'Progressive Approval' Coming? US FDA Considers Accelerated Approval Without Surrogates

Commissioner Gottlieb tells Congress that concept could be used for some clinical endpoints when a large benefit is seen in a small trial, which could benefit rare disease drug development.

Capitol House

The US FDA is exploring a potentially dramatic expansion of the accelerated approval mechanism. Under the concept, products showing a substantial, early effect on a clinical endpoint – as opposed a surrogate endpoint used for the existing accelerated approval process – could qualify for the new expedited pathway.

Commissioner Scott Gottlieb said Nov. 30 during a House Energy and Commerce Subcommittee on Health hearing that targeted therapies sometimes show early "outsized benefits," particularly in oncology.

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