Innovation in Drug Research
and Development for
Prevalent Chronic Diseases
PROCEEDINGS OF A WORKSHOP
Andrew March, Amanda Wagner Gee, Robert Pool, 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
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, Inc.; 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: 978-0-309-09214-2
International Standard Book Number-10: 0-309-09214-0
Digital Object Identifier: https://doi.org/10.17226/26291
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. Innovation in drug research and development for prevalent chronic diseases: Proceedings of a workshop. Washington, DC: The National Academies Press. https://doi.org/10.17226/26291.
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PLANNING COMMITTEE ON INNOVATION IN DRUG RESEARCH AND DEVELOPMENT FOR PREVALENT CHRONIC DISEASES1
CARLOS O. GARNER (Co-Chair), Eli Lilly and Company
ANANTHA SHEKHAR (Co-Chair), University of Pittsburgh
MELINDA BUNTIN, Vanderbilt University School of Medicine
GRACE COLÓN, InCarda Therapeutics
BETTINA DRAKE, Washington University School of Medicine and Alvin J. Siteman Cancer Center
ALYSON KARESH, Center for Drug Evaluation and Research, U.S. Food and Drug Administration
CHRONIS MANOLIS, UPMC Health Plan
PHYLLIS PETTIT NASSI, Huntsman Cancer Institute, The University of Utah
HOWARD ROSEN, BonVelo Ventures and Stanford University
SUSAN SCHAEFFER, Patients’ Academy for Research Advocacy
AMIR TAMIZ, National Institute of Neurological Disorders and Stroke, National Institutes of Health
Health and Medicine Division Staff
CAROLYN SHORE, Director, Forum on Drug Discovery, Development, and Translation
AMANDA WAGNER GEE, Program Officer
LEAH CAIRNS, Program Officer
ANDREW MARCH, Associate Program Officer
MELVIN JOPPY, Senior Program Assistant
ANDREW M. POPE, Senior Director, Board on Health Sciences Policy
Consultant
ROBERT POOL, 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.
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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
AMY ABERNETHY, Office of the Commissioner, U.S. Food and Drug Administration
CHRISTOPHER P. AUSTIN, National Center for Advancing Translational Sciences, National Institutes of Health
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, Broad Institute of Massachusetts Institute of Technology and Harvard Medical School
ESTHER KROFAH, FasterCures, Milken Institute
LISA M. LAVANGE, University of North Carolina
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, Takeda Pharmaceuticals
KELLY ROSE, Burroughs Wellcome Fund
SUSAN SCHAEFFER, Patients’ Academy for Research Advocacy
JOSEPH SCHEEREN, Critical Path Institute
ANANTHA SHEKHAR, University of Pittsburgh
JAY SIEGEL, Retired
ELLEN V. SIGAL, Friends of Cancer Research
___________________
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.
LANA R. SKIRBOLL, Sanofi
AMIR TAMIZ, National Institute of Neurological Disorders and Stroke, National Institutes of Health
ANN TAYLOR, AstraZeneca
PAMELA TENAERTS, Clinical Trials Transformation Initiative
JOANNE WALDSTREICHER, Johnson & Johnson
ROBERT WALKER, Biomedical Advanced Research and Development Authority
JONATHAN WATANABE, University of California, Irvine, School of Pharmacy and Pharmaceutical Sciences
CARRIE WOLINETZ, Office of Science Policy, National Institutes of Health
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
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:
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 BRADFORD GRAY, Urban Institute (retired). 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 challenges and opportunities for spurring drug research and development for prevalent chronic diseases; to the members of the planning committee for their work in developing the workshop agenda and shaping the discussions; and to additional National Academies staff, without whom this workshop and the accounting thereof would not have been possible: Christie Bell, Robert Day, Sadaf Faraz, Devona Overton, Esther Pak, Marguerite Romatelli, Bettina Seliber, Lauren Shern, and Taryn Young.
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Contents
Organization of the Proceedings
2 PERSON-CENTERED DRUG RESEARCH AND DEVELOPMENT
Chronic Pain: A Patient’s Journey
The Importance of the Patient Voice
Including Underserved Populations
The Value of Research on Underserved Populations
3 NEW TECHNOLOGIES TO ENABLE RESEARCH IN PREVALENT CHRONIC DISEASE
New Innovations in Drug Discovery and Development
Holistic Approaches to Innovation
The National Institutes of Health BRAIN Initiative
Artificial Intelligence Applications for Drug Discovery
A Regulatory Perspective on New Technologies in Clinical Research
Chronic Obstructive Pulmonary Disease
Investing in One Disease, Applying to Multiple Diseases
6 LESSONS LEARNED FOR THE FUTURE
A Public–Private Partnership Perspective
7 REFLECTIONS AND FINAL THOUGHTS
Engaging Patients Throughout the Research Process
Considerations for Designing Clinical Trials
Boxes and Figures
BOXES
2-1 Models for Incorporating Patients’ Input in Research
4-1 Prevention Can Be Profitable
6-1 Minimizing the Total Cost of Care
FIGURES
2-1 Genomics is failing on diversity
3-1 AI-assisted drug discovery
5-1 Increasing risk with increasing number of metabolic syndrome criteria
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