The 2014 Numbers Are In: FDA’s Orphan Drug Program Shatters Records

February 16, 2015

By Kurt R. Karst

Earlier this month in a post concerning the Orange Book we mentioned our love of data and discussed how solid figures can reveal some interesting conclusions.  After all, it’s rather difficult – and dangerous – to come to a conclusion without having as many facts and as much data as possible.  Or, as Sir Arthur Conan Doyle wrote in The Adventure of the Copper Beeches, one of the Sherlock Holmes series of stories: “‘Data! Data! Data!’ he cried impatiently.  ‘I can’t make bricks without clay.’”  Indeed!  And the clay we have to play with today to make some bricks are the latest data from FDA’s Office of Orphan Products Development (“OOPD”) on orphan drug designations and approvals.

Although FDA’s agency-wide performance management system, known as FDA-TRACK, provides some information on orphan drug designations and approvals, that system is not up-to-date.  So we have to get our data from other sources – primarily (though not exclusively) from FDA’s Orphan Drug Designations and Approvals database.  As we’ve done in some previous years, we culled information from that database, which OOPD populates with data on a rather frequent basis.   The database is also constantly being refined to better reflect what orphan drug designations have been granted, what orphan drugs have been approved, and what the relevant periods of orphan drug exclusivity apply to.   We understand that the database will be revamped soon (perhaps later this year) to make it a more useful and user-friendly resource. 

The three metrics we’ve historically followed are: (1) the number of orphan drug designation requests received by OOPD; (2) the number of orphan drug designation requests granted by OOPD; and (3) the number of orphan drugs approved.  In 2014, records were shattered for all three metrics, with an astounding 467 designation requests (a nearly 35% increase over 2013), an astonishing 293 orphan drug designations granted (a nearly 13% increase over 2013), and a whopping 49 orphan drug approvals (a 53% increase over 2013).  Wow!  That’s an amazing output for FDA’s orphan drug program (and, in particular, for OOPD).

Below are three tables – one for each metric – showing the year-by-year numbers since 1983.



When we add up all of the numbers since 1983, FDA has approved 511 orphan drugs, granted 3,280 orphan drug designations, and received 4,738 orphan drug designation requests.  Of the 511 approvals, some drugs have been approved for more than a single rare disease, and sometimes a single orphan drug designation has been the platform for multiple orphan drug approvals (and multiple periods of 7-year exclusivity).

So what does it all mean?  Well, clearly orphan drugs are trending up – way up!  And there’s no indication of a slowdown any time soon.  (In fact, we understand that OOPD is already on pace to break the 2014 record for the number of orphan drug designation requests received by the Office.)  The data also show that the Orphan Drug Act has been an overwhelming success (for both patients and the drug and biotechnology industries).  Of course, a successful program breeds copycats.  We’ve seen that with the creation of orphan drug programs in other countries modeled after the Orphan Drug Act.  It’s also happening on out own backyard, however.  Consider, for example, the Generating Antibiotic Incentives Now Act (“GAIN Act”) (FDC Act § 505E), and the Dormant Therapies Act provisions included in the draft 21st Century Cures Act (see our previous post here).  The roots for both of those items can almost certainly be traced back to the Orphan Drug Act.