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Totality Of ‘Breakthrough’ Evidence Guides FDA Designation Decisions

Executive Summary

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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Duke's McClellan: Changing Drug Development Policy From Outside FDA

In an interview, Margolis Center for Health Policy's Mark McClellan and Gregory Daniel talk about their recent move from Brookings to Duke, the breadth and impact of their work with FDA on drug development issues, and opportunities for potential future collaborations under PDUFA VI.

Dispute Resolution Strategies: FDA Clarifies Which Issues Can Be Appealed

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.

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.

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