By Maximilian Klietmann, VP of Marketing, LSN
The FDA launched its “Breakthrough Therapy Designation” program just over a year ago, and the initial data is in. Though results are mixed, the BTD could have a significant impact on investor behavior in the near future, as major indications could become as attractive as orphan therapies when it comes to time-to-market. This article will dive into exactly what the BTD is, what the initial program results look like, and what it means to the industry at large.
The BTD, as it is often referred to, is a program intended to accelerate the development and review of drugs for serious or life-threatening conditions. According to the FDA, an asset with at least some preclinical data showing “substantial improvement on at least one clinically significant endpoint over available therapy” qualifies for BTD. A company that has secured BTD is granted all of the FDA fast-track benefits as well as more intensive FDA guidance on an efficient drug development. Basically, this allows a drug with the potential to outperform existing therapies in a significant way to have the same shortened time to market that orphan therapies are able to secure. In short – FDA approval timelines for significantly improved cardiovascular drugs could be fast-tracked just like rare disease drugs.
So how does the data look on the first batch of applications to the program? So far the FDA approved 30% of the 113 applications for the BTD it has received, and about 60% have been denied from the program or voluntarily withdrew. However, the picture is still not clear on what the impact will be on actual drug approvals and whether the program will significantly reduce time to market. So far, the non-orphan BTD beneficiaries of the BTD have been mostly oncology treatments and anti-infectives, but the potential for other therapies to take advantage of the program is huge.
It will be important to keep an eye on this program, because the implications could spell some changes in terms of investor interests. This is especially true for investors in orphan diseases who are motivated by the fast-track to market. However, this is only true if the program can actually deliver what it promises, so watch for the first wave of program graduates. If approval rates are high and development time is short, it could spell big things for investors and entrepreneurs alike.
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