In 14-6 vote, advisory committee said use of external control comparison to contextualize single-arm trial data demonstrated efficacy and reliance on preclinical data as confirmatory evidence was justified.
A majority of US Food and Drug Administration advisory committee members on 4 October voted that US WorldMeds, LLC provided sufficient evidence to conclude that eflornithine (DFMO) improves event-free survival in pediatric patients with high-risk neuroblastoma despite a nontraditional data package.
In a 14-6 vote by the Oncologic Drugs Advisory Committee, panelists in the majority said they found the sponsor’s use...
A real-world evidence study also does not meet the regulatory standard for an adequate and well-controlled clinical investigation, the agency said. The FDA should allow the accelerated approval drug to remain available like other treatments that failed their confirmatory trials, Intercept said.
Recent and upcoming US FDA advisory committee meetings and a summary of the topics covered.
New marketing authorization applications filed with the European Medicines Agency's human medicines committee (the CHMP) for review under the EU's centralized procedure. Updated monthly.
Proposal to have panelists rate the strength of efficacy and safety evidence on a numeric scale also draws support in written comments on ways to optimize the advisory committee process.
A real-world evidence study also does not meet the regulatory standard for an adequate and well-controlled clinical investigation, the agency said. The FDA should allow the accelerated approval drug to remain available like other treatments that failed their confirmatory trials, Intercept said.
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Recent and upcoming US FDA advisory committee meetings and a summary of the topics covered.
