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Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2020. Exploring Novel Clinical Trial Designs for Gene-Based Therapies: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25712.
×

Exploring Novel
Clinical Trial Designs for
Gene-Based Therapies

PROCEEDINGS OF A WORKSHOP

Siobhan Addie, Meredith Hackmann, Joe Alper, and Sarah H. Beachy,
Rapporteurs

Forum on Regenerative Medicine

Board on Health Sciences Policy

Health and Medicine Division

images

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Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2020. Exploring Novel Clinical Trial Designs for Gene-Based Therapies: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25712.
×

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This activity was supported by contracts between the National Academy of Sciences and Advanced Regenerative Manufacturing Institute; Akron Biotech; Alliance for Regenerative Medicine; American Society of Gene & Cell Therapy; Burroughs Wellcome Fund; California Institute for Regenerative Medicine; Centre for Commercialization of Regenerative Medicine; Department of Veterans Affairs (Contract No. VA268-16-C-0051); Food and Drug Administration (Grant #1R13FD0066—14-01); GE Healthcare; International Society for Cell & Gene Therapy; International Society for Stem Cell Research; Johnson & Johnson; The Michael J. Fox Foundation for Parkinson’s Research; National Institute of Standards and Technology; National Institutes of Health: National Institute of Dental and Craniofacial Research (Contract No. HHSN263201800029I; Order No. 75N98019F00847), National Institute of Diabetes and Digestive and Kidney Diseases (PO No. 75N94019P00304); The New York Stem Cell Foundation; Parkinson’s Foundation; Sanofi (Contract No. 55630791); and United Therapeutics Corporation (Contract No. 10003921). 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: 978-0-309-67298-6
International Standard Book Number-10: 0-309-67298-8
Digital Object Identifier: http://doi.org/10.17226/25712

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Copyright 2020 by the National Academy of Sciences. All rights reserved.

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Suggested citation: National Academies of Sciences, Engineering, and Medicine. 2020. Exploring novel clinical trial designs for gene-based therapies: Proceedings of a workshop. Washington, DC: The National Academies Press. http://doi.org/10.17226/25712.

Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2020. Exploring Novel Clinical Trial Designs for Gene-Based Therapies: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25712.
×

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Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2020. Exploring Novel Clinical Trial Designs for Gene-Based Therapies: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25712.
×

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Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2020. Exploring Novel Clinical Trial Designs for Gene-Based Therapies: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25712.
×

PLANNING COMMITTEE ON EXPLORING NOVEL CLINICAL TRIAL DESIGNS FOR GENE-BASED THERAPIES1

KRISHANU SAHA (Co-Chair), Associate Professor and Retina Research Foundation Kathryn and Latimer Murfee Chair, Department of Biomedical Engineering, University of Wisconsin–Madison

CELIA WITTEN (Co-Chair), Deputy Director, Center for Biologics Evaluation and Research, Food and Drug Administration

MILDRED CHO, Research Professor of Pediatrics and Associate Director, Center for Biomedical Ethics, Stanford University

MICHAEL DeBAUN, Professor of Pediatrics and Medicine, Vanderbilt University School of Medicine

CYNTHIA DUNBAR, Senior Investigator, National Heart, Lung, and Blood Institute, National Institutes of Health

DEREK ROBERTSON, Co-Founder and President, Maryland Sickle Cell Disease Association

KATHERINE TSOKAS, Regulatory Head of Regenerative Medicine and Advanced Therapy, Johnson & Johnson

Forum on Regenerative Medicine Staff

SARAH H. BEACHY, Senior Program Officer and Forum Director

SIOBHAN ADDIE, Program Officer

MEREDITH HACKMANN, Associate Program Officer

MICHAEL BERRIOS, Senior Program Assistant

Board on Health Sciences Policy Staff

BRIDGET BOREL, Program Coordinator (from October 2019)

MARIAM SHELTON, Program Coordinator (until October 2019)

ANDREW M. POPE, Senior Board Director

___________________

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. 2020. Exploring Novel Clinical Trial Designs for Gene-Based Therapies: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25712.
×

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Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2020. Exploring Novel Clinical Trial Designs for Gene-Based Therapies: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25712.
×

FORUM ON REGENERATIVE MEDICINE1

TIM COETZEE (Co-Chair), Chief Advocacy, Services and Research Officer, National Multiple Sclerosis Society

KATHERINE TSOKAS (Co-Chair), Regulatory Head of Regenerative Medicine and Advanced Therapy, Johnson & Johnson

JAMES C. BECK, Vice President, Scientific Affairs, Parkinson’s Foundation

SANGEETA BHATIA, John J. and Dorothy Wilson Professor, Institute for Medical Engineering and Science, Electrical Engineering and Computer Science, Massachusetts Institute of Technology

GEORGE Q. DALEY, Director, Stem Cell Transplantation Program, Boston Children’s Hospital and Dana-Farber Cancer Institute; Dean, Harvard Medical School

BRIAN FISKE, Senior Vice President, Research Programs, The Michael J. Fox Foundation for Parkinson’s Research

LAWRENCE GOLDSTEIN, Distinguished Professor, Department of Cellular and Molecular Medicine, Department of Neurosciences; Director, University of California, San Diego, Stem Cell Program; Scientific Director, Sanford Consortium for Regenerative Medicine; Director, Sanford Stem Cell Clinical Center, University of California, San Diego, School of Medicine

AUDREY KUSIAK, Scientific Program Manager, Rehabilitation Research and Development Service, Office of Research and Development, Department of Veterans Affairs (until September 2019)

ROBERT S. LANGER, David H. Koch Institute Professor, Massachusetts Institute of Technology

CATO T. LAURENCIN, University Professor, Albert and Wilda Van Dusen Distinguished Professor of Orthopaedic Surgery, Professor of Chemical, Materials Science, and Biomedical Engineering; Director, The Raymond and Beverly Sackler Center for Biomedical, Biological, Physical, and Engineering Sciences; Chief Executive Officer, Connecticut Convergence Institute for Translation in Regenerative Engineering, University of Connecticut

TERRY MAGNUSON, Sarah Graham Kenan Professor, Vice Chancellor for Research, University of North Carolina at Chapel Hill

MICHAEL MAY, President and Chief Executive Officer, Centre for Commercialization of Regenerative Medicine

___________________

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.

Page viii Cite
Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2020. Exploring Novel Clinical Trial Designs for Gene-Based Therapies: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25712.
×

RICHARD McFARLAND, Chief Regulatory Officer, Advanced Regenerative Manufacturing Institute

JACK MOSHER, Senior Manager, Scientific Affairs, International Society for Stem Cell Research

DUANQING PEI, Director General, Guangzhou Institutes of Biomedicine and Health, Chinese Academy of Sciences

THOMAS PETERSEN, Vice President, Regenerative Medicine, United Therapeutics Corporation

ANNE PLANT, Chief of the Biosystems and Biomaterials Division, National Institute of Standards and Technology

KIMBERLEE POTTER, Scientific Program Manager, Biomedical Laboratory R&D Service, Office of Research and Development, Department of Veterans Affairs (from September 2019)

DEREK ROBERTSON, Co-Founder and President, Maryland Sickle Cell Disease Association

GRIFFIN RODGERS, Director, National Institute of Diabetes and Digestive and Kidney Diseases, National Institutes of Health

KELLY ROSE, Program Officer, Burroughs Wellcome Fund

KRISHNENDU ROY, Robert A. Milton Chair and Professor in Biomedical Engineering; Technical Lead, National Cell Manufacturing Consortium; Director, Marcus Center for Therapeutic Cell Characterization and Manufacturing, Georgia Institute of Technology

KRISHANU SAHA, Associate Professor and Retina Research Foundation Kathryn and Latimer Murfee Chair, Department of Biomedical Engineering, University of Wisconsin–Madison

RACHEL SALZMAN, Society Officer, American Society of Gene & Cell Therapy

JAY P. SIEGEL, Chief Biotechnology Officer and Head, Scientific Strategy and Policy, Johnson & Johnson (Retired)

LANA SKIRBOLL, Vice President, Science Policy, Sanofi

MARTHA SOMERMAN, Director, National Institute of Dental and Craniofacial Research, National Institutes of Health

LISA STROVINK, Chief Strategy Officer, The New York Stem Cell Foundation

SOHEL TALIB, Associate Director of Therapeutics and Industry Alliance, California Institute for Regenerative Medicine

PHIL VANEK, General Manager, Cell Therapy Growth Strategy, GE Healthcare

DANIEL WEISS, Chief Scientific Officer, International Society for Cell & Gene Therapy

MICHAEL WERNER, Co-Founder and Senior Policy Counsel, Alliance for Regenerative Medicine

Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2020. Exploring Novel Clinical Trial Designs for Gene-Based Therapies: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25712.
×

CELIA WITTEN, Deputy Director, Center for Biologics Evaluation and Research, Food and Drug Administration

CLAUDIA ZYLBERBERG, Founder and Chief Executive Officer, Akron Biotech

Forum on Regenerative Medicine Staff

SARAH H. BEACHY, Senior Program Officer and Forum Director

SIOBHAN ADDIE, Program Officer

MEREDITH HACKMANN, Associate Program Officer

MICHAEL BERRIOS, Senior Program Assistant

Board on Health Sciences Policy Staff

BRIDGET BOREL, Program Coordinator (from October 2019)

MARIAM SHELTON, Program Coordinator (until October 2019)

ANDREW M. POPE, Senior Board Director

Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2020. Exploring Novel Clinical Trial Designs for Gene-Based Therapies: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25712.
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Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2020. Exploring Novel Clinical Trial Designs for Gene-Based Therapies: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25712.
×

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 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. 2020. Exploring Novel Clinical Trial Designs for Gene-Based Therapies: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25712.
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Page xiii Cite
Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2020. Exploring Novel Clinical Trial Designs for Gene-Based Therapies: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25712.
×

Acknowledgments

The support of the sponsors of the Forum on Regenerative Medicine was crucial to the planning and conduct of the workshop Exploring Novel Clinical Trial Designs for Gene-Based Therapies, and for the development of this Proceedings of a Workshop. Federal sponsors were the Department of Veterans Affairs; Food and Drug Administration; National Institute of Standards and Technology; and National Institutes of Health: National Institute of Dental and Craniofacial Research and National Institute of Diabetes and Digestive and Kidney Diseases. Nonfederal sponsorship was provided by the Advanced Regenerative Manufacturing Institute; Akron Biotech; Alliance for Regenerative Medicine; American Society of Gene & Cell Therapy; Burroughs Wellcome Fund; California Institute for Regenerative Medicine; Centre for Commercialization of Regenerative Medicine; GE Healthcare; International Society for Cell & Gene Therapy; International Society for Stem Cell Research; Johnson & Johnson; The Michael J. Fox Foundation for Parkinson’s Research; The New York Stem Cell Foundation; Parkinson’s Foundation; Sanofi; and United Therapeutics Corporation.

The Forum on Regenerative Medicine wishes to express gratitude to the expert speakers who explored the design complexities and ethical issues associated with developing clinical trials for gene-based therapies. The forum also wishes to thank the members of the planning committee for their work in developing an excellent workshop agenda. The project director would like to thank the project staff who worked diligently to develop both the workshop and the resulting Proceedings of a Workshop.

Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2020. Exploring Novel Clinical Trial Designs for Gene-Based Therapies: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25712.
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Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2020. Exploring Novel Clinical Trial Designs for Gene-Based Therapies: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25712.
×
Page xviii Cite
Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2020. Exploring Novel Clinical Trial Designs for Gene-Based Therapies: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25712.
×
Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2020. Exploring Novel Clinical Trial Designs for Gene-Based Therapies: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25712.
×

Acronyms and Abbreviations

AAV adeno-associated virus
ADA adenosine deaminase
BEST Biomarkers, EndpointS, and other Tools
CAR T chimeric antigen receptor T cell
CCSS Childhood Cancer Survivor Study
CFTR cystic fibrosis transmembrane conductance regulator
CLL chronic lymphocytic leukemia
CNS central nervous system
DDT drug development tool
DMD Duchenne muscular dystrophy
Dx diagnosis
FDA Food and Drug Administration
GAA acid alpha-glucosidase
GT gene therapy
GVHD graft-versus-host disease
HL Hodgkin lymphoma
HLA human leukocyte antigen
Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2020. Exploring Novel Clinical Trial Designs for Gene-Based Therapies: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25712.
×
IRB institutional review board
MLMT multi-luminance mobility test
NCATS National Center for Advancing Translational Sciences
NCI National Cancer Institute
NDI National Death Index
NeuroNEXT Network for Excellence in Neuroscience Clinical Trials
NHL non-Hodgkin lymphoma
NHLBI National Heart, Lung, and Blood Institute
NIH National Institutes of Health
RPE65 retinal pigment epithelium 65
SCD sickle cell disease
SCID severe combined immunodeficiency
SJLIFE St. Jude Lifetime cohort
SMA spinal muscular atrophy
Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2020. Exploring Novel Clinical Trial Designs for Gene-Based Therapies: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25712.
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Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2020. Exploring Novel Clinical Trial Designs for Gene-Based Therapies: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25712.
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Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2020. Exploring Novel Clinical Trial Designs for Gene-Based Therapies: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25712.
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Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2020. Exploring Novel Clinical Trial Designs for Gene-Based Therapies: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25712.
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Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2020. Exploring Novel Clinical Trial Designs for Gene-Based Therapies: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25712.
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Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2020. Exploring Novel Clinical Trial Designs for Gene-Based Therapies: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25712.
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Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2020. Exploring Novel Clinical Trial Designs for Gene-Based Therapies: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25712.
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Page viii Cite
Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2020. Exploring Novel Clinical Trial Designs for Gene-Based Therapies: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25712.
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Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2020. Exploring Novel Clinical Trial Designs for Gene-Based Therapies: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25712.
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Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2020. Exploring Novel Clinical Trial Designs for Gene-Based Therapies: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25712.
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Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2020. Exploring Novel Clinical Trial Designs for Gene-Based Therapies: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25712.
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Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2020. Exploring Novel Clinical Trial Designs for Gene-Based Therapies: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25712.
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Page xiii Cite
Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2020. Exploring Novel Clinical Trial Designs for Gene-Based Therapies: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25712.
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Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2020. Exploring Novel Clinical Trial Designs for Gene-Based Therapies: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25712.
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Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2020. Exploring Novel Clinical Trial Designs for Gene-Based Therapies: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25712.
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Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2020. Exploring Novel Clinical Trial Designs for Gene-Based Therapies: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25712.
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Page xvii Cite
Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2020. Exploring Novel Clinical Trial Designs for Gene-Based Therapies: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25712.
×
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Page xviii Cite
Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2020. Exploring Novel Clinical Trial Designs for Gene-Based Therapies: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25712.
×
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Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2020. Exploring Novel Clinical Trial Designs for Gene-Based Therapies: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25712.
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Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2020. Exploring Novel Clinical Trial Designs for Gene-Based Therapies: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25712.
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Recognizing the potential design complexities and ethical issues associated with clinical trials for gene therapies, the Forum on Regenerative Medicine of the National Academies of Sciences, Engineering, and Medicine held a 1-day workshop in Washington, DC, on November 13, 2019. Speakers at the workshop discussed patient recruitment and selection for gene-based clinical trials, explored how the safety of new therapies is assessed, reviewed the challenges involving dose escalation, and spoke about ethical issues such as informed consent and the role of clinicians in recommending trials as options to their patients. The workshop also included discussions of topics related to gene therapies in the context of other available and potentially curative treatments, such as bone marrow transplantation for hemoglobinopathies. This publication summarizes the presentation and discussion of the workshop.

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