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.
Preparing for the Road Ahead: Rare Disease Advocates Unite
Patient Involvement Value Dossier
Increasing evidence supports that patient involvement in development of clinical trials provides value to sponsors by de-risking trial efficiency
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…