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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.
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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.
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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.
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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.
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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.
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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.
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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.
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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.
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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.
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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.
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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.
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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.
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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.
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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.
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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.
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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.
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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.
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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.
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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.

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 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 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. (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-XXXXX-X International Standard Book Number-10: 0-309-XXXXX-X 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. 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. 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. 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

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 rapporteur and the institution. 2 Affiliation listed at the time of the workshop. PREPUBLICATION COPY: UNCORRECTED PROOFS v

FORUM ON DRUG DISCOVERY, DEVELOPMENT, AND TRANSLATION12 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. 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 Diabetics 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 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 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. PREPUBLICATION COPY: UNCORRECTED PROOFS vii

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 PREPUBLICATION COPY: UNCORRECTED PROOFS viii

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. PREPUBLICATION COPY: UNCORRECTED PROOFS ix

Acknowledgments This Project was funded in whole or in part with federal funds from the Applicable Institute/Center, National Institutes of Health, U.S. Department of Health and Human Services, under Contract No. HHSN263201800029I. PREPUBLICATION COPY: UNCORRECTED PROOFS xi

Contents ACRONYMS AND ABBREVIATIONS xvii 1 INTRODUCTION 1 Defining Virtual Clinical Trials, 3 Organization of the Proceedings of a Workshop, 4 2 OPPORTUNITIES TO IMPROVE CLINICAL TRIALS 5 A Patient Perspective, 6 An Industry Perspective, 7 An Academic Perspective, 8 Discussion, 12 3 EXPLORING VIRTUAL CLINICAL TRIALS 15 Lessons Learned from Clinical Care, 16 Lessons Learned from Observational Studies, 17 Lessons Learned from Interventional Studies, 20 Panel Reactions, 25 Discussion, 27 4 ACCESS AND EQUITY 29 A Patient Perspective, 30 Recruitment for Clinical Trials, 31 Underserved Community Outreach and Engagement, 32 Lessons Learned from Behavioral Interventions, 35 Discussion, 38 5 POLICY CONSIDERATIONS 41 A Regulatory Perspective, 42 Clinical Trials Transformation Initiative: Decentralized Clinical Trials Project, 44 Privacy Protections for Virtual Clinical Trials, 46 Informed Consent for Passive Data Collection, 48 Discussion, 49 6 REFLECTIONS ON THE WORKSHOP AND POTENTIAL FUTURE DIRECTIONS 53 Session 1: Opportunities to Improve Clinical Trials, 53 Session 2: Exploring Clinical Trials, 53 Session 3: Access and Equity, 54 Session 4: Policy Considerations, 54 Next Steps, 55 REFERENCES 57 PREPUBLICATION COPY: UNCORRECTED PROOFS xiii

xiv CONTENTS APPENDIXES A WORKSHOP AGENDA 65 B WORKSHOP SPEAKER BIOGRAPHICAL SKETCHES 71 C EXAMPLES OF VIRTUAL CLINICAL TRIALS INCLUDED IN WORKSHOP HANDOUT 81 D VIRTUAL CLINICAL TRIALS PRESENTED BY SPEAKERS AT WORKSHOP 87 PREPUBLICATION COPY: UNCORRECTED PROOFS

Boxes, Figures, and Tables BOXES 1-1 Workshop Statement of Task, 3 3-1 Investigation of Genetics Data to Identify Drug Targets and Drug Response, 19 4-1 Facilitating Early Communication Between Community Organizations and Technology Developers: The Battle for Our Babies Campaign, 34 FIGURES 2-1 Drug discovery, development, and approval process, 9 2-2 Typology of clinical research based on location and methods of data captured, 10 3-1 Attrition of interested and eligible participants at each step of the REMOTE trial protocol, 21 4-1 Our Healthy Community model for community engagement, 34 4-2 Mobile phone and smart phone ownership trends across socioeconomic demographic groups, 36 4-3 Reasons for minority participation and non-participation in mHealth research, 37 5-1 CTTI’s Mobile Clinical Trials (MCTs), 44 TABLES 2-1 Current and Future Models for Clinical Trials, Categorized by Stages in Clinical Trials, 9 2-2 The Many Advantages of Virtual Clinical Trials, 12 PREPUBLICATION COPY: UNCORRECTED PROOFS xv

Acronyms and Abbreviations ALS amyotrophic lateral sclerosis CCPA California’s Consumer Protection 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 Rule 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 MCT mobile clinical trial NIH National Institutes of Health ONC The Office of the National Coordinator for Health Information Technology UNC13A Unc-13 Homolog A gene VA U.S. Department of Veterans Affairs PREPUBLICATION COPY: UNCORRECTED PROOFS xvii

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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.

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