Modern adaptive randomized clinical trials : statistical and practical aspects PDF
Preview Modern adaptive randomized clinical trials : statistical and practical aspects
Modern Adaptive Randomized Clinical Trials Statistical and Practical Aspects © 2016 by Taylor & Francis Group, LLC K23296_FM.indd 1 6/8/15 12:08 PM Editor-in-Chief Shein-Chung Chow, Ph.D., Professor, Department of Biostatistics and Bioinformatics, Duke University School of Medicine, Durham, North Carolina Series Editors Byron Jones, Biometrical Fellow, Statistical Methodology, Integrated Information Sciences, Novartis Pharma AG, Basel, Switzerland Jen-pei Liu, Professor, Division of Biometry, Department of Agronomy, National Taiwan University, Taipei, Taiwan Karl E. Peace, Georgia Cancer Coalition, Distinguished Cancer Scholar, Senior Research Scientist and Professor of Biostatistics, Jiann-Ping Hsu College of Public Health, Georgia Southern University, Statesboro, Georgia Bruce W. Turnbull, Professor, School of Operations Research and Industrial Engineering, Cornell University, Ithaca, New York Published Titles Adaptive Design Methods in Bayesian Methods in Epidemiology Clinical Trials, Second Edition Lyle D. Broemeling Shein-Chung Chow and Mark Chang Bayesian Methods in Health Economics Adaptive Designs for Sequential Gianluca Baio Treatment Allocation Bayesian Missing Data Problems: EM, Alessandro Baldi Antognini and Data Augmentation and Noniterative Alessandra Giovagnoli Computation Adaptive Design Theory and Ming T. Tan, Guo-Liang Tian, Implementation Using SAS and R, and Kai Wang Ng Second Edition Bayesian Modeling in Bioinformatics Mark Chang Dipak K. Dey, Samiran Ghosh, Advanced Bayesian Methods for Medical and Bani K. Mallick Test Accuracy Benefit-Risk Assessment in Lyle D. Broemeling Pharmaceutical Research and Advances in Clinical Trial Biostatistics Development Nancy L. Geller Andreas Sashegyi, James Felli, and Rebecca Noel Applied Meta-Analysis with R Ding-Geng (Din) Chen and Karl E. Peace Biosimilars: Design and Analysis of Follow-on Biologics Basic Statistics and Pharmaceutical Shein-Chung Chow Statistical Applications, Second Edition James E. De Muth Biostatistics: A Computing Approach Stewart J. Anderson Bayesian Adaptive Methods for Clinical Trials Causal Analysis in Biomedicine and Scott M. Berry, Bradley P. Carlin, Epidemiology: Based on Minimal J. Jack Lee, and Peter Muller Sufficient Causation Mikel Aickin Bayesian Analysis Made Simple: An Excel GUI for WinBUGS Clinical and Statistical Considerations Phil Woodward in Personalized Medicine Claudio Carini, Sandeep Menon, Bayesian Methods for Measures of and Mark Chang Agreement Lyle D. Broemeling © 2016 by Taylor & Francis Group, LLC K23296_FM.indd 2 6/8/15 12:08 PM Clinical Trial Data Analysis using R DNA Methylation Microarrays: Ding-Geng (Din) Chen and Karl E. Peace Experimental Design and Statistical Analysis Clinical Trial Methodology Sun-Chong Wang and Arturas Petronis Karl E. Peace and Ding-Geng (Din) Chen DNA Microarrays and Related Genomics Computational Methods in Biomedical Techniques: Design, Analysis, and Research Interpretation of Experiments Ravindra Khattree and Dayanand N. Naik David B. Allison, Grier P. Page, Computational Pharmacokinetics T. Mark Beasley, and Jode W. Edwards Anders Källén Dose Finding by the Continual Confidence Intervals for Proportions and Reassessment Method Related Measures of Effect Size Ying Kuen Cheung Robert G. Newcombe Elementary Bayesian Biostatistics Controversial Statistical Issues in Lemuel A. Moyé Clinical Trials Empirical Likelihood Method in Survival Shein-Chung Chow Analysis Data Analysis with Competing Risks and Mai Zhou Intermediate States Frailty Models in Survival Analysis Ronald B. Geskus Andreas Wienke Data and Safety Monitoring Committees Generalized Linear Models: A Bayesian in Clinical Trials Perspective Jay Herson Dipak K. Dey, Sujit K. Ghosh, Design and Analysis of Animal Studies in and Bani K. Mallick Pharmaceutical Development Handbook of Regression and Modeling: Shein-Chung Chow and Jen-pei Liu Applications for the Clinical and Design and Analysis of Bioavailability and Pharmaceutical Industries Bioequivalence Studies, Third Edition Daryl S. Paulson Shein-Chung Chow and Jen-pei Liu Inference Principles for Biostatisticians Design and Analysis of Bridging Studies Ian C. Marschner Jen-pei Liu, Shein-Chung Chow, Interval-Censored Time-to-Event Data: and Chin-Fu Hsiao Methods and Applications Design and Analysis of Clinical Trials for Predictive Medicine Ding-Geng (Din) Chen, Jianguo Sun, and Karl E. Peace Shigeyuki Matsui, Marc Buyse, Introductory Adaptive Trial Designs: and Richard Simon A Practical Guide with R Design and Analysis of Clinical Trials with Mark Chang Time-to-Event Endpoints Joint Models for Longitudinal and Time- Karl E. Peace to-Event Data: With Applications in R Design and Analysis of Non-Inferiority Dimitris Rizopoulos Trials Measures of Interobserver Agreement Mark D. Rothmann, Brian L. Wiens, and Reliability, Second Edition and Ivan S. F. Chan Mohamed M. Shoukri Difference Equations with Public Health Medical Biostatistics, Third Edition Applications A. Indrayan Lemuel A. Moyé and Asha Seth Kapadia © 2016 by Taylor & Francis Group, LLC K23296_FM.indd 3 6/8/15 12:08 PM Meta-Analysis in Medicine and Health Sample Size Calculations for Clustered Policy and Longitudinal Outcomes in Clinical Dalene Stangl and Donald A. Berry Research Chul Ahn, Moonseong Heo, and Mixed Effects Models for the Population Song Zhang Approach: Models, Tasks, Methods and Tools Sample Size Calculations in Clinical Marc Lavielle Research, Second Edition Shein-Chung Chow, Jun Shao Modeling to Inform Infectious Disease and Hansheng Wang Control Niels G. Becker Statistical Analysis of Human Growth and Development Modern Adaptive Randomized Clinical Yin Bun Cheung Trials: Statistical and Practical Aspects Oleksandr Sverdlov Statistical Design and Analysis of Stability Studies Monte Carlo Simulation for the Shein-Chung Chow Pharmaceutical Industry: Concepts, Algorithms, and Case Studies Statistical Evaluation of Diagnostic Mark Chang Performance: Topics in ROC Analysis Kelly H. Zou, Aiyi Liu, Andriy Bandos, Multiple Testing Problems in Lucila Ohno-Machado, and Howard Rockette Pharmaceutical Statistics Alex Dmitrienko, Ajit C. Tamhane, Statistical Methods for Clinical Trials and Frank Bretz Mark X. Norleans Noninferiority Testing in Clinical Trials: Statistical Methods for Drug Safety Issues and Challenges Robert D. Gibbons and Anup K. Amatya Tie-Hua Ng Statistical Methods in Drug Combination Optimal Design for Nonlinear Response Studies Models Wei Zhao and Harry Yang Valerii V. Fedorov and Sergei L. Leonov Statistics in Drug Research: Patient-Reported Outcomes: Methodologies and Recent Measurement, Implementation and Developments Interpretation Shein-Chung Chow and Jun Shao Joseph C. Cappelleri, Kelly H. Zou, Statistics in the Pharmaceutical Industry, Andrew G. Bushmakin, Jose Ma. J. Alvir, Third Edition Demissie Alemayehu, and Tara Symonds Ralph Buncher and Jia-Yeong Tsay Quantitative Evaluation of Safety in Drug Survival Analysis in Medicine and Development: Design, Analysis and Genetics Reporting Jialiang Li and Shuangge Ma Qi Jiang and H. Amy Xia Theory of Drug Development Randomized Clinical Trials of Eric B. Holmgren Nonpharmacological Treatments Translational Medicine: Strategies and Isabelle Boutron, Philippe Ravaud, and Statistical Methods David Moher Dennis Cosmatos and Shein-Chung Chow Randomized Phase II Cancer Clinical Trials Sin-Ho Jung © 2016 by Taylor & Francis Group, LLC K23296_FM.indd 4 6/8/15 12:08 PM Modern Adaptive Randomized Clinical Trials Statistical and Practical Aspects Edited by Oleksandr Sverdlov EMD Serono USA © 2016 by Taylor & Francis Group, LLC K23296_FM.indd 5 6/8/15 12:08 PM CRC Press Taylor & Francis Group 6000 Broken Sound Parkway NW, Suite 300 Boca Raton, FL 33487-2742 © 2016 by Taylor & Francis Group, LLC CRC Press is an imprint of Taylor & Francis Group, an Informa business No claim to original U.S. Government works Version Date: 20150515 International Standard Book Number-13: 978-1-4822-3989-8 (eBook - PDF) This book contains information obtained from authentic and highly regarded sources. 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Visit the Taylor & Francis Web site at http://www.taylorandfrancis.com and the CRC Press Web site at http://www.crcpress.com © 2016 by Taylor & Francis Group, LLC Contents Preface xi Contributors xv I Introduction 1 1 An Overview of Adaptive Randomization Designs in Clinical Trials 3 Oleksandr Sverdlov II Restricted Randomization 45 2 Efron’s Biased Coin Design Revisited: Statistical Properties, Randomization-Based Inference and Sequential Monitoring 47 Victoria Plamadeala 3 Adaptive Biased Coins: Achieving Better Balance without Compromising Randomness 55 Alessandro Baldi Antognini1 and Maroussa Zagoraiou2 4 Brick Tunnel and Wide Brick Tunnel Randomization for Studies with Unequal Allocation 83 Olga M. Kuznetsova1 and Yevgen Tymofyeyev2 III Covariate–Adaptive Randomization 115 5 Development of Novel Covariate–Adaptive Randomization Designs 117 Wenle Zhao 6 Optimal Model-Based Covariate–Adaptive Randomization Designs 131 Anthony Atkinson 7 Statistical Inference Following Covariate–Adaptive Randomization: Recent Advances 155 D. Stephen Coad vii © 2016 by Taylor & Francis Group, LLC viii Contents 8 Covariate–Adaptive Randomization with Unequal Allocation 171 Olga M. Kuznetsova1 and Yevgen Tymofyeyev2 IV Response–Adaptive Randomization 199 9 OptimalAllocationDesignsforaMulti-ArmMulti-Objective Clinical Trial 201 David Azriel 10 Response–AdaptiveRandomization:AnOverviewofDesigns and Asymptotic Theory 221 Li-Xin Zhang 11 Statistical Inference Following Response–Adaptive Randomization 251 Yanqing Yi1 and Xikui Wang2 12 Sample Size Re-Estimation in Adaptively Randomized Clinical Trials with Missing Data 269 Ruitao Lin and Guosheng Yin 13 Some Caveats for Outcome Adaptive Randomization in Clinical Trials 287 Peter F. Thall1, Patricia S. Fox1 and J. Kyle Wathen2 V Covariate-Adjusted Response–Adaptive Randomization 307 14 Efficient and Ethical Adaptive Clinical Trial Designs to Detect Treatment–Covariate Interaction 309 Seung Won Hyun1, Tao Huang2 and Hongjian Zhu3 15 Longitudinal Covariate-Adjusted Response–Adaptive Randomization: Impact of Missing Data 327 Tao Huang1 and Hongjian Zhu2 16 Targeted Covariate-Adjusted Response–Adaptive LASSO-Based Randomized Controlled Trials 345 Antoine Chambaz1, Mark J. van der Laan2 and Wenjing Zheng2,3 17 Covariate-Balanced Bayesian Adaptive Randomization: Achieving Tradeoff between Inferential and Ethical Goals in Small and Moderate Size Trials 371 Ying Yuan and Jing Ning © 2016 by Taylor & Francis Group, LLC Contents ix VI Randomized Designs with Treatment Selection 387 18 Multi-Arm Multi-Stage Designs for Clinical Trials with Treatment Selection 389 James Wason 19 Sequential Elimination in Multi-Arm Selection Trials 411 Christina Yap1, Xuejing Lin2 and Ying Kuen K. Cheung2 20 Accounting for Parameter Uncertainty in Two-Stage Designs for Phase II Dose–Response Studies 427 Emma McCallum1 and Bj¨orn Bornkamp2 VII Application and Practical Aspects 451 21 A Single Pivotal Adaptive Trial in Infants with Proliferating Hemangioma: Rationale, Design Challenges, Experience and Recommendations 453 Stephane Heritier1, Caroline C. Morgan-Bouniol2, Serigne N. Lˆo3, Stephanie Gautier4 and Jean Jacques Voisard5 22 Practical Implementation of Dose–Response Adaptive Trials 483 Tom Parke and Martin Kimber 23 Statistical Monitoring of Data in Response–Adaptive Randomized Clinical Trials 505 Paul Gallo © 2016 by Taylor & Francis Group, LLC © 2016 by Taylor & Francis Group, LLC
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