Statistics in Clinical Development of Cancer Drugs

Cancer clinical trials have become increasingly complex, requiring statistical methodologies that are both rigorous and flexible across diverse study designs. This book provides a comprehensive and practice-oriented overview of the statistical methods underpinning modern oncology drug development, covering the full continuum from early-phase studies through confirmatory trials and regulatory submission. Organized by development phase, the text presents principled approaches to dose-escalation and dose-optimization, proof-of-concept decision-making, and master protocol designs. It further details methodologies for late-stage trials, including sample size determination, group-sequential monitoring, time-to-event analysis, multiplicity adjustment, and adaptive designs, with particular attention to challenges such as delayed treatment effects. In addition to confirmatory trial methodology, the book addresses advanced analytical topics, including subgroup evaluation, treatment switching, multi-phase treatment strategies, and bias adjustment techniques. Contemporary issues in oncology research—such as the estimand framework, real-world evidence, seamless and platform trials, and emerging applications of artificial intelligence and machine learning—are also discussed. Accessible yet rigorous, this book is an essential resource for biostatisticians, clinical researchers, and graduate students who want to design smarter trials, make better decisions, and accelerate the development of life-saving cancer therapies.