FDA Orphan Drug Designations Are On the Rise

By Kurt R. Karst –      

2008 was a banner year for orphan drug designations.  FDA’s Office of Orphan Products Development (“OOPD”), which, since September 2007, has been under the leadership of Timothy Coté, M.D., M.P.H., designated a record 165 products for orphan (i.e., rare)  diseases and conditions.  FDA also approved 15 orphan products in 2008.  The 2008 orphan drug designation figure continues a recent trend in an uptick in designations.  From 2004 to 2007, OOPD granted 130, 122, 141, and 119 designations, respectively.  From 1983 to 2003, the greatest number of orphan drug designations in a single year was 95.  The table below illustrates OOPD’s designation and FDA’s orphan drug approval track record since the enactment of the Orphan Drug Act of 1983 (“ODA”).

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.    

Orphan drug designation qualifies a company for several benefits under the ODA, as amended.  These benefits include a 7-year period of orphan drug exclusivity upon product approval (which generally prevents FDA from approving another firm’s version of the “same drug” for the same disease or condition for 7 years), a tax credit for certain clinical testing expenses for the orphan drug, written guidance on the non-clinical and clinical studies needed to obtain marketing approval of an orphan drug, and orphan drug grants.  In addition, there are certain user fee, pediatric assessment (i.e., FDC Act § 505B(k)), and “extra-statutory” benefits for orphan drugs (e.g., the quantity and quality of evidence needed to support the approval of an orphan product seems to reflect the understanding that there is a limited subject population in which to study the product – see ICH/FDA, Guideline for Industry: The Extent of Population Exposure to Assess Clinical Safety: For Drugs Intended for Long-term Treatment of Non-Life-Threatening Conditions, at 4 (Mar. 1995)).