FDA Accelerated Approval For Rare Diseases Again Under Congressional Pressure
This article was originally published in The Pink Sheet Daily
Letter being circulated by Rep. Bilirakis states the agency’s draft expedited approval guidance does not provide clarity for rare disease sponsors to use accelerated approval.
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Drugs intended to prevent a serious condition or reduce likelihood of disease progression may qualify, as may products intended to diminish serious adverse events associated with current treatment.
Final guidance on expedited regulatory pathways suggests FDA is open to accelerated approval for some acute conditions; agency also provides examples outside of HIV/cancer arena where surrogate and intermediate clinical endpoints have supported approval.
Draft appropriations committee report instructs FDA to undertake tasks both profound (offer a better accounting of how user fees are spent) and routine (meet with patient communities for specific diseases).