2009 – Another Banner Year for Orphan Drug Designations

By Kurt R. Karst –      

FDA’s Office of Orphan Products Development (“OOPD”) surpassed the 2,000 orphan drug designation mark in 2009 and designated a near-record 160 products for orphan (i.e., rare) diseases and conditions.  FDA also approved 17 orphan products in 2009, according to data taken from OOPD’s new orphan drug designation database.  The 2009 orphan drug designation figure continues a trend in an increase in designations in recent years.  As we previously reported, in 2008, OOPD designated a record 165 products. 

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.

Coincidentally, the Tufts Center for the Study of Drug Development just issued an Impact Report (subscription required) analyzing orphan drug designations.  According to that report:

• During the 2000s, orphan products comprised 22% of all new molecular entities (NMEs) and 31% of all significant biologics (SBs) receiving U.S. marketing approval.

• Orphan products receiving priority review status rose from 35% of all orphan NMEs in 2000-02 to 50% in 2006-08; during the same time the share of orphan SBs receiving priority review status rose from 17% to 67%.

• While biotech firms during the 2000s garnered, on average, about one-third of all orphan drug approvals, they received just over 50% of orphan drug designations.

• Sponsors engaged in clinical development funded through orphan grants reported that 22% of their programs led to approvals, which compares with a clinical approval success rate of 16% among mainstream drug developers.