[House Hearing, 115 Congress]
[From the U.S. Government Publishing Office]











                  DEPARTMENTS OF LABOR, HEALTH AND HUMAN
                SERVICES, EDUCATION, AND RELATED AGENCIES
                         APPROPRIATIONS FOR 2018
_______________________________________________________________________

                                 HEARINGS

                                 BEFORE A

                           SUBCOMMITTEE OF THE

                       COMMITTEE ON APPROPRIATIONS

                         HOUSE OF REPRESENTATIVES

                      ONE HUNDRED FIFTEENTH CONGRESS

                              FIRST SESSION

                                  _______

    SUBCOMMITTEE ON LABOR, HEALTH AND HUMAN SERVICES, EDUCATION, AND 
                            RELATED AGENCIES

                       TOM COLE, Oklahoma, Chairman

  MICHAEL K. SIMPSON, Idaho             ROSA L. DeLAURO, Connecticut
  STEVE WOMACK, Arkansas                LUCILLE ROYBAL-ALLARD, California
  CHARLES J. FLEISCHMANN, Tennessee     BARBARA LEE, California
  ANDY HARRIS, Maryland                 MARK POCAN, Wisconsin
  MARTHA ROBY, Alabama                  KATHERINE CLARK, Massachusetts
  JAIME HERRERA BEUTLER, Washington
  JOHN R. MOOLENAAR, Michigan

  NOTE: Under committee rules, Mr. Frelinghuysen, as chairman of the 
full committee, and Mrs. Lowey, as ranking minority member of the full 
committee, are authorized to sit as members of all subcommittees.

                        Susan Ross, Jennifer Cama,
              Justin Gibbons, Kathryn Salmon, and Lori Bias
                            Subcommittee Staff

                                 ________

                                  PART 8

                                                                            Page
Role of Facilities and Administrative Costs in Supporting NIH-
  Funded Research......................................................        1
Down Syndrome: Update on the State of the Science and Potential 
  Discoveries Across Other Major Diseases..............................       61
                                        
[GRAPHIC(S) NOT AVAILABLE IN TIFF FORMAT]


                                 _______

          Printed for the use of the Committee on Appropriations
                                 _______

                   U.S. GOVERNMENT PUBLISHING OFFICE

27-444                WASHINGTON: 2017
                   








                      COMMITTEE ON APPROPRIATIONS

                                ----------                              
             RODNEY P. FRELINGHUYSEN, New Jersey, Chairman


  HAROLD ROGERS, Kentucky \1\           NITA M. LOWEY, New York
  ROBERT B. ADERHOLT, Alabama           MARCY KAPTUR, Ohio
  KAY GRANGER, Texas                    PETER J. VISCLOSKY, Indiana
  MICHAEL K. SIMPSON, Idaho             JOSE E. SERRANO, New York
  JOHN ABNEY CULBERSON, Texas           ROSA L. DeLAURO, Connecticut
  JOHN R. CARTER, Texas                 DAVID E. PRICE, North Carolina
  KEN CALVERT, California               LUCILLE ROYBAL-ALLARD, California
  TOM COLE, Oklahoma                    SANFORD D. BISHOP, Jr., Georgia
  MARIO DIAZ-BALART, Florida            BARBARA LEE, California
  CHARLES W. DENT, Pennsylvania         BETTY McCOLLUM, Minnesota
  TOM GRAVES, Georgia                   TIM RYAN, Ohio
  KEVIN YODER, Kansas                   C. A. DUTCH RUPPERSBERGER, Maryland
  STEVE WOMACK, Arkansas                DEBBIE WASSERMAN SCHULTZ, Florida
  JEFF FORTENBERRY, Nebraska            HENRY CUELLAR, Texas
  THOMAS J. ROONEY, Florida             CHELLIE PINGREE, Maine
  CHARLES J. FLEISCHMANN, Tennessee     MIKE QUIGLEY, Illinois
  JAIME HERRERA BEUTLER, Washington     DEREK KILMER, Washington
  DAVID P. JOYCE, Ohio                  MATT CARTWRIGHT, Pennsylvania
  DAVID G. VALADAO, California          GRACE MENG, New York
  ANDY HARRIS, Maryland                 MARK POCAN, Wisconsin
  MARTHA ROBY, Alabama                  KATHERINE M. CLARK, Massachusetts
  MARK E. AMODEI, Nevada                PETE AGUILAR, California
  CHRIS STEWART, Utah
  DAVID YOUNG, Iowa
  EVAN H. JENKINS, West Virginia
  STEVEN M. PALAZZO, Mississippi
  DAN NEWHOUSE, Washington
  JOHN R. MOOLENAAR, Michigan
  SCOTT TAYLOR, Virginia
  ----------
  \1\ Chairman Emeritus

                   Nancy Fox, Clerk and Staff Director

                                   (ii)
 
DEPARTMENTS OF LABOR, HEALTH AND HUMAN SERVICES, EDUCATION, AND RELATED 
                    AGENCIES APPROPRIATIONS FOR 2018

                              ----------                     

                                         Tuesday, October 24, 2017.

   THE ROLE OF FACILITIES AND ADMINISTRATIVE COSTS IN SUPPORTING NIH-
                            FUNDED RESEARCH

                               WITNESSES

KELVIN DROEGEMEIER, VICE PRESIDENT FOR RESEARCH, UNIVERSITY OF OKLAHOMA
KEITH YAMAMOTO, VICE CHANCELLOR FOR SCIENCE POLICY AND STRATEGY, 
    UNIVERSITY OF CALIFORNIA, SAN FRANCISCO
BRUCE T. LIANG, M.D., DEAN, UNIVERSITY OF CONNECTICUT SCHOOL OF 
    MEDICINE
GARY GILLILAND, M.D., PRESIDENT AND DIRECTOR, FRED HUTCHINSON CANCER 
    RESEARCH CENTER
    Mr. Cole. Good morning. It is my pleasure to welcome all of 
you to the Subcommittee on Labor, Health and Human Services, 
and Education for a hearing to discuss the role of facilities 
and administrative costs, sometimes referred to as F&A or 
indirect costs, in supporting NIH-funded research at 
universities and independent research institutions across the 
country.
    Since World War II, the Federal Government and research 
institutions have partnered in a cost-sharing relationship that 
has led to discoveries that have cured diseases and improved 
the quality of life for people around the world. That 
partnership made possible the creation of a biomedical research 
enterprise that has made the U.S. the leader in innovation and 
economic competitiveness.
    We all acknowledge this cost-sharing relationship is valid 
and necessary to maintaining U.S. leadership in research and 
development. But, as stewards of taxpayer dollars, it is our 
responsibility to examine whether the system, as currently 
designed, still works to fairly and efficiently support the 
cost of doing critical research, given the increasing 
complexity of biomedical research today.
    I have expressed great concern with the administration's 
proposal in the fiscal year 2018 budget to cap NIH indirect-
cost reimbursement at 10 percent of total research costs, a 
large reduction from the 28 percent spent by NIH in fiscal year 
2017. Based on many discussions with researchers, 
administrators, and other research funders, both in Oklahoma 
and across the country, I am very concerned that the proposed 
F&A rate cut would drastically reduce the amount and quality of 
research conducted in the United States and that public 
universities would be particularly hard hit.
    We are here today to examine the current policy and 
determine whether there are ways we can make it better, as well 
as whether we can take steps to reduce the administrative 
burden imposed by the Federal Government on research 
institutions and scientists so that researchers can spend more 
time doing research and less time doing paperwork.
    We are fortunate to have four outstanding witnesses from 
universities and independent research institutions to help us 
understand these issues today.
    First, Dr. Kelvin Droegemeier is the vice president of 
research, regents' professor of meteorology, Weathernews Chair 
Emeritus, and Teigen Presidential Professor at the University 
of Oklahoma. He is serving his second 6-year term on the 
National Science Board, the last 4 years of which he served as 
Vice Chairman, and was appointed this year by Governor Mary 
Fallin as Oklahoma's fourth Secretary of Science and 
Technology, and, for the record, my very good friend and very 
wise counselor.
    Dr. Keith Yamamoto is a professor of cellular and molecular 
pharmacology at the University of California, San Francisco, 
and is UCSF's first vice chancellor for science, policy, and 
strategy. He chairs the Coalition for Life Sciences and sits on 
the National Research Council Governing Board Executive 
Committee and serves as vice chair of the National Academy of 
Medicine's Executive Committee and Council.
    Dr. Bruce Liang is an internationally recognized 
cardiovascular physician/scientist and serves as Dean of the 
University of Connecticut School of Medicine. He also serves as 
chief of UConn's Division of Cardiology and is director of the 
Pat and Jim Calhoun Cardiology Center at UConn Health.
    Finally, Dr. Gary Gilliland is the president and director 
of the Fred Hutchinson Cancer Research Center. Dr. Gilliland 
has worked as a researcher and clinician, specializing in blood 
cancers like leukemia.
    As a reminder to everyone, we will abide by the 5-minute 
rule so that we will be able to keep closely to the schedule we 
outlined for members and participants.
    Before we begin, I would like to turn to my ranking member, 
the gentlelady from Connecticut, for any remarks she cares to 
make.
    Ms. DeLauro. Thank you very much, Mr. Chairman.
    And let me welcome our expert panel this morning. And what 
a distinguished group of doctors, all. But I want to point out 
that UConn is not only for known for its great basketball team 
but for its outstanding scholarship, as well.
    Welcome, Dr. Liang.
    I want to say a thank you to all of you for being here 
today to share your perspective on indirect costs associated 
with biomedical research. The talent on your side of the table 
cannot be overstated, and the research at your institutions 
represents the power to do so much good for more people than 
almost anything else within the purview of government.
    The issue of indirect costs gets to the heart of the 
Federal Government's role in basic research. As a community, we 
have determined that basic research should be fully supported 
by the Federal Government, that the government should pay for 
the true cost of research, which includes both direct and 
indirect costs. Both components are necessary to fund world-
class research programs like the ones that are represented by 
our panelists this morning. I should note that this has been a 
guiding principle behind Federal funding for research for 
decades.
    Why do we, as a government, commit to funding the true 
costs of research? Because basic research benefits the common 
good. It creates knowledge, improves our understanding of 
biology, health, and medicine, and advances treatments and 
cures for many of the diseases that affect millions of people. 
It improves our quality of life. It saves lives. And, as a 
survivor of ovarian cancer, this is very personal to me.
    Basic research is the building block of scientific 
knowledge. We all know that. The private sector will step in 
once research can be translated into commercially viable 
products. However, if it is not for the federally funded basic 
research, we would never reach that point.
    The term ``indirect costs'' is a bit of a misnomer. 
Indirect costs include research facilities' daily operations 
and maintenance and program administration, to name a few. In 
short, indirect costs are necessary to support world-class 
research.
    Unfortunately--well, let me just say, as Tony DeCrappeo, 
president of the Council on Government Relations, observed, 
quote, ``If all you are concerned about is the direct costs, it 
won't take long for your facilities to deteriorate. You can't 
do research on the quad.''
    Unfortunately--and the Chairman alluded to this--the 
administration thinks that research can be done on the cheap, 
and the budget shortsightedly proposed a cut of $7,500,000,000 
below NIH's current funding level. And once there was a 
realization that the proposal to cut NIH research was 
unpopular, it is my view that there was a surge for 
justification for a proposed cut, and that worked its way back 
to settling on indirect costs.
    And, again, in my view, I believe that the administration's 
rigid cap of 10 percent for indirect costs would have a 
chilling impact on research. My colleagues on the committee 
have heard me say it many times, but it bears repeating: You 
cannot do more with less. You can only do less with less.
    So I reject the administration's proposal to reduce our 
investment in NIH research. I appreciate Chairman Cole's 
efforts to work in a bipartisan fashion to include language in 
the House bill and in the short-term CR to block this 
shortsighted proposal.
    Now, I understand that there are concerns, including 
concerns from some NIH-funded researchers, about the share of 
research dollars that are allocated to facilities, 
administration, and related costs. So I am eager to hear from 
our panel as you address these often-heard critiques.
    I will take one second to--it is going to be a few more 
seconds--to remind us that, while the NIH is now funded at 
$34,100,000,000 thanks to two consecutive $2,000,000,000 
increases, funding has not kept pace with the rising costs of 
biomedical research. In fact, NIH's budget has declined by 
nearly $6,500,000,000 since 2003 when you adjust for inflation.
    Sixteen years ago, NIH funded about one in three 
meritorious research proposals. Today, the rate has fallen to 
about one in five. Thereby we miss opportunities to work toward 
cures for life-altering diseases that affect far too many 
people.
    The unfunded grants translate to medical discoveries not 
being made and lives not being saved. We choose to hamper our 
progress as a Nation. I wonder, if you add a cap to indirect 
costs on top of this decline in opportunity, how does that 
impact your ability to carry on?
    I have introduced the bipartisan Accelerating Biomedical 
Research Act, which would reverse the devastating funding cuts 
to the NIH and provide stable, predictable growth for years to 
come. It would untie the hands of the committee, allow us to go 
above the caps--the same mechanism that we use for healthcare 
fraud and abuse. This would set us, in my view, on a path to 
doubling the NIH budget.
    I am hopeful that this year we will be able to provide 
another increase in $2,000,000,000 for NIH research--I know the 
chairman feels absolutely the same way--if we have a bipartisan 
agreement to raise the discretionary funding cap.
    Thank you again to our panel. We look forward to hearing 
from each of you and getting a better understanding of the true 
cost of basic research, including both direct and indirect 
costs.
    Thank you.
    Mr. Cole. I thank the gentlelady.
    And we are privileged this morning to be joined by the 
ranking member of the full committee. And, of course, in that 
capacity, she is a member of every subcommittee, but I know we 
are her favorite because she is always here.
    So I want to recognize the gentlelady from New York for any 
remarks she cares to make.
    Mrs. Lowey. Well, I would like to thank Chairman Cole and 
Ranking Member DeLauro. And because I always want to be here, I 
will be very brief. And I apologize to the panelists in advance 
that I cannot stay, but I certainly support everything that is 
in your briefs, and I thank you for submitting them.
    And I really do appreciate, Mr. Chairman, that we have such 
distinguished panelists to share their views with us this 
morning.
    I was extremely troubled, which is the understatement, by 
the administration's budget request to not only make a dramatic 
$7,500,000,000 cut to the NIH but, in particular, to slash 
funding for research facilities that host NIH grantees and pay 
for things as basic as keeping the lights on.
    Universities, hospitals, and other research centers in my 
district and throughout the country have appropriately raised 
alarms that this cut would significantly harm an institution's 
ability to fund biomedical research. We cannot afford to let 
that happen.
    While I do not share the chairman's support for the fiscal 
year 2018 appropriations bill--I should say, his public support 
for the fiscal year 2018--I don't want to get the chairman into 
difficulty here.
    Mr. Cole. Really.
    Ms. DeLauro. We have been careful.
    Mrs. Lowey. I truly recognize his work and the work of all 
those on this subcommittee to increase funding at the NIH by 
$1,100,000,000 and include language prohibiting HHS from 
changing the rates on facilities and administrative costs.
    If the HHS decides to move unilaterally to cap funds to 
research facilities, it would be catastrophic for biomedical 
research in our country, would prevent future scientists from 
obtaining NIH grants. A unilateral move by HHS would also be 
alarming, as it directly opposes congressional intent.
    So today's hearing will be helpful in discussing what 
reforms may and may not be needed. And, hopefully, your message 
will provide the administration with the knowledge necessary to 
make a more informed budget request next year.
    So thank you very much, Mr. Chairman.
    Mr. Cole. Always a pleasure to have the gentlelady.
    Before I turn it over to the first of our witnesses, at my 
request, Dr. Droegemeier has provided a comprehensive and 
informative paper on the history of the Federal Government's 
role in partnering with universities to support research and 
the role of F&A cost reimbursement in supporting this research.
    I think this paper is really exceptional, quite frankly. 
Just thank you. It brings a lot of things together in a single 
place. And I would highly recommend it as a resource for our 
members and would ask unanimous consent that we be able to 
enter it in full in the record.
    Ms. DeLauro. Absolutely.
    Mr. Cole. Without objection.
    Mr. Cole. Okay. Now I would like to recognize the first of 
our four witnesses.
    Dr. Droegemeier, you are up.
    Mr. Droegemeier. Thank you very much, Chairman Cole, 
Ranking Member DeLauro, and members of the subcommittee. It is 
my privilege to testify before you today. I am testifying as an 
academic researcher, an administrator, a teacher, and also an 
adviser on matters of science and technology policy.
    I just wish to make four brief points today in my 
testimony.
    The first point is that, to understand any proposed changes 
in F&A, we really need to start not with budgets but, rather, 
with a history of how the current research enterprise came to 
be.
    In the 1930s, virtually all research in higher education 
was funded either by philanthropy or by private foundations, 
not by the Federal Government. You may find it surprising that 
most institutions at that time had no interest, in fact, in 
receiving Federal funds for research, owing to fears about 
government intrusion.
    But then, by 1939, President Roosevelt began mobilizing the 
Nation for war. Vannevar Bush, who would go on to write the 
manifesto that led to creation of the National Science 
Foundation, was put in charge by President Roosevelt to fund 
academic institutions, to fund research.
    Taking into account the financial interests and idealogical 
values of universities, Bush established a funding model in 
which indirect costs would be fully reimbursed at a rate of 50 
percent, a flat rate, which is one-half of that being charged 
by industry.
    That moment really marked the beginning of the academic-
government research partnership that we enjoy today. And I 
think we all know how important universities were in our 
victory in World War II.
    Now, from 1950 to 1966, various caps were instituted on 
indirect-cost rates for grants, which halted the previous 
practice of full reimbursement. In 1966, however, those caps 
were removed, but a new requirement of cost-sharing--that is, 
mandatory cost-sharing--was added so that higher education 
would formally assume some of the financial risk. And, also, 
those caps were put in place to help stretch Federal dollars.
    To that point, which is my second point, four specific 
types of cost-sharing are important today and are linked to the 
F&A issue.
    The first form is that mandated by agencies or by States or 
offered voluntarily by institutions. In fiscal 2015, U.S. 
universities paid $1,300,000,000 in cost-sharing.
    The second form of cost-sharing concerns the unrecovery of 
F&A. In fiscal year 2015, U.S. colleges and universities did 
not recover $4,860,000,000, or in other words 30 percent, of 
the F&A they were allowed by law to receive. This under-
recovery results from agencies that limit F&A on certain 
programs, from other organizations that charge no or reduced 
F&A, including State governments. And it also results from a 
26-year-long cap on the administrative component, the ``A'' 
component of F&A.
    The third form of cost-sharing involves the use of 
university funds themselves. In fiscal year 2015, this 
represented 24 percent of all R&D funds expended by higher 
education, or $16,800,000,000. And that number has been growing 
steadily for the past 20 years, which is now really approaching 
a point of unsustainability. This money goes toward funding 
unfunded compliance mandates, which total around 100 today 
compared to just a few 25 years ago.
    The final form of cost-sharing lies in the fact that the 
F&A negotiated rate with the Federal Government is, in fact, 
lower than it should be based upon institutional analyses, 
which is also due in part to the cap that is placed on the A 
rate. At my own campus, the negotiated rate of 55 percent is 
notably less than the 61.6 percent that the rate should 
actually be.
    Also, at my campus, looking at the actual recovery rate 
that we achieve based on money we actually bring in, the F&A 
rate is only 33 percent, which means that my institution puts 
22 cents for every dollar that it brings in on the table to 
bring in that research dollar.
    My third point concerns the growth in F&A. Over the past 10 
years, the average growth has been only 0.8 percent per year 
for research universities, even though administrative costs 
have grown dramatically during this time. For NIH research 
project grants from 1998 through 2014, the ratio of F&A to 
total project costs was nearly constant at slightly more than 
30 percent. And it was constant for nearly 20 years.
    My fourth and final point concerns implications of capping 
the NIH F&A rate at 10 percent. The proposed cap would decrease 
F&A from $4,600,000,000 to roughly $1,900,000,000. If this 
money were removed entirely from the NIH budget, the same 
amount or, possibly, likely, somewhat less research would be 
performed, but--and I really want to stress this point--only 
institutions that have sufficient resources to make up that 
loss in F&A or a substantial fraction of it would be able to 
accept the funding. And, ultimately, eventually, those 
universities also would be unable to make up that loss. The 
U.S. would therefore see diminished involvement in research by 
perhaps hundreds of universities and would have other profound 
implications.
    Now, if, on the other hand, that saved F&A went to the 
direct-cost category, more research would be funded in direct 
costs. But, again, only those institutions having sufficient 
resources would be able to play. Those institutions that have 
resources that aren't sufficient to make up the lost F&A would 
be left out entirely or have greatly diminished activities.
    Addressing today's health challenges requires an all-hands-
on-deck participation of every researcher in every corner of 
the Nation. The risks associated with making what might seem 
like minor changes to the highly successful but tenuous 
academic-government partnership for purposes of financial 
expediency have profound consequences for our Nation and must 
be carefully weighed.
    Thank you very much for the opportunity to comment, and I 
look forward to your questions.
    [The information follows:]
    
    
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    Mr. Cole. Thank you very much.
    Dr. Yamamoto, you are recognized for any comments you care 
to make.
    Mr. Yamamoto. Good morning, Chairman Cole, Ranking Member 
DeLauro, members and staff of this esteemed subcommittee. I am 
Keith Yamamoto, vice chancellor for science policy and strategy 
and an NIH-funded molecular biologist at the University of 
California, San Francisco.
    The topic today, the role of facilities and administrative 
costs, F&A, in supporting NIH-funded research, bears centrally 
on the remarkable compact between the Federal Government and 
the Nation's research enterprise that Dr. Droegemeier referred 
to. This compact was launched over 70 years ago when Vannevar 
Bush urged the Federal Government to greatly expand the crucial 
link it had established with scientists during World War II.
    Dr. Bush addressed science broadly but singled out the, 
quote, ``war on disease,'' close quote, noting that, quote, 
``if we are to maintain progress in medicine, the government 
should extend financial support to basic biomedical research in 
the medical schools and universities.''
    What evolved is a Federal academic cost-sharing grant-in-
aid agreement. By awarding my lab an NIH grant, the government 
helps to pay for my proposed experiments and a portion of 
infrastructure, the F&A, needed for those experiments.
    This partnership aids both government and academia, 
compared especially to another concept that was under 
consideration at the time, which was for the government to 
construct, maintain, and populate a vast network of Federal 
laboratories. And the cost-sharing is genuine. Universities and 
research institutes are the second-largest funders of 
biomedical research, behind only NIH.
    Let me make three quick points about that F&A matter.
    First, the separation of research costs into direct and F&A 
is a rational accounting strategy. Direct funding partially 
covers the experiments in my lab, whereas F&A funding helps to 
support the resources at UCSF, without which those experiments 
could not be done: lights, water, information technology, my 
sophisticated laboratory building, security, staff to ensure 
responsible stewardship of the grant funds and compliance with 
Federal regulations and guidelines and the like. Such costs are 
better accounted for in aggregate, covering all of the NIH-
funded research at UCSF, compared, for example, to installing 
separate water and electricity meters in every laboratory. But, 
like direct costs, F&A costs are essential to the research.
    Second point: The F&A rate determination is rigorous and 
institution-specific--institution-specific--and, thus, very 
complex.
    Funding decisions for direct costs are made by expert 
scientific peer-review panels. Only one application in five is 
funded. F&A support is then awarded as a percentage of direct 
support that is negotiated for each institution, with, in our 
case, the HHS auditors. In addition, there are predefined caps 
on F&A for certain types of grants and for administrative 
costs.
    All of this complexity produces complicated outcomes--
different F&A reimbursement rates for different institutions, 
always well below the actual cost but still very different. And 
with few stakeholders truly understanding the complexities, 
including even some Members of Congress who are strong 
advocates for the NIH and certainly including some faculty, as 
Ms. DeLauro said--I used to be among those who thought, ``Well, 
the F&A costs are just money that I got by winning this grant. 
The money should come to me.''
    Third point: The rate basis for F&A reimbursement differs 
between the Federal Government and various philanthropic 
institutions, so the rates are not directly comparable. For 
example, the Bill & Melinda Gates Foundation counts facilities, 
utilities, and communications as direct costs, whereas NIH does 
not.
    A recent study showed that if a government and 
philanthropic organization's F&A rates were compared on the 
same basis then they would not be different. At UCSF, we have 
done that calculation, so we have recalculated the F&A costs 
using NIH criteria for the $138,000,000 in Gates funding 
received between 2013 and 2017 and found that the rate 
approximates that for Federal grants.
    Like all complicated procedures and policies, the F&A 
reimbursement process is not perfect. It merits periodic 
evaluation. And I might submit to you that a congressionally 
mandated analysis and assessment by the Natural Academy of 
Sciences and the National Academy of Medicine could serve the 
process very well.
    However, draconian cuts or caps would damage research, 
researchers, research outcomes, American health, and American 
competitiveness. If the Federal Government adopted OMB's F&A 
cap, our large enterprise at UCSF would suddenly be short 
$120,000,000, with no way--no way--to make up the deficit. 
Instead, we would have to decide which research and health 
programs, which training programs, which facilities are going 
to be maintained, which would be cut back, which would be 
closed.
    And make no mistake that smaller institutions that are 
actively, vigorously building up their biomedical research 
enterprises--it could lead to total shutdown of those efforts 
that are so vital to their regions and their congressional 
districts.
    Clearly, NIH F&A is an important matter with big 
ramifications, so let me thank the subcommittee for dedicating 
your time to learn about it and evaluate its merits and 
impacts.
    This concludes my testimony. I would be happy to respond to 
your questions.
    [The information follows:]
    
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    Mr. Cole. Thank you very much.
    Dr. Liang, you are recognized now for your opening 
statement.
    Dr. Liang. Chairman Cole, Ranking Member DeLauro, and 
subcommittee members, I thank you for the opportunity to 
testify today before this subcommittee regarding the 
administration proposal to drastically reduce and cap the 
reimbursement of F&A costs to academic research institutions.
    My name is Bruce Liang. I am the dean of the UConn School 
of Medicine and also a practicing cardiologist in Farmington, 
Connecticut.
    I also want to begin by thanking the subcommittee for 
acting on July 13th to soundly reject this attempt to reduce 
the costs associated with cutting-edge medical research and for 
its bold and bipartisan proposal to actually increase the 
Nation's investment in NIH by $1,100,000,000 in fiscal year 
2018.
    A 10-percent cap on the F`A rate would weaken our ability 
to innovate and to develop new medicine and cures, would shut 
down research programs, and would also in Connecticut adversely 
affect biotechnology innovation by UConn, by Jackson Laboratory 
for Genomic Medicine, which is located in Farmington, 
Connecticut, the Mount Sinai Genomic Research Center in 
Branford, Connecticut, and Yale and New Haven's growing 
bioscience businesses, as well as academic institutions such as 
those associated with AAMC, APLU, and AAU.
    As you know, F&A costs are the shared expenses related to 
buildings and use of research facilities and the administrative 
backbone functions that make such places run. F&A 
reimbursements pay for building depreciation and maintenance, 
academic library materials, shared research equipment, 
departmental administration office supplies, and grant 
oversight activities, such as pre-award application and, 
hopefully, post-award work--not very exciting stuff but 
absolutely critical funding to keep academic medical centers 
and research institutions operating in an efficient manner.
    The F&A reimbursement for costs incurred by academic 
institutions is tightly regulated and audited by the Federal 
Government, and the methodology for negotiating indirect costs 
has been in place since 1965. The rates have remained largely 
stable across NIH grantees for a number of decades.
    The administrative component of the Federal F&A is capped 
at 26 percent, despite the fact, as we heard earlier, that the 
actual costs to administer grants substantially exceeds the 26-
percent recovery. So, again, these are shared costs. The work 
associated with F&A rate setting and reporting is minor and is 
far outweighed by the critical importance of F&A reimbursement 
to universities and research institutions.
    The F&A dollars are needed to support the real costs of 
doing research, without which universities would need to rely 
on other sources of funding, such as tuition dollars or 
philanthropy. Reallocating these costs to other funding sources 
would have a detrimental impact on not only the student support 
but universities' ability to recruit highly talented students 
and faculty. Do we really want to raise tuition to compensate 
for a capped F&A rate and discourage talented individuals 
because of their concern for greater indebtedness or divert 
resources away from supporting a promising young physician 
scientist?
    And like a boat crew using all oars to propel us forward as 
the leader in the world, every supporter, including Federal 
Government, States, universities, and private donors and 
nonprofit foundations, all must continue to share the load.
    I want to close on a point that might be overlooked when 
debating about the minutia of facilities and administrative 
costs, which also speaks to my experience helping teach the 
next generation of academic researchers. Without Federal 
support that shares with the States and the universities the 
cost of building the innovative cardiology and genomic research 
centers we have at UConn, we cannot attract great scientists, 
like Dr. Travis Hinson, who studies the genetics of disease of 
the heart muscle that can lead to early heart failure, or Dr. 
Se-Jin Lee, who has elucidated a new protein target to treat 
frailty.
    These investigators are just some of the innovators who 
have attracted talented students to work with them and are the 
true beneficiary of Federal F&A reimbursement. And they are the 
future of medical discovery and innovation in this country.
    Again, I thank the subcommittee for the opportunity to 
testify. I am happy to answer any questions.
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    Mr. Cole. Thank you very much.
    Before we move to Dr. Gilliland--actually, he is a 
constituent of one of our members of this committee, and Ms. 
Herrera Beutler, I think, requested the opportunity to formally 
introduce him to the group.
    Ms. Herrera Beutler. I thank the chairman.
    I really am excited to be a part of and hear this testimony 
and to introduce someone who we are very proud of, Dr. Gary 
Gilliland, president and director of the Fred--I was going to 
say the ``Fred Hutch,'' the slang--Fred Hutchinson Cancer 
Research Center from Washington State, who is going to be 
sharing with us today.
    We are really proud of this global-leading institution that 
is at the center of life sciences research in our community in 
Washington State. As many of you know, Fred Hutchinson has been 
at the forefront of disease research since being founded in 
1975. And they are known--really, they are world-renowned for 
their cutting-edge work in the elimination of cancer and 
related diseases, particularly with bone marrow transplants and 
immunotherapy.
    And as a researcher and clinician specializing in blood 
cancers, Dr. Gilliland has made major contributions to the 
understanding of the genetic basis of blood diseases, 
particularly leukemia. And in addition to his own research on 
personalized cancer treatments, Dr. Gilliland has successfully 
led to a breakthrough immunotherapy drug--has led it to market 
at Merck and has spearheaded a new model for personalized 
medicine at the University of Pennsylvania system.
    So Dr. Gilliland has earned a Ph.D. in microbiology from 
the University of California, Los Angeles, in 1980 and a 
medical degree from the University of California, San 
Francisco, in 1984.
    And before coming to Fred Hutchinson Cancer Research 
Center, the Fred Hutch, in 2015, Dr. Gilliland has held 
numerous clinical research and leadership titles, including 
professor at Harvard Medical School, director of the leukemia 
program at Dana-Farber/Harvard Cancer Center, senior vice 
president and global oncology franchise head at Merck & 
Company, and the vice dean of the Perelman School of Medicine 
at the University of Pennsylvania.
    He has also had many awards and honors that are really too 
numerous to list here today.
    Dr. Gilliland and Fred Hutch also provide inspiration and 
hope for everyone fighting their own battle with cancer. And we 
are very grateful for your work and look forward to supporting 
you in your efforts to find curative therapies for cancer.
    Thank you, Mr. Chairman.
    Mr. Cole. Thank you very much.
    Pretty great introduction there.
    Dr. Gilliland. That was great.
    Mr. Cole. Dr. Gilliland, you are recognized for your 
opening statement.
    Dr. Gilliland. Well, thank you, Congresswoman, for that 
very kind introduction. I am very grateful.
    Chairman Cole, Ranking Member DeLauro, and members of the 
committee, thank you for inviting me to testify this morning.
    At the Fred Hutch, our mission is to eliminate cancer and 
related diseases as causes of human suffering and death. As 
noted, we were founded in 1975. Today, we have more than 3,000 
employees. We occupy 1.3 million square feet. We run over 400 
clinical trials each year.
    Our breakthrough discoveries began with Dr. Don Thomas' 
pioneering work in bone marrow transplantation, which has 
boosted survival rates for certain blood cancers from zero to 
90 percent. Dr. Thomas' work earned a Nobel Prize in 1990 and 
led to the potentially lifesaving option to treat more than a 
million people worldwide.
    Though originally developed for leukemia patients, this 
procedure is now used to treat more than 50 different diseases, 
including autoimmune disease, sickle-cell anemia, 
myelodysplastic syndromes, inherited immune system disorders, 
and metabolic disorders.
    The development of bone marrow transplantation also 
provided the first definitive and reproducible proof of the 
human's immune system's ability to cure cancer. Today our 
researchers continue to refine these approaches, which harness 
the power of our own immune cells and molecules to eliminate 
cancer, through a world-leading immunotherapy program. And this 
is just one of our areas of focus.
    NIH funding drives scientific innovation at research 
organizations across the country, including the Fred Hutch, and 
I am proud that the results of our exceptional science also 
demonstrate excellent stewardship of NIH funding. Our research 
has led to dramatic returns on the Federal taxpayers' 
investment, both in dollars and in lives saved.
    For example, one study of the Fred Hutch-based Women's 
Health Initiative led to discoveries that have helped prevent 
up to 20,000 cases of breast cancer each year and yielded a net 
economic return of $37,100,000,000 over 10 years, which is a 
return of approximately $140 on every dollar that was invested 
in the trial. And just this year, Fred Hutch research found 
that for the $418,000,000 in NCI-funded Southwestern Oncology 
Group clinical trials, cancer patients in the U.S. have gained 
3.34 million years of life.
    As the committee knows, NIH grants include both direct 
costs and indirect costs. By Federal law and regulation, quote, 
``Direct costs are those costs that can be identified 
specifically with a particular sponsored project relatively 
easily with a high degree of accuracy and can include supplies, 
certain equipment, researcher salary support.''
    In contrast, indirect costs are those that benefit multiple 
research projects, so we are not easily tied to just one 
project. These include sophisticated environmental controls, 
compliance with State, local, and Federal regulations, and even 
equipment and space rental costs. They efficiently enable us to 
protect research participants' safety and privacy, report the 
results of publicly funded research, safely use and dispose of 
potentially hazardous research chemicals and biomaterials, and 
to store and process and analyze very complex data sets.
    Together, direct and indirect costs represent the true 
total cost of research. Indirect funds are not, as some have 
said, quote, ``money that goes for something other than 
research.'' And an institution's indirect cost is completely 
unrelated to its administrative efficiency. It is one method of 
accounting, with formulas that are created by the Office of 
Management and Budget and set by each research center's 
cognizant agency.
    At the Fred Hutch, following generally accepted accounting 
principles for nonprofit organizations results in a general and 
administrative cost rate of approximately 12 percent of our 
total costs.
    At Fred Hutch, our sole focus is research that saves lives. 
So every dollar, direct and indirect, funds research. Patient 
outcomes cannot be improved without funding the true total cost 
of research. This includes shared scientific resources and 
resources that enable us, for example, to generate therapeutic 
T cells for immunotherapy trials, analyze and protect patients' 
genomic data, provide support for exploratory pilot projects.
    By supporting experts and equipment that no single project 
or lab could supply on its own, shared resources, as one 
example, save money, and they drive team science that 
accelerates the translation of bench science into tomorrow's 
bedside cures.
    Two years ago, I pledged that we should have curative 
therapies for most, if not all, cancers by 2025, and I stand by 
that. Advances in bioscience, technology, and data science have 
brought us to this inflection point. This is not a time to pull 
back; this is a time to double down. The choice to invest fully 
in biomedical research is an investment in a healthy, thriving 
Nation.
    Thank you.
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    Mr. Cole. Thank you.
    I think Ms. Herrera Beutler had a quick point.
    Ms. Herrera Beutler. Quick clarification. Please forgive 
me. It is ``Gilliland.'' I am having such a hard time with J's 
and G's. This is my first cup of coffee.
    Dr. Gilliland. I can't say my own name in the morning, so 
you did very well.
    Ms. Herrera Beutler. Thank you.
    Mr. Cole. Well, thank you all again very much for your 
testimony, and let me start with the questioning. And I have a 
question that, really, I would pose to each of you, just 
sequentially, starting with Dr. Droegemeier.
    How would your institution react if the administration's 
proposal to cap indirect-cost reimbursement at 10 percent of 
total costs were actually enacted? Would your institution 
continue to apply for the same number and types of grants, or 
would your institution do less to cover its increased share of 
the administrative costs?
    I am sure each of you would have a different strategy, 
depending on your position, frankly, financially, as to how you 
would approach this, but I am curious what the range of 
responses might be.
    Mr. Droegemeier. Thank you, Chairman Cole.
    At our health sciences center campus in Oklahoma City, a 
10-percent cap would result in the loss of about $11,000,000 of 
F&A for the last fiscal year, fiscal year 2016. On the Norman 
campus, where we actually do NIH-funded research, we would lose 
a significant amount, probably about $7,000,000 or so, over 
about a 3-year period.
    How we would respond to that would be that we would have to 
shut down some labs. We would have to pull back in terms of 
hiring students. We would have to scale back our research, 
ultimately, because we don't have the resources to make up 
those lost F&A costs.
    And I think that is true for a lot of the universities in 
the Midwest that share our same structure, where we don't have 
large research endowments, we don't have resources that are 
gifts and things like that that are already predetermined--the 
use is predetermined. So they are not fungible, flexible money 
that we could apply there. I think we are very loathe to raise 
tuition costs, so we would not balance the books on the backs 
of our students. So we would have to scale back our research.
    And, also, the Oklahoma Medical Research Foundation, 
likewise, told me that about $25,000,000 a year would be 
impacted by this cap, which would translate into about a 
$100,000,000 loss of research.
    So we would have to scale back because we just don't have 
the financial resources, including at the State budget. 
Chairman, you know that our State budget is very challenged 
right now. So we couldn't look to the State or to private 
resources very effectively to raise the cost to make up the 
lost F&A.
    Mr. Cole. Thank you.
    Dr. Yamamoto.
    Mr. Yamamoto. I would echo those comments. In fact, you 
have in front of you three representatives of State public 
institutions, and Dr. Droegemeier's comments are really, I am 
sure, true for all of us.
    We have a large research enterprise. The UCSF has four 
professional schools, are number one in NIH funding in the 
country, have been for the last 3 years running. So there is 
lots of resources there, and you might think, ``Oh, well, 
everything is going to be okay.'' As I mentioned in my 
testimony, the consequence of pulling back to a 10-percent cap 
would be a $120,000,000 shortfall in 1 year. We don't actually 
have that money sitting around in the backyard.
    And so the consequence would be to pull back on 
maintenance, building facilities, being able to maintain them, 
being able to acquire and utilize sophisticated research 
instrumentation, training, and research programs that are 
making a real impact on health. And so that would be the 
consequence.
    It has been said by some colleagues of mine on faculties 
that this would be a great thing because it would open up more 
money for research. But, in fact, that is not the case, because 
the indirect costs of the F&A has to be covered in some way, 
one way or another. And we really don't have a choice for that. 
And so, in fact, it would not benefit new investigators coming 
into the fold. They would not be hired.
    So there is really--it is not an equal matter of being able 
to move money around back and forth. It is just this constant 
pool that we need to deal with. And the consequences would be 
dire for our institution as well as for these others.
    Mr. Cole. Dr. Liang.
    Dr. Liang. In 2016, UConn's total research enterprise 
totaled about $260,000,000, which included about $40,000,000 of 
F&A support from the Federal agencies, including the NIH. So, 
if the F&A were to be capped at 10 percent, we would lose most 
of that $40,000,000.
    And so then the question is, what alternative sources of 
funding could there be? Well, tuition and fees is an important 
part of it, but, being the State's flagship public university, 
we try to keep the tuition and fees down to attract 
outstanding, talented students from Connecticut, also from 
outside Connecticut as well, and we would be forced to raise 
tuition and fees. And, even then, it would not go anywhere near 
to cover the shortfall if the F&A cost is capped at 10 percent.
    And the other source would be philanthropy and donations, 
but, as we know, many of the donations have stipulations. We 
would have to comply with philanthropists' wishes in terms of 
how the funds are expended, and so it really cannot be diverted 
to cover the F&A costs, such as building maintenance.
    And so the end result would be that we have to close down 
research programs, and not only that we cannot innovate and 
stimulate and support young, promising scientists, we have to 
actually shut down some of the research programs. So it would 
be devastating for us.
    Mr. Cole. I have gone over my time, but, with the consent 
of the ranking member, I certainly want to give you, Dr. 
Gilliland, an opportunity to respond as well.
    Dr. Gilliland. Well, thank you, Chairman.
    We are the top NIH-funded research institute in the 
country. We have been since 1992. The impact on the Hutch would 
be calamitous. We have 82 percent of our support that comes 
from the NIH. This 10-percent cap would represent a $60,000,000 
operating loss for us. And the consequence of that is that we 
simply would not be able to accept the same number of NIH 
grants that we do now. We compete pretty effectively for those 
one in five, but we could not move that forward.
    And, Mr. Chairman, the tragedy around that is not that we 
would lose labs or we would close down labs or we would lose 
scientists. The tragedy is that people will die from cancer 
because we are not able to advance our work at a time when we 
are at an inflection point where we have potentially curative 
approaches.
    There are people who have died of cancer while we are 
having this hearing. That is a very important sense of urgency. 
And we will get there someday, but it would slow us down, and 
people will sacrifice their lives as a consequence of that.
    Mr. Cole. Thank you.
    I want to thank my friend for the indulgence, and we will 
now move to the ranking member for her questions.
    Ms. DeLauro. Thank you very much.
    And just to say, this is about saving lives, you know, and 
this is where our opportunity is.
    Let me just--and I want to do something similar. That was 
going to be my first question, but let me get to what is my 
second question, which includes all of you.
    The topic of indirect costs is a discussion about the true 
cost of research and whether the Federal Government is, in 
fact, covering the true cost of that research. So I am going to 
ultimately get to all four of you on this, but, Dr. Yamamoto, 
your testimony notes that, at UCSF, you estimated your indirect 
costs in 2016, $253,000,000. You were reimbursed $160,000,000.
    Dr. Droegemeier, your testimony was that unrecovered 
indirect costs at the University of Oklahoma's Norman campus is 
averaged at $17,500,000 per year from 2011 to 2015. You also 
note that the campus recovered about 52 percent of its indirect 
costs, while the university picked up the tab for the remaining 
48 percent.
    So, ultimately, as I say, I want to get all four of you to 
answer this. But, Dr. Yamamoto and Dr. Droegemeier, I would be 
interested about a further explanation of the discrepancy 
between your estimated indirect costs, the amount that is 
reimbursed by the NIH. How do you fill the gap?
    And I would like to ask that same question of both Dr. 
Liang and Dr. Gillibrand--Gilliland. Gillibrand is my colleague 
in the Senate, so we have all kinds of things happening here 
today.
    Dr. Gilliland. Almost anything.
    Ms. Herrera Beutler. It is ``gill''----
    Ms. DeLauro. Gill.
    Ms. Herrera Beutler [continuing]. Like a fish gill, for the 
record.
    Dr. Gilliland. Fish gill. That is good. Okay.
    Ms. DeLauro. Thank you.
    Mr. Yamamoto. Thank you for that question. Maybe we should 
start calling him Jerry Gilliland.
    So the shortfall, we have to make it up. So the discrepancy 
comes from----
    Ms. DeLauro. Could you talk about the discrepancy?
    Mr. Yamamoto. So the discrepancy comes from two sources.
    One is the outcomes of these negotiations that we go 
through every 3 to 5 years with HHS. It is really hand-to-hand 
combat. It is, line by line, moving through the budgets, where 
the money is coming from that is actually going to NIH-funded 
investigators, not just other scientists or clinicians at UCSF. 
And so that process sort of keeps everyone on track. It is just 
a way to prevent problems, fraud or other kinds of abuse, 
because those things come up every 3 to 5 years and we have to 
take it through.
    When we do that, then it is a negotiation with the 
auditors. And they allow certain amounts, sometimes--always 
less than what we think would be justified and always, as you 
said, resulting in a big shortfall that has to be covered in 
some way. So that is one source of the discrepancy.
    The other is, as Dr. Droegemeier said, that decades ago--I 
guess it was 1991--NIH installed a cap on the amount of 
indirect costs for certain kinds of grants and certain kinds of 
activities. Administrative costs capped at 26 percent, as we 
have heard. Again, a big shortfall. And there is just nothing 
we can do about that. That is not even up for negotiation.
    So, in order to keep the enterprise functioning, we need to 
make up the shortfall. It used to be that we could draw on 
State funds and State support. That is no longer true. UCSF 
doesn't have undergraduates, so even if we were to take this 
dramatic step of increasing tuition, we would have no impact on 
the operating budget at UCSF.
    And so it is really through philanthropy and pulling 
together other sorts of funds and endowments that can be 
liberated for activities like this that we are able to cover 
those costs. We know that is the case, and we are able to do it 
now with the negotiated rate that we have. But if there would 
be this draconian cut, then we would be short, as I said, 
$120,000,000 each year.
    Mr. Droegemeier. I would agree with that. I think, as time 
has gone on, you know, we have learned to deal with some of 
these things. It has sort of been creeping along and growing 
up, but if you look at the 24 percent of funding that now 
universities put on the table, almost one-quarter of all the 
funding that they expand every year is from their own money. We 
heard the Fred Hutch Center, you know, a very substantial 
amount of money, compared to NIH money coming in. So that, I 
think, is really, you know, a very, very big problem.
    I think the other thing is the fact that we--my campus, we 
basically only recover half of the F&A, essentially, that we 
are due. And so we have made up that funding in other ways. We 
have scrimped and saved and stuff, but we have also stopped 
providing faculty agreements to cost-share. Even if it is 
required by the agency, we basically say, ``No, we can't do 
that.'' Major equipment proposals that have a 30-percent 
mandated cost-sharing by the Federal Government, sometimes we 
say, ``You know what? We can't do that. We can't afford to be 
successful because we can't pay the cost of bringing that in.''
    So it is not just the F&A rate; it is also the cost-
sharing. When that all piles on together, essentially what 
universities have to do, they have to say, you know, ``We can't 
do that anymore.'' And it has really hit that point now.
    Ms. DeLauro. Dr. Liang.
    Dr. Liang. Thank you, Ranking Member DeLauro.
    So we are already putting in another 20-some percent of our 
own funds to cover the unreimbursed F&A. And so, you know, we 
will have a gap there if the cap is capped at 10 percent, and 
we will have to go to tuition and try to find more philanthropy 
sources. And the State of Connecticut has reduced the block 
grant to the university, and so we will be really in a bind. 
And I think we are going to have to be very strategic in a way 
we don't want to be by closing down certain research programs.
    I think, you know, as a scientist, I can attest to the fact 
that a lot of the discovery is by serendipity. And if you are 
trying to be too top-down and strategic, you are going to lose 
a lot of basic knowledge and that scientific talent, and it 
would be really devastating.
    Thank you.
    Ms. DeLauro. Dr. Gilliland.
    Dr. Gilliland. Yes, thank you.
    Not too much to add, except that we do--and thank you for 
the proper pronunciation. I am very grateful.
    We do renegotiate our rates as a research institute every 
year with the HHS. It is a civilized process, but, nonetheless, 
we don't recover about $30,000,000 to $40,000,000 a year in 
indirect costs. And the way that we make that up is through 
philanthropy. I would like to spend my time focusing on curing 
cancer, but I spend about 30 percent of my time in 
philanthropic efforts to help support our budget.
    Ms. DeLauro. Thank you.
    Thank you, Mr. Chairman.
    Mr. Cole. Thank you.
    We next go, by order of arrival, to Mr. Fleischmann. Is----
    Ms. DeLauro. He just left.
    Mr. Cole. He just left, so there you go. Okay. We now go 
to, then, my good friend from Maryland, Dr. Harris.
    Mr. Harris. Thank you very much. Thank you very much, Mr. 
Chairman, for calling a hearing on a very important topic.
    First of all, I am just--and you don't have to answer--I am 
just puzzled by this discrepancy, because, you know, HHS does 
pay a fairly large indirect-cost percent. So I assume the 
discrepancy really occurs from all those other grants that 
aren't NIH grants.
    The ones, for instance, listed on this--and I don't need an 
answer, because I only have 5 minutes. For instance, all these 
groups listed on this slide vary from the American Lung 
Association, with no indirect-cost reimbursement; American 
Cancer Society, 20 percent; American Heart, 10 percent.
    And, you know, the testimony that Bill & Melinda Gates, you 
know, covers facilities--but you have to mention that change 
was made in February of this year, after the administration had 
said, let's take a look at indirect costs because of this 
discrepancy.
    Now, let me go to the next slide here. Now, here is one of 
the problems. The question is, what are we doing in our medical 
research universities, and are we, in fact, training or 
educating the next generation of researchers?
    This slide--all of you, I am sure, are familiar with it--is 
the graph of the age at which people make their great 
discoveries. So, for instance, the physician you mentioned at 
Fred Hutch, the one who developed the transplantation of 
leukocytes to treat leukemia, made his discovery in his late 
thirties.
    Next slide.
    If you look at when RO1 grants, the major grants--go one 
more slide, this one--when RO1, the major grant over at NIH, 
was made and the age distribution, in 1980, strangely enough, 
the peak of distribution was in the late thirties. That is when 
the NIH was awarding these grants, to people in their late 
thirties. That is when people actually, statistically, make 
their great discoveries.
    Now, let's go to the other slide. Let's fast-forward 35 
years, with what has happened over at the NIH. This is the 
graph from 2015. Markedly different. The peak is now at 50. 
Now, I am 60. I used to do NIH research. I am not going to make 
a great discovery, because you know what? Once you reach that 
age, you put blinders on.
    So we have asked the academic institutions over the years 
to come up with ideas for how to solve this. And I have had 
many presidents of universities in my office say, ``It is easy. 
Just give us more money.''
    Next slide.
    This shows the NIH funding from 1980 on the left to 2015 on 
the right. So, as we were increasing funding from 
$4,000,000,000 to $32,000,000,000, the grants were going to 
older and older individuals.
    Now, I am going to ask Dr. Yamamoto, I have a letter here--
because, you know, I talked with Dr. Collins about this 
problem: Are we doing the right thing at the NIH, and methods 
with which we are trying to solve this. So he came up with the 
GSI, you know, the grant scoring index, the one that says: You 
know what? There is actually evidence from the NIH that, once 
you get multiple grants, the last grants are actually much less 
productive than the first grants. It makes human sense. You 
know, you only have so many things you can think about at once. 
Your major projects are going to be the ones you think about; 
the less major ones are not.
    But remember, the indirect costs are the same for all those 
grants. Whether it is your first grant or your fifth grant, the 
indirect costs are the same.
    You said in your letter to your faculty that there are 42 
UCSF investigators who were threatened by the GSI. And, 
strangely enough, the GSI plan was shot down a month and a half 
later, withdrawn by NIH, because academic universities, on the 
whole, opposed the GSI.
    Now, why? You have to ask the question, why? Is it because 
we really want all the senior investigators to get their fourth 
and fifth and sixth grants that have 52-percent or 60-percent 
indirect costs?
    I was in this business. Universities love NIH grants 
because they come with large indirect costs associated with 
them. Allowed them to build big buildings. Allowed them to 
build great facilities. Great facilities actually have the 
option of curing cancer.
    But have we lost a very important point there? And I am 
going to ask each of you. Have we lost our ability to say one 
of our major goals is to train young investigators?
    Now, I was involved, and I know that there are theories 
that, you know, this was cooked up in order to just lower the 
NIH budget. No, it wasn't, because I know the indirect-costs 
subject was brought up to make universities think about the 
future of research in America.
    And remember, the GSI press release by Dr. Collins said 
there was 6,000 more grants that could be funded if we 
instituted GSI. And I would make certain--I would do my best to 
make certain these went to young investigators.
    So a method to reduce NIH expenditures at some point by 
perhaps capping indirect costs, like is done in other 
institutions, why couldn't we use that money to turn over and 
start shifting that curve back to young investigators? A very 
quick response from all of you. Shouldn't we do that? Shouldn't 
we put more emphasis on young investigators? Quickly. It could 
be a ``yes'' or a ``no'' response.
    Thank you. I yield back.
    Mr. Droegemeier. I think we absolutely should. I think it 
is a very important problem. NSF has dealt with this same 
thing, of getting the young investigators. I don't think the 
F&A is the way to solve that problem, though.
    In fact, to your earlier point about those percentages you 
showed, it is important to realize that the Federal Government 
funds 55 percent of the research in this country; the 
universities fund 24 percent; the philanthropic organizations 
fund 6 percent. Their role is very, very different than the 
Federal Government. So, although we have low indirect-cost 
rates, they represent a really small fraction of the money we 
expend.
    So, therefore, we eat some of that fund, but if that 
happened to NIH, that is why we would be dead in the water, 
because it is 55 percent the Federal Government. You start 
monkeying with that 55 percent, we are in serious trouble. We 
can handle a little bit with the foundations because they have 
a much different role and they are a much smaller percentage.
    Mr. Yamamoto. So I agree with that.
    Let me just take a different tack to your question, Dr. 
Harris, and that is that this increased age of investigators 
and your zeal, which you have had for a long time, to support 
young investigators--and I share that with you, as we have 
discussed in your office--would not be addressed in a 
substantial way by changes in F&A.
    The capacity to fund more investigators or, actually, to 
issue more grants is a positive one when resources are limited, 
as they are. But whether the--my letter and those of other 
investigators about the concerns about the GSI was not to 
oppose those ideas, but, in fact, because we felt that the 
metrics by which these measurements were being made were not 
correct. Different kinds of research cost very different 
amounts of money. And so simply measuring dollars and 
restricting the researchers in that way is not appropriate.
    I know that the NIH--we are working with them to look for 
other ways to try to measure this, and I know that the Deputy 
Director for Extramural Research is working on this very 
actively. So whether this can be done and whether it would have 
an impact on the support for young investigators, I think, is 
something that we need to look very carefully at.
    You probably know that the National Academy of Sciences is 
undertaking two studies right now--I am on one of the study 
committees--to look at the way that graduate education works in 
STEM disciplines. Another one is explicitly targeted to support 
for new investigators. Those reports we will be issuing in the 
next year. I think that you will--I hope that you will be 
excited about what some of the things are that we are putting 
forward.
    And so I think there are approaches to that problem that 
you have so long focused on that can be very productive. I am 
not convinced that changing the F&A is one of them.
    Dr. Liang. Thank you.
    My answer is fairly straightforward. I think if you take 
away the F&A reimbursement, it will affect the young 
investigators more than it would the established investigators, 
because their labs do not have all the equipment and the know-
how that build up over the years.
    And we will have to find other sources to support the F&A 
costs, which, as we heard earlier today, is really a real cost. 
And, as Representative DeLauro said, it is a misnomer. It is 
really part of the real cost.
    So I think you are going to really more adversely affect 
the young investigators. And you wouldn't be able to use 
sources of funding to stimulate high-risk-but-high-reward 
research pilot studies in which the young people have these 
wonderful, thinking-outside-the-box ideas, and you are going to 
have to use those resources to fund the real costs, which is 
the F&A. And so I think it is going to be worse for the young 
investigators.
    Thank you.
    Dr. Gilliland. Dr. Harris, I share your passion for 
training young investigators. And it is also terrific to have a 
committee member who has been the recipient of NIH funding. So 
I do appreciate your perspectives.
    We have a variety of mechanisms for supporting young 
investigators at the Hutch. As one example, every new recruit 
that comes in--and we are actively recruiting--has a 
$2,500,000-or-so startup package that comes from philanthropy 
that we use to ensure that they are not dependent upon NIH 
support moving forward.
    I would also say that there are certain NIH grants, like 
training grants, that are really important for young, 
burgeoning investigators that only bring in 8-percent indirect-
cost rates by stipulation. So we do need to make up that 
balance.
    And, lastly, I do appreciate you showing the slide on the 
foundations, but I would say that it is a bit of an apples-and-
oranges comparison because many foundations will allow 
accounting methods that take into account indirect costs. For 
example, foundations will help support our infrastructure for T 
cell immunotherapy. And the Gates Foundation, in particular, 
from whom we can't accept all the resources we could get 
because we can't afford to, they do have an infrastructure 
grant that they have awarded to the Hutch because they 
understand the challenges in trying to support our global HIV 
vaccine trials network.
    But very important points. And it is really important for 
us to dial back and have people that look less like you and me 
and people that are younger leading the charge.
    Mr. Cole. I guess we send people over 60 to Congress, 
right?
    If we could, again, on order of arrival, we will go to my 
good friend, Mr. Pocan.
    Mr. Pocan. Great. Thank you, Mr. Chairman.
    And thank you to the panelists.
    You know, first, I think the good news is, you know, in 
what has been at times--it is a difficult Congress to get 
things through both houses and signed into law. The good news 
is, on NIH, there is bipartisan agreement. Thanks to our 
subcommittee chairman and our ranking member and other 
advocates, we have been really fortunate in being able to get 
additional dollars for NIH.
    And I think, even on this issue, when we had Secretary 
Price here, who was a fiscal hawk, on this issue--no longer is 
here, I am not sure if the same emphasis is going to be as much 
on this issue. Because, as I talk to Members and hear Members, 
it seems like people really understand this indirect-cost issue 
in a better, more comprehensive way.
    So there are two things I would like to get to you. So, on 
the first one, if we could get, like, 30- to 40-second answers.
    To be fair, if we are putting more dollars and we want more 
dollars going to research, what are some of the barriers that 
you are having as you get the money? For example, the 
University of Wisconsin, which would lose about $50,000,000 
under this proposal, has said some of the--in order to justify 
the cost, it is something that is a real burden on the 
regulation side, so to speak.
    Just 30 to 40 seconds each, if you could, what could make 
it so the money is even more being delivered to care or to 
finding cures?
    Mr. Droegemeier. Irrespective of F&A, right?
    Mr. Pocan. Yes.
    Mr. Droegemeier. Yeah. I would say, absolutely, the 
regulatory framework that we have right now. Two studies 
successively have shown, by the Federal Demonstration 
Partnership, that faculty who are funded by the Federal 
Government, on average, spend 42 percent of their time on 
administrative activities. And we all agree that for human-
subjects research, animal research, things like that, there are 
very, very important regulations that need to be followed--
laboratory safety and so on. But a lot of these regulations 
really have no impact at all, have been shown to have no 
impact, but yet they really are suppressing our research 
system.
    So we think about waste, fraud, and abuse. I think one of 
the biggest abuses and the wastes that we have is the 
intellectual talent of our faculty and researchers because of 
all this administrative burden, some of it very, very 
important; other of it really isn't needed at all.
    And so I think focusing on that, which Congress is doing, 
and so is the National Academy, very effectively, I think that 
would be my answer.
    Mr. Pocan. Thank you.
    Mr. Yamamoto. I don't have much to add to that. I totally 
agree with that. I got my first NIH grant when I was 30, and I 
should clearly be shown the door by now. But it was a different 
world. When I got that grant when I was 30, I was able to spend 
all my time in the lab doing work, and now it is exactly what 
Dr. Droegemeier said. I spend all my time kind of chasing down 
regulatory reports, compliance reports, and doing things that 
actually could be addressed well.
    There are several reports that have come out, some from the 
National Academies, that deal with the administrative-burden 
issue. And I really hope that members of this committee or 
other Congress Members will take up that charge, because that 
really is the biggest problem. It not only is very wasteful of 
resources, but it really changes the way that we--the time and 
energy going completely into thinking about science. And simply 
just sitting back and reflecting on what is going on in the lab 
makes all the difference, and that is the time that goes away 
first.
    Dr. Liang. I don't have much to add. I would just echo the 
comments.
    And I think, as an administrator now, I do my best to 
protect the young investigators' time and to protect their time 
and to try to steer their attention and effort to ask the right 
science questions and to give them the resources that they need 
to build their scientific research programs.
    So I think less time spent on regulatory areas, other than 
IRB and the IACUC, the Animal Ethics Committee, would be 
helpful.
    Thank you.
    Dr. Gilliland. I echo those comments, Congressman, but I 
would also add that our faculty spend, on average, 50 to 55 
percent of their time writing grants, responding to reviews on 
grants, recycling grants, administrative support. Some of them 
are very good at it, but that is not what we select them for. 
We select them to be brilliant, creative, innovative 
scientists. And I would rather have them spending time doing 
that and not as much time in the grant-writing process.
    So we do a lot to support them. We are trying to move to a 
model where we can give our faculty resources that we can raise 
from exogenous sources like philanthropy so that they are able 
to focus on their science and think about their science and not 
think about the mechanics.
    I will give one example that is terrific, though, from the 
National Cancer Institute, which is that we have an 
investigator named Denise Galloway. She is the woman who 
connected the dots between human papilloma virus and cervical 
cancer that led to the vaccine that was developed at Merck that 
has the potential to prevent more than 90 percent of cervical 
cancer worldwide. That is what she should be focused on.
    And the NCI gave her this fantastic grant called an 
Outstanding Investigator Award that runs for 7 years with 
nominal reporting strategies. You usually only get this when 
you get to be my age, but she is still doing incredible work. 
And it is a great chance for her to think about her science and 
not to have to focus on some of the administrative 
responsibilities.
    Ms. DeLauro. That is potentially 4,000 lives a year for 
women.
    Dr. Gilliland. In the United States. But worldwide, Sub-
Saharan Africa, China, cervical cancer is an enormous 
challenge. And it is a lot better to prevent cancer than it is 
to treat, I can tell you that, as you well know.
    Mr. Pocan. Thank you.
    Mr. Cole. I feel like I ought to give you extra time, but 
you have actually gotten us back to the 5-minute clock, so I 
want to thank my good friend for that.
    We will next go to Mr. Moolenaar.
    Mr. Moolenaar. Thank you, Mr. Chairman.
    And I want to thank all of our guests here.
    A number of you mentioned that the F&A cap would impact 
training programs, either shrinking or terminating some of 
those, and would hinder the professional and educational 
opportunities for the next generation.
    I wondered if you have thoughts on how that would impact 
the future of our healthcare workforce, especially as we are 
facing a shortage of healthcare workers, and what this would 
mean for the next generation of scientists.
    Mr. Droegemeier. I will be happy to start.
    I think one of the things that is important to recognize--
we haven't talked about this--is that a lot of the 
universities, I think, who would sort of succumb to this cap 
are in areas that have large populations of underrepresented 
individuals. So I think we would see a decline in the 
increasing participation of those populations in research, 
which, if you look at the trends of the demographics in this 
country, means that we are not going to have enough folks to do 
the research.
    Also, those same universities produce a lot of the faculty 
that are hired by the top institutions in the country. They 
also hire those individuals. So if those--and I can refer to 
some data I have in my report for NIH in terms of the funding. 
The F&A goes to the top 50 institutions of 500; 10 percent of 
the institutions get 70 percent of the F&A. So, if those other 
institutions, those other 450, start to decline and, you know, 
get pushed aside, basically, they are not a supplier of talent 
to the other institutions, nor are they hiring the individuals 
who become their faculty.
    So it really is underrepresented groups get harmed and also 
the supply and demand of faculty positions and researchers gets 
changed.
    Mr. Moolenaar. The other thing, just to follow up on that, 
I wanted to ask, you know, it seems like right now we have 
large and small, public and private universities, nonprofit. Do 
you think the smaller, less established institutions, 
laboratories, would be able to compete for the funding if we 
remove that?
    Mr. Droegemeier. I think it would be very difficult for 
them because they don't have the infrastructures and they also 
don't have some of the financial resources that larger 
institutions do. But I think, ultimately, even the well-
resourced institutions would find it very difficult, as time 
goes on, to make up the difference of the lost F&A.
    Mr. Yamamoto. I think that the up-and-coming institutions 
would suffer first. And we need that kind of diversity in our 
scientific enterprise as much as any of the other kinds of 
diversity that we are thinking about.
    We have to have different ways of thinking about problems, 
because our chances of having our ideas being correct are 
actually small. There is one way to be right and an infinite 
number of ways to be wrong. Right? And if everybody in the 
enterprise trained together and thinks together, then you could 
actually have the whole enterprise going down the wrong 
pathway.
    So having that diversity is really a crucial thing, and I 
think it is something that we really need to be attentive to.
    I just want to make one other quick point, and that is that 
Gary mentioned this ``inflection point'' term, and I want to 
address it in the terms of our training and our training 
population.
    And that is that we are at a place in research, biomedical 
research and other research as well, where the potential for 
Ph.D.-trained individuals in the sciences to be able to 
contribute to society not only as faculty members in our 
research institutions that we are discussing but in a whole 
range of other fields--as you all well know, decisions that 
society is going to be making now and in the future are going 
to be increasingly technology-driven, and they are going to 
have to be wise decisions that are based on real knowledge and 
understanding of what those technologies are, what the 
potentials are, and what the potential downsides are.
    That kind of understanding, we really need people, you 
know, all parts of our society, to be able to advise us about 
that, not just in science and medicine, but these trained 
people going out to fill positions in policy, as you are all 
doing, and in communication and education and business and law, 
because those are the stakeholders that are going to be 
involved in making and driving these decisions.
    So we lose the workforce because of a drop-off of the 
capacity to support NIH research. And we are losing not just 
the kinds of a scientific and health advances that we will be 
hearing about, but we really affect society's capacity to be 
able to make the right decisions.
    Mr. Moolenaar. Thank you.
    And, Mr. Chairman, I just wanted to ask one last question 
that may go a little bit over.
    If I understood what you said, you said your researchers 
spend almost 50 percent of their time doing compliance-
related----
    Dr. Gilliland. I said that.
    Mr. Moolenaar. Are there some key areas that we could look 
at to improve that so that they are actually spending more time 
on the research function? And is that something you could make 
recommendations to the committee moving forward?
    Dr. Gilliland. Well, I think I can address that, that it 
really does come down to what the paylines are. At the National 
Cancer Institute, it is around 9 percent, so that is 1 in 10 
grants. It just means you have to continually be writing grants 
and rewriting grants. It means, when you submit the grant, it 
often isn't funded the first cycle; you need to come back for 
the next cycle.
    So I think it is largely a function of paylines. When the 
paylines were where they were when Keith and I were young, in 
the 20- to 25-percent range, it was much more tractable.
    But to your point about the impact on training, for us, if 
the lower cap is imposed, we just won't be able to accept as 
many grants. So we will go through a process where we will have 
to do internal prioritization before we even allow somebody to 
send a grant in. And then they go through the 9-percent 
paylines at the NCI for our institution.
    And that scares off kids who are thinking about going into 
science. We have a lot of them that get their Ph.D.s in the 
biomedical sciences, and, at least in Seattle, they will go off 
and work for Amazon or Microsoft or Google. So I really worry 
about the impact that we won't be able to measure, who is it 
that didn't go into science because it looks so onerous in 
terms of trying to obtain grant support.
    And to the point about diversity of thought that Keith 
made, you should take the sequester as an example. The larger 
institutions have an easier time weathering that, for a variety 
of reasons. We can't afford to lose the diversity of thought 
around this country and to have the big get bigger and have the 
small go away. We can't afford to have that happen.
    Mr. Moolenaar. Thank you.
    Thank you, Mr. Chairman.
    Mr. Cole. You are welcome.
    We will go now to my good friend from California, Ms. Lee.
    Ms. Lee. Thank you, Mr. Chairman.
    [Off-mike] 350 research agreements, approximately 
$63,000,000, paying 57-percent rate that NIH and other Federal 
offices currently agreed to in order to access university 
research infrastructure.
    So, if the cap were 10 percent and universities were forced 
to turn to other financial realities, what would that mean for 
global competitive research, and who would universities turn to 
for this research?
    Secondly, in the letter, it was mentioned, the fact that a 
10-percent cap would have adverse impacts on fostering job 
creation and American economic competitiveness and healthcare 
outcomes, of course, for all citizens.
    So I would like for you to respond to those two questions: 
Where would universities go for additional research money if 
the 10 percent were capped? And what would this do in terms of 
our economic growth and competitiveness and job creation?
    Mr. Yamamoto. So I think that we have addressed your 
question in part, Congresswoman Lee. And it is nice to see you, 
as well.
    You know, UCSF received approximately $624,000,000 in 
funding in 2016 from the NIH. $465,000,000 of that was for 
direct costs, and $159,000,000 was for F&A. That left us with a 
shortfall at which UCSF had to come up with $77,000,000 to be 
able to cover the F&A shortfall. That means for every thousand 
dollars of NIH funding that UCSF received in 2016 UCSF 
contributed an additional $166 that it had to pull together 
from other sources.
    If the 10-percent cap were installed, as I said, we would 
be facing a really dire shortfall that we really wouldn't be 
able to address if it were installed in this draconian way. 
Yes, of course, we would scramble to try to find other 
resources, but the outcome of it, as you implied, is that U.S. 
scientific competitiveness would take an immediate hit.
    It is great that you brought this up. I mean, we live in a 
world economy, and other countries are moving very rapidly to 
try to--or they are sort of incorporating this notion that the 
U.S. has moved forward because its prowess in scientific 
research. If you look at what is happening in India and China 
and South Korea, they are putting double-digit increases into 
their scientific budgets every year.
    And so, if NIH were to pull back, even if it were in this 
factional way of changing the F&A costs, the impact on all of 
our institutions would be dire, and it would just allow these 
other countries to move forward. So the competitive issue is a 
serious one we need to keep in mind as we look at these 
policies.
    Mr. Droegemeier. I would just say that I think, really, 
this cut really potentially endangers the partnership that 
dates back 70 years between the Federal Government and 
universities that won World War II. And I think, ultimately, 
the success of our Nation and, really, competing in the world 
really requires the government and the universities to work 
hand-in-hand.
    And, in fact, if you look at the current OMB guidance, it 
talks about paying their fair share. A 10-percent cap would 
shift a massive amount of the funding burden to universities, 
and that fundamentally changes the role of the Federal 
Government in the research enterprise. It really puts it on the 
backs of the universities, who are now problematically funded 
by States and things like that that don't have the resources, 
that aren't going to raise tuition and so on.
    So I think the partnership model starts to come undone, and 
that has really massive consequences for us when we are facing 
other nations that are investing, as Dr. Yamamoto said, and 
moving very, very rapidly. China, India, you look at the number 
of papers, the number of students they are producing, the 
number of patents and so on. They are investing massively. If 
we pull back and undo that partnership, it is going to be very, 
very difficult to recover.
    Dr. Liang. I would just add that the infrastructure that is 
allowed by the F&A reimbursements are critical not just to 
support the research at the university, but an important 
byproduct is the science and invention to start up companies. 
So it is a very important way to get return on the investment. 
And if we take that away, the downstream consequences would be 
adverse for the economic job creation piece of it.
    Dr. Gilliland. I would just add, Congresswoman, that one 
unintended consequence, as I alluded to, would be that it will 
be even more difficult to get grants in that context. It 
certainly would be at our institution. And the consequence is 
that people won't focus on the cool, creative, innovative, 
high-risk-high-reward type of science. They will focus on 
things that can get funded and things that are inherently 
predictable. And, yes, it is likely to yield ``important new 
information,'' is one of the phrases that comes out of NIH 
study sections.
    So I do have some concerns about the impact on our ability 
to maintain our world-leading pace in the creative and 
innovative space, which is where the most important advances 
will come.
    Ms. Lee. Thank you.
    Thank you.
    Mr. Cole. I thank the gentlelady.
    We will go next to my good friend from Idaho, Mr. Simpson.
    Mr. Simpson. Thank you, Mr. Chairman.
    Thank you all for being here today.
    First, let me get rid of one of my pet peeves. Over 80 
percent of the NIH funding goes to extramural grants to you 
all. And when a discovery is made of a new drug that cures 
whatever or helps whatever, the press releases all that kind of 
stuff, newspaper articles, all about the researcher, all about 
the university, all about the Hutch or whatever, you know. 
Nothing about NIH in any of this stuff.
    What I am saying is: Help us help you by educating the 
public that it is their tax dollars that are doing this through 
NIH and stuff. Because if I go out on the street, most people 
don't even know what NIH is. I understand where you are coming 
from, but help us in that regard.
    Secondly, I guess we can thank the administration for this 
hearing today. Had they not proposed their proposal in the 
budget which we rejected, we probably wouldn't even be having 
this hearing on F&A costs and that kind of stuff, and I think 
it is a vital hearing that we have.
    I am a big fan of what goes on at NIH and what goes on at 
the universities and the research that is done. I have to 
preface my remarks saying that, because it might not sound 
friendly when I ask this question.
    I appreciate Dr. Harris' input on this and the challenges 
that we face. But this is what happens out at the universities, 
and you all know this. All the universities want to become 
research facilities. They want to expand their role, attract 
students because they have more research and stuff. So they go 
out and they raise money, private foundation, maybe through the 
State building authority, to build a new biomedical research 
facility. They get a bill. Then comes the cost of operating it.
    And I am sure, in the preplanning, part of the cost of 
operation is, how many grants can we get that will help us 
offset the cost of operating it? Is that true? I mean, that is 
an obvious thing that you would look at, at how we are going to 
do it.
    So then when we talk about whether 26 percent is the right 
percent or not, it is kind of like, well, if we don't get this 
26 percent, we are going to have to close this facility that we 
went out and initiated to build so that we could expand our 
research.
    How much of that 26 percent of the overall facilities 
administrative costs of a grant would still exist if you didn't 
get the grant?
    Dr. Gilliland. Well, I can take a first stab at that, 
Congressman. And it is a really important question.
    I will say, first of all, that our buildings, at least on 
our site, are built with debt and philanthropy. We don't 
receive any Federal funds or State funds to support those.
    We do use indirect-cost rates to support, in essence, the 
space or the rental costs, if you want to think of it that way. 
But if you compare the cost in terms of our indirect rate by 
using a building that we put up with debt and philanthropy to 
what we would have to pay in the South Lake Union area for 
market rental, that is about a 50-percent savings on what it 
would cost if we didn't have those facilities in place.
    So we are very sensitive to that issue. This should not be 
a strategy for putting up buildings, and it should not be a 
strategy where you have to close the building down. But the 
government doesn't take any risk, we take the risk, associated 
with the cost of the building and the mortgages and the debt.
    Mr. Simpson. And I understand what you are saying, because 
you are a private entity as opposed to a university. And 
universities are always competing against one another.
    Are we doing it wisely, and are we using this as a way of 
helping us expand our research capabilities as a university so 
that we become a more modern research university and that kind 
of stuff and then saying, the Federal Government needs to help 
us in this arena?
    Mr. Droegemeier. I think when the NIH budget doubled in the 
late 1990s and early 2000s, I think there was a large amount of 
building going on. In fact, people have written papers on this. 
Bruce Alberts and other people have written papers that talked 
about----
    Mr. Simpson. And let me say, I am not saying that that 
increased building and stuff is a bad thing.
    Mr. Droegemeier. Right. Right. Right. No, you are 
absolutely right. And, of course, we educate students in there, 
so when we actually do the audits for the F&A rate setting, we 
only look at the space that is set aside for research. But how 
do you differentiate when you are educating a student versus 
doing research, especially if you are undergraduates and so on 
and so forth?
    So I think universities are much more sensitive to that now 
in the past 10 years, probably, or so, to not overbuild. They 
look at Federal budgets; they see that they are not going to be 
growing and doubling and so on and so forth.
    So I think there was some of that that happened in the late 
1990s with the doubling, but I don't think it is happening now. 
If you look at the last year for which data are available--I 
just, actually, looked this up the other day--in terms of new 
construction, the government funded 15.8 percent of buildings, 
and the institutions themselves funded 63.7 percent. So, by and 
large, the government isn't funding a lot of new construction.
    But when the buildings are built in debt service, we do 
include the interest in the F&A rate. But I think there is a 
lot of scrutiny on that now, and I think universities have 
gotten wise to the fact that it is very, very difficult to 
build lots and lots of buildings, because you won't be able to 
fund their usage. And, at the end of the day, you have to pay 
the debt service on them if you don't, you know, fully pay for 
the buildings up front.
    So I don't think that is as much of an issue as it was 15 
or 20 years ago, but that is a very, very good point that you 
raise.
    Mr. Simpson. I appreciate it.
    Thank you, Mr. Chairman.
    Mr. Cole. You are welcome.
    Ms. Roybal-Allard.
    Ms. Roybal-Allard. We have spoken a lot about the impact of 
the 10-percent cap on your universities and on students, but I 
would like to discuss the impacts of these potential cuts on 
larger research communities.
    Most of your institutions are located in communities that 
are also home to biotech companies that benefit from university 
research collaborations. If F&A funding is cut and universities 
are forced to support smaller research portfolios, what would 
be the impact on the biotech companies that were attracted to 
robust university research communities, and what impact would 
it have on the overall economy in your regions?
    Mr. Yamamoto. You are absolutely right that one of the 
great outcomes of the real revolution in our understanding of 
biomedical processes and disease processes has been on the 
economic end. And the first biotech company in the Nation, 
Genentech, was actually started by a UCSF researcher. And it 
was really a watershed moment, because it indicated for the 
first time that basic biomedical research could actually be 
made into a product that could make money in a company and 
actually have impact, direct impact, on people's health and 
well-being. Those two things are incredibly powerful, and it 
has led to the biotech revolution you are talking about.
    And so, a pullback in the capacity of this government 
compact with the academic research community would actually 
have a very strong detrimental effect on the capacity to start 
up new companies and to keep them healthy and going strong.
    UCSF's Mission Bay campus is surrounded by biotech and 
pharma companies, many of which were started from the research 
that goes on there, and many of them are actually my trainees 
who were at UCSF at the time. And so now pulling back from that 
commitment, that compact, would actually have dire consequences 
on this economic engine that has developed.
    Dr. Gilliland. Yeah, I would echo those comments. We are 
also surrounded by biotech in the Seattle area, and it comes 
back to this issue around how do you foster creativity and 
innovation. Because that is the real value. That is where the 
intellectual property comes into play that is attractive for 
biotech.
    And I will also add to that that, although some academic 
institutions have--well, they probably all have a certain 
amount of hubris, but--have hubris around their ability to 
actually make a drug or develop a drug within house, I can tell 
you, having been on the other side of the fence in pharma, that 
that is simply not tractable. We absolutely depend on biotech 
and pharma to take our discoveries and translate them into 
clinical benefit. And that is the model that we use at the 
Hutch, and I do think it would be deleteriously impacted.
    Dr. Liang. And I will also echo those comments. It is a 
very good question. Yale-New Haven area, with bioscience 
ventures, that has been growing for quite some time in 
Farmington with UConn Health and Jackson Lab for Genomic 
Medicine and the University of Connecticut as doors. We are 
also seeing growing activities in the biotech innovation 
attracting folks and companies from outside the State, in fact, 
to come to Farmington.
    We have built the facilities basically based on State 
support, and we are being able to rent out space to companies 
from both within UConn invention as well as outside UConn.
    But thank you very much for that very relevant question.
    Ms. Roybal-Allard. Just one more question. The United 
States is currently number one in biomedical research. However, 
as Federal funding for research has declined in the U.S., young 
researchers have left for other countries or have left the 
field altogether.
    Fewer researchers means fewer scientific publications. How 
will a potential decrease in peer-reviewed journal articles and 
the global release of new scientific data impact the standing 
of the U.S. in global scientific communities?
    Dr. Gilliland. Thank you, Congresswoman. That is a great 
question, and it will have an impact.
    I was just in China a few weeks ago. And competition is a 
good thing. I think it spurs us all on. But if you want to see 
a very impressive scientific economy and worry about our world-
leading position, spend a few weeks walking around their 
universities and their biotech companies. It is really 
impressive. And I do have concerns around our maintaining our 
leadership role in that context.
    Mr. Yamamoto. And you might say that, relevant to my 
earlier comments about the global futures of this endeavor now, 
that it doesn't matter, that we just want science to move 
forward and it will all be okay. But it turns out that U.S. 
science has set the standard for the way that research should 
be done for discovery, for innovation, for really bold 
thinking. And for years, we were able to float along on that 
even if the budgets weren't very strong. But the other 
countries have realized that this is their ticket to being able 
to become really powerfully developed nations. And so they are 
pushing hard for this.
    And I think that what I come down to, when you think about 
this, is the imperative for U.S. science to maintain this top 
position. Because we have set the standards for the way that 
things are done. We have set, kind of, the behavioral ways that 
scientists act as colleagues and competitors to be able to try 
to move forward. And those elements are not really baked into 
these other cultures yet, and so I think it is really crucial 
that we maintain that role. And I think being mindful of your 
kinds of insights and questions is something that we really 
need to maintain going forward.
    Ms. Roybal-Allard. Thank you.
    Mr. Cole. Ms. Herrera Beutler.
    Ms. Herrera Beutler. Thank you, Mr. Chairman.
    You know, I wanted to ask Dr. Gilliland about ROI, and I 
will, but I want to also refer first to a comment you made. I 
think it was in response to Dr. Harris' question, but you were 
talking about getting private dollars from some of these 
foundations. And you said, specifically, ``I can't accept''--I 
think you were talking about Bill and Melinda Gates, but you 
said, ``I can't accept all the money because I can't afford 
to.'' And I was hoping you could elaborate on that really 
quickly.
    Dr. Gilliland. The Gates Foundation does use a different 
accounting method for calculating indirect-cost rates, so they 
do provide more than just the 20-percent support if you think 
about it from that perspective, that they do allow for certain 
infrastructure elements, for the global HIV vaccine trials 
network, for example. But they don't cover the full costs.
    So we need to make that up. And we have only two ways of 
doing that, sort of, as a research institute, which is to use 
philanthropy or to try to capitalize on some of the value that 
has been brought from our intellectual property and from 
spinout companies. But, all that being said, we put in about 
$69,500,000 last year from philanthropic sources towards 
balancing our indirect-cost rates.
    There are grants that one of our divisions would love to 
have from the Gates Foundation, and I have to say, no, we can't 
afford to do that unless I get a little bit better at 
fundraising. And I am working on that. But that is a sad thing 
for me, because these are great projects. But we have to 
prioritize which are the ones that we think are really 
important. Because we do lose money on them, and we will make 
that up. But the things that are really important, like a 
vaccine for HIV, we are out there trying to support.
    Ms. Herrera Beutler. So it is costing you--so you have 
researchers who want to do certain things and want to go after 
money, but you can't necessarily cover all the back-end costs. 
You can't allow them to just go for it, basically.
    Dr. Gilliland. Well, that is correct, and that is true for 
foundations. So we do have a prioritization process that 
evaluates any project that has unrecovered F&A, because we have 
to make that balance up to be in compliance with Federal 
regulations. And there are things we just say, no, you can't 
apply for this, because, even though it is a cool idea, 
collectively, as an institution, we have to prioritize that 
against things that we think are really important to our 
mission and our strategic plan. So, yes.
    Ms. Herrera Beutler. And to what role the NIH funding is 
going to play, keeping whole the other pieces?
    Dr. Gilliland. I am not sure I understand.
    Ms. Herrera Beutler. So, like, if that money isn't there--
or maybe I am not understanding it right--to basically cover 
costs--like, if--how much can you backfill with other, 
whether--you said fundraising or philanthropy.
    Dr. Gilliland. If we had an infinite amount of 
philanthropic support--and there is a lot of wealth in the 
Seattle area, so we are working on that--we could open our arms 
and say, do anything that you want to do. But there are 
projects that we have to prioritize as not being high enough 
priority; we just can't afford to do that in a fiscally 
responsibility way.
    Ms. Herrera Beutler. On the ROI front, obviously, we have 
heard that the Federal resources--all resources are very 
precious, and we want to steward those as appropriately and 
correctly.
    Can you please discuss a little bit about how the Hutch is 
promoting, kind of, the ROI concept?
    Dr. Gilliland. Well, thank you for that question.
    We are trying to do everything that we can to publicize the 
return on investment. To Congressman Simpson's point, it is 
really important to make sure that the NIH is included in that.
    So, whenever we talk about the Women's Health Initiative 
that was funded by the NIH, we talk about the value 
proposition, the return on investment of $37,000,000,000 and 
lives saved, primarily from a reduction in breast cancer risk 
but also cardiovascular risk. We make sure that the NIH is 
applauded for that, but also point out the ROI, which is: That 
was a $180,000,000 trial. It was expensive--160,000 women 
followed over 10 years. But the return on investment for our 
society is spectacular.
    I have mentioned the example with Denise Galloway and the 
HPV vaccine--huge return on investment worldwide for the work 
that she did. And the cooperative group trials, which in cancer 
sometimes take a hit, that data from the SWOG trials showing 
that there were 334 million years of patient life extended with 
the $418,000,000 that was spent on the trials. That is $125 per 
patient-year, which is extraordinary.
    So there is value there, but the other value, 
Congresswoman, is that, when we have the opportunity to be 
creative, we can think about strategies like taking a cancer 
patient's own immune cells out of their body, genetically 
reprogramming them to put in a laser guidance system and a 
molecular explosive device to seek out and destroy cancer 
cells. So we are getting cure rates in people who had death 
sentences for diseases like acute lymphoblastic leukemia, 93-
percent complete response rates, with this technology that is 
out-of-the-box thinking.
    That return on investment is harder to measure right now. 
There will be commercial value to that, ultimately. But the 
value is seeing women named Stephanie, for example, who was 
dead, I mean, literally a few weeks to live, now going out to 
meet with our donors and our philanthropists to say, ``Look at 
me. I shouldn't be here today.''
    That return for me, personally, that one life saved, is 
extraordinarily important. So we have a saying, ``No lives left 
behind.'' That is what we are trying to do in our approach to 
treating cancer. We have to show the value proposition 
economically to our Federal regulators and our government, but 
that is the kind of return that we are looking for.
    Ms. Herrera Beutler. Thank you.
    I yield back.
    Mr. Cole. We don't have much time left, because, frankly, 
this has been a really good panel, and the members have been 
really engaged, but I want to give everybody at least an 
opportunity for one more. So, if we could, I am going to drop 
this to 2 minutes. And then, obviously, Ms. DeLauro and I, if 
you have any final thoughts, we will have an opportunity for 
that too.
    I will forego my time, since we don't have a lot here, and 
I want to go directly to my friend, the gentlelady from 
Connecticut.
    Ms. DeLauro. If you could, one or two of you, we have 
alluded to the negotiating process for indirect costs with the 
appropriate Federal agency--the documentation, et cetera. Can 
you just walk us quickly through that process and what it 
entails so that we get some idea there of--and equate it to 
whether or not frivolous expenses can get uncovered or not 
covered. Just walk us quickly through the process so we get 
some idea of what you are going through.
    Mr. Droegemeier. Sure.
    So, at my own university, we go through a very extensive 
physical inventory of all of our space, for example. I mean, 
the book is about 4 inches thick. And we literally--every 
building, every room. And then we do an analysis of what 
portion of that room is dedicated to research, who funds that 
research, what portion of it is education, and so on and so 
forth. So we do the whole space analysis.
    Then we go through and we analyze all of our cost systems. 
We look at the HR system, we look at cost accounting, we look 
at the student records system, anything that is associated with 
any administration that touches research, including the 
library. We look at our debt service, the whole analysis of our 
so-called general ledger.
    That all goes into this massive amount of information that 
is then given to, in our case, the Department of Health and 
Human Services, the negotiators. And sometimes, frankly, it is 
a phone call, and after 30 minutes you say, ``Okay, well, here 
is what we have agreed to.'' And you think, ``Gee, we put all 
that work in it, and it just comes down to a phone call.'' Now, 
that is not an unusual circumstance, I think. But that is how 
the process plays out.
    And, in some cases, they will come and do a visit to your 
university. They will look it over in great detail. But if they 
have had some experience, some of the auditors, they come in 
and say, okay, yeah, we remember 2, 3, 4 years ago or whatever, 
we see that this is all consistent, so we will give you this 
rate, and it is over. Sometimes they will give you a 
provisional rate that goes for a few years and then they adjust 
it and so on.
    So it is as much of an art as it is a science, but it is 
based on rigorous analyses. And that is why, in my university, 
61.6 percent should be the actual rate. It is really negotiated 
to be 55 percent, so we lose to 6 percentage points, almost 7, 
in the negotiation process.
    It varies across the country. People have looked at this; 
the GAO has looked at this. Why is there different--you know, 
why is there a regional variability? Why is the ONR, in terms 
of how they do it, slightly different than HHS?
    Ms. DeLauro. Right.
    Mr. Droegemeier. So, as I think Dr. Gilliland mentioned, it 
is an imperfect process, for sure. And GAO just wrote a report 
a few months ago on this. So it constantly gets looked at, I 
think, in terms of the process.
    But I think it works pretty well, and it is getting better 
all the time. But it is a very laborious, time-intensive 
process.
    Ms. DeLauro. Right.
    And just very quickly, the opportunity for hiding, you 
know, a frivolous expense. You know, we went back all those 
years and talked about the yacht in San Francisco----
    Mr. Droegemeier. Right.
    Ms. DeLauro [continuing]. Whenever it was----
    Mr. Droegemeier. Right.
    Ms. DeLauro [continuing]. You know. But----
    Dr. Gilliland. Stanford.
    Mr. Yamamoto. Stanford.
    Mr. Droegemeier. Stanford.
    Ms. DeLauro. Stanford. Sorry.
    Mr. Droegemeier. Yeah, we are all quick to say who it was. 
Boy.
    Ms. DeLauro. I am sorry. I am sorry. I am sorry. Please. 
Yes. Right.
    It wasn't Connecticut, though, Dr. Liang, in any case.
    But is that--to the best possible--there is always the 
outlier, but is that--it is uncoverable if you are trying to--
--
    Mr. Droegemeier. Right. That is a great question. I put 
that very question to COGR. We heard COGR mentioned earlier. 
And what I was told was that, since that particular incident, 
there really have been no major findings.
    There was one university that had a $9,000,000 finding 
fairly recently, I guess, because they allowed research to be 
conducted in a building that should have been charged a 
different rate. It wasn't a fraudulent kind of thing. They went 
back and said, ``You know, you are right. It was in a building 
that was owned by the university, so it should have been just 
charged the administrative rate rather than the full F&A.''
    But, to their knowledge, that was the only, kind of, claim 
that had been found in the last 25 or so years. So I think the 
system is working pretty well in that regard.
    Ms. DeLauro. Okay.
    Does everybody concur, or is there----
    Dr. Liang. Yeah, I concur.
    Mr. Cole. I am just terrible at enforcing the clock today, 
but----
    Ms. DeLauro. You are.
    Mr. Cole  [contuinuing]. Okay.
    Dr. Liang. No, I don't really have anything more 
substantial to add. At UConn, we have a similar process, and 
there is no hidden illegitimate expenses.
    Ms. DeLauro. Uh-huh. Thank you.
    Thank you, Mr. Chair.
    Mr. Cole. Absolutely.
    Mr. Simpson, I am prepared to be tougher on you.
    After we are established, we go in order. So we will just 
go right down the panel here.
    Mr. Simpson. I don't have any other questions, but to thank 
you for your work and stuff.
    And you said that you would lose $10,000,000 in overhead 
costs? You don't know where you get it? Let me suggest you get 
it out of the university football program so the rest of us 
could compete with you.
    Mr. Droegemeier. I have to tell you, that is a great point, 
and our football program actually gives several million dollars 
a year to the academic programs. It is one of the very few in 
the country that does that. But we are still looking for a 
national championship.
    Sorry. Sorry.
    Mr. Cole. Just think how much we could plow into research 
if we win the national title this year.
    I want to go next to my good friend, Dr. Harris.
    Mr. Harris. Thank you very much.
    And, again, thank you all for being here.
    And, you know, Dr. Gilliland, thanks for your honest 
testimony about applying for outside foundation money. The fact 
of the matter is they don't pay as much indirect costs. And 
that was your testimony, although earlier testimony was, 
``Yeah, they all pay about the same.'' They don't.
    And any researcher in the field knows that is why your 
first grant goes to the NIH. It doesn't go to the Gates 
Foundation, it doesn't go to American Heart, it doesn't go to 
American Cancer Society, it goes to the NIH, because the NIH is 
the most generous funder.
    So the question before us is not whether we are going to 
fund it but whether the amount we fund is an appropriate 
amount.
    And I will just make one observation, because I just sat 
here thinking about, now, how can we use indirect costs to 
actually fund young investigators or to encourage it. And I am 
thinking, you know, the senior investigators I knew, they had 
nice--they had their laboratories, but when they added the 
fourth and fifth project into that lab, there was minimal 
indirect cost added into that lab. The university really--I 
mean, it was just gravy for the university. That fourth and 
fifth grant was just gravy.
    So the GSI was kind of maybe a crude attempt to go after 
it. But what if we just said, you know, your third, fourth, and 
fifth grant, we are going to just get capped indirect costs--
because, you know, by the time you get to the third, fourth, 
fifth grant in a laboratory, you don't really have all those 
costs--and then turn that money over and fund more--and, again, 
because we are Congress, we can actually direct how the NIH 
funds money. We could direct them to spend those savings on 
funding young investigators. Doesn't that work?
    I mean, again, you are shaking your head. Because, look, I 
get it. Universities love that third, fourth, and fifth grant 
from the senior investigator. The question is, are we going to 
get our clock cleaned internationally because we are willing to 
say we need to fund that--and, look, I will agree with Mr. 
Simpson. I came from an institution; they built a Taj Mahal 
research building in the late 1990s. I mean, this was the most 
beautiful building. It was wonderful to do research in. It was 
beautiful. But you had to pay the costs for 20 or 30 years, 
however long the debt service was. And that gravy train didn't 
run forever.
    So my only comment is, I think we can think of ways, and we 
should be clever thinking of ways, that we can fund young 
investigators and turn that clock--because we have not turned 
it back. The Director of the NIH has said we have only 
plateaued it. We maybe have stopped the progress, but we have 
certainly not turned the clock back to funding young 
investigators.
    And I yield back.
    Mr. Cole. Well, if somebody wants to make a response, I 
will certainly allow that.
    Mr. Harris. Absolutely.
    Dr. Liang. Thank you, Dr. Harris.
    One suggestion could be, which we are seeing more and more 
at UConn, is--we have those research millionaires, we call 
them, the many NIH grants. We have asked them to put young 
investigators as co-PI or multiple PI as a mechanism to get 
them supported under the supervision and tutelage of their more 
senior principal investigators.
    We actually think that is a better way to cultivate the 
next generation of young scientists, rather than capping the 
indirect costs, because I think that is really part of the 
overall cost. And we want to keep that by encouraging the young 
investigators to be multiple PI with the more senior ones as a 
way to grow the next generations of scientists.
    Dr. Gilliland. And I would only add to that, Dr. Harris, 
that, if you will recall the scoring system for the GSI, it 
actually penalized people that went in as co-PIs, that you got 
more points than you should if you had a single RO1 site.
    There are some fixes, potentially, in the ways we look at 
it, because I think Bruce is right, that you want to try to 
encourage senior investigators to mentor junior investigators 
in how you write a grant and how you put it together, and being 
co-PIs is a fantastic way to do that.
    Mr. Yamamoto. I want to just go back to the comments that 
Dr. Droegemeier so beautifully put forth in the beginning, and 
that is that--so, first of all, Dr. Harris, as you know, 
faculty members don't apply for NIH grants first because they 
give higher indirect costs or F&A they apply for their NIH 
grants because that is where most of the money is.
    And so, to go back to this compact that has been made 
between the Federal Government and the economic research 
community, the power that that compact has had because the 
Federal Government has maintained that agreement and been loyal 
to it is just immeasurable. I mean, it is a huge impact.
    And so, yes, it is true that, at the outset, the Federal 
Government said, ``Yes, we need to contribute to this. We will 
do this on a cost-sharing basis.'' As I said, universities and 
research institutes are the second-highest funders of 
biomedical research, behind the NIH. So that compact has been 
maintained in a very powerful way.
    And so the fact that the government started with this 
principle that it is important to do this, make this kind of 
cost-sharing agreement, in order to drive forward the research 
endeavor, and the fact that foundations may not share that same 
commitment but, instead, are able to say, isn't it great that 
the Federal Government has done what it has done, that public 
funds have been made available in the way that they have been, 
that they have built this fantastic super-structure that is 
generating so much impact in our society, and so we are going 
to come in on top of that and be able to supplement it in that 
way.
    It is leveraging for every partner--for the foundations 
that are able to come in and support research in a building 
that has been put up in part with NIH funds and not have to do 
that themselves; certainly for the government that has made 
that commitment that now sees that there are commitments from 
private philanthropy, from industry in some cases, to be able 
to add on to that. Everybody is leveraging each other's 
efforts, and it is really because the NIH started in the way 
that it did.
    Mr. Cole. Thank you.
    Mr. Droegemeier. Could I just really quickly----
    Mr. Cole. Real quick.
    Mr. Droegemeier. I think your presumption is very 
reasonable, but I think, in fact, just the opposite happens. 
When a faculty member becomes a senior investigator and they 
get more and more grants, their lab actually grows. They hire 
post-docs, more students. They hire collaborators, researchers. 
Staff come in. And so it actually becomes more expensive to run 
their labs. It is not a steady state, where they get grant 
after grant and the sunk costs are already there. They are 
constantly growing their facilities, because they are senior 
and they are doing more research.
    So I think it is not that they have been there and done it 
and they are just in a steady state. No, they are actually 
increasing. Those costs never go away, because the acquisition 
of equipment, that is not an indirect cost; that is a direct 
cost.
    Mr. Cole. Thank you.
    Ms. Herrera Beutler.
    Ms. Herrera Beutler. My last question was kind of along--I 
think some of it has been answered, but I wanted to give Dr. 
Gilliland and anybody else, I guess, if you have maybe kind of 
a closing comment or something.
    Dr. Harris always brings up good questions, and we didn't 
always have enough time to have you respond, but it is helpful 
for me to see that exchange. And so, if there is something that 
maybe you didn't have a chance to cover, I am kind of just 
throwing it open.
    Or not.
    Mr. Cole. Wow. That is proof positive it has been a very 
thorough hearing, I guess.
    But I want to go to my friend, the ranking member, for any 
closing thoughts she might have.
    Ms. DeLauro. I appreciate that very, very much, Mr. 
Chairman.
    And I would like to just make reference, if I can--it is 
just a statement, no question--to Dr. Droegemeier's paper, 
which is really--you know, all the papers are great, don't 
misunderstand, but the history, for me, personally, was very, 
very informative.
    And we began by saying that virtually all research in 
higher education was funded either by philanthropy or private 
foundation. We then found that the institutions of higher 
education had no interest in receiving Federal funds for 
research owing to fears of government intrusion in curricular 
research topics and governance--always a fear.
    We then move to 1933, where we do say that, you know, the 
National Research Council, National Academy of Science involved 
in creating the concept of this partnership calmed the fears 
among academics that funding decisions would be made by experts 
and peers and not by bureaucrats.
    And 1940, Franklin Roosevelt, the direction he went, where 
the point was organizing and sponsoring academic and industrial 
research for national defense, which was the watershed moment, 
as you point out, the seed of a decades-long partnership, U.S. 
Government, U.S. higher education institutions in conducting 
research and development for the public good.
    What has been created here is the opportunity which has 
made us the leaders in the world in discovery and in saving 
lives. Above all, we need to preserve that decades-long 
partnership. We can fix around the edges. We cannot slash and 
burn what that has meant to date in the lives of the people of 
this country but, most importantly, what it means for the 
future of discovery based on science, not government intrusion 
in that process, in order to be able to safeguard the public 
and their lives.
    Thank you for what you do. Hold steadfast, my friends.
    Mr. Cole. Well, let me--I am not going to try and top that. 
My friend is a brilliant communicator. But I do want to begin 
by thanking all of you for being here. I thought this was an 
exceptionally thoughtful and, for this committee, a very, very 
informative process. So I really appreciate you taking your 
time out to come and travel a great distance for all of you, 
really, and to help educate us so we make decisions here.
    And I also want to make a couple other closing remarks. And 
I want to be careful how I say it, because I don't intend to be 
critical of the administration. This administration I support. 
I didn't support this particular proposal, but, broadly, I am 
supportive.
    But, you know, I have been around budgets a lot, and I used 
to help write budgets at the State level, and if you have been 
an appropriator, you deal with it. And, look, this really 
wasn't, in my view, originally from OMB, about getting more 
bang for the buck in research. If it were, you would have left 
the money the same and just lowered the reimbursement rate. 
Instead, we lowered the reimbursement rate, and we took the 
money and directed it someplace else.
    And we do that in budgets all the time. That is a 
reasonable thing to do. In this case, that money was directed 
towards defense, and I don't disagree with that. I disagree 
with this particular part of it, but I don't disagree with 
beefing up defense. I think it is actually something that has 
to be done. And I recognize they had very little time in which 
to work, and you are looking for savings where you can get 
them, and you have a priority, and so you are going to fund 
that priority.
    I will add for the record, it is interesting to notice they 
didn't propose this same 10-percent cap for defense research, 
which is at least as big as biomedical research. I mean, 
actually, if you look at this, they are pretty comparable, in 
terms of the cost ratio, what DOD does and what NIH does. So, 
if this is really true, then you would want to apply this to 
defense research as well as biomedical research out of NIH.
    And I would just urge--and I have had this discussion with 
my friends at OMB and with the administration. They need to 
look at NIH and Center for Disease Control like defense, too, 
because it just really is. You are much more likely to die in a 
pandemic than a terrorist attack.
    And I hope we don't have any terrorist attacks. I know 
every American hopes that. But I will guarantee you, in the 
next 4 years, there will be another Ebola, there will be 
another SARS, there will be another Zika. So maintaining a 
robust capability here at all times--because you can't create 
it overnight any more than you create a defense overnight--I 
think is really, really important for the security of the 
American people. And it is really important for us that we get 
this balance right.
    I know when I took this job and my counterpart over in the 
Senate, my good friend and our former colleague, Senator Blunt, 
we had seen in this area we had been flat-funded for a dozen 
years. And, you know, there is all this talk about setting 
goals, and Senator Blunt is a very wise guy. He said, you know, 
if you set a goal in government, like we are going to double 
it, then when you get there, you quit. And that is basically 
what happened the last time we did this.
    So we had a really good discussion, what should our goal 
be. And our goal should be sustainable investment over time, 
you know, something that is regular. And that is what we tried 
to do the last 2 years on a very bipartisan basis, I might add. 
Our friends, the Democrats, agree on this, as well. And that is 
what we are going to try and do if we are able to come to a 
larger agreement this year. And I think that is very important.
    Within that, I do think you are always looking for 
economies and savings. And, you know, I think Dr. Harris' 
contribution here is a real one, because this is a discussion 
that needs to be had.
    And I appreciate those of you that pointed out some of the 
administrative costs, that while we have kept your cap 
constant--again, I commend you, really, that 31-page, you know, 
miniature masterpiece, because it is really good. But look at 
the explosion in mandates while the costs stayed the same. And 
that is actually an area I think we can all come together on.
    And as a number of you pointed out in your testimony, we 
have studies underway. Our friends on Energy and Commerce have 
done some really good work on this in 21st Century Cures. And 
we need to do that, because we do have very restricted 
resources. I mean, we do have a budget deficit that is 
beginning to move the wrong way because we won't do entitlement 
stuff, and that is going to crowd out all of these type things 
where there is defense on one side of the discretionary ledger 
or it is biomedical research on the other. These are important 
national priorities too. Everything can't just be Social 
Security, Medicare and Medicaid, and the remaining entitlement 
programs. That is 70 percent of our budget now. So, sooner or 
later, we are going to have to go there.
    But these investments really do save lives. And this 
partnership--and I really appreciate all of you that talked 
about that. Because, you know, as Dr. Yamamoto said toward the 
end, look, we all leverage one another's money in this, and 
that is one of the reasons why it has been so productive. It is 
productive for philanthropy and private enterprise. It is 
productive for the Federal Government. We don't have to go out 
and build the labs; you guys have already done it. You know, 
your taxpayers or your contributors or your donors have ponied 
up for part of the cost of what is really important national 
work.
    So I just want to make sure as we go forward that we don't 
throw out the baby with the bath water, you know, looking for 
savings and disrupting what is really a pretty complex, now-70-
year-old ecosystem that has produced extraordinary benefits for 
the American people and extraordinary benefits, to be quite 
frank, for all of mankind. This is one of America's great 
contributions to humanity. A lot of the cures that are found 
here don't stay here. Thank goodness, they go all over the 
world, and they help lots of folks that aren't fortunate enough 
to have the resources that this country has been able to 
generate, both financial and intellectual, to actually do this 
kind of work.
    So thank you for coming here. It is amazingly helpful to us 
to get your testimony and benefit from the insights of a 
lifetime that each of you have dedicated to research and each 
of you have dedicated to making life better, frankly, for your 
fellow Americans and for all of humanity. So we very much 
appreciate you being here.
    And, with that, we are closed.

                                       Wednesday, October 25, 2017.

 OVERSIGHT HEARING--DOWN SYNDROME: UPDATE ON THE STATE OF THE SCIENCE 
       AND POTENTIAL FOR DISCOVERIES ACROSS OTHER MAJOR DISEASES

                               WITNESSES

                               Panel One

HON. CATHY McMORRIS RODGERS, A REPRESENTATIVE IN CONGRESS FROM THE 
    STATE OF WASHINGTON
HON. CHERI BUSTOS, A REPRESENTATIVE IN CONGRESS FROM THE STATE OF 
    ILLINOIS
HON. PETE SESSIONS, A REPRESENTATIVE IN CONGRESS FROM THE STATE OF 
    TEXAS

                               Panel Two

MICHELLE SIE WHITTEN, FOUNDER, CEO, AND PRESIDENT, GLOBAL DOWN SYNDROME 
    FOUNDATION
DR. WILLIAM MOBLEY, EXECUTIVE DIRECTOR, DOWN SYNDROME CENTER FOR 
    RESEARCH AND TREATMENT, AND FLORENCE RIFORD CHAIR OF ALZHEIMER'S 
    DISEASE RESEARCH, UNIVERSITY OF CALIFORNIA--SAN DIEGO
DR. JOAQUIN M. ESPINOSA, EXECUTIVE DIRECTOR, LINDA CRNIC INSTITUTE FOR 
    DOWN SYNDROME UNIVERSITY OF COLORADO SCHOOL OF MEDICINE
FRANK STEPHENS, QUINCY JONES ADVOCATE, GLOBAL DOWN SYNDROME FOUNDATION
    Mr. Cole. We are going to go ahead and convene the meeting 
and Chairman McMorris Rodgers will join us in just a few 
minutes. I didn't want to keep the other members waiting.
    Before we start, however, I want to recognize my good 
friend, the gentleman from Arkansas, for the purpose of an 
introduction.
    Mr. Womack. Thank you, Mr. Chairman. And if it please the 
committee, I would like to, first of all, disclose that some 
time around 11 o'clock I have to depart early for a previously 
scheduled meeting.
    But I do want to take just a moment to recognize a young 
lady in the audience today. And each one of my colleagues have 
been provided a nice glossy bio of this young lady, but for the 
benefit of the rest of the audience and for hearing purposes, 
to recognize Mary Lorraine Wood Borman.
    Now she is in the back. Mary is a freshman at the 
University of Arkansas.
    [Applause.]
    Mr. Cole. We don't have to holler ``sooie'' or anything 
like that.
    Mr. Womack. We don't have to call the hogs today. Given our 
record in the SEC right now, we probably shouldn't be calling 
the hogs right now.
    But that said, as the information provided to each of my 
colleagues so states, this is a very special young lady, doing 
very well in her college studies at the University of Arkansas. 
And while she is not on the dais today and not one of the 
people testifying, she has just a terrific story.
    So I commend her biographical information to each one of my 
colleagues. And I want her to know how honored and privileged 
we are today to have her in our audience.
    So, Mary, thank you for how you represent our great State 
and this cause. And welcome.
    Thank you, Mr. Chairman. I yield back.
    Mr. Cole. Thank you.
    Good morning. It is my pleasure to welcome all of you to 
the Subcommittee on Labor, Health and Human Services, and 
Education for a hearing to discuss the state of science for 
Down syndrome research. This hearing is timely as October is 
Down Syndrome Awareness Month, which is a time to expand 
awareness of the accomplishments and abilities of people with 
Down syndrome.
    Today we will have two panels of witnesses. The first panel 
includes three fellow Members of Congress who are tireless 
advocates for improving the lives of individuals with Down 
syndrome. The second panel includes researchers and advocates 
who are committed to expanding our knowledge about Down 
syndrome and related disorders and improving the quality of 
life for Americans with Down syndrome.
    Research on Down syndrome is increasingly important because 
as the number of births of babies with Down syndrome increases 
and the life span of individuals with Down Syndrome expands, we 
are likely to see a large increase in the number of Americans 
living with Down syndrome.
    Research targeted at further understanding this disorder 
will contribute to improving the lives of individuals with Down 
syndrome. Additionally, recent discoveries suggest that Down 
syndrome research has the potential to contribute to our 
knowledge of a variety of other diseases and disorders that 
affect all Americans. We will hear more about this exciting 
research today.
    I am pleased to introduce the witnesses to our panel. It 
seems odd to read your introduction, Mr. Chairman, but I am 
going to do it.
    Mr. Pete Sessions is chairman of the House Committee on 
Rules, and I am proud to say I am his vice chairman on that 
committee. He is the proud father of a young man by the name of 
Alex, who we all have met on many occasions, an outstanding 
young man who has Down syndrome.
    Mr. Sessions has been a passionate advocate and leader in 
Congress for people with disabilities. As the lead sponsor, he 
worked tirelessly to enact the Family Opportunity Act in 2006, 
which gives States the option to create a Medicaid buy-in for 
low- to moderate-income families with children with 
disabilities.
    He is the co-chair of the Congressional Down Syndrome 
Caucus, serves as an adviser to the President for Special 
Olympics Texas, and has served as a board member of the Best 
Buddies International. And all of us know him as genuinely one 
of the outstanding leaders of Congress.
    Mrs. Cheri Bustos represents Illinois' 17th Congressional 
District and is the co-chair of the Democratic Policy and 
Communications Committee. She is also the co-chair of the 
Congressional Down Syndrome Caucus. Again, a distinguished 
member with an extraordinary reputation.
    I will introduce Cathy even though she is not here. Mrs. 
Cathy McMorris Rodgers is eastern Washington's chief advocate 
in Congress. As chair of the House Republican Conference she is 
the fourth-highest-ranking Republican in the House and the 
highest ranking woman in Congress.
    In 2007, she gave birth to Cole Rodgers. Cole was born with 
Trisomy 21 and inspired McMorris Rodgers to become a leader in 
the disabilities community. She is also a co-chair of the 
Congressional Down Syndrome Caucus and played an instrumental 
role in securing passage of the ABLE Act in 2014, which created 
tax-free savings accounts to empower individuals with 
disabilities to save and invest in their futures.
    After that, we will move to the second panel, and just to 
get the introductions done now, a little bit early.
    Ms. Michelle Sie Whitten is the co-founder, president, and 
CEO of the Global Down Syndrome Foundation, as well as the 
executive director of the Anna and John J. Sie Foundation, 
which is the largest private funder of grants for Down 
syndrome-related research and programs.
    Dr. Joaquin Espinosa is the executive director of the Linda 
Crnic Institute for Down Syndrome. He is also a professor in 
the Department of Pharmacology at the University of Colorado 
Anschutz Medical Campus School of Medicine.
    Dr. William Mobley is a distinguished professor and chair 
of the Department of Neurosciences at the University of 
California, San Diego. He also serves as the executive director 
of UCSD's Down Syndrome Center for Research and Treatment, and 
is the Florence Riford Chair of Alzheimer's Disease Research.
    And Mr. Frank Stephens was awarded the Quincy Jones 
Excellence in Advocacy Award by the Global Down Syndrome 
Foundation in 2016. As an advocate, he has spoken all over 
North America and Europe, promoting the inclusion of 
individuals with intellectual disabilities.
    As a reminder to everybody, when the regular panel gets up 
here, the star--the star--of course I have the stars in front 
of me--I will pay for that later, I am sure--but when the 
expert witness panel gets here we will be operating by the 5-
minute clock. But our colleagues obviously have very busy 
schedules of their own, so I am going to allow them to give 
their testimony. We will open it up just broadly for any 
comment or question anybody cares to include. We won't hold 
them to the normal rigor of our routine so they can get about 
their business.
    With that, Mr. Chairman, it is a delight to have you here 
as my good friend and a recognized leader in this area, a 
strong voice for people with disabilities across the spectrum 
in our country. And the gentleman is recognized for whatever 
opening remarks he would care to make.
    Mr. Sessions. Chairman Cole, thank you very much, and 
Members of the Committee, I am delighted to be before you, not 
just my colleagues----
    Mr. Cole. I forgot, I wasn't paying attention. My good 
friend, the ranking member, had an opening statement. So I 
apologize very much for that. I apologize to my friend.
    Ms. DeLauro. Thank you very much, Mr. Chairman. And I 
apologize to my colleagues as well, but it is a delight to have 
you here today. It is a distinguished panel.
    We really do welcome you to the Labor, HHS Subcommittee and 
look forward to your testimony because just a heartfelt thanks 
for your dedicated work in collaboration with this wonderful, 
wonderful community and a generous community in this important 
research and efforts to look into Down syndrome.
    I am also pleased as well to welcome our second panel, Dr. 
Mobley, Dr. Espinosa. But I want to recognize Michelle Sie 
Whitten, president and CEO of the Global Down Syndrome 
Foundation.
    What an enormous impact on the lives of people with Down 
syndrome that you have made through research, medical care, 
education and advocacy.
    And Michelle's family is with her, her extended family is 
with her today, mom and dad, in-laws, daughter Sophia, son 
Patrick.
    And I have to say this, Mr. Chairman. Her mom is from 
Naples, from Napoli. So I have got to recognize my own roots in 
this effort.
    So it is wonderful to see you again.
    Last but not least, obviously, is Frank Stephens. And, 
Frank, thank you for your dedicated advocacy and for sharing 
your experiences today.
    I also want to recognize, as my colleague from Arkansas 
did, constituents from New Haven, Connecticut, and that is 
Stevie, Lisa and Leah Stevenson. Why don't you just stand for a 
moment? So they are here today advocating for Down syndrome 
research.
    So, you know, it is wonderful and enlightening, I think, 
for American people to see the ways in which we do debate 
issues here. We hear from experts and enact legislation in this 
body. And I am grateful that they are here today.
    I am looking forward to today's hearing. As the witnesses 
made clear in their written testimony, the potential for 
scientific breakthroughs related to Down syndrome has never 
been greater than it is today. And those breakthroughs are so 
important as individuals with Down syndrome face higher health 
risks than the rest of the population, including additional 
risks for congenital heart problems, vision problems, and 
leukemia.
    In addition, as they age, those affected by Down syndrome 
have an elevated risk of developing dementia related to 
Alzheimer's disease. As Dr. Mobley notes in his written 
testimony, all of the pathological manifestations of 
Alzheimer's disease are present in the brains of those with 
Down syndrome by age 40. He adds, by age 60, 90 percent of 
individuals with Down syndrome will suffer the effects of 
dementia.
    Those are heartbreaking statistics for individuals with 
Down syndrome, for their families, and for all of us. And as 
Frank Stephens says, let's make our goal to be Alzheimer's 
free.
    At the same time, the same time, individuals with Down 
syndrome are unlikely to have heart attacks, hypertension, or 
solid tumor cancers.
    Dr. Espinosa mentions a different, quote, ``disease 
spectrum'' in the population with Down syndrome. As our 
panelists can attest, we are discovering that individuals with 
Down syndrome could help to identify cures or treatments for 
diseases that continue to kill millions of Americans, including 
cancer and cardiovascular disease, which is why I have the 
honor to advocate and to include report language in the Labor, 
HHS bill that would encourage the NIH to explore a trans-NIH 
initiative to better understand the molecular, cellular, and 
physiological mechanisms that predestined individuals born with 
a third copy of human chromosome 21, or Trisomy 21, to be 
vulnerable to a range of diseases that cause nearly 60 percent 
of deaths today in the U.S.
    This is important work that should not be siloed in a 
single NIH institute. As we have learned from witnesses, our 
testimony this morning, we will further explore this in the 
hearing, the promising research crosses a wide spectrum of 
health conditions and should be pursued by experts in each of 
those research disciplines.
    The research presents an opportunity to make scientific 
advances that will improve the health and quality of life of 
hundreds of thousands of Americans with Down syndrome and 
possibly to discover the keys to preventing diseases that 
affect millions of American families every year.
    Our panelists, one of them, Michelle, calls this, quote, 
``therapeutic leverage.'' I think it is a great term, and I 
hope we can use that leverage to improve the lives of 
individuals with Down syndrome, as well as millions of others 
who suffer from debilitating diseases.
    Mr. Chairman, in the interest of time and our colleagues' 
time and the panel's time, I will just only make reference, 
what I usually make reference to at this point, which is that 
even while we have at the NIH $34 billion, thanks to two 
consecutive $2 billion increases, our increases have not kept 
pace with biomedical research, and our budget has declined at 
the NIH by about $6.5 billion since 2003 when we adjust for 
inflation.
    So with that, our work is cut out for us to increase that 
funding and find the ways in which to do it for individuals 
with Down syndrome and for all the scientific discoveries that 
we can make studying the disease spectrum. We need to take a 
look at the investments that we do make into research through 
the Federal Government.
    Welcome to my colleagues and welcome to the panel that 
follows. Thank you so much for being here this morning.
    And thank you, Mr. Chairman.
    Mr. Cole. I thank the gentlelady for her testimony. I again 
apologize to her for just plowing ahead there. I didn't mean to 
do that.
    Ms. DeLauro. Oh, please, no apologies.
    Mr. Cole. Again, I want to start once again with my good 
friend, the chairman of the Rules Committee, a distinguished 
advocate for people with disabilities, and a very good personal 
friend.
    Mr. Chairman, you are recognized for whatever remarks you 
care to make.
    Mr. Sessions. Chairman Cole, thank you very much, Ranking 
Member DeLauro, thank you very much, and to the committee 
members who are taking their time to attend this important 
hearing, I want to thank each and every one of you.
    I come to the table today as a father, I come to you as a 
Member of Congress, I come to you as a Member of Congress that 
is interested in policy that would be good for a lot of people, 
but good for America.
    I come to you today to give you some insights. As a result 
of what the chairman and the ranking member are doing today, it 
will open many eyes and ears of people across this country who 
have recognized people who are in their community, people who 
may go to their church, maybe a next-door neighbor, but it 
opens up the eyes of the general public about how important 
each of our people are who have Down syndrome.
    They are persons with Down syndrome and they are people who 
were born just like you and I, but were selected in a different 
way by God to be special people. Some people refer to them as 
angels. But they are people who are in our midst who very much 
want and need not just to be respected, but to be included in 
the general, I think, attributes of society to make their lives 
better too.
    And I think what you will hear over and over today from not 
only the families and the advocates, but it is a recognition of 
how important it is for Congress to turn not only their resolve 
into making life better for them, but by helping all people.
    So today I will tell you Alexander Gregory Sessions, my 
son, who is 23 years old, cannot be here today. Here he is as 
an Eagle Scout with me. And Alex, then a year or so ago, we 
went to an SMU football game and he had a chance to be there. 
Alex works for Home Depot. So the business community has caught 
on it that there are attributes about each of our people that 
might include them in a work relationship.
    Today we are talking about a number of factors, including 
how the NIH might be more involved in the research, in 
advancing the things that we know that exist within our Down 
syndrome community, about how it can prepare all of us for the 
future. But what I would like to say to you is that there are 
facts and factors about our advancements that have made life 
better.
    You will hear Dr. Mobley today, my very dear friend, who 
for a number of years has been really my adviser, my pusher, my 
guide that I pray for because he makes advances. And what Bill 
would tell you, in the fifties, the average age of a Down 
syndrome person that lived would be 9 years old. In the 
eighties, it went to 25. It is now almost 60. And that is 
because the attitudes of the American people, including the 
medical community, who would look at a person and think that 
perhaps they did not have the tools to adequately take care of 
them, perhaps it was because of maybe some bias maybe on some 
intellectual ability model, perhaps it might be from their own 
bias of not really understanding the importance of that person.
    What I want to suggest to you today is by you taking the 
opportunity to hear our story, you will see that there are 
people that are behind me that are all over our great land. 
They are families who work in their Down syndrome societies 
back in their homes. They are people who when a new baby is 
born will make calls to that family and say, as I have done 
many times, including to Cathy McMorris Rodgers when Cole was 
born, that this is a gift, that this is an opportunity as a 
parent to accept a relationship with a special person and to 
try and grow them to not only a lifetime of activity, but one 
that we would better their lives and so many others.
    Alexander Gregory Sessions, while he is an Eagle Scout, I 
think he taught each of those boys as much about life and about 
the opportunities as any scouting experience that they have. 
But I will also tell you that Alex now at age 23 does not 
remember a day in scouting. Some of the greatest parts of my 
life and his life he can't even remember.
    And so it tells me and us that we need to turn to research. 
We need to turn to research, not just the medical community, 
but the researchers at NIH and those who are like Dr. Mobley 
and Dr. Espinosa, who can look at the general population of 
Down syndrome people and begin working on those intellectual 
areas, those areas not only of the brain, but of the receptors, 
the synapse, and dendrites that allow the brain to function and 
to talk back and forth to each other.
    And so that is the specialty type of work that you as 
members of this Appropriations Committee get into. I encourage 
you and push you and applaud you to please understand that what 
is here behind us today is embodied by the co-chairman of our 
Down Syndrome Caucus. But we are asking you to please know how 
important today's hearing is and to thank each and every one of 
you.
    As I look at this committee, Mr. Chairman and Ranking 
Member DeLauro, I see not only colleagues. I see kind people 
who recognize that research and development, that the R&D 
models that us pushing, not just oversight, us pushing the NIH 
in directions that we believe are important, aids and helps 
them to not only develop their content, but to work forward.
    Lastly I will say this. Dr. Francis Collins and I are very 
dear friends and I have asked and received Dr. Collins six 
different times to come up to the Rules Committee, Chairman 
Cole has been there, Ranking Member DeLauro has been there, as 
we talked about issues that the NIH handles. They need $300 
million, that is it, $300 million more a year to give them a 
better opportunity to grow the scientific community of young 
people who have gone through college and maybe give them their 
first taste of an opportunity to be a leader in this area and 
for us to grow our young people.
    So I would say not only are we here with the opportunity, I 
think, to discuss Down syndrome, but overall, Mr. Chairman, as 
you look at a pot of money that might give the director, 
Director Collins, a chance to offer seedlings, evidence that we 
can grow the number of researchers and investigators, it would 
be helpful.
    I want to give my thanks to each of you on this committee, 
not only for your kindness to be here today, but your 
thoughtfulness as you make tough decisions about the direction 
of this country. And I trust that not only will you do that 
well, but this body will support your activity.
    Mr. Chairman, I would ask unanimous consent that anything I 
brought in writing would be available today.
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    Mr. Cole. Without objection.
    Mr. Sessions. Thank you, sir.
    Mr. Cole. Thank you, Mr. Chairman. And I suspect that young 
Alex learned something from his dad, who was also an Eagle 
Scout.
    Mr. Sessions. Yes, sir. Thank you, sir.
    Mr. Cole. Good to have you here, my friend.
    We will next go to Mrs. Bustos. And a delight to have you 
here.
    And I just want to point out for the record, and my 
colleagues know this and I am sure the audience does, but we 
are quite often accused of being very partisan and polarized in 
Congress and to some degree that is a legitimate remark and 
observation. But there are things that bring us together. This 
cause is one of them. And I am delighted to see a bipartisan 
delegation here to advocate on behalf of people with Down 
syndrome and their families.
    So my colleague is recognized for whatever opening 
statements she cares to make.
    Mrs. Bustos. Great. Thank you for mentioning that, Chairman 
Cole. I want to thank you and Ranking Member DeLauro for having 
us here, for hosting this. I want to thank the beautiful people 
behind us, the advocates, the medical professionals, and the 
people who are here with Down syndrome. And thanks for all of 
you for taking the time today.
    I know you all get this, but back home, in my State of 
Illinois, I have mostly a rural district. We teach our own 
kids, our sons and our daughters, to make that we are lifting 
each other up to build strong communities and to make sure that 
everyone, everyone has a chance to succeed, no matter where 
they come from. So it is important to me that we treat everyone 
with value.
    And it is in that capacity that I am proud to be one of the 
co-chairs of the Congressional Task Force on Down syndrome, 
along with these two Members of Congress here, because it is 
the right thing to do. So it is a pleasure to be with you 
today.
    I have met some absolutely wonderful families and wonderful 
children, wonderful adults with Down syndrome through this task 
force. And I am going to share one of my favorite stories, and 
that was back in 2014 when I think just about everybody sitting 
up there, and certainly the three of us, the day that we voted 
for the ABLE Act.
    And a colleague of mine had brought a young lady named 
Briana on the House floor. If you are under a certain age we 
are allowed to bring children on the House floor with us. And I 
just, like, instantly had this bond with Briana and later found 
out that she had some roots in Illinois. And so we were 
together, and as some of us do when there is children with us, 
we will put our little card into our voting booth and we will 
say, ``Do you want to press the `yes' button?''
    And so Briana was there with me and I said, ``Why don't you 
press the `yes' button?'' And it was that very vote that took 
us over the number that we needed to pass the ABLE Act. And so 
as soon as she pressed that button, the whole gallery, if you 
remember that, went nuts, and it was with that vote that the 
ABLE Act passed. So to this day I keep in touch with Briana's 
family, with her mom and her dad.
    And I know most people here know this already, but for the 
record I want to talk a little bit about some things that I 
hope that we all understand about Down syndrome. We know that 
when someone with Down syndrome walks into the room, that they 
light it up. We know that they often live long, happy, 
successful lives, as Congressman Sessions talked about Alex and 
his new job at Home Depot, 1 week into his new job.
    Mr. Sessions. One week in.
    Mrs. Bustos. And so we are very pleased to hear about that. 
But there are also some other facts about Down syndrome that 
are leading to this debate that we are having today and this 
discussion that we are having today.
    So while the National Institutes of Health funding has seen 
some tremendous growth over the last 20 years, the funding for 
Down syndrome research has remained somewhat flat. So let's 
talk about that a little bit.
    Just yesterday I spoke with Briana's dad, Brad, and he 
shared with me that his entire family got Lyme disease about 6 
months ago. And so while Briana's mom and dad have recovered, 
made pretty much a full recovery, Briana is still having a 
rough go of it.
    And so this is 6 months after her diagnosis and she is 
still struggling to walk as a result of this. So her dad Brad 
has to carry her now to the kitchen table, to the restroom, to 
go to bed at night.
    And it is because people with Down syndrome have a weaker 
immune system, and there is really little research into that 
part of the condition. So today's testimonies will show why it 
is so important to fix that.
    People with Down syndrome have some remarkable differences 
in their health compared with the rest of us, and Ranking 
Member DeLauro mentioned that in her opening remarks, but they 
are nearly immune to some of the most common health threats 
that the rest of us deal with. They are naturally resistant to 
almost all forms of cancer, they almost never suffer heart 
attacks, and high blood pressure is almost entirely unheard of.
    So developing a better understanding of Down syndrome may 
help scientists find breakthroughs to fight cancer, to address 
heart attacks, which affect so many of our own loved ones.
    Additionally, we need to do more with those with Down 
syndrome lead longer and healthier lives. Congressman Sessions, 
I didn't know that back in the fifties that the average age was 
only 9, and so we are making obviously some tremendous progress 
in that. But we need more. We want that people with Down 
syndrome live well past their sixties.
    And we talked about dementia and Alzheimer's, and your own 
son's memory already only being in his twenties. But right now 
almost 100 percent of people with Down syndrome will display 
some kind of signs of Alzheimer's by the time they are in their 
forties.
    So we have got to make all of these a challenge to address 
and one of our top priorities as we look at this. I really do 
believe that with a stronger commitment from Congress these are 
some of the issues that we can tackle together, to your point, 
Chairman Cole, in bipartisan fashion. This is something that 
certainly does not need to have any kind of Republican or 
Democratic label when we want to address something like that.
    I am here to say that I want to be a partner in addressing 
this, and I am very pleased to be up here with my colleagues.
    And with that, Mr. Chairman, I yield back.
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    And as those of you in the audience know, you have some 
very passionate and very powerful advocates for your cause on 
both sides of the aisle. So it is a delight to have my 
colleagues here.
    With that, give us just a second, we will rearrange. No, 
that is all right. You can come on up. They are getting 
everything set up here so we can get the appropriate name tags 
and get everybody in their place.
    Mr. Cole. I thank the gentlelady for her testimony.
    Madam Chairman, we have actually already done a formal 
introduction, but if you will forgive me, I want to quickly 
note for the record what a delight it is to have you here.
    The highest ranking woman in Congress, clearly the most 
capable person in the Republican leadership team, there is no 
question about that, and somebody that I think--I once 
described the chairman as somebody who knows how to throw a 
punch with a smile. And she does. She is extremely effective on 
the floor.
    But she also brings us together, and this is one of those 
causes in which she has reached across the aisle and brought us 
together and played a really significant role in the passage of 
the ABLE legislation and many other things.
    So, Madam Chairman, it is a delight to have you here, and 
you are recognized for whatever remarks you care to make.
    Mrs. McMorris Rodgers. Thank you so much, Chairman Cole and 
Ranking Member DeLauro, for holding this hearing on the 
tremendous potential of research related to those with Down 
syndrome. And it is not just for those with Down syndrome. It 
is for millions of others, as my colleagues have also pointed 
out. And I am thrilled to be here as a part of this community 
today and to be joined with a packed-out room that all 
represent part of the Down syndrome community.
    Ten years ago, our oldest son Cole was born and tested 
positive for that extra 21st chromosome. It is also known as 
Trisomy 21 or Down syndrome. And today Cole is a happy, healthy 
fifth grader. He has mastered his multiplication table. In 
fact, his classmates want him to be on their team because he 
does so well on the quizzes. He is on Cub Scouts, on his way to 
being an Eagle Scout. He loves to play basketball. And without 
medical research, children like Cole wouldn't have the 
opportunities that they have today.
    Down syndrome's discovery began with Dr. John Langdon Down. 
In 1866 this brilliant English doctor of the Royal London 
Hospital laid out the groundwork for what would become a rich 
history of dramatic medical breakthroughs.
    In the 1950s it led to the discovery that humans have 46 
chromosomes in each cell, but an individual with Down syndrome 
has 47. And in 2000 an international team of scientists 
successfully identified and catalogued each of the 
approximately 329 genes on the chromosome 21. And, in fact, 
today the Alzheimer's gene has also been associated with the 
21st chromosome. You will hear more about that.
    As a mom and Member of Congress, I have dedicated my time 
for advocating for more research to improve outcomes and 
increase opportunities for those born with Down syndrome.
    Today there is still so much more to discover. Consider 
this: 50 percent of babies born with Down syndrome are born 
with a congenital heart defect, although there is no case of 
one with Down syndrome suffering from a heart attack.
    Children with Down syndrome have a higher likelihood of 
developing juvenile leukemia, although those with Down syndrome 
do not have solid tumor cancers.
    And thanks to improved medical care, 80 percent of adults 
with Down syndrome live beyond 60 years, compared to in 1960, 
on average, a person would live to only be 9 or 10 years old. 
This is a dramatic increase in life expectancy. However, almost 
every individual develops Alzheimer's or dementia.
    Even though Congress has substantially and consistently 
increased the NIH budget in recent years, and in very large 
part to the work of this committee, funding for Down syndrome 
research makes up less than one hundredth of a percent of the 
total budget.
    The year Cole was born, the NIH provided $16 million of 
research for those with Down syndrome. Today it is 
approximately $28 million. With more than 400,000 Americans 
living with Down syndrome, that is less than $100 of research 
per individual.
    By comparison, 1.5 million live with autism, which received 
$243 million. Cystic fibrosis receives $91 million and affects 
nearly 30,000. Fragile X is funded at $46 million, with an 
estimated 50,000 people living with the disease across the 
Nation.
    With additional funding, more new and innovative research 
could take place. A great example is Global Down Syndrome's 
Human Trisome Project. It is an ambitious longitudinal--tough 
word for me to say--cross-sectional study, several layers of 
genomics information, thousands of individuals with Down 
syndrome, and 500 typical individuals.
    This research will help us understand why are individuals 
with Down syndrome are protected from some medical conditions 
and diseases, while also highly predisposed for others.
    And it won't just help those with Down syndrome, but also 
millions of others with life-threatening diseases through the 
potential development of new diagnostic and therapeutic tools.
    Therefore, my question to this committee is: Why aren't we 
dedicating more research to continue to unlock the mysteries of 
the 21st chromosome? I urge my colleagues to consider increased 
NIH funding for Down syndrome research, especially studies that 
incorporate much needed cross-institute collaboration. The 
history of the research related to Down syndrome and the 21st 
chromosome is rich with breakthroughs and dramatic outcomes. 
Let's go unlock some more.
    Thank you, Mr. Chairman.
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    Mr. Cole. I thank the chairman for her testimony.
    As I mentioned earlier, we know you all have busy 
schedules, but I do want to give any of our Members who care to 
make a comment or ask a question an opportunity to do that 
before we excuse you and bring the expert panel up. So I want 
to recognize first my good friend, the ranking member.
    Ms. DeLauro. I don't have any questions, Mr. Chairman. But 
I think you get a sense, we all get a sense that--and to our 
audience--we are blessed to be a part of this institution.
    And the strength of this institution is its potential. 
Sometimes it doesn't do everything you want it to do every day, 
but its potential is enormous. And its potential with regard to 
looking at Down syndrome, looking at cancer, looking at all the 
diseases that our health agencies, the NIH, the CDC, the FDA, 
these are jewels.
    And we do have the ability and the power to push the edge 
of the envelope for scientific research through these blessed 
jobs that we have to help to make a difference.
    So thank you for the difference that you all are making in 
this effort. Thank you.
    Mr. Cole. Anybody on this side have any comment, questions, 
introduction that they care to make?
    Looks like you are mercilessly excused. But before you are, 
I want to thank each of you for being here.
    And as those of you in the audience know, you have some 
very passionate and very powerful advocates for your cause on 
both sides of the aisle. So it is a delight to have my 
colleagues here.
    With that, give us just a second, we will rearrange. No, 
that is all right. You can come on up. They are getting 
everything set up here so we can get the appropriate name tags 
and get everybody in their place.
    And, Mr. Coffman, please come and join us, because I know 
you will have a couple of introductions to make.
    Can I just say for the record, there is a lot of hugging 
and kissing today. Not normally what we see here.
    I want to welcome each of our panelists and guests here 
today as our witnesses. You have already been formally 
introduced, so I will forego that, other than I want to 
recognize my good friend from Colorado who has a couple of 
constituents here and wanted to have the opportunity to 
introduce them to the committee.
    The gentleman from Colorado is recognized, Mr. Coffman.
    Mr. Coffman. Thank you, Mr. Chairman.
    Mr. Chairman, I would like to begin by thanking you and the 
ranking member for holding today's hearing and recognizing 
Colorado's efforts to study the link between Down syndrome and 
Alzheimer's disease in search for a cure.
    I am honored to be here today to introduce Ms. Michelle Sie 
Whitten, the president, CEO, and cofounder of the Global Down 
Syndrome Foundation in Denver, Colorado, and Dr. Joaquin 
Espinosa, the executive director of the Linda Crnic Institute 
for Down Syndrome at the University of Colorado.
    Michelle has served as the executive director of the Anna 
and John Sie Foundation and was fundamental to the 
establishment of the Linda Crnic Institute for Down Syndrome, 
as well as the Sie Center for Down Syndrome at the Children's 
Hospital of Colorado. Most importantly, she is the mother of 
two children and one of whom has Down syndrome.
    Dr. Espinosa currently directs research that is 
investigating how gene networks control cellular behavior and 
the function of organisms in cancer biology and Down syndrome. 
Additionally, he is leading the Human Trisome Project at the 
Crnic Institute. This is the largest and most comprehensive 
study of its kind.
    As you can see, Michelle and Dr. Espinosa have dedicated 
their lives and careers to studying and advancing Down syndrome 
research, especially the link with Alzheimer's disease and the 
connections to cancer, immune dysregulation, and heart disease, 
to name a few.
    Mr. Chairman, it has been a privilege to learn about their 
innovative research that has identified a strong link between 
Down syndrome and Alzheimer's disease. The fact that people 
with Down syndrome have a significantly increased risk of 
developing Alzheimer's makes it a compelling reason for the 
research community to further study this connection. We can 
better understand how Alzheimer's progresses and why certain 
people are more susceptible to the disease.
    Additionally, this area of research provides a valuable 
opportunity for us to better understand how to protect our 
constituents from developing Alzheimer's disease. And Michelle 
Sie Whitten and Dr. Espinosa are at the forefront of this 
critical research.
    Mr. Chairman and Ranking Member, thank you again for having 
me here today and for your focus on advancing Down syndrome 
research.
    I yield back.
    Mr. Cole. I thank the gentleman for coming. And for that, 
my friend is of course excused. I know has a busy schedule to 
make. But thank you for joining us this morning.
    And we will move straight to the testimony. So, Ms. 
Whitten, you are recognized for whatever opening comments you 
care to make.
    Ms. Whitten. Great. Thank you.
    And thank you, Congressman Coffman.
    So, Chairman Cole and Ranking Member DeLauro, thank you for 
convening today's hearing and for your work and leadership in 
significantly increasing Federal support for biomedical 
research and efforts to improve the quality of life for 
Americans with Down syndrome.
    Thank you, Representatives McMorris Rodgers, Bustos, and 
Sessions for all you have done and continue to do for our 
children and adults with Down syndrome.
    Representatives McMorris Rodgers and Sessions have been 
great mentors to me and advocates for my organization's work, 
and Congresswoman DeLauro has been really infallible in that 
sense.
    My name is Michelle Sie Whitten. First and foremost, I am a 
daughter, a wife, and a mother. I am the daughter of two 
immigrants, my mother from Italy and my father from China, and 
I am the mother of two fabulous children, Sophia, who is 14 and 
happens to have Down syndrome, and Patrick, who is a typical 
11-year-old. They are all here in the audience today with my 
husband and his mom and sister from English, and I thank them 
for allowing me to work hard every day to improve the future 
not just for Sophia, but for Cole, for Alex, and millions more 
with Down syndrome.
    When I was pregnant with Sophia I had an amnio. The genetic 
counselor told me my baby would die by 3 and essentially 
pressured me to terminate. My husband and I did some soul 
searching and continued with the pregnancy and we never looked 
back. We consider Sophia a gift who has enriched our lives and 
all those around her.
    During my pregnancy, I discovered the life span a person 
with Down syndrome was not 3, but 50 at the time. I also 
discovered there was little or no clinical research addressing 
health outcomes for people with Down syndrome.
    Shortly after I gave birth, I found myself in Bethesda, as 
I would, meeting the then-Director of the National Institutes 
of Health, Dr. Elias Zerhouni. It was Dr. Zerhouni who pointed 
out to me that Down syndrome was one of the least funded 
genetic conditions by the NIH and who told me, if you do just 
one thing, establish an academic home and rebuild the pipeline 
for science, and the science needs to be there.
    We did just that. In 2008 my family and I organized the 
Down syndrome scientific summit, and the conclusion was 
twofold. First, shock that there was not more funding for Down 
syndrome. And second, conviction that research would not only 
help the 300,000 to 400,000 people with Down syndrome in the 
U.S., but could help millions more.
    And we have talked about the therapeutic leverage, which my 
father actually coined. So 100 percent, we have talked about, 
will have the brain pathology of Alzheimer's. Thirty percent 
will have autoimmune disorders. And 50 to 500 times more likely 
to get certain kinds of leukemia. Alternatively, it is 
extraordinarily rare for a person with Down syndrome to suffer 
a heart attack or a solid tumor cancer.
    So based on this knowledge, we established the Down 
Syndrome Foundation, and we collaborate with several other 
national Down syndrome organizations doing excellent work, and 
they support us in taking the lead in this issue.
    Global has worked tirelessly and as good partners with 
Congress and NIH to stimulate Down syndrome research funding. 
Shortly after we incorporated, Global worked with 
Representative Sessions to help start the Congressional Caucus 
with Representative Patrick Kennedy and, of course, Cathy 
McMorris Rodgers.
    We reached across the aisle. We have worked with U.S. 
Senators Tom Harkin, the late Arlen Specter, Richard Shelby. 
Today we are privileged to have close friends and allies and 
many other colleagues who have supported language, like 
Congresswoman DeLauro, to really highlight the disparity of 
funding for Down syndrome research.
    In December of 2010, we jointly organized the first-ever 
Down syndrome conference held by the Eunice Kennedy Shriver 
National Institute of Health Child Health and Human 
Development, the NICHD. And that conference was focused on 
national registries and databases, and the conference findings 
were published in 2001 and were a catalyst to NICHD's Down 
syndrome registry called DS Connect.
    Most importantly, we established a strong pipeline of 
excellent science. And they can no longer say--nobody can 
actually say that there is a lack of good science as a reason 
for the disparity of funding for Down syndrome research.
    So today Global's gross revenue is approximately $8 
million, with the majority of our proceeds going to research, 
and our programs reach about 20,000 people with Down syndrome 
in the U.S., including medical care to patients from 28 States 
and 7 countries. We have 38 labs and over 140 scientists 
working on Down syndrome with key focuses like Alzheimer's, 
autoimmune disorder, and cancer.
    Today we are ready to work with the trans-NIH platform that 
builds on these amazing breakthroughs and allows us to recast 
Down syndrome as an autoimmune disorder or as an immune system 
disorder. And I am sure that Dr. Espinosa will talk more about 
some of these exciting findings.
    The science stands ready, but now we need our colleagues at 
NIH to think in new ways with us to leverage these amazing 
breakthroughs and consider for a moment how this can be done. 
If we take the key comorbidities associated with Down syndrome, 
then it would follow that at least 10--at least 10--institutes 
at NIH should be key stakeholders in Down syndrome research. 
And I have included them in a handout so you can see those.
    And I think that people with Down syndrome stand ready to 
participate in the science, as seen in some of the work that 
Dr. Espinosa is doing and his enrollments rates being double 
what we had anticipated in a very short timeframe.
    On behalf of my daughter's future, our constituents with 
Down syndrome and their families, and others that stand to gain 
from our science, we hope that Congress can help effectuate a 
trans-NIH approach to Down syndrome research consistent with 
the 21st Century Cures law, and that our mutual desire for NIH 
to engage in outside-the-box thinking and incorporation of new 
funding into this research is finally fulfilled.
    Despite our advocacy and advancement, there has been 
negative to flat funding for Down syndrome research over the 
last couple of decades. This is also a significant disparity as 
compared to other developmental conditions or comparable 
disorders, and my written testimony also includes a kind of 
chart tracking those budgets.
    As you can see, despite being the leading cause of 
developmental delay in the U.S. and the world, Down syndrome is 
one of the least-funded genetic conditions by the NIH. From 
2001 to 2006, NIH annual funding for Down syndrome research 
plummeted from $29 million to $14 million, despite significant 
growth of the NIH budget during this time.
    From 2001 to 2017, if Down syndrome had increased at the 
same rate as the NIH budget up or down, we would be at $744 
million today. Over those two decades were at about $350 
million.
    I will leave you with these final numbers. Based on the 
CDC, there are 300,000 to 400,000 people estimated with Down 
syndrome living in the U.S. today. Live births have increased 
from 1 in 1,000 in 2002 to 1 in 691. The life span of a person 
with Down syndrome has more than doubled to 60 years today, up 
from 28 years in the 1980s. With increased live births and 
doubling of life span there will be a relative population 
explosion of people with Down syndrome in the United States.
    To be clear, the number of people with Down syndrome is 
getting larger, not smaller, and so is the need. Our children 
and adults with Down syndrome, who are American citizens, 
deserve to know that there is research funding and medical care 
available to them that allows them to reach their true 
potential.
    Thank you for caring about the future of this special 
population and allowing me to testify at this milestone 
hearing. Thank you.
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    Mr. Cole. Thank you for your testimony and your advocacy.
    Dr. Espinosa, you are recognized for whatever opening 
comments you care to make.
    Dr. Espinosa. Thank you, Chairman Cole and Ranking Member 
DeLauro, for hosting this hearing and inviting me to testify.
    I am here today to share with you my understanding of what 
I believe is one of the most spectacular developments in the 
history of medicine and biomedical research.
    I know that you know that one in four Americans is going to 
die of cancer. Despite a massive investment in cancer research 
over the past 5 decades, cancer remains a leading cause of 
death in the USA and many developed countries.
    I know that you are also aware of the tremendous impact of 
Alzheimer's disease on our society. One in three of our senior 
citizens dies with Alzheimer's or related dementia. The medical 
care associated to Alzheimer's, the costs are $260 billion a 
year, and the number is expected to quadruple by 2050.
    So clearly our Nation and the world at large could use new 
ideas and new resources to address these major biomedical 
issues.
    Now, what if I told you that there is a special population 
of American citizens for whom the statistics do not apply? What 
if I told you that this population is naturally protected from 
developing most cancers, yet at the same time they are the 
largest human population with a genetic predisposition to 
Alzheimer's disease? I hope you would agree with me that this 
population would deserve special attention.
    I am talking, of course, about people with Down syndrome. 
People with Down syndrome are very special. Typical people have 
the DNA packaging of 46 chromosomes. People with Down syndrome 
they have 47 chromosomes. They have an extra copy of chromosome 
21, three instead of two, and this is why the condition is 
known as Trisomy 21.
    This small extra piece of DNA cause a different disease 
spectrum of in people with Down syndrome whereby either they 
are strongly protected or strongly predisposed to major medical 
conditions that affect the typical population. So clearly these 
fascinating observations deserve much research inquiry and 
medical observations.
    Now, cancer and Alzheimer's are just the tip of iceberg. 
For instance, it is very rare to hear of somebody with Down 
syndrome dying of a heart attack. On the other hand, they are 
highly predisposed to autoimmune conditions such as type 1 
diabetes, rheumatoid arthritis, celiac disease, autoimmune 
hyperthyroid disease.
    I told you that they are protected from most solid cancers, 
such as, say, breast cancer or prostate cancer. At the same 
time, they are highly predisposed to developing leukemias. And 
I could give you many, many more examples.
    Now, how come then Down syndrome is one of the least 
understood medical conditions and one that has been underserved 
by the biomedical research enterprise?
    Perhaps many thought that the population with Down syndrome 
was going to eventually disappear due to prenatal screening and 
early terminations, but that is definitely not the case. There 
are four times more people with Down syndrome in the USA today 
than in the 1950s. The rate of live births has actually 
increased. The life expectancy for people with Down syndrome 
has more than doubled since the 1980s.
    So at the most conservative estimate there are at least 
220,000 people with Down syndrome in the USA. There could be as 
many as 400,000. Simply people, with Down syndrome are here to 
stay.
    Now, many may have thought that it was impossible or very 
difficult to reverse the ill effects of the extra chromosome. 
That is also not true. That more than doubling of the life 
expectancy was due to some simple interventions, getting people 
with Down syndrome out of institutions, give them proper 
medical care, and some standard medical practices, such as 
surgery for those with a congenital heart defect or hormone 
management with hypothyroidism. I am convinced that with more 
specialized research people with Down syndrome will live even 
longer, better lives.
    Regardless of the historical reasons, the truth remains 
that Down syndrome research has been underfunded. And clearly 
it is a multi-organ, multi-system condition that could not 
possible fit under the scope of a single NIH institute. 
However, historically the bulk of the research in this area has 
been funded by a single institute, the National Institute of 
Child Health and Development.
    Given the obvious potential of Down syndrome research to 
advance our understanding of Alzheimer's, cancer, leukemias, 
autoimmune conditions, and much more, I think the time is right 
to think about a trans-NIH initiative to investigate this 
condition involving many, many institutes at NIH.
    Now, the concept of Congress and NIH working together on a 
trans-NIH initiative to address an emergent medical problem is 
not new. Back in 1988, Congress and NIH worked swiftly to 
create the Office for AIDS Research. This was in the face of 
the imminent HIV/AIDS epidemic. They created the Office for 
AIDS Research. That is a trans-NIH agency that orchestrates a 
large grant portfolio crossing the boundaries of individual 
institutes and centers within NIH.
    Within a few years, the new cases of AIDS started to 
decrease, they continue to decrease, and today deaths caused by 
HIV are less than a third of what they were in the 1990s. So 
clearly the strategy and investment paid off with big 
dividends.
    Now, in 1988, when Congress worked with NIH to create the 
Office for AIDS Research, there were fewer than 100,000 people 
with AIDS in the USA. That same year, there were more than 
150,000 people with Down syndrome. Today the Office of AIDS 
Research administers a budget of $3 billion. That is 150 times 
more money than that devoted to Down syndrome research.
    So how much longer should people with Down syndrome wait to 
receive the due share of the resources devoted to medical 
research? How many more people with Down syndrome should there 
be to have a trans-NIH initiative? I think the time for action 
is now. People with Down syndrome stand ready to participate 
research, for their own benefit of course, but also for the 
benefit of the rest of humankind.
    Thank you for hosting this historic hearing and allowing me 
to testify.
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    Mr. Cole. Thank you very much for your testimony.
    We will move next to you, Dr. Mobley. And I just want to 
say, I am sure you are aware of this, when we decided to do 
this hearing, of course one of the first people I talked to was 
Pete Sessions. And he immediately said: This is the guy you 
have got to have. You have got to make sure that Dr. Mobley 
comes and shares all he knows with his testimony.
    So we are delighted you took the time to come, and you are 
recognized for whatever opening remarks you care to make.
    Dr. Mobley. Thank you, Chairman Cole and Ranking Member 
DeLauro and the esteemed members of the committee. I really 
want to thank you for the invitation to testify on the very 
important topic of Down syndrome research.
    I am going to just depart a little bit from my comments to 
make the case that this is an enormously positive time in our 
history in science. We have the power to really understand the 
genes and mechanisms that cause the problems that people with 
Down syndrome have. And we have that same power to understand 
how to prevent solid tumors. They are going to teach us a lot.
    There is absolutely no one in this room that should ever 
have to suffer from Alzheimer's disease. We are all there. All 
of us are susceptible to this. And I am going to make the case 
that an investment in research in Down syndrome is going to 
make it possible for not just those people with Down syndrome, 
but for all of the rest of us to avoid it.
    How about a world without Alzheimer's disease? My argument 
is, if you want that to happen--and we all very much do--one 
should encourage a very robust investment in research on Down 
syndrome. We can prevent it in those folks. And I think, 
because of the work we do for them, we can prevent it in all of 
the rest of us.
    I have a number of other comments, but let me just quickly 
say how pleased I am that it is a new day for Down syndrome 
research. When I got started in this, my colleagues told me: Do 
not study Down syndrome, please don't do that. You are going to 
ruin your career. And why? Because it is too complex to 
understand, too difficult to study, and treatments will come 
too late.
    Those are not true, those statements are all false. We are 
showing increasingly that in spite of the complex biology of 
Down syndrome--and it is complex--genes and mechanisms causing 
adverse effects are being discovered, targets for treatment are 
being defined, and one can confidently forecast the emergence 
of successful therapies for children and adults.
    But we have problems. There are a lot of unmet needs. And I 
want to go to this important question. Would understanding the 
genetic and mechanistic basis for a disorder in Down syndrome 
serve not just those with Down syndrome, but the population at 
large? And I think the answer to that question is yes, that 
studies in Down syndrome will positively impact met the care of 
those who do not have Down syndrome.
    We have talked about Alzheimer's disease. I won't mention 
it again except to say that it is a scourge. Imagine how those 
with Down syndrome and their families view the oncoming threat 
of Alzheimer's disease. I can promise you, it is a nightmare, a 
nightmare from which they cannot awake.
    Some years ago I committed to understanding what is going 
on here. We discovered in mouse models of Down syndrome, and 
later others in people with Down syndrome, that an extra copy 
of one gene, an extra copy of one gene is necessary. We can 
target that gene. We can devise therapies that attack that gene 
and its products. We can lower the level of that gene's 
expression to normal. And I think in so doing we will prevent 
Alzheimer's disease in Down syndrome.
    This work needs help. This work needs investment and 
energy. And that investment has to be really thoughtfully, 
carefully designed, and I would argue has to be designed with 
the consideration of both the private sector, as well as NIH.
    We have enjoyed so much the contributions from the LuMind 
RDS Foundation under its late leader Dr. Michael Harpold, a 
great friend and a great advocate and a great champion for this 
work, the Global Down Syndrome Foundation, the Alzheimer's 
Association, the National Down Syndrome Society, the Lejeune 
Foundation, the Cure Alzheimer's Fund, and AC Immune.
    NIH's increasing role in Down syndrome has been highly 
significant. Not long ago, DS research was a low priority for 
NIH. That changed substantially with the establishment of the 
Down Syndrome Working Group recommended by Congress in 2006. We 
are very grateful to Congress for their interest and support 
for Down syndrome research.
    With this investment, with this recommendation came a new 
energy for Down Syndrome research. Among the manifestations 
that we can now see are new initiatives to identify biomarkers 
and track Alzheimer's in people with Down syndrome, support 
under a private-public partnership of a clinical trial to 
prevent Alzheimer's disease and Down syndrome.
    And we are very grateful at the recruitment of Dr. Diana 
Bianchi to the directorship of the NICHD. She is a terrific 
Diana Bianchi researcher and somebody we really look forward to 
working with.
    So I ask Congress to urge NIH to build upon its existing 
efforts to accelerate the pace and expand the scope of its work 
to enable an era of unprecedented success in understanding and 
caring with people Down syndrome, and I thank the committee for 
the chance to testify. Thank you so much.
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    Mr. Cole. Thank you very much for your testimony.
    And, next, we would like to go to Mr. Frank Stephens, an 
outstanding advocate on behalf of Down syndrome research and on 
behalf of the families who have to deal with the issue. So, Mr. 
Stephens, you are recognized for whatever opening comments you 
care to make.
    Mr. Stephens. Mr. Chairman and members of the committee, 
just so there is no confusion, let me say that I am not a 
research scientist. However, no one knows more about life with 
Down syndrome than I do.
    Whatever you learn today, please remember this: I am a man 
with Down syndrome, and my life is worth living. Sadly, across 
the world, a notion is being sold that maybe we don't need 
research concerning Down syndrome. Some people say prenatal 
screens will identify Down syndrome in the womb, and those 
pregnancies will just be terminated. It is hard for me to sit 
here and say those words. I completely understand that the 
people pushing this particular final solution are saying that 
people like me should not exist. That view is deeply prejudiced 
by an outdated idea of life with Down syndrome.
    Seriously, I have a great life. I have lectured at 
universities, acted in an award-winning film and an Emmy-
winning TV show, and spoken to thousands of young people about 
the value of inclusion in making America great. I have been to 
the White House twice, and I didn't have to jump the fence 
either time.
    Seriously, I don't feel I should have to justify my 
existence. But to those who question the value of people with 
Down syndrome, I would like--no, I would make three points. 
First, we are a medical gift to society, a blueprint for 
medical research into cancer, Alzheimer's, and immune system 
disorders. Second, we are an unusually powerful source of 
happiness. A Harvard-based study has discovered that people 
with Down syndrome as well as their parents and siblings are 
happier than society at large. Surely happiness is worth 
something. Finally, we are the canary in the coal mine. We are 
giving the world a chance to think about the ethics of choosing 
which humans get a chance at life. So we are helping to defeat 
cancer and Alzheimer's, and we make the world a happier place.
    Is there really no place for us in the world? Is there 
really no place for us in the NIH budget?
    On a deeply personal note, I cannot tell you how much it 
means to me that my extra chromosome might lead to the answer 
to Alzheimer's. It is likely that this thief will one day steal 
my memories, my very life from me. This is very hard for me to 
say, but it has already begun to steal my mom from me. Please, 
think about all those people you love the way I love my mom. 
Help us make this difference, if not for me and my mom, then 
for you and the ones you love. Fund this research. Let's be 
America, not Iceland or Denmark. Let's pursue answers, not 
final solutions. Let's be America. Let's make our goal to be 
Alzheimer's-free, not Down-syndrome-free.
    Thank you.
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    Mr. Cole. Wow. Before we proceed to questions, Mr. 
Stephens, I want to say, in your testimony, I think you 
answered every question you laid out. Just thank you very much 
for your very powerful testimony. Thank you for being here 
today.
    I want to open the questions--I want to move first to Dr. 
Espinosa and Dr. Mobley. And my question is this: You all in 
your testimony referred to how dramatically--and, Dr. Espinosa, 
you discussed this with some specificity--how dramatically 
lifespans for people with Down syndrome have increased in the 
last three decades. Could you elaborate a little bit more on 
what the reasons for that increase are? How much of it has been 
due to research? How much has it just been due to changing 
conditions in terms of how we approach people with Down 
syndrome?
    Dr. Mobley. Thank you so much for the question.
    In large part, this is simply making available to people 
with Down syndrome what we were already making available to the 
rest of us.
    I am going to tell you a story. When I was a brand new 
pediatric resident at Stanford, I went to work one day, and I 
was told: There is a little girl in this room, and she is 12 
years old. She has a low-grade fever and some belly pain. Would 
you mind investigating, figure out what is going on?
    I went to see her, and it was very clear she had an acute 
appendix. So I felt terrific; I can make a difference here. 
Here I am, a brand new resident, and I can actually make a 
diagnosis. And I called the surgeons, and they came and said: 
Well, we are not sure.
    And 8 hours went by, and they came again, and they said: We 
are not sure.
    They waited 72 hours until she perforated her appendix and 
nearly died. And what was her problem? She had Down syndrome. 
They were simply unwilling at that time to take seriously the 
rights that a person with Down syndrome had to great medical 
care. I will never forget that incident. It has changed my 
life.
    So I submit to you that we are increasingly doing for 
people with Down syndrome what we have done for others forever. 
Yes, there have been advances. Congenital heart disease surgery 
is cooler, better, faster, stronger. Yes, we know how to 
measure hormone levels more effectively. Yes, we know how to 
treat infections. All those things are true. But the biggest 
breakthrough so far is bringing those people home, putting them 
in school, and treating their medical issues the way they 
should have been treated all along.
    Mr. Cole. Thank you.
    Dr. Espinosa.
    Dr. Espinosa. Yeah. I agree with everything that my 
colleague, Dr. Mobley, said. I would like to add that there is 
a lot of so-called low-hanging fruit in the Down syndrome 
research field. And it is totally possible and I am confident 
that if we got an investment in research, we can find other 
simple interventions that could have a massive impact on the 
lives of people with Down syndrome. One example is research 
coming out of our institution shows that the immune system of 
people with Down syndrome, a particular aspect of it, is super 
activated all the time, which leads to autoimmune conditions 
but also exhaustion of other aspects of the immune system 
leading to more particular infections, you know, like the 
example of Briana with Lyme's disease. What do you know? This 
aspect of the Down syndrome can be modulated with FDA-approved 
drugs for the conditions. So now we are testing the possibility 
of immune therapies for the conditions.
    So, if we were to invest in more research, chances are that 
there may be drugs that can be repurposed, that were developed 
for other conditions but that can be now used--of course after 
appropriate testing for safety and efficacy--in people with 
Down syndrome. So I am very optimistic that an effort in 
research will pay off big dividends.
    Mr. Cole. Thank you.
    In the limited time I have left, I want to address the 
questions to Ms. Whitten and Mr. Stephens which are: You have 
seen dramatic changes, obviously, in the last few years. What 
are the most important changes you have seen? What are the 
changes you would like to see in terms of quality of life for 
people with Down syndrome?
    Ms. Whitten. Frank, do you want to go first, or do you want 
me to go first?
    Mr. Stephens. I can go first.
    Ones that we would like to see is better healthcare because 
NIH needs to fund this research. Why? Because people like us 
who were born differently need better care because, like I said 
in my speech about Alzheimer's and all that I have said, those 
are the diseases that we really need to try and cure.
    Mr. Cole. Ms. Whitten.
    Ms. Whitten. Absolutely.
    I kind of echo Dr. Mobley and Dr. Espinosa. I think one of 
the great advances, which ironically really were only kind of 
finalized in the early 1990s, was kind of 
deinstitutionalization in this country. It is kind of a dark 
past of ours. But the overwhelming majority of people with Down 
syndrome were put in institutions, most of which were inhumane. 
And as Dr. Mobley and Dr. Espinosa had said, bringing them 
home, allowing them, through IDEA, you know, standing on the 
shoulders of the human rights, civil rights activists of the 
sixties and seventies, and including a great advocate of ours, 
Senator Tom Harkin, that then they were able to, you know, 
actually live a longer life. And then, as Dr. Espinosa said, 
with additional research, we believe that that 60 sound barrier 
can be upwardly mobile.
    And then the quality of life: I think the quality of life 
is the number one concern that I as a parent have and that we 
hear in the Down syndrome community. I think, first and 
foremost, it is health because, if you suffer from bad health, 
everything is secondary, so getting the great healthcare at all 
stages of life. And we are in new territory with adults 
doubling their lifespan.
    This is the first generation of people with Down syndrome 
who will outlive their parents. And that is a scary thing. And 
when we close our eyes and we leave this world, we want to make 
sure that they are safe. So I think safety, healthcare, and 
then just their quality of life that includes education, jobs, 
all these things that other Down syndrome and organizations as 
well as Cathy McMorris Rodgers and others are fighting for. But 
I am right there on the healthcare and the research.
    Mr. Cole. Thank you very much.
    I now recognize my good friend, the ranking member of the 
subcommittee.
    Ms. DeLauro. Thank you very much, Mr. Chairman, and thank 
you all for very powerful testimony. It is the advantage of 
this wonderful committee that we have the opportunity to listen 
to people who are experts in the field and who are also experts 
in their own lives as to what this--what Down syndrome can mean 
in their lives.
    Now, I wanted to ask a question. The subcommittee has 
followed advances in cancer immunotherapy very, very closely. 
And very exciting, I might add, about researchers who are 
looking at the ways in which we harness the power of our own 
bodies to deal with being immune against diseases.
    Dr. Espinosa, your research has shown that Down syndrome 
has an adverse effect on the immune system. Can you tell us 
about your discoveries? Explain how the research is similar or 
different in advances--to advances in the cancer immunotherapy. 
There are some researchers trying to harness the power of the 
immune system to fight diseases like cancer. But it sounds like 
your research could help calm the immune system with an 
individual with Down syndrome so that their own body isn't 
attacking itself.
    I am not a scientist as well, Frank.
    But is that accurate? What could other researchers who are 
working on immunotherapy learn from your work, and how would 
what you are requesting in terms of that further research 
capability add to this effort?
    Dr. Espinosa. Thank you for an outstanding question. You 
are not a scientist, but you think very clearly. There is an 
obvious connection here between the immune disregulation in 
people with Down syndrome and the protection from cancer and 
also the increased risk of leukemia. So what we found is that 
there is a branch of the immune system that is hyperactive in 
people with Down syndrome; it is the branch of immune system 
that we usually use only when fighting off a viral infection or 
fighting off a tumor, right? So it is called the interferon 
response. Interferons are the molecules that our cells produce 
when they have been infected with a virus to alert the 
neighboring cells and the immune system that there is a virus. 
They can also be produced in response to the presence of a 
small tumor. And then that alerts the immune system to come to 
the side and start attacking either the virus-infected cell or 
the incipient tumor cell. So that is always on in people with 
Down syndrome. That is not good.
    The immune system should be like the National Guard, you 
know: being quiet in times when it is not needed but then being 
deployed only in the case of an emergency and then coming back 
to the barracks. When you have the immune system constantly 
reactive, it can lead to problems like an attack of the self. 
And that is why people with Down syndrome have so many 
autoimmune conditions. When the immune system attacks the 
thyroid, you could have hypothyroidism. When it attacks the 
pancreas, you could have type 1 diabetes. When it attacks your 
hair follicle cells, you could have alopecia areata, a loss of 
hair which is very prevalent in people with Down syndrome. You 
can have attack of your pigment-producing cells in the skin 
that leads to vitiligo. All conditions that we see more oft in 
people with Down syndrome.
    So, coming back to your initial question, can we learn from 
this about cancer immunotherapy? Yes. What we have seen in 
people with Down syndrome is that they have elevated numbers of 
the cells in the immune system that attack tumors, and these 
are the cells of the immune system that are tumors, when they 
are successful at being tumors, fool--you know, and prevent 
that attack. What cancer immunotherapy does is unblock those 
restraints of the immune system so that immune cells can come 
in and attack and clear the tumor. So there is an obvious 
connection there where by studying more the protective aspects 
of the immune system with people with Down syndrome, we may 
find a way to modulate that in a therapeutic way in typical 
people that have tumors. The potential is definitely there.
    Ms. DeLauro. The potential is there is the--in terms of the 
research that you are looking to increase, is that an area that 
you are looking at? What are the areas that you want to try to 
focus on in terms of this additional research? Is this an 
avenue for additional resources for you to try and----
    Dr. Espinosa. Well, since I am a cancer researcher, from my 
background, that is an area where I personally am interested 
in. But I don't think that should be the only area where we 
should be investing in. I think we need a transdisciplinary 
approach across NIH institutes and centers, where we can look 
at Alzheimer's, like in the research program of Dr. Mobley; we 
can look at the cancer connection; we can look at the 
autoimmune connection. I think there is potential here for a 
lot of different things.
    Me, personally, my research team back in Denver is looking 
at this particular aspect.
    Ms. DeLauro. Thank you. Thank you.
    Mr. Cole. Let's go--with all due respect, we have these 
expert witnesses. We have our own experts too up here. So I 
want to go to my good friend Dr. Harris for whatever questions 
he would care to ask.
    Mr. Harris. Thank you very much.
    And thank you, Mr. Chairman, for calling the hearing.
    Mr. Stephens, I have been in Congress 7 years. That is the 
most powerful testimony I have heard at a committee hearing in 
7 years.
    Mr. Stephens. Thank you.
    Mr. Harris. I graduated medical school in 1980. And there 
is a reason why this is stuck in the Institute of Childhood 
Diseases, because, in 1980, it basically was a childhood 
disease, because we were taught that children with Down 
syndrome didn't live very long. They had congenital anomalies, 
which, at the time, were difficult to treat surgically, or high 
mortality rates and didn't. And it spawned an era where we 
started developing things like prenatal testing, and then 
thought I guess it is a good idea now that if we can measure 
this and we can find this disease, then, as you suggest, we can 
eliminate the disease through elective abortion. And I think 
that is something we always have to be careful about when we 
look at disability communities and try to solve that problem--
and, you know, we are having ongoing debates about end-of-life 
care and, you know, the disability concerns, end of life; we 
just have to be thoughtful of that.
    Anyway, it is very interesting. I want a little feedback 
from the panel because you are following on a hearing yesterday 
on NIH funding and, in general, global issues and about, you 
know, how great a job they do; we just ought to keep on doing 
exactly what we do and maybe give them a little more money.
    But your perception is absolutely correct. This needs more 
than just a little bit more money. It needs to be done smartly. 
It needs to be done wisely. It needs to be thought of outside 
the box. There is a reason why--and I have the sheet here. The 
per-affected individual, how much the NIH spends on various 
disease, and I think it was pointed that, for HIV/AIDS, we 
spend $2,500 in research per affected individual. And if you 
estimate the number of people living with Down's in the United 
States is 250,000, which is the low estimate, it is $111 per 
year per person. $111. You spend 25 times more per person on 
HIV/AIDS than we do in Down's. And, remember, in 1981, when I 
graduated from medical school, both diseases had similar 
reputations. Okay. And here we--now, why is that?
    And we have to ask, why is that? And I will suggest because 
we don't think outside the box when we--and a lot of Federal 
funding agencies don't think outside the box. They do have to 
be pushed by a committee like this to say, how can you have 
missed this? How can you have missed that, you know, these 
discoveries that you talk about and whether it is immunology, 
whether it is oncogenesis, how can we have missed this 
connection and not have particularly invested in that?
    So I am just going to ask the--do you think that, and you 
may not know this because this may be our purview to figure out 
how to solve this problem, that do we do it by just urging the 
NIH to look at this with--and, you know, because to come and 
ask--because you are asking for $300,000,000. Now, there are 
two ways you can do it. You can add more funding or you can 
just say: Look, we are going to increase funding every year 
anyway. Should we just earmark some of that and encourage the 
NIH to do this multidisciplinary approach? And I would suggest 
the latter one probably makes more sense. It might be easier to 
do to look at these new avenues.
    Where do you think--how do you think it is best done? How 
do we change the NIH's mind about this to say--and maybe, Dr. 
Mobley, since you know--since you kind of bridge it; your 
credential is you head an Alzheimer's institute and the Down 
syndrome research division--how do we convince conventional 
scientists, conventionally thinking scientists, to think 
outside the box on this?
    Dr. Mobley. Thanks. I have no perfect formula. But here is 
what I would--here is what I would say. The science is so 
powerful now that were NIH to pay more attention to it, were 
they to look across the field of Down syndrome research to 
identify gaps in understanding, to define priorities, to 
establish the bridge between discovery and therapeutic target, 
between target and target development, between target 
development and clinical trials, if they were given the 
opportunity, encouraged to pay attention to the great science 
that is there, the net result would be more funding. Forget 
about earmarks. The result of an NIH who pays attention to the 
science that is already there and who is empowered to make it 
better will be fantastic.
    And so I agree with this trans-NIH approach. I think it is 
exactly right. It needs--I need to hear from people who study 
immunology. And I can tell you right now: Immune-mediated 
mechanisms are making a difference and causing problems in the 
Alzheimer's-diseased brain. We simply can't--this chromosome 
and the things that it creates are a wealth of information 
across institutes to change the game.
    So ask NIH to pay attention, ask them to study it, ask them 
to define priorities and close gaps, and you will see a change.
    Mr. Harris. Thank you very much.
    I yield back.
    Mr. Cole. Thank you.
    We will next go to my good friend from California, Ms. 
Roybal-Allard.
    Ms. Roybal-Allard. Okay.
    Thank you, Mr. Stephens, for your very compelling testimony 
to the subcommittee. All you have accomplished in your life is 
very impressive.
    And, yes, your life, and the life of all those with DS, has 
value and is truly worth living.
    As has been highlighted today, that individuals living with 
Down syndrome are a gift to society, and it is not because you 
have that extra chromosome but because of your extra special 
heart. Unfortunately, many in our society are too slow to 
recognize this, and the world has not always been kind to the 
people with Down syndrome. For that reason, it is not 
surprising that many who have DS and their families are 
skeptical of government-sponsored research involving Down 
syndrome. So, as a result, one of the issues that has been 
brought to my attention by advocates in California is that many 
families with a Down syndrome child are hesitant to sign up for 
the Down syndrome patient registry because they are concerned 
about the government knowing too much about their health and 
their personal lives. What would you tell the parents of young 
children with Down syndrome to encourage them to sign up for 
the DS connect and to participate in the research?
    Mr. Stephens. I would tell them there is no need to be 
afraid because I know we can sign this. Why? Because it is our 
life. It is our blood. We have the right to celebrate who we 
are, to know that people with Down syndrome--like I said in my 
speech, we are men and women with Down syndrome, and I know 
that our lives are worth living.
    Ms. Roybal-Allard. Thank you.
    Dr. Mobley, the connection between Down's and dementia was 
first recognized in the seventies, and Alzheimer's pathology 
was discovered in the brains of individuals with DS in the 
eighties. And it has been almost 40 years that have passed, and 
only now is this research getting the attention that it 
deserves, and we have the baby boomer generation of adults with 
Down syndrome who are now well into their 50s and 60s, and we 
still have no drug approved to treat dementia, and many primary 
care providers across the country are still unaware or unable 
to make the diagnosis for Alzheimer's in those with Down 
syndrome. Why do you think it has taken so long for the medical 
community to embrace this critical connection between Down's 
and Alzheimer's?
    Dr. Mobley. It is a really good question. I want to say 
that the medical community suffers in the same way that the 
general community suffers. If people with Down syndrome are 
just different somehow, if their needs are not as important as 
other people, then why would you bother helping people with 
Down syndrome?
    Now, I don't want to mitigate the difficulties in 
diagnosing Alzheimer's disease in the general population. I 
don't want to say that we have had such great success with 
Alzheimer's disease. There are no therapies, yes. There are no 
therapies right now.
    But I can say that, in the many years that I have been 
invested in this, for a very long time, there was a pushback 
that, even in spite of the brain pathology, in spite of the 
changes in cognition, this wasn't the same thing. I was told by 
a Director of NIH one time: Well, it is not the same disease.
    It is the same disease. It looks different a little bit, 
but it is fundamentally the same disease.
    So I would argue that the reluctance to accept this 
realization is partly a reluctance to consider carefully the 
livelihood, the well-being, the importance of people with Down 
syndrome. Partly, it is that.
    Partly, it is that it is a little more difficult to make a 
diagnosis of dementia in somebody who starts off a little 
different cognitively, but that is all changing. I think there 
is a sea change in the way people think about Down syndrome. A 
few years ago, I gave a talk at UCLA, and I said I think we 
should certainly be thinking about treatments for Down 
syndrome. And one the scientists said: I have never even heard 
that concept before. Treat Down syndrome? Really? That is news 
to me.
    So, partly, it is recognition of the similarities. Partly, 
it is recognition of the personhood of the person with Down 
syndrome. And, partly, it is just getting the word out. And I 
think this committee has a chance to make a difference there. 
If every practitioner in America knew that this risk was there 
of Alzheimer's and Down syndrome, I think we would change their 
views of things. And I think that would be very positive.
    Ms. Roybal-Allard. You know, there are no drugs approved in 
this country to treat individuals with Down's or Alzheimer's, 
yet Britain has approved three cholinesterase. Is that----
    Dr. Mobley. Cholinesterase.
    Ms. Roybal-Allard [continuing]. Inhibitors for dementia in 
Down syndrome patients. Do you know why?
    Dr. Mobley. So, about 14 years ago, we had the FDA approve 
cholinesterase inhibitors for Alzheimer's disease in this 
country. That is the last major breakthrough--apart from 
memantine, that is the last major breakthrough. In fact, it was 
longer than that. It was like 25 years ago for cholinesterase 
inhibitors and 13 years ago for memantine. Those are drugs that 
are out there that are available to treat--symptomatically 
treat Alzheimer's disease right now. Those drugs are available 
for people with Down syndrome. But I think that they are 
underutilized. It is also fair to say that the effects--those 
side effects of those drugs may be different in people with 
Down syndrome than in the general population.
    But the bottom line really that you need to hear is, in 
spite of many, many billions of dollars invested, we are not 
yet there with disease-modifying treatments for Alzheimer's 
disease. And my argument is that here is a chance for people 
with Down syndrome to change that.
    Ms. Roybal-Allard. Thank you, Mr. Chairman. You have been 
generous with your time.
    Mr. Cole. You are generous to recognize that. Thank you.
    I next want to go to my good friend from the great State of 
Alabama, Mrs. Roby, for any questions she cares to ask.
    Mrs. Roby. Thank you, Chairman Cole, for convening this 
informative hearing today, and to all of you on the panel for 
sharing your information, and, Mr. Stephens, for your personal 
testimony; I appreciate it so much.
    But to highlight the importance of biomedical research and 
development in your own lives, it is truly fascinating to 
learn, as a member of this committee, that research could 
potentially translate to tremendous breakthroughs in 
Alzheimer's, certain cancers, and autoimmune diseases, just to 
name a few, as has already been discussed today.
    Mr. Chairman, today, I would like to recognize Melinda 
McClendon and her two sons, Buck and Charlie.
    Will you all stand up?
    Melinda and her two sons are constituents of mine from 
Dothan, Alabama. And I am so proud to have you here as 
advocates from my community to share your personal story. And I 
had the opportunity--and, Mr. Chairman, you met Buck on the 
floor yesterday--to take Buck down with me during votes. And I 
got to see firsthand Buck's infectious personality and his love 
of life. So thank you for sharing your day with me yesterday, 
and I am so grateful that all are here. So thank you so much.
    As a mother myself, I am particularly touched by your 
personal testimony, Mr. Stephens. Every life is precious. And I 
want you to know that I am with you and I support you. I, too, 
am deeply troubled by the recent reports out of Iceland and 
Denmark and other places boasting of being Down-syndrome-free 
by 2030 by means of abortion. I want you to know that I am 
unapologetically pro-life, and I will always fight to make sure 
that there are protections for life under our laws here in the 
United States. Every baby should be treated like the miracle 
that they were created to be.
    So, as we talk about growing research capabilities, 
including funding through this very committee, I think it is 
important, Mr. Chairman, that we have assurances that the 
medical community won't slip down the slippery slope towards 
eugenics however indirect the practice may be. And so, if you 
want to offer some comments on that, I think this is important. 
I know Dr. Harris touched on it, but, Mr. Stephens, you did as 
well. And I think this is a very, very important theme as we 
meet here today.
    Dr. Espinosa. Yeah. I would like to comment to that.
    You know, as the executive director of the largest agency 
studying Down syndrome in the world right now, you know, the 
Linda Crnic Institute for Down Syndrome, 34 active labs, more 
than 140 scientists, we do not fund any research that could 
even tangentially lead to that eugenics part that you 
recognize. And it is not that, now and then, we don't receive a 
proposal, you know, to try to investigate the origin of that 
extra chromosome, you know, how to prevent that,you know, which 
asks to try that proposal--we don't go there. Our mission is to 
improve the lives of people with Down syndrome. Within that, 
there are possibilities for prenatal treatments. You know, it 
is, of course, something that one needs to very cautious about, 
about intervening, you know, prebirth. But that is within the 
realm of possibilities with more research and advancing 
technology. So you can have assurance, you know, from the Linda 
Crnic Institute that we will not fund any research that could 
even tangentially be tied to selective terminations.
    Mrs. Roby. Thank you. I appreciate that.
    Real quickly, I don't have much time left, but can you 
expound--either one of you--can you expound on the timeline of 
researching Down syndrome? We have already gone over how--some 
advancements that we have made. But, you know, what technology 
resources and advocacy do you think are essential for 
discovering the next major medical breakthrough for individuals 
with Down syndrome in 5, 10, 15 years, 20 years? And what, if 
any, specific research or studies do you know at the present 
that focus on the adult population? If you could just go there 
with me, that would be great.
    Dr. Mobley. I will briefly start.
    The timeline depends on you. The timeline depends on you. 
The technology is there. The ideas are there. We need a 
concerted effort to solve these problems. And I think if you 
insist upon it, it will happen. So, just to make it clear, we 
know--with respect to Alzheimer's disease, we know what to do, 
we know when to do it, we know who to do it with. It is just a 
matter of putting our will to that purpose.
    I give you a specific example. We are now--under NIH 
funding, we developed a very promising compound that in mouse 
models of Down syndrome eliminates--eliminates--the effects of 
that extra chromosome. The molecule is well advanced. We hope 
to file an IND on that molecule very soon. I hope that we will 
do our first-in-man studies in Down syndrome. That could happen 
in 2 years. But, of course, it won't if there is not funding. 
And right now we don't have funding for that.
    So I want to say that it is a rich time for us. There is 
great promise, but we are going to need your help in heating up 
the argument and directing attention to this very important 
possibility. And I would argue that what we are hearing about 
Alzheimer's disease is true across the board. There are many, 
many projects that could go much more rapidly. So I would say 
the timing is up to you, Mrs. Roby.
    Mrs. Roby. Thank you very much for that.
    And I have gone over my time, but, Mr. Chairman, thank you 
for this very powerful hearing today in the subcommittee. I 
really, really appreciate you doing this.
    Mr. Cole. Well, thank you. And you can thank your excellent 
staff. You know, they are the ones that put these things 
together for us and help us identify the topics. So thank you.
    We always go by order of arrival before we begin. So my 
good friend from Massachusetts, Ms. Clark, is recognized for 
whatever questions she cares to ask.
    Ms. Clark. Thank you, Mr. Chairman and Ranking Member 
DeLauro, for this incredible hearing.
    And thank you to all the panelists, especially to Frank, 
for your powerful testimony here this morning.
    But I have some powerful people with me from my home State 
as well. I want to introduce Jan Tobin from Arlington. And we 
are very pleased to have Maureen Gallagher here, who is the 
executive director of the Massachusetts Down Syndrome Congress. 
But most of all, I want to introduce you to Kate Bartlett.
    Kate, if you would stand up and just say hello.
    Kate is one powerful advocate. I met Kate when she came to 
my office and signed me up as a cosponsor of the ABLE Act. We 
share a love of politics. I know this is a very bipartisan 
hearing and topic. We are kind of partisan, Kate and myself. 
And she is just an inspirational young woman, as I meet so many 
young women across my district. And I am delighted to have her 
here today. We also share a first name, spelled correctly with 
a K.
    But we also share something not so positive. Kate has 
statistically a great chance of Alzheimer's. I have lost my 
grandfather, my great aunt, my aunt, and now my mom, who is 
suffering.
    So, Dr. Mobley, what do you see as the possibility for 
isolating the gene that looks at Alzheimer's and the 
connection, if you can talk a little more about the connection 
between the much needed research for people with Down syndrome 
and the connection to Alzheimer's?
    Dr. Mobley. Yeah. Thank you.
    We know that Down syndrome is complex. We know that a lot 
of genes may contribute. But we know that the gene called APP 
is necessary for Alzheimer's disease and Down syndrome. The 
argument is that, if we target that gene and its products, so 
if we turn down its expression, if we eliminate its toxic 
products, that we can prevent Alzheimer's disease and Down 
syndrome. And the relevance of this more generally to 
Alzheimer's disease, to your mom, to my mom, to lots of moms 
and dads, is that that gene features prominently in all the 
thinking about Alzheimer's research and in Alzheimer's disease. 
One product of that gene is found in aggregates in the brains 
of folks with--from the general population. There is a familial 
form of Alzheimer's disease in which only the extra copy of 
that one gene is present to cause it. So the message here is 
that a very focused approach that sees the bridge between what 
we learn in Down syndrome, what we have learned in the genetics 
of Alzheimer's in the general population, that we focus on 
that. We pay attention to that. We invest in that in a very 
serious way. And by doing so, we basically find a way to 
prevent Alzheimer's disease, not just in Down syndrome but in 
the whole approximate population.
    I want to mention that therapeutically one should target 
APP, but there are other genes that make a difference in Down 
syndrome very likely, and in the population in general, very 
likely these other genes are playing an important role. So we 
don't want to it be only APP, but we want a program that 
robustly addresses those other genes as well because what we 
are facing is an epidemic that is going to affect all of us. We 
need to act now to avoid that epidemic.
    Ms. Clark. Thank you. And I just--I wanted to quickly say 
to Ms. Sie Whitten--is it Sie? Yeah. I thank you for your 
comment about rebuilding the pipeline of science. And it is so 
important for Down syndrome, despite what--you know, what--
translational research. Just on its own, we need to do this for 
the quality of life of people in our community. And the 
translational research is important but almost additional good 
things that can come.
    Some of the concerns I have is we are also debating a 
budget this week where we are looking at potentially removing 
trillions of dollars out of Federal spending. And there was a 
proposal by this administration to cut NIH overall by 22 
percent. That was rejected by this committee. Can you talk 
about what a 22-percent reduction in NIH would look like?
    Ms. Whitten. I can definitely speak on behalf of Global and 
our affiliates. And I don't know if--guys, do you think I speak 
for everybody in the community? It would be devastating. It 
would be devastating to a lot of people, millions of people. 
But when you look at the Down syndrome community, even 
furthermore compounded if we continue on this trajectory. If 
you look at the estimate of the budget for 2018 that kind of 
tracks with that cut, you know, we would drop down to 
$20,000,000. I mean, we were at 29 in 2001. You can just see, 
compounded year on year, what that has done for the lack of 
health outcomes for our children and adults. So I am so 
appreciative to every single one of you.
    And this is great because it is like, you know, both sides 
of the aisle, everybody is getting together, and everybody 
says: This is important for the health of all Americans. We 
need to maintain or increase, ideally, the budget at NIH. And 
certainly what we would hope is that, with yet another 
increase, that we could get some fair share of funding to help 
people with Down syndrome first and foremost and then, well, by 
God, if it helps people with Alzheimer's and cancer and 
autoimmune disease, that would be fabulous. So thank you very 
much.
    Ms. Clark. Thank you.
    I yield back. Thank you for your indulgence.
    Mr. Cole. I must say, I am being very indulgent today. But 
I will gently admonish my friends on both sides of the aisle, 
please refrain from asking the questions 2 seconds before the 
time is out.
    Ms. Clark. I think it was 20.
    Mr. Cole. You know, 20, 19, actually. But we will try and--
this is an exceptional hearing, and we appreciate your 
testimony so much. We are going to be running a little late. I 
am going to make a chairman's prerogative decision here. I want 
to make sure that every member has an opportunity to ask at 
least one set of full questions. So we are, with the indulgence 
of witnesses, go a little bit longer than we are scheduled for. 
And, certainly, I want to offer the ranking member and myself 
any opportunity to make any closing remarks we care to at the 
end of the hearing.
    With that caveat, I am going to go next to my good friend, 
Ms. Herrera Beutler, from the State of Washington.
    Ms. Herrera Beutler. Thank you. And I echo the comments of 
everyone you have heard. This has been amazing and eye-opening 
and enlightening. Cathy is one of my best friends. She had to 
sneak off. She is a busy lady. But, you know, watching her go 
through this process--and I have a few questions and a few 
thoughts, but one thing I want to say, just even to the 
families, to the advocates, I mean, you represent just a force 
that has been laboring and laboring and laboring just to get to 
this point, and I want to say thank you.
    You know, I had a challenging pregnancy, was told there 
would be no hope for my kiddo and to terminate, and there was 
no treatment even if she would have been born and breathing. It 
was a totally different condition. And we decided we wanted 
to--we didn't want to make that decision, right? We wanted to 
give her the best chance we could. And, you know, I was told by 
so many doctors: This won't work. This won't work.
    I was told all sorts of things that are not factual. And it 
wasn't because I think their hearts are in the wrong place, but 
I think the static mindset around people who are different. I 
mean, it just--it baffles me that we talk about diversity; we 
pay it lip service. It is total lip service. It is on every TV 
commercial. It is on ads. Yet we don't truly walk it out. And 
this is such an example of that. This is an example of this is 
true diversity. It is--there are truly differences. And we can, 
as a society, decide we are going to look at these differences, 
and we are going to truly celebrate them by treating them just 
like we would anybody else, or we are going to do--you know, I 
Googled the Iceland--your comment about Iceland and Denmark, 
and my stomach turned. And I think we need to recognize, as a 
society--you know, we are talking about how we can move the 
ball forward with regard to NIH funding.
    And I think you have captured the attention of the panel. I 
think we are going to do everything we can. But we also need to 
know, how do we move this ball forward in society? Because, 
ultimately, that is why this has gone where it has. You know, I 
have a bill on prenatal screenings. I think they are great, but 
they are not accurate, right? They are not--the people who get 
those screenings are not told the inaccuracies. And they make 
life-changing decisions based on it. I mean, I think what I 
would like to hear is, how do we help move this ball forward in 
society? Like, how do we truly celebrate this diversity?
    You know, and this may go to the parents but, really, to 
the whole panel because I think, in each of your respective 
spheres of influence, you have faced this and you have had to 
battle this and get through, really, just these hurdles. So I 
want to say thank you for doing that. I mean, it is your kid, 
right? So you are going to do whatever it takes. But to those 
who have had to, you know, go toe-to-toe with colleagues, how 
do we change this in the communities? Because that is where 
real change occurs. I always tell people: It doesn't occur in 
Congress. We are the tip of the spear. We can help things. We 
can push things. But true change is going to come from the 
hearts and the minds of the people in our communicates. And how 
do we help effect that change? And that is to the panel.
    Ms. Whitten. Thank you. That was just an amazing statement. 
And everything was true. We talk about people with Down 
syndrome as being differently-abled, and that is the diversity. 
I think that we as a community have an obligation to be here, 
to advocate in D.C., but also advocate throughout everywhere we 
are, in our hometowns, when we go travel. I mean, just talking 
to a young OB/GYN doctor whose son goes to school with another 
10-year-old with Down syndrome has a completely different idea 
than a doctor that was practicing 30 years ago. So I think by 
including our children, having them out in the community, in 
school, getting them jobs, helping them get jobs--just like you 
would a typical kid, by the way; you know, we all help our kids 
to get jobs--then they are proving themselves as role models 
and changing society.
    And the other thing is we support a lot of people who are 
in the performing arts. And that makes a huge difference. I 
don't know if you have heard of ``Born This Way,'' these TV 
shows and these actors, including Frank, in films, having those 
role models out there really does make a difference to 
mainstream America. All of a sudden, their lens starts to 
change, and they start to see a person with Down syndrome not 
only as a human being, which, if you haven't met a person with 
Down syndrome, could be the case, unfortunately, still in this 
country, but as that blonde cute gal from whatever State who is 
really good at cheerleading or math. So that is what we need to 
do as a community. And we have fabulous local Down syndrome 
organizations across the country who are doing that in their 
locations, and we work with them.
    Dr. Mobley. Just very quickly, I never--I don't know how to 
answer the question, but I know it is the right question. But 
just as a thought, if the National Academy of Medicine decided 
that physicians needed to take seriously the diverse folks in 
their practice, if they were to come out with a statement that 
looked at it--is it a problem, how much of a problem is it, who 
does it affect--if they were to come out with an authoritative 
study on this, it may well change the practice of medicine 
across the country. I could recommend that.
    Dr. Espinosa. Yeah. I would like to make a brief comment 
about promoting diversity in the scientific enterprise. And we 
have this amazing experience, you know, in Denver with the 
Linda Crnic Institute for Down Syndrome where, in 4 years, we 
recruited 38 labs that were not doing research on Down 
syndrome. You put the money; that is where your values are at. 
Scientists will come to it because the observations are 
fascinating, because scientists really do care, and they see an 
opportunity for major discoveries. So we have created a very 
diverse group of scientists who were not thinking about Down 
syndrome simply by putting the researchers there, you know, 
$5,200,000 in grants just in the last 5 years. Scientists will 
come to the Down syndrome research field if we give them an 
opportunity to have funded research.
    Ms. Herrera Beutler. Thank you.
    Mr. Cole. To Dr. Espinosa's point, this committee, just for 
the record, has done a lot in the area of Alzheimer's research 
for years. And I happen to be talking to the director in charge 
of that particular institute. I said: Is it making a 
difference?
    He said: You know, you begin to measure the difference by 
the research proposals you get, and they are up over 28 percent 
in a single year.
    Your point is very well made, that this money is a driver, 
obviously, in the decisions that are made.
    With that, I want to go to my good friend from California, 
Ms. Lee, for whatever questions she cares to pose to the panel.
    Ms. Lee. Thank you very much, Mr. Chairman. I want to thank 
yourself and our ranking member for this very important and 
powerful hearing. And I have a story I want to share in just a 
minute. But let me ask Julie Paulson from my district, Albany, 
California, to stand with Zach and Adeline.
    Thank you for coming so far. It is really wonderful to see 
you here. They traveled a long way. So thank you so much.
    I, first of all, want to thank you all so much for your 
very powerful testimony. But let me take a moment to share a 
personal story.
    My music teacher, her name was Mrs. Nixon. I grew up in El 
Paso, Texas. Mrs. Nixon and her husband--I think her husband 
was probably the first African-American physician or dentist in 
El Paso--they had a daughter with Down syndrome named Annie. 
Now, she brought Annie to our house for piano lessons every 
single Saturday. Mind you, I started playing the piano when I 
was 3 years old. I took piano lessons every Saturday from 3 to 
13--age 13. Annie, Mr. Stephens, lit up my life. She taught me 
so much as a child. I learned--you would not believe what I 
learned from Annie. And this is way in the day. We had fun 
playing together, and she made my life so much happier. And I 
just want you to know that because today I am thinking of 
Annie. And I listen to your testimony. I am looking at you, and 
I am so happy to see you here. So thank you very much.
    Let me say that I also have a sister living with multiple 
sclerosis. And so I wanted to ask you the relationship and the 
association with multiple autoimmune diseases as it relates to 
MS, and have you found any evidence of the connection between 
Down syndrome and multiple sclerosis?
    And then, secondly, as it relates to the issue of 
diversity, of course, we have young people who are transgender, 
we have huge gaps with people of color in terms of the research 
as it relates to Down syndrome in terms of many, many barriers, 
and so I would like to hear how this diversity plays out with 
people of color and with transgender individuals and others in 
terms of the research and what we are doing in terms of the 
standards of care.
    And, finally, I will just say I am one who wants to 
increase NIH's budget in all diseases by extra, by the millions 
and the billions, because NIH is a lifesaving institute, and it 
is really helping, you know, to shape the world in terms of 
diseases. And it is really important that we never ever cut NIH 
and don't rob Peter to pay Paul. I don't want to see us taking 
money from one research program and putting it in another 
because you all have so brilliantly and eloquently told us that 
we have got to tackle everything if we really are a life-
affirming country.
    So thank you very much.
    Dr. Espinosa. Yeah. That is a great question. I would 
address first the part about autoimmune conditions. The 
population with Down syndrome is likely the largest human 
population with a genetic predisposition to autoimmune 
disorders. With that being said, they don't have a higher risk 
of all autoimmune disorders. MS, which you mentioned, actually 
is not significantly up, as far as we can tell, in people with 
Down syndrome. Lupus is not up, but Hashimoto's hyperthyroidism 
is very prevalent. Celiac disease is very prevalent. Type 1 
diabetes is up. Autoimmune skin conditions are up.
    Without going into the technicalities of autoimmune 
conditions, this makes sense that we would get a particular 
flavor of autoimmune conditions based on the type of immune 
disregulation that they have, right? So the molecules that 
drive MS are different than the molecules themselves that drive 
other autoimmune conditions. So we definitely need more 
research to understand why they are predisposed to some 
conditions but not others.
    With regards to the comment about diversity, this is a very 
important area for us, because there are differences depending 
on the fragment of the community they look at. For example, the 
life expectancy for people with Down syndrome of African origin 
lineage is lower. You know, they haven't done as well in these 
massive increases in life expectancy that I shared with you 
about, and we don't know why. So we need more research in that 
area, what is going on with the African-American community.
    Another important fact is that the increase in the 
prevalence of live births is actually higher among Hispanics. 
We don't know why. We assume that may be because of religious 
beliefs, and, you know, a large fraction of that community 
being Catholic, maybe they choose to terminate less. But, 
again, we don't know why the rise in incidence is preferential 
to that fragment of the population.
    And then, finally, with regards to transgender, you know, 
and gender identity, to be honest with you, we don't have any 
research on that area to have an informed discussion about it. 
I don't know if Bill or Michelle can comment to that.
    Ms. Lee. Well, Mr. Chairman, I would just ask, how do we 
get funding for more research to address the entire population? 
That is what I want to know. We heard it.
    Mr. Cole. Well, that is--I have no more passion and 
advocate for this committee than my ranking member; I can tell 
you that.
    But we have, on a bipartisan basis, frankly, have tried to 
make this a priority the last couple of years. I think we will 
continue to do that. The real aim here long term is to not go 
through ever again a dozen years of a flat funding. And now I 
will point out directly we did that under both administrations. 
What we need is to shoot toward not a particular goal but a 
particular process whereby we add to this on a regular basis. 
This should be like a national investment. It yields enormous 
benefits to this country.
    And I am very proud of my ranking member's having been a 
partner in that. And I can assure you, when we negotiate with 
our friends on the other side of the rotunda, we have got some 
partners there too.
    Ms. Lee. Thank you, Mr. Chairman.
    I know your commitment and our ranking member's commitment. 
But I also want to make sure that we know and understand, as we 
conduct these negotiations, that, while we increase the 
funding--while we fight to increase the funding, we also look 
at where the gaps are and increase the funding for research 
based on where many of these gaps are, which, oftentimes, we 
don't do, so thank you very much.
    Mr. Cole. You will be proud to know your ranking member and 
I are already exchanging notes.
    Ms. Lee. Thank you very much.
    Thank you all. Good to see you.
    Mr. Cole. I want to go to our--go ahead, you should give 
her applause.
    [Applause.]
    Terrific member of this committee.
    I want to go to our last questioner, my friend, Mr. 
Moolenaar from Michigan. Before I do, I just want to point out 
and I hope you take note of this.
    Number one, it is very unusual, Members have a really busy 
schedule, so when Mr. Moolenaar comes, he came here at the very 
beginning, he stayed for the entire meeting. We have other 
members who have asked questions. They have very busy schedules 
too. Here they are still here. And you should take that as a 
compliment, honestly, as to how compelling and how informative 
the testimony is. Members really do--are listening very 
closely, and you have been very helpful, and this has been 
exceptional.
    With that, I am going to go to my friend from Michigan for 
whatever questions he cares to ask.
    Mr. Moolenaar. Thank you, Mr. Chairman. I want to thank you 
and the ranking member and the staff for putting this together. 
And I want to thank the panel for their testimony.
    I want to also thank the families who are here and for your 
patience. I know it has been a long hearing. I am at the end of 
the line here, new member of the committee, so I am the last 
one, I think, that will ask an original question.
    I especially want to thank Mr. Stephens. I know a lot of 
people have mentioned how compelling your testimony is. I have 
to tell you, I think that was one of the best speeches I have 
heard since I have been in Congress. So thank you for that. It 
reminded me of some of our founding documents about being 
endowed by our Creator with the right to life, and you 
certainly spoke so eloquently to that.
    And, Ms. Sie Whitten, I want to thank you for your work. 
You know, it struck me as you shared in your testimony about 
feeling somewhat pressured to terminate.
    Ms. Whitten. Oh, definitely.
    Mr. Moolenaar. And the fact that you and your husband chose 
not to and the fact--I don't think you would be here and your 
family wouldn't be here this way. And I think that is a very 
powerful thing, the impact that your entire family is having in 
educating all of us, because I had no idea of the research that 
is being done and the multiplier effect of that research as 
well. So thank you so much.
    And I also want to follow up, Dr. Espinosa and Dr. Mobley, 
with your comments. You have said a number of times about the 
need for a focused approach, a concerted effort. What strikes 
me is in so many areas it takes sort of this unifying message, 
the momentum, you know, to occur. You think of the cancer moon 
shot, you know. It kind of gave people a picture of what we are 
trying to do here. You know, you think about the original 
landing a man on the moon concept and that promoting kind of 
STEM education.
    And I just wonder, you have really talked about sort of the 
synergies here with this research, and again, as I mentioned, 
the multiplier effect. Is there a way to communicate this so 
that we kind of get the sense, the urgency that you have 
described today, and the tremendous bang for the buck in terms 
of the research dollar, so that we can help advocate for this 
with NIH and with our colleagues in Congress?
    Dr. Espinosa. I would like to comment on that. First of 
all, I would like to acknowledge that we have great allies at 
NIH. But we cannot set this up in a way that Peter robs Paul, 
because that is going to put our friends at NIH in a very 
difficult situation where they have to defund or fund less of 
something to fund more Down syndrome research.
    So if you as a committee allocate funds specifically for 
research on Down syndrome, our friends at NIH are going to find 
the creative ways to fulfill this mandate of a trans-NIH 
enterprise.
    When we go to NIH, and actually we do, all of us, go quite 
a lot and talk to the program officers there, they also learn 
about some of these fascinating observations and they want to 
help us, but they don't have any more money. Every cent is 
being budgeted for.
    So there are great success stories. I talk about the Office 
of AIDS Research. What about the Women's Health Initiative? 
That is a population that has a different chromosomal content. 
We realized that we were doing science in a gender, sex-
agnostic way. We mandated NIH to start paying attention, have 
scientists looking at both sexes in their experiments.
    So we can ask NIH for ideas on how to do the trans-NIH 
initiative. It is going to be very difficult for them to be 
creative without additional resources.
    Mr. Moolenaar. And it brings me to the point I think you 
made earlier. In 2006 there was a Down Syndrome Working Group 
established, and I believe that was congressionally directed. 
Has there been anything since that? And what would be the next 
steps to build on that momentum?
    Dr. Mobley. Yeah, thanks. No, that is true. And in 2006 
Congress asking for an effort to look at Down syndrome 
seriously and Down syndrome research, I think it changed the 
game. A lot has happened since that time.
    We found a champion in Yvonne Maddox, who was deputy 
director of NICHD at that time, and she as a singular person 
drove a task force that began to sketch the Down syndrome 
research landscape. We have seen a lot of development since 
then. There were meetings in 2013 and 2014 focused specifically 
on Alzheimer's disease and Down syndrome. The energy at NIH has 
increased. The number of new initiatives is multiplying.
    So it works. So when you say something, it makes a 
difference. I would ask you to say now: Let's harvest the 
wonderful ideas that are possible right now, looking at Down 
syndrome research, to make the world a better place for them 
and for the rest of us. Let's create a Down syndrome initiative 
at the NIH level that looks across disorders to understand how 
the science that we already know is there and that we will 
explore further can really change the game, not just for those 
with Down syndrome, but for the rest of us.
    I think if you name it, if you call it out, if you energize 
it, if you bring attention to this issue, great things will 
happen.
    Mr. Moolenaar. Thank you very much.
    Thank you, Mr. Chairman.
    Mr. Cole. Thank you.
    I want to go next to my ranking member for whatever closing 
remarks or thoughts she cares to give.
    Ms. DeLauro. Thank you very much, Mr. Chairman.
    And just to add to what everyone has said, it is an 
extraordinary panel, people who are in the medical profession, 
folks who enable the medical profession to do what you do, and 
in the person of Frank understanding what the wide range of 
abilities that exist and what we can really do to change, 
change people's lives. So I am grateful for all of that.
    I think in so many respects we need to--let me just ask, I 
would very, very much like to get information back on what the 
consortium is doing so to get some idea of all the institutes 
which are currently working. And I know the trans-NIH platform 
that you want to work with and how that might in essence be--is 
that possible under the consortium?
    How we can move given what is there so that we know exactly 
where we are going to look at where the gaps are and how to 
encourage it, in addition to the--not for answering now--but 
using the Precision Medicine Initiative, how you that might be 
applied to the Human Trisome Project.
    And so that would be enormously helpful in terms of the 
deliberations that we can make here.
    Just one last comment, Mr. Chairman. I said in my--at the 
moment the funding for the NIH is $6.5 billion below where it 
was. That means that in so many instances we are robbing Peter 
to pay Paul and that there has to be an understanding, I 
believe particularly in the health area, which is why I 
proposed doing what we do in terms of how we look at fraud and 
abuse in that account, is that we do not attribute that to the 
budget. So that is outside.
    And so for the NIH and the funding of NIH, which I have 
suggested for the last several years that we do something that 
is very similar, the Accelerating Biomedical Research Act, 
which would allow us to go above the caps. It is a mechanism, 
as I say, we use for looking at fraud and abuse. Why not allow 
us to use that kind of mechanism to go above on the caps?
    Because, I say that because we do a lot of things at the 
Federal level. We build helicopters. We deal with agricultural 
subsidies. We do roads, bridges, all kinds of things. But in my 
view, there is nothing more important that we do than to save 
people's lives.
    And as I said to my colleagues who are here, we are all 
blessed, because we are in an institution that has the ability 
to push that edge of the envelope in order to be able to save 
lives. And we ought to think about what it is that we do to 
connect those dots and look at where our priorities lie, and 
that is the overall, overarching goal that we have. And the 
utilization of this institution to do that seems to me to be a 
profound obligation and a moral responsibility that we have.
    You are doing what you do and you have the moral 
responsibility, the social responsibility, which you are 
carrying out your mission. I want us to be able to do that with 
the kind of resources we need to be able to effectively allow 
the discovery that you speak about.
    Thank you.
    Mr. Cole. I will say, as I have told my good friend before, 
she is endlessly creative about how you get around caps and 
allocations. But it is always for a good cause, always for a 
good cause.
    I want to begin just number one by thanking all of you. I 
want to thank the witnesses that were here. I want to thank the 
advocates and obviously the young people and some not so young 
that are dealing with this disease that are here today. It is a 
very powerful impression and very, very helpful to us.
    Ms. Herrera Beutler in her remarks made a really important 
point, which is Congress is usually not where things begin. 
They usually begin someplace else and work their way toward 
Congress. And you have all done an extraordinary service simply 
by educating this committee and offering powerful personal 
testimony and expert medical testimony that is really 
profoundly moving.
    I have had the great privilege to chair this subcommittee 
for 3 years and all of us have participated in lots of 
committee hearings, but I don't think any of us have ever in 
one quite like this, quite this powerful and quite this 
helpful.
    My home State's favorite philosopher is Will Rogers, and he 
used like to say, we are all ignorant, only about different 
things. And you have really informed this committee in a way 
and helped us overcome maybe some areas where some of at least 
were not as well informed as we would like to be. That is 
extraordinarily helpful.
    So again, I want to thank you.
    I want to thank the committee. I thought this was some of 
the best questions we have had and some of the, frankly, the 
most intensity in terms of feeling on both sides of the aisle. 
And that is a good thing. This is something that brings us 
together. It doesn't tear us apart. Quite the opposite. So it 
was good to hear.
    And I want to tell you that we are listening very carefully 
to what you had to say. And we are very careful about intruding 
too deeply in terms of making decisions, but there are times 
when there are gaps, and you very powerfully, I think, pointed 
to where we have a gap.
    And if we have our collective way, we might not agree on 
the amount, but this subcommittee will once again figure out a 
way to add money over at NIH as we have the last 2 years, and I 
know my ranking member wants to do again, and, frankly, I know 
my colleagues in the Senate want to do again as well. And this 
is something we will certainly take into consideration as we do 
that.
    So again, I just want to thank each of you for taking the 
time. I know it is a disruption to your day to travel this way.
    You were all superb. Mr. Stephens, you are clearly the star 
of the show, and no wonder you are advocate extraordinaire. But 
I thought that was, like my friend Mr. Moolenaar, one of the 
most moving testimonies I have ever heard. And it was not just 
the technical expertise, but the profound questions that we 
wrestle with and that you highlighted very, very well. And I 
want to personally thank you for coming here.
    I want to thank you two gentlemen too in terms of spending 
your time and your expertise in the area. I was very touched, 
Mr. Mobley, when you talked about how you ended specializing 
here and the discouragement I am sure each of you have gotten 
because maybe we have not--I used to be an academic and 
resources do attract you. And you guys clearly made decisions 
when there wasn't much in the way of resources and in that you 
have made an extraordinary contribution.
    And, Ms. Sie Whitten, I mean, my goodness, how to take a 
tragedy and turn it into a triumph for all people and to take 
your personal experience and make it meaningful for the country 
and for a particular community that has been overlooked. You 
should be extraordinarily proud of what you have done and how 
you have dealt with. It is an amazing effort.
    So with that, I am going to end the hearing, but again, I 
want to thank each and every one of you for coming.


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