April 14, 2015
The FDA recently confirmed that it has been informally instituting a policy to expedite the review of certain breakthrough therapy-designated applications for the past several months. FDA’s informal policy has now found its way into a Manual of Policies and Procedures (MAPP). Under this pathway, FDA review teams are instructed to plan to act at least one month prior to the Prescription Drug User Fee Act (PDUFA) goal date. ’s MAPP describes the characteristics that breakthrough therapy-designated drugs eligible for “expedited review” should have. These include a demonstration of substantial improvement over existing therapies, designation for priority review, and a determination by the review team for a first cycle approval to be likely. Not all breakthrough therapy-designated drugs will receive expedited review as decisions will be made on a case-by-case basis. The MAPP also explains that the Center for Drug Evaluation and Research (CDER) review teams can determine that an expedited review is no longer appropriate and change the review to the default priority review timeline.