Rethinking evidence generation for ultra-rare diseases 

FDA’s RDEP in Context Developing treatments for ultra-rare diseases presents fundamental challenges for traditional evidence generation. Patient populations for these conditions range from fewer than 10 to up to 1,000, making traditional randomized clinical trial (RCT) designs impractical and possibly unethical. These constraints require sponsors to adopt flexible evidence approaches without compromising the rigor needed… The post Rethinking evidence generation for ultra-rare diseases  appeared first on Drug Discovery and Development.

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