This quarterly publication will keep you up to date on PRA’s Rare Disease Team focus, achievements, and new initiatives.
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Blog
A Critical Success Factor in Rare Disease Clinical Research: Patient Advocacy Collaboration
In most public sectors, understanding the end user’s perspective and building out a product strategy around it is nothing short of dogma. However, in…
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Resource
Rapid Participation Burden Survey
Quick, flexible, validated patient survey tool for rapid feedback on trial-specific participation burden, helps sponsors refine risk analyses and…
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Blog
Decoding FDA Gene Therapy Guidances Part Three: Long-term Follow-ups
In Part Three of our Decoding FDA Gene Therapy Guidances series, we discuss the need for thorough long-term follow-up studies for these products, as…
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