Agency's agreement with rare disease advocacy group will facilitate patient meetings with review divisions, as well as enhance communication of safety information.
The US FDA is partnering with a prominent rare disease advocate to increase the patient voice in drug approval and development in what appears to be another evolution in the agency's patient-focused drug development efforts.
A new memorandum of understanding (MOU) with the National Organization for Rare Disorders will allow collaboration to plan workshops,...
Cardiovascular and Renal Drugs Advisory Committee will consider whether open-label extension data from a randomized trial that failed its primary endpoint, along with a historical control comparison, are enough to support approval in the ultra-rare disease.
The US FDA commissioner also said the hub is looking for an executive director who will work with the CBER and CDER heads, who will serve as co-leads.
Under current law, drugs or biologics must receive rare pediatric disease designation before 1 October to be eligible for a priority review voucher. The sunset provisions have led to a spike in designation requests at the FDA and a push by pediatric and rare disease advocates for an extension, either through a continuing resolution or reauthorizing legislation.
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.
Revised recommendation is based on most recent data on circulating strains in US, where KP.3 is now dominant. Novavax sticks with plans targeting the JN.1 ‘parent strain.’
Pink Sheet reporter and editors discuss the US FDA’s changes to the COVID-19 vaccine indication and its revocation of the vaccine emergency use authorizations, as well as new vaccine coverage and reimbursement questions.
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