On the RISE: Controls in Rare Disease Clinical Trials for Small and Diminishing Populations

Conducting clinical trials in rare disease populations continues to pose many unique challenges, especially for small and diminishing populations, despite the existence of regulatory guidance on how to perform controls in rare disease trials and an increase in approvals for drugs and biologics to treat patients with rare diseases. This 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, will explore considerations when choosing a control, discuss existing and innovative control options internal and external to the trial, and identify how these controls can be used to generate evidence that supports regulatory decision-making.

This project is supported by the Food and Drug Administration (FDA) of the U.S. Department of Health and Human Services (HHS) as part of a financial assistance award U19FD006602 totaling $5,192,495 with 100 percent funded by FDA/HHS. The contents are those of the author(s) and do not necessarily represent the official views of, nor an endorsement, by FDA/HHS, or the U.S. Government.