Sverdlov Modern Adaptive Randomized Clinical Trials

Statistical and Practical Aspects
Erscheinungsjahr 2015
ISBN: 978-1-4822-3989-8
Verlag: Taylor & Francis
Format: PDF
Kopierschutz: Adobe DRM (»Systemvoraussetzungen)

E-Book, Englisch, 533 Seiten

Reihe: Chapman & Hall/CRC Biostatistics Series

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.

Autoren/Hrsg.

Sverdlov, Oleksandr

Fachgebiete

• Mathematik | Informatik Mathematik Stochastik
• Medizin | Veterinärmedizin Medizin | Public Health | Pharmazie | Zahnmedizin Medizin, Gesundheitswesen Epidemiologie, Medizinische Statistik
• Medizin | Veterinärmedizin Medizin | Public Health | Pharmazie | Zahnmedizin Medizinische Fachgebiete Pharmakologie, Toxikologie

Inhaltsverzeichnis

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 Björn 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. Lô, 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