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Statistical Design, Monitoring, and Analysis of Clinical Trials, Second Edition concentrates on the biostatistics component of clinical trials. This new edition is updated throughout and includes five new chapters.
Developed from the authors' courses taught to public health and medical students, residents, and fellows during the past 20 years, the text shows how biostatistics in clinical trials is an integration of many fundamental scientific principles and statistical methods. The book begins with ethical and safety principles, core trial design concepts, the principles and methods of sample size and power calculation, and analysis of covariance and stratified analysis. It then focuses on sequential designs and methods for two-stage Phase II cancer trials to Phase III group sequential trials, covering monitoring safety, futility, and efficacy. The authors also discuss the development of sample size reestimation and adaptive group sequential procedures, phase 2/3 seamless design and trials with predictive biomarkers, exploit multiple testing procedures, and explain the concept of estimand, intercurrent events, and different missing data processes, and describe how to analyze incomplete data by proper multiple imputations.
This text reflects the academic research, commercial development, and public health aspects of clinical trials. It gives students and practitioners a multidisciplinary understanding of the concepts and techniques involved in designing, monitoring, and analyzing various types of trials. The book's balanced set of homework assignments and in-class exercises are appropriate for students and researchers in (bio)statistics, epidemiology, medicine, pharmacy, and public health.
List of contents
1. Overview. 2. Concepts and Methods of Statistical Designs. 3. Efficiency with Trade-Offs and Crossover Designs. 4. Sample Size and Power Calculations. 5. Analysis of Covariance and Stratified Analysis. 6. Regression Analysis of Survival Data 7. Sequential Designs and Methods-Part I: Expected Sample Size and Two-Stage Phase II Trials in Oncology 8. Sequential Designs and Methods-Part II: Monitoring Safety and Futility 9. Sequential Designs and Methods-Part III: Classical Group Sequential Trials 10. Monitoring the Maximum Information and Adaptive Sample-Size Designs 11. Multiplicity Issues and Methods for Controlling the Type-I Error Rate. 12.Clinical Trials with Predictive Biomarkers. 13. Seamless Phase II/III: Select-the-Winner Design. 14. Statistical Significance and p-Values. 15. Estimand, Intercurrent Events, and Missing Data.
Summary
This book concentrates on the biostatistics component of clinical trials. This new, second edition is updated throughout and includes three new chapters. This text reflects the academic research, commercial development, and public health aspects of clinical trials.
Report
"This book gives a good overview a bout various aspects of statistics in the design, monitoring and analysis of clinical trials and covers also modern topics. In my opinion this book can not only be helpful for teaching, but could also be a very helpful guidebook for inexperienced statistic ians as well as other researchers who are just starting to work in the field of clinical trials. It may also be a good tool to help the communication between multidisciplinary trial teams, as it covers the topics from several angles and does not purely foc us on statistics."
- Stefanie Hayoz, ISCB News, September 2022.
