Accelerated Approval: US FDA Explains When A Confirmatory Trial Is ‘Underway’

The agency will consider study enrollment, accrual rates and the number of clinical site activations, among other factors, in determining whether a confirmatory trial is underway prior to accelerated approval, but the new draft guidance does not specify the proportion of patients that must be enrolled at the time of approval.

Cruise ship
It may not be as obvious as a cruise ship when a confirmatory trial gets underway. (Shutterstock)
Key Takeaways
  • A new draft guidance describes the FDA's considerations when deciding whether a confirmatory trial is underway prior to accelerated approval.
  • If the agency determines that continued enrollment and retention after a drug product is on the market is likely to be especially challenging, it may require confirmatory trial enrollment to be completed at the time of approval.
  • Some situations may exist involving infectious diseases and rare disorders where an ongoing confirmatory trial is neither required nor feasible at the time of accelerated approval.

The US Food and Drug Administration will consider study enrollment, accrual rates and clinical site activations, among other factors, in determining whether a confirmatory trial is sufficiently underway and available...

A new draft guidance, “Accelerated Approval and Considerations for Determining Whether a Confirmatory Trial is Underway,” is intended to ensure that the time between accelerated...

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