Rare Disease Innovation, Science, and Exploration (RISE) Workshop Series

The Rare Disease Innovation Hub’s inaugural RISE Workshop, entitled On the RISE: Controls in Rare Disease Clinical Trials for Small and Diminishing Populations, took place on September 3, 2025. The hybrid, public workshop, co-convened by the Duke-Margolis Institute for Health Policy and the U.S. Food and Drug Administration (FDA) Rare Disease Innovation Hub under a cooperative agreement with the FDA, explored some of the unique challenges of conducting clinical trials in rare disease populations, especially for small and diminishing patient populations. FDA and external panelists discussed considerations when choosing a control for rare disease trials, discussed existing and innovative control options internal and external to the trial, and identified ways these controls can be used to generate evidence that supports regulatory decision-making.

A video recording of the full workshop and Duke Margolis’ written report on the meeting is available here.