This quarterly publication will keep you up to date on PRA’s Rare Disease Team focus, achievements, and new initiatives.
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Case Study
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…
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Blog
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…
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Resource
Patient Involvement Value Dossier
Increasing evidence supports that patient involvement in development of clinical trials provides value to sponsors by de-risking trial efficiency
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