Primer On FDA’s "Breakthrough Therapy" Program

The designation resulted, in part, from a November 2011 cancer research conference sponsored by the Friends of Cancer Research and the Engelberg Center for Health Care Reform at the Brookings Institution. A panel of agency representatives and stakeholders discussed proposals for expediting the development of drugs with early evidence of large treatment effects. (See Also see "Expedited Approval Pathway Concept Draws Strong Support But Different Proposals" - Pink Sheet, 5 December, 2011..)

The concept of a progressive approval pathway also was included in the Biotechnology Industry Organization’s list of “big ideas” for PDUFA V reauthorization. Under this proposal, a drug could be...