Orphan Drug Approvals and Designations Dipped in 2016, But Orphan Drug Designation Requests Skyrocketed

January 25, 2017By Kurt Karst

Hardly a day goes by that we don’t see a press release or announcement that a company has requested or received from FDA’s Office of Orphan Products Development (“OOPD”) designation of its drug or biological product as an “orphan drug.” There’s a reason for that: FDA is receiving orphan drug designation requests at a record rate of 1.6 per day (2.23 per work day in 2016), and is granting designation requests at a near-record rate of 0.9 per day (1.28 per work day in 2016)!  If you haven’t already guessed, it’s time for the FDA Law Blog’s annual rundown of orphan drug designations and approval!

In 2016, FDA’s orphan drug program continued to break new ground with massive numbers of orphan drug designation requests. Orphan drug designations and approvals (which include not only approvals of NDAs for new molecular entities and BLAs for original biological products, but also applications approved for new orphan uses of previously approved drugs and biologics) dipped slightly from 2015, but not by much (see our previous post here).  2016 was still the second best year for orphan drug designations, and the third best year for orphan drug approvals since the enactment of the Orphan Drug Act.

FDA’s OOPD received an astonishing 582 requests for orphan drug designation in 2016.  That 110 more than the record set in 2015.  OOPD also granted an amazing 333 orphan drug designations in 2016.  That’s 21 fewer designations than the 2015 record, but still not too shabby.  There were 9 fewer orphan drug approvals in 2016 compared to the near-record-tying 48 approvals in 2015. (FDA set a record in 2014 with 49 orphan drug approvals.)  Since 1983, FDA has approved almost 600 orphan drugs, has granted nearly 4,000 orphan drug designations, and has received nearly 5,800 orphan drug designation requests.

Below are three tables – one for each metric we track – showing the year-by-year numbers since 1983. The numbers are largely based on information from FDA’s Orphan Drug Designations and Approvals Database.

ODDR2016
ODD2016

ODA2016Over the years, we’ve pointed out the fact that while OOPD’s workload continues to increase, the Office’s staff and resources have not kept pace. Despite the massive increase in workload and added responsibilities over the years, OOPD’s staff has remained at about 25 employees for quite some time.  Moreover, funding for OOPD has remained relatively flat over the past few years.

The stress on limited OOPD resources was apparent in a FDA Voice blog post from July 2016. There, Dr. Gayatri Rao, OOPD Director, noted the increased workload and the effect on meeting internal Office review goals:

[T]he sustained increase in designation requests over the last three years, coupled with the increasing number of incentive programs and competing workload priorities, have forced us to reconsider our internal review target. Reviewing these applications in an efficient and timely manner continues to be a top priority, but to ensure we continue to conduct these reviews with the appropriate level of care and consideration, our current goal is to review on average 75% of designation requests within 120 days of receipt.

Perhaps the soon-to-be-released budget will include some much needed additional funding for OOPD . . . . but we’re not holding our breath.