Methods and Applications of Statistics in Clinical Trials

Contributors xix Preface xxiii 1 Analysis of Over- and Underdispersed Data 1 2 Analysis of Variance (ANOVA) 10 3 Assessment of Health-Related Quality of Life 26 4 Bandit Processes and Response-Adaptive Clinical Trials: The Art of Exploration Versus Exploitation 40 5 Bayesian Dose-Finding Designs in Healthy Volunteers 51 6 Bootstrap 62 7 Conditional Power in Clinical Trial Monitoring 102 8 Cost-Effectiveness Analysis 111 9 Cox-Type Proportional Hazards Models 126 10 Empirical Likelihood Methods in Clinical Experiments 146 11 Frailty Models 166 12 Futility Analysis 174 13 Imaging Science in Medicine I: Overview 187 14 Imaging Science in Medicine, II: Basics of X-Ray Imaging 213 15 Imaging Science in Medicine, III: Digital (21st Century) X-Ray Imaging 264 16 Intention-to-Treat Analysis 313 17 Interim Analyses 323 18 Interrater Reliability 334 19 Intrarater Reliability 340 20 Kaplan-Meier Plot 357 21 Logistic Regression 365 22 Metadata 380 23 Microarray 392 24 Multi-Armed Bandits, Gittins Index, and Its Calculation 416 25 Multiple Comparisons 436 26 Multiple Evaluators 446 27 Noncompartmental Analysis 457 28 Nonparametric ROC Analysis for Diagnostic Trials 483 29 Optimal Biological Dose for Molecularly Targeted Therapies 496 30 Over- and Underdispersion Models 506 31 Permutation Tests in Clinical Trials 527 32 Pharmacoepidemiology, Overview 536 33 Population Pharmacokinetic and Pharmacodynamic Methods 551 34 Proportions: Inferences and Comparisons 570 35 Publication Bias 595 36 Quality of Life 608 37 Relative Risk Modeing 622 38 Sample Size Considerations for Morbidity/Mortality Trials 633 39 Sample Size for Comparing Means 642 40 Sample Size for Comparing Proportions 653 41 Sample Size for Comparing Time-to-Event Data 664 42 Sample Size for Comparing Variabilities 672 43 Screening, Models of 689 44 Screening Trials 721 45 Secondary Efficacy End Points 731 46 Sensitivity, Specificity, and Receiver Operator Characteristic (ROC) Methods 740 47 Software for Genetics/Genomics 752 48 Stability Study Designs 778 49 Subgroup Analysis 793 50 Survival Analysis, Overview 802 51 The FDA and Regulatory Issues 815 52 The Kappa Index 836 53 Treatment Interruption 846 54 Trial Reports: Improving Reporting, Minimizing Bias, and Producing Better Evidence-Based Practice 860 55 U.S. Department of Veterans Affairs Cooperative Studies Program 876 56 Women's Health Initiative: Statistical Aspects and Selected Early Results 901 57 World Health Organization (WHO): Global Health Situation 914 Index 925