National Academies Press: OpenBook
Suggested Citation:"Front Matter." 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. doi: 10.17226/26349.
×

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

images

THE NATIONAL ACADEMIES PRESS
Washington, DC
www.nap.edu

Suggested Citation:"Front Matter." 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. doi: 10.17226/26349.
×

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 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

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. Envisioning a transformed clinical trials enterprise for 2030: Proceedings of a workshop. Washington, DC: The National Academies Press. https://doi.org/10.17226/26349.

Suggested Citation:"Front Matter." 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. doi: 10.17226/26349.
×

Image

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.

Suggested Citation:"Front Matter." 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. doi: 10.17226/26349.
×

Image

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.

Suggested Citation:"Front Matter." 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. doi: 10.17226/26349.
×

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.

Suggested Citation:"Front Matter." 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. doi: 10.17226/26349.
×

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.

Suggested Citation:"Front Matter." 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. doi: 10.17226/26349.
×

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

Page viii Cite
Suggested Citation:"Front Matter." 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. doi: 10.17226/26349.
×

This page intentionally left blank.

Suggested Citation:"Front Matter." 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. doi: 10.17226/26349.
×

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.

Suggested Citation:"Front Matter." 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. doi: 10.17226/26349.
×

This page intentionally left blank.

Suggested Citation:"Front Matter." 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. doi: 10.17226/26349.
×

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.

Suggested Citation:"Front Matter." 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. doi: 10.17226/26349.
×

This page intentionally left blank.

Suggested Citation:"Front Matter." 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. doi: 10.17226/26349.
×
Suggested Citation:"Front Matter." 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. doi: 10.17226/26349.
×

This page intentionally left blank.

Page xvii Cite
Suggested Citation:"Front Matter." 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. doi: 10.17226/26349.
×

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
Page xviii Cite
Suggested Citation:"Front Matter." 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. doi: 10.17226/26349.
×
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
Suggested Citation:"Front Matter." 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. doi: 10.17226/26349.
×
PageR1
Suggested Citation:"Front Matter." 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. doi: 10.17226/26349.
×
PageR2
Suggested Citation:"Front Matter." 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. doi: 10.17226/26349.
×
PageR3
Suggested Citation:"Front Matter." 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. doi: 10.17226/26349.
×
PageR4
Suggested Citation:"Front Matter." 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. doi: 10.17226/26349.
×
PageR5
Suggested Citation:"Front Matter." 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. doi: 10.17226/26349.
×
PageR6
Suggested Citation:"Front Matter." 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. doi: 10.17226/26349.
×
PageR7
Page viii Cite
Suggested Citation:"Front Matter." 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. doi: 10.17226/26349.
×
PageR8
Suggested Citation:"Front Matter." 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. doi: 10.17226/26349.
×
PageR9
Suggested Citation:"Front Matter." 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. doi: 10.17226/26349.
×
PageR10
Suggested Citation:"Front Matter." 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. doi: 10.17226/26349.
×
PageR11
Suggested Citation:"Front Matter." 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. doi: 10.17226/26349.
×
PageR12
Page xiii Cite
Suggested Citation:"Front Matter." 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. doi: 10.17226/26349.
×
PageR13
Suggested Citation:"Front Matter." 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. doi: 10.17226/26349.
×
PageR14
Suggested Citation:"Front Matter." 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. doi: 10.17226/26349.
×
PageR15
Suggested Citation:"Front Matter." 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. doi: 10.17226/26349.
×
PageR16
Page xvii Cite
Suggested Citation:"Front Matter." 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. doi: 10.17226/26349.
×
PageR17
Page xviii Cite
Suggested Citation:"Front Matter." 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. doi: 10.17226/26349.
×
PageR18
Next: 1 Introduction »
Envisioning a Transformed Clinical Trials Enterprise for 2030: Proceedings of a Workshop Get This Book
×
Buy Paperback | $30.00
MyNAP members save 10% online.
Login or Register to save!
Download Free PDF

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.

  1. ×

    Welcome to OpenBook!

    You're looking at OpenBook, NAP.edu's online reading room since 1999. Based on feedback from you, our users, we've made some improvements that make it easier than ever to read thousands of publications on our website.

    Do you want to take a quick tour of the OpenBook's features?

    No Thanks Take a Tour »
  2. ×

    Show this book's table of contents, where you can jump to any chapter by name.

    « Back Next »
  3. ×

    ...or use these buttons to go back to the previous chapter or skip to the next one.

    « Back Next »
  4. ×

    Jump up to the previous page or down to the next one. Also, you can type in a page number and press Enter to go directly to that page in the book.

    « Back Next »
  5. ×

    Switch between the Original Pages, where you can read the report as it appeared in print, and Text Pages for the web version, where you can highlight and search the text.

    « Back Next »
  6. ×

    To search the entire text of this book, type in your search term here and press Enter.

    « Back Next »
  7. ×

    Share a link to this book page on your preferred social network or via email.

    « Back Next »
  8. ×

    View our suggested citation for this chapter.

    « Back Next »
  9. ×

    Ready to take your reading offline? Click here to buy this book in print or download it as a free PDF, if available.

    « Back Next »
Stay Connected!