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FDA Unveils Fast-Track Approval for Rare Disease Drugs

9/4/2025

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Summary

Story summary

The U.S. FDA proposed a new approval process for drugs targeting rare diseases with fewer than 1,000 patients.
Eligibility will require a lack of adequate alternative therapies and may permit single-arm clinical trials for effectiveness evidence.
FDA gene therapy chief Vinay Prasad introduced the "plausible mechanism pathway" to expedite approvals for tailored therapies.

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Story summary

The U.S. FDA proposed a new approval process for drugs targeting rare diseases with fewer than 1,000 patients.
Eligibility will require a lack of adequate alternative therapies and may permit single-arm clinical trials for effectiveness evidence.
FDA gene therapy chief Vinay Prasad introduced the "plausible mechanism pathway" to expedite approvals for tailored therapies.