ARPA-H: The Next Frontier of Biomedical Research (House Hearing, 117 Congress)
Summary
Transcript of the House Committee on Energy and Commerce Subcommittee on Health hybrid hearing ARPA-H: The Next Frontier of Biomedical Research, convened February 8, 2022 during the 117th Congress, Second Session, published as Serial No. 117-66 (GPO 59-386). Subcommittee Chairwoman Anna G. Eshoo presided with Ranking Member Brett Guthrie and Committee Chairman Frank Pallone, Jr., each delivering opening and prepared statements. The subcommittee examined biomedical research priorities and adjourned at 2:58 p.m. with ten business days for members to submit additional written questions.
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“ARPA-H: The Next Frontier of Biomedical Research (House Hearing, 117 Congress).”
Original at www.govinfo.gov. Archived at Pandemic Darlings, https://pandemicdarlings.com/source-documents/chrg-chrg-117hhrg59386-chrg-117hhrg59386-e411686abb/
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ARPA-H: THE NEXT FRONTIER OF BIOMEDICAL
RESEARCH
HYBRID HEARING
BEFORE THE
SUBCOMMITTEE ON HEALTH
OF THE
COMMITTEE ON ENERGY AND
COMMERCE
HOUSE OF REPRESENTATIVES
ONE HUNDRED SEVENTEENTH CONGRESS
SECOND SESSION
FEBRUARY 8, 2022
Serial No. 117–66
(
Published for the use of the Committee on Energy and Commerce
govinfo.gov/committee/house-energy
energycommerce.house.gov
U.S. GOVERNMENT PUBLISHING OFFICE
59–386 PDF WASHINGTON : 2026
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 CÁRDENAS, 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 Chair NEAL P. DUNN, Florida
NANETTE DIAZ BARRAGÁN, California JOHN R. CURTIS, Utah
A. DONALD MCEACHIN, Virginia DEBBIE LESKO, Arizona
LISA BLUNT ROCHESTER, Delaware GREG PENCE, Indiana
DARREN SOTO, Florida DAN CRENSHAW, Texas
TOM O’HALLERAN, Arizona JOHN JOYCE, Pennsylvania
KATHLEEN M. RICE, New York KELLY ARMSTRONG, North Dakota
ANGIE CRAIG, Minnesota
KIM SCHRIER, Washington
LORI TRAHAN, Massachusetts
LIZZIE FLETCHER, Texas
PROFESSIONAL STAFF
TIFFANY GUARASCIO, Staff Director
WAVERLY GORDON, Deputy Staff Director
NATE HODSON, Minority Staff Director
(II)
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 Chair MICHAEL C. BURGESS, Texas
PETER WELCH, Vermont H. MORGAN GRIFFITH, Virginia
KURT SCHRADER, Oregon GUS M. BILIRAKIS, Florida
TONY CÁRDENAS, California BILLY LONG, Missouri
RAUL RUIZ, California LARRY BUCSHON, Indiana
DEBBIE DINGELL, Michigan MARKWAYNE MULLIN, Oklahoma
ANN M. KUSTER, New Hampshire RICHARD HUDSON, North Carolina
ROBIN L. KELLY, Illinois EARL L. ‘‘BUDDY’’ CARTER, Georgia
NANETTE DIAZ BARRAGÁN, California NEAL P. DUNN, Florida
LISA BLUNT ROCHESTER, Delaware JOHN R. CURTIS, Utah
ANGIE CRAIG, Minnesota DAN CRENSHAW, Texas
KIM SCHRIER, Washington JOHN JOYCE, Pennsylvania
LORI TRAHAN, Massachusetts CATHY MCMORRIS RODGERS, Washington
LIZZIE FLETCHER, Texas (ex officio)
FRANK PALLONE, JR., New Jersey (ex
officio)
(III)
C O N T E N T S
Page
Hon. Anna G. Eshoo, a Representative in Congress from the State of Cali-
fornia, 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 .......................................................................... 10
Prepared statement .......................................................................................... 12
Hon. Cathy McMorris Rodgers, a Representative in Congress from the State
of Washington, opening statement ..................................................................... 14
Prepared statement .......................................................................................... 16
WITNESSES
Keith R. Yamamoto, Ph.D., Vice Chancellor for Science Policy and Strategy,
University of California San Francisco .............................................................. 22
Prepared statement .......................................................................................... 25
Esther Krofah, Executive Director, Fastercures and Center for Public Health
at the Milken Institute ........................................................................................ 36
Prepared statement .......................................................................................... 38
Answers to submitted questions ...................................................................... 187
Geoffrey Shiu Fei Ling, M.D., Ph.D., CEO, Colonel, U.S. Army (ret), on
Demand Pharmaceuticals, U.S. Department of Health and Human Services 41
Prepared statement .......................................................................................... 44
Answers to submitted questions ...................................................................... 191
Brett P. Giroir, M.D., Former Assistant Secretary for Health, Acting FDA
Commissioner, Admiral, U.S. Public Health Service ........................................ 46
Prepared statement .......................................................................................... 48
Answers to submitted questions ...................................................................... 203
Brian J. Miller, M.D., M.B.A., M.P.H., Professor of Medicine, John Hopkins
Medicine Professor of Neurology, Johns Hopkins School Medicine ................. 58
Prepared statement .......................................................................................... 60
Answers to submitted questions ...................................................................... 205
SUBMITTED MATERIAL
Eric S. Lander, Director, Office of Science and Technology Policy, Executive
Office of the President, prepared statement ...................................................... 123
Letter of September 8, 2021, from Peter Kolchinsky, et al., to President
Biden, et al., submitted by Ms. Eshoo ................................................................ 129
Statement of February 8, 2022, from Sharon Pearce, Senior Vice President,
Government Relations, the National Kidney Foundation, submitted by Ms.
Eshoo ..................................................................................................................... 145
Article of February 10, 2022, ‘‘Beyond Diversity—Time for New Models of
Health’’ Perspective, submitted by Ms. Eshoo ................................................... 148
Statement of February 8, 2022, from the Alzheimer’s Association and Alz-
heimer’s Impact Movement, submitteed by Ms. Eshoo ..................................... 151
Press release of February 5, 2022, from the American Society for Bio-
chemistry and Molecular Biology in support of H.R. 5585, submitted by
Ms. Eshoo .............................................................................................................. 153
Letter of October 20, 2021 from George Vradenburg, Co-Founder and Chair,
et al., USAgainstAlzheimer’s,submitted by Ms. Eshoo ..................................... 155
Research ‘‘Facilities and Administrative Costs of Federally Sponsored Univer-
sity,’’ submitted by Ms. Eshoo ............................................................................. 156
(V)
VI
Page
Letter of February 7, 2022, from Noubar Afeyan, Ph.D., Founder and Chief
Executive Officer, Flagship Pioneering, to Ms. Eshoo and Mr. Guthrie,
submitted by Ms. Eshoo ...................................................................................... 162
Letter of February 1, 2022, from Dr. Jeffrey H. Kleck, to Ms. Eshoo and
Mr. Guthrie, submitted by Ms. Eshoo ................................................................ 164
Letter of February 7, 2022, from David Arrons, Chief Executive Officer,
and Danielle Leach, Chief of Comunicity and Government Relations, Na-
tional Brian Tumor Society, to Ms. Eshoo and Mr. Guthrie, submitted
by Ms. Eshoo ........................................................................................................ 166
Statement of February 8, 2022, from Rajarshi Banerjee, M.D., Ph.D., MSc.,
MRCP, Chief Executive Officer, Perspectum, submitted by Ms. Eshoo .......... 168
Letter of November 11, 2021, from Lee Hoo, M.D., Ph.D., Co-Founder and
Chief Strategy Officer ISB, Seattle, CEO, et al., Phenome HEALTH, to
Ms. Eshoo, submitted by Ms. Eshoo ................................................................... 176
Statement of February 8, 2022, from Catharine E. Krebs, Ph.D., Physicians
Committee for Responsible Medicine, submitted by Ms. Eshoo ....................... 178
Statement of February 8, 2022, from Christian Eusemann, Ph.D., Vice Presi-
dent, Research and Innovation, Collaboration Officer, Siemens
Healthineers, submitted by Ms. Eshoo ............................................................... 182
Article ‘‘What is China’s Thousand Talents Plan?’’ NatureJobs Career Guide,
submitted by Ms. Eshoo ...................................................................................... 186
ARPA-H: THE NEXT FRONTIER OF
BIOMEDICAL RESEARCH
TUESDAY, FEBRUARY 8, 2022
HOUSE OF REPRESENTATIVES,
SUBCOMMITTEE ON HEALTH,
COMMITTEE ON ENERGY AND COMMERCE,
Washington, DC.
The subcommittee met, pursuant to notice, at 10:34 a.m. in the
John D. Dingell Room 2123, of the Rayburn House Office Building,
and remotely via Cisco Webex online video conferencing, Hon.
Anna Eshoo (chairwoman of the subcommittee), presiding.
Members present: Representatives Eshoo, Butterfield, Matsui,
Castor, Sarbanes, Welch, Schrader, Cárdenas, Ruiz, Dingell,
Kuster, Kelly, Barragán, Blunt Rochester, Craig, Schrier, Trahan,
Fletcher, Pallone (ex officio); Guthrie (subcommittee ranking mem-
ber), Upton, Burgess, Griffith, Bilirakis, Long, Bucshon, Carter,
Curtis, Crenshaw, Joyce, and Rodgers (ex officio).
Also present: Representative Rush and DeGette
Staff present: Elizabeth Ertel, Office Manager; Waverly Gordon,
Deputy Staff Director and General Counsel; Tiffany Guarascio,
Staff Director; Mackenzie Kuhl, Press Assistant; Una Lee, Chief
Health Counsel; Meghan Mullon, Policy Analyst; Juan Negrete,
Junior Professional Staff Member; Kaitlyn Peel, Digital Director;
Caroline Rinker, Press Assistant; Chloe Rodriguez, Clerk; Andrew
Souvall, Director of Communications, Outreach, and Member Serv-
ices; Asad Ramzanali, Legislative Director; Kate Arey, Minority
Content Manager and Digital Assistant; Sarah Burke, Minority
Deputy Staff Director; Grace Graham, Minority Chief Counsel,
Health; Nate Hodson, Minority Staff Director; Peter Kielty, Minor-
ity General Counsel; Emily King, Minority Member Services Direc-
tor; Bijan Koohmaraie, Minority Chief Counsel, Oversight and In-
vestigations Chief Counsel; Clare Paoletta, Minority Policy Analyst,
Health; Kristen Shatynski, Minority Professional Staff Member,
Health; Olivia Shields, Minority Communications Director; Michael
Taggart, Minority Policy Director; and Everett Winnick, Minority
Director of Information Technology.
Ms. ESHOO. 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.
I would just like to make a brief statement, and then move on
with our hearing.
On the first day of President Biden’s presidency, he announced
a very high standard in terms of conduct in his Administration. Dr.
Eric Lander mistreated subordinates. It is a long record, and I be-
(1)
2
lieve that, because he didn’t live up to that standard that the Presi-
dent set, that his resigning was the right thing to do. And so he,
obviously, is not here this morning. He resigned last evening, one
of our Nation’s most brilliant scientists. And so he has stepped
down from being the director of the Office of Science and Tech-
nology Policy. Again, I think that was the right thing to do. Women
are not lesser beings.
For members and witnesses taking part in person, we are fol-
lowing the guidance of the CDC and the Office of the Attending
Physician. So we are asking everyone to please wear your 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 obviously need to unmute your microphone
when you wish to speak.
Since we have some witnesses appearing virtually today, I need
to ask my colleagues in the hearing room to mute themselves
whenever they are not speaking, so we can clearly hear the wit-
nesses’ responses. If there is background noise, it really diminishes
the voices of those that are testifying.
Since members are participating from different locations at to-
day’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 the staff. All documents will
be entered into the record at the conclusion of the hearing.
The Chair now recognizes herself for 5 minutes for an opening
statement. And before I begin that, I want to thank our witnesses
this morning for being so cooperative to move your presence up in
the hearing, given the absence of and the resignation of Dr. Land-
er.
OPENING STATEMENT OF HON. ANNA G. ESHOO, A REP-
RESENTATIVE IN CONGRESS FROM THE STATE OF CALI-
FORNIA
ARPA-H, the Advanced Research Projects Agency for Health,
presents a unique opportunity to take a major leap forward in bio-
medical sciences by funding high-risk, high-reward innovation that
will improve the quality of life for all.
Let me start by describing where ARPA-H fits by painting a pic-
ture of the current landscape of biomedical innovation, which I
think of as a tale of two mountains, with a valley in between.
On one end of the landscape, we have a mountain called basic
research, which is supported by the National Institutes of Health,
a research lab that traces its roots to a 1887 lab in the Marine Hos-
pital Service. Basic research is curiosity-driven, motivated by a de-
sire to expand humanity’s knowledge. Discoveries in basic research
are the critical building blocks for modern medicine. Everyone on
this subcommittee supports NIH. We have worked to strengthen it
and fund it, and we take great pride in it.
On the other end of the biomedical innovation landscape is a
mountain called applied research. Companies have a profit motive
to commercialize scientific discoveries with market potential. Inves-
tors take risks in applied research, but only within a narrow band
3
of what is foreseeable from the industry’s perspective. The public
depends on private investments to bring biomedical discoveries to
market.
In between these mountains of basic and applied research, it is
what is called the valley of death. There are countless ideas that
have the potential to be breakthrough cures, but the needed invest-
ment can’t be raised because the risk is too great for private actors
and is outside the realm of basic research. ARPA-H aims to turn
this sunken valley into a lofty mountain, where breakthrough dis-
coveries can be realized on the deadliest diseases we face.
How will this work in practice? For that answer, we turn to
DARPA, which is the inspiration for ARPA-H. In many ways,
DARPA mirrors the culture of Silicon Valley, which I am very
proud to represent. In the Valley, every successful entrepreneur
stands on the shoulders of failed bets that came before them. Inves-
tors take many bets within a given area, and then they quickly
double down on what works.
This similarity in cultures between Silicon Valley and DARPA is
not a coincidence. Many of DARPA’s successes happened in the
Valley. ARPANET, the precursor of the modern internet funded by
DARPA, had one of its four original network nodes at Stanford Re-
search Institute. DARPA also funded major developments in semi-
conductors made of silicon, the namesake of my region. And
DARPA is the source of GPS, which has countless academic and
commercial linkages to Silicon Valley.
ARPA-H, as proposed in H.R. 5585, the ARPA-H Act, would be
an independent agency within HHS designed to make high-risk,
high-reward investments. I have worked on the legislation for sev-
eral months after the President convened a small group of bipar-
tisan representatives—and bicameral—in the West Wing last
March to describe his vision for the agency.
Like DARPA, my legislation proposes ARPA-H to be made up of
highly empowered program managers who are not career govern-
ment employees, but are instead experts in their field who dedicate
their time to short-term projects for long-term results. Some of
these program managers could be NIH-funded career academic sci-
entists ready to break the mold. Others could be leading computer
scientists that build new methods of deploying AI to find discov-
eries for rare diseases.
I have talked to many members of this subcommittee personally
about the legislation to create ARPA-H, and it is my top legislative
priority in this Congress, and I welcome your ideas on the topic.
So if you haven’t expressed them, make sure you do to me.
Let me thank my colleagues, Congresswoman Diana DeGette and
Congressman Fred Upton. They have also put a great deal of time
and thought into this issue, and I am pleased with their support
of the legislation. Their work on Cures 1.0—we hear a continuing
refrain about the effectiveness of that—with that legislation that
became law, and now their work on Cures 2.0—many scientists
have told me that the two bills are complementary, and I look for-
ward to advancing both.
Finally, this hearing was noticed, as we said—as I said at the be-
ginning, as a two-panel, and I want to welcome the panelists that
4
changed their schedules to be the starting brilliant panel that I
know that you are.
[The prepared statement of Ms. Eshoo follows:]
PREPARED STATEMENT OF HON. ANNA ESHOO
5
6
Ms. ESHOO. So, he Chair is now pleased to recognize Mr. Guth-
rie, the ranking member of our subcommittee, for his 5 minutes for
an opening statement.
OPENING STATEMENT OF HON. BRETT GUTHRIE, A REP-
RESENTATIVE IN CONGRESS FROM THE COMMONWEALTH
STATE OF KENTUCKY
Mr. GUTHRIE. Thank you, Chair Eshoo, and thank you. I want
to associate with your words on Director Lander. Everybody de-
serves to be respected. Everybody deserves to be respected. And the
ones who always think they are the smartest people in the room,
sometimes other people are a lot smarter than you think they are,
and we need to be mindful of that, and I am just disappointed
that—I am glad he is not here today, but, you know, he has an-
swers that we need to—hopefully, the White House will have some-
body that can answer the questions as well.
Today we are discussing the proposal creating the Advanced Re-
search Projects for Health, ARPA-H, that would establish a
DARPA-like agency housed in the National Institutes of Health.
Others and I on this committee have been strong supporters of
healthcare innovation, specifically biopharmaceutical and bio-
medical research, and the great success stories—Operation Warp
Speed, which was established by President Trump at the beginning
of the COVID–19 pandemic.
I think very early in President Biden’s term a group of us went
to the Oval Office. I remember being in the Oval Office very early
in the term, and we were talking about this, and I made the com-
ment when we left—I don’t know if any of you remember—I think
I said that people are getting tired of votes going 220 to 215 in the
House. People are ready to have agencies and things that we can
work on and work on together, and healthcare is something we
have always been able to work on together.
And you know, we just—we have seen—we didn’t see that, we
didn’t see bills that were—could generate bipartisan support mov-
ing forward, and we want to work together. But there are a couple
of questions that we really need to ask, and I had told Dr. Lander
before that these would come up, and—because we had a phone
call before all of his situation.
And the questions is NIH itself, and one is just the role of a new
agency. And there is one point at NIH. Last Congress, as ranking
member of the Oversight and Investigation Subcommittee, I co-led
a letter to Director Collins of NIH and the Director Wray of the
FBI to request information on how their agencies are working to
remove foreign influence from biomedical research.
NIH is the largest funder of biomedical research in the world.
And Director Wray described how researchers from China can
mask their identity to accept millions of U.S. grant dollars to steal
U.S.-backed biomedical research to give China a competitive edge.
And I know that some of my Republican colleagues have actively
led on trying to get other information on grants from the NIH, and
we have not been able to move forward. So it makes this more dif-
ficult to try to create a new agency that would be part of NIH.
To be clear, I believe these hearings are important, and we need
to understand the gaps in care across our healthcare system, and
7
how an agency like ARPA-H could close these gaps. So we need
to—but we do have questions about how this new agency would im-
pact research efforts being led by similar Federal agencies in addi-
tion to our private sector partners.
ARPA-H is anticipated to be housed within NIH, whose mission
is to conduct fundamental basic research to ensure we have
foundational understanding of how biological systems work. But re-
member the private sector, not NIH, is responsible for bringing the
breakthrough therapies to market. Only five percent of NIH-funded
research initiatives result in treatments that come to the market,
and only yield six new patents for every 100 million spent on re-
search. However, the research is built upon by the private sector
to move forward.
And the CBO data further shows the pharmaceutical industry in-
vested 83 billion in research and development in Fiscal Year 2019
alone, with over 60 percent more drugs first coming to the market
in the U.S. between 2010 and 2019, which underscores how effec-
tive private markets are in quickly adapting to patient needs. We
ought to be finding out more about these opportunities and the in-
novation that they require, as well.
Further, the U.S. Food and Drug Administration, the Biomedical
Advanced Research Development Authority, and Defense Advanced
Research Projects Agency, DARPA, are all Federal entities working
directly with our partnering—with industry leaders, developing
cutting-edge technologies, and we want to know how ARPA-H will
fit within these ongoing efforts within the private sector, and these
agencies, and especially since there are still some unanswered
questions of how the agency will function, and I think still where
it will actually be housed.
So we are looking forward to working with members of this com-
mittee. We want to get answers to these questions about ARPA-H,
and we—but we do want to work and find policies that will bring
about innovations and cures that will help the lives of our fellow
citizens. And we thank you so much.
[The prepared statement of Mr. Guthrie follows:]
8
PREPARED STATEMENT OF HON. BRETT GUTHRIE
9
10
Mr. GUTHRIE. And Madam Chair, I will yield back.
Ms. ESHOO. Thank you. The gentleman yields back.
The Chair is now pleased to recognize the chairman of the full
committee, Mr. Pallone, for your 5 minutes of——
Mr. PALLONE. Thank you.
Ms. ESHOO [continuing]. Or an opening statement.
OPENING STATEMENT OF HON. FRANK PALLONE, JR., A REP-
RESENTATIVE IN CONGRESS FROM THE STATE OF NEW JER-
SEY
Mr. PALLONE. Thank you, Chairwoman Eshoo. Today the com-
mittee will examine the Biden Administration’s proposal to estab-
lish the Advanced Research Projects Agency for Health, or ARPA-
H, and how this proposal could enhance the landscape of bio-
medical research.
There is a lot of excitement for ARPA-H. Patient groups, aca-
demia, industry, and many others have voiced their support of this
novel model to catalyze society-changing medical breakthroughs.
Imagine a world with cancer-curing vaccines, no overdose deaths,
a genetic test to detect and actually prevent illness, and a truly eq-
uitable health delivery system. It is hard to imagine at this point
with COVID, but the possibilities with ARPA-H are endless.
Today America’s existing biomedical research ecosystem is the
best in the world. It is supported by the best universities, compa-
nies, and scientists. But there are still gaps and missed opportuni-
ties. Fundamental research conducted by universities, non-profits,
and government agency requires a high degree of scrutiny in order
to produce strong and objective knowledge. Once fundamental
knowledge is established, translational science within the commer-
cial sector takes over to develop cures, treatments, and technologies
that address patient needs.
And this process involves a degree of risk that often times stands
in the way of making lifesaving discoveries. Some of the risk fac-
tors include cost and recruitment for clinical trials, scalability, reg-
ulatory pathways, and the question of profit. At the end of the day,
the priorities of the academic and commercial sectors may result in
ideas simply not being pursued that are considered too high-risk,
having a significant cost, or where the potential commercial market
would not support the cause.
So advanced research agencies like the Defense Advanced Re-
search Projects Agency, or DARPA, have addressed these gaps.
DARPA has done this by building on established fundamental re-
search, and funding time-limited, milestone-based translational re-
search. This high-risk, high-reward model allows the defense re-
search ecosystem to understand what works and what does not,
without spending decades of resources on trial and error. And as
a result, DARPA has developed platform technologies that have
changed the world, technologies that we use every day, including
cell phones, the internet, flat screen displays, and global posi-
tioning systems.
DARPA is part of the Department of Defense and, importantly,
it has not supplanted any function of the Department’s vast struc-
ture because its mission is unique and specific. So the same is true
for ARPA-H. Its mission is to make pivotal investments in break-
11
through technologies that can’t readily be accomplished through
traditional research and commercial activity. And I look forward to
hearing from our panel of expert witnesses who have decades of ex-
perience in academia, fundamental research, Federal public health
agencies, and, of course, DARPA to discuss the mission structure,
authority, timing, and funding of ARPA-H.
Now, although this is not a legislative hearing, appropriators
have introduced legislation that will fund ARPA-H at $3 billion in
the House and 2.4 billion in the Senate. And that funding is contin-
gent on this subcommittee writing and passing authorizing legisla-
tion. There are two existing proposals that would do so, one intro-
duced by Chairwoman Eshoo and the other introduced by Rep-
resentative DeGette. And I want to thank both of them for their
outstanding leadership on this important issue.
This subcommittee has a strong bipartisan history of supporting
Federal biomedical research. The proposal for ARPA-H is another
opportunity for us to work together and establish an agency that
will have a direct impact on fundamental research, breakthrough
technologies, and healthier patient outcomes, and, of course, keep
us at the forefront of biomedical research in the United States.
[The prepared statement of Mr. Pallone follows:]
12
PREPARED STATEMENT OF HON. FRANK PALLONE JR.
13
14
Mr. PALLONE. So I thank you again, Chairwoman Eshoo, and I
yield back the balance of my time.
Ms. ESHOO. The gentleman yields back. And now the Chair now
recognizes the ranking member of the full committee, Representa-
tive Cathy McMorris Rodgers.
OPENING STATEMENT OF HON. CATHY MCMORRIS RODGERS,
A REPRESENTATIVE IN CONGRESS FROM THE STATE OF
WASHINGTON
Mrs. RODGERS. Thank you, Madam Chair. I too want to associate
myself with your comments regarding Dr. Lander. The question be-
fore this committee this morning is whether to create a new agen-
cy, ARPA-H, partly driven by the growing concerns as to the cul-
ture at NIH. Given Dr. Lander’s sudden resignation last night, it
only raises more questions in my mind as to what is really going
on at NIH, and the culture at NIH.
I too want to thank the second panel for their flexibility in
being—appearing before this committee earlier than you first an-
ticipated.
We are discussing the proposal to address and create the Ad-
vanced Research Agency for Health, ARPA-H. It is a new bio-
medical research agency with the initial price tag of 6.5 billion over
three years.
We are all proud that the United States is the leading nation
leading the world in biomedical research and innovation. Still, mil-
lions continue to suffer from diseases that do not have any treat-
ment. I have been a long-time supporter of NIH. I have supported
doubling the funding at NIH. I have supported projects like the
Brain Initiative, intended to speed scientific research necessary to
accelerate cures for neurologic diseases. But I do have some con-
cerns with this particular proposal, and I want to address it in
three main areas.
Its intent ignores actions by the Biden Administration and
Speaker Pelosi that will destroy medical innovation, such as the
government price controls.
NIH is not cooperating or being transparent with Congress on
how existing research funded by taxpayer dollars is being spent, es-
pecially in China.
And last, many questions about the ARPA-H proposal itself re-
main unanswered.
Regarding innovation, my colleagues and I will send a letter to
Secretary Becerra today to detail how the proposed national cov-
erage determination for all biologics targeting amyloid for Alz-
heimer’s will devaState innovation, and hurt patients who rely on
it. Bipartisan members of this committee have also written in oppo-
sition to the Administration’s decision to repeal a final rule that
would have provided Medicare coverage for FDA-approved break-
through medical devices.
I am concerned that innovation-crushing decisions like these are
a preview of how the Administration would abuse its power under
government price controls. If innovation is truly what ARPA-H is
about, re-proposing the NCD for Alzheimer’s patients and
innovators, giving them hope, reinstating the innovative medical
15
device regulations, and abandoning government price controls are
reasonable steps we should all take.
Second, I am not convinced that a brand new agency is the an-
swer to or will be able to overcome the institutional, cultural, and
bureaucratic barriers that are present at our Federal scientific
agencies. Our COVID–19 origins investigation has revealed that
NIH has failed to do proper oversight and ensure accountability
over research dollars, especially the risky research in China. Right
now NIH has a long way to go to build trust. It should start by
providing complete transparency by complying with congressional
oversight.
Before we give the executive branch more authority and re-
sources, let’s make sure that we get the answers on what is being
spent today, and why.
Onto ARPA-H itself. There is a fundamental question about the
role of the private sector and the role of the Federal Government.
Right now ARPA-H seems to lack a clear mission. I have asked for
clarity from passionate advocates, researchers, the Biden Adminis-
tration, Dr. Collins. I asked Dr. Lander the last time we spoke. Ev-
eryone has a different answer. How can we hold an agency account-
able for success without clear, measurable goals?
I am concerned about duplication. In 2006 the NIH launched the
Common Fund Program using a venture capital framework to tack-
le high-risk, milestone-driven projects to remove roadblocks in med-
ical research that impede basic scientific discovery. In 2021 the
program received over $640 million. In 2011 a new NIH center for
biomedical science, NCATS, was established to ‘‘catalyze a genera-
tion of innovative methods and technologies.’’
In the 21st Century Cures Act Congress established the Cures
Acceleration Network to reduce significant barriers between re-
search, discovery, and clinical trials. 21st Century Cures gave NIH
other tools to advance biomedical research, such as funding oppor-
tunities for young investigators, a specific program called High-
Risk, High-Reward Research, and funded the Cancer Moonshot and
the Brain Initiative, the Regenerative Medicine Innovation Project,
and the All of US research program. Are these existing programs
not working?
Let’s do the oversight. A new agency brings a lot of other costs.
Again, I am totally supportive. I want America to lead in innova-
tion and medical research, but let’s make sure that we are doing
the job we need to do, this committee oversight of existing pro-
grams.
[The prepared statement of Mrs. Rodgers follows:]
16
PREPARED STATEMENT OF HON. CATHY MCMORRIS RODGERS
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Mrs. RODGERS. I yield back. Thank you.
Ms. ESHOO. The gentlewoman yields back.
The Chair would like to remind Members that, pursuant to com-
mittee rules, all Members’ written opening statements will be made
part of the record.
Now this is one of the best parts of the hearing, is to introduce
our witnesses. And we—colleagues, this is really a sterling panel
of witnesses.
The first, Dr. Keith Yamamoto, and he is here with us in person.
He is the vice chancellor for science policy and strategy, director of
precision medicine, and professor of cellular and molecular pharma-
cology at the University of California, San Francisco.
Welcome to you, Dr. Yamamoto. It is an honor to have you with
us.
Virtually we have Dr. Esther Krofah. She is the executive direc-
tor of FasterCures and the Center for Public Health at the Milken
Institute.
Thank you to you for being with us.
Dr. Geoffrey Ling, here in person, is the CEO of On Demand
Pharmaceuticals, a Johns Hopkins School of Medicine professor,
and John Hopkins Hospital attending physician. Dr. Ling also
served as the founding director of DARPA’s biological technologies
office, and is a retired colonel with 21 years of service as an Army
medical officer.
It really is difficult to abbreviate your backgrounds. So, col-
leagues, I am just giving a snapshot. But if you go into the testi-
mony and the bios, you will be reading, single space, for quite a
while. Our country is blessed with the leadership of each one of
these individuals.
Dr. Brett Giroir has served as the assistant secretary of health
at HHS. He has testified many times at our committee, the acting
FDA commissioner, director of the defense sciences office at
DARPA, and admiral in the U.S. Public Health Service Commis-
sioned Corps.
Welcome to you, Dr. Giroir. It is good to have you with us once
again.
Dr. Brian Miller is here with us in person. He is a practicing
hospitalist, and an assistant professor of medicine and business at
the Johns Hopkins University School of Medicine.
Welcome to you.
So to each one of you, we are proud to have you here. We are
grateful to you. We look forward to your testimony.
For those that are here with us in person, you are probably fa-
miliar with the system of lights here in front of you. You have 1-
minute remaining when the light turns yellow, and I think every-
one knows what a red light is signaling.
So, Dr. Yamamoto, thank you again. You have 5 minutes for your
testimony.
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STATEMENT OF KEITH R. YAMAMOTO, PH.D., VICE CHAN-
CELLOR FOR SCIENCE POLICY AND STRATEGY, UNIVERSITY
OF CALIFORNIA SAN FRANCISCO; ESTHER KROFAH, EXECU-
TIVE DIRECTOR, FASTERCURES AND CENTER FOR PUBLIC
HEALTH AT THE MILKEN INSTITUTE; GEOFFREY SHIU FEI
LING, M.D., PH.D., CEO, ON DEMAND PHARMACEUTICALS
PROFESSOR OF NEUROLOGY, JOHNS HOPKINS MEDICINE;
BRETT P. GIROIR, M.D., ADMIRAL, FORMER ASSISTANT SEC-
RETARY FOR HEALTH, U.S. DEPARTMENT OF HEALTH AND
HUMAN SERVICES; AND BRIAN JAMES MILLER, M.D., M.B.A.,
M.P.H., PROFESSOR OF MEDICINE, JOHN HOPKINS MEDI-
CINE
STATEMENT OF KEITH R. YAMAMOTO, Ph.D.
Dr. YAMAMOTO. Good morning, Chairman Eshoo, Ranking Mem-
ber Guthrie, and members of the subcommittee. It is also nice to
see my friend, Congressman DeGette, here, as well. And it is an
honor to present a statement before you today.
I shall address two questions: first, why at this moment of spec-
tacular discoveries about biological mechanisms and disease, most
of them NIH-sponsored, should Congress be establishing another
agency, ARPA-H; and second, why should Congress ensure that
ARPA-H is fully independent, with a culture and practices that
seem almost polar opposites to NIH’s successful model?
First, why ARPA-H now? The policy framework for Federal sup-
port of U.S. science and technology was set in 1945 by President
Roosevelt’s science adviser. Government would fund basic re-
search—that is, discovery of new knowledge—and training of fu-
ture generations of scientists. Industry would then develop the new
knowledge into products from which they would profit, and the
American people would enjoy a happier, more secure quality of life.
In the case of health, for example, we needed basic research to
understand biological processes, how molecules collaborate to make
cells, tissues, organs, and healthy human beings. Basic research
has to be untargeted. We don’t know what we don’t know. So NIH
created a competitive funding program giving university scientists
freedom to let their curiosity, ingenuity, and expertise determine
which biological processes they wish to study, and how they would
study them.
NIH’s funding apparatus is driven by peer review. Working sci-
entists serve on committees to decide which proposals submitted by
their colleagues will win funding. With many scientists each pur-
suing whichever biological process intrigues them, the gaps in our
knowledge are being filled.
Now, peer review is not perfect. Dr. Francis Collins, just retired
as NIH director, acknowledged that the NIH process ‘‘is a little
slow, a little conservative, not necessarily going to embrace the
transformative projects.’’ Yet this curiosity-driven model is, by any
measure, the world’s greatest knowledge discovery engine for bio-
medical research. For example, 99 of the 230 Nobel Prizes ever
awarded in chemistry, physiology, or medicine have gone to 163
NIH-supported scientists. Astonishing dominance.
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However, new knowledge alone is insufficient to motivate indus-
try to develop applications. Among the 9,000 known human dis-
eases, there are approved treatments for only about 500. And
among the 24 most impactful drugs on the market, the median
time between the key bit of knowledge discovery and FDA approval
was 32 years.
Clearly, biotechs face many barriers: economic risk too high,
near-term markets too small, scope too broad for any one company
to realize profit, industry alone unable to do the job. Thus, Federal
science and technology policy needs a revise. Government support
is required to de-risk industry participation, and government co-
ordination and management are required to set and meet auda-
cious goals. That is ARPA-H.
So question No. 2, why should Congress endow ARPA-H with a
drastically different culture and operating model? Consider first
ARPA-H’s distinctive goals. Its starting point is NIH’s endpoint:
discovered knowledge is the foundation for ARPA-H development of
platform technologies, devices, therapeutics. It seeks applications,
rather than discovery of knowledge to demonstrate feasibility of
transformative concepts, de-risking development.
Next, look at the ARPA-H operating model, which draws on
DoD’s DARPA, which Drs. Ling and Giroir will discuss, and may
have been mentioned here already. Support and manage program-
specific, transdisciplinary, multi-sector partnerships and teams to
meet contract goals set in and enforced by ARPA-H program man-
agers. Embrace bold approaches, tolerate failure, create advanced
technologies, computational tools, novel materials, imaging meth-
ods all leveraged on chronic and infectious diseases, and on count-
less rare diseases which afflict millions, but receive scant attention
due largely to market size concerns.
None of this looks like NIH, so ARPA-H needs to look and act
differently. At the same time, though, NIH must continue to thrive
for ARPA-H to succeed. Thus, Congress should install safeguards
to prevent ARPA-H funding from supplanting NIH investments or
threatening its culture.
For ARPA-H itself, Congress should provide independence to con-
struct a flat, nimble operating model that supports program man-
agers, each overseeing a daunting health challenge and a bold path
to its solution. Congress should grant the director and program
managers authorities, flexibilities, and, yes, appropriations for hir-
ing, diversity, contracting, broad partnering, and ethical and effi-
cient IP and tech transfer.
Importantly, ARPA-H should be authorized as an independent
agency within HHS, rather than as a component of NIH. Dr. Re-
gina Dugan, a renowned former director of DARPA, puts it this
way: ‘‘An organization like the ARPA-H exists to challenge conven-
tional wisdom. Don’t put it inside the very organization that holds
the conventional wisdom.’’ Creating a new culture and operating
model is always difficult, but creating it within a very different and
long-established one is likely impossible.
Thus, the actions of Congress in authorizing these agencies will
influence, if not determine, its success or failure. Thankfully, the
legislation developed by Chairwoman Eshoo and all of you wisely
24
recognizes the critical elements of independence, authority, culture,
policy, and practice that will place ARPA-H on a positive trajectory.
ARPA-H repairs a weakness in our Federal science and tech-
nology policy. ARPA-H will consolidate new scientific knowledge
and devise strategies and tools that improve and extend lives for
all, including those long disadvantaged.
This concludes my testimony. I would be pleased to answer any
questions. Thank you again for the opportunity to?
[The prepared statement of Dr. Yamamoto follows:]
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Ms. ESHOO. Thank you, Dr. Yamamoto. Next we will hear testi-
mony from Ms. Esther Krofah.
You have 5 minutes for your testimony.
STATEMENT OF ESTHER KROFAH
Ms. KROFAH. Thank you, Chairwoman Eshoo, Ranking Member
Guthrie, and members of the Subcommittee on Health, for the op-
portunity to provide input on the proposed Advanced Research
Projects Agency for Health, ARPA-H. My name is Esther Krofah,
and I am executive director of two centers of the Milken Institute:
FasterCures and the Center for Public Health.
FasterCures is driven by a singular goal, to save lives, by speed-
ing scientific advancements to all patients. We like to say our name
is our mission. With an independent voice, FasterCures is working
to build a system that is effective, efficient, and patient centered.
During the pandemic we have witnessed the rapid development of
effective COVID–19 vaccines in under a year, and development of
therapeutics and diagnostics that demonstrate how critical sci-
entific discovery translated into real products and interventions
save lives. But many patients are asking, if it can be done for
COVID–19, can it also be done for my disease condition?
This morning I speak to you as my father is fighting stage four
cancer, hoping for some more time so that he can see his children
and grandchildren achieve their dreams. ARPA-H holds the prom-
ise to work at the cutting edge of science to take risks and achieve
breakthroughs that can improve lives like my father’s, but count-
less others.
FasterCures has long supported NIH DARPA-like authorities
and capabilities for more high-risk, solutions-oriented R&D. So we
are gratified that this concept is being seriously considered at this
time.
My comments today will be in the following areas: the structure
of the proposed new agency, including its location, leadership, au-
thorities, and funding; second, its activities, including priority set-
ting and coordination with other agencies and sectors.
Some have questioned whether ARPA-H should be housed within
NIH, as currently proposed. There is, of course, a more recent ex-
ample of the DARPA model that has been stood up and can be
looked to for lessons learned. And that is ARPA-E, which resides
within the Department of Energy, but employs an operating model
like DARPA’s. We have seen with ARPA-E that an entity like this
can exist within a larger Federal agency, and still foster a different
culture and operating model with the right toolkit and key ingredi-
ents. As such, we do not see a reason ARPA-H could not be situ-
ated within NIH and still accomplish its mission, including advan-
tages to having easy access to other NIH infrastructure, personnel,
programs, and expertise.
We would like to emphasize that this new entity should not be
considered as a substitute for the National Center for Advancing
Translational Sciences, NCATS. NCATS has a broad remit to sup-
port the whole field and discipline of translational and clinical re-
search. That needs to remain distinct and well-supported.
37
Who leads a new ARPA-H entity will be critical, especially as its
first leader, and should be selected by their visionary capacity and
ability to inspire and empower a new team, directing milestone-
driven initiatives. ARPA-H will need to ensure expertise from the
private sector is engaged, both internally and externally. It should
ideally have a leader with experience outside academia, with a
proven track record of success and managing through failures. It
will need an external advisory body comprised of patient organiza-
tions, industry, academia, and other non-profits to inform the agen-
cy’s priorities.
Representatives should also include those from under-served mi-
nority communities, defining problems that are most important to
be solved.
DARPA’s program managers are a critical asset, and should also
be for ARPA-H. Ensuring key people are recruited for those posi-
tions is central to the culture necessary for the success of this ef-
fort. This is likely to require freedom from the usual constraints of
the Federal hiring process, in order to bring in the right people for
limited durations, do it quickly, and pay them appropriately.
Perhaps more important than the exact budget number is a con-
sistency of funding and sustainability over time. This needs to be
a multi-year commitment of effort and funding. It is bigger than a
3-year budget line item.
Collaboration with other Federal agencies is necessary for the
success of ARPA-H. FDA is, obviously, a critical link in the process
that gets exciting new science and products into the hands of pa-
tients. We need to make sure they have the resources and expertise
to keep pace and effectively regulate new technologies coming to
them for review through efforts like ARPA-H.
There also needs to be active and regular engagement with other
agencies critical to advancing solutions to patients, such as CMS
and CDC.
ARPA-H should develop a data-driven and transparent process
for setting priorities, including and prioritizing conditions with
high unmet need and low innovation activity.
In creating this new entity, we should heed key lessons from the
COVID–19 pandemic. Investment should be prioritized in platform
technologies and research infrastructure.
I would like to conclude by thanking you for the opportunity to
offer input. I am happy to discuss these ideas and answer any
questions you may have.
[The prepared statement of Ms. Krofah follows:]
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Ms. ESHOO. Thank you for your testimony.
Next, the Chair recognizes and thanks Dr. Geoffrey Ling for
being with us today in person.
You have to know that this is some—it is a treat for us to have
people here in person. It has been a rarity now for two years. So
welcome to you, and you have 5 minutes for your testimony, Doc-
tor.
STATEMENT OF GEOFFREY SHIU FEI LING, M.D., Ph.D.
Dr. LING. Thank you, Chairwoman Eshoo. Good morning, Rank-
ing Member Guthrie and distinguished Members of the Congress.
I have to tell you that—you have been thanking us, but I have to
thank you. This is a life memory for me. So thank you all.
My name is Geoffrey Ling, and I want to start by saying I am
an Army officer. So the way I talk, please forgive me, because I
spent 21 years as a military officer. I served in the United States
Army. I served in Afghanistan. I served in Iraq as a military physi-
cian.
I was fortunate, since leaving government service, to be able to
go ahead and become CEO of my own company called On Demand
Pharmaceuticals. I am also a professor of neurology at the Johns
Hopkins, where I still practice medicine on occasion. I am getting
kind of old for it, but it is what it is. So my comments are my own,
please. They do not reflect that of Hopkins, On Demand, or U.S.
Army.
Relevant to this hearing, I was the founding director of the bio-
logical technologies office at DARPA, and I served at the agency for
11 years. Also relevant to this hearing, I served for the NIH for 14
years. I was on advisory councils and study sections. So from this
perspective, I am going to address my comments.
DARPA was found in 1958 in response to an existential threat:
Sputnik. ARPA-H is being considered in the shadow of a real
threat, COVID.
But in adversity, there is opportunity, there is a real chance to
do something new, do something special, do something bold, and
that is create an agency that says yes. Everybody else looks to say
no. No is the easiest answer. It means you don’t have to do any-
thing, it means you are happy with the status quo. It means that
you are just fine to go back to do what you were doing.
But you want an agency that says yes, yes, I will go after autism;
yes, I will go after Alzheimer’s; yes, I will go after glioblastoma
multiforme, a brain tumor. And it is just not saying yes, it is know-
ing how to say yes. It is not about more money, it is about how
to spend the money. Not what to spend the money on, but how to
spend the money on.
When I was at DARPA we recognized that this is taxpayer
money, not my money. This is not the investigator’s money, it is
taxpayer money, the people work every doggone day who expect
you to perform against it to deliver something for them. And how
do you do that? You do that by making sure that everybody knows
what they are trying to accomplish.
This is about affirming, changing, or rejecting current clinical
care. If you are not doing one of those three things, you are not
doing the job. You want to affirm it, you want to reject it, you want
42
to change it. You have got to come to work every day, knowing
that. You have got to come to work thinking that you have autism,
or your child has autism. When we looked at DARPA performers,
we expect them to have that level of commitment.
And what was given in return? DARPA program managers—
ARPA program managers will work with those groups from end to
end. It is not about doing the science. That is the first step. You
have got to get through regulatory. Then someone has got to make
it, then somebody has got to distribute it. And then the patient
community has to embrace it. It is an end-to-end solution. Pro-
grams are meant to address all of these.
At the start of a DARPA BTO project, it is not just the scientists.
It is the scientist who has been reviewed by, not peers, I am sorry,
but they are reviewed by NIH officers, DoD science officers, FDA
science officers. Why the FDA, you would ask? Because the FDA
should be there at the beginning, because you got to get through
them. It is a fact of life. If they are not there at the beginning, they
are not going to be there at the end.
You have to resource these performers, give them the people they
need, give them the equipment they need, give them the money
they need. But do not give them the time they ask for. Time is the
worst enemy we have. ARPA-H is going to be like DARPA. Hold
them to time. You are going to say—a lot of people are saying, ‘‘Oh,
I need more time, I need more time.’’ Those are not the people you
want. You want the person who says, ‘‘Put me in, Coach. Put me
in,’’ and recognize that it is not a gift. It is taxpayer money. If they
are not delivering, fire them, get somebody else.
If you look at national baseball teams, who makes it to the World
Series? It is the people who recognize that the inside infielder may
not be the one you started with, may not be the one you are going
to end with, because at the end of the day you are trying to get
into the World Series. It is not about who, it is about the what. It
is about the mission. Get the mission done.
So when I—when you ask, should there—an ARPA-H exist, of
course. I am not going to go through all of the wonderful things
that all of my friends have said. I agree with them completely. This
is about how it should be constructed.
It should be independent. It needs to be independent, because
you need people there who are going to have the determination,
have the drive, have the urgency to get the job done, end to end.
And it means, at the very outset, the scientists, the regulators, in-
dustry, and the patient advocacy groups, and the clinicians. If you
don’t have them all there at the beginning, and you don’t have
them throughout this, you will have the valley of death the Con-
gresswoman—Chairwoman Eshoo, you are right.
We cross that by bridging it at the beginning. Look at how
DARPA does this. The model exists. You are not looking for an-
other model. It exists. All the authorities needed have been—al-
ready been awarded by the Congress. Just institute them in this
agency of ARPA-H.
We talk about U.S. losing ground. Let me tell you right now, U.S.
will not lose ground. I tell you why that is. Because of this. No to-
talitarian government would ever set up a DARPA, ever. Put smart
people, fund them, turn them loose? Who in heaven’s name would
43
do that, if you were a totalitarian dictator? You wouldn’t. You
would only do it in our kind of environment.
So at the end of the day, I want to thank the committee for al-
lowing me to speak. But I say that now is the time, and make it—
please make it independent.
[The prepared statement of Dr. Ling follows:]
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Ms. ESHOO. Spoken like a general in the Army. Thank you very
much.
Next the Chair is so pleased to recognize Dr. Brett Giroir.
You have 5 minutes for your testimony, and welcome again to
the subcommittee.
STATEMENT OF BRETT P. GIROIR, M.D.
Dr. GIROIR. Chairwoman Eshoo, Ranking Member Guthrie, sub-
committee members, thank you for the opportunity to testify at this
historic moment in American history, the creation of ARPA-H.
Long before I became assistant secretary for health, DARPA was
my passion. In 1998 I joined a technical assessment council spon-
sored by DARPA to probe scientific frontiers, and recommended
new DARPA initiatives. Five years later, I formally joined DARPA
as the deputy director, and then director of the defense sciences of-
fice, where I had the honor of working with Dr. Ling. I was the
first physician to lead an office at DARPA in its then–50 year his-
tory.
On numerous occasions I have assessed concepts for organiza-
tions modeled on DARPA. Frequently, these have failed to achieve
their potential because of fatal flaws that condemn them to medioc-
rity. America cannot afford to have a mediocre ARPA-H.
There are two overriding principles that must be the foundation
of the agency.
First, at all costs, ARPA-H must nurture a culture of the innova-
tion, where the staff seek transformational advances, not incre-
mental change; where there is no disincentive for failure, only for
not being bold enough; where conventional wisdom is generally re-
jected in favor of novel approaches; where the power of ideas is al-
ways more important than a proposal—proposer’s institutional
brand; and where the goal is to create interactive collaboration,
rather than stovepipe competition.
Second, program managers will make or break ARPA-H. The di-
rector must have the ability to motivate, attract, hire, and enable
program managers from the government, academia, non-profits,
and industry. Program managers must be diverse, entrepreneurial,
and excel across multiple scientific disciplines.
Next, let me address a few specific issues.
One, I believe it would be a fatal mistake to organize ARPA-H
within the NIH. To a great degree, we need ARPA-H because the
NIH cannot maintain a culture of radical innovation, disciplined
execution, specific accountability, and streamlined processes that
are essential for ARPA-H.
Don’t misunderstand me. The NIH is outstanding at what it
does, but it will never be DARPA or ARPA-H. As such, I strongly
support that ARPA-H be independent of the NIH, and that the di-
rector report directly to the Secretary of Health and Human Serv-
ices.
Two, ARPA-H must have rapid, streamlined, and non-burden-
some processes to make financial awards within weeks, not months
or years.
Three, ARPA leadership must be able to select performers based
on the overall likelihood of the program’s success. They must be
47
empowered to select diverse technical approaches and manage
overall risks.
Four, with the exception of core business and legal components,
ARPA-H staff must have term limits.
Five, like DARPA, ARPA-H must employ disciplined execution.
Awardees’ progress should be reviewed weekly by program man-
agers, quarterly by office directors, and annually by the director.
There are milestones and timelines, and if these are not met the
entire agency must address the root causes and attempt to remedy
them.
Six, the ARPA-H director must have deep technical knowledge in
a field, but by definition cannot be expert in everything that ARPA-
H will address. More importantly, the director must be strategic,
visionary, and able to recruit the best and the brightest. And need-
less to say, the director’s ability to communicate is paramount.
Seven, the initiatives at ARPA-H must be informed by national
priorities, but they must also intersect with scientific opportunities.
It may not be possible to ‘‘cure stage four cancer next year,’’ but
it is possible to develop systems for extraordinarily early diagnosis
of multiple cancers at home on a rapid test, enabling early treat-
ment.
In the short term, I believe it is possible for ARPA-H to develop
systems to monitor improved health equitably in the home, to pre-
vent falls among the elderly, to dramatically lessen maternal mor-
tality, to develop a new paradigm to solve neurodegeneration, and
to shield our Nation from future pandemics.
In conclusion, when the Soviet Union launched Sputnik, the
United States channeled its shock into action: DARPA. DARPA has
changed the world: the Internet, GPS, stealth, NASA, microelec-
tronics, autonomous vehicles, the bionic arm developed by Dr. Ling,
and mRNA vaccines. Much like our Nation’s Sputnik moment, it is
time for America to admit that the health of our Nation is intoler-
ably poor, that health disparities have worsened, and that we
spend far too much to get so little in return. ARPA-H is our best
opportunity to catalyze a healthier future for all Americans.
Thank you, and I look forward to your questions.
[The prepared statement of Dr. Giroir follows:]
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Ms. ESHOO. Thank you very much, Dr. Giroir. You really offered
compelling testimony to my colleagues and myself.
Next, the Chair is pleased to recognize and welcome Dr. Brian
Miller. Dr. Miller—I introduced him earlier, but we love the word
‘‘practicing,’’ practicing hospitalist.
So we are delighted to have you—honored, actually—and extra
pleased, because you are here with us in the hearing room. You
have 5 minutes for your testimony, Doctor.
STATEMENT OF BRIAN J. MILLER, M.D., M.B.A., M.P.H.
Dr. MILLER. Thank you, Chairwoman Eshoo, Ranking Member
Guthrie, and distinguished members of the Subcommittee on
Health for inviting me to share my views on biomedical research
and ARPA-H, the question for today.
As you mentioned, I am a practicing hospitalist, and my other
hat is actually as a health policy researcher. I run a 15-person
group, and have experience at four regulatory agencies, which are
sometimes barriers to innovation or, at other times, they facilitate
it. Today I am here in my own capacity, and my views don’t rep-
resent those necessarily of Hopkins or Johns Hopkins Medicine.
I think we all know that innovation—and no one would disagree
that innovation is important to us—as consumers or patients, as
citizens, as clinicians, and as a country. That is why we spend over
$70 billion in taxpayer funds across ten agencies—more than that,
actually; there is quite a list—and $31 billion through the NIH’s
Extramural Grant Program. The private sector accompanies us on
this journey, and spends around $89 billion annually, and venture
capitalists join us with an additional $36 billion.
ARPA-H, as proposed, has multiple challenges, as I see it.
The first is structural. We are duplicating core functions, admin-
istrative and otherwise.
The lack of a clear scientific mission and a strategic plan, to me,
is most concerning. I have heard all kinds of good visions about
things that we can and should do, and agree that we can and
should support innovation. However, after meeting with 5,100
stakeholders, the White House should release a clear report identi-
fying the scientific and medical research gaps, along with a stra-
tegic plan, before, as my colleagues mentioned, we spend taxpayer
money to the tune of $6.5 billion.
I do agree with all of my colleagues that the basic principles of
tenure-limited leadership, and program managers’ managerial
independence, and minimal bureaucracy are to be applauded. And
in fact, we could think about applying them to some of the existing
agencies.
But ARPA-H, even before this, is not enough. China is a rising
threat. The biopharmaceutical company market cap of Chinese
companies rose from 3 billion to 380 billion, a 100fold increase over
the last five years. There were 23 Chinese bioscience IPOs in 2020,
and of the world’s largest bioscience IPOs, seven of ten were from
China. China has more researchers than we do, and more patents
granted it. The Chinese Communist Party has made research and
development—and, in fact, dominance thereof with specific GDP
targets—a national priority, representing political risk for us. The
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Thousand Talents program, launched in 2008, recruited 7,000 sci-
entists, and even featured an ad in Nature Magazine.
So ARPA-H is really not enough if we are going to respond to
China. We need to protect our greatest achievement, which is our
biomedical research industrial complex. Instead, we should apply
the principles that we are talking about here today for ARPA-H to
part of a program that we already fund, the NIH Extramural
Grant Program, where we invest $31 billion of taxpayer funds
every year.
Let’s look to change part of the NIH and the Extramural Grant
Program into ARPA-H. Let’s have transparency and accountability
for indirect costs. Let’s have indirect cost reforms to find more
funds for researchers without an additional burden on taxpayers,
who already face inflation. Let’s decrease investigator administra-
tive burdens and, most importantly, that sort of enthusiasm and
the three shots of espresso that my colleague Dr. Ling had, as did
I, probably, this morning, let’s get that into the NIH Extramural
Grant Program.
We talk about problems. Well, we all admit that there are prob-
lems there. Let’s address them.
I also want to say that, in addition to looking at the Extramural
Grant Program and just the innovation overall, we can’t, you know,
ignore regulatory burdens such as the Medicaid Drug Rebate Pro-
gram or the need for new FDA regulatory pathways for software-
driven medical devices and other potential future states.
Thank you, and I look forward to your questions.
[The prepared statement of Dr. Miller follows:]
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Ms. ESHOO. Thank you, Dr. Miller.
OK, we have heard from all of our panelists, and the Chair rec-
ognizes herself for 5 minutes for questions.
Dr. Yamamoto, you have spent and continue to—I mean, you
have an illustrious career working with and around NIH. Why do
you feel so strongly that ARPA-H should be located outside of the
agency?
And I would also like to have Dr. Giroir and Dr. Ling answer
that question.
Dr. YAMAMOTO. Thank you for that question. The——
Ms. ESHOO. Perhaps, Dr. Yamamoto, if you could, it would be in-
teresting if you would rebut Dr. Miller.
Dr. YAMAMOTO. Excuse me? I am sorry, I missed what you said.
Ms. ESHOO. To rebut Dr. Miller, whether you agree or disagree,
helpful to us.
Dr. YAMAMOTO. But you asked specifically whether—the argu-
ment why it should be located outside of NIH. And I think that the
main force of that argument is that the mission and goals of ARPA-
H are different.
NIH is a masterful agency at discovery of new knowledge, but
does not actually extend to being able to develop applications for
that new knowledge. And the route for being able to do that has
already been cast and demonstrated extremely well in DARPA and
in ARPA-E.
Ms. ESHOO. Thank you.
Dr. YAMAMOTO. And so I think that is the reason that it should
be outside. Setting up that new culture and operating model within
the culture and operating model of NIH, as successful as it is,
right, would be challenging, at best. And my fear is that the agency
would actually fail, if it were to try to be within the NIH.
Ms. ESHOO. Dr. Ling?
Dr. LING. I was actually——
Ms. ESHOO. You need to turn your microphone on so we can hear
your commanding voice.
Dr. LING. I never want to go ahead of a ranking officer.
So anyway, I do believe that ARPA-H needs to be independent.
As we had—we have talked about the construct of this new agen-
cy, ARPA-H. To put it inside of NIH—which does its job and does
it well—it has created a certain structure, a certain organization,
and, of course, a certain culture. That culture has been successful
for what it does. We are asking ARPA-H to do something com-
pletely different, to take a bold, initial view of things, to do an end-
to-end type of approach toward building capability. That is A-num-
ber-one. The goal of ARPA-H is to create capability. The role of
NIH is to create new knowledge, which it does beautifully.
So when you ask an agency that exists, does their job well, and
you subsume another one underneath it, it is going to lose its iden-
tity, it is going to lose its structure, it is going to lose its way. So
ARPA-H, if it is going to be something new, something bold, some-
thing innovative, then it needs to be set free to do exactly that.
And I would point out that we are not doing something brand
new, never heard of before. It is following a model that has worked,
DARPA. DARPA’s model has worked. So that—and so that is really
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why I feel passionately that, for ARPA-H to work, it needs to follow
the same model.
Ms. ESHOO. Thank you.
Dr. Giroir?
Dr. GIROIR. I completely agree with my colleagues. Tom Brady
may be the best quarterback in history, but probably would not
have been a good linebacker. The NIH is outstanding at what it
does, but it does not have that type of culture, execution, and meth-
odology that an ARPA-H does need.
And look, at DARPA we frequently work with the NIH investiga-
tors, right? These are the foundations of the knowledge, that one
little glimpse to say yes to people funded by the National Science
Foundation, by the Office of Naval Research. These are very com-
plementary and synergistic organizations, but you can’t fuse them
because you lose the culture of both if you do that.
And again, you cannot condemn ARPA-H to mediocrity from the
moment it is born. I feel really strongly, as my colleagues do, that
being independent but highly interactive with all the basic re-
search agencies is the way to do this.
Dr. YAMAMOTO. Doctor—Eshoo, Ms. Eshoo, if I may.
Ms. ESHOO. Yes, Dr. Yamamoto?
Dr. YAMAMOTO. If I may, I would just comment on one point
made by our colleague, Ms. Krofah, that ARPA-H would be able to
work well within the NIH because of the demonstration of ARPA-
E being—existing within DoE. And actually, these are non-parallel
situations. And Regina Dugan, former DARPA director, has made
that point and I think that others have, as well, that the focus of
the mission agencies is narrow and really tightly defined.
And so if ARPA-H were in the NIH, it would be like—would be
much more like DARPA being within the Air Force. DARPA in the
DoD, and so we are talking about ARPA-H being within the HHS.
Ms. ESHOO. The HHS, mm-hmm. Thank you, that is very helpful.
Now I would like to recognize the ranking member of our sub-
committee, Mr. Guthrie, for your 5 minutes of questions.
Mr. GUTHRIE. Thank you very much.
And first to Dr. Krofah, my prayers are with your father. You
said your father is—in your testimony—struggling with stage four
cancer. And you are saying that he hopes that he lives long enough
to see his children and grandchildren live their dreams. My guess,
if he is watching his accomplished daughter this morning, heavily
credentialed, testifying before Congress, he is living one of his
dreams. So our thoughts and prayers go with you. And people’s
personal stories matter, and they bring us forward.
So when we were going to have a previous witness from the
White House—and I talked to him this week—one of my questions
that we wanted to answer is why was ARPA-H needed, and where
should it be, in NIH or outside, and which we are talking about
today. And so when we compare DARPA and Sputnik, it appears
to me—I wasn’t around in the 1950’s. I was a child of the—born
in the 1060’s. But there was really no private sector doing Sputnik,
or those types of things.
And so, all of a sudden, we had to put all forces of government
to move forward, and which we did this summer with COVID–19
and the mRNA vaccine. And I know that Pfizer and BioNTech used
73
their experience to develop mRNA cancer vaccines to create vac-
cines for COVID. So this is for Dr. Miller.
In fact, the company announced in October 2021 they would
begin phase two trials in U.S., Germany, Spain, and Belgium to
use technology to create colorectal cancer
[sic]. And so the—and in the ARPA-H concept paper, they use
this as an example, said if we have ARPA-H, we are going to be
able to use—take NIH’s research and use mRNA vaccines, an ex-
ample of transformative projects.
So the question is that is happening in the private sector, and
I think Dr. Yamamoto said that we have NIH doing basic research,
which we all agree, and then it is executed. That is when we need
ARPA-H to execute that research. And so my question, Dr. Miller,
is that being done in the private sector? And do we need another
government agency to do that? And we are just trying to get to the
bottom of that.
Dr. MILLER. Thank you. I agree wholeheartedly. I think that this
highlights that we need to actually define what the gap is.
So we have the NIH spending $31 billion, industry spending 89
billion, the life sciences venture capital community—largely Cali-
fornia Research Triangle Park—spending $36 billion. What are the
specific disease targets? What are the specific platform technologies
that we see as gaps? We haven’t really seen that. I have seen clear
suggestions that ARPA-H is a good idea, but not yet a clear stra-
tegic priority.
And of course, on top of this, if we are actually going to occupy
with a government agency that translational enterprise, we are po-
tentially going to crowd out private sector investment.
Mr. GUTHRIE. So I think an 83 billion in resources through the
private sector alone was invested in 2019. How do you see the mar-
ketplace, as you just kind of got to, currently adapting to unmet
clinical needs?
And what—so there—what we are trying to find—when we
talked with Dr. Lander before, it was we have NIH, as we talked
about here today, then we have a gap, and then we have the execu-
tion in the private sector. So what is the gap? And then, is the
gap—how can the gap be filled?
Dr. MILLER. I think the gap is already being filled by the private
sector, frankly, and industry. If we look at orphan indications, rare
diseases, we now have gene therapy, we have the accelerated ap-
proval pathway, which allows early market entry for oncologic
therapies for products and disease indications, which people pre-
viously had no hope.
I think, if we want to change how we do research, we should
think about getting more money out of the indirect costs that we
already spend at the NIH in the Extramural Grant Program, rath-
er than burdening taxpayers further.
Mr. GUTHRIE. So Dr. Yamamoto and Dr. Ling, or one of the two
of you who have advocated for ARPA-H, would you argue why
our—why the private sector isn’t filling that, and why ARPA-H is
needed to fill the gap? Because you are, obviously, arguing there
is a gap. And so why there is a gap, and why the private sector
is not filling it.
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Dr. LING. There is a gap. There is a gap. There are 10,000 known
diseases, there are only 700 cures and treatments. That is a pretty
big gap. I am a practicing physician, also. And I am a neurologist,
which is the worst. So there is so much in the gap in terms of what
I am able to provide patients: Alzheimer’s Disease, you can go
down the list. I am not going to do that.
There are some very simple things that an ARPA-H could do that
do—where the private sector is not filling. The private sector is ac-
tually quite conservative. And the point is that early government
investment can yield a huge amount of private industry pull.
The trick is going through the valley of death. We have talked
about that again and again. And so that is why I am saying that
this construct of an ARPA-H is to create that end-to-end, going-for-
ward type of approach. That is really what is needed. You have to
de-risk it at each step of the way. We do this at DARPA all the
time. You have to de-risk it at each handle. You have to de-risk it
for the FDA. You have to de-risk it for the industry. You have to
de-risk it for the patients and the clinicians. There is de-risking
along the way.
Government support—government, being that they don’t have
the constraints that the private industry has, for example. I mean,
I am a CEO of my own company that I founded. There was a lot
of elements to it that government funding does, in fact, really help.
But I will give you a tangible example, a very tangible example,
all right? And forgive me, this may take a little bit more than
two——
Ms. ESHOO. Yes, you need to summarize, Dr.——
Dr. LING. I will summarize it——
Ms. ESHOO [continuing]. So Dr. Yamamoto can give his an-
swer——
Dr. LING. I will tell you what. I will just yield to Dr. Yamamoto.
Ms. ESHOO. OK.
Dr. LING. I am just going to yield to Dr. Yamamoto, an old
friend.
Dr. YAMAMOTO. Thanks, Geoff.
There is a gap. I mentioned the fact that the time between the
basic science observation that eventually leads to an FDA-approved
drug is 32 years, median time. That is a gap, right? We need to
fill that gap, because patients are sick and dying in that—during
that period.
Mr. GUTHRIE. OK, I guess I will yield back, and just say I know
that some of the 10,000 diseases are still—are at the NIH level,
and not moving forward. So I mean, not saying they won’t get to
the private sector to move forward, as well, and the gap. But thank
you so much for your answers, I really appreciate it.
Sorry I went over. I apologize. I yield back.
Ms. ESHOO. You don’t have to apologize. Everything that is being
discussed is important, and there are so many facets to this.
So the gentleman yields back.
The Chair is pleased to recognize the chairman of the full com-
mittee, Mr. Pallone, for your 5 minutes of questions.
Mr. PALLONE. Thank you, Madam Chair——
Ms. ESHOO. And thank you for allowing us to have this hearing.
Mr. PALLONE. It is a consensus.
75
Dr. Ling, over the course of your career you have participated in
and led biomedical discoveries that have transformed medicine, no-
tably your work at DARPA. And you—the stated goal of ARPA-H
is to expedite cross-cutting technologies and discoveries like the Ge-
nome Project not every 20 or 30 years, but every five to ten.
We have seen the success of programs like DARPA and ARPA-
E. But yet engineering systems are not direct comparisons to
human biology and complex diseases, so I just want to better un-
derstand why ARPA-H is the appropriate model for achieving bio-
medical breakthroughs, and why now is the right time.
And my first question really expands on what you have already
said today, which is, you know, the—what is the gap between the
fundamental research and commercial sectors that ARPA-H seeks
to fill?
Dr. LING. No, thank you, Congressman.
The—what ARPA-H offers is the opportunity to leverage against
the successes of the NSF and the NIH and other agencies that do
fundamental research. Biology is not engineering, that is true. But
the way to do research can follow a model that does work in engi-
neering.
So a timeline-driven, a milestone-gated, with clear deliverables
at the end of a performance period are, in fact, good ways to fun-
damentally support advancement of R&D so that it can get into
and across the valley of death. That is the most important thing.
It is not a question of if it is biology or is it chemistry or physics
or engineering. It is really of how to manage the research, and how
to manage the research dollars.
As Dr.—I gave you an outline of—DARPA takes a very dis-
ciplined approach to it, much like ARPA-H must do, as well. To dif-
ferentiate itself, it must take a very disciplined approach on how
the dollars are spent. Milestone-driven, timeline-limited, with clear
deliverables: those are the essential elements of success in an
ARPA-like construct. And therein lies—so it is not really a question
of is this amenable, it is the—it is how the dollars are managed.
That is very, very important.
The accountability of that money has to lie with the program
manager. The accountability of the program manager lies with the
director. And a director’s accountability lies, of course, with the
Congress, as well as the Secretary of the HHS. There has to be ac-
countability to the money.
Mr. PALLONE. OK. Now, let me just combine my second and third
question, which are how will ARPA-H be more effective at advanc-
ing cures and treatments for the medical field’s biggest challenges,
and why is ARPA-H additive, rather than duplicative of existing
programs addressing translational research, such as NCATS, if I
could combine those two?
Dr. LING. Again, it comes to exactly what I said, it is the process.
So one of the things that, when we set out to do the robot arm
that you had heard about, the prosthetic arm, it was that it had
to be delivered within four years. There were clear gates to go
through it, and so on and so forth. We then achieved it. But at the
beginning of that program we had NSF science officers and FDA
officers helping review the program, and being part of the program
so that, by the time, at the fouryear mark, it was ready to transi-
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tion over to going through the FDA regulatory process, we got the
arm regulated, fully approved, within eight years of start of pro-
gram. Eight years, not thirty-three. Eight years. And that arm is
now with the Veterans Administration.
So again, we involved the Veterans Administration early on, too,
so that they had clinicians and they had patient advocates already
going through the steps. That is one example. There is many oth-
ers, but I don’t want to take up your time, Congressman. But I
think that that is the point, is that you have to start at the begin-
ning, where you are going to go through all these gates. Plan for
them, structure it into the process. And it is the how you spend the
money and how you design the program and—to make it a contrac-
tual obligation to the performers.
Mr. PALLONE. I appreciate that. I also appreciate your enthu-
siasm. I can’t help thinking about the Super Bowl. Like—you seem
like you are, you know, organizing a football team here. It is really
great, the energy. Thank you.
I yield back, Madam Chair.
Ms. ESHOO. The gentleman yields back. It is a pleasure to recog-
nize the ranking member of our full committee, Representative
Cathy McMorris Rodgers.
You have 5 minutes for your questions.
Mrs. RODGERS. Thank you, Madam Chair.
Leadership matters, accountability matters, character matters,
people who do the right thing when nobody’s looking, that matters.
Unfortunately, I am fundamentally concerned that NIH is not re-
sponding to Congress right now. So I will set that aside, because
we are focused on ARPA-H right now.
So, Dr. Lander, Dr. Giroir, I would like to start just better under-
standing how you view what currently is happening at NIH, some
of the programs that exist that are intended to be innovative and
cutting edge. Congress is funding the Common Fund; the National
Center for Advancing Translational Science; High-Risk, High-Re-
ward Program; the Accelerating Medicines Partnership. And that is
just at NIH. I would just like to hear you share with me, to share
your insights with me as far as are these programs not working,
if they are working. Do we still need ARPA-H? And if they are not,
should they be eliminated, and these dollars spent part of pro-
moting ARPA-H?
That was Dr.—oh, Dr. Miller. Dr. Miller, sorry. Dr. Miller and
Dr. Giroir, if you would answer those questions, please.
Dr. MILLER. Thank you. I guess my question even before that is,
you know, with Dr. Ling’s comment about program managers and
these gated timelines, we have these other programs at the NIH.
It sounds like they are not doing that. Why don’t we make those
NIH programs do that, and transition funding from indirect costs
to make a mini-ARPA-H at one of those other programs that are
already there?
It sounds like the NIH is also not being responsive. If we think
that we have gaps—and there are gaps, and it does take a long
time to develop a drug or a device—we should use the existing pro-
grams that we have, rather than make a whole new program be-
cause we say that the other programs aren’t working well. It seems
sort of like common sense.
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Mrs. RODGERS. OK, thank you.
Dr. Giroir?
Dr. GIROIR. Thank you for that question, and it is a very impor-
tant one.
ARPA-H is going to be—if so authorized—is going to be a major
part of the ecosystem. But it is not the entire ecosystem. As Dr.
Ling and others have said, ARPA-H will rely on the basic discovery
and ideas that occur at the NIH, at the National Science Founda-
tion, funded by the Gates Foundation, the Leap Fund at Wellcome
Trust.
It will also need the kinds of advanced translation that comes out
of ARPA-H. ARPA-H will never cure pancreatic cancer; it will cre-
ate the technical abilities, the novel approaches, de-risk that, so it
could be translated and transition to the private sector, and poten-
tially other aspects of NIH, like——
Mrs. RODGERS. OK, if I could just clarify, why do some of these
existing programs, that I believe are intended for that same pur-
pose, are not working as intended?
[No response.]
Ms. ESHOO. Dr. Giroir, we lost your audio.
Dr. GIROIR. Sorry. I am going to say that I don’t feel I have the
ability to comment, you know, extensively on any of these programs
currently.
Mrs. RODGERS. OK, OK.
Dr. GIROIR. I certainly have been involved with them, but I can’t
comment negatively on this program.
Mrs. RODGERS. OK, thank you.
Ms. ESHOO. Yes.
Mrs. RODGERS. And I hear that this agency will be accountable
to Congress, yet it is a Presidential appointment without any con-
gressional oversight.
Dr. GIROIR. So——
Mrs. RODGERS. Another question, Dr. Miller. Are there things
that we can reform at our existing science agencies, such as NIH
and BARDA, to change the culture and the risk tolerance without
eroding their core missions?
Dr. MILLER. Absolutely. That is an excellent question. I think
having tenure-limited program managers and leadership in various
parts of the NIH, in conjunction with making that decision with
the NIH director and/or oversight, would be an important transi-
tion to happen over time.
Indirect costs, which represent billions of dollars at the NIH, lack
of transparency and accountability, and we should demand from
funded institutions where that comes—where those dollars are
spent.
Mrs. RODGERS. OK, thank you. Well, and I will attempt one more
for Dr. Giroir.
I appreciate your perspective here. You know, we are talking
about ARPA-H, and that the rules should not be to implement and
bring to the market preventions or cures, it should be to develop
and prove the viability of these concepts. Can you explain why
ARPA-H should be a proof of concept agency?
Dr. GIROIR. Because it really fills—it does fill that gap, it bridges
that gap. This is not meant to supplant the private sector or any-
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thing down the line, but that gap is real. It needs to de-risk to cre-
ate new approaches. We don’t even have a paradigm for
neurodegeneration now. These are the things that it needs to do.
And let me just make a comment. My last year at DARPA I did
113 meetings and briefings. About half of those were to Congress
or professional staff, and about half of those were within stake-
holder groups. You know, ARPA-H has to have that degree of re-
sponsiveness and interaction for it to be successful, and I share
your concern about that, and that should be an expectation.
Mrs. RODGERS. OK, thank you. I have more questions, but I have
run out of time.
I will yield back. Thank you.
Ms. ESHOO. The gentlewoman yields back.
The Chair is pleased to recognize the gentlewoman from Cali-
fornia, Ms. Matsui, for her 5 minutes of questions.
Ms. MATSUI. Thank you very much, Madam Chair, and thank
you to everyone who is joining this hearing today to think critically
about the future of the biomedical technology pipeline.
Now, there is an ongoing and healthy debate on the appropriate
placement of ARPA-H. Generally, there is a consensus that ARPA-
H will need an independent structure and novel culture to deliver
innovative ideas in health and medicine.
Dr. Giroir, you mentioned ARPA-H needs to be independent, dis-
tinct from the traditional NIH institutes and centers. Can you
elaborate on how your experience at DARPA helped shape that per-
spective?
Dr. GIROIR. Thank you very much for that question, ma’am. As
Dr. Ling said, it is the whole process of how you do business. It
is the whole process and thought process of accountability, but also
sort of being a counter-cultural organization that doesn’t go with
the flow, that doesn’t rely on your peers for incremental change.
It relies on teaming, where we bring academia, together with in-
dustry, with non-profits, and teams of teams. Nobody really com-
petes against each other. They are competing all for the same goal.
Look, I am a big fan of the NIH. The NIH has done remarkable
things for this country. But it could never create the culture that
is needed at DARPA. We have a model, as Dr. Ling has said. I do
believe it needs to be independent, but it does need to be housed
within HHS, the same way as DARPA is within DoD, ARPA-E is
within Energy, and ARPA-H should be within HHS.
Ms. MATSUI. Well, can I say this, though? HHS is still a massive
agency, with its own levels of bureaucracy, as we know. And accel-
eration is a key to ARPA-H’s mission to transform and improve
healthcare. Now, placed at HHS, how can we ensure that ARPA-
H has that capability to move quickly and efficiently?
Dr. GIROIR. It really depends on your authorizations. And as I
put in my written testimony, I think the legislation by Chair-
woman Eshoo has deep insight into the authorities that are needed
to make sure that this happens. Picking the first director is going
to be critical, allowing rapid contracting, having term limits, being
able to hire the best and brightest program managers, and having
that independent culture: I really think that legislation provides
the basis for what is needed.
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This—reporting to the Secretary does not mean the Secretary
runs the agency, but it is vital that the director report at the high-
est level, and I do believe the Secretary is the appropriate level for
that.
Ms. MATSUI. OK. Well, thank you very much, Dr. Giroir.
Talking about necessary authorities, DARPA is a unique agency
that benefits from numerous special authorities. For example,
DARPA can capitalize on flexible hiring and procurement authori-
ties, including grants, contracts, and cooperative agreements, and
other transaction authorities. Ms. Krofah, can you explain why spe-
cial contracting authorities are needed for ARPA-H, and provide an
example of when they are best applicable?
[No response.]
Ms. MATSUI.
Ms. KROFAH?
Ms. MATSUI. Yes?
Ms. KROFAH. Thank you, thank you so much for that question.
And you know, I want to make a comment, in terms of some of the
prior conversation, to say that the momentum for which we are
seeing the acceleration and the need for driving innovation is com-
ing out of COVID–19. And we have seen NIH be successful, par-
ticularly with a RADx program, in being able to stand up a collabo-
ration with public and private quite quickly.
In terms of your question with other transactional authorities,
that is an example where you can leverage such authorities to do
hiring, to do contracting in a way where you are not going through
significant levels of delay or barriers. That type of authority also
needs to be embedded within ARPA-H, including leveraging other
hiring practices such as FDA, in bringing top-notch scientists out-
side of government to play a role in regulatory approval processes.
Ms. MATSUI. OK. Well, that is very good, Ms. Krofah.
I want to quickly touch on a related issue of the critical role that
indirect cost recovery associated with NIH grants plays in sup-
porting research at the University of California. From building
labs, to quickly processing data, to keeping patients safe, there are
indirect but essential costs of conducting research and the Federal
Government’s support is vital to advancing the research mission.
Dr. Yamamoto, I know you have a long history of helping Con-
gress understand the importance of indirect cost issues. Can you
please provide some insight into why research institutions like
UCSF receive indirect cost recovery?
Quickly.
Dr. YAMAMOTO. Indirect costs are part of supporting research.
Direct costs pay for salaries of the investigators, the equipment
that is needed, and so forth. Indirect costs help the institutions to
be able to support the infrastructure required for that research to
be—take place, whether it is buildings, providing distilled water
and electricity, or having administrators who will oversee the
grants and make sure that they are managed properly and come
into compliance.
So those—so indirect costs have kind of a—have a funny ring to
them, because it seems like it is not related to what the investiga-
tors are doing. But in fact, those are—those things are crucial. And
so they are an important part of allowing the research enterprise
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to go forward. Institutions do not recover their full costs of sup-
porting research. Indirect costs provide a portion of that. That is
essential.
Ms. MATSUI. OK, thank you very much, Dr. Yamamoto.
I yield back.
Ms. ESHOO. The gentlewoman yields back. The Chair is pleased
to recognize the gentleman from Michigan, Mr. Upton, former
chairman of the full committee, and one of the authors of Cures
2.0.
Five minutes for your questions.
Mr. UPTON. Well, thank you, Madam Chair.
Nine thousand diseases, five hundred remedies. Every disease
group that is out there is looking for that breakthrough therapy.
They have got to have the hope to help.
We have got some real champions, bipartisan, on both sides of
the Capitol, looking for ARPA-H, knowing that DARPA was so suc-
cessful, and mainly because they cut through the chaff. They got
the job done.
I am not from the Sputnik generation either, but I do remember
Lee Iacocca. ‘‘Lead, follow, or get out of the way.’’ That is exactly
the charge that this organization needs.
So the appropriators have already funded it to billions of dollars
if we end up getting out of a continuing resolution. Dr. Giroir, how
do we ensure that ARPA-H runs like DARPA? How do we cut
through the chaff to make sure that that happens?
Dr. GIROIR. Well, thank you very much, and you are exactly cor-
rect.
And I will make another comment. It is not—the money is nec-
essary, but it is not sufficient. You could allocate ten times that
amount of money and not achieve the goal, if you don’t create the
right authorities, the right culture, the right director, the right pro-
gram managers. I was hired as an IPA from academia, where I was
a full professor all these are critically important. But sir, it is all
about the culture, and it is all about execution and creating that
environment. And that is going to require this committee to protect
that environment and the program managers, so they can do ex-
actly what you said which is exactly where it needs to be.
Mr. UPTON. So that is why, to me, that is why we need more
than just an appropriation. We need to have the proper oversight
and controls to make sure that it runs the way that Lee Iacocca
would. Coming from Michigan, he was a good Michigander.
Dr. GIROIR. Yes, sir.
Mr. UPTON. Ms.—he was a great Michigander.
Ms. Krofah, I am so glad the Milken Institute is here. And for
those that are watching, or those members that weren’t here when
we did 21st Century Cures, the Milken Institute just did a wonder-
ful job for us to tap them for ideas to include in 21st Century
Cures.
So one of the things that we are looking on in the next version
2.0, is the pandemic response and drug development. I am worried
a bit by the current State of the drug development incentives to
prevent future pandemics. As you know, Diana DeGette and I are
huge proponents of encouraging new antibiotic developments, and
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we included our colleague Mr. Doyle’s PASTEUR Act in legislation
this summer, calling 2.0.
The use of real-world evidence, RWE, has been a big priority.
And as—you, as—I want to ask if you are concerned, as I am,
about the lack of new antibiotics in the pipeline. And if so, how
might RWE or other components of the agreement before us help
search for new cures as we look to the—stop a future pandemic?
Ms. KROFAH. Thank you so much, Mr. Upton, for your question.
And absolutely, a delight to be here and to partner with you on
21st Century Cures 1.0, and now very supportive of 21st Century
Cures 2.0.
I am glad that you brought up the antibiotic issue because, as
many of us say, it is silentthat is happening right now. So many
are losing their lives to drug-resistant infections.
We do believe that inclusion of the PASTEUR Act is going to
play a critical role because, as you know, the market is broken, in
terms of our—process for new antibiotics. On the one hand, we
want to use fewer antibiotics. On the other hand, because of drug
resistance, we need more antibiotics that are able to cut through
those particular bacteria.
The challenge, of course, is that we don’t have a marketplace
that will reimburse innovation at the levels that are needed. PAS-
TEUR will go a long way in ensuring that we are de-linking vol-
ume and value, eventually providing incentives along the way that
we are supporting innovation tied to the value of those antibiotics,
not just prescribing more.
At the same time, we need more than just PASTEUR. We need
to ensure that the private sector can be brought in, as well. The
Milken Institute has been doing some work around creating inno-
vative financing mechanisms to ensure that we are tying the pri-
vate sector investment dollars, as well as other incentives such as
PASTEUR to make sure it is a level playing field.
You also talked about real world data evidence. We need to col-
lect that wherever they may be found. We need to ensure that they
have regulatory grade where they can be used, from decision-
making perspectives. So we welcome what we saw during the
COVID–19 pandemic. We do think there is much more we need to
do to learn from that experience and ensure that we are capital-
izing on that antibiotic R&D pipeline.
Mr. UPTON. So do you think we can get to where we ought to be
in the next five to ten years?
Ms. KROFAH. Absolutely, if we make sure that we put these in-
centives in place. We need to make sure that we get PASTEUR
over the finish line, absolutely. We need to make sure that we are
continuing to invest in these companies upstream. They are doing
their best right now, but they are not going to be successful—of
course, we saw bankruptcy over time in the antibiotic space. We
don’t want to continue that trend, so absolutely we . We need to
make sure AMR is on the same level as other pandemic prepared-
ness initiatives.
Mr. UPTON. Thank you.
I yield back.
Ms. ESHOO. The gentleman yields back. I just would like to say
that it is the intention of the Chair that we move to, you know, leg-
82
islative and markup on Cures 2.0 and ARPA-H. They are highly
complementary of one another.
And you know, when you use the figures 9,000 diseases, 500
solved, we have our work cut out for us, and the people across the
country, families, are—they deserve to have hope. Every life is a
valuable one. And we always, you know, offer our—not only our
sympathy, but our caring to one another when we know something
has stricken the family of one of our colleagues. So this is as real
as our getting up in the morning and doing our work.
So I think that Congress has the capacity to do this, the wisdom
to do it, and that we work hard together to refine whatever needs
to be refined, but we should not be in the way of what I just de-
scribed. So thank you for allowing me the time to say that.
The Chair is really pleased to recognize the gentlewoman from
Florida, Ms. Castor, for your 5 minutes of questions.
Ms. CASTOR. Well, thank you, Chair Eshoo, and good morning,
everyone. And thank you to our witnesses for their insights today.
And I really want to thank Chair Eshoo, and Representative
DeGette, and Representative Upton for their leadership. And I look
forward to working on this very exciting concept for advanced re-
search projects, the whole initiative that will help us build on the
existing research, so that we are not leaving innovative discoveries,
treatments, and cures on the table.
But we have to set up this endeavor for success, and I think that
includes transparency and accountability. Because, unfortunately,
what we have seen over the past two years, data evidence and
interventions to address the pandemic have often been met with
distrust, outright distrust, and misrepresentation. It has led to
poor uptake in the effective and lifesaving interventions such as
the COVID–19 vaccine. So for ARPA to succeed, we are going to
need to build trust among the public and all of the stakeholders out
there.
Stakeholders have specifically requested that ARPA-H be trans-
parent about the selection criteria and the decisionmaking process
for its broader investment goals, as well as the selection of indi-
vidual research projects. So Dr. Ling, what is your perspective on
how ARPA-H can be both transparent and accountable?
Dr. LING. Thank you, Congresswoman. It absolutely should be
transparent and accountable. It needs to be transparent and ac-
countable at every level, and it begins with the performers. It be-
gins with the scientists and the engineers.
Again, the structure of it is very DARPA-like. And in this case
there—it is not a DoD type of project, but it is a health project. So
transparency is—absolutely, should be built within the system
fully.
So let me be more specific. The performers should be accountable
to the program manager. The program managers have the author-
ity to move money around, as well as move the investigator pool
around, as well. That program manager is accountable to the office
director, and the office director up to the director, and then for—
ultimately, the director to the Congress, as well as to the Secretary.
In this whole construct is that—we said before it is going to be
a timeline-driven, milestone-regulated process. Those milestones
should be very, very transparent. Those should be published, actu-
83
ally, so that you know exactly what the taxpayers dollars are spent
on and, in fact, how well they are doing against those milestones,
those phase gates, as it were. And money is tied to it. I will be
blunt, money is tied to it. So if they don’t perform, they don’t get
the money. It is real simple.
The other element of this is the engagement. As I said to you be-
fore—and I really reiterate this point—engagement has to be just
not the investigators. The science is not the end of this. You still
have to get through regulatory, you have to get through industry,
and you have got to get to the consumer groups, which in this case
are the patient advocacy groups, as well as the clinicians that sup-
port them.
So at all stages, they have to be engaged from the very begin-
ning, and they have to be engaged throughout the entire process.
So that is, in fact, how you build in accountability and how you
build in trust, quite frankly. You don’t want to bring something in
the eleventh hour and say, ‘‘Here it is.’’ You want them to be part
of the process from the get-go. And in fact——
Ms. CASTOR. Great.
Dr. LING [continuing]. That is how—again, we have a model. It
is called DARPA. That is the model, if you look at the BTO, how
they do a lot of their medically related portfolio——
Ms. CASTOR. Right.
Dr. LING [continuing]. Because it is a small part of their port-
folio. It is still a Defense Department agency. That is why an
ARPA-H, which is only devoted to health, is really something that
we need here and now.
Ms. CASTOR. OK, thank you.
And Dr. Yamamoto, I was very enthused to see in President
Biden’s concept paper for ARPA-H he explicitly mentioned the idea
of using mRNA vaccines to prevent cancers as an example of poten-
tial transformative projects for the new agency. We know the—
about the success of mRNA for COVID. Most traditional vaccines
take years to develop. Here was a medical miracle, really.
And back home I represent the Moffitt Cancer Center in Tampa,
and they are—the researchers there tell me that the mRNA cancer
vaccines could potentially be the most—some of the most cost-effec-
tive methods for preventing recurrences in the high cost of cancer
care. Do you agree, and what do you see as the future here for
immunotherapy treatments and prevention?
Dr. YAMAMOTO. Thank you for that question, Congresswoman.
The mRNA vaccines do represent a really revolutionary break-
through that depended upon years of basic research that came be-
fore that to understand the elements that drive stability of mes-
senger RNA, you know, the—inside the cells to be able to create
the little packaging molecules that protect the RNA on the way in.
And so it was a great victory in that sense.
And your colleagues in Tampa are correct that the potential
promise of cancer—mRNA-driven cancer vaccines is enormous, and
DARPA had a role in these RNA technologies. So I think that that
is correct, and is worthy of all of the excitement that has been gen-
erated.
If we step back—and I think it is worth making an explicit point
about how the kinds of—the collaborations that come together that
84
drive the kinds of advances we are talking about. There are about
two dozen Federal agencies that are doing science and technology.
There is nothing—except for the ARPA models, there is nothing in
the mission statements or goals of those agencies that drives them
to work together and collaborate.
It is not—there is nothing explicit in the ARPA agencies that say
that they should be out talking to all of the agencies around to find
out what the capabilities are that could come together to—that
would allow them to really make advances. But instead, it is the
mission of the program managers and the director that say, ‘‘How
are we going to get to this really hard point?’’ And they realize that
the only way they can do that is to bring together the talents of
other agencies, to bring together—bring in the private sector, and
research foundations, and so forth, academia and so forth, to be
able to accomplish these goals.
So it is not in the mission statement of the ARPAs to drive col-
laborations. But the directors know that that is the only way they
are going to get there. It is the only way that it is going to work.
And so the—so this is a aggregating, convening force that actually
does something that no other science agencies in the Federal Gov-
ernment do, to bring together these talents and skills to accomplish
hard things.
Ms. CASTOR. Very helpful, thank you.
Ms. ESHOO. The gentlewoman’s time has expired.
Colleagues, I think you have already noticed that I have been
very generous with time on both sides of the aisle. But there are
excellent questions that are being asked. They deserve to be asked.
But we have brilliance here, in terms of our witnesses, and their
answers to these questions are just so important. So thank you for
indulging my generosity, which I will continue to put out there.
How is that? OK.
The Chair is pleased to recognize the gentleman from Virginia,
Mr. Griffith, for your 5 minutes of questions.
Mr. GRIFFITH. Thank you very much, Madam Chair. Innovation
and medical research has been a priority of mine since I arrived
in Congress. I believe there is a role for both the Federal Govern-
ment and the private sector in this space, and that Congress has
a responsibility to fund these activities.
My line of questions that was originally written out said, ‘‘OK,
tell me why we need a new agency. Let me get my head around
this.’’ You all have answered those questions to a certain extent al-
ready, so I am not going to be redundant on this occasion.
But Dr. Ling, you caught my imagination in the spirit of trying
to create something new and different. And you said, you know, if
we are trying to build—and you used the baseball analogy—if we
are trying to get to the World Series, we might like that person a
whole lot, but if they are not getting the job done, if they are not
performing, we should fire them. But firing people is very difficult
at the Federal level, as you know.
So I am interpreting—and I want you to confirm, yes or no, if
you agree—I am interpreting that you think, when we pass this
legislation, we need to have specific language on, if they are not
meeting the requirements of their particular contract, that that
85
contract will be terminated and they will be fired. Is that correct?
Did I understand you?
Mike, so that everybody in the world can hear you.
Dr. LING. Correct, Congressman. In DARPA there is a phrase. It
says, ‘‘At any time, for any reason, without prior notice, this con-
tract may be terminated at the decision and—of the U.S. Govern-
ment.’’
Mr. GRIFFITH. And I appreciate that. And Ranking Member
Cathy McMorris Rodgers pointed out, OK, well, what are we talk-
ing about with accountability? We need to have some accountability
language in there, as well, not just for the executive branch, but
also for the congressional branch. Would you agree with that, as
well, Dr. Ling?
Dr. LING. There needs to be accountability throughout the sys-
tem. Exactly, Congressman.
Mr. GRIFFITH. And that would be both executive branch and, two,
the Congress of the United States.
Dr. LING. Absolutely. And all the way down to the performer
level. They need to feel accountable. This is not their money, it is
the taxpayer money.
Mr. GRIFFITH. And we are trying to make sure we get big things
done. And if we are going to create a whole new agency inside of
our NIH, or inside of whatever we are going—wherever we are
going to put it, we need to make sure that they are performing and
getting that research done for the American people. I appreciate
that very much.
Dr. Giroir, I know you said some good things about the NIH, and
I agree. I am involved in some stuff with the NIH. I think they do
a great job in their lane, and so forth. But one of the concerns we
have had of late is it appears that, through third-party contractors,
there is a lot of money being funneled to China. Do you think that
we can stop that with ARPA-H?
And should we put some specific language in there that this re-
search needs to be done on American soil?
Dr. GIROIR. Thank you for that. You know, I do believe that the
NIH investigators do need to—well, the NIH program managers in
NIH offices do need to be more accountable.
In general, if you are an NIH grantee, you get your grant, you
put a report together at the end of a year, you send it in, and that
is it. And you really don’t become accountable until every five
years, when that is renewed. Contrast that to DARPA, where, lit-
erally, every week the program manager is reviewing the per-
former. Every month the office director is reviewing all the pro-
grams, and every year the director reviews every single [inaudible].
I can also say that, when I was at DARPA—to your staff and the
appropriations staff, or from the Defense side, I briefed every single
program in my office, what they were doing, and what they were
accountable for. That is the degree of transparency and account-
ability.
So yes, I agree. I do believe the NIH needs to raise the account-
ability, particularly for overseas investigators, without being bur-
densome on U.S. investigators. But ARPA-H would be accountable
at every level and transparent. That is the nature of the beast.
86
Mr. GRIFFITH. Well, one of the frustrations that I have had is,
in trying to figure out what was going on at the Wuhan lab with
American money, was getting answers, and they won’t give us the
answers. And the—well, I never expect to get answers from the
Chinese. And so that creates a problem.
If we are going to spend this money, Dr. Miller, you said, you
know, the Chinese are ahead of us on research, they have got all
these extra people doing research, and that the United States
needs to catch up, and we need to be doing this research, too. What
is the point of putting this money into ARPA-H or anything else,
if we are then just going to subcontract with the Chinese?
Don’t you think we ought to be doing that here, in the United
States?
Dr. MILLER. I agree wholeheartedly.
Mr. GRIFFITH. That is what I like, a short, quick answer.
[Laughter.]
Mr. GRIFFITH. Do you—and so we would be a whole lot better off
if we were doing that.
And then, if there is some kind of a question of accountability,
you would agree, Dr. Miller and both—and Dr. Ling—it is a whole
lot easier to get it if we are dealing with people who are in this
country and who are answering to us directly, as opposed to a gov-
ernment that does not have any reason to show us anything, or
give us any information?
Dr. MILLER. Yes.
Mr. GRIFFITH. Dr. Ling?
Dr. LING. We should only be working with trusted allies and, of
course, the United States citizenry. That is exactly what it should
be.
Mr. GRIFFITH. Well, and I would say I think it needs to be mostly
American. I suppose, if we had a trusted ally that we could actually
trust, but when we can’t get answers on what happened at Wuhan
with American money, I have serious problems about expanding
any program that doesn’t have language to protect us.
I yield back.
Ms. ESHOO. The gentleman yields back. The Chair is pleased to
recognize the gentleman from Maryland, Mr. Sarbanes, for your 5
minutes of questions.
Mr. SARBANES. Thanks very much, Madam Chair. I appreciate
the hearing, and I thank the witnesses for their testimony.
There were, I think, 15 listening sessions that were conducted
last year, broadly, to invite input around the proposal of ARPA-H.
I had a chance to listen to a kind of wrap-up session of that that
took place in October. And some of the themes there in describing
ARPA-H included positive disruption, establishing benchmarks so
that this high-risk, high-reward ambition could be deployed
through the opportunity to fail fast. Equity and diversity was a big
part of the description of what is being sought through ARPA-H.
Use-driven, not curiosity-driven, was a phrase, as well. Measuring
success, not through publication, but health improvement for the
public, not duplicating existing initiatives, et cetera. And I assume
that these are features and characteristics that we saw in DARPA,
as well, when it was being developed and obviously now, that it has
been deployed.
87
I wanted to ask Dr. Ling—you and Dr. Yamamoto—to comment
on—to put the Human Genome Project and Operation Warp Speed
in some context relative to this discussion of ARPA-H, are those ef-
forts ones that, if an ARPA-H had been in place, would have re-
sided there, or would it have been sort of the tip of the spear for
those kinds of efforts?
Or are they different in the sense that they were assembling on,
at least in the case of Operation Warp Speed, assembling in a kind
of emergency fashion existing resources across many different
agencies, and therefore they should be distinguished from the kinds
of projects that we will see in the ARPA-H space?
But it would be useful to me, as a kind of reference point, for you
to make some observations about what ARPA-H would look like,
how it would operate, et cetera, against that idea of the Operation
Warp Speed or Human Genome, or both.
So why don’t we start with Dr. Yamamoto and then Dr. Ling? I
would be interested in hearing from you.
Dr. YAMAMOTO. Thank you for that question. Important—an im-
portant one.
My view on this is that the two examples that you gave, the
Human Genome Project and Operation Warp Speed, if the ARPA-
H office had been established, might well have been projects that
were carried out under that aegis.
If you remember the way that the Human Genome Project ran,
first of all, it took a long time. And second, it turned into this sort
of kind of entertaining competition between a private company and
the government effort. If ARPA had been in place, that collabora-
tion would have been formed early on, and the progress on the
Human Genome Project would have gone much faster. Instead, you
had these two camps that weren’t communicating with each other,
they were collaborating—they were not collaborating, and the work
went more slowly.
Operation Warp Speed worked pretty well, and it was in—as you
said, in response to an urgent need, right. But Francis Collins him-
self has said that it was a difficult lift because the culture and
practices were not in place at the NIH to really make it work well.
So those examples are actually good examples of things that I
think an ARPA structure could be able to accomplish, accomplish
well, accomplish better.
Mr. SARBANES. Dr. Ling?
Dr. LING. Yes, I fully agree with Dr. Yamamoto, that the—if
there was an ARPA, the opportunity there would have been lever-
aged much, much earlier.
Let me talk specifically about Operation Warp Speed, which was
really—if you take a look at the mRNA vaccines, DARPA had actu-
ally invested in this program. It was called the ADEPT program
run by my colleague, Dr. Dan—Colonel Dr. Dan Wattendorf,
bought back in 2010. And the two performers that he chose was
Moderna and GSK. So they were—already, industry was being
brought into this ten years before anybody knew that there was a
COVID threat around, and they were doing it to develop vaccines
against Ebola, actually, at that time.
And so the beauty of this was that they brought in a public-pri-
vate partnership already at the very beginning, in 2010, 2010—
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that—look what it yielded here, now, in 2020, when COVID finally
reared its ugly head. And that is why we are talking about—you
want to say good gravy, we are really lucky to have an ARPA in-
vestment at that time. I think that speaks loudly for it.
And again, let me point out that it was a true private-public
partnership. They—it involved laboratories, of course. It involved
the FDA, of course. But Moderna and GSK, Moderna and GSK,
they were performers in this program.
Mr. SARBANES. Let me ask you, Dr. Ling, since you are speaking
now, and I have only got 25 seconds here. But on this discussion
around where ARPA-H should be situated, whether it should reside
formally inside of NIH or more broadly inside HHS, should it be
in the orbit of NIH, either physically or organizationally, be some-
how tethered there?
Or do you see it as, in a sense, free-floating within HHS? Can
you comment on that a little bit?
Dr. LING. I believe it should be a separate, distinct, and inde-
pendent agency, just like the CDC is, the FDA is, the NSF, and the
NIH. So they are all complementary. They all work together, but
they are separate and distinct, and they each have their own chain
of command. That is very, very critical.
Mr. SARBANES. Thank you. I yield back.
Ms. ESHOO. The gentleman yields back. The Chair is pleased to
recognize the gentleman from Florida, Mr. Bilirakis, for his 5 min-
utes of questions.
Mr. BILIRAKIS. Thank you, Madam Chair, and I thank all of you
for testifying today. Thank you for your patience.
Dr. Miller, you talked in your testimony about the critical need
for our Nation to have a strong response to China in the area of
biomedical research, and I agree. Rather than focusing on ARPA-
H, you say we need to transform the NIH’s Extramural Grant Pro-
gram, which consists of an almost $32 billion-per-year budget.
One of your reform proposals is to improve accountability and
transparency of taxpayer dollars by specifying where indirect dol-
lars go for extramural projects. I couldn’t agree more, which is why
I introduced the Protecting Integrity of our Biomedical Research
Act, which would require disclosure of participation in foreign tal-
ent programs as a condition of receiving Federal extramural bio-
medical research grant dollars.
This was in direct response to cases where—you probably know
this—where NIH-funded research had concealed support they had
received from the Chinese Government. In fact, the GAO has
issued reports stating the need for NIH to address undue foreign
influence and increased transparency through disclosure. Despite
its direct relevance to research integrity and combating Chinese in-
fluence, I am disappointed that the majority decided to ignore my
comments in this proposal, and not place it in the COMPETES Act.
Dr. Miller, can you elaborate how adding more transparency re-
quirements to NIH extramural grants will ensure the appropriate
use of funds?
And can you give additional ideas of how we can protect against
inefficient project selection, please?
Dr. MILLER. Thank you. First and foremost, the average indirect
cost rate is around 52 percent. Originally, when the NIH started,
89
it was around eight percent. The Gates Foundation pays ten per-
cent, and the European Union pays 20 percent. It is unclear to me
why our average would be twice that of the EU, or five times that
of the Gates Foundation. If a billionaire is getting a better deal
than the U.S. Government, that is not exactly a great look for us.
Ways to fix that would include putting a cap, and having tiers,
and having a tier tied to your three year prior rolling average,
meaning that universities that get more money are in a lower indi-
rect rate group—say, 10, 17.5, 25 percent as an example.
I think the important thing to emphasize is that the current dis-
tribution is highly inequitable, where HBCUs and a lot of public
schools in the plain states and also the southeast and the south-
west don’t receive a lot of NIH grants. It is not because there aren’t
great researchers there, there are. It is just, if you are getting a
lot of NIH grants, and you have a lot of administrative funds, you
build a massive grant-making apparatus. And then that apparatus
helps your researchers compete and comply with the 154-page, I
believe it was, guide for NIH extramural grants, right? That takes
a lot of infrastructure to respond to that.
So I think that sort of breaking that cycle is a way that we could
promote innovation and growth, and also free up funds for direct
research funding.
Mr. BILIRAKIS. Thank you. My next question is for Dr. Miller and
Dr. Giroir.
I have spent much of my time here in Congress advocating for
policies that encourage medical innovation by removing red tape,
and incentivizing companies to develop cures as fast, as safe—and
safely as possible, particularly for rare diseases. Because of the na-
ture of the 7,000 known rare diseases affecting small patient popu-
lations, it often falls through the cracks, unfortunately.
Can you provide examples of how ARPA-H could fill in these
gaps and prioritize rare disease research, especially without a spec-
ified strategic plan before its creation?
And again, for Dr. Miller and Dr. Giroir.
Dr. GIROIR. Well, thank you——
Dr. MILLER. I will be brief, thank you.
[Pause.]
Dr. MILLER. I was going to say, I will be brief.
We should have a list of what those priorities are before we fund
it.
Mr. BILIRAKIS. Thank you.
Dr. Giroir?
Dr. GIROIR. I think, for rare diseases, ARPA-H could be incred-
ibly important.
I am a pediatrician. For example, genetic cures can be remark-
able in the future. But if they cost 2 or $3 million per cure because
we have limiting technologies, they are not going to be equitably
accessible across the country. So DARPA investing in core types of
technologies that can dramatically increase access in an equitable
way across a number of rare genetic diseases is just one example
that really is biology, but it has an engineering flair, and it is com-
pletely there.
We can drive down the cost of cell-based therapies from a half-
a-million and a million dollars down tenfold, so we increase the ac-
90
cess. So these kind of platforms, as opposed to just an individual
disease, is where ARPA-H can make an enormous contribution.
Mr. BILIRAKIS. Thank you very much.
I yield back, Madam Chair. Thank you.
Ms. ESHOO. The gentleman yields back. Let me just say to the
gentleman the points that you have made about your legislation,
which I was not aware of, we will work with you on that, because
I think that there are some really important elements relative to
transparency and the other things that you raised. So I would be
happy to do that.
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 Chair. I really ap-
preciate this hearing. Very timely, very critical. COVID is shining
a light, as some of our witnesses have showed us, on the need for
this type of program, going forward, in this very complex world we
live in.
I guess the biggest concern I have is, like I said—comment to Dr.
Yamamoto—is how do we assure that ARPA-H is not, frankly, just
doing the research that industry would do anyway?
I mean, how do we make sure that we are complementary, and
assisting, and getting to what industry cannot afford to do, that
high risk beyond which they draw the line? How do we evaluate
that?
Dr. YAMAMOTO. I think this really depends on the wisdom and
insights of the program directors, program managers, to be able to
identify de-risking opportunities, areas where industry can’t afford
to go, or feel that there is not an open—not a big enough market,
or that the challenge for getting to—across the valley of death is
too great to risk, economically.
And those opportunities are actually all around us. They are
really at the heart of why it takes a median time of 32 years to
move from a basic research discovery to an approved drug. And so
we will be able to find within ARPA-H the program managers that
that have identified or maybe even lived those barriers. And it will
be—come into ARPA-H.
You know, these program managers, by the way, get their jobs
by competing with each other to come to the director of the agency
and say, ‘‘I have a problem that I think really needs to be solved,’’
right? ‘‘And I have a way that I think it can be solved, and here
is what it is going to take to get there,’’ right?
And a part of that evaluation and—that the director will make
is, you know, what is unique about this? There is something called
the Heilmeier Catechism that says, what is unique about this,
where it is not being done elsewhere? And what is it that you think
can be done that makes it specifically an ARPA-like project?
Mr. SCHRADER. All right, very good. Thank you. I guess that
brings up a second question I had.
I guess Dr. Ling or whoever, I—the question of intellectual prop-
erty. Would the collaboration between the government and—you
know, government equals taxpayer—and industry, which equals
shareholder and, you know—how do we allocate the intellectual
property rights in those types of collaborations? What has been
done?
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Mike.
Dr. LING. That has already been legislated, Congressman: the
Bayh-Dole Act of 1980, and then the—you know, the Federal regu-
lations, the contract for Federal regulations, are very clear. They—
the IP belongs to the private entity. The U.S. Government doesn’t
make anything. They don’t own any factories. So the intellectual
property actually belongs to the private entity——
Mr. SCHRADER. Even in case where we are going beyond just
basic research?
I understand that argument with basic research. But now that
we are going into applied research, should that still apply, in
that——
Dr. LING. To my understanding, it does, Congressman.
The one thing that I would point out, though, in all contracts—
contracts, not grants, but contracts—is march-in rights. The U.S.
Government reserves the right to march in. They can create excep-
tions that allow them march-in. They can march in because the pri-
vate partner did not, you know, commercialize it within an X pe-
riod of time——
Mr. SCHRADER. What, march in, meaning they can acquire those
rights?
Dr. LING. Oh, absolutely. They can call—we—at DARPA, we used
to call it clawback.
Mr. SCHRADER. OK. OK, very good. I guess my last question is
for Dr. Krofah.
That discussed a little bit about we need both NCAT and ARPA-
H. They seem—what is the difference, and why do we need both?
Ms. KROFAH. Well, as I mentioned earlier, the broad portfolio for
NCATS is actually quite diverse in terms of their reach and activi-
ties.
A large part of their portfolio funds the CTSAs. These are the
academic institutions that are quite focused on translational re-
search, and that needs to continue. It is about 80 percent of
NCATS budget. They help to talk about issues such as de-risking,
the vaule of death, but really a significant way that affects the en-
tire sector, the entire industry.
NCATS also has a program, for example, the—program or the
chip for example, and they also have programs for rare diseases.
Some of these programs may be appropriate for an ARPA-H-like
entity, and others may not be necessarily appropriate for an ARPA-
H-like entity. But when we are talking about the point of high-risk,
high-reward projects that would be captured under an ARPA-H,
those types of projects are quite distinct in supporting the overall
field for example of translational sciences, which includes: things
like capturing the clinical trial inventory, which happened during
COVID–19.
Those—mentioned also contributed data into a platform called—
C3, which allowed all of that data that is being generated in these
trials and studies and academic settings to be—for COVID–19.
Those kinds of programs ought to continue—and may not be the
right types of projects for ARPA-H.
Mr. SCHRADER. Very good. Thank you, and I yield back, Madam
Chair.
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Ms. ESHOO. The gentleman yields back. It is a pleasure to recog-
nize the gentleman from Indiana, Dr. Bucshon, for your 5 minutes
of questions.
Mr. BUCSHON. Thank you, Madam Chairwoman. I think most of
the questions about ARPA-H have pretty much been answered, so
I am going to focus on research, in general.
And in a September letter from numerous biotech and venture
capital executives to President Biden, Secretary Becerra, and bipar-
tisan congressional leadership, the executive state—and this is a
quote from the letter—‘‘As written, Build Back Better would cause
investors and researchers to de-prioritize small molecule drugs for
diseases predominantly covered by Medicare, including many can-
cers and Alzheimer’s.’’
Although many of the small companies represented by signato-
ries to this letter will feel that shift in priorities immediately, it
will take years for the public and Congress to see the impact of this
mistake on the kinds of new drugs that do and don’t come to the
market. We can assume that it will take a long time for Congress
to come around to fixing its mistake, so BBB could cost us decades
of small molecule progress. And I know all too well, as a physician,
that eliminating any—even one drug is—has substantial con-
sequences.
So on one hand, some members are stressing the need for addi-
tional research and development—and a new department, in fact,
or agency—while on the other hand actively working to potentially
curtail the industry’s efforts to find new cures through price con-
trol-type policies.
So Admiral Giroir, as you know, President Biden recently re-
launched his Cancer Moonshot, which I agree with. Considering
small molecule oral cancer drugs account for a significant percent-
age of the biotech industry’s oncology pipeline moving forward, do
you share some of the concerns raised by the biotech research and
investment community in this letter?
Dr. GIROIR. Well, thank you for the question. I think we have to
listen to the biotech community in the letter. Of course, this needs
to be overseen by Congress, and really ask the probing questions.
But we cannot—we can absolutely not bias the system against
any class of therapeutic, whether it be large molecules, small mol-
ecules, biologics, vaccines, types of vaccines. The aperture has to be
completely open, and the competition needs to go forward.
So again, I can’t comment specifically on—but you need to listen
to industry, and generally—and to ask the right questions. But I—
by the fact that they raised it and raised it in such a passionate
fashion, I think we need to be very careful about that, because
ARPA-H will need to develop small molecules. And in general, they
are a lot cheaper, easier to manufacture, and easier to keep on
shore than other types of interventions.
Mr. BUCSHON. Thank you. And more broadly, just broadly, do
you think that drug price controls will take us backward in cancer
research and innovation for patients, and potentially could result
in fewer cures discovered if we set Federal-level drug price controls
on the industry?
Dr. GIROIR. You know, I am going to give you my own personal
feeling here. I think there is a balance that needs to be set, right?
93
There is a balance that needs to be set, that—and it can be set.
It is hard to set it, but, you know, if you have drugs that are too—
are not affordable, and people can’t have them, it is no use having
the drugs. On the other hand, we have seen—and particularly in
antibiotics that were brought up—if there is not the proper incen-
tives on that side, then no one will develop it. I do think it is a
balance.
Unfortunately, I am not going to give you a simple answer, be-
cause it is not a simple answer. Both sides are very valid points,
I believe, in my opinion. And this is exactly why we have a com-
mittee like yours to work out that balance, because there is.
Mr. BUCSHON. Yes, thank you for that. I mean, it is a difficult
policy discussion to have. And you know, the Trump Administra-
tion proposed some price control-type things that I disagreed with
from that Administration, and I disagree with price controls in gen-
eral proposed in BBB.
But on the other hand, of course, as you mentioned, patients
should have access to drugs, and we need to strike a balance here,
and figure out what policies that we can put in place, like some of
the bipartisan ones we passed in the last Congress, that can get
drug prices down.
I mean, obviously, I am an avid supporter of innovation, and
have spent my time in Congress advocating for increased funding
for research and development, including at the NIH and other
agencies. I think it is important to examine all the different gov-
ernment-funded programs, in addition to the work currently taking
place at the NIH, and see how we can, you know, pull or pool our
resources together to stretch taxpayer dollars further through bet-
ter collaboration and assurance that more of the money actually
goes toward R&D for innovative drugs, and not toward indirect
costs at agencies.
I yield back.
Ms. ESHOO. The gentleman——
Dr. GIROIR. May I—OK.
Ms. ESHOO. Excuse me, did you have something else you wanted
to add, Doctor?
Dr. GIROIR. I just wanted to say, from the ARPA-H point of
view—and that relates to cost—remember Moore’s Law for semi-
conductors, that every year the capability goes up twofold and it
drops by half. That is why your laptop is so relatively inexpensive.
ARPA-H could absolutely do things—like the cost of a genetic
cure, just the cost of goods, are a million dollars now. That is why
you have to charge $3 million. ARPA-H could have a program to
lower that to 100,000, to decrease that by tenfold, thus increasing
access, lowering costs, and helping everyone out. Those are the
kinds of things that DARPA—ARPA-H could easily do as part of
its mission that will help take the, you know—as an orthogonal ap-
proach to drug prices, yes, we need to do what you are doing, but
let’s lower them across the board by making them more inexpen-
sive because of technologies.
Ms. ESHOO. Thank you, Dr. Giroir. And thank you for quoting my
constituent, Moore’s Law.
It is a pleasure to recognize the gentleman from California, Mr.
Cárdenas, for his 5 minutes of questions.
94
Mr. CÁRDENAS. Thank you very much, Madam Chairwoman, and
I would also like to thank Ranking Member Guthrie. I really appre-
ciate both of you for having this ARPA-H hearing. It is really im-
portant for medical innovation and for progress.
Biomedical research is absolutely critical to understanding our
most insurmountable medical challenges, and ARPA-H is a particu-
larly exciting opportunity, because it will allow our researchers to
investigate with more flexibility, and take on more risk than they
can through our more conventional processes.
To me, one of the greatest promises of ARPA-H is its potential
to address so many conditions that disproportionately impact com-
munities of color and poor people in America. As we come up on
three years of a pandemic that has shown an even brighter light
on the glaring health inequities in our country, I am hopeful that
ARPA-H could provide a lifeline in the form of treatments, medical
devices, and even cures for so many diseases out there.
Dr. Ling, again, thank you so much for sharing your valuable
perspective on ARPA-H’s incredible potential for a biomedical—for
biomedical innovation. How can ARPA-H be leveraged to focus on
bridges—bridging the gap in health outcomes across different de-
mographic groups?
Dr. LING. Thank you, Congressman. I would say that it is about
what ARPA-H should be about, which is developing capability. De-
veloping capability, that is how you are able to help broadly across
the enterprise of not only specific diseases, but also of groups that
have been under-represented, you—by being what—the goal of an
ARPA-H is not a thing, but a capability, not a particular piece of
knowledge, but a capability.
So for example, if you had a much better imaging capability be-
yond that of MRI, it helps everybody. It helps all diseases. Those
are the kind of things that should be an investment of an ARPA-
H. DARPA always created capability, and that led to all the other
wonderful outcomes that we see today.
Mr. CÁRDENAS. Thank you, Dr. Ling. Also Dr. Ling, is it possible
or actually happening out there, where a pursuit of finding a cure
could be a company—a private entity could spend $10 million, $100
million, or even more, and then, oops, find out that they got at the
end of their research and they couldn’t find a cure and have to
shelve it? Has that happened? Does that happen out there?
Dr. LING. Yes, it does. So the—there is always risk in any en-
deavor such as this. But the way you manage that risk is through
the process.
So we had a saying at DARPA, which I think would apply equal-
ly to ARPA, which is, ‘‘Fail early, fail fast.’’ And what you do is,
by having this gated milestone approach, you can see how you are
progressing. You don’t want to find out that you are going to lose
the race at the end of the race. You want to know along the way
that it is not possible to get there.
What it allows you to do is pivot. You don’t have to abandon the
pursuit of that cure. You might have to pivot. You may say that,
oh, a small molecule may not work, I might have to use a large
molecule. I may not have to use a large molecule. Surgery, may be
more—whatever. But the point is, by having this phased-gate ap-
proach, as opposed to just throwing money at the problem, you now
95
have a way of monitoring accountability of the money, and account-
ability toward achieving the goal, which is, as you point out, the
cure.
But remember, in any of these journeys, if you push the knowl-
edge forward as you do this—you want to push it as fast as you
can—you can build on it, even if it looks like you are not going to
get there.
Mr. CÁRDENAS. Dr. Ling, it is unfortunate, in my opinion, that
in America today some of these innovators, some of these compa-
nies, private entities that are investing 100 million, in some cases
even $1 billion, and don’t bring something to market, that there is
something evil about that.
What is the reality and practicality when an entity has just in-
vested 300 million, 400 million, 600 million, 700 million, and they
are just not getting the results to find that cure, for example? Is—
does that make them evil for having to figure out maybe it is time
to shelve it, and then let’s focus on other things?
Dr. LING. Sometimes the science is not there. Sometimes the way
it was conducted. There is many reasons for so-called failure, and
I think that is——
Mr. CÁRDENAS. So therefore, in our limited time, Dr. Ling, so
therefore, is it that ARPA-H will actually help create more opportu-
nities where those investments are—the willing of—the will of
those investments can actually result in more cures, more results,
more positive results, as you mentioned earlier, having the ability
to have this support system, and then shift, and then continue,
rather than abandon?
Dr. LING. Absolutely. Absolutely, you can—you—when—we learn
mostly from our mistakes. We learn mostly from our failures. It is
the pivot and move that becomes very essential. And that is, in
fact, the model of an ARPA. An ARPA does exactly that.
Mr. CÁRDENAS. Yes, thank you very much. That is why I have
my wife, she definitely reminds me when I make mistakes, and
then I learn, and then I[inaudible].
Thank you so much. I yield back.
[Laughter.]
Ms. ESHOO. Dr. Yamamoto, you were nodding. Did you want to
add something to this, briefly?
Dr. YAMAMOTO. Yes, I did. Thank you. I would just like to say
that the other way that ARPA can contribute to solving the prob-
lem that you raised—and a very important one—of a company
making a huge investment and coming up with failure, is getting
back to capabilities.
Something that Dr. Ling and I talked a lot about when he was
founding the BTO was being able to use big—was having DARPA
be able to generate computational tools that would allow us to
evaluate massive amounts of data that could then be serving, in
the case that you are raising, to changing the nature, the size, and
the composition of clinical trials.
The more we know about the mechanism behind a given disease
that—which can be gathered by, in fact, pulling together lots of
data, including real-world evidence—that was raised earlier—the
better we are able to be able to construct patient cohorts in clinical
96
trials that actually have a bigger chance of having the tested drug
be successful.
There is great examples of this, but in breast cancer medicine,
for example, the first precision medicine drug, if you will, call
Herceptin, the drug made by Genentech, a biotech—a small, rel-
atively small, biotech company at that time—was able to structure
a clinical trial based on what they knew at the molecular level
about the cause of a certain kind of metastatic breast cancer. And
by being able to limit the size of that cohort, they were able to
carry out a small clinical trial that went relatively rapidly, and suc-
ceeded, and Herceptin came out as a drug.
If they didn’t have that information, if they hadn’t collected that
data, they would have had to put together a big trial, with many
more patients, and it would have lasted longer. And in fact, we
know from Sue Desmond-Hellmann, the president of product devel-
opment at Genentech during that time, they would not have car-
ried out the trial because it cost, as you said, hundreds of millions
of dollars, and it would have failed.
So one of the things that ARPA-H can do is to be able to carry—
put together capabilities of that sort that would, in fact, lead to
clinical trials that are more—that are successful, and lead to drugs
that are successful.
Ms. ESHOO. Thank you. The gentleman’s time has expired.
Dr. Yamamoto, it is nice to hear Susan Hellmann’s name raised
and mentioned.
The Chair is now pleased to recognize the gentleman from Penn-
sylvania, Dr. Joyce.
Mr. JOYCE. Thank you, Chair.
Ms. ESHOO. Five minutes.
Mr. JOYCE. Thank you, Chair Eshoo, for yielding, and to our wit-
nesses for appearing——
Ms. ESHOO. And happy birthday to you.
Mr. JOYCE. Thank you for the birthday greetings.
Ms. ESHOO. Happy birthday from all of us.
Mr. JOYCE. Thank you, I appreciate that.
Thank you to our witnesses for appearing here today. This is an
incredibly important topic.
Dr. Ling, thank you for your passionate testimony on this sub-
ject. You have worked at Johns Hopkins. You have worked in aca-
demic medicine there. You have worked in the military, and you
have worked in the private sector. I think the many prongs that
you bring into this equation are very important.
We recently saw a controversial coverage decision from a govern-
ment agency regarding a breakthrough treatment for Alzheimer’s
disease, specifically related to amyloid and the deposition of
amyloid. How would you envision this ARPA-H agency working
with and complementing work being done already with the private
sector, and not being resistant to private sector innovation?
Dr. LING. Thank you, Dr. Joyce, and—Congressman Joyce. The
drug that you are speaking of, I believe, is the Biogen drug
Aducanumab, which was for Alzheimer’s, and one of the first to ac-
tually show some efficacy against this horrible disease.
So again, I am not privy to understanding the reimbursement de-
cisions, or anything like that, but I can simply say this, is that
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comes back to what Dr. Yamamoto just said, and that is developing
those capabilities that you can work with a company like Biogen,
or you can work with a company like GSK and these others, so that
they can better use the data that is available to make it better and
easier for them to conduct their clinical trials to be able to save the
money up front, to determine more quickly what, in fact, is the
benefit.
Because to my understanding of the study right now, is it is—
the question is it doesn’t work for everybody, but it is very effective
in some. And so how do you identify those ‘‘some’’ that could do
well? So—because that is true for cancer, that is true for infections.
There is always a cohort that seems to do much better from that
therapy than, say, another, for whatever reasons it may be.
And elucidating those reasons using new computational capabili-
ties, perhaps using new diagnostic capabilities, all these things
within the purview of an ARPA-H to build that capability, Dr.
Joyce, so that it can be brought to bear so that then these decisions
will have much better evidence to work by, that is the way an R&D
agency such as an ARPA would be able to contribute in a positive
way to the point that you are making.
Mr. JOYCE. Dr. Ling, you mentioned the research that is nec-
essary. What do you believe can be taken from DARPA and ARPA-
E when it comes to securing that research, securing the confiden-
tial and sensitive nature of research from nefarious foreign actors?
And how can we do that successfully, while being able to work
with the private sector and the academic researchers, all at the
same time?
Dr. LING. No, the point you raise is very, very important. You
know, these are investments made by the American taxpayer, by
the citizenry of our country, and they should be, first and foremost,
be to the benefit of the United States.
And I said to you before, and I say it again, that what we are
proposing is something a totalitarian government would never pro-
pose. That is, having a free and independent agency that would be
able to work on behalf of the citizenry. That is totally against what
a totalitarian government, such as the adversaries of which we
speak of.
As we come back to ensuring that they do this, I think we can
take the model of the Department of Defense and DARPA. DARPA
agonizes over this all of the time. DARPA agonizes that the things
that they create will work their way into an adversarial’s arsenal,
quite frankly. And so, in many ways, we can learn to use those
safeguards and to use those processes that already exist by the
model agency that we are working on right now to incorporate
them back into an ARPA-H, much as it is in an ARPA defense,
which is what DARPA is.
Mr. JOYCE. And I thank you, Dr. Ling, for mentioning the time
restraints to achieve these goals, to listen to industry, and we, as
Members of Congress, to be responsible stewards of the taxpayer
dollars.
Thank you, Madam Chair. Thank you for holding this incredibly
important meeting, and I yield the remainder of my time.
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Ms. ESHOO. And the Chair thanks you, always, for your thought-
ful questions and the—such a respectful way of presenting them.
I think we all appreciate that.
The Chair is pleased to recognize another one of the doctors—we
are blessed, because we have several doctors as members of this
subcommittee, and we benefit from their membership here.
Dr. Ruiz from California, 5 minutes of questions.
Mr. RUIZ. Thank you, Madam Chair, for holding this important
hearing.
Over the course of the last two years, the COVID–19 pandemic
has exposed the magnitude of health inequities plaguing our coun-
try. As a doctor who grew up and practiced medicine in a medically
underserved community where health inequities are rampant, I am
encouraged that these disparities are at the forefront of our policy
conversations. It is imperative that, as we consider the structure
and implementation of the ARPA-H program, that we prioritize
health equity.
Ms. Krofah, the Administration is committed to promoting and
prioritizing health equity in every decision made by ARPA-H. How
can this be achieved, and should such a directive be included in
statutory language authorizing ARPA-H?
Ms. KROFAH. Congressman, thank you so much for raising that.
You know, when I think about the last two years with the pan-
demic, it has been absolutely devastating on communities of color,
while at the same time there are essential healthcare workers that
were suffering disproportionately.
And the issues around health equity are not new, right? We have
known these issues for many, many decades, but we have not gal-
vanized the full attention both of government, but also all of our
private sector, non-profit sectors, and others to really address this
problem. I think we have the unique opportunity to do so now, and
I absolutely agree with you that ARPA-H is a model and a vehicle
that we should use to really start to understand what is different
in the underlying biology of diseases that may affect some popu-
lations differently than another.
You know, an analogy that was used the other day that I really
appreciated is that, when we think about our clinical trials, it is
like testing a car down a straight road and expecting that it is
going to perform when conditions change. What we really need to
do is to test our products on windy roads, for all populations, to un-
derstand when they may not work for subsets of populations.
Mr. RUIZ. Well, I appreciate you saying that, because I have a
bill specifically to do just that, Diversity in Clinical Trials Act. Do
you think that we need to add statutory language authorizing
ARPA-H to do the health equity work?
Ms. KROFAH. I think we need to give the program managers and
ARPA-H the flexibility. However, I do think that we need to have
a patient advisory board that really provides the diverse represen-
tation. That should be written in, where there is consultation in
which projects are prioritized.
Mr. RUIZ. And how can we—how can ARPA-H ensure that its
workforce is sufficiently diverse?
And how do you see workforce diversity play a role in ARPA-H
supporting projects to advance health equity?
99
Ms. KROFAH. I would agree we need diversity in the program
managers, we need diversity in even the director of ARPA-H, and,
particularly given that it is time-limited, who those directors will
be over time.
We know, particularly at the provider level, that patients are
more likely to see people who look like them. We need program
managers who look like the communities that are suffering from
the burden of disease. So I absolutely agree that part of the consid-
erations for hiring needs to include diversity, and that should be
embedded in the legislation.
Mr. RUIZ. Thank you, Ms. Krofah. I am a man of science. I like
to measure things. And if you can measure it, then there is a way
you can improve it. So how can we measure societal improvements
in health equity catalyzed by ARPA-H?
Ms. KROFAH. We need metrics. Earlier we talked about we need
a strategic plan. I would add that we need metrics. We need spe-
cific metrics for programs that are identified, for which populations
may benefit, and for what outcomes we are looking for.
Mr. RUIZ. And how would ARPA-H guarantee that products de-
veloped through their pipeline are made available and affordable in
an equitable manner to all consumers?
Ms. KROFAH. You know, that is the role of the private sector. We
talked a bit earlier about what the private sector offers. They offer
that manufacturing, they offer that end to end.
What we need to ensure early on with these high-risk projects
with ARPA-H is that we have the ability to reach communities that
we are not reaching already. Let’s talk about capabilities——
Mr. RUIZ. Well, currently—you know, I understand it is the pri-
vate sector, but current—in the private sector, often times people
who can’t afford a certain price are left out of lifesaving remedies
that—they have no choice over whether they live or die if they
don’t get the lifesaving remedy. So government has a role in influ-
encing and incentivizing equity and promotion.
So I hope that, as—Ms. Krofah, that ARPA-H can help advise on
ways that we can promote equity like the Biden Administration
has done in purchasing some of the vaccines and incentivizing the
pharmaceutical companies to use 40 percent in underserved com-
munities.
And with that, I yield back my time.
Ms. KROFAH. Yes, I will just——
Ms. ESHOO. The gentleman yields back.
Ms. KROFAH [continuing]. Comment, just one more——
Ms. ESHOO. Oh, I am sorry.
Ms. KROFAH [continuing]. Comment on that last point, which is
just to say that the regulatory piece of the collaboration with
ARPA-H is quite critical. We need FDA who’s responsible for the
review and approval of those products to make sure participants
are diverse, and that is what I would add to that.
Ms. ESHOO. Thank you.
The gentleman from Georgia, Mr. Carter—oh, you want to take
Mr. Curtis first? Wonderful.
Mr. Curtis, good to see you. You are recognized for 5 minutes for
your questions.
Mr. CURTIS. Thank you, Madam Chair, Mr. Ranking Member.
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Well, those who know me know I am excited to have another
chance to brag about the community in Utah, and our advances in
life sciences, and the great things that we are doing there. The
Utah BioHive and Utah Health Care System is very active and
very healthy. As a matter of fact, BioHive is made up of about
14,000 companies across the State of Utah, and I am really con-
fident that it has been a really dynamic and powerful combination
of public-private investment and success.
And I have got to tell you, I am just really pleased with the panel
that we have had here today. I have really enjoyed the discussion,
and I feel like there is a strong sense that we all want to do some-
thing, and we all want to do the right thing, and that we are here
to have a thoughtful discussion about what that is. And from the
tone of my questions, I wouldn’t want anybody to imply that I am
opposed to ARPA-H. I just have questions that I want to have re-
solved in my mind.
Maybe, Dr. Miller, if I could start with you, I am reminded, as
we talked today about a famous quote from Ronald Reagan that he
made famous, ‘‘The most terrifying words in the English language
are ’I am from the government, and I am here to help’.’’ And it feels
like sometimes Congress wants to solve all our problems by throw-
ing a lot of money at it, and instead of analyzing maybe what we
could be doing to more facilitate what is happening in the private
sector.
So I wonder if we should also be talking about less regulation,
faster approval process, how removing incentives like I think H.R.
3 does impacts less giving away of IP, as I think we have done with
COVID, and less competition. And if I understand some of what
has happened today, it almost feels like ARPA-H could come into
Utah and compete with some of my private investors, right? Maybe
in an IP or otherwise.
So could you just talk about is that a possibility, and what could
we be doing to—Dr. Ling, I loved your passion when you talked
about unleashing this power, right? But I got to tell you, having
spent most of my time in the private sector, I feel the same way
about the private sector.
And what can we do, Dr. Miller, to fully unleash the private sec-
tor?
Dr. MILLER. Thank you, Representative Curtis, for that question,
and good to see you. A couple of things.
First of all, a $6.5 billion investment in biomedical research does
not counterbalance the threat of administrative pricing in drug
markets. So the—that doesn’t really compute for me.
I think you are right, all—this could potentially, actually, mis-
place—displace the private sector. I hear phrases like ‘‘time-gated’’,
‘‘performance metrics’’, ‘‘fail early,’’ holding program managers re-
sponsible. All that sounds like is what I hear my colleagues from
pharmaceutical companies and device manufacturers saying. So I
worry that ARPA-H will be potentially directly displacing them.
I think we have to look at regulatory barriers, as you said. The
Medicaid Drug Rebate Program is one, for example. We have these
high-cost, million-dollar therapies, and we want equitable access to
them for everybody. Well, the Medicaid Drug Rebate program
means that, if you have a value-based contract, and your pharma-
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ceutical company doesn’t meet the milestone, and the value is zero
dollars, that means the price for your drug is zero dollars. So I
think we need to address barriers like that.
Mr. CURTIS. Thank you. There has been a pretty healthy debate,
but I would kind of like to give Ms. Krofah a chance to weigh in
on this, or any of you that would like to, this concept of is the best
place for this under NIH.
And I have just got to tell you, from a—here again, from a busi-
ness perspective, you typically would not put an organization that
you wanted very different—under an organization that the culture
was so different. So, Ms. Krofah, you seemed to disagree with that.
I would love to hear from you, and any of the—else that want to
weigh on that that haven’t had a chance to express your thoughts
on that.
Ms. KROFAH. Yes. Well, thank you so much, Congressman. And
I do seem to be in the minority view at least with my panelists on
this topic. So I do you appreciate you pointing that out.
My perspective comes from a few different places. One is I just
experienced coming out of the COVID–19 pandemic. I absolutely
agree, in terms of the culture, NIH really is a culture that allows
us to do scientific discovery. What we saw differently during
COVID was NIH put together public-private partnerships through
active through Red X that helped us achieve in, really, a tremen-
dously short amount of time, movement in vaccine therapeutics
that really translated into saving of lives.
If we can take those platforms and leverage those learnings,
there is an opportunity for ARPA-H to succeed and do well within
NIH, but with safeguards. We talked earlier that we need—abso-
lutely we need those safeguards. We need ?
Mr. CURTIS. I——
Ms. KROFAH [continuing]. That there is a level of independence,
which——
Mr. CURTIS. I regret that we are out of time, so I am going to
cut you off before I get cutoff.
I—once again, before I end, I would just like to thank—the dis-
cussion, I think, has been healthy and vibrant, and I appreciate
being part of it. Madam Chair, I yield my time.
Ms. ESHOO. The gentleman yields back.
The Chair is now pleased to recognize the gentlewoman from
Washington, another one of our wonderful doctors, Dr. Schrier.
Ms. SCHRIER. Well, thank you, Madam Chair, and thank you to
our panelists for coming today. I am very excited to talk with all
of you about the prospect of ARPA-H. And I think I can speak for
all of us when I know just how remarkable it was when the power
of the Federal Government joined with private industry to rapidly
roll out—develop and roll out coronavirus immunizations. And we
had shots in arms in less than a year, and that was remarkable,
and it is this sort of power that we are looking to harness in ARPA-
H.
This presents such tremendous opportunities, specifically for my
home State of Washington, for the researchers who are working on
things like CAR-T gene therapies, and precision medicine, and
more. And in my State innovative researchers spent more than 8
years pioneering personalized cancer immunotherapies for patients
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with lymphoma that hadn’t responded to traditional treatment, and
that CAR-T therapy represents not just another treatment or the
last ditch effort, but is a cure, as we have seen recently, as that
immunity lasts a lifetime, and will continue to kill any residual
cancer cells that might arise.
I will tell you that another brilliant Washington researcher is
studying the ways we might be able to tailor medicine according to
individual genomes and phenoms. So using genetic information,
coupled with environmental factors, this theory can inform, even
from infancy, a child’s lifetime risks for disease, and give parents—
and then that child later—the tools to mitigate those risks and
keep them well.
So, Dr. Yamamoto, I wanted to ask a few things, if you wouldn’t
mind answering briefly about how ARPA-H could impact just ev-
eryday people in Washington State. Like, in your opinion, would
ARPA-H be a catalyst for expanding CAR-T gene therapy, maybe
to see if it will work in solid tumors?
Dr. YAMAMOTO. Thank you. CAR-T therapy is, in many ways, is
powerful and amazing and important, as it is. This is really the tip
of the sword for being able to do cell engineering. It enables us to
use—develop cell therapies that deliver new therapeutics to the
point of action, to the exact cells that are responsible for the dis-
ease, for example.
And so I think that there is every possibility that, under an
ARPA-like management, that cell therapies could be developed that
will definitely be able to serve people throughout your State, and
throughout the country, and the world, in fact, because they are—
they provide the kind of targeting that allows for early diagnosis,
for highly effective therapy early in the state—in disease. And that
sort of detection and treatment is really what is needed in order
to really counter disease——
Ms. SCHRIER. That is great. And——
Dr. YAMAMOTO [continuing]. Early detection and focused, tar-
geted treatment.
Ms. SCHRIER. And speed is of the essence, right? I mean, eight
years to get CAR-T, it is remarkable in one sense, but with the
force that we put into Operation Warp Speed, imagine what we
could do for, say, pediatric solid tumors, brain tumors, and they
just don’t have time.
Also, I——
Dr. YAMAMOTO. I will just add that, as a matter of fact, there is
a project that UCSF—actually being undertaken right now to be
able to use CAR-T therapies in glioblastoma, a very important and
devastating brain disease.
Ms. SCHRIER. That is phenomenal. Thank you. I wanted to also
ask, in your opinion, as we talk about precision medicine—and I
think of this, as a doctor, and who is enrolled in studies. We had
to work really hard to get a diverse population in vaccine studies.
I was wondering how precision medicine could make a difference,
specifically for how health outcomes for women and people of color.
Dr. YAMAMOTO. It is essential that—so precision medicine, as you
know, really capitalizes on being able to aggregate and integrate
and analyze vast amounts of data about many, many different indi-
viduals, right, to be able to gain the knowledge that will allow us
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to be able to then take a focused approach to a given disease. And
collecting that data then, in a way that is equitable, is very—then
becomes very essential.
Clinical trials in this country have not been carried out in an eq-
uitable fashion, and it is—and it is the reason that we have found,
at the end of the day, disparities that are very damaging. And at
the root of them is the failure to be equitable in collecting the infor-
mation and analyzing it.
Ms. SCHRIER. I only have ten seconds left, so I just want to say,
as a pediatrician, how exciting all of this as I think about my pa-
tients, patients with autism, patients with threatening diseases—
for them we can find cures through something like ARPA-H. So
thank you very much. I yield back.
Ms. ESHOO. Thank you, Dr. Schrier. Now, who is next?
OK, the gentleman from Georgia, the pharmacist on our sub-
committee, you have five—the only one, too, that is right—you
have 5 minutes, Mr. Carter, for questions.
Mr. CARTER. OK, and thank you, Madam Chair, and thank all
of you for being here.
I want to start—and bear with me here, before I get into my
questions—but it seems like we have a bullying problem with this
Administration. I mean, our first witness that was supposed to be
here this morning is not here because he resigned last night be-
cause of supposedly bullying.
Dr. Fauci, another example of bullying, bullying us into having
to take vaccines and having to wear a mask. All examples of bul-
lying.
You look at Dr. Kestner and Dr. Levine bullying Governors into
not using monoclonal antibodies, which have been proven to be ef-
fective, and then limiting the supplies of those.
The Administration bullied Americans into not using Ivermectin,
calling it a horse de-wormer, when actually it has worked for many
people, been effective.
And then you have got U.S. Trade Representative Katherine Tai,
who has tried to bully American pharmaceutical companies into
giving their intellectual property, free of charge, to China.
You know, it just—there is a common theme here, and it is bul-
lying, and it needs to stop with this Administration.
Now that I got that off my chest, Dr. Miller, I want to ask you.
You know, we have got a lot of serious public health challenges in
this country. There is no question about that. And I recognized that
in my 30-plus years of practicing pharmacy. And we need innova-
tion, we need treatment innovation for lymphedema, cancer, hyper-
tension, and all kinds of things. But I am especially concerned
about the prevalence of antimicrobial resistance, and the need for
new medications. ARPA-H proposals suggest an entirely new de-
partment is needed to address the lack of cures and treatments for
these issues.
And, I don’t know, one of our colleagues on this committee, Mor-
gan Griffith, had recommended a book, ‘‘The Perfect Predator.’’ I
don’t know if you have read that or not, but I am right in the mid-
dle of it, and it is a fascinating, true story, a fascinating read. But
I will tell you, NIH gives out billions of dollars every year to dif-
ferent agencies, to different companies. And I get it. I understand
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what their role is, and what they are supposed to be doing here.
But sometimes you have to wonder if we are getting the return on
our taxpayers’ money that we should be. And it looks like there is
a lack of transparency and accountability at the NIH, and it is con-
cerning to a lot of us.
Dr. Miller, what can Congress do, what could we do to reform
and modernize the NIH and its mission for advanced research be-
fore opening an entirely new department, as is being proposed?
Dr. MILLER. Thank you. I think this brings just a couple core
questions to us about sort of how to run a research enterprise. Like
my colleagues have all said—and I agree with—you want to mini-
mize bureaucracy, right?
So just blowing that bureaucracy away, why is the grant guide
154 pages long? I mean, that is longer than most people’s grants.
Could you imagine writing a grant in response to that?
So I think blowing that away, creating a culture of risk tolerance,
and that culture of tolerating failure and supporting failure as we
think about platforms, as we think about new big ideas, all these
principles that my colleagues are mentioning are principles that we
should try and integrate into the NIH, and we need to change that
culture.
Mr. CARTER. Well, thank you for that. Let me ask you, again—
Admiral Giroir, is he with us?
Dr. GIROIR. Yes, he is.
Mr. CARTER. Yes, thank you. I wanted to ask you, I have men-
tioned my concerns about the prevalence of antimicrobial resist-
ance, the drugs that are currently on the market, and I have cer-
tainly witnessed this in my practice of pharmacy throughout the
years in the overuse of antibiotics, the lack of pharmaceutical com-
panies investing into research and development for new antibiotics.
I believe it is like the early 1980’s, the last that we had, and we
need to address that. We have got to address that in this country,
and I would rather government stay out of it, but at the same time
I don’t know how we are going to afford not to. We have got to
stimulate this in some way.
But looking back on your time at HHS, Admiral, I wonder if you
can fill us in on why BARDA and other Federal agencies aren’t
equipped to address these needs. Is there room for reform at
BARDA and NIH and other research programs in the Federal Gov-
ernment to address these type of gaps?
Dr. GIROIR. Well, thank you, and it is good to see you again. And,
you know, I am a big pharmacist fan, and I think ARPA-H could
do a lot to support pharmacy and distribution of care.
These are fundamentally different organizations. BARDA has
turned into—and it is fine—a truly advanced development group on
a limited mission set that is for biodefense. So you see investments
being made like taking an underlying technology that might have
been developed by DARPA, and making sure it gets approval for
influenza, or making sure it gets approval for COVID. So it is in
a fundamentally different operating space, and I would say its
processes are highly bureaucratic, and not really that quick, agile
type of program.
On the other end, you have the NIH, which we have discussed
very much, so there is, of course, room for reform across the board.
105
But that bridging of the gap that ARPA-H can do—not to displace
the private sector, but to empower the private sector—to decrease
risk for the private sector, to create technologies that a rising tide
will raise all boats, this is all the kinds of things that ARPA-H will
do.
And let me just say, in terms of equity, we talk a lot about cures.
Just as you and I talk so much about distributing healthcare by
using pharmacists, I think the explicit goal of DARPA should be to
get care and prevention in the homes, particularly of the under-
served in the world. That may not be as sexy as curing stage four
cancer, but it is vitally important, and is along the lines of distrib-
uting equitable healthcare and meeting people where they are.
Mr. CARTER. Thank you, Admiral.
And I thank you for your indulgence, Madam Chair, and I yield
back.
Ms. ESHOO. Let me just say we have two votes that are up on
the floor, so we are going to have to go over to vote.
We have two, four—Mr. Crenshaw came in, that is five, plus a
waive-on. Let’s take two more members, and then we will recess.
I think the—our witnesses need a break, as well. And then we will
come back to take the questions of members that have not been
recognized yet.
So at this point I will recognize the gentlewoman from New
Hampshire, Ms. Kuster, for her 5 minutes of questions.
Ms. KUSTER. Thank you, Madam Chair, and I want to thank all
the witnesses for being here today, and for this hearing. I want to
take some time to discuss ARPA-H’s relationship to existing efforts
by the National Institutes of Health.
The NIH has run large, complex programs before, using DARPA-
like approaches to drive highly managed, use-inspired, break-
through research. For example, the NIH Rapid Acceleration of
Diagnostics, RADx, initiative utilized an innovative funnel ap-
proach to rapidly advance promising COVID–19 diagnostic tech-
nologies. Other NIH programs have similar goals to ARPA-H, such
as the National Center for Advancing Translational Sciences and
the Accelerating Medicines Partnership. The ARPA-H program,
then, will need to complement NIH’s existing research portfolio,
rather than duplicate it, as we have heard in this hearing today.
Ms. Krofah, can you explain how you see ARPA-H comple-
menting NCATS and other research programs at NIH, and how can
its structure avoid redundancies to existing programs?
Ms. KROFAH. Thank you so much for that question. And in fact,
you just referenced some good examples from NIH about use-driven
research that would be applicable in the ARPA-H context.
I referenced in my prior talk that we need to look at RADx, and
the model that was used in RADx, as a potential for what ARPA-
H could mean and be in an NIH context. So I agree with you, that
those particular case studies—learning from NCATS, learning from
AMP, learning from RADx—are absolutely great examples in terms
of NIH has performed these types of activities before, and they
could be successful in the future, again, with those guardrails that
we talked about and the independence that is needed.
Ms. KUSTER. Great. Now, one of the cornerstones of Federal sup-
port of research has been around the use of peer review, which
106
plays a critical role in determining project merit and, ultimately,
whether a project is supported.
Dr. Yamamoto, can you speak to how peer review will be lever-
aged at ARPA-H, and how is it different from the process leveraged
by NIH and other Federal agencies?
Dr. YAMAMOTO. Well, if we learn from DARPA, what we know is
that there is not actually peer review there, but it doesn’t mean
there is not review—there is extensive review—and that one of the
powers of an ARPA agency will be that it will gain expertise and
input and advice, not just from peers, those that are actually car-
rying out the kind of work that is being proposed, but from across
the research spectrum, looking—getting experts from different
agencies within the Federal Government, something the NIH
doesn’t do, and being able then to being able to establish a judg-
ment about the kinds of programs they will undertake, and being
transparent about it.
So there will actually be extensive review within the ARPA agen-
cy. This is what we have learned from DARPA, very powerful
mechanisms that work very, very well.
Ms. KUSTER. Do you think it will be more of a collaboration ap-
proach, is that what you are saying?
Dr. YAMAMOTO. Collaborative in the sense that you are bringing
voices in from different groups that are not—voices that are not
tapped in the NIH peer review system. Remember, that it is an im-
portant, I think, distinction to make, that what NIH is trying to
do is knowledge discovery, and that—and that the—really, the best
way to do that, we now know from many decades of experience, is
to give working scientists their own head in—with the problems
that they choose to study, using their—the drive of their curiosity
to understand something, to do that.
And so peer review of just calling on other fellow scientists to
make a judgment about the merits of a given proposal actually
works very well. It is—and as Francis Collins has said, it is—does
tend to be conservative, because peers are the ones that made the
existing paradigms that are going to be defended. They will choose
things that are relatively high feasibility, so higher-risk projects
are not as well celebrated within the NIH. Those are problems.
They are problems that I think can actually be solved by modifying
the peer review system.
But the ARPA system actually depends on being able to cast a
wide net, get out into the communities, and listen to what is need-
ed, and what is available, and what is possible in order to bring
together groups to build the kinds of capabilities that Dr. Ling
talks about.
Ms. KUSTER. Well, I think your testimony has been so helpful
and important for us today to understand that collaboration is
going to be essential, and it is distinct from the other NIH efforts,
and I think that is an important distinction.
Thank you, Madam Chair, I yield back.
Ms. ESHOO. We thank you.
Let’s see if we can get two more in. Is that all right with you,
Brett?
OK.
107
The Chair is pleased to recognize the gentleman from Texas, Mr.
Crenshaw, for your 5 minutes of questions.
Mr. CRENSHAW. Thank you, Madam Chair, and thank you to the
ranking member. Thank you to the witnesses for being here for
this really important subject. It is fascinating to all of us and I
think there is certainly broad agreement that we need to do more
to help biomedical innovation and healthcare innovation. There is
a lot we can do.
I do have concerns about the ARPA-H proposition, that it may
be duplicative, and that it doesn’t address some of the problems
with innovation, the core problems with CMS, with the FDA. You
know, I wonder if that valley of death is really lack of funding or
problems with the FDA and CMS not agreeing to pay for a par-
ticular project or treatment.
For Dr. Miller, if ARPA-H will cover investment when the pri-
vate sector fails, we have to ask the question: Why is private sector
not investing in a particular product?
OK, so why wouldn’t they want to invest in a particular project?
Dr. MILLER. Thank you, Representative Crenshaw. I get pro-
posals from biotech companies and device manufacturers probably
every week, and I read them, and usually my answer is no, this
isn’t going to go anywhere. And it is not necessarily because it is
not a good scientific idea, but because, one, there is usually not a
payment policy framework for it.
I mentioned the Medicaid Drug Rebate program earlier. That is
a common barrier for these sort of curative dream-type therapies
that we would like to see that can cure rare diseases.
I think other things are, like at the FDA, we don’t have a path-
way for software-driven medical devices. And so you are not going
to develop that product, because there is no path to market. If we
want to turn into the Borg—which, you know would be great, I
could run faster, I wouldn’t have to worry about getting a knee re-
placement eventually, and other things—those products aren’t
there, and no one is going to create them, because you invest hun-
dreds of millions of dollars, years of time, untold thousands of
human hours of labor, and then you don’t get FDA clearance be-
cause the FDA says, ‘‘Oh, am I going to evaluate this as AI? Am
I going to evaluate this as machine learning? Am I going to evalu-
ate this as software as a medical device? Am I going to evaluate
this as a traditional medical device? And should it go through all
four offices before it gets cleared?’’
So I think a lot of these are regulatory barriers, and we have to
address them.
Mr. CRENSHAW. And maybe I will move the question to Dr. Ling.
You know, is there anything in these proposals that would
change that?
I mean, what assurances could ARPA-H give to therapeutic de-
velopers that their product might be greenlit by regulatory agen-
cies?
Dr. LING. Well, that is a wonderful question, Congressman.
Thank you for it.
Part of it, again, comes from—is that there needs to be innova-
tion at all levels. CMS needs to innovate. FDA needs to innovate.
CDC needs to innovate. NIH needs to innovate.
108
Mr. CRENSHAW. OK.
Dr. LING. Within these different groups. to keep up with the 21st
century—because that is where we are right now, facing 21st cen-
tury problems—in fact, you are correct, across the enterprise this
needs to be done, but this is where ARPA-H could be very helpful.
Mr. CRENSHAW. I—and I agree. Like, I think there is places for
that. But it does seem like the—you know, we might be putting the
horse before the cart here, or the cart before the horse. The horse
does go before the cart.
[Laughter.]
Mr. CRENSHAW. Anyway, you know what I mean.
Maybe—and help us paint a picture, Dr. Ling, of, if ARPA-H was
created right now, in your perfect vision, do you have any examples
of some projects that it could immediately undertake?
I mean, who out there, what startup out there right now, is just
waiting for investment, but just can’t get any?
Dr. LING. Thank you, Congressman. One project I think that
would be very helpful, to be illustrative, is what I came back to be-
fore, is imaging.
So right now—in the 1920’s, we came with X-ray, great. In the
1960’s we came up with CT scan, great. In the 1980’s we came out
with MRI, super duper. What has happened since then? Nothing.
So an ARPA-H project would be get me an imaging platform that
would have performance metrics at least an order of magnitude
better than MRI. Boom. Make it so that it has to operate at room
temperature. That drives cost down. Make it so that it is not using
ionizing radiation, much as X-ray and CT do, so it doesn’t hurt pa-
tients. What technologies can bring to bear that you can do this
right now?
And I am a geek, all right? So, for example, quantum orbital res-
onance spectroscopy could be an example. What you want—and
there are small groups doing it right now, they can’t get the money
to do it. Siemens doesn’t want to do it. Why? Because they sell
MRIs. Why in heaven’s name would they do that? So it is a white
space. It is a technological solution.
Now, what would be the benefit of doing such a thing, you would
ask, Congressman. Well, if you had an order of magnitude better
performance, you could actually diagnose cancer earlier. And I
don’t mean just a cancer. I mean, all cancers. Then you now have
the opportunity of treating cancers when in stage one and stage
two. We may not have to invent new drugs. We may actually im-
prove health because we are able to—that is an example of build-
ing a capability.
Now, you talked about the regulatory on that. You need to drag
the FDA in, right then and there, as we start, and say, ‘‘Look, this
is coming. You need to come up with ways to regulate this. That
is your job.’’ But they—but you can’t bring it to them 4 years later,
after it is done, and say, ‘‘Now you have got to do it.’’ You have
got to bring them in on day one. That is the point I am trying to
make, is that that end-to-end solution—and ARPA-H would call
that—as much as Dr. Yamamoto said, you have got to bring these
groups together now, early.
You have got to bring the patients in, because they are not going
to want to lie down in this thing if they don’t understand what the
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heck it is. So you have got to bring them in early, early, early. You
have got to bring them in at the very outset, Congressman. That
is, in fact, how DARPA does it. It doesn’t tell the Marines you are
going to have this new thingamajig. They bring them in right away
and say, ‘‘Look, we are going to develop this thingamajig. You need
to go—how to figure out how to make it, and then—and put it in
to your combat system and your tactics.’’
So you don’t do—you have got to do it from the beginning, Con-
gressman. That is the how you do it.
Mr. CRENSHAW. I appreciate your answer, and I have gone well
over.
Thank you, Madam Chairwoman. I yield back.
Ms. ESHOO. The gentleman yields back. I am going to call on one
more member, because she can’t return when we resume the hear-
ing—is the gentlewoman from Illinois, Ms. Kelly, and then we will
break for the two votes.
And what time should we say we will be back, Mr. Brett?
VOICE. Mr. Brett?
Mr. GUTHRIE. Twenty minutes after the first vote—after the last
vote.
Ms. ESHOO. OK, but how long is that? Just to give the witnesses
an idea.
Well, why don’t we—well, let’s see what time we walk out, and
then we will better estimate the time.
So the gentlewoman from Illinois, you have 5 minutes——
Ms. KELLY. Thank you so much, Madam Chair and Ranking
Member Guthrie, for holding this hearing on ARPA-H.
It is well-established that our country faces large gaps in access
to care, and that the color of your skin can determine the quality
of your care and your health outcomes. Despite these inequities,
Black and Latinx scientists who are well aware of the role these
inequities play in their communities continue to be funded at low
rates.
According to the NIH, in 2020 only two percent of funded NIH
research project grant applications have Black or African American
lead scientists, and five percent had Hispanic or Latinx-led sci-
entists.
Ms. Krofah, how can ARPA-H prevent repeating the mistakes of
the past, and fund a diverse pool of researchers working across bio-
medical and community-based settings?
Ms. KROFAH. Well, thank you so much for your question. It is a
problem. It is exactly a problem, just as you have stated. And what
we need to do is to make sure that we don’t repeat the mistakes
of the past, which is a nice to have, but not a must have.
What we typically say is, ‘‘Write a community action plan, and
send us your ideas of how you are going to do outreach,’’ but there
is no accountability in the end.
I think we have the opportunity to take all of the learnings that
we have gone through to really put that into place with ARPA-H.
We should have targets. We should say, ‘‘What are the diseases
that are affecting the most number of people that are bearing the
highest burden of disease, and how do we innovate there?’’
We also need to make sure that we are hiring the right program
managers from these diverse perspectives, and we need to set some
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targets, and we need to have a patient advisory council that is di-
verse. I think those are issues that we just should not shortchange.
We should not broaden into language that is not specific enough
that we do nothing about. So I completely agree with you. I think
we can write that in to make sure it happens.
Ms. KELLY. So would you consider those to be metrics that would
be used to determine if ARPA-H is funding a diverse pool of sci-
entists, or are there other things you would suggest?
Ms. KROFAH. I do think that we need to make sure that we have
metrics. Again, we do want to make sure that ARPA-H has the
flexibility to pursue the science and the innovation, wherever that
lies—of course, understanding that there is a public health need at
the end of the day.
Outlining a strategic plan that includes metrics and it is trans-
parent—what diseases, and why, for whom, to what benefit, and
who are we bringing along—during those research studies needs to
be clearly outlined.
And that also needs to occur in partnership with FDA, because
a regulatory approval process happens there.
Ms. KELLY. As you know, some of the most successful and inno-
vative interventions to improve health come from within impacted
communities. For example, community health centers, hyperlocal
healthcare hubs serving marginalized patients are often the epi-
center of new ideas and cutting-edge innovation, from community-
based emergency departments to trauma-informed behavioral
healthcare.
How do you envision ARPA-H partnering with local communities
to buildupon the wealth of community-based knowledge that really
already exists?
Ms. KROFAH. I think the program managers need to go out, and
not rely on the communities to come in. We have these models. We
use mobile clinics to go out into the communities. We need to do
listening sessions. We need to take the managers and those mes-
sages out, wherever they are.
We can leverage platforms that were used to communicate
around COVID–19 vaccination, turn those infrastructure into op-
portunities to listen to the communities.
But if we are going to stay in ivory towers with program man-
agers who are all located centrally, and expecting those diverse
communities to come to them, that just will not happen. So I do
agree with you——
Ms. KELLY. Yes——
Ms. KROFAH [continuing]. And I do think those messages need to
be taken out on the road, and to have those listening sessions with
broad and diverse communities.
Dr. GIROIR. Ma’am, if you will, at DARPA my program managers
in my office needed to be in the office two afternoons per month.
I want to reinforce how important it is to get out into the commu-
nities. They are not going to be cooped up, they are not going to
be in an ivory tower. They can’t be behind walls. They have to go
interact.
And the second point—and this is so important—is if you have
an NIH system that ranks proposals 1 to 100, and only the top five
get picked, you are going to lose all the diversity, because they are
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not in the mainstream. They don’t have the grantsmanship offices
that Harvard or Yale or Hopkins do.
The whole goal of DARPA in the review process is maybe to take
number one and three, but to pick that diverse approach, number
25 that is not from, you know, Harvard. And I am just using that
stereotypically, but it might be from a community health center, or
it might be from an HBCU. They would never make it to a tradi-
tional 1-to-end review process, but having that—diversity is a
means to success. It is a goal in and of itself, but more than any-
thing it is a means to success, particularly in the health realm.
Ms. KELLY. Well, thank you both so much. Thank you for your
patience. Thank you for being witnesses, and I yield back.
Thank you, Madam Chair.
Ms. ESHOO. You are most welcome. I am glad we could accommo-
date you.
Ms. KELLY. Thank you.
Ms. ESHOO. All right. I think that—well, we are going to go over
to vote. We will recess, and we are going to try to be back here by
2:20. All right?
And then, I don’t know, I know I have two members from our
side of the aisle, and a third that wishes to waive on. But we will
make that determination when we come back.
Mr. GUTHRIE. Somebody can——
Ms. ESHOO. OK. Well, I am not—no one is going to be penalized
if they come back.
Mr. GUTHRIE. I know——
Ms. ESHOO. They should be heard.
OK, the committee will recess until——
[Recess.]
Ms. ESHOO. The Subcommittee on Health will now come back to
order.
The Chair is pleased to recognize the gentlewoman from Cali-
fornia, Ms. Barragán, for your 5 minutes of questions.
Ms. BARRAGÁN. Thank you, Madam Chairwoman, for hosting this
important hearing today. This bold proposal to create a new agency
exclusively tasked to drive medical breakthroughs is so important
because, among the 9,000 or so known human diseases, there are
FDA-approved treatments for only about 500 of them.
To date, more than 146 drugs have been tested for Alzheimer’s
disease and rejected. While the first new drug for Alzheimer’s in
nearly 20 years was approved—recently approved by FDA, there is
still a long way to go to truly treat and prevent the progression of
Alzheimer’s disease. ARPA-H has the opportunity to play a critical
role in revolutionizing how we prevent, treat, and cure a range of
diseases with unmet medical needs, like Alzheimer’s, which affects
millions of families across the country, including my own.
My first question is for you, Dr. Yamamoto. I am interested in
how ARPA-H could help accelerate the discovery of Alzheimer’s bio-
markers as a means of tracking responses to potential Alzheimer’s
therapies and exploring the use of digital technologies for diag-
nosis, assessment, and disease monitoring, among other important
research initiatives. Can you discuss how Congress can ensure
there is transparency around ARPA activities, including data shar-
ing and the open resources development of data and information?
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Dr. YAMAMOTO. Thank you for that question, and let me start by
saying that my dad died of Alzheimer’s, so I know something about
that from—at a very, very personal level.
Everyone knows that early diagnosis is probably the clearest
route to being able to cure diseases, and neurological diseases are
kind of the king of the failure to be able to diagnose early. My dad
died when he was 84. In retrospect, I can think back to not quite
a decade before that, but a number of—let’s say, eight years before,
when I could then, in retrospect, begin to think, oh yes, there was
something going on, my dad. But we didn’t know that.
But, in fact, right, there are things that are going on in the brain
when the baby is born, or maybe before, right? We just don’t know
what they are. So the impact of being able to diagnose neurological
disease early could go back to decades, seven decades. And think
of the impact that we could have in being able to cure or prevent
those diseases, if we knew what those early markers were. I think
that ARPA has the capability—can develop the capabilities to be
able to achieve such early diagnoses.
As we said before, when—what precision medicine does is collect
enormous amounts of data about lots of people and experimental
organisms, to be frank, that allow us to be begin to pool that infor-
mation, and begin to perceive biomarkers, indications of what the
diseases are. So I can easily see an ARPA undertaking a project
where at least the access to information to be able to establish
those early markers is there to say that they have—a project man-
ager could walk in and say, ‘‘I think the goal is that we want to
diagnose Alzheimer’s 15 years earlier than we can right now.’’
Ms. BARRAGÁN. Thank you, Dr.——
Dr. YAMAMOTO. I think we are beginning to pull together the
data to be able to do that.
Ms. BARRAGÁN. Thank you.
Dr. Ling, part of the proposed mission of ARPA-H is to make piv-
otal investments in breakthrough technologies and broadly applica-
ble platforms, capabilities, resources, and solutions that have the
potentially—potential to transform important areas of medicine
and health. This is a broad mission.
But what do you see as some of the greatest unmet needs facing
human health, and how could ARPA-H help solve some of these
unmet needs?
Dr. LING. Thank you, Congresswoman. I think that Dr.
Yamamoto hit it right on the head.
One of the really ripe areas where a good period of investment
could really make a big difference is the early diagnostics. And it
is just not finding the widget that is going to identify the blood
marker. It is also the analytical techniques that Dr. Yamamoto
speaks of.
What does that mean? It means bringing in mathematicians and
statisticians and physicists, potentially, into this discussion. It
can’t just be biologists and physicians. That is what an ARPA-H
does. It brings in the people who are not commonly in the eco-
system into the ecosystem, bringing the chemists, bringing the en-
tomologists, bringing the marine biologists, bringing—honestly no
joke—the meteorologist, who looks at data at a large ecosystem-
level, with the techniques that they do, to do something straight-
113
forward as weather. Apply those to the data sets that we are talk-
ing about right now.
You don’t have to rediscover the wheel; you have to readapt the
wheel. That is what an ARPA-H could do. It could bring in and cre-
ate a new ecosystem, Congresswoman.
Ms. BARRAGÁN. Well, thank you all for your responses. I didn’t
get to any of my other questions, but hopefully I will submit them.
Thank you, Madam Chairwoman, I yield back.
Ms. ESHOO. The gentlewoman yields back and now the Chair is
pleased to recognize the gentlewoman from Delaware, Ms. Blunt
Rochester, for your 5 minutes of questions.
Ms. BLUNT ROCHESTER. Thank you so much, Madam Chair-
woman, and thank you to the witnesses for sharing your testimony
today. I am pleased that we are discussing the Advanced Research
Projects Agency for——
VOICE. Are you ready——
Ms. BLUNT ROCHESTER [continuing]. ARPA-H, which will accel-
erate the development of lifesaving treatments and cures in this
country.
Last week the House passed the America COMPETES Act, a
comprehensive package of—to bolster America’s global economic
competitiveness. And today we turn our attention to ensuring that
the U.S. remains the leader in global biomedical innovation.
I am particularly interested in understanding how the interests
of the American people can be protected, and how preventions and
cures derived from the work of ARPA-H will be distributed equi-
tably and ethically.
One of the goals of ARPA-H is to create platform technologies
upon which others can build and innovate. Admiral Giroir, based
on your previous experience with BARDA, how can the U.S. Gov-
ernment ensure that contractual agreements for the technology
transfer and commercialization of products that use research spon-
sored by ARPA-H reflect the contributions of the American Govern-
ment and the American people?
Dr. GIROIR. Yes, thank you for that question, and it is good to
work with you again.
No. 1, the agency needs the flexible contracting authority to put
that in. One cookie cutter does not fit. And as we heard, again,
other transactions, cooperative agreements, all of those things.
No. 2, I would like to switch the paradigm a little bit. Instead
of making sure that the next thing is distributed equitably, we
ought—the next thing ought to be focused on equitable distribution
as its primary goal, right?
So just as an early diagnostic could be, I could see a program
that says we want to assure in the next five years that all the un-
derserved communities have a projected life expectancy equivalent
to those who are White and affluent. Now that is a big goal, but
that is a DARPA hard goal. That is going to be prevention, pre-
venting kidney disease, preventing hypertension, getting in the
home.
So I think you have to design it from the start, not just do it as
an afterthought.
Ms. BLUNT ROCHESTER. I love that, and it gets to the disparities
issues that we have been talking about for years.
114
What guardrails could be put in place so that technology devel-
oped within the U.S. Government research funding is not exported
for manufacturing overseas?
Dr. GIROIR. You know, that is not going to be necessarily the pur-
view of ARPA, but I think it is very important.
Now, we talk about ARPA-H, you know, that the things that are
going to be developed are going to benefit the world, just like the
vaccines for COVID. America is, literally, saving the world. But we
want to do our best to keep that technology here, so it supports
jobs here, it supports the infrastructure here, that we don’t get it
copied by foreign—you know, overseas, that we don’t get hacked,
and all that gets done away. Again, these are all vital things.
And again, as Geoff said earlier, DARPA has been managing
this. You know, things that are developed now won’t necessarily be
public for 30 years. The technology needs to be protected. I think
we have the model. I think we have to employ the model and adapt
the model, but I think your concerns are incredibly right on target,
ma’am.
Ms. BLUNT ROCHESTER. Thank you. Thank you so much. And I
am going to ask this question of the panel, and I will call you, and
if I don’t get to you, if you could submit it in writing, that would
be great.
And how will we know if ARPA-H is successful, given the lag
time between product development and commercialization?
And if we could, start with Dr. Yamamoto.
Dr. YAMAMOTO. So I think that we want to be able to look at the
nature of the projects that are undertaken, and think about the
scope, the breadth of application that—if the capability is devel-
oped. So we want things that don’t focus on one disease, but things
that will be effective for big clusters of them. I think that would
be one measure that we could begin to apply to examining and
evaluating the projects that ARPA-H comes forward with.
Ms. BLUNT ROCHESTER. Great, thank you.
And Ms. Krofah?
Ms. KROFAH. Yes, I would say understanding the impact on
health outcomes through an ARPA-H program or intervention.
Have we actually seen a difference in a disease or classes of disease
states and, in particular to what Admiral Giroir just mentioned, eq-
uitably, across different populations who suffer disproportionately?
Ms. BLUNT ROCHESTER. Great, thank you.
And Dr. Ling?
[Pause.]
Ms. BLUNT ROCHESTER. Microphone.
Dr. LING. I think that there are metrics that we can use that are
in existence. How many patents is one of them. The second is how
many have transitioned into commercial practice. That is No. 2.
No. 3 is how many have transitioned into clinical use and adopted
by patients.
These are all metrics that have existed. DARPA, for example,
has metrics looking at the dollars that are invested, and the return
on investment in terms of commerce-produced, and it is a 10-to–
1 ratio if you don’t include the internet. If you include the internet,
then it becomes, you know, astronomical.
115
So there are metrics that we can use that at all different levels
that can actually be brought to bear to look at metrics of success—
and failure, I might add.
Ms. BLUNT ROCHESTER. Yes, my time has expired. But Dr. Mil-
ler?
Dr. MILLER. Yes, I think we have to look at measures that are
clinically relevant. So did we change how people practice medicine?
Did we change how consumers access healthcare?
And then I think we also need to measure if that is distributed
fairly and equitably because, frequently, it is not.
Ms. BLUNT ROCHESTER. Great, thank you so much.
Dr.—or Admiral Giroir, we will get back to you later.
And again, thank you so much, Madam Chairwoman, for this im-
portant hearing. I yield back.
Ms. ESHOO. Well, thank for your patience and for participating.
Let’s see who—oh, we have——
The Chair is pleased to recognize the gentlewoman from Massa-
chusetts, Mrs. Trahan, for 5 minutes.
Mrs. TRAHAN. Well, thank you, Madam Chairwoman, and thank
you to the witnesses here today. I am pleased that this sub-
committee is holding important discussions on the establishment of
ARPA-H.
Developing multiple vaccines within one year to effectively pre-
vent serious illness and death from COVID–19 was unprecedented,
and this outcome was made possible by significant Federal invest-
ment and collaboration with the private sector. Seeing what we ac-
complished in such a short time to save lives from COVID–19, it
only makes sense for the numerous research projects out there to
receive that same level of investment and urgency.
Those increased levels of investment and urgency is critical for
people like my dad, who has been living with MS for 25 years, and
who will benefit from the next innovation or lifesaving cure. How-
ever, barriers and gaps exist within the public and private bio-
medical research ecosystem, which can lead to the stalling or fail-
ure of innovative projects.
So to address this research gap, the Biden Administration has
proposed the establishment of ARPA-H, and I am a big fan of the
DARPA model, which ARPA-H will follow, including the
groundbreaking discoveries we all know so well: internet, GPS—we
could go on and on. So I am excited to witness the groundbreaking
discoveries that will come out of ARPA-H.
Dr. Ling, how can we ensure the appropriate resources will be
allocated toward high-potential projects?
Dr. LING. Again, it begins with the program managers and the
construct of the agency itself. If we are following the DARPA
model, which I totally advocate, completely and totally, what it is
is that program managers develop the program that they think is
appropriate.
Let’s say, using Dr. Yamamoto’s case, it is for a new analytical
approach that could be applied broadly across a number of different
diseases. That program manager will then construct the program.
What are going to be the milestones? What, in fact, are going to
be the performers staff? And then they ask for the money.
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So the money isn’t allocated initially. One program maybe gets
$10 million. Another program might get $50 million, and another
program might get $100 million. It is dependent upon resourcing
properly the program, and what the program hopes to achieve. As
I said to you before, the most critical element is proper use of the
dollars. And that is—it is the how. I always said it before, it is
the—how the dollars are spent.
And so what you want to do is be sure to adequately resource
the performers to achieve the goals of the program, as they are out-
lined from the get-go. The one thing you don’t want to give them
is time. Time is the one resource that we are not going to want to
give to the performers. We will give them money, we will give them
people, we will give them equipment, but not time.
Mrs. TRAHAN. I appreciate that.
Ms. Krofah, I am wondering if you could just expound a little bit
on how we can ensure that, you know, profit will not be the main
driver for the development of a device, a treatment, or a tech-
nology, or a cure under ARPA-H.
Ms. KROFAH. Yes, thank you for that question. You know, what
is the most important, as we think about these sets of challenges,
is to go back to that patient perspective. Where are we seeing high
burdens of disease? What are the issues that are affecting that
high burden of disease? What is the role of technology? What is the
role of analytics? What is the role of diagnostics? How do we ad-
dress those sets of issues?
And then work backward from there to really address the science
and the innovation question. Once we do that, that should yield the
results that we are looking for. And that is why we make sure that
we translate that science into what can be accessible for those pa-
tients at the end of the day.
It is not about the money, it is not about the profit motives at
the end, because that is the issue why we are not seeing particular
types of innovation targeted to patients who are suffering on the
ground from diseases that we all know very well that we have not
made a difference in decades and decades.
Mrs. TRAHAN. Great. And then, you know, my colleague from
Delaware asked about metrics, and I think that is on all of our
minds. How do we know if we are going to be successful?
I guess, once established, how long do you expect it to take to
measure the success of the new agency through some of the metrics
that were mentioned?
And I know that the clock is ticking down, so, Ms. Krofah, I will
start with you.
Ms. KROFAH. I think metrics should start from day one. How
quickly does the agency get up and running?
And then we should go and talk about how quickly do the pro-
gram managers get established, and how quickly do those projects
get funded and get started?
And then, of course, what kinds of science yields the outcomes
over time? This could be a year, it could be 5, it could be ten years,
but the metrics need to start on day one.
Mrs. TRAHAN. Great. Well, I appreciate you all being here. I look
forward to working closely with the Administration and the impor-
tant stakeholders as the ARPA-H proposal comes into fruition.
117
Thank you so much, I yield back.
Ms. ESHOO. Thank you. It is always enlightening to hear the
questions that you pose.
Is Mrs. Fletcher with us? Is she poised to question?
Mrs. FLETCHER. Yes, Chairwoman Eshoo.
Ms. ESHOO. Oh, there you are.
Mrs. FLETCHER. Yes.
Ms. ESHOO. OK, I am pleased to recognize you, the gentlewoman
from Texas, Congresswoman Fletcher, 5 minutes.
Mrs. FLETCHER. Thank you so much, Chairwoman Eshoo. Thank
you for allowing me this time to participate in this hearing, and
thank you for holding this hearing on the proposed creation of
ARPA-H. I want to thank all of our witnesses for being here today,
as well. This has been a really interesting and informative hearing.
And I know I have said before on this committee one of the rea-
sons I was so excited to become a member of this subcommittee
was because of its jurisdiction over medical research, and that is
because of the community that I represent in Houston, Texas. I—
my district lies just west of the Texas Medical Center, which is the
largest medical center in the country. It employs more than
300,000 people, many of whom live in my district, including some
of the researchers that have done this incredible research we have
been talking about today.
I know Dr. Schrier was talking a little bit about CAR-T therapies
earlier, and, of course, Dr. Jim Ellison, who won the Nobel Prize
for his cancer research on this very issue lives in my district, and
was my guest at the State of the Union two years ago. So I am just
so proud to represent these incredibly creative, innovative, thought-
ful, and really pioneering researchers in my district.
So I am very supportive of the ARPA-H proposal that is before
us today. I am glad to have co-sponsored both Chairwoman Eshoo’s
bill, the ARPA-H Act, and Congresswoman DeGette’s bill, the
Cures 2.0 Act.
And as we have discussed throughout the hearing, the agency is
modeled on DARPA, which has led to key innovations like GPS and
the internet, things that we now couldn’t imagine living without.
And it could be that ARPA-H leads us to that next medical break-
through that will revolutionize healthcare as we know it.
But there are key differences between DARPA and ARPA-H, and
one of them that is really notable is that DARPA has, one, major
customers that helps set its priorities, right? The Department of
Defense. Whereas, when it comes to the biomedical ecosystem,
there are various players, ranging from scientists to pharma-
ceutical companies to patients that are setting the agenda. And all
these players have a crucial role in developing biomedical solutions
that improve the lives of patients every day.
So I am interested in talking more about and learning about
your thoughts on the role that ARPA-H will play within this larger
system, and I want to direct my first question first to Ms. Krofah.
You discuss how ARPA-H must be engaged with other agencies.
We have talked about that throughout the hearing, engaging with
CMS and FDA. And you know, these things are critical to advanc-
ing solutions for patients. How do you envision ARPA-H working
in a collaborative manner with these other agencies?
118
Ms. KROFAH. Thank you so much for the question. I mentioned
in my written testimony and earlier this morning that I believe it
is critically important that we have an advisory council and en-
gagement with patients, engagement with industry, engagement
with non-profits, engagement with academia to listen.
And of course, we need to listen throughout the country. We
don’t need to sit in the ivory tower, as I mentioned earlier before.
I think that would be critically important to understand the kinds
of issues that are motivating people to ask questions: What is hold-
ing back science for me, and why? And to bring forward their solu-
tions to inform the program managers as they outline the proposals
that they put forward to the ARPA-H director.
Mrs. FLETCHER. Terrific, thank you. You also mentioned in your
testimony that the private sector will be an important partner for
ARPA-H. Can you discuss—and I know we have touched on it
throughout the day, but with the kind of minute-and-a-half I have
left, there varying opinions here, and we have heard some of our
colleagues on the other side of the aisle really want to defer more
to the private sector. But can you discuss why it is so important
for the private sector and other stakeholders to help work together
to ensure that the project that ARPA-H supports ultimately lead to
improvements in health for patients?
Ms. KROFAH. Yes, absolutely. The biomedical innovation is an
ecosystem. It is not one actor. And each part of the ecosystem mat-
ters to all the other parts.
So when we talk about the totality that end-to-end solution set,
as was outlined by some colleagues, we need R&D upstream to be
connected downstream to where manufacturing occurs and, impor-
tantly, where those products end up going to patients. And that is
really the role of the private sector. That last mile is an area where
the private sector has significantly innovated the manufacturing
capacity that we see.
Even when we got the COVID–19 vaccines over the finish line,
we needed vast manufacturing capacity and capabilities in order
for those vaccines to reach patients. And then the delivery points,
right, the retail sites, the mobile clinics, outreach to the community
settings. These are all the roles that we need to engage the private
sector.
But also, importantly, they need to bring their expertise to bear.
Many of these companies are also investing in different types of
technologies. They can identify where the gaps are. Data analytics
is a big gap. Our data ecosystem is quite siloed. We do not talk
well to each other. If you are in one hospital setting, your data is
not carried over to another hospital setting. If you are in one phar-
macy, your data is not carried over to the other pharmacy.
How do we break down these barriers in data silos? The private
sector can come forward and say, ‘‘We may not be able to solve it
as one company,’’ but we can put forward a project like ARPA-H,
where it can break down those issues and those barriers.
Mrs. FLETCHER. That is terrific. Well, thank you so much. I see
I have gone over my time, so I just want to thank you for that ex-
planation, and really reminding us all about the ecosystem, and
how we can accomplish so much when everyone works together.
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So Chairwoman Eshoo, thank you so much for bringing us to-
gether for this hearing, and I yield back.
Ms. ESHOO. I thank the gentlewoman, and you should know that
this big screen carries your beautiful face and voice. It is exciting,
you know? We really get—with the screen you get a real close-up.
[Laughter.]
Ms. ESHOO. I am glad it is not of me, but it is wonderful to see
colleagues on the big screen.
Now we welcome—we are grateful to Congresswoman Diana
DeGette. She is not a member of our subcommittee, but she is
waiving on, an important member of the Full Energy and Com-
merce Committee and one of the authors of Cures 1.0, now with
2.0.
And I want to reState my intent that ARPA-H and Cures 2.0,
they are complementary, and the Chair will seek to move them to-
gether so that we could advance the legislation not only through
the full committee, but the full House of Representatives, as it is
so important that we do.
And with that, welcome, Diana, and you have 5 minutes.
Ms. DEGETTE. Thank you so much——
Ms. ESHOO. And if you go over a little, it is OK, we are just about
done.
[Laughter.]
Ms. DEGETTE. OK, thank you. Thank you so much——
Ms. ESHOO. You have waited all day.
Ms. DEGETTE. I will just be happy to bat clean-up. Thank you
so much, Madam Chair. Thanks for letting me waive on, although
I do consider myself to be sort of an adjunct member of this sub-
committee, anyway. And I want to thank you for your commitment
to working with me and Congressman Upton on both this impor-
tant initiative, ARPA-H, and also Cures 2.0, which really, really
have synchronicity and need to go together.
When Fred and I first teamed up in 2015 to draft the 21st Cen-
tury Cures bill, we couldn’t have imagined the incredible success
it would have for this country. And because of that Act, we have
a better understanding of the human brain. We have made huge
strides in regenerative medicine. We have increased funding for
Alzheimer’s research and cancer research. Congresswoman Trahan
talked about Operation Warp Speed. Many people don’t think we
would have been able to get the vaccine that we did without the
pathways that we had in 21st Century Cures. And, as Fred likes
to say, it passed out of this committee 57 to 0. And of course,
through the House and Senate.
And so I want to talk both about ARPA-H and Cures 2.0 today
to hear how they can work together to have even more stunning
advances in U.S. biomedical research. I would like to start with
you, Ms. Krofah.
What are pathways and programs that were included in 21st
Century Cures—or why are they, like the Moonshot and the Break-
through Devices program, critical to the continued success of tech-
nological innovation?
Ms. KROFAH. Well, thank you so much, Congresswoman DeGette,
and I very much applaud the efforts for Cures 2.0, and certainly
your commitment to 21st Century Cures 1.0 that passed a number
120
of years ago that has had a tremendous impact on the biomedical
innovation ecosystem.
You know, I will start by saying the role that patients have in
providing their data toward medical research, contributing their
data into the ecosystem, was supported through 21st Century
Cures, and continues to be continue on the landscape. At the same
time, seeking opportunity for breakthrough therapies and break-
through designations has allowed us to see many new therapies
reach the market to treat conditions like cancer, and many dif-
ferent types of cancer we would not have seen, otherwise.
And as we think about ARPA-H and the potential to merge the
proposals that are in Cures 2.0, with an ARPA-H model, what it
reminds me of is that close collaboration that is needed between
ARPA-H and FDA, similar to what is in Cures 2.0, in terms of en-
couraging communication between FDA and—being able to bring
that regulatory science up front at the time of determining the pro-
gram or project is and creating that regulatory throughout that
process will create clarity through the breakthrough process, will
enable us, ultimately, to get the product create ARPA-H into prod-
ucts that are approved by FDA and, ultimately, to patients.
Ms. DEGETTE. Great. So what you are really saying is you see
Cures 2.0 and ARPA-H as complementary, not substitutes for each
other. Is that correct?
Ms. KROFAH. That is absolutely correct.
Ms. DEGETTE. And I—Dr. Yamamoto, I see you nodding your
head yes. Do you agree?
Dr. YAMAMOTO. Absolutely, I agree. And I think the other—the
thing I would add to what you just heard is that I think that—I
said earlier that there is nothing in the Federal Government that
incentivizes agencies with different focused missions to really co-
operate and work together. And in many ways, Cures 2.0—and
Cures 1.0, but also Cures 2.0—begins to do that. It doesn’t——
Ms. DEGETTE. Thank you.
Dr. YAMAMOTO. It doesn’t create the natural drive for collabora-
tion that ARPA-H does, because that is what the—the only—the
program managers know that the only way they are going to get
there is to bring together different groups.
Ms. DEGETTE. Right.
Dr. YAMAMOTO. But——
Ms. DEGETTE. And Dr. Ling, I saw—I also saw you nodding your
head. Do you agree with that?
Dr. LING. I fully agree with that. The ultimate goal here is to im-
prove healthcare for everybody in this country, for every American
citizen. And to do that you have to attack the entire problem in
toto. ARPA-H is just a small piece. Cures 1.0, Cures 2.0, in fact,
actually embrace much more of the health reform, the policy issues,
working with FDA, and a number of other things that are abso-
lutely essential to realizing this ultimate goal that we have.
Ms. DEGETTE. And Admiral Giroir, I can’t see you, but I am hop-
ing you were nodding your head, too. What is your view on that?
Dr. GIROIR. I actually could not agree more with you. These are
not only complementary, but synergistic. They can’t live without
each other.
121
And I will just make one comment, as I do believe a significant
minority of program managers at ARPA-H come from FDA or NIH,
because they will have been frustrated at things they couldn’t get
done within their own agency, will come to ARPA to get it done,
and then return richer and the country better for it.
Ms. DEGETTE. Great, thank you. Well, thank you to all of you for
your leadership. I appreciate it.
Madam Chair, I just would like to take a moment of personal
privilege on something else. One of our colleagues was attacking
Dr. Fauci earlier, and calling him names.
And I want to say that, for Dr. Fauci, for all of our research sci-
entists and our public servants who have worked hard to get us
through this pandemic, including all of you, I want to say I appre-
ciate what you are doing. I appreciate the advice and the science
that you are relying on. And I want to apologize on behalf of the
U.S. Congress that many of you have had to get security details be-
cause you have been under the threat of violence and worse. So
thank you. Thank you to all of our advisors and scientists for get-
ting through this. We will continue to do that.
And I yield back.
Ms. ESHOO. The gentlewoman yields back. I thank her for her—
for not only her questions, but for—to recognize the extraordinary
service of people: Dr. Fauci, so many others.
You know, I often think that those that are in the public sector,
like he is, like so many are, they could be making millions of dol-
lars a year in the private sector. Millions, tens of millions a year,
a year. He devoted an entire lifetime to serve the American people.
And you know, whether we agree with each other or not, to—that
honorable servants of the people are attacked, that really has no
place. That is not America. That just isn’t America. So thank you
for raising that.
I want to thank all the witnesses. I love hearings. I have to
admit that. So if I sit here for 7 hours, the only regret is that my—
or ranking member that I am dragging along with me, although he
is a very, very attentive member—you have, I think, filled this
room, whether you were—are with us virtually or in person, with
very, very rich testimony, and very direct answers to our direct
questions.
And so you have enlarged the issue that is before us. And your
expertise, I think, whomever is listening in at home, that they
would really be applauding the experts that we have in our coun-
try. And that you have come forward to advise the Congress is your
gift to our country, and you have added immeasurably to our think-
ing today and helped us to improve the legislative vehicle that is
before us. So on behalf of all of the members of the subcommittee,
we all thank you and applaud you.
Now I need to get a—I request unanimous consent to enter the
following documents into the record. Do you want me to read them?
Mr. GUTHRIE. No, no. No objection.
Ms. ESHOO. Oh, no objection? So ordered.
[The information appears at the conclusion of the hearing.]
Ms. ESHOO. They will be entered into the record.
That—our panel has completed their work today. And I think
that you all deserve at least a martini this evening before dinner.
122
And Members have ten business days to submit additional ques-
tions for the record. So, to the witnesses, when we get those to you,
please answer as promptly as you can to any of the questions that
you receive. That is—it is not just appreciated, but it is an impor-
tant weighing in, and we always want members’ questions to be
answered.
So with great gratitude, at this time the subcommittee is ad-
journed.
[Whereupon, at 2:58 p.m., the subcommittee was adjourned.]
[Material submitted for inclusion in the record follows:]
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