‘Threading The Needle:’ Finding A Regulatory Foundation For Ultra Rare Disease Development

 
• By Derrick Gingery

Sometimes even the US FDA’s regulatory flexibility may not be enough to approve rare disease treatments in very small populations, but agency officials worry that creating a new pathway could be problematic.

needle and thread
Changing FDA standards could increase the risks for reimbursement, Peter Marks said. • Source: Shutterstock

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