Rare endpoints: Delivering on unmet patient needs

By Jamie Pierson, Senior Project Leader, Parexel International
Kim MacDonnell, AD, Rare Disease Center of Excellence

The latest episode of Preparing for a Cell and Gene Future features a discussion between Jamie Pierson (Program Lead within Parexel’s Cell and Gene Center of Excellence) and Kim MacDonnell (Associate Director for Rare Diseases). They consider uses of surrogate markers as endpoints in rare disease development and look ahead to positive directions established by FDA's current initiatives.

Contributing Expert