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US FDA’s Makary Floats Vague ‘Plausible Mechanism’ Approval Pathway For Rare Diseases

 
• By Sarah Karlin-Smith

Ascertaining the novelty and benefits of Makary’s new drug approval idea – along with what it would take to implement it – requires more clarity on the types of studies he envisions being used to grant and confirm “plausible mechanism’ approvals.

black and white image of fog
Makary's vision for a new rare disease approval pathway appears to still be a bit foggy. (Shutterstock)
Key Takeaways
  • New FDA Commissioner Makary said he is going to roll out a new pathway for rare disease drugs that would allow for approval without a randomized controlled trial, "based on a plausible mechanism." Approvals would be on a "conditional basis."

US Food and Drug Administration Commissioner Martin Makary said he plans to roll out a new drug approval pathway for...

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