The FDA established a new type of rapid designation to drugs that address unmet medical needs or have shown especially efficacy treatments addressing a serious or life-threatening illness. In order to qualify the the sponsor must provide early clinical evidence that the treatment is a substantial improvement over currently available therapies.
In April 2015 the FDA held a symposium about the fist 2,5 years of experience with the BT Designation. The event was filmed. On the symposium a number of new facts came up.
As of March 2015, FDA had received a total of 293 requests for breakthrough therapy designation.
Of these, the Agency granted a total of 82—less than 1 in 3. Of the 244 requests submitted to the Center for Drug Evaluation and Research (CDER), 71 (or 30%) were granted. Of the 49 requests submitted to the Center for Biologics Evaluation and Research (CBER), 11 (or 22%) were granted. In contrast, between 1998 and 2007 CDER received 566 fast track designation requests, and granted 424 (75%), while CBER received 311 fast track designation requests, and granted 194 (62%).10,11 The two designations have different criteria—the “bar” for granting a BTD request is higher, for example—but the higher rate of success for fast track designation requests suggests that sponsors may have relatively less clarity on what qualifies as a breakthrough therapy as compared to what qualifies for fast track. In order to shed more light on this issue—as well as improve the success rate of future applications—the agency is currently conducting an in-house analysis of breakthrough therapy designation requests and the FDA review of these requests.