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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Accelerated Approval: Midodrine Experience Shows Regulatory Challenge US FDA Faces With Makena
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.
Supplemental Oncology Approvals: 'Reverse Accelerated Approval,' Not Lowering The Bar
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.
When Seeking ‘Breakthrough’ Status, Beware Of Manufacturing Changes
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.