US FDA asked a very typical question of its advisory committee about Cytokinetics’ omecamtiv mecarbil: is there ‘substantial evidence’ of efficacy? But in this case the issue wasn’t a trial that was too small or clearly flawed – it was whether such a large, rigorous trial should have shown more than it did.
There is nothing unusual about the US Food & Drug Administration convening an advisory committee to weigh in on an application that relies on a single pivotal trial, with the primary question being whether the sponsor has met its burden of showing “substantial evidence” of efficacy.
The agency wrestled with that very question in a remarkable tandem of meetings for Amylyx Pharmaceuticals, Inc.’s ALS treatment...
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.
