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Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2021. Envisioning a Transformed Clinical Trials Enterprise for 2030: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/26349.
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Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2021. Envisioning a Transformed Clinical Trials Enterprise for 2030: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/26349.
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Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2021. Envisioning a Transformed Clinical Trials Enterprise for 2030: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/26349.
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Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2021. Envisioning a Transformed Clinical Trials Enterprise for 2030: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/26349.
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Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2021. Envisioning a Transformed Clinical Trials Enterprise for 2030: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/26349.
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Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2021. Envisioning a Transformed Clinical Trials Enterprise for 2030: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/26349.
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Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2021. Envisioning a Transformed Clinical Trials Enterprise for 2030: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/26349.
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Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2021. Envisioning a Transformed Clinical Trials Enterprise for 2030: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/26349.
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Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2021. Envisioning a Transformed Clinical Trials Enterprise for 2030: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/26349.
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Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2021. Envisioning a Transformed Clinical Trials Enterprise for 2030: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/26349.
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Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2021. Envisioning a Transformed Clinical Trials Enterprise for 2030: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/26349.
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Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2021. Envisioning a Transformed Clinical Trials Enterprise for 2030: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/26349.
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Page xiii Cite
Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2021. Envisioning a Transformed Clinical Trials Enterprise for 2030: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/26349.
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Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2021. Envisioning a Transformed Clinical Trials Enterprise for 2030: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/26349.
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Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2021. Envisioning a Transformed Clinical Trials Enterprise for 2030: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/26349.
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Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2021. Envisioning a Transformed Clinical Trials Enterprise for 2030: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/26349.
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Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2021. Envisioning a Transformed Clinical Trials Enterprise for 2030: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/26349.
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Page xviii Cite
Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2021. Envisioning a Transformed Clinical Trials Enterprise for 2030: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/26349.
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Envisioning a Transformed Clinical Trials Enterprise for 2030 PROCEEDINGS OF A WORKSHOP Theresa Wizemann, Amanda Wagner Gee, and Carolyn Shore, Rapporteurs Forum on Drug Discovery, Development, and Translation Board on Health Sciences Policy Health and Medicine Division PREPUBLICATION COPY—Uncorrected Proofs

THE NATIONAL ACADEMIES PRESS   500 Fifth Street, NW   Washington, DC 20001 This activity was supported by contracts between the National Academy of ­Sciences and Amgen Inc.; Association of American Medical Colleges; ­AstraZeneca; Biogen; Biomedical Advanced Research and Development Authority; Burroughs Wellcome Fund (Contract No. 1021334); Critical Path Institute; Eli Lilly and Com- pany (Contract No. 4900709231); FasterCures, Milken Institute; Foundation for the National Institutes of Health; Friends of Cancer Research; Johnson & Johnson; Medable; Merck & Co., Inc. (Contract No. MRLCPO-21-138769); National Institutes of Health (Contract No. HHSN263201800029I; Task Order No. HHSN26300007): National Cancer Institute, National Institute of Allergy and Infectious Diseases, National Institute of Mental Health, National Institute of Neurological Disorders and Stroke, Office of Science Policy; New England Journal of Medicine; Sanofi (Con- tract No. 65873711); Takeda Pharmaceuticals; U.S. Food and Drug Administration (Grant No. 1R13FD007302-01). Any opinions, findings, conclusions, or recommen- dations 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/10.17226/26349 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 2021 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. 2021. Envisioning a transformed clinical trials enterprise for 2030: Proceedings of a workshop. Washington, DC: The National Academies Press. https://doi.org/10.17226/26349. 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 institu- tion 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 char- ter 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 typi- cally 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 opin- ions 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

ENVISIONING A TRANSFORMED CLINICAL TRIALS ENTERPRISE FOR 20301 STEVEN GALSON (Co-Chair), Retired ESTHER KROFAH (Co-Chair), FasterCures, Milken Institute AMY ABERNETHY, Verily Life Sciences ANITA ALLEN, University of Pennsylvania Carey School of Law CHRISTOPHER AUSTIN, Flagship Pioneering HOWARD BURRIS, Sarah Cannon LUTHER CLARK, Merck & Co., Inc. GISELLE CORBIE-SMITH, University of North Carolina School of Medicine M. KHAIR ELZARRAD, Center for Drug Evaluation and Safety, U.S. Food and Drug Administration JENNIFER GOLDSACK, Digital Medicine Society RICHARD MOSCICKI, PhRMA AMY PATTERSON, National Heart, Lung, and Blood Institute, National Institutes of Health JOSEPH SCHEEREN, Retired ANANTHA SHEKHAR, University of Pittsburgh PAMELA TENAERTS, Medable CHRISTOPHER YOO, Systems Oncology Health and Medicine Division Staff CAROLYN SHORE, Director, Forum on Drug Discovery, Development, and Translation AMANDA WAGNER GEE, Program Officer JULIE LIAO, Program Officer ANDREW MARCH, Associate Program Officer MELVIN JOPPY, Senior Program Assistant ANDREW M. POPE, Senior Director, Board on Health Sciences Policy Consultant THERESA WIZEMANN, Science Writer 1 The National Academies of Sciences, Engineering, and Medicine’s planning ­committees are solely responsible for organizing the workshop, identifying topics, and choosing ­speakers. The responsibility for the published Proceedings of a Workshop rests with the workshop rapporteurs and the institution. v PREPUBLICATION COPY—Uncorrected Proofs

FORUM ON DRUG DISCOVERY, DEVELOPMENT, AND TRANSLATION1 ROBERT M. CALIFF (Co-Chair), Verily Life Sciences GREGORY SIMON (Co-Chair), Kaiser Permanente Washington Health Research Institute and University of Washington CHRISTOPHER P. AUSTIN, Flagship Pioneering LINDA BRADY, National Institute of Mental Health, National Institutes of Health JOHN BUSE, University of North Carolina School of Medicine BARRY S. COLLER, The Rockefeller University THOMAS CURRAN, Children’s Mercy, Kansas City RICHARD DAVEY, National Institute of Allergy and Infectious Diseases, National Institutes of Health KATHERINE DAWSON, Biogen JAMES H. DOROSHOW, National Cancer Institute, National Institutes of Health JEFFREY M. DRAZEN, New England Journal of Medicine STEVEN K. GALSON, Retired CARLOS O. GARNER, Eli Lilly and Company JULIE L. GERBERDING, Merck & Co., Inc. DEBORAH HUNG, Harvard Medical School LYRIC JORGENSON, Office of Science Policy, National Institutes of Health ESTHER KROFAH, FasterCures, Milken Institute LISA M. LaVANGE, University of North Carolina LEANNE MADRE, Clinical Trials Transformation Initiative ROSS McKINNEY, JR., Association of American Medical Colleges JOSEPH P. MENETSKI, Foundation for the National Institutes of Health ARTI RAI, Duke University School of Law MARK ROGGE, Retired KLAUS ROMERO, Critical Path Institute KELLY ROSE, Burroughs Wellcome Fund SUSAN SCHAEFFER, Patients’ Academy for Research Advocacy JOSEPH SCHEEREN, Retired ANANTHA SHEKHAR, University of Pittsburgh JAY SIEGEL, Retired ELLEN V. SIGAL, Friends of Cancer Research LANA R. SKIRBOLL, Sanofi 1 The National Academies of Sciences, Engineering, and Medicine’s forums and roundtables do not issue, review, or approve individual documents. The responsibility for the p ­ ublished Proceedings of a Workshop rests with the workshop rapporteurs and the ­institution. vi PREPUBLICATION COPY—Uncorrected Proofs

AMIR TAMIZ, National Institute of Neurological Disorders and Stroke, National Institutes of Health ANN TAYLOR, AstraZeneca PAMELA TENAERTS, Medable JOANNE WALDSTREICHER, Johnson & Johnson ROBERT WALKER, Biomedical Advanced Research and Development Authority JONATHAN WATANABE, University of California, Irvine, School of Pharmacy and Pharmaceutical Sciences ALASTAIR WOOD, Vanderbilt University JANET WOODCOCK, U.S. Food and Drug Administration Forum Staff CAROLYN K. SHORE, Forum Director AMANDA WAGNER GEE, Program Officer ANDREW MARCH, Associate Program Officer MELVIN JOPPY, Senior Program Assistant ANDREW M. POPE, Senior Director, Board on Health Sciences Policy vii PREPUBLICATION COPY—Uncorrected Proofs

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Reviewers This Proceedings of a Workshop 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 comments that will assist the National Academies of Sciences, Engineer- ing, and Medicine in making each published proceedings as sound as possible and to ensure that it meets the institutional standards for quality, objectivity, evidence, and responsiveness to the charge. The review com- ments and draft manuscript remain confidential to protect the integrity of the process. We thank the following individuals for their review of this proceedings: BARBARA BIERER, Harvard Medical School BARRY COLLER, The Rockefeller University M. KHAIR ELZARRAD, U.S. Food and Drug Administration JOHN ORLOFF, Alexion (Retired) Although the reviewers listed above provided many constructive comments and suggestions, they were not asked to endorse the content of the proceedings nor did they see the final draft before its release. The review of this proceedings was overseen by ELI ADASHI, Brown Univer- sity. He was responsible for making certain that an independent exami- nation of this proceedings was carried out in accordance with standards of the National Academies and that all review comments were carefully considered. Responsibility for the final content rests entirely with the ­rapporteurs and the National Academies. ix PREPUBLICATION COPY—Uncorrected Proofs

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Acknowledgments Support from the sponsors of the Forum on Drug Discovery, Devel- opment, and Translation is crucial to support this and other work of the National Academies. The National Academies staff wish to express their gratitude to the speakers whose presentations helped inform workshop discussions on the state of drug research and development for adults across the older age span; to the members of the planning committee for their work in devel- oping the workshop agenda and shaping the discussions; and to National Academies staff without whom this workshop and the accounting thereof would not have been possible: Christie Bell, Robert Day, Sadaf Faraz, Eeshan Khandekar, Devona Overton, Esther Pak, Marguerite Romatelli, Bettina Seliber, Lauren Shern, and Taryn Young. xi PREPUBLICATION COPY—Uncorrected Proofs

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Contents ACRONYMS AND ABBREVIATIONS 1 INTRODUCTION Organization of the Workshop and Proceedings, 2 DEFINING THE VISION Envisioning a More Person-Centered and Inclusive Clinical Trials Enterprise, Envisioning an Optimized Clinical Trials Enterprise Through the Use of Technologies, Envisioning a More Resilient, Sustainable, and Transparent Clinical Trials Enterprise, 3 ENHANCING OUTCOMES IN A MORE PERSON-CENTERED AND INCLUSIVE CLINICAL TRIALS ENTERPRISE The Road to 2030: Perspectives from the Field, The Road to 2030: Visions of What Is Possible, Reflections on Achieving Person-Centered and Inclusive Trials, 4 PRACTICAL APPLICATIONS FOR TECHNOLOGY TO ENHANCE THE CLINICAL TRIALS ENTERPRISE The Road to 2030: Perspectives from the Field, The Road to 2030: Visions of What Is Possible, xiii PREPUBLICATION COPY—Uncorrected Proofs

xiv CONTENTS Reflections on Realizing the Potential of Technology in Clinical Trials, 5 BUILDING A MORE RESILIENT, SUSTAINABLE, AND TRANSPARENT CLINICAL TRIALS ENTERPRISE The Road to 2030: Perspectives from the Field, The Road to 2030: Visions of What Is Possible, Reflections on Resilience, Sustainability, and Transparency of the Clinical Trials Enterprise, 6 OPPORTUNITIES FOR TRANSFORMATION Opportunities to Transform the Clinical Trials Enterprise, Innovating for 2030 Now, Taking the Lessons from the COVID-19 Pandemic Response Forward, Closing Remarks, REFERENCES APPENDIXES A HEALTH AFFAIRS BLOG POSTS B SPEAKER AND MODERATOR BIOGRAPHIES C WORKSHOP AGENDA PREPUBLICATION COPY—Uncorrected Proofs

Boxes and Figures BOXES 1-1 Workshop Statement of Task, 5-1 Lessons from RECOVERY, FIGURES 2-1 One sponsor’s qualitative impression of the adoption and impact of remote approaches on the conduct of clinical trials during a pandemic, 5-1 Basic protocol design for the first phase of RECOVERY trial, 5-2 Complexity of community, xv PREPUBLICATION COPY—Uncorrected Proofs

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Acronyms and Abbreviations AAMC Association of American Medical Colleges ACTIV Accelerating COVID‑19 Therapeutic Interventions and Vaccines (NIH) CBER Center for Biologics Evaluation and Research (FDA) CDER Center for Drug Evaluation and Research (FDA) CEAL Community Engagement Alliance (NIH) CEO chief executive officer CMS Centers for Medicare & Medicaid Services CoVPN COVID-19 Prevention Network CTTI Clinical Trials Transformation Initiative EHR electronic health record FDA U.S. Food and Drug Administration FDASIA U.S. Food and Drug Administration Safety and Innovation Act GDPR General Data Protection Regulation HBCU Historically Black College and University HEAL Healthy Eating Active Living HRSA Health Resources and Services Administration xvii PREPUBLICATION COPY—Uncorrected Proofs

xviii ACRONYMS AND ABBREVIATIONS IRB institutional review board NCATS National Center for Advancing Translational Sciences NHS National Health Service (United Kingdom) NIH National Institutes of Health (United States) NIMHD National Institute on Minority Health and Health Disparities OMHHE Office of Minority Health and Health Equity PCORI Patient-Centered Outcomes Research Institute PDUFA Prescription Drug User Fee Act R&D research and development RECOVERY Randomized Evaluation of COVID-19 Therapy Trials PREPUBLICATION COPY—Uncorrected Proofs

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The evolution of health care is expanding the possibilities for integration of clinical research into the continuum of clinical care; new approaches are enabling the collection of data in real-world settings; and new modalities, such as digital health technologies and artificial intelligence applications, are being leveraged to overcome challenges and advance clinical research. At the same time, the clinical research enterprise is strained by rising costs, varying global regulatory and economic landscapes, increasing complexity of clinical trials, barriers to recruitment and retention of research participants, and a clinical research workforce that is under tremendous demands.

Looking ahead to 2030, the Forum on Drug Discovery, Development, and Translation of the National Academies of Sciences, Engineering, and Medicine convened a public workshop for stakeholders from across the drug research and development life cycle to reflect on the lessons learned over the past 10 years and consider opportunities for the future. The workshop was designed to consider goals and priority action items that could advance the vision of a 2030 clinical trials enterprise that is more efficient, effective, person-centered, inclusive, and integrated into the health care delivery system so that outcomes and experiences for all stakeholders are improved. This Proceedings of a Workshop summarizes the presentations and discussions that took place during the four-part virtual public workshop held on January 26, February 9, March 24, and May 11, 2021.

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