The Future of Biomedicine: Translating Biomedical Research into Personalized Health Care (Hearing, 2021)
Summary
The printed record of a hybrid hearing before the Subcommittee on Health of the Committee on Energy and Commerce, House of Representatives, dated December 8, 2021 (Serial No. 117-60), on translating biomedical research into personalized health care. Opening statements are listed for Hon. Anna G. Eshoo, Hon. Brett Guthrie, Hon. Frank Pallone, Jr. and Hon. Cathy McMorris Rodgers. Eshoo opens by saying the subcommittee is there to hear from leading researchers about where biomedical innovation is headed. Witnesses include Amy Abernethy, M.D., Ph.D., of Verily Life Sciences, Atul Butte, M.D., Ph.D., of UCSF and UC Health, Adolph P. Falcon of the National Alliance for Hispanic Health and Leroy Hood, M.D., Ph.D., of the Institute for Systems Biology.
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“The Future of Biomedicine: Translating Biomedical Research into Personalized Health Care (Hearing, 2021).”
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[House Hearing, 117 Congress]
[From the U.S. Government Publishing Office]
THE FUTURE OF BIOMEDICINE: TRANSLATING BIOMEDICAL RESEARCH INTO
PERSONALIZED HEALTH CARE
=======================================================================
HYBRID HEARING
BEFORE THE
SUBCOMMITTEE ON HEALTH
OF THE
COMMITTEE ON ENERGY AND COMMERCE
HOUSE OF REPRESENTATIVES
ONE HUNDRED SEVENTEENTH CONGRESS
FIRST SESSION
__________
DECEMBER 8, 2021
__________
Serial No. 117-60
GRAPHIC(S) NOT AVAILABLE IN TIFF FORMAT
Published for the use of the Committee on Energy and Commerce
govinfo.gov/committee/house-energy
energycommerce.house.gov
_______
U.S. GOVERNMENT PUBLISHING OFFICE
56-897PDF WASHINGTON : 2024
COMMITTEE ON ENERGY AND COMMERCE
FRANK PALLONE, Jr., New Jersey
Chairman
BOBBY L. RUSH, Illinois CATHY McMORRIS RODGERS, Washington
ANNA G. ESHOO, California Ranking Member
DIANA DeGETTE, Colorado FRED UPTON, Michigan
MIKE DOYLE, Pennsylvania MICHAEL C. BURGESS, Texas
JAN SCHAKOWSKY, Illinois STEVE SCALISE, Louisiana
G. K. BUTTERFIELD, North Carolina ROBERT E. LATTA, Ohio
DORIS O. MATSUI, California BRETT GUTHRIE, Kentucky
KATHY CASTOR, Florida DAVID B. McKINLEY, West Virginia
JOHN P. SARBANES, Maryland ADAM KINZINGER, Illinois
JERRY McNERNEY, California H. MORGAN GRIFFITH, Virginia
PETER WELCH, Vermont GUS M. BILIRAKIS, Florida
PAUL TONKO, New York BILL JOHNSON, Ohio
YVETTE D. CLARKE, New York BILLY LONG, Missouri
KURT SCHRADER, Oregon LARRY BUCSHON, Indiana
TONY CARDENAS, California MARKWAYNE MULLIN, Oklahoma
RAUL RUIZ, California RICHARD HUDSON, North Carolina
SCOTT H. PETERS, California TIM WALBERG, Michigan
DEBBIE DINGELL, Michigan EARL L. ``BUDDY'' CARTER, Georgia
MARC A. VEASEY, Texas JEFF DUNCAN, South Carolina
ANN M. KUSTER, New Hampshire GARY J. PALMER, Alabama
ROBIN L. KELLY, Illinois, Vice NEAL P. DUNN, Florida
Chair JOHN R. CURTIS, Utah
NANETTE DIAZ BARRAGAN, California DEBBBIE LESKO, Arizona
A. DONALD McEACHIN, Virginia GREG PENCE, Indiana
LISA BLUNT ROCHESTER, Delaware DAN CRENSHAW, Texas
DARREN SOTO, Florida JOHN JOYCE, Pennsylvania
TOM O'HALLERAN, Arizona KELLY ARMSTRONG, North Dakota
KATHLEEN M. RICE, New York
ANGIE CRAIG, Minnesota
KIM SCHRIER, Washington
LORI TRAHAN, Massachusetts
LIZZIE FLETCHER, Texas
------
Professional Staff
JEFFERY C. CARROLL, Staff Director
TIFFANY GUARASCIO, Deputy Staff Director
NATE HODSON, Minority Staff Director
Subcommittee on Health
ANNA G. ESHOO, California
Chairwoman
G. K. BUTTERFIELD, North Carolina BRETT GUTHRIE, Kentucky
DORIS O. MATSUI, California Ranking Member
KATHY CASTOR, Florida FRED UPTON, Michigan
JOHN P. SARBANES, Maryland, Vice MICHAEL C. BURGESS, Texas
Chair H. MORGAN GRIFFITH, Virginia
PETER WELCH, Vermont GUS M. BILIRAKIS, Florida
KURT SCHRADER, Oregon BILLY LONG, Missouri
TONY CARDENAS, California LARRY BUCSHON, Indiana
RAUL RUIZ, California MARKWAYNE MULLIN, Oklahoma
DEBBIE DINGELL, Michigan RICHARD HUDSON, North Carolina
ANN M. KUSTER, New Hampshire EARL L. ``BUDDY'' CARTER, Georgia
ROBIN L. KELLY, Illinois NEAL P. DUNN, Florida
NANETTE DIAZ BARRAGAN, California JOHN R. CURTIS, Utah
LISA BLUNT ROCHESTER, Delaware DAN CRENSHAW, Texas
ANGIE CRAIG, Minnesota JOHN JOYCE, Pennsylvania
KIM SCHRIER, Washington CATHY McMORRIS RODGERS, Washington
LORI TRAHAN, Massachusetts (ex officio)
LIZZIE FLETCHER, Texas
FRANK PALLONE, Jr., New Jersey (ex
officio)
C O N T E N T S
----------
Page
Hon. Anna G. Eshoo, a Representative in Congress from the State
of California, opening statement............................... 2
Prepared statement........................................... 4
Hon. Brett Guthrie, a Representative in Congress from the
Commonwealth of Kentucky, opening statement.................... 6
Prepared statement........................................... 8
Hon. Frank Pallone, Jr., a Representative in Congress from the
State of New Jersey, opening statement......................... 12
Prepared statement........................................... 14
Hon. Cathy McMorris Rodgers, a Representative in Congress from
the State of Washington, opening statement..................... 16
Prepared statement........................................... 18
Witnesses
Amy Abernethy, M.D., Ph.D., President Of Clinical Studies
Platforms, Verily Life Sciences................................ 22
Prepared statement........................................... 25
Answer to submitted questions................................ 143
Atul Butte, M.D., Ph.D. Distinguished Professor and Director of
the Bakar Computational Health Science Institute, UCSF, and
Chief Data Scientist, UC Health................................ 33
Prepared statement........................................... 35
Answer to submitted questions \1\
Adolph P. Falcon, M.P.P., Executive Vice President, National
Alliance for Hispanic Health................................... 42
Prepared statement........................................... 44
Answer to submitted questions................................ 155
Leroy Hood, M.D., Ph.D., President, Institute for Systems
Biology, Affiliate Professor of Immunology, University of
Washington..................................................... 49
Prepared statement........................................... 51
Answer to submitted questions................................ 171
Lloyd B. Minor, M.D., Dean, Stanford University School of
Medicine....................................................... 64
Prepared statement........................................... 66
Answer to submitted questions................................ 183
Submitted Material
Statement of December 8, 2021, from the Alzheimer's Association
and Alzheimer's Impact Movement, submitted by Ms. Eshoo........ 120
Statement of December 8, 2021, from Representative Yvette D.
Clarke of New York, Member of Congress, submitted by Ms. Eshoo. 124
Statement of December 8, 2021, from Representative Eric M.
Swalwell of California, Member of Congress, submitted by Ms.
Eshoo.......................................................... 125
Statement of December 8, 2021, from Amrit Chaudhuri, Chief
Executive Officer, SmartLabs................................... 126
Study ``Regenerative Medicine and Cell Therapies: An Overview of
Clinical Studies in the United States'', Alliance for Cell
Therpary Now................................................... 130
Statement of December 8, 2021, from Representative Tom Emmer of
Minnesota, Member of Congress, submitted, by Ms. Eshoo......... 137
Article of December 2, 2021, ``On Health Policy, Donald Trump
Beats Joe Biden Hands Down'', by Joel White, Wall Street
Journal, submitted by Ms. Eshoo................................ 139
THE FUTURE OF BIOMEDICINE: TRANSLATING BIOMEDICAL RESEARCH INTO
PERSONALIZED HEALTH CARE
----------
WEDNESDAY, DECEMBER 8, 2021
House of Representatives,
Subcommittee on Health,
Committee on Energy and Commerce,
Washington, DC.
The Subcommittee met, pursuant to call, at 10:30 a.m., in
the John D. Dingell Room, 2123 of Rayburn House Office
Building, and remotely via Cisco Webex online video
conferencing, Hon. Anna Eshoo (Chairwoman of the Subcommittee),
presiding.
Members present: Representatives Eshoo, Matsui, Castor,
Sarbanes, Welch, Schrader, Cardenas, Ruiz, Dingell, Kuster,
Kelly, Barragan, Blunt Rochester, Craig, Schrier, Trahan,
Fletcher, Pallone, ex officio; Guthrie (subcommittee ranking
member), Upton, Burgess, Griffith, Bilirakis, Long, Bucshon,
Mullin, Hudosn, Carter, Dunn, Curtis, Crenshaw, Joyce, and
McMorris Rodgers, ex officio.
Also present: Representatives DeGette and Schakowsky
Staff present: Lydia Abma, Health Fellow; Tania Calle,
Health Fellow; Waverly Gordon, Deputy Staff Director and
General Counsel; Tiffany Guarascio, Staff Director; Zach Kahan,
Deputy Director Outreach and Member Service; Mackenzie Kuhl,
Press Assistant; Meghan Mullon, Policy Analyst; Juan Negrete,
Junior Professional Staff Member; Kaitlyn Peel, Digital
Director; Asad Ramzanali, Legislative Director; Tim Robinson,
Chief Counsel; Chloe Rodriguez, Clerk; Kylea Rogers, Staff
Assistant; Andrew Souvall, Director of Communications,
Outreach, and Member Services; Kimberlee Trzeciak, Chief Health
Advisor; C.J. Young, Deputy Communications Director; Alec
Aramanda, Minority Staff Member; Sarah Burke, Minority Deputy
Staff Director; Grade Graham, Minority Chief Counsel, Health;
Nate Hodson, Minority Staff Director; Peter Kielty, Minority
General Counsel; Emily King, Minority Member Services Director;
Bijan Koohmaraie, Minority Chief Counsel, Oversight and
Investigations Chief Counsel; Clare Paoletta, Minority Policy
Analyst, Health; Kristin Seum, Minority Counsel, Health; and
Michael Taggart, Minority Policy Director.
Ms. Eshoo. Good morning, everyone. Good morning,
colleagues. The Subcommittee on Health will now come to order.
And due to COVID-19, today's hearing is being held remotely
as well as in person.
For members and witnesses taking part in person, we are
following the guidance of the CDC and the Office of the
Attending Physician. So please wear a mask when you are not
speaking.
For members and witnesses taking part remotely, microphones
will be set on mute to eliminate background noise. Members and
witnesses, you will need to unmute your microphone when you
wish to speak.
Since members are participating from different locations at
today's hearing, recognition of members for questions will be
in the order of subcommittee seniority.
Documents for the record should be sent to Meghan Mullon at
the email address we have provided to your staff, and all
documents will be entered into the record at the conclusion of
our hearing.
The Chair now recognizes herself for 5 minutes for an
opening statement.
OPENING STATEMENT OF HON. ANNA G. ESHOO, A REPRESENTATIVE IN
CONGRESS FROM THE STATE OF CALIFORNIA
Colleagues, we are here today to hear from our country's
leading researchers about where biomedical innovation is headed
and what we can do to accelerate innovation to improve the
health and the lives of every American.
This is, I believe, one of the most important topics we
could be discussing at our Subcommittee. This year marks 20
years since the initial results of the Human Genome Project
were first published. The outcomes of the project provided a
glimpse into DNA's potential for advancing research and
launched a new era of biomedicine where genetic discoveries
paved the way for new treatment options and improved human
health.
The Human Genome Project was and remains the world's
largest collaborative biological project. Ambitious for its
time, the project sequenced the three billion pairs of DNA
letters of the human genome in just over ten years with $2.7
billion in funding.
This success is due to the multidisciplinary research
efforts of 20 international institutions, the coordination of
high-performance computing centers, and the successful
management by the NIH and the Department of Energy.
Incredible advances in the field of genomics and the
creation of state-of-the-art technologies now allow us to
understand human biology much better than ever before. A human
genome can now be sequenced in a matter of days for less than
$1,000 on a single ``deep sequencing'' machine.
Genetic testing can now be done at home to find increased
risk for certain health problems. And CRISPR gene editing can
uniquely modify genetic code, offering hope for the time for
treating rare genetic disorders in clever ways.
It is through groundbreaking scientific breakthroughs like
these that the U.S. continues to be on the cutting edge of
discovery. Fundamental discoveries and basic research continue
to help scientists identify genetic variants that increase the
risk of diseases like cancer and diabetes. And novel
discoveries in translational research will pave the way toward
innovative treatments.
As we meet today, Americans still face the highest disease
burden and the highest rate of avoidable deaths when compared
to similarly large and wealthy countries. Traditional
medicine's approach of treating the average patient with a one-
size-fits-all approach does not appropriately serve our
country's diverse patient population.
We need to capitalize on the new tools and technologies
that are being created to treat each patient as what they are--
a unique individual.
I am greatly looking forward to hearing from today's
witnesses about where they see the biomedical sciences heading
and what Congress should be investing in to accelerate
innovation for the betterment of the American people in the
third decade of the 21st century.
[The prepared statement of Ms. Eshoo follows:]
Prepared Statement of Hon. Ann Eshoo
[GRAPHIC] [TIFF OMITTED] T6897.001
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Ms. Eshoo. The Chair now recognizes Mr. Guthrie, the
distinguished Ranking Member of our Subcommittee, for 5 minutes
for his opening statement.
OPENING STATEMENT OF HON. BRETT GUTHRIE, A REPRESENTATIVE IN
CONGRESS FROM THE COMMONWEALTH STATE OF KENTUCKY
Mr. Guthrie. Thank you, thank you, Chair Eshoo for holding
this hearing.
And today we are discussing ways to promote and advance
American biomedical innovation.
I thank the Chair for holding this hearing, and I look
forward to hearing from our witnesses today about how
healthcare delivery can be transformed through data generation
and innovative technologies.
However, we cannot talk about advancing healthcare
innovation without addressing the harmful drug pricing
provision the Democrats' reckless tax and spending spree bill
that will ultimately restrict patients' access to timely care
and lead to less cures.
This past year several indicators have shown the Biden
administration is moving the country in the wrong direction. We
are facing the highest levels of drug overdoses in our Nation's
history, in part, because of the influx of deadly drugs,
including fentanyl, exacerbated by the Biden administration's
failure to secure our southern border.
Additionally, Americans are experiencing the highest levels
of inflation in over 30 years. This is the most expensive
Thanksgiving holiday most have seen.
Despite all of this, the White House and Democrats in
Congress continue to ignore these flashing red lights and are
downplaying the true risks that increasing Federal spending
will have on American households.
Even the former Chair of the White House Counsel of
Economic Advisors in the Obama administration is sounding alarm
bells on inflation by stating that the Biden administration's
officials are systematically underestimating inflation and
further saying they poured kerosene on the fire by signing the
massive $1.9 trillion American Rescue Plan.
The Democrats' most recent partisan effort would force drug
manufacturers to accept a government-mandated price of a drug
or potentially face up to a 95 percent excise tax for refusing
to accept the government's bad deal. Make no mistake. This is
not a negotiation. This is government price setting.
The University of Chicago published an issue brief last
week on the impact of this tax and spending bill on
biopharmaceutical innovation and patient health. The studies in
the summary brief found the drug pricing provisions would lead
to a decrease in research and development investments by over
660 billion through 2039, resulting in 135 fewer drugs brought
to the market during this time.
Most consequentially, they found it could lead to the loss
of approximately 331 million life-years, which is 31 times
higher than loss due to COVID.
Most Americans are against this. A survey from the Kaiser
Family Foundation found that 72 percent of Americans oppose
drug price negotiation if it leads to fewer medications being
developed in the future.
The bill punishes our innovators and undermines the
significant strides of this committee. Operation Warp Speed
under President Trump's leadership, and others have made
throughout the pandemic to get needed treatments to healthcare
settings as quickly and safely as possible.
The good news is there are existing bipartisan proposals
introduced by members of this committee to address the rising
cost of prescription drug medications for patients without
harming innovation.
H.R. 19, the Lower Cost, More Cures Act, includes 40
bipartisan proposals that would bring needed market-based
reforms like addressing pay-for delay tactics in order to lower
the cost of prescription medications without threatening the
development of future cures.
H.R. 19 would specifically give seniors relief by capping
their annual out-of-pocket spending and reducing the cost of
insulin for seniors by capping monthly insulin costs, once
deductibles are met. In fact, Build Back Better does have some
of these proposals from H.R. 19, which shows that there are
areas of agreement on how to lower drug costs for Americans.
Where we do not and we will never agree is on the idea that
forcing companies to accept whatever price the government feels
like paying for prescription medications is the correct way to
lower the cost of prescription medications.
I am deeply concerned about the estimated loss of more than
100 new cures if Build Back Better is signed into law and hope
this bill ultimately fails.
Moving forward, I encourage my colleagues to work with me
on bipartisan reforms to lower drug prices and find solutions
that prioritize getting more affordable treatments to market
for patients living with life-threatening and debilitating
diseases like ALS and other neurodegenerative diseases. Doing
so can improve the quality of life for millions across the
country.
Thank you, Madam Chair, and I yield back.
[The prepared statement of Mr. Guthrie follows:]
Prepared Statement of Hon. Brett Guthrie
[GRAPHIC] [TIFF OMITTED] T6897.003
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[GRAPHIC] [TIFF OMITTED] T6897.006
Ms. Eshoo. The gentleman yields back.
The Chair is now pleased to recognize the Chairman of the
Full Committee, Mr. Pallone, for his 5 minutes for an opening
statement.
OPENING STATEMENT OF HON. FRANK PALLONE, Jr., A REPRESENTATIVE
IN CONGRESS FROM THE STATE OF NEW JERSEY
Mr. Pallone. Thank you, Chairwoman Eshoo.
I know this is an issue that is very important to you and
you have been involved with for a long time and that is the
cutting edge of biomedical research.
As we celebrate the 20th anniversary of the mapping of the
human genome this year, it is an opportunity to examine how far
we have come and how scientists are charting a path forward to
lead to new discoveries to improve public health.
The purpose of today's hearing is to examine the current
state of biomedical research in the U.S. and explore the
opportunities for the future of innovation, investment, and
equity in healthcare.
Our nation is fortunate to have the greatest biomedical
researchers in the world, working every day in clinics and labs
to advance our basic understanding of disease in living
organisms and apply that foundational knowledge to the
development of treatments and cures.
Historically, our health system has focused on treating or
preventing diseases broadly in the average patient. This has
resulted in treatments and drugs that work well for some but
have little to no effect in others.
In the last decade, however, we have seen transformative
changes in the field of biomedical research. One such example
is the advancement of precision medicine, which seeks to
individualize treatment and care by accounting for patient-
specific genes, environment, and lifestyle.
For example, research and development in precision medicine
has helped advance immunotherapy treatments for oncology. If we
are to continue to build on this work, we will need to leverage
new technological tools and methods of study, such as genetic
phenotyping, quantum computing, novel clinical trial designs,
as well as traditional basic and translational research.
As we examine the current state of biomedical research, we
must keep equity at the forefront of our efforts. The ongoing
COVID-19 pandemic has demonstrated what many have known all
along, that our health system disadvantages minority
communities and inadequately addresses their needs.
We must examine and account for diverse populations in data
collection, as well as recognition of potential biases in
artificial intelligence, biomedical research, and the
development of drugs, devices, and treatments.
It is also important that we ensure Americans can access
these drugs and treatments and that is a critical component of
the Build Back Better Act that the House passed last month.
Today, far too many Americans are being forced to ration their
medications, go without needed treatments, or exhaust their
life savings because prescription drug costs are too high.
It is simply not fair that Americans pay three, four, or
ten times as much for the exact same drugs as people in other
countries pay.
The Build Back Better Act will make prescription drugs more
affordable by finally giving Medicare the ability to negotiate
lower drug prices with the pharmaceutical companies. Seniors
will also pay no more than $2,000 a year in out-of-pocket costs
for their drugs and the legislation penalizes pharmaceutical
companies that unfairly raise prices.
The bill also allows the Federal Government to negotiate
insulin prices and lowers those prices to no more than $35 a
month for Americans with diabetes.
And this legislation finally begins to provide relief to
Americans at the pharmacy counter without threatening
innovation.
So I look forward to hearing from our panel of experts who
have significant experience in academic, clinical, regulatory,
and commercial settings. The future of biomedical research
depends on the synergy between these fields and the fruits of
their labor will transform our health system to promote
wellness for all Americans.
So, again, I thank the Chairwoman for convening this
hearing and continuing her work to move the ball forward on
such an important topic.
And I yield back.
[The prepared statement of Mr. Pallone follows:]
Prepared Statement of Hon. Frank Pallone Jr.,
[GRAPHIC] [TIFF OMITTED] T6897.007
[GRAPHIC] [TIFF OMITTED] T6897.008
Ms. Eshoo. The gentleman yields back.
The Chair is now pleased to recognize Representative Cathy
McMorris Rodgers, the Ranking Member of our Full Committee, for
your 5 minutes for an opening statement.
OPENING STATEMENT OF HON. CATHY McMORRIS RODGERS, A
REPRESENTATIVE IN CONGRESS FROM THE STATE OF WASHINGTON
Mrs. Rodgers. Thank you, Madam Chair.
And to our witnesses, I want to extend this special thank
you to Dr. Leroy Hood for making the trip all the way from the
great State of Washington to be with us.
The story of American biomedical innovation is one that
should be celebrated. Through the NIH's Human Genome Project,
we know that there are over 20,000 human genes.
To help discover new cures, this information is being used
to identify genes found in conditions like Alzheimer's, cancer,
and rare diseases.
The 21st Century Cures Act gave the NIH the resources to
advance basic biomedical research across the spectrum.
As co-chair of the Neuroscience Caucus, I have been a
strong supporter of the BRAIN Initiative, which is aimed at
finding new ways to treat, cure, and prevent brain disorders by
exploring how the brain enables the body to store and retrieve
information quickly.
The All of Us Research Program is also revitalizing
thehealthcare system by teaching us more about precision
medicine and personalized care plans. I am excited about these
researchers' innovative work that will reduce cost and, more
importantly, save lives and improve people's quality of life.
America's biopharmaceutical sector is vital to our global
competitiveness. There are over 4,000 cancer drugs in the R&D
pipeline, 700 for neurological conditions, and 450 for
cardiovascular disease.
We are on the verge of amazing breakthroughs. America is
leading the way in bringing hope to patients here and around
the world.
Unfortunately, in the reconciliation package pending before
Congress, this would be reversed, and the incredible work would
be lost. And it would eliminate hope for future cures.
Price controls, price controls that are being included
right now in President Biden's plan will kill innovation and
lurch us more toward government-controlled healthcare. We see
in countries like Canada and the U.K. the power rests with the
government to measure lives in dollars and cents before
politicians decide whether a cure is worth it.
It would mean no hope for many people who deserve a
fighting chance at life. It would also push private innovators
further overseas and empower countries like China, which is
already racing to lead the world in biotechnology.
We all sadly saw what happened during the pandemic when
China dominated the market for certain medical supplies.
Surely, we all agree that less innovation, fewer cures, and a
dependency on China cannot be America's future.
There was bipartisan agreement on this just a few months
ago. That is why H.R. 3, government price control, failed in
this committee. Unfortunately, this policy has been resurrected
in the bill that has now passed the House floor.
I look forward to hearing today about how we can unleash
more biomedical information, not destroy it with government
price controls.
The Congressional Budget Office confirmed that there will
be fewer new medicines as a result of government price controls
that ultimately passed the House. Hopefully it will not pass
the Senate.
A University of Chicago study estimates that it would
shrink R&D spending by 18, 18.5 percent and lead to 135 fewer
new cures. The study found price controls would generate a loss
331 million life-years, which measures the lost potential of
saved lives and longer years lived.
This study found that government price controls would lead
to 21 to 43 fewer new antiviral drugs. They estimate four to
nine fewer new HIV drug approvals, and about two to five
million life-years lost as a result of price controls.
We have heard some suggest that this reduction in cures and
treatments is just a feature of built-in cost of bringing down
drug cost. It has been suggested that it is, quote, worth it
and a tradeoff Americans are willing to accept.
I do not believe it. It is a false choice. It is a false
choice on families like Crystal Davis who believed in the
promise of America so that her son with SMA can live a full
life. We should be doing all that we can to encourage hope in
the next generation of cures.
Let's reject price controls and focus on bipartisan work
like solutions in H.R. 19, which will result in increased
competition and lower patient cost without sacrificing the
future of biomedical innovation in the United States.
Now more than ever we should be working together on
uniquely American solutions to save lives, lower cost, and
uphold the dignity and right of every person to live a full
life. Energy and Commerce can lead the way.
I look forward to the discussion today.
I yield back.
[The prepared statement of Mrs. Rodgers follows:]
Prepared Statement of Hon. Cathy McMorris Rodgers
[GRAPHIC] [TIFF OMITTED] T6897.009
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Ms. Eshoo. The gentlewoman yields back.
Pursuant to committee rules, all members' written opening
statements shall be made part of the record.
I now would like to introduce our witnesses. First, Dr. Amy
Abernethy is the President of Clinical Studies Platforms at
Verily Life Sciences.
Welcome to you and thank you for being with us today.
Remotely, Dr. Atul Butte is the Priscilla Chan and Mark
Zuckerberg Distinguished Professor and the Inaugural Director
of the Bakar Computational Health Sciences Institute at UCSF.
That is University of California at San Francisco. He is also
the Chief Data Scientist for the entire University of
California Health System.
Welcome to you, Doctor, and thank you very much for being
with us.
Mr. Adolph Falcon is the Executive Vice President of the
National Alliance for Hispanic Health.
Welcome to you and thank you for being with us.
Dr. Leroy Hood is here at the witness table. He is the
President of the Institute for Systems Biology and an Affiliate
Professor of Immunology at the University of Washington.
Welcome to you, Dr. Hood, and thank you.
And last but certainly not least, Dr. Lloyd Minor who is
the Dean of the Stanford University School of Medicine, which I
have the privilege of representing.
Welcome to you, Dr. Minor, and thank you for the on again,
off or putting up with the on again, off again changes in
schedules for this hearing. I appreciate it. We all do, and it
is an honor to have you with us this morning.
So, thank you for joining us today. We look forward to your
testimony.
You are probably familiar with the lights that are in front
of you. You have a minute remaining when the light turns yellow
and we all know what red means.
So, let's begin with Dr. Abernethy. You are recognized for
5 minutes for your testimony.
STATEMENT OF AMY ABERNETHY, M.D., Ph.D., PRESIDENT OF CLINICAL
STUDIES PLATFORMS, VERILY LIFE SCIENCES; ATUL BUTTE, M.D.,
Ph.D., DISTINGUISHED PROFESSOR AND DIRECTOR OF THE BAKAR
COMPUTATIONAL HEALTH SCIENCE INSTITUTE, UCSF, AND CHIEF DATA
SCIENTIST, UC HEALTH; ADOLPH P. FALCON, M.P.P., EXECUTIVE VICE
PRESIDENT, NATIONAL ALLIANCE FOR HISPANIC HEALTH; LEROY HOOD,
M.D., Ph.D., PRESIDENT, INSTITUTE FOR SYSTEMS BIOLOGY,
AFFILIATE PROFESSOR OF IMMUNOLOGY, UNIVERSITY OF WASHINGTON;
AND LLOYD B. MINOR, M.D., DEAN, STANFORD UNIVERSITY SCHOOL OF
MEDICINE
STATEMENT OF AMY ABERNETHY, M.D.
Dr. Abernethy. Thank you, Chair Eshoo, Ranking Member
Guthrie, and members of the Health Subcommittee.
Thank you for inviting me to speak with you today about
personalized healthcare. I have spent my career working on ways
to make sure patients are getting the care that is tailored to
their unique circumstances and to make sure that that care is
based on the best available evidence.
For many years before my tour at FDA, I was a Professor of
Medicine at Duke University. My clinical training is in
oncology and palliative care. I focused on patients with
melanoma.
I also directed the Center for Learning Health Care at
Duke, where the vision was to enable whole-person care to meet
the individual where they are in their health journey and
seamlessly bring together research and clinical care so that
they inform each other in a patient-centric way.
It is, therefore, a real honor and humbling that after many
years I am here with this panel discussing ways that we can
come together to make personalized healthcare a reality.
If there is one message I want to emphasize today, it is
that we are not going to reach the goal of personalized
healthcare unless we make big gains in how we generate and
analyzehealthcare data, data that tell us how new treatments
work at the individual personalized level.
Data and the clinical evidence generated from these data
are the fundamental underpinning of personalized healthcare
decisions.
Let's take cancer diagnosis as one example. With
personalized healthcare, it will not be just a matter of
matching a certain cancer mutation with the appropriate drug
for that cancer mutation, but rather a selection of the
appropriate intervention, and it will depend on many features
blended together, including a person's symptom experience, such
as what symptoms are bothering them the most; the genetic basis
for the disease; and lots of additional details, such as the
likelihood for the intervention to work based on a person's
background, genetics or environmental exposures.
Finally, the selection of the intervention should account
for the personal values of the patient.
Personalization in healthcare means far more than just
matching a treatment to specific biologic markers. It is the
ability to consider many features and circumstances together to
support ultra-tailoring, matching to the intervention and the
patient.
What gaps do we have before this vision can be realized?
Diversity in clinical trials, a topic I hope that we cover
today, is just one example.
Traditional clinical trials are an extremely powerful way
for understanding what treatment works. But the ways we have
historically done clinical trials also have significant
drawbacks, not the least of which is that these trials only
capture a segment of the population.
As a result, such trials typically cannot account for the
great diversity of people in our society. We can do better.
Diversity in clinical trials is only one of the challenges that
we need to address, but it is a big one.
When I say that we need to address the gaps in evidence
generation, I do mean ``we.'' Congress and the Executive Branch
agencies involved, including HHS, FDA, CMS, and others, are
extremely important for setting the requirements and goal posts
for developing healthcare evidence.
For example, 21st Century Cures represent a big leap in the
right direction and included a multitude of provisions that
have helped to put us on a course to make better use of real-
world data and real-world evidence.
The Cures 2.0 Act has some very important proposals that
can continue the momentum, including on themes like
decentralized trials, use of real-world data, and diversity in
clinical trials.
But I will reiterate a point that I made over and over
again when I was at FDA. In addition to government, industry
also has a critical responsibility to push the field forward,
to generate the tools needed to translate biomedical
discoveries into personalized healthcare.
Different components of industry are now playing different
roles in this work. On the discovery side, there are many
companies, large and small, doing revolutionary work with gene
and cell-based therapies, advanced diagnostics, and digital
therapeutics. These are just a few categories where we are
seeing solutions.
On the health tech side, we, including the company where I
now work, Verily, are working to develop the machinery of
evidence generation. We are hyper focused on making clinical
trials run more efficiently and reach a broader, more diverse
set of patients than ever before.
We are building data sets that allow us to combine the best
features of clinical trials and real-world data, and we are
developing methods for monitoring the performance ofhealthcare
products when they are deployed in the real world, especially
important for monitoring tools, such as artificial
intelligence-based tools of medicine.
And we need to do all of this with robust, transparent, and
secure works in a way that protects personal privacy. This work
is complex and takes collaboration between clinicians, data
scientists, privacy experts, and of course, excellent software
energy and engineers.
I look forward to talking with you today. Thank you.
[The prepared statement of Dr. Abernethy follows:]
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Ms. Eshoo. Thank you very much, Doctor.
The Chair is now pleased to introduce Dr. Butte for 5
minutes for your testimony.
STATEMENT OF ATUL BUTTE, M.D.
Dr. Butte. Thank you, Chair Eshoo, Ranking Member Guthrie,
and committee members.
My name is Atul Butte. I am a physician scientist and
institute Director at the University of California at San
Francisco and the Chief Data Scientist for the whole UC Health
System.
I have to start by saying the views expressed here are my
own and do not represent the views of the University of
California or any of these organizations.
Since 2013, when White House Office of Science and
Technology Policy, Dr. John Holdren directed Federal agencies
to enable the results of federally funded research to be made
freely available to the public, our national data resources are
growing, but we do not often think about this biomedical
infrastructure like we think about other national resources,
like our national labs or our national parks or our roads and
bridges.
I will highlight a few examples here, all of which are
housed at or through NIH of this national data resource.
GenBank contains all publicly available DNA sequences. While
nearly 40 years old, it is still a relevant----
Ms. Eshoo. Doctor, I am sorry to interrupt. The sound is a
bit jerky. So can you just speak a little slower so that we can
absorb every word. I am not catching it all, and I think it may
be a little difficult for the rest of the members as well.
Every word you say counts. So if you could just slow down a
little bit and if you need a little more time, I will certainly
grant it. OK?
Thank you.
Dr. Butte. Surely. I will slow down.
I am highlighting a few examples of our national data
resources, which are growing, and we should think of these as
national resources, like our national labs and national parks,
to be protected.
GenBank, for example, nearly 40 years old now, is a
relevant worldwide home for even SARS-CoV-2 sequences.
Cancer Genome Atlas, one of many disease-specific databases
funded by NIH, has led to tens of thousands of cancer patients
being studied, led to many discoveries, and while the program
has formally ended, the data is still there publicly
accessible.
And many others, tens of thousands of data resources around
the world emphasizing the volume and complexity of data needed
to understand the human condition. And all of this is just the
tip of an iceberg of the data we are going to need to develop
the next generation of cures and treatments.
And we are expecting much more to come. Starting in January
2023, it is great that NIH will be executing on their new
policy requiring all NIH supported research include a data
management and sharing plan. It will be important to ensure
these plans are good plans and in force.
A newer source of biomedical data is electronic health
record data surrounding clinical care. One of the most exciting
roles I have now is that of Chief Data Scientist for the
entirety of the University of California Health System, and
across our six medical schools and 12 hospitals, we have
treated over seven million patients in the past ten years.
We have a secure data warehouse now that we use to improve
the quality of our care and, when deidentified, to enable the
next generation of clinical research with data on hundreds of
millions of encounters, procedures, and nearly a billion
medication orders and prescriptions.
The narrative I want to make sure I communicate is that we
have spent billions of dollars to acquire this data on
patients. In fact, I call it the most expensive data in America
now. It will be a national tragedy if we do not use this data,
of course, safely and responsibly; if we do not use this data
to improve the practice of medicine.
This clinical data can inform patients as to the detail of
what is going on in their care and what is next. Data on the
pricing of services can help patients select the right level of
care at an affordable price, and we can start to use this data
to eliminate unnecessary use of specific medications.
And I am proud that the University of California Health
System recently signed a health equity pledge, along with 40
other institutions, to leverage our clinical data to document
and address health equity or specifically inequities.
I am going to end with some specific recommendations.
First, more funding should be made available for training in
biomedical data sciences at all stages, teaching statistics,
programming, and database skills, design and visualization.
Second, Federal funding for these important biomedical data
repositories remains quite variable, too arbitrary, and too
fragile, and needs to be stabilized.
Third, can we open more Federal Government-related data to
others? Imagine if the millions of chest X-ray images, for
example, from federally run hospitals and clinics were
carefully and safely shared with available AI engineers to
build novel tools to help lead them and then companies around
those schools.
We should invest in technological solutions, enable broader
and better use of our national data resources.
Fourth, let's ensure that the 2023 NIH policies for data
sharing do carry through, and that we create a better culture
of research data that is disseminated with the public.
And fifth and final, we need to build on programs like the
new NIH AIM-AHEAD to not only make sure diversity is properly
covered in our biomedical data set and artificial intelligence
models, but diversity is promoted and enhanced among the data
scientists themselves.
Thank you for enabling me to give my signature. Thank you.
[The prepared statement of Dr. Butte follows:]
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Ms. Eshoo. Thank you, Dr. Butte.
It is almost encyclopedic what you just shared with us, and
I look forward to the questions that we are going to ask you to
answer.
Next, Mr. Falcon. You are recognized for 5 minutes, and
thank you, again, for being with us today.
STATEMENT OF ADOLPH P. FALCON
Mr. Falcon. Well, thank you, Chairwoman Eshoo and Ranking
Member Guthrie and members of the Health Subcommittee. I thank
you for the opportunity to testify today on behalf of the
National Alliance for Hispanic Health, the Alliance and the
Healthy Americas Foundation, which is the supporting
organization of the Alliance.
The Alliance is the Nation's premier science-based and
community-driven organization that focuses on the best health
for all. We work to ensure that health incorporates the best of
science, culture, and community.
Our community-based members I am proud to report deliver
health and human services to over 15 million in underserved
communities every year, and over the past two years, we have
been at the front line of our Nation's COVID-19 response.
We, as an organization, know the benefit of biomedical
research, but we have challenges, and I have submitted written
testimony to the subcommittee, but I would just like to cover a
few of those challenges.
One is the ongoing inadequate inclusion of underrepresented
population groups, and the lack of inclusion is not a new
issue. Dealing with the lack of inclusion was a central
recommendation of the 1985 report of Secretary Heckler's Task
Force on Black and Minority Health.
In fact, out of that task force work in 1989, we added a
Hispanic identifier for the first time to the model death
certificate. Adding that data was transformative. It showed us
that regardless of country of heritage Hispanics actually live
longer than non-Hispanic whites, and that is true despite
additional risk factors like diabetes, excess weight, lack of
health insurance.
The one-size model never served anyone, and it only created
distorted models of health. Good science, good epidemiological
practice, and development of safe products require adequate
inclusion of all.
But we are not there. For example, Hispanics represent one
in five persons in the U.S., but we only represent about five
percent of participants in clinical trials. An analysis of a
decade of clinical trials that led to approved cancer drugs
found that only one in ten of those trials reported data for
Hispanics.
And tragically, Hispanics represent only one percent of
individuals in genome-wide association studies.
This lack of inclusion not only limits our ability to
translate biomedical research into healthcare. It also is
ignoring the law. The 1993 NIH Revitalization Act required
inclusion of all groups.
The good news is that we know it works. We have existing
standards of community-based participatory research where
researchers and community members collaborate as equal partners
in design, carrying out the assessment and analysis of
research. We know these standards work and deliver inclusive
science.
For example, the Hispanic Community Health Study at NIHhas
already enrolled 16,000 Hispanic adults from four diverse
communities in a long-term study, and the NIH's All of Us
Research Program is a shining example of inclusion of
community-based participatory research.
Right now with all of us we have over 400,000 participants
and participants from racial and ethnic groups underrepresented
in biomedical research represent a majority of those
participants.
And we have seen the importance of this kind of research in
a response to COVID-19. With All of Us, we were able to quickly
test over 24,000 participant samples to look for antibodies
against SARS-CoV-2, providing significant information to our
Nation's response.
We can do better in terms of inclusion. Three items I would
draw your attention to are: one, the importance of passing the
Diversifying Investigation Via Equitable Research Study for
Everyone, DIVERSE Trials Act.
And I thank many of the members of this committee for their
support of that Act.
Two, we should be mandating inclusion of community-based
participatory research standards, both as a part of FDA review
of new drug applications and as a part of NIH's review of
research funding proposals.
And, thirdly, it is time to require the establishment of a
task force and a report by HHS on efforts for collecting and
analyzing data for population underrepresentation in biomedical
research. It has been 36 years since the report of Secretary
Heckler's Task Force on Black and Minority Health. The time for
an update is long overdue.
I thank you for the opportunity to present testimony to you
here today.
[The prepared statement of Mr. Falcon follows:]
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Ms. Eshoo. Thank you for your excellent testimony.
The Chair is now pleased to recognize Dr. Hood for your 5
minutes of testimony.
STATEMENT OF LEROY HOOD
Dr. Hood. Thank you, Chairman Eshoo, for inviting me to
testify today.
I would also like to commend you for authorizing the ARPA-H
Program. With this initiative, I think we can say comes big,
bold, paradigm-changing effort to transform healthcare.
And one such program is the Beyond Human Genome I will
speak about that will bring actionable outcomes to patients
from the very beginning.
Ms. Eshoo. Dr. Hood, can you move the microphone a little
closer?
Dr. Hood. Yes.
Ms. Eshoo. That is it. Good. Thank you.
Dr. Hood. We face in healthcare today five major
challenges: one, the quality of healthcare; two, the escalating
cost of healthcare; three, an aging population; four, an
explosion of chronic diseases, and five, the need to have
equity in outcomes and opportunity for healthcare.
I am going to discuss this program Beyond the Human Genome
Project, and I will argue it will bring novel approaches for
each of these challenges.
The essence is the following. We know with the data-driven
approach can follow the health trajectory of each individual
over time and optimize their health, minimizing disease, and
bringing people hopefully into their 90s or 100s mentally alert
and physically capable.
To execute this kind of program, I have proposed this
initiative Beyond the Human Genome, and my suggestion is it be
directed initially by a nonprofit I have created called Genome
Health, and that we analyze the genomes and phenomes of a
million persons over a ten-year period, i.e., the second genome
project supported by the Federal Government just as we did for
the first.
The genome is a static, unchanging digital source code of
life. The phenome represents the state of an individual as you
change across your lifetime, and that is determined by three
things: your genome, your lifestyle, and the environmental
exposures that you have.
We can assay this phenome at different times by measuring
and quantifying up to 1,000 or more protein analytes; by
looking at your gut microbiome, the species in your gut that
determine very much about your health; and digital cognition
measurements and digital physical health measurements.
And these are the tools we will use to follow trajectories.
Thus, the longitudinal phenome is what is Beyond the Human
Genome Project, and it provides deep actionable insights into
body and brain.
We have two proofs of principles. One, we have looked at
5,000 people over four years, and by using this data-driven
approach, we have been able to identify a powerful set of
actionable possibilities we call scientific wellness that are
validated by the literature.
Number two, we can create a metric that measures how
rapidly you age and makes suggestions about how to optimize
aging.
And there we have seen now the ability to discover the
earliest transitions of chronic disease years before they
manifest themselves, giving us the opportunity to reverse them
when they are at simple stage.
Our partner Posit has actually done the same kind of thing
for the brain, that is, 40 digital brain measurements that
assess 25 different cognitive elements in a way that measures
them and optimizes them for optimal brain health.
We have three key partners in this endeavor. Guardian
Research Network, they are connected to 100 hospitals, 30
million patients in 13 States across the South and the
Southeast. The important point is they lie across populations,
Latino, Black, and economically disadvantaged, and we plan in
the million-person project to have the proper ratios of all of
these individuals in the program.
The second partner is Providence with coverage in brain
health.
And the third partner just announced to us recently is
Google that has made available to us its search, its cloud, its
digital health, and its hyper scale AI techniques to optimize
the platforms we need to make this program a reality.
If you look around the world, all----
Ms. Eshoo. You need to wrap up, Doctor.
Dr. Hood. Oh, OK. I have got much more to say. We will
answer any questions.
[The prepared statement of Dr. Hood follows:]
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Ms. Eshoo. Wonderful. Thank you very much.
Dr. Minor, it is a pleasure to welcome you to our
Subcommittee. Thank you for your special leadership and if
members think I am biased. You are absolutely right. I am.
You are recognized for 5 minutes for your testimony.
STATEMENT OF LLOYD B. MINOR
Dr. Minor. Thank you.
Good morning, Chairwoman Eshoo, Ranking Member Guthrie,
Chairman Pallone, Ranking Member McMorris Rodgers, and members
of the subcommittee. I am honored to appear before you on
behalf of the Stanford University School of Medicine.
I would like to express my gratitude to Representative
Eshoo for her years of support for Stanford Medicine. I also
thank this committee and Congress for your leadership and
longstanding investments in biomedical research through the
National Institutes of Health.
This bipartisan support of biomedicine is enabling people
to live healthier lives while strengthening our Nation's
economy and its future competitiveness.
At the heart of this progress is basic science, which
provides the foundational knowledge upon which all novel
therapeutics, interventions, and diagnostics are developed.
NIH funding drives our Nation's preeminence in basic
science research and robust investment is critically important
to our health, our economy, and our global standing.
COVID-19 mRNA-based vaccines underscore the extraordinary
return on basic science investment. Beyond the hundreds of
thousands of lives saved, these vaccines, built on decades of
research, are blunting the pandemic's financial burden, which
some economists estimate could reach $16 trillion if left
unchecked.
These vaccines also exemplify the power of preventive
medicine, a central focus of Stanford Medine's precision health
vision. This proactive approach to healthcare seeks to
transform our system of sick care into true healthcare, using
Data Health, using health data, AI, emerging technologies, and
lab-based discovery. Precision Health emphasizes predicting,
preventing, and curing disease precisely, critically in that
order.
More than ever we are challenged by diseases that demand
Precision Health solutions. Consider the growing physical and
economic burdens of mental illness in the United States. From
2009 to 2019, spending on mental health treatment and services
increased 52 percent, reaching $225 billion.
SAINT, or Stanford Accelerated Intelligent Neuromodulation
Therapy, exemplifies one Precision Health approach to
addressing this challenge. This experimental treatment uses a
magnetic coil to directly stimulate underactive parts of the
brain in people with clinical depression.
In October, results from the latest clinical trial showed
that 80 percent of participants went into remission after
receiving the therapy.
NIH grants have made this promising endeavor possible,
among many others at Stanford Medicine, which are detailed in
my written testimony. And though we are fortunate to have this
support, the funding landscape grows ever more competitive.
Since 2000, NIH applications have doubled. The success
rates have declined sharply. Research proposals that Federal
agencies rate as excellent are often not funded due to limited
resources.
For this reason, I urge you to continue to strongly
increase funding for basic research. Supporting this pursuit of
knowledge has produced stunning medical advances, generated new
fields of research, and made the unimaginable possible. And it
will continue to so long as scientists are supported in
exploring the unknown.
Basic research remains the bedrock of innovation, but
translational research is also critical. At Stanford Medicine,
we are encouraged by the promise of a model that supports basic
science and the translation of discovery through creation of an
Advanced Research Project Agency for Health, or ARPA, also
known as ARPA-H.
Legislative efforts to fund and establish ARPA-H, such as
Chairwoman Eshoo's recently introduced authorizing legislation,
recognize the critical importance of our country's biomedical
enterprise and reflect our aspirations to move discoveries from
lab bench to bedside.
To help bridge this gap and accelerate the translation of
promising therapies, we recently launched the Innovative
Medicines Accelerator, or IMA. Serving as an ARPA-H of sorts,
the IMA provides researchers from across Stanford University
access to the technology, resources, expertise, and funding
needed to advance their discoveries to improve human health.
Though only recently formed, the IMA has already had a
significant impact. Originally designed to aid development of
medicines for diseases such as cancer and rare disorders, the
IMA pivoted early in the pandemic, enabling our faculty to
better address the public health crisis.
In the months following the beginning of the pandemic, the
IMA awarded research grants, supported two trials on repurposed
drugs, and initiated two industry-sponsored trials, all the
while building out the infrastructure to enable further
research.
I am more optimistic than ever about the future of
biomedicine in the United States for many reasons. Our world
class academic medical centers, longstanding congressional
support, and a diverse population uniquely position us to
continue to lead the scientific revolution.
However, as competing economies around the world pump money
into biomedical research, remaining at the forefront will
require increased investment in the research that fuels our
technological and scientific progress.
Moreover, it is critical that we continue to invest in the
diversity of our scientific research community and support
those from underrepresented groups, which I described in my
written testimony.
I hope you agree that these are urgent issues for our
Nation's health, our economy, and our standing as a global
leader, and our future.
Thank you.
[The prepared statement of Dr. Minor follows:]
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Ms. Eshoo. Thank you, Dr. Minor.
We will now move to members' questions and the Chair
recognizes herself for 5 minutes to do so.
I will go to Dr. Minor first.
Obviously, I take great pride in representing Stanford
because it is an institution where you, Dr. Minor, and your
colleagues are at the cutting edge of so many fields.
Can you broaden out what you state in your testimony,
about, and tell us what key enabling technologies you think
will play the biggest role in medical innovation in the coming
decade and where we should be directing Federal investments?
Is it AI? Is it quantum computing? Is it something else
completely? Is it all of the above?
And also your testimony, and others as well, discuss the
importance of diversity. What is your experience with what
works for increasing diversity in biomedical and life sciences
research?
Dr. Minor. Well, thank you, Congresswoman Eshoo.
On the first question, and we have heard from the
distinguished experts giving testimony this morning in many of
the areas that I think are important for advancing the future
of biomedical research and improving the health of Americans,
indeed, the health of everyone in the world.
I see the future as really recommending the convergence of
three related but distinct areas, the first being biomedical
science, biotech, med tech., the second being information
science, and the third technology.
It is the fusion and the virtuous triangle created by the
synergies among these three disciplines that I think will
transform the biomedical landscape in the next decade-plus and
will help us to bring about even greater improvements than we
have seen in the past in predicting, preventing, and curing
disease precisely.
On your second question with regard to diversity, I think
the first point and the first important point is being very
intentional about how we as leaders in higher education,
leaders in academic medical center, how we reach out, how we
design training programs, how we create the environment that
attracts and nurtures the careers of young people from diverse
backgrounds.
At Stanford, one of the things we have been able to do,
thanks to generous philanthropic gifts, is to establish a
program that is going to bring people from historically
underrepresented communities to post-baccalaureate programs at
Stanford as well as increase the recruitment of
underrepresented minorities to our faculty and our outreach
through our programs with historically black colleges and
universities, our outreach to institutions that have not had
the opportunities that many institutions in our country have
had because of our country's history of racism and disparities
that we really have to take a proactive stance in curing and
correcting moving forward.
Ms. Eshoo. Thank you, Dr. Minor.
To Dr. Falcon, thank you for your excellent testimony.
I want to stick with the last point around diversity. The
statistics you shared about the lack of diversity in clinical
trials I found really chilling and your testimony points to
several efforts to increase inclusion and diversity in
research.
Why do you think to date the efforts have fallen short so
far? What have we been doing wrong?
There have been efforts. Tell us what you think about that.
Mr. Falcon. Sure. Clearly, we have put out standards. FDA
has standards for inclusion. NIH has standards for inclusion,
and this body has mandated development of those standards.
But standards are not enough. It has come time to move
beyond standards and start mandating inclusion, and we have
seen that work. For example, four years ago, as part of FDA's
appropriations, FDA was required to start reporting data for
inclusion of Hispanics as part of their clinical trials drug
snapshot.
Simply knowing that that data was going to start being
reported publicly made a change. We have seen the numbers for
inclusion of Hispanics in FDA-reviewed clinical trials improve
since that was required.
We need to require that any review of FDA clinical trial
research have a score with regard to inclusion that as those
clinical trials are conducted, they report regularly to FDA
whether or not they are meeting their inclusion standards, and
if not, we should be kicking in new efforts to bring those
trials, while they are in the field, up to the standards of
inclusion.
Similarly, NIH, as part of their review of proposals for
funded research, should have a relevancy score that would look
at its not only inclusion by race, ethnicity, sex, gender, but
also whether or not the studies are powered to report that data
separately.
We have seen, for example, just last month the New England
Journal of Medicine is now going to require all published
research to include a table that specifically states what
populations were included in order to increase diversity in all
of New England Journal of Medicine's publications.
Ms. Eshoo. I think I have to ask you to stop because I am
way over my time, but each of us has 5 minutes to ask
questions. I could easily use a half hour to field questions to
those that have testified today, but we can submit question,
written questions, to our witnesses.
So thank you to each one of you.
The Chair now recognizes Mr. Guthrie, the wonderful Ranking
Member of our Subcommittee, for his 5 minutes to ask questions.
Mr. Guthrie. Thank you, Madam Chair. I appreciate it.
And before I begin my questions, I want to mention how
important it is for the Biden administration to keep crucial
components of the Trump administration era, Medical Coverage
for Innovative Technology, or the MCIT, interim final rule
intact to ensure timely access for care for Medicare
beneficiaries.
I recently read a letter with over 660 bipartisan members
calling for the Biden administration to work with industry
partners to resolve outstanding implementation issues while
providing temporary coverage, both prospective and retroactive,
of these approved devices to allow patients access to care.
Dr. Abernethy, how can we build off policies like MCIT, the
MCIT rule to ensure we are incentivizing future investments in
innovative technologies and apply requisite data to make
informed coverage decisions and subsequently advancing future
investments in novel healthcare technologies?
Dr. Abernethy. Thank you, Mr. Guthrie. This is an important
question.
Really as we think about the goal of getting innovative
technologies as another intervention to patients, the real
question is how do we make sure we have the mechanisms in place
to provide those innovative therapies and also the mechanisms
in place to evaluate how they perform, including in the real
world.
I think the MCIT discussion or proposal really brought to
the forefront that critical discussion that, for Medicare
beneficiaries and beyond, how do we really think beyond even
the approval state to continue to evaluate how these
interventions perform for all people.
I think one of the things that I saw when I was at FDA, the
importance also that if we are going to have continued
evaluation of interventions, that CMS has the capabilities and
resources to also support that evaluation across time.
Mr. Guthrie. Unlike better integration of the FDA approval
that you were involved in, CMS coverage and data collection
increase our potential for truly personalized medicine, and
what more can Congress do to make that happen?
Dr. Abernethy. This is certainly an area, sir, that was of
great interest to me when I was in FDA. We were thinking about
how could we use data, the same data, in support of questions
of safety and effectiveness that FDA has while also questions
about coverage and implementation for Medicare beneficiaries
that CMS has.
I see that we can really help FDA and CMS understand the
value of working together with the tools needed and help to
develop both the tools and the methodologies needed to do that
and also to be able to evaluate data from those different
perspectives across time.
There is a huge opportunity here to not only make this
continuous evaluation of interventions become a reality, but
also develop the method that we can apply to other parts of the
personalized healthcare setting.
Mr. Guthrie. OK. Thank you.
Dr. Hood, you mentioned your partners as you were moving
forward. I think some nonprofit and Google for-profit. Can you
explain why it is so important to take a public-private
approach whenever we are advancing complex healthcare research
initiatives that involve large data sets?
Dr. Hood. [Microphone turned off.]
Ms. Eshoo. Doctor, you need to turn your microphone on.
Dr. Hood. Whether----
Ms. Eshoo. There you go.
Dr. Hood. It is going to require fundamental changes in how
industry looks at this, and we see this project as an
opportunity to recruit industry in to catalyze this change,
have them be a participant of it, and put it in the context of
how in the future they are going to direct their efforts to be
a part of this big swing from disease orientation to wellness
orientation.
And I will tell you it is going to come. It is a question
of how long, and together we can really accelerate this
process.
The really important point is if you move to wellness, you
create enormous cost savings, and you can transfer what you
have saved into creating active opportunities to really
emphasize wellness and emphasize prevention.
And I think this is the opportunity Google saw. This is the
opportunity Kavoit, one of our partners, has seen, and I think
this is the opportunity that Guardian Research Network and
Posit have all seen. They will be a part of the new revolution
in medicine that is pointed toward healthcare.
And the only question is are you going to get in at the
leading edge or are you going to be dragged in behind.
Mr. Guthrie. Thank you.
My time has expired.
Ms. Eshoo. The gentleman yields back.
The Chair is pleased to recognize the Chairman of Full
Committee, Mr. Pallone, for his 5 minutes of questions.
Mr. Pallone. Thank you, Chairwoman Eshoo.
Modern medical researchers have a tremendous amount of data
available to them, but not all data is relevant, reliable, or
useful evidence for proving a product is safe and effective to
regulators.
So, Dr. Abernethy, my questions are to you. I wanted to ask
you about how FDA can determine the appropriate use for
available data when making regulatory decisions.
And first, you mentioned in your testimony that considering
real-world data in addition to randomized clinical trial data
can improve scientific decision making.
However, real-world data introduces a number of variables,
including data sources and quality, and while one size does not
always fit all, it's important for regulators like FDA to have
standards for data quality before considering it in the context
of a regulatory decision.
So, let me ask first how can FDA develop clear standards
for how the agency should assess real-world data and should
these standards differ by the type or source of data?
Dr. Abernethy. Thank you very much for this important
question.
Well, so first of all, 21st Century Cures compelled FDA to
start to sort out how to use real-world data; did so within the
context of two critical use cases, extending labels, making
label expansions and also post marketing commitments and
requirements.
In December of 2018, FDA, therefore, put forward a
framework of how to start to consider to use real-world data,
specifically understanding data that are fit for purpose,
understanding analyses are fit for purpose, and really
demonstration of when these analyses could be done.
But this was not FDA saying we will use real-world data,
but we need to learn how.
In September of this year, FDA put forth a guidance on the
expectations around using real-world data in the context of
regulatory decision making, and that guidance came back to
exactly what you said, Mr. Pallone, the importance of
understanding data quality and understanding when data can be
fit for purpose, for especially high-risk questions and
concerns that FDA has.
I still think we have a lot of work to do to develop the
methods, develop the data sets, to develop an understanding of
when we can leverage both real-world data and real-world data
combined with clinical trial data to make confident decisions.
But I see progress, and I see that we have the opportunity
to do this better and better in the future, and FDA will be a
critical part of making that happen.
Mr. Pallone. Well, thank you.
Now, your testimony also mentioned the use of patient
experience data to generate evidence. However, here, too, FDA
has reported that patient experience data submitted with
applications can vary widely in quality and completeness and
relevance.
So is there anything that can be done? Well, two questions.
Is there anything that can be done to improve the quality and
relevance of patient experience data?
And how can regulators ensure the patient experience data
is free from bias?
Dr. Abernethy. So patient experience data is critical for
helping to put the medical interventions into context and also
understanding how do medical interventions change our
experience within the context of our own health and, our own
lives.
The challenge with patient experience data is that it is by
definition subjective that is about experience, and so we have
critical work to do to both make sure that we can collect
patient experience information, for example, direct patient
reports, in as complete of a way as possible.
So leveraging, for example, software technologies that meet
patients where they are and allow the collection of complete
information directly from people in their homes rather than
needing to come to clinic.
We need to make sure that as patient personal information
or patient experience is being collected, we understand the
reliability and the credibility of that information and how it,
for example, corresponds with increasing fatigue and decreasing
ability to function and move along in real life.
And then how can that information be used to make credible
assessments of the performance of medical interventions?
This is an area that we cannot let go but is going to need
continued technology development, scientific methodology
development, and understanding of how to integrate that into
regulatory decisions.
Mr. Pallone. Now, you may have touched upon the last
question, but let me ask it an easy way. Well, what lines, if
any, should FDA draw around patient experience data?
And, you know, should it be considered in regulatory
decisions?
Dr. Abernethy. So, first of all, the ability to consider
patient experience data should be in the context of any
regulatory decision, at least to help understand and put that
decision into context.
Whether patient experience data, patient reported
information is the specific endpoint that the regulator makes
the decision on for that particular treatment really depends on
the credibility and essentially the qualification of that
endpoint using rigorous scientific methods.
But the ability to incorporate the understanding of how an
intervention works for real people in their experience can be a
part of the complete story for any particular evaluation.
Mr. Pallone. Oh, thank you so much.
Thank you, Madam Chair.
Ms. Eshoo. The gentleman yields back.
The Chair is pleased to recognize the Ranking Member of the
Full Committee, Mrs. McMorris Rodgers, for her 5 minutes of
questions.
Mrs. Rodgers. Thank you, Madam Chair.
Dr. Hood, your innovative approach to predictive
preventive, personalized, and participatory framework that
embraces wellness and early detection is fascinating. It is
encouraging to imagine a future in the United States where
biomedical leadership syncs up with a community-based,
personalized and preventative system to capitalize on our
country's leadership in biomedical foundations.
I am concerned that that foundation is in jeopardy and
potentially being dismantled by the proposed government price
controls. It selectively applies a negotiating process to
certain drugs at staggered intervals and cumbersomely applies
these price controls to new indications as well.
Would you share more with us of what you mentioned in your
testimony as developing clinical research approaches and data
resources that are disease agnostic, and any insights you might
be willing to give as far as the impact of price controls that
push investment in certain diseases over others and certain
indications over others, and how much that might disrupt the
opportunity ahead of us?
Ms. Eshoo. Doctor, your microphone please.
Dr. Hood. [Microphone turned off.]
Ms. Eshoo. Is your microphone on, Doctor? Oh, OK.
Dr. Hood. Can you hear me now?
Ms. Eshoo. Yes.
Dr. Hood. OK. Sorry.
Ms. Eshoo. You have to turn it on each time.
Dr. Hood. With regard to drug-controlled prices, I think
there is a much better way to approach it, and I will tell you.
Out of the data we are going to be generating on each
individual, we will be able to get biomarkers that tell which
patients can respond to which drugs.
The fact is if you take the ten most common drugs today
sold in the U.S., less than ten percent of the patients respond
properly to those drugs.
With these biomarkers, we will be able to match patients
against drugs and get 100 percent results.
The cost of drugs today is roughly $600 billion a year. So
if we could save 90 percent of that, and that is, you know, a
hypothetical stream, that would be a very powerful way to deal
with drug efficiency and to think about changing the price of
drugs. OK?
I think the second question of this program being disease
agnostic is really an important point because, for example,
there are 7,000 rare diseases that have been defined to date.
Ten percent of the American population has one of these rare
diseases. Eighty percent of those diseases comes from a single
defective gene in the cases that have been studied.
And often if you know the gene at birth, you can begin to
make accommodations that do not imprison the patient in that
defective gene for the rest of their life.
And we will be looking at entire populations with no bias
for disease whatsoever, and we will identify the correlations,
and we will have the data to begin getting even deeper insights
into therapies for all of these rare diseases.
Exactly the same is true for rare recessive mandilion
diseases like hemochromatosis or so forth. At birth we will be
able to diagnose those and we will be able to make
modifications that will improve the health.
One of the things that is really missing in today's data is
the deep data approach from the infant up to 16. Those early
stages of childhood development we have very little data on,
and if we were to have a similar program on that initiative, it
could be transformational for how we deal with kids.
So the agnostic disease looking kind of at everything--and
you can do that with a million patients, that is why we chose
the number--is, I think, going to transform the whole landscape
of how we deal with diseases.
Mrs. Rodgers. That is great. I appreciate your insights.
As the Chair said, I think we all have more questions that
we could ask. I think I am going to yield back at this point
and look forward to hearing from more.
Thanks.
Ms. Eshoo. The gentlewoman yields back.
The Chair is now pleased to recognize the gentlewoman from
California, Ms. Matsui, for her 5 minutes of questions.
Ms. Matsui. Thank you very much, Madam Chair, and I want to
thank the witnesses for being with us here today to testify at
this very important hearing.
I want to ask about something that we have not talked about
here. Advancement in the field of computer science and
artificial intelligence paves the way for healthcare
innovation, delivery, and outcomes.
For example, just a few years ago researchers at UC-Davis
Health and UC-San Francisco developed an artificial
intelligence algorithm to teach a computer to define the
hallmarks of Alzheimer's disease and human brain tissue.
During the pandemic, engineering researchers for UC-Davis
demonstrated that artificial intelligence algorithms may be
useful in protecting newly infected COVID-19 patients on
ventilators from developing serious long-term lung injuries.
These are really noteworthy achievements and hold a lot of
promise for the future of healthcare. However, challenges
remain in addressing biases within the machine learning and
artificial intelligence.
Dr. Butte, there have been calls for increased
accountability and transparency in coding and computer science.
What biases exist in artificial intelligence and what are their
effects onhealthcare?
Dr. Butte.
Dr. Butte. All right. Thank you. Great. Thank you for the
question.
So all of us as computational innovators have to do better
by working with data in an open, fair, accountable, and
governed way. And as your question states, collecting more data
is not just the challenge. Making sure we collect data in a
fair, responsible, and transparent way and ensuring the data
collected properly represents all of our patients is of utmost
importance.
As an analogy that I use, imagine considering the purchase
of a self-driving car that was only trained on roads in
Mountain View, California. You would never accept such a car
that did not know how to run in deep snow or blinding rain.
So similarly, we should never utilize a self-driving
medical algorithm trained only on a quarter of American
patients.
Ms. Matsui. OK.
Dr. Butte. Go ahead.
Ms. Matsui. Mr. Butte, thank you.
I can understand that analogy, but how can these biases be
addressed and eventually eliminated?
Dr. Butte. We have got to know; we should know and document
what is in the algorithms we are building, sharing, and buying,
and ensure that they are trained on data covering the diversity
of Americans. That is of utmost importance.
Ms. Matsui. OK.
Dr. Butte. Further, we need to make sure they are
engineered by data scientists that cover the diversity of
America as well, and I think the NIH AIM-AHEAD program is a
great start by NIH and should be continued, renewed, and grown.
Ms. Matsui. OK. Thank you, Dr. Butte.
And while I have you, I want to quickly touch on related
and important work coming from my district. I am enormously
proud of UC-Davis, how it participates in NIH and all of its
precision medicine network.
Can you share how the use of data from initiatives like
All of Us is improving care for patients?
How do we make sure all of these networks are tools to
address health disparities?
Dr. Butte. I am really proud of what we have accomplished
in the All of Us Research Program in the State of California.
Dr. Anton Colburn and others have led this effort to register
nearly 50,000 participants so far at UC-Davis and across the UC
Health System, and importantly 80 percent are considered
underrepresented in biomedical research and 53 percent are
racial minorities.
We ourselves have already used this data to study how
specific drugs are being used and prescribed in Americans,
especially looking at the differences in how these drugs are
prescribed across races and ethnicities.
We can study these drugs like antihypertensives and
diabetes drugs and compare them with each other to find the
better, safer, and more cost-effective drugs.
Ms. Matsui. OK. Thank you, Dr. Butte, very much.
A technology like this brings tremendous potential to
advancing health equities.
I have a question here. Dr. Abernethy, I want to follow up
on the chairman's questions about patient experience data. This
Congress I have introduced the BENEFIT Act, legislation that
would require FDA to consider patient experience and patient-
focused drug development, PFDD, data within the benefit-risk
framework for drug approval.
Stakeholders, particularly patient groups play a big role
in collecting this data. You mentioned the importance of
credibility in collecting patient experience information. What
suggestions do you have for how these groups can better work
with FDA to ensure data collected is useful to overall
evaluations.
Dr. Abernethy, quickly.
Dr. Abernethy. Thank you.
Patient experience data is critical, and the patient
advocacy space as well as public-private partnerships that
incorporate FDA is critical to not only make sure it is clear
what FDA needs to understand better and how to prioritize the
patient's voice, but also to figure out how do we develop the
tools and methodologies going forward in the future. The
patient voice is critical.
Ms. Matsui. OK. Thank you very much.
And I yield back.
Ms. Eshoo. The gentlewoman yields back.
It is a pleasure to recognize the gentleman from Michigan,
Mr. Upton, who has been at the forefront of helping to make the
investments so that we have a brighter health future in our
country.
You are recognized for your 5 minutes, sir.
Mr. Upton. Well, thank you, Madam Chair.
Earlier this week I had the chance to visit with some of
the NIH folks and others, and I am so excited about what is
coming about.
And for me in my district, it just so happens Pfizer is my
largest employer, and that is where they package and
manufacture and put together the vaccine, and because of the
21st Century Cures Act, it is pretty clear that we actually got
a vaccine approved probably eight or ten months earlier than it
otherwise would have been, saving literally hundreds of
thousands of Americans.
So it is really exciting for me to be there as they play
such a prominent role.
Dr. Abernethy, as a follow-up to the 21st Century Cures
Act, as you know, we have introduced H.R. 6000, 2.0. It has a
real focus on increasing the use of real-world evidence in the
regulatory decision-making.
Can you tell us a little bit about how we can further
maximize and expand the use of RWE to help new treatments for
patients?
Dr. Abernethy. Thank you very much.
Thank you for 21st Century Cures and----
Mr. Upton. You can thank everybody here. It passed in this
committee 53 to nothing.
Dr. Abernethy. I am so proud to be sitting with all of you
here today. So thank you for 21st Century Cures.
And thank you for the conversation around the potential of
extending into the future.
Real world data can be a part of the conversation and the
decision making at FDA and really across personalized
healthcare in a number of ways.
First of all, as highlighted, we need to continue to
develop the data sets and make sure the data sets are
appropriately fit for purpose.
We need to make sure that we continue innovation in that
space and that FDA has the opportunity to see not only what is
possible today but comment on and continue to point forward
with what types of innovation in the data space we are going to
need for tomorrow, including incorporating patient experience
data.
We also need to continue to innovate on the analyses that
are possible going into the future. So how do we make sure that
we have confident output that can then inform not only
decisions based on real world data, but decisions that can
combine real world data with clinical trial data so we can get
a complete 360-degree view using totality of the evidence?
Also importantly I think as seen, we can now start to think
about how do we leverage solutions such as decentralized
clinical trial solutions that meet patients where they are in
the home, but also pair that information with information
coming in directly from, for example, clinical research sites
that we are able to get a complete view of the patient and the
data really through the ability to leverage technology.
Mr. Upton. Well, that is what we hope to accomplish with
our provisions regarding RWD.
Quickly, one common refrain that we have heard so often
from patient groups is that CMS has taken such a long time to
make payment decisions on new drugs that make it through the
approval process at FDA.
Are there ways or what ways do you see where we can have
FDA and CMS better communicate so that once a drug is approved
by the FDA, in fact, it will make it through the payment
process quicker?
Dr. Abernethy. So certainly the ability to make sure that
Medicare beneficiaries have access to interventions that work
is critical.
One thing that I think that we can do is align CMS and FDA
thinking, especially through data. How can the same data sets
that FDA is already requesting for continued evaluation of
interventions post approval now also provide the confidence to
CMS that they need to understand how this intervention is going
to perform across time for Medicare beneficiaries?
I think we can begin to develop solutions that leverage the
same data for both tasks, but really give capabilities to those
two different agencies to do their work.
Mr. Upton. Again, I hope that we can see that happen when
we get this thing moving along.
Dr. Hood, a quick question as a follow-up to Ms. Rodgers'
question about the All of Us initiative.
You talked a little bit about youngsters one to 16 and how
it is so important to measure that data. Where are you in terms
of not the demographics but the actual numbers of ages one to
16?
What emphasis do you have on collecting that database from
those youngest, I will say?
Dr. Hood. Well, I can tell you we have just started talking
with Kaiser about the possibility of setting up a longitudinal
phenome analysis of just that population group, to begin taking
in some of the data that we have talked about here.
I would like to add just one comment about real-world data,
and that is one should realize the longitudinal phenome
analysis, for example, of 6,000 different blood elements gives
you entirely new spectrum of real-world data, and what those
6,000 elements let us do, because blood base all of the organs
and they secrete informational molecules into the blood, is
having a global assessment of your internal as well as your
external health and so forth.
So real-world data is going to expand exponentially in the
future as we bring more and more people into the longitudinal
phenome analysis, and the pediatric examples, I think, will
just change the whole spectrum of how we view disease in the
pediatric population.
Mr. Upton. Thanks very much.
I yield back.
Ms. Eshoo. The gentleman yields back.
It is a pleasure to recognize the gentlewoman from Florida,
Ms. Castor, for your 5 minutes of questions.
Ms. Castor. Well, thank you, Chair Eshoo, for holding this
important hearing.
Thank you to our witnesses for sharing your expertise. It
gives us an opportunity to mark the 20th anniversary of the
Human Genome Project. So to Dr. Hood and all of the scientists
and all of the policy makers who play an important role, thank
you for your remarkable contribution.
And when you look out, think about the next 20 years. It is
very important for us now to concentrate on how we combine
this, one of the greatest feats in recent scientific history,
the sequencing of the human genome, now with advances in
bioengineering, biomedicine, to prevent and cure disease. So
the opportunities are enormous here.
I am fortunate to represent a top health innovation center
in the Tampa Bay area anchored by the Moffitt Cancer Center and
the University of South Florida Health.
Moffitt has been leading the charge on precision medicine
and immunotherapy for cancer patients. For example, scientists
at Moffitt have been working on a range of research to better
understand how the immune system can successfully fight cancer.
But I have also heard from folks who think we are making a
lot of advances on precision medicine when it comes to cancer
but maybe not other diseases. Dr. Thomas McDonald from USF
highlighted to me that while we have made great strides in
personalized therapies for cancers, we are kind of lagging
behind in other diseases.
His laboratory established a cardiogenic program top search
for novel therapeutics for hereditary heart disease.
Do you agree that we need to focus more maybe?
Dr. Abernethy, I will start with you.
How do we ensure research tackles broadest, deepest health
problems?
Where are we headed?
Dr. Abernethy. Thank you very much.
As a fellow Floridian, I appreciate this question.
I certainly think that we need to take the lessons learned
in the cancer and rare disease space and now start to apply to
all therapeutic areas.
Practically speaking, it may not always be the same science
and the same methodologies, but the premise, the premise to
individualize and to tailor, to blend the best of what we
understand of biology on a personhood together to figure out
treatments, I think we can take to all therapeutic areas.
Ms. Castor. So NIH, they had to strike this balance before,
and I guess our funding decisions will address that as well.
And maybe, Dr. Minor, you could address that a little bit,
and I also want you to focus in on the advances due to CRISPR
technology.
I have been fascinated by this. I have met with Dr.
Jennifer Doudna at Cal-Berkeley across the bay, rap SU, about
the significant or the potential advancements across biology,
medicine, agriculture.
If you just take a peek at what is going on now with
CRISPR, sickle cell clinical trials; mosquitoes, how we reduce
the spread of malaria; impact of climate change on crops in
agriculture, vision and blindness.
Dr. Minor, talk to us about this and the enormous
opportunities. Is ARPA-H a way to help advance CRISPR
technology and all of the advances there?
And what else do we need to keep in mind?
Dr. Minor. Well, thank you, Congresswoman Castor.
Yes, I think ARPA-H offers tremendous promise in making the
types of advances that you have described.
And one of the reasons I think that we have seen so much
progress as you outlined from the great institution in your
district and other places in cancer immunotherapy is that for
basic research, we have a much better understanding today than
we did as recently as five years ago of the immune mechanisms
and cancer, and that enabled the development of these
immunotherapies in many other diseases, most notably
degenerative neurological diseases.
We do not have nearly as complete an understanding of the
basic mechanisms, and that underscores again the importance of
basic science research in driving the advances that then fuel
better therapeutics and diagnostics in the future.
I agree with you that CRISPR is truly transformative
technology. We want to make sure that it is used ethically and
with appropriate standards and regulation from the government
and certainly with input from bioethicists.
But as you so well described, it has the potential to
improve our development of crops, to extend the productivity of
so many things in the agricultural space as well as to treat
both genetic, clear monogenic disorders that Dr. Hood
described, but also treat disorders that have a genetic basis,
of which most do, such as cancer, degenerative neurological
diseases, to be able to treat those much more effectively than
we have in the past.
We have only begun to scratch the surface, and I am very
excited about the future of CRISPR.
Ms. Castor. Thank you very much.
Ms. Eshoo. The gentlewoman yields back.
She mentioned UC-Berkeley. I'm very fond of saying that I
get to represent the greatest private university in the world
that lives in the shadow of the greatest public university in
the world.
With that I recognize one of the doctors on our
subcommittee, Dr. Burgess, for his 5 minutes of questions.
Mr. Burgess. I thank the chair.
Dr. Abernethy thank you for being with us today, and thank
you for your time. Speaking with your colleagues at Verily and
Alphabet earlier this year on a Zoom call certainly gave me
some significant insight, and I appreciate the fact that you
have spent some time in the belly of the beast over at the FDA,
and you may have some insights that you are able to share with
us.
Operation Warp Speed I think we all acknowledge was a
significant win, and a vaccine was worked on and produced
within what seemed to be a very short period of time, but are
there lessons that you can help us with?
We have got an FDA reauthorization bill coming up I mean
literally in hours where we are going to need to be working on,
and it has got to be done by the end of July. So the time frame
is fairly short, but the good news is that gives us an
opportunity to talk to your former colleagues over at the FDA a
lot about things because the user fee agreements are what
allows for probably 75 percent of their funding.
So help us with the lessons learned from Operation Warp
Speed and how we might incorporate that into our better
understanding of the work of the FDA.
Dr. Abernethy. Thank you very much, sir.
The interesting thing as we look back across the pandemic,
we have seen some incredible work that has really helped with
managing the care of our population, but also pointed towards
the future.
As I reflect on your question, I think that this story has
taught us the importance of not only making sure that our
discovery engines are working as quickly as possible and we are
scaling the regulatory apparatus, but we have really not only
revolutionized clinical trials, but really taken this fast
forward so that we can make sure that clinical trials are being
conducted as quickly as possible and also incorporate and meet
all people where they are.
Another thing that is going to be important as we think
about FDA going forward is to make sure that FDA is enabled
with the tools of evaluating the totality of the evidence,
clinical trials data, and real-world data, and we certainly saw
that in the context of the pandemic.
We also saw in the context of the pandemic the opportunity
to make sure that when possible and when needed that we can
move forward with thinking about manufacturing early so that we
can be ready with solutions when people need it.
So I think there is a number of things to learn from the
pandemic.
Mr. Burgess. Thank you.
And we will certainly look forward to your continued
participation as we go through that process.
Dr. Hood, I want to just ask you. You mentioned you were
working on--and thanks for bringing up the four Ps, by the way.
My very first meeting when I started this committee years ago
was with Dr. Zerhouni, and he articulated about the four Ps,
and the participatory part of that is probably one of the most
important things.
But you mentioned the ability to optimize aging, and I am
just wondering do you have an app for that.
Dr. Hood. The optimization for aging.
Mr. Burgess. With your longitudinal look at the genome with
the phenome?
Dr. Hood. Yes. The algorithm that we have developed right
now employs about 50 metabolites, and those 50 metabolites not
only give us the ability to estimate your global age, OK, but
we can estimate the age of your major organs, your immune
system, your heart, and your kidney, and so forth.
And from the metabolites----
Mr. Burgess. Let me restate the question. Do you have an
app for that? I may not live long enough to hear the answer to
your question.
Dr. Hood. Yes, yes. We do have an app for it, and I can
send you where you can get your biological age done, but the
key thing is to do it repeatedly because you want to show that
you are going down rather than going up.
Mr. Burgess. I do not need to know that. I actually know
that.
Dr. Minor, I just wanted to ask you quickly. You brought up
the ARPA-H and, as you know, there are a couple of proposals
before our committee introduced by members, and you have heard
the testimony of Dr. Abernethy. One of the things that we are
going to need the most amount of help with, as I see, is just
what the up-front funding and the basic research, but you
mentioned translational, how you get through the guardians at
the FDA, how you get past the bureaucrats at CMS for coverage
determinations.
All of these things almost need to happen simultaneously,
and now we have set the public expectation with Warp Speed that
we can, in fact, get things done quickly.
So could you help us with that?
Dr. Minor. Well, Congressman, I think you described it
really well. I think we did set the expectation with Warp
Speed. We showed what is possible by bringing together the
various branches of government, various government agencies in
collaboration with public institutions and with industry.
I think that expectation will continue, and so far as the
success of ARPA-H, you know, DARPA probably serves as a good
model overall in that DARPA serves the Defense Department, is
charged with innovating in ways that will improve the ability
to defend our country and protect our troops.
Similarly, ARPA-H will likely have a similar charge for
doing the same thing in healthcare and the health and wellbeing
of America.
I am optimistic that because of what we demonstrated is
possible during COVID with the development and deployment of
vaccine that a similar mandate will exist for ARPA-H.
Mr. Burgess. Thank you.
Madam Chair, I yield back.
Ms. Eshoo. The gentleman yields back.
The Chair now is pleased to recognize the gentleman from
Maryland, Mr. Sarbanes, for your 5 minutes of questions.
Mr. Sarbanes. Madam Chair, thanks very much, and thanks for
the hearing.
Like all of us, I hear from constituents about the need to
fund and advance research on a variety of diseases, whether it
is cancer, diabetes, or other diseases and what we can do to
detect and treat and cure those diseases, improve the quality
of life for our families and communities.
I also bring the perspective of being an original cosponsor
of the critical bill, the Henrietta Lacks Enhancing Cancer
Research Act, which as you know directed the GAO to complete a
study and provide recommendations on how Federal agencies can
address barriers to participation for underrepresented
populations in federally funded cancer clinical trials. And
this bill became law earlier this year.
Mr. Falcon, could you speak to the critical need, again,
for increased diversity that you laid out in your testimony?
And particularly the importance of the All of Us Research
Program which we have heard a little bit about today already.
But this program aims to build a diverse biomedical
research database on health information. How can that program,
the All of Us Research Program, inform our knowledge of
differences between and among certain types of cancer or other
diseases?
Mr. Falcon. Well, first of all, Congressman Sarbanes, I
would be remiss if I did not thank you and the committee for
the Henrietta Lacks Act because it has helped put a focus on
the importance of diversity.
Yes, All of Us is an important national resource. We are
working to get to one million persons enrolled. We are a little
bit over 400,000. There are over 300,000 bio samples collected,
over a quarter million electronic health records collected.
But the central feature is that equity was at the
organizational core of All of Us. It was part of the mission
statement. It was part of the metrics that were measured. It
was part of the funding for community engagement partners.
It showed that equity can be done in health research
because over half of the participants are members of racial and
ethnic groups that have been previously underrepresented in
biomedical research.
This was a significant change, and it can happen with ARPA-
H as well. I am pleased to see that the proposals for APRA-H do
have equity explicitly stated as a goal of APRA-H, and that
kind of organizational capacity around equity makes research
happen.
I would also point out to the committee, and I think
Congressman Ruiz and others, that as clinical trials are
advancing, we are moving more and more to decentralized
clinical trials.
But as we make that move, we are creating another barrier
for underrepresented populations, and that those decentralized
trials depend in large part on technology and being able to
have access to technology.
And we do need to take care and create some safe harbors
for clinical trial sponsors to be able to provide funding, to
provide the technology, but also funding in order to
participate around transportation, childcare, and all those
other issues as we move to decentralize trials.
Mr. Sarbanes. I appreciate very much that observation
because, as you know, we can suffer up here a lot from
unintended consequences. So as we rally around the idea of
decentralizing, it is important to have you flagging some of
the new obstacles that can present, and we have to have
strategies for getting over and around those obstacles.
Dr. Abernethy, as we continue to collect health information
and employ these innovative technologies, can you speak a
little bit about the ethical or privacy considerations that we
should be thinking about in terms of future policy making?
Dr. Abernethy. Thank you very much.
As we think about leveraging new solutions and technologies
to make clinical trials easier to participate in, we need to
make sure that we stick to our core principles of privacy, of
security, and making sure people, real people, understand what
they are participating in, and have a full understanding across
the time of their participation, including how their
information is going to be used.
I think we should be leveraging the best of what software
development and technology has to offer with respect to user
experience and user design so we can get that right.
And we also need to reduce the burden of participation for
all people in order to be able to be in clinical trials.
Mr. Sarbanes. Thanks very much.
As I conclude, Madam Chair, I would note there is some
intersection there with that last observation with Dr. Falcon's
or Mr. Falcon's observation.
As we decentralize, we have got to make sure that people
who are participating in these trials understand fully what
that participation means and that their interests are being
absolutely protected.
With that I yield back.
Ms. Eshoo. Excellent points.
The gentleman yields back.
The Chair is pleased to recognize the gentleman from
Virginia, Mr. Griffith, for his 5 minutes of questions.
Mr. Griffith. Thank you very much, Madam Chair.
Thanks to all of the witnesses here today.
I have been listening to the testimony, read some of the
written testimony as well, and want to note that several of you
who have expressed support for bills that would increase NIH
funding.
I am a long-time supporter of funding for NIH because of
the important work they do, and as my fellow committee members
know, I am currently participating in an NIH study on those who
had mild cases of coronavirus or COVID-19, and I think the NIH
team is great. They have been doing great work on that. I have
been very impressed.
However, in my role working on the Oversight and
Investigations Subcommittee of this committee and on this
committee, I have been disappointed by the agency's disregard
of requests for information and documents on the origins of
COVID-19.
Members of this committee have sent five oversight letters
to the NIH and have not received a response to our concerns and
questions.
They have been unresponsive to Congress on vital questions,
and that is not acceptable. I think any discussion about
additional funding and new programs has to be coupled with an
understanding that while the NIH has the job to do the science,
we have the job to do the oversight and to make sure that what
is being done is being done in accordance with the principles
and the guidelines and so forth that we have set out.
And if somebody has made a mistake, and mistakes will
happen in every organization, that we have the ability to look
at it and try to figure out how do we do it better going
forward.
That is our job, and so I have been very concerned about
that, and here is the problem when we do not get answers. I
know you all cannot answer any questions. I am going on a
little diatribe. I apologize to you all for that.
But here is the problem that I have and I think lots of
others have. If we cannot get somebody to get us answers to the
questions that we have, then we must assume that the answer is
the worst-case scenario, and then we have to act accordingly.
All right. That being said, I am done with the diatribe and
I appreciate you all letting me do that.
Dr. Abernethy, you talked extensively about personalized
medicine and incorporation of real-world data into medical
care. So I am going to go right into what would have been high
tech ten years ago but is now becoming low tech.
My watch, a friend of mine has a better one than I do or
his son did, and it picked up and I do not know how; probably
with AI that you talked about in your testimony as well. It
picked up an afib about a year ago. Went in to see the doctors
immediately. They could not find anything. Then he went for a
much more extensive test. Sure enough they found a heart valve
problem, and even though the family had been told it was not
genetic, it is the same heart valve problem that killed his
mother a couple decades ago.
They were able to go in because they identified it. They
were able to go in and fix it, and he is fine. Everything is
going to be great.
But that came from a wearable. What are your thoughts on
wearables?
And I also would note that you indicated with our studies
we have to go to the people. Is it possible we can use
wearables in that regard, too, to get the data instantly, but
also get feedback from wearables?
Dr. Abernethy. Thank you for the question about wearables.
Practically speaking, being able to leverage all available
data, including the sensors in our watches, is going to be a
critical way of being able to not only collect information
across time, but also do so in a way that helps to personalize,
as you described that story being able to leverage the heart-
related information to now direct care, but also do so in a way
that reduces the burden of a person having to fill in a form or
enter information through secondary purposes.
We have a lot of work to do though to make sure that the
information coming, for example, from a wearable is reliable,
does not create biases that would amplify disparities in
ourhealthcare system, and that we understand where to switch
out data points, such as an endpoint from a wearable in the
sensor in your watch as opposed to the way we traditionally
conduct clinical trials.
I suspect we have got a lot of work to do across time, but
this is a huge area of opportunity.
Mr. Griffith. Well, I envision we may have the opportunity
to cut down on the number of visits, say, to the NIH,
particularly if you come from a distance and you are perhaps
living in a rural area. We could combine maybe the wearables
with telemedicine as well, and things I probably have not even
thought of yet.
But thank you all so much for being here.
Yes? Did you have another comment? I have got a couple of
seconds.
Dr. Abernethy. I was just about to say that the combination
of telemedicine, wearables, et cetera, not only help us get
more information data. They meet people where they are, and
they reduce the burden of participation. We see that as really
important for the future.
Mr. Griffith. Absolutely.
I yield back. Thank you, Madam Chair.
Ms. Eshoo. The gentleman yields back.
The Chair is pleased to recognize the gentleman from
Oregon, Mr. Schrader, for your 5 minutes of questions.
Mr. Schrader. Thank you very much, Madam Chairwoman. I
really appreciate it.
I guess I will start off by making some comments on the
prescription drug bill that is incorporated in the PPP plan
because of the inaccuracies, inflation comments, unfortunately
that I have heard here today regarding what it is all about.
That prescription drug bill is an excellent, excellent balance
between H.R. 3 and what others would perhaps like to do. It
incorporates most of H.R. 19 that my colleagues on the other
side of the aisle have referenced.
Our problem solvers group that is made up of Republicans
and Democrats looked at H.R. 19, looked at H.R. 3, tried to
figure out a good balance between these to not stifle
innovation, but make sure that people could afford the
medication that we are talking about here today that are truly
lifesaving for a lot of folks that are out there.
It incorporates Part D redesign, the insulin caps, frankly,
the biosimilar ASP plus eight provisions, trying to get at some
of the key gaming of the system that goes on by the
pharmaceutical industry, but at the same time respecting the
fact it costs a lot of money to innovate these drugs.
I want that to occur in America. I want it to occur in the
districts that some of these members in this room represent,
and I think we found that balance.
The idea that it is going to cut off all innovation is
ridiculous. H.R. 3, yes, it had a greater impact on the number
of drugs that might come to market, according to the
Congressional Budget Office. I respect that, and that was our
goal, to respond to that.
Our bill, according to the Congressional Budget Office,
might result in maybe one drug over the next decade not coming
to market. All right. That is one drug. I get that, but I will
balance that any day against seniors may not be able to afford
the medication.
We reduce the out-of-pocket expense for seniors to $2,000 a
year that they can pay over the course of the year to make it
as affordable as possible to them. They are no longer subject
to the donut hole that they are on hook for thousands, tens of
thousands of dollars for some of these very exciting new,
innovative drugs that unfortunately cost quite a little bit.
The same thing, insulin is capped at $35. That is huge.
That is huge for seniors. Diabetes is--I am a veterinarian. It
is prevalent in the animal population, prevalent in the human
population, absolutely critical to prevent that sort of thing
from becoming endemic in our population.
And limiting the cap on everyday drugs we have used for
years to the price of inflation when they try and jack the cost
up on us. I think that is a good deal. That is great deal for
Americans, Oregonians, being able to afford medication.
So the idea that this is stifling innovation is completely
complete you know what and inaccurate, and I resent the fact
that that is being put out. We have incorporated a lot of my
Republican concerns. It is a really good bill, and I hope it
actually passes the Senate. It is a boon for our America's
seniors to enjoy quality of life along the lines of what we are
talking here.
Dr. Hood, if I could ask you a question here. I am really
excited about the work you are doing. The phenome thing is
critical. Medicine is a lot more than about just the disease
entity itself. It is about the environment. It is about these
predispositions, all those things.
So it is very, very exciting. What is the role between NIH
and industry in getting involved in the phenome project itself?
Dr. Hood. Well, we have talked with the All of Us Program
at NIH, and conceded we have really complementary
possibilities. We are exploring it, and we plan to work
together in the future.
I think the industries that we have brought in that are
going to enable us to get this program started very early on
are going to push us to points where we can begin delivering
the longitudinal phenome data that will push us towards
wellness but will let us really transform diseases.
And, again, let me just say this agnostic view of disease
is interesting because with a million people, we will have
hundreds of thousands of people with the major diseases,
diabetes, cardiovascular disease, cancer, Alzheimer's. OK?
From the data we get, we will be able to create and
identify I would guess the 20 to 40 different subtype each of
those major diseases have, and each of those will be targets
for new drug approaches.
Mr. Schrader. Very cool. If I may, I am sorry to interrupt,
but a question, like a little bit now and, of course, later
because my time is running out, but how do we adopt lifestyle
and environmental concerns into what we do in the
pharmaceutical domain and how we approach patients and their
medications without unfortunately discriminating.
I mean, some people have genetic predispositions, certain
races, certain cultures.
Dr. Hood. Absolutely.
Mr. Schrader. How do we improve outcomes without
discrimination?
And you can answer that later or----
Dr. Hood. Again, the key point I want to make is
thisGuardian Research Network. Why does it cross all of the
major ethnic groups? And we will put them into the million-
person project in proportion to their level in the general
population.
And with a million people we will get a lot of data on
Latinos, on Blacks, and the whole thing. So you are absolutely
right. We need that data because we treat different people
different ways according to what their genetic predispositions
are.
And the same may be true of these diseases like diabetes
and Alzheimer's. We may see different tendencies in different
groups, and with the million-person project, this will come out
absolutely beautifully in a non-selected manner that gives you
what you see in the population.
Mr. Schrader. Very good. Thank you very much.
I yield back. Thank you, Madam Chair.
Ms. Eshoo. The gentleman yields back.
I just want to take a moment to salute the gentleman from
Oregon for the important work that he has done to lower the
price of prescription drugs for the American people. The costs
are simply unsustainable.
And when this becomes law, everyone's constituents,
Republican constituents, Democratic constituents, those of us
that, you know, who are Republican members, that are Democrats.
All of our constituents are going to benefit from it.
And I doubt then that my Republican colleagues will go out
and say that this is a bad thing.
So with that I would like to recognize the gentleman from
Florida, Mr. Bilirakis, for his 5 minutes of questions.
Mr. Bilirakis. Thank you, Madam Chair, again, for holding
this hearing on biomedical research and innovation and the
future of personalized medicine, so very important.
I said before that data drives decision making. I think the
panel would agree that we should focus on generating quality
data in order to make advances in biomedical research and
quality, evidence-based decisions. This is, indeed, a key part
of the puzzle to translating basic research into breakthrough
cures.
With this said, I am disappointed that the Biden
administration decided to repeal the Medicare Coverage for
Innovative Technology, or the MCIT rule, that provides our
seniors with access to breakthrough devices.
We created the FDA Breakthrough Device Program to allow for
a priority review process for groundbreaking breakthrough
technologies that have no approved alternatives, offer
significant advantages to the existing options or availability
of which would be in the best interest of our patients.
I applaud the FDA for setting up a rigorous pathway to
ensure these medical devices, the device technologies, are safe
and effective upon approval, and these manufacturers must
collect the necessary data to show its benefit to patients.
Unlike their drug counterparts, these devices do not
received coverage by CMS upon approval until they embark on a
costly and year's long clinical study process. By contrast, the
MCIT rule allowed for temporary national coverage under
Medicare during which FDA continues to collect data and conduct
its post market surveillance requirements.
This rule had broad bipartisan support, and I even co-led a
bipartisan letter with Representative DelBene and
Representative Cardenas and Representative Walorski that would
codify coverage for these breakthrough devices.
Despite this, CMS bureaucrats decided to repeal the MCIT
rule leaving these patients with less access to care. I am
grateful that the leadership on this subcommittee wrote a
letter to CMS asking them to reconsider their decision.
Dr. Abernethy, you mentioned in your testimony the
importance of longitudinal post-market monitoring of medical
products to ensure we are establishing evidence-based
decisions.
Can you tell me more about the importance of incentivizing
this evaluation of evidence over time?
Dr. Abernethy. Thank you for your important question, and
another fellow Floridian.
I really think that one of our core tasks as we encounter
not only breakthrough devices but other interventions that can
impact health and personalize our care is that we continue to
evaluate how medical products perform across time, and we
generate the data sets needed for that data-driven decision
making.
Importantly, evaluating medical products across time not
only allows us to continuously understand safety and
effectiveness but also helps us understand what is the best way
for a doctor and a patient together to make the right decision
for this particular patient and within the context of that
patient's needs because we develop more and more data across a
more diverse and richer population, as well as more
understanding of which specific scenarios and what timing.
So that is this critical issue of continuing to collect
longitudinal data.
Mr. Bilirakis. Thank you.
It makes sense.
Do you agree that Medicare coverage of innovative
technologies, which would lead to more widespread access,
patient access, could result in more robust evidence generation
in the post-market setting?
Dr. Abernethy. It is really important that we continue to
think through how we are going to get Medicare beneficiaries
and others the interventions that they need, and this is an
important topic for us all.
Mr. Bilirakis. Very good.
Thank you, Madam Chair. I yield back.
Ms. Eshoo. The gentleman yields back.
It is a pleasure to recognize the gentleman from Missouri.
Oh, I am sorry. Well, you are after the next person. How is
that, Mr. Long? I am sorry. I was anxious to get to you because
you were the very first one in the hearing room this morning.
The Chair now has the pleasure of recognizing the
gentlewoman from Michigan followed by the gentleman from
Missouri, Mrs. Dingell for your 5 minutes.
Mrs. Dingell. Thank you, Madam Chair, and thank you,
Ranking Member Guthrie.
And I am sorry to my Missouri colleague that I came in
front of you because I always love hearing from you.
But I really want to thank the Chair for convening today's
hearing on advances in biomedicine and personalized treatment
because it really, really is so critical.
But I also think it is important to note that public
funding of research through institutions by the NIH is
fundamental to the development of cutting-edge therapies and
personalized medicine that we have been discussing today.
In fact, NIH contributed to published research for every
one of the 210 new drugs approved by FDA from 2010 to 2016.
So we have got to continue to provide the resources
necessary at the Federal level to translate basic research into
medical breakthrough. This starts with supporting early career
scientists and investigators who are the future of medical
innovation in this country.
Dr. Minor, you mention in your testimony that since 2000
NIH applications have doubled, but the success rate of those
applications declined from 32 percent to 21 percent, leaving
many quality proposals unfunded.
Can you discuss the importance of NIH funding for early
career scientists?
What impact has this development had on those aspiring to a
career in research and how can we in Congress help support
early career researchers?
Dr. Minor. Well, thank you very much, Congresswoman
Dingell, for your question, and, yes, I think that the decline
in the proportion of investigators supported by the NIH, the
proportion of grants that are actually funded by the NIH, the
decline has had an effect on our biomedical infrastructure in
the country and, in particular, on young investigators.
I applaud the NIH for doing a number of things to address
that, such as early career investigator awards that are
reviewed specifically for the cohort of young investigators.
When I mentioned young investigator now, it is a relevant term
since the average age at which an investigator received their
first NIH grant is now in the early to mid-40s, and that is
because of the length of training that is required to get to
the level to compete effectively, even for these early career
investigator awards.
There is no more precious resource, I would say, in our
country than our biomedical workforce, and we are privileged to
have still an extraordinarily dedicated group of young people
who want to go into biomedicine and physicians, physician
scientists, basic scientists.
We need to make sure that our governmental support,
particularly from the NIH, rewards their years of dedicated and
minimally compensated effort in order to give them an
opportunity to build their research programs.
And that is why, as you mentioned, it is so important that
NIH funding be increased to enable that work force to continue
to thrive.
Mrs. Dingell. Thank you, Dr. Minor.
And I do believe that my colleagues and I in a very
bipartisan way on this committee will continue to prioritize
support for the NIH moving forward.
In the time I have left, I would also like to touch on the
issue of data privacy. We have heard from our witnesses about
the potential to leverage large data sets of health information
to drive advances in care.
However, we have also seen that personal health information
is an attractive target in countless research institutions and
healthcare providers have been the victims of data theft and
cyberattacks.
In fact, I myself have signed up for NIH's data collection,
but was very worried about my own privacy because it can result
in the theft of medical identifiers, prescription information,
treatment information and other sensitive medical data.
Dr. Abernethy, you mentioned the importance of data
governance and privacy in your testimony. What policies should
Congress be looking at to reduce the risk of unauthorized
access to personal health information, given the proliferation
of electronic health records and aggregated health data?
Dr. Abernethy. Thank you so much for this important
question.
Practically speaking, we must prioritize privacy, security,
and consent if we are going to advance this critical work in
personalized healthcare. We need to make sure that we continue
to look at current rules and think about what are current
implications with respect to capabilities and technologies of
today and how those rules are performing for us and whether or
not we need to update those rules, such as HIPAA, across time.
We also need to think about how do we incentivize
innovations that continue to improve on privacy and privacy
sparing solutions, such as leveraging tokenization, so we do
not have to include personal identifiers and leveraging, for
example, synthetic data sets that obscure personal information
but maintain the ability to analyze data sets to understand
important outcomes around healthcare intervention.
If we do not prioritize privacy, we will not be able to
maintain trust and trustworthiness, which is critical to the
system going forward.
Mrs. Dingell. Thank you.
It is very important. I am out of time. I want to pursue it
more.
Madam Chair, I yield back.
Ms. Eshoo. The gentlewoman yields back.
A very important line of questioning that we need to
pursue, Mrs. Dingell.
Now, Mr. Long, the gentleman from Missouri, you are
recognized for 5 minutes for your questions.
Mr. Long. Thank you, Madam Chair.
And if I was going to yield to anyone in the John Dingell
Room, I could not think of anyone better than Debbie Dingell to
have yielded to.
So you kind of set that up for me. Thank you.
Dr. Abernethy, thank you for being here to share your
perspective. Can you talk about how traditional clinical trials
are conducted and what makes them resource intensive and
inconvenient and overly burdensome for their participants?
Dr. Abernethy. Thank you for this important question, sir.
In the context of a traditional clinical trial, we
prespecify in a very carefully worded study protocol, like a
recipe book, all of the actions that a study coordinator and a
patient and other actors in the clinical trial system must take
in order to collect that data.
Often it requires an individual to go to a clinical trial
site. So, for example, my father needed to go to Houston if we
were going to consider him for a specific cancer trial rather
than his hometown of Orlando.
It also often requires a person to get an extra or many
extra tests that may be duplicative of tests that they have
already had in the past, such as extra biopsies or additional
scans.
And then it requires a person to be followed over time,
oftentimes going back to that site way far away from their
hometown.
The opportunities in the future are to leverage the ability
for a person to participate in the clinical trial leveraging
video means or digital health solutions to collect data.
However, we need to make sure that when we leverage these new
solutions in the future, we do not sacrifice participant safety
or the ability to collect high quality, credible data that can
answer questions confidently.
Mr. Long. My second question was going to be on the use of
digital health tools to alleviate some of the burdens, but I
think you kind of covered that in that portion.
So my next question is what are the challenges to
generating adequate and acceptable evidence using these new
tools and trial designs and how do we overcome them?
Dr. Abernethy. Thank you. I appreciate the opportunity to
follow on here.
Practically speaking, we have the opportunity to leverage a
number of tools, some of which were made possible by this
committee within the context of 21st Century Cures and
opportunities going forward. These tools include, for example,
leveraging all available data, including data that had been
passively collected in the electronic health records, claims
data assessed, and tools such as the censor in a watch to be
able to collect information, for example, about movement.
We have got the opportunity to leverage these tools.
However, we are going to have to do the hard work of
understanding how to make sure that the data are cleaned up,
are of high quality, and are representative of longitudinal
reflections of care that also help us understand how a person
performs across time.
We also have the responsibility to make sure we develop the
scientific methods that allow us to make sure that we can
responsibly and reliably analyze these data sets, including
when we pair clinical trial data sets with real-world data
sets.
And we also have to make sure, as I mentioned before, that
we critically make sure that patients are kept safe whenever
being involved in clinical research and we never sacrifice the
issue of patient safety.
Mr. Long. OK. Thank you.
And, Madam Chair, this is my eleventh year in Congress, and
I have picked up recently on the fact that when you are in
Congress not everyone is a huge fan, and every time I wear my
neckwear today, I get people that maybe are not real big fans
say, ``What is that goofy tie Long has on?''
But I always like to point out this is actually the St.
Jude Children's Research Center tie, and being a father of a
cancer survivor, I always wear it with pride. So before
everybody starts saying, ``What is that goofy tie?'' now you
know.
I yield back.
Ms. Eshoo. The gentleman yields back.
I do not know anyone in Congress that does not think the
world of you, Mr. Long. So just disabuse yourself of that
notion, and your necktie is beautiful because it represents
something that is magnificent in our country, St. Jude's and
the care that they give to children day in and day out.
The Chair is pleased to recognize Dr. Bucshon, another of
our outstanding doctors on our subcommittee, for your 5 minutes
of questions.
Mr. Bucshon. Thank you, Madam Chairwoman.
And today's hearing comes at an opportune time. Promoting
innovation and advanced research in our biomedical industry is
crucial for not only America's leadership in the world, but
also more importantly, it is crucial for America's patients.
That is why I am concerned about the drug pricing protocols
in the Build Back Better Act, and they would have the opposite
effect limiting research and hampering innovation, in my view.
In fact, a recent analysis conducted by the University of
Chicago found that the drug pricing protocols found in the
Build Back Better Act would lead to 135 fewer new drugs by
2039.
The study also said that it would generate a loss of 331.5
million life-years in the U.S. during that time. That is 31
times as large as the 10.7 million life-years lost from COVID
to the U.S.
As a physician, I sometimes had to share bad news with
patients and families, and I know all too well that eliminating
just one new drug is one drug too many. What if one of those
new drugs is a cure for Alzheimer's, cancer, or ALS?
Dr. Abernethy, obviously, the Federal Government plays a
role in funding research to the NIH and other programs, but it
cannot stand on its own. Can you explain how important private
research and development is to innovation and discovery of new
cures?
Dr. Abernethy. Thank you very much for this important
question, sir.
I learned during my time at the agency the criticality of
public-private partnerships. When I was at the agency, I was a
co-author of a committee report for the National Academy of
Medicine on digital health in COVID, and what we realized was
that we had data, data everywhere, but really the inability
oftentimes to put it to work the way that we were hoping in
managing the pandemic.
And one of the key recommendations was to build on public-
private partnerships, something that we had learned as an
important element when I was at FDA.
What can public-private partnerships do for us? Well, first
of all, they can help us move faster by ensuring coordination
across industry, across government, across academia, to solve
hard problems.
They can make sure that there is learning from each other,
but also co-investment in solving problems that matter and
amplify the work done by those different sectors.
The other thing is when regulators are also part of that
conversation or at the table, regulators can learn and start to
think about what is possible into the futured and start to
think about how that might be regulated in the future so that
the regulatory actions are not falling behind of what is
possible in the private sector or in academia.
Mr. Bucshon. Well, thanks for that answer.
And I think we saw during the pandemic with the development
of the vaccines this cooperation between the Federal Government
and the private sector and resulting in therapeutics and
vaccines available in really record time.
Madam Chairwoman, I yield back.
Ms. Eshoo. The gentleman yields back.
The Chair is pleased to recognize the gentlewoman from
Texas, Mrs. Fletcher, for your 5 minutes of questions.
Mrs. Fletcher. Thank you so much, Chairwoman Eshoo, and
thank you for convening this hearing today.
Thank you to all of our witnesses for your testimony. It
has been a very interesting and informative morning.
And it is not really surprising. Apart of the reason that I
was so excited to become a member of this committee and this
subcommittee and this subcommittee, in particular, is because
of the committee's jurisdiction over medical research and the
hugely important and interesting issues in front of us.
So I thank you very much for doing this today.
Part of the reason that I was so interested in this is
because I represent Houston, Texas, and it is the site of the
Texas Medical Center, the largest medical center in the world
and home to some of the country's and the world's greatest
researchers, and it is such a privilege to get to represent so
many of them here in Washington.
Institutions like the Texas Medical Center Institution, the
research institution there, the incredible care they provide,
they are ready and willing to tackle some of these biggest
health research challenges.
But an important piece of turning research into treatment
and advancement is academic, clinical, commercial, and public
partnerships.
So, Dr. Minor, I want to thank you again for being with us
today, and from your testimony, it is clear that you know what
it takes to conduct research both responsibly and efficiently
and also what the biggest barriers to progress can be.
So I want to direct my questions and the time I have to
you. Based on your decades of experience in the academic space,
as you noted in your testimony, basic research served as the
foundation for innovative technology and treatments. However,
translational research is also critical to disclose what
strategies or further investments should Congress consider to
help advance translational research and the advancement of
research from academic labs to commercialization.
Dr. Minor. Well, thank you for your question, Congresswoman
Fletcher, and congratulations on representing the district that
has in it the Texas Medical Center, which is a truly amazing
complex, truly amazing work in all spectrums ofhealthcare, from
patient care, research, and training the next generation of
leaders.
I think there are several things that can be done, and many
of which we have discussed today already to accelerate the
translation of basic science discoveries into new therapeutics
and diagnostics and improved health.
We have talked about the importance of Federal funding,
also the importance of making sure that appropriately monitored
and governed collaborations between academic institutions,
nonprofit institutions, and the commercial sector, making sure
that there are no impediments to those interactions because
really to translate and to bring developments to the benefit of
patients, there is a commercialization step that absolutely is
essential.
So making sure that proceeds at a facile way, and I would
just look back to something that Congress did many, many years
ago with the Buy Gold Act, which really fueled the beginnings
of the biomedical revolution in our country and in the world.
Similarly, looking for ways that we can incentivize public
and private collaborations in the future will be really
important.
And finally, one thing perhaps that we have not touched on
as much in today's hearing but that is critically important is
really focusing on training the next generation of leaders.
You know, the level of existing scientific knowledge today
compared to when I was training is unfathomably greater than it
was before, and we need to focus on how we train our scientific
workforce and the opportunities we provide to them to receive
outstanding training.
Training grant programs from the NIH are critically
important. Recently the funding for those programs has been
challenged, and that is the critical pipeline that will drive
innovation and drive this translation of discoveries into new
therapies and diagnostics for decades to come.
So I think focusing on our training programs enabling our
academic institutions that run those programs to be successful
and making sure that the Federal Government at all levels is
providing the appropriate support for those training program is
critically important, from training Ph.D.'s to training medical
students in medical school, and also postgraduate medical
education which plays a hugely important role.
Mrs. Fletcher. Well, thank you so much, Dr. Minor, and that
is certainly something that I have heard from our institutions
as well, the importance of finding and training and retaining
talented folks who are doing the kind of work that has payoffs
today.
I am sorry that I am almost out of time to ask more
questions, but I would just like to say for anyone on the panel
who would like to weigh in on that question, if you wanted to
submit an answer to that for the record, I would be so
grateful.
And I really am grateful for all of your time today and to
you, Chairwoman Eshoo, for holding this hearing.
And I yield back.
Ms. Eshoo. The gentlewoman yields back.
The Chair is pleased to recognize another outstanding
doctor on our subcommittee. We are so proud of those that are.
Dr. Dunn of the State of Florida, you are recognized for your 5
minutes of questions.
Mr. Dunn. Thank you very much, Madam Chair, and thank you,
Ranking Member Guthrie, for hosting this hearing today to
discuss biomedical research and personalized medicine.
I am very proud of America's robust tradition of innovation
in the biomedical industry. We are living in a fascinating age.
The success of Operation Warp Speed is a great example of the
ability of the industry to step up to that when we really
needed them very much.
This committee should be considering and advancing policies
that continue to incentivize innovation unimpeded by regulatory
barriers and excessive red tape.
And I agree with the witnesses who spoke earlier today that
said that government price fixing of pharmaceuticals does not
serve that goal.
I want to shift my attention though to another important
and topical area of medicine, which I think can help tailor
treatment decisions for patients today, and that is T-cell
testing.
Dr. Abernethy, you wrote recently an article entitled
``Winning the War on COVID Requires a Complete Understanding of
Immunity. So Why Aren't We Demanding It?''
Well, let me start by saying kudos to you for writing that
article. There are a few of us who are in the trenches trying
to fight for T-cell testing.
I introduced a bill that provides for coverage of T-cell
tests and T-cell immunity. I have discussed and written about
this at length with Drs. Fauci and Collins and others over at
NIH and I must say to a fairly poor reception.
Can you please elaborate for the rest of them here who may
not be as familiar with T-cell testing as you and I are what
kind of information we are missing out on due to our public
health authorities' narrow focus on antibodies rather than T-
cells as well?
Dr. Abernethy. Thank you for this interesting and important
question, sir.
Practically speaking, as we look towards the science to
help us combat the pandemic and address both SARS-CoV-2 as well
as think about management of pandemics in the future and other
healthcare concerns, it is important that we leverage the
complete portfolio of solutions in front of us.
Our immune system has more than just one compartment. We
have the ability to not only build antibodies, but also
leverage cellular immunity, T-cells, to also attack and combat.
Mr. Dunn. And I wonder if you would also compare, you know.
We knew about T-cell testing when we studied SARS-CoV-1, right?
Twenty years ago, and so this is not entirely novel science we
are talking about here.
Dr. Abernethy. An interesting point. Sometimes science
takes pace at not always with exactly the same pace in all
areas, and so one of the things that we have seen in the
immunology space is sometimes the story on the antibodies side
is amplified with not as much discussion going on on the T-cell
side.
But I think that we have the opportunity to really look at
how the entire immune system is performing and making sure that
we really are amplifying how the whole immune system works, and
at least just making sure that we are figuring out how our
interventions are performing across the spectrum.
Mr. Dunn. Thank you for that.
Dr. Hood or Dr. Abernethy, are either of you familiar with
any large-scale T-cell testing, screening, if you will, for
SARS-CoV-2?
Dr. Hood. Well, I have participated at the Institute for
Systems Biology and Swedish Hospital in some large-scale COVID
tests. One of----
Mr. Dunn. Were they T-cell?
Dr. Hood. Well, one of the really powerful tools for being
able to separate B-cell and T-cell specificity is to take it
each blood draw as you follow these patients, 5,000 white blood
cells, and sequence all the information in each of those.
Mr. Dunn. That is a lot of sequencing, is it not, Doctor?
Dr. Hood. So it defines the T-cells. It defines the
different classes of T-cells, and we found new classes of T-
cells.
Mr. Dunn. Yes, yes, I am familiar. So you are going to get
lost in the details of T-cells.
Dr. Hood. So it is heartily clear that T-cells play a
critical role in COVID immunity.
Mr. Dunn. I think it does. Let me just say we agree with
each other.
In the interest of the short time we have, I want to turn
to Dr. Abernethy and say how do you think vaccine manufacturers
could be, maybe should be using T-cells as they develop their
vaccines and work on whether or not there is a need for
boosters?
Dr. Abernethy. Interesting question. In line with the
science, we have the opportunity for vaccine manufacturers to
really explore the full immune system response to vaccines.
We also have the opportunity to make sure that that
information becomes available not only to regulators but to the
entire clinical and scientific community, and that is going to
be something that I hope to see more of in the future.
Mr. Dunn. Yes. I would very much like to see this.
And in the few seconds that remain let me say that I have
been recommending to people that if they wonder if they need a
booster say, ``You may need a booster, you may not. Get a T-
cell test. Find out if you need a booster. If you have got
immunity, why are you going and getting a booster? And if you
do not have immunity, why are you not getting the darn
vaccine?''
So those are my thoughts when I am advising patients. I
should have prefaced that, I guess. Chairwoman Eshoo has told
you that I am a doctor.
Thank you very much. I yield back, Madam Chair.
Ms. Eshoo. I thank the good doctor. He yields back.
The Chair is more than pleased to recognize the gentlewoman
from Massachusetts, Mrs. Trahan, for her 5 minutes of
questions.
Mrs. Trahan. Thank you, Madam Chair.
And thank you, all the witnesses, for being here today.
A key thing in this discussion of biomedical research has
been the importance of collaboration, and this comes in many
forms, including data sharing. We know that data plays a
critical role in advancing biomedical research and translating
into new diagnostics and treatments.
And we have seen that for viruses like COVID-19, but also
for chronic diseases, mental health issues, maternal health
outcomes, and substance use disorder.
Dr. Butte, you have successfully worked to combine
healthcare data from across six University of California
medical schools and health systems.
And, Dr. Abernethy, likewise you have extensive data
science experience in both public and private settings. This
question is for both of you.
How can we improve communication between State and Federal
public health agencies and private health systems?
And what special considerations should be made to ensure
communications between public and private health systems is
efficient, effective, and secure?
Dr. Butte. Great. Maybe I will go first.
As we all know, the United States has a competitive
healthcare system. While we all want to enable and empower
patients with their own data, especially using Federal
standards, our pharma, biotech, and AR providers do not often
want to share data with each other for competitive reasons.
However, the Federal Government has the ability to convene
data, and that could be enhanced. So, for example, the National
Public COVID Collaborative or N3C Program by NIH is one great
example, with nearly 10 million COVID tested patients now and
across many of the Nation's health systems.
With the right governance, NIH has shown that clinical data
can actually be shared with each other to drive the best
treatments and practices, for example, with patients with
COVID.
Mrs. Trahan. Great. Dr. Abernethy, can you please add?
Dr. Abernethy. Thank you. I will expand on Dr. Butte.
But first of all, we can continue to leverage technology
that enables the ability to share, whether that is pooled data
coming to one particular place or federated data where data
sits in different places, but we share essentially insights.
The second thing I would say is that we do need to continue
to incentivize sharing. Incentivize in hoarding gets us into
trouble, but incentivizing learning how to use data together is
one of the things that has become progressively more important,
and we have seen that in the pandemic.
But it has to be safe sharing where we are focusing on
patient privacy and security as we have talked about earlier
today.
The last thing I would say is we have mentioned today the
value of public-private partnerships, and you have also
mentioned the value of, for example, State and Federal work
together, and learning how to do this work well and learning
from each other is going to be important as we try and work
forward with patient data sharing.
Mrs. Trahan. Great. Thank you.
Researchers, like many of you, are also pioneering in
answers for AI in healthcare. Ensuring that minority groups are
properly represented in data sets is one possible way to
mitigate the risk of a predictive healthcare tool performing
poorly for minority populations.
And so, Dr. Abernethy, can you explain the importance of
training these AI models on diverse data sets and comment on
how feasible it is for manufacturers to diversify their data
sets?
Dr. Abernethy. Thank you very much for this important
question.
As you critically point out, the building of algorithms is
based, first, on the data that informed the development of
those algorithms, and therefore, first of all, algorithms can
become biased just by virtue of bias in the underlying data
sets.
Therefore, it is incumbent on our future if we are going to
leverage artificial intelligence for us to build data sets that
do not systematically exclude specific populations.
We need to be leveraging those data sets that are as
complete as possible and also documenting or essentially
measuring the bias in data sets so that we can continue to
improve.
The other thing that we need to do is document the
performance of artificial intelligence solutions, including the
output as well as how they perform in terms of their intended
task and document that performance across time if we want to
make sure that artificial intelligence continues to work for
us.
Mrs. Trahan. Great. Those are great points.
Well, let me just close by thanking the witnesses for their
time today and for your contributions to biomedical research,
especially through this pandemic.
Thank you, Madam Chair. I yield back with time to spare.
Ms. Eshoo. The gentlewoman yields back.
A pleasure to recognize the gentleman from Oklahoma, Mr.
Mullin, for his 5 minutes of questions.
Mr. Mullin. Thank you, Madam Chair.
Dr. Abernethy, am I saying that right?
Dr. Abernethy. Yes, sir.
Mr. Mullin. OK. We all know that the precision medicines
are typically incredibly expensive. Can you speak to why these
drugs have such a high price tag?
Dr. Abernethy. Thank you for your question, sir.
While I am not an expert in drug pricing or cost, I think
that one of the critical issues as we think about personalized
healthcare and precision medicine is that there is important
adjustment in biology, in clinical development, in moving
through the regulatory expectations, and importance of
ultimately development interventions that are oftentimes for
smaller and smaller populations, which may ultimately get
reflected in the context of price.
But practically speaking, I hope towards a landscape where
innovation across this space is going to increase the
availability of precision medicine for all.
Mr. Mullin. Thank you.
I agree with that, too.
In your testimony, you had highlighted that your company
has payment structures that were based on patient outcomes.
Can you speak to the importance of this innovative payment
structure that you guys have?
Dr. Abernethy. I am not specifically sure exactly which
payment structures to which you are referring. I think one of
the things that I have thought a lot about and been considering
in the future is the availability of data and the same data
sets and processes that we use for clinical trials and clinical
studies, the ability to interrelate patient treatment and
outcome to help inform, for example, outcomes-based pricing in
the future, where we can interrelate the performance of
intervention to then the ability to think about how to pay for
value across time.
Mr. Mullin. Right. I believe in your written testimony you
said that you have only been able to use these payment
structures in limited circumstances. Am I correct on that?
Dr. Abernethy. I think this was more generally from a
policy perspective, the ability to use these kinds of
interventions rather than specifically for our company.
Mr. Mullin. Well, you know, I am part of the Innovation
Caucus and co-chair that, and we are always looking for ways to
incentivize the medical industry to look at new directions, new
ways. Instead of us just trying to fit everything into the same
doughnut that we have always done business in, let's figure out
a way to go around that.
And the payment structure is one of them. You know, in
anything we do in life, be it get an attorney or call a service
company, the payment is based upon the outcome. In most cases
it is based upon did you complete the job or did you not. Did
it work? Did the product you installed, did it work? The same
thing with our mechanics that work on our vehicles.
And if we can look at a new payment model and tie it to the
patient's outcome, that is something for us to look into, and
as we are looking through innovation, I think we have got to
look at the payment structure, too.
And so as we move forward, I hope we can have more of
these conversations.
Madam Chair, with that I yield back and thank you for
having this hearing.
Ms. Eshoo. The Chair thanks the gentleman for your
interesting line of questions.
The Chair is now pleased to recognize another one of our
fine doctors, a member of our committee, Dr. Schrier of
Washington State, for your 5 minutes of questions.
Ms. Schrier. Thank you, Madam Chair.
And thank you to the witnesses who testified today. I was
thrilled to hear about your research and work, and I am very
proud to see a Washingtonian here in the room today. Washington
State is the hub for innovation, and, Dr. Hood, I am so glad
that you are with us today.
Dr. Hood, I want to talk to you first and the work that you
have done through phenyl health and the Beyond Human Genome
Project. Briefly, can you tell me how this project, the Beyond
Human Genome Project, is different from the Human Genome
Project?
Dr. Hood. Yes. The Beyond the Human Genome Project differs
in that it employs for all the million people longitudinal
phenome analyses, and the phenome, as I said before, is the
reflection of the interaction of your genome, your lifestyle,
and your entire environment.
So it gives us deep insights into what is happening at
different points in your life to lead to actionable
possibilities that we will be able to use on patients.
So it differs fundamentally with a whole new technology
that is opening up probably thousands of new actionable
possibilities we will be able to use to treat patients.
Ms. Schrier. I love the idea of finding risk and disease
early and being able to treat more appropriately. Research is
paramount to finding treatments and cures, and that is why I
was so proud to co-lead the Pediatricians Accelerate Childhood
Therapies Act, the PACT Act, with Dr. Joyce.
I think it is imperative that we invest specifically in
pediatric research if we are going to address the greatest
public health threats facing children in the 21st century like
obesity and malnutrition and cancer, diabetes, asthma and,
frankly, COVID-19. We have got to incentivize pediatric
research.
In the meantime, you talked about prevention, identifying
people at risk, finding the interaction of the genome and the
phenome, and then addressing early detection, and we detected
diseases as well as we can.
Can you briefly give me just like a sense of what sorts of
diseases you can screen for in the Beyond Genome Project so
that people have an understanding of what this means in their
specific lives?
Dr. Hood. Well, I think the first answer is we can really
screen for approximately virtually any disease that we know
about. As I said earlier, there are about 7,000 rare diseases,
each for the most part caused by a single gene defect, and the
complete genome sequence analysis at birth would
instantaneously let us diagnose those diseases.
And, again, with single gene defects and the information we
get with longitudinal phenome, we have the possibility of being
able to generate early therapies and not let a person go
through life with that gene defect.
There are many recessive mandilion diseases like cystic
fibrosis and hemochromatosis and some of the Tay-Sachs-like
diseases and so forth. And, again, these can be detected, and
the individuals followed to be able to treat them at the
earliest instance of moving toward a disease process.
We will be able to take the----
Ms. Schrier. Sir, if I can----
Dr. Hood [continue]. And stratify them into their different
subtypes and go after therapies that can attack each of these
subtypes.
So those are the kind of things we could do in a precision
population approach to pediatrics.
Ms. Schrier. That is great.
Dr. Hood, you know, I will tell you this as a pediatrician.
I think about Type 1 diabetes. We now know how to identify
those genes. Screening for them at birth would be incredibly
important.
And we know that while we cannot cure it yet, that we can
delay its onset, and every year that onset is delayed is a year
less with the potential risk for complications.
Cystic fibrosis we now have gene therapies for. See, you
could do that before any lung scarring develops.
And hemochromatosis is simply treated with blood draws, and
if you could do that, you can protect the liver and other
organs.
This is very exciting research. Thank you very much for
leading the way, and I think that the last thing I would say is
now we have to get doctors and teach them onboard because
sometimes it is carrying through on those preventive lifestyle
teams that can be the toughest part of all.
Thank you very much. I yield back.
Dr. Hood. Thank you.
Ms. Eshoo. The doctor yields back.
And now finally, the patient and deliberative gentleman
from Utah, Mr. Curtis. You have 5 minutes for your questions.
And really, thank you for your patience. Mr. Curtis was the
second person in the chamber this morning before we began. So
thank you, and you are recognized.
Mr. Curtis. Madam Chair, you are too kind. Thank you, and
thank you to our ranking member. This is obviously a very
important hearing.
I have to say I am very impressed with all of our witnesses
today and the expertise that you bring.
Madam Chair, you have rightly so bragged about your
district and Stanford, but I must take this occasion to brag
about mine and to share some information about what is
happening in Utah with life sciences. It is the fastest growing
life sciences community in the United States in Utah, and the
Beehive State is home to a vibrant healthcare innovation
ecosystem that is a key driver in both life changing
interventions and tools.
Utah has referred to their State life sciences industry as
``BioHive,'' a reference to the Beehive State.
This community consists of approximately 1,400 companies
across the State working as a collective in order to have real
impact.
There are a few interesting statistics I would love to
share with everybody today. These individuals specialize in
research, medical device manufacturing, biotech, and
pharmaceuticals and diagnostics. They are second in the Nation
for medical device employment concentration.
They provide $13 billion in [audio malfunction] GDP from
industry. They account for eight percent of Utah's total GDP,
and they filed for 538 bioscience related patents in 2019.
The BioHive includes partnerships with our top-notch
universities, start-up accelerations, health systems, and State
and local government, which collectively drive healthcare
research and innovation in the State.
I believe it is particularly important to promote public-
private partnerships and to prevent policies from being enacted
that impede private sector investments. One of those 1,400
companies is Queen. It is a clinical stage biopharmaceutical
company that was founded in 2013 working on--get this--the
reverse cellular energetic failure and to enhance repair of
nerve cells.
Boy, if we could strike that, that would be an amazing
thing.
But they are concerned. Sweeping government drug price
controls, such as those proposed in H.R. 3, worry them and
worry their investors. The Queen team is passionate about their
work, but ultimately advanced potential solutions that will
make a difference in patients' lives need investment, need
predictability from the community.
And I would be remiss if I did not address that in our
comments today.
Dr. Hood, I have been fascinated as you talked about your
research, and I am curious. This committee and myself
personally have a deep tie with ALS and the suffering that we
see from them, MS, Parkinson's, some of these diseases that
attack the nervous system.
Are you seeing anything in your research that are flags for
these diseases that would help us help these good people?
Dr. Hood. Well, one of my major areas of research now is
Alzheimer's disease, and I will make a couple of general
statements.
One is that there have up until very recently not been more
than 500 clinical trials on drugs for Alzheimer's. All have
failed, and it is because they have entirely the wrong
hypothesis about what it is as----
Mr. Curtis. And I am going to push you just because we have
all got tight time constraints.
Dr. Hood. I am just saying what we are learning about
Alzheimer's is almost certainly going to apply to the other
neurological diseases.
Mr. Curtis. We are all cheering for you.
The frustration has come before this committee and those
who have addressed this committee, is the inability of these
patients to access treatments. Can you give us any advice on
how to speed that up?
Dr. Hood. I think the key to access to the patient really
lies with better treatment and a focus on wellness and
prevention rather than always attempting to attack----
Mr. Curtis. I just wish we had so much more time, but I am
just going to keep moving on and just press the fact that some
of these cures that are potential but undefined, they do not
have access to them, despite the fact that they have been given
a life sentence.
So I would just like to lay down a marker in this hearing
as well for these good people.
Dr. Minor, let me switch to you quickly. You have also been
part of an ecosystem that is very healthy as I think I have
described in Utah. Tell us what we have to worry about with
government destroying these ecosystems.
We have talked a little bit about what government can do to
help. What are your worries about what government might do that
would be detrimental to these ecosystems?
Dr. Minor. Well, thank you for your question.
I think that harmonizing and developing greater clarity on
regulatory requirements coming from the Federal Government
between the various agencies is one proactive step that
government can take.
Certainly all of us as Americans should be grateful for the
FDA and the CDC for the critically important role that they
play, long before COVID, but during COVID as well. But we have
learned from COVID ways that perhaps the FDA and CDC could look
at better coordination, particularly when things are moving
quickly.
So I think there are a number of areas, Congressman, that
could be explored.
Mr. Curtis. Doctor, I am over my time, and the Chair has
been very patient with me. I would like to just end by pointing
out that we have to be as careful not to hurt the industry as
we are in our efforts to try to help the industry.
Thank you, Madam Chair. I yield my time.
Ms. Eshoo. Did you get the answer to your question? We
should give time for the answer.
Who did you direct it to?
Mr. Curtis. So to Dr. Minor, but I would be curious if any
of our other witnesses want to weigh in on that.
Ms. Eshoo. Dr. Minor, would you like to weigh in on that?
Dr. Minor. Well, I think there are opportunities for the
government to look at impediments that may exist and get
feedback from industry.
And, yes, I do think that there are concerns with regard to
the speed at which things can be developed and commercialized,
and regulatory issues may play a role in that.
Mr. Curtis. Thank you.
Thank you, Madam Chair.
Ms. Eshoo. Certainly. The gentlewoman from New Hampshire,
Ms. Kuster, is recognized for your 5 minutes of questions.
Ms. Kuster. Great. Thank you so much, Madam Chair.
And, Mr. Curtis, I will pick up where you left off.
I want to thank the witnesses for being with us to discuss
advances in biomedical research and how Congress, the research
community, and industry can work together to advance innovative
technologies and treatments.
Well, basic research plays critical roles in achieving
these goals. We also must ensure the knowledge gained from the
basic research can translate into the development of new
diagnostic and therapeutic tools that can be used in clinical
practice.
And it is this translational research that gives many of us
hope one day it will provide the foundation for new diagnostic
devices and promising therapies for patients with diseases like
Alzheimer's or cancer.
Dr. Minor, as a scientist, surgeon, and academic leader,
you are a strong proponent of translational research. As such,
you are well aware of the significant challenges associated
with crossing the Valley of Death, the phase between research
and innovation that can be so difficult.
As technology advances and leads to breakthroughs in fields
like defense and energy, breakthroughs in disease treatment and
cures seem to lag behind.
So, Dr. Minor, this committee has supported efforts to
address the Valley of Death by supporting the advancement of
novel clinical trial designs and streamlining the regulatory
process when we passed the 21st Century CURES Act.
Have these efforts been effective?
And could you explain why or why not?
Dr. Minor. Thank you very much for your question,
Congresswoman.
I do think that the efforts have been effective, but there
is so much more that we can do and that we should be doing.
Yes, I think the best evidence of the efficacy is what was
accomplished with Operation Warp Speed and the development and
deployment of messenger RNA vaccines, a completely new class of
vaccines, in about 11 months from the sequencing of the genome
to the emergency use authorization.
That is an example of what can be accomplished. A lot more
needs to be done. ARPA-H, I think, is a great step in that
direction of accelerating translation initiatives such as the
innovative medicine's accelerator at Stanford that I described
in my testimony earlier. I think it is also an important step
there.
Today is the difference between how we train basic
scientists and the way basic science is done and how we do
translation, and so assisting basic scientists in the process
of getting their discoveries in a translational pipeline is a
big responsibility for those of us in academia, and I think it
is something that the government through programs such as ARPA-
H, Operation Warp Speed, and the things that were done during
that period can be very beneficial.
Ms. Kuster. Well, I would certainly agree with you, and it
was quite extraordinary. I wish we could be as successful in
convincing the American people to take the vaccine.
What policies have been useful to you?
And could you recommend any changes in the statutes or
changes in directions that this committee could take to
increase advances in contemporary science and increase
diagnostics and therapeutics?
Dr. Minor. Thank you.
Encouraging innovation is critically important. NIH does a
fantastic job of funding research. Most of NIH funding is
directed at projects that are already up and going.
Preliminary data plays a major role in NIH applications for
understandable reasons, but oftentimes the most innovative
discoveries and advances come from a spark of an idea. Many of
those ideas may fail to yield results. Yet we still need to
give those ideas an opportunity to see the light of day or not.
And right now we do not have a grouping of governmentally
sponsored programs that fund the most innovative research.
Again, I think the notion behind ARPA-H will advance that goal,
and what we do at universities is important.
But we have heard the word ``innovation'' and several
members of Congress today on this committee have talked about
its importance, and I think there are ways that we can pursue
legislation that encourages innovation and entrepreneurship in
our biomedical communities.
Ms. Kuster. Well, I thank you all for being with us, and I
thank the chairwoman for her leadership in this area.
And with that I am going to yield back and hopefully you
will save the ten seconds that Mr. Curtis went over.
[Laughter.]
Ms. Eshoo. Thank you very much, Congresswoman Kuster.
It is a pleasure to recognize the gentleman from Georgia,
the pharmacist on our committee, Mr. Carter, your 5 minutes for
questions.
Mr. Carter. Thank you, Madam Chair.
And thank all of you for being here. This is extremely
important. This is the wave of the future.
I started practicing pharmacy back in 1980, and what I have
seen in healthcare and the progress that we have made has been
nothing short of miraculous. I can only imagine what we are
going to see in the next few years, to be quite honest with
you, with this type of discoveries that will result from this
innovation, and that is exactly what it is.
Dr. Hood, I want to ask you. Biomarker testing, what does
it mean in the context of precision medicine?
I heard you say earlier today, and I found it fascinating,
being a pharmacist, and I am thinking of it in terms of from
the way that the insurance companies now, you know, you have a
formulary, and you have to take that medication on that
formulary.
But what you are talking about, you would be able to
identify specific drugs that work for specific patients, and I
am trying to get my arms around how the insurance companies are
going to adjust to that and how we in CMS and Medicare and
Medicaid, how we are going to adjust to all of that because it
is no longer going to be a competitive bidding system to get
your drug on the formulary if it is, indeed, as you say,
patient-specific in precision medicine like this.
Dr. Hood. Well, my feeling is it will not be quite patient
specific, but there will be different categories of patients
and smaller categories than they are used to.
So blood biomarkers are the most common. The things they
can do are, one, I can identify biomarkers that for statins can
tell me who can take that successfully and who can avoid the
complications of statins, diabetes, muscle pain, and all of the
other things.
Another use is to be able to see the wellness to disease
transition at its earliest stage, and that marker lets us think
about therapy at that point in time, at a reversal of disease
before it ever manifests.
So blood biomarkers can look at any state change or they
can look at populations that respond to external stimuli like
drugs.
Mr. Carter. You know, I believe you are going to run into
some barriers here because we run into it now. You know, let's
stick with statins. You know, there are certain statins that
work better for patients than others because of the side effect
profile or because of the patient's biology, if you will,
whatever.
But, you know, again, I think this is going to be a barrier
with coverage. I mean, let's face it. Some patients, they have
to take whatever the insurance company is going to pay for.
They cannot afford to pay out of pocket.
What other barriers are----
Dr. Hood. Well, if, in fact, the drugs that the insurance
companies pay for work every time, then that is going to be an
enormous improvement in the long run for better response.
Mr. Carter. Absolutely.
Dr. Hood. OK? And, again, on the ten most common drugs,
about ten percent of the people responded effectively. Ninety
did not. So if we can clear those 90 away, we are going to save
hundreds of billions of dollars in cost for drugs.
Mr. Carter. That is good news.
Dr. Hood. And that is not saying we have to reprice or
anything. That is just making sure if we match the right drug
to the right patient.
Mr. Carter. Thank you, Dr. Hood.
Dr. Abernethy, I want to get to you really quick and I only
have a minute left.
We all know what is happening in China, and we all know
that the United States has been on the forefront of genomic
testing, and it remains the world leader. However, China
recently has begun a significant push into genomics and through
state-backed entities.
And how these companies and the Chinese Communist Party use
the data that will become available through genomics research
is a major concern with the Intelligence Committee. I have been
in hearings here at the Capitol where they have expressed
concerns to us about what they are going to do with that.
How concerned are you about the threat of China and what we
can do to counter China's advances and potential data theft?
Dr. Abernethy. Thank you for that very interesting
question.
Practically speaking, I think that we have the opportunity
and responsibility to do the critical work, especially in
genomics and other areas here in the United States.
Practically speaking, the more of that that gets done
outside of our country takes away from our ability to innovate
here and also increases risk of, for example, specific
information about our population to be in other places where
the people can do things that----
Mr. Carter. But do you think it can be used adversely?
Dr. Abernethy. I do not have specific knowledge about how
it can or cannot be used adversely, but I certainly can imagine
ways that genomic information can be used by bad actors when
they want to.
Mr. Carter. Dr. Hood, do you think it could be used
adversely?
Ms. Eshoo. Doctor, you need to turn your microphone on
please.
Mr. Carter. Microphone, microphone.
Dr. Hood. The major threat to us in longitudinal phenome
analysis is China. They are the only one, apart from us, that
is doing this in a major way, and I think the key is going to
be to fund this in an aggressive way so we will remain a world
leader in this particular area.
Mr. Carter. If they stay with it, and they will stay with
it. They are no friend of ours.
Ms. Eshoo. You have----
Mr. Carter. And I know I have gone over, and I appreciate
your indulgence, Madam Chair.
Thank you all, both.
Ms. Eshoo. Certainly. The gentleman yields back.
The Chair now recognizes the gentleman from California, Mr.
Cardenas, for his 5 minutes of questions, followed by Mr.
Crenshaw, followed by Ms. Kelly, and then finally followed by
Dr. Joyce.
Mr. Cardenas. Thank you, Madam Chairwoman Eshoo and also
Ranking Member Guthrie, for holding this important hearing.
And I want to thank all of you who are witnesses today and
experts helping to educate us policy makers in the Energy and
Commerce Committee here in Congress.
Biomedical research is the foundation for the development
of future medical treatments and cures, and this hearing will
help inform us, the legislators, on how to improve on these
processes.
Today I would like to focus on the importance of diversity
and inclusion in biomedical research. We know that there are
many groups that are underrepresented in clinical trials and
biomedical research, including racial and ethnic minority
groups, sexual and gender minority, people living with
disabilities, people who have low income or low educational
attainment, and rural residents.
Mr. Falcon, I want to also thank you for your decades of
work in this area. Based on your testimony, you have extensive
experience in community engagement efforts and providing
traditionally underrepresented groups the opportunity to be
heard on issues related to their health. So thank you for that.
In your testimony, you state that the biomedical research
enterprise has not met the standards for diversity and
inclusion set forth by the NIH, the Revitalization Act of 1993.
It has been offered that one reason for this is that much
of the Food and Drug Administration and the National Institutes
of Health guidance on inclusion is nonbinding. I will repeat.
It is nonbinding.
That means that it is recommended and not required. And as
you mentioned, this unfortunately does not seem to be enough. I
want to provide a very recent example of this.
Aduhelm, which costs about $56,000 a year, was approved
just a few months ago by the FDA for the treatment of
Alzheimer's disease, despite older Black adults being estimated
to have Alzheimer's at double the rate of White adults. Only .6
percent of the study participants were Black.
Additionally, only three percent were Hispanic, and .03
percent--that is one person in the study--was Native American.
Clearly, the nonbinding approach is not working.
From your vantage point, Mr. Falcon, what further actions
could Congress take to improve representation in clinical
trials?
And what are the consequences of inadequate inclusion in
biomedical research?
Mr. Falcon. Well, thank you, Representative Cardenas, for
the question and for your leadership on this issue.
Very clearly recommendations have not worked. We have had
decades of recommendations. It is time to make the
recommendations binding, and that can be done in some very
clear and, frankly, very straightforward ways.
At NIH, there is a review process. That review process now
should include a score tied to funding on whether or not the
study meets standards of community-based participatory
research, which NIH itself has said is the gold standard of
research.
That score should include whether or not there is adequate
inclusion of underrepresented groups, whether or not the study
is designed to power to report out findings specific for those
underrepresented groups, and whether or not the study design
includes all the principles of community-based participatory
research, and finally, all study research should be reported to
report out data by race, ethnicity, sex and gender, as recently
required now by the New England Journal of Medicine.
Those changes would dramatically change the landscape of
research being approved and research being reported out of NIH.
With regard to the FDA, in a very straightforward way,
again, the FDA review standards of clinical trial proposals
should include a review of inclusion and should set metrics for
clinical trials as they progress for meeting those standards of
inclusion.
And if those standards are not being met, there should be
enhancement required during the clinical trial process so that
we do not get to the end of a clinical trial without adequate
inclusion.
The effect on healthcare has been dramatic of there being a
lack of diversity. Right now one in five cancer clinical trials
fail because of lack of enrollment. It is very expensive to get
to the point of starting a clinical trial, and if we are
failing simply because we cannot achieve enrollment, we are
failing in terms of innovation, and we are failing in terms of
delivering on the promise of healthcare for all.
Mr. Cardenas. Thank you, and that is why I am working with
my UC colleagues that are Representatives Kelly, Butterfield,
and Clark on a bill to improve inclusion in clinical trials,
and I am so grateful for the impact that you have been able to
give us today.
With that, I apologize for going over my time, Madam
Chairwoman. I yield back.
Ms. Eshoo. I would just add that is the beauty of a hearing
in the Congress of the United States.
It is a pleasure to recognize the gentleman from Texas, Mr.
Crenshaw for your 5 minutes of questions.
Mr. Crenshaw. Thank you, Madam Chair. Thank you and the
ranking member for holding this hearing.
I know that this is of particular importance to the Chair
of the subcommittee because she represents many of the firms
that make these drugs and is also very passionate about finding
new cures.
In Houston, we also have a very strong biomedical
innovation sector, and we are very proud of that. I am going to
talk about one of my constituents. This committee paved the way
for her innovation with the CURES Act working on adult stem
cells.
So Donna Chang works on this future of medicine, curing a
person's disease with their own stem cells, and she is doing
clinical trials with stem cells on Parkinson's, long COVID, and
other neurodegenerative diseases.
And these are not unsafe treatments. They are not fringe
doctors. They are FDA approved trials and procedures that show
promise, but they do get stuck in the regulatory framework set
up for stem cell therapies.
So, Dr. Abernethy, if you would indulge me for these
questions, stem cell therapies are supposed to be regulated
under the RMAT pathway established in CURES. For these more
advanced treatments, specifically what I am talking about, they
sometimes do not meet those strict requirements set by the RMAT
pathway and, therefore, are regulated as a drug through that
pathway.
Stem cells are part of a person's body. Could they be
regulated in the same way as we regulate other autonomous
bodily tissue?
Dr. Abernethy. Thank you very much, Mr. Crenshaw. An
interesting question, and certainly we are all looking for
better ways to take care of patients and to personalize.
I do not really have an opinion as to whether or not they
can be regulated outside of their current pathway, and
practically speaking, I do think regulatory innovation is going
to be important across this space.
Mr. Crenshaw. Do you think the RMAT pathway could be
amended to broaden the number of stem cell therapies that can
be safely approved?
Dr. Abernethy. I honestly have not explored this specific
question. So I do not know the answer to that question.
Mr. Crenshaw. Yes or no?
Do you think we need to act on that as a Congress to design
new drug pathways for stem cell therapies?
Dr. Abernethy. It seems like this is an important question
to this committee. So this is an important time to look in
detail.
Mr. Crenshaw. Oh, boy, here we go. Mesenchymal stem cells
are more and more commonly extracted from adipose tissue. OK?
So we are talking about just taking stem cells from fat. All
right? Let's have a normal conversation.
Unfortunately, the FDA has ruled that the stem cells taken
from fat tissue just do not pass the muster of the definition
of minimally manipulated, and in my conversations with the FDA,
I learned that they are hesitant on adipose tissue stem cells.
It does not come from a safety concern but a lack of
knowledge on this tissue. They do not know how the process of
removing the adipose tissue changes the function of the stem
cells.
And maybe you still cannot answer it, but maybe you can
shed some light since you spent some time at the FDA. I mean,
short of writing a bill that tells the FDA how to interpret
what minimally manipulated means, what can Congress do to help
close their knowledge gap on stem cells derived from adipose
tissue?
Dr. Abernethy. So again this is not an area where I can
specifically comment with discrete knowledge. I think your
question about what can Congress do to help FDA in these areas
is an important one.
Critically, there are often areas where there is evolving
science and FDA needs the opportunity to have the personnel, so
essentially the scientists at FDA have the scientific dialogue,
for example, together with the National Academy of Medicine and
others and also the opportunity to update a regulatory pathway
that is needed.
And so as Congress there is the opportunity to make sure
that the FDA has the right resourcing and also the right sense
of urgency to solve these problems.
Mr. Crenshaw. And based on your answers, it seems like we
do need to tell the FDA what to do. I think maybe that is the
point I want make here.
And I think there could be some great bipartisan work to
get really cutting edge, effective treatment to people that
right now are just tied up in a web of paralysis by analysis at
the FDA. And ironically some of these treatments are potential
treatments for paralysis. We have actually seen some real
interesting case studies from this particular biomedical
research firm that I was talking about.
So I hope that is something this committee can work on.
And I yield back.
Ms. Eshoo. The gentleman yields back.
I would suggest to the gentleman perhaps a briefing, you
know, to meet with the FDA. I would be happy to work with you
on that if you so choose to do it.
The gentlewoman from Illinois, Ms. Kelly, a wonderful
member of this committee, the subcommittee, the full committee.
You are recognized for 5 minutes.
Ms. Kelly. Thank you, Madam Chair and Ranking Member
Guthrie, for holding this hearing on the future of biomedicine.
COVID-19, as we all know has had----
Ms. Eshoo. Is your microphone on?
Ms. Kelly. Yes.
Ms. Eshoo. OK.
Ms. Kelly. COVID-19 has had a devastating impact on our
country's physical and mental health. According to a U.S.
Census Bureau survey, Black and Latinas individuals were
disproportionately affected by mental health issues during
COVID-19.
However, according to the National Institute of Health,
clinical trials for depression treatments funded in 2018 had a
median participation of 67 percent white participants but only
11 percent Black and seven percent Latinx participants.
These numbers are not reflective of racial and ethnic
diversity in the United States and future clinical trials need
to reflect the disproportionate impact these conditions have on
communities of color.
Unfortunately, this example is not an outlier and similar
disparities can be found in many clinical trials, from anxiety
and prostate cancer to heart disease. The lack of progress
highlights the need for increased racial and ethnic diversity
in clinical trials.
Mr. Falcon, as you mentioned in your testimony, the NIH
Revitalization Act established many of NIH's current guidelines
around inclusion of women and members of minority groups.
Are there any gaps in current NIH policies to increase
clinical trial diversity specifically around accountability for
clinical trial sponsors?
Mr. Falcon. Yes. Again, the fact that the guidance is not
mandatory is the most significant action that does need to be
taken by NIH, and unfortunately the government is following
rather than leading as I have mentioned before.
Just last month, the New England Journal of Medicine is
requiring all of its publications to include specific data for
underrepresented populations. The government should also be
doing that as well, and NIH should mandate that.
The FDA should follow a similar path, and with regard to
the FDA, I would recommend that it is time for this committee
to receive an update on the 907 plan to increase diversity in
clinical trials.
Ms. Kelly. Thank you.
Would there be any benefit to empowering NIH with greater
authority to work with clinical trial sponsors to establish
clear and measurable goals for diverse recruitment and
retention in the funding applications.
Mr. Falcon. Yes, absolutely, and in fact, the Diverse
Trials Act, one of the three components, actually asks the
Secretary to set standards around decentralized trials that
could be implemented.
I think if you talked to some of the major trial sponsors,
what they are most looking for is clarity on how to deal with
the issue of inclusion, and I do think there will be recent
activity, in fact, to a greater partnership with NIH in terms
of this issue.
Ms. Kelly. Thank you.
We need to make sure we increase accountability to ensure
that clinical trials represent the racial and ethnic
communities impacted by the disease or condition being studied.
That is why I am working on a clinical trial diversity bill
focused on the NIH with my Energy and Commerce colleagues,
Representatives Cardenas, Butterfield, and Clark.
We look forward to working with our colleagues on both
sides of the aisle to advance this important bill.
Dr. Abernethy, can you provide any examples of how
developing drugs without racially and ethnically diverse trials
can lead to drugs that might not be effective or even cause
adverse effects for certain populations?
Dr. Abernethy. Thank you for this important question.
Practically speaking, if we think about developing drugs
and we leave parts of our population, segments of patients out
of the story, it can have an adverse effect because we do not
understand, for example, whether or not there might be specific
consequences, whether those are as it relates to personal
health, like renal function, symptom experience or also other
issues, such as the issues around managing comorbidities and
co-administration of medications.
If we are going to make progress in this space, we have
three critical things that we need to do. First is we are going
to need to update our approach to design the clinical trials
themselves. So, for example, make sure that generalizability,
the eligibility criteria in clinical trials do not exclude
patients unnecessarily.
We see, for example, that some clinical trials exclude
people with HIV when there is no real reason that people with
HIV should be excluded in a particular population within that
clinical trial context.
The second is we need to make sure clinical trials meet
patients where they are whenever possible. So, for example,
decentralized clinical trial initiatives are very important.
And the third is that when specific parts of our population
cannot be involved in clinical trials for some reason, for
example, people with advanced hepatic failure, we need to make
sure that we leverage real world data sources to fill in that
knowledge gap so we understand the performance for all
patients, whether that is due to medical comorbidities, due to
racial and ethnic backgrounds, or due to inability to
participate for some other personal reason.
Ms. Kelly. Thank you so much.
And I yield back.
Ms. Eshoo. The gentlewoman yields back.
It is a pleasure to recognize the gentleman from
Pennsylvania, yet another one of the doctors on our
subcommittee that we benefit so much from, Dr. Joyce. You are
recognized for 5 minutes.
Mr. Joyce. Thank you, Chair Eshoo and Ranking Member
Guthrie, for convening this critically important hearing.
Biomedical research and the advancement of cutting-edge
therapies and their cures, they save lives. It is critical in
our role as policy makers that we acknowledge this and work
together to facilitate innovation.
That is why it is so concerning that we are still
discussing the Build Back Better Act, which would cripple this
innovation, especially with regard to rare diseases.
We also must realize that we cannot limit private research
and only fund the NIH and expect the same advancements and new
cures. Almost 90 percent of new drugs originate in their
entirety from industry, and pursuing policies that discourage
this type of lifesaving investment is counterproductive and
will ultimately harm the patients.
As a physician and as a legislator, simply this is
unacceptable.
With that said, I would like to thank the witnesses for
testifying regarding advancements in biomedical research, and I
truly appreciate your expertise in these efforts
Dr. Abernethy, like you, I am trained in internal medicine,
and I am also trained in dermatology and have spent more than
25 years treating patients with melanoma, similar to you.
I have seen what advancements in therapies have done for
patients with metastatic melanoma. That has ultimately led to
cures and lives being maintained.
Dr. Abernethy, as we move toward more individualized
medicine, such as cell-based therapies, do you believe that
regulators are prepared to review and to advance these types of
products to approval?
Dr. Abernethy. Thank you very much for your question, sir.
And you are absolutely right. In the space of melanoma, we
have watched how the landscape has changed, and with patients
sitting in front of us who are 20 years old, who are dying, now
have treatments that improve their lives.
Practically speaking, I do believe that the regulatory
framework is ready to continue to allow for the continued
approval of therapies as they become available. However, I
think that the FDA is going to need several things.
The FDA is going to continue to need to make sure that
there are enough scientists and experts at FDA to review those
applications as the science gets progressively more complex.
I think the FDA is going to need to be ready to scale from
a regulatory perspective. There are a thousand cell and gene
therapies in front of the FDA right now, and we are going to
need, for example, data and technology tools to be able to do
that work faster.
And we are also going to need to make sure that the FDA has
the tools in place to allow the evaluation of therapies across
time because as these new innovations come forward, we are
going to need to study them not for one or two years, but five,
ten, 15 years, which is going to require new ways of thinking.
Mr. Joyce. Dr. Abernethy, I agree. These new innovations,
they save lives, and given that it may not make sense to adopt
the same regulatory requirements for patients with specific
therapies as we have for more traditional treatments, what
actions should we here in Congress and regulators take to
ensure that the government keeps pace with science so that
patient access is no longer delayed?
Dr. Abernethy. So I sincerely believe that we need to make
sure that we are developing the solutions that allow us to
evaluate individual therapies across time and hone our
understanding around which patients work or for which patients
any particular intervention works.
That means that we do need the continued tools that allow
for appropriate earlier approval when it is appropriate for
both our understanding of safety and effectiveness of a
potential therapy and at least adequate understanding of that,
but also continue to evaluate that across time.
This way we are able to leverage the opportunity of earlier
approval with the balanced expectation of understanding when we
should pull back that approval or adjust it if we find out an
intervention is not working as expected.
Mr. Joyce. Thank you, Dr. Abernethy.
Dr. Schrier, who is here present today, and I have worked
and introduced bipartisan legislation to deal with the issue of
creating pathways for pediatric research.
With the time that I have remaining, can you please speak
to why it is important to make the distinction between adults
and pediatric research and the importance of this research
towards developing future cures to childhood cancers?
Dr. Abernethy. Thank you.
Three critical things. One, children are not small adults.
We have to understand how interventions work within the context
of real people, including our children.
So that is the first reason this is critical, is that we
have to make sure that we are actually doing the work with the
people for whom it matters, not just translating what we think
from adults into children.
The second is that the afflictions of children are
different than adults, and so we have to make sure that we have
done the science to address the problems that are affecting our
children, which is often different diseases.
And the third thing is that we have to make sure we
incentivize essentially research and regulatory work that needs
to happen for populations that are oftentimes not the focus of
investment for essentially the communities that capitalize the
clinical development of the future.
Mr. Joyce. Madam Chair, my time has expired. I thank you
for allowing me to extend.
And, Dr. Abernethy, thank you for being here for your
important insights.
And I yield.
Ms. Eshoo. The good doctor yields back.
We do not have any more members that wish to question. I
want members to know that they have ten business days to submit
additional questions for the record.
And, witnesses, we ask you to respond as promptly as you
can to the questions, the written questions that are submitted
to you.
Also, I would like to request unanimous consent to enter
the documents that I shared with the minority into the record.
Mr. Guthrie. No objection.
Ms. Eshoo. No objection. So moved.
[The information follows:]**********COMMITTEE
INSERT**********
Ms. Eshoo. Let me thank the witnesses, Dr. Abernethy, Dr.
Butte, Mr. Falcon, Dr. Hood, and to my constituent, Dr. Minor.
You have been with us since 10:30 this morning. So that is
what, almost three and a half hours?
But these three and a half hours have been highly
instructive because you have been excellent witnesses. We
certainly are going to put our heads together to identify the
key areas that you have brought forward. They all deserve
legislation that addresses how best to advance for the
betterment of the American people.
And when we advance in terms of all of this, it is a gift
to the world because America leads.
So thank you so much for the time and effort that you have
put into this hearing. I thank all of the members.
And at this time the subcommittee is adjourned.
[Whereupon, at 1:54 p.m., the subcommittee was adjourned.]
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