The Peripheral and Central Nervous System Drug Advisory Committee (PCNSDAC) of the US Food and Drug Administration (FDA) makes recommendations for the approval or denial of drugs that treat nervous system diseases. Before issuing recommendations, the PCNSDAC hears public testimony about these drugs in an open forum.1 For example, eteplirsen, a treatment for Duchenne muscular dystrophy, was approved after 52 public speakers provided testimony at the PCNSDAC meeting.2 After approval of eteplirsen, critics spoke out against its approval, citing weak evidence from a 12-patient trial based on a surrogate end point.3 Because public speakers overwhelmingly spoke in favor of eteplirsen,3 critics raised concerns regarding the influence of public testimony on FDA drug approval.4
Arthur W, Austin J, Wayant C, Vassar M. Association of Conflicts of Interest for Public Speakers for the Peripheral and Central Nervous System Drugs Advisory Committee of the US Food and Drug Administration With Their Statements. JAMA Neurol. Published online December 17, 2018. doi:10.1001/jamaneurol.2018.3997
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