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 Professors 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 |
| 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, enrollment, and consent for gene-based therapies and how they differ from conventional clinical trials.
- Identify what resources are available to help patients and providers 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 |
| 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 |
| 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 |
| 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 |
