Orphan Drug Designations and Applications Took Off in 2010 While Orphan Drug Approvals Tapered Off

By Kurt R. Karst –      

FDA’s Office of Orphan Products Development (“OOPD”) was busy in 2010!  According to recent data obtained by FDA Law Blog, after a banner year in 2009 in which OOPD surpassed the 2,000 orphan drug designation mark and designated a near-record 160 products for orphan (i.e., rare) diseases and conditions, in 2010, the Office saw a record 323 orphan drug designation requests come through the door and granted 192 designations (for a grand total of 2308).  Despite the intense interest in orphan drugs, however, FDA approved only 14 orphan products in 2010, according to data taken from OOPD’s recently updated orphan drug designation database.  That figure is down from 17 approvals in 2009, and from a 1996 high of 25 approvals. 

It is unclear why, exactly, there was such an increase in orphan drug designations and requests in 2010.  One explanation might be OOPD’s public outreach efforts.  The Office has held a series of workshops on orphan drug designation, and just announced another one last week that will take place from February 28 – March 1, 2011 in Claremont, California.  OOPD also created the Rare Disease Repurposing Database (“RDRD”) in 2010.  The RDRD “offers sponsors a new tool finding special opportunities to develop niche therapies that are already well-advanced through development.” 

The tables below illustrate OOPD’s designation and FDA’s orphan drug approval track record since the enactment of the Orphan Drug Act in 1983.

 
And while we’re on the topic of orphan drug education efforts, we’ll put in a plug for North America’s first industry-led orphan drug conference – the World Orphan Drug Congress USA.  The conference will be held in Washington, D.C. from April 12-15.  Hyman, Phelps & McNamara, P.C. is an associate sponsor of the conference.  Additional information on the conference is available here.