Requests for Orphan Drug Designation Explode in 2009; But Designation Success Rate is Low

By Kurt R. Karst & Frank J. Sasinowski –      

We recently reported on the banner year FDA’s Office of Orphan Products Development (“OOPD”) had in 2009, surpassing the 2,000 orphan drug designation mark and designating a near-record 160 products for orphan (i.e., rare) diseases and conditions.  That followed a record 165 designations in 2008, which we reported on here.  As additional information has been made available, however, the 2009 designation figure is tempered by what appears to be a low success rate. 

According to our data (shown in the tables below), OOPD received 250 orphan drug designation requests in 2009, but only granted 160 designations, for a designation rate of 64%.  That is below the historical (cumulative) success rate of 70%, and far below the 2008 success rate of 89%.  To be fair, not all designations submitted in a particular calendar year are acted on in that year.  For example, a designation request submitted in December might not be acted on until February or March of the next calendar year.  And in some cases, it takes a couple of years for OOPD to grant designation because the data and information submitted with an orphan drug designation request are insufficient to support designation at that time, but later-acquired information adequately addresses OOPD concerns.  Nevertheless, the 2009 figure seems to validate a comment we made about the 2008 designation figure: 

It is unclear whether the recent increase in orphan drug designations reflects a shift in OOPD policy, or whether the Office is receiving more designation requests.  Our experience is that the depth and thoroughness of OOPD review has certainly not decreased.  In fact, OOPD seems to be placing a greater emphasis on the scientific rationale and medical plausibility components of an orphan drug designation request than it has in the past.  Thus, we believe the increase in orphan drug designations is largely a function of the quantity of requests OOPD is receiving.    

As orphan drug designation requests continue to stream into OOPD, companies need to be reminded that quality counts, and that OOPD appears to be scrutinizing designation requests more so today than it has in the past.  We’ll see how the 2010 designation figures pan out when we present them next year.