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- 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....
- Perspective
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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