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Comment & Response
November 20, 2018

Breakthrough Therapy Designation for New Drugs

Author Affiliations
  • 1Dana-Farber Cancer Institute, Boston, Massachusetts
JAMA. 2018;320(19):2042. doi:10.1001/jama.2018.15938

To the Editor Mr Puthumana and colleagues1 cataloged the trial characteristics of drugs granted Breakthrough Therapy designation by the US Food and Drug Administration (FDA), noting that pivotal trials supporting these approvals commonly lacked randomization, double blinding, and control groups; used surrogate markers; and enrolled small numbers of patients. The Breakthrough Therapy designation was developed with the explicit intent of enabling use of efficient trial designs incorporating the aforementioned elements if early evidence warranted it. When a drug shows a substantial improvement over available therapy early in development, optimizing further study to ensure timely patient access prevents patients from having to wait unnecessarily long for new treatment.

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