Accelerated Approval Conversion Could Mean ‘Breakthrough’ Loss
Sponsors of ‘breakthrough therapy’-designated investigational oncology products may need to identify a niche subpopulation that remains underserved when a competing product with accelerated approval in the same indication completes confirmatory trials.
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The orthostatic hypotension treatment midodrine has been on the market for more than 20 years despite the failure of postmarketing studies to confirm clinical benefit; an advisory committee soon will weigh the future of AMAG’s preterm birth drug Makena, which failed its confirmatory trial required as a condition of accelerated approval.
Sometimes data supporting drug's initial approval can serve as confirmatory evidence for full approval of a subsequent indication based on an intermediate endpoint, US FDA Oncology Center of Excellence Director Pazdur says; proposed framework discussed at Friends of Cancer Research meeting would establish more systematic approach to streamlining supplemental approvals.
Clinical data supporting a designation request should come from a product under development, not from a study using an earlier formulation, CBER reps say. Rolling submissions help ensure that potential manufacturing and facility concerns are identified early.