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Confirmatory Studies For Rare Diseases Shouldn’t Need To Start Before Accelerated Approval, Stakeholders Argue

 
• By Derrick Gingery

Advocates and sponsors looking to shape implementation of US FDA’s new mandate want to ensure firms are not required to make significant investments in surrogate endpoints before they are validated.

clinical trial use
A blanket mandate to start confirmatory trials when accelerated approval is granted could force significant investment in surrogate endpoints that are not accepted. • Source: Shutterstock

BOSTON – Having a confirmatory study ongoing at the time of an accelerated approval may be a laudable goal, but some stakeholders worry a blanket requirement could hinder rare disease development.

Under the 2022 Food and Drug Omnibus Reform Act, the US Food and Drug Administration gained the ability to require...

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