Breakthrough therapy designation: A primerInteresting article. Dated of 2015, but it gives some insights. Here are some excerpts:
Breakthrough therapy designation: A primer BTD was established in 2012 under the Food and Drug Administration Safety and Innovation Act, and is intended to expedite the development and review of drugs that show signs of extraordinary benefit at early stages of the clinical development process. However, BTD is not an automatic approval. The drug still has to undergo clinical testing and review by the FDA. Rather, BTD is designed to facilitate and shorten the clinical development process, which can otherwise take many years to complete. However, the FDA denies many more requests than it grants. In fact, as of March 2015, less than one in three of the BTD requests submitted have been granted. By contrast, roughly 75% of the requests for fast track designation (another of the Agency’s expedited programs) were granted between 1998 and 2007. This discrepancy suggests ongoing uncertainty over what exactly constitutes a “breakthrough” according to the FDA’s criteria.
... HOW DOES BTD RELATE TO THE OTHER THREE EXPEDITED PROGRAMS?
The other three expedited review and development programs—fast track designation, priority review, and accelerated approval—are also geared at facilitating the development and approval of drugs for serious conditions. These other programs have been in place for over 15 years, and have played a significant role in accelerating patient access to new therapeutics In 2014 alone, 66% of the 41 drugs approved by FDA’s Center for Drug Evaluation and Research used at least one of these four pathways, and 46% received at least two of the designations in combination. ... HOW MANY THERAPIES HAVE RECEIVED THE DESIGNATION?
As of March 2015, FDA had received a total of 293 requests for BTD. Of these, 82 received the designation, and 23 have since been approved for marketing. Ten of these approvals were new indications for already approved drugs, rather than novel therapies that had never before received FDA approval.
WHAT ARE THE BENEFITS OF BTD?
For drug manufacturers, it is about the intensity and frequency of their interactions with FDA. Once the designation is granted, the FDA takes an “all hands-on-deck” approach to providing the manufacturer with ongoing guidance and feedback throughout the clinical development process. Products that receive BTD are also able to submit portions of their marketing application on a rolling basis (rather than all at once at the end of clinical trials) and BTD can also be used in combination with other expedited programs in order to further reduce the product’s time to market.