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Promising Pathway Act Would Fill Gap For Rare Disease Treatments That Don’t Fit Accelerated Approval, Advocates Say

 
• By Sue Sutter

Bill would enable two-year provisional approval of drugs to treat life-threatening illnesses, such as ALS, based on substantial evidence of safety and early evidence of efficacy. At a Senate committee hearing, rare disease advocates said it would remedy the FDA’s inconsistent application of regulatory flexibility, but a bioethicist cautions it would lower the bar for approval.

Two doors
ALS patients are willing to accept greater risk because they know 'what is behind door number two,' advocates say. • Source: Shutterstock

A provisional drug approval pathway is needed for serious, life-threatening conditions that lack good biomarkers and cannot use the US Food and Drug Administration’s accelerated approval pathway, rare disease advocates said in urging Congress to pass the Promising Pathway Act.

Patients, advocacy group representatives and industry representatives who spoke at a 26 October Senate Aging Committee hearing, and at a press conference that preceded it, also said the legislation would help to ensure the US Food and Drug Administration consistently

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