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When to request Fast Track, Breakthrough or RMAT designations, and common denial reasons
Summary
Panelists said Fast Track, Breakthrough and RMAT should be requested with an original IND or as an amendment (ideally before end-of-phase 2); denials commonly arise when data do not show potential to address an unmet medical need or when preliminary clinical evidence is not persuasive.
Beatrice Colangal and Mara Miller described the evidentiary expectations for expedited designations. Beatrice said Fast Track requests are denied when a product does not show potential to address an unmet medical need or when the drug-development plan is inadequate. "Requests have been denied by OTP if the product does not show potential to address an unmet medical need," she said.
Mara noted Breakthrough therapy requests are denied when preliminary clinical evidence fails to demonstrate substantial improvement on clinically significant endpoints or is difficult to interpret (for example, because of small sample size or short follow-up). Jessica Behmer advised that Fast Track, Breakthrough and RMAT requests are generally submitted with an original IND or (as an amendment) before the end of phase 2.
