FDA & EMEA Adopt Common Orphan Drug Designation Application Form to Ease Sponsor Burden; Independent Reviews will Continue

November 28, 2007

Earlier this week, the European Medicines Agency (“EMEA”) announced that EMEA and FDA have adopted a common application form for sponsors seeking orphan drug designation in the European Union (“EU”) and the U.S.  The move to a common application form was anticipated when FDA and EMEA announced earlier this year that the two regulatory authorities agreed to expand regulatory cooperation under a September 2003 Confidentiality Arrangement to include orphan drugs (see 7/18/07 FDA Law Blog post). According to the November 26th EMEA press release:

To be eligible for receiving orphan incentives, sponsors of orphan medicines have had to submit separate applications for orphan designation to the EMEA and to the FDA using different submission formats to satisfy the respective regulatory requirements. These different formats have imposed an additional burden on sponsors. Hence, the parties have agreed to harmonise the application form to simplify part of the orphan medicines designation process.

This common application format will now allow sponsors to apply to both jurisdictions at the same time with one application. A common format will also establish a favourable environment for the EMEA and FDA to share common experiences and gain an understanding of the similarities and differences of the process of obtaining orphan designation in the two regulatory systems.

FDA has not yet formally announced the adoption of a common designation application form; however, earlier this month the Agency issued a Federal Register notice requesting emergency Office of Management and Budget processing of a proposed collection of information to enable FDA to jointly announce with EMEA the adoption of the common EMEA/FDA application form at the EU-wide Administrative Simplification Workshop on November 28, 2007.  FDA also recently updated its website to include the new common application form – Form FDA 3671.

The Orphan Drug Act of 1983, as amended (“ODA”), amended the FDC Act to provide drug manufacturers with incentives to develop and market products for rare (i.e., orphan) diseases and conditions — most notably, a 7-year period of market exclusivity.  To be eligible for this exclusivity, the approved drug must have been designated as an orphan drug by FDA.  The ODA provides two routes for obtaining designation of a drug for an orphan disease or condition.  A request can be made either on the basis that a product is intended to treat a disease or condition that has a prevalence of 200,000 or less affected persons in the U.S, or if a disease or condition affects over 200,000 individuals, then if a sponsor can show that there is no reasonable expectation that the costs of developing and making available the drug will be recovered from sales in the U.S.  Most orphan drug designations are based on a U.S. prevalence under 200,000 persons. 

The EU orphan drug system, established in December 1999 under European Commission Regulation No. 141/2000 (later amended under European Commission Regulation No. 847/2000), is largely based on the ODA; however, there are differences between the two systems.  In particular, a rare disease is defined as one affecting fewer than 5 in 10,000 people in the EU.  This means that a disease or condition could be considered orphan in the EU, but not in the U.S.  The common orphan drug designation application form contains sections for requirements unique to each regulatory authority.

Although the creation of the common application form might decrease the burden on sponsors seeking orphan drug designation, it is not intended to signal a greater likelihood of dual FDA/EMEA designation.  According to the EMEA announcement, the “EMEA and the FDA will still conduct independent reviews of such submissions to assure the data submitted meet the legal and scientific requirements of their respective jurisdictions.”


  • FDA Office of Orphan Products Development website
  • EMEA orphan drugs website

Categories: Drug Development