Report From the NEI/FDA Endpoints Workshop on Age-Related Macular Degeneration and Inherited Retinal Diseases.

The Association for Research in Vision (ARVO), together with the Foundation Fighting Blindness (FFB), organized the National Eye Institute (NEI)–Food and Drug Administration (FDA) endpoints workshop on age-related macular degeneration (AMD) and inherited retinal diseases (IRD) on November 9, 2016. The public workshop convened basic researchers, clinicians, and regulatory authorities in an interactive forum to discuss the latest clinical findings on AMD and IRD with the goal of optimizing clinical trial design and developing trial endpoints. Specifically, the aim of the workshop was to assemble stakeholders from academic and regulatory spheres to discuss clinical data on potential primary and secondary clinical trial endpoints; patient stratification for disease monitoring, interventions, and evaluation of treatment response; and identification of future research avenues. The workshop was jointly organized by Dr. Frederick Ferris III, MD, Clinical Director at the NEI; Dr. Emily Chew, MD, Deputy Clinical Director at the NEI; Dr. Karl Csaky, MD, PhD, Managing Director and Head of Molecular Ophthalmology Laboratory at the Retina Foundation of the Southwest; Dr. Jacque L. Duncan, MD, Professor of Clinical Ophthalmology at the University of California, San Francisco; and Janet K. Cheetham, PharmD, from the FFB. The workshop format was a series of presentations followed by panel discussions, in which FDA representatives offered regulatory perspectives on potential endpoints for clinical trial design. The following key topics were discussed.