to work together 
to develop a list of these relevant targets and that list is 
now developed through a series of meetings between the NCI and 
the FDA. It has been published online and it is seven pages of 
molecular targets that, if you are making a drug to this 
target, you have to have a plan to test it in children.
    Now, we eagerly await to see how this is implemented. We 
have every expectation the pharmaceutical companies will comply 
with this law and will really change their practices.
    Senator Bennet. Well, that is good to hear. Thank you very 
much.
    Finally, Dr. Collins, appreciate the update you provided on 
the Precision Medicine Initiative, particularly with respect to 
the All of Us Research campaign you were talking about. Saying 
it is going to give researchers a lot more data to predict 
prevention and treatment needs.
    As we begin to think about the future of precision 
medicine, I just wanted to know whether you think N.I.H. needs 
additional authorities to keep up with the fast pace of 
science.
    Researchers in Colorado have been at the forefront of some 
of these biomedical advances. There are more than 720 
biomedical companies in my state employing almost 160,000 
Coloradans through direct and indirect jobs, many of which, 
almost all of which actually pay extremely well.
    When we think about the hope of personalized medicine and 
the level of innovation we are seeing, what is the best way for 
us to follow-up on 21st Century Cures as we think about it?
    Dr. Collins. Again, I think what the 21st Century Cures 
bill provided over a very thoughtful two years of selecting and 
hearing from various stakeholders about what would be most 
useful did, in fact, incorporate from our perspective, a number 
of legislative authorities that we greatly value.
    There was a question from Senator Hassan about this other 
transaction authority being granted, our ability to use that in 
the common fund and to use it in the All of Us Precision 
Medicine Initiative has made a lot of difference in the ability 
to move quickly.
    We would actually be grateful to have an even broader 
authority for other transaction authority in other places. The 
Chairman and I have talked about that. As we have gotten more 
experience with it, it is perhaps more rapid moving. Maybe 
people worry it is a little bit riskier because it can be rapid 
moving, but in certain instances, has made all the difference. 
So that would be an area.
    Another area if we had the opportunity to expand our 
authorities, when we get to a place where we really have an 
opportunity to do an assessment of a precision medicine 
strategy, it is not interesting to the private sector. The 
ability to carryout Phase 3 trials within the National Center 
for Advancing Translational Sciences would be of value. At the 
present time, that is not something we have the authority to 
do. That is just another example of something that could help 
us.
    But again, I cannot say enough about the way in which 21st 
Century Cures basically took our list of things that we hoped 
to be able to do and pretty much checked the boxes one by one, 
and has made it so much more possible for us to move quickly.
    Senator Bennet. Thank you, and thanks to everybody.
    I actually cannot leave. I cannot resist asking Dr. Fauci, 
before we go, what are you worrying about these days?
    Dr. Fauci. Thank you for that question, Senator.
    As you probably would have guessed, I always worry about 
the emergence of an infectious disease such as we usually use 
the prototype of pandemic influenza, a respiratory illness that 
spreads rapidly and that has a high degree of morbidity and 
mortality.
    It is for that reason that I have been, and my colleagues 
and I have been, working on that for the last at least a 
decade, but more intensively over the last couple of years, on 
the development of a universal influenza vaccine that would not 
only be important to obviate the need to get a vaccine every 
single season and try to guess, hopefully correctly, what the 
next season's flu is going to be.
    But also to be able to immunize children at a very early 
age like we do with measles, mumps, and rubella to protect them 
from the possibility of an unexpected catastrophic outbreak 
like we saw in the pandemics that we have experienced.
    As a matter of fact, we have just very recently had a major 
meeting of individuals from throughout the country and world to 
help us formulate a strategic plan to develop a research agenda 
for the development of universal flu. You have asked Dr. 
Collins and I, many people do, when is this going to happen?
    We now have phases of Phase 2 and Phase 3 clinical trials 
that look very promising. And just literally in the next day or 
so, there is going to be an announcement from the University of 
Pennsylvania of a very, very interesting approach toward 
vaccines that involves recombinant DNA technologies that are 
really going to be very important.
    I have here just for your staff if you want it, a paper 
that we just recently published in the ``Journal of Infectious 
Diseases,'' which outlines our strategic plan for the universal 
influenza vaccine and our research agenda.
    That is what I worry about, but we are trying to do 
something about, but we are trying to do something about what I 
worry about.
    Senator Bennet. Thank you, Mr. Chairman.
    The Chairman. Thanks, Senator Bennet.
    Thank you, Dr. Collins, and to each of you for your 
extraordinary service to our country. Dr. Fauci, that was one 
of Dr. Collins's bold predictions about the universal vaccine 
and it is good to hear that it is on the way.
    We are glad to see a significant new and consistent source 
of funding directed toward the National Institutes of Health. 
But we want to make sure that we spend every single dollar as 
wisely and effectively as possible.
    We hope this hearing and other tools that we give you, 
either through 21st Century Cures or the authority to use money 
in different ways, if you will let us know what you need. 
Senator Bennet has been a leader in many of these bills. A lot 
of bipartisan support for breakthrough initiatives and we want 
to create an environment where you can succeed.
    The hearing record will remain open for 10 days. Members 
may submit additional information for the record within that 
time, if they would like.
    The HELP Committee will meet again on Wednesday, August 29 
when we will hear from Dr. Scott Gottlieb, Commissioner of the 
Food and Drug Administration.
    Thank you for being here.
    The Committee will stand adjourned.

                         QUESTIONS AND ANSWERS

   Response by Francis Collins to Questions from Senator Alexander, 
Senator Roberts, Senator Young, Senator Enzi, Senator Collins, Senator 
 Burr, Senator Murray, Senator Casey, Senator Baldwin, Senator Warren, 
             Senator Kaine, Senator Smith and Senator Jones
             

[GRAPHICS NOT AVAILABLE IN TIFF FORMAT]

                                ------                                

    [Whereupon, at 11:28 a.m., the hearing was adjourned.]

                                  [all]