Modern Adaptive Randomized Clinical Trials : Statistical and Practical Aspects

This book aids in identifying when to apply adaptive randomization designs in practice. Featuring contributions from statisticians, clinical trialists, and subject-matter experts in academia and industry, the text discusses restricted, covariate-adaptive, response-adaptive, and covariate-adjusted response-adaptive randomization designs, as well as randomized designs with treatment selection. It describes a successful implementation of a single pivotal phase II/III adaptive trial in infants with proliferating hemangioma, addresses practical aspects of phase II dose-ranging studies, and examines statistical monitoring and interim analysis issues in response-adaptive randomized clinical trials.

Is adaptive randomization always better than traditional fixed-schedule randomization? Which procedures should be used and under which circumstances? What special considerations are required for adaptive randomized trials? What kind of statistical inference should be used to achieve valid and unbiased treatment comparisons following adaptive randomization designs?

answers these questions and more.

From novel designs to cutting-edge applications, this book presents several new and key developments in adaptive randomization. It also offers a fresh and critical look at a number of already-classical topics.

Featuring contributions from statisticians, clinical trialists, and subject-matter experts in academia and the pharmaceutical industry, the text:

• Clarifies the taxonomy of the concept of adaptive randomization
• Discusses restricted, covariate-adaptive, response-adaptive, and covariate-adjusted response-adaptive (CARA) randomization designs, as well as randomized designs with treatment selection
• Gives an exposition to many novel adaptive randomization techniques such as brick tunnel randomization, targeted least absolute shrinkage and selection operator (LASSO)-based CARA randomization, multi-arm multi-stage (MAMS) designs, to name a few
• Addresses the issues of statistical inference following covariate-adaptive and response-adaptive randomization designs
• Describes a successful implementation of a single pivotal phase II/III adaptive trial in infants with proliferating hemangioma
• Explores some practical aspects of phase II dose-ranging studies and examines statistical monitoring and interim analysis issues in response-adaptive randomized clinical trials

Modern Adaptive Randomized Clinical Trials: Statistical and Practical Aspects covers a wide spectrum of topics related to adaptive randomization designs in contemporary clinical trials. The book provides a thorough exploration of the merits of adaptive randomization and aids in identifying when it is appropriate to apply such designs in practice.