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Enhancing NIH Research on Autoimmune Disease (2022)

Chapter:Front Matter

Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2022. Enhancing NIH Research on Autoimmune Disease. Washington, DC: The National Academies Press. doi: 10.17226/26554.
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Below is the uncorrected machine-read text of this chapter, intended to provide our own search engines and external engines with highly rich, chapter-representative searchable text of each book. Because it is UNCORRECTED material, please consider the following text as a useful but insufficient proxy for the authoritative book pages.

Committee for the Assessment of NIH Research on Autoimmune Diseases Board on Population Health and Public Health Practice Health and Medicine Division A Consensus Study Report of PREPUBLICATION COPY—Uncorrected Proofs

THE NATIONAL ACADEMIES PRESS   500 Fifth Street, NW   Washington, DC 20001 This activity was funded with federal funds from the National Institutes of Health, U.S. Department of Health and Human Services, Contract/Task Order No. HHSN263201800029I/75N98020F00006. Any opinions, findings, conclusions, or recommendations expressed in this publication do not necessarily reflect the views of any organization or agency that provided support for the project. International Standard Book Number-13: 978-0-309-XXXXX-X International Standard Book Number-10: 0-309-XXXXX-X Digital Object Identifier: https://doi.org/10.17226/26554 Additional copies of this publication are available from the National Academies Press, 500 Fifth Street, NW, Keck 360, Washington, DC 20001; (800) 624-6242 or (202) 334-3313; http://www.nap.edu. Copyright 2022 by the National Academy of Sciences. All rights reserved. Printed in the United States of America Suggested citation: National Academies of Sciences, Engineering, and Medicine. 2022. Enhancing NIH research on autoimmune disease. Washington, DC: The National Academies Press. https://doi.org/10.17226/26554. PREPUBLICATION COPY—Uncorrected Proofs

The National Academy of Sciences was established in 1863 by an Act of Congress, signed by President Lincoln, as a private, nongovernmental institution to advise the nation on issues related to science and technology. Members are elected by their peers for outstanding contributions to research. Dr. Marcia McNutt is president. The National Academy of Engineering was established in 1964 under the charter of the National Academy of Sciences to bring the practices of engineering to advising the nation. Members are elected by their peers for extraordinary contributions to engineering. Dr. John L. Anderson is president. The National Academy of Medicine (formerly the Institute of Medicine) was established in 1970 under the charter of the National Academy of Sciences to advise the nation on medical and health issues. Members are elected by their peers for distinguished contributions to medicine and health. Dr. Victor J. Dzau is president. The three Academies work together as the National Academies of Sciences, Engineering, and Medicine to provide independent, objective analysis and advice to the nation and conduct other activities to solve complex problems and inform public policy decisions. The National Academies also encourage education and research, recognize outstanding contributions to knowledge, and increase public understanding in matters of science, engineering, and medicine. Learn more about the National Academies of Sciences, Engineering, and Medicine at www.nationalacademies.org. PREPUBLICATION COPY—Uncorrected Proofs

Consensus Study Reports published by the National Academies of Sciences, Engineering, and Medicine document the evidence-based consensus on the study’s statement of task by an authoring committee of experts. Reports typically include findings, conclusions, and recommendations based on information gathered by the committee and the committee’s deliberations. Each report has been subjected to a rigorous and independent peer-review process and it represents the position of the National Academies on the statement of task. Proceedings published by the National Academies of Sciences, Engineering, and Medicine chronicle the presentations and discussions at a workshop, symposium, or other event convened by the National Academies. The statements and opinions contained in proceedings are those of the participants and are not endorsed by other participants, the planning committee, or the National Academies. For information about other products and activities of the National Academies, please visit www.nationalacademies.org/about/whatwedo. PREPUBLICATION COPY—Uncorrected Proofs

COMMITTEE FOR THE ASSESSMENT OF NIH RESEARCH ON AUTOIMMUNE DISEASES BERNARD M. ROSOF (Chair), Professor, Department of Medicine, Donald and Barbara Zucker School of Medicine at Hofstra/ Northwell GLINDA S. COOPER, Director of Science and Research, Innocence Project1 DEIDRA C. CREWS, Professor of Medicine, Division of Nephrology, Department of Medicine, Deputy Director, Center for Health Equity, Johns Hopkins University School of Medicine WILLIAM R. DUNCAN, Vice Provost for Research Emeritus, East Tennessee State University DELISA FAIRWEATHER, Associate Professor of Medicine and Immunology, Director of Translational Research, Department of Cardiovascular Medicine, Mayo Clinic SONIA FRIEDMAN, Associate Professor of Medicine, Harvard Medical School; Director of Women’s Health, Crohn’s and Colitis Center, Brigham and Women’s Hospital LISA I. IEZZONI, Professor of Medicine, Harvard Medical School, Massachusetts General Hospital ANDREA M. KNIGHT, Associate Professor of Pediatrics, University of Toronto; Staff Physician, Division of Rheumatology, Hospital for Sick Children, Toronto; Adjunct Assistant Professor of Pediatrics, Perelman School of Medicine, University of Pennsylvania; Faculty Scholar, PolicyLab, Children’s Hospital of Philadelphia SCOTT M. LIEBERMAN, Associate Professor of Pediatrics, Division of Rheumatology, Allergy, and Immunology, Carver College of Medicine, University of Iowa MICHAEL D. LOCKSHIN, Professor of Medicine, Weill Cornell Medicine, Director, Barbara Volcker Center for Women and Rheumatic Disease, Hospital for Special Surgery JILL M. NORRIS, Professor and Chair, Department of Epidemiology, Colorado School of Public Health, University of Colorado Anschutz Medical Campus EMILY C. SOMERS, Professor and Epidemiologist, Departments of Internal Medicine, Environmental Health Sciences and Obstetrics & Gynecology; Michigan Institute for Clinical & Health Research (MICHR) Interdisciplinary Research and Team Science Faculty Lead, University of Michigan Schools of Medicine & Public Health 1 Director of Science and Research at the Innocence Project until November 2021. v PREPUBLICATION COPY—Uncorrected Proofs

BARBARA G. VICKREY, Professor and System Chair, Department of Neurology, Icahn School of Medicine at Mount Sinai Study Staff ROSE MARIE MARTINEZ, Study Director (from July 2021), Senior Board Director H. AWO OSEI-ANTO, Study Director (until July 2021) KRISTIN E. WHITE, Associate Program Officer DARA ROSENBERG, Research Associate (from November 2020) LEILA MEYMAND, Senior Program Assistant (October 2020– January 2022) GRACE READING, Senior Program Assistant (from January 2022) MISRAK DABI, Finance Business Partner Y. CRYSTI PARK, Administrative Assistant Consultants JOSEPH ALPER, Consulting Science Writer CHRIS BAROUSSE, Consulting Researcher NANCY DONOVAN, Consulting Advisor CHRISTINE LAGANA RIORDAN, Senior Director of Research & Evaluation, Ripple Effect vi PREPUBLICATION COPY—Uncorrected Proofs

Reviewers This Consensus Study Report was reviewed in draft form by indi- viduals chosen for their diverse perspectives and technical expertise. The purpose of this independent review is to provide candid and critical com- ments that will assist the National Academies of Sciences, Engineering, and Medicine in making each published report as sound as possible and to ensure that it meets the institutional standards for quality, objectivity, evidence, and responsiveness to the study charge. The review comments and draft manuscript remain confidential to protect the integrity of the deliberative process. We thank the following individuals for their review of this report: ANNE E. BURKE, Johns Hopkins University School of Medicine HERMINE I. BRUNNER, Cincinnati Children’s Hospital Medical Center DAVID A. HAFLER, Yale University JUDITH A. JAMES, Oklahoma Medical Research Foundation, University of Oklahoma Health Sciences Center SUE JINKS-ROBERTSON, Duke University FREDERICK W. MILLER, National Institutes of Health GARY W. MILLER, Columbia University Mailman School of Public Health vii PREPUBLICATION COPY—Uncorrected Proofs

viii REVIEWERS DAVID S. PISETSKY, Duke University & Durham VA Medical Center D. BRENT POLK, University of California San Diego MARIAN REWERS, Barbara Davis Center for Diabetes, University of Colorado School of Medicine TERRY J. SMITH, University of Michigan Medical School ROBERT B. WALLACE, The University of Iowa Although the reviewers listed above provided many constructive comments and suggestions, they were not asked to endorse the conclu- sions or recommendations of this report nor did they see the final draft before its release. The review of this report was overseen by BETTY A. DIAMOND, The Feinstein Institute for Medical Research, and ALAN I. LESHNER, American Association for the Advancement of Science (AAAS). They were responsible for making certain that an independent examina- tion of this report was carried out in accordance with the standards of the National Academies and that all review comments were carefully consid- ered. Responsibility for the final content rests entirely with the authoring committee and the National Academies. PREPUBLICATION COPY—Uncorrected Proofs

Preface As a practicing gastroenterologist, I treated patients who had Crohn’s disease and ulcerative colitis. Both are inflammatory bowel diseases (IBD) and among the most common autoimmune diseases. Over the last two decades, we have made progress in diagnosing IBD, but we have not yet identified biomarkers that enable us to screen for it. We have made revo- lutionary advances in targeting pharmaceutical therapies to manage IBD, but these options are still limited, many patients have refractory disease, and the therapies themselves may have significant side effects. There is currently no cure for IBD, so for much of their lives, patients may be at risk for developing additional autoimmune diseases as well as complica- tions such as cancer, infections, and maternal/fetal complications. During the course of this study, I was also reminded that we do not have reliable U.S. incidence and prevalence data for IBD. A goal of autoimmune disease research is to empower physicians to more accurately diagnose, treat, minimize, and ideally, cure disease. There is a critical research gap in personalized medicine, for example, the ability to measure a combination of individual genetics, serologies, phenotypes, and biological mechanisms that predict response to a specific therapy. Side effects and eventual failures, over time, of current medica- tions dictate a need for continuing research into disease pathogenesis and candidate therapeutics. Congressional legislation has often assisted in focusing National Institutes of Health (NIH) efforts to meet urgent demands related to the ix PREPUBLICATION COPY—Uncorrected Proofs

x PREFACE health and welfare of U.S. citizens. In 2019 Congress called for the NIH to contract with the National Academies of Sciences, Engineering, and Medicine to identify and review NIH’s research efforts in the broad area of autoimmune diseases with a particular emphasis on the risk factors, diagnostic tools, barriers to diagnosis, treatments and prospects for cure. Given the complexity of autoimmune diseases and the fact that they encompass many conditions, the committee’s expertise included clini- cians and researchers in numerous specialties that focus on autoimmune diseases as well as epidemiologists, health disparities researchers, and persons familiar with NIH’s research administration processes. NIH has conducted research that has contributed significantly to the advances in care of autoimmune disease, and it is important to continue to translate research knowledge into more precise diagnostic criteria and clinical interventions to achieve the best outcomes and benefit the lives of our patients. Progress in medical research requires visionary strategic thinking, the ability to meet constant challenges in disease prevention and therapeutics, new findings in genetics, coordination, and interdisciplinary guidance. The recommendations of the committee in chapter 7 are preceded by a thoughtful analysis of Institute and Center autoimmune disease research activity along with the committee findings and conclusions. The number and complexity of these diseases requires a concerted strategic effort that leverages the many research activities that occur across Institutes and Centers. The committee identifies opportunities and options for enhanc- ing autoimmune disease research at the NIH. The committee’s recommen- dations provide a basis for developing a strategy with metrics that yield data that can be reviewed periodically. It is our hope that a subsequent review would show advancement in autoimmune disease research and improvements in outcomes. On behalf of the committee, I would like express our thanks for the responsiveness of the NIH’s Institutes, Centers, and Offices in aiding the committee to gather information. A special thanks to Lisa Begg (OD/ ORWH), Susan Cooper (NIAID) and Ellen Goldmuntz (NIAID) for their guidance and support. I would also like to thank the committee members for their tremen- dous commitment and hard work, all the more meaningful in view of increased responsibilities both in their practices and their lives as a result of the pandemic, and the need to meet and work together virtually. On behalf of the committee, I would like to express our thanks and appreciation to the National Academies leadership and staff: Rose Marie Martinez, Senior Director of the Board on Population Health and Public PREPUBLICATION COPY—Uncorrected Proofs

PREFACE xi Health Practice, and acting Study Director; Kristin White, Associate Pro- gram Officer; Dara Rosenberg, Research Associate; Grace Reading, Senior Program Assistant. I would also like to thank former staff members Awo Osei-Anto, Senior Program Officer and Leila Meymand, Senior Program Assistant for their contributions to the study process. Bernard M. Rosof, M.D., Chair, Committee on the Assessment of NIH Research on Autoimmune Diseases PREPUBLICATION COPY—Uncorrected Proofs

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Contents ACRONYMS AND ABBREVIATIONS xix SUMMARY 1 PART I 1 INTRODUCTION 17 References, 26 2 BACKGROUND ON AUTOIMMUNE DISEASES 31 Defining Autoimmune Diseases, 31 Occurrence and Course of Autoimmune Diseases, 38 The Life-Course Framework and Autoimmune Diseases, 74 Summary and Research Implications, 76 References, 78 3 OVERVIEW OF SELECT AUTOIMMUNE DISEASES 95 Sjögren’s Disease, 97 Systemic Lupus Erythematosus, 105 Antiphospholipid Syndrome, 114 Rheumatoid Arthritis, 118 Psoriasis, 124 Inflammatory Bowel Disease, 128 Celiac Disease, 136 xiii PREPUBLICATION COPY—Uncorrected Proofs

xiv CONTENTS Primary Biliary Cholangitis, 143 Multiple Sclerosis, 150 Type 1 Diabetes, 158 Autoimmune Thyroid Diseases, 164 Summary and Research Implications, 169 References, 172 4 CROSSCUTTING ISSUES IN AUTOIMMUNE DISEASES 223 Commonalities Across Autoimmune Diseases, 223 Maximizing Advances in Technology, 254 Summary and Research Implications, 258 References, 261 PART II 5 NATIONAL INSTITUTES OF HEALTH AUTOIMMUNE DISEASE RESEARCH EFFORTS 289 NIH Overview and Organizational Structure, 289 NIH Research Process, 294 Congressional Actions and NIH Autoimmune Disease Research, 320 Current Coordination of Autoimmune Disease Research at NIH, 326 Summary, 337 References, 338 6 ANALYSIS OF INSTITUTE AND CENTER AUTOIMMUNE DISEASE RESEARCH ACTIVITY 345 Characteristics of Research Activities Supported by ICs, 349 IC Funding of Grants, 352 Research Grants by Autoimmune Disease, 364 Research Focus of Funded Grants, 378 Indicators of NIH Autoimmune Research Portfolio Accomplishment, 392 Summary, 406 References, 407 7 OPPORTUNITIES AND OPTIONS FOR ENHANCING RESEARCH AT NIH 411 Planning, Collaboration, and Innovation, 415 Recommendations, 433 References, 441 PREPUBLICATION COPY—Uncorrected Proofs

CONTENTS xv APPENDIXES A COMMITTEE MEMBER BIOGRAPHIES 443 B LIST OF AUTOIMMUNE DISEASES 451 C OPEN SESSION AGENDAS 455 D EPIDEMIOLOGY: SELECT DISEASES 461 E MISSION STATEMENTS OF NIH’S INSTITUTES AND CENTERS 485 F NIH OFFICE OF AUTOIMMUNE DISEASE RESEARCH ACT OF 1999 493 G ANALYSIS OF THE NIH AUTOIMMUNE RESEARCH GRANT PORTFOLIO: METHODOLOGY 499 H TOPIC ANALYSIS OF NIH AUTOIMMUNE RESEARCH GRANT ABSTRACTS 509 PREPUBLICATION COPY—Uncorrected Proofs

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Acknowledgments The committee and staff are grateful for the support of the National Academies of Sciences, Engineering, and Medicine’s Health and Medicine Division (HMD) staff who contributed to producing this report. The com- mittee and staff thank Tina Seliber, Lauren Shern, Leslie Sim, and Taryn Young in the HMD Executive Office; Anne Marie Houppert, Christopher Lao-Scott, Rebecca Morgan, in the National Academies Research Center; Sadaf Faraz, Devona Overton, and Marguerite Romatelli in the Office of the Chief Communications Officer; Stephanie Miceli in the Office of News and Public Information; Julie Eubank in the Office of Congressional and Government Affairs; and Mandy Enriquez, Thomas Holland, Dempsey Price, and Alejandro Velazquez, in the Office of Conference Management. The committee’s work was enhanced by the systematic analysis of Christine Lagana Riordan and her team from Ripple Effect, as well as the technical advice provided by Nancy Donovan who served as a consultant to the project. The committee thanks Chris Barousse for her assistance in conducting topic modeling of research grants. The committee thanks Joseph Alper for his editorial assistance in the production of this report. The committee also thanks Aimee Mead for her assistance in reviewing the report. The committee thanks all the speakers and moderators who partici- pated in committee meetings, as well as others who provided information, input, and assistance. They include the following: James M. Anderson, Karyl S. Barron, Lisa Begg, Jane Buckner, Stephanie Burrows, Susan Coo- per, Mary K. Crow, Cindy Danielson, Sanjoy Dutta, Julia Gichimu, Ellen xvii PREPUBLICATION COPY—Uncorrected Proofs

xviii ACKNOWLEDGMENTS Goldmuntz, Brian Haugen, Caren Heller, Deborah Hodge, Michael C. Humble, Judith A. James, Calvin Johnson, Daniel Kastner, Alex Keenan, Ruth Kirby, Virginia Ladd, Nicholas LaRocca, Jessica Lobo, Marie Man- cini, James McNamara, Sara Myers, John O’Shea, Mark Pedersen, Nishadi Rajapakse, Lisa G. Rider, Judy Riggie, Pamela L. Schwartzberg, Lisa Spain, Yaron Tomer, Ursula Utz, Emily von Scheven, and Victoria P. Werth. Finally, we give special thanks to the National Institutes of Health for generously funding this project. PREPUBLICATION COPY—Uncorrected Proofs

Acronyms and Abbreviations 25OHD 25-hydroxyvitamin D AARDA American Autoimmune Related Diseases Association1 ACE Autoimmunity Centers of Excellence ACF Administration for Children and Families ACL Administration for Community Living ACP American College of Physicians ACR American College of Rheumatology AD-ADRD Alzheimer’s Diseases and Alzheimer’s Disease Related Dementias ADCC NIH Autoimmune Diseases Coordinating Committee AHRQ Agency for Healthcare Research and Quality AI artificial intelligence AIDS acquired immune deficiency syndrome AIM autoimmune and immune-mediated diseases AIT autoimmune thyroiditis ALP alkaline phophatase ALPS autoimmune lymphoproliferative syndrome AMA antimitochondrial antibodies AMP® Accelerating Medicines Partnership® ANCA antineutrophil cytoplasmic antibodies 1 In 2021, AARDA changed its name to The Autoimmune Association. xix PREPUBLICATION COPY—Uncorrected Proofs

xx ACRONYMS AND ABBREVIATIONS Anti SSA/Ro anti Sjögren’s syndrome related antigen A autoantibodies Anti SSB/La anti Sjögren’s syndrome related antigen B autoantibodies APECED autoimmune polyendrocrinopathy candidiasis ectodermal dystrophy aPL antiphospholipid antibodies APS antiphospholipid antibody syndrome APS-ACTION Antiphospholipid Antibody Syndrome Alliance for Clinical Trials and International Networking AS ankylosing spondylitis ASBT apical sodium-dependent bile acid transport ASPE HHS Assistant Secretary for Planning and Evaluation ATD autoimmune thyroid disease ATSDR Agency for Toxic Substances and Disease Registry B&F Buildings and Facilities BA Budget Authority BARDA Biomedical Advanced Research and Development Authority BPA bisphenol A BRAIN Brain Research through Advancing Innovative Neurotechnologies Initiative BRIDA BACH2-Related Immunodeficiency and Autoimmunity CAPS catastrophic antiphospholipid syndrome CAR chimeric antigen receptor CARRA Childhood Arthritis and Rheumatology Research Alliance CC NIH Clinical Center CD Crohn’s disease CD40 cluster of differentiation 40 CDC Centers for Disease Control and Prevention CeD celiac disease CHOP Children’s Hospital of Philadelphia CIT Clinical Islet Transplantation Consortium CMS Centers for Medicare & Medicaid Services CNS central nervous system COPD chronic obstructive pulmonary disease COVID-19 coronavirus disease 2019 CRD Committee RePORTER Datasets CRS Congressional Research Service cSLE childhood-onset systemic lupus erythromatosus PREPUBLICATION COPY—Uncorrected Proofs

ACRONYMS AND ABBREVIATIONS xxi CSR Center for Scientific Review CVD cardiovascular disease DAIDS Division of AIDS Research DAISY Diabetes Autoimmunity Study in the Young DAIT Division of Allergy, Immunology, and Transplantation DCCT/EDIC Diabetes Control and Complications Trial/ Epidemiology of Diabetes Interventions and Complications DED dry eye disease DGP deamidated gliadin peptide DIPP Type 1 Diabetes Prediction and Prevention Study DNA deoxyribonucleic acid  DOC dental, oral, craniofacial DPCPSI Division of Program Coordination, Planning, and Strategic Initiatives EAE experimental autoimmune encephalomyelitis EAP Expanded Access Program EBV Epstein-Barr virus ELISA enzyme-linked immunoassay EmA immunoflourescent anti-edmysium EPA Environmental Protection Agency ERA Electronic Research Administration ESRD end-stage renal disease FDA Food and Drug Administration FGF fibroblast growth factor FIC Fogarty International Center FOA funding opportunity announcement FXR Farnesoid X Receptor FY fiscal year GGT gamma-glutamyl transferase HHS Department of Health and Human Services HIV human immunodeficiency virus HLA human leukocyte antigen  HRSA Health Resources and Services Administration HSS Hospital of Special Surgery IBD inflammatory bowel disease IC NIH Institute or Center PREPUBLICATION COPY—Uncorrected Proofs

xxii ACRONYMS AND ABBREVIATIONS ICD International Classification of Diseases iEdison Interagency Edison Database IFNs interferons Ig immunoglobulin IHS Indian Health Services IL interleukin IMSGC International Multiple Sclerosis Genetics Consortium IOM Institute of Medicine IRP National Institutes of Health Intramural Research Program ISN International Society of Nephrology ITN Immune Tolerance Network JAK janus kinase inhibitor JIA juvenile idiopathic arthritis JIF journal impact factor LDA latent dirichlet allocation LFA Lupus Foundation for America LFT liver function tests LHHS Labor, Health, and Human Services LFWG Lupus Federal Working Group MG myasthenia gravis MGNet Myasthenia Gravis Rare Disease Network MOG-Antibody myelin oligodendrocyte glycoprotein MRI magnetic resonance imaging mRNA messenger ribonucleic acid MS multiple sclerosis NAM National Academies of Medicine NASEM National Academies of Sciences, Engineering, and Medicine NCAPG National Coalition of Autoimmune Patient Groups NCATS National Center for Advancing Translational Sciences NCCIH National Center for Complementary and Integrative Health      NCI National Cancer Institute NEI National Eye Institute NHANES National Health and Nutrition Examination Survey NHGRI National Human Genome Research Institute NHLBI National Heart, Lung, and Blood Institute NIA National Institute on Aging PREPUBLICATION COPY—Uncorrected Proofs

ACRONYMS AND ABBREVIATIONS xxiii NIAID National Institute of Allergy and Infectious Diseases NIAAA National Institute on Alcohol Abuse and Alcoholism NIADAR National Institute of Autoimmune Disease and Autoimmunity Research NIAMS National Institute of Allergy and Infectious Diseases NIBIB National Institute of Biomedical Imaging and Bioengineering NICHD National Institute of Child Health and Human Development NIDA National Institute of Drug Abuse NIDCD National Institute of Deafness and Other Communications Disorders NIDCR National Institute of Dental and Craniofacial Research NIDDK National Institute of Diabetes and Digestive and Kidney Diseases NIEHS National Institute on Environmental Health Sciences NIGMS National Institute on General Medical Sciences  NIH National Institutes of Health 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 NLP natural language processing NMSS National Multiple Sclerosis Society NOD non-obese diabetic NORD National Organization of Rare Diseases norUDCA norursodeoxycholic acid NOSI notice of special interest NPP National Priorities Partnership NPSLE neuropsychiatric systemic lupus erythematosus NQF National Quality Forum NSAIDs nonsteroidal anti-inflammatory drugs OAD/AR Office of Autoimmune Disease/Autoimmunity Research OAR Office of AIDS Research OBSSR Office of Behavioral and Social Sciences Research OCA obeticholic acid OD Office of the Director OPA Office of Portfolio Analysis PREPUBLICATION COPY—Uncorrected Proofs

xxiv ACRONYMS AND ABBREVIATIONS OS Office of the Secretary ORWH Office of Research on Women’s Health PA program announcement pANCA perinuclear anti-neutrophil cytoplasmic antibody PBC primary biliary cholangitis (historically: primary biliary cirrhosis) PPAR peroxisome proliferated activated receptor PROMIS® Patient-Reported Outcomes Measurement Information System PROTECT Study Predicting Response to Standardized Pediatric Colitis Therapy PSC primary sclerosing cholangitis RA rheumatoid arthritis RCDC NIH Research, Condition, and Disease Categorization RCR relative citation ratio RCT randomized controlled trial RePORT Research Portfolio Online Reporting Tools RePORTER NIH Research Portfolio Expenditures and Results system RF rheumatoid factor RFA request for applications RFI request for information RFP request for proposals RHYTHM Rheumatoid Arthritis Study of the Myocardium RNA ribonucleic acid RPS Renal Pathology Society SAMHSA Substance Abuse and Mental Health Services Administration SARS-COV-2 severe acute respiratory syndrome Coronavirus 2 SEER surveillance, epidemiology, and end results SGMRO Sexual and Gender Minority Research Office sJIA systemic juvenile idiopathic arthritis SLE systemic lupus erythematosus SpA spondyloarthropathy SPIRES Scientific Publication Information Retrieval and Evaluation System SRG Scientific Review Group SRO Scientific Review Officer SSc systemic sclerosis PREPUBLICATION COPY—Uncorrected Proofs

ACRONYMS AND ABBREVIATIONS xxv ST suppression of tumorigenicity STTR small business technology transfer T1D type 1 diabetes TEDDY The Environmental Determinants of Diabetes in the Young Th t helper TLR toll-like receptor TNF tumor necrosis factor TPN total parenteral nutrition TRAPS tumor necrosis factor receptor-associated periodic syndrome tTG tissue transglutaminase tTGA tissue transglutaminase autoantibodies UC ulcerative colitis UDCA ursodeoxycholic acid  USPTO U.S. Patent and Trade Office VA Department of Veterans Affairs VEXAS vacuoles, E1 enzyme, X-linked, autoinflammatory and somatic syndrome WHO World Health Organization PREPUBLICATION COPY—Uncorrected Proofs

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Autoimmune diseases occur when the body's immune system malfunctions and mistakenly attacks healthy cells, tissues, and organs. Strong data on the incidence and prevalence of autoimmune diseases are limited, but a 2009 study estimated the prevalence of autoimmune diseases in the U.S. to be 7.6 to 9.4 percent, or 25 to 31 million people today. This estimate, however, includes only 29 autoimmune diseases, and it does not account for increases in prevalence in the last decade. By some counts, there are around 150 autoimmune diseases, which are lifelong chronic illnesses with no known cures. The National Academies of Sciences, Engineering, and Medicine was asked to assess the autoimmune disease research portfolio of the National Institutes of Health (NIH).

Enhancing NIH Research on Autoimmune Disease finds that while NIH has made impressive contributions to research on autoimmune diseases, there is an absence of a strategic NIH-wide autoimmune disease research plan and a need for greater coordination across the institutes and centers to optimize opportunities for collaboration. To meet these challenges, this report calls for the creation of an Office of Autoimmune Disease/Autoimmunity Research in the Office of the Director of NIH. The Office could facilitate NIH-wide collaboration, and engage in prioritizing, budgeting, and evaluating research. Enhancing NIH Research on Autoimmune Disease also calls for the establishment of long term systems to collect epidemiologic and surveillance data and long term studies (20+ years) to study disease across the life course. Finally, the report provides an agenda that highlights research needs that crosscut many autoimmune diseases, such as understanding the effect of environmental factors in initiating disease.

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