FDA and European Medicines Agency agree to accept a single orphan drug designation annual report

In recognition of World Rare Disease Day which took place yesterday, the US Food and Drug Administration and the European Medicines Agency (EMEA) last Friday announced a more streamlined process to help regulators better identify and share information throughout the development process of orphan drug and biologic products, which are developed specifically to treat rare medical conditions. Both agencies have agreed to accept the submission of a single annual report from sponsors of orphan drug and biologic products designated by both the USA and the European Union.

Currently, if an orphan product was granted designation on the exact same day in both the USA and EU, sponsors must submit separate reports to their respective regulatory agency. The use of one annual report will also benefit sponsors by eliminating the duplication of efforts and by simplifying the process that meets the annual reporting requirements of both the USA and EU for orphan designated products.

'This process provides benefits for both agencies,' said Timothy Cote, Director of the FDA's Office of Orphan Products Development. 'Additionally, it reduces the duplication involved for sponsors in reporting to two separate regulatory agencies,' he added.