The FDA has five expedited designations for speeding up the availability of drugs for serious diseases. These are summarized in table below. We will go into a bit more detail on each of the programs in the next few sections.
Therapies receiving special designations must meet the evidentiary standards for approval, including demonstrating effectiveness (regardless of whether the product receives accelerated or traditional approval). Fast track designation, Breakthrough Therapy designation, and Regenerative Medicine Advanced Therapy (RMAT) designation are distinct designation programs with different programmatic requirements. It is possible to apply for and receive more than one designation for a given product, but you should apply for each designation separately. Information that supports more than one designation may be submitted in each separate designation request. [5]
Summary of Expedited Programs
Expedited program type | Type of data required | Qualifying criteria | Benefits |
|---|---|---|---|
Preliminary non-clinical, mechanistic, or clinical data | A drug that is intended to treat a serious condition AND non-clinical or clinical data demonstrate the potential to address unmet medical need OR a drug that has been designated as a qualified infectious disease product |
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Preliminary clinical data | A drug that is intended to treat a serious condition AND preliminary clinical evidence indicates that the drug may demonstrate substantial improvement on a clinically significant endpoint(s) over available therapies |
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Not specified; Sponsor should make justification of alternate endpoint based scientific support | A drug that treats a serious condition AND generally provides a meaningful advantage over available therapies AND demonstrates an effect on a surrogate endpoint that is reasonably likely to predict clinical benefit, or on a clinical endpoint that can be measured earlier than irreversible morbidity or mortality (IMM) that is reasonably likely to predict an effect on IMM or other clinical benefit (i.e., an intermediate clinical endpoint) |
Note: The FDA requires clinical trials to be conducted post approval to confirm clinical benefit | |
Data contained in the final NDA submission | An application (original or efficacy supplement) for a drug that treats a serious condition AND, if approved, would provide a significant improvement in safety or effectiveness OR any supplement that proposes a labeling change pursuant to a report on a pediatric study under 505Ab OR an application for a drug that has been designated as a qualified infectious disease product OR any application or supplement for a drug submitted with a priority review voucher |
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Preliminary clinical evidence | A drug that is a regenerative medicine therapy intended to treat, modify, reverse, or cure a serious or life-threatening disease or condition, AND preliminary clinical evidence indicates that the drug has the potential to address unmet medical needs for such a disease or condition |
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(*) Accelerated Approval is a pathway, unlike the other four which are designations (**) Still relatively new and not easy to get | |||