FDASIA 2012 and the origins of the expedited programmes

United States, federal statute. The Food and Drug Administration Safety and Innovation Act (FDASIA) was signed on 9 July 2012 (Public Law 112-144). Section 902 created Breakthrough Therapy designation; other titles made the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act permanent, reauthorised prescription drug user fees for a fifth time, and created generic and biosimilar user fees. Primary text: the Congress.gov record of S.3187.

The expedited programmes have three statutory parents. The Prescription Drug User Fee Act of 1992 let the FDA charge companies fees in return for review-time goals, which is where the ten-month standard and six-month priority review clocks come from. The Food and Drug Administration Modernization Act of 1997 (FDAMA) created Fast Track designation and wrote accelerated approval into statute as section 506. FDASIA added Breakthrough Therapy for drugs whose early clinical data suggest substantial improvement over available therapy, giving them intensive FDA guidance and senior staff involvement. The FDA's expedited programmes guidance (2014) describes how the four fit together.

What it changed for oncology: cancer drugs receive the largest share of breakthrough designations, and the combination of breakthrough, priority review and accelerated approval is how single-arm response-rate data in a biomarker-defined population became a route to market in a few years. Critics note that designation is announced in press releases and can move share prices while adding no evidence, and that a substantial minority of breakthrough drugs later show no survival benefit.