Assessing Novel Efficacy Endpoints in Ophthalmologic Rare Disease Drug and Biologics Development September 17, 2025

https://www.fda.gov/drugs/news-events-human-drugs/assessing-novel-efficacy-endpoints-ophthalmologic-rare-disease-drug-and-biologics-development?utm_medium=email&utm_source=govdelivery Assessing Novel Efficacy Endpoints in Ophthalmologic Rare Disease Drug and Biologics Development Date: Wed, Sept. 17, 2025; 9:30 a.m. - 2:30 p.m. The Duke-Margolis Institute for Health Policy, under a cooperative agreement with the FDA, is convening a one-day hybrid public meeting on September 17, 2025, focused on novel efficacy endpoints used in interventional clinical trials for drugs and biologics intended for patients with severe vision loss to support regulatory decision making. The meeting will focus on full-field stimulus threshold testing and ellipsoid zone data. Researchers, clinicians, and other parties will present and discuss evidence and data that may support the use of these tools in regulatory decision-making, such as clinical and statistical considerations for quantifying a clinically meaningful change. Discussion will also include current limitations and potential strategies to advance the use and implementation of these tools to support regulatory decision-making https://healthpolicy.duke.edu/events/assessing-novel-efficacy-endpoints-ophthalmologic-rare-disease-drug-and-biologics?utm_medium=email&utm_source=govdelivery