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