Alternative evidence in drug development and regulatory science

While Randomized Controlled Trials (RCTs) and Real-World Evidence (RWE) have traditionally served complementary roles, the role of RWE has been expanded by legislation and regulatory mandates. This expanded role reflects the evolution of clinical trial endpoints that include precision medicine, pharmacogenomics, and health economics and outcomes research. These clinical trial endpoints influence both regulatory approval, reimbursement, and health policy. The process of drug development using RCTs is slow, expensive, and generally lacks patient diversity. RWE now ranges from its traditional role in Phase IV pharmacovigilance to external controls in prospective clinical trials for FDA submissions. In the context of accepted standards for RCTs and management of mega-data through artificial intelligence, the role of RWE has evolved to advance patient care in a timely and resource-efficient manner.