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.
Living with a Rare Disease: Daizha’s Story
Good Clinical Practice: A PRA Perspective
Three Necessary Steps When Designing your New World Protocol
This article explores the design of a protocol that includes decentralized components. After a clinical-trial protocol has been developed, adding…