FDA smooths reviews of rare disease treatments
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The Food and Drug Administration on Monday laid out a new pathway aimed at spurring novel individualized treatments targeting rare diseases.
Why it matters: Officials said the draft rules would make it easier for industry to develop more types of promising new treatments for rare diseases, including with CRISPR gene-editing technology.
Driving the news: The agency is establishing a way to evaluate rare disease treatments that can't be put through traditional clinical trials because the patient populations are too small.
- Treatments with a similar mechanism could be grouped together to allow for a single approval that can be adapted based on different genetic mutations, for example.
The big picture: The move was inspired by "Baby KJ," an infant born with a rare metabolic disease who became first person to be successfully treated with a personalized CRISPR therapy last year at Children's Hospital of Philadelphia.
What they're saying: FDA commissioner Marty Makary said Monday that rare diseases would no longer be an "afterthought" at the agency.
- He said 30 million Americans have a rare disease. "For too long they have been under-appreciated, under-recognized, the research has been underfunded, because our system is built for common diseases," he added.
- Tracy Beth Høeg, acting head of the FDA's drug center, said the draft guidance is "going to inspire industry to start developing these treatments in a way that we haven't seen before."
