New approval paths are needed, says Woodcock

FDA director of drugs Janet Woodcock says she is so optimistic that biomedical science and the war on cancer will produce “startlingly better” therapies that the research community should develop new approval paths.

At the Conference on Clinical Cancer Research sponsored by the Engelberg Center on Health Care Reform and Friends of Cancer Research in November, she said that, for example, with many metastatic solid tumors, if many complete responses are seen in an initial Phase 1 trial, “we should all stop and think about what to do next.”

Woodcock was on a panel that wrote the issue brief  “Development Paths for New Drugs with Large Treatment Effects Seen Early.” It noted: “Randomized Phase 2 and Phase 3 trials provide the most reliable evidence,” but  they “can be extremely time consuming, expensive and difficult to perform.”

That brief proposed an alternative pathway to full approval consisting of a small randomized Phase 2b trial, with about 120 to 150 patients. The purpose, said the brief, “is to demonstrate a large treatment effect in a small number of patients, while maintaining the same statistical significance currently used in trials that seek small benefits.”
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