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Rare Diseases: Growing Understanding Will Unlock Accelerated Approval, FDA Says

 
• By Derrick Gingery

US FDA officials say they need knowledge of the disease to have the confidence to use accelerated approval.

FDA entrance sign 2016

US FDA officials believe sponsors may be able to help eventually make abbreviated approval pathways more accessible for rare disease drugs, but cautioned that preparations should begin now.

Billy Dunn, director of the Division of Neurology Products in FDA's Office of New Drugs, which handles many rare disease treatment applications, offered a hopeful prediction for the advancement of...

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• By Vibha Sharma

The International Council for Harmonisation has identified four new topics that can benefit from global regulatory alignment, with timelines for initiating work to be determined later.