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5 National Institutes of Health Autoimmune Disease Research Efforts
Pages 289-344

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From page 289...
... 1 The other 11 operating divisions are: Administration for Children and Families, Adminis tration for Community Living, Agency for Healthcare Research and Quality, Agency for Toxic Substances and Disease Registry, Center for Faith-Based and Neighborhood Partnerships, Centers for Disease Control and Prevention, Centers for Medicare & Medicaid Services, Food and Drug Administration, Health Resources and Services Administration, Indian Health Service, and Substance Abuse and Mental Health Services Administration (see HHS, 2022)
From page 290...
... 2016 to 2020, and the most recent plan covers FY 2021 to 2025 (NIH, 2021d) .4 The Act also changed guidance on reporting on NIH research activities from the earlier mandated biennial report to a triennial report, the most recent of which covered FY 2016 to 2018.5 Congress annually appropriates funds for NIH overall and the ICs specifically, and sometimes Congress qualifies funds with specific requirements or instructions (NIH, 2021e)
From page 291...
... 116-94) , available in the Congressional Record, vol.
From page 292...
... . NIH ICs have different missions that guide their activities, and may focus, for example, on a specific disease, organ system, life stage, field of science, or training or technology.
From page 293...
... . NOTE: B&F; Buildings and Facilities; FIC, Fogarty International Center; FY, Fiscal Year; IC, Institutes and Centers; NCATS, National Center for Advancing Translational Sciences; NCCIH, National Center for Complementary and Integrative Health; NCI, National Cancer Institute; NEI, National Eye Institute; NHGRI, National Human Research Institute; NHLBI, National Heart, Lung, and Blood Institute; NIA, National Institute of Aging; NIAAA, National Institute on Alcohol Abuse and Alcoholism; NIAID, National Institute of Allergy and Infectious Diseases; NIAMS, National Institute of Arthritis and Musculoskeletal and Skin Diseases; NIBIB, National Institute of Biomedical Imaging and Bioengineering; NICHD, Eunice Kennedy Shriver National Institute of Child Health and Human Development; NIDA, National Institute on Drug Abuse; NIDCD, National Institute on Deafness and Other Communication Disorders; NIDCR, National Institute of Dental and Craniofacial Research; NIDDK, National Institute of Diabetes and Digestive and Kidney Diseases; NIEHS, National Institute of Environmental Health Sciences; NIGMS, National Institute of General Medical Sciences; NIMH, National Institute of Mental Health; NIMHD, National Institute on Minority Health and Health Disparities; NINDS, National Institute on Neurological Disorders and Stroke; NINR, National Institute of Nursing Research; NLM, National Library of Medicine; OD, Office of the Director.
From page 294...
... , which are publicly available documents in which NIH describes its intentions to make discretionary awards for which it selects the awardees and/or the amounts of funding via a competitive process.7 FOAs can range from an NIH-wide umbrella announcement that channels investigator-initiated research applications, to a single IC's announcement to solicit applications for a clearly-defined scientific area in order to fulfill specific objectives8 (NIH Office of Extramural Research, 2021)
From page 295...
... . Chartered, also called standing or recurring, study sections review most investigator-initiated research applications; the sections comprise members who are "primarily non-federal scientists with expertise in relevant scientific disciplines and current research areas" (NIH, 2018)
From page 296...
... 296 ENHANCING NIH RESEARCH ON AUTOIMMUNE DISEASE FIGURE 5-3  NIH CSR review process. NOTE: CSR, Center for Scientific Review; IC, Institute and Center; SRG, Scientific Review Group.
From page 297...
... Chapter 6 discusses CSR study sections that review applications that focus on autoimmune diseases. The ICs use a similar process to review grant applications submitted in response to solicited FOAs, but they convene ad hoc special emphasis panels with expertise appropriate for each separate FOA (NIAID, 2020)
From page 298...
... ICs may propose their own initiatives with or without dedicated funding. NIH initiatives may also derive from and be coupled with private sector initiatives, with variable sharing of funds, Examples of the latter include large registries or clinical care consortia created by advocacy groups, strengthened in structure and resources by NIH or NIH-sponsored research, that pharmaceutical companies can use.
From page 299...
... , which are areas of focus for NIAMS and NIDDK, while others are not lethal but cause life disruption treatable with today's technologies, such as Hashimoto's thyroiditis and Graves' disease, a focus of NIDDK. The complexity and diversity in the group of autoimmune diseases has contributed to how research and other efforts to understand the diseases are dispersed across NIH ICs, which in turn may lead to siloing of efforts if there are no systematic attempts to coordinate activities across NIH.
From page 300...
... NIDDK, National Institute of Diabetes and Digestive and Kidney Diseases; NIAID, National Institute of Allergy and Infectious Diseases; NIAMS, National Institute of Arthritis and Musculoskeletal and Skin Diseases; NINDS, National Institute on Neurological Disorders and Stroke; NHLBI, National Heart, Lung, and Blood Institute; NCI, National Cancer Institute; NEI, National Eye Institute; NIDCR, National Institute of Dental and Craniofacial Research; NIGMS, National Institute of General Medical Sciences; NIEHS, National Institute of Environmental Health Sciences; NICHD, Eunice Kennedy Shriver National Institute of Child Health and Human Development; NHGRI, National Human Research Institute; NCATS, National Center for Advancing Translational Sciences; OD, Office of the Director.
From page 301...
... This lack of standardization across ICs and consistency in the release of strategic plans for individual ICs has important implications since it may pose an obstacle for ICs to coordinate their strategic planning efforts, and subsequently, their funding and research efforts for autoimmune diseases. While the timeframe covered by some of the strategic plans overlap, the plans are at various stages of implementation.
From page 302...
... prevention of Psoriatic Arthritis) ; Inflammatory clinical trials and arthritis and Myopathies; Scleroderma; observational musculoskeletal Sjögren's Syndrome; studies, and and skin diseases; Antiphospholipid Syndrome; and mechanistic the training of Vasculitis (Giant Cell Arteritis, studies with basic and clinical ANCA-Associated Vasculitis, a focus on scientists to Granulomatosis with Polyangitis, mechanisms of carry out this and Behçet's disease)
From page 303...
... . steady and diverse pool of talented new investigators; fostering exceptional research training and mentoring opportunities; and ensuring that science based health information reaches patients, their families, health care providers, and the public through communications and outreach" continued PREPUBLICATION COPY -- Uncorrected Proofs
From page 304...
... . of immune mechanisms to develop immunotherapies; and the use of improved animal models; and support clinical trial networks and investigator initiated clinical trials to evaluate immune-based treatment, such as cellular therapies, tolerance approaches, and other courses of action, each with integrated mechanistic studies (McNamara, 2021)
From page 305...
... National Heart, "The mission of Vasculitis – Immune Supports basic, Lung and the NHLBI is to Thrombocytopenia; Autoimmune translational and Blood Institute provide global hemolytic anemia; Autoimmune clinical research (NHLBI) leadership for a thrombocytopenic purpura; in the causes research, training, coexisting diseases of and coexisting and education systemic SLE (Atherosclerosis; diseases of program to Hypertension; Platelet autoimmune promote the activity)
From page 306...
... 306 ENHANCING NIH RESEARCH ON AUTOIMMUNE DISEASE TABLE 5-3 Continued Types of Research Supported for Autoimmune Diseases Relevant Autoimmune IC Name Mission to Missiona Diseases National Eye The mission of the Autoimmune uveitis; Supports basic, Institute (NEI) NEI is to conduct autoimmune retinopathy; translational, and support autoimmune dry eye diseases and clinical research, training, (DED)
From page 307...
... . "advance Health Sciences the environment research on (NIEHS)
From page 308...
... . National Cancer The mission of Supports Institute (NCI)
From page 309...
... The Institute also supports research in certain clinical areas, primarily those that affect multiple organ systems. To assure the vitality and continued productivity of the research enterprise, NIGMS provides leadership in training the next generation of scientists, enhancing the diversity of the scientific workforce, reducing disparities, and developing research capacities throughout the country" (NIGMS, 2021)
From page 310...
... . National "The mission of Supports Institute of the NICHD is research of Child Health to lead research of immune and Human and training to tolerance in Development understand human reproduction and (NICHD)
From page 311...
... A blank cell indicates that while the IC's mission may not focus on autoimmune diseases specifically, its research may benefit autoimmune disease research indirectly or may involve autoimmune diseases as a result of its broad mission. PREPUBLICATION COPY -- Uncorrected Proofs
From page 312...
... The various strategic plans reference autoimmune diseases and issues of autoimmunity to varying degrees or not at all, as the discussion below notes for each individual IC. The institutes that funded the most autoimmune diseases research and whose missions and focus align directly with autoimmune diseases -- NIAMS, NIDDK, and NIAID -- were more likely to mention the autoimmune diseases in their strategic plan.
From page 313...
... and the Autoimmune Diseases Coordinating Committee (ADCC)
From page 314...
... . NIAID The most recent NIAID-wide strategic plan is from 2017, and development of new one is not yet scheduled.11 The budget and planning page of NIAID's website12 does include strategic plans for various diseases and conditions and research areas, such as COVID-19, hepatitis B, tickborne disease research, vaccine adjuvants, and tuberculosis (NIAID, 2021b)
From page 315...
... . While the strategic plan does not mention specific autoimmune diseases, NINDS seeks to advance research on the basic science and mechanisms of nervous system function in order to advance efforts to prevent and treat neurological disorders.
From page 316...
... This critical challenge was the only explicit reference to autoimmune diseases in the strategic plan. The critical challenges, along with a set of compelling questions, constitute NHLBI's strategic research priorities (NHLBI, 2016)
From page 317...
... In addition, NIEHS's goals for advancing environmental health sciences include supporting research on the effects of the environment on biological systems and processes, which may have implications for many diseases, including autoimmune diseases (NIEHS, 2018)
From page 318...
... NIGMS has supported some autoimmune diseases-focused research, so its goals may have relevance for the field of autoimmune diseases. NIDCR NIDCR released an RFI in August 2019 to solicit stakeholder input for its 2020 to 2025 strategic plan, but decided to postpone the plan release following the retirement of the former director in December 2019 and pending the installation of a new director who would provide new leadership and direction for the institute (NIDCR, 2021b)
From page 319...
... The report is organized around five themes, which include an emphasis on understanding the biological basis of development; promoting gynecologic, andrologic, and reproductive health; promoting healthy pregnancies and lifelong wellness; improving the health of children and adolescents as they transition into adulthood; and safe, effective therapies and devices for pregnant women, children, and individuals with disabilities. While the strategic plan does not focus on specific diseases or conditions, the plan mentions how the results from NICHD-funded researchers working with maternal mouse models that showed the persistence of fetal cells post-delivery could offer insight into diseases that affect women, including autoimmune diseases (NICHD, 2019)
From page 320...
... CONGRESSIONAL ACTIONS AND NIH AUTOIMMUNE DISEASE RESEARCH On numerous occasions, Congress has issued mandates pertaining to autoimmune disease research at NIH. Constituents, advocacy groups, and other stakeholders who request congressional action on autoimmune diseases research have contributed greatly to such action, often through organized advocacy efforts (Autoimmune Association, 2021; Keenan, 2020)
From page 321...
... Annual funding for the program has grown over time from $30 million to the current level of $150 million.14 The Children's Health Act of 200015 called for the NIH Director to "expand, intensify, and coordinate research and other activities involving autoimmune diseases among the institutes." It also requested establishing an Autoimmune Diseases Coordinating Committee (ADCC) to coordinate activities across NIH and with other federal health programs.
From page 322...
... In addition, the Act directed NIDDK to conduct or support long-term epidemiology studies in those who have or who are at risk for type 1 diabetes in order examine the causes, characteristics, and complications of the disease, and to undertake an effort to support regional clinical research centers for preventing, detecting, treating, and curing juvenile arthritis and related conditions. NIH was to join HHS in a national effort to increase "research and development of prevention strategies, including consideration of vaccine development, coupled with appropriate ability to evaluate the effectiveness of such strategies in large clinical trials of children and young adults." NIDDK was to be appropriated sums for FY 2001 through 2005, but the Act did not specify what those sums should be.
From page 323...
... The Office was appropriated "such sums as already have been appropriated for FY 2002, and $4,000,000 for each of the FY 2003 through 2006" for the purpose of cooperative agreements or grants for the regional centers of excellence for rare diseases. Congress enacted the Pancreatic Islet Cell Transplantation Act of 200418 to "increase the supply of pancreatic islet cells for research, to provide for better coordination of Federal efforts and information on islet cell transplantation." Annual reports prepared by the NIDDK-led Diabetes Mellitus Interagency Coordinating Committee were directed to evaluate the federal activities and programs related to pancreatic islet transplantation and research, recommend legislation to increase the supply of pancreas tissue for islet cell transplantation, and address the adequacy of federal funding.
From page 324...
... In 2018, the National Clinical Care Commission Act22 established an HHS Commission, which included NIH as a member, to evaluate and make recommendations on how to better leverage and coordinate federal programs related to as many as two metabolic or autoimmune diseases with insulin-related etiology. In making its recommendations, the Act instructed the Commission to consider preventing and reducing the diseases and complications, "gaps in Federal efforts to support clinicians in providing integrated high-quality care," "improvement in and improved coordination of Federal education and awareness activities related to the prevention and treatment of the diseases," methods for dissemination of educational materials, and opportunities for consolidating overlap in federal programs related to the diseases.
From page 325...
... The Director of the Office was also to promote coordination and cooperation among the ICs involved in autoimmune disease research and those supported by their research; promote NIH allocation of resources for such research; annually report on the research, including identifying projects that should be conducted or supported; and serve as the principal advisor to HHS, NIH, CDC, and FDA and other agencies regarding autoimmune diseases. The 1999 bill authorized a FY 2000 appropriation of $950,000 (20 percent more than that of the 1998 bill)
From page 326...
... in 1998 in response to congressional interest in the autoimmune diseases and the need to coordinate activities across the ICs and with other federal health programs and activities for autoimmune diseases (ADCC, 2000) .35 The inaugural ADCC membership consisted of representatives of the ICs that are engaged in autoimmune diseases research, and members from other federal departments and agencies that focus on autoimmune diseases, such as CDC, Veterans Administration, and FDA.36 In addition, although Congress did not require it, the ADCC extended membership invitations to advocacy groups and representatives from 31 Prevention, Awareness, and Research of Autoimmune Disease Act of 2006, HR 6214, 109th Cong., 2nd sess., Congressional Record 152, no.
From page 327...
... was an autoimmune diseases research plan that had been requested by Congress, and it was informed by an expert panel that identified priority areas for autoimmune diseases research and considerations for implementing the research plan. The research plan had four major areas of emphasis: • "reducing the burden of disease; • enhancing understanding of disease etiology; 37 Personal communication.
From page 328...
... The field of autoimmune disease research at NIH has expanded over time, yet the amount of reporting 39Expressing the sense of the House of Representatives with respect to the level of funding provided to the National Institutes of Health for carrying out the Autoimmune Diseases Research Plan., H.Res.610, 108th Cong. 2nd sess.
From page 329...
... In addition, in FY 2021, NIAID is "developing a Notice of Special Interest to encourage investigator-initiated celiac disease research." The ADCC is now using the process it used for celiac disease to explore other gaps and topics of interest to the community.41 An earlier public meeting held in April 2019 introduced the Accelerating Medicines Partnerships (AMP) , a collaborative initiative for autoimmune diseases sponsored by NIAMS, NIAID, and other non-NIH partners (ADCC, 2019)
From page 330...
... Daniel Kastner, MD, PhD, Scientific Director, Division of Intramural Research Head, Inflammatory Disease Section, National Human Genome Research Institute, NIH, August 30, 2021. 44 Personal communication.
From page 331...
... The Offices release strategic plans, and the NIH triennial report to Congress describes Office activities. 45 Available at https://www.niams.nih.gov/about/working-groups/lupus-federal/ac tion-plan (accessed December 6, 2021)
From page 332...
... . 50 Website available at https://www.citisletstudy.org/what.html (accessed December 2, 2021)
From page 333...
... . 51 Website available at https://www.autoimmunitycenters.org/mission.php (accessed December 2, 2021)
From page 334...
... • Description: This Office facilitates and coordinates NIH-wide activities that involve research for a broad range of rare diseases, including autoimmune diseases. Duties of the Office include:  Developing and maintaining a centralized database on rare diseases.
From page 335...
...  ORWH  FDA (NIH, 2021b)  European Medicines Agency  Multiple biopharmaceutical and life science companies  Multiple nonprofit organizations • Description: The purpose of the AMP® and AMP® AIM pro gram is to establish disease teams that will focus on autoimmune and immune-mediated diseases, including rheumatoid arthritis, 54 Website available at https://www.rarediseasesnetwork.org/mgnet (accessed December 2, 2021)
From page 336...
... identify new biomarkers of tolerance and disease." ITN conducts research on organ and cellular transplantation, autoimmune diseases, allergy, asthma, and type 1 diabetes. ITN collaborates with academic, government, and industry leaders to develop and fund cutting edge immune tolerance research.
From page 337...
... Currently, 12 of 24 ICs provide the majority of funding to support extramural investigator-initiated autoimmune disease research, and 11 of 24 ICs provide funding to support intramural autoimmune disease research programs. Each IC has a different mission, and often has different research priorities and focuses on different autoimmune diseases than the other ICs.
From page 338...
... Paper read at NASEM Committee for the Assessment of NIH Research on Autoimmune Diseases: Meeting 3, March 4, Webinar. CIT (Clinical Islet Transplantation)
From page 339...
... Paper read at NASEM Committee for the Assessment of NIH Research on Autoimmune Diseases: Meeting 2 (Continuation) , February 1, 2021, Webinar.
From page 340...
... 2021a. Autoimmune Diseases Coordinating Committee member organizations.
From page 341...
... Bethesda, MD: Eunice Kennedy Shriver National Institute of Child Health and Human Development.
From page 342...
... 2021b. Notice of intent to publish a funding oppor tunity announcement for Accelerating Medicines Partnership Autoimmune and Immune-Me diated Diseases: Disease teams for rheumatoid arthritis, lupus, psoriatic spectrum diseases and Sjögren's syndrome (UC2 clinical trial optional)
From page 343...
... Paper read at NASEM Committee for the Assessment of NIH Research on Autoimmune Diseases: Meeting 3, March 4, 2021, Webinar. SGMRO (NIH Sexual & Gender Minority Research Office)
From page 344...
... PREPUBLICATION COPY -- Uncorrected Proofs


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