New Draft Guidance for Industry on Breakthrough Therapy Designation and Accelerated Approval: Review of FDA Webinar

by Deborah Toscano 05 Aug 2013
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The FDA hosted a live webinar this morning to present the new draft guidance for industry for fast track designation, breakthrough therapy designation, accelerated approval, and priority review designation. This is the first such guidance published by the FDA on the new Breakthrough Therapy Designation and Accelerated Approval. The intent of these programs is to encourage drug development to address unmet needs for serious conditions. Breakthrough Therapy designation can be given to a new drug or biologic that demonstrates “substantial” (and this is admittedly a subjective term) improvement over available therapies for a serious condition, and it is the term “substantial improvement” that largely differentiates Breakthrough Therapy from Fast Track designation. According to the presentation, the FDA would consider “substantial” to be a clear benefit with regard to both the magnitude of the effect (which could include duration and/or degree) and the importance of the effect. Particularly valuable would be demonstration of treating the underlying disease rather than just treating the symptoms. A new drug with similar efficacy but that avoids a serious toxicity or a side effect that may not be serious but leads to poor compliance or withdrawals would also be considered as well. Add-on therapies would be considered as well, as long as they meet the criteria. Breakthrough Therapy Designation gives the sponsor all of the benefits of Fast Track status, plus more intensive guidance from the FDA and the attention of senior management. Accelerated Approval could be granted to a new drug that demonstrates efficacy on a surrogate endpoint that is reasonably likely to predict an important long-term clinical endpoint such as irreversible morbidity or mortality, or an intermediate clinical endpoint, also that is reasonably likely to be predictive of long term clinical benefit. In these cases the sponsor will usually be required to demonstrate post-marketing that the drug does indeed provide the predicted long-term clinical benefit. The FDA encourages sponsors to submit an application for potential Breakthrough Therapy Designation early in the development process, as soon as there is sufficient evidence of the clinical benefit of the drug.

The FDA is not liberally giving out Breakthrough Designations, however. Since May 31, 2013, the FDA has received 59 requests for Breakthrough Designation, of which 20 have been granted and 20 denied, so far.  

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