Rare diseases are finally getting their due in the US Food and Drug Administration’s advisory committee system.
On 12 December, the FDA announced the establishment of a new expert panel – the Genetic Metabolic Diseases Advisory Committee...
Genetic Metabolic Diseases Get Their Own US FDA Advisory Committee
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In a first for rare diseases, a new standing committee will provide advice and recommendations on technical, scientific and policy issues related to medical products for genetic metabolic diseases.
Learn what's happening at the US FDA. On the go.
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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.
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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.
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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.