Agency plans guidance on whether observational studies can provide real-world evidence to support regulatory decisions about drug effectiveness; however, RWE framework includes critical questions about retrospective studies, and reflects agency concerns about transparency.
The US FDA’s real-world evidence (RWE) framework takes a cautious approach to the use of observational studies to support new efficacy claims for approved drugs.
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The FDA struck an upbeat tone during its annual Generic Drugs Forum despite an unprecedented period of cutbacks and disruptions and reiterated a desire to continue meeting user fee goals.
Ascertaining the novelty and benefits of Makary’s new drug approval idea – along with what it would take to implement it – requires more clarity on the types of studies he envisions being used to grant and confirm “plausible mechanism’ approvals.
The Pink Sheet's list of EU centralized approvals of new active substances has been updated to include four new products, one of which is Tivdak, Pfizer/Genmab’s treatment for previously treated recurrent or metastatic cervical cancer.
Industry feedback on a European Medicines Agency pilot that introduced a “stepwise” approach to pediatric investigation plans was positive, but the future of the new model will depend on the outcome of the pharmaceutical legislation negotiations, the agency says.
The preliminary White House 2026 budget plan cuts agency funding, but not so sharply that user fees are excluded from collection.
The Pink Sheet's list of EU centralized approvals of new active substances has been updated to include four new products, one of which is Tivdak, Pfizer/Genmab’s treatment for previously treated recurrent or metastatic cervical cancer.
Industry feedback on a European Medicines Agency pilot that introduced a “stepwise” approach to pediatric investigation plans was positive, but the future of the new model will depend on the outcome of the pharmaceutical legislation negotiations, the agency says.
