The selection of study variables and effort required for validation depends on the necessary level of certainty and the impact of potential misclassification on study inference, the US FDA’s final guidance says, a change from the September 2021 draft.
The US Food and Drug Administration’s final guidance on assessing electronic health records and medical claims data for regulatory use gives sponsors more leeway on issues related to study variable validation.
However, the final guidance does not appear to address some of industry’s other concerns about the September 2021 draft, including...
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the potential consequences for sponsors if enrollment goals are not met, stakeholders say in comments on the DAP draft guidance.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.
