The Year of the Orphan; Orphan Drug Designations and Approvals Hit All-Time High in 2011

February 8, 2012

By Kurt R. Karst –      

It’s about that time of year again when we do some number crunching and take stock of the year that was in orphan drug designations and approvals.  And what a year it was!  FDA not only approved a record 26 marketing applications for products for orphan (i.e., rare) diseases and conditions, but FDA’s Office of Orphan Products Development (“OOPD”) granted a record 199 orphan drug designation requests.   OOPD also received 306 orphan drug designation requests in 2011, which is only second to the record of 323 designation requests set in 2010 (see our previous post here).  Of the 3,659 orphan drug designation requests submitted to FDA since the enactment of the Orphan Drug Act in 1983, OOPD has granted 2,507 of them, or about 68.5%. 

The tables below show OOPD’s designation and FDA’s orphan drug approval track record.  (Additional historical information on the year-by-year designation/approval numbers since the enactment of the Orphan Drug Act in 1983 is available here.) 


In addition to the record numbers of orphan drug designations and approvals, 2011 also saw a change in OOPD leadership with the installment of Gayatri Rao, M.D., J.D. as Acting Director (see our previous post here), and the first U.S. Conference on Rare Diseases and Orphan Products.  At the conference, Chairman of the NORD Board of Directors and Hyman, Phelps & McNamara, P.C. Director, Frank J. Sasinowski announced the release of a landmark report he authored on the flexibility in FDA’s review of potential treatments for patients with rare diseases (see our previous post here).  Shortly thereafter, FDA issued a proposed rule intended to clarify regulatory provisions and make minor improvements to address issues that have arisen since the Agency promulgated its orphan drug regulations in December 1992 (see our previous post here).  FDA also issued a report to Congress with findings and recommendations to improve the current regulatory/scientific armamentarium to facilitate the development of products for rare and neglected diseases (see our previous post here). 

The 2011 orphan drug designation and approval numbers may be difficult marks to beat; however, FDA appears to be off to a decent start in 2012 with 8 designations and 2 approvals in January.

REMINDER: HP&M is hosting FDA Appeals – Improving Your Odds of Success: Trends, Expectations, Strategies, a webinar on March 21, 2012, 12:30 – 2:00 p.m. ET.  Click here to register.