The Interface between Randomized Controlled Trials and Real-World Evidence Studies - Jie Chen;Naitee Ting;Feinan Lu

Integrate RCTs and real-world evidence for stronger regulatory submissions

Since the passage of the 21st Century Cures Act, the landscape of clinical research has transformed dramatically. Today, the integration of real-world data (RWD) and real-world evidence (RWE) with traditional randomized controlled trials (RCTs) is not just innovative - it is essential for modern drug development and regulatory decision-making.

This comprehensive volume bridges the methodologies of conventional RCTs and emerging RWE studies, offering systematic approaches for leveraging diverse data sources throughout the clinical development lifecycle. Grounded in regulatory statistics yet forward-looking in scope, the book explores Stein's continuum of study designs - from traditional RCTs to pragmatic trials and observational studies - and demonstrates how RWD can enhance trial feasibility, inform outcome selection, provide external controls for single-arm trials, and strengthen post-approval safety monitoring.

Readers will find detailed explorations of:

Designed as both a reference and a graduate-level textbook, this book serves clinical researchers such as clinicians and biostatisticians, regulatory professionals, public health scientists, and industry practitioners seeking to harness the power of RWD and RWE to generate credible, actionable evidence for modern medicine.