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Ultra-Rare Disease Approvals By US FDA Could Take More ‘Mechanistic,’ Less ‘Empirical’ Approach

 
• By Nielsen Hobbs

Acting Commissioner Woodcock says ‘we weren’t as flexible as we should be’ and notes that center directors are reviewing the criteria for rare disease assessments.

Watch mechanism (Alamy)
• Source: Alamy

The US Food and Drug Administration is considering how to create more flexibility for the approval of ultra-rare diseases, acting commissioner Janet Woodcock said.

Unlike in Europe, which defines the conditions as those affecting one patient per 50,000 people, there is no statutory definition...

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