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
THE NATIONAL ACADEMIES PRESS
Washington, DC
www.nap.edu
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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 Company (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 (Contract No. 65873711); Takeda Pharmaceuticals; U.S. Food and Drug Administration (Grant No. 1R13FD007302-01). 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: 978-0-309-26928-5
International Standard Book Number-10: 0-309-26928-8
Digital Object Identifier: https://doi.org/10.17226/26349
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Suggested citation: National Academies of Sciences, Engineering, and Medicine. 2022. Envisioning a transformed clinical trials enterprise for 2030: Proceedings of a workshop. Washington, DC: The National Academies Press. https://doi.org/10.17226/26349.
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ENVISIONING A TRANSFORMED CLINICAL TRIALS ENTERPRISE FOR 20301
STEVEN GALSON (Co-Chair), Amgen Inc.
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 Inc.
CHRISTOPHER YOO, Systems Oncology
Health and Medicine Division Staff
CAROLYN K. 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.
FORUM ON DRUG DISCOVERY, DEVELOPMENT, AND TRANSLATION1
ROBERT M. CALIFF (Co-Chair), Duke University and Verily Life Sciences and Google Health
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, Amgen Inc.
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 published Proceedings of a Workshop rests with the workshop rapporteurs and the institution.
AMIR TAMIZ, National Institute of Neurological Disorders and Stroke, National Institutes of Health
ANN TAYLOR, AstraZeneca
PAMELA TENAERTS, Medable Inc.
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
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Reviewers
This Proceedings of a Workshop was reviewed in draft form by individuals 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, Engineering, 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 comments 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 University. He was responsible for making certain that an independent examination 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.
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Acknowledgments
Support from the sponsors of the Forum on Drug Discovery, Development, 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 developing 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.
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Contents
Organization of the Workshop and Proceedings
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
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
Taking the Lessons from the COVID-19 Pandemic Response Forward
Boxes and Figures
BOXES
1-1 Workshop Statement of Task
FIGURES
5-1 Basic protocol design for the first phase of the RECOVERY trial
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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 |
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 |