The Call for Progress in Evidence-Based Medicine

Randomized controlled trials (RCTs) are foundational in evidence-based medicine but are often limited by their reliance on controlled conditions and highly selective patient populations, which may not reflect real-world healthcare outcomes. This commentary highlights the value of pragmatic controlled trials (PCTs) as a complementary method for evaluating real-world effectiveness (RWE). By employing Bayesian statistical approaches, PCTs enable comprehensive assessment of healthcare interventions under everyday conditions, bridging the gap between experimental efficacy and practical applicability. A three-dimensional strategy is proposed, addressing efficacy, effectiveness, and value to improve the relevance and generalizability of healthcare research. The commentary also emphasizes the need for endpoint-specific risk profiling to optimize patient outcomes and decision-making. Integrating PCTs into evidence-based medicine provides a robust framework for advancing healthcare efficiency and delivering patient-centered care in diverse populations.