Published Online: Tuesday, January 1, 2008

The European Commission, the European Medicines Agency (EMEA), and the FDA have adopted a common application form for pharmaceutical manufacturers seeking orphan drug designation for medicines developed to treat rare diseases in both the European Union (EU) and the United States.

The move eases the orphan drug application process by allowing companies to apply to both jurisdictions simultaneously. Previously, sponsors of orphan medicines had to submit separate applications to the EMEA and the FDA, using different submission formats to satisfy each jurisdiction's respective regulatory requirements.

About 25 million Americans and 30 million individuals living in the EU have >6000 rare diseases. As well as providing an additional incentive to develop promising therapies for rare diseases, it is hoped that the streamlined application process will help establish a favorable environment for the EMA and the FDA to share common experiences and gain an understanding of each others' regulatory systems.

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