Thursday, April 2
9:05– 9:15 AM
Welcome and Opening Remarks
Amy Comstock Rick, President & CEO, Food and Drug Law Institute (FDLI)
Laura Brown, Director, Educational Programs, FDLI
9:15 – 10:00 AM
Keynote Address
Dr. Peter W. Marks, Director, Center for Biologics Evaluation and Research, FDA
This panel will explore definitions, types of products, mechanisms of action, and potential benefits associated with the rapidly innovating field of regenerative medicine. Speakers will discuss FDA’s comprehensive regenerative medicine policy framework, the regulation of human cells, tissues, and cellular and tissue-based products (HCT/Ps) under Public Health Service Act Sections 351 and 361, how FDA’s regulation of regenerative medicine therapies interacts with the practice of medicine, and the impact the end of FDA’s pending enforcement discretion period for certain HCT/Ps in November 2020 will have on companies.
Catherine M. Cook, Executive Vice President, Drugs and Biological Products, Greenleaf Health, Inc.
Julia C. Tierney, Chief of Staff, Center for Biologics Evaluation and Research, FDA
Moderated by Denise Esposito, Partner, Covington & Burling LLP
11:00 – 11:15 AM
Break
Despite gene and cell therapies’ many practical differences, several regulatory pre- and post-approval considerations intersect for these therapies. This session will explore these intersections, which include trends in expedited development and approval programs for regenerative medicine therapy; novel clinical trial design challenges; conducting comparability studies; FDA’s interpretation of “sameness” for orphan drug exclusivity; manufacturing, distribution, and tracking issues; and FDA’s capacity to review and support the increasing volume of regenerative medicine submissions.
Julie Allickson, Director, Regenerative Medicine Clinical Center, Wake Forest Institute for Regenerative Medicine
Edward Berg, Vice President, Deputy General Counsel, BioMarin Pharmaceutical Inc.
Michael N. Druckman, Partner, Hogan Lovells US LLP
Moderated by James Valentine, Attorney, Hyman, Phelps & McNamara, PC
12:30 – 1:30 PM
Lunch Break
Due to its long-term or permanent therapeutic effects, gene therapy presents many unique challenges in both the pre-and post-approval setting. Speakers will first contemplate how historical safety failures have led to a more cautionary approach to clinical trial safety and risk/benefit considerations. Panelists will then discuss the current landscape of clinical trials, manufacturing, and long-term follow up. Impacts of FDA’s January 2020 suite of draft and final guidance documents on clinical development, manufacturing, and orphan drug designation will also be discussed.
Wilson Bryan, Supervisory Medical Officer, Center for Biologics Evaluation and Research, FDA
Mantej (Nimi) Chhina, Senior Director and Head, Global Regulatory Policy, BioMarin Pharmaceutical Inc.
Lance L. Shea, Partner, BakerHostetler
Moderated by Barbara Binzak Blumenfeld, Shareholder, Buchanan Ingersoll & Rooney
2:45 – 3:00 PM
Break
Over the past several years FDA has taken decisive action against cell therapy clinics promoting unapproved, unproven, and potentially harmful products. This panel will explore lessons learned from FDA’s completed and ongoing enforcement actions and litigation against cell therapy companies. Speakers will also discuss FDA’s compliance assistance programs and projected agency steps after the November 2020 enforcement discretion deadline, as well as the potential public health effects of Google’s recent decision to stop advertising unapproved stem cell therapies.
Todd H. Halpern, Partner, Venable LLP
Melissa Mendoza, Deputy Director, Office of Compliance and Biologics Quality, Center for Biologics Evaluation and Research, FDA
Elizabeth Richardson, Director, Health Care Products Project, The Pew Charitable Trusts
Moderated by Beth P. Weinman, Counsel, Ropes & Gray LLP
What does the future hold for regenerative medicine? This panel will discuss the future of innovation and regulation in this exploding area. Access and reimbursement issues for these generally expensive therapies, including interactions between FDA, the Centers for Medicare and Medicaid Services (CMS), and payors will also be addressed.
Amy Garabedian, Associate General Counsel, Spark Therapeutics
Bruce Nash, Chief Physician Executive and Senior VP for Health and Medical Management, Blue Cross Blue Shield of Massachusetts
Michael Werner, Partner, Holland & Knight LLP
Moderated by Preeya Noronha Pinto, Partner, King & Spalding LLP
5:00 PM
Closing Remarks and Adjournment