Totality Of ‘Breakthrough’ Evidence Guides FDA Designation Decisions
Sponsors should think about more than just a single efficacy data point when assembling their designation requests because the agency looks at the ‘entire puzzle’ in deciding whether ‘breakthrough’ status is warranted.
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Matters ripe for appeal above review division level include denial of request for breakthrough therapy designation, revised guidance states, but general advice – such as how to conduct a clinical research program – are not appealable.
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.