Orphan Drug Approvals Dipped in 2015, While Designations and Designation Requests Continue Upward Trend

February 9, 2016

By Kurt R. Karst

Now that the dust from 2015 has settled, we’re able to take a look at and evaluate the year that was in orphan drug designations and approvals. Our annual review has become quite popular.  We often see the numbers we provide repeated on various websites and cited in published reports (e.g., here).  As we noted last year, in 2014 FDA’s orphan drug program shattered records in each of the three categories we track: orphan drug approvals, orphan drug designations, and orphan drug designation requests submitted to FDA’s Office of Orphan Products Development (“OOPD”). In 2015, FDA’s orphan drug program continued to break new ground with massive numbers of orphan drug designations and orphan drug designation requests. Orphan drug 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 – so-called “repurposed drugs”) dipped slightly from 2014, but 2015 was still the second best year for orphan drug approvals since the enactment of the Orphan Drug Act.

In 2015, FDA’s OOPD received an avalanche of 472 requests for orphan drug designation (5 more than the record set in 2014), and designated an amazing 354 products as orphan drugs (about a 22% increase over 2014). There were 5 or 7 fewer orphan drug approvals in 2015 compared to 2014, depending on whether or not you accept the 2014 number in FDA’s Orphan Drug Designations and Approvals Database or the 2014 number provided by OOPD in a report last year (we use the OOPD report number instead of the database number for 2014).  (There are also other FDA databases and reports – here and here – that show differing numbers, making it difficult to identify a true count.)  For 2015, we added one orphan drug approval for Cysteamine Bitartrate (NDA 203389) to our count because that approval does not seem to be captured in the OOPD database.  Since 1983, FDA has approved about 552 orphan drugs, has granted 3,633 orphan drug designations, and has received 5,210 orphan drug designation requests.   

Below are three tables – one for each metric we track – showing the year-by-year numbers since 1983. A few of the numbers we provided last year changed slightly as FDA has updated the Agency’s designation and approval database.  The most current numbers are in the tables below.



With the growing number of designation requests submitted to FDA’s OOPD each calendar year (about 1.9 per day in 2015 based on a 251-day working year), as well as all of the other programs OOPD is responsible for implementing (e.g., the Humanitarian Use Device Program, the Pediatric Device Consortia, the Orphan Drug Grants Program, and designation requests under the Rare Pediatric Disease Priority Review Voucher Program), you might think that the staff in OOPD has grown accordingly.  You would be wrong.  Despite the massive increase in workload and added responsibilities, OOPD’s staff has remained at about 25 employees for many years now.  Moreover, funding for OOPD has remained relatively flat over the past few years.

While OOPD is a pretty lean, mean working machine, resources can be stretched only so thin. We’re already starting to see some of the strains of the increasing workload on OOPD.  Usually OOPD sets an internal target date for responding to orphan drug designation requests at about 60-90 days from receipt of a request.  We understand that the target date is now at around 90-120 days.  Those folks in charge of the purse strings should really consider dedicating greater funding to OOPD.  But that may not happen soon.  Earlier today we learned that FDA's Fiscal Year 2017 Budget Request is $26,099,000 in budgeting authority for OOPD (see here at pages 105-113).  That figure mirrors the actual Fiscal Year 2016 enacted level.