News Briefs

FDA and European Union
Adopt Shared Form

The United States and European drug regulatory agencies announced this week that they have adopted a single application form that may be submitted to both agencies by sponsors seeking orphan designation of medicines in the European Union and the U.S. The initiative is aimed at simplifying the process of obtaining orphan status for medicines intended for rare diseases in both jurisdictions.

“This is a good first step,” said NORD President Abbey S. Meyers. “It will make the orphan product designation process more efficient and less costly. Since many of the companies developing products for rare diseases are small, that’s important.”

In the U.S., legislation was adopted in 1983 (the Orphan Drug Act) that provides federal financial incentives for companies developing treatments for rare diseases, which are defined as ones affecting fewer than 200,000 Americans. Similar legislation was adopted by the European Union in 1999.

To date, sponsors seeking to have medicines designated as orphans to qualify for the incentives have had to submit different application forms to the U.S. Food and Drug Administration (FDA) and its European Union counterpart agencies, the European Commission and the European Medicines Agency (EMEA). The shared application includes a section for common information required by both the EMEA and the FDA. In addition, it has sections for requirements unique to each agency.