US, EUROPE DEVELOP COMMON ORPHAN DRUG DESIGNATION FORM

Pharmacy Times, Volume 0, 0

The EuropeanCommission,the EuropeanMedicinesAgency(EMEA), andthe FDA have adopted a common applicationform for pharmaceutical manufacturersseeking orphan drug designationfor medicines developed to treatrare diseases in both the EuropeanUnion (EU) and the United States.

The move eases the orphan drugapplication process by allowing companiesto apply to both jurisdictionssimultaneously. Previously, sponsors oforphan medicines had to submit separateapplications to the EMEA and theFDA, using different submission formatsto satisfy each jurisdiction'srespective regulatory requirements.

About 25 million Americans and 30million individuals living in the EU have>6000 rare diseases. As well as providingan additional incentive to developpromising therapies for rare diseases, itis hoped that the streamlined applicationprocess will help establish a favorableenvironment for the EMA and theFDA to share common experiences andgain an understanding of each others'regulatory systems.