Opportunities to Improve Dose-Finding and Optimization for Rare Disease Drug 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 virtual workshop focused on sharing effective strategies for dose-finding and optimization in rare diseases and best practices for early-phase trial design. Participants will discuss innovative trial designs, modeling, use of biomarkers, and other strategies that can lead to more efficient identification of appropriate drug dosages, as well as the use of real-world data to evaluate use of alternate dosages in the post-marketing setting. Discussion will also include consideration of the nuances of disease subtypes, population characteristics, therapy types, and specifics of drug administration and action.

This event in supported by the FDA of the U.S. Department of Health and Human Services (HHS) as a part of a financial assistance award U01FD006807 totaling $3,493,089 with 100 percent funded by FDA/HHS. The contents are those of the author(s) and do not necessarily represent the official view of, nor an endorsement by, FDA/HHS or the U.S. Government.