Statistical Design, Monitoring, and Analysis of Clinical Trials

Weichung Joe Shih, PhD, has been tenured professor and chair of the Department of Biostatistics, Rutgers School of Public Health, Rutgers University, New Brunswick, New Jersey, and director of Biometrics Division at the Rutgers Cancer Institute of New Jersey, New Brunswick, New Jersey. He is an elected fellow of the American Statistical Association (1996) and an elected member of the International Statistical Institute (2001). Prior to joining academia, he spent his formative years (1982-1999) at Merck Research Laboratories, Rahway, New Jersey. He served in the Advisory Board of the US FDA for reviewing new drug applications, and was associate editor of professional journals, including Statistics in Medicine, Controlled Clinical Trials, Clinical Cancer Research, Statistics in Biopharmaceutical Research, and Statistics in Bioscience. He pioneered in the field of sample size re-estimation for clinical trials, which has evolved into the field of adaptive designs. He also first advocated the use of consistency criterion for international bridging studies, which is now adopted by the ICH guidance for global multiregional clinical trials. He has collaborated extensively with physicians in various therapeutic areas and authored numerous papers in statistical methodology in clinical trials. His research interests include adaptive designs and missing data issues. He has been honored as professor emeritus of Rutgers University since July 2019. Joseph Aisner, MD, is a professor of medicine and a professor of environmental and occupational medicine at the Robert Wood Johnson Medical School of Rutgers University, New Brunswick, New Jersey, director of Medical Oncology Unit at the Robert Wood Johnson University Hospital, New Brunswick, New Jersey, and co-leader of the Clinical Investigations Program at the Rutgers Cancer Institute of New Jersey. He has published extensively and has served on the editorial board of multiple journals, including Journal of Clinical Oncology, Cancer Therapeutics, Medical Oncology, Clinical Cancer Research, and Hematology-Oncology Today. He is a fellow of the American College of Physicians and the American Society of Clinical Oncology. He serves on and chairs several National Data Monitoring Committees, has served on multiple National Institutes of Health (NIH) Study Sections, and has headed two National Cooperative Cancer Study Groups. His research interests include cancer clinical trials and evaluation of therapeutic interventions.

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.