STAT·4 min read·medium

FDA is misapplying tools meant to speed rare disease cures

K
Kat Bryant Knudson
FDA is misapplying tools meant to speed rare disease cures
AI Summary

A patient advocate argues that the FDA is misapplying the platform technology designation, a tool intended to accelerate drug development for rare diseases. The author claims that inconsistent regulatory flexibility is creating uncertainty and delaying life-saving treatments.

Bryant Knudson is the founder and CEO of The Speak Foundation, a patient-led nonprofit advancing care, research, advocacy, and treatment development for people living with limb girdle muscular dystrophy.

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