FDA and EPA Simplify Orphan Drug Development Process

4 March 2010 (Last Updated March 4th, 2010 18:30)

The US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have agreed to accept the submission of a single annual report from sponsors of orphan drug and biologic products designated by the US and the EU. The more streamlined process, announced in recognition of

The US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have agreed to accept the submission of a single annual report from sponsors of orphan drug and biologic products designated by the US and the EU.

The more streamlined process, announced in recognition of World Rare Disease Day, eliminates the duplication of efforts by the sponsors to submit separate reports to the two agencies if they were granted orphan designation on the same day in the US and EU.

The single annual report will contain information on the development of orphan medical products, including a review and status of ongoing clinical studies, a description of the investigation plan for the coming year and anticipated or existing problems in the process that may impact their designation as an orphan product.

The optional new process for submission will not introduce any additional regulatory requirements and each regulatory body will conduct its own review and assessment of the annual report.