Expanded Access Programs Eyed For Data-Gathering Purposes
Protocol-driven approach could generate reliable data about investigational drug's efficacy and safety in patients who do not qualify for clinical trials, PhRMA's Moscicki says; Johns Hopkins' Sharfstein cautions this could turn expanded access programs into burdensome studies that take away from primary purpose of providing access to investigational therapies.
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Independent assessment finds positive perceptions of expanded access program but calls on agency to do more to correct manufacturer misperceptions about adverse events and clarify when data from expanded access would support labeling extensions.
US FDA’s approval of Advanced Accelerator Applications’ radiopharmaceutical for gastroenteropancreatic neuroendocrine tumors was driven by robust Phase III study results in midgut carcinoid tumors and data from an investigator-initiated, expanded access study in the Netherlands that enrolled patients with a host of other neuroendocrine tumor types.
Commissioner Gottlieb’s recent remarks on pending federal 'Right-to-Try' legislation were his most positive to date; he pushed back on earlier versions that would have removed FDA from process.