FDA and EMA To Accept a Single Annual Report for Orphan Designated Drugs

The US Food and Drug Administration and the European Medicines Agency (EMA) have agreed to 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 drugs and biologic products designated by the US and the European Union.

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

The single annual report will provide information to both agencies on the development of orphan drugs, including a review and status of ongoing clinical studies, a description of the investigation plan for the coming year, and anticipated or current problems in the process that may affect their designation as an orphan product. The single annual report submission is voluntary and will apply only to sponsors who have obtained an orphan designation status for their product from both the FDA and EMA.

The optional new process for submission will not introduce any additional regulatory requirements. Each regulatory body will conduct its own review and assessment of the annual report to assure the information meets all the legal and scientific requirements of each agency. The FDA and EMA will exchange the annual reports electronically through a secure portal. Starting February 28, 2010, sponsors may send a single orphan drug designation annual report to both agencies.