NIH IN THE

21st CENTURY:
ENSURING TRANSPARENCY AND
AMERICAN BIOMEDICAL LEADERSHIP
REPORT TITLE GOES HERE:
AND THE REPORT SUBTITLE WILL GO HERE
Introduction

The National Institutes of Health (NIH) funds more biomedical research than any other public institution in the
world, making the agency a key driver of the United States’ strategic advantage in science and technology. NIH
consists of 27 institutes and centers (ICs) and has a budget of more than $49 billion. Yet NIH’s authorization of
appropriations lapsed at the end of fiscal year (FY) 2020, and Congress’ last large-scale review of the agency’s
activities and authorities occurred nearly a decade ago, culminating in the enactment of the 21st Century Cures
Act.

As Ranking Member of the Senate Health, Education, Labor, and Pensions (HELP) Committee, I issued a
request for information (RFI) from stakeholders in September 2023 seeking feedback on NIH’s current activities
and statutory framework. Respondents to the RFI ranged from academic institutions; relevant professional
societies, think tanks, and trade associations; patient advocates; and individual researchers sharing their
observations from careers working within the NIH ecosystem. Respondents identified multiple opportunities for
legislative action that would build upon past congressional efforts to strengthen NIH and the U.S. biomedical
research enterprise. In this white paper, I put forward a selection of proposals to invite discussion of policies
that may be appropriate for legislative action. I look forward to working with my colleagues in Congress and all
interested stakeholders to ensure that NIH is well prepared to seize scientific opportunities to advance the health
and wellbeing of Americans in the decades to come.

Brief Legislative History

NIH began in 1887 with the creation of the Laboratory of Hygiene as part of the Marine Hospital Service, the
predecessor to the modern Public Health Service. Over the next 40 years, Congress repeatedly charged the
Marine Hospital Service via the Hygiene Laboratory with researching specific infectious diseases and other
conditions.1 In 1930, Congress passed legislation sponsored by then-Louisiana Senator Joseph E. Ransdell to
rename the Hygiene Laboratory the National Institute of Health and created research fellowships within the
Institute.2 The National Institute of Health began building its current Bethesda, Maryland headquarters through
a series of land donations and congressional appropriations later that decade.3

In 1944, Congress enacted the Public Health Service Act, which revised the previous patchwork of federal
public health laws into a more cohesive framework to support biomedical research, disease control, and patient
care. The Public Health Service Act reorganized the National Cancer Institute into the National Institute of
Health, provided authorities that eventually led to the establishment of the NIH Clinical Center, and created
other divisions to support NIH’s biomedical research mission.4 Between the 1940s and 1970s, Congress enacted
multiple laws to establish new ICs within NIH, make “institutes” plural in the agency’s name, expand its
authorities to support biomedical research capacity, and prioritize certain areas of research.5

1 Chronology of Events, NATIONAL INSTITUTES OF HEALTH, https://www.nih.gov/about-nih/what-we-do/nih-almanac/

chronology-events (last updated Oct. 5, 2023) (during this period, Congress changed the name from the Laboratory of Hygiene to the
Hygiene Laboratory).
2 WWI and the Ransdell Act of 1930, NATIONAL INSTITUTES OF HEALTH, https://history.nih.gov/display/history/
WWI+and+the+Ransdell+Act+of+1930 (last visited Apr. 29, 2024).
3 Id.
4 Id.
5 NATIONAL INSTITUTES OF HEALTH, supra note 1.
1
In the 1980s, Congress focused on incentivizing the commercialization of federally funded research through
laws such as the Bayh-Dole Act, the Small Business Innovation Development Act, and the Stevenson-Wydler
Act.6 While not limited to NIH, these laws profoundly reshaped NIH’s intramural and extramural research
operations and emphasized the importance of translational science. In the 1990s, Congress authorized the
Foundation for NIH, which supports intramural scientists and helps broker NIH’s public-private partnerships,
and passed the NIH Revitalization Act, which established multiple new components of NIH and the
Department-level Office of Research Integrity, allowed NIH to support extramural construction activities,
established the Institutional Development Award (IDeA) program, and created scholarship and loan repayment
programs.7

Over the past two decades, Congress has enacted multiple laws to continue improving NIH operations. In
2000, Congress passed the Public Health Improvement Act, which supported biomedical research capacity,
and the Children’s Health Act, which established the NIH Pediatric Research Initiative. Congress enacted the
NIH Reform Act in 2007, which addressed NIH’s structure, operations, and public transparency.8 Specifically,
the NIH Reform Act created the Common Fund to support crosscutting NIH research initiatives of strategic
and scientific importance and the Council of Councils to advise the Director on administering the Common
Fund and carrying out other crosscutting NIH research initiatives. The law also consolidated authorizations
of appropriations for individual NIH programs into a single authorization level for the agency, created the
Scientific Management Review Board to advise on NIH’s structure and organization, and directed NIH to
establish an electronic system for reporting and categorizing NIH-funded research.9 The current system
is known as the Research Portfolio Online Reporting Tools (RePORT). As part of the Food and Drug
Administration Amendments Act of 2007, Congress directed NIH to expand the information included about
ongoing studies on clinicaltrials.gov and required certain trials to be registered with and report results on the
website. The 21st Century Cures Act (commonly referred to as “Cures”), enacted in 2016, built on the NIH
Reform Act by emphasizing the importance of strategic planning, streamlining administrative requirements,
providing additional authorities to the NIH Director, and establishing a process for the periodic review of each
IC Director’s performance.10 The 21st Century Cures Act also included policies to encourage more high-quality
research, support the next generation of scientists, and facilitate the participation in research of pregnant and
postpartum women, children, and other underrepresented populations. The law also provided one-time funding
for certain large-scale research initiatives. Despite these bipartisan legislative successes and consistent, annual
investment in NIH, opportunities remain to improve NIH’s operations to better support the U.S. biomedical
research enterprise.

6 Id.
7 Id.
8 Id.
9 Judith A. Johnson & Kavya Sekar, The National Institutes of Health (NIH): Background and Congressional Issues, CON-
GRESSIONAL RESEARCH SERVICE 5 (Apr. 19, 2019), https://www.crs.gov/reports/pdf/R41705/R41705.pdf.
10 Amanda K. Sarata, The 21st Century Cures Act (Division A of P.L. 114-255), CONGRESSIONAL RESEARCH SERVICE
(Dec. 23, 2016), https://www.crs.gov/reports/pdf/R44720/R44720.pdf.
2
 Maximizing the Effectiveness of Current NIH Funding
Balancing NIH’s Portfolio

Each year, NIH dedicates 80 percent of its budget to extramural research activities and an additional 11 percent
to its intramural research program.11 NIH annually supports approximately 50,000 new or ongoing extramural
projects, over 30,000 of which (60 percent) are R01-equivalent grants.12 R01-equivalent grants support specific,
often investigator-initiated research activities for a limited duration of three to five years.

A recent analysis from NIH’s Office of Extramural Research found that applied projects represent a growing
portion of NIH’s extramural portfolio. In FY22, over 45 percent of R01 grants and 50 percent of all awards were
for applied projects, compared to approximately 38 percent and 42 percent, respectively, in FY09.13 Figure 1 is
taken from this Office of Extramural Research analysis and demonstrates this shift toward applied research in
terms of overall number of projects.14

This shift toward applied research is consistent with recent government-wide trends because applied research
often has a more immediate, tangible impact on Americans’ daily lives (evident in developments like the
creation of the Advanced Research Projects Agency for Health and the National Science Foundation’s (NSF)
new Directorate for Technology, Innovation, and Partnerships).15 However, many respondents expressed
concern that NIH’s increasing focus on translational and clinical research will come at the expense of
investigator-initiated, basic science, the historic heart of NIH’s business model. The U.S. Government plays a
unique role in supporting basic discovery, which enables long-term biomedical innovation. One RFI respondent
noted that the Office of Management and Budget (OMB) has historically directed NIH to dedicate at least 55

Figure 1. NIH Extramural Research Portfolio, FY 2009-2022

14

11 Francis S. Collins, NIH-Wide Strategic Plan for Fiscal Years 2021–2025, NATIONAL INSTITUTES OF HEALTH 1 (July
30, 2021), https://www.nih.gov/sites/default/files/about-nih/strategic-plan-fy2021-2025-508.pdf.
12 Id.; see also NIH Data Book, R01-Equivalent Grants, NATIONAL INSTITUTES OF HEALTH,
https://report.nih.gov/nihdatabook/category/3 (last visited Apr. 29, 2024).
13 Mike Lauer, Trends in NIH-Supported Basic, Translational, and Clinical Research: FYs 2009-2022, NATIONAL INSTI-
TUTES OF HEALTH OFFICE OF EXTRAMURAL RESEARCH (Oct. 31, 2023),
https://nexus.od.nih.gov/all/2023/10/31/trends-in-nih-supported-basic-translational-and-clinical-research-fys-2009-2022/.
14 Id.
15 Technology, Innovation and Partnerships, U.S. NATIONAL SCIENCE FOUNDATION, https://new.nsf.gov/tip/latest (last
visited Apr. 29, 2024).
3
percent of its portfolio to basic research in recognition of this unique role, and some previous NIH directors
have sought to exceed that recommended level. Waning federal focus on basic research could lead to a decline
in treatments and cures eventually developed through private funding. Unlike certain areas of clinical research,
the private sector would not be equipped to fill gaps in support for basic research.

Another challenge within NIH’s research portfolio is balancing the need to direct research toward specific topics
of significant public health interest (referred to as “targeted research”) with investigator-initiated research. In
FY23, targeted research comprised 12 percent of all new research project grants made by NIH, a total of 20
percent of all new research project grant funding awarded.16 This targeted funding ties back to more than 60
discrete funding levels specified by Congress in the FY23 annual appropriations report language.17 On the one
hand, direction from Congress and NIH senior
leadership can be beneficial to focus the research Box 1. Nutrition and Obesity Research
community on high-impact areas of research and
ensure NIH is appropriately prioritizing work Obesity is correlated with multiple leading causes
to address diseases of significant unmet need. of death in the U.S. and contributes more than $147
For example, Box 1 briefly describes NIH’s billion annually in medical costs. However, one
underinvestment in nutrition and obesity research, analysis found that NIH dedicated a mere $1.9 billion
an area of public health importance. However, to nutrition research in FY19, and only 1.3 percent of
too much direction can warp the portfolio as a all NIH-funded research projects addressed the role
whole, dissuading investigators from pursuing of diet and nutrition in the prevention and treatment
other research topics that could have broad benefit
of disease. This compares to over $3.8 billion spent
and to create a naturally diversified portfolio.18
in the same year on cardiovascular disease and $16.9
Further analysis of NIH’s internal data could
billion on Type 2 diabetes research.
help the research community, policymakers,
and stakeholders better understand how to
appropriately balance these competing interests. At the same time, NIH attempted to shut down
its intramural metabolic research unit, one of a
Reducing Redundancy and Finding Efficiencies handful of facilities around the country capable of
conducting complex clinical research to evaluate the
Reducing redundancy could improve the balance metabolic effects of diet. This historic devaluing of
of NIH’s portfolio by making more resources the importance of nutrition science likely resulted in
available within NIH’s existing budget. Under missed opportunities to improve the rigor of nutrition
statute, the NIH director is responsible for research and indicates a misalignment between NIH
classifying and publicly reporting NIH-funded spending and public health impact.
research projects and conducting priority-setting

16 NIH Data Book, Research Project Grants, NATIONAL INSTITUTES OF HEALTH, https://report.nih.gov/nihdatabook/cate-
gory/4 (last visited Apr. 29, 2024).
17 National Institutes of Health (NIH) Funding: FY1996-FY2024, CONGRESSIONAL RESEARCH SERVICE 13-17 (May 17,
2023), https://crsreports.congress.gov/product/pdf/R/R43341.
18 Box 1 Citations; NIH RePORTER, NATIONAL INSTITUTES OF HEALTH, https://reporter.nih.gov/search/4iuRVEm-
SKUG9nSmDJCJB4A/projects/charts (last visited Apr. 29, 2024); Sheila Fleischhacker et al., Strengthening national nutrition re-
search: rationale and options for a new coordinated federal research effort and authority, AMERICAN SOCIETY FOR NUTRITION
(Jul. 20, 2020), https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7454258/; Catherine Boudreau & Helena Bottemiller Evich, How
Washington keeps America sick and fat, POLITICO (Nov. 4, 2019), https://www.politico.com/news/agenda/2019/11/04/why-we-dont-
know-what-to-eat-060299; Obesity Basics, CENTERS FOR DISEASE CONTROL AND PREVENTION, https://www.cdc.gov/obesi-
ty/basics/index.html (last updated June 3, 2022).
4
reviews for the agency.19 However, it is unclear what steps current and former directors have taken to formally
conduct these reviews and whether analysis of NIH’s internal and public-facing data contributes to the reviews.
These reviews and corresponding analyses should provide a unique opportunity for the NIH director to identify
areas of research duplication and potential collaboration between ICs.

In addition to duplication within IC research portfolios, NIH should also consider potentially redundant
research infrastructure. For example, each IC supports its own clinical trial networks that may have overlap in
their capabilities and purposes. During the planning process for NIH’s Accelerating COVID-19 Therapeutic
Interventions and Vaccines (ACTIV) initiative, an NIH working group assessed the capabilities of 60 clinical
trial networks to identify the most appropriate sites through which to carry out ACTIV trials.20 NIH should
build on this assessment to explore integrating its existing trial networks into a more cohesive, enterprise-
wide clinical trials capability. As part of this process, NIH should consider how best to reach patients who
may be prospective research participants. For example, the National Cancer Institute’s Community Oncology
Research Program (NCORP) is a community-based model for trial design that brings clinical trials directly to
patients through their local hospitals and doctors’ offices. The Council of Councils recently approved a proposal
from NIH Director Bertagnolli to establish an NIH-wide primary care clinical trial network over a five-year
period.21 Community-based clinical trial networks that more centrally involve non-degree granting institutions
could improve the accessibility of clinical trials for patients across the country. However, any new network
must account for and build upon existing capabilities without exacerbating existing duplication within NIH’s
portfolio.

Learning from Scientific Successes and Failures

Other untapped resources include data NIH and its extramural partners possess on applications for funding
and the outcomes of funded projects. NIH currently publishes data on which projects and researchers receive
funding (commonly referred to as the “success rate”).22 However, more granular data about how specific
proposals fare through the peer review process and are ultimately selected or rejected for funding are not
available. Several respondents to my RFI stated that access to this data would enable researchers to conduct
metascience research on the scientific process. NIH’s Office of Portfolio Analysis has supported some
metascience initiatives by aggregating information related to citations of published papers and contributing
to the development of best practices in the field.23 Piloting a process for the secure sharing of NIH application
and review data with trusted researchers would help identify or validate trends within NIH processes and
recommend process improvements.

Researchers also often lack insight when experiments produce null or inconclusive results (collectively
referred to as “negative results”) because these findings are typically not published in academic journals. Yet,
negative results can themselves advance lines of scientific inquiry, such as questioning or ultimately refuting a

19 Public Health Service Act § 42 U.S.C. § 282(b); 42 U.S.C. § 282(b)(3).

20 Clinical Trial Capacity Working Group, NATIONAL INSTITUTES OF HEALTH, https://www.nih.gov/research-training/
medical-research-initiatives/activ/clinical-trial-capacity-working-group (last updated Dec. 23, 2020).
21 Council of Councils Meeting, Agenda, NATIONAL INSTITUTES OF HEALTH, https://dpcpsi.nih.gov/council/april-5-2024-
agenda (last updated Apr. 5, 2024).
22 NIH Success Rates, NATIONAL INSTITUTES OF HEALTH, https://report.nih.gov/funding/nih-budget-and-spending-data-
past-fiscal-years/success-rates (last visited Apr. 29, 2024).
23 George M. Santangelo, Office of Portfolio Analysis Strategic Plan FYs 2021-2025, NATIONAL INSTITUTES OF HEALTH
OFFICE OF PORTFOLIO ANALYSIS 19 (Aug. 27, 2023), https://dpcpsi.nih.gov/sites/default/files/OD-OPA-FY2021-2025-SP-Pub-
lic-508_11_1_2021.pdf#page=19.
5
hypothesis, pointing investigators in more fruitful directions, or identifying flaws in a methodology. While some
peer-reviewed journals do accept negative results for publication, the direct expenses and opportunity costs to
the researcher associated with preparing an article, along with any perceived reputational costs associated with
an unsuccessful experiment, may serve as a disincentive to publish these results. NIH should consider strategies
to encourage the voluntary sharing of negative results, such as the establishment of a negative results repository
within the National Library of Medicine.

Sustaining the United States’ Competitive Advantage in Biomedical Research

According to some estimates, every dollar NIH spends on research generates $2.46 in economic activity.24
FY23 investments provided support for over 400,000 jobs and generated nearly $93 billion.25 However, China’s
aggressive investment in biomedical research, such as through the Thousand Talents program and Made in
China 2025, threatens the US’ long-term competitive advantage. Winning the biomedical research arms race
requires a critical review of NIH’s current policies and practices to better support the domestic biomedical
research enterprise.

Incentivizing Innovation

Respondents to my RFI noted that NIH’s extramural research programs tend to reward incremental science,
rather than high-risk, but potentially transformational studies and identified the current approach to peer review
as a driver of incrementalism. Peer review is foundational to the NIH model, and peer reviewers provide
an invaluable service to the research enterprise by volunteering their time and expertise. However, some
respondents noted that the heavy involvement of subject matter experts—rather than “generalists” who are not
as embedded in a particular area of research—can bias study sections toward the approaches and methodologies
favored by such experts. Respondents also noted that this phenomenon leads peer review committees to focus
heavily on the mechanics of the proposed methodology and likelihood of success, rather than the overall
scientific vision and potential impact of the proposal. The structure of funding applications lend themselves
to such a discussion during peer review: R01 applications consist of a one-page statement of objectives and a
12-page research strategy, which includes discussion of proposed methodology and preliminary results.26 As
a result, respondents noted that researchers face significant pressure to demonstrate proof of concept through
robust preliminary data prior to applying for funding. Respondents also noted inconsistencies between peer
reviewers, review cycles, and situations when peer reviewers turn over mid-review. In some cases, researchers
might even tailor their application to target specific study sections that may be more receptive to their
proposal. To address these myriad challenges, NIH should pilot and evaluate multiple approaches to changing
the application and peer review process, including staffing committees with more generalists, streamlining
application discussion of methodology and preliminary results, regularly reviewing the focus and members of
study sections to promote alignment with current science, and improving training of peer reviewers to make
reviews more consistent.

NIH’s Small Business Innovation Research (SBIR) and Small Business Technology Transfer (STTR) programs

24 NIH’s Role in Sustaining the U.S. Economy, UNITED FOR MEDICAL RESEARCH 1 (Mar. 2024), https://www.unitedfor-
medicalresearch.org/wp-content/uploads/2024/03/UMR-NIHs-Role-in-Sustaining-the-US-Economy-2024-Update.pdf.
25 Id.
26 Write Your Research Plan, NATIONAL INSTITUTE OF ALLERGY AND INFECTIOUS DISEASES, https://www.niaid.nih.
gov/grants-contracts/write-research-plan#:~:text=The%20Research%20Strategy’s%20page%20limit (last updated July 26, 2017).
6
also demonstrate room for administrative improvement to better achieve program goals. Respondents
specifically cited as barriers NIH’s longer window to make awards compared to other Federal departments that
operate their own SBIR and STTR programs. For example, the Department of Defense takes three months,
on average, to notify successful applicants of an award, compared to NIH’s six and a half months.27 NIH
also uses similar program management and peer review structures for its SBIR and STTR programs to those
described above for other extramural research. A recent National Academies of Sciences, Engineering, and
Medicine review recommended that Congress and NIH explore piloting ways to make NIH SBIR and STTR
programs more timely and responsive to the needs of small businesses.28 The John S. McCain National Defense
Authorization Act for Fiscal Year 2019 directed the Department of Defense to establish a similar pilot program
to speed up its SBIR and STTR review and awards process.29 Some respondents to my RFI recommended
NIH take a more venture capital-style approach to its administration of the program by exempting from, or
dramatically reworking, the peer review process for SBIR and STTR to speed application review timeframes
and hiring individuals with venture capital or other business expertise to serve as program officers. NIH’s
successful Rapid Acceleration of Diagnostics (RADx) program during the COVID-19 pandemic provides
one model for how NIH could quickly and effectively assess the promise and feasibility of SBIR and STTR
proposals.

Program officers also play a key role in NIH’s extramural research prioritization and funding decisions. As
staff scientists responsible for managing grant portfolios, program officers advise prospective applicants on the
relevance of their proposed projects, develop requests for proposals and targeted research opportunities, and
provide expertise to the agency.30 NIH program officers are typically permanent employees of the agency who
develop specific areas of interest and expertise over time. Respondents pointed out that other agencies, such
as the NSF, employ temporary program officers through “rotator” programs.31 Similarly, advanced research
projects agencies across the federal government limit the term of their program managers to only a few years.
These time-limited appointments enable more individuals to work within the agency, inject the agency with
fresh ideas and up-to-date scientific knowledge, and take their understanding of the agency and its priorities
back to their research positions following the conclusion of their rotation. NIH should pilot a rotator approach to
fill program officer positions and related key roles.

Fundamental to the success of NIH’s model is collaboration: with researchers and their institutions, small
businesses, and larger biopharmaceutical firms via formal public-private partnerships. Without robust
partnerships, NIH would not have the same degree of impact on the advancement of science and economic
activity. As Congress and NIH explore strategies to strengthen and improve these partnerships, policymakers
must avoid the temptation of policies, such as the abuse of march-in rights, that would ultimately have a chilling
effect on collaboration. In addition to undercutting the goals of the Bayh-Dole Act, the use of march-in rights to
address drug prices would explicitly violate congressional intent, according to the bipartisan authors of the law,
former Senators Birch Bayh (D-IN) and Bob Dole (R-KS).32

27 Assessment of the SBIR and STTR Programs at the National Institutes of Health, NATIONAL ACADEMIES OF SCIENCES,
ENGINEERING, AND MEDICINE 78 (Feb. 9 2022), https://www.ncbi.nlm.nih.gov/books/NBK577834/.
28 Id. at ix.
29 15 U.S.C. § 638 as amended by P.L. 115-232 § 854(b).
30 Program Officers SOP, NATIONAL INSTITUTE OF ALLERGY AND INFECTIOUS DISEASES, https://www.niaid.nih.
gov/research/program-officers (last updated June 9, 2020).
31 Rotator Programs, U.S. NATIONAL SCIENCE FOUNDATION, https://new.nsf.gov/careers/rotator-programs (last visited
Apr. 29, 2024).
32 Robert Dole & Birch Bayh, Our Law Helps Patients Get New Drugs Sooner, THE WASHINGTON POST (Apr. 10, 2002),
https://www.washingtonpost.com/archive/opinions/2002/04/11/our-law-helps-patients-get-new-drugs-sooner/d814d22a-6e63-4f06-
7
Supporting the Biomedical Research Workforce

A core component of the modern scientific research and development enterprise is investment in the scientific
workforce to enable future discoveries.33 Along with other Federal research agencies, NIH invests heavily
in fellowships and training programs and promoting STEM education. However, the realities of extramural
research funding have resulted in a system that works well for certain institutions and investigators, at times to
the detriment of others.

Respondents cited institutions’ use of “soft money”—reliance upon NIH funding to reimburse themselves, in
part or in whole, for researchers’ salaries. One respondent referred to this practice as a rarely discussed but
glaring conflict of interest: if researchers are dependent upon successfully securing NIH grants in order to retain
their jobs, they are likely to avoid taking risks in the projects they propose for NIH funding. This practice also
has a disproportionately negative effect on early-stage investigators, who typically have lower success rates than
established investigators (see Figure 2).

Other respondents pointed to the use of clinical margins to cover research costs. In some cases, this could give
physician-scientists more ability to remain in research compared to basic scientists who do not have these types
of alternative funding sources. However, it could also create a perverse incentive for physician-scientists to
dedicate most of their time to clinical care, rather than research activities. These points mirror findings from the
NIH Advisory Committee to the Director (ACD) Next Generation Researchers Initiative Working Group.34
Other respondents highlighted that postdoctoral researchers are increasingly choosing careers in industry or
nonacademic research institutions over academia. A 2014 National Academies study attributed this trend to

8da3-d9698552fa24/.
33 Vannevar Bush, Science the Endless Frontier: A Report to the President, OFFICE OF SCIENTIFIC RESEARCH AND DE-
VELOPMENT (July 1945), https://www.nsf.gov/od/lpa/nsf50/vbush1945.htm#ch4.
34 NIH Advisory Committee to the Director (ACD) Next Generation Researchers Initiative Working Group Report, NATIONAL
INSTITUTES OF HEALTH 21 (Dec. 2018), https://acd.od.nih.gov/documents/presentations/12132018NextGen_report.pdf.
8
more clearly defined roles, higher salaries, and
more clear career development opportunities.35 Box 2. Next Generation Researcher Initiative
NIH’s funding model relies heavily on academic
research institutions and the complementary The 21st Century Cures Act (P.L. 114-255)
role that academic researchers play relative to established a Next Generation Researchers
government intramural or industry-conducted Initiative within the Office of the NIH director.
science. Respondents noted that established Senators Susan Collins (R-ME) and Tammy Baldwin
academic investigators also are expected to cover (D-WI) championed this policy. In response to
training costs through research grants but are often the directive, then-director Dr. Francis Collins
not incentivized to dedicate time to mentoring commissioned a working group of the Advisory
trainees.
Committee to the Director, which led to the
development of 30 recommendations, including
Taken together, these observations demonstrate
recommended definitional changes, monitoring
severe structural problems in how government and
progress under the Initiative, updating relevant
academia together finance biomedical researchers.
Some respondents recommended increasing data (including a 2007 report on how much NIH
the amount of a single NIH grant that can be funding goes toward extramural salaries), providing
used for salaries. On the other hand, the Next professional development opportunities for trainees
Generation Researchers Initiative Working Group listed on NIH funding applications, and reducing
previously explored lowering this amount to reduce programmatic bias that benefits established
institutions’ reliance on NIH funding for salaries.36 investigators. NIH has updated its policies in
Congress and NIH should explore this problem in response to these and related recommendations.
further detail and identify policy options that ensure For example, NIH announced that, beginning in
NIH and institutions each provide appropriate 2025, a new peer review scoring rubric will go into
degrees of support for federally funded academic effect that is intended to reduce reputational bias.
researchers. These changes benefit both early-stage investigators
and those who are established but from less well-
Reimagining the Intramural Program
resourced institutions.
NIH’s Intramural Research Program is the
world’s largest biomedical and behavioral
research institution, consisting of over 1200 principal investigators, 1800 clinicians and scientists, and 5000
trainees.37 Respondents noted that the intramural program in many cases performs research that is similar to,
or functionally the same as, projects funded under the extramural program. While the intramural program does
currently possess unique capabilities, such as the NIH Clinical Center, this observation raises questions about
how to distinguish the intramural program’s role and reduce potential duplication between the two portfolios.

Respondents suggested reorganizing the intramural program into interdisciplinary, inter-IC programs, similar
to NIH’s Porter Neuroscience Research Center, in which over 800 scientists from across NIH work together
on research that cuts across the expertise of individual ICs.38 This reorganization would enable the intramural

35 The Postdoctoral Experience Revisited, NATIONAL ACADEMIES OF SCIENCES, ENGINEERING, AND MEDICINE
(2014), https://nap.nationalacademies.org/catalog/18982/the-postdoctoral-experience-revisited.
36 NATIONAL INSTITUTES OF Health, supra note 30, at 20-21.
37 Collins, supra note 11, at 12.
38 The John Edward Porter Neuroscience Research Center, NATIONAL INSTITUTES OF HEALTH, https://www.nih.gov/
about-nih/john-edward-porter-neuroscience-research-center (last reviewed July 21, 2015).
9
program to more easily tackle complex problems that cannot be easily addressed through extramural projects,
for reasons such as their capital-intensive nature or anticipated time horizon. One respondent recommended
looking to the National Aeronautics and Space Administration’s (NASA) practice of using “decadal surveys,”
through which the National Academies convenes scientists to identify “the most compelling science
questions.”39 These surveys guide NASA’s strategic investments over the successive decade. The use of this
model should not preclude the reallocation of resources to address unforeseen needs over the next ten years.
For example, NIH would need to be able to adapt to the emergence of a new pathogen of pandemic potential or
disruptive technology. Yet, a decadal survey could help create buy-in across the scientific community and the
general public on NIH’s strategic priorities and ensure its intramural portfolio addresses areas of true unmet
need. NIH’s existing five-year strategic planning process could be adapted to account for a decadal survey, with
interim updates.

This reimagined intramural program could also benefit from a mix of staffing approaches. Principal
investigator-led permanent laboratories may still play a necessary role under this new model. At the same time,
a rotator model could bring in academic researchers for a defined period of time to work on interdisciplinary
projects to advance the priorities established in the decadal survey.

Restoring Public Trust in Science

NIH-funded research provides significant opportunities to improve the health and wellbeing of all Americans.
However, public sentiment toward scientific institutions has degraded in recent years, exacerbated by a
perceived lack of transparency and concerns about political biases during the COVID-19 pandemic response. In
order for the potential of future NIH research to be fully realized, the agency must make restoring public trust in
the scientific process and scientific institutions a priority.

Ensuring Transparency into NIH Operations

Rebuilding public trust in NIH will require improving meaningful transparency and public discourse about NIH
operations. While NIH is a highly visible agency, members of Congress have expressed concern about a lack
of engagement with congressional oversight requests related to the COVID-19 pandemic response. Similarly,
respondents noted instances of the agency deprioritizing statutory requirements, such as the lack of engagement
of the Science Management Review Board (SMRB). Established under the NIH Reform Act, the SMRB is
tasked with reviewing NIH’s structure and making recommendations every seven years. However, NIH has not
convened the SMRB since 2015, and one of its only recommendations prior to that time—the consolidation of
two ICs with similar research focuses—was disregarded by agency leadership. As an initial step to improving
transparency into NIH operations, Congress should reconstitute the SMRB and incorporate the perspectives of
individuals from outside the scientific community to inform SMRB recommendations to the director.

Transparency and evaluation are hallmarks of the scientific process. Many of the policies proposed in this white
paper could have significant effects, both intended and unintended, on the U.S. biomedical research enterprise,
and should therefore be piloted on a voluntary basis. Respondents also noted that NIH is still heavily reliant on
paper-based processes and human review, despite having significant technological capabilities. Respondents
suggested applying a more scientific approach to how NIH runs its programs, such as using machine learning

39 Decadal Survey, Biological and Physical Sciences Research in Space, THE NATIONAL AERONAUTICS AND SPACE
ADMINISTRATION, https://science.nasa.gov/biological-physical/resources/decadal-surveys/ (last visited Apr. 29, 2024).
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to better predict which proposals will yield the most transformative science. NIH should establish an initiative
to use scientific tools to evaluate the impact of NIH operations and policies closer to real time. NIH should
publicize the results of these evaluations to inform policy recommendations. While NIH does engage in these
types of analyses for specific issues, a more formal initiative would help NIH measure the value and tradeoffs of
piloted policy reforms and identify other interventions.
Promoting Research Integrity

Research misconduct is another major issue facing NIH. Recent high-profile cases of research misconduct,
specifically within Alzheimer’s research, and the potential applications of artificial intelligence to data
fabrication and falsification raise questions about how NIH can protect the integrity of its research investments.
HHS’ Office of Research Integrity (ORI) is responsible for developing research misconduct policies and
conducting investigations on behalf of NIH and other HHS public health agencies. Last fall, ORI proposed
updates to its research misconduct regulations for the first time since 2005. The office has a small footprint
relative to the volume of NIH-funded research and does not have independent investigative authorities. A recent
editorial in Science noted that NSF’s Office of the Inspector General (OIG) tends to identify more instances
of research misconduct than ORI, despite NIH having a significantly larger extramural research portfolio.40
Given these factors, ORI’s ability to proactively identify or prevent research misconduct within the NIH-funded
research portfolio is likely limited.

Within NIH’s authorities, the agency recently issued a data sharing policy that will enable researchers to
more easily validate or refute claims.41 New technologies could also play a role in helping to quickly identify
inconsistencies in research claims. For example, the Defense Advanced Research Projects Agency (DARPA)
previously carried out the Systematizing Confidence in Open Research and Evidence (SCORE) program,
which demonstrated the feasibility of using algorithms to validate claims.42 Congress and NIH should identify
opportunities to leverage these types of technologies, coupled with data sharing policies, to restore public
confidence in research claims. Congress should also review the statutory authorities of HHS’ ORI to identify
any areas that could be strengthened, clarified, or updated to reflect current research misconduct challenges.
This review should also take into consideration the complementary role of the Department of Health and
Human Services’ (HHS) OIG.

Adhering to Grants Management Processes

In order for NIH to rebuild public trust, the public must first have confidence that NIH is appropriately
overseeing its research portfolio. Over the past year, HHS-OIG has issued three reports describing deficiencies
in NIH’s oversight of award and subaward recipient compliance with grant requirements.43 Box 3 describes

40 Ivan Oransky & Barbara Redman, Rooting out scientific misconduct, 383 SCIENCE 131 (2024), https://www.science.org/
doi/10.1126/science.adn9352.
41 Data Management & Sharing Policy Overview, NATIONAL INSTITUTES OF HEALTH SCIENTIFIC DATA SHAR-
ING, https://sharing.nih.gov/data-management-and-sharing-policy/about-data-management-and-sharing-policies/data-manage-
ment-and-sharing-policy-overview#after (last visited Apr. 29, 2024).
42 Systematizing Confidence in Open Research and Evidence (SCORE), DEFENSE ADVANCED RESEARCH PROJECTS
AGENCY, https://www.darpa.mil/program/systematizing-confidence-in-open-research-and-evidence (last visited Apr. 29, 2024).
43 Christi A. Grimm, The National Institutes of Health and Ecohealth Alliance Did Not Effectively Monitor Awards and Sub-
awards, Resulting in Missed Opportunities to Oversee Research and Other Deficiencies, U.S. DEPARTMENT OF HEALTH AND
HUMAN SERVICES OFFICE OF INSPECTOR GENERAL (Jan. 2023), https://oig.hhs.gov/oas/reports/region5/52100025.pdf; Amy
J. Frontz, The National Institutes of Health Did Not receive 81 of 109 Required Audit Reports For Foreign Grant Recipients, U.S.
DEPARTMENT OF HEALTH AND HUMAN SERVICES OFFICE OF INSPECTOR GENERAL (Dec. 2023), https://oig.hhs.gov/
documents/audit/7901/A-05-21-00019-Complete%20Report.pdf.
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OIG’s findings in detail.
Box 3. HHS-OIG Reviews of NIH Grants Management
Collectively, OIG’s findings demonstrate and Oversight
systematic deficiencies in how NIH conducts post-
award monitoring. These findings are echoed by In 2023, OIG issued three reports finding deficiencies
RFI respondents: one respondent noted that, while with NIH’s grants management processes.
many researchers do comply with award terms and Specifically, OIG found:
conditions, such as the requirement for submission
of annual progress reports, NIH program officers 1. NIH did not effectively monitor activities carried
do not typically engage with researchers in a out by EcoHealth Alliance and its subaward
material way. NIH staff do not generally ask recipient, the Wuhan Institute of Virology (WIV),
follow-up questions about the information including by ensuring the submission of progress
contained in progress reports or otherwise
reports and required laboratory records. OIG’s
demonstrate that the agency uses the reports for
review ultimately led HHS to propose the WIV for
any specific purpose. Yet, these reports and other
debarment.
compliance activities require significant time and
2. NIH did not receive 81 out of 109 required audit
effort on the part of researchers to prepare.
reports from foreign award recipients. Of the
This creates a significant oversight challenge: on 28 audit reports that were received, 10 required
the one hand, these findings demonstrate issues corrective action, but NIH only followed up
with the performance of certain funding recipients within required timeframes for three out of the 10
that need to be quickly identified and addressed. recipients.
However, clearly existing grants oversight tools, 3. NIH generally did not comply with single audit
such as annual progress reports, clearly create a review requirements, taking more than twice the
disproportionate burden for researchers who are allotted time to respond to findings that warranted
genuinely seeking to comply. corrective action. Government funding recipients
with multiple awards are required to commission
Moving forward, Congress and NIH should an annual audit (known as the “single audit”)
explore options to balance administrative burden conducted by a third party to determine financial
with truly meaningful oversight tools. For
health and test internal controls. In some cases,
example, in addition to the annual single audit (a
NIH’s delay likely jeopardized the agency’s ability
process led by the funding recipient and focused
to take legal action in response to findings.
on financial metrics), NIH could use a risk-based
framework to commission randomized audits
focused on compliance with award terms and conditions and research integrity issues. This approach could be
balanced with consideration of streamlined recurring reporting for lower risk projects.

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Conclusion

Since the enactment of 21st Century Cures, the scientific landscape has changed exponentially. Artificial
intelligence and machine learning have advanced at an unexpectedly rapid pace, and their potential applications
within biomedical research and health care are seemingly endless. The COVID-19 pandemic accelerated
the adoption of telehealth and the digitization of public health data, shifted how we develop medical
countermeasures, and proved the pace at which science can occur under the right circumstances. These trends
underscore the importance of fully realizing the goals of prior legislative efforts. While we now have more
opportunities to advance the health and wellbeing of the American people through biomedical innovation, the
risks of failure—whether by failing to harness research opportunities, the erosion of the domestic biomedical
research workforce, the proliferation of low-quality research, or poor oversight that threatens public trust
in science—are greater than ever before. I look forward to working with all interested stakeholders and my
colleagues on the HELP Committee to harness this opportunity to strengthen NIH for the next generation of
Americans.

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