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Suggested Citation:"Appendix A: Workshop Agenda." 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 83
Suggested Citation:"Appendix A: Workshop Agenda." 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 84
Suggested Citation:"Appendix A: Workshop Agenda." 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 85
Suggested Citation:"Appendix A: Workshop Agenda." 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 86
Suggested Citation:"Appendix A: Workshop Agenda." 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 87
Suggested Citation:"Appendix A: Workshop Agenda." 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 88

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Appendix A Workshop Agenda EXPLORING NOVEL CLINICAL TRIAL DESIGNS FOR GENE-BASED THERAPIES: A WORKSHOP November 13, 2019 National Academy of Sciences Building Lecture Room 2101 Constitution Avenue, NW Washington, DC 20418 AGENDA 8:30 a.m. Opening Remarks Kathy Tsokas, Forum Co-Chair Regulatory Head of Regenerative Medicine & Advanced Therapy Johnson & Johnson 8:35 a.m. Charge to Workshop Speakers and Participants Krishanu Saha, Workshop Co-Chair Associate Professor 83 PREPUBLICATION COPY—Uncorrected Proofs

84 NOVEL CLINICAL TRIAL DESIGNS FOR GENE-BASED THERAPIES Retina Research Foundation Kathryn and Latimer Murfee Chair Department of Biomedical Engineering University of Wisconsin–Madison 8:45 a.m. Keynote Lecture Trial by “Firsts”: Lessons from the Frontlines of Clinical Trials in Gene Therapy Katherine High President and Head of Research and Development Spark Therapeutics 9:05 a.m. Clarifying Questions from Workshop Participants SESSION I: DEVELOPING FIRST-IN-HUMAN GENE THERAPY CLINICAL TRIALS Session Objective: • Explore the issues arising in the design of early-stage clinical gene therapy trials, including choice of endpoints, relevance, and requirements for preclinical data and identifying and using appropriate controls or natural history datasets. Session Moderator: Cindy Dunbar, National Heart, Lung, and Blood Institute, National Institutes of Health 9:15 a.m. Jonathan Kimmelman Director of Biomedical Ethics Unit McGill University 9:30 a.m. Richard Finkel Division Chief Neurology Department of Pediatrics Nemours Children’s Health System 9:45 a.m. Petra Kaufmann Vice President R&D Translational Medicine AveXis PREPUBLICATION COPY—Uncorrected Proofs

APPENDIX A 85 10:00 a.m. Panel Discussion with Speakers and Workshop Participants 10:30 a.m. Break SESSION II: UNDERSTANDING THE COMPLEXITIES OF PATIENT SELECTION, ENROLLMENT, AND THE CONSENT PROCESS Session Objectives: • Understand ethical issues surrounding patient selection, enroll- ment, and consent for gene-based therapies and how they differ from conventional clinical trials. • Identify what resources are available to help patients and pro- viders accurately understand the potential risks and benefits of participating in a gene-based clinical trial. • Explore communication strategies aimed at helping patients make informed decisions about participating in trials for gene-based therapies. Session Moderator: Mildred Cho, Stanford University 10:45 a.m. Courtney Fitzhugh Lasker Clinical Research Scholar Laboratory of Early Sickle Mortality Prevention National Heart, Lung, and Blood Institute National Institutes of Health 11:00 a.m. John Tisdale Senior Investigator and Director Cellular and Molecular Therapeutics Laboratory National Heart, Lung, and Blood Institute National Institutes of Health 11:15 a.m. Jennifer Puck Professor, Department of Pediatrics University of California, San Francisco 11:30 a.m. Pat Furlong Founding President and Chief Executive Officer Parent Project Muscular Dystrophy 11:45 a.m. Patient and Family Perspectives Ron Bartek PREPUBLICATION COPY—Uncorrected Proofs

86 NOVEL CLINICAL TRIAL DESIGNS FOR GENE-BASED THERAPIES 11:50 a.m. María José Contreras 11:55 a.m. Tesha Samuels 12:00 p.m. Panel Discussion with Speakers and Workshop Participants 12:30 p.m. Working Lunch SESSION III: CONSIDERING THE CHALLENGES WITH DEVELOPING ENDPOINTS FOR GENE THERAPY CLINICAL TRIALS Session Objective: • Learn about successes and challenges in accurately measuring clinical endpoints and outcomes for gene-based therapies and moving products through the translational pathway. Session Moderator: Larissa Lapteva, Food and Drug Administration 1:30 p.m. Larissa Lapteva Associate Director for Clinical and Nonclinical Regulation Division of Clinical Evaluation, Pharmacology, and Toxicology Office of Tissues and Advanced Therapies Center for Biologics Evaluation and Research Food and Drug Administration 1:45 p.m. Dwight Koeberl Professor Department of Pediatrics/Division of Medical Genetics and Department of Molecular Genetics and Metabolism Medical Director Biochemical Genetics Laboratory Duke University 2:00 p.m. Albert Maguire Professor of Ophthalmology Hospital of the University of Pennsylvania Presbyterian Medical Center of Philadelphia 2:15 p.m. Julie Kanter Associate Professor Hematology and Oncology University of Alabama at Birmingham School of Medicine PREPUBLICATION COPY—Uncorrected Proofs

APPENDIX A 87 2:30 p.m. Panel Discussion with Speakers and Workshop Participants 3:00 p.m. Break SESSION IV: INTEGRATING A GENE-BASED THERAPY INTO CLINICAL PRACTICE: EXPLORING LONG-TERM PATIENT MANAGEMENT AND FOLLOW-UP ISSUES Session Objectives: • Explore the implications of long-term clinical management of patients who participate in gene-based clinical trials (e.g., side effects, immunological implications). • Discuss how data from a limited number of patients can be effectively used to determine if a gene-based therapy is safe and/ or effective. Session Moderator: Michael DeBaun, Vanderbilt University 3:15 p.m. Tejashri Purohit-Sheth Director Division of Clinical Evaluation, Pharmacology, and Toxicology Office of Tissues and Advanced Therapies Center for Biologics Evaluation and Research Food and Drug Administration 3:30 p.m. Leslie Robison Chair Department of Epidemiology and Cancer Control Co-Leader Cancer Control and Survivorship Program St. Jude Children’s Research Hospital 3:45 p.m. David Chonzi Vice President Pharmacovigilance and Epidemiology Allogene 4:00 p.m. Bruce Marshall Senior Vice President of Clinical Affairs Cystic Fibrosis Foundation PREPUBLICATION COPY—Uncorrected Proofs

88 NOVEL CLINICAL TRIAL DESIGNS FOR GENE-BASED THERAPIES 4:15 p.m. Patient Perspective Bob Levis 4:20 p.m. Panel Discussion with Speakers and Workshop Participants SESSION V: NEXT STEPS AND WRAP-UP SESSION Session Objectives: • Discuss ways forward to support the clinical development of safe and effective gene-based therapies. • Summarize the lessons learned and topics discussed throughout the workshop. Session Moderator: Celia Witten, Food and Drug Administration 4:50 p.m. Final Panel Discussion Ron Bartek Mildred Cho Richard Finkel Pat Furlong Katherine High Julie Kanter 5:20 p.m. Final Remarks from Workshop Co-Chairs Celia Witten, Workshop Co-Chair Deputy Director Center for Biologics Evaluation and Research Food and Drug Administration 5:30 p.m. Adjourn Evening Reception – East Court PREPUBLICATION COPY—Uncorrected Proofs

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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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