The Numbers Are In! 2013 Was An Across-the-Board Record Breaker for Orphan Drugs

By Kurt R. Karst –      

Since we started this blogging gig seven years ago (March 6th was our anniversary) we’ve strived to cover topics of interest to FDA-regulated companies, fellow food and drug and healthcare lawyers and regulatory personnel, as well as people just generally interested in FDA law.  Among other things, this has meant compiling and providing information on topics and happenings not usually reported on by other media outlets.  FDA’s activities with respect to orphan drugs is one such area.  But we’re not talking about reporting on your regular orpan drug approval, such as FDA’s recent approval of IMPAVIDO (miltefosine) to treat leishmaniasis (resulting in the third Tropical Disease Priority Review Voucher) (see here).  No, we’ve tended to look at things in the orphan drug space more broadly.

Over the years we’ve kept track of the success of the Orphan Drug Act (“ODA”), which President Ronald Reagan signed into law on January 4, 1983.  We have measured that success by the number of orphan drug designations submitted to and granted by FDA, and the number of orphan drug approvals resulting from those designations.  The ODA turned 30 last year, and we celebrated that anniversary – as did FDA (see here) – with a February post titled “The Orphan Drug Act: 30 Years and Still Going Strong!”  Although we looked at some new orphan drug metrics in that previous post, we didn’t have any records to report; just some near-records showing that interest in orphan drugs remains quite high.  Well, our 2013 numbers are in . . . . and it was an across-the-board record breaking year for orphan drugs.  That’s quite appropriate for the 30th anniversary year of the ODA. 

In 2013, FDA approved 31 orphan drugs – 5 more than the previous record year (2011) – pushing the total number of orphan drugs approved past the 450 mark.  Both orphan drug designations and orphan drug designation requests skyrocketed in 2013.  There were an astounding 260 orphan drug designations and a similarly astounding 346 orphan drug designation requests submitted to FDA’s Office of Orphan Products Development (“OOPD”) in 2013.  Previously, records for those metrics were set in 2011 with 203 orphan drug designations and in 2010 with 325 orphan drug designation requests, as shown in the tables below.  FDA’s grant rate for orphan drug designation requests has remained steady at about 70% of the requests submitted to the Agency since 1983 (2,989 requests granted of 4,271 submitted to OOPD).

Just as astounding as the 2013 numbers is the fact that the funding for (and the number of personnel in) FDA’s OOPD has reportedly remained relatively steady over the past several years.  Presumably, at some point an infusion of new funding and personnel will need to be made.  Indeed, FDA continues to push orphan drug development and, in addition to OOPD, has a Rare Diseases Program.  In January 2014, FDA held a two-day public workshop on “Complex Issues in Developing Drug and Biological Products for Rare Diseases” (Docket No. FDA-2013-N-0985).  In March 2014, there was an internal FDA all-day training session in which about 100 FDA reviewers participated, titled “Meeting the Challenges of Rare Disease Drug Review: Thinking Outside the Regulatory Box.”  In addition to presentations from FDA officials, Hyman, Phelps & McNamara. P.C.’s Frank J. Sasinowski presented on “Cataloguing FDA’s Flexibility in Regulating Therapies for Persons with Rare Disorders.”  Mr. Sasinowski previously authored a landmark report on flexibility in FDA’s review of potential treatments for orphan diseases (see our previous post here).  He also authored an analysis of FDA's Accelerated Approval/Subpart H/Fast Track approvals (see ourprevious post here).  The growing interest in orphan drug development likely means that many record breaking years are ahead.