Design and Analysis of Bridging Studies PDF
Preview Design and Analysis of Bridging Studies
Biostatistics D e s i g As the development of medicines has become more globalized, the n geographic variations in the efficacy and safety of pharmaceutical a Design and n products need to be addressed. To accelerate the product d development process and shorten approval time, researchers are beginning to design multiregional trials that incorporate subjects A Analysis of n from many countries around the world under the same protocol. a l Design and Analysis of Bridging Studies addresses the issues y Bridging s arising from bridging studies and multiregional clinical trials. For i bridging studies, the book explores ethnic sensitivity, the necessity s of bridging studies, types of bridging studies, and the assessment o Studies f of similarity between regions based on bridging evidence. For B multiregional clinical trials, the text considers regional differences, r assesses the consistency of treatment effect across regions, and i d discusses sample size determination for each region. g i n Taking into account the International Conference Harmonisation g (ICH) E5 framework for bridging studies, the book provides a unified S summary of the growing literature and research activities in this area. t It covers the regulatory requirements, scientific and practical issues, u and statistical methodology for designing and evaluating bridging d i studies and multiregional clinical trials, with the goal of inspiring new e s research activities in the field. Edited by Jen-pei Liu HC Shein-Chung Chow shL iaoiu ow Chin-Fu Hsiao K12075 K12075_Cover.indd 1 6/8/12 3:35 PM Design and Analysis of Bridging Studies 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 Jen-pei Liu Biometrical Fellow Professor Statistical Methodology Division of Biometry Department of Agronomy Integrated Information Sciences National Taiwan University Novartis Pharma AG Taipei, Taiwan Basel, Switzerland Karl E. Peace Bruce W. Turnbull Georgia Cancer Coalition Professor Distinguished Cancer Scholar School of Operations Research Senior Research Scientist and and Industrial Engineering Professor of Biostatistics Cornell University Jiann-Ping Hsu College of Public Health Ithaca, New York Georgia Southern University Statesboro, Georgia Adaptive Design Methods in Clinical Trial Methodology Clinical Trials, Second Edition Karl E. Peace and Ding-Geng (Din) Chen Shein-Chung Chow and Mark Chang Computational Methods in Biomedical Adaptive Design Theory and Research Implementation Using SAS and R Ravindra Khattree and Dayanand N. Naik Mark Chang Computational Pharmacokinetics Advanced Bayesian Methods for Medical Anders Källén Test Accuracy Controversial Statistical Issues in Lyle D. Broemeling Clinical Trials Advances in Clinical Trial Biostatistics Shein-Chung Chow Nancy L. Geller Data and Safety Monitoring Committees Basic Statistics and Pharmaceutical in Clinical Trials Statistical Applications, Second Edition Jay Herson James E. De Muth Design and Analysis of Animal Studies in Bayesian Adaptive Methods for Pharmaceutical Development Clinical Trials Shein-Chung Chow and Jen-pei Liu Scott M. Berry, Bradley P. Carlin, Design and Analysis of Bioavailability and J. Jack Lee, and Peter Muller Bioequivalence Studies, Third Edition Bayesian Analysis Made Simple: An Excel Shein-Chung Chow and Jen-pei Liu GUI for WinBUGS Design and Analysis of Bridging Studies Phil Woodward Jen-pei Liu, Shein-Chung Chow, Bayesian Methods for Measures of and Chin-Fu Hsiao Agreement Design and Analysis of Clinical Trials with Lyle D. Broemeling Time-to-Event Endpoints Bayesian Missing Data Problems: EM, Karl E. Peace Data Augmentation and Noniterative Design and Analysis of Non-Inferiority Computation Trials Ming T. Tan, Guo-Liang Tian, Mark D. Rothmann, Brian L. Wiens, and Kai Wang Ng and Ivan S. F. Chan Bayesian Modeling in Bioinformatics Difference Equations with Public Health Dipak K. Dey, Samiran Ghosh, Applications and Bani K. Mallick Lemuel A. Moyé and Asha Seth Kapadia Biostatistics: A Computing Approach DNA Methylation Microarrays: Stewart J. Anderson Experimental Design and Statistical Causal Analysis in Biomedicine and Analysis Epidemiology: Based on Minimal Sun-Chong Wang and Arturas Petronis Sufficient Causation DNA Microarrays and Related Genomics Mikel Aickin Techniques: Design, Analysis, and Clinical Trial Data Analysis using R Interpretation of Experiments Ding-Geng (Din) Chen and Karl E. Peace David B. Allison, Grier P. Page, T. Mark Beasley, and Jode W. Edwards Dose Finding by the Continual Randomized Clinical Trials of Reassessment Method Nonpharmacological Treatments Ying Kuen Cheung Isabelle Boutron, Philippe Ravaud, and David Moher Elementary Bayesian Biostatistics Lemuel A. Moyé Sample Size Calculations in Clinical Research, Second Edition Frailty Models in Survival Analysis Shein-Chung Chow, Jun Shao Andreas Wienke and Hansheng Wang Generalized Linear Models: A Bayesian Statistical Design and Analysis of Perspective Stability Studies Dipak K. Dey, Sujit K. Ghosh, Shein-Chung Chow and Bani K. Mallick Statistical Evaluation of Diagnostic Handbook of Regression and Modeling: Performance: Topics in ROC Analysis Applications for the Clinical and Kelly H. Zou, Aiyi Liu, Andriy Bandos, Pharmaceutical Industries Lucila Ohno-Machado, and Howard Rockette Daryl S. Paulson Statistical Methods for Clinical Trials Measures of Interobserver Agreement Mark X. Norleans and Reliability, Second Edition Mohamed M. Shoukri Statistics in Drug Research: Methodologies and Recent Medical Biostatistics, Third Edition Developments A. Indrayan Shein-Chung Chow and Jun Shao Meta-Analysis in Medicine and Health Statistics in the Pharmaceutical Industry, Policy Third Edition Dalene Stangl and Donald A. Berry Ralph Buncher and Jia-Yeong Tsay Monte Carlo Simulation for the Translational Medicine: Strategies and Pharmaceutical Industry: Concepts, Statistical Methods Algorithms, and Case Studies Dennis Cosmatos and Shein-Chung Chow Mark Chang Multiple Testing Problems in Pharmaceutical Statistics Alex Dmitrienko, Ajit C. Tamhane, and Frank Bretz Design and Analysis of Bridging Studies Edited by Jen-pei Liu Shein-Chung Chow Chin-Fu Hsiao CRC Press Taylor & Francis Group 6000 Broken Sound Parkway NW, Suite 300 Boca Raton, FL 33487-2742 © 2013 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: 20120525 International Standard Book Number-13: 978-1-4398-4635-3 (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 Contents Preface .....................................................................................................................xv Contributors ........................................................................................................xvii 1. Bridging Diversity: Extrapolating Foreign Data to a New Region ......1 Shein-Chung Chow and Chin-Fu Hsiao 1.1 Introduction ...........................................................................................1 1.2 Impact of Ethnic Differences ...............................................................2 1.3 Regulatory Guidelines .........................................................................4 1.3.1 ICH E5 Guideline on Bridging Studies .................................4 1.3.2 Regulatory Guidelines in Asia-Pacific Region .....................6 1.4 Current Issues ........................................................................................7 1.4.1 Criteria for Similarity ..............................................................7 1.4.2 Sample Size Estimation and Allocation ................................8 1.5 Examples ..............................................................................................10 1.6 Concluding Remarks ..........................................................................12 1.7 Aim and Structure of the Book .........................................................14 References .......................................................................................................15 2. Two-Stage Designs of Bridging Studies ..................................................17 Chin-Fu Hsiao, Hsiao-Hui Tsou, Jen-pei Liu, and Yuh-Jenn Wu 2.1 Introduction .........................................................................................17 2.2 Two-Stage Design for Bridging Studies ...........................................18 2.2.1 Two-Stage Design ..................................................................19 2.2.2 Determining Sample Size and Critical Values ..................19 2.3 Group Sequential Approach to Evaluating Bridging Studies ......25 2.3.1 Group Sequential Approach.................................................25 2.3.2 Sample Size Determination ..................................................26 2.4 Concluding Remarks ..........................................................................30 References .......................................................................................................31 3. Consistency of Treatment Effects in Bridging Studies and Global Multiregional Trials .......................................................................33 Weichung J. Shih and Hui Quan 3.1 Introduction .........................................................................................33 3.2 Global Clinical Trial ............................................................................34 3.3 Consistency in Making Inference Regarding the Overall Treatment Effect θ .......................................................................................35 3.4 Consistency as a Method for Region-Specific Registration ..........39 vii viii Contents 3.5 Other Considerations .........................................................................43 3.5.1 Time-to-Event and Binary Endpoints .................................43 3.5.2 Noninferiority Trial ...............................................................44 3.5.3 MRCT Monitoring .................................................................44 3.6 Example ................................................................................................45 3.7 Discussion ............................................................................................46 References .......................................................................................................47 Shein-Chung Chow, Ying Lu, and Lan-Yan Yang 4.1 Introduction .........................................................................................49 4.2 Criteria for Assessing Similarity ......................................................50 4.3 Reproducibility and Generalizability ..............................................51 4.3.1 Test for Reproducibility ........................................................52 4.3.1.1 Estimated Power Approach ..................................52 4.3.1.2 Confidence Bound Approach ...............................56 4.3.1.3 Bayesian Approach ................................................57 4.3.2 Evaluating the Generalizability Probability ......................63 4.4 Assessing the Similarity-Based Sensitivity Index ..........................64 4.4.1 Population Shift between Regions ......................................64 4.4.2 Case Where Both ε and C Are Fixed ...................................65 4.4.3 Case Where ε Is Random and C Is Fixed ............................66 4.4.3.1 E-Step .......................................................................67 4.4.3.2 M-Step ......................................................................67 4.4.4 Case Where ε Is Fixed and C Is Random ............................68 4.4.5 Case Where Both ε and C Are Random ..............................71 4.5 Concluding Remarks ..........................................................................75 References .......................................................................................................77 5. Combining Information in Clinical Drug Development: Bridging Studies and Beyond ....................................................................79 Kuang-Kuo Gordon Lan and José Pinheiro 5.1 Introduction .........................................................................................79 5.2 Leveraging Existing Information in Bridging Studies ..................80 5.2.1 Weighted Z-Test Approach ...................................................81 5.2.2 Discount Factor Approach ....................................................83 5.3 Application: Bridging of Blood Pressure Drug ...............................84 5.4 Discussion ............................................................................................85 References .......................................................................................................87 6. A Bayesian Approach for Evaluation of Bridging Studies...................89 Chin-Fu Hsiao, Hsiao-Hui Tsou, Jen-pei Liu, and Yuh-Jenn Wu 6.1 Introduction .........................................................................................89 6.2 Mixture of Prior Information ............................................................91 6.3 Determination of Sample Size ...........................................................94 6.4 Examples ..............................................................................................97
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