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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."
  • Without more detail, including on the types of studies Makary would require preapproval, experts said its difficult to assess whether Makary's idea represents a truly novel approval option at FDA and what the pros and cons of that option would be for patients.
  • There was also some skepticism that post-market tools like registries would be able to provide the data necessary to support these approvals post-market.

US Food and Drug Administration Commissioner Martin Makary said he plans to roll out a new drug approval pathway for rare diseases that would allow certain medicines to come to...

Rare disease patient advocates have long clamored for more flexibility from FDA to get treatments approved, particularly for ultra-rare conditions which may affect hundreds of people...

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