Advisory committee’s recommendations on secondary endpoints for pivotal studies reflects patient and caregiver comments during the open public hearing about the disease’s clinical complications, including ear infections and sleep apnea, and challenges performing activities of daily living.
A US FDA advisory committee’s recommendations on secondary endpoints for achondroplasia drug studies highlight the impact that patient and caregiver testimony can have on clinical trial design.
At their May 11 meeting, the agency’s Pediatric Advisory Committee and Endocrinologic and Metabolic Drugs Advisory Committee said pivotal studies...
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.
