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Breakthrough Therapies - A New FDA Paradigm

The flexibility promised in the 2012 Food and Drug Administration Safety and Innovation Act (FDASIA) continues to be rolled out at the Agency. FDASIA includes a provision that allows sponsors to request that their drug be designated as a "Breakthrough Therapy". FDA is in the process of developing guidance related to this designation, but Sponsors can now submit requests for Breakthrough Therapy designation. Presumably, breakthrough therapies will cover such agents as those which appear to provide unmet medical needs for important healthcare conditions.

Breakthrough therapy designation is intended to expedite the development and review of drugs for serious or life-threatening conditions. The criteria for breakthrough therapy designation require preliminary clinical evidence that demonstrates the drug may have substantial improvement on at least one clinically significant endpoint over available therapy. A breakthrough therapy designation conveys all of the current fast track program advantages, as well as more intensive FDA guidance on an efficient drug development program. The FDA also has an organizational commitment to involve senior management in such guidance.

"FDASIA requires the following Agency actions, as appropriate:

A request for Breakthrough Therapy designation can be submitted concurrently with, or as an amendment to, an Investigational New Drug Application (IND) with a cover letter, a completed form 1571, and the following information:

How has this been working? As of the end of March, 2013, there had been about 40 submissions for breakthrough therapy designation and about 10 had been granted. Incidentally, an Agency decision to approve, or to refuse to approve, and application for breakthrough designation is an action taken within the IND and remains confidential unless the Sponsor chooses to make it public.