This quarterly publication will keep you up to date on PRA’s Rare Disease Team focus, achievements, and new initiatives.
Creating Solutions to Ensure Endpoint Protection in Challenging Rare Disease Trial
The collection of high-quality endpoint data was paramount to the success of this Phase II program. Because this was the first clinical trial ever…
Decoding FDA Gene Therapy Guidances Part Two: Rare Disease
In Part Two of Decoding FDA Gene Therapy Guidances, we discuss three separate guidances that provide further insight into how sponsors can safely and…
Patient Involvement Value Dossier
Increasing evidence supports that patient involvement in development of clinical trials provides value to sponsors by de-risking trial efficiency