Clinical development involving rare diseases poses a unique set of medical, scientific, and operational challenges. PRA understands these challenges and the critical success factors necessary to develop drugs for rare disease patients. It’s not a cookie-cutter formula, but rather a nimble approach that embraces the flexibility, creativity, and innovation needed to succeed in this arena. In the last 5 years, we have managed over 170 Phase I-IV rare disease studies, across 11 different therapeutic areas, resulting in the successful marketing approval of 16 drugs.
RARE DISEASES NEWSLETTER Volume 13, May 2019
Welcome to the thirteenth edition of the rare diseases newsletter. This quarterly publication strives to keep you up to date on PRA's Center for Rare…
Rare Disease Registries: How to Become Part of the Cure
Easing the Rare Disease Patient’s Burden in Clinical Trials with Innovative Technology
In this webinar, you will learn how technology can influence rare disease clinical study design, increase the speed of research and thereby reduce…