Ultra Rare Disease: Is Perpetual IND a Regulatory Solution for Access?
Friday, May 31, 2024 09:15 AM - 10:15 AM
Room 208-209
Regulation & Policy
Roundtable
Organized by: ISCT North America Legal & Regulatory Affairs Committee
Moderator
Key Learning Objectives
1. What may constitute ultra rare diseases?
2. What are existing regulatory mechanisms to provide access to these types of CGTs/advanced therapies?
3. What are the bottlenecks for IND maintenance and marketing approval of ultra-rare products where commercialization is unlikely?
4. What are possible solutions to these challenges?
Moderator
- Michael Mendicino, PhD, Hybrid Concepts International (HCI), LLC, United States
- Donald B. Kohn, MD, University of California, Los Angeles, United States
- Julie Douville, PhD, n-Lorem Foundation, United States
- Nicole Verdun, MD, Food and Drug Administration (FDA), United States
Key Learning Objectives
1. What may constitute ultra rare diseases?
2. What are existing regulatory mechanisms to provide access to these types of CGTs/advanced therapies?
3. What are the bottlenecks for IND maintenance and marketing approval of ultra-rare products where commercialization is unlikely?
4. What are possible solutions to these challenges?
