As the development of medicines has become more globalized, the geographic variations in the efficacy and safety of pharmaceutical products need to be addressed. To accelerate the product development process and shorten approval time, researchers are beginning to design multiregional trials that incorporate subjects from many countries around the world under the same protocol.
Design and Analysis of Bridging Studies addresses the issues arising from bridging studies and multiregional clinical trials. For bridging studies, the book explores ethnic sensitivity, the necessity of bridging studies, types of bridging studies, and the assessment of similarity between regions based on bridging evidence. For multiregional clinical trials, the text considers regional differences, assesses the consistency of treatment effect across regions, and discusses sample size determination for each region.
Taking into account the International Conference Harmonisation (ICH) E5 framework for bridging studies, the book provides a unified summary of the growing literature and research activities in this area. It covers the regulatory requirements, scientific and practical issues, and statistical methodology for designing and evaluating bridging studies and multiregional clinical trials, with the goal of inspiring new research activities in the field.
Bridging Diversity: Extrapolating Foreign Data to a New Region, Shein-Chung Chow and Chin-Fu Hsiao
Impact of Ethnic Differences
Aim and Structure of the Book
Two-Stage Designs of Bridging Studies, Chin-Fu Hsiao, Hsiao-Hui Tsou, Jen-pei Liu, and Yuh-Jenn Wu
Two-Stage Design for Bridging Studies
Group Sequential Approach to Evaluating Bridging Studies
Consistency of Treatment Effects in Bridging Studies and Global Multiregional Trials, Weichung J. Shih and Hui Quan
Global Clinical Trial
Consistency in Making Inference Regarding the Overall Treatment Effect θ
Consistency as a Method for Region-Specific Registration
Assessing Similarity Using the Reproducibility and Generalizability Probabilities and the Sensitivity Index, Shein-Chung Chow, Ying Lu, and Lan-Yan Yang
Criteria for Assessing Similarity
Reproducibility and Generalizability
Assessing the Similarity-Based Sensitivity Index
Combining Information in Clinical Drug Development: Bridging Studies and Beyond, Kuang-Kuo Gordon Lan and Jose Pinheiro
Leveraging Existing Information in Bridging Studies
Application: Bridging of Blood Pressure Drug
A Bayesian Approach for Evaluation of Bridging Studies, Chin-Fu Hsiao, Hsiao-Hui Tsou, Jen-pei Liu, and Yuh-Jenn Wu
Mixture of Prior Information
Determination of Sample Size
Issues of Sample Size in Bridging Trials and Global Clinical Trials, Hsien-Ming James Hung, Sue-Jane Wang, and Robert O’Neill
Issues of Sample Size Planning for Bridging Trial
Issues of Sample Size Planning in Global Clinical Trial
Impacts of Consistency Assessment on Sample Size
Design and Sample Size Considerations for Global Trials, Christy Chuang-Stein, Yoichi Ii, Norisuke Kawai, Osamu Komiyama, and Kazuhiko Kuribayashi
Points to Consider When Designing a Multiregional Trial
Determination of Regional Sample Size in a Multiregional Study
Additional Analysis to Estimate Treatment Effects in Different Regions
Real-Time Monitoring of Multiregional Trials
Application of Genomic Technologies for Bridging Strategy Involving Different Race and Ethnicity in Pharmacogenomics Clinical Trials, Sue-Jane Wang
Pharmacogenomics and Pharmacogenetics
Bridging Study Evaluation
Challenges of Bridging Strategy in Pharmacogenomics
Interaction Effects in Bridging Studies, Eric Tsung-Cheng Hsieh and Jen-pei Liu
Noninferiority Hypothesis for Treatment Similarity
Statistical Testing Procedures
Discussion and Final Remarks
Multiregional Clinical Trials, Yi Tsong and Hsiao-Hui Tsou
Different Regional Requirements
Bridging the Overall Treatment Effect to Regions
Multiregional Clinical Trials for Global Simultaneous Drug Development in Japan, Kihito Takahashi, Mari Ikuta, and Hiromu Nakajima
MRCTs as a Solution to Drug Lag
Improvements in Clinical Trial Environment in Japan
Growing Trends of MRCTs in Japan
MRCTs in the Field of Oncology
MRCTs in the Field of CVM
Issues in MRCTs
Feasibility and Implementation of Bridging Studies in Taiwan, Mey Wang, Yeong-Liang Lin, and Herng-Der Chern
Regulatory History of Bridging Strategy
Assessment Experiences Based on ICH E5
Bayesian Statistical Approach to Bridging Studies
Designing an External (Supplemental) Bridging Study
References appear at the end of each chapter.
Jen-pei Liu is a professor in the Division of Biometry, Department of Agronomy, and Institute of Epidemiology and Preventative Medicine at National Taiwan University. He is also an adjunct investigator in the Institute of Population Health Sciences at the National Health Research Institutes in Taiwan. A fellow of the ASA, Dr. Liu is an associate editor of the Journal of Biopharmaceutical Statistics and an editor of the Chapman & Hall/CRC Biostatistics Series. He has authored ten books, over 120 peer-reviewed research articles, and over 40 book chapters. His research interests include statistical methodology in bioequivalence evaluation, clinical trials, equivalence and noninferiority testing, bridging testing, statistical quality control in biopharmaceutical products, diagnostic testing, statistical QA/QC for biochip diagnostic products, and evaluation of substantial equivalence in GMO crops and food.
Shein-Chung Chow is a professor in the Department of Biostatistics and Bioinformatics at Duke University School of Medicine. Dr. Chow is also a professor of clinical sciences at Duke–National University of Singapore Graduate Medical School. He is the editor-in-chief of the Journal of Biopharmaceutical Statistics and editor-in-chief of the Chapman & Hall/CRC Biostatistics Series. He has authored or co-authored over 200 papers and 19 books, including Adaptive Design Methods in Clinical Trials, Second Edition, Handbook of Adaptive Designs in Pharmaceutical and Clinical Development, and Controversial Statistical Issues in Clinical Trials. A fellow of the ASA and member of the ISI, Dr. Chow has received the ASA Chapter Service Recognition Award, the DIA Outstanding Service Award, and the ICSA Extraordinary Achievement Award.
Chin-Fu Hsiao is an investigator in the Institute of Population Health Sciences at the National Health Research Institutes in Taiwan. Dr. Hsiao is also an adjunct associate professor at National Chiao Tung University. His research interests encompass Bayesian analysis, clinical trials, and genetic study.
"… a place to start acquiring an understanding of bridging and multi-regional studies and as a supplement to ICH-E5 and local region’s current regulatory implementation guidance. … this book is also intended to stimulate additional research in bridging and multi-regional trials and will be of interest to those looking for research topics."
—Scott D. Patterson, Australian & New Zealand Journal of Statistics, 56, 2014
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