The Orphan Drug Act: 30 Years and Still Going Strong!

By Kurt R. Karst & Frank J. Sasinowski –

The Orphan Drug Act (“ODA”), which President Ronald Reagan signed into law on January 4, 1983, turned 30 years old in January.  The milestone came just days after the death of actor Jack Klugman, who has been credited as being an important driving force behind the passage of the law.  Klugman, the star of “Quincy, M.E.,” testified before Congress on the ODA, and two episodes of the show focused on the issue of rare diseases: “Seldom Silent, Never Heard” (1981) and “Give Me Your Weak” (1982). 

FDA celebrated the ODA’s 30th anniversary with a series of articles and interviews (see here and here).  And the National Organization for Rare Disorders (“NORD”) put out a great spread, including ODA milestones and 2013 events at which the anniversary will be celebrated, including at Rare Disease Day 2013 (February 28, 2013).  Representative Henry Waxman (D-CA), who was instrumental in the passage of the ODA, also commemorated the anniversary and Klugman’s passing (see here).  

We at Hyman, Phelps & McNamara, P.C. are celebrating the ODA’s 30th anniversary as well . . . . and here on the FDA Law Blog in our own special way.  For several years now we have taken stock of the year that was in orphan drug designations and approvals.  For example, in 2011, we noted that 2010 saw a record number of orphan drug designation applications.  Also in 2011, we saw the publication of a landmark report authored by former Chairman of the NORD Board of Directors and HP&M Director, Frank J. Sasinowski, on the flexibility in FDA’s review of potential treatments for patients with rare diseases (see our previous post here), as well as FDA’s publication of 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).  In 2012, we celebrated a record number of orphan drug approvals and orphan drug designations in 2011.   In 2013, we don’t have any new records to report on reached in 2012; however, there are some near records, showing that interest in orphan drug remains quite high.  And we’ve looked at some new orphan drug metrics based on information available from FDA’s searchable database for Orphan Designated and or Approved Products that we think readers will find interesting.

In 2012, FDA surpassed the 400 orphan drug approval mark, ending up with 25 orphan drug approvals – just one shy of the record 26 orphan drug approvals set in 2011.  Both orphan drug designations and orphan drug designation requests were down in 2012, with 188 orphan drug designations and 264 orphan drug designation requests.  Records for those metrics were set in 2011 and 2010, respectively, as shown in the table below.  Overall, FDA has granted designation to nealy 70% of the requests submitted to the Agency since 1983. 

The new metrics we decided to look at this year, as reflected in the last two columns of the table below, required some significant number crunching based on data from FDA’s database.  The first metric is the number of drugs designated in a particular year that ended up with an approval.  Thus, for example, in 1984, FDA designated 41 products as orphan drugs, and 24 of those designations ended up with an orphan drug approval.  The second metric is perhaps of greater interest.  It shows the number of days – expressed as an average, median, and range – between orphan drug designation and approval for products designated in that particular year.  Overall, it takes an average of about 4 years and a median of about 3 years from designation to approval.  Excluding the zeros in the table below, the quicked designation-to-approval was 2 days (for epirubicin as a component of adjuvant therapy in patients with evidence of axillary node tumor involvement following resection of primary breast cancer), and the longest designation-to-approval was 9,529 days (for factor XIII concentrate (human) for the routine prophylactic treatment of congenital factor XIII deficiency). It should be noted that the figures in the last column include a small percentage – about 2.9% – of products that were approved before designation was granted.  Those anomalies occurred in 1984, 1985, 1987, 1991, 2008, 2010, and 2011, and, in most cases, before the ODA was amended to require that a company request designation before submission of a marketing application for the orphan drug.  Instead of counting those days in the negative, we counted them towards the overall figures. 

Outside of the 2012 statistics, it is also worth noting that 2012 saw significant litigation involving the ODA, as well as the first ever rescission of orphan drug exclusivity – for  Octapharma USA, Inc.’s WILATE (von Willebrand Factor/Coagulation Factor VIII Complex (Human)) for von Willebrand disease (see our previous post here).  The first piece of litigation was brought against FDA by K-V Pharmaceutical Company to “restore” orphan drug exclusivity for the pre-term birth drug MAKENA (hydroxyprogesterone caproate) Injection.  As we previously reported, the case was dismissed in the DC District Court, but is on appeal to the DC Circuit (Case No. 12-5349).  The second piece of litigation was brought against FDA by Depomed, Inc., which, as we previously reported, filed a Complaint in the DC District Court challenging FDA’s denial of orphan drug exclusivity for GRALISE (gabapentin) Tablets.   In January, Depomed filed a Motion for Summary Judgment.  Last week, FDA filed a Motion to Dismiss/Motion for Summary Judgment in the case.