CDER real world evidence policy head John Concato said the FDA could approve a new labeled use based only on real-world evidence, but made clear the approach is challenging.
A novel FDA-approved indication for an existing drug can be supported by real-world evidence alone, but the sponsor still must meet the traditional evidentiary standard, Office of Medical Policy Associate Director for RWE John Concato reaffirmed.
The FDA expects that a novel product could be approved based only on real-world evidence.
“The short answer is, ‘yes’ potentially,” Concato said in response to a direct question about whether the FDA would approve...
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.
Pharmaceutical companies should only use AI in evidence generation and reporting where there is “demonstrable value from doing so,” according to England’s health technology assessment body, NICE.
Results from the first pilot real-world evidence study commissioned by the European Medicines Agency to help HTA bodies and payers characterize patients with multiple myeloma have drawn positive feedback.
Overreliance on “problematic” trials while overlooking the value of real-world evidence could affect the development of other rare disease treatments, according to Advanz’s CEO.
The agency also should explain the potential consequences for sponsors if enrollment goals are not met, stakeholders say in comments on the DAP draft guidance. Some commenters urged the FDA to apply waiver criteria flexibly, especially for rare diseases.
As the planet heats up and sea levels rise, the need to reduce carbon emissions is becoming ever more urgent. The life sciences industry is just one of many that are looking to cut the carbon it produces.
An FDA official said the agency is considering allowing MIDD meeting requests to be submitted more often, but must have a plan to deal with the likely increased demand.
