# The National Institutes of Health (NIH): Background and Congressional Issues > Briefs, arguments, decisions, and more. URL: https://www.frixlaw.com/law-library/documents/crs%3AR41705 ## Record - **Collection:** Congressional research report - **Document type:** CRS Report - **Published:** January 13, 2025 - **Citation:** R41705 ## Text The National Institutes of Health (NIH): Background and Congressional Issues Updated January 13, 2025 Congressional Research Service https://crsreports.congress.gov R41705 SUMMARY The National Institutes of Health (NIH): Background and Congressional Issues The National Institutes of Health (NIH), under the Department of Health and Human Services (HHS), is the leading federal agency for biomedical and health research. In FY2024, NIH used its over $47 billion budget to support more than 300,000 scientists and research personnel working at over 2,500 institutions across the United States and abroad, as well as to conduct research and training at its own facilities. The agency consists of the Office of the Director, in charge of overall policy and program coordination, and 27 institutes and centers, each of which focuses on particular diseases, research areas, or agency support services. Over 80% of NIH-supported research is funded through a highly competitive system of peer-reviewed grants and contracts. R41705 January 13, 2025 Kavya Sekar Analyst in Health Policy For a copy of the full report, please call 7-5700 or visit www.crs.gov. The Public Health Service Act (PHSA) provides the statutory basis for NIH programs, and funding levels are provided mostly through the annual appropriations process. In December 2016, Congress introduced major reforms and programs at NIH through the 21st Century Cures Act (P.L. 114-255). Prior to 2016, the last time Congress addressed NIH with comprehensive legislation was in December 2006 through the NIH Reform Act (P.L. 109-482). Congress also gives some direction to NIH through appropriations report language and some program-specific authorizations. In recent decades, Congress has accepted, for the most part, the scientific and public health priorities established by the agency through its planning and grant-making activities that involve members of the scientific community and the general public. NIH has seen budget fluctuations throughout its history. From FY1998 to FY2003, Congress doubled the NIH budget from $13.7 billion to $27.1 billion, which rapidly increased NIH’s purchasing power and subsequently the nation’s funded research workforce and projects. The agency then saw low funding growth or cuts from FY2004 to FY2015, which resulted in increased competition for NIH funding as the agency’s purchasing power decreased. Starting in FY2016, Congress provided NIH with funding increases each year, raising the program level from about $30 billion in FY2015 to $47.7 billion in FY2023. In FY2024, NIH’s funding level slightly decreased from the prior year. NIH officials and scientific observers have cited funding variability and uncertainty as a challenge for the agency. Along with funding uncertainty, other challenges facing the agency and the broader research enterprise include • • • • whether to change NIH’s large and decentralized structure, and if so, how; • how to address geopolitical and security dimensions of NIH research, particularly interference from foreign governments and other potential biosecurity issues; • • how to balance the public and private sectors’ relative roles in biomedical research; determining NIH’s research priorities across disease types, areas of human health, and types of research; how to balance new and existing funding commitments amid budget fluctuations; how to ensure a robust research workforce pipeline, particularly to enable early-career researchers to enter the field; whether and how to formulate policies around pharmaceutical drugs developed, in part, through NIHfunded research, and, in particular, how to address issues associated with access and affordability of such drugs. This CRS report provides background and analysis on NIH’s organization, mission, budget, and history; outlines the agency’s major responsibilities; and discusses some of the issues facing Congress as it works to guide and monitor the nation’s investment in medical and health research through NIH. Congressional Research Service The National Institutes of Health (NIH): Background and Congressional Issues Contents Introduction ..................................................................................................................................... 1 Background on NIH ........................................................................................................................ 2 History ....................................................................................................................................... 2 Organizational Structure ........................................................................................................... 3 Recent Major Legislative History ............................................................................................. 4 Authority ................................................................................................................................... 6 Recent Authorization of Appropriations ............................................................................. 7 NIH Research Activities .................................................................................................................. 7 Types of Research at NIH ......................................................................................................... 8 Extramural Research ............................................................................................................... 10 Scientific Peer Review Process for Extramural Funding ................................................... 11 Grants Policy..................................................................................................................... 15 Grants Administration and Oversight ............................................................................... 16 Research Grants: By the Numbers .................................................................................... 17 Intramural Research ................................................................................................................ 18 Research Training.................................................................................................................... 19 Information Dissemination...................................................................................................... 20 Budget ........................................................................................................................................... 20 Foundation for the NIH ........................................................................................................... 23 Setting NIH Research Priorities .................................................................................................... 23 NIH Process in Setting Research Priorities ............................................................................. 24 Strategic Planning ............................................................................................................. 25 Coordinating Across NIH ................................................................................................. 25 Coordinating Across the Federal Government .................................................................. 26 Congressional Involvement in NIH Research Priorities ......................................................... 27 Appropriations .................................................................................................................. 27 Authorizations ................................................................................................................... 28 Research Restrictions ........................................................................................................ 29 Selected Recent Research Initiatives ............................................................................................. 30 Alzheimer’s Disease and Related Dementias Research .......................................................... 30 21st Century Cures Act Innovation Projects ............................................................................ 32 Coronavirus Disease 2019 (COVID-19) and Long COVID Research.................................... 35 Selected Issues for Congress ......................................................................................................... 40 Changing NIH’s Structure ....................................................................................................... 40 Determining NIH’s Research Priorities................................................................................... 42 Balancing New and Existing Funding Commitments ............................................................. 45 NIH and the Research Workforce Pipeline ............................................................................. 48 Next Generation Researchers Initiative ............................................................................ 49 Postdoctoral Workforce..................................................................................................... 50 Geopolitical and Security Dimensions of NIH Research ........................................................ 52 Balancing Federal and Industry Support of Research ............................................................. 55 NIH Funded Research and Pharmaceutical Drug Development ............................................. 59 Intramural Research .......................................................................................................... 61 Extramural Research ......................................................................................................... 63 Looking Ahead .................................................................................................................. 64 Congressional Research Service The National Institutes of Health (NIH): Background and Congressional Issues Figures Figure 1. NIH Organization ............................................................................................................. 4 Figure 2. FY2023 NIH Obligations, by Funding Mechanism ......................................................... 8 Figure 3. “Continuum” of Biomedical Research at NIH ................................................................. 9 Figure 4. NIH Scientific Peer Review Process for Extramural Funding ....................................... 14 Figure 5. Research Grants Awarded, by Fiscal Year, 2009-2023................................................... 17 Figure 6. R01-Equivalent Grant Average Cost: 2000 to 2023 ....................................................... 18 Figure 7. NIH Funding, FY1998-FY2024 ..................................................................................... 22 Figure 8. Research Project Grants (RPG) Awarded by NIH and Success Rates for New Grant Applications...................................................................................................................... 46 Figure 9. R01-Equivalent Grant Application Success Rates for First-Time and Established Investigators ............................................................................................................................... 50 Figure 10. NIH Distribution of Budget Authority, Basic and Applied Research........................... 58 Tables Table 1. NIH Alzheimer’s Disease Research Funding Directed by Congress ............................... 30 Table 2. Authorization of Appropriations for NIH Innovation Projects Under the Cures Act .............................................................................................................................................. 33 Table 3. Components of NIH, with History and Scope ................................................................. 65 Appendixes Appendix. Selected NIH Research Policies .................................................................................. 70 Contacts Author Information........................................................................................................................ 72 Congressional Research Service The National Institutes of Health (NIH): Background and Congressional Issues Introduction The National Institutes of Health (NIH) is the leading federal agency for biomedical and health research. The agency has major roles in training biomedical researchers and disseminating health information. The NIH mission is “to seek fundamental knowledge about the nature and behavior of living systems and the application of that knowledge to enhance health, lengthen life, and reduce illness and disability.”1 NIH supports two categories of research: extramural research, performed by nonfederal scientists using NIH grants or other awards, and intramural research, performed by federal NIH scientists in NIH-operated research facilities. Congress maintains a high level of interest in NIH for a variety of reasons. NIH funds research in every state, and widespread constituencies contact Congress about funding for particular diseases and levels of research support in general. NIH is the largest and most visible contributor to the federal medical and health research effort; it represents about one-fifth of total federal research and development (R&D) funding and represents close to half of all federal R&D spending outside of the Department of Defense.2 NIH has the largest budget of the nine health-related agencies that make up the Public Health Service (PHS) within the Department of Health and Human Services (HHS).3 NIH-funded research has contributed to major scientific advances. To date, 174 NIH-funded researchers have received Nobel Prizes for their work.4 NIH-funded research has led to major medical innovations, such as treatments for heart disease, cancer, and HIV/AIDS. Such advances have been credited with helping increase life expectancy and prevent millions of deaths.5 In recent years, NIH has drawn Congress’s attention for many additional reasons. First, during the Coronavirus Disease 2019 (COVID-19) pandemic, NIH played a major role in supporting research related to the novel virus; in particular, NIH helped develop new tests, vaccines, and therapeutics. In addition, NIH leaders became a public face of the federal government during the pandemic, communicating health and scientific information. At the same time, NIH faced increased scrutiny, in particular, during investigations into the origins of the virus. These investigations drew public attention to NIH’s funding of coronavirus research in China and to the agency’s challenges in monitoring laboratories in China that received subawards from other NIH grantees. These discussions also drew attention to NIH’s support of so-called “gain-of-function” research, or research that can make viruses more transmissible or pathogenic. These conversations have led to policy discussions around broader geopolitical and security issues associated with NIH research. NIH has also seen a major leadership shift recently: NIH’s longtime Director Dr. Francis Collins stepped down in 2021, after serving under three presidential administrations, and was replaced by Director Dr. Monica Bertagnolli in 2023. Additionally, a new complementary independent agency 1 National Institutes of Health (NIH), “About the National Institutes of Health,” at https://www.nih.gov/about-nih/what- we-do/mission-goals. 2 CRS analysis of federal research and development budget data provided by the Office of Management and Budget. 3 The Public Health Service also includes the Centers for Disease Control and Prevention (CDC), the Food and Drug Administration (FDA), the Agency for Healthcare Research and Quality (AHRQ), the Health Resources and Services Administration (HRSA), the Substance Abuse and Mental Health Services Administration (SAMHSA), the Indian Health Service (IHS), the Agency for Toxic Substances and Disease Registry (ATSDR), and the Administration for Strategic Preparedness and Response (ASPR). For further information, see CRS Report R48060, Department of Health and Human Services: FY2025 Budget Request. 4 NIH, “Nobel Laureates,” https://www.nih.gov/about-nih/what-we-do/nih-almanac/nobel-laureates. 5 NIH, “Report of the Director: National Institutes of Health: Fiscal Years 2014 and 2015,” pp. 9-10, https://dpcpsi.nih.gov/sites/default/files/NIH_Directors_Biennial_Report-2014-2015.pdf. Congressional Research Service 1 The National Institutes of Health (NIH): Background and Congressional Issues housed within NIH was established in 2022, the Advanced Research Projects Agency for Health (ARPA-H), which is focused on boosting health and medical innovation. In recent years, many within and outside of Congress have discussed whether NIH warrants further reform in light of the challenges the agency faces as discussed further in this report. NIH is the largest single funder of health and medical research in the world, though the agency’s funding has fluctuated over time. From FY1998 to FY2003, Congress doubled the NIH budget over a five-year period, from $13.7 billion to $27.1 billion. The agency then saw low funding growth or cuts from FY2004 to FY2015. From FY2016 through FY2023, Congress provided NIH with funding increases each year, until FY2024 when NIH saw a slight decrease in its overall budget.6 Aside from funding, other potential issues of interest for many in Congress and the research community include • • • • • • • whether to change NIH’s large and decentralized structure, and if so, how; determining NIH’s research priorities across disease types, areas of human health, and types of research; how to balance new and existing funding commitments amid budget fluctuations; how to ensure a robust research workforce pipeline, particularly how to support early-career researchers to enter the field; how to address geopolitical and security dimensions of NIH research, particularly interference from foreign governments and other potential security biosecurity issues; how to balance the public and private sectors’ relative roles in biomedical research; whether and how to formulate policies around pharmaceutical drugs developed, in part, through NIH-funded research, and, in particular, how to address issues associated with access to and affordability of such drugs. This report provides background and analysis on NIH’s history, organization, authorities, and budget; outlines the agency’s major responsibilities; and discusses some of the issues facing Congress as it works to guide and monitor the nation’s investment in medical research through NIH. Background on NIH History NIH traces its roots to 1887, when a one-room Laboratory of Hygiene was established at the Marine Hospital in Staten Island, NY. Relocated to Washington, DC, in 1891 and renamed the Hygienic Laboratory, it operated for its first half century as a research lab for the Public Health Service.7 Congress designated the research laboratory the National Institute of Health in 1930 (P.L. 71-251). NIH moved to donated land in the Maryland suburbs in 1938. By 1948, several new institutes and divisions had been created, and the agency was renamed as the National Institutes of Health (P.L. 80-655). Congress and the executive branch created new institutes and 6 CRS Report R43341, National Institutes of Health (NIH) Funding: FY1996-FY2025. 7 NIH, “History,” “Chronology of Events,” at https://www.nih.gov/about-nih/who-we-are/history. Congressional Research Service 2 The National Institutes of Health (NIH): Background and Congressional Issues centers (ICs) over the following decades, resulting in the 27 ICs that exist today (see Table 3). The most recent institute was established in 2011, the National Center for Advancing Translational Sciences (NCATS, P.L. 112-74; see Table 3). NIH now occupies a 322-acre main campus in Bethesda, MD, and several off-campus sites, including locations in Maryland, North Carolina, Montana, and Arizona.8 In addition, the Advanced Research Projects Agency for Health (ARPA-H) was established recently in 2022 as an independent agency housed within NIH to advance “high-potential, highimpact” biomedical and health research. ARPA-H was first established by an appropriation in FY2022 (P.L. 117-103) and then codified in FY2023 (P.L. 117-328). ARPA-H is modelled after other “ARPA” agencies within the federal government, in particular, the Defense Advanced Research Projects Agency (DARPA). The ARPA-H Director directly reports to the HHS Secretary; ARPA-H is not considered an NIH IC. 9 In March 2023, the HHS Secretary exempted ARPA-H from all of NIH policies, except where the ARPA-H Director identifies a need to follow NIH policy.10 Therefore, most of the policies discussed in this report do not apply to ARPA-H. For more information on ARPA-H, see CRS Report R47568, Advanced Research Projects Agency for Health (ARPAH): Overview and Selected Issues. Organizational Structure Selected NIH Resources https://www.nih.gov/ Background: https://www.nih.gov/about-nih. Budget: https://officeofbudget.od.nih.gov/index.htm. Research, condition and disease funding estimates: https://report.nih.gov/funding/categoricalspending#/ Health information: https://www.nih.gov/healthinformation. Office of the Director, Institutes and Centers: https://www.nih.gov/institutes-nih. Grants and grants policy: https://grants.nih.gov/ grants/oer.htm. Funded projects database: https://reporter.nih.gov/. Peer review: https://grants.nih.gov/grants/peerreview.htm. Chronologies (historical and legislative): https://www.nih.gov/about-nih/what-we-do/nihalmanac/timelines. Congressional Liaison: 301-496-3471, https://www.nih.gov/institutes-nih/nih-office-director/ olpa. Today, NIH is a large and complex organization. NIH consists of the Office of the Director and 27 components—20 research institutes, three research centers, the National Library of Medicine (NLM), and three other support centers (see Figure 1). As detailed in Table 3, NIH’s ICs were established over time through separate laws or administrative actions, starting with the National Cancer Institute, established in 1937. The Office of the Director (OD) sets overall policy for NIH and coordinates the programs and activities of all NIH components, particularly transinstitute research initiatives and issues. The individual ICs focus on particular diseases (e.g., the National Cancer Institute), body systems (e.g., National Heart, Lung, and Blood Institute), life stages (e.g., the National Institute on Aging), and scientific fields (e.g., the National Institute of Biomedical Imaging and Bioengineering). Each IC plans and manages its own research programs in coordination with OD. Congress provides separate appropriations to 24 of the 27 ICs. This includes all 20 institutes, 8 NIH Intramural Research Program, “Research Campus Locations,” at https://irp.nih.gov/about-us/research-campus- locations. 9 CRS Report R47568, Advanced Research Projects Agency for Health (ARPA-H): Overview and Selected Issues. 10 Pursuant to statutory authority provided in Public Health Service Act Section 499A(a)(3). See Advanced Research Projects Agency for Health, Department of Health and Human Services, “Exemption of the Advanced Research Projects Agency for Health (ARPA-H) From Policies and Requirements of the National Institutes of Health (NIH),” 88 Federal Register 19157, March 30, 2023. Congressional Research Service 3 The National Institutes of Health (NIH): Background and Congressional Issues NLM, the three research centers, to OD, and to a buildings and facilities account (see the “Budget” section). The three research centers include the John E. Fogarty International Center for Advancing Study in the Health Sciences, the National Center for Advancing Translational Sciences, and the National Center for Complementary and Integrative Health. The three support centers are funded through transfers from other NIH components: the Clinical Center, the Center for Information Technology, and the Center for Scientific Review.11 The institutes, NLM, the three research centers, and OD have the authority to award research grants; the three operational support centers do not award research grants.12 Figure 1. NIH Organization Office of the Director and 27 Institutes and Centers Source: Adapted based on information from NIH, “Organization,” https://www.nih.gov/about-nih/who-we-are/ organization and Public Health Service Act Title IV. Notes: Does not include the Advanced Research Projects Agency for Health (ARPA-H), an independent agency housed within NIH. The ARPA-H Director reports to the HHS Secretary. Recent Major Legislative History Since the 1980s, Congress has passed comprehensive NIH laws about once a decade. This is in addition to many laws that have addressed specific NIH programs (discussed further in the “Authorizations” section). Major laws have included the following: The Health Research Extension Act of 1985 (P.L. 99-158) created the current structure of Public Health Service Act (PHSA) Title IV, NIH’s main authorizing title (see the “Authority” 11 The three support centers are financed by the NIH Management Fund, through collections from other NIH ICs for services provided by the support centers. See NIH, “FY2020 Congressional Budget Justification, Overview Vol. I,” p. 94, https://officeofbudget.od.nih.gov/pdfs/FY20/br/Overview-Volume-FY-2020-CJ.pdf. 12 Authorities of the ICs are detailed in Title IV of the Public Health Service Act (PHSA). See the “Authority” section. Congressional Research Service 4 The National Institutes of Health (NIH): Background and Congressional Issues section), providing explicit statutory authority for all of NIH and its institutes, including the authority and duties of the NIH Director and the institute directors.13 Specifically, the law authorized 14 research institutes and centers, the National Library of Medicine, and the Division of Research Resources. Some of these institutes and components were newly authorized in this law. The law also included authority for the HHS Secretary to establish new or abolish existing NIH institutes with advance notice to Congress. The law also included new statutory requirements for the ethical treatment of both humans and animals in research. The NIH Revitalization Act of 1993 (P.L. 103-43) extended NIH authorizations of appropriations for three years and included many amendments throughout PHSA Title IV. A major provision was the establishment of requirements to include women and minorities in clinical research as appropriate for the scientific question under study (see the “Inclusion Policies” section). The law also codified several offices at NIH, such as the Office of Women’s Health, the Office of Research on Minority Health, and the Office of Behavioral and Social Sciences Research. The law included several provisions focused on research integrity, and it codified an Office of Research Integrity, along with new protections for whistleblowers and new requirements to protect against financial conflicts of interest among researchers. In addition, the law included many provisions focused on specific diseases, particularly many HIV/AIDS provisions to enhance NIH-wide coordination on related research. The NIH Reform Act of 2006 (P.L. 109-482) included major revisions to NIH’s authorizations. Specifically, it sought to limit the creation of new NIH institutes and centers, as well as to enhance the NIH Director’s ability to coordinate across the agency. The law followed a congressionally requested report published in 2003 by the Institute of Medicine and National Research Council (IOM/NRC) that had examined NIH’s structure and need for organization reform.14 The report noted some challenges with NIH’s large and decentralized organizational structure, made up of 27 Institutes and Centers (ICs), but stated that any proposals for changing the number of ICs or OD program offices should be subject to a public evaluation process.15 Many of the recommendations in the 2003 IOM/NRC report were incorporated into the NIH Reform Act of 2006 (P.L. 109-482). Among many other reforms, the act created the Scientific Management Review Board (SMRB) to provide advice on NIH’s organization and management. SMRB is charged with formally and publicly reviewing NIH organizational structure at least once every seven years. The law also required a biennial report to Congress on NIH activities and strategic planning, as well as the creation of a comprehensive database on NIH research.16 The Reform Act also consolidated many authorizations of appropriations for specific NIH programs to a single authorization of appropriations for the entire agency, extended from FY2007 to FY2009. 13 Prior to enactment, most of NIH’s Institutes and Centers were explicitly authorized in law, whereas others were not. See U.S. Congress, House Energy and Commerce Committee, Health Research Extension Act of 1985, 99th Cong., 1st sess., June 4, 1985, 99-158, pp. 19-20. 14 Institute of Medicine and National Research Council, Enhancing the Vitality of the National Institutes of Health: Organizational Change to Meet New Challenges, 2003, https://nap.nationalacademies.org/catalog/10779/enhancingthe-vitality-of-the-national-institutes-of-health-organizational. The IOM is now known as the National Academy of Medicine. 15 Ibid., p. 7. The IOM/NRC report recommended more rigorous and frequent review of the performance of top NIH and IC leaders, including the possibility of term limits; reassessment by Congress of the National Cancer Institute’s special status regarding appointments and budget authority; and reform of the advisory council system so that it is more independent and protected from political influences. 16 Now known as the Research Portfolio Online Reporting Tools (RePORT) system. See https://report.nih.gov/. All other duplicative reporting requirements were eliminated. The law added new reporting requirements on clinical trials, human tissue storing and tracking, whistleblower complaints, and special consultant hires (all had been the subject of investigations by the House Energy and Commerce Committee, the committee of jurisdiction for NIH). Congressional Research Service 5 The National Institutes of Health (NIH): Background and Congressional Issues The 21st Century Cures Act (P.L. 114-255), enacted in 2016, reauthorized appropriations for NIH until FY2020 and introduced several administrative reforms at NIH. The act required the NIH Director to develop and make publicly available an NIH-wide Strategic Plan every six years (see the “Strategic Planning” section). The act also introduced accountability measures, such as five-year terms for IC Directors. Other reforms included efforts to reduce administrative burden at NIH, such as by exempting NIH research from requirements of the Paperwork Reduction Act, and efforts to prevent and eliminate duplicative research across the agency. The act also authorized several programs and research efforts at NIH, in particular, by creating a new NIH Innovation Account for funding four major research initiatives (detailed further in the “21st Century Cures Act Innovation Projects” section). Authority NIH derives most of its statutory authority for its programs from the Public Health Service Act (PHSA) of 1944, as amended (42 U.S.C. §§201-300mm-61): PHSA Section 301 (42 U.S.C. §241) grants the HHS Secretary broad and permanent authority to conduct and sponsor research. PHSA Title IV, “National Research Institutes,” (42 U.S.C. Chapter 6A, Subchapter III) is the main authorizing title for NIH. It defines the agency’s overall structure, the responsibilities of the NIH Director and the IC Directors, overall policy requirements, and the research areas of each IC. Key governing provisions include the following: • • • • • PHSA Section 401 (42 U.S.C. §281) establishes NIH as an agency within the Public Health Service that consists of 27 ICs and the Office of the Director, and caps the number of ICs at 27. This section also provides authority for NIH reorganization to the HHS Secretary, as advised by the Scientific Management Review Board (see the “Recent Major Legislative History” section). PHSA Section 402 (42 U.S.C. §282) establishes the position of the NIH Director and outlines its responsibilities. PHSA Section 405 (42 U.S.C. §284) establishes the positions of IC Directors and specifies their responsibilities, including to oversee funded research and to make final decisions for new research grant awards. PHSA Section 406 (42 U.S.C. §284a) establishes advisory councils and boards for each of the ICs to oversee their programs. PHSA Title IV, Part C-Part E (42 U.S.C. Chapter 6A, Subchapter III, Part CPart E) outlines the specific statutory authorizations for each of the research ICs. All of the research ICs are covered by specific provisions in these sections, but the provisions vary considerably in the amount of detail included in the statutory language. Some NIH programs are authorized elsewhere in the PHSA or in other laws. For example, PHSA Title XXIII authorizes the NIH Office of AIDS Research (PHSA Section 2351)17 and other HIV/AIDS research programs and authorities.18 As another example, the 21st Century Cures Act (P.L. 114-255) authorizes NIH Innovation projects (see the “21st Century Cures Act Innovation Projects” section). 17 42 U.S.C. §300cc-40. 18 42 U.S.C. §§300cc-1 et seq. Congressional Research Service 6 The National Institutes of Health (NIH): Background and Congressional Issues Recent Authorization of Appropriations In 2016, the 21st Century Cures Act (P.L. 114-255) amended the PHSA (§ 402A), authorizing overall appropriations for NIH in FY2018 ($34,851,000,000), FY2019 ($35,585,871,000), and FY2020 ($36,472,442,775) to carry out activities authorized in Title IV of the PHSA. The current authorization of appropriations for NIH expired at the end of FY2020. The overall authority for NIH, or explicit authorizations of individual ICs, has lapsed at times, including currently. However, NIH has continued to receive annual appropriations even when its authorization of appropriations has lapsed. In general, when Congress appropriates funds for a program whose funding authorization has expired, that appropriation provides sufficient legal basis to continue the program during that period of availability absent indication of congressional intent to terminate the program.19 NIH Research Activities NIH research spans all fields of medical, health, and behavioral research, from basic investigation of biological mechanisms to testing new therapeutics in clinical research. The ICs sponsor two categories of research: extramural research, performed by nonfederal scientists using NIH grants or other awards, and intramural research, performed by federal scientists in the NIH-operated research facilities and the Clinical Center. NIH also supports a range of extramural and intramural research training programs, especially to prepare early-career investigators for research careers, and it engages in a number of information dissemination activities to reach various audiences. Funding for research makes up most of NIH spending. Figure 2 shows the breakdown of NIH obligations by funding mechanism. Displaying budget data by mechanism reveals the balance between extramural (e.g., research grants, research centers, and R&D contracts) and intramural funding, as well as the relative emphasis on support of individual investigator-led research (e.g., research grants and intramural research) versus funding of contracted projects (e.g., R&D contracts). 19 CRS Report R46497, Authorizations and the Appropriations Process. Congressional Research Service 7 The National Institutes of Health (NIH): Background and Congressional Issues Figure 2. FY2023 NIH Obligations, by Funding Mechanism Dollars in Millions Source: Developed by CRS using budget data from NIH, “FY2025 Justification of Estimates for Appropriations Committees, Vol I: Overview–History of Obligations by Total Mechanism,” p. 87. Amounts shown do not account for the Public Health Service Evaluation Set-Aside funding or ARPA-H. Notes: “Total main extramural” category includes the main NIH mechanisms of research support, including research project grants, contracts, research center grants, training grants, and other mechanisms of support. Some extramural programs are integrated into the “Other” category, particularly for the “Office of the Director, Superfund, and Other” category. Figure is based on NIH’s categorization system used in source linked above. Types of Research at NIH According to NIH, the agency conducts and supports the “full continuum” of biomedical, health, and behavioral research to understand the causes and mechanisms of disease, and then translates Congressional Research Service 8 The National Institutes of Health (NIH): Background and Congressional Issues that knowledge into clinical practice and health interventions. NIH defines the continuum of research as follows (see Figure 3):20 • • • • • Basic research involves studying the fundamental mechanisms of biology and behavior. Preclinical translational research involves developing and testing new diagnostics, therapeutics, and preventive measures. This research is conducted using laboratory animals, cell cultures, samples of human or animal tissues, computer modeling, or other approaches. Clinical research is conducted with human subjects. Clinical research can include (1) clinical trials of diagnostics, therapeutics, and preventive measures, as well as any basic or other research conducted with patients; (2) epidemiological and behavioral studies; and (3) outcomes research and health services research. Postclinical translational research investigates the best methods to enhance access to and the implementation of newly discovered biomedical interventions. Clinical and community practice involves translating new biomedical research discoveries into widespread clinical and community practice. It includes NIH’s effort to ensure that scientific findings are communicated rapidly and clearly to the public. Figure 3. “Continuum” of Biomedical Research at NIH Source: NIH, “Report of the Director of the National Institutes of Health: Fiscal Years 2012 & 2013,” at https://report.nih.gov/biennialreport1213/NIH_OD_Biennial_report_2012-2013_508complete.pdf, p. 25. The same figure appears in NIH’s most recent triennial report. See page 55 of NIH, Report of the Director National Institutes of Health: Fiscal Years 2019-2021, https://dpcpsi.nih.gov/sites/default/files/2023-09/FY1921%20Triennial_Report_FINAL_508C.pdf. In addition, NIH has identified population-based, epidemiological research as one of the key drivers behind the research continuum. This type of research provides statistical evidence of the association between disease and human biology, behavior, and environmental circumstances. In addition, NIH’s investment in research tools and resources helps drive the continuum.21 20 All definitions based on NIH, Report of the Director of National Institutes of Health: Fiscal Years 2019- 2021, pp. 55-57, at https://dpcpsi.nih.gov/sites/default/files/2023-09/FY19-21%20Triennial_Report_FINAL_508C.pdf. 21 Ibid. Congressional Research Service 9 The National Institutes of Health (NIH): Background and Congressional Issues NIH reports that about half of its funding is for basic research.22 NIH emphasizes that the research continuum is not always linear. Progress in research may involve moving back and forth between different stages. For instance, a failed clinical trial on a therapeutic for a given disease may lead to new questions that then require more basic research to make progress in treating that disease, rather than advancing directly into postclinical translational research or other stages.23 Extramural Research NIH extramural research funding makes up nearly 83% of the overall NIH budget and supports 300,000 scientists and research personnel affiliated with over 2,500 universities, medical schools, and other research institutions in every state and around the world.24 Extramural awards include multiple types of research and training grants (see the text box below), cooperative agreements, and contracts, Within the large “research grants” category, the bulk of the funding goes to research project grants (RPGs) awarded to individual investigators and small teams, most of whom are based at universities and medical centers. Other types of grants are provided to groups of researchers who work in collaborative programs or in multidisciplinary centers that focus on particular diseases or areas of research, often called “centers of excellence.” Data on awards and recipients by state, congressional district, type of institution, and subject of the research are available on the NIH website.25 Types of Extramural NIH Grants NIH awards many types of grants and uses activity codes to differentiate its programs. Not all NIH ICs fund all types of grant programs. Common types of grants and their activity codes include the following: Research Grants: NIH’s research grants fall into its R-series. NIH’s most commonly used grant program, the standard independent Research Project Grants (RPG, R01) are used to support a discrete, specified research project carried out by an independent Principal Investigator. This grant mechanism is used by all ICs, and grants are generally awarded for three to five years. NIH can also award RPGs for smaller, more exploratory grants (e.g., R03, R15, R21) and award grants to small businesses under the federal governmentwide Small Business Technology Transfer (STTR)/Small Business Innovative Research (SBIR) program (e.g., R41 R43, and R44). (For more information on STTR/SBIR, see CRS Report R43695, Small Business Research Programs: SBIR and STTR.) Career and Training Grants: Several NIH grant programs support training and career development, including its Research Career Development Awards (K-series), Institutional Training Grants (T-series), and Individual Fellowships program (F-series). (For more information, see the “Research Training” section.) Program Project/Center Grants: The P-series consists of grants for large, multiproject efforts, including grants for research centers (e.g., P30, P50) that support shared resources and facilities or projects by a number of different investigators within the same research category (e.g., National Cancer Institute Cancer Centers). Resource Grants: NIH also awards research resource grants that fund research-related support or access to resources, rather than a specific research project, which includes support for research resources or infrastructure (e.g., R24) or for research education projects (e.g., R25). Sources: NIH, “Types of Grant Programs,” https://grants.nih.gov/grants/funding/funding_program.htm, and NIH Research Training and Career Development, “Programs,” https://researchtraining.nih.gov/programs. 22 NIH, “FY 2003–FY 2023 Distribution of Budget Authority: Percentage for Basic and Applied Research,” https://officeofbudget.od.nih.gov/pdfs/FY25/spending_hist/Basic%20and%20Applied%20FY%202003%20%20FY%202023%20(V).pdf. 23 NIH, Report of the Director of National Institutes of Health: Fiscal Years 2019- 2021, pp. 57, at https://dpcpsi.nih.gov/sites/default/files/2023-09/FY19-21%20Triennial_Report_FINAL_508C.pdf. 24 NIH, “What We Do—Budget,” https://www.nih.gov/about-nih/what-we-do/budget. 25 See the NIH Awards by Location & Organization, at https://report.nih.gov/award/index.cfm. See also the “Selected NIH Resources” text box above for other resources on awards. Congressional Research Service 10 The National Institutes of Health (NIH): Background and Congressional Issues Scientific Peer Review Process for Extramural Funding Scientists who seek to compete for NIH extramural research funding, whether for new proposals or for the renewal of previous awards, submit detailed plans in their funding applications describing the research they plan to undertake. In 2023, NIH received 70,746 applications for competing (new) research and training grants, and awarded a total of 16,670 grants.26 All NIH grant, cooperative agreement, and R&D contract concept applications (referred to as “grants” throughout this report for simplicity and as consistent with NIH sources) undergo review through a two-tiered system of peer review, which includes a competitive and committee-based process to evaluate the applications.27 The peer review system is pursuant to statute, especially Section 492 of PHSA (42 U.S.C. §289a),28 and federal regulations (42 C.F.R. Part 52h). The first stage of peer review assesses the application on scientific and technical merit. In the second stage, the NIH IC makes a funding decision, weighing the project’s scientific merit against the IC’s research priorities and funding availability (see Figure 4).29 According to one IC, it typically takes between 8 to 20 months after the due date for an investigator to receive an award (known as the “Notice of Award”).30 Grant solicitations and receipt: Researchers can submit applications in response to NIH Notices of Funding Opportunity (NOFO); applications are either investigator-initiated or in response to a specific notice for targeted research.31 Most applications are investigator-initiated, meaning that a scientist or group of scientists generates an original research project idea and then submits a grant application through an NIH-wide submission process.32 Some applications are submitted in response to solicitations by ICs for research areas the ICs seek to target or for which they have set aside funding.33 NIH’s Center for Scientific Review (CSR) receives most applications. CSR assigns each application that meets basic eligibility requirements to a Scientific Review Group for the first stage of review and then to a potential awarding IC for the second stage.34 The potential awarding 26 NIH Data Book, “Research and Training Grants” https://report.nih.gov/nihdatabook/category/24. 27 42 C.F.R. §52h.1. 28 Other statutes also govern aspects of NIH peer review requirements, such as PHSA Sections 402(b)(16) and PHSA Sections 402(b)(25), 405(b)(1)(B), 405(b)(2), and 406(a)(3)(A). 29 NIH, NIH Peer Review: Grants and Cooperative Agreements, https://grants.nih.gov/grants/ peerreview22713webv2.pdf. 30 NIH National Institute of Allergy and Infectious Diseases, “Timeline for Funding Decisions,” September 30, 2024, https://www.niaid.nih.gov/grants-contracts/timelines-funding-decisions. 31 NIH, “Understanding Funding Opportunities,” https://grants.nih.gov/grants/how-to-apply-application-guide/prepareto-apply-and-register/understand-funding-opportunities.htm. 32 Called “Parent Announcements”; see NIH, “Parent Announcements (For Unsolicited or Investigator-Initiated Applications),” https://grants.nih.gov/grants/guide/parent_announcements.php. 33 These include Program Announcements and Requests for Applications. Program Announcements are issued by one or more ICs to highlight areas of scientific interest, and Requests for Applications are funding opportunities issued by one or more ICs to highlight well-defined areas of scientific interest to accomplish specific program objectives. See NIH, “Understanding Funding Opportunities,” https://grants.nih.gov/grants/how-to-apply-application-guide/prepare-toapply-and-register/understand-funding-opportunities.htm. 34 NIH, NIH Peer Review: Grants and Cooperative Agreements, https://grants.nih.gov/grants/ peerreview22713webv2.pdf. Congressional Research Service 11 The National Institutes of Health (NIH): Background and Congressional Issues IC is the one whose mission best aligns with the objectives of the research proposal.35 In some cases, ICs directly receive and provide initial review of applications.36 First stage: In the first stage of peer review, the applications are assigned for review by a Scientific Review Group (SRG), which is a peer-review committee composed of roughly 12 to 22 scientists who are experts in the relevant fields of research.3738 NIH convenes many SRGs to review applications. As of 2023, there were over 250 chartered or recurring study sections (or SRGs) in addition to temporary SRGs. About 19,000 distinct peer reviewers participated in approximately 1,200 peer review meetings each year.39 Per statute, no more than one-fourth of the members of any SRG may be federal employees.40 Peer reviewers are expected to disclose conflicts of interest and may not participate in evaluations of grant applications where they have conflicts of interest.41 The SRG is responsible for evaluating a grant proposal on the basis of scientific merit and potential impact of the research.42 (See the “Changes to Peer Review Criteria for Research Grants” text box below for new developments). SRGs also review applications for certain NIH policy requirements, such as plans for protecting animals and humans involved in research (see the Appendix).43 After discussing the application, each member gives the application a final score for scientific and technical merit, and an overall impact score is determined from the average of members’ final scores.44 The application may also be given a percentile ranking based on how the overall impact score compares with other applications reviewed by the SRG in the preceding two review rounds.45 Second stage: In the second stage, the funding decisions are refined by the National Advisory Councils or Boards of the potential awarding ICs, which are advisory committees that oversee each IC’s research priorities and portfolios.46 Advisory Councils and Boards are composed of scientific and lay representatives.47 No federal employees may serve as regular voting members.48 35 NIH, Report of the Director of the National Institutes of Health: Fiscal Years 2019-2021, p. 14, https://dpcpsi.nih.gov/sites/default/files/2023-09/FY19-21%20Triennial_Report_FINAL_508C.pdf. 36 NIH, 2.4.1 Initial Review, NIH Grants Policy Statement, April 2024, https://grants.nih.gov/grants/policy/nihgps/ HTML5/section_2/2.4.1_initial_review.htm. 37 NIH, NIH Peer Review: Grants and Cooperative Agreements, https://grants.nih.gov/grants/ peerreview22713webv2.pdf; NIH, NIH Peer Review: Grants and Cooperative Agreements, https://grants.nih.gov/ grants/peerreview22713webv2.pdf; and Jeffrey Mervis, “Peering into peer review,” Science, vol. 343 (February 7, 2014), pp. 596-598. 38 NIH Office of Extramural Research, “Managing Conflict of Interest in NIH Peer Review of Grants and Contracts,” https://grants.nih.gov/grants/peer/peer_coi.htm. 39 NIH Center for Scientific Review, “CSR Data and Evaluations: CSR Overview,” https://public.csr.nih.gov/ AboutCSR/Evaluations#overview. 40 PHSA Section 402(b)(29); 42 U.S.C. §282(b)(29). 41 42 C.F.R. §52h.5. 42 Review criteria are outlined broadly in regulations at 42 C.F.R. §§52h.8 and 11 and then further specified in each notice of funding opportunity announcement for the specific award. See NIH, NIH Peer Review: Grants and Cooperative Agreements, https://grants.nih.gov/grants/peerreview22713webv2.pdf. 43 42 C.F.R. §§52h.8 and 11 and NIH, NIH Peer Review: Grants and Cooperative Agreements, https://grants.nih.gov/ grants/peerreview22713webv2.pdf. 44 NIH, “Peer Review-Scoring,” https://grants.nih.gov/grants/peer-review.htm#scoring2. 45 NIH, “Funding Decisions,” https://grants.nih.gov/grants-process/award/funding-decisions. 46 Authorized in PHSA Section 406, 42 U.S.C. §284a. 47 PHSA Section 406(b)(3), 42 U.S.C. §284a(b)(3). 48 CRS Communication with NIH Office of Federal Advisory Committee Policy, June 30, 2022. Congressional Research Service 12 The National Institutes of Health (NIH): Background and Congressional Issues These groups examine summary statements of applications recommended for funding, place their impact scores and percentile rankings in the context of the IC’s research priorities, and then make recommendations for final funding decisions.49 Many ICs establish a “payline,” or percentile cutoff for applications that get funded, though ICs may prioritize applications outside of the payline based on other considerations.50 The IC director then makes final funding decisions.51 The 21st Century Cures Act of 2016 (P.L. 114-255) added a requirement that the IC Director weigh the Advisory Council or Board’s advice against the IC’s mission and research priorities, the NIHWide Strategic Plan, and programs or projects funded by other ICs on similar topics before awarding a research grant.52 49 NIH, 2.4.3 National Advisory Council or Board Review, NIH Grants Policy Statement, April 2024, https://grants.nih.gov/grants/policy/nihgps/HTML5/section_2/2.4.1_initial_review.htm. 50 NIH, Report of the Director of the National Institutes of Health: Fiscal Years 2019-2021, pp. 15-16, https://dpcpsi.nih.gov/sites/default/files/2023-09/FY19-21%20Triennial_Report_FINAL_508C.pdf. 51 PHSA §405(b)(3). 52 See Section 2033 of the act. Congressional Research Service 13 The National Institutes of Health (NIH): Background and Congressional Issues Figure 4. NIH Scientific Peer Review Process for Extramural Funding Sources: Developed by CRS based on 42 C.F.R. Part 52h, NIH, Public Health Service Act Section 405; NIH, 2.4. The Peer Review Process, NIH Grants Policy Statement, April 2024, https://grants.nih.gov/grants/policy/nihgps/ HTML5/section_2/2.4_the_peer_review_process.htm. NIH Peer Review: Grants and Cooperative Agreements, https://grants.nih.gov/grants/peerreview22713webv2.pdf; and NIH, “Grants and Funding: Peer Review,” https://grants.nih.gov/grants/peer-review.htm. Congressional Research Service 14 The National Institutes of Health (NIH): Background and Congressional Issues Changes to Peer Review Criteria for Research Grants In October 2023, NIH announced simplified peer review criteria for the scientific and technical review of research proposals, to be in effect for grant receipt deadlines as of January 25, 2025, and beyond. These new criteria were developed in response to concerns about the complexity of peer review and the burden on reviewers. NIH also sought to mitigate reputational bias in review. The agency has faced long-standing concerns that its review process favors experienced—and therefore often older—researchers at the expense of enabling new researchers to enter the field (see the “NIH and the Research Workforce Pipeline” section for further discussion). In addition, NIH working groups that helped develop the criteria stated that “persistent racial disparities in NIH funding raise the question of whether review criteria in any way perpetuate an unfair advantage or disadvantage.” The new criteria are designed to reduce NIH’s five-factor rating framework to a three-factor framework. Prior to these changes, the five factors were: (1) Significance, (2) Innovation, (3) Approach, (4) Investigator, and (5) Environment, derived from factors listed in regulations governing the peer review process (42 C.F.R. §52h.8). The new criteria group these five into three factors: (1) Importance of the Research (Significance, Innovation); (2) Rigor and Feasibility (Approach); and (3) Expertise and Resources (Investigators, Environment). The first and second new factors are to receive scores from reviewers, while the third (Expertise and Resources) is to be considered, but not scored, in an effort to reduce reputational bias. The new criteria are the result of a multiyear process to solicit feedback on the peer review criteria, develop proposed changes, and then solicit feedback on the proposal. According to an NIH summary of public comments, the majority of respondents favored the changes. Some disagreed with certain aspects of the changes. For example, some disagreed with making “Expertise and Resources,” an unscored factor because they thought such criteria are critical to the work. Others argued that the changes did not go far enough to address bias in peer review. Many stressed the need for reviewer training to make the new criteria effective. Sources: NIH, “Simplified Peer Review Framework,” https://grants.nih.gov/policy/peer/simplifying-review/ framework.htm; “Developing the Simplified Framework” in NIH, “Background—NIH Peer Review Process,” https://grants.nih.gov/policy/peer/simplifying-review/background.htm; NIH Center for Scientific Review, “Announcing a Simplified Review Framework for NIH Research Project Grant Applications,” October 19, 2023, https://www.csr.nih.gov/reviewmatters/2023/10/19/announcing-a-simplified-review-framework-for-nih-researchproject-grant-applications/; Center for Scientific Review Advisory Council Simplifying Review Criteria Working Groups, “Recommendations for Simplifying R01 Review Criteria,” April 27, 2021, https://public.csr.nih.gov/sites/ default/files/2021-04/Recommendations_of_the_CSRAC_Working_Group_on_Simplifying_Review-nonCT_and_CT.pdf; and NIH, “Simplifying Review Framework: Feedback from the Request for Information,” April 28, 2023, https://grants.nih.gov/sites/default/files/ NIH%20SRF%20RFI%20Content%20Analyses%20April%202023%20508c.pdf. Grants Policy NIH grantees must comply with NIH policies governing their award. NIH grant requirements are based in laws, regulations, and NIH-developed policies. As summarized in the “Authority” section above, NIH’s statutory authorizations, especially in PHSA Title IV, form the statutory basis for NIH grant requirements along with other HHS and federal-wide requirements elsewhere in law. Key regulations underpinning NIH grants include general HHS award regulations (45 C.F.R. Part 75)53 and NIH-specific regulations (42 Parts 50-52i and 59a, with variation by grant type). NIH maintains an annually updated “Grants Policy Statement” on the standard terms and conditions of NIH grant awards.54 Grantees are also informed of specific award requirements in their “Notice of Award.” For the most part, extramural researchers must comply with the same 53 These regulations reflect HHS’s adoption of uniform federal award regulations promulgated by the Office of Management and Budget in 2 C.F.R. Part 200. (Uniform Administrative Requirements, Cost Principles, and Audit Requirements for Federal Awards). HHS follows the federal government-wide requirements with a few variations specific to the department. 54 NIH Office of Extramural Research, “NIH Grants Policy Statement,” https://grants.nih.gov/policy/nihgps/index.htm. Congressional Research Service 15 The National Institutes of Health (NIH): Background and Congressional Issues research policy requirements as intramural researchers. Some of these policy requirements are summarized in the Appendix. Selected NIH Grants Terminology Authorized Organization Representative (AOR): The individual, named by the applicant organization, who is authorized to act for the applicant and to assume the obligations imposed by the federal laws, regulations, requirements, and conditions that apply to grant applications or grant awards. Grants Management Officer (GMO): An NIH official responsible for the business management aspects of grants and cooperative agreements, including review, negotiation, award, and administration, and for the interpretation of grants administration policies and provisions. GMOs are delegated the authority from the Chief Grants Management Officer to obligate NIH to the expenditure of funds and permit changes to approved projects on behalf of NIH. Each NIH IC that awards grants has one or more GMOs with responsibility for particular programs or awards. Program Director/Principal Investigator (PD/PI): The individual(s) designated by the applicant organization to have the appropriate level of authority and responsibility to direct the project or program to be supported by the award. The applicant organization may designate multiple individuals as program directors/principal investigators (PD/PIs), who share the authority and responsibility for leading and directing the project, intellectually and logistically. When multiple PD/PIs are named, each is responsible and accountable to the applicant organization or, as appropriate, to a collaborating organization for the proper conduct of the project or program, including the submission of all required reports. The presence of more than one PD/PI on an application or award diminishes neither the responsibility nor the accountability of any individual PD/PI. Program Official/Program Officer/Project Officer (PO): The NIH official responsible for the programmatic, scientific, and technical aspects of a grant or cooperative agreement. Source: NIH, “Grants-Glossary,” https://grants.nih.gov/grants/glossary.htm. Grants Administration and Oversight While NIH-funded research projects are typically led by Program Directors/Principal Investigators (PD/PIs; see the text box above), grant awards are generally made to the institutions that employ those researchers. At funded institutions, an Authorized Organization Representative (AOR) is responsible for the administrative aspects of NIH grants. AORs are responsible for signing grant applications submitted by PD/PIs and, in doing so, ensure that the institution will comply with all applicable federal requirements. According to NIH, AORs, PD/PIs, and other research administration staff share overall responsibility for the successful implementation of an NIH grant.55 If an NIH awardee collaborates with another research institution on the grant, the awardee is responsible for overseeing their collaborators (with prior approval required from NIH in some cases), regardless of whether the NIH awardee provides any funding or support to the collaborator.56 The NIH Division of Grants Compliance and Oversight provides training and resources to grantees and institutions to ensure compliance.57 NIH monitors its awardees through reporting requirements, such as financial reporting and research progress reports.58 Every funded NIH grant has an assigned Grants Management Officer (GMO) responsible for overseeing business and nonprogrammatic (e.g., financial) aspects of the grant, as well as a Program Official (PO) responsible for overseeing programmatic, scientific, and 55 NIH, 2.1.2 Roles and Responsibilities-Recipient Staff, NIH Grants Policy Statement, April 2024, https://grants.nih.gov/grants/policy/nihgps/HTML5/section_2/2.1.2_recipient_staff.htm. 56 See NIH, 2.15.1 Consortium Agreements: General, NIH Grants Policy Statement, April 2024, https://grants.nih.gov/ grants/policy/nihgps/HTML5/section_15/15.1_general.htm. 57 NIH Grants & Funding, “Grants Compliance & Oversight,” https://grants.nih.gov/policy/compliance.htm. 58 NIH Grants & Funding, “Post-Award Monitoring and Reporting,” https://grants.nih.gov/grants/post-awardmonitoring-and-reporting.htm. Congressional Research Service 16 The National Institutes of Health (NIH): Background and Congressional Issues technical aspects of the grant (see the text box above).59 NIH may also conduct site visits to ensure compliance as needed.60 NIH and funded research institutions share responsibility for ensuring that NIH-funded research complies with federal requirements.61 Research Grants: By the Numbers In FY2023, NIH funded over 52,000 research grants (including RPGs, center grants, and other research awards). Figure 5 illustrates funding trends, showing the percentage of research grants each year for R01-equivalent awards. Figure 5. Research Grants Awarded, by Fiscal Year, 2009-2023 Includes number and percentage of R01-equivalent grants of total research grants awarded Source: CRS analysis of data from NIH Data Book, including “Research Grants: Awards, by Institute/Center,” https://report.nih.gov/nihdatabook/report/205, and “R01-Equivalent Grants: Awards as a Percentage of All Research Grants,” https://report.nih.gov/nihdatabook/report/32. Notes: Research grants are defined as extramural awards made for Research Centers, Research Projects, Small Business Innovation Research/Small Business Technology Transfer (SBIR/STTR) Grants, and Other Research Grants. Research Grants are defined by the following NIH activity codes: R, P, M, S, K, U (excluding UC6), DP1, DP2, DP3, DP4, DP5, D42, and G12. R01-equivalent grants are defined as grants awarded under NIH activity codes DP1, DP2, DP5, R01, R37, R56, RF1, RL1, U01, and R35 from select NIGMS and NHGRI program announcements (PAs). 59 NIH, 2.1.1 Roles and Responsibilities-NIH and HHS Staff, NIH Grants Policy Statement, April 2024, https://grants.nih.gov/grants/policy/nihgps/html5/section_2/2.1.1_nih_and_hhs_staff.htm. 60 NIH Grants & Funding, “Grants Compliance & Oversight,” https://grants.nih.gov/policy/compliance.htm. 61 Ibid. Congressional Research Service 17 The National Institutes of Health (NIH): Background and Congressional Issues As shown in Figure 5, the total number of grants rose slightly following the end of the NIH budget doubling period (FY2003) and then began to fall when NIH saw decreases in its purchasing power by FY2009 (see the “Budget” section). With funding increases, the total number of grants awarded began to increase in FY2016 and eventually exceeded the prior peak level in FY2004 (47,464 grants) by FY2019 (49,092). The majority of grants awarded are R01equivalent independent research project grants, ranging from 56% to 64% of all grants awarded in any year. In FY2022, the average cost of an R01-equivalent grant was $585,307.62 Average costs vary by type of grant. For example, the average cost of research center grants in FY2022 was over $2.4 million.63 As shown in Figure 6, while the average cost of an R01-equivalent grant rose in current dollars from FY2000 to FY2023, the average cost rose slightly when adjusting for inflation ($288 thousand in 2022 compared with $247 thousand in FY1998). Figure 6. R01-Equivalent Grant Average Cost: 2000 to 2023 Source: NIH Data Book, “R01-Equivalent Grants: Average Size,” https://report.nih.gov/nihdatabook/report/158. Notes: Inflation adjustment used the Biomedical Research and Development Price Index (BRDPI). Intramural Research The NIH intramural research program (IRP), at about $5.0 billion in FY2023, accounts for approximately 11% of the total NIH budget.64 IRP employs approximately 1,150 principal investigators and 6,000 trainees, ranging from high school students to postdoctoral and clinical 62 NIH Data Book, “R01-Equivalent Grants: Average Size,” https://report.nih.gov/nihdatabook/report/158. 63 NIH Data Book, “Research Center Grants: Average Size,” https://report.nih.gov/nihdatabook/report/160. 64 CRS analysis of data from NIH, “FY2025 Justification of Estimates for Appropriations Committees, Vol I: Overview–History of Obligations by Total Mechanism,” p. 50, https://officeofbudget.od.nih.gov/pdfs/FY25/br/ Overview%20of%20FY%202025%20Presidents%20Budget%20corrected%20Jul%2015.pdf. Congressional Research Service 18 The National Institutes of Health (NIH): Background and Congressional Issues fellows in NIH-operated laboratories.65 Other IRP personnel include administrative support staff, guest researchers, and contractors. Intramural research takes place at the 322-acre main campus in Bethesda, MD, and several off-campus sites, including locations in Maryland, North Carolina, Montana, Arizona.66 Almost all of the ICs have an intramural research program, but the size, structure, and activities of the programs vary greatly.67 As with extramural funding, most intramural research proposals are investigator-initiated. However, NIH sets the direction for its intramural research program by hiring scientists of targeted expertise, allocating resources to certain laboratories and programs, and conducting reviews by panels of external experts. 68 In addition, intramural researchers are generally subject to the same research policy requirements as extramural researchers (see the Appendix section) but adhere to these requirements through internal NIH processes.69 Each intramural scientist is evaluated by an external Board of Scientific Counselors from their IC every four years to review their work and research portfolio. Each IC’s intramural research program is also reviewed by an external panel every 10 years, concerning the entire research portfolio and impact of the research.70 Some intramural scientists work in the Clinical Center, which houses both basic research laboratories and clinics for scientists involved with patient care in clinical research studies. The Clinical Center is the nation’s largest hospital devoted solely to clinical research. Along with scientists, the Clinical Center employs over 1,000 nurses and allied health professionals to support its work.71 Most ICs with intramural research programs fund research at the Clinical Center. Research Training As stated by the agency, “NIH’s ability to ensure that it remains a leader in scientific discovery and innovation is dependent upon a pool of creative, diverse, and highly talented researchers.”72 Research training activities are designed to support every stage of a biomedical research career (see the “Stages of a Research Career” text box below) in both the extramural and intramural research programs. Programs range from summer internships for high school students to mentoring programs for independent investigators. Predoctoral and postdoctoral training opportunities are available through a variety of training grants, fellowships, and loan repayment programs.73 The largest extramural program is called the Ruth L. Kirschstein National Research Service Awards (NRSA) program, authorized by PHSA Section 487, which supports pre- and 65 NIH, Report of the Director of the National Institutes of Health: Fiscal Years 2019-2021, p. 17, https://dpcpsi.nih.gov/sites/default/files/2023-09/FY19-21%20Triennial_Report_FINAL_508C.pdf. 66 NIH Intramural Research Program, “Research Campus Locations,” at https://irp.nih.gov/about-us/research-campuslocations. 67 See links to individual IC programs at https://irp.nih.gov/about-us/our-programs. ICs that do not have an intramural research component are the National Institute of General Medical Sciences (NIGMS) and the Fogarty International Center (FIC). 68 NIH, Report of the Director of the National Institutes of Health: Fiscal Years 2019-2021, p. 18, https://dpcpsi.nih.gov/sites/default/files/2023-09/FY19-21%20Triennial_Report_FINAL_508C.pdf. 69 See NIH, “Policy Manual,” https://policymanual.nih.gov/. 70 NIH, Report of the Director of the National Institutes of Health: Fiscal Years 2019-2021, p. 18, https://dpcpsi.nih.gov/sites/default/files/2023-09/FY19-21%20Triennial_Report_FINAL_508C.pdf. 71 Ibid, pp. 19-20. 72 NIH, “The Biomedical Research Workforce,” https://researchtraining.nih.gov/dbrw/biomedical-research-workforce. 73 NIH, “Research Training and Career Development,” https://researchtraining.nih.gov/. Congressional Research Service 19 The National Institutes of Health (NIH): Background and Congressional Issues postdoctoral research training awards to both institutions and individuals. In 2023, the NRSA program supported over 17,000 graduate students and postdoctoral fellows.74 Stages of a Scientific Research Career • Undergraduate and Postbaccalaureate. Current students or recent recipients of bachelor’s degrees who are studying or working in scientific research. • Predoctoral/Graduate Training. Graduate students working toward a research or clinical doctorate degree. Usually involves working on highly structured research projects under the supervision of an experienced mentor. • Postdoctoral/Clinical Residency. New doctorate recipients who gain further training to help transition to a career as an independent researcher. • Early Career Researcher. Scientists who have recently obtained independent positions as investigators, faculty members, clinician scientists, or industry scientists. • Established Investigator. Scientists who have demonstrated expertise in their research field through a record of independent and original scientific contributions. They often serve as mentors to trainees at undergraduate, predoctoral, and postdoctoral levels. Source: NIH, “Research Training and Career Development- Career Path,” at https://researchtraining.nih.gov/ career-path. Information Dissemination NIH has important roles in translating the knowledge gained from biomedical research into medical practice and useful health information for the general public. The individual ICs carry out many relevant activities, such as sponsoring seminars, meetings, and consensus development conferences to inform health professionals of new findings; answering thousands of telephone, mail, and online inquiries; publishing physician and patient education materials on the internet and in print; supporting information clearinghouses and running public information campaigns on various diseases; making specialized databases available; and fostering partnerships for educating clinicians and other health care professionals on the latest science.75 Budget At roughly $47 billion for FY2024 (excluding ARPA-H funding), NIH’s budget is much larger than those of other PHS agencies such as the Food and Drug Administration (FDA), Centers for Disease Control and Prevention (CDC), Health Resources and Services Administration (HRSA), Indian Health Service (IHS), and the Substance Abuse and Mental Health Services Administration (SAMHSA). In FY2023, about 32% of all discretionary HHS funding was provided to NIH.76 Moreover, as of FY2023, NIH represented about one-fifth of total federal R&D funding and close to 45% of federal spending on R&D outside of the Department of Defense.77 NIH has seen budget fluctuations, as shown in Figure 7. Prior to 2004, Congress had doubled the NIH program level over a five-year period, from its FY1998 base of $13.7 billion to the FY2003 74 CRS analysis of numbers available in NIH, “Data Book- Kirschstein-NRSA Training Grants and Fellowships: Pre- and Post-Doctoral Full-Time Training Positions Awarded,” https://report.nih.gov/nihdatabook/report/52. 75 NIH, Report of the Director of the National Institutes of Health: Fiscal Years 2019-2021, pp. 50-53, https://dpcpsi.nih.gov/sites/default/files/2023-09/FY19-21%20Triennial_Report_FINAL_508C.pdf. 76 Analysis of data used for CRS Report R48060, Department of Health and Human Services: FY2025 Budget Request. 77 CRS analysis of federal research and development budget data provided by the Office of Management and Budget. Congressional Research Service 20 The National Institutes of Health (NIH): Background and Congressional Issues level of $27.2 billion. Subsequently, NIH experienced a decade of stagnant growth in the agency’s budget. Congress provided budget increases generally around 1.0%-3.2% from FY2004 to FY2015, often lower than the rate of inflation for biomedical research, which resulted in reduced purchasing power for the agency. In some years (FY2006, FY2011, and FY2013), funding for the agency decreased in nominal dollars. Starting in FY2016 through FY2023, Congress provided NIH with funding increases each year, mostly over 5% annually, increasing the program level from $30.3 billion in FY2015 to $47.7 billion in FY2023.78 NIH once again saw a slight reduction in its overall program level in FY2024, decreasing -0.7% compared with FY2023. In inflation-adjusted FY2023 dollars, the FY2024 NIH program level remains roughly 6% below the peak 2003 level.79 For more information, see CRS Report R43341, National Institutes of Health (NIH) Funding: FY1996-FY2025. 78 FY2023 level excludes funding for the Advanced Research Projects Agency for Health (ARPA-H). 79 Analysis excludes funding for ARPA-H. Congressional Research Service 21 The National Institutes of Health (NIH): Background and Congressional Issues Figure 7. NIH Funding, FY1998-FY2024 Program Level Funding in Current and Constant (FY2023) Dollars Source: FY2024 request and FY2024-enacted numbers from Congressional Record, daily edition, vol. 170, no. 51, Book 11, March 22, 2024, pp. H2022-H2025, accessed at https://www.congress.gov/118/crec/2024/03/22/170/ 51/CREC-2024-03-22-bk2.pdf, and P.L. 118-47. FY2023 final and FY2025 request numbers from NIH, Overview of FY2025 President’s Budget, pp. 100, 101, at https://officeofbudget.od.nih.gov/pdfs/FY25/br/ Overview%20of%20FY%202025%20Presidents%20Budget.pdf, and ARPA-H, Congressional Justification: FY2025, p. 9, accessed at https://arpa-h.gov/sites/default/files/2024-03/ARPA-H%20FY%202025.pdf. The FY2022 (and earlier) program levels are from NIH Budget Office, Appropriations History by Institute/Center (1938 to Present), at http://officeofbudget.od.nih.gov/approp_hist.html. Inflation adjustment reflects the Biomedical Research and Development Price Index (BRDPI), updated January 2024, at https://officeofbudget.od.nih.gov/ gbiPriceIndexes.html. Notes: Program level includes all budget authority, including transfers noted in budget documents. Amounts provided to NIH designated for emergency requirements are excluded from these totals (e.g., the FY2020 and FY2021 amounts do not include the amounts provided in the coronavirus supplemental appropriations acts). Congressional Research Service 22 The National Institutes of Health (NIH): Background and Congressional Issues Foundation for the NIH NIH also works with the Foundation for the National Institutes of Health (FNIH), a 501(c)(3) charitable organization that raises private funding and manages public-private partnerships to support NIH’s mission. FNIH was established by statute in 1990 (P.L. 101-613) and was amended in 1993 (P.L. 103-43). The organization initially began operations in 1996. FNIH supports research projects and programs, education and training, conferences and events, and other support activities for NIH.80 Pursuant to PHSA Section 499 (42 U.S.C. §290b), there are terms and restrictions on activities, requirements for the board of directors, reporting requirements, and other requirements for FNIH. As of 2023, FNIH has raised over $1.5 billion in support of NIH’s mission.81 FNIH also receives some transfers of NIH’s appropriations for its administrative and operational expenses (averaging less than 0.01% of NIH’s annual budget).82 For more information on FNIH, see the relevant section in CRS Report R46109, Agency-Related Nonprofit Research Foundations and Corporations. Setting NIH Research Priorities NIH funds research on hundreds of diseases, conditions, and areas of human health.83 NIH funding is highly competitive—21.4% of all research project grant applications were funded in FY2023.84 NIH and Congress face trade-offs in allocating funding in a fair manner that balances the scientific merit of proposals with meeting the diverse health needs of the population. Funding decisions are especially difficult because science is a process of discovery. Even experts cannot always predict which proposals will lead to breakthroughs. Historic tensions have included • • • • • whether to designate funding for specific diseases and areas of research or to allow untargeted funding for the most meritorious proposals identified through the peer review process; how to balance funding for basic scientific research with applied research; whether funding should go to certain ethically contentious research areas, such as embryonic stem cell research; how to fund research on the most pervasive diseases and conditions while also funding research on rare diseases or emerging health issues; how to allocate funding among established and successful scientists while enabling new scientists to enter the field; 80 FNIH, “About Us,” https://fnih.org/about, and FNIH, “Our Programs,” https://fnih.org/our-programs/. 81 FNIH, “FNIH Health Impact Report: 2023 Facts and Figures,” https://fnih.org/story-2023-facts-figures/. 82 CRS Report R46109, Agency-Related Nonprofit Research Foundations and Corporations. 83 The NIH “Estimates of Funding for Various Research, Condition, and Disease Categories (RCDC)” table includes over 280 categories of diseases, conditions, and research areas for which NIH categorizes its funding. The table is not comprehensive of all possible ways to categorize NIH research. See NIH, “Estimates of Funding for Various Research, Condition, and Disease Categories (RCDC)” table, last updated May 14, 2024, https://report.nih.gov/funding/ categorical-spending#/. 84 NIH, “Justification of Estimates for Appropriations Committees FY2025, Overview Vol. I,” p. 113. Congressional Research Service 23 The National Institutes of Health (NIH): Background and Congressional Issues • and how to determine the appropriate way to fund research among available mechanisms, including extramural grants, contracts, and intramural research.85 In recent decades, Congress allowed NIH ICs, for the most part, to fund research based on their own internal prioritization process, which involves scientific experts, patient advocates, and other constituencies. At times, including in recent years, Congress has provided direction to NIH funding in both appropriations report language and legislation. The following sections summarize (1) NIH internal processes for setting research priorities through strategic planning and advisory groups, and (2) congressional involvement in NIH research priorities, including recent major efforts, legislation, and research restrictions. NIH is not the only federal agency that supports biomedical and health research. The Department of Defense (DOD) and the Department of Veterans Affairs (VA) and others also support medical research programs.86 In addition, other HHS agencies support health research, such as the Centers for Disease Control and Prevention (CDC) and the Agency for Healthcare Quality and Research (AHRQ). Although the below discussion focuses on research priorities at NIH, Congress may consider how to prioritize and coordinate funding for medical and health research across the federal government. NIH Process in Setting Research Priorities Each NIH IC has separate research priorities, which are specified in statutory authority in varying levels of detail.87 IC research priorities are also broadly captured by their mission statements.88 ICs establish research priorities through strategic planning, annual planning, and periodically reviewing and assessing their research portfolios. Each IC has an advisory council that makes recommendations for IC research priorities and funding decisions. Per statute, the advisory councils consist of no more than 18 members appointed by the HHS Secretary. Two-thirds of the members represent leading representatives of the relevant health and scientific disciplines. Onethird of the members are appointed from the general public and include leaders from other fields, such as law, health policy, economics, and management.89 According to the agency, decisionmakers at NIH seek advice from many groups when setting research priorities, including scientific researchers and professional science societies, patient organizations and voluntary health associations, IC Advisory Councils, Congress and the Administration, the Advisory Committee to the NIH Director, the SMRB, and NIH staff.90 85 See discussions in U.S. Congress, House Energy and Commerce Committee, Health Subcommittee, Scientific Opportunities and Public Needs: Balancing NIH’s Priority Setting Process, 108th Cong., 2nd sess., June 2, 2004, and U.S. Congress, Senate Health, Education, Labor, and Pensions Committee, Labor Subcommittee, Biomedical Research Priorities: Who Should Decide?, 105th Cong., 1st sess., May 1, 1997. 86 See CRS In Focus IF10349, Congressionally Directed Medical Research Programs Funding for FY2024, and section on “Medical Care and Medical Research Discretionary Programs Funding” in CRS Report R48056, Department of Veterans Affairs FY2024 Appropriations. 87 Title IV of PHSA includes statutory authorities for all NIH ICs. See the “Authority” section of this report. 88 NIH, “List of Institutes and Centers,” last updated July 2023, https://www.nih.gov/institutes-nih/list-institutescenters. 89 Most NIH advisory councils are authorized in PHSA Section 406; 42 U.S.C. §284a. That section specifies membership and responsibilities for each advisory council. 90 NIH, “NIH Research Planning,” https://www.nih.gov/about-nih/nih-research-planning. Congressional Research Service 24 The National Institutes of Health (NIH): Background and Congressional Issues Strategic Planning For many years, most ICs have undergone a periodic strategic planning process to determine its funding priorities among the research areas in each IC’s broadly defined mission and programmatic areas.91 Statute specifies that the NIH Director “shall ensure that scientifically based strategic planning is implemented in support of research priorities as determined by the agencies of the National Institutes of Health.”92 The Cures Act (P.L. 114-255) added a requirement for an NIH-Wide Strategic Plan, in part to facilitate IC collaboration and coordination, to be updated every six years.93 This followed a prior directive for NIH to develop an agency-wide strategic plan in the FY2015 appropriations law (P.L. 113-235).94 (See the “Recent Major Legislative History” section.) In the latest NIH-wide strategic plan for 2021-2025, NIH states that it seeks to meet its mission “by pursuing scientific opportunities when they arise, responding to ongoing and emerging public health needs, and addressing rare diseases.” The plan specifies overarching agency objectives around advancing research, supporting research capacity, and ensuring research integrity. In addition, the plan names specific themes that NIH intends to support across its research portfolios. These include (1) Improving Minority Health and Reducing Health Disparities, (2) Enhancing Women’s Health, (3) Addressing Public Health Challenges Across the Lifespan, (4) Promoting Collaborative Science, and (5) Leveraging Data Science for Biomedical Discovery.95 According to NIH, the Strategic Plan was developed with input from external stakeholders, including “members of the scientific and health care communities, professional societies, advocacy organizations, industry, other federal agencies, and the general public” and in collaboration with leadership and staff of NIH’s Institutes, Centers, and Offices.96 Coordinating Across NIH The NIH Reform Act of 2006 (P.L. 109-482) enhanced the authority of the NIH Director’s Office to perform strategic planning, especially facilitating and funding transdisciplinary, cross-institute research initiatives. The Reform Act also created a special office, the Division of Program Coordination, Planning, and Strategic Initiatives (DPCPSI), that “identifies important areas of emerging scientific opportunity or rising public health challenges to assist in the acceleration of research investments in these areas.”97 The Office of Strategic Coordination within DPCPSI manages the NIH Common Fund, which supports large, complex research efforts that involve the collaboration of two or more research institutes or centers. The Office of Strategic Coordination works with staff and leadership across NIH to identify and promote NIH-wide scientific opportunities that receive Common Fund support.98 Each individual IC strategic plan specifies its planning process; from NIH, “NIH Strategic Plans and Visions,” at https://report.nih.gov/reports/strategic-plans. 92 PHSA Section 402(b)(5); 42 U.S.C. §282(b)(5). 93 42 U.S.C. §282(m). 94 See 128 STAT. 2475. 95 NIH, “NIH-Wide Strategic Plan: Fiscal Years 2021-2025,” p. 3, https://www.nih.gov/sites/default/files/about-nih/ strategic-plan-fy2021-2025-508.pdf. 96 Ibid, pp. 44-45. 97 NIH, “NIH Research Planning,” http://www.nih.gov/about/researchplanning.htm. 98 NIH, “Office of Strategic Coordination—The Common Fund,” https://commonfund.nih.gov/about. 91 Congressional Research Service 25 The National Institutes of Health (NIH): Background and Congressional Issues Coordinating Across the Federal Government As mentioned above, several agencies within and outside of HHS fund health research. NIH program staff use applicant information and other agencies’ funding databases to avoid duplicating funding with another agency for the same recipient for the same project.99 In addition, NIH holds regular meetings to collaborate and align priorities with other HHS research agencies, DOD, and VA.100 NIH publishes an annual report and maintains a database of its collaborations with other HHS agencies.101 The executive branch and Congress have also established some interagency strategies and committees aimed at aligning research priorities across agencies, usually in an effort to ensure that funded research across agencies furthers overarching health goals. These strategies or committees are often specific to certain disease or research areas. The next section provides a few examples established by Congress. Selected Examples of Interagency Coordinating Committees Interagency Pain Research Coordinating Committee (IPRCC): The Patient Protection and Affordable Care Act (ACA; P.L. 111-148, as amended) established the Interagency Pain Research Coordinating Committee,102 which is now led by NIH.103 The committee, made up of federal and nonfederal members, oversees scientific progress across the government under the Federal Pain Research Strategy, which focuses on advancing pain prevention and management, along with other efforts to advance pain research.104 Muscular Dystrophy Coordinating Committee (MDCC): As authorized in statute,105 the Muscular Dystrophy Coordinating Committee coordinates research across NIH and with other federal agencies on all forms of muscular dystrophy. The committee, currently supported by National Institute of Neurological Disorders and Stroke (NINDS), includes both federal and nonfederal members and is tasked with developing a plan for research and education on muscular dystrophy across HHS to encompass health, psychosocial, public services, and rehabilitative issues related to the disease.106 Interagency Autism Coordinating Committee: As initially established by the Children’s Health Act of 2000 (P.L. 106-310), the Interagency Autism Coordinating Committee (IACC), composed of federal and nonfederal public members, coordinates federal efforts related to autism spectrum 99 U.S. Government Accountability Office (GAO), Biomedical Research: Actions Needed to Adopt Collaboration Practices to Address Research Duplication, GAO-24-106757, February 2024, https://www.gao.gov/assets/870/ 866837.pdf, and GAO, Biomedical Research: Observations on DOD’s Management of Congressionally Directed Medical Research Programs, GAO-22-105107, January 31, 2022, pp. 6-7, https://www.gao.gov/assets/gao-22105107.pdf. 100 Ibid. 101 See NIH, “Report on NIH Collaborations with Other HHS Agencies for Fiscal Year 2022,” https://crs.od.nih.gov/ CRSPublic/. 102 Authorized at PHSA Section 409J(b); U.S.C. 42 §284q. 103 NIH, “About the NIH IPRCC,” https://www.iprcc.nih.gov/about/nih-iprcc. Specifically, the Director of the National Center for Complementary and Integrative Health currently chairs the committee. See “Membership,” https://www.iprcc.nih.gov/about/membership. 104 NIH, “Federal Pain Research Strategy Overview,” https://www.iprcc.nih.gov/federal-pain-research-strategyoverview. 105 PHSA Section 404E(d); 42 U.S.C. §283g(d). 106 PHSA Section 404E(e) and NIH, “Charter: Muscular Dystrophy Coordinating Committee,” https://www.ninds.nih.gov/sites/default/files/documents/MDCC_Charter_508C.pdf. Congressional Research Service 26 The National Institutes of Health (NIH): Background and Congressional Issues disorder (ASD), including both research and services and supports activities.107 The National Institute of Mental Health manages the committee and provides administrative support.108 Congressional Involvement in NIH Research Priorities Congress has shaped NIH by establishing its overall authorizing statutes (see the “Authority” section), which govern the agency’s overall structure, its award and review processes, and the responsibilities of each of its ICs. Congress also provides annual appropriations to the IC accounts, which drive NIH’s overall research direction by setting different funding levels for ICs with different missions. From time to time, Congress has also authorized or funded specific research programs—often disease-specific research programs—either within or across NIH ICs. Congress has supported major large-scale research initiatives on specific diseases at NIH, including during the War on Cancer in the 1970s, for the HIV/AIDS epidemic in the 1980s and 1990s, and through several research initiatives discussed in the “Selected Recent Research Initiatives” section.109 A long-standing debate has centered on whether and to what extent Congress should specify funding for certain diseases or programs within NIH, or whether Congress should allow the agency to determine research funding allocations through its own priority setting and review processes.110 The following sections discuss how Congress has shaped NIH’s research priorities through both appropriations and authorizations legislation. Congress at times has also enacted certain restrictions on NIH research. Appropriations For many years prior to FY2015, appropriators avoided specifying dollar amounts for particular disease areas, fields of research, or mechanisms of funding in both report and bill text, aside from the level of the IC accounts. Generally, specific amounts were appropriated to each IC, and then funding was awarded through competitive grants, through contracts, or to intramural researchers.111 Changes in congressional practice have occurred most notably with research funding for Alzheimer’s disease (discussed further in the “Alzheimer’s Disease and Related Dementias Research” section). From FY2001 through FY2014, Congress provided broad directives to NIH in report language, encouraging the agency to prioritize Alzheimer’s disease and to increase resources toward its research through the National Institute on Aging (NIA).112 The explanatory statement accompanying the FY2014 omnibus included the following language: In keeping with longstanding practice, the House and Senate Appropriations Committees do not recommend a specific amount of NIH funding for this purpose or for any other individual disease. Doing so would establish a dangerous precedent that could politicize the NIH peer review system. Nevertheless, in recognition that Alzheimer’s disease poses a serious threat to the Nation’s long-term health and economic stability, the agreement 107 PHSA Section 399CC; 42 U.S.C. §280i-2. 108 NIH, “Interagency Autism Coordinating Committee Charter,” https://iacc.hhs.gov/about-iacc/charter/. 109 NIH National Cancer Institute, “National Cancer Act of 1971,” https://www.cancer.gov/about-nci/overview/history/ national-cancer-act-1971, and Department of Health and Human Services (HHS), “A Timeline of HIV and AIDS,” HIV.gov, https://www.hiv.gov/hiv-basics/overview/history/hiv-and-aids-timeline. 110 Rachel Kahn Best, “Chapter 4: Ranking Diseases,” in Common Enemies: Disease Campaigns in America (New York, NY: Oxford University Press, 2019), pp. 84-108. 111 CRS review of appropriations documents. 112 Based on CRS search of “Alzheimer’s” and related terms in enacted appropriations laws, accompanying committee reports, and House and Senate committee appropriations bills from FY2001 to FY2014. Congressional Research Service 27 The National Institutes of Health (NIH): Background and Congressional Issues expects that a significant portion of the recommended increase for NIA should be directed to research on Alzheimer’s. The exact amount should be determined by the scientific opportunity of additional research on this disease and the quality of grant applications that are submitted for Alzheimer’s relative to those submitted for other diseases. 113 The explanatory statement for the FY2015 omnibus included similar language but noted that the agreement provided a $25 million increase for Alzheimer’s disease research at NIA; still, it did not direct NIH to reserve a specific total dollar amount.114 Then, in a departure from recent precedent, the explanatory statements accompanying FY2016 appropriations directed NIH to reserve a specific amount for Alzheimer’s disease research.115 In recent years, appropriations reports for NIH have specified dollar amounts for some research related to certain diseases or topics, though annual appropriations have left most of each IC’s funding flexible and untargeted. For example, the explanatory statement accompanying FY2024 appropriations for NIH included over 40 line items directing specific dollar amounts for certain research or program areas.116 A Senate FY2024 appropriations report (S.Rept. 118-84) explained the committee’s approach to targeting NIH funding as follows: As in previous years, the Committee has targeted NIH funding in areas of promise of scientific advancement and urgency, while allowing NIH to maintain flexibility to pursue unplanned scientific opportunities and address unforeseen public health needs. 117 Authorizations At times, Congress has enacted authorizations for specific programs or research areas within NIH ICs. Congress has, for example, enacted provisions targeting new types or approaches to research to be supported by NIH. For example, the Cures Acceleration Network (P.L. 111-148) in 2010 sought to advance technologies to improve drug development. Laws have also been enacted targeting specific disease or health program areas within NIH ICs. For example, since 2010, specific laws were enacted related to research on hearing loss screening and detection (P.L. 111337), pancreatic and lung cancer (P.L. 112-239), pediatric cancer (P.L. 113-94, P.L. 115-180), muscular dystrophy (P.L. 113-166), and pain (P.L. 114-198), to name a few examples. In some cases, these laws have provided NIH or its ICs with new authorities or funding sources for research and, at times, have included new requirements for NIH research (e.g., strategic planning or reporting requirements).118 However, in many cases, NIH does not need a specific authorization to fund research on a certain health topic. NIH is able to support research on nearly all areas of human health through its existing authorizations. Some policymakers have long questioned whether considering disease-specific legislation for NIH research is a productive use of limited committee and floor time, and whether such 113 Congressional Record, January 15, 2014, vol. 160, no. 9—Book II, H1037. 114 Congressional Record, December 11, 2014, vol. 160, no. 151—Book II, H9832. 115 Congressional Record, December 17, 2015, vol. 161, no. 184—Book III, H10285. 116 See Table A-1 in CRS Report R43341, National Institutes of Health (NIH) Funding: FY1996-FY2025. Includes funding directives incorporated by reference from S.Rept. 118-84. 117 S.Rept. 118-84, p. 89. 118 For example, the Gabriella Miller Kids First Research Act (P.L. 113-94) created a new funding source for research by directing transfers of certain amounts from the Presidential Election Campaign Fund to a new 10-Year Pediatric Research Initiative Fund to be made available for pediatric research as authorized by the law. The authorization for the Cures Acceleration Network (P.L. 111-148) granted NIH new authorities to support research, including through Other Transactions authority and through requiring matching funds from certain recipients under the program. P.L. 115-180 added new reporting requirements for NIH on childhood cancer research projects. Congressional Research Service 28 The National Institutes of Health (NIH): Background and Congressional Issues legislation leads to the best outcomes at NIH.119 At the same time, Members of Congress frequently hear from stakeholders—particularly disease-specific advocates—calling for NIH research on certain topics.120 These stakeholders may express concern that NIH is inadequately funding research in certain areas or that the agency’s funded research approaches are not meeting health needs.121 In an effort to be responsive to these stakeholders, Congress has sometimes considered and passed disease-specific legislation for NIH research. To illustrate, in 1993, an exchange between a House Representative and then-HHS Secretary during a hearing on the NIH Revitalization Act (H.R. 4, 103rd Congress) showed the considerations at hand with disease- and program-specific provisions in the bill: Rep Greenwood: “H.R. 4 is a nice, big, thick, 170-page bill that gives a lot of direction to the NIH with regard to research. The question I have is this: Is there anything in here that the institutes could not do without this legislation? Do you really need this kind of direction? Or, are we guilty of micro-managing in response to all of the well intentioned disease groups, if you will, pressuring for research funding.” Sec. Shalala: “Of course, there is nothing that we could not do without those directives. The question is whether our own strategic planning process would produce that specific list which is what you are asking. My answer to this is that this is government money and those elected by the government have a right to give us-to nudge us, to set the standards for us, to give us a list of what they think is important. We also have a right to come back and argue what we think the priorities ought to be privately or publicly. It is just if I am going to keep my own integrity as part of this process, I am not going to pretend that we would not prefer to have more flexibility. I certainly wanted that as I headed any agency, but what I am suggesting to you is that this bill, as structured, is one that we believe on balance we can support. There are obviously parts of it that we might not think are terrific at this point in time.”122 Research Restrictions From time to time, Congress has placed restrictions on NIH research, often in annual appropriations legislation. Restrictions for FY2024 related to, for example, advocating or promoting gun control, payment for abortions, human embryo research, and promoting 119 See, for example, statement by Rep. Bliley in 1993 during consideration of the NIH Revitalization Act (H.R. 4, 103rd Congress), “I personally have serious reservations that world-renowned institution such as the NIH really needs this much detailed Congressional direction in order to conduct the best possible scientific research” (from U.S. Congress, House Energy and Commerce Committee, Health and Environment Subcommittee, NIH Revitalization Act, 103rd Cong., 1st sess., February 3, 1993), and statement by Senator Kennedy in 1997 hearing, “Setting research priorities, a complex process that must be informed by the concerns of many groups—the patients, women, children and the elderly. But the final judgment on the direction of the biomedical research must be left largely to NIH. They have the knowledge and experience to make the wisest decision” (from U.S. Congress, Senate Committee on Labor and Human Resources, Subcommittee on Public Health and Safety, Biomedical Research Priorities: Who Should Decide?, 105th Cong., 1st sess., May 1, 1997). 120 To illustrate, an analysis found that beginning in the 1980s, witnesses representing disease-specific organizations or patient advocacy groups made up over 20% of all witnesses at annual House LHHS appropriations hearings that are open to all public witnesses. By the 1990s, such witnesses made up about one-third of all witnesses at such hearings. This is particularly notable because LHHS appropriations fund a wide a wide array of health, education, labor, social services, and other programs. See Rachel Kahn Best, “Chapter 4: Ranking Diseases,” in Common Enemies: Disease Campaigns in America (New York, NY: Oxford University Press, 2019), pp. 87-88. 121 Rachel Kahn Best, “Chapter 4: Ranking Diseases,” in Common Enemies: Disease Campaigns in America (New York, NY: Oxford University Press, 2019), pp. 84-108. 122 U.S. Congress, House Energy and Commerce Committee, Health and Environment Subcommittee, NIH Revitalization Act, 103rd Cong., 1st sess., February 3, 1993. Congressional Research Service 29 The National Institutes of Health (NIH): Background and Congressional Issues legalization of controlled substances.123 In many cases, these restrictions reflected ethical and political decisions that Congress made about the types of research that NIH should support. As an example, the restriction on human embryo research dates back to FY1996 and prohibits HHS from creating human embryos for research purposes or “for research in which a human embryo or embryos are destroyed, discarded, or knowingly subjected to risk of injury or death greater than that allowed for research on fetuses in utero” under referenced laws and regulations.124 Selected Recent Research Initiatives Alzheimer’s Disease and Related Dementias Research As noted in the “Appropriations” section, Congress began directing specific funding levels for Alzheimer’s and related dementias research in appropriations reports from FY2015 through FY2024, reflecting an overall recent change in congressional practice around specifying funding for certain diseases at NIH. Table 1. NIH Alzheimer’s Disease Research Funding Directed by Congress Fiscal Year Amount FY2015 Increase of $25 million (no total specified) FY2016 $926 million (+350 million) FY2017 $1,391 million (+400 million) FY2018 $1,828 million (+414 million) FY2019 $2,340 million (+425 million) FY2020 $2,818 million (+350 million) FY2021 $3,118 million FY2022a Increase of $289 million FY2023 Increase of $226 million FY2024 Increase of $100 million Source: Reports and explanatory statements accompanying annual Departments of Labor, Health and Human Services, and Education, and Related Agencies Appropriations (LHHS) appropriations laws. Notes: Amounts shown in parentheses from FY2016 to FY2020 show increases from the prior fiscal year. In some years, language directed funding for Alzheimer’s disease research. In other years, language directed funding for Alzheimer’s disease and related dementias research. The table does not show allocations to specific institutes and centers named in the directives. a. Beginning in FY2022, the reports did not state a total provided for Alzheimer’s disease and related dementias research, but rather stated that the appropriations law provided an increase for such research. These funding increases have been driven by the National Plan to Address Alzheimer’s Disease, first announced in 2012.125 Established by the National Alzheimer’s Project Act (NAPA; P.L. 111375), the National Plan includes “Prevent and Effectively Treat Alzheimer’s Disease and Related 123 NIH, “Notice of Legislative Mandates in Effect for FY2024,” from https://grants.nih.gov/grants/guide/notice-files/ NOT-OD-24-110.html. 124 Specifically, 45 C.F.R. §46.204(b) and PHSA Section 498(b) (42 U.S.C. §289g(b)). See Section 508 of Division D in Further Consolidated Appropriations Act, 2024 (P.L. 118-47), for current language of the restriction. 125 HHS, “Obama Administration Presents National Plan to Fight Alzheimer’s Disease,” press release, May 15, 2012, https://aspe.hhs.gov/obama-administration-presents-national-plan-fight-alzheimers-disease. Congressional Research Service 30 The National Institutes of Health (NIH): Background and Congressional Issues Dementias by 2025” as the first of five key goals.126 To help meet this goal, NIH began to publish an annual bypass budget in FY2015 to estimate funding needs for Alzheimer’s disease research, starting for FY2017. A bypass budget, also known as a professional judgement budget, is a budget proposal submitted directly by NIH to Congress to estimate research funding needs based on scientific opportunity, rather than as determined by the regular budgeting process. The bypass budget was mandated by the Consolidated and Further Continuing Appropriations Act of 2015 (P.L. 113-235), which specified that the NIH Director is to submit an annual independent Alzheimer’s research budget request directly to Congress, pursuant to the National Alzheimer’s Plan. To determine its bypass budget proposal, NIH has convened research summits starting in 2012 and has worked across its ICs to determine recommendations and funding needs for Alzheimer’s disease research. To meet its research goals, NIH has used targeted funding opportunity notices to solicit research proposals related to Alzheimer’s disease from scientists.127 Alzheimer’s disease research represents an area of major congressional involvement, in which large amounts of research funding are directed toward a specific disease. In recent years, some advances have been made in preventing, diagnosing, and treating Alzheimer’s disease and related dementias, many of which have been linked to NIH research. For example, recent diagnostic advances in imaging and certain fluid-based tests have helped improve the ability to identify and diagnose Alzheimer’s disease and related dementias in conjunction with other clinical evaluations.128 NIH helped fund the development of the first blood test of amyloid, a biomarker (or biological indicator) of Alzheimer’s disease.129 NIH-funded research contributed to the imaging technologies used in clinical trials to assess the efficacy of drugs to treat Alzheimer’s disease and related dementias.130 In addition, NIH-funded research has contributed to an understanding of prevention; for instance, that controlling high blood pressure may reduce agerelated cognitive impairment that may ultimately lead to Alzheimer’s disease.131 In recent years, the U.S. Food and Drug Administration has approved three drugs for treating mild or early-stage Alzheimer’s disease.132 NIH funded basic research into amyloid, the brain protein targeted by these drugs that helped inform their scientific basis.133 There is still much progress to be made in research on Alzheimer’s disease and related dementias, including better understanding the contributions of genetics, environmental exposures, and life events to the disease and for improving diagnosis, including through digital tools or improved 126 HHS, National Plan to Address Alzheimer’s Disease, 2012, p. 6, https://aspe.hhs.gov/system/files/pdf/102526/ NatlPlan2012%20with%20Note.pdf. 127 NIH, Open Science, Big Data, and You: Working Together to Treat and Prevent Alzheimer’s Disease and Related Dementias. NIH Bypass Budget Proposal for Fiscal Year 2020, July 30, 2018, https://www.nia.nih.gov/sites/default/ files/2018-07/fy2020-bypass-budget-report-final.pdf. 128 W.M van der Flier, M.E de Vugt, E.M.A Smets, et al., “Towards a Future Where Alzheimer’s Disease Pathology is Stopped Before the Onset of Dementia,” Nature Aging, vol. 3 (May 18, 2023), pp. 494-505. 129 National Institute on Aging (NIA), “Small Business Spotlight: C₂N Diagnostics’ Blood Test Detects Alzheimer’s,” November 1, 2022, https://www.nia.nih.gov/news/small-business-spotlight-c2n-diagnostics-blood-test-detectsalzheimers, and National Institute on Aging, “Biomarker Research,” in 2020–2021 Report of Scientific Advances for the Prevention, Treatment, and Care of Alzheimer’s Disease and Related Dementias, https://nia.nih.gov/report-20202021-scientific-advances-prevention-treatment-and-care-dementia/biomarker-research. 130 NIH, “10 Years of Alzheimer’s Disease and Related Dementias Research,” September 2023, https://www.nia.nih.gov/10-years-alzheimers-disease-and-related-dementias-research. 131 NIA, “Intensive Blood Pressure Control May Slow Age-Related Brain Damage,” press release, August 13, 2019, https://www.nia.nih.gov/news/intensive-blood-pressure-control-may-slow-age-related-brain-damage. 132 Alzheimer’s Association, “FDA-Approved Treatments For Alzheimer’s,” https://www.alz.org/media/Documents/ alzheimers-dementia-fda-approved-treatments-for-alzheimers-ts.pdf. 133 NIH, “2024 NIH Alzheimer’s and Related Dementias Research Progress Report: Advances and Achievements,” https://www.nia.nih.gov/sites/default/files/2024-08/2024-alzheimers-progress-report.pdf. Congressional Research Service 31 The National Institutes of Health (NIH): Background and Congressional Issues fluid-based tests, among many other topics. In addition, while drugs for treating Alzheimer’s disease are now available, they serve a relatively small patient population with early or mild disease and can have significant side effects.134 As of March 2024, the National Institute on Aging reported funding 72 different clinical trials on potential pharmacological treatments for Alzheimer’s disease and related dementias that take many different treatment approaches.135 More broadly, as of January 2024, there were 171 clinical trials assessing 134 drugs for treating Alzheimer’s disease (funded by both the public and private sectors). One expert has argued that this level of drug development is much less than the level of development for cancer drugs.136 It may take decades to see the full scientific and medical impact of recent NIH investments in Alzheimer’s disease and related dementias research. New drugs for any given disease generally build upon a body of science that takes decades to fully develop and then translate to practical application. According to a 2015 study, some of the first drugs approved to treat symptoms associated with Alzheimer’s disease were approved 22 years after the body of science underpinning those drugs became established.137 For the recent FDA-approved drugs that target amyloid, the body of science underlying those technologies became established in the early 2000s, fitting the same general trend with the related drug approvals occurring in 2022 to 2024.138 21st Century Cures Act Innovation Projects The 21st Century Cures Act (P.L. 114-255; the Cures Act), enacted in December 2016, authorized $4.8 billion for NIH for four specific innovation projects over a 10-year period (FY2017FY2026), with varying amounts allocated each fiscal year (see Table 2). The Cures Act established the “NIH Innovation Account,” to which specified amounts were transferred for each of FY2017 through FY2026 (see Table 2) for the purpose of carrying out the following four NIH Innovation Projects, with funds made available in subsequent appropriations acts: • The All of Us Research Program ($1.5 billion for FY2017 through FY2026), which aims to collect clinical, environmental, lifestyle, and genetic data from a large patient cohort over many years—with a goal of recruiting over 1 million 134 National Academies of Sciences, Engineering, and Medicine, “Preventing and Treating Alzheimer’s Disease and Related Dementias: Promising Research and Opportunities to Accelerate Progress: Proceedings of a Workshop–in Brief,” Washington, DC, 2024, https://nap.nationalacademies.org/catalog/27784/preventing-and-treating-alzheimersdisease-and-related-dementias-promising-research-and-opportunities-to-accelerate-progress. 135 NIA, “NIA-Funded Active Alzheimer’s and Related Dementias Clinical Trials and Studies,” last updated March 2024, https://www.nia.nih.gov/research/ongoing-AD-trials. 136 National Academies of Sciences, Engineering, and Medicine, “Preventing and Treating Alzheimer’s Disease and Related Dementias: Promising Research and Opportunities to Accelerate Progress: Proceedings of a Workshop–in Brief,” Washington, DC, 2024, https://nap.nationalacademies.org/catalog/27784/preventing-and-treating-alzheimersdisease-and-related-dementias-promising-research-and-opportunities-to-accelerate-progress. 137 Jennifer M. Beierlein, Laura M. McNamee, Michael J. Walsh et. al, “Patterns of Innovation in Alzheimer’s Disease Drug Development: A Strategic Assessment Based on Technological Maturity,” Clinical Therapeutics, vol. 37, no. 8 (August 1, 2015). 138 Jennifer M. Beierlein, Laura M. McNamee, Michael J. Walsh et. al, “Patterns of Innovation in Alzheimer’s Disease Drug Development: A Strategic Assessment Based on Technological Maturity,” and email communication with Dr. Fred Ledley, one of the study authors in September 2024. Congressional Research Service 32 The National Institutes of Health (NIH): Background and Congressional Issues • • • participants139 (formerly named the Precision Medicine Initiative Cohort Program).140 The Brain Research through Advancing Innovative Neurotechnologies (BRAIN) Initiative ($1.5 billion for FY2017 through FY2026), which involves developing and implementing new technology to understand how individual cells and the neural circuits they form interact in time and space—scientific understanding that may help treat, cure, or prevent brain-related disorders.141 The Beau Biden Cancer Moonshot ($1.8 billion for FY2017 through FY2023), which began in 2016 and sought to make a decade’s worth of progress in preventing and treating cancer in just five years.142 The Regenerative Medicine project ($30 million for FY2017 through FY2020), which supported clinical research using adult stem cells in coordination with FDA.143 To date, amounts authorized for the Innovation Projects shown in Table 2 have been fully appropriated. Table 2. Authorization of Appropriations for NIH Innovation Projects Under the Cures Act Millions of dollars Fiscal Year 2017 2018 2019 2020 2021 2022 2023 2024 2025 2026 Total PMI/All of US 40 100 186 149 109 150 419 235 BRAIN 10 86 115 140 100 152 450 172 36 91 31 1,455 195 1,511 Cancer Moonshot 300 300 400 195 195 194 216 Regenerative Medicine 2 10 10 8 Total Innovation Account 352 496 711 492 404 496 1,085 407 127 1,800 30 226 4,796 Source: P.L. 114-255, Section 1001(b)(4). 139 NIH, Implementation of Funding Plan for the NIH Innovation Projects Under the 21 st Century Cures Act, https://www.nih.gov/sites/default/files/research-training/initiatives/nih-cures-innovation-plan.pdf, and NIH, “About,” All of Us Research Program, https://allofus.nih.gov/about. 140 NIH, “PMI Cohort Program announces new name: the All of Us Research Program,” October 13, 2016, https://allofus.nih.gov/news-events/announcements/pmi-cohort-program-announces-new-name-all-us-researchprogram. 141 NIH, Implementation of Funding Plan for the NIH Innovation Projects Under the 21 st Century Cures Act, https://www.nih.gov/sites/default/files/research-training/initiatives/nih-cures-innovation-plan.pdf, and NIH, The BRAIN Initiative, “Overview,” https://braininitiative.nih.gov/about/overview. 142 National Cancer Institute, “History of the Cancer Moonshot,” December 2023, https://www.cancer.gov/research/ key-initiatives/moonshot-cancer-initiative/history. 143 NIH, Implementation of Funding Plan for the NIH Innovation Projects Under the 21 st Century Cures Act, https://www.nih.gov/sites/default/files/research-training/initiatives/nih-cures-innovation-plan.pdf. Congressional Research Service 33 The National Institutes of Health (NIH): Background and Congressional Issues Cures Act Innovation Project funding is unique from most of the funds NIH receives through the annual appropriations process. These funds are subject to different budget enforcement rules: for appropriated amounts to the account—up to the limit authorized for each fiscal year—the amounts are subtracted from any cost estimate for enforcing discretionary spending limits (i.e., the budget caps). In effect, appropriations to the NIH Innovation Account as authorized by the Cures Act are not subject to discretionary spending limits.144 Therefore, Congress does not need to consider the Innovation Account funds when determining the amount of NIH funding within the discretionary spending allocations. The f [Text truncated at 120,000 characters. The full text is on the page linked above.] --- Source: Frix Law Library, https://www.frixlaw.com/law-library/documents/crs%3AR41705. 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