FDA director of drug evaluation and research Janet Woodcock says she is amazed that FDA has had more than 30 requests for its new breakthrough therapy designations and it has granted 11 already. The provision for breakthrough designations was part of the FDA Safety and Innovation Act (FDASIA) passed by Congress last year. The designation must be for a product for a serious condition and it must show preliminary clinical evidence that it may demonstrate substantial improvement over available therapies.

Addressing the annual meeting of the Food and Drug Law Institute in Washington, DC, Woodcock said that FDA is   “establishing a paradigm for these drugs that have a very high treatment effect.”

Woodcock said many people are asking how the program differs from accelerated approval or fast track approval. The agency, she said, will issue guidance to clarify that and she has told staff she wants to have a side-by-side comparison. “We have to work with the sponsors. Then, once we have identified breakthrough therapy on what will the development plan be…we want a rapid development plan,” she told the meeting.

Woodcock added that some of the therapies that have received the designation are for rare diseases and many are targeted therapies using genetics.