In February 2026, the Food and Drug Administration (FDA) outlined a meaningful evolution in the Agency’s evidentiary approach to drug approval: establishing one adequate and well-controlled trial, supported by confirmatory evidence, as the default basis for marketing authorization. This position...
Seth DePuy
Navigating rare disease drug development requires coordinated, adaptive strategies as science and regulation evolve in parallel. Small, geographically dispersed patient populations, limited natural history data, and heterogeneous disease progression make trial design and execution unusually complex. At the same time,...
Since PDUFA VII was signed into law in September 2022, the U.S. Food and Drug Administration (FDA) has introduced a series of initiatives aimed at addressing the persistent challenges of rare disease development. Collectively, these efforts form a more complete...
Determining the right regulatory pathway for a drug product is a critical early decision that can shape the entire development strategy. While many products may appear to fit the generic model, nuances in formulation, dosage form, route of administration, or...