Assessing Novel Efficacy Endpoints in Ophthalmologic Rare Disease Drug and Biologics Development

The Duke-Margolis Institute for Health Policy, under a cooperative agreement with the U.S. Food and Drug Administration, is convening a one-day public workshop focused on novel efficacy endpoints used in interventional clinical trials for drugs and biological products intended for patients with severe vision loss to support regulatory decision making. The workshop will focus in particular on full-field stimulus threshold testing (FST) and ellipsoid zone data (EZ). Researchers, clinicians, and other stakeholders 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.