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FDA Breakthrough Therapy Designation Statistics (07.03.13)

July 3, 2013

To expedite the development and review of drugs for life-threatening and serious diseases, the FDA had three programs before the Breakthrough Therapy Designation is created :

– Fast Track

– Accelerated Approval

– Priority Review.

The three Designations are in existence for at least 20 years. What is different about the Breakthrough Therapy Designation is the “ … requirement of early clinical data demonstrating an unprecedented effect …”. For example, the Fast Track Designation can be granted off the back of promising preclinical data, whereas the Accelerated Approval Designation is about surrogate trial endpoints. For a Breakthrough Therapy Designation, the FDA is expected to work closely with drug sponsors to “ …. design collaborative, multidisciplinary development plans that hasten timelines to approval and minimize the number of patients exposed to less efficacious treatment or placebos.”

The FDA has a “Frequently Asked Questions: Breakthrough Therapies” section on their website where the latest statistics on the FDA Breakthrough Therapy Designations are published and updated as of June 28, 2013 :

Breakthrough   Therapy (BT) Designation Category

# of BT   Designations (10/01/12-06/28/13)

Total   # of BT Requests Received


Total   # of BTs Granted


Total # of   BTs Denied


Total   # of BTs Pending


Seventeen (17) Breakthrough Therapy Designations have been identified by the sponsor companies as of June 28, 2013.  With 23 total Breakthrough Therapy Designations granted, there are 6 that have not been identified by the sponsor company.

The FDA doesn’t disclose information regarding sponsors who submitted requests for or who have been granted or denied breakthrough therapy designations. Per the FDA website,  “Breakthrough therapy designation requests are typically submitted to an IND, and the FDA cannot disclose the existence of an IND, or any submissions that have been submitted to the IND, unless it has previously been publicly disclosed or acknowledged per 21 CFR 312.130(a).”

The FDA publishes on June 26, 2013,  a draft guidance outlining both the qualifying criteria and benefits for the agency’s four expedited programs for serious or life-threatening conditions – Fast Track, Accelerated Approval, Priority Review, and the Breakthrough Therapy.

Comments on the draft guidance are due August 26, 2013. Further discussion will be provided by the Blog this week.

Please Note: “Idea” PNG by Producer at ar.wikipedia (Transferred from ar.wikipedia) [Public domain] | Wikimedia Commons.

Copyright © 2012-2013, Orphan Druganaut Blog. All rights reserved.

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