Breakthrough Therapy Status, AdComms and Approvals

In recent years FDA has undertaken several means to facilitate the consideration of new drug applications by creating various mechanisms for expedited review for either new drugs or new indications for existing drugs. Prior to 2012, there had long been in place Accelerated Approval and Fast Track and Priority Review. In 2012, as a consequence of the enactment of the Food and Drug Administration Safety and Innovation Act (FDASIA), Breakthrough Therapy (BT) status was added as a means to gain speedier consideration of new drugs that would make an impact in the treatment environment.

All of the expedited means have had impact. In fact, by 2015, which was a banner year for the approval of new molecular entities, fully half of them had had some form of expedited review.

Breakthrough Therapy designation has been increasingly common since 2013 when there were a handful of drugs granted the status. In 2014, the number increased dramatically. By the end of the first quarter of this year, FDA has stated that it has granted breakthrough status to 111 out of 342 requests. FDA does not announce the status outcomes for specific companies.

What exactly does Breakthrough Therapy mean and what does FDA do with it?  The agency has said that the purpose is to indicate early in the drug development process that a compound has the potential to represent a true breakthrough in the long run. That, in turn, means that the agency will invest a number of resources to see that the compound gets evaluated in a highly efficient manner with regular input and advice from key FDA personnel. Receiving Breakthrough Therapy status means that the drug is treating a serious condition and that there is an indication of a potential to be a substantial improvement over existing treatments.

While the agency does not announce the designations when they occur, FDA does list the approvals that have occurred for compounds that have been designated as a Breakthrough Therapy. When you look at the list of drugs and biologics breakthrough therapies that have been approved and compare it to the list of drugs and biologics that have had advisory committee meetings in the course of their approval process reveals a surprising number.

Per the CDER list of Breakthrough Therapy approvals by calendar year – here and here, there have been 44 BT approvals as of June 30 of this year. However, FDA advisory committees have met only four times to consider investigative compounds for approval that have had breakthrough status. The balance – and bulk – of compounds that have had Breakthrough Therapy status were approved without an advisory committee meeting.

Does that mean that breakthrough status is a sort of surrogate marker for drugs that will not require an AdComm?  Probably not, but it does perhaps say that the process that is signified by breakthrough status – of working closely with FDA regarding key issues in the drug approval process – may for the most part make an advisory committee meeting less necessary under most circumstances. That said, there will still be circumstances where the agency would appear to believe that it still needs the input of advisory committee members in considering the drug application.

I was able to identify four instances in which there was an advisory committee meeting where a breakthrough status compound was the subject of approval deliberations.  In all four of these meetings the committees all voted to approve and FDA took the advice of the committee and ultimately approved all four.

For more on Breakthrough Therapy status, you can read an FDA Q&A here, a Fact Sheet here, and an informative interview here.

No Weekly Roundup today – but back next week.

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