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

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.