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Confirmatory Trials: Flexibility In Timing Hinges On Accelerated Approval Indication Size – FDA’s Marks

 
• By Sue Sutter

CBER director suggests more flexibility on confirmatory study timing is warranted for accelerated approvals in rare diseases and even in some more common scenarios such as infectious disease outbreaks, although a ‘relatively stronger approach’ will apply to most large indications.

balance beam
CBER will consider regulatory flexibility for rare diseases when it comes to confirmatory trial timelines. • Source: Shutterstock

The US FDA Center for Biologics Evaluation and Research’s decision-making on when to require that confirmatory trials be up and running at the time of accelerated approval will rest heavily on the size of the patient population at issue, CBER Director Peter Marks said.

The Food and Drug Omnibus Reform Act provision that allows the agency to require a confirmatory trial be ongoing at the time of approval “helps us have some teeth around...

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