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Stretching accelerated approval

Why FDA wants to make overall survival an endpoint for accelerated approval

FDA wants to stretch its accelerated approval pathway beyond traditional surrogate endpoints to include overall survival that has been demonstrated in small trials, such as exploratory studies.

The change is a response to advances in targeted drug development that are making it increasingly possible to identify potential breakthroughs early in clinical development.

It also reflects intense patient demand for access to new medicines as rapidly as possible.

FDA Commissioner Scott Gottlieb said the problem FDA is trying to solve is how to determine what the agency should do “when we have a targeted drug, introduced in a properly selected group of patients, which has an outsized effect on overall survival in a rare or deadly cancer, but where that benefit is seen in a small trial.”

Gottlieb was speaking at a November congressional hearing on implementation of the 21st Century Cures Act. He described FDA’s ideas for getting potential breakthroughs to patients more rapidly, including by granting accelerated approval based on preliminary OS data. The case is especially strong for cancer treatments, Gottlieb said.

Accelerated approval, which was created in the 1990s in response to the AIDS crisis, allows FDA to approve drugs based on the demonstration of efficacy as measured by a surrogate or interim endpoint that is likely to

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