Randomization, Masking, and Allocation Concealment

266 Pages·2018·5.81 MB·English

by  Vance Berger

Randomization,
Masking, and Allocation
Concealment
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 Clinical Basic Statistics and Pharmaceutical
Trials, Second Edition Statistical Applications, Second Edition
Shein-Chung Chow and Mark Chang James E. De Muth
Adaptive Designs for Sequential Bayesian Adaptive Methods for
Treatment Allocation Clinical Trials
Alessandro Baldi Antognini Scott M. Berry, Bradley P. Carlin,
and Alessandra Giovagnoli J. Jack Lee, and Peter Muller
Adaptive Design Theory and Bayesian Analysis Made Simple:
Implementation Using SAS and R, An Excel GUI for WinBUGS
Second Edition Phil Woodward
Mark Chang
Bayesian Designs for Phase I–II
Advanced Bayesian Methods for Clinical Trials
Medical Test Accuracy Ying Yuan, Hoang Q. Nguyen,
Lyle D. Broemeling and Peter F. Thall
Analyzing Longitudinal Clinical Trial Data: Bayesian Methods for Measures
A Practical Guide of Agreement
Craig Mallinckrodt and Ilya Lipkovich Lyle D. Broemeling
Applied Biclustering Methods for Big Bayesian Methods for Repeated Measures
and High-Dimensional Data Using R Lyle D. Broemeling
Adetayo Kasim, Ziv Shkedy,
Bayesian Methods in Epidemiology
Sebastian Kaiser, Sepp Hochreiter,
Lyle D. Broemeling
and Willem Talloen
Bayesian Methods in Health Economics
Applied Meta-Analysis with R
Gianluca Baio
Ding-Geng (Din) Chen and Karl E. Peace
Bayesian Missing Data Problems: EM,
Applied Surrogate Endpoint Evaluation
Data Augmentation and Noniterative
Methods with SAS and R
Computation
Ariel Alonso, Theophile Bigirumurame,
Ming T. Tan, Guo-Liang Tian,
Tomasz Burzykowski, Marc Buyse,
and Kai Wang Ng
Geert Molenberghs, Leacky Muchene,
Nolen Joy Perualila, Ziv Shkedy,
and Wim Van der Elst
Published Titles
Editor-in-Chief Bayesian Modeling in Bioinformatics Clinical Trial Optimization Using R
Shein-Chung Chow, Ph.D., Professor, Department of Biostatistics and Bioinformatics, Dipak K. Dey, Samiran Ghosh, Alex Dmitrienko and Erik Pulkstenis
Duke University School of Medicine, Durham, North Carolina and Bani K. Mallick
Cluster Randomised Trials:
Benefit-Risk Assessment in Second Edition
Series Editors
Pharmaceutical Research and Richard J. Hayes and Lawrence H. Moulton
Byron Jones, Biometrical Fellow, Statistical Methodology, Integrated Information Sciences,
Development
Novartis Pharma AG, Basel, Switzerland Computational Methods in Biomedical
Andreas Sashegyi, James Felli,
Research
Jen-pei Liu, Professor, Division of Biometry, Department of Agronomy,
and Rebecca Noel
Ravindra Khattree and Dayanand N. Naik
National Taiwan University, Taipei, Taiwan
Benefit-Risk Assessment Methods in
Karl E. Peace, Georgia Cancer Coalition, Distinguished Cancer Scholar, Senior Research Scientist Computational Pharmacokinetics
Medical Product Development: Bridging
and Professor of Biostatistics, Jiann-Ping Hsu College of Public Health, Anders Källén
Georgia Southern University, Statesboro, Georgia Qualitative and Quantitative Assessments
Confidence Intervals for Proportions
Qi Jiang and Weili He
Bruce W. Turnbull, Professor, School of Operations Research and Industrial Engineering, and Related Measures of Effect Size
Cornell University, Ithaca, New York Bioequivalence and Statistics in Clinical
Robert G. Newcombe
Pharmacology, Second Edition
Published Titles Controversial Statistical Issues in
Scott Patterson and Byron Jones
Clinical Trials
Adaptive Design Methods in Clinical Basic Statistics and Pharmaceutical Biosimilar Clinical Development:
Shein-Chung Chow
Trials, Second Edition Statistical Applications, Second Edition Scientific Considerations and New
Data Analysis with Competing Risks
Shein-Chung Chow and Mark Chang James E. De Muth Methodologies
and Intermediate States
Adaptive Designs for Sequential Bayesian Adaptive Methods for Kerry B. Barker, Sandeep M. Menon,
Ronald B. Geskus
Treatment Allocation Clinical Trials Ralph B. D’Agostino, Sr., Siyan Xu, and Bo Jin
Data and Safety Monitoring Committees
Alessandro Baldi Antognini Scott M. Berry, Bradley P. Carlin, Biosimilars: Design and Analysis of
in Clinical Trials, Second Edition
and Alessandra Giovagnoli J. Jack Lee, and Peter Muller Follow-on Biologics
Jay Herson
Adaptive Design Theory and Bayesian Analysis Made Simple: Shein-Chung Chow
Design and Analysis of Animal Studies
Implementation Using SAS and R, An Excel GUI for WinBUGS Biostatistics: A Computing Approach
in Pharmaceutical Development
Second Edition Phil Woodward Stewart J. Anderson
Shein-Chung Chow and Jen-pei Liu
Mark Chang
Bayesian Designs for Phase I–II Cancer Clinical Trials: Current and
Design and Analysis of Bioavailability
Advanced Bayesian Methods for Clinical Trials Controversial Issues in Design and
and Bioequivalence Studies, Third Edition
Medical Test Accuracy Ying Yuan, Hoang Q. Nguyen, Analysis
Shein-Chung Chow and Jen-pei Liu
Lyle D. Broemeling and Peter F. Thall Stephen L. George, Xiaofei Wang,
Analyzing Longitudinal Clinical Trial Data: Bayesian Methods for Measures and Herbert Pang Design and Analysis of Bridging Studies
Jen-pei Liu, Shein-Chung Chow,
A Practical Guide of Agreement Causal Analysis in Biomedicine and
and Chin-Fu Hsiao
Craig Mallinckrodt and Ilya Lipkovich Lyle D. Broemeling Epidemiology: Based on Minimal
Applied Biclustering Methods for Big Bayesian Methods for Repeated Measures Sufficient Causation Design & Analysis of Clinical Trials for
and High-Dimensional Data Using R Lyle D. Broemeling Mikel Aickin Economic Evaluation & Reimbursement:
An Applied Approach Using SAS & STATA
Adetayo Kasim, Ziv Shkedy, Bayesian Methods in Epidemiology Clinical and Statistical Considerations in
Iftekhar Khan
Sebastian Kaiser, Sepp Hochreiter, Lyle D. Broemeling Personalized Medicine
and Willem Talloen Claudio Carini, Sandeep Menon, and Mark Chang Design and Analysis of Clinical Trials
Bayesian Methods in Health Economics
for Predictive Medicine
Applied Meta-Analysis with R Gianluca Baio Clinical Trial Data Analysis Using R
Shigeyuki Matsui, Marc Buyse,
Ding-Geng (Din) Chen and Karl E. Peace Ding-Geng (Din) Chen and Karl E. Peace
Bayesian Missing Data Problems: EM, and Richard Simon
Applied Surrogate Endpoint Evaluation Data Augmentation and Noniterative Clinical Trial Data Analysis Using R and SAS,
Design and Analysis of Clinical Trials with
Methods with SAS and R Computation Second Edition
Time-to-Event Endpoints
Ariel Alonso, Theophile Bigirumurame, Ming T. Tan, Guo-Liang Tian, Ding-Geng (Din) Chen, Karl E. Peace,
Karl E. Peace
Tomasz Burzykowski, Marc Buyse, and Kai Wang Ng and Pinggao Zhang
Geert Molenberghs, Leacky Muchene, Design and Analysis of Non-Inferiority Trials
Clinical Trial Methodology
Nolen Joy Perualila, Ziv Shkedy, Mark D. Rothmann, Brian L. Wiens,
Karl E. Peace and Ding-Geng (Din) Chen
and Wim Van der Elst and Ivan S. F. Chan
Published Titles
Difference Equations with Public Health Inference Principles for Biostatisticians
Applications Ian C. Marschner
Lemuel A. Moyé and Asha Seth Kapadia
Interval-Censored Time-to-Event Data:
DNA Methylation Microarrays: Methods and Applications
Experimental Design and Statistical Ding-Geng (Din) Chen, Jianguo Sun,
Analysis and Karl E. Peace
Sun-Chong Wang and Arturas Petronis
Introductory Adaptive Trial Designs:
DNA Microarrays and Related Genomics A Practical Guide with R
Techniques: Design, Analysis, and Mark Chang
Interpretation of Experiments
Joint Models for Longitudinal and Time-
David B. Allison, Grier P. Page,
to-Event Data: With Applications in R
T. Mark Beasley, and Jode W. Edwards
Dimitris Rizopoulos
Dose Finding by the Continual
Measures of Interobserver Agreement
Reassessment Method
and Reliability, Second Edition
Ying Kuen Cheung
Mohamed M. Shoukri
Dynamical Biostatistical Models
Medical Biostatistics, Fourth Edition
Daniel Commenges and
A. Indrayan
Hélène Jacqmin-Gadda
Meta-Analysis in Medicine and
Elementary Bayesian Biostatistics
Health Policy
Lemuel A. Moyé
Dalene Stangl and Donald A. Berry
Emerging Non-Clinical Biostatistics in
Methods in Comparative Effectiveness
Biopharmaceutical Development and
Research
Manufacturing
Constantine Gatsonis and Sally C. Morton
Harry Yang
Mixed Effects Models for the Population
Empirical Likelihood Method in
Approach: Models, Tasks, Methods
Survival Analysis
and Tools
Mai Zhou
Marc Lavielle
Essentials of a Successful Biostatistical
Modeling to Inform Infectious Disease
Collaboration
Control
Arul Earnest
Niels G. Becker
Exposure–Response Modeling: Methods
Modern Adaptive Randomized Clinical
and Practical Implementation
Trials: Statistical and Practical Aspects
Jixian Wang
Oleksandr Sverdlov
Frailty Models in Survival Analysis
Monte Carlo Simulation for the
Andreas Wienke
Pharmaceutical Industry: Concepts,
Fundamental Concepts for New Clinical Algorithms, and Case Studies
Trialists Mark Chang
Scott Evans and Naitee Ting
Multiregional Clinical Trials for
Generalized Linear Models: A Bayesian Simultaneous Global New Drug
Perspective Development
Dipak K. Dey, Sujit K. Ghosh, and Joshua Chen and Hui Quan
Bani K. Mallick
Multiple Testing Problems in
Handbook of Regression and Modeling: Pharmaceutical Statistics
Applications for the Clinical and Alex Dmitrienko, Ajit C. Tamhane,
Pharmaceutical Industries and Frank Bretz
Daryl S. Paulson
Published Titles Published Titles
Noninferiority Testing in Clinical Trials: Statistical Analysis of Human Growth
Difference Equations with Public Health Inference Principles for Biostatisticians
Issues and Challenges and Development
Applications Ian C. Marschner
Tie-Hua Ng Yin Bun Cheung
Lemuel A. Moyé and Asha Seth Kapadia
Interval-Censored Time-to-Event Data:
Optimal Design for Nonlinear Response Statistical Design and Analysis of Clinical
DNA Methylation Microarrays: Methods and Applications
Models Trials: Principles and Methods
Experimental Design and Statistical Ding-Geng (Din) Chen, Jianguo Sun,
Valerii V. Fedorov and Sergei L. Leonov Weichung Joe Shih and Joseph Aisner
Analysis and Karl E. Peace
Sun-Chong Wang and Arturas Petronis Patient-Reported Outcomes: Statistical Design and Analysis of
Introductory Adaptive Trial Designs:
Measurement, Implementation and Stability Studies
DNA Microarrays and Related Genomics A Practical Guide with R
Interpretation Shein-Chung Chow
Techniques: Design, Analysis, and Mark Chang
Joseph C. Cappelleri, Kelly H. Zou,
Interpretation of Experiments Statistical Evaluation of Diagnostic
Joint Models for Longitudinal and Time-
Andrew G. Bushmakin, Jose Ma. J. Alvir,
David B. Allison, Grier P. Page, Performance: Topics in ROC Analysis
to-Event Data: With Applications in R
Demissie Alemayehu, and Tara Symonds
T. Mark Beasley, and Jode W. Edwards Kelly H. Zou, Aiyi Liu, Andriy Bandos,
Dimitris Rizopoulos
Quantitative Evaluation of Safety in Drug Lucila Ohno-Machado, and Howard Rockette
Dose Finding by the Continual
Measures of Interobserver Agreement
Development: Design, Analysis and
Reassessment Method Statistical Methods for Clinical Trials
and Reliability, Second Edition
Reporting
Ying Kuen Cheung Mark X. Norleans
Mohamed M. Shoukri
Qi Jiang and H. Amy Xia
Dynamical Biostatistical Models Statistical Methods for Drug Safety
Medical Biostatistics, Fourth Edition
Quantitative Methods for
Daniel Commenges and Robert D. Gibbons and Anup K. Amatya
A. Indrayan
HIV/AIDS Research
Hélène Jacqmin-Gadda Statistical Methods for Healthcare
Meta-Analysis in Medicine and Cliburn Chan, Michael G. Hudgens, and
Elementary Bayesian Biostatistics Performance Monitoring
Health Policy Shein-Chung Chow
Lemuel A. Moyé Alex Bottle and Paul Aylin
Dalene Stangl and Donald A. Berry
Quantitative Methods for Traditional
Emerging Non-Clinical Biostatistics in Statistical Methods for Immunogenicity
Methods in Comparative Effectiveness Chinese Medicine Development
Biopharmaceutical Development and Assessment
Research Shein-Chung Chow
Manufacturing Harry Yang, Jianchun Zhang, Binbing Yu,
Constantine Gatsonis and Sally C. Morton
Randomization, Masking, and Allocation
Harry Yang and Wei Zhao
Mixed Effects Models for the Population Concealment
Empirical Likelihood Method in Statistical Methods in Drug Combination
Approach: Models, Tasks, Methods Vance W. Berger
Survival Analysis Studies
and Tools
Randomized Clinical Trials of
Mai Zhou Wei Zhao and Harry Yang
Marc Lavielle
Nonpharmacological Treatments
Essentials of a Successful Biostatistical Statistical Testing Strategies in the
Modeling to Inform Infectious Disease Isabelle Boutron, Philippe Ravaud,
Collaboration Health Sciences
Control and David Moher
Arul Earnest Albert Vexler, Alan D. Hutson,
Niels G. Becker
Randomized Phase II Cancer
and Xiwei Chen
Exposure–Response Modeling: Methods
Modern Adaptive Randomized Clinical Clinical Trials
and Practical Implementation Statistics in Drug Research:
Trials: Statistical and Practical Aspects Sin-Ho Jung
Jixian Wang Methodologies and Recent
Oleksandr Sverdlov
Repeated Measures Design with
Developments
Frailty Models in Survival Analysis
Monte Carlo Simulation for the Generalized Linear Mixed Models for
Shein-Chung Chow and Jun Shao
Andreas Wienke
Pharmaceutical Industry: Concepts, Randomized Controlled Trials
Statistics in the Pharmaceutical Industry,
Fundamental Concepts for New Clinical Algorithms, and Case Studies Toshiro Tango
Third Edition
Trialists Mark Chang
Sample Size Calculations for Clustered
Ralph Buncher and Jia-Yeong Tsay
Scott Evans and Naitee Ting
Multiregional Clinical Trials for and Longitudinal Outcomes in Clinical
Survival Analysis in Medicine and
Generalized Linear Models: A Bayesian Simultaneous Global New Drug Research
Genetics
Perspective Development Chul Ahn, Moonseong Heo,
Jialiang Li and Shuangge Ma
Dipak K. Dey, Sujit K. Ghosh, and Joshua Chen and Hui Quan and Song Zhang
Bani K. Mallick Theory of Drug Development
Multiple Testing Problems in Sample Size Calculations in Clinical
Eric B. Holmgren
Handbook of Regression and Modeling: Pharmaceutical Statistics Research, Third Edition
Applications for the Clinical and Alex Dmitrienko, Ajit C. Tamhane, Shein-Chung Chow, Jun Shao, Hansheng Translational Medicine: Strategies and
Pharmaceutical Industries and Frank Bretz Wang, and Yuliya Lokhnygina Statistical Methods
Daryl S. Paulson Dennis Cosmatos and Shein-Chung Chow
http://taylorandfrancis.com
Randomization,
Masking, and Allocation
Concealment
Edited by
Vance W. Berger
CRC Press
Taylor & Francis Group
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© 2018 by Taylor & Francis Group, LLC
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Library of Congress Cataloging-in-Publication Data
Names: Berger, Vance, editor.
Title: Randomization, masking, and allocation concealment / [edited by] Vance Berger.
Description: Boca Raton : Taylor & Francis, a CRC title, part of the Taylor & Francis
imprint, a member of the Taylor & Francis Group, the academic division of T&F
Informa plc, 2018. | Includes bibliographical references.
Identifiers: LCCN 2017030500 | ISBN 9781138033641 (hardback : acid-free paper)
Subjects: LCSH: Clinical trials--Statistical methods. | Clinical trials--Moral and
ethical aspects.
Classification: LCC R853.C55 R35 2018 | DDC 610.72/4--dc23
LC record available at https://lccn.loc.gov/201703050
Visit the Taylor & Francis Web site at
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Contents
Editor........................................................................................................................xi
Contributors .........................................................................................................xiii
1. Randomization and Bias in Historical Perspective .................................1
J. Rosser Matthews
2. Proper Randomization Reduces the Chance of Wasted
Biomedical Research ......................................................................................9
Arturo Martí-Carvajal
3. Sympathetic Bias: A Neglected Source of Selection Bias .....................29
William C. Grant
4. The Alleged Benefits of Unrestricted Randomization .........................39
Vance W. Berger
5. Restricted Randomization: Pros and Cautions ......................................51
Jonathan Chipman
6. Evolution of Restricted Randomization with Maximum
Tolerated Imbalance .....................................................................................61
Wenle Zhao
7. Evaluating the Evaluation ...........................................................................83
Adriana C. Burgos and Ross J. Kusmick
8. Selection Bias in Studies with Unequal Allocation ..............................89
Olga M. Kuznetsova
9. Unrecognized Dual Threats to Internal Validity Relating
to Randomization .......................................................................................123
Vance W. Berger, Adriana C. Burgos, and Omolola A. Odejimi
10. Testing for Second-Order Selection Bias Effect in Randomised
Controlled Trials Using Reverse Propensity Score (RPS) ..................141
Steffen Mickenautsch and Bo Fu
ix