Design and Analysis of Bridging Studies

285 Pages·2012·6.972 MB·\285

by  Jen-pei Liu (Editor), Shein-Chung Chow (Editor), Chin-Fu Hsiao (Editor)

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
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Version Date: 20120525
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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