Toward a Framework to Improve Diversity and Inclusion in Clinical Trials

Convened May 20, 2024

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Theresa Wizemann, Kyle Cavagnini, Alex Helman, and Carolyn Shore, Rapporteurs

Forum on Drug Discovery, Development, and Translation

National Cancer Policy Forum

Board on Health Sciences Policy

Health and Medicine Division

Proceedings of a Workshop

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

This project has been funded in whole or in part with contracts between the National Academy of Sciences and 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; National Institutes of Health Office of the Director; National Institutes of Health; and Department of Health and Human Services, under Contract No. HHSN263201800029I. Other federal funders include Centers for Disease Control and Prevention (Award No. 002396) and U.S. Food and Drug Administration (Award No. 001021). Other sponsors of this activity are American Association for Cancer Research; American Cancer Society (Award No. 001561); American College of Radiology; American Society of Clinical Oncology; Amgen, Inc.; Association of American Cancer Institutes; Association of American Medical Colleges; Association of Cancer Care Centers; AstraZeneca; Biogen; Burroughs Wellcome Fund (Award No. 002235); Critical Path Institute; Eli Lilly and Company; FasterCures, Milken Institute; Flatiron Health; Foundation for the National Institutes of Health; Johnson & Johnson (Award No. 002261); Medable; Merck & Co. (Award No. 002241 & 002242); National Comprehensive Cancer Network; National Patient Advocate Foundation; New England Journal of Medicine; Novartis (Award No. 002199); Oncology Nursing Society; Partners in Health; Pfizer Inc.; Sanofi; and the University of Illinois, Urbana-Champaign. 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: 978-0-309-73145-4
International Standard Book Number-10: 0-309-73145-3
Digital Object Identifier: https://doi.org/10.17226/28587

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Suggested citation: National Academies of Sciences, Engineering, and Medicine. 2025. Toward a framework to improve diversity and inclusion in clinical trials: Proceedings of a workshop. Washington, DC: The National Academies Press. https://doi.org/10.17226/28587.

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PLANNING COMMITTEE ON TOWARD A FRAMEWORK TO IMPROVE DIVERSITY AND INCLUSION IN CLINICAL TRIALS¹

FREDA LEWIS-HALL (Chair), Retired

BARBARA BIERER, Brigham and Women’s Hospital and Harvard Medical School

SILAS BUCHANAN, Institute for eHealth Equity

LUTHER CLARK, Merck & Co., Inc.

MARIANNE HAMILTON LOPEZ, Duke-Margolis Center for Health Policy

MORGAN HANGER, Clinical Trials Transformation Initiative

ESTHER KROFAH, Milken Institute

MARTIN MENDOZA, Centers for Medicare & Medicaid Services

CARLA RODRIGUEZ-WATSON, Reagan-Udall Foundation for the Food and Drug Administration

MICHELLE TARVER, Food and Drug Administration

ROBERT A. WINN, Virginia Commonwealth School of Medicine

Health and Medicine Staff

CAROLYN SHORE, Director, Forum on Drug Discovery, Development, and Translation

SHARYL NASS, Codirector, National Cancer Policy Forum; Senior Director, Board on Health Care Services

FRANCIS AMANKWAH, Codirector, National Cancer Policy Forum (from July 2024)

ERIN BALOGH, Codirector, National Cancer Policy Forum (until June 2024)

KYLE CAVAGNINI, Associate Program Officer

BRITTANY HSIAO, Associate Program Officer (until March 2024)

NOAH ONTJES, Associate Program Officer

JENNIFER ZHU, Associate Program Officer

EMMA WICKLAND, Research Associate

MELVIN JOPPY, Senior Program Assistant

CLARE STROUD, Senior Director, Board on Health Sciences Policy

Consultants

THERESA WIZEMANN, Science Writer

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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

GREGORY SIMON (Cochair), Kaiser Permanente Washington Health Research Institute; University of Washington

ANN TAYLOR (Cochair), Retired

BARBARA E. BIERER, Harvard Medical School; Brigham and Women’s Hospital

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

LUTHER T. CLARK, Merck & Co., Inc.

BARRY S. COLLER, The Rockefeller University

TAMMY R. L. COLLINS, Burroughs Wellcome Fund

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, Retired

CARLOS O. GARNER, Eli Lilly and Company

MORGAN HANGER, Clinical Trials Transformation Initiative

SALLY L. HODDER, West Virginia University

TESHEIA JOHNSON, Yale School of Medicine

LYRIC JORGENSON, Office of the Director, National Institutes of Health

ESTHER KROFAH, Milken Institute

LISA M. LaVANGE, University of North Carolina

ARAN MAREE, Johnson & Johnson

CRISTIAN MASSACESI, AstraZeneca

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

ANAEZE C. OFFODILE II, Memorial Sloan Kettering Center

HEATHER PIERCE, Association of American Medical Colleges

ARTI RAI, Duke University School of Law

KLAUS ROMERO, Critical Path Institute

JONI RUTTER, National Center for Advancing Translational Sciences, National Institutes of Health

SUSAN SCHAEFFER, The Patients’ Academy for Research Advocacy

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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.

ANANTHA SHEKHAR, University of Pittsburgh School of Medicine

MARK TAISEY, Amgen Inc.

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

PAMELA TENAERTS, Medable Inc.

JONATHAN WATANABE, University of California, Irvine School of Pharmacy and Pharmaceutical Sciences

ALASTAIR WOOD, Vanderbilt University

CRIS WOOLSTON, Sanofi

JOSEPH C. WU, Stanford University School of Medicine

Forum Staff

CAROLYN SHORE, Director, Forum on Drug Discovery, Development, and Translation

KYLE CAVAGNINI, Associate Program Officer

BRITTANY HSIAO, Associate Program Officer (until March 2024)

NOAH ONTJES, Associate Program Officer

MELVIN JOPPY, Senior Program Assistant

CLARE STROUD, Senior Director, Board on Health Sciences Policy

NATIONAL CANCER POLICY FORUM¹

ROBERT A. WINN (Chair), Virginia Commonwealth University

ROHIT BHARGAVA, Cancer Center at Illinois

SMITA BHATIA, The University of Alabama at Birmingham

GIDEON BLUMENTHAL, Merck

CHRIS BOSHOFF, Pfizer Inc.

OTIS W. BRAWLEY, Johns Hopkins University

CHRISTINA CHAPMAN, Baylor College of Medicine; Michael E. DeBakey VA Medical Center

GWEN DARIEN, Patient Advocate Foundation

CRYSTAL DENLINGER, National Comprehensive Cancer Network

JAMES H. DOROSHOW, National Cancer Institute

S. GAIL ECKHARDT, Baylor College of Medicine

CHRISTOPHER R. FRIESE, University of Michigan

STANTON L. GERSON, Case Western Reserve University

SCARLETT LIN GOMEZ, University of California, San Francisco

JULIE R. GRALOW, American Society of Clinical Oncology

ROY S. HERBST, Yale University; American Association for Cancer Research

HEDVIG HRICAK, Memorial Sloan Kettering Cancer Center

CHANITA HUGHES-HALBERT, University of Southern California

ROY A. JENSEN, University of Kansas; Association of American Cancer Institutes

RANDY A. JONES, University of Virginia

BETH Y. KARLAN, University of California, Los Angeles

SAMIR N. KHLEIF, Georgetown University; Society for Immunotherapy of Cancer

ELENA MARTINEZ, University of California, San Diego

LARISSA NEKHLYUDOV, Brigham and Women’s Hospital; Dana-Farber Cancer Institute; Harvard Medical School

CLEO A. RYALS, Flatiron Health

RICHARD L. SCHILSKY, ASCO TAPUR Study; University of Chicago

JULIE SCHNEIDER, Oncology Center of Excellence, U.S. Food and Drug Administration

SUSAN M. SCHNEIDER, Duke University

LAWRENCE N. SHULMAN, University of Pennsylvania

HEIDI SMITH, Novartis Pharmaceuticals

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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.

KATRINA TRIVERS, Centers for Disease Control and Prevention

ROBIN YABROFF, American Cancer Society

Forum Staff

SHARYL NASS, Codirector, National Cancer Policy Forum; Senior Director, Board on Health Care Services

FRANCIS AMANKWAH, Codirector, National Cancer Policy Forum (from July 2024)

ERIN BALOGH, Codirector, National Cancer Policy Forum (until June 2024)

LAURENE GRAIG, Senior Program Officer (June–July 2024)

JENNIFER ZHU, Associate Program Officer

EMMA WICKLAND, Research Associate

TORRIE BROWN, Program Coordinator

ANNA ADLER, Senior Program Assistant

JULIE WILTSHIRE, Senior Finance Business Partner

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 ELLEN CLAYTON, Vanderbilt University Medical Center. She 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

The Forum on Drug Discovery, Development, and Translation is grateful for the generous support of our sponsors: Amgen, Inc.; Association of American Medical Colleges; AstraZeneca; Biogen; Burroughs Wellcome Fund; Critical Path Institute; Eli Lilly and Company; FasterCures, Milken Institute; Foundation for the National Institutes of Health; Johnson & Johnson; Medable; Merck & Co., Inc.; 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; National Institutes of Health Office of the Director; New England Journal of Medicine; Sanofi; and the U.S. Food and Drug Administration.

The National Cancer Policy Forum is grateful for the support of our many annual sponsors. Federal sponsors include the Centers for Disease Control and Prevention and the National Cancer Institute/National Institutes of Health. Nonfederal sponsors include the American Association for Cancer Research; American Cancer Society; American College of Radiology; American Society of Clinical Oncology; Association of American Cancer Institutes; Association of Cancer Care Centers; Flatiron Health; Merck & Co. Inc; National Comprehensive Cancer Network; Patient Advocate Foundation; Novartis Oncology; Oncology Nursing Society; Partners In Health; Pfizer Inc; and Cancer Center at Illinois.

The National Academies staff wish to express their gratitude to the speakers whose presentations helped inform workshop discussions on improving diversity and inclusion in the national clinical trials enterprise; the members of the planning committee for their work in developing the

workshop agenda and shaping the discussions; and additional National Academies staff, without whom this workshop and the accounting thereof would not have been possible: Christie Bell, Lori Brenig, Samantha Chao, Robert Day, Amber McLaughlin, Marguerite Romatelli, and Taryn Young.

Contents

ACRONYMS AND ABBREVIATIONS

1 INTRODUCTION

Toward a National Action Plan

Organization of the Proceedings

2 COMMUNITY ENGAGEMENT AND INVESTMENT

Establishing Equitable Research Partnerships

Relationships and Reciprocity

Community Health Through Church Initiatives

Sustainable Community Engagement

A Role for Changemakers

Inclusive Research Beyond Enrollment

The Role of Faith-Based Organizations

Additional Strategies for Promoting Equitable Participation in Clinical Trials

3 DEFINING, COLLECTING, AND SHARING COMPREHENSIVE AND CONSISTENT DATA ON TRIAL DIVERSITY

ONC Initiatives to Support Diverse Clinical Trials

Categorizing Race and Ethnicity for Data Collection

Improving Race and Ethnicity Data Collection and Use

FDA Initiatives to Support Diverse Clinical Trials

Elements of Sustainable Change in Clinical Trial Diversity

Tools and Implementation

Data on Workforce and Provider Diversity

4 CLINICAL TRIAL SITE ENABLEMENT

The NCATS Clinical and Translational Science Award Program

Community Health Centers as Partners

Valuing Diverse Representation in Device Trials

Empowering Participants

Fostering Community Readiness to Conduct Trials at Scale

Becoming Trustworthy

Creating Sustainable Funding and Reimbursement Models

Sustainable Partnerships

5 AGENCY PERSPECTIVES: CMS, FDA, AND NIH

CMS

FDA and NIH in Conversation

6 CHALLENGING THE CLINICAL TRIAL ECOSYSTEM

A Patient Perspective on Trust and Access

Implementing FDA Authorities and Mandates

Public–Private Partnerships

Sponsors Building Community Capacity

Diversity in Device Clinical Trials

The Role of CMS Coverage in Health Equity

Making the Business Case for Diversity in Trials

Training Clinical Investigators and Trial Staff

Sustaining Improvements in Clinical Trial Diversity

Closing Remarks

REFERENCES

APPENDIXES

A Workshop Agenda

B Biographical Sketches of the Workshop Planning Committee and Speakers

Boxes

BOXES

1-1 Workshop Statement of Task

1-2 National Action Plan Domains and Goals

6-1 Highlights of Key Points Made by Individual Speakers Relevant to Each of the Eight National Action Plan Domains

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Acronyms and Abbreviations