
Setting a New Pace: Real-World Evidence is Finding Its Footing in HEOR
In 2021, something unprecedented occurred in the pharmaceutical arena—the US Food and Drug Administration (FDA) approved a medication based exclusively on retrospective real-world data (RWD), without any evidence from clinical trials.Vijoice (alpelisib) had been administered on a compassionate-use basis to patients with severe symptoms of PIK3CA-related overgrowth spectrum, and a study of data from their charts led to the FDA’s accelerated approval.1That protocol will be less likely to raise eye...