Materials
Session I: Introductions & Project Overview
Introductions and Welcome
Claire M. Gelfman, Foundation for Fighting Blindness
Veronica Miller, The Forum for Collaborative Research
Jim Wang, Adverum Biotechnologies
Session II: Understanding the Global Regulatory Perspective
The US Regulatory Landscape
Peter Marks, US Food and Drug Administration (CBER)
The European Regulatory Landscape
Kerstin Wickstrom, Icelandic Medicines Agency
The Japanese Regulatory Landscape
Shinichi Noda,Pharmaceuticals and Medical Devices Agency
Session III: Identifying Gaps and Barriers
Case Study: Luxturna
Daniel Chung, SparingVision
Interpreting the New FDA Draft Guidance for AMD and its Implications for Clinical Trial Design
Peter Kaiser, Cleveland Clinic
Session IV: PROs and PROMs
Fit for Purpose PROMs
Todd Durham, Foundation for Fighting Blindness
Session V: Patient Perspective
Uni-Rare Study
Jose-Alain Sahel,University of Pittsburgh MedicalCenter
Session VI: Identifying Opportunities for Collaboration
Working Groups Overview
Sehyr Khan,The Forum for Collaborative Research