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February 2018

Deflazacort—New Costs of an Old Medicine

Author Affiliations
  • 1Department of Physiology and Functional Genomics, University of Florida, Gainesville
  • 2Department of Biochemistry and Molecular Biology, University of Florida, Gainesville
JAMA Neurol. 2018;75(2):143-144. doi:10.1001/jamaneurol.2017.3704

Two thousand dollars vs $89 000. That is the cost of deflazacort per year before and after the recent US Food and Drug Administration (FDA) approval for symptomatic treatment in Duchenne muscular dystrophy (DMD).1 On February 9, 2017, the FDA granted approval for labeled use of deflazacort to mitigate DMD symptoms.2 Prior to its approval in the United States, deflazacort had been available overseas for decades; in accordance to the Federal Food, Drug, and Cosmetic Act,3 it was then legal to obtain it overseas. (Briefly, this law states that patients are allowed to import foreign drugs if several criteria are met, including an absence of commercialization in the United States.) After the domestic approval and commercialization of deflazacort, it is now illegal to purchase foreign deflazacort, which was previously affordably priced.

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