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 and the Promise of Gene Therapy: What You Need to Know
Cross-Border Enrollment of Rare Disease Patients
Clinical trials in rare diseases present unique challenges unseen in trials for more common conditions. Cross-border enrollment can be key to…
RARE DISEASES NEWSLETTER Volume 6, May 2017
This quarterly publication will keep you up to date on PRA’s Rare Disease Team focus, achievements, and new initiatives.