FDA Lists the Reasons for Denial of BTD Requests
(Thursday, November 16, 2023)
Since its inception a little more than 10 years ago, the Breakthrough Therapy Designation (BTD) has become the most recognized indication of the FDA’s strong support for the therapeutic potential of a new drug. However, a BTD designation is hard to get; about two-thirds of the requests for BTD are denied or withdrawn by the applicants before the impending denial. This week FDA published an internal survey for the most common reasons for the denial of BTD requests, and it's consistent with what the FDA has said earlier about such denials.
The BTD designation was created in 2012 to encourage faster approval of exceptionally promising drugs. In the last 10 years, the FDA has granted the BTD to 467 drugs and 70 biologics and approved 264 drugs and 27 biologics, making it one of the FDA’s most successful expedited approval programs. The BTD is highly coveted, particularly by small and medium-sized companies with sole drug development programs. So, any tips on how to ensure that the BTD request is successful would be highly appreciated. FDA provides just that.
Just like its original survey published in 2016 with the early data from BTD requests, the new data from the requests submitted in the years 2017-2019, shows that the most common reason for the denial of the BTD request is insufficient treatment effect observed in the preliminary clinical trials, followed by inadequate trial design. The grant of BTD is not influenced much by whether or not there are available treatments for an indication for which the request is made or if the indication is an orphan indication. Most of the supporting data came from Phase 2 clinical trials.
The good news is that the FDA process has been consistent across the years. This data confirms that BTD requests are based on the quality of the clinical efficacy data more than any other factor, which should give clear directions to the applicants.
Dr. Mukesh Kumar
Founder & CEO, FDAMap
Linkedin: Mukesh Kumar, PhD, RAC