[Senate Hearing 117-]
[From the U.S. Government Publishing Office]
DEPARTMENTS OF LABOR, HEALTH AND HUMAN SERVICES, AND EDUCATION, AND
RELATED AGENCIES APPROPRIATIONS FOR FISCAL YEAR 2022
----------
WEDNESDAY, MAY 26, 2021
U.S. Senate,
Subcommittee of the Committee on Appropriations,
Washington, DC.
The subcommittee met at 10:01 a.m., in room SD-562, Dirksen
Senate Office Building, Hon. Patty Murray (chairwoman)
presiding.
Present: Senators Murray, Reed, Shaheen, Schatz, Baldwin,
Murphy, Manchin, Blunt, Shelby, Graham, Moran, Kennedy, Hyde-
Smith, Braun, and Rubio.
DEPARTMENT OF HEALTH AND HUMAN SERVICES
National Institutes of Health
STATEMENT OF FRANCIS S. COLLINS, M.D., PH.D., DIRECTOR
ACCOMPANIED BY:
DIANA BIANCHI, M.D., DIRECTOR, EUNICE KENNEDY SHRIVER NATIONAL
INSTITUTE OF CHILD HEALTH AND HUMAN DEVELOPMENT
ANTHONY FAUCI, M.D., DIRECTOR, NATIONAL INSTITUTE OF ALLERGY
AND INFECTIOUS DISEASES
GARY GIBBONS, M.D., DIRECTOR, NATIONAL HEART, LUNG, AND BLOOD
INSTITUTE
ELISEO PEREZ-STABLE, M.D., DIRECTOR, NATIONAL INSTITUTE ON
MINORITY HEALTH AND HEALTH DISPARITIES
NED SHARPLESS, M.D., DIRECTOR, NATIONAL CANCER INSTITUTE
BRUCE TROMBERG, PH.D., DIRECTOR, NATIONAL INSTITUTE OF
BIOMEDICAL IMAGING AND BIOENGINEERING
opening statement of senator patty murray
Senator Murray. Good morning. The Senate Appropriations
Subcommittee on Labor, Health and Human Services, Education and
Related Agencies will please come to order.
Today, we are having a hearing on the Biden
Administration's fiscal year 2022 Budget Request for the
National Institutes of Health. Senator Blunt and I will each
have an opening statement, and then I will introduce our
witnesses. And after the witness testimony, Senators will each
have 5 minutes for a round of questions.
Before we begin, I do want to walk through the COVID-19
safety protocols that are in place today. And again, I really
want to thank all of our clerks and everyone who has really
worked hard to get this set up and help us all stay safe and
healthy. So, thank you to them.
For today, we are going to be conducting this hearing
following similar COVID protocols to what we have used in the
past. Committee members are seated at least 6 feet apart. Some
Senators are participating by videoconference. However, I do
expect that this will be our final hybrid hearing, and we will
be able to return to regular, in-person hearings at our next
hearing.
Consistent with CDC guidance, those who are fully
vaccinated do not need to wear a mask, though they may still
choose to do so. And while we are unable to have the hearing
fully open to the public or media for in-person attendance,
live video is available on our committee website. And if you
are in need of accommodations, including closed captioning, you
can reach out to the committee or the Office of Congressional
Accessibility Services.
As of today, almost half of U.S. adults are fully
vaccinated. And while we have a lot of work left yet to do to
reach communities who still cannot get vaccines and reassure
people who still have many questions about them, we can see the
light at the end of the tunnel. And, I really want to thank all
of our witnesses, especially Dr. Collins and Dr. Fauci, for
putting in long hours and putting science first.
Where we are at today is a testament to the tireless work
scientists at NIH have been doing to study this disease and how
we can best fight it, and oversee clinical trials for vaccines
and therapeutics and more, to ensure they are safe and
effective. And, of course, as our witnesses know, our
historically fast progress in fighting COVID-19 and developing
safe and effective vaccines was actually years in the making.
The pace of discovery we have seen this past year was made
possible by research into mRNA vaccines we funded in response
to Ebola and other viruses, and biomedical research enterprise
that has been built over decades to become one of the most
cutting edge in the world.
This should be an important reminder when it comes to
biomedical research. You can never fully predict how the
discoveries of today will prepare you for the challenges of
tomorrow. That is why you have to build the robust research
enterprise and recruit diverse, world class talent, and make
sure scientists can do their work free from political
interference.
And President Biden's budget, which proposes over $40
billion for NIH (National Institutes of Health), the largest
increase in the agency's history, will go a long ways towards
making sure we can continue to prioritize this. This budget
will reinforce our work to fight COVID-19, along with many
other diseases and disorders that threaten families in my home
State of Washington, or Missouri, or across the Country.
It includes funding to improve treatments for addiction and
substance use disorders, and funding to aid the fight against
cancer, Alzheimer's disease, and rare diseases families across
the Country are grappling with.
President Biden's budget request will also fund research to
help us study the health effects of climate change, which may
be increasing the number of infectious disease outbreaks;
identify solutions to gun violence, which continues to claim
tens of thousands of lives each year in this Country; and root
out the health inequities in our Country, which are undermining
the health of people of color, people with disabilities, rural
communities, those paid low incomes, and more.
The President has also proposed $6.5 billion for a new
initiative--the Advanced Research Projects Agency for Health.
Like the defense initiative it is inspired by, ARPA-H is
envisioned as breaking the mold for how cutting-edge research
is conducted, speeding up the development of medical treatments
by funding innovative projects. I am interested to hear more
about how it can add to NIH's work and operate as something
truly distinct from its other traditional, biomedical research
programs.
Of course, at the end of the day, innovation is not just
driven by new programs and new investments. It is driven by
people, which is why with as much as we invest in NIH each
year, and as important as its work to its families, our
families, we cannot afford to have this agency's potential
limited or its success threatened by bias, discrimination,
harassment, or assault in the workplace.
Unfortunately, we know that in the biomedical research
community, the prevalence of researchers of color is too low,
and the prevalence of sexual harassment is too high. These are
real problems with real consequences for biomedical research
and the people who do the lifesaving work we are all
benefitting from today.
I commend NIH for the efforts it has taken on both of these
fronts so far. NIH has done work to examine barriers to
diversity among its researcher ranks and how its own practices
have reinforced structural biases that allow discrimination to
persist. But, more work remains to tear down barriers and
create lasting change.
And when it comes to sexual assault, Director Collins, I am
glad you have taken some forceful action to address the problem
among the NIH workforce, but NIH must do more to use its
enormous influence with the research community to enforce
change in the Nation's universities and research institutions.
I expect NIH to continue building on its efforts so far to
remove racism, discrimination, and harassment from research,
and I will continue to follow up on that progress.
Finally, as proud as we all are of our Nation's biomedical
research institutions, we do not invest billions of dollars in
biomedical research out of pride, nor do we invest in them to
help pharmaceutical companies make astronomical profits. We do
it to bring new treatments, cures, and hope to people across
the Country and across the world. It is important that we never
lose sight of this because even the most brilliant miracle cure
can only save people if they can actually get it.
Just as I hope to work with my colleagues on both sides of
the aisle to make lifesaving investments in biomedical research
like those proposed in the President's budget, I also hope we
can work together to bring down the cost of healthcare,
especially for prescription drugs; keep working towards
universal health coverage; and bring the cures we are investing
in to the families who need them.
With that, I will turn it over to Senator Blunt for his
remarks.
statement of senator roy blunt
Senator Blunt. Well, thank you, Chair Murray. I appreciate
having this hearing today and appreciate being able, again, to
start this process with you as we did last week on our first
hearing.
I am certainly glad that Dr. Collins and the Institute
directors are here with us today. I think two of the directors
are testifying before the committee for the first time, and,
so, welcome to the two of you. And this is a helpful
relationship for us, and hopefully for you.
Certainly, the challenges we have faced over the past year
have been unanticipated and significant. I think the global
pandemic reinforced the importance of the National Institutes
of Health. In less than a year, NIH was able to take this novel
coronavirus and help develop two FDA (Food and Drug
Administration)-authorized vaccines, two FDA-authorized
therapeutics, and 16 rapid diagnostic tests, including the
first FDA-authorized point-of-care diagnostic test for COVID-19
to combat its spread and its effects.
A year ago, when we would have had a similar discussion,
one of the big topics would be, why can't we get enough tests?
NIH stepped up and really played a big role in seeing that we
had enough tests. We have not heard that discussion for a long
time. And that does not mean that millions of tests are not
being taken every day. It just means we figured out at this
committee and NIH to be part of meeting that need.
It was revolutionary to watch NIH work, but it did not just
happen. In a time of crisis, during shutdowns, during social
distancing, dealing with a disease that has never been seen
before, the system and its nationwide grantees were able to use
their expertise and infrastructure to, again, develop tests,
treatments, and vaccines. Our research infrastructure was
tested like never before and, in my opinion, it succeeded in
remarkable ways.
I believe there are really three reasons for that. First,
in the past 6 years, this committee and the Congress, in a
bicameral, bipartisan way have prioritized and invested in NIH.
Within that 6-year timeframe, funding for medical research
increased by almost $13 billion, or nearly 43 percent over that
6 years after a decade at virtually level funding. This
investment encouraged young scientists, young researchers, and
mid-career researchers that were leaving the field before that
to stay in the field. And, with your insistence, Dr. Collins,
some of that money every time was set aside to be sure that it
was going to first-time grantees.
We were able to shore up the research infrastructure across
the Country and provide research into mRNA, an idea that had
never produced a vaccine before and, of course, became the
foundation for the two principal vaccines that were developed
very much with the involvement of NIH.
Our ability to pivot so quickly and so successfully to
fighting COVID-19 could not have been accomplished had we
stayed at the funding levels we were at 7 years ago. The buying
power was not where it needed to be. Young researchers were
leaving the field. Tough budgetary decisions meant that people
were not only getting their applications rejected at
significant levels; they just, frankly, stopped making a lot of
applications. That is not your problem, by the way, today.
Second, at the height of the pandemic, Congress gave the
Department of Health and Human Services significant funding and
flexibility to create Operation Warp Speed. It was successful
in developing two FDA-authorized COVID-19 vaccines and
commercializing another with the help of NIH because we united
in our effort to make that happen.
One of the things we did was to really invest in vaccines
that we did not think were certain to work, but thought were
likely to work, and that meant that vaccines were available
when they got FDA authorization rather than months after they
got FDA approval. Because of that, fully half of all adults
have been vaccinated now in the United States as we work toward
a bigger number than that.
We pushed private industry and worked with private industry
in ways we had not before. I have said at the time, one way to
win the horse race is to bet on all the horses. And I think to
a great extent we did in the vaccine effort, bet on all the
horses we thought had a chance to finish the race, and it made
a difference.
Finally, one of the most important lessons learned from the
pandemic is the value of having the Federal Government, on
occasion, as a more active partner in research and development
instead of just a sponsor. The ambitious speed and goals that
pushed private companies to research, develop, and manufacture
a COVID-19 vaccine, along with what we did in testing, really
created the kind of breakthroughs we needed.
RADx and Warp Speed, I think put us in a different place
than we would have been 2 years ago in thinking about how we
can look at some of our research efforts in another way. That
is why I want to work with the Administration to support the
ARPA-H initiative. This will be a new institute, or is proposed
to be a new institute, and I think that is what should be the
case. They will have the flexibility and tools necessary to
both nimbly and innovatively respond to both the next pandemic
and also some of the big health issues we face today.
This is a critical moment in a rapidly changing healthcare
world. Finding those things that the kind of Warp Speed, Shark
Tank, RADx relationship could enhance in cancer, in
Alzheimer's, in every disease where there is an opportunity;
where we see that moment and know that this is something that
does not necessarily call for a 5-year research grant, but some
sort of partnership different than that that moves toward a
real conclusion sooner than we might otherwise be able to do
that.
ARPA-H should not do what the other institutes do, but it
should do what the other institutes cannot do in a crosscutting
way that goes throughout the institutes, looking for
opportunities, frankly, in the other institutes where there is
a breakthrough moment that we could look at differently. I
think we can help fill gaps here that otherwise would not be
filled and look forward to that discussion.
Now, also, as someone working with Senator Murray for the
last 6 years to increase the funding and the focus in what NIH
has been doing, we clearly want to be sure that this somehow
does not take away from the solid research that proves so
effective in getting us ready for what we just saw.
So, Dr. Collins, I look forward to working with you and
Chair Murray and the Administration in making ARPA-H a reality.
I think the moment is ready for that. I think because of what
has happened in the last 2 years, NIH is ready for that, and
look forward to the discussion today.
[The statement follows:]
Prepared Statement of Senator Roy Blunt
Thank you, Chair Murray. I appreciate Dr. Collins and the other
Institute Directors for being here today.
The challenges we have faced over the past year in a global
pandemic reinforced the importance of the National Institutes of
Health.
In less than a year, NIH was able to take this novel coronavirus
and develop two FDA-authorized vaccines, two FDA-authorized
therapeutics, and 16 rapid diagnostic tests, including the first FDA-
authorized point-of-care diagnostic test for COVID-19, to combat its
spread and effects.
This was revolutionary, and it didn't happen without decades of
preparation.
In a time of crisis, during shutdowns and social distancing, for a
disease never seen before, the NIH and their nationwide system of
grantees were able to use their expertise and infrastructure to develop
tests, treatments, and vaccines for COVID-19. Our research
infrastructure was tested like never before, and it succeeded. And I
believe there were three key reasons behind this success.
First, for the past six years, this Committee and Congress have
prioritized and invested in NIH. Within this timeframe, funding for
medical research increased by $12.85 billion, or nearly 43 percent,
after having spent the previous decade at virtually level funding.
This investment encouraged young and mid-career scientists in the
field, who often have the most novel and innovative research ideas,
shored-up the research infrastructure across the country, and provided
research into mRNA, which is the foundation for two of the COVID-19
vaccines.
Our ability to pivot so quickly and so successfully to fighting
COVID-19 could not have been accomplished had this Committee let NIH
funding stagnate for another decade, dragging down its buying power,
and letting young researchers leave the field. Making the tough
budgetary decisions necessary to prioritize the NIH paid off.
Second, at the height of the pandemic, Congress gave the Department
of Health and Human Services significant funding and flexibility to
create Operation Warp Speed. It was successful in developing two FDA-
authorized COVID-19 vaccines and commercializing another, with the help
of NIH, because it united the federal government, private companies,
and researchers around a common goal.
The reason that we have been able to fully vaccinate half of all US
adults is because there was a deliberate strategy in the last
Administration to focus and provide funding for any COVID-19 vaccine or
therapeutic that had the likelihood to work. We took financial risks to
manufacture vaccines as the development process was still underway.
We pushed private industry to innovate their own approaches. And we
forever changed the drug approval process. As I have said before, the
way to win a horse race is to bet on all the horses. That is what this
Committee and the previous Administration did.
Finally, one of the most important lessons learned from the
pandemic is the value of having the Federal Government become a more
active partner in research and development, instead of just a sponsor.
The ambitious speed and goals that pushed private companies to
research, develop, and manufacture a COVID-19 vaccine through Operation
Warp Speed demonstrated that active collaboration in public-private
partnerships, in conjunction with significant funding, are game
changers in creating scientific breakthroughs.
Now we must learn from these lessons. There is an opportunity to
build upon Operation Warp Speed and NIH's RADx diagnostic testing
program to leverage public-private partnerships to dramatically
accelerate the development and approval of new treatments and
technologies. What two years ago would have been termed risky and
financially unpalatable now is possible.
And that is why I want to work with this Administration to support
the ARPA-H initiative. This will be a new Institute that will have
flexibility and tools necessary to nimbly and innovatively respond to
both the next pandemic and also to some of the biggest health issues
Americans face today, like cancer and Alzheimer's disease.
ARPA-H should do what other NIH Institutes cannot. It needs to be
cross-cutting throughout all the NIH Institutes and collaborative both
internally with NIH and HHS and externally with partners. It needs to
be innovative. And it should help fill the gaps we clearly saw during
the pandemic between basic science and commercialization of COVID-19
vaccines and therapeutics.
Simply put, there are aspects of NIH research that could move much
faster outside the traditional NIH grant cycle. The NIH peer review
process is the gold standard, but we also need to recognize that it
doesn't work for all research at all times.
I look forward to working with you, Dr. Collins, and you, Chair
Murray, on making ARPA-H a reality.
It will take collaboration between the Administration, NIH, and
Congress. But as we work toward a new Institute to accelerate the
application and implementation of health discoveries, we must make sure
that basic science is not abandoned. ARPA-H should not be the shiny new
toy we all focus on, especially not to the detriment of the NIH
research community as a whole.
If there is one lesson we must take from this pandemic, it is that
our nation's success depends on the medical research infrastructure
across this country supported by the NIH. Now is not the time to
abandon it. Now is the time to make it even stronger.
Thank you.
Senator Murray. Thank you very much, Senator Blunt.
I will now introduce our witnesses.
Dr. Francis Collins is the director for the National
Institutes of Health.
Dr. Diana Bianchi is the director of the Eunice Kennedy
Shriver National Institute of Child Health and Human
Development.
Dr. Anthony Fauci is the director of the National Institute
of Allergy and Infectious Diseases.
Joining us virtually is Dr. Gary Gibbons. He is the
director of the National Heart, Lung and Blood Institute.
Dr. Eliseo Perez-Stable is the director of the National
Institute on Minority Health and Health Disparities.
Dr. Ned Sharpless is the director of the National Cancer
Institute.
And, finally, Dr. Bruce Tromberg is the director of the
National Institute of Biomedical Imaging and Bioengineering.
So, Dr. Collins, we will turn to you for your opening
remarks.
SUMMARY STATEMENT OF DR. FRANCIS S. COLLINS
Dr. Collins. Thank you, Chair Murray and Ranking Member
Blunt and distinguished members of the subcommittee. I am
honored to be here today with my colleagues representing the
National Institutes of Health, the NIH.
I could spend hours describing the exciting work the
President's budget is proposing for NIH, including major
investments to address impacts of the COVID-19 pandemic, reduce
health disparities in maternal mortality, improve mental
health, broaden approaches to pain and opioid addiction, and
establish a bold, new agency within NIH called ARPA-H.
But, in our brief time together, it is also important to
emphasize how steady funding increases that you have provided
to NIH, starting well before the pandemic, made it possible for
NIH to meet the challenges of the pandemic and to prepare for
what comes next.
Often at these hearings, I share a story of a patient whose
life has been saved by NIH research, but in this uniquely
challenging year, it is hard to single out any one person. In
fact, all of the more than 160 million Americans who have
received COVID-19 vaccines as of today are success stories made
possible by the sustained investment that this committee made
years ago to basic biomedical research.
The road to these mRNA vaccines actually started back in
the 1960s when the function of messenger RNA was first
understood. These messengers carry instructions from the cell's
DNA manual to produce the proteins that do the work. Now, for
vaccines, we knew that certain proteins, like the spike
proteins on the coronavirus, could spur an immune response.
But, might it be safer and just as effective to use the RNA,
the codes for those spike proteins, to instruct the patient's
body to produce them? And it took a lot of obstacles to
surmount to get there over more than 20 years, but we are blown
away by how well it works.
In parallel, other NIH-supported scientists, including some
at our own Vaccine Research Center, learned that locking those
spike proteins into the right configuration could make an even
better vaccine. So, when COVID hit, we knew exactly what to do,
but we needed the help of the American people enrolling in
clinical trials to finish the job. To facilitate that, NIH
opened a dialogue with communities disproportionately affected
by COVID to ensure that they had access to the vaccine trials.
The Community Engagement Alliance, or CEAL, c-e-a-l,
Initiative built on some existing, long-term partnerships with
trusted leaders in underserved communities to engage directly
on trial enrollment, and later with hesitant individuals on
issues related to vaccine safety and efficacy.
We were able to use the enrollment techniques we learned in
the large, longitudinal studies, such as All of Us, that you
have championed. The result is that all Americans can look at
the major vaccine trials and see that people like them were
included.
While the vaccines were in early trials, the world was
clambering for rapid diagnostics to understand and manage our
risks. Members of this committee, most notably Senator Blunt,
asked what NIH could do to ramp up innovation. And thanks to
your support, and using a novel Shark Tank approach, NIH took
on a new role as a venture capitalist through the Rapid
Acceleration of Diagnostics, or RADx program.
Today, there are 33 novel testing platforms helping perform
just today, millions of tests daily, due to RADx. This program
demonstrated the remarkable innovations that are possible when
NIH brings together experts in engineering, business, and
manufacturing to fund big ideas.
Now, the President's budget proposes a major investment to
build on this momentum the Advanced Research Projects Agency
for Health, or ARPA-H. This new agency within NIH will catalyze
novel strategies to speed transformational and innovative
ideas, ideas such as simple blood tests to detect free-floating
DNA or protein markers that signal a cancer is growing
somewhere in the body; a micro needle patch that delivers a
vaccine to hard-to-reach communities in the mail; using an
innovation funnel to recruit, test, and scale up new
technologies for ambulatory blood pressure measurement with the
potential to transform the management of hypertension.
These are just a few of the bold ideas that ARPA-H could
tackle, but they are not science fiction. With standard
approaches, well, they might happen in a decade or two. With
ARPA-H, we believe it could take half that time.
The President believes that with your help, we can learn
from the lessons of pandemic and transfer this scientific
momentum into big improvements in the health of all Americans.
I do, too.
My colleagues and I would be pleased to answer your
questions.
[The statement follows:]
Prepared Statement of Francis S. Collins, M.D., Ph.D., Diana W.
Bianchi, M.D., Anthony S. Fauci, M.D., Gary H. Gibbons, M.D., Eliseo J.
Perez-Stable, M.D., Norman E. Sharpless, M.D., and Bruce J. Tromberg,
Ph.D.
Good morning, Chairwoman Murray, Ranking Member Blunt, and
distinguished Members of the Subcommittee. 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.
First, I want to thank this Subcommittee for your commitment to
NIH, which allowed the biomedical research enterprise to respond
quickly to the greatest public health crisis in our generation over the
past year. We mounted vigorous research efforts to understand the viral
biology and pathogenesis of the coronavirus disease 2019 (COVID-19),
develop vaccines in record time, support and commercialize diagnostics
at the point of care, and test therapeutics for both outpatient and
inpatient settings. This work is far from finished.
The President's Discretionary Request proposes budget authority of
$51 billion for NIH in fiscal year (FY) 2022. The Biden Administration
places great emphasis on research and development in general. At NIH in
particular, the Request proposes to build on the successes of pandemic
era research and to put the research enterprise to work on some of our
Nation's most persistent and perplexing health challenges, including
cancer, Alzheimer's disease, opioid use disorder, health disparities,
maternal mortality, HIV/AIDS, gun violence, climate change, and other
areas with major implications for our Nation's health.
First and foremost, the President's Request proposes $6.5 billion
to establish the Advanced Research Projects Agency for Health--ARPA-H
to drive transformational innovation in health research and speed
application and implementation of health breakthroughs. ARPA-H will
tackle bold challenges requiring large scale, cross-sector
coordination, employing a non-traditional and nimble approach to high
risk research, modeled after DARPA in the Department of Defense. To
achieve this, ARPA-H will invest in emergent opportunities by
conducting advanced systematic horizon scans of academic and industry
efforts, leveraging novel public-private partnerships, recruiting
visionary program managers, and using directive approaches that provide
quick funding decisions to support projects that are results-driven and
time-limited. Potential areas of transformative research driven by
ARPA-H include: the use of the mRNA vaccines to teach the immune system
to recognize any of the 50 common genetic mutations that drive cancer;
development of a universal vaccine that protects against the 10 most
common infectious diseases in a single shot; development of wearable
sensors to measure blood pressure accurately 24/7; and leveraging of
artificial intelligence technology to advance care for individual
patients and improve detection of early predictors of disease.
ARPA-H represents the kind of transformative idea for biomedical
research that only comes along once in a long while. Our confidence
that NIH is ready has been greatly advanced by our experience in
addressing the COVID-19 pandemic--developing vaccines in record time,
establishing an unprecedented public-private partnership on
therapeutics that has made it possible to test more than a dozen
possible therapeutics in rigorous trials, and building a venture
capital model for assessing SARS-CoV-2 diagnostic technologies that has
yielded millions of daily tests in just months.
But while we begin to imagine a life after COVID-19, we must
acknowledge that there are COVID-related impacts that we have yet to
understand and address, including the full impact of the pandemic on
children. Children were largely spared from COVID-19 but for some
children, exposure to the COVID-19 virus led to Multisystem
Inflammatory Syndrome in Children (MIS-C), a severe and sometimes fatal
inflammation of organs and tissues. The Eunice Kennedy Shriver National
Institute of Child Health and Human Development (NICHD) is leading a
multi-institute initiative known as the Collaboration to Assess Risk
and Identify loNG-term outcomes for Children with COVID (CARING for
Children with COVID), which will assess both short-term and long-term
effects of MIS-C and other severe illness related to COVID-19 in
children, including cardiovascular and neurodevelopmental
complications.
For many Americans, this pandemic and its related socioeconomic
effects have had an overwhelming impact on their mental health. Prior
research on disasters and epidemics has shown that in the immediate
wake of a traumatic experience, large numbers of affected people report
distress, including new or worsening symptoms of depression, anxiety,
and insomnia. To aid in mental health recovery from the COVID-19
pandemic, NIH will continue to focus on research in this area. This
will be done, in part, by utilizing participants in existing cohort
studies, who will be surveyed on the effect of the pandemic and various
mitigation measures on their physical and mental health.
The COVID-19 pandemic has brought into sharp focus the dramatic
health disparities that exist across the American population. In
addition, the Nation has been shaken by the killing of George Floyd and
other attacks on people of color, forcing a recognition that our
country is still suffering the consequences of centuries of racism. NIH
will continue to address these disparities, specifically through
research managed by the National Institute on Minority Health and
Health Disparities (NIMHD), the National Heart, Lung, and Blood
Institute (NHLBI), the National Institute of Nursing Research (NINR)
and the Fogarty International Center (Fogarty).
NIMHD looks to better understand the human biological and
behavioral mechanisms and pathways that affect disparity populations,
better understand the long-term effects of disasters on health care
systems caring for populations with health disparities and research
focusing on the societal-level mechanisms and pathways that influence
disease risk, resilience, morbidity and mortality. NINR and Fogarty
both look to better understand and reduce rural health disparities in
low-income counties in the southern United States, support nursing
science focused on racial, ethnic, and socioeconomic health
disparities, with the goal of closing the gap in health inequities and
increase health disparity research in low and middle income countries.
In addition to the core health disparities research, the
President's Request puts an additional specific focus on maternal
morbidity and mortality (MMM), which disproportionately affect specific
racial and ethnic minority populations. Black and American Indian/
Alaska Native individuals are two to four times more likely to die from
pregnancy-related or pregnancy-associated causes compared to white
individuals. Furthermore, Black, Hispanic and Latina Americans, Asian,
Pacific Islander, and American Indian/Alaska Native individuals all
have higher incidence of severe maternal morbidity (SMM) compared to
white individuals. The Implementing a Maternal Health and Pregnancy
Outcomes Vision for Everyone (IMPROVE) initiative supports research on
how to mitigate preventable MMM, decrease SMM, and promote health
equity in maternal health in the United States.
As the climate continues to change, the risks to human health will
grow, exacerbating existing health threats and creating new public
health challenges. Major scientific assessments document a wide range
of human health outcomes associated with climate change. While all
Americans will be affected by climate change, underserved populations
are disproportionately vulnerable. These populations of concern include
children, the elderly, outdoor workers, and those living in
disadvantaged communities. NIH is poised to lead new research efforts
to investigate the impact of climate on human health, with the goal to
understand all aspects of health-related climate vulnerability.
Therefore, the President's Request includes a $100 million increase for
research on the human health impacts of climate change.
The FY 2022 President's Discretionary Request makes a major
additional investment to address the opioid crisis. The crisis of
opioid misuse, addiction, and overdose in the United States is a
rapidly evolving and urgent public health emergency that has been
exacerbated by the coronavirus pandemic. Since the declaration of a
public health emergency for COVID, illicit fentanyl use and heroin use
have increased, and overdoses in May 2020 were 42 percent higher than
in May 2019.
The use of opioids together with stimulants, such as
methamphetamine, is increasing; and deaths attributed to using these
combinations are likewise increasing. Taking note of these trends, FY
2021 appropriation language expanded allowable use of Helping to End
Addiction Long-term (HEAL) funds to include research related to
stimulant misuse and addiction. Identifying how opioids and stimulants
interact in combination to produce increased toxicity will enhance our
ability to develop medications to prevent and treat comorbid opioid and
stimulant use disorders and overdoses associated with this combination
of drugs.
Finally, I'd like to take a moment to thank this Subcommittee for
its recognition over the last two years that America's continuing
leadership in biomedical research requires infrastructure and
facilities that are conducive to cutting-edge research. With your
support, we will break ground in the near future on a new Surgical,
Radiological, and Laboratory Medicine division of our Clinical Center,
which will replace severely outdated and deteriorating operating suites
and lab space with state-of-the-art facilities. NIH continuously works
to ensure that the buildings and infrastructure on its campuses are
safe and reliable and that these real property assets evolve in support
of science--but NIH's backlog of maintenance and repair is now nearly
$2.5 billion. The President's FY 2022 Discretionary Request includes
$250 million to make progress on reducing this backlog and requests
flexibility for Institutes and Centers to fund construction, repair,
and improvement projects.
COVID-19 compelled us to perform a stress test on biomedical
research enterprise. The enterprise performed nobly. We found what
worked, and also identified barriers we hadn't fully appreciated
before, and invented new ways around them. The President's FY 2022
Discretionary Request is a roadmap for how to build on the successes of
research, address our gaps, and apply our insights to the most
important problems we face as a nation. With your support, the future
is filled with opportunity. My colleagues and I look forward to
answering your questions.
Senator Murray. Thank you very much, Director Collins. I
have to say, I have always loved your success stories. They are
usually really beautiful. But, I will say, I think many of us
in this room are grateful to be your success story this time.
So, thank you.
We will now begin our 5-minute rounds of questions, and Dr.
Collins, I will start with you.
As you just talked about, the President's budget includes
$6.5 billion to create the ARPA-H within NIH that is modeled
after DARPA. DARPA is a small, $3.5 billion agency that is
composed mostly of program managers and empowered to push the
limits of their disciplines and shape some milestone-driven
breakthrough technologies in short 3- to 5-year stints.
Given that the nature of NIH's work is different, relying
on a peer review system or multi-year grants that is
traditionally risk-adverse, where progress is often measured in
decades, how do you envision ARPA-H fitting into the NIH
ecosystem?
ARPA-H STRUCTURE
Dr. Collins. Senator, it is a great question. I think you
are right that much of what NIH does requires this kind of
careful, deliberative, investigator-initiated, hypothesis-
driven research, and that is going to be the mainstay of what
we do going forward. That has been the success story of NIH for
many decades.
But, there are opportunities, as we have seen happen during
COVID, such as the need to develop diagnostics in a hurry, to
develop vaccines in a hurry, that are not really amenable to
that approach, where you need to have program managers that are
empowered to move things swiftly and have the flexibility and
the resources to do so. And that is the DARPA model. We have
studied that closely, and we do think that there are projects
in biomedicine now that would be greatly advantaged by that.
That is not the typical peer review process that may take a
year from the idea to the first award. With RADx, we made those
first awards 5 days after the Congress gave us the budget for
it, and that played out really well.
So, we want to incorporate that mindset, and we want to
bring on perhaps a hundred of these program managers, give them
the opportunity to build the kind of collaborative ventures
that include such organizations as small businesses that might
otherwise not be likely to write an NIH grant.
Ride herd over these things carefully so that if they are
not doing well, they get basically stopped immediately. We
expect there will be failures--this is high risk--but identify
the areas of greatest opportunity. And every Institute at NIH
is now coming forward saying, I have at least five ideas of
what I would like to do with ARPA-H that I cannot do right now.
So, this should not be seen as competing with the
Institutes. It is going to be a synergistic relationship that
will allow us to do things otherwise that would take a very
long time.
Senator Murray. Okay. Well, you have said that it should be
within the office of the director. In that structure, how would
decisions be made about what projects to fund?
Dr. Collins. So, we will need to hire a director for ARPA-
H, who will need to be a visionary person, and the idea is to
bring on somebody who is not probably going to be doing this as
their long-term career, but maybe for one term, 5 years, with
one possible renewal.
That person will be very much engaged then in bringing
onboard these very creative program managers who have to make a
pitch about what kind of projects they think are worth
investing in and convince the director that that is the case.
And, then, they are given the flexibilities to go out and find
the right partners and see what can happen. But, that is all
going to be done in a way that is quite nimble. It is not going
to involve our traditional peer review process.
Senator Murray. Okay.
STRUCTURAL RACISM AND HEALTH EQUITY
Dr. Perez-Stable, your career has really focused on
improving the health of communities of color and underserved
populations. And NIH recently released a $30 million funding
opportunity to study the impact of structural racism and
discrimination in order to promote health equity and eliminate
health disparities. Can you talk to us a little bit about what
more can NIH, and particularly NIMHD (National Institute on
Minority Health and Health Disparities), be doing to address
those issues, and what would be the benefit of making
additional investments?
Dr. Perez-Stable. Thank you, Senator Murray, for that
question. So, first of all, we had to recognize that structural
racism could be operationalized as a research construct and not
just an organizational construct, and we went through a
workshop and scientific reflection on this. I think the moment
earlier this year for all of the NIH Institutes and Centers
agreed that this was an area that we needed to move on and
advance more quickly in the research side. And, so, we had a
commitment from all the institutes that do this, although NIMHD
was leading it from the beginning.
We believe that two areas are susceptible for improvement.
One would be the healthcare setting, where I think through
interventions at the structural, as well as the clinician and
the patient level will help. And, also, in promoting healthy
communities so that we can have easier access to green space,
to healthy food, accessible healthcare in community health
centers.
These are two areas that we believe are susceptible for
improvement, although we will depend on our scientific
community to promote and submit ideas that will be reviewed and
hopefully funded within fiscal year 2022.
Senator Murray. Okay. Thank you very much. I look forward
to working with you and hearing more about that.
Senator Blunt.
Senator Blunt. Thank you, Chairman.
ARPA-H FUNDING LEVEL
Dr. Collins, on the ARPA-H budget request, $6.5 billion,
one part of the question will be, how do you think that number
was arrived at, and is that a realistic number to commit in
year one?
And two, our concern would also be that we do not get in a
position that--we have already given NIH $6.5 billion and level
fund everything else. I do like the President's $2.5 billion. I
am sure you could figure out how to spend more than that in the
other institutes. That is pretty close to the average of the
last 6 years from our committee. I would certainly like to stay
at least at that level.
But, how do you think those two numbers compete with each
other? And how do you feel about actually being able to commit
$6.5 billion in that first fiscal year of ARPA-H?
Dr. Collins. That is a great question, Senator, and we have
thought a lot about it. I am pleased the President's budget
proposes that this would be 3-year money because, obviously,
you are going to start from a standing start whenever the
budget actually gets approved for fiscal year 2022. We hope
that will be September 30th, right? Well, it might not be. So,
at any rate, we would then really be benefitted by being able
in that first year to stretch those dollars over a little bit.
I do think we could, with a hundred program managers,
readily come up with a number of projects that would fit within
that envelope on an annual basis. But, I hear what you are
saying about a concern because I have heard it also that this
might in some way compromise the interest of the Institutes. I
guess I would look at it a different way, though.
As I said earlier, every one of the Institutes is coming
forward with great ideas about how they would like to use ARPA-
H. They think of this as an augmentation of their capabilities,
not a subtraction. And, so, they will be feeding ideas into
this and have a lot to do about how those are chosen. So, even
though the base number that is being proposed, $2.5 billion for
the ICs (NIH Institutes and Centers), may sound like a sort of
average one, in terms of the science they can do, ARPA-H is
going to add to that.
Senator Blunt. All right. Thank you.
ARPA-H AND CANCER RESEARCH
Well, Dr. Sharpless, one of the things the President, of
course, talks about in this issue, in this topic, is more
rapidly moving toward ending cancer. Obviously, we want to do
that. We also want to make the point that that is not the only
thing that ARPA-H would be focused on, nor would it just be
cancer or Alzheimer's. But, on that topic, how do you envision
the ARPA-H role in cancer research and what might you be able
to do with ARPA-H that you are not able to do in the
traditional restraints of the National Cancer Institute?
Dr. Sharpless. Thank you for the question, Senator Blunt.
It is great to be testifying in front of this committee again.
Good to see you virtually, at least, today.
Yes, as the President has said, ending cancer as we know it
is a top domestic priority for this Administration. We are
obviously, the cancer research community, is galvanized by this
notion and very excited.
I think, as you know, the National Cancer Institute does
some things really well. You know, we fund basic foundational
science very well. We can do clinical trials quite well. But,
there are some areas where we are challenged, where we have
struggles, and I think the scale and nimbleness and ability to
interact with industry is very appealing about ARPA-H for
certain kinds of cancer projects.
I think a good example of that is this blood-based cancer
detector technology that Dr. Collins mentioned in his opening
statement where you can, you know, find cancers at a very early
stage in otherwise asymptomatic, healthy people, and that could
have a profound effect on cancer mortality.
So, you know, getting up a huge trial of that technology as
quickly as possible is the kind of thing that I think would be
a good fit for ARPA-H.
Senator Blunt. Okay. Thank you, Dr. Sharpless.
RADX PARTNERSHIPS
Dr. Tromberg, let me see if I can get one more question in.
I think what you were part of at RADx is one of the reasons
that gives me real optimism about new kinds of relationships
that we might develop at ARPA-H. But, would you talk just a
little bit about RADx and how that partnership continued right
through the entire process of these companies that you were
choosing to invest money with, going ahead and making the first
home-based test, and I think producing well over two million
tests every day now, in addition to the tests that would have
come through the regular process?
Dr. Tromberg. Yes. Thank you so much, Senator Blunt, and
thank you for your question and for your generous support of
the RADx program.
The bioengineering-technology community has formed
partnerships all across the government. That has included
working with BARDA, FDA, DOD (Department of Defense), CDC
(Centers for Disease Control and Prevention), HHS (Department
of Health and Human Services), and the White House Testing
Board. More than 900 scientists are working across government,
academia, and the private sector in a very unique way to make
this work.
And, as you have mentioned, if we fast-forward to now,
about 1 year later, we now have 33 RADx-supported companies
that have increased the Nation's testing capacity by more than
300 million new tests, and there have been 23 new FDA
authorizations. And we have really changed the dialogue from
laboratory testing of symptomatic folks to over-the-counter,
widely available tests, point-of-care tests that are accessible
to all. Greater choice and greater capabilities. And this has
really happened because of all of these partnerships that we
formed, the accelerated innovation.
We have brought out new technologies. About 20 percent of
our portfolio actually--not many people know about--has been
based in nanoscience and nanotechnology.
Senator Blunt. Good.
Dr. Tromberg. So it has been a tremendous surge for
innovation.
Senator Blunt. Thank you, Doctor.
Thank you, Chairman.
Senator Murray. Yes. Senator Reed.
Senator Reed. Thank you very much.
I want to welcome all the panelists and thank them for
their distinguished service to the Nation, particularly during
this difficult and challenging COVID pandemic.
Dr. Collins, one of the things that is becoming
unfortunately and painfully obvious is the increase in
suicides, and this is very disturbing. And we are concerned,
also, about the impact of COVID-19 on accelerating, perhaps,
that phenomenon.
SUICIDE PREVENTION
So, the question I would have is, what research is NIH
doing on suicide prevention so that we can recognize the
warning signs, better communicate with friends and family, and
also give healthcare providers more insight? I am told that
many suicide victims visit emergency rooms frequently before
their suicide and those signs are not picked up. So, your
comments would be appreciated.
Dr. Collins. Well, I appreciate the question, Senator, and
it is a source of great concern and obviously great heartache
for the way in which this is taking a toll amongst people
across our Nation, and certainly at a time where mental health
issues have been even further heightened by all the stresses of
COVID-19. One can see this also becoming even more of a threat
to people who have lost hope.
NIH is deeply engaged in trying to understand ways to
prevent this terrible outcome, and the National Institute of
Mental Health has in fact invested in a number of new
initiatives as a result of that concern.
One that I would point to that has turned out to be a
pretty encouraging development is the recognition that the drug
Ketamine, which is used in anesthesia and sometimes used as a
party drug, unfortunately. It also turns out to have benefits
for people with serious depression, including people with
suicidal ideation. Now approved by FDA, and the drug
Esketamine, this is now available and it is being used in those
acute situations of acute suicidal threat.
You also mentioned that many people who are on the brink do
end up visiting healthcare facilities. We have worked hard to
try to make sure that this idea of having a screening tool that
was used in emergency rooms for individuals who are there, even
if they do not appear to be there for psychiatric reasons, gets
used to identify, particularly with adolescents, whether they
might be in a situation of contemplating self-harm.
On top of that, certainly NIMH is investigating other means
of treating depression, and also thinking hard about other
interventions that might be beneficial here in terms of
cognitive behavioral therapy combined with pharmacotherapy to
try to assist those individuals who are in this difficult
place. But, it is a terribly difficult problem.
I will say, it is interesting, but it is not necessarily
that encouraging, the actual suicide rate, as best we know, in
the course of the last year has not gone up. It has actually
gone down slightly, and that has tended to be the case in
national crises before. But, what I worry about is what happens
when we seem to be getting past the crisis, is there a pent up
backup there that might in fact result in an even greater risk
in the coming months.
I would be glad to give you more information. I am sure Dr.
Gordon would, as well, in terms of all the things that we are
doing.
Senator Reed. Thank you very much.
LONG COVID
Very quick question to both--to Dr. Fauci. The long haul
COVID-19 is beginning to trouble a lot of people. They never
seem to be able to recover from it and recurrences. What
attention are we paying to that issue?
Dr. Fauci. Thank you for that question, Senator. We are
paying a considerable amount of attention to it. In fact, we
have a program to the tune of $1.15 billion, looking at
developing cohorts of individuals so that we can study them for
the incidence, the prevalence, underlying pathogenesis, and, if
possible, if we can find this out, anything that we can do from
an intervention. So, the NIH is taking this very seriously.
Thank you.
Senator Reed. Thank you very much.
I have to commend Dr. Sharpless for his efforts on
childhood cancer. I was teamed up with Senator Capito. We
passed the Childhood Cancer STAR Act. We have been funding it,
thanks to the Chairwoman, at $30 million a year, and I want to
commend NIH on its renewed emphasis on childhood cancer, not
only treatments, but also gathering data about these victims as
they age so that we can see if there is any interventions that
we can use later on. So, thank you, Dr. Sharpless, and thank
you, panelists. Thank you very much.
Senator Murray. Thank you. Senator Graham.
Senator Graham. Thank you, Madam Chairman.
VACCINE DEVELOPMENT
The vaccine, developing the vaccine as fast as we did, what
is your biggest takeaway, Dr. Collins? How did we do that? And
how can we do it again if we have to?
Dr. Collins. It is really important to look and see that
this was built upon decades of research in basic science that
many people might have said would not probably end up being as
relevant as it turned out to be.
Senator Graham. So, all of our money in the past paid off
here, right?
Dr. Collins. Absolutely. This committee, and then the
Congress, especially over the course of the last 6 years where
you have increased the NIH support by 40 percent, has made it
possible for us to do a lot of things that otherwise we would
still not have been able to start. So, yes, it is all built
upon that foundation.
Senator Graham. Do you feel like the budget request being
made is enough to continue to build on what we have done?
Dr. Collins. I am very supportive of the President's budget
request, as you might expect I would be. And I am particularly
excited about this new proposal of ARPA-H, a new component of
NIH that would give us kind of a DARPA attitude that we could
bring to projects that are waiting for that kind of
opportunity.
Senator Graham. Well, I just hope we can memorialize what
we did to get the vaccine out so quickly.
GLOBAL VACCINE DISTRIBUTION
The developing world--Dr. Fauci, one thing I worry about is
getting the vaccine out into the developing world, particularly
Africa. What can we do better in that regard? And why should
we?
Dr. Fauci. Well, first of all, the answer to your second
question, which is very relevant, Senator, is why should we?
And the reason we should is that a global pandemic requires a
global response. And even though, as you well know from the
numbers, we are doing extremely well in this Country--we now
have over 60 percent of adults having at least one dose, and
about almost 50 percent of the adult population in this Country
fully vaccinated.
However, even if we get this pandemic under control, which
I believe we will within a period of a few months, there is
always the danger, when you have viral dynamics in other parts
of the world, for the generation of variants that might
actually undermine the protectiveness of the vaccines that we
have.
Senator Graham. So, it is in America's interest to get the
vaccine out to as many people as possible?
Dr. Fauci. It is absolutely to our interest. I believe--not
only do I think it is a humanitarian, moral responsibility, but
it is in what I call enlightened self-interest for us to do
that.
ORIGIN OF COVID-19
Senator Graham. So, let's talk about our enlightened self-
interest for a moment. Has there ever been a pandemic that we
know of that started in a laboratory somewhere?
Dr. Fauci. To our knowledge, no.
Senator Graham. Okay. If this were in fact a breach of
protocols in China, if it did come out of a lab, that would be
a first for the world; is that right?
Dr. Fauci. I believe so. There was a situation with an
influenza where there was a suspicion that it might have
escaped from a laboratory in Russia.
Senator Graham. But this----
Dr. Fauci. But that has never been validated or confirmed.
Senator Graham. So, have we found any animals that carry
COVID-19 that could have been the source of the transmission to
humans thus far?
Dr. Fauci. Thus far, not. I mean, if what you are referring
to, Senator, is an intermediate host----
Senator Graham. Right.
Dr. Fauci [continuing]. We know clearly, for example, with
SARS-CoV-1 that a bat virus went into a civet cat, which then
transmitted it into the human population. With MERS, it was a
bat to a camel to human.
The intermediate host, if there is one, has not yet been
found.
Senator Graham. And we have been looking for that
intermediate host; is that fair to say?
Dr. Fauci. That is fair to say, sir.
Senator Graham. At what point in time would it become more
likely it came from the lab if we do not find an intermediate
animal host? How much longer?
Dr. Fauci. I do not think we can give a time element on
that, Senator, for the simple reason we still have not yet
confirmed what the host is from Ebola. We know that Ebola jumps
from an animal reservoir to human, and it has been many years
now since the original Ebola outbreaks, and we have not yet
nailed that down.
Senator Graham. But we believe that Ebola did not come from
a lab?
Dr. Fauci. Yes.
Senator Graham. Okay.
Dr. Fauci. Yes.
Senator Graham. So, I guess my point is, who should look,
what should we be doing to make sure we find out how it
started?
Dr. Fauci. Right.
Senator Graham. And finally, what should be the
consequences to any country, China included that allowed this
to happen? What should the world expect of a country if they in
fact allowed this virus to come from one of their labs through
negligence?
Dr. Fauci. Well, first of all, when you said, who should,
you know, the WHO (World Health Organization) did what they are
referring to now as phase one of an investigation, which they
felt was not completely adequate, as you know. You have heard
me and Dr. Collins and others in the Administration calling for
a continuation of the investigation.
I do not think I can comment on your second question. It
would have to be the circumstances under which something like
that happened, if indeed it happened.
Senator Graham. Well, just very briefly--I know my time is
out--I think we should send a clear signal to China--seems to
be a source of a lot of pandemics--that if this did occur in
the lab, expect something to happen because if we do not, we
are just going to reinforce this in the future. And what that
something is, I am open-minded to, but I am closed-minded to
the idea of doing nothing.
Senator Murray. Thank you. Senator Shaheen.
Senator Shaheen. Thank you, Madam Chairman, and thank you
to you, Dr. Collins, and everyone at NIH for all of your hard
work over the last very difficult year and for everything else
you are doing.
ARPA-H AND DIABETES
As you are aware, diabetes is one of the most expensive and
pervasive of our chronic diseases, and I was pleased that in
the authorization at the end--re-authorization at the end of
the year, we funded the Special Diabetes Program for 3 years
and the work that is being done to advance treatment for Type
1.
But, can you talk about this new ARPA-H agency and to what
extent it might be looking at ways to help address diabetes?
Dr. Collins. I would love to, and thank you for the
question, Senator. This is the hundredth anniversary year of
the discovery of insulin, so we have come a long way in those
hundred years, but we are not where we really need to be to say
we have conquered this one.
ARPA-H, because of its ability to tackle problems in a
team-oriented, nimble way, offers us some new opportunities
here. Certainly, one of the ones that the Diabetes Institute
has been promoting to me of late, sending me ideas, is to
transform the way that we actually develop and test
therapeutics, shouldn't we at this point be able to come up
with therapeutics for diabetes that do not require injections.
A totally new approach to how we would treat this disease.
Another one that I am excited about, and I know you have
done a lot of encouragement about this, is the artificial
pancreas.
Senator Shaheen. Right.
Dr. Collins. And we have made real progress there, Senator.
But, I think we could go a lot faster if we had this
coordinated, ARPA X kind of attitude brought to this, both for
artificial pancreases that are built on engineering and sort of
a feedback loop that gives insulin when it needs to, but maybe
even more so the ones that built upon the patient's own stem
cells that can be converted into that.
Senator Shaheen. And how do we make sure that diabetes is
one of those diseases that ARPA-H addresses?
Dr. Collins. Well, fortunately, because I think we do have
a pretty good budget being proposed here, and diabetes is
already mentioned by the President as one of the three areas of
interest, I think diabetes is extremely likely to be on the
list.
Senator Shaheen. Good. Thank you. I am glad to hear that.
COVID-19 VACCINE BOOSTER SHOTS
Dr. Fauci, the question that everybody is asking is, are we
going to need a booster shot to complement our COVID
vaccination? Do you have any sense of that and what the timing
might be for that?
Dr. Fauci. Two parts to that question, and they are
separate but important. I do not anticipate that the durability
of the vaccine protection is going to be infinite. It is just
not.
Senator Shaheen. Right.
Dr. Fauci. So, I would imagine we will need at some time a
booster. What we are figuring out right now is what that
interval is going to be. We know from studies following people
from the original clinical trials that the protection goes out
at least 6 months, and likely a year. But, we do not know right
now how long that will be.
So, what we are doing is we are following those cohorts
because there is a level of protection that is called a
correlate of immunity, and we know that if you are above that
level, you are in quite good shape to be protected.
The vaccine itself gives you a level up here. So, how long
it takes to start coming back down, we are following it, and
two ways of understanding that. One, does, from a lab
standpoint, it get below a certain level; or, do we start
seeing a lot more breakthrough infections. Either of those
would be a trigger. But, we are following that very carefully.
So, in answer to your first part of your question, I
believe we will need a booster. I am not exactly sure when.
Senator Shaheen. Thank you.
SUBSTANCE USE DISORDER AND METHAMPHETAMINE RESEARCH
And, Dr. Collins, you may remember that New Hampshire is
one of the hardest hit States by the substance use disorder
epidemic. And we have seen a decline over the last year because
of the pandemic, but we have also seen a replacement of many of
those opioids by meth. I think there is a belief among some
people who use substances that meth cannot kill you in the same
way that an opioid can. And, yet, as I talk to providers, they
tell me there are very few treatments that they have available
to them to deal with meth.
So, can you tell me what the National Institute on Drug
Abuse is doing to try and address the meth piece of substance
misuse?
Dr. Collins. Absolutely. This is an area of intense
interest and concern because what was primarily an opioid
crisis is now very much becoming a mixed crisis of opioids and
stimulants, and particularly methamphetamine.
I was pleased to see that NIDA (National Institute on Drug
Abuse) ran a trial, a phase three trial, on treatment for
methamphetamine addiction, which is a combination of injectable
Naltrexone and oral Bupropion, and showed benefit. We have not
previously had anything to offer to help people who are
addicted to meth. That is one step forward.
We also now are running this effort to vaccinate people
against methamphetamine. I know that sounds odd, but you could
immunize against that compound in a way that it would no longer
provide anybody much of a benefit if they decided to use it
anyway. We are doing that for heroin and Fentanyl, and we are
doing it for meth. But it is very helpful.
Senator Shaheen. Excuse me for interrupting. Does that work
if people have already been users?
Dr. Collins. It will. So, basically, getting your immune
system to make an antibody so that in the future, if you
encounter that drug, it cannot get to your brain because the
antibodies grab onto it.
Senator Shaheen. I will have to learn more about that.
Thank you. My time is up.
Thank you, Madam Chair.
Senator Murray. That is very interesting. Thank you.
Senator Kennedy.
Senator Kennedy. Thank you, Madam Chairman, Chairwoman.
GAIN-OF-FUNCTION RESEARCH IN CHINA
Dr. Fauci, I believe you have testified that you did not
give any money to the Wuhan lab to conduct gain-of-function
research. Is that right?
Dr. Fauci. That is correct.
Senator Kennedy. How do you know they did not lie to you?
Dr. Fauci. Excuse me, sir?
Senator Kennedy. How do you know they did not lie to you
and use the money for gain-of-function research anyway?
Dr. Fauci. Well, we have seen the results of the
experiments that were done and that were published and that the
viruses that they studied are on public databases now. So, none
of that was gain-of-function, so----
Senator Kennedy. How do you know they did not do the
research and not put it on their website?
Dr. Fauci. There is no way of guaranteeing that, but in our
experience with grantees, including Chinese grantees, which we
have had interactions with for a very long period of time, they
are very competent, trustworthy scientists. I am not talking
about anything else in China. I am talking about the
scientists. That you would expect that they would abide by the
conditions of the grant, which they have done for the years
that we have had interactions.
Senator Kennedy. So you do not think the Chinese would lie
to you?
Dr. Fauci. Well, when you say the Chinese, the Chinese are
a rather broad group. I know the scientists that we have dealt
with have been trustworthy.
Senator Kennedy. You think all the scientists have told the
truth in terms of the origin of the Wuhan virus and not been
influenced by the communist party of China, do you?
Dr. Fauci. I do not have enough insight into the communist
party in China to know the interactions----
Senator Kennedy. Right.
Dr. Fauci [continuing]. Between them and the scientists,
sir.
Senator Kennedy. Right. Why are we giving them money in the
first place?
Dr. Fauci. Well, that is a very good question, and thank
you for giving me the opportunity to----
Senator Kennedy. You are welcome.
Dr. Fauci [continuing]. Answer it. Well, SARS-CoV-1 started
in China in Guangdong Province, and it went from a bat to a
civet cat to a human.
Senator Kennedy. Yes, and excuse me, Doc, for interrupting
you, but our time is so limited.
Dr. Fauci. No, no. I am going to be real quick.
Senator Kennedy. Our time is so limited. Why are we giving
money to the labs in China to study virology?
Dr. Fauci. Well, I am going to give you a rather succinct
answer to that, sir.
Senator Kennedy. I would appreciate that.
Dr. Fauci. And that is why I was saying the SARS-CoV-1,
clearly the bats that have the viruses that are the
coronaviruses are in China. As I said a couple of times, it is
not in Fairfax County, Virginia or is it in New York. It is in
China. So, if you want to show and study importantly the
animal-human interface, the viral----
Senator Kennedy. Because that is where the bats are?
Dr. Fauci. Yes, the bats.
Senator Kennedy. Okay. I got it.
Dr. Fauci. That is where the bats are.
Senator Kennedy. I want to be sure I understand your
testimony. You did not give money to the Wuhan lab to do gain-
of-function research?
Dr. Fauci. That is correct.
Senator Kennedy. And you believe they did not do gain-of-
function research because they told you they did not?
Dr. Fauci. We have seen the results of the studies that
they conducted and they were not gain-of-function.
Senator Kennedy. Including any private studies?
Dr. Fauci. Excuse me? Including?
Senator Kennedy. Any private studies.
Dr. Fauci. I am not sure what you are getting at, sir.
Senator Kennedy. Here is what I am getting at. You gave
them money and you said, don't do gain-of-function research.
Dr. Fauci. Correct.
Senator Kennedy. And they said, we won't?
Dr. Fauci. Correct.
Senator Kennedy. And you have no way of knowing whether
they did or not except you trust them; is that right?
Dr. Fauci. Well, we generally always trust the grantee to
do what they say, and you look at the results----
Senator Kennedy. Have you ever had a grantee lie to you?
Dr. Fauci. I cannot guarantee that a grantee has not lied
to us because you never know.
Senator Kennedy. Yes. Can we agree that if you took
President Xi Jinping and turned him upside down and shook him,
the World Health Organization would fall out of his pocket?
Dr. Fauci. I do not think I can answer that question, sir.
I am sorry.
Senator Kennedy. Well, do you think President Xi Jinping
has undue influence over the World Health Organization, do you?
Dr. Fauci. I have no way of knowing the influence of the
president of China over the WHO.
Senator Kennedy. Okay. So you think the WHO is a completely
independent body and level playing field, call-it-like-you-see-
it, and they really want to get to the bottom of the origin of
the virus? Do you believe that?
Dr. Fauci. My interaction with the WHO and for Dr. Tedros,
the Director General, has been one----
Senator Kennedy. Okay.
Dr. Fauci [continuing]. That I do believe he is a person of
high degree of integrity.
INVESTIGATION INTO ORIGIN OF COVID-19
Senator Kennedy. I got it. I want to ask one last question.
Why did you guys spike--not guys, and ladies. Why did you all
spike the prior administration's investigation into the origins
of the coronavirus and whether it could have come out of the
Wuhan lab?
Dr. Fauci. Sir, I--we did not spike anything in the prior
administration. I am not sure what you mean by spike. But, we
have no influence----
Senator Kennedy. The State Department spiked the prior
administration's study.
Dr. Fauci. But that has nothing to do with the National
Institutes of Health.
Senator Kennedy. So they did not consult with you all?
Dr. Fauci. They did not.
Senator Kennedy. Did they consult with you, Dr. Collins?
Dr. Collins. I read about it in the press this morning.
Senator Kennedy. Doc.
Dr. Bianchi. No.
Senator Kennedy. They just spiked it without talking to
their experts?
You do not want to answer that one, do you?
Dr. Collins. I just read about it.
Senator Kennedy. Thank you, Madam Chair.
Senator Murray. Senator Murphy.
Senator Murphy. Thank you very much, Madam Chair.
Listen, the World Health Organization is the most
influential global public health institution in the world,
whether my friends like it or not. They have more people and
more influence on the ground across the world than anybody
else, including the United States.
And, so, if the complaint is that any country, including
China, has too much influence, the answer is not for the United
States to walk away. The answer is for the United States to
double down and make sure that any grievances we have are
addressed. Otherwise, the problem for which you are identifying
is exacerbated by the United States not being at the table with
the WHO.
And while the major donors to that organization certainly
have lots of influence, as is the case with every international
organization, it is an oversimplification to suggest that they
are in the pocket of the Chinese government. China has
influence. The United States has influence, as well, so long as
we are at the table.
FIREARMS RESEARCH
I have two areas to cover, and the first I wanted to raise
with you, Dr. Collins, and that is around the budget request to
double the firearm injury and mortality prevention research
account. Let me place myself solidly behind that request. Thank
you for making it, and I was hoping you might--I apologize if
you have gotten a question on this already. I have been
listening but in and out a bit.
I am hoping that you might be able to talk a little bit
about how you might prioritize that additional funding,
especially as it might relate to research on community-based
interventions and what works and what does not. And, then, you
know, how to make sure that all that information gets out to
community partners, folks who are boots on the ground, maybe
not the exact set of players that NIH is used to disseminating
information to.
Dr. Collins. Well, I appreciate the question, and we are
enthusiastic about expanding our approach and the amount of
funds we can put into research on firearm violence. After all,
some 40,000 deaths happen each year from firearms. About 60
percent of those are suicides, which is another topic that came
up earlier and is also part of our suicide prevention, is to
think about availability of guns.
I think you are right, though, that community approaches
are very much ripe for this kind of approach, where you might
not just try to change one thing in the community, but see if
by coordinating the efforts across multiple different ways in
terms of making sure that firearms are not accessible to those
people who might misuse them; in terms of particularly
adolescent and youth risks of violence and how to intervene.
Maybe we could take an approach that would be more holistic
as opposed to trying to fix one thing at a time. With a larger
amount of funding here and a community focus, I think we might
be able to do that.
Senator Murphy. The President has proposed, I think, $5
billion to support these community-based interventions. Maybe
some of that will be used for assessment and study. But, given
the fact that I think we probably can get bipartisan agreement
about supporting these investments in prevention, it really
would be helpful to use some of this increased funding to
assess which ones work and which ones do not.
SOCIAL DETERMINANTS OF HEALTH
Second broad topic, and maybe I will address this both to
Dr. Collins and I think, via video, Dr. Perez-Stable, is on the
topic of social determinants of health. And I am just
interested to hear a little bit about how we have adjusted
research based upon our growing understanding that people's
health is dictated by where they live and how much money they
make and how close they are to pollution sources.
My sense is that, you know, this is not an easy sort of
thing to incorporate into a research community that is sort of
used to working in labs and not always used to thinking about
how factors outside the body impact health. What have we
learned? How has that changed the way that we fund research and
encourage applications to come to NIH that might support social
determinant research?
Dr. Collins. I am going to ask Dr. Perez-Stable to respond.
Dr. Perez-Stable: Thank you, Dr. Collins, and thank you,
Senator Murphy, for that important question.
At the National Institute on Minority Health and Health
Disparities, and throughout NIH, the topics of social
determinants of health have always been present. We consider
self-identified race and ethnicity and socioeconomic status
standard measures to be fundamental factors that influence
health in ways that we do not really understand, and that is
why we believe that all research with human beings should
measure these routinely and follow them.
In addition to these two, though, there are other
demographic and individual social determinants of health, of
which many are issues related to age and gender, sexual
orientation, but then structural social determinants of health
that you refer to. Where one lives, plays, and prays, relate to
both transportation, housing, and issues around green space
and, of course, Internet access, which has become incredibly
important, as we know, in the last year. So, we have these
fundamentally incorporated into our standard research, and
community engagement is really part of everything that we do at
NIMHD, and increasingly across the Agency.
Senator Murphy. Well, thank you for that. I appreciate the
new focus you are putting on this. Again, this is an area of
potential bipartisan agreement. Senator Sullivan and I have
legislation in this space and look forward to working with you
on it.
Thank you, Madam Chair.
Senator Murray. Thank you. Senator Shelby, are you ready?
You want me to----
Senator Shelby. Yes, I am ready.
Senator Murray. Okay.
Senator Shelby. I just got here. Thank you. I have been at
another hearing, and this question may have been asked.
Dr. Collins, always good to see you.
Dr. Collins. Likewise.
Senator Shelby. I agree with a lot of people on this
committee that the money we put in to biomedical research
benefits mankind, period. Not just our people, but the world,
what it has taught.
AUTOIMMUNE RESEARCH BREAKTHROUGHS
Two or three promising areas, biomedical research in the
area of autoimmune--that is a big, big topic. You know it
better than anybody. What are we--what are the breakthroughs
there, the hopes, in two or three of those top areas?
Dr. Collins. Well, thank you, Senator. It is good to see
you, and I know you are running from one place to another. I am
glad you are here.
I just had a wonderful experience yesterday afternoon
listening to presentations from a consortium of researchers
that we have funded jointly with industry. So, this is called
the Accelerating Medicines Partnership, and it is focused on
rheumatoid arthritis and lupus.
What they have done is to take this field, which was
looking at immunology in a way that was pretty cutting edge 5
years ago, and now completely transformed it by looking at
individual immune cells in the synovium of people with
rheumatoid arthritis--the lining of the joint--and say, what
are you doing there, immune cells, and how does that teach us
what the real pathogenesis about----
And for lupus, they are looking at kidney biopsies,
because, of course, lupus affects the kidney and that is one of
its serious consequences. Same thing, looking at individual
cells.
It has completely revamped our understanding of these
diseases. We have learned, for instance, that the pericyte,
which was just sort of a cell that we thought was hanging out
watching in the kidney of somebody with lupus, might be the
driver of what is really happening there as far as the immune
response. This is not p-a-r-a. This is p-e-r-i, cyte, in case
that is not clear. For rheumatoid arthritis, it is the
fibroblasts.
And we are so excited about this. We are now planning to
expand that same approach to other autoimmune diseases, to
psoriasis, to psoriatic arthritis, to Sjogren's Syndrome, and
maybe others, as well.
So, you hit me at a great moment. I was so jazzed yesterday
to see what has been possible.
Senator Shelby. All based on bacteria, is it?
Dr. Collins. It is all based on this ability to look at
single cells, one at a time. We have not really been able to do
that until about 5 years ago. We would have to look at
thousands of cells and try to infer what was there, and now you
can ask each one. And the cell is, after all, the basic unit of
all life, and it has been outside of our reach, but not
anymore.
Senator Shelby. What could that do for the autoimmune area?
Dr. Collins. I think it can have a huge impact because we
now have new targets coming out of this recognition that I
think in the next 4 or 5 years, we are going to see a whole new
generation of drugs for autoimmune diseases based upon that
insight that is just now emerging.
CYSTIC FIBROSIS RESEARCH
Senator Shelby. I brought this up many a time, but in the
area of cystic fibrosis, there have been so many breakthroughs
in that area, extending children's lives, adults' lives, and
everything. Where are we going there? We have come a long way,
but we are not there yet.
Dr. Collins. We are not completely there, but, oh, boy,
have we come a long way, especially in the last 2 years now
with this 30-year effort, and I have been deeply engaged in
this having had a role in----
Senator Shelby. I know.
Dr. Collins [continuing]. Discovering the gene back in
1989. And, now, we have this triple drug therapy, which for 90
percent of patients with cystic fibrosis is dramatically
beneficial. I get messages almost every week from somebody who
was really in tough shape, and now they are back at work; or
somebody who was on a transplant list, and now they were taken
off of it because their lungs are doing so much better.
But, there is still that 10 percent. This is where I think
the gene-editing approach, where you actually figure out how to
fix that misspelling of the cystic fibrosis gene in the lungs
of somebody who is affected, might be the way to get to 100
percent, and there is a lot of work going on that.
LUPUS RESEARCH
Senator Shelby. What promises are out there that you have
talked about before dealing in lupus, which is an autoimmune
disease?
Dr. Collins. Well, as I mentioned, we have this ability now
to be able to see individual immune cells, what are they up to
in lupus, both in the kidney and in other areas, as well. I
think that is teaching us some new things about what the real
fundamental cause is. And it will tell us that some of the
treatments we have been giving, like steroids, are kind of a
little bit too much of a sledgehammer, and what we need now is
something much more subtle to go after the fundamental problem.
We have a better chance at that now.
PANCREATIC CANCER RESEARCH
Senator Shelby. What about the area of pancreatic cancer?
That is a fast-moving thing, I know.
Dr. Collins. It is, indeed. And if Dr. Sharpless is
listening, maybe he would like to quickly give a response since
that is his area at the Cancer Institute. Ned, are you there?
Dr. Sharpless. Sure. Yes. Thank you, Francis.
Pancreatic cancer is an area where we have not seen the
success that we have seen in other cancers, but it is not for
lack of good ideas. So, there are a number of----
One of the realizations is that pancreatic cancer comes in
lots of flavors, and each one needs its own treatment. So, now
we are working on the subset approach to pancreatic cancer. I
think there is also a real opportunity to detect pancreatic
cancer earlier at a more curable stage.
So, I think those are the exciting areas of pancreatic
cancer research.
Senator Shelby. Thank you. I would like to get in--I know
my time is moving on. The chairperson has been very kind.
CTSA PROGRAM
Dr. Collins, in the area of the CTSA Program, the Clinical
and Translational Science Award Program. The CTSA hubs and
their partners, I think, have done a lot of good work in that
area, and valuable work, especially during the COVID-19 thing.
It is my understanding that the NIH, National Institutes of
Health that you head, is considering significant changes to
that program that would discourage hubs, like UAB, for example,
in Birmingham, from forming partnerships with certain non-
clinical universities in research questions.
Is this true, and why is that?
Dr. Collins. That is not a correct assumption. I know there
are some rumors flying around about that, and there will be a
public announcement about this.
Basically, just, without trying to get too far ahead of
what has not been revealed publicly, I think we are trying to
simplify the application process to make it easier for those
hubs, and we intend to keep them going in vigorous ways; to
apply when they are up for renewal in a way that does not
require an application of 2,000 pages, which is what it has
been. But, we would not want to do anything to discourage these
collaborations that you are mentioning. Take that from me.
Senator Shelby. Thank you. Madam Chair, thank you.
Senator Murray. Thank you. Senator Manchin.
Senator Manchin. Thank you, Madam Chairman, and thank all
of our presenters. I appreciate very much them being here.
DOMESTIC DRUG SUPPLY CHAIN
My first question will go to Dr. Fauci. The Food and Drug
Administration reports that nearly 40 percent of finished drugs
and roughly 80 percent of active pharmaceutical ingredients are
manufactured abroad. During the COVID-19 pandemic, we saw
factories shut down in order to prevent the spread of virus,
drug supply chains disrupted, and drug shortages increase. As a
result, America's access to essential medicines was really put
into jeopardy.
As a preeminent infectious disease doctor, you know better
than anyone how important it is to have access to essential
medicines. So, my question will be, Doctor, can you comment on
the importance of a strong domestic supply chain for essential
medicines? And how can we ensure we do not experience future
drug shortages when the global supply chains are disrupted?
Dr. Fauci. Thank you very much for the question, Senator
Manchin. I think it is absolutely critical that we have the
capability, independent of supplies from foreign countries, to
be able to supply the necessary medicines that we need in the
United States. I have been of that opinion for a very long
period of time.
The solution to the problem is to be doing much less of the
outsourcing to foreign countries for the important ingredients
of many of our medications. So, right now, we are not in that
good position, and I believe, particularly since the
disruptions of the supply chain that have occurred with the
COVID-19 pandemic, that this might be a good lesson for us for
the future to make sure we have much more dependency on what we
can do domestically as opposed to in foreign nations.
Senator Manchin. Doctor, have you all looked at why? Why
has most of the manufacturing left the United States and why
are we not able to manufacture? Are we at a disadvantage in the
United States for other reasons, cost wise, or basically
different types of things, that we make people jump through
hoops and everything else as far as permitting and all that?
What would be the cause?
Dr. Fauci. You know, Senator, to be honest with you, I do
not know why that has happened. I think it was because it was
felt it would be much less expensive to get this done outside,
but I do not really know the answer to your question of why we
have so much of a dependency of important materials outside of
the Country. But, certainly, whatever the reason, I believe it
needs to be corrected.
Senator Manchin. Well, I need to work with you on that,
Doctor, if I can, basically, in making sure this
Administration--I think they understand the urgency we need to
start basically manufacturing again, not only just our drugs,
but so many things in our Country. So, I look forward to your
support on that.
RURAL HEALTH OUTCOMES
Dr. Collins, West Virginia is constantly ranked last in the
Nation for health outcomes. In 2020, the America's Health
Rankings reported my State of West Virginia 50th for premature
deaths, frequent mental distress, and multiple chronic
conditions. We also ranked last in life expectancy.
What is the NIH doing to bridge this gap in health
outcomes? And how do you ensure that the medical research that
you do benefits people in poor, rural communities?
Dr. Collins. Well, it is very troubling to see the fact
that you have just cited that health outcomes are not what we
would all want them to be. And, of course, there are many
factors that play into that, Senator, and we are deeply engaged
in research in trying to identify the ones that are
addressable.
Certainly, one of the things I might point to is the
increasing focus we have on disease prevention. If we simply
are limiting ourselves to trying to help people who have
already developed a serious disease, we have kind of missed the
opportunity. Unfortunately, our healthcare system does not do a
great job in that situation of providing support for disease
prevention, and it seems happier to pay for things once people
are already quite ill, so there is additional work that needs
to be done there.
One of the things that I think I would point to is a series
of large-scale efforts to really understand what are the
factors that play out in people staying healthy or getting a
chronic disease or how you manage that.
The All of Us Program, which this Congress has supported,
on the way to enrolling a million participants, including in
West Virginia, is a way in which we can collect that kind of
evidence, including their electronic health records and lots of
information about their environmental exposures, and try to
figure out in a holistic way, how can we take that information
and bring forward a better chance for people to live not just a
good lifespan, but a good health span. So, we are----
Senator Manchin. Thank you, Doctor.
Dr. Collins [continuing]. Deeply engaged.
Senator Manchin. Thank you, sir.
Dr. Fauci, finally, you know, my home State of West
Virginia is battling an epidemic during the middle of a
pandemic. We have been devastated by the drug epidemic, COVID-
19, and now--we now lead the Nation in new HIV infection rates.
You spent much of your career focused on prevention, diagnosis,
and treatment of HIV/AIDS, and your research has been
instrumental in saving countless lives in the United States and
around the world.
INFECTIOUS DISEASE SURVEILLANCE EFFORTS
So, Doctor, what is being done to replicate testing and
surveillance efforts we saw put into place for COVID-19 for
other infectious disease, like HIV/AIDS? And what public health
infrastructure would be required to bring better infectious
disease testing and surveillance to fruition?
Dr. Fauci. Thank you for that question, Senator. The HIV
testing situation, unfortunately, has been somewhat interrupted
by the COVID-19 pandemic because of the interruption of
multiple services.
But, as you know, we have a 10-year plan to end HIV as an
epidemic in the United States, and that is going to require
access to testing for those who are not infected to put them
on, if they are at risk, to pre-exposure prophylaxis; and those
who are infected to immediately put them on antiretroviral
therapy. Because, as we know, when you bring the level of virus
to below detectable, not only do you save the life of the
individual, but you make it essentially impossible for that
individual to infect someone else.
So, testing is really at the fundamental basis of how you
address the epidemic and, for that reason, it is going to be
extremely important to get our testing capabilities back up to
snuff once we get the Country back on a degree of normality
following control of the COVID-19 pandemic.
Senator Manchin. Thank you. Thank you, Madam Chairman.
Senator Murray. Thank you. Senator Braun.
Senator Braun. Thank you, Madam Chair.
Dr. Fauci, I was listening with interest in Senator
Kennedy's line of questioning, which probably was asking you to
maybe answer some things based upon what the WHO should do or
not.
INVESTIGATION INTO ORIGIN OF COVID-19
I would like to discuss something that is probably a little
simpler to answer in terms of transparency in general. From the
time I have known you and Dr. Collins, it has generally been in
this seat, and we have been talking about something related to
COVID. Would you agree that in the whole process of--now that
there are second thoughts on how this thing derived, that it
may have come from a lab, that we should emphasize as much
transparency as possible in pursuit of getting the answer?
Dr. Fauci. Without a doubt, Senator. No doubt.
Senator Braun. And the next logical question would be that
we do not know what we are going to get from the communist
regime or the WHO, but we do know that through our Director of
National Intelligence and probably DHS (Department of Homeland
Security), from Haines and Mayorkas, that they have probably
got information there. And, so, since you believe in
transparency, wouldn't you think that we should declassify all
the information that we own so that you, Americans, independent
researchers, can see what we have got to sort through how this
thing started?
Dr. Fauci. Well, Senator, I have said publicly and most
recently that I believe that there should be transparency, and
open, fair, and independent, continue to look. As I have said,
I still believe that the most likely scenario is that this was
a natural occurrence, but no one knows that 100 percent for
sure. And since there is a lot of concern, a lot of
speculation, and since no one absolutely knows that, I believe
we do need the kind of investigation where there is open
transparency and all the information that is available to be
made available to scrutinize.
Senator Braun. So, since you have been the point person on
just a variety of topics through the COVID saga, does that mean
then that you will ask President Biden to declassify that
information?
Dr. Fauci. I do not think I can promise you----
Senator Braun. But, I mean, would you ask him since you
believe in transparency? Wouldn't it make sense that we get the
information that we have? And I think if it does not come from
you, Dr. Collins, someone that has been in the mix from the
get-go, that we will not see it. And we owe it to the American
people with what we have been through to at least look at the
information that we have.
Dr. Fauci. Yes. I am not sure the information we have,
but--I am not sure if it is my place to tell the President of
the United States to declassify----
Senator Braun. But you have been very engaging on a wide
range----
Dr. Fauci. Right.
Senator Braun [continuing]. Of topics, and I think he would
respect your opinion as much as anyone.
Dr. Collins, where are you at on that subject of giving the
American people the information that we house?
Dr. Collins. Well, I am very much where Dr. Fauci is with
the desire to be as transparent as possible in this situation
and really try to find out what happened. I agree with him that
it is most likely that this is a virus that arose naturally,
but we cannot exclude the possibility of some kind of a lab
accident. That is why we have advocated very strongly that WHO
needs to go back and try again after the first phase of their
investigation really satisfied nobody, and this time we need a
really expert-driven, no-holds-barred collection of
information, which is how we are mostly really going to find
out what happened.
I am just not in a position to know what might be in the
classified documents and what else might be there that would
not be relevant to this and might actually be harmful to
national security. I get--I take your point. But, I know the
President is very interested, also, in seeing truth come out
here, so it may not require Tony or me to tell him that this
would be good, to make this as visible as possible.
Senator Braun. Well, I think for the American public, if we
are relying on the WHO to do it again, even though it seems
like they have had somewhat of an epiphany that we need to dig
deeper. I think if it does not come from the two of you to ask
for simply the release of information, of course, keeping
hidden anything that would be something that could not be
exposed. But, I am guessing there is a good bulk of that that
would be benign in terms of just the information we have about
the origin of the disease.
So, I think for many of us, many Americans, with what we
have gone through, we ought to at least be willing to look at
the information that we have to get people satisfied that we
are getting to the bottom of it. So, I would ask each one of
you to think about that and see if it makes sense, have our
President declassify it so we can see it.
Dr. Collins. Thank you.
Senator Braun. Thank you.
Senator Murray. Thank you. Senator Moran.
Senator Moran. Chairman, thank you.
Dr. Collins--well, Doctors, welcome. Good to be here with
you, and I appreciate your presence and your work.
Let me talk about clinical and translational science, if I
could. Under Dr. Austin's prior leadership, the National Center
for Advancing Translational Science at NIH has been essential
in facilitating clinical and translational research, and I have
seen it in Kansas. In fact, I have seen it with the director of
that directorate.
CTSA PROGRAM
In Kansas, NCATS' Clinical and Translational Science Award
Program has served for a catalyst to bring lots of
organizations in the research community and community partners
together to advance research.
I have concerns with potential changes that are under
consideration for the CTSA Program. In particular, changes that
would lower hub awards and limit CTSA partners.
Moving forward, will there continue to be consideration for
ensuring that CTSA centers are located in regions in the U.S.
which do not already have those hubs? There is already a
limited number in the Mid-West, and I would be concerned if any
new changes to the program that would make it more difficult
for these hubs to compete.
And, then, I would ask the question about partners. At the
University of Kansas, for example, they partner with Children's
Mercy, Kansas City University of Medicine and Biosciences,
Kansas State University, St. Luke's Health, University of
Kansas Health System, KU Office of Research, KU School of
Medicine in Wichita, and University of Missouri in Kansas City.
Since the CTSA Program is focused on partnerships between
regional research hubs and community partners, why would NCATS
limit the ability of the program, in my view, to accomplish its
goal?
Dr. Collins. Well, Senator, thank you for the question. I
am a big fan of the CTSA Program and enjoyed my opportunity to
travel to Kansas with you and see some of the things they were
doing a few years ago.
And this is, I think, one of those circumstances where
there seems to be some anxiety in the CTSA community about
something that has not actually been announced yet, and I would
like to be reassuring about this. The real intention of the
change that is being proposed is to de-complicate the renewal
process, which currently requires an application of about 2,000
pages that I do not think anybody enjoys putting together, and
to try to make this more straightforward.
There is no intention to reduce the number of hubs.
Certainly, every hub has to compete to show that they are
actually using the funds wisely, and we will continue that
process. And this notion that somehow the new process will
discourage collaborations with other institutions I find a
little hard to understand because I have no knowledge that that
is at all intended to be the case, and I would personally
oppose that.
Senator Moran. Thank you for your reassurance. My question
was more complicated than I wanted it to be, but your answer
was very comforting.
Let me ask just a couple of specific questions.
NCATS RARE DISEASE RESEARCH
What can this committee do to support NCATS' efforts to
enable and facilitate advanced important research in rare
diseases for patients living particularly in rural communities?
Dr. Collins. Well, the NCATS is deeply engaged in rare
diseases. Our former director, Chris Austin, not only was a
personal promoter of that; he was the head of the international
committee for rare diseases, and that tradition will continue
under Acting Director, Dr. Rutter.
Certainly, the support that this committee has provided to
NCATS to make it possible for that kind of investment to happen
in rare diseases, for which companies probably are not going to
make an investment because the market is too small, is one of
the reasons that we have now made really significant progress
in dozens of these rare diseases.
We are also engaged right now in a serious conversation
with industry about whether there is a way, with gene therapy
emerging as an even more attractive opportunity for rare
diseases, to make sure that we move that forward at all due
speed and not have it held up by such things as a limitation in
manufacturing of viral vectors.
So, they are right in the middle of that, and the support
that you all have provided has made that possible, particularly
through the Cures Acceleration Network, which is part of NCATS.
ALZHEIMER'S DISEASE RESEARCH
Senator Moran. Can one of the directors talk about the
improved science this additional investment in Alzheimer's
research will help fund, including a better understanding of
risks and protective factors in individuals, again perhaps with
a focus on rural populations?
Dr. Collins. That is probably me because Dr. Hodes is not
here. So, yes, this committee, this Congress, has increased
funding for Alzheimer's research by five-fold over the course
of the last 7 or 8 years, and that has made possible all kinds
of bold approaches we otherwise would not have had.
We now have dozens of new drug targets that have emerged
from the very careful analysis of who gets Alzheimer's and who
does not. Of course, we are all waiting to see what happens
maybe next month when FDA makes a review decision about the
monoclonal antibody from Biogen, Aducanumab, and that will make
a big difference if they decide there is something there. But,
we are not depending on that.
So, yes, I might add, this ARPA-H proposal, which is part
of the President's budget, specifically calls out Alzheimer's
as an area of great opportunity to do some of these very bold,
aggressive, and nimble approaches that would probably not
happen so easily by our standard grant mechanism.
Senator Moran. Dr. Collins, I was confused by what I
thought was all the directors were appearing, although just not
all of them in person. But, thank you. You can pinch-hit for
each and every one of them and you did it----
Dr. Collins. I will try.
Senator Moran [continuing]. This morning. I am going to see
if I can get Dr. Sharpless to come to Kansas and join us again
on a visit.
Dr. Collins. Well, he is listening, so he heard you.
Dr. Sharpless. Oh, I look forward to that.
Senator Moran. All right. Consider yourself invited, and I
consider you just accepted.
[Laughter]:
Senator Murray. Thank you. Senator Schatz.
Senator Schatz. Thank you, Chair Murray and Ranking Member.
PSYCHEDELIC DRUG THERAPIES
Dr. Collins, in 2019, I wrote to you and the then-FDA
commissioner requesting an update on efforts by NIH and FDA to
research psychedelic drugs to treat mental health illnesses.
Since then, there have been a number of potentially promising,
peer-reviewed clinical research on this topic. Can you give me
an update on what the next steps may be?
Dr. Collins. I appreciate the question. Yes, there has been
a resurgence, I think, of interest in psychedelic drugs, which
for a while were sort of considered like not an area that
researchers legitimately ought to go after. And I think as we
have learned more about how the brain works, we have begun to
realize that these are potential tools for research purposes
and might be clinically beneficial.
I will just mention one, which is Psilocybin, which has now
been tried in no less than three randomized, controlled trials
for depression, and is showing a signal there of potential
interest, and that could be quite exciting because we are
looking for new approaches to that.
But, there are other trials going on with MDMA, even with
Psilocybin--with LSD. I think at the moment, it is the
Psilocybin that has gotten the greatest attention.
Senator Schatz. And what are your next steps?
Dr. Collins. I have been talking with the Drug Abuse
Institute--and I am sorry they are not here--and the Mental
Health Institute--and they are not here, so I am pinch-hitting
for them, as well--about whether it is a good moment to
consider having perhaps a workshop to say, okay, what have we
learned so far, and what more might we want to do as far as
designing the next generation of clinical trials, to see where
these provide benefit going beyond depression to such things as
PTSD (Post-Traumatic Stress Disorder).
So, I think over the course of the next year, we are going
to want to have a hard look at this.
MARIJUANA RESEARCH
Senator Schatz. Thank you. In 2019, you wrote to me that
the NIH is committed to advancing research on the risks and
potential benefits of marijuana for therapeutic uses. In that
letter, you cited a number of barriers to advancing this type
of research. Are we making any progress?
Dr. Collins. We are making some progress. You may know
that, in the past, researchers who wanted to do a clinical
study on marijuana had all kinds of limitations. It took
generally at least a year to get through the process of
paperwork to be allowed to utilize marijuana because it is a
Schedule 1 agent.
But, it was also an issue that there was only one source,
which was our marijuana farm in Mississippi. When I became NIH
director, I was told, hey, you are running a marijuana farm.
Who knew? And that, of course, is an issue because it is a
limited opportunity for access. DEA (Drug Enforcement
Administration) has now given permission to expand the number
of suppliers. That will help.
But, frankly, what we really need is to moderate the
Schedule 1 limitation. Dr. Volkow and I have been proposing for
a while something called Schedule 1-R, which would be basically
a different pathway if you are going to use this material for
research.
Senator Schatz. So, I have a bill with Senators Feinstein
and Grassley, which passed the Senate, did not pass the House,
to address some of these barriers. Do I have your commitment to
work with my office on this legislation?
Dr. Collins. I would be glad to.
NON-OPIOID ALTERNATIVES TO CHRONIC PAIN
Senator Schatz. Thank you. I want to talk to you finally
about chronic pain and non-opioid alternatives. I passed a
couple of laws in this area to enable research. And I think
when people think about alternatives to opioids, they move
right to--in their mind, they move right into alternative
medicine. And, what I am talking about is a non-opioid,
pharmaceutical solution to chronic pain, and I am wondering
whether we are making progress in that space.
Because, certainly, if people find other ways to alleviate
their pain--physical therapy, yoga, whatever, mindfulness--I am
for all of it. But, there is still a space here for a pill that
you can take to alleviate chronic pain without getting you
hooked on an opioid. Where are we with this?
Dr. Collins. That is a critical issue, and this Congress
has supported NIH in something we call the HEAL Initiative,
which is--stands for Helping End Addiction Long Term. Part of
that is about how to better treat people who are addicted to
opioids, but a big part of it is coming up with alternatives
for chronic pain management that are not addictive, that are
not opioids.
As a result of that, we have partnered up with industry to
basically identify promising therapeutics that attack different
targets in the pain mechanism that might, therefore, be
beneficial. Such things as a sodium channel, for instance,
called Nav1.7, that is involved in the pain transmission. But,
if you block that, it should not give you any risk of
addiction. We are making real progress there.
We have something called EPPIC-Net, which is bringing
onboard promising compounds, getting them into Phase 2 trials
as part of the HEAL Initiative. I could give you a lot more
information about that if you would like.
Senator Schatz. Thank you. And I will just submit this one
that you can consider for the record.
The U.S. has the same Federal trust responsibility for
native Hawaiians as it applies to Alaska natives and American
Indians, and I am hoping that you will consider expanding the
scope of the Tribal Health Research Office to include native
Hawaiians. I will get you a more full question for the record
and look forward to your response. Thank you.
Dr. Collins. Glad to look at that.
Senator Murray. Thank you. Senator Hyde-Smith.
Senator Hyde-Smith. Thank you, Madam Chairman. Thank you
for holding the hearing, and thanks to all the witnesses who
are participating today, and I certainly appreciate your
willingness to serve. That is not lost here, for sure, with the
past year that we have had.
FIREARMS RESEARCH AND FIREARM REGISTRIES
Dr. Collins, I wrote to you last November to express my
concerns that projects recently funded by NIH disregard the
spirit, long-established policies against creation of a Federal
firearms registry. And particularly, an NIH grant to Northwell
Health of New York provided Federal funds for the hospital to
ask the questions about lawful gun ownership of every patient
seeking healthcare for any reason whatsoever at the hospital's
emergency department.
Even more concerning, every member of the advisory
committee overseeing the grant has been a very outspoken
advocate for expansive gun control, including bans on large
classes of common and popular firearms.
I have long been concerned about how firearm registries can
undermine the ability of law-abiding citizens to exercise their
Second Amendment rights. Several provisions of Federal law
already prohibit data collection related to lawful gun
ownership, and I have introduced legislation to strengthen
these provisions even further.
Dr. Collins, given that President Biden is seeking
increased funding for grants like the one awarded to Northwell,
how are you making sure that such projects do not infringe on
Americans' constitutional gun rights or violate Federal
statutory prohibitions on gun registries as they stand right
now?
Dr. Collins. Senator, I recall your letter, and we looked
closely at that particular grant from Northwell and what they
were proposing to do.
First of all, I think we can all agree that gun violence,
which takes about 40,000 lives every year, is something that
does deserve close attention and scrutiny as far as the
research that we might be able to do to understand what are the
causes and how to save those lives if it is possible to do so.
So, we will actually be glad to pursue those opportunities.
But, we are mindful of the prohibition that Congress has
put forward many years ago about not advocating for gun
control, and we have been pretty careful about that. I think in
that instance, the particular grant, while you are right that
they were asking for this information, it fell somewhat short
of what most people would have called a broad concept of a gun
registry. And I think that is, if I remember, what we said in
the letter in response to you.
But, I want to promise you, we are going to be very
sensitive to those issues, as we now, with the President's
budget, seek to see if we can do more to try to identify
reasons that gun violence is so prominent and what research
might teach us about how to save lives.
Senator Hyde-Smith. Thank you. I appreciate your
consciousness of that.
ORIGIN OF COVID-19
And this question may have been asked before. I have been
in another hearing. I hope I am not being redundant. But, like
many of my colleagues, I firmly believe we need to get to the
bottom of the origin of COVID-19, and this seems even more
important after this week's Wall Street Journal report that
three researchers from China's Wuhan Institute of Virology
sought hospital care in November 2019--for symptoms consistent
with COVID-19.
First, I want to go down the line for all of our witnesses
of how strongly do you believe that it is possible that the
origin of the COVID-19 pandemic resulted from a leak of the
virus from the Chinese lab?
And second, Dr. Fauci, I would like to ask you
specifically, how is your institute working to get to the
bottom of the origins of COVID-19, including exploring the
laboratory leak theory?
So, I am going to start with the entire panel for the first
question of, how strongly do you believe that this is possible?
Dr. Collins. Well, I will start, and then others can
respond. Again, I will say, I think the most likely reason,
mechanism, by which SARS-CoV-2 arose was a natural process of
transfer from an animal to humans, but it is certainly possible
that other options might have occurred, including a possible
lab leak. We just do not have evidence to be able to say what
that likelihood is.
Dr. Bianchi.
Dr. Bianchi. Yes. So, I would agree with Dr. Collins. We
have no personal knowledge of anything that might have happened
in China at the National Institute of Child Health and Human
Development, but we fully support a full investigation of
getting at the facts.
Dr. Collins. Dr. Gibbons. Dr. Gibbons, are you there?
Dr. Gibbons. Yes. I concur with my colleagues in terms of
transparency is a critical part of this.
Dr. Collins. Dr. Sharpless, I think I saw you on the
screen.
Dr. Sharpless. Sure. Yes, Senator Hyde-Smith, I saw the
same report and I found that concerning. I think lab accidents
happen and we need to investigate the possibility. Although I
think many of us feel zoonotic transfer is perhaps more likely,
I think we should investigate all possible explanations.
Dr. Collins. Dr. Perez-Stable.
Dr. Perez-Stable. I concur with my colleagues. I think of
concern, but certainly we need evidence.
Dr. Collins. And Dr. Tromberg.
Dr. Tromberg. Yes, I agree with my colleagues, as well, and
would like to see more investigation.
Dr. Collins. Dr. Fauci.
Dr. Fauci. Yes. As I have said many times, I feel the
likelihood is still high that this is a natural occurrence.
But, since we cannot know 100 percent whether it is or is not,
other possibilities exist and, for that reason, I and my
colleagues have been saying that we are very much in favor of a
further investigation to the next phase from the WHO, who has
already done a phase one. And, we are strongly in support of
continuing that to a phase two investigation.
Senator Hyde-Smith. Thank you----
Senator Murray. Thank you.
Senator Hyde-Smith [continuing]. Very much, and I yield my
time.
Senator Murray. Thank you so much. Senator Baldwin.
Senator Baldwin. Thank you, Madam Chair.
Last week, I had the privilege of joining some of my
colleagues on a visit to the National Institutes of Health.
While much of our discussion was centered on the response to
the COVID-19 pandemic, I was struck by the broad applications
of the innovation that we have seen during this time.
ADVANCES IN VACCINE AND THERAPEUTIC DELIVERY SYSTEMS
(RADX PROGRAM)
And, I have often spoken about the Wisconsin-based company,
FluGen, which is working to make vaccines that can be
administered as a nasal spray. I also believe that this type of
innovation is key in terms of how we think about our ability to
respond to future pandemics.
Dr. Tromberg, it was great to see you on that trip to NIH.
I wonder if you could describe how engineering advancements
have contributed to our response to COVID-19. And, how are you
thinking about the future of delivery and administration of
vaccines and therapeutics? And, how will these advancements
help us prepare for the future?
Dr. Tromberg. Thank you, Senator Baldwin, for the question,
and it was great to meet you last week, or I guess it was 2
weeks ago when you came to visit.
So, for COVID, we have supported a wide range of
technologic advances in medical imaging and artificial
intelligence, digital health platforms, PPE (Personal
Protective Equipment), ventilators, new therapeutic approaches.
Of course, the biggest probably and most impactful has been the
RADx testing program, which has delivered, as you have seen,
more than 300 million tests, including over-the-counter tests
with very advanced technologies from nanoscience.
In terms of vaccines, this is a very exciting area. Another
one that we have had in our portfolio, one of the strategies
that we have been supporting, are micro needle patches. So,
imagine a dime-sized micro needle patch that has got--the
needles are entirely soluble in water, and as soon as you put
them into your skin, they start to deliver the vaccine. After
the delivery, the needles are all gone, and you throw the patch
away. You get a new one in the mail. So, this has moved into
Phase 1 clinical trials. Efficacy has been shown.
I might, if you have a moment, toss it over to Dr. Fauci
because we have collaborated with his institute in the
development of these new delivery approaches and they may have
some other approaches, as well.
Senator Baldwin. Please. Dr. Fauci.
Dr. Fauci. Thank you, Bruce. Yes. We have an active
collaboration with Dr. Tromberg's Institute and we are looking
towards the future about how we can make it much easier to get
people vaccinated. This is of particular relevance right now
because, with COVID-19, even though we are doing really very
well with vaccination, we still have a group of individuals who
were really difficult to get to. And hopefully, when we have a
much easier way to administer the way Dr. Tromberg has
mentioned, that will make it easier for us.
Senator Baldwin. Excellent. In April, the University of
Wisconsin launched the Center for Health Disparities Research
Center, which has a leadership team comprised entirely of
women, will focus on how physical environment and social
conditions intersect to influence an individual's health.
Their first initiative, funded by the NIH, will use data
from 22 Alzheimer's disease research centers throughout the
U.S. to examine how social determinants of health throughout a
person's lifetime impact their brain health.
The pandemic has made it clear that we need to do more
research like this to better understand and respond to health
inequities, and I applaud the work of Dr. Amy Kind and the
University of Wisconsin. It is imperative that we maintain our
commitment to this into the future.
COVID-19 AND HEALTH DISPARITIES
So, Dr. Perez-Stable, how has the impact of the COVID-19
pandemic on communities of color informed how NIH thinks about
studying health disparities going forward? And what additional
investments are needed to fill these gaps?
Dr. Perez-Stable. Thank you, Senator Baldwin, for that
question. I think a year ago, when we understood the dimension
of the dramatic, disproportionate burden by race, ethnicity,
and socioeconomic status on the population, there was sort of
an aha moment for all of NIH to say, this problem has been with
us for a long time. We have made limited progress. It is time
we put our innovation, our efforts, to address this.
Out of this effort, we created the Community Engagement
Alliance Against COVID-19. Dr. Gibbons and I are co-chairing
that. Dr. Collins mentioned it in his opening statement. And I
think to heighten the importance of community engagement, so
talk to the people that are affected, bring them in as full
partners, identify the problems, and then mobilize all sectors
that we can mobilize. Not just the researchers and the
healthcare clinicians, but also the housing, transportation,
zoning, all the different sectors of society, to see how we can
begin to make a difference in this setting.
And I applaud the effort of Dr. Kind. She was a grantee of
ours, as well as others, and also applaud the effort of looking
at existing data with standardized measures to address problems
of this kind, like Alzheimer's disease.
Senator Baldwin. Yes. Thank you so much.
Madam Chair, I yield back.
Senator Murray. Thank you. Senator Rubio.
Senator Rubio. Thanks, all of you, for being here.
I think I will direct this to Dr. Fauci, but I welcome
everybody's answer. I just want to go through, so, what we do
know. We have heard a lot about what we do not know.
So, here are the things that we do know, okay?
ORIGIN OF COVID-19
So, SARS-1, we identified the host animal within 4 months.
MERS, I believe, we identified the host animal within 9
months.
It has now been 15 and a half, 16 months, we have still not
seen and China has not produced any evidence of the host animal
that transmitted COVID-19 to a human.
We know that China has a history of lab accidents. I think,
Dr. Fauci, you answered Senator Graham's question. I think he
phrased it as, has there ever been a pandemic that came out of
a laboratory, and the answer was no.
But, we know of outbreaks that came out of a laboratory. I
believe back in 2004, two researchers in Beijing were infected
doing research on SARS and it led to an outbreak. China has a
history of lab accidents.
This outbreak happened in a city that happened to be the
home, coincidentally, of a lab which we know is involved in
extensive research. And, what they do is they take this
naturally-occurring virus and they manipulate it and they
change it to make it infectious to humans. We know that they do
that there. They have published about it.
And, it also happened in a city in a lab where a Rutgers
biosecurity expert raised concerns about its safety, and our
diplomats in 2018 were cabling back to Washington expressing
concern about the safety.
So, I take all those facts together, right?
SARS, we knew the host in 4 months.
MERS we knew the host in 9.
We still do not know the host in--for COVID, even though--
and China is not being transparent about it even though they
have a vested interest in producing the host so they can put
all this down.
In a lab that we know is involved in changing viruses
synthetically so that they become infectious for humans.
In a lab that diplomats have told us is unsafe.
In a country that had history of lab leaks.
And, by the way, in a virus that we know can be
synthetically-created because the Swiss did it. The Swiss
created an exact replica of this virus in the lab for purposes
of answering it.
All of these facts were available to us last May, last
April. Why--I will start with Dr. Fauci. Why did you dismiss
the lab leak theory as credible?
Dr. Fauci. I have always said that the high likelihood is
that this is a natural occurrence. I did not dismiss anything.
I just said it is a high likelihood that this is a natural
occurrence from the environment of an animal reservoir that we
have not yet identified, and I still maintain that.
But, as I just mentioned in response to other questions,
that since you do not know 100 percent about that, because no
one knows, including me, 100 percent what the origin is, is the
reason why we are in favor of further investigation.
Senator Rubio. Well, given everything I have just cited--
and if anything I just cited is incorrect, I hope I will be
corrected. I am relying--obviously, not my field of study, so I
am relying on what other experts have published. What is the
basis for this high likely--what is the basis for the
conclusion that it is likelier to have been naturally occurring
than a lab accident?
I asked a specific question to the Director of National
Intelligence, and how I posed it is, is it not true that it is
the assessment that they are equally likely, based on our
information that we have.
So, as I outline all of these things here, is she wrong
when she answered me yes? And, based on everything I have just
cited, why the--what is it that we are basing the higher
likelihood of naturally occurring? Is it simply because that is
all we have ever seen in the past?
Dr. Fauci. Well, we have historical experience that
happened with SARS-CoV-1. It happened with MERS. It happened
with HIV. It happened with virtually all the influenza
pandemics. So, the historical basis for pandemics evolving
naturally from an animal reservoir is extremely strong, and it
is for that reason that we felt that something similar like
this has a much higher likelihood.
But, again, getting back to what I said--and let me repeat
so there is no lack of clarity in that. No one knows, not even
I, 100 percent at this point, which is the reason why we are in
favor of further investigation.
Senator Rubio. But, going back to precedent, precedents
require them to be similar. The difference between this one and
that one is--as I said, 4 months we knew the host for SARS, at
9 months we knew the host for MERS. China has all the incentive
in the world to produce this host and has not done so. And,
then, you add up all these other things, I mean, is it just a
coincidence it happened in the city that is doing this kind of
research, which, by the way, is controversial? I know you and
others have been supportive of it, but it is controversial. It
is not widely accepted as good.
My whole point is there are people out there who had
Facebook posts taken down. They are called kooks, conspiracy
theorists, for saying publically a year ago what we now say may
be possible. I think those people deserve an apology, at a
minimum.
Thank you.
Senator Murray. Thank you.
COVID-19 AND MIS-C
Dr. Bianchi, thank you. NICHD (National Institute of Child
Health and Human Development) is trying to develop ways to
identify children at high risk for multi-system inflammatory
syndrome in children. It is a rare and life-threatening after
effect of COVID-19. Now, while most children who become
infected, I know, have mild or no symptoms, some do go on to
develop this severe and sometimes fatal condition. I know your
research is still in the early stages, but could you describe
the NICHD's efforts to develop clinical, predictive models
using machine learning to identify children at risk and how
physicians are using this testing device and data?
Dr. Bianchi. Thank you very much for your question, Senator
Murray. As you know, there are almost four million children who
have been infected with SARS-CoV-2, but the key is to figure
out which is the one-in-a-thousand child who is going to get
very sick with this MIS-C, and that child could get critically
ill, although most do recover. So, as a parent, you would want
to know if my child tests positive, what is going to happen.
And, so, as part of the RADx RAD program--NIH is supporting
this. It is four different programs CARING for Children with
COVID, but the predictive one that is using artificial
intelligence and machine learning is called the PreVAIL Kids
Program. And what that is, is it is eight different programs
around the Country, with some international partners, that are
using existing cohorts, as well as prospectively enrolled
cohorts, to collect biospecimens and use artificial
intelligence in conjunction with the electronic health records.
The program started within the past few months, so we do
not have evidence yet. But, the enrollments are on target, and
we are expecting to enroll about 12,000--actually, we have
already enrolled about 12,000 children out of 16,000 that are
expected.
A.I. DETECTION OF CANCERS
Senator Murray. Okay. And Dr. Sharpless, artificial
intelligence has been shown to help improve the detection of
breast cancer in mammograms, and lung cancer in CT scans. And
suggesting that AI appears well suited for imaging, are you
looking at the potential for AI to help early detection of
other cancers?
Dr. Sharpless. Oh, yes. This is a very important topic. I
think artificial intelligence has really the ability to
transform cancer research and cancer clinical care in dramatic
ways.
We have a very lively set of collaborations going on with
the Department of Energy that has extensive expertise in this
topic. To use, you know, AI to try and identify drug targets
for medicinal chemistry, or to use AI to read 600,000 pathology
reports that we get for the SEER database every year, or to use
artificial intelligence for image analysis, both pathology
images and radiology images.
So, I think this is a tremendously exciting technology that
has real opportunities to advance cancer research and cancer
care in many important ways.
I think we were also worried about the ethical issues of
AI, and we want to make sure that we use practices that will
not reinforce biases that are latent in some of our data sets.
But, overall, I think the promise of AI is very exciting
for cancer research.
Senator Murray. Interesting. Okay.
CLIMATE CHANGE AND HEALTH
Dr. Gibbons, the request, budget request, includes $110
million to study the impact climate change is having on health.
Talk to us about what kind of serious effects have we been
seeing from climate change, and what kinds of research do you
expect NHLBI (National Heart, Lung, and Blood Institute) to
support with this kind of funding?
Dr. Gibbons. Yes. Thank you for that question. As we know,
climate change often involves these changes in our air, in our
air quality, particularly it is likely to promote more air
pollution. Certainly, the constituents on the West Coast are
familiar with the impact of wild fires on air quality.
And although air is all around us, air pollution tends to
concentrate and have its greatest impact on certain
communities, particularly communities in which those
neighborhoods are closer to sources of air pollution, and
therefore, the impact is also inequitable in terms of the
health consequences of air pollution, and that is falling on
the most vulnerable.
We know that it exacerbates certain chronic conditions,
certainly cardiopulmonary ones like chronic obstructive
pulmonary disease, asthma, heart failure. Heart attacks are
increased in the context of higher air pollution promoted by
climate change.
And, we anticipate that there will be a need to not only
mitigate the impact of climate change, but also to enhance
resilience to the effects of air pollution on health, and we
anticipate that that will involve enhancing healthy communities
that are disproportionately affected by the consequences of air
pollution derived from climate change. And our programs that
are community-engaged research with that health equity lens
should be promising in that regard.
Senator Murray. Okay. I think this is really important, and
I think we all should recognize that this is an area we need to
look at, so I appreciate your work on this and we will be
following it closely.
I will turn to----
SEXUAL HARASSMENT AT NIH
Okay. I have one additional question and that is for Dr.
Collins. In 2018, the National Academies, as you know, released
a report that found that nearly 60 percent of women in academia
have experienced--60 percent--have experienced sexual
harassment on the job and recommended that Federal research
agencies require institutions to notify them when individuals
on grants have violated harassment policies or put on
administrative leave due to harassment allegation. And other
science agencies, like National Science Foundation, have
implemented these changes.
Tell me, what is NIH doing to require its research
institutes to do the same?
Dr. Collins. Senator, I share the sense that this is an
extremely important issue. The National Academy report that you
mentioned I think really got everybody to recognize how
pervasive sexual harassment is and what a significant negative
it has been for far too long for women in our scientific
workforce.
We conducted our own working group in the Advisory
Committee to the Director that reported to me in December of
2019 and made a series of very significant recommendations
about how we might change our approach to this. We have been
working through those and have already implemented a
significant fraction of them. There are some that still require
some additional legal authority that is hard for us to be able
to do at the present time.
In terms of what you are particularly pointing to, we have
had now more than 300 allegations that have been brought to us
about sexual harassment in our grantee institutions; others
within our own intramural program. Of those 300, about 30
percent of them have turned out to be actually entirely
validated. That has resulted in a hundred different changes in
grants that--particularly, removal of principal investigators
and replacement of those with other individuals.
One hundred and twenty-five individuals have been taken out
of our pool of peer reviewers because of this kind of concern
about the bias that they bring to that experience.
And we have made it very clear to our institutions that we
expect them to report any circumstance----
Senator Murray. Well, expecting them does not require them
to.
Dr. Collins. And, Senator, you and I are in an interesting
discussion here that I agree--I wish we were able to simply say
require. At the present time, legally, we are told we do not
have that authority. We would have to go through a 2-year
rulemaking effort, or we would need statutory assistance.
Senator Murray. Well, okay. This is really important, and
whatever we need to do, I do not--you know, I know you have
worked on it, I know you have focused on it, but I know of
women who have left our scientific research institutes because
of this. We cannot afford to have that happen for a thousand
reasons. So, whatever it is we need to do here, we need to know
what it is so we can do it.
Dr. Collins. I am so with you. And if there is another
iteration we can take at this to try to figure out--I will say
that what we have said in terms of the expecting response from
our institutions has gotten their attention in a pretty
remarkable way. Even without requiring it, we are seeing
reporting coming through.
Senator Murray. Well, to every one of them that is
listening, I am not done with this.
Dr. Collins. Okay.
Senator Murray. Senator Blunt.
Senator Blunt. Thank you, Chair.
I have three or four questions. Let me eliminate a couple
of other topics by just making a couple of comments on some
things that have already been said, one, and one thing that has
not been, I do not believe, brought up today.
One is on the CTSA awards. None of the people talking to us
that are current recipients think that this simplifying the
process makes it more likely that they will get the research
bench-to-bedside result that they think you want and they think
is the key to this award.
And, you have heard a number of schools mentioned, and
University of Washington would be one of them that Senator
Murray would be very familiar with. Washington University in
St. Louis collaborates through this program with Saint Louis
University and the University of Missouri to get to more rural
hospital settings and do things. So, I suspect you have heard a
number of concerns about that today.
I have not heard brought up one of my concerns, which I am
just going to mention. I do not think you need to respond to
it. I do think that waiving the intellectual property rights on
COVID-19 vaccines is a problem. I think it is a problem because
I do not think it actually would increase the number of
vaccines, the capacity to produce a vaccine that has efficacy,
in the timeframe we need to make it. It probably is not
benefitted much by waiving the rights to the research. The WTO
(World Trade Organization) has to unanimously agree, which I do
not think they do. But, if they do, we give our research to
everybody.
And third, when this comes up again, companies would have
less willingness, I think, to step forward. At least one of the
companies, Dr. Collins that we dealt with in Warp Speed, there
was no agreement at all that if they were not successful--we
had a contract. We would buy 100,000 doses, but only if they
were FDA authorized. So, they were out there totally on their
own, as these companies you would expect to be.
I do not think this is likely to happen because of the WTO,
but I have some concerns that I suspect are shared by others at
NIH.
IMPACT OF COVID-19 PANDEMIC ON CHILDHOOD DEVELOPMENT
Dr. Bianchi, just the title--let's just take the title of
your Institute and look at COVID. What do you think the impact
on child health and development of COVID and the COVID
environment, the pandemic environment, the quarantine
environment, has been? And how are we going to be looking at
what the long-term ramifications of that might be and what
advice we may be able to give to schools and moms and dads and
behavioral health and other health providers as it relates to
child development impacted by this?
Dr. Bianchi. Thank you so much for that question, Senator
Blunt, because children, you know, have not--I think they are
so important in terms of our Nation's future, first of all.
But, the fact that children have been home from school has
affected the entire family, has affected the workforce, et
cetera.
But, because children in general have been asymptomatic or
mildly symptomatic, they have not gotten as much attention, and
yet being at home, being away from in-person schooling, I think
may have significant impact for years to come. And, for that
reason, we are trying to get the kids back to school as soon as
possible.
And as part of the RADx Underserved Population program, we
are also leading an initiative to really develop, evaluate, and
implement testing, along with mitigation, of, you know, hand
washing, social distancing, et cetera, to get evidence to
reassure people to get kids back to school. Two of the sites
are actually in Missouri, and one is in Washington State. There
is a program in Yakima, and there is a special program in
Missouri that is looking at how you deal with kids who have
intellectual disabilities and cannot mitigate in the same way.
So, to answer your question, I think there will be long-
term effects. I think the answer is to get kids back to school
safely, with evidence. And, this program is based on a funded
project that was very successful in North Carolina that showed
with all the mitigation, with the work with the superintendents
of schools, that the secondary infection rate in schools was
extremely low compared to the community.
Senator Blunt. Yes. I would think here that some of the
developmental issues, and they will be different with 4 and 5
year olds and kindergarten and first grade than they will
people in seventh grade, and those may be different than
people----
Dr. Bianchi. Absolutely.
Senator Blunt [continuing]. In the eleventh and twelfth
grade and how--you know, I think we are going to have to watch
this carefully and try to get data and then share that data.
FUTURE OF MRNA TECHNOLOGY
On vaccines--actually, on--maybe more on mRNA than
vaccines, what do we think the impact may be as it relates to
cancer, to HIV? We will start, Dr. Fauci, with you. Can we look
at the flu shot in a different way? And what do we think the
mRNA impact, now that we know this different use for it, may
have on other healthcare settings? And Dr. Sharpless, I am
going to come to you second on this.
Dr. Fauci. It is going to--I believe, and many of my
colleagues believe, that the mRNA technology, as it has been so
spectacularly successful with SARS-CoV-2 to develop a vaccine
against COVID-19, is already being pursued for other
infections, including HIV and including influenza. So, there
are a couple of things that are going on now. Even as we see
the successes with COVID-19 in using the mRNA technology for
the development, for example, of universal flu vaccines, as
well as now having HIV vaccine researchers now looking at the
possibility of an mRNA platform technology to use for HIV. So,
it is already happening.
Senator Blunt. Dr. Sharpless, on mRNA, I mean, we know the
impact in just the last half dozen years of immunotherapy on
cancer treatment. What about this mRNA intervention and how it
might impact the way we look at fighting cancer?
Dr. Sharpless. Yes, this is a very exciting topic. You
know, people interested in this space have been working on
this, you know, long before the pandemic. So, using mRNA for
cancer therapy has many potential applications because you can
really get the body to make a protein, and that protein could
have a desirable effect against cancer, for cancer therapy, in
a lot of ways.
The furthest advance, as you mentioned, is the use of mRNA
vaccines, you know, cancer vaccines. And clearly, they tend to
be highly personalized, the ideas that you can sequence
someone's own tumor and then make the vaccine to their very own
tumor in a way that will not cause them autoimmune side
effects, and this is an idea to augment other kinds of
autoimmune cancer--or anti-immune cancer therapies.
So, it is a very promising area. It is in clinical trials,
and we just need to see how this develops.
Senator Blunt. Thank you. My last question, Chair.
IMPACT OF COVID-19 PANDEMIC ON RESEARCH AND RESEARCHERS
Dr. Collins, in the pandemic, particularly with lab
closings, we obviously lost some time, and lost research that
is going to take a long time to recreate. Are the lab
reopenings happening in the way they need to? And, do you have
the flexibility to extend a grant to overcome the disruption?
And probably just not this disruption of the time closed, but
the research lost by closing, as well.
Dr. Collins. I am glad you are asking because this is yet
another of the terrible casualties of this terrible pandemic.
It has been very hard on researchers, especially those who need
a laboratory to do their work or who were running a clinical
trial that was very hard to enroll participants. And, yes, we
did have to have many of those folks staying away from the
workplace for their own safety.
They are coming back. Our own program at NIH, our
intramural program, now is up to about 50 percent occupancy,
but it is not anywhere near where it was pre-pandemic. We have
done everything we can with our flexibilities to try to make
sure, particularly, that trainees and early-stage investigators
do not get further injured by this by extending the periods of
their training; or by allowing grants if they are able to put
forward a special request to be extended for an extra year,
either without extra funds, or with, if the case is strong.
And yes, I also think we need to be cognizant of the way in
which this is affecting people in other ways. We have now come
up with a way to provide childcare support for our trainees who
otherwise have not had that, and that has been one additional
burden on their shoulders.
Our estimates are that it is about a $16 billion loss that
has occurred because of the way in which this has affected
research in our extramural institutions; that they are in a
tough place to try to make up. So, I appreciate your asking the
question.
We are going to have a really big challenge getting
ourselves back into the place that we were before this
happened.
Senator Blunt. Well, let us know what we need to be
thinking about as we think about the rest of this bill on that
topic. And thank you, Chair.
Senator Murray. Thank you very much. And I want to thank
all of our witnesses today for their really--for a really
productive hearing. I think we all learned a lot. So, thank you
very much.
ADDITIONAL COMMITTEE QUESTIONS
For any Senators who wish to ask additional questions,
questions for the record will be due one week after the
President's budget is delivered at 5 p.m. The hearing record
will also remain open until then for members who wish to submit
additional materials for the record.
[The following questions were not asked at the hearing, but
were submitted to the Department for response subsequent to the
hearing:]
Questions Submitted to Dr. Francis Collins
Questions Submitted by Senator Patty Murray
Question. The President's fiscal year 2022 skinny budget proposed a
major new biomedical research effort by establishing ARPA-H. While the
skinny budget was light on details regarding the structure of the
program, the Administration's statement indicated that the initial
focus of ARPA-H would be `on cancer and other diseases such as diabetes
and Alzheimer's.'
Assuming Congress and the Administration work together to establish
ARPA-H, how would you envision ARPA-H setting priorities for research
into additional diseases?
Answer. Over the long term, the proposed structure for the Advanced
Research Projects Agency for Health (ARPA-H) is intended to empower the
ARPA-H leadership and staff to set and execute on research priorities
for a variety of high-risk, high-reward, milestone-driven projects that
can lead to novel capabilities, platforms, and resources that are
applicable to a range of diseases.
For the initial direction, the Administration is working to set up
multiple pathways, both within the government and the broader
stakeholder community, for priority setting and for exploring new areas
ripe for research at ARPA-H. At the time of this hearing, the White
House Office of Science and Technology Policy (OSTP) and the National
Institutes of Health (NIH) are in the planning phases of convening
multiple listening sessions with key stakeholder groups including
patient organizations, industry, venture capitalists and
philanthropists, and others from the academic and research communities.
During these sessions, stakeholders will be asked to offer their
perspective on what they see as the greatest research challenges and
opportunities that could be addressed using the ARPA-H model. This
input will help refine the scope and provide a wealth of ideas for the
first ARPA-H director to consider as they develop the agency's vision.
In mid-July, the Administration established a Joint Fast Track
Action Committee (FTAC) to help steer the creation of ARPA-H and lay
the groundwork for strong interagency coordination. OSTP and NIH serve
as co-chairs of this committee that includes representatives from
Department of Agriculture, DARPA, Office of the Under Secretary of
Defense for Research & Engineering, ARPA-E, BARDA, CDC, CMS, FDA, VA,
EPA, NSF, and the Smithsonian Institution, among others.
Question. Some of the greatest advances in medical innovation in
the last decade have been brought on through genetic analyses and use
of sophisticated computer programs that can shorten the time taking
drug candidates through clinical studies. In fact, the development of
COVID-19 vaccines benefited from the use of 21st century technology
like cloud computing and AI to help stop the virus' spread and save
lives.
How will the President's budget build on the use of modern tools
like cloud computing, AI, and genetic analyses to further accelerate
the delivery of cures to patients?
Answer. Over the last decade, pharmacogenetics has advanced the
frontier of personalized medicine such that drug therapeutics are
developed based on the genetic aberrations of disease. This approach is
most notably applied for cancer treatments and also other diseases.
Cancers of various types are treated by first knowing the genetic
mutations and/or deletion of genes. Then drug candidates are screened
and developed by computer modeling of the target sites along with
potential drug candidates. Such modeling requires various large
datasets and analytics that, if stored in the cloud and interoperable,
can be mined to find the best drug candidates that bind to the target
sites for treatment. Storing large datasets in the cloud is only the
first requirement for cloud computing. Such computation requires new
tools, and support for tool development is essential to realize the
opportunities for cloud computing.
Artificial intelligence (AI) has advanced the pace of drug
discovery and development via predictive models of drug/target
interactions and also facilitates clinical trial design based on
algorithms for go/no go decisions during the trials.
The President's Budget Request supports the application of AI to
improve diagnostics for diseases as diverse as coronavirus disease 2019
(COVID-19) and cancer. In each case, information-rich data sources that
are stored, aggregated together, and analyzed in the cloud are used to
rapidly train and test these new capabilities. New programs like the
Artificial Intelligence/Machine Learning Consortium to Advance Health
Equity and Researcher Diversity program, or AIM-AHEAD, and Bridge2AI
will harness AI for health by generating AI-ready datasets and best
practices for machine learning. This will allow researchers to
accelerate data-driven discovery for grand challenges in biomedicine
using AI-based technologies. Additionally, NIH's partnership with cloud
services providers--Google, AWS and now Microsoft Azure--further
enhances researchers' abilities to leverage industry technologies and
utilize AI-ready data for drug discoveries and therapeutic treatments.
______
Questions Submitted by Senator Richard J. Durbin
Question. I have worked with the Subcommittee Chair and Ranking
Member for years on sustained, predictable increases to the NIH
budget--with the goal of providing at least 5 percent real growth year-
over-year. We have had success, leading to a 42 percent increase over
the past 6 years, along with supplemental funding in COVID-19 relief
packages. The President's fiscal year 22 budget calls for a 19 percent
increase to the NIH overall budget. The vast majority of that comes
from the proposed creation of a new advanced research effort, called
ARPA-H. When I toured the NIH campus recently with many members of this
Subcommittee, you discussed how innovative efforts during the
pandemic--such as with the RADx testing program or Warp Speed vaccine
development--align with the ARPA-H proposal, incorporation closer
partnerships with industry and coordination at different stages in the
research and development of promising breakthroughs. Your testimony
discusses application of this nimble ARPA-H proposal for cancer,
infectious diseases, and autoimmune diseases.
As we evaluate this proposal, what are the core aspects of this
ARPA-H policy that you want us to keep in mind?
Answer. We envision that the Advanced Research Projects Agency for
Health (ARPA-H) will be able to tackle large-scale challenges using a
proven high-risk, high-reward approach that embraces nimbleness and
flexibility with the broader goal of delivering rapid breakthroughs
that serve all patients. Being successful in this endeavor requires
close communication and collaboration across government and with key
stakeholders in the external biomedical community. This could include
undertaking projects with Federal agencies, private companies,
independent research institutes, medical centers, as well as academic
institutions--all collaborating to advance innovative health research.
NIH deployed similar approaches in response to the COVID-19 pandemic
(Accelerating COVID-19 Therapeutic Interventions and Vaccines, or ACTIV
and Rapid Acceleration of Diagnostics, or RADx)--which yielded life-
saving results for Americans, and also served as a learning opportunity
to appreciate further the value of employing a DARPA--like model to
support research. With Congressional support, we believe we can
leverage these models in other areas of health research to drive
transformative change and impact.
Question. We have spoken in the past about two seemingly divergent
issues. On one hand, we talk about the need to invest in medical
research to find breakthroughs and cures for patients, so we rightfully
appropriate billions into NIH-funded research--sign me up for that. But
then these drugs come to market--the vast majority of them benefitting
from NIH research (e.g. a study finding that all 210 drugs approved by
FDA between 2010 and 2016 benefitted from NIH-funded research in some
form)--and too many of them with exorbitant price tags. Recent studies
show that high costs contribute to poor medication adherence, including
with one-quarter of cancer patients choosing not to fill a prescription
due to cost. I know Dr. Sharpless has talked about the ``financial
toxicity'' for cancer patients. Americans pay the highest prices for
medications in the world, with a recent GAO report finding that the
U.S. pays two- to four-times more for certain medications than other
developed countries. It is counterintuitive and an outrage that
taxpayers fund cutting-edge research, which leads to drugs, that we
often cannot afford once they hit the market. I understand NIH does not
set drug prices and does not want to limit the handoff or development
of its research to stakeholders that commercialize the discoveries. But
the current system does not maximize the benefits for patients.
Given the role of NIH research in contributing to FDA-approved
medications, many of which come with extremely high price tags, what
specific steps can NIH take to ensure that patients are able to afford
the incredible discoveries made at NIH?
The NIH has received several petitions to exercise march-in rights
(35 U.S.C. Sec. 203), but has never done so.
--Under what circumstances would NIH consider doing so?
--Under that statutory authority, how does NIH define and evaluate
the term ``practical application'' for the purposes of how a
contractor or assignee makes a subject invention funded by NIH
available to the public on reasonable terms?
--What are the factors used in such definition and evaluation?
--Can you provide an example of the analysis undertaken in evaluation
of a previously filed march-in-petition?
Answer. The National Institutes of Health (NIH) shares your concern
about the high price of drugs and the impact on public health. The
article you reference shows that all of the 210 drugs approved by U.S.
Food and Drug Administration from 2010 to 2016 were based on at least
one scientific publication reporting on research funded by the NIH.\1\
The researchers reported that 96 percent of the NIH funded projects
were identified based on a search for the ``target'' rather than the
drug itself. Identifying a drug target, meaning a protein in a cell
that has a function in a disease process, opens the door for any
researcher in industry or academia to screen for drugs that bind to the
target to slow or arrest disease processes. This research is key to a
vibrant drug discovery process in the United States and does not limit
discovery to one drug for each target. The development of multiple
drugs for a particular disease allows the patient and physician to
choose the best one for them and can lead to price competition in the
market. Drug pricing is a complex problem that involves various
segments of the market, much of which NIH has no control over. A
smaller number of important drugs utilize patented inventions funded by
the NIH. When NIH has been asked to consider march-in under the Bayh-
Dole Act based on the price of such drugs, NIH has stated that the
issue of drug pricing is one that should be address by Congress, as it
considers these matters in a larger context.\2\
---------------------------------------------------------------------------
\1\ Cleary et al., 2018, www.ncbi.nlm.nih.gov/pmc/articles/
PMC5878010/.
\2\ NIH march-in responses from 1997-2013 at ott.nih.gov/policy/
policies-reports under ``NIH March-In Response''.
---------------------------------------------------------------------------
The Bayh-Dole march-in provision (See 35 U.S.C. 203) allows a
government funding agency to require a grantee to grant a license to a
patent of an invention made under that agency's awarded grants or
contracts and allows other ``responsible applicants'' to obtain the
license if one of four circumstances are met:
1. the contractor or assignee has not taken, or is not expected to
take within a reasonable time, effective steps to achieve practical
application of the subject invention in such field of use
2. to alleviate health or safety needs which are not reasonably
satisfied by the contractor, assignee, or their licensees
3. to meet requirements for public use specified by Federal
regulations and such requirements are not reasonably satisfied by the
contractor, assignee, or licensees
4. the agreement required by section 204 [a requirement that
patented products be manufactured substantially in the United States
unless a waiver is granted]
The first two criteria are typically cited in petitions to consider
a march-in by the National Institutes of Health (NIH). For example, if
a company has rights to a government funded patent for a drug candidate
but is not making reasonable efforts to bring it to market, the company
may be failing to meet the requirements to achieve practical
application of the invention. These criteria are considered on a case-
by-case basis by the agency in view of the facts presented in each
case.
If NIH were to march-in, the grantee could appeal that decision
through the Federal courts. Only after the company had lost all legal
appeals could NIH grant a license to a second company, should there be
one interested in developing a new version. Additionally, the drug
could be covered by other patents that cover certain aspects of the
drug, such as methods of making and administering it. In such
instances, the march-in could be ineffective, because the original
company could stop a new company from making the generic until the
other patents expire.
After the court appeals and expiration of any other patents, a
company would typically have to conduct clinical trials or otherwise
establish equivalency with the brand drug to obtain U.S. Food and Drug
Administration approval. The entire process, including administrative
hearings, court appeals and new clinical trials, could take years
before the new product reached the market. In the meantime, alternative
therapies may have become available or the patent subject to march-in
may have expired.
NIH has considered march-in on several occasions and was either
able to work with parties to reach an agreement to address the issues
raised, such as the case with CellPro and Fabrazyme, or decided that
the march-in legal requirements were not met to march-in to address the
public health and safety issues raised, such as was the case with
Norvir.\3\
---------------------------------------------------------------------------
\3\ See ott.nih.gov/policy/policies-reports under NIH March-In
Response.
---------------------------------------------------------------------------
Question. The COVID-19 pandemic has impacted every major sector of
the economy of the United States, including our nation's biomedical
research. I have heard from countless universities across the state of
Illinois about the impact that this pandemic has had on the medical
research pipeline. From shuttered labs, to interrupted or delayed
clinical trials, to unforeseen pandemic-related costs, they have
estimated that this pandemic has caused over $10 billion in lost
research. Last year, Senator Moran and I sent a bipartisan letter to
Senate leadership, requesting at least $10 billion in additional
funding to help make-up for the unforeseen disruptions and costs to
medical research nationwide.
Dr. Collins, I am wondering if you can speak to the toll that the
pandemic has taken on medical research nationwide and what Congress
might be able to do to help.
Answer. The National Institutes of Health (NIH) remains deeply
concerned and mindful about how the spread of coronavirus disease 2019
(COVID-19) has negatively affected the biomedical research
enterprise.\4\ Last summer, the NIH estimated it would cost at least
$10 billion to restart labs which were forced to rapidly close. That
original estimate proved overly optimistic as the pandemic subsequently
continued, and as such, the NIH now estimates the financial impacts to
be approximately $16 billion on the biomedical and behavioral research
enterprise.
---------------------------------------------------------------------------
\4\ https://nexus.od.nih.gov/all/2020/11/04/continued-impact-of-
covid-19-on-biomedical-research/.
---------------------------------------------------------------------------
The estimates considered many factors:
--Key resources, such as animal colonies, cell lines and expired
reagents that need to be re-established.
--Access to core facilities that was limited due to a backlog of
requests.
--Delicate and complicated equipment that required recalibration and
quality control testing prior to returning to routine use.
--Requirements for social distancing to protect staff and clinical
trial participants coupled with anticipated reluctance by
participants to travel, which slowed the rate of clinical trial
accrual and progress and increased the cost of conducting
trials.
In addition to the financial estimates, the NIH fielded two online
surveys to objectively document COVID-19's impact on the extramural
research workforce.\5\ The main finding from the surveys was that the
majority of respondents noted concerns about research functions,
research productivity, and financial status.\6\ Well into the pandemic,
many NIH-supported research labs enforced social distancing, inherently
restricting access and severely limiting the ability to generate
research results and preliminary data at a crucial time in career
development of early stage investigators and trainees. Junior faculty,
often with only a single NIH award and unable to access their labs to
generate additional data, are at risk of losing all funding and may
have insufficient data to write papers while working from home. Some
investigators, especially women with dependent care responsibilities,
are more negatively affected. Investigators supported by training or
career development awards are experiencing hiring freezes and job
revocations, jeopardizing the ability of early-stage career
investigators to transition to independence, particularly as they come
to the end of their current funding. Clinical investigators have been
diverted from their research labs to meet the clinical demands of
COVID-19 patient care.
---------------------------------------------------------------------------
\5\ https://nexus.od.nih.gov/all/2020/10/05/encouraging-
participation-in-upcoming-nih-surveys-to-identify-impacts- of-covid-19-
on-extramural-research/.
\6\ https://nexus.od.nih.gov/all/2021/03/25/the-impact-of-the-
covid-19-pandemic-on-the-extramural-scientific-workforce-outcomes-from-
an-nih-led-survey/.
---------------------------------------------------------------------------
Considering these effects, the NIH is concerned about potential
pandemic-related losses of scientists exiting the biomedical research
workforce and abandoning scientific careers to seek alternative
employment. In an effort to address the unanticipated impacts of the
pandemic on the career trajectories of early career scientists, the NIH
has provided several policy flexibilities, including grant award
extensions (both funded and un-funded), opportunities for investigators
to extend the timeline for early career status, provided administrative
supplements, and more.
______
Questions Submitted by Senator Brian Schatz
Question. At the hearing, we discussed psychedelic drug research
and the potential of these drugs to treat mental health illness. You
stated that the NIH would consider having a workshop on this subject.
What is the current status of NIH-funded clinical trials involving
human subjects on the potential benefits of psychedelics combined with
psychotherapy?
Are there statutory or regulatory barriers to NIH pursuing or
funding human subject research on psychedelic drugs?
When does NIH plan to convene a workshop on psychedelic drug
research?
Answer. The National Institutes of Health (NIH) supports research
on the development and testing of pharmacological interventions--
including the use of hallucinogens such as ketamine, and psychedelic
drugs such as psilocybin--for the treatment of illnesses. In
particular, the National Institute of Mental Health (NIMH) requires an
experimental therapeutic approach for the development and testing of
therapeutic interventions for mental illnesses, in which the studies
not only evaluate the clinical effect of an intervention, but also
generate information about the mechanisms underlying a disorder or an
intervention response. Research on psychedelic drugs holds promise for
uncovering mechanisms of mental illnesses and possible interventions,
ultimately leading to novel treatments with fewer side effects and
lower abuse potential. Further research is needed to examine the
efficacy and long-term safety of psychedelic drugs, including with
repeated exposure and potential interactions with existing treatments.
The dissociative anesthetic ketamine has recently emerged as an
effective fast-acting antidepressant.\7\ The NIMH Director's Message,
``New Hope for Treatment-Resistant Depression: Guessing Right on
Ketamine,'' describes the role of NIMH and other researchers in the
development of esketamine, a U.S. Food and Drug Administration-
approved, rapid-acting medication that targets treatment-resistant
depression.\8\ Within the NIMH Intramural Research Program, Dr. Carlos
Zarate is now conducting clinical trials to better understand how
ketamine rapidly reduces depressive symptoms in people with treatment-
resistant depression or bipolar depression.\9,10\
---------------------------------------------------------------------------
\7\ pubmed.ncbi.nlm.nih.gov/27839782/.
\8\ www.nimh.nih.gov/about/director/messages/2019/new-hope-for-
treatment-resistant-depression-guessing-right-on- ketamine.
\9\ clinicaltrials.gov/ct2/show/NCT03065335.
\10\ clinicaltrials.gov/ct2/show/NCT03973268.
---------------------------------------------------------------------------
The National Institute on Drug Abuse (NIDA) currently supports a
clinical trial which aims to assess the efficacy of ketamine, in
combination with behavioral therapy, in the treatment of cocaine use
disorders.\11\
---------------------------------------------------------------------------
\11\ clinicaltrials.gov/ct2/show/NCT03344419.
---------------------------------------------------------------------------
Additionally, a privately funded clinical trial is assessing the
potential efficacy of the psychedelic drug psilocybin for the treatment
of obsessive-compulsive disorder.\12\ While the NIH is not directly
funding this trial, NIMH does support the trial's principal
investigator through a Mentored Patient-Oriented Career Development
Award.\13\
---------------------------------------------------------------------------
\12\ clinicaltrials.gov/ct2/show/NCT03356483.
\13\ reporter.nih.gov/project-details/10127338.
---------------------------------------------------------------------------
Further, a number of NIH-funded researchers are conducting basic
and preclinical research to investigate the use of psychedelic drugs as
potential therapeutic interventions for mental illnesses. For example,
NIMH-funded researchers are examining the mechanisms underlying the
antidepressant effects of psychedelic drugs in an effort to develop
novel, non-hallucinogenic treatment strategies that are both safer and
more effective than existing treatment options.\14\
---------------------------------------------------------------------------
\14\ reporter.nih.gov/project-details/10003396.
---------------------------------------------------------------------------
As with all human subjects research, clinical research on
psychedelic drugs is governed by several statutes, regulations, and
policies intended to protect the rights and welfare of research
participants. For example, NIH has specific requirements for research
staff and policies regarding research conduct, safety monitoring, and
reporting of information about research progress.\15\ In accepting an
award that supports human subjects research, the recipient institution
assumes responsibility for all research conducted under the award,
including protection of human subjects at all participating and
consortium sites.\16\ All human subjects research must also be
reviewed, approved, and monitored by an Institutional Review Board.\17\
---------------------------------------------------------------------------
\15\ grants.nih.gov/policy/humansubjects/policies-and-
regulations.htm.
\16\ grants.nih.gov/grants/policy/nihgps/html5/section_4/
4.1.15_human_subjects_
protections.htm.
\17\ www.fda.gov/regulatory-information/search-fda-guidance-
documents/institutional-review-boards-frequently-asked-questions.
---------------------------------------------------------------------------
Because psychedelic drugs are controlled substances, clinical
research using psychedelic drugs must also follow Drug Enforcement
Administration requirements, including registration, inspection, and
certification of the drugs.\18\
---------------------------------------------------------------------------
\18\ grants.nih.gov/grants/policy/nihgps/html5/section_4/
4.1.5_controlled_substances.htm.
---------------------------------------------------------------------------
From April through June 2021, the Trans-NIH Integrative Medicine
Course Organizing Committee hosted a series of research talks on
psychedelic drugs.\19\ Building on these research talks, NIMH and NIDA
are now working together to convene a scientific workshop in winter
2021. This workshop will bring together leading researchers to examine
the state of the evidence for the use of psychedelics in the treatment
of mental illnesses.
---------------------------------------------------------------------------
\19\ events.cancer.gov/nci/psilocybinresearch/agenda.
---------------------------------------------------------------------------
Question. The United States shares a unique political relationship
with the Native Hawaiian community. Different Federal agencies within
HHS are responsible for the administration of Native healthcare
programs, but the same Federal trust responsibility requires the
provision of comprehensive, quality healthcare to Native Hawaiians,
Alaska Natives and American Indians. In 2015, NIH established the
Tribal Health Research Office within the Office of the Director to
coordinate tribal health research activities across NIH. However, no
such research office exists for Native Hawaiians.
Would you consider expanding the scope of the Tribal Health
Research Office to include Native Hawaiians? Would this help to
increase the number of Native Hawaiian researchers and the amount of
Native Hawaiian research being conducted across the country?
Has NIH set any goals for the Tribal Health Research Office, and
how will you measure its success and impact across NIH's Institutes and
Centers?
Some funding opportunities at NIH, such as the Native American
Research Centers for Health program, do not permit entities serving
Native Hawaiian communities to apply. Why are these entities excluded,
and would NIH consider including these entities in the eligibility for
these grant opportunities?
Answer. The National Institutes of Health (NIH) Tribal Health
Research Office (THRO) does not conduct disparity research on Native
American populations. THRO ensures that the NIH fulfills its
obligations to Indian Tribes as federally recognized sovereign nations,
conducts government to government interactions appropriately, and holds
formal Consultations with Tribal governments on policy, regulatory, and
legislative issues that have a significant direct impact on Indian
Tribes.
The National Institutes of Health (NIH) published the NIH Strategic
Plan for Tribal Health Research with input from American Indian/Alaska
Native (AI/AN) Communities and the NIH Tribal Advisory Committee (TAC).
The plan includes four agency-wide strategic goals: enhancing
communication and collaboration; building research capacity for AI/AN
communities; expanding research; and enhancing cultural competency and
community engagement. The Tribal Health Research Office (THRO), along
with the NIH Institutes and Centers (ICs), developed processes and
metrics for evaluating progress on the strategic objectives and their
supporting action items to achieve these goals. THRO regularly collects
data on AI/AN health research activities from all ICs through an
automated process to analyze the NIH research portfolio, assess
progress towards the strategic goals, and measure impact across NIH.
The National Institute of General Medical Sciences in conjunction
with multiple NIH Institutes, Centers, and Offices (ICOs) partner with
Indian Health Service (IHS) to support the Native American Research
Centers for Health (NARCH). NARCH grant applications are submitted by
and awarded to a tribe or tribal organization, who are sovereign
nations with distinct governing bodies. Awarding the grant directly to
the tribe or tribal organization allows for the community to dictate
and oversee research priorities, while drawing upon necessary expertise
from the research community to accomplish its scientific goals.
______
Questions Submitted by Senator Joe Manchin, III
Question. West Virginia is consistently ranked last in the nation
for health outcomes. In 2020, the America's Health Rankings Report
ranked West Virginia 50th for premature deaths, frequent mental
distress, and multiple chronic conditions. We also rank last in life
expectancy. West Virginia has, in many ways, been left behind as
medical advances have saved lives in other places.
What is NIH doing to bridge this gap in health outcomes?
How do you ensure that the medical research that you do benefits
people in poor, rural communities?
How can we better expand the access rural Americans have to
successful medical treatments, particularly in states like mine where
the disease burden is so high?
Answer. The National Institutes of Health (NIH) recognizes the
unique health disparities that rural communities face, and as such,
rural health is an important area of research for the agency.
Through diverse collaborations and partnerships with communities,
academic institutions, and state agencies, NIH supports and conducts
rural health research to improve health outcomes and reduce rural
health disparities with a special emphasis on the poor in rural
communities. In fiscal year 2020, NIH supported more than 1,000 rural
health-related grants for approximately $728 million. In 2020, West
Virginia received approximately $45.7 million in funding from NIH, of
which about $6.4 million supported research and research capacity-
building activities related to rural health.
In 2019, NIH held the Inaugural NIH Rural Health Seminar, a
collaboration of several NIH Institutes and Centers to explore topics
in rural health and opportunities for research collaborations to
improve rural health outcomes. In 2020, NIH hosted a virtual rural
health conference entitled, NIH Rural Health Seminar: Challenges in the
Era of COVID-19. In October 2021, NIH will host the Pathways to
Prevention Workshop: Improving Rural Health Through Telehealth-Guided
Provider-to-Provider Communication, a virtual event to identify
research gaps, explore barriers, and facilitate successful, sustainable
implementation of provider-to-provider telehealth in rural settings.
NIH's rural health research focuses on key areas aimed at
addressing health disparities that rural populations in West Virginia
and around the United States experience. In fiscal year 2020, in
response to the disproportionate impact of coronavirus disease 2019
(COVID-19) on racial and ethnic minority, and other vulnerable
communities including rural populations, NIH established the Rapid
Acceleration of Diagnostics for Underserved Populations (RADx-UP)
initiative. The overreaching goal of the RADx-UP initiative is to
understand the factors associated with disparities in COVID-19
morbidity and mortality and to lay the foundation to reduce disparities
for those underserved and vulnerable populations more impacted by
COVID-19. One example of a RADx-UP project in your state, is the
Developing Novel Strategies to Increase COVID-19 Testing among
Underserved and Vulnerable Populations in West Virginia through
Community and State Partnerships. This project will implement
collaborative strategies to increase availability and uptake of severe
acute respiratory syndrome coronavirus 2 (SARS CoV-2) testing among the
medically underserved, rural West Virginia population that includes
multiple vulnerable groups at risk for severe COVID-19 and death. This
initiative will test whether those implemented strategies, including
home test kit and mobile unit mechanisms, successfully increase
testing, and if not, determine why the interventions did not work to
inform future sustainable testing policy.
In addition, NIH supports the West Virginia University Health
Sciences TME CoBRE project, which focuses on the microenvironment of
different tumor types, including cancers initiating in the bone marrow,
head and neck, breast, and brain. This project will increase
understanding of the constant interaction between the tumor and its
environment, provide diverse training opportunities and mentoring
strategies for junior faculty, and develop critical infrastructure and
recruit additional tumor microenvironment focused scientists to West
Virginia. Another project, the West Virginia Clinical and Translational
Science Institute: Improving Health through Partnerships and
Transformative Research (WVCTSI), leads statewide collaborations and
innovation in clinical and translational research. This project will
build sustainable research infrastructure, recruit clinician scientists
and translational researchers that excel in team science, and actively
engage with multiple stakeholders that include communities, medical
providers, and policy makers to improve the health of West Virginians.
NIH is committed to ensuring that there are opportunities for poor
rural Americans to access the benefits of research and that research
addresses the unique strengths and challenges of rural communities by
supporting several initiatives focused on human immunodeficiency virus
(HIV), cardiovascular disease, cancer, drug addiction, and other
chronic diseases disproportionately affecting rural communities. First
announced in April 2018, the NIH Helping to End Addiction Longterm\SM\
Initiative, or NIH HEAL\SM\ Initiative, is an expansive agency-wide
effort. It spans basic, translational, clinical, and implementation
science and promotes collaborations of all types of research to address
the crises of opioid misuse, addiction, and overdose in the United
States. Launched in fiscal year 2020, Strategies to Improve Health
Outcomes and Reduce Disparities in Rural Populations supports research
to promote a greater understanding of the challenges faced by rural
populations in developing or adapting evidence-based interventions that
can reduce health risks faced by rural Americans. A total of eight
awards were funded including: Harnessing the Power of Peer Navigation
and mHealth to Reduce Health Disparities in Appalachia which is using a
community-based approach to integrate peer navigation and mobile health
strategies to develop a culturally congruent, bilingual intervention to
increase the use of HIV, sexually transmitted infection, and Hepatitis
C prevention and care services among individuals with health
disparities living in rural Appalachia. Another study, Heart of the
Family: A Cardiovascular Disease and Type 2 Diabetes Risk Reduction
Intervention in High-Risk Rural Families is examining the effects of a
family focused, lifestyle intervention that is culturally tailored for
use with rural Hispanic or Latino and non-Hispanic or Latino adults. In
2020, the National Institute on Minority Health and Health Disparities
(NIMHD) funded four rural Resource Hubs to focus on rural health
research. These hubs will involve coalitions of researchers and
community partners to build research capacity in an identified rural
catchment area and offer opportunities to share resources and data
across collaborators.
NIH continues to support the Accelerating Colorectal Cancer
Screening and Follow-Up Through Implementation Science (ACCSIS)
Program, a Cancer Moonshot? Initiative, designed to reduce cancer
screening disparities. The aim is to identify evidence-based
interventions and identify promising approaches for bringing these
interventions to unscreened populations. Researchers test interventions
such as mailing programs for home testing, provider education, and
clinic-based patient navigation among Medicaid, rural, and racial and
ethnic minority groups. In fiscal year 2020, NIH reissued and released
the Pragmatic Research in Healthcare Settings to Improve Diabetes and
Obesity Prevention and Care funding opportunity announcement. This
initiative aims to improve diabetes and obesity prevention and/or
treatment that are adapted for implementation in healthcare settings
where individuals receive routine medical care. One of the funded
grants, Telemedicine for Reach, Education, Access, Treatment and
Ongoing Support (TREAT-ON), is a diabetes educator-driven, primary
care-based telemedicine model that redesigns primary care practice to
provide access to real-time ongoing support and help high risk
participants in an underserved rural community to achieve and sustain
improvements in clinical, psychosocial and behavioral outcomes. The NIH
Minority Health and Health Disparities Strategic Plan 2021-2025 aims to
test best practices for dissemination and implementation of minority
health and health disparities research in diverse diseases and
conditions into rural communities.
Continued collaborations and partnerships with scientists and
organizations from rural communities, such as West Virginia, will
contribute to NIH's reach in rural communities and support our work to
combat rural health disparities.
Question. The NIH funds the WV Clinical and Translational Science
Institute at West Virginia University through a 5-year $20 million
grant. The Institute provides critical health research across West
Virginia and has successfully mentored early career investigators,
established pilot project funding, and created a research network
across 27 primary care sites. Their research has focused on important
health issues in my state including lung disease in coal miners, opioid
addiction, and the hepatitis C epidemic, as well as cancer, heart
disease, and stroke. Most recently, the Institute has been on the front
line of COVID-19 research, having received a $1.5 million NIH Grant to
lead an 8-state effort so that data from COVID-19 patients could be
analyzed to develop the most impactful COVID-19 research. They're also
responsible for utilizing the NIH RADx grant to scale up COVID-19
testing in WV Communities.
Can you comment on the importance of continued collaboration
between the NIH and research institutions like the WV Clinical and
Translational Science Institute at West Virginia University?
What more can we be doing to support young researchers, such as
those mentored through this Institute?
Answer. One of the core programs supported by the National
Institute of General Medical Sciences (NIGMS) Institutional Development
Award (IDeA) is the IDeA Networks for Clinical and Translational
Research (IDeA-CTRs), which includes the West Virginia Clinical and
Translational Science Institute (WV CTSI). The IDeA-CTR network aims
to:
--Support the development and/or enhancement of infrastructure and
human resources required to address clinical and translational
research needs in IDeA-eligible states and jurisdictions;
--Strengthen clinical and translational research that addresses the
broad spectrum of health challenges faced by populations in
IDeA-eligible regions; and
--Foster and coordinate collaboration in clinical and translational
research within an IDeA-CTR network and with other
institutions.
Strengthening and expanding the capacity for clinical and
translational research in IDeA-eligible states is a pressing need,
since health conditions such as obesity, diabetes, cardiovascular
diseases, cancer, infectious diseases, chronic obstructive pulmonary
disease, maternal health issues, and substance use disorders are
disproportionally present in and borne by communities in these states.
The IDeA-CTR networks support health research professionals who have
first-hand knowledge of these challenges in order to understand and
improve the health outcomes of residents in affected jurisdictions.
Having the WV CTSI in place during the coronavirus disease 2019 (COVID-
19) pandemic, for instance, has allowed it to act as a springboard for
West-Virginia-based research aimed at studying and addressing the
virus. The $1.5 million supplemental award referenced in this question
facilitated the development of an eight-state consortium that created
an IDeA State COVID-19 Patient Registry. Through the collaboration
between the NIH and WVU, the Registry has become a key component of the
National COVID Cohort Collaborative, making important contributions in
addressing the unique challenges brought by COVID-19 to traditionally
underserved groups such as rural populations. Another supplement to the
WV CTSI supports a network for conducting COVID-19 testing in West
Virginia that includes the state health department, the national guard,
and rural clinics. This collaborative effort is playing a major role in
facilitating the state's testing efforts. Finally, the WV CTSI is also
a key participant of an NIH-sponsored multi-site Post-Acute Sequelae of
SARS-CoV-2 (PASC) study of ``Long COVID'' patients who continue to
experience symptoms long after initial infection.
Both NIGMS and NIH remain committed to supporting IDeA-CTR networks
like the WV CTSI, given the very important role that such networks play
in developing research infrastructure and improving health outcomes
within IDeA states.
The National Institutes of Health (NIH) believes that supporting
early career researchers is crucial to maintaining a productive,
innovative, and diverse biomedical research workforce that can continue
to advance the vitality of the scientific research enterprise. NIH's
Next Generation Researchers Initiative (NGRI) is developing and
implementing strategies to identify, support and retain investigators
across early career stages.
As part of the NGRI, NIGMS has prioritized and included several
strategies for supporting trainees and early-stage investigators (ESIs)
within its 2021-2025 Strategic Plan, along with targets for
implementing those strategies that provide accountability and the
ability to measure progress. Career development initiatives such as the
recently launched Maximizing Opportunities for Scientific and Academic
Independent Careers (MOSAIC) program focus on retaining and supporting
postdoctoral scholars from diverse backgrounds through the critical
point of transitioning them into independent faculty careers.
Cooperative agreements with professional organizations support
educational activities that equip MOSAIC scholars with professional
skills, mentoring, and career networks. At the individual level, grants
such as NIGMS' Maximizing Investigators' Research Award (MIRA) offer
support to early-stage investigators (ESIs) by providing them both the
opportunity to perform creative and ambitious research as well as the
flexibility to follow important new research directions and scientific
insights. Since launching this award mechanism in 2015, MIRA has
supported 628 early-stage investigators (ESIs), at least two of whom
were in West Virginia. In fiscal year 2020 alone, NIGMS funded 200 ESIs
through MIRA. As these examples illustrate, both the NIGMS and NIH
remain committed to supporting promising early career investigators in
every state in the nation.
______
Questions Submitted by Senator Roy Blunt
Question. Dr. Collins, I am a big supporter of the Clinical and
Translational Science Award (CTSA) program. I believe we should look
for ways to strengthen the CTSA program and reinforce the hubs around
the country. That is why I am troubled to hear about a possible CTSA
reorganization that will be announced in June. This reorganization
comes with limited discussion and consultation with the CTSA directors.
I am concerned, specifically, with the proposal to break up hub awards
into smaller pieces, requiring CTSAs to write several grant
applications instead of just one. Dr. Collins, I have two questions.
First, as you know, this Committee pays a lot of attention to CTSAs and
has been concerned in the past about communication between NCATS and
the CTSA community. For example, NCATS emailed relevant stakeholders to
combat the rumors about changes to the CTSAs, but did not provide any
relevant data to explain what they want to do and why they want to do
it. That did nothing but add to the concerns and speculation in the
community.
Why haven't these specific changes been discussed broadly within
the CTSA community? I believe if there was open dialogue and a stronger
partnership between NCATS and CTSAs, there would likely be more buy-in
from the community.
Two, how does cutting the hub award and requiring CTSAs to compete
for multiple awards strengthen the program? It appears to me that this
change would bring uncertainty to the program and jeopardize the
stability of the hubs.
Answer. The Clinical and Translational Sciences Award (CTSA)
program is indeed a very valuable and important program for the
National Center for Advancing Translational Sciences (NCATS), NIH, and
the nation. NCATS understands that there are often concerns when there
are planned updates to a program, particularly one as large and
impactful as the CTSA Program. The planned updates are part of the
regular NIH business process for reissuing Funding Opportunity
Announcements (FOAs), which is required because FOAs expire after 3-4
years. The planned updates will maintain the structure of the program
and reflect the public input received--much of which was provided by
the CTSA hub institutions and investigators. The planned updates are
designed to strengthen the program, by prioritizing hub strengths,
streamlining the overall application process, emphasizing clinical
partnerships which are critical to achieving the objectives of this
national program, and stabilizing the funding provided to the hub
institutions by allowing up to 7 years of funding (rather than the
typical five-year award period for NIH awards).
How NCATS Engages with the CTSA Community: NCATS agrees that a
strong partnership is extremely important and works closely with the
CTSA community on a regular basis.
--Regular Meetings: A CTSA Steering Committee \20\ including
leadership from NCATS and the CTSA Principal Investigator
community, meets monthly. A monthly webinar for all CTSA
Program investigators also shares information about the
program. NCATS CTSA leadership and program officers also
routinely engage with investigators and institutional
leadership across the CTSA Program as part of their regular
duties for implementing a program of this size and complexity.
In addition, there are yearly multi-day conferences where the
CTSA investigators and NCATS staff engage deeply on important
issues related to the CTSA program.
---------------------------------------------------------------------------
\20\ clic-ctsa.org/groups/steering-committee.
---------------------------------------------------------------------------
--Engaging the Community on Updates to the Planned FOA: To maintain
fair and open competition for funding opportunities, NCATS
cannot discuss specific details about a draft FOA with select
groups of the public, particularly those who already have
funding and would be re-competing for the funds. The level of
engagement must be framed to ensure that all investigators and
institutions, not only the current awardees, have an equal
opportunity to compete for the program funds and that NCATS
officials act impartially and not give preferential treatment
to any organization or individual.\21\ In following these NIH
policies, NCATS provided multiple opportunities to ask for and
receive input from the broader public, including the CTSA
community, on how to improve the CTSA Program.
---------------------------------------------------------------------------
\21\ ethics.od.nih.gov/principles-ethical-conduct-government-
officers-and-employees.
---------------------------------------------------------------------------
--A key approach for input was a Request for Information (RFI)
released in the Fall of 2019. The comments received, many
from the CTSA community, significantly influenced the
updates to the CTSA Program that NCATS is planning. (RFI;
NOT-TR-19-027 \22\)
---------------------------------------------------------------------------
\22\ grants.nih.gov/grants/guide/notice-files/NOT-TR-19-027.html;
(see this video,www.youtube.
com/watch?v=LDBJSl-_QbQ, for an overview presentation of feedback
received).
---------------------------------------------------------------------------
--General feedback was sought from CTSA application peer reviewers
over multiple study sections; many of whom are also CTSA
investigators.
--Informal discussions occurred with CTSA Program consortium
members, individually and in small group settings, over the
course of typical program oversight and interactions.
--Often the first public discussion about a future FOA occurs when
NCATS, like other NIH Institutes and Centers, seeks concept
approval from its Advisory Council during a session open to
the public. This occurs on June 11, 2021. Of note, the
NCATS Advisory Council includes three members that are
Principal Investigators from the CTSA Program.
--In addition, NCATS has built in additional time after the release
of the new FOA--6 months, instead of 2-4 months, prior to
the first application receipt date--to familiarize all
potential applicants with the new FOA, including hosting of
webinars to provide technical assistance to the applicant
community.
--NCATS widely shared a communication to address inaccuracies and
rumors about changes to the CTSA Program FOA. The letter
did not discuss planned changes to the CTSA Program nor
provide data, as sharing details about the FOA in a non-
public manner prior to its posting is not permissible.
--Summary of Stakeholder Feedback: From the input received through
the multiple approaches described above, stakeholder
feedback centered around four distinct areas: (1)
decreasing application administrative burden, (2)
increasing Hub flexibility and Hub specialization
opportunities, (3) expanding Hub funding options, and (4)
preserving partnerships and collaborations. Three
additional areas were identified by NCATS for improvement:
(1) ensuring the CTSA Program's sustainability (in terms of
avoiding the need to reduce the number of hubs or cut
budgets), which requires updates to budget formulas and
calculations; (2) increased emphasis towards addressing
health disparities; and (3) strengthening clinical research
capabilities, which have been critical to the national
responses to the opioid epidemic and the coronavirus
disease 2019 (COVID-19) pandemic.
Hub Budgeting: NCATS takes the proper stewardship of taxpayer funds
very seriously. NCATS does not intend to change the number of hubs or
the amount of funding dedicated to the hub core awards. Future award
amounts will be based on the amount requested by each applicant and
will follow a revised formula for classifying the size of awards from
what is currently used. In addition to incorporating feedback from
different stakeholders, one of NCATS' objectives is to ensure the long-
term sustainability of the program while avoiding a reduction in the
number of hubs or reducing hub budgets to stay within the appropriated
budget for the program. Requested budgets for CTSA awardees have been
increasing to the highest award size under the CTSA graduated award
structure, which is not sustainable under current funding for the
program, so a restructured award calculation is needed. The total award
size of future hubs is anticipated to be similar to the current awards
for the vast majority of awardees.
Structure of the Program Applications: NCATS considered extensive
public feedback, outlined above, in updating the CTSA Program FOA,
including how these updates could contribute to stabilization for the
awardees and to sustainability of the program. To date, the application
process for institutions applying for CTSA hub awards has been
complicated and burdensome, linking up to three separate activities
together into one package, the U54 application. Linking the Hub, Career
Development, and Training activities together for application
submission and peer review is primarily for the benefit of NIH in being
able to track these activities. However, based on feedback, it places
substantial burden on the applying institution in the form of
developing large, complex applications, often containing several areas
of duplicate information. The review of three separate activities in
one application risks pulling an institution out of funding range, due
to one of the activities not faring well in peer review. Applicants
that do not successfully compete face a prolonged period of uncertainty
for funding, while having to address, revise, and resubmit the entire
U54 application package for a subsequent review cycle. These factors
combined with the duration of the awards--five years --raises the
stakes of each application and contributes to an environment where
applying and awarded institutions are in a constant state of
application preparation.
Stakeholder concerns about the complexity of the current
application are an important and consistent piece of feedback NCATS
received. Separating the applications will streamline the submission
process for each component, will reduce duplication of information in
an application, will result in less reliance on the success of one part
of the application, will avoid the risk of significant delays in
awarding a hub if the Training or Career Development components are not
strong, and may allow better alignment of Training and Career
Development awards with the clinical training calendar. Separating the
Hub application from the training and career development applications
will also allow the Hub application, which is the key institutional
award, to be awarded for up to 7 years, more than the standard 5 years.
With this strategy, NCATS intends to provide further stability to an
institution's funding by extending the Hub award. Combining all
applications together does not allow for that seven-year Hub award
option, as NIH limits training and career development awards to 5
years. Separating the applications and providing the additional planned
funding opportunities will also give the institutions more control over
where they place their priorities based on their own strengths, another
key piece of feedback received through stakeholder input.
In closing, we hope that these responses have addressed your
concerns. If not, NCATS is happy to provide additional information.
NCATS recognizes the significance of the CTSA Program. The pandemic has
further served to highlight the importance of this program in
responding to emerging clinical and translational needs at local,
regional, and national levels. NCATS' intent with the proposed updates
to the CTSA FOA is to strengthen the program, provide additional
funding stability, and continue to incorporate research to tackle
health disparities through this program. NCATS also wants to address
important concerns raised by the CTSA community to streamline
application and award preparation processes, continue to emphasize the
importance of partnerships, and allow institutions more flexibility to
leverage their strengths in contributing to this important national
resource.
Question. Dr. Collins, the impact of COVID-19 has been
significant--both to Americans physical health, but also to their
mental health. The fiscal year 2022 budget includes $25 million for
focused research on the impact of the pandemic on mental health.
Can you discuss what research areas this funding will be focused on
and how the All of Us research initiative will play a role in
understanding the full impact of the pandemic?
Answer. The All of Us Research Program's participants come from
diverse communities across the United States and generously donate
their data and time to drive a wide range of biomedical discoveries,
which are vital for informing public health strategies and
preparedness. Due to the diverse nature of the program, the All of Us
Research Program will play a vital role in understanding the mental and
physical impact of the pandemic across the United States and within
some of the hardest-hit communities. All of Us began to address the
challenge of the coronavirus disease 2019 (COVID-19) pandemic in May
2020 by leveraging its significant and diverse participant base to seek
new insights into COVID-19 and its impact through an online COVID-19
Participant Experience (COPE) survey.\23,24\ The COPE surveys focused
on understanding the mental and physical impacts of the COVID-19
pandemic on participants and included questions on symptoms, stress,
social distancing, social determinants of health, and economic impacts.
Participants were invited to take the survey in May, June, July,
November, and December 2020, and February 2021. This multi-pronged
assessment will enable researchers to study the effects of COVID-19
over time and better understand how COVID-19 affects people's mental
and physical health differently. To date, over 10,000 participants
completed all six COPE surveys and over 100,000 completed at least one
COPE survey during the pandemic, with 70 percent of those participants
coming from a community that is historically underrepresented in
biomedical research.
---------------------------------------------------------------------------
\23\ allofus.nih.gov/news-events-and-media/announcements/all-us-
research-program-launches-covid-19-research- initiatives.
\24\ www.nlm.nih.gov/dr2/
COPE_Survey_NIH_All_of_Us_Clean_4.27.20.pdf.
---------------------------------------------------------------------------
In addition to COPE, All of Us tested blood samples from over
24,000 participants collected between January 2 and March 18, 2020, for
the presence of SARS-CoV-2 antibodies, which provided evidence of
infection in five states prior to initial reports. The program
anticipates making the full results of this study available in June
2021.\25\ Additionally, All of Us is collecting relevant electronic
health record (EHR) information from more than 246,000 participants,
some of whom have been diagnosed with COVID-19 or sought healthcare for
related symptoms, to help researchers look for patterns and learn more
about the physical and mental health impacts of COVID-19 and the
effects of different medicines and treatment. As data are made
available from all of these efforts, researchers will look for new
leads that may bring greater precision to the diagnosis, treatment, and
prevention of COVID-19, including those communities that have been hit
the hardest. The program will make data gathered through these
activities broadly accessible to approved researchers on a rolling
basis, in future releases of its secure data platform, the Researcher
Workbench.\26\ The program will continue to explore additional ways it
can leverage its unique and diverse dataset to answer critical research
questions to enhance our understanding about the full impact of the
pandemic, especially with a focus on mental health.
---------------------------------------------------------------------------
\25\ The results of this study were announced on June 15, 2021;
complete details at: allofus.nih.gov/news-events-and-media/
announcements/nih-study-offers-new-evidence-early-sars-cov-2-
infections-us.
\26\ www.researchallofus.org/.
---------------------------------------------------------------------------
Question. Dr. Collins, the COVID-19 pandemic highlighted the need
to use non-human primates (NSP) in research. The budget requests $30
million for NSP infrastructure.
Can you provide further details to the Committee on the need for
this funding and details on how this funding would be allocated and to
whom?
What types of research would be at jeopardy if NSPs were not
replaced or expanded?
Answer. The National Institutes of Health (NIH) remains committed
to protecting animal welfare while, at the same time, advancing
biomedical research and human health. The budget request for $30
million for nonhuman primate infrastructure would cover facilities used
to house nonhuman primates which require continual updates and
maintenance to ensure responsible stewardship over these invaluable
resources. The funds in the budget request would be distributed by
soliciting applications from NIH grantees to improve existing
facilities, not to establish new nonhuman primate facilities. Several
nonhuman primate facilities have existed for over 60 years and housing
enclosures require frequent repair and replacement. New construction
for research facilities would include animal holding rooms, necessary
equipment such as surgical tables, centrifuge, ultrasound, clinical
analyzer, procedure, and veterinary clinical support in order to meet
or exceed the current high-level care of the nonhuman primates.
Additionally, the COVID-19 pandemic highlighted the need for new
construction to expand animal biosafety level 3 areas in order to have
biocontainment facilities associated with nonhuman primate facilities.
In addition to ethically appropriate housing, nonhuman primates require
a proper diet, clinical/veterinary care as well as psychological and
environmental enrichment, which necessitates skilled staff and
additional resources including supplemental produce, various enrichment
devices such as foraging devices for food, various toys, and puzzles.
NIH would support expansion at existing NIH-supported facilities to
leverage the investment. The NIH Office of Research Infrastructure and
Programs (ORIP) supports a well-coordinated national consortium of
seven National Primate Research Centers (NPRCs) and other breeding
colonies that collectively address research needs and trends, best
husbandry practices, maintenance of genetic diversity, standardization
of models, ethics, rigor, and reproducibility. NPRCs are national
resources serving not only NIH-funded investigators but other federally
funded investigators, foundations, and industry, including many SARS-
CoV-2 projects in the last year.
Research with animal species, including nonhuman primates, remains
critical for modeling human physiology and is essential for developing
new prevention strategies, treatments, and cures for disease beyond the
need for responding to emerging infectious diseases. Nonhuman primates
have been essential for understanding human biology and developing
treatments for diseases, mostly because of our shared anatomy,
physiology, and behavior. Importantly, the genetic sequence
similarities between nonhuman primates and humans can reach up to 98.77
percent, which has made nonhuman primates models critical for studying
neurobiology, transplant tolerance and rejection, infectious diseases,
reproductive biology, and regenerative medicine. More recent
applications have been in regenerative medicine and gene therapy and
editing. There is a rapidly emerging need for marmosets in the
neurosciences where recent National Academies of Sciences, Engineering,
and Medicine (NASEM) reports and the Brain Research Through Advancing
Innovative Neurotechnologies (BRAIN) Initiative community have pointed
out that demand far exceeds supply.\27\ Another critical area of
intense need and research development is nonhuman primate models of
Alzheimer's disease to develop therapies. Nonhuman primate models are
commonly used for studies of visual systems, auditory systems,
cognitive function, and brain connectivity. The single largest
application of nonhuman primates continues to be in developing vaccines
and therapies for HIV/AIDS.
---------------------------------------------------------------------------
\27\ www.nap.edu/read/25356/chapter/7.
---------------------------------------------------------------------------
Research using animal models, including nonhuman primate models,
has led to tremendous advances critical for saving countless lives and
extending human life expectancy around the world. Until suitable non-
animal models are developed, the complexity of human systems, both in
health and in disease, can only be truly understood through
complementary model systems with sufficient complexity, and nonhuman
primates remain invaluable for this effort. When animal models are
required, NIH will only conduct and support research in accordance with
the highest scientific and ethical principles. To uphold these
principles, the NIH budget includes investments in nonhuman primate
facilities, resources, and enrichment.
Question. Dr. Collins, how much funding, broken down by Institute
or Center, has NIH repurposed for COVID-19 related lab reopenings or
lost research activities?
Answer. To support our recipients affected by the pandemic, the
National Institutes of Health (NIH) provided extensions, both funded
and unfunded, as well as administrative supplements, to address the
unanticipated impacts of the pandemic. The NIH has also issued multiple
funding opportunities for current recipients to repurpose existing
awards and expand the scope of ongoing research to include coronavirus
disease 2019 (COVID-19) research activities.\28\ Continued support for
these projects is contingent on satisfactory progress, the availability
of funds, and NIH Institute and Center (IC) funding priorities, which
continue to change as the pandemic, and research on COVID-19
progresses.
---------------------------------------------------------------------------
\28\ grants.nih.gov/grants/guide/COVID-Related.cfm.
---------------------------------------------------------------------------
Decisions related to individual awards are made by the funding NIH
IC on a case-by-case basis, taking into account those critical factors.
All requests to change the scope of an NIH grant award require prior
approval from the awarding NIH IC, as stipulated in the NIH Grants
Policy Statement, section 8.1.2.5.\29\
---------------------------------------------------------------------------
\29\ grants.nih.gov/grants/policy/nihgps/HTML5/section_8/
8.1.2_prior_approval_
requirements.htm#Change4.
---------------------------------------------------------------------------
The NIH continues to analyze the data on the impact of COVID-19 on
the biomedical research community, and its potential impact on NIH
budget and grant activities.
Question. It is my understanding that one of the main issues NIH
faced related to COVID-19 expenses was for post-doctoral candidates
finishing their training, research, or fellowship.
How has this issue been addressed and do you expect to see a
funding issue related to the extension of some of these grant awards
into fiscal year 2022?
Answer. The coronavirus disease 2019 (COVID-19) pandemic, along
with extensive mitigation measures, has adversely affected progress in
many biomedical research settings. Evidence from multiple sources,
including results from a survey during the fall of 2020, indicates
legitimate concerns about career trajectory for early career
scientists.\30\ Hearing these concerns, the National Institutes of
Health (NIH) issued a Guide Notice detailing our approach to support
early career scientists whose career trajectories may have been
significantly affected by the pandemic.\31\ Specifically, NIH is
providing an opportunity for recipients in their last year of NIH
Fellowship (F) and NIH Career Development (K) awards who have been
impacted by COVID-19 to request extensions.\32\ Such extensions will be
considered on a case-by-case basis, within the existing availability of
funds.
---------------------------------------------------------------------------
\30\ nexus.od.nih.gov/all/2021/03/25/the-impact-of-the-covid-19-
pandemic-on-the-extramural-scientific-workforce-outcomes-from-an-nih-
led-survey/.
\31\ grants.nih.gov/grants/guide/notice-files/NOT-OD-21-052.html.
\32\ https://nexus.od.nih.gov/all/2021/02/08/extensions-for-early-
career-scientists-whose-career-trajectories-have-been-significantly-
impacted-by-covid-19/.
---------------------------------------------------------------------------
Generally speaking, the NIH typically makes between 500 to 600 F
and K extensions per year, the vast majority (more than 95 percent) of
which are no-cost extensions. Only seven funded extensions were awarded
in fiscal year 2019. In fiscal year 2020, the NIH awarded 548
extensions, with 75 (14 percent) of these being funded extensions. Thus
far in fiscal year 2021, 15 funded extensions are linked to NOT-OD-21-
052, but we will have a much better sense of uptake as the fiscal year
concludes. Though there appears to be a relative increase in the number
of funded extensions commensurate with the pandemic, the absolute
numbers remain low.
______
Questions Submitted by Senator Cindy Hyde-Smith
Question. What is the fully intended scope of ARPA-H? Will it
address diseases beyond cancer, diabetes, and Alzheimer's, such as ones
with more challenging markets? Do you have examples?
Answer. The scope of the Advanced Research Projects Agency for
Health (ARPA-H) is intended to be broad and, indeed, stretch beyond the
areas initially identified by the President. There are a number of
areas with substantial unmet needs--some examples include emerging
infectious disease, rare and ultra-rare disease, and antimicrobial
resistance--and, with targeted investments over time, breakthrough
progress could be made. In addition to specific disease areas, ARPA-H
intends to build capabilities and explore various platform
technological approaches which may have broad applicability across a
range of diseases and conditions. A recent commentary in Science \33\
outlined some exciting concepts such as developing mRNA vaccines to
prevent most cancers; creating molecular ``zip codes'' to more
precisely target tissues and cell types while minimizing side effects;
deploying holistic interventions that identify those at high-risk and
leverage new telehealth approaches to eliminate racial disparities in
maternal morbidity and mortality rates and premature births; and
developing small, highly accurate, inexpensive, non-intrusive, wearable
24/7 monitors for blood pressure and blood sugar. While these examples
are meant to illustrate the breadth of potential projects that ARPA-H
could support, we believe it is projects like these that can have a
significant impact for patients who are relying on biomedical research
and innovation to live longer, healthier lives.
---------------------------------------------------------------------------
\33\ science.sciencemag.org/content/373/6551/165.
---------------------------------------------------------------------------
Question. Additionally, how will ARPA-H fit into the larger health
focused R&D structure? How will its role be defined as unique among the
various funding programs, and will there be coordination with other
entities such as BARDA to ensure cooperation and avoid duplication?
Answer. The Advanced Research Projects Agency for Health (ARPA-H)
is meant to become an integral component of the constellation of
agencies focused on promoting health and research and development--both
within and beyond NIH and HHS. As described in a recently published
commentary in Science,\34\ ARPA-H should be housed as a new entity
within NIH. The rationale for this organizing principle is two-fold.
First, the goals of ARPA-H fall squarely within the mission of the NIH,
which is ``to seek fundamental knowledge about the nature and behavior
of living systems and the application of that knowledge to enhance
health, lengthen life, and reduce illness and disability.'' Second, the
NIH offers a rich source of fundamental health research that will be
foundational for a constructive, collaborative, and productive
relationship with ARPA-H. We envision robust collaborations on
synergistic topics with the existing NIH Institutes and Centers, along
with organizations both outside and within the government. The added
benefit of housing ARPA-H within NIH is that it will create
administrative efficiencies so that more resources can be directed
toward the mission and help avert duplication of effort.
---------------------------------------------------------------------------
\34\ science.sciencemag.org/content/373/6551/165.
---------------------------------------------------------------------------
In mid-July, the Administration launched a Federal Joint Fast Track
Action Committee (FTAC) intended to help steer the creation of ARPA-H
and lay the groundwork for strong interagency coordination. OSTP and
NIH serve as co-chairs of this committee that includes representatives
from the Department of Agriculture, DARPA, Office of the Under
Secretary of Defense for Research & Engineering, ARPA-E, BARDA, CDC,
CMS, FDA, VA, EPA, NSF, and the Smithsonian Institution, among others.
Bringing these entities together at an early stage will help ensure
strong collaboration and coordination among the various research-
focused organizations throughout the Federal Government. The agency
personnel who sit on the FTAC will also be a valuable source of insight
and advice as ARPA-H is launched.
______
Questions Submitted by Senator Patrick J. Leahy
Question. I strongly support the Administration's renewed approach
to innovation in medical research through the establishment of the
Advanced Research Projects Agency for Health (ARPA-H). COVID-19 has
shown that a commitment to breakthrough innovation, directed allocation
of resources, and collaborative approaches can accelerate how
scientific breakthroughs can be transitioned to treatments and cures.
The administration has proposed that the agency will focus on
innovative treatments in cancer, Alzheimer's disease, and opioid
disorders. Several institutions in Vermont are national leaders in
these stated research fields despite their smaller and more rural
nature. While I strongly support any efforts to accelerate innovation,
I am concerned that valuable collaborators could be left out or lose
out on Federal funding, particularly if there is no traditional grant
application process.
What role will smaller and more rural research institutes play in
ARPA-H? If projects are funded outside a grant application process,
will there be established guidelines to include collaborators from
rural or traditionally underrepresented areas?
Answer. Over the long term, the proposed structure for the Advanced
Research Projects Agency for Health (ARPA-H) is intended to empower the
ARPA-H leadership and staff to set and execute on research priorities
for a variety of high-risk, high-reward, milestone-driven projects that
can lead to novel capabilities, platforms, and resources that are
applicable to a range of diseases. These priorities include the
opportunity to fund smaller and more rural research institutes.
For the initial direction, the Administration is working to set up
multiple pathways, both within the government and the broader
stakeholder community, for priority setting and for exploring new areas
ripe for research at ARPA-H. At the time of this hearing, the White
House Office of Science and Technology Policy (OSTP) and the National
Institutes of Health (NIH) are in the planning phases of convening
multiple listening sessions with key stakeholder groups including
patient organizations, industry, venture capitalists and
philanthropists, and others from the academic and research communities.
During these sessions, stakeholders will be asked to offer their
perspective on what they see as the greatest research challenges and
opportunities that could be addressed using the ARPA-H model. This
input will help refine the scope and provide a wealth of ideas for the
first ARPA-H director to consider as they develop the agency's vision.
In mid-July, the Administration established a Joint Fast Track
Action Committee (FTAC) to help steer the creation of ARPA-H and lay
the groundwork for strong interagency coordination. OSTP and NIH serve
as co-chairs of this committee that includes representatives from
Department of Agriculture, DARPA, Office of the Under Secretary of
Defense for Research & Engineering, ARPA-E, BARDA, CDC, CMS, FDA, VA,
EPA, NSF, and the Smithsonian Institution, among others.
Soliciting a diversity of perspectives and approaches will be a key
tenet of the Advanced Research Projects Agency for Health (ARPA-H).
Much like DARPA and ARPA-E, it will do so by supporting the best
strategies to solve an identified challenge and by pursuing multiple
approaches. Program managers will also have the authority to combine
proposals from different institutions to assemble the boldest, most
innovative portfolio, allowing each team to build on their strengths
while benefiting from the knowledge, expertise, and resources from
other institutions. ARPA-H will also provide awards that range in size
and mechanism--from smaller, pilot projects to develop a prototype, to
complex multi-site trials, to prizes that stimulate healthy competition
and ingenuity. Further, ARPA-H will support a Small Business Innovation
Research (SBIR) and Small Business Technology Transfer (STTR) program
with business development, commercialization, and other resources to
provide small businesses with the tools they need to be successful.
These approaches are examples of mechanisms that ARPA-H will utilize to
support a range of organizations across the country which may include
small and/or rural institutions, and its portfolio will be regularly
evaluated to ensure there is diversity of perspective. Because ARPA-H
will be a nimble, dynamic organization, it will be able to readily
pivot to experiment with new approaches.
Question. Chronic pain is a significant public health issue
affecting an estimated 50.2 million Americans each year. Based on data
from the National Health Interview Survey (NHIS), the total value of
lost productivity due to chronic pain is estimated to be nearly $300
billion annually. With little known about alternatives for treating and
managing relief from pain, medical providers are often limited to
prescribing highly addictive opioids or muscle relaxants to help
patients mitigate symptoms from pain. Scientific research suggests that
long term use of such medications can result in the body's reduction of
its own ability to fight pain. Even for patients who do not experience
direct abuse or addiction with long term use, scientists have found
that withdrawal symptoms are present when patients stop taking these
medications. Unfortunately, research into addiction and alternatives to
treatment has historically lagged at NIH. Enhanced research on chronic
pain management and treatment, other than through the use of highly
addictive opioid painkillers, has the potential to reduce substance
abuse and promote better methods for addressing pain.
I strongly support the NIH Heal Initiative to find solutions to
curb the national opioid public health crisis by understanding,
managing, and treating pain. Please describe any progress made by the
HEAL Initiative on medication development to alleviate pain and to
treat addiction. What remains the biggest barrier to research to
investigate new and alternative options to treat chronic pain?
Answer. The National Institutes of Health (NIH) recognizes the need
to improve pain management without risk of addiction and other serious
side effects. NIH is taking a multi-pronged approach to develop safe
and effective therapies to reduce our reliance on opioids and treat
addiction. The NIH Helping to End Addiction Long-term (HEAL) Initiative
launched in 2018 has awarded over $1.5 billion for research to discover
and accelerate development of non-addictive pharmacological and non-
pharmacological pain treatments, as well as treatments for opioid use
disorder (OUD) and overdose.
Through the HEAL Initiative, NIH supports over 70 targeted studies
to accelerate the development of treatments for OUD, including novel
medications and biologic agents, as well as novel formulations of
approved medications to treat OUD and prevent opioid overdose. To date,
16 Investigational New Drug Applications were filed with the U.S. Food
and Drug Administration and authorized to proceed for human studies.
These studies focus on a variety of drug targets, as well as vaccines
that could prevent opioids from entering the brain. HEAL currently
funds nine opioid vaccine projects including vaccine candidates
targeting oxycodone,\35\ fentanyl \36\ and heroin.\37\ This strategy
could offer more accessible, manageable treatment through longer-
lasting vaccines to reduce the risk of relapse.
---------------------------------------------------------------------------
\35\ reporter.nih.gov/search/Pcd2IghkPU6lnJkOT7FlFQ/project-
details/9778811.
\36\ reporter.nih.gov/search/Wp_sHzUhIUuYqDimSa90iw/project-
details/9737173.
\37\ reporter.nih.gov/search/GNnJWbYvQUeIlbwhgFofXA/project-
details/9734921.
---------------------------------------------------------------------------
HEAL-supported work also includes studies to identify, optimize and
test promising molecules, biologics, and devices for treating pain that
target non-opioid pathways in the nervous system. Biomarker studies to
enhance clinical trials and improve best practices are moving forward.
In addition, non-pharmacological approaches to manage many different
pain conditions are being evaluated through effectiveness and
implementation research approaches.
In these ways, HEAL is providing much needed resources to advance
research on new and safe alternatives to opioids for chronic pain. The
complexity and diverse nature of chronic pain itself along with a high
prevalence of other co-occurring chronic conditions such as diabetes,
depression, and autoimmune disorders create an enormous challenge for
advancing research.
Mechanisms for the causes of different pain conditions vary,
biomarkers for patient response to treatment and likelihood for
progression of disease also are characteristic of the disease
condition. In addition, treatments for co-morbidities require careful
balancing and often long- term multidisciplinary care. These and other
factors require an expanded breadth and scope of pain research to
better provide personalized care for those with chronic pain. The
Federal Pain Research Strategy \38\ describes research priorities to
relieve the burden of pain. The NIH HEAL initiative provided support to
move many of the report's recommendations forward.
---------------------------------------------------------------------------
\38\ www.iprcc.nih.gov/federal-pain-research-strategy-overview.
---------------------------------------------------------------------------
Specifically, the NIH HEAL initiative established essential pain
research infrastructure to accelerate development of new medications
and devices to treat pain. An analgesic screening platform uses animal
and human cell-based models such as neural tissue chips for rapid
screening of molecules or devices for analgesic-relevant biological and
pain behavioral activity. HEAL, with input from academic and industry
partners, established an Early Phase Pain Investigation Clinical
research network (phase 2 studies) to test safety and efficacy of novel
therapeutics and a later stage pain management Effectiveness Research
Network (ERN) to compare effectiveness of pharmacological and non-
pharmacological approaches in many different pain conditions. The
Pragmatic and Implementation Studies for the Management of Pain to
Reduce Opioid Prescribing (PRISM) network focuses on clinical trials of
non- pharmacologic pain therapies in healthcare systems. The Phase 2
network will launch trials on two new analgesics in late 2021. The ERN
is supporting eight large trials for various pain management
strategies. PRISM is supporting six large trials in healthcare systems.
In addition, HEAL established an analgesic development pipeline to
accelerate the development and testing of novel drugs and devices. This
comprehensive program uses team-based science coupled with a
comprehensive set of research resources to bring new therapeutics
rapidly to the clinic. To advance the discovery and validation of new
drug targets, HEAL has funded over 30 projects to discover and verify a
diverse set of drug target types across multiple pain conditions, six
drug optimization studies on new safe and effective pain treatments,
and 11 projects to test the effectiveness of implanted devices and
noninvasive stimulation of nerves in the brain or throughout the body
to reduce perception of pain. In addition, to improve the efficacy of
clinical trials for pain treatments, and to increase the chance that
new therapeutics will advance along the regulatory path to approval,
HEAL tests the development of biomarkers to objectively measure pain,
including pain associated with sickle cell disease, musculoskeletal
disease, nerve pain and headache. Promising biomarkers identified
through this program may advance to clinical validation through the
Early Phase Pain Investigation Clinical Network (EPPIC-Net). Findings
from these studies could improve quality of life for millions of people
in the United States who experience pain daily. Recent HEAL
accomplishments toward new therapeutics include two patent filings for
small molecule modulators of pain receptors involved in chronic pain
and migraine.
New directions for HEAL will also continue to pursue goals laid out
in the Federal Pain Research Strategy,\39\ including demonstration
projects to aid in the development of a coordinated approach to pain
management in healthcare systems. This effort would assess multi-
disciplinary and multimodal approaches to pain management embedded in
healthcare systems. Research within systems of pain care would allow
for effective interventions to be adopted into the healthcare system
and improve access for patients. Focused discussion with select
healthcare program leadership would identify pain conditions of
greatest opportunity, with an emphasis on effectiveness research,
quality management and team-based care. This effort would seek to
leverage existing infrastructure through ongoing collaborative and
interagency efforts.
---------------------------------------------------------------------------
\39\ www.iprcc.nih.gov/federal-pain-research-strategy-overview.
---------------------------------------------------------------------------
Another specific effort in development aims to advance health
equity to address the wide disparities in care and treatment for pain
and addiction, known to result in both the undertreatment and
overtreatment with opioids, increased risk of addiction and overdose,
lack of access to effective non-pharmacological options for pain
treatment, and lack of access to evidence-based addiction care.
Disparities in pain management exist across multiple levels: pain
assessment, treatment, and management at the patient, provider,
community, and healthcare system levels. Planned expansion to HEAL
includes the development and implementation of culturally appropriate
interventions for the prevention and management of pain and addiction
in diverse populations, with a focus on sustainable and scalable
interventions that can be rapidly implemented by healthcare systems.
In addition, recent discoveries in human genetics and molecular
biology will be incorporated into the development of a novel team-based
platform to rapidly test targets and candidate therapeutics for diverse
human pain conditions and share findings with the wider pain research
community. This research will address pain systems and allow for a
variety of research questions including conditions of chronic analgesic
use, other drug use, substance use disorders (SUDs) and other co-morbid
conditions, and will enable and accelerate human gene- and cell- based
validation of pain therapeutic targets through the HEAL initiative and
other pipelines. This will build on existing HEAL research on
preclinical and translational research in pain, and ongoing efforts to
accelerate the development of novel treatments for pain. Through these
and other efforts at HEAL and across the NIH, we aim to continue to
improve our understanding of pain and develop non-addictive, effective
therapies.
Question. Migraine is currently the second leading cause of all
global disability. Unfortunately, due in part to limited research and
treatment, inappropriate opioid prescriptions for migraine present
Americans with ongoing risks of opioid use disorders and have worsened
outcomes in patients. Overall, 6 million Americans living with
migraines are active opioid users. I strongly support the NIH Heal
Initiative to find solutions to curb the national opioid public health
crisis by understanding, managing, and treating pain. While migraine
grant proposals are eligible for consideration under the HEAL request
for applications (RFAs) issued for pain research, less than 1 percent
of HEAL Initiative appropriations have funded headache disorders
research--the least funded NIH area among all the nation's burdensome
diseases. I am very concerned about the failure to attract enough
investigators to this historically under-funded research area.
Does NIH have plans to issue specific RFA programs for headache
disorders research, comparable in scope to the Back Pain Consortium
(BACPAC) group of RFAs for research on back pain?
Answer. The National Institutes of Health (NIH) recognizes the
burden of pain at the individual and population levels and that
headache disorders are prevalent and disabling conditions which affect
millions of Americans. The NIH launched the HEAL Initiative (Helping to
End Addiction Long-term) to improve pain care and better prevent and
treat opioid use disorder. Priorities of the HEAL initiative, developed
with our stakeholders with expertise in pain research and care, include
enhanced understanding of pain, discovery and validation of novel pain
therapeutic targets, testing therapies in clinical settings, and
accelerating the process to bring new therapies to patients. The
initiatives are, or were, open to all pain conditions. The HEAL
initiative also established much needed research infrastructure to
support innovative science. Headache research fits within the scope of
all these initiatives and will benefit from the enhanced
infrastructure.
HEAL funding solicitations call for proposals across all pain
conditions. NIH staff recognizes the low submission rate of headache
applications and broadly disseminates information on HEAL and other
funding announcements to the research community to encourage
submissions. Most funding announcements specifically cite headache as
an area of interest and others are inclusive of headache. Low back pain
is an exception among pain conditions in that it has unique research
gaps such as lack of diagnostic tools and technologies, no accepted
common data elements, poor diagnostic criteria, complex etiology, and
lack of an adequate evidence base for effective practice guidelines.
The HEAL Back Pain Consortium (BACPAC) initiative was launched to fill
these extensive gaps to improve pain care across the spectrum of low
back pain.
Migraine and other headache disorders have good classification
schemas, a range of effective treatment therapies whose development was
supported by NIH research, and evidence-based diagnostic categories and
treatment protocols (International Headache Society). Our understanding
of migraine etiology is more advanced than that for back pain. NIH has
supported transformative basic research that advanced our knowledge of
migraine mechanisms, causes, and predictors, biomarker identification,
and new therapy development. For example, NIH supported investigators
provided the foundation for development of CGRP antibodies now used
widely for migraine therapy. NIH sponsored research also contributed to
understanding how migraine auras activate nociceptors and initiate a
migraine, and the mechanism of action for new migraine therapies such
as vagus nerve stimulation. Basic research on potassium channels,
delta, or kappa opioid receptors, and TRP channels fundamentally
increased our understanding of trigeminal nociceptors and their
involvement in initiating a migraine, giving us new targets for
potential treatments. An NIH sponsored pivotal pediatric migraine
clinical trial changed clinical practice for children with chronic
daily headaches.
NIH and HEAL leadership recognize that far too many headache
sufferers are prescribed opioids despite clear clinical practice
guidelines that call for non-opioid effective alternatives rather than
opioids. This practice reflects the sparsity of headache specialists
and the lack of and education of our primary care providers who are
often the first to treat those with disabling migraines. NIH also
recognizes the need to expand the headache research workforce. The HEAL
initiative recently released funding announcements to support training
and mentorship of early and mid- career researchers in the field of
basic, translational, and clinical pain research. We encourage those
interested in headache research to benefit from these opportunities.
______
Questions Submitted to Dr. Anthony Fauci
Questions Submitted by Senator Richard J. Durbin
Question. I have received a lot of questions from Illinois
families, who are hoping for more clarity on the CDC's most recent mask
guidelines. Many vaccinated parents--with unvaccinated children at
home--are wondering if they should be wearing masks when out in public.
What advice would you give to vaccinated parents who have
unvaccinated children at home?
When do you think we will have a COVID vaccine approved for
children younger than 12 years of age?
Answer. Currently authorized coronavirus disease 2019 (COVID-19)
vaccines meet the U.S. Food and Drug Administration's (FDA's) rigorous
standards for safety and effectiveness, and current data suggest that
fully vaccinated people are less likely to transmit severe acute
respiratory syndrome coronavirus 2 (SARS-CoV-2) to others. According to
the Centers for Disease Control and Prevention (CDC), fully vaccinated
people--including those living with unvaccinated children or
adolescents--can resume activities without wearing masks or physically
distancing, except where required by Federal, state, local, tribal, or
territorial laws, rules, and regulations. Individuals ages 2 and older
who are unvaccinated, however, should continue to wear masks in public
and when around people who do not live in their household, except when
eating or sleeping. CDC will continue to evaluate and update public
health recommendations for fully vaccinated people as more information,
including on Delta and other new variants, becomes available.
Efforts to evaluate COVID-19 vaccines in children under age 12
currently are underway, and a COVID-19 vaccine may be available for
this age group by the end of 2021. On March 16, 2021, Moderna, in
collaboration with the National Institute of Allergy and Infectious
Diseases (NIAID) and the Biomedical Advanced Research and Development
Authority (BARDA), launched KidCOVE, a Phase 2/3 study to evaluate the
safety and efficacy of the Moderna COVID-19 vaccine in children ages 6
months to less than 12 years. Pfizer also is conducting a Phase 1/2/3
trial to evaluate its COVID-19 vaccine in this age group. In addition,
other vaccine developers are planning to begin trials to test their
vaccine candidates in children. Until a COVID-19 vaccine is available
for children under age 12, it will be important for all individuals,
especially children and other unvaccinated individuals, to continue to
follow all public health measures for COVID-19 advised by the CDC,
including frequent hand washing and the use of masks and social
distancing in certain settings.
______
Questions Submitted by Senator Joe Manchin, III
Question. My home state of West Virginia is battling an epidemic
during the middle of a pandemic. My state has been devastated by the
drug epidemic, COVID-19, and we now lead the nation in new HIV
infection rates. You have spent much of your career focused on the
prevention, diagnosis, and treatment of HIV/AIDS. Your research has
been instrumental in saving countless lives in the United States and
around the world. The National Institute of Allergy and Infectious
Diseases supports initiatives focused on diagnosing, treating,
preventing and responding to the HIV epidemic in the United States.
These efforts represent steps in the right direction, but will not
alone end West Virginia's increasing numbers of new HIV infections and
other opioid-related infectious diseases.
What is being done to replicate testing and surveillance efforts we
saw put into place for COVID-19 for other infectious diseases, like
HIV/AIDS?
What public health infrastructure would be required to bring better
infectious disease testing and surveillance to fruition?
Answer. The Federal response to coronavirus disease 2019 (COVID-19)
relied heavily on the utilization and expansion of existing resources
for human immunodeficiency virus (HIV) and other infectious diseases.
By leveraging available resources, we have been able to accelerate the
development of diagnostic tests and other medical countermeasures, as
well as surveillance and community engagement efforts. In turn,
knowledge gained from the COVID-19 response may inform strategies to
address other infectious diseases such as HIV. This includes efforts
undertaken by the U.S. Department of Health and Human Services (HHS) to
end HIV in the United States by 2030 through the Ending the HIV
Epidemic in the U.S. (EHE) initiative. EHE is coordinating across HHS
agencies and with patient, community, academic, and other partners to
plan, design, and deliver local HIV prevention and care services. This
``whole-of-society'' approach is a model for ending both the HIV
epidemic as well as the COVID-19 pandemic. Proper diagnosis and
treatment of HIV are key components of this initiative, and efforts to
improve testing and surveillance for HIV are ongoing.
An important aspect of the response to the COVID-19 pandemic as
well as the HIV epidemic is community engagement. The National
Institute of Allergy and Infectious Diseases (NIAID), in cooperation
with the Department of Defense, established the COVID-19 Prevention
Network (CoVPN) by leveraging existing NIAID-funded clinical trials
networks, including networks focused on HIV treatment and prevention.
The CoVPN built on existing community relationships to enhance trust
and meaningful engagement in key racial and ethnic minority communities
throughout the United States to promote diverse participation in
clinical trials for COVID-19. The community relationships enhanced by
the CoVPN may be further leveraged to advance efforts, including
testing and surveillance, for HIV and other infectious diseases.
The National Institutes of Health (NIH) also anticipates that the
rapid establishment of COVID-19 testing and surveillance may help to
address HIV and other infectious diseases. NIH launched the Rapid
Acceleration of Diagnostics (RADx) initiative to speed innovation in
technologies to test for severe acute respiratory syndrome coronavirus
2 (SARS-CoV-2), in partnership with the Biomedical Advanced Research
and Development Agency (BARDA), the Centers for Disease Control and
Prevention (CDC), the U.S. Food and Drug Administration (FDA), and the
Defense Advanced Research Projects Agency (DARPA). As part of RADx, NIH
and CDC are evaluating whether frequent self-administered, at-home
SARS-CoV-2 testing helps reduce community transmission of SARS-CoV-2.
Efforts to develop and deploy rapid, point-of- need diagnostics for
SARS-CoV-2--including at-home testing kits--may inform community-based
testing and surveillance strategies for other infectious diseases,
including HIV.
NIH and NIAID will continue to build on investments in improved
diagnostic tests for SARS- CoV-2 to support the development of novel
diagnostic tests for other infectious diseases such as HIV. In
addition, lessons learned on the best way to integrate and expand on
existing research efforts and infrastructure will be invaluable as we
continue to prepare for--and respond to-- other existing and emerging
infectious disease threats.
As discussed in response to part a of this question, the Federal
response to the COVID-19 pandemic has strengthened existing
partnerships and coordination mechanisms, as well as established new
partnerships that will inform the response to future infectious disease
pandemics and existing epidemics, such as the HIV/AIDS epidemic in the
United States. The coordinated efforts through RADx and the CoVPN
allowed us to leverage the intrinsic strengths from public and private
sector partners to achieve an unprecedented level of scientific
achievement and community engagement. When the COVID-19 pandemic ends,
lessons learned from our experiences with RADx and the CoVPN will
continue to help inform efforts to address other infectious disease
threats.
NIH and NIAID will continue to work with HHS Operating Divisions
and other Federal agencies to identify the actions that were most
effective in responding to the COVID-19 pandemic. This information may
result in new initiatives, strategic plans, and/or formal assessments
of pandemic preparedness.
______
Questions Submitted by Senator Richard C. Shelby
Question. As America begins to assist the world to vaccinate all
who want it, the current vaccine options can be problematic for
countries without the infrastructure to store vials in a cooled or
frozen environment.
How beneficial could an effective, intranasal vaccine option be for
developing countries that cannot store the current vaccines at frigid
temperatures or produce the healthcare workers to give the shot?
Do you see this option benefitting Americans who may be hesitant to
receive the current vaccine dosage in a shot?
Answer. Global access to safe, effective vaccines will be critical
to address the coronavirus disease 2019 (COVID-19) pandemic. Limiting
the spread of the severe acute respiratory syndrome coronavirus 2
(SARS-CoV-2) virus in foreign countries helps to control the pandemic
in those countries while also limiting the development and spread of
variants that could eventually be introduced into the United States. To
enhance vaccine availability in foreign countries, the Biden
Administration has supported and contributed to COVAX, a global
mechanism for equitable access to COVID-19 vaccines. COVAX has
delivered COVID-19 vaccines to more than 100 countries, the majority of
which have lower-income economies. The United States also has made
millions of doses of COVID-19 vaccines available to other countries to
support vaccination campaigns around the world.
Existing COVID-19 vaccines are being successfully administered
globally, and several COVID-19 vaccines authorized for emergency use or
in clinical testing in the United States can be shipped and stored at
refrigerator temperatures (2-8 degrees Celsius). Still, the development
of vaccines that can be administered with less skill and/or stored at
warmer temperatures have the potential to expand vaccination efforts
both in the United States and abroad. The National Institute of Allergy
and Infectious Diseases (NIAID) is supporting the development of
vaccine candidates and platforms that may be more accessible and
convenient than currently available COVID-19 vaccines, including a
single-dose intranasal SARS-CoV-2 vaccine candidate called ChAd-SARS-
CoV-2-S. NIAID scientists and collaborators recently showed that the
intranasal ChAd-SARS-CoV-2-S vaccine candidate limited infection in
non-human primates. Novel vaccines with alternative administration
strategies, such as intranasal vaccines, may reduce barriers to
transporting and administering vaccines in developing countries. It is
important to note, however, that these vaccines may still need to be
kept at low temperatures or may require administration by a healthcare
provider with specialized training to ensure accurate dosing and
administration. For example, FluMist Quadrivalent--a U.S. Food and Drug
Administration-approved intranasal vaccine against influenza--must be
administered by a healthcare provider in the United States.
In addition, National Institutes of Health (NIH) scientists and
NIH-supported researchers are studying additional vaccine delivery
technologies, including vaccines that can be orally administered or
that utilize microneedles in patches placed on the skin to deliver the
vaccine. For example, NIH scientists have begun preclinical evaluation
of a virus-like-particle-based vaccine candidate for SARS-CoV-2 that
can be administered orally, and NIH-supported researchers are
evaluating a patch-based vaccine for SARS-CoV-2. An NIH-supported Phase
I trial of a patch-based vaccine candidate for influenza showed that
individuals that received the vaccine had a similar immune response to
those receiving the influenza vaccine via intramuscular injection. NIH
also is supporting the development of another promising patch-based
vaccine candidate for influenza that uses biodegradable microneedles
originally developed through NIH-supported research to stabilize
vaccines and antibiotics outside of the cold chain. Although additional
testing will be necessary, orally administered and patch-based vaccines
may prove to be an invaluable tool in resource-limited settings as they
may require little to no refrigeration, as well as less training to
administer correctly.
As we work to address the COVID-19 pandemic, as well as other
infectious disease threats, recent innovations in vaccine technology
will help make it easier to get vaccines to areas that can be difficult
to serve with traditional vaccines. NIH continues to support research
on intranasal, oral, and patch-based vaccine platforms, all of which
could be highly adaptable for use against a number of infectious
pathogens.
Vaccines that can be administered intranasally may be considered
less invasive than those that require an injection. Such an option may
encourage individuals who are hesitant to receive the COVID-19 vaccines
currently authorized for emergency use in the United States, which are
all administered via intramuscular injection, to become vaccinated.
Additional vaccine delivery technologies, such as oral or patch-based
vaccines may also provide additional flexibilities when trying to reach
individuals in resource-limited areas or who are vaccine hesitant or
needle adverse. As noted in the response to part a of this question,
NIAID is supporting and will continue to support the development of
vaccine candidates with different delivery technologies to reduce
vaccine hesitancy as well as barriers to vaccine access.
______
Questions Submitted to Dr. Diana Bianchi and Dr. Eliseo Perez-Stable
Questions Submitted by Senator Richard J. Durbin
Question. Our nation continues to struggle with racial disparities,
especially in maternal health. The U.S. is one of only 13 countries
where our nation's maternal mortality rates are worse now than they
were 25 years ago. Every year, 700 women in the U.S. die as a result of
their pregnancy--and more than 60 percent of these deaths are
preventable. Tragically, African American and Hispanic women are three
times as likely as White women to die from pregnancy-related issues.
For years, I have introduced the MOMMA's Act with Rep. Robin Kelly, and
I'm so pleased that a major component of our bill was recently signed
into law as part of the American Rescue Plan. Now states can follow in
Illinois' footsteps by allowing new moms to keep their Medicaid
coverage for a full year, versus just 60 days.
What research NIH is doing in this space?
How is NIH working to actually improve maternal and infant
healthcare?
Answer. Maternal health is a priority for the National Institutes
of Health (NIH) and multiple NIH institutes have heavily invested in
research to prevent maternal morbidity and mortality (MMM) and improve
health for women, before, during, and after pregnancy. In fiscal year
2020 NIH supported $407 million in research on maternal health and $224
million in research on MMM.
In a year that was dominated by both the coronavirus disease 2019
(COVID-19) pandemic and renewed calls to combat health disparities and
inequities, NIH ensured these challenges were integrated into efforts
to reduce MMM. In March 2020, researchers in the Eunice Kennedy Shriver
National Institute of Child Health and Human Development's (NICHD)
Maternal-Fetal Medicine Units Network designed the Gestational Research
Assessments for COVID-19 (GRAVID) study, which evaluated data from more
than 1,200 pregnant women at 33 hospitals across the country and found
that pregnant COVID-19 patients with severe disease are at higher risk
for cesarean delivery, postpartum hemorrhage, hypertensive disorders of
pregnancy, and preterm birth. Data from the study is being shared with
a larger registry to inform future studies of COVID-19's effects on
pregnancy and maternal health.
Tackling the challenge of reducing maternal MMM requires strong
partnerships with and among local communities and resources,
particularly with racial and ethnic minority populations that
experience stark health disparities. To that end, several NIH
Institutes, Centers, and Offices (ICOs) held community engagement
activities to hear first-hand how patient communities can inform future
research and what engagement strategies might enhance local efforts to
improve maternal health. A common refrain was that research conducted
in a community should be developed with and vetted by the community to
ensure success and improved outcomes. These engagement activities
informed the development of the IMPROVE (Implementing a Maternal health
and PRegnancy Outcomes Vision for Everyone) Initiative, which aims to
build an evidence base that will improve maternal care and outcomes
from pregnancy through 1 year postpartum. IMPROVE is co-led by NICHD
and the NIH Office of Research on Women's Health and engages over 30
ICOs to research the leading causes of maternal mortality in the United
States--cardiovascular disease, infection, and immunity--as well as
contributing health conditions or social factors, such as mental health
disorders, diabetes, obesity, substance use disorders, and structural
and healthcare system issues that disproportionately affect Black
pregnant and postpartum women. IMPROVE prioritizes comprehensive,
interdisciplinary research that engages communities with high rates of
maternal deaths and complications. This work will help create tailored,
evidence-based solutions for pregnant and postpartum women.
NIH research on MMM generates evidence that improves outcomes and
clinical care, and several NIH Institutes have strong investments in
this space. For example, an NICHD-funded study demonstrated that when
hospitals implemented evidence-based recommendations for clinical
practice there was a reduction in the risk of severe maternal morbidity
from obstetric hemorrhage, a common complication of childbirth. The
reduction was more dramatic for Black women more than for White women,
reducing disparities and improving outcomes. NICHD is also supporting a
machine learning framework to predict severe maternal morbidity.
Researchers aim to analyze population-based data from Maryland state
databases and hospital surveys to develop techniques that can predict
maternal risks early. Identifying key predictors of severe maternal
morbidity can help ascertain health disparities, strengths and
weaknesses in obstetric care, and prevent adverse maternal and neonatal
outcomes.
In fiscal year 2020, the National Institute on Minority Health and
Health Disparities (NIMHD) started an initiative entitled Addressing
Racial Disparities in Maternal Mortality. This initiative supports
multidisciplinary research projects that examine the clinical, social,
behavioral, and healthcare system interventions to address racial
disparities in MMM in the United States. Additionally, NIMHD funded the
Maternal and Developmental Risks from Environmental and Social
Stressors (MADRES) project in collaboration with the National Institute
on Environmental Health Sciences, to examine prenatal environmental
exposures and social stressors in relation to depression and
cardiovascular risk factors postpartum.
The National Heart, Lung, and Blood Institute (NHLBI) is weaving
together a network of community-engaged researchers who will not only
work to improve women's heart health and reduce maternal mortality, but
will also address other health disparities. For example, NHLBI's new
Maternal Health Community Implementation Program, will fund three or
four regional coalitions to pilot test community-based strategies in
areas where maternal death rates are high, particularly in the
southeast. Additionally, NHLBI's Early Intervention to Promote
Cardiovascular Health of Mothers and Children (ENRICH) will tap into
existing Federal home health/wellness programs that serve at-risk
families to determine if adding a cardiovascular intervention will
enhance maternal and early childhood outcomes. Approximately 3,000
mother- child pairs across various sites will be reached as part of
this effort.
These are just a few examples of how NIH's broad investment in
addressing MMM is improving maternal and infant care.
______
Questions Submitted by Senator Jeanne Shaheen
Question. I am hopeful that our continued investment in the Special
Diabetes Program, and diabetes research at NIH as a whole, can help
spur a new wave of breakthroughs, and maybe one day a cure for
diabetes.
Now that Congress has secured longer-term funding for the Special
Diabetes Program, can you please provide information on NIH's priority
areas for Special Diabetes Program research in the years to come?
Answer. The National Institutes of Health (NIH) appreciates the
recent extension of the Special Diabetes Program, which will allow us
to continue critical ongoing research programs and to support new
research to improve the health and quality of life of people with or at
risk for type 1 diabetes and its complications. For example, the recent
extension will allow the National Institute of Diabetes and Digestive
and Kidney Diseases (NIDDK) to continue the Human Islet Research
Network, which is working to better understand how insulin-producing
cells are lost in type 1 diabetes and to find strategies to replace or
protect them in people, toward curing the disease. NIDDK plans to begin
new clinical trials through the Type 1 Diabetes TrialNet network,
testing agents to prevent onset of clinical type 1 diabetes. Such
research will build on the landmark success of previous TrialNet
research demonstrating for the first time ever that early preventive
treatment can delay onset of clinical type 1 diabetes in high-risk
individuals. NIDDK also plans to support research building on the
tremendous recent progress in developing transformative diabetes
management technologies, such as artificial pancreas devices. For
example, future research is needed to improve components of artificial
pancreas devices (e.g., glucose sensors, hormone formulations), develop
simpler and more user-friendly devices, and test devices in
understudied populations (e.g., older adults, pregnant women, people
with poorly controlled blood glucose levels). This type of research
will move us closer to our goal of developing multiple different
artificial pancreas technologies for people of all ages so that they
can choose the technology best suited to their clinical needs. NIDDK
also plans to support new research to identify novel ways to detect and
monitor type 1 diabetes onset and progression, such as by determining
whether ``extracellular vesicles'' that originate from pancreatic
tissue may be useful to detect earlier stages of type 1 diabetes than
currently possible. NIDDK is collaborating with the National Heart,
Lung, and Blood Institute on new research toward reducing
cardiovascular disease in people with type 1 diabetes, as very little
is known about how best to prevent and treat this life-threatening
complication. To inform other future research directions, NIDDK is
spearheading a planning meeting in spring 2022 under the auspices of
the statutory Diabetes Mellitus Interagency Coordinating Committee to
obtain input from external scientific and lay experts on critical new
and emerging research opportunities that could be supported by the
Special Diabetes Program.
Question. New Hampshire continues to be one of the hardest-hit
states in the substance use disorder epidemic, with one of the highest
overdose death rates in the country. I am very supportive of the
ongoing work at the National Institute on Drug Abuse (NIDA) to research
potential non-addictive alternatives to opioids for pain management.
Could you discuss progress on any research within NIDA to study
these types of alternatives?
Answer. The National Institutes of Health (NIH) recognizes the need
to improve pain management without risk of addiction and other serious
side effects. NIH is taking a multi-pronged approach to develop safe
and effective therapies to reduce our reliance on opioids.
To avoid replay of the spike in opioid deaths related to over-use
of medical opioids for pain management we need more effective, non-
addictive pain medications and data that can inform best practices in
pain care. The NIH Helping to End Addiction Long-term (HEAL) Initiative
was launched in 2018 and significantly expanded research to discover
and accelerate development of non-addictive pharmacological and non-
pharmacological pain treatments. HEAL has awarded over $1.5 billion for
research to improve pain management and address opioid use disorder and
overdose. Studies supported by HEAL, the Blueprint Neurotherapeutics
Program, and multiple NIH Institutes, in particular the National
Institute for Neurological Disorders and Stroke (NINDS), are underway
to identify, optimize and test promising molecules, biologics, and
devices that target non-opioid pain pathways in the nervous system.
Biomarker studies to help with diagnosis of pain conditions and to
identify patients most likely to respond to a particular treatment will
enhance pain clinical trials and improve best practices are moving
forward. In addition, non-pharmacological approaches to manage many
different pain conditions are being evaluated through effectiveness and
implementation research approaches.
The NIH HEAL initiative established essential pain research
infrastructure to accelerate development of new medications and devices
to treat pain. An analgesic screening platform uses animal- and human
cell-based models such as neural tissue chips for rapid screening of
molecules or devices for analgesic relevant biological and pain
behavioral activity. HEAL, with input from academic and industry
partners, established an Early Phase Pain Investigation Clinical
research network (phase 2 studies) to test safety and efficacy of novel
therapeutics and a later stage pain management Effectiveness Research
Network (ERN) to compare effectiveness of pharmacological and non-
pharmacological approaches in many different pain conditions. The ERN
is supporting eight large trials for various pain management
strategies. The Pragmatic and Implementation Studies for the Management
of Pain to Reduce Opioid Prescribing (PRISM) network focuses on
clinical trials of non-pharmacologic pain therapies in healthcare
systems.
The Phase 2 network will launch trials on two new analgesics in
2021. The ERN is supporting eight large trials for various pain
management strategies. PRISM is supporting six large trials in
healthcare systems. In addition, HEAL established an analgesic
development pipeline to accelerate the development and testing of novel
drugs and devices. This program uses team-based science coupled with a
comprehensive set of research resources to bring new therapeutics
rapidly to the clinic. To advance the discovery and validation of new
drug targets, HEAL has funded over 30 projects to discover and verify a
diverse set of drug target types across multiple pain conditions, six
drug optimization studies on new safe and effective pain treatments,
and 11 projects to test the effectiveness of implanted devices and
noninvasive stimulation of nerves in the brain or throughout the body
to reduce perception of pain. This effort greatly expands on NINDS
supported studies in these areas.
Recent HEAL accomplishments toward new therapeutics include two
patent filings for small molecule modulators of pain receptors involved
in chronic pain and migraine. One ongoing study received
Investigational New Drug (IND) approval for use of buprenorphine with
nonpharmacological treatment to relieve pain in patients undergoing
kidney dialysis. Through the NIH Blueprint Neurotherapeutics Program
researchers are developing non-addictive kappa opioid receptor
antagonists for treatment of migraine and a safe, non-opioid epoxide
hydrolase inhibitor to reduce diabetic nerve pain. Earlier, NIH
supported basic science research led to calcitonin gene-related peptide
therapy for migraine and nerve growth factor therapy for inflammatory
pain. Drugs that target these molecules are now approved by the U.S.
Food and Drug Administration to treat migraine and osteoarthritis pain.
Through the Brain Research through Advancing Innovative
Neurotechnologies (BRAIN) Initiative, which is a major effort to
develop tools to map, monitor, and modulate neural circuits, NIH has
supported studies that will enhance diagnostics and therapies for
chronic pain and other neural circuit disorders.
Question. The Institutional Development Award (IDeA) program at NIH
has proven critical in funding New Hampshire researchers, including
especially the innovative work at Dartmouth College and Dartmouth-
Hitchcock Health. I am hopeful that Congress can continue to support
funding for this program.
Can you provide any insight into how NIH is currently making use of
Institutional Development Award funds and whether more funding for the
program would be helpful?
Answer. The Institutional Development Award (IDeA) supports basic,
clinical, and translational research, faculty development, and
infrastructure improvements at institutions in states and territories
that have historically received a lower aggregate level of NIH funding.
The program aims to strengthen biomedical research capacity, enhance
the competitiveness of investigators in securing research funding, and
enable clinical and translational research that addresses the specific
needs of rural and medically underserved communities. Currently,
institutions in 23 States and Puerto Rico are eligible for funding
through the IDeA Program, the various components of which include:
--IDeA Networks of Biomedical Research Excellence (INBRE). INBRE
enhances, extends, and strengthens the research capabilities of
biomedical research faculty in IDeA states through a statewide
program that links a research-intensive institution with
primarily undergraduate institutions. INBRE supports
institutional research and infrastructure development; research
by faculty, postdoctoral scientists, and students at
participating institutions; and targeted outreach to build
science and technology knowledge within a state's workforce.
Only one INBRE award is made per IDeA-eligible state. The New
Hampshire INBRE, which is led by Dartmouth and co-led by the
University of New Hampshire, is in its twelfth year of
operation and has used the program's support to improve and
expand research capacity at all eight of its partner
institutions, including adding additional labs, cores and
instrumentation/infrastructure; establishing fully functional
Office of Sponsored Programs for faculty members to
competitively seek extramural grants; training and mentoring of
both faculty and students; and enhancing a vibrant
institutional research culture. In fiscal year 2020, the
National Institute of General Medical Sciences (NIGMS)
supported 24 INBRE awards.
--Centers of Biomedical Research Excellence (COBRE--Phases I, II, and
III). COBRE supports the establishment and development of
innovative, state-of-the-art biomedical and behavioral research
centers at institutions in IDeA-eligible states that: (a)
galvanize multidisciplinary research to develop a critical mass
of investigators that are competitive for peer-reviewed
research funding; (b) provide improvements to research
infrastructure; and (c) maintain research cores to sustain a
collaborative, multidisciplinary research environment that
includes pilot project programs, mentoring, and workforce
training. In fiscal year 2020, NIGMS supported 112 COBRE
awards. One such example, a Phase I COBRE at Dartmouth's Geisel
School of Medicine called iTarget (Institute for Biomolecular
Targeting), aims to catalyze the development of new therapeutic
approaches to address cancer, chronic obstructive pulmonary
disease, and respiratory syncytial virus, a common viral
infection that can be dangerous to young children and the
elderly. This COBRE is providing unique resources to
investigators at Dartmouth and its IDeA partners, thus
enhancing research productivity and funding competitiveness
across the region.
--IDeA Networks for Clinical and Translational Research (IDeA-CTR).
IDeA-CTRs develop a network infrastructure and capacity in
IDeA-eligible states to conduct clinical and translational
research focused on health concerns that disproportionately
affect rural and medically underserved populations and/or that
are prevalent in IDeA states. IDeA-CTR awards support mentoring
and career development activities in clinical and translational
research. In fiscal year 2020, NIGMS supported 12 IDeA-CTR
awards.
--Regional Technology Transfer Accelerator Hubs. NIGMS established
the Regional Technology Transfer Accelerator Hubs for IDeA
states in each of the four IDeA regions (central, northeast,
southeast, and western regions). The hubs provide both
consulting services and skills development in entrepreneurship,
technology transfer, small business finance, and other areas
needed to transform important discoveries made in the
laboratory into potentially viable commercial products that
address human health. In fiscal year 2020, NIGMS supported four
accelerator hubs. The northeast hub is located at Celdara
Medical in Lebanon, New Hampshire.
--Research Co-Funding. NIGMS provides co-funding for applications
from IDeA state institutions that have been judged meritorious
by NIH peer-review committees and national advisory councils
but that may also fall outside the usual range of support by a
given NIH Institute or Center (IC). In fiscal year 2020, NIGMS
co-funded 42 research project grants at 20 NIH ICs; one of
these was at Dartmouth College.
______
Questions Submitted to Dr. Ned Sharpless
Questions Submitted by Senator Patty Murray
Question. The American Cancer Society's Annual Report to the Nation
on the Status of Cancer highlighted that we are making good progress in
the battle against cancer, with the incidence and mortality rates for
most cancers have dropped significantly. However, among the 20 most
common cancers, relative survival for patients significantly improved
since the mid-1970s except for those with uterine cancer.
What plans does the NCI have in fiscal year 2022 to develop a
paradigm of increased research to improve hope for survival for
patients with uterine cancer?
Answer. The National Cancer Institute (NCI) shares the committee's
commitment to research on uterine cancers, including endometrial cancer
(cancer of the inner lining of the uterus), and improving outcomes for
patients.
Today, nearly 40 percent of adults are obese, and without
intervention, the obesity epidemic will result in more cancers. Uterine
cancer incidence and mortality have increased in recent years,\40\
believed to be partially associated with rising rates of obesity.\41\
Women who are obese or overweight are approximately two to four times
as likely as normal weight women to develop uterine cancer, including
endometrial cancer, making interventions to address weight and obesity
vital to combatting uterine cancer incidence and mortality. Examples of
NCI-supported research on this topic include a study of how changes in
body composition following weight loss impact inflammatory biomarkers
in biopsy-collected endometrial tissue and blood samples and whether
these processes differ between Black and White women; \42\ the
development of a weight loss intervention among Appalachian residents;
\43\ and a study of the Deep South Interactive Voice Response (IVR)-
supported Active Lifestyle (DIAL) Intervention to increase physical
activity levels among residents of the Deep South.\44\
---------------------------------------------------------------------------
\40\ pubmed.ncbi.nlm.nih.gov/30521505/,seer.cancer.gov/
report_to_nation/statistics.html#
factors.
\41\ www.cancer.gov/about-cancer/causes-prevention/risk/obesity/
obesity-fact-sheet.
\42\ reporter.nih.gov/project-details/10129305.
\43\ reporter.nih.gov/project-details/10065366.
\44\ reporter.nih.gov/project-details/10163139.
---------------------------------------------------------------------------
Researchers at the University of North Carolina Lineberger
Comprehensive Cancer Center are directly examining the metabolic and
molecular differences of endometrial tumors in obese and non-obese
women. In addition, this research team is exploring how metformin,
widely used to treat type II diabetes, may also exhibit anti-tumor
activity through its effects on a patient's metabolism.\45\
---------------------------------------------------------------------------
\45\ reporter.nih.gov/project-details/10104456.
---------------------------------------------------------------------------
Translational research to bridge the gap between basic research on
endometrial cancer and potential therapies is also essential to
improving outcomes for patients. NCI supports a Specialized Program of
Research Excellence (SPORE) focused on translational research for
endometrial cancer at the University of Texas/MD Anderson Cancer
Center. This SPORE is conducting research aimed at developing
therapeutic strategies for advanced/recurrent endometrial cancer and
aggressive subtypes, addressing unmet clinical needs in prevention and
conservative therapy of high-risk precancerous lesions and low-grade
endometrial cancer, and incorporating molecular diagnostics into
clinical decisionmaking.\46\
---------------------------------------------------------------------------
\46\ trp.cancer.gov/spores/endometrial.htm.
---------------------------------------------------------------------------
As of July 2021, NCI is supporting over 150 clinical trials with a
primary focus on uterine (including endometrial) cancer. Examples of
these projects include studies of the use of an immunotherapy agent, in
combination with other cancer therapies, to treat high risk endometrial
cancer; \47,48\ a trial examining a combination therapy to treat
endometrial cancers that express the HER2 protein; \49\ and a study
evaluating the use of the experimental therapy triapine to treat
endometrial serous adenocarcinoma, a difficult to treat subtype of
uterine cancer.\50\ Clinical trials are an integral part of advancing
research in this important topic area, and NCI is committed to reaching
out to disparate, at-risk communities to explain, educate, and
encourage clinical trial participation.
---------------------------------------------------------------------------
\47\ clinicaltrials.gov/ct2/show/NCT04214067.
\48\ clinicaltrials.gov/ct2/show/NCT03914612.
\49\ clinicaltrials.gov/ct2/show/NCT04585958.
\50\ clinicaltrials.gov/ct2/show/NCT04494113.
---------------------------------------------------------------------------
As part of the National Institutes of Health (NIH) efforts to
identify future research directions, NCI and the Eunice Kennedy Shriver
National Institute of Child Health and Human Development (NICHD)
explored research opportunities into the progression of benign
gynecologic conditions to cancers through a collaborative workshop in
April 2019. Currently, NICHD funds research on benign gynecologic
conditions such as endometriosis and uterine fibroids, while NCI funds
research on women's cancers. The workshop sought to bridge the two
research areas and identify gaps in the biologic, epidemiologic, and
clinical understanding of progression from benign conditions to cancer.
The workshop addressed three gynecologic disease types: (1)
endometriosis or endometrial cancer and endometrial-associated ovarian
cancer, (2) uterine fibroids (leiomyoma) or leiomyosarcoma, and (3)
denomyosis or adenocarcinoma. Working groups were formed for each
disease type, and key questions and current challenges that emerged
from the discussions, along with potential research opportunities to
advance understanding of progression of gynecologic benign conditions
to cancer, were published. Specific research questions and gaps were
identified in all three focus areas, and several cross-cutting topics
emerged. The results of this workshop, as well as ongoing horizon-
scanning activities, will continue to inform NIH's next steps to
address uterine cancer.
Question. Non-Hispanic Black women are two time as likely as non-
Hispanic White women to die from uterine or cervical cancer (https://
www.ajog.org/article/S0002-9378(16)46212- 5/pdf).
Can NIH/NCI please share with the Committee the research activities
the NCI is supporting to address this disparity, particularly with
regards to access to care, prevention, early diagnosis, treatment
completion and developmental therapeutics?
Answer. The National Cancer Institute (NCI) shares the Committee's
concern regarding cervical and uterine/endometrial cancer disparities
and is working to support research to eliminate these disparities, as
well as cancer disparities more broadly. Examples of research aimed at
addressing disparities in uterine and cervical cancer outcomes are
provided below.
NCI is a leader in developing and supporting definitive, practice-
changing gynecologic (GYN) clinical trials, as well as responding to
areas of scientific inquiry that are unaddressed by private industry.
The NCI GYN Cancers Steering Committee sets clinical trials strategic
priorities that address areas of unmet clinical need, important
unanswered clinical questions, and potential new approaches to disease
treatment.\51\ The Institute has supported and advanced GYN cancer
research that will provide greater insight into these cancers,
additional options for drug therapies, and improved surgical techniques
with the intent of increasing survivorship and quality of life. As of
July 2021, NCI is supporting over 150 interventional clinical trials
with a primary focus on uterine (including endometrial) cancer, two
trials on the rare uterine sarcoma, and nearly 100 trials for cervical
cancer patients. NCI also has several trials that are ``disease
agnostic,'' meaning that they are open to patients with certain genetic
alterations rather than traditional cancer types, creating
opportunities for patients to potentially benefit from precision
medicine and targeted therapy.
---------------------------------------------------------------------------
\51\ www.cancer.gov/about-nci/organization/ccct/steering-
committees/nctn/gynecologic.
---------------------------------------------------------------------------
A recent study led by NCI intramural researchers used population
data from NCI's Surveillance, Epidemiology, and End Results (SEER)
database to evaluate trends of hysterectomy-corrected uterine cancer
incidence rates for women overall and by race and ethnicity, geographic
region, and histologic subtype. Correct estimation of these rates
requires accounting for hysterectomy prevalence, which varies by race,
ethnicity, and region. The researchers found that incidence rates of
common subtypes of uterine cancer were stable in non-Hispanic White
women over the study period and increased in women of other racial/
ethnic groups. By contrast, incidence rates of aggressive subtypes have
been increasing dramatically over time in all racial/ethnic groups; in
particular, much higher rates of these aggressive subtypes were
observed in Black women than in other racial/ethnic groups. The
researchers also observed that survival rates were lower among all
women with aggressive subtypes than among women with common subtypes,
and Black women had the lowest survival rates within each stage at
diagnosis or histologic subtype.
Uterine serous carcinoma (USC) is a rare but aggressive type of
endometrial cancer. In about one-third of women with USC, their tumor
cells overproduce a protein called HER2 (human epidermal growth factor
receptor 2), which is associated with poor prognosis in women with
endometrial cancer. Black women with endometrial cancer are more likely
than White women to be diagnosed with UCS and are more likely than
women of other races/ethnicities to have HER2 overproducing UCS tumors.
NCI clinical studies for patients with HER2 overproducing uterine
serous cancer and carcinosarcoma are currently in development.
NCI-supported researchers are working to describe additional
differences in subtypes of uterine and endometrial cancers, with the
eventual goal of targeting therapies to treat each disease subtype. For
example, investigators at Brigham and Women's Hospital, using data from
the NCI-supported Epidemiology of Endometrial Cancer Consortium
(E2C2),\52\ are studying genomic variation across the full spectrum of
endometrial tumors, distinct risk factor profiles across tumor types,
and the role of underlying tumor biology to better understand the
disparities in outcomes between African-American and non-African-
American women.\53\ NCI-supported investigators at Wayne State
University are examining aggressive subtypes of high-grade endometrial
tumors, including endometrioid, serous, clear cell and mixed
carcinomas, by analyzing both clinical and genetic data in 500 women
(250 African-American, 250 White) diagnosed with these cancers.\54\ In
addition, NCI is supporting a planning grant to establish a Specialized
Program of Research Excellence (SPORE) at Northwestern University
focused on gynecologic cancer disparities. One of the pilot projects
will focus on the tumor genomics of endometrial cancer.\55\
---------------------------------------------------------------------------
\52\ epi.grants.cancer.gov/eecc/.
\53\ reporter.nih.gov/search/o5KPkwNzZUavBogOfHXfCgproject-details/
10156374.
\54\ reporter.nih.gov/search/frdhnx_EQkONjxE8GPyxvQ/project-
details/9916725.
\55\ reporter.nih.gov/search/-UP_KUgEu0G9_0Zt655Nsg/project-
details/9961257.
---------------------------------------------------------------------------
To more accurately evaluate the risk of cervical precancer and
study novel biomarkers in women undergoing cervical cancer screening,
intramural researchers in NCI's Division of Cancer Epidemiology and
Genetics have partnered with the University of Mississippi Medical
Center and the Mississippi State Department of Health in the STRIDES
Study (Studying Risks to Improve Disparities of cervical cancer in
Mississippi). This study, based in one of the top five states for
cervical cancer incidence and mortality, combines the expertise of
clinicians, laboratory scientists, epidemiologists, and implementation
scientists to address all aspects of cervical cancer prevention and
control.\56\
---------------------------------------------------------------------------
\56\ dceg.cancer.gov/research/cancer-types/cervix/cervix-
mississippi.
---------------------------------------------------------------------------
In 2020, NCI launched the ``Last Mile Initiative,'' with the goal
of improving cervical cancer screening coverage to underserved, never
screened, and under-screened women. This initiative will evaluate an
alternative cervical cancer screening approach: self-collection of
samples (self- sampling) by women, which are then sent to labs for
human papillomavirus (HPV) testing. This approach aims to identify
cervical cancer cases in these groups of women, which account for over
half of cervical cancer cases in the United States each year. Self-
sampling offers several benefits, including ease of collection at the
time and place of the patient's choosing, without the need for a clinic
appointment or speculum exam. To conduct this assessment, NCI
established a public-private partnership between Federal agencies,
industry partners, and professional societies/clinical guidelines
organizations, and will support a nationwide, multicentric screening
trial in diverse settings, the Last Mile Initiative Self-sampling for
HPV Testing to Improve Cervical Cancer Prevention Trial (LMI-SHIP
Trial).\57\
---------------------------------------------------------------------------
\57\ prevention.cancer.gov/major-programs/nci-cervical-cancer-last-
mile-initiative.
---------------------------------------------------------------------------
Additionally, NCI is collaborating with the NIH Office of Research
on Women's Health (ORWH) and other NIH Institutes and Centers to
participate in an ORWH Advisory Committee on Research on Women's Health
Consensus Conference to be held in October 2021. The conference will
include a focus on cervical cancer disparities and research
opportunities to continue to address disparities in incidence and
mortality.
NCI will continue to identify opportunities to better understand
and address cancer health disparities, including for cervical and
uterine/endometrial cancers.
______
Questions Submitted by Senator Richard J. Durbin
Question. Approximately 20,000 people in the United States have
germline mutations in the gene RUNX1. Patients with RUNX1-familial
platelet disorder are at a heightened risk for developing blood
cancers. NCI supports a longitudinal natural history study of patients
with such germline mutations and their families. While germline RUNX1
mutations are rare, I understand that NIH-funded research in this area
holds promise for the fields of hematology and oncology.
How can deepening our understanding of, and ultimately developing
cancer prevention strategies for, inherited blood cancer predisposition
syndromes like RUNX1 familiar platelet disorder advance the entire
cancer research field forward?
Answer. The RUNX1 gene regulates the development of blood cells
(hematopoiesis), controlling other genes that help determine the fate
of hematopoietic stem cells, which have the potential to develop into
all types of mature blood cells, including platelets. Platelets are
cells that help blood to clot. Inherited mutations in the RUNX1 gene
cause familial platelet disorder with associated myeloid malignancies
(RUNX1-FPDMM) and predispose individuals to some types of blood
cancers. Although genetic predisposition to solid tumors such as breast
and colon cancers has been widely recognized over the past several
decades, the contribution of inherited genetic disorders related to
blood cancer is a more recent field of study.
There are many instances where understanding the molecular basis
for a rare inherited disease has provided insight into more common
forms of a particular disease. For example, BRCA1 and BRCA2 mutations
were discovered as hereditary breast cancer genes but are also relevant
to sporadic (non-hereditary) breast cancers, ovarian cancers, and some
hereditary forms of colon cancer. Similarly, understanding the blood
cancers associated with RUNX1-FPDMM may lead to improved understanding
of other types of blood cancers as well.
Research efforts across the National Institutes of Health (NIH) are
underway to better understand RUNX1-FPDMM. Investigators funded by the
National Heart, Lung, and Blood Institute (NHLBI) are studying cells
from people with this disorder to better understand key target genes
regulated by RUNX1 and their role in hematopoiesis.\58\ This work could
also yield a better understanding of genetic pathways that lead to
blood cancers, as well as the blood clotting mechanisms that contribute
to cardiovascular disease. Investigators at the National Human Genome
Research Institute (NHGRI), along with intramural scientists at the
National Cancer Institute (NCI), are conducting a natural history study
at the NIH Clinical Center that is intended to identify and follow
patients with RUNX1 mutations to hopefully identify biomarkers that can
predict which patients will develop cancers.\59\ To date, the study has
enrolled 198 patients from 55 families, representing the largest FPDMM
cohort being followed prospectively at a single institution in the
world.
---------------------------------------------------------------------------
\58\ reporter.nih.gov/project-details/10083753.
\59\ www.genome.gov/Current-NHGRI-Clinical-Studies/hematologic-and-
premalignant-conditions-associated-with-RUNX1-
mutation;clinicalstudies.info.nih.gov/ProtocolDetails.
aspx?id=2019-HG-0059; clinicaltrials.gov/ct2/show/NCT03854318.
---------------------------------------------------------------------------
Studying RUNX1-FPDMM will have broader significance than just this
rare disease. Germline (inherited) predisposition to hematopoietic
malignancies is often under-diagnosed, with recent studies indicating
that 10-30 percent of RUNX1 mutations detected in acute myeloid
leukemias are inherited, which is much more common than previously
appreciated.\60\ In addition, FPDMM can serve as a model to study the
development of leukemia, since researchers can monitor individuals with
the RUNX1 mutation before they develop leukemia to identify factors
associated with cancer risk and to map tumor evolution.
---------------------------------------------------------------------------
\60\ pubmed.ncbi.nlm.nih.gov/32315381/.
---------------------------------------------------------------------------
______
Questions Submitted by Senator Jack Reed
Question. The fiscal year 2021 Appropriations law included full
funding--$30 million--for the Childhood Cancer STAR Act, which I
authored.
Could you provide an update on how that funding will be spent in
the coming year?
How will that work be coordinated with the childhood cancer data
initiative?
Answer. NCI is supporting several new and ongoing Childhood Cancer
STAR Act research projects in fiscal year 2021, for a total planned
investment of $28 million. The Centers for Disease Control and
Prevention continues to support enhancements to expand capacity within
the National Program of Cancer Registries (NPCR) to help cancer
registries collect and make the data on pediatric cancer cases
available more rapidly, a $2 million effort in fiscal year 2021.
Consistent with provisions in Section 101 of the STAR Act, NCI's
fiscal year 2021 appropriation for STAR Act activities is supporting
new and expanded projects focused on the collection and storage of
biospecimens for future research. Several projects are conducted
through the NCI-supported Children's Oncology Group (COG) to focus
additional attention to rare cancer subtypes that are currently
underrepresented in NCI-supported biorepositories, as well as tumor
types with a high risk of treatment failure. For example, particularly
rare subtypes of pediatric cancers for which COG does not have open
clinical trials, tumor tissue collection options are limited. STAR Act
appropriations are supporting the COG Rare and Under-Represented Cancer
Tissue Banking project to enable tumor tissue and associated germline
(e.g., blood) sample collection for specific groups of patients for
which current tumor tissue collection is lacking or inadequate, with
priority for tumor types such as sarcomas and brain and central nervous
system (CNS) tumors, which have high risk of treatment failure.
The COG Rare and Under-Represented Cancer Tissue Banking project
was launched in fiscal year 2020 and is expanding in scope in fiscal
year 2021. This initiative is collaborating closely with CCDI, and with
the use of fiscal year 2021 CCDI funds, tumor tissue will undergo
clinically-relevant molecular profiling through the CCDI Molecular
Characterization Protocol. The data generated will be returned to
treating physicians to help guide the diagnosis and treatment of
patients, and the data will additionally be stored and made available
to the research community through CCDI data platforms. In addition to
rare cancer populations, the CCDI Molecular Characterization Protocol
will initially support characterization of tumors from children with
CNS tumors and from children with soft tissue sarcomas. The Protocol
aims to collect, store, and make available detailed clinical and
molecular information for each child participating in the study,
including data that will help a pediatric oncologist treat that patient
and help researchers learn more about childhood cancers.
NCI is continuing support in fiscal year 2021 for other STAR Act
biobanking projects launched in fiscal year 2020. Through the COG Rapid
Autopsy Specimen Collection project, NCI and COG are working with
patient organizations to support rapid autopsy collection of tumor
samples from children and adolescents and young adults (AYAs) who have
died of their disease. Foundations and families within the pediatric
brain tumor community have been leaders in such programs, and NCI
continues to learn from their experiences to expand this model to other
childhood cancers. We are incredibly grateful to these parents and
caregivers, who amidst unimaginable grief and loss, contribute to
future research to advance science and help other families.
NCI is also supporting the COG to continue to expand the collection
of specimens taken at the time of relapse, as well as collecting
diagnostic samples for children and AYAs who have already submitted
samples at relapse through NCI's Pediatric Molecular Analysis for
Therapy and Choice (MATCH) Precision Medicine Trial. An important
impediment to understanding mechanisms of treatment failure for
childhood solid tumors is the limited numbers of paired specimens from
both diagnosis and relapse that are available for researchers to study.
Specimens at relapse are critical for evaluating biological changes
between diagnosis and relapse that can lead to the identification of
mechanisms of treatment failure and to the development of strategies
for circumventing these mechanisms. Through CCDI, Pediatric MATCH tumor
specimens from diagnosis and from relapse are being molecularly
characterized to identify the changes in gene mutations and gene
expression that occur between diagnosis and relapse, which could inform
better treatments.
Consistent with Section 202 of the STAR Act, in fiscal year 2021,
NCI will continue to conduct and support childhood cancer survivorship
research. NCI has supported two new Requests for Applications (RFAs)
since fiscal year 2019 that are directly aligned with survivorship
research areas emphasized in the STAR Act. Issued in fiscal year 2019,
RFA CA-19-033: \61\ Improving Outcomes for Pediatric, Adolescent and
Young Adult Cancer Survivors focused on projects to develop and test
interventions that prevent, mitigate or manage adverse outcomes in
pediatric and/or AYA cancer survivors and/or evaluate models of care
that strengthen coordination, continuity, and quality, or that reduce
access barriers to needed services including follow-up care, and that
improve outcomes across the survivor's lifespan. Development of
interventions to address disparities in outcomes and/or access to
needed care, and to address the needs of minority or medically
underserved pediatric and/or AYA populations were also prioritized. NCI
is supporting seven awards in response to this RFA, and the awards will
focus on various patient sub-populations (e.g. disease site),
developmental groups, specific late and long-term effects, and the
types of interventions (both preventive and supportive care).
---------------------------------------------------------------------------
\61\ grants.nih.gov/grants/guide/rfa-files/RFA-ca-19-033.html.
---------------------------------------------------------------------------
Issued in fiscal year 2020, RFA CA-20-027 \62\ and RFA CA-20-028:
\63\ Research to Reduce Morbidity and Improve Care for Pediatric, and
Adolescent and Young Adult (AYA) Cancer Survivors invite applications
for research projects to improve care and health-related quality of
life for childhood and AYA cancer survivors, with a focus on six key
domains that align with research priorities emphasized in the STAR Act:
(1) disparities in survivor outcomes; (2) barriers to follow-up care
(e.g. access, adherence); (3) impact of familial, socioeconomic, and
other environmental factors on survivor outcomes; (4) indicators for
long-term follow-up needs related to risk for late effects, recurrence,
and subsequent cancers; (5) risk factors and predictors of late/long-
term effects of cancer treatment; and (6) development of targeted
interventions to reduce the burden of cancer for pediatric/AYA
survivors.
---------------------------------------------------------------------------
\62\ grants.nih.gov/grants/guide/rfa-files/RFA-CA-20-027.html.
\63\ grants.nih.gov/grants/guide/rfa-files/rfa-ca-20-028.html.
---------------------------------------------------------------------------
In fiscal year 2021, NCI will support subsequent years for grants
initially awarded in fiscal year 2019 and fiscal year 2020, as awards
were made for five-year terms, and the Institute will be making several
new grant awards through the RFA launched in fiscal year 2020. The
first round of applications is in the final stages of review, and
awards will be made before the close of fiscal year 2021. The second
round of applications are due on July 30, 2021, and awards are
anticipated to be made in fiscal year 2022.
NCI also continues to make additional investments in childhood
cancer survivorship research beyond the STAR Act appropriation, funding
several notable initiatives and projects with resources provided
through the Institute's general appropriation. For example, NCI
continues to fund long-standing investments in the Childhood Cancer
Survivor Study (CCSS),\64\ which the Institute has supported
continuously since establishing CCSS in 1994. This cohort of more than
38,000 childhood cancer survivors diagnosed between 1970 and 1999 (and
5,000 siblings of survivors who serve as the comparison group for the
study) serves as a foundational resource for the survivorship research
community.
---------------------------------------------------------------------------
\64\ cancer.gov/types/childhood-cancers/ccss.
---------------------------------------------------------------------------
Additionally, NCI continues to support research projects that
investigators develop and submit independent of specific childhood and
AYA cancer survivorship funding opportunities such as the STAR Act RFAs
described above. These investigator-initiated research projects provide
critical contributions to this field, and awards made to date in fiscal
year 2021 include a project to compare symptom burdens (toxicity),
neurocognitive change, and functional outcomes in children with
pediatric brain tumors treated with proton versus photon radiotherapy.
Proton beam radiotherapy (PBRT) is often thought to be a promising
treatment for children with brain tumors as it may preserve cognitive
functioning without sacrificing disease control. This will be the first
large-scale study to prospectively compare the two therapies to assess
important measures of daily functioning that will quantify the clinical
significance of any differences identified between groups in
survivorship. This project aims to help physicians and families better
understand the relative effect of PBRT on symptoms and neurocognitive
functioning to inform treatment decisions.\65\ Another award is
supporting further study of psychosocial risk in young survivors of
pediatric cancer diagnosed in early childhood, including the role of
both physical and neurocognitive late effects. This project aims to
identify specific medical and neurocognitive late effects that increase
psychosocial morbidity, as well as protective factors, to inform more
effective interventions to optimize quality of life in children
affected by cancers diagnosed in early childhood.\66\ In addition, the
NCI-supported ASPIRES (Activating cancer Survivors and their Primary
care providers to Increase coloREctal cancer Screening) study aims to
prevent the development of subsequent cancers among childhood cancer
survivors treated with abdominal or pelvic radiotherapy, who are almost
four times more likely to develop colorectal cancer (CRC) compared to
the general population. The study will test a remote intervention aimed
at promoting early CRC screening and detection.\67\
---------------------------------------------------------------------------
\65\ reporter.nih.gov/search/kPIDddsyREmcoShhVEYN4Q/project-
details/10146799.
\66\ reporter.nih.gov/search/5Nb7PgFn7kyHJnjYOFzMQA/project-
details/10122486.
\67\ reporter.nih.gov/search/5Nb7PgFn7kyHJnjYOFzMQA/project-
details/10096080.
---------------------------------------------------------------------------
NCI remains committed to implementing the research sections of the
STAR Act directed toward the Institute, and to ensuring that these
efforts continue to complement the Institute's broader portfolio of
childhood and AYA cancer research. This includes CCDI, the COG, the
CCSS, and many other research programs and projects working together to
support much needed progress for children with cancer and their
families, including survivors and caregivers facing the challenges of
managing the late effects of cancer and its treatments.
______
Questions Submitted by Senator Roy Blunt
Question. Dr. Sharpless, one of the goals I had when I was Chairman
of this Subcommittee was to increase NIH funding, in an effort to
increase the success rates of grants--meaning more research grants
would be funded. This is important because the NIH peer review system
does not always reward high-risk science or young researchers' grant
applications. But, if you have additional funding, you can fund more
than just the `safest' science grants from the most established
researchers. NCI has seen an increase of more than 50 percent in the
number of grant applications since 2013, keeping your success rates and
paylines lower than most NIH Institutes. While the positive aspect of
this statistic is that the cancer research community is energized and
applying for NCI funding, you can only fund a certain amount of
applications because of the significant increase in grant applications.
The last two LHHS bills have included specific funding for NCI to
increase their Research Project Grants.
How has this allowed you to increase success rates, raise the
payline, and make more awards?
Answer. The intense competition and demand for NCI funding reflects
incredible scientific opportunities in cancer research and presents a
major challenge for the NCI to carefully balance increasing demand for
competing grant funding while sustaining previous years' commitments to
multi-year grants.
Investigator-initiated research has proven itself to be one of the
biggest drivers of progress in cancer research, and accordingly is the
biggest driver of NCI's budget, with long-term investments into funding
new and continuing awards constituting more than 40 percent of NCI's
annual budget. These awards have been the source of some of the most
innovative and transformative ideas in cancer research, leading to
direct benefits for patients in the form of new oncology drug
approvals, the development of immune checkpoint inhibitor therapy
(Nobel Laureate Jim Allison), CAR-T (chimeric antigen receptor-T) cell
immunotherapy (Carl June), and novel drug design strategies such as
PROTACs (proteolysis targeting chimeras) \68\ that use normal cellular
processes to identify and destroy proteins in cancer cells that drive
cancer growth (Raymond DeShais and Craig Crews).
---------------------------------------------------------------------------
\68\ www.cancer.gov/research/annual-plan/scientific-topics/protac-
infographic.
---------------------------------------------------------------------------
Considering all funding mechanisms, NCI supported 109 additional
awards in fiscal year 2020 as compared to fiscal year 2019 (from 6,053
in fiscal year 2019 \69\ to 6,162 in fiscal year 2020 \70\). Across
fiscal year 2020 and 2021, the successive funding increases allowed NCI
to increase the R01 payline from the 8th percentile in fiscal year 2019
to the 11th percentile in fiscal year 2021. With the fiscal year 2020
budget increase, NCI increased R01 paylines by 25 percent compared to
fiscal year 2019 and restored continuing grants to 100 percent of their
committed level, providing researchers the full fiscal year 2020 budget
approved during the initial grant award. Funding increases in fiscal
year 2021 allowed NCI to further raise the payline for R01 research
awards, for an overall 35 percent increase compared to 2019, as well as
to keep funding continuing awards at 100 percent. In addition, for
those two consecutive years (fiscal year 2020 and fiscal year 2021),
NCI also raised the payline for Early-Stage Investigators, reflecting
NCI's commitment to developing and supporting early career scientists
to build the next generation of cancer researchers.
---------------------------------------------------------------------------
\69\ www.cancer.gov/about-nci/budget/congressional-justification/
fy2021-nci-congressional-justification.pdf.
\70\ www.cancer.gov/about-nci/budget/congressional-justification/
fy2022-nci-congressional-justification.pdf.
---------------------------------------------------------------------------
We have the final success rate and total number of awards results
for fiscal year 2020, the year when Congress targeted an additional
$212.5 million for new and continuing grants, but we will not have
final results for fiscal year 2021 until after the first quarter of
fiscal year 2022. Our fiscal year 2020 results show that NCI increased
the number of competing R01s we issued within the payline by more than
100 awards, a jump of more than 15 percent from the prior year. The
funding increase also allowed us to pay other meritorious R01
applications that scored just outside the payline. Overall, our success
rate for fiscal year 2020 rose to 12.7 percent, from 11.6 percent in
the prior year.
The targeted increases that Congress has provided allows NCI to
increase paylines, achieve a corresponding increase in the overall NCI
application success rate, and issue more grant awards. This funding has
been critical to awarding new grants, while also allowing NCI to
support ongoing research and the breadth of core NCI research
investments, such as NCI's designated cancer centers, Specialized
Programs of Research Excellence (SPOREs), and large national networks
of clinical trials. All of these awards and programs will continue to
fuel broad, sustained progress that serves the needs of individuals
with cancer and those at risk of cancer, leading to a deeper
understanding of the biology of cancer and new strategies to prevent,
screen, diagnose, and treat cancer, in all its forms.
______
Questions Submitted by Senator Shelley Moore Capito
Question. The NCI is doing tremendous work in implementing the new
Childhood Cancer Data Initiative, which holds the promise of vastly
improving the treatment of childhood cancer and the quality of life for
survivors. The Childhood Cancer STAR Act calls for a major investment
in biorepository and bio-specimen collection.
Can you tell us how these two vital initiatives are working
together? NIH Response:
Answer. The National Cancer Institute (NCI) agrees that it is vital
for biospecimen collection and storage efforts supported through the
STAR Act and data generation, analysis, and sharing supported through
Childhood Cancer Data Initiative (CCDI) to continue to contribute to
and enhance each initiative's progress in a complementary manner. To
that end, NCI is utilizing STAR Act appropriations to support the
Children's Oncology Group (COG) Rare Tumor Populations Biobanking
project, which enables tumor tissue and germline (e.g., blood)
collection for specific groups of patients for which current tumor
tissue collection is lacking or inadequate, with priority for tumor
types such as sarcomas and brain and central nervous system tumors,
which often have the highest risk of treatment failure.
The COG Rare Tumor Populations Biobank was launched in fiscal year
2020 and is expanding in scope in fiscal year 2021. This initiative is
collaborating closely with CCDI, and with the use of fiscal year 2021
CCDI funds, tumor tissue will undergo clinically-relevant molecular
profiling through the CCDI Molecular Characterization Protocol. The COG
Rare Tumor Populations Biobank provides a critical foundation for these
characterization efforts within CCDI. The data generated will be
returned to treating physicians to help guide the diagnosis and
treatment of patients, and the data will be stored and made available
to the research community through CCDI data platforms. In addition to
rare cancer populations, the CCDI Molecular Characterization Protocol
will initially support characterization of tumors from children with
Central Nervous System (CNS) tumors and from children with soft tissue
sarcomas. The Protocol aims to collect, store, and make available
detailed clinical and molecular information for each child
participating in the study, including data that will help a pediatric
oncologist treat that patient and help researchers learn more about
childhood cancers.
NCI is also supporting a STAR Act biobanking project through the
COG to continue to expand the collection of specimens taken at the time
of relapse, as well as collecting diagnostic samples for children and
adolescents and young adults (AYAs) who have already submitted samples
at relapse through NCI's Pediatric Molecular Analysis for Therapy and
Choice (MATCH) Precision Medicine Trial. An important impediment to
understanding mechanisms of treatment failure for childhood solid
tumors is the limited numbers of paired specimens from both diagnosis
and relapse that are available for researchers to study. Specimens at
relapse are critical for evaluating biological changes between
diagnosis and relapse that can lead to the identification of mechanisms
of treatment failure and to the development of strategies for
circumventing these mechanisms. Through CCDI, Pediatric MATCH tumor
specimens from diagnosis and from relapse are being molecularly
characterized to identify the changes in gene mutations and gene
expression that occur between diagnosis and relapse, which could inform
better treatments.
These are specific examples of early and ongoing collaboration
between STAR Act and CCDI- supported projects, and more broadly, there
will be additional opportunities for data generated through STAR Act
specimen collection and survivorship research efforts to contribute to
the CCDI data ecosystem. For example, other STAR Act biobanking
projects have supported additional biospecimen collection within the
NCI-supported Childhood Cancer Survivor Study (CCSS), focused on
subsequent cancers and chronic health conditions. CCDI funds were used
to molecularly characterize specimens from patients who developed
second cancers to enhance understanding of the genetic factors that
lead to increased risk of second malignant tumors. Additionally, CCDI
funds have supported submission and management of CCSS data to NCI and
other NIH repositories so that they can be linked within the CCDI data
ecosystem and more easily shared with the broader research community.
As NCI's CCDI continues to link data resources across the childhood
cancer research field, we envision these linkages and the data
ecosystem they create serving as a resource for continued research, and
as a growing repository for all types of data generated through NCI and
other funded childhood and AYA cancer research. Similar to the CCSS,
individual research projects, including preclinical studies and
clinical trials, will have the opportunity to contribute data to CCDI,
linking this additional data to CCDI resources such as the Molecular
Characterization Protocol and the National Childhood Cancer Registry,
two foundational CCDI initiatives.
______
Questions Submitted by Senator Cindy Hyde-Smith
Question. I, along with many members of the committee remain
concerned with the lack of targeted therapies for rare cancer patients.
It is my understanding that rare cancers account for 380 of 400
distinct forms of cancer and almost 1/3 of all diagnoses and include
all pediatric cancers. A recent analysis showed that 80 percent of all
patients who lacked an FDA-targeted therapy were rare cancer patients.
In addition, of the 3,994 clinical trials in phases 1, 2, and 3 from
January 1, 2012 to January 1, 2017, almost 75 percent did not include a
rare cancer by name. While rare cancer affects every population,
translational research and commercial drug development has
traditionally neglected small patient populations. Each subtype of
cancer requires a targeted therapy in order to save a life or to
significantly improve lifespan.
What is NIH's plan to ensure there are adequate investments for
treatments for rare cancer patients and what can Congress and this
committee do to help?
Answer. The National Institutes of Health (NIH) remains committed
to supporting research to advance the understanding of all cancers,
including rare cancers, and to inform the development of targeted
cancer therapies for rare cancers and rare subtypes of cancers,
including pediatric cancers (all types and subtypes of pediatric
cancers are considered ``rare'' by definition).
The cancer research community--thanks to NIH-supported developments
in understanding the specific genes, proteins, and other unique
molecular characteristics driving certain cancer subtypes--continues to
recognize that cancer is made up of a collection of hundreds, if not
thousands, of subtypes defined by these characteristics. As a result of
National Cancer Institute (NCI)-supported efforts and other relevant
research, ``cancer'' is increasingly becoming a collection of rare
cancer subtypes.
This evolved understanding of cancer is reflected in NCI's current
clinical trials portfolio and investments in translational and basic
research, including several initiatives in the intramural Center for
Cancer Research (CCR).
Increasingly, clinical trials are examining targeted therapies
based on molecular subtypes. For example, NCI's National Clinical
Trials Network (NCTN) is currently supporting trials assessing
therapies to treat gliomas with certain genetic alterations \71\ and
pancreatic cancers with specific gene alterations.\72,73\ NCI also
supports trials that are dedicated to patients with rare tumors,
including the NCTN-supported Dual Anti-CTLA-4 and Anti-PD1-Blockade in
Rare Tumors (DART) Trial \74\ and the Rapid Analysis and Response
Evaluation of Combination Anti-Neoplastic Agents in Rare Tumors (RARE
CANCER) Trial,\75\ which is supported by NCI's Experimental
Therapeutics Clinical Trials Network.
---------------------------------------------------------------------------
\71\ www.clinicaltrials.gov/ct2/show/NCT00887146.
\72\ www.clinicaltrials.gov/ct2/show/NCT04858334.
\73\ www.clinicaltrials.gov/ct2/show/NCT04548752.
\74\ www.clinicaltrials.gov/ct2/show/NCT02834013.
\75\ www.clinicaltrials.gov/ct2/show/NCT04449549.
---------------------------------------------------------------------------
To ensure that researchers have a strong pipeline of therapy
candidates to consider for use in clinical trials, NCI supports several
initiatives to support the preclinical stage of development of
therapeutics to treat rare cancers, including the NCI Experimental
Therapeutics (NeXT) Program and the Pediatric Preclinical Testing
Consortium (PPTC). The mission of NeXT is to advance clinical practice
and bring improved therapies to patients with cancer by supporting the
most promising new drug discovery and development projects. The PPTC
addresses key challenges associated with the development of new
therapies for children with cancer by developing reliable preclinical
testing data for pediatric drug candidates that can be used to inform
new agent prioritization decisions.
The first step in identifying new therapeutic targets, however, is
elucidating the basic biological mechanisms that give rise to cancers.
To further these research efforts, NCI supports the development of
resources for broad use across the cancer research community. These
resources include cell lines, organoid models, patient derived
xenograft (PDX) models, biospecimens, and other biological samples. NCI
makes drug information summaries available on its website, along with
extensive cancer treatment summaries. Additional resources include the
Developmental Therapeutics Program, the National Clinical Trials
Network (NCTN) Navigator, Patient-Derived Xenograft (PDX) Centers, PDX
Finder, the NCI Mouse Repository, and the Physician Data Query (PDQ)
Database.\76\
---------------------------------------------------------------------------
\76\ A more extensive list is available at www.cancer.gov/research/
resources/.
---------------------------------------------------------------------------
The Rare Tumor Patient Engagement Network, launched in fiscal year
2018 and part of NCI's CCR, leverages the resources of the NCI
intramural research program and the NIH Clinical Center to bring
together investigators, patients, and advocacy groups to study rare
tumors. Under the umbrella of this effort, NCI launched the My
Pediatric, Adolescent, and Adult Rare Tumor (MyPART) Network, a
collaboration of scientists, patients, family members, advocates, and
healthcare providers to find treatments for rare cancers. The MyPART
Network collects samples like blood, saliva, and archived biopsy tissue
from people with rare solid tumors as part of the Natural History Study
of Rare Solid Tumors. The purpose of the study is to engage rare tumor
patients and their families in the research process, study how rare
tumors grow, track participants' health history over a long period of
time, share data with other scientists, build new ways of testing new
treatments, and design new clinical trials for rare cancers. MyPART
scientists also hold clinics on rare tumors to facilitate
collaborations between researchers, patients, and advocacy
organizations; to date, MyPART has hosted clinics on chordomas, SDH-
deficient gastrointestinal stromal tumors, and medullary thyroid
cancer, and more clinics are in the planning stages. Additionally, the
NCI Comprehensive Oncology Network Evaluating Rare CNS Tumors (NCI-
CONNECT) program aims to advance the understanding of rare adult
central nervous system (CNS) cancers by establishing and fostering
patient-advocacy-provider partnerships and networks to improve
approaches to care and treatment; seven clinical studies and trials are
currently open through NCI-CONNECT.\77\
---------------------------------------------------------------------------
\77\ www.cancer.gov/rare-brain-spine-tumor/refer-participate/
clinical-studies.
---------------------------------------------------------------------------
Because of these and similar investments, the U.S. Food and Drug
Administration (FDA) has approved a number of therapies in recent years
for patients with rare cancer subtypes and related conditions. For
example, in May 2021, the FDA granted accelerated approval to sotorasib
(Lumakras) for patients with locally advanced or metastatic non-small
cell lung cancer (NSCLC) with alterations in the KRAS G12-C gene, a
mutation which is present in only 13.8 percentsa of NSCLC patients.
Similarly, the FDA approved selumetinib (Koselugo) in 2020 for the rare
tumor condition neurofibromatosis type 1, in patients over the age of
two, as the first approved treatment for this condition. In 2018, the
FDA granted accelerated approval to larotrectinib (Vitrakvi) for adult
and pediatric patients with solid tumors with a neurotrophic receptor
tyrosine kinase (NTRK) gene fusion. NTRK gene fusions are prevalent in
nearly all cases of certain rare cancer subtypes, including secretory
carcinoma of the breast or salivary gland and infantile fibrosarcoma;
they have also been observed in some patients with more common types of
cancer, such as glioma, melanoma, and carcinomas of the thyroid, lung,
and colon.\78\
---------------------------------------------------------------------------
\78\ www.ncbi.nlm.nih.gov/pmc/articles/PMC6859817/.
---------------------------------------------------------------------------
NIH will continue to support research efforts that reflect the
scientific understanding of the many subtypes of cancers, including
work that will enable the development of therapies for rare tumor
subtypes.
______
Questions Submitted to Dr. Gary Gibbons
Questions Submitted by Senator Roy Blunt
Question. Dr. Gibbons, we have all heard about the plight of COVID-
19 ``long-haulers'' who have symptoms after their acute COVID-19
infection has subsided. A growing number of studies suggest that many
patients experience some type of heart damage after contracting the
infection, even in those not sick enough to be hospitalized. According
to the American Heart Association, nearly one-fourth of those
hospitalized with COVID-19 have been diagnosed with cardiovascular
complications. A study in the Journal of the American Medical
Association stated that researchers found abnormalities in the hearts
of 79 percent of recovered patients and ``ongoing myocardial
inflammation'' in 60 percent.
Who is most at-risk of this type of heart damage, and is there
indication that this damage is permanent?
With heart damage appearing to be widespread, will screenings to
detect cardiovascular damage be included as routine follow-up care for
COVID-19 patients?
Do you have any sense of how long longitudinal studies should last
to follow long-haulers?
Answer. While severe acute respiratory syndrome coronavirus 2
(SARS-CoV-2) enters the body through the respiratory tract, the virus
also infects many other cell types and can damage multiple organs and
tissues, including the heart and blood vessels. In rare cases, acute
infection has been associated with cardiovascular complications
including acute myocardial injury, myocarditis (heart inflammation),
and arrhythmias (irregular heartbeat). This is not surprising given
that viruses frequently trigger inflammation, and as the body's immune
system fights off the virus, the inflammatory process can damage
healthy tissues, including the heart. Many different viruses are known
to cause myocardial injury and myocarditis.
Many patients with coronavirus disease 2019 (COVID-19) experience
damage to their blood vessels, leading to the formation of blood clots
(thrombosis) that can develop in or travel to vital organs, including
the heart. Blood clots in the coronary arteries can starve the heart of
oxygen and damage the heart muscle. NIH's ACTIV-4 Antithrombotics
adaptive master protocols have made progress in evaluating the safety
and effectiveness of various types of blood thinners (e.g., aspirin,
heparin, apixaban) for treating adults with signs of blood vessel
damage and thrombosis from COVID-19, known as COVID-19-associated
coagulopathy.\79\ Clinical trials are ongoing across three patient
populations (inpatient, outpatient, and convalescent or patients
recovering from COVID-19). These trials are providing valuable
information about how to help prevent moderately ill patients with
COVID-19 from progressing to intensive care, and could perhaps help
mitigate future cardiac complications. For example, ACTIV-4 has shown
that full-dose heparin is safe and effective at preventing blood clots
in moderately ill hospitalized patients and reduced the need for life
support.
---------------------------------------------------------------------------
\79\ www.nih.gov/research-training/medical-research-initiatives/
activ/covid-19-therapeutics-prioritized-testing-clinical-trials#activ4.
---------------------------------------------------------------------------
Studies have shown that patients with COVID-19 may show signs of
cardiac injury, detected by a release of the cardiac muscle protein
troponin into the bloodstream.\80\ Such injury is associated with worse
short-term outcomes and higher mortality. An analysis of more than 40
studies involving more than 8,000 COVID-19 patients found that venous
thromboembolism (VTE; blood clots originating in a vein) occurred in
approximately 21 percent of patients.\81\ Among COVID-19 patients
admitted to intensive care, the VTE rate was as high as 31 percent. A
review of myocarditis associated with acute COVID-19 estimated that the
incidence is less than five percent; although less than previously
thought, this could still mean a large number of patients with acute
myocarditis given that COVID-19 cases in the United States have
surpassed 33 million.
---------------------------------------------------------------------------
\80\ www.heartrhythmjournal.com/article/S1547-5271(20)30625-1/
fulltext#tbl1.
\81\ pubmed.ncbi.nlm.nih.gov/33251499/.
---------------------------------------------------------------------------
The incidence of continuing or new cardiac problems after COVID-19
or asymptomatic SARS-CoV-2 infection remains unknown. Although most
people with COVID-19 get better within weeks of illness, some people
experience post-acute sequelae, including chest pains, shortness of
breath, exhaustion, heart palpitations, and chest pain. In addition,
patients diagnosed with cardiac injury, thrombosis, or myocarditis
during acute COVID-19 could sustain damage to the heart that persists
long after the acute illness has passed. There is still much to be
learned about the long-term cardiovascular consequences of SARS-CoV-2
infection.
NIH's Researching COVID to Enhance Recovery (RECOVER) initiative
seeks to understand, and ultimately to prevent and treat, long COVID
and other post-acute sequelae of SARS-CoV-2 (PASC) across the
lifespan.\82\ At the center of the Initiative is an observational study
that will include adults and children recruited from ongoing studies of
COVID-19, long COVID clinics, and other cohorts. RECOVER is designed to
significantly expand both our knowledge about the full clinical
spectrum, long term outcomes, and underlying biology of PASC; as well
as our ability to provide safe and effective therapeutic interventions.
---------------------------------------------------------------------------
\82\ recovercovid.org/.
---------------------------------------------------------------------------
Current diagnostic protocols generally include physical, cognitive,
and psychological assessments. The evaluation of patients hospitalized
with COVID-19 includes elements of a cardiovascular evaluation,
including assessment of known cardiovascular disease and risk factors
for cardiovascular disease, assessment of symptoms that may be caused
by respiratory or cardiac disease, laboratory testing (including a
complete blood count and complete metabolic panel), chest radiograph,
electrocardiogram (ECG), and troponin testing (which is followed if
elevated). A more targeted cardiac evaluation may be needed depending
on the patient's symptoms. Patients who develop new onset heart
failure, for example, may need an echocardiogram (echo) to determine
the best course of action. One of the goals of the RECOVER meta-cohort
study is to develop core defining characteristics and diagnostic
criteria for long COVID and other forms of post-acute sequelae of SARS-
CoV-2 infection (PASC), including understanding the impact the virus
has on the cardiovascular system.
NIH plans to, and has support to follow the RECOVER meta-cohort for
at least 3 years. In addition to addressing the public health impact of
SARS-CoV-2 infection, RECOVER also has the potential to enhance our
understanding of other chronic syndromes theorized to have a viral
origin, at least in some individuals, such as chronic fatigue syndrome
and postural orthostatic tachycardia syndrome (POTS).
______
Questions Submitted by Senator Shelley Moore Capito
Question. Pulmonary fibrosis (PF) means scarring in the lungs. Over
time, the scar tissue can destroy the normal lung and make it hard for
oxygen to pass through the walls of the air sacs into the bloodstream.
PF is not just one disease--it is a group of more than 200 different
lung diseases that all look very much alike.
The most recent studies show that more than 200,000 Americans are
living with PF today. Approximately 50,000 new cases are diagnosed each
year and as many as 40,000 Americans die each year. With no known cure,
certain forms of PF, such as idiopathic pulmonary fibrosis, (IPF), may
take the lives of patients within three to 5 years from diagnosis.
PRECISIONS is the first-ever clinical trial to apply the principles
of precision medicine to the diagnosis and treatment of idiopathic
pulmonary fibrosis. PRECISIONS is supported by a $22 million grant from
the National Institutes of Health (NHLBI grant number HL145266) and
Three Lakes Foundation, a philanthropic organization.
PRECISIONS is designed as a double-blind, multi-center, randomized,
placebo-controlled trial investigating the safety and efficacy of NAC
in patients with IPF who have a specific genetic variant which is
present in 25 percent of IPF patients. The trial will enroll 200
patients from approximately 20 PFF Care Center Network (CCN) sites.
Initial recruitment into the study is being facilitated by looking at
phenotypic data from patients that are enrolled in the PFF Registry.
Can you provide an update on the NHLBI-funded PRECISIONS grant,
which seeks to shed more light on the role of genetics in pulmonary
fibrosis?
How has the COVID pandemic affected this study?
Answer. The National Heart, Lung, and Blood Institute (NHLBI) is
committed to supporting research on pulmonary fibrosis, which leads to
progressive scarring of the lungs that makes it increasingly more
difficult to breathe. PRECISIONS \83\ is a five-year study that aims to
enroll 200 patients with idiopathic pulmonary fibrosis (IPF) and use
genetic testing to identify those patients most likely to respond to an
experimental treatment, an antioxidant known as N-acetylcysteine or
NAC. This first-of-its-kind precision medicine trial builds on an
earlier study suggesting that a gene called TOLLIP influences how
patients respond to NAC, such that it might be helpful only for a
subgroup of patients who have a particular version of the gene. The
trial will enroll only that subgroup, in order to increase the
likelihood of detecting a benefit.
---------------------------------------------------------------------------
\83\ reporter.nih.gov/project-details/9822535.
---------------------------------------------------------------------------
PRECISIONS is co-funded by the Three Lakes Foundation, a non-profit
philanthropy that supports education and research efforts to improve
the time to diagnosis and accelerate new therapies for IPF. The study
also involves a partnership with the Pulmonary Fibrosis Foundation,
whose patient registry is being leveraged to perform molecular analyses
on biospecimens obtained from patients with IPF. These analyses are
intended to uncover novel genetic risk factors that will improve IPF
diagnosis, predict its clinical course, and understand its underlying
disease mechanisms--all of which could yield further insight into
potential targeted therapies.
The study was delayed in the latter half of fiscal year 2020 due to
COVID-19-related institutional research restrictions, which led to
NHLBI approval of a six-month interim no-cost extension. By December
2020, the investigators had successfully completed all pre-specified
project milestones for the first phase of their biphasic research plan,
including enrollment of the first study participant. NHLBI approved the
transition to the second phase of the project in March 2021. To date,
six study sites have been activated, the percentage of eligible
participants who meet the study's genotype inclusion criteria has been
exactly as expected, and recruitment has proceeded on target.
During COVID-19-related delays and uncertainty regarding the
feasibility of in-person lung function assessments (spirometry),
PRECISIONS initiated an ancillary study to understand the utility of
home spirometry to monitor patients with IPF. The study also intends to
add a COVID-19--specific questionnaire to baseline and follow-up visits
in the clinical trial as a means of leveraging this existing patient
cohort to capture additional data on the epidemiological and clinical
characteristics of COVID-19.
______
Questions Submitted by Senator Cindy Hyde-Smith
Question. Concerned about other countries' ability to obtain
vaccines quickly for their populations, the Administration recently
announced that it will support a waiver of the World Trade Organization
TRIPS Agreement, which would waive intellectual property protections
for COVID-19 vaccines. It is my understanding, however, that there are
no guarantees that the companies or countries who seek to use vaccine
manufacturer's intellectual property to make copies will be able to
deliver safe and effective vaccines, or that their manufacturing
processes will meet the strict regulatory standards necessary for
authorization. Furthermore, there are already reports of counterfeit
vaccines being used to exploit vulnerable populations in the U.S. and
around the world.
Are you concerned that giving away intellectual property via a
TRIPS waiver could make worse the problem of counterfeit and low-
quality vaccines in the market? What effect could this have on
endangering lives and undermining public confidence in the vaccines
that have been proven safe and effective?
Answer. The National Institutes of Health (NIH) is concerned about
counterfeit and low-quality vaccines; however, NIH does not have the
expertise or authority to investigate these matters. The degree to
which any TRIPS waiver addresses these issues of concern will not be
known unless and until the terms are agreed upon.
Question. The Administration recently endorsed the idea of waiving
intellectual property (IP) protections for COVID-19 vaccines, in the
hopes that it will speed up manufacturing of the vaccines around world.
However, it is my understanding that some vaccine developers are
already experiencing constraints in everything from raw materials to
fill-finish capacity critical to producing and administering vaccines.
Are you concerned that diverting critical supplies from
manufacturers with proven track records for delivering high-quality,
safe and effective vaccines could actually worsen the supply chain
constraints we're currently seeing, and not just for COVID vaccines,
but also non-COVID-19 medicines such as oncology and other infectious
diseases?
Answer. The National Institutes of Health (NIH) fully supports
efforts to ensure reliable supply chains for vaccines and other
medicines; however, NIH is not directly involved in these efforts.
______
Questions Submitted to Dr. Perez-Stable
Questions Submitted by Senator Roy Blunt
Question. Dr. Perez-Stable, we typically talk about getting
researchers into the NIH field and staying there as a pipeline.
However, when we look at the pipeline for minority researchers, it can
easily be called a funnel. We have a lot of work to do in increasing
the diversity of NIH researchers. And as the COVID-19 pandemic has
highlighted, NIH must also focus on health disparities research. The
problems to these two solutions may go hand-in-hand. I know that Dr.
Collins has started the UNITE program to look at racial inequities
within the NIH community and has started a Common Fund program to fund
transformative research into health disparities. While I commend these
steps, many of the fundamental issues these programs are trying to
address are reasons we started the Institute you fund--the National
Institute for Minority Health and Health Disparities.
Can you provide your perspective on how we get more minority
scientists into the NIH community?
And, specifically, what role should NIH take in making sure
minorities have the educational background necessary to go into STEM
fields--which often starts at the high school level, if not earlier?
Answer. The National Institutes of Health (NIH) is committed to
diversifying the research workforce and will continue to identify
opportunities to increase its focus on building and supporting a
diverse scientific workforce. The NIH UNITE initiative was developed to
address inequity in biomedical research and will help NIH to identify
more strategies and opportunities to strengthen its efforts to
diversify the research workforce and attract and prepare more students
from underrepresented backgrounds for STEM careers. The NIH already has
several efforts to diversify the STEM pipeline and to train students at
all levels of education as described below.
NIH supports several initiatives to attract and recruit more
minority scientists into the NIH intramural community. For example, the
NIH Equity Committee systematically tracks and evaluates diversity,
inclusion, and equity metrics in the intramural research program. In
addition, the Distinguished Scholars Program (DSP) enhances the
diversity of principal investigators in the NIH Intramural Research
Program (IRP) by supporting first year tenure-track investigators with
supplemental funds to start their research lab and engaging in
activities designed to foster a sense of belonging and to promote
research and career success. Moreover, the IRP provides a diverse
environment for NIH-wide scientific recruitments through the Stadtman
Tenure-Track Investigators, Lasker Clinical Research Scholars, and
Early Independent Scientists recruitment programs. This approach has
led to a greater proportion of women and scientists from
underrepresented backgrounds recruited to NIH. The 2019 DSP cohort was
comprised of approximately 7 percent Hispanics or Latinos, 27 percent
African Americans or Blacks, 27 percent Asians, 40 percent White, and
73 percent female. Among the fiscal year 2020 cohort, 21 percent was
African American or Black, 21 percent Hispanic or Latino, 21 percent
Asian, 36 percent White, and 50 percent female. Of the 15 Distinguished
Scholars selected in the 2019 cohort, nine were Stadtman Tenure-Track
Investigators, and two were Lasker Clinical Research Scholars. Of the
14 Distinguished Scholars selected in the 2020 cohort, 10 were Stadtman
Investigators, and three were Lasker Scholars.
Extramurally, NIH has dedicated efforts to recruit diverse
scientists from underrepresented groups to prepare successful NIH
grants. NIH provides Diversity Research Supplements to enhance the
diversity of the research workforce by recruiting and supporting
graduate students, post-doctoral fellows, and eligible investigators
from diverse backgrounds, including those from groups that have been
shown to be underrepresented in health-related research. These
supplements to existing grants provide a pathway to career success for
scientists from diverse backgrounds and remains relatively
underutilized. There are several other NIH programs that promote
diversifying the research workforce and some are highlighted below.
First, the NIH/National Institute on Minority Health and Health
Disparities Loan Repayment Program (NIMHD LRP), which aims to increase
the pool of qualified researchers who conduct health disparities
research. Over a 15-year period, recipients of an LRP award from NIMHD
are more likely to be awarded a subsequent NIH grant than their
counterparts who were not successful. The LRP Health Disparities
applications have now been extended to all NIH Institutes as of 2019.
Second, the Native American Research Centers for Health promote a cadre
of scientists and health research professionals interested in American
Indian/Alaska Native health research. Third, NIMHD established the
NIMHD Health Disparities Research Institute to support the research
career development of promising early-career minority health and health
disparities research scientists. Fourth, the NIH's Faculty
Institutional Recruitment for Sustainable Transformation (FIRST)
program, announced in 2020, will increase the participation of
researchers dedicated to inclusive excellence, including minority
researchers, in biomedical research at NIH-funded institutions. The aim
of the program is to enhance institutional inclusive excellence, with
diversity and equity at its core enabling biomedical research
institutions to hire a diverse cohort of early-stage research faculty
committed to inclusive excellence and diversity. The current pipeline
of underrepresented scientists is not empty with about 14 percent of
new U.S.-granted Science, Technology, Engineering and Math (STEM) PhDs
awarded to underrepresented groups and similarly 14 percent of current
medical students are from these groups.Lastly, the Science Education
Partnership Award (SEPA) Program funds innovative pre-kindergarten to
grade 12 science, technology, engineering, and mathematics (STEM) and
Informal Science Education (ISE) educational projects. SEPA projects
create partnerships among biomedical and clinical researchers and
teachers and schools, museums and science centers, media experts, and
other educational organizations. The NIH will continue to identify
opportunities to increase its focus on building and supporting a
diverse scientific workforce.
SUBCOMMITTEE RECESS
Senator Murray. The meeting is adjourned. Thank you.
[Whereupon, at 12:08 p.m., Wednesday, May 26, the
subcommittee was recessed, to reconvene subject to the call of
the Chair.]