The US Food and Drug Administration’s (FDA’s) February 2026 announcement, made public in the New England Journal of Medicine (NEJM), outlines that one adequate and well-controlled pivotal study, together with confirmatory evidence, will now be the agency’s default basis for product approval. This differs from the FDA’s previous general expectation of two independent, adequate, and well-controlled pivotal trials to demonstrate substantial evidence of effectiveness.

FDA’s Rare Pediatric Disease (RPD) Priority Review Voucher (PRV) program was established in 2012 as a financial incentive to encourage the development of therapeutics for rare pediatric diseases. The program officially ended as of September 30, 2024 due to a sunset clause. 

On December 15, 2025, the FDA published the final guidance on the “Use of Real-World Evidence to Support Regulatory Decision-Making for Medical Devices” Use of Real-World Evidence to Support Regulatory Decision-Making for Medical Devices  Dr. Makary said that the FDA is, “removing unnecessary barriers that have prevented us from using powerful real-world evidence to get life-changing treatments to patients faster.”

FDA’s draft guidance streamlines biosimilar approval by reducing the need for comparative efficacy studies (CES). The guidance provides revised criteria outlining the circumstances in which conducting CES may be unnecessary for a Biologics License Application submitted through the 351(k) biosimilar and interchangeable biosimilar pathway.