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.
The Importance of Patient Education for Rare Disease Trials
Easing the Rare Patient's Burden in Clinical Trials
PRA Successfully Rescues Phase II Hematology Study
A phase II single-blind, randomized, placebo-controlled trial to study the efficacy and safety of Study Drug administered as adjunctive treatment to…