Rare Endpoints: Delivering On Unmet Patient Needs

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

In this episode of "Preparing for a Cell and Gene Future," Jamie Pierson, Program Lead at Parexel’s Cell and Gene Center of Excellence, engages in a compelling discussion with Kim MacDonnell, Associate Director specializing in Rare Diseases. Together, they explore the innovative use of surrogate markers as endpoints in the development of treatments for rare diseases.

The conversation not only delves into the practical applications of these surrogate markers but also anticipates promising advancements fostered by the FDA’s latest initiatives. Pierson and MacDonnell reflect on the evolving regulatory landscape and its impact on accelerating therapeutic breakthroughs in the field of rare diseases, underscoring the pivotal role of surrogate endpoints in this journey towards more effective treatments and improved patient outcomes.