FDA is misapplying tools meant to speed rare disease cures
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.
Why it matters
Regulatory hurdles in rare disease drug development directly impact the speed at which patients receive life-saving therapies.
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.
When I was first diagnosed with limb girdle muscular dystrophy (LGMD), I was devastated to learn there wasn’t any treatment or cure. For those of us living with progressive diseases, time is not neutral. Every month a promising therapy is delayed, we will have irreversible loss of muscle function.
Rare disease drug development is in the midst of an extraordinary era of scientific innovation. Researchers are advancing gene therapies and targeted treatments unimaginable just a decade ago. The Food and Drug Administration has opportunities today with new leadership to modernize, including the utilization of innovative trial designs and tools so the United States continues to lead the world in rare disease drug development.
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