Developing drugs for rare diseases has long proved challenging from both a regulatory and commercial perspective, and the rarer the disease, the further these problems are exacerbated.
Clearer Regulatory Frameworks Needed For Ultra-Rare Conditions, Experts Say
Meeting the regulatory gold standard for drug candidates in ultra-rare diseases can be impractical, a regulatory expert says, but greater collaboration and shared insights from regulatory reviews could help find a viable path forward.
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