National Academies Press: OpenBook
Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2019. Virtual Clinical Trials: Challenges and Opportunities: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25502.
×

Virtual Clinical Trials

CHALLENGES AND OPPORTUNITIES

PROCEEDINGS OF A WORKSHOP

Joe Alper, Eeshan Khandekar, 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. 2019. Virtual Clinical Trials: Challenges and Opportunities: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25502.
×

THE NATIONAL ACADEMIES PRESS 500 Fifth Street, NW Washington, DC 20001

This activity was supported by contracts between the National Academy of Sciences and AbbVie Inc.; American Society for Microbiology; Amgen Inc. (Contract No. GHCCOPS-CSARF-126570); Association of American Medical Colleges; AstraZeneca; Burroughs Wellcome Fund (Contract No. 1019001); Critical Path Institute; Eli Lilly and Company; FasterCures; Foundation for the National Institutes of Health; Friends of Cancer Research; GlaxoSmithKline (Contract No. OTH-PPL-30507); Johnson & Johnson; Merck & Co., Inc. (Contract No. CMO-180308-002499; MRLCPO-18-2641); National Institutes of Health (Contract No. HHSN263201800029I; Task Order No. HHSN26300007): National Cancer Institute, National Center for Advancing Translational Sciences, 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; Pfizer Inc.; Sanofi (Contract No. 38532911); Takeda Pharmaceuticals (Contract No. 53108); and U.S. Food and Drug Administration (Grant No. 5R13FD005496-03, 5R13FD005496-04): Center for Drug Evaluation and Research, Office of the Commissioner. 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: 978-0-309-49488-5
International Standard Book Number-10: 0-309-49488-5
Digital Object Identifier: https://doi.org/10.17226/25502

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 2019 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. 2019. Virtual clinical trials: Challenges and opportunities: Proceedings of a workshop. Washington, DC: The National Academies Press. doi: https://doi.org/10.17226/25502.

Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2019. Virtual Clinical Trials: Challenges and Opportunities: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25502.
×

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. C. D. Mote, Jr., 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. 2019. Virtual Clinical Trials: Challenges and Opportunities: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25502.
×

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. 2019. Virtual Clinical Trials: Challenges and Opportunities: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25502.
×

PLANNING COMMITTEE ON VIRTUAL CLINICAL TRIALS: CHALLENGES AND OPPORTUNITIES1,2

LINDA BRADY (Co-Chair), National Institute of Mental Health, National Institutes of Health

S. CLAIBORNE JOHNSTON (Co-Chair), Dell Medical School, The University of Texas at Austin

STEVEN ANDERSON, Center for Biologics Evaluation and Research, U.S. Food and Drug Administration

ROBERT M. CALIFF, Duke University and Verily Life Sciences

JOSH DENNY, Vanderbilt University

ROBERT GENTLEMAN, 23andMe

ROBERT GOODWIN, Pfizer Inc.

KATHY L. HUDSON, People-Centered Research Foundation

DAVID H. LEDBETTER, Geisinger

DAVID MCCALLIE, Cerner Corp.

REBECCA PENTZ, Emory University

LEONARD SACKS, Center for Drug Evaluation and Research, U.S. Food and Drug Administration

TODD SHERER, The Michael J. Fox Foundation for Parkinson’s Research

KELLY SIMCOX, Sanofi

JOHN WILBANKS, Sage Bionetworks

Project Staff

CAROLYN SHORE, Forum Director

REBECCA ENGLISH, Program Officer

EESHAN KHANDEKAR, Research Associate

MELVIN JOPPY, Senior Program Assistant

Consultant

JOE ALPER, Consulting 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.

2 Affiliation listed at the time of the workshop.

Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2019. Virtual Clinical Trials: Challenges and Opportunities: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25502.
×

This page intentionally left blank.

Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2019. Virtual Clinical Trials: Challenges and Opportunities: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25502.
×

FORUM ON DRUG DISCOVERY, DEVELOPMENT, AND TRANSLATION1,2

RUSS B. ALTMAN (Co-Chair), Stanford University

ROBERT M. CALIFF (Co-Chair), Duke University and Verily Life Sciences

CHRISTOPHER P. AUSTIN, National Center for Advancing Translational Sciences, National Institutes of Health

LINDA BRADY, National Institute of Mental Health, National Institutes of Health

TANISHA CARINO, FasterCures, Milken Institute

RICHARD DAVEY, National Institute of Allergy and Infectious Diseases, National Institutes of Health

JAMES H. DOROSHOW, National Cancer Institute, National Institutes of Health

JEFFREY M. DRAZEN, New England Journal of Medicine

STEVEN K. GALSON, Amgen Inc.

CARLOS GARNER, Eli Lilly and Company

JULIE L. GERBERDING, Merck & Co., Inc.

LYNN D. HUDSON, Critical Path Institute

JEFF HURD, AstraZeneca

S. CLAIBORNE JOHNSTON, Dell Medical School, The University of Texas at Austin

KATHARINE KNOBIL, GlaxoSmithKline

FREDA C. LEWIS-HALL, Pfizer Inc.

ALLISON MCELVAINE, American Diabetes Association

ROSS MCKINNEY, JR., Association of American Medical Colleges

JOSEPH P. MENETSKI, Foundation for the National Institutes of Health

BERNARD MUNOS, InnoThink Center for Research in Biomedical Innovation

MICHAEL SEVERINO, AbbVie Inc.

RACHEL E. SHERMAN, Office of the Commissioner, U.S. Food and Drug Administration

ELLEN V. SIGAL, Friends of Cancer Research

LANA R. SKIRBOLL, Sanofi

BRIAN L. STROM, Rutgers, the State University of New Jersey

AMIR TAMIZ, National Institute of Neurological Disorders and Stroke, National Institutes of Health

___________________

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.

2 Affiliation listed at the time of the workshop.

Page viii Cite
Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2019. Virtual Clinical Trials: Challenges and Opportunities: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25502.
×

PAMELA TENAERTS, Clinical Trials Transformation Initiative, Duke University

JOHN WAGNER, Takeda Pharmaceuticals

JOANNE WALDSTREICHER, Johnson & Johnson

CARRIE WOLINETZ, Office of Science Policy, National Institutes of Health

JANET WOODCOCK, Center for Drug Evaluation and Research, U.S. Food and Drug Administration

Forum on Drug Discovery, Development, and Translation Staff

CAROLYN SHORE, Forum Director

REBECCA ENGLISH, Program Officer

AMANDA WAGNER GEE, Program Officer

SYLVIA NCHA, Associate Program Officer

EESHAN KHANDEKAR, Research Associate

MELVIN JOPPY, Senior Program Assistant

ANDREW M. POPE, Director, Board on Health Sciences Policy

Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2019. Virtual Clinical Trials: Challenges and Opportunities: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25502.
×

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:

ANDREA CORAVOS, Elektra Labs

PENNY RANDALL, Central Nervous System Center of Excellence, IQVIA

Although the reviewers listed above provided many constructive comments and suggestions, they were not asked to endorse the content of this proceedings nor did they see the final draft before its release. The review of this proceedings was overseen by LESLIE BENET, University of California, San Francisco. 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. 2019. Virtual Clinical Trials: Challenges and Opportunities: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25502.
×

This page intentionally left blank.

Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2019. Virtual Clinical Trials: Challenges and Opportunities: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25502.
×

Acknowledgments

The support of the following people from the Health and Medical Division at the National Academies of Sciences, Engineering, and Medicine was essential for the planning and conduct of the workshop and for the development of the Proceedings of a Workshop: Rebecca English, Bettina Seliber, Lauren Shern, and Taryn Young. The assistance of Laura Penny, independent health and medical editor, was also essential for the editing of the proceedings.

The support of the sponsors of the Forum on Drug Discovery, Development, and Translation was crucial to the planning and conduct of the workshop Virtual Clinical Trials: Challenges and Opportunities and for the development of the proceedings.

This project was funded in whole or in part with federal funds from the National Institutes of Health, U.S. Department of Health and Human Services, under Contract No. HHSN263201800029I: National Cancer Institute, National Center for Advancing Translational Sciences, National Institute of Allergy and Infectious Diseases, National Institute of Mental Health, National Institute of Neurological Disorders and Stroke, and the Office of Science Policy; along with the U.S. Food and Drug Administration: Center for Drug Evaluation and Research and Office of the Commissioner.

Nonfederal sponsorship for this project was provided by AbbVie Inc., Amgen Inc., American Society for Microbiology, Association of American Medical Colleges, AstraZeneca, Burroughs Wellcome Fund, Critical Path Institute, Eli Lilly and Company, FasterCures, Foundation for the National

Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2019. Virtual Clinical Trials: Challenges and Opportunities: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25502.
×

Institutes of Health, Friends of Cancer Research, GlaxoSmithKline, Johnson & Johnson, Merck & Co., Inc., New England Journal of Medicine, Pfizer Inc., Sanofi, and Takeda Pharmaceuticals.

Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2019. Virtual Clinical Trials: Challenges and Opportunities: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25502.
×
Page xvii Cite
Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2019. Virtual Clinical Trials: Challenges and Opportunities: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25502.
×

Acronyms and Abbreviations

ALS amyotrophic lateral sclerosis
CCPA California’s Consumer Privacy Act
CTTI Clinical Trials Transformation Initiative
CYP2C19 Cytochrome P450 2C19 Enzyme
D2P direct-to-participant
EHR electronic health record
FDA U.S. Food and Drug Administration
GDPR Global Data Protection Regulation
GWAS genome-wide association study
HbA1c Hemoglobin A1c (glycated hemoglobin)
HiMSS Healthcare Information and Management Systems Society
HIPAA Health Insurance Portability and Accountability Act
ICD International Statistical Classification of Diseases and Related Health Problems
IRB Institutional Review Board
LRRK2 leucine-rich repeat kinase gene
Page xviii Cite
Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2019. Virtual Clinical Trials: Challenges and Opportunities: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25502.
×
MCT Mobile Clinical Trials
NIH National Institutes of Health
ONC The Office of the National Coordinator for Health Information Technology
PCSK9 Proprotein Convertase Substillsin/KexIn Type 9
UNC13A Unc-13 Homolog A gene
VA U.S. Department of Veterans Affairs
Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2019. Virtual Clinical Trials: Challenges and Opportunities: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25502.
×
Page R1
Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2019. Virtual Clinical Trials: Challenges and Opportunities: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25502.
×
Page R2
Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2019. Virtual Clinical Trials: Challenges and Opportunities: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25502.
×
Page R3
Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2019. Virtual Clinical Trials: Challenges and Opportunities: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25502.
×
Page R4
Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2019. Virtual Clinical Trials: Challenges and Opportunities: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25502.
×
Page R5
Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2019. Virtual Clinical Trials: Challenges and Opportunities: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25502.
×
Page R6
Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2019. Virtual Clinical Trials: Challenges and Opportunities: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25502.
×
Page R7
Page viii Cite
Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2019. Virtual Clinical Trials: Challenges and Opportunities: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25502.
×
Page R8
Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2019. Virtual Clinical Trials: Challenges and Opportunities: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25502.
×
Page R9
Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2019. Virtual Clinical Trials: Challenges and Opportunities: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25502.
×
Page R10
Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2019. Virtual Clinical Trials: Challenges and Opportunities: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25502.
×
Page R11
Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2019. Virtual Clinical Trials: Challenges and Opportunities: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25502.
×
Page R12
Page xiii Cite
Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2019. Virtual Clinical Trials: Challenges and Opportunities: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25502.
×
Page R13
Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2019. Virtual Clinical Trials: Challenges and Opportunities: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25502.
×
Page R14
Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2019. Virtual Clinical Trials: Challenges and Opportunities: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25502.
×
Page R15
Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2019. Virtual Clinical Trials: Challenges and Opportunities: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25502.
×
Page R16
Page xvii Cite
Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2019. Virtual Clinical Trials: Challenges and Opportunities: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25502.
×
Page R17
Page xviii Cite
Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2019. Virtual Clinical Trials: Challenges and Opportunities: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25502.
×
Page R18
Next: 1 Introduction »
Virtual Clinical Trials: Challenges and Opportunities: Proceedings of a Workshop Get This Book
×
Buy Paperback | $55.00 Buy Ebook | $44.99
MyNAP members save 10% online.
Login or Register to save!
Download Free PDF

Successful drug development relies on accurate and efficient clinical trials to deliver the best and most effective pharmaceuticals and clinical care to patients. However, the current model for clinical trials is outdated, inefficient and costly. Clinical trials are limited by small sample sizes that do not reflect variations among patients in the real world, financial burdens on participants, and slow processes, and these factors contribute to the disconnect between clinical research and clinical practice.

On November 28-29, the National Academies of Sciences, Engineering, and Medicine convened a workshop to investigate the current clinical trials system and explore the potential benefits and challenges of implementing virtual clinical trials as an enhanced alternative for the future. This publication summarizes the presentations and discussions from the workshop.

  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!