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