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January 14, 2020

Reform at the FDA—In Need of Reform

Author Affiliations
  • 1Johns Hopkins Bloomberg School of Public Health, Baltimore, Maryland
JAMA. 2020;323(2):123-124. doi:10.1001/jama.2019.20538

On December 23, 2016, the US Food and Drug Administration (FDA) announced the approval of nusinersen, the first drug for the devastating disease spinal muscular atrophy. The approval was based on an interim analysis of 82 patients in a single randomized trial; 40% of treated children improved, compared with none in the control group. The FDA had requested that the interim analysis be conducted. A press release emphasized the speed of agency action, with the review division director saying, “We worked hard to review this application quickly.”1

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