Hemophilia Gene Therapy Studies Could Use Non-Inferiority Design, US FDA Suggests
Development guidance from CBER recommends comparing investigational gene therapy products to existing prophylaxis treatment.
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Although clotting factor activity levels may someday be validated as a surrogate endpoint, the currently recommended primary efficacy measure for traditional approval remains annualized bleeding rate, agency says in final guidance.
Outgoing commissioner say he has 'extreme confidence' in soon-to-be-acting Commissioner Ned Sharpless and other agency leaders, and expects initiatives advanced during his tenure will be ‘encapsulated and enshrined in regulation and policy’ because they are built on a solid administrative record.
A Pink Sheet analysis illustrates how the outgoing commissioner enhanced FDA's ability to facilitate the further development of innovative drugs.