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? Modern Adaptive Randomized Clinical Trials: Statistical and Practical Aspects 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.
Preface Contributors Introduction An Overview of Adaptive Randomization Designs in Clinical Trials Oleksandr Sverdlov Restricted Randomization Efron's Biased Coin Design Revisited: Statistical Properties, Randomization-Based Inference, and Sequential Monitoring Victoria Plamadeala Adaptive Biased Coins: Achieving Better Balance without Compromising Randomness Alessandro Baldi Antognini and Maroussa Zagoraiou Brick Tunnel and Wide Brick Tunnel Randomization for Studies with Unequal Allocation Olga M. Kuznetsova and Yevgen Tymofyeyev Covariate-Adaptive Randomization Development of Novel Covariate-Adaptive Randomization Designs Wenle Zhao Optimal Model-Based Covariate-Adaptive Randomization Designs Anthony Atkinson Statistical Inference Following Covariate-Adaptive Randomisation: Recent Advances D. Stephen Coad Covariate-Adaptive Randomization with Unequal Allocation Olga M. Kuznetsova and Yevgen Tymofyeyev Response-Adaptive Randomization Optimal Allocation Designs for a Multi-Arm Multi-Objective Clinical Trial David Azriel Response-Adaptive Randomization: An Overview of Designs and Asymptotic Theory Li-Xin Zhang Statistical Inference Following Response-Adaptive Randomization Yanqing Yi and Xikui Wang Sample Size Re-Estimation in Adaptively Randomized Clinical Trials with Missing Data Ruitao Lin and Guosheng Yin Some Caveats for Outcome Adaptive Randomization in Clinical Trials Peter F. Thall, Patricia S. Fox, and J. Kyle Wathen Covariate-Adjusted Response-Adaptive Randomization Efficient and Ethical Adaptive Clinical Trial Designs to Detect Treatment-Covariate Interaction Seung Won Hyun, Tao Huang, and Hongjian Zhu Longitudinal Covariate-Adjusted Response-Adaptive Randomization: Impact of Missing Data Tao Huang and Hongjian Zhu Targeted Covariate-Adjusted Response-Adaptive LASSO-Based Randomized Controlled Trials Antoine Chambaz, Mark J. van der Laan, and Wenjing Zheng Covariate-Balanced Bayesian Adaptive Randomization: Achieving Tradeoff Between Inferential and Ethical Goals in Small and Moderate Size Trials Ying Yuan and Jing Ning Randomized Designs with Treatment Selection Multi-Arm Multi-Stage Designs for Clinical Trials with Treatment Selection James Wason Sequential Elimination in Multi-Arm Selection Trials Christina Yap, Xuejing Lin, and Ying Kuen K. Cheung Accounting for Parameter Uncertainty in Two-Stage Designs for Phase II Dose-Response Studies Emma McCallum and Bjorn Bornkamp Application and Practical Aspects A Single Pivotal Adaptive Trial in Infants with Proliferating Hemangioma: Rationale, Design Challenges, Experience, and Recommendations Stephane Heritier, Caroline C. Morgan-Bouniol, Serigne N. Lo, Stephanie Gautier, and Jean Jacques Voisard Practical Implementation of Dose-Response Adaptive Trials Tom Parke and Martin Kimber Statistical Monitoring of Data in Response-Adaptive Randomized Clinical Trials Paul Gallo