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Friday, November 15, 2013

Expediting Drug Development — The FDA's New “Breakthrough Therapy” Designation

Today's post was shared by NEJM and comes from

Many people with serious or life-threatening illnesses for which there are no satisfactory treatments are understandably eager to gain access to new therapies and are willing to trade off greater certainty about a drug's performance for speed of access. 

Because the typical clinical drug-development program takes about 7 years, during which a substantial body of safety and efficacy data is generated, the Food and Drug Administration (FDA) has long-standing expedited pathways available for drugs being studied for such illnesses. However, many patients and their advocates continue to believe that clinical development is sometimes prolonged beyond what is necessary. 

During the congressional considerations leading up to passage of the FDA Safety and Innovation Act of 2012 (FDASIA), a variety of provisions related to this theme were put on the table. 

When the bill was enacted, two modifications of the Federal Food, Drug, and Cosmetic Act addressed the issue of drug development for serious illnesses: a new “breakthrough therapy” designation for investigational drugs and expansion of the statute regarding accelerated approval

The breakthrough-therapy designation has since been introduced into the FDA portfolio of expedited programs for serious conditions....
Rachel E. Sherman, M.D., M.P.H., Jun Li, J.D., Ph.D., Stephanie Shapley, M.B.A., Melissa Robb, R.N., and Janet Woodcock, M.D.
N Engl J Med 2013; 369:1877-1880November 14, 2013DOI: 10.1056/NEJMp1311439
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