FDA to unlock interim endpoints for accelerated approvals

FDA Commissioner Scott Gottlieb Thursday announced the agency’s intention to broaden the use of intermediate clinical endpoints to support accelerated approvals, teed up the agency’s release of new antibiotic drug development guidance and gave an early look at use of a new pathway for qualifying biomarkers. He also endorsed in principle proposals to create a new pathway for regulation of diagnostics, including laboratory-developed tests (LDTs).

Speaking at a hearing of the House Energy & Commerce Committee marking the first anniversary of the 21st Century Cures Act, Gottlieb noted that accelerated approval is usually based on surrogate endpoints such as tumor shrinkage. He said the agency is developing policy to clarify its ability to grant accelerated approval based on an “outsized” effect demonstrated in a small trial on a clinical endpoint such as survival. Such endpoints would be considered interim clinical endpoints and could be the basis for accelerated approval, he said. ...