Primer On FDA’s "Breakthrough Therapy" Program

A look at the genesis of the US expedited regulatory pathway for drugs and biologics, its benefits, and how the program is working today.

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...

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