Stakeholders want wider FDA use of accelerated approval

At least two stakeholders called for greater use of accelerated approval by FDA at a Tuesday hearing held by the U.S. House of Representatives Energy and Commerce Committee as part of the committee's 21st Century Cures initiative. Frank Sasinowski, director of the National Organization for Rare Disorders, said FDA should increase the use of accelerated approval by considering it for each new therapy and recommended greater use of intermediate clinical endpoints for accelerated approval. Sarah Radcliffe, EVP for health for the Biotechnology Industry Organization, called on FDA to clarify the process for validating novel endpoints and to discuss with sponsors the use of surrogates or intermediate clinical endpoints earlier in development.

Radcliffe also stressed that the President's Council of Advisors on Science and Technology (PCAST) urged FDA to use accelerated approval more broadly, beyond HIV/AIDS and oncology, which could result in "fewer, smaller, or shorter clinical trials." The E&C committee held Tuesday's hearing to review recommendations from PCAST for FDA included in a 2012 report for the agency (see BioCentury Extra, Sept. 25, 2012). ...