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.
Laura Iliescu, Manager of Patient Advocacy & Engagement, Center for Rare Diseases
We measure our HQ with actions, experiences, and intentions. Nowhere is that more evident that in the stories of our subject matter experts. We want…
The Patient Voice - Engaging Rare Disease Patients to Accelerate Clinical Trial Enrollment
Rare disease patients face a number of unique challenges and complexities of their conditions, Often, this means that their families and advocates…
RARE DISEASES NEWSLETTER Volume 7, October 2017
This quarterly publication will keep you up to date on PRA’s Rare Disease Team focus, achievements, and new initiatives.