Methods and Applications of Statistics in Clinical Trials, Volume 2: Planning, Analysis, and Inferential Methods includes updates of established literature from the Wiley Encyclopedia of Clinical Trials as well as original material based on the latest developments in clinical trials. Prepared by a leading expert, the second volume includes numerous contributions from current prominent experts in the field of medical research. In addition, the volume features:
- Multiple new articles exploring emerging topics, such as evaluation methods with threshold, empirical likelihood methods, nonparametric ROC analysis, over- and under-dispersed models, and multi-armed bandit problems
- Up-to-date research on the Cox proportional hazard model, frailty models, trial reports, intrarater reliability, conditional power, and the kappa index
- Key qualitative issues including cost-effectiveness analysis, publication bias, and regulatory issues, which are crucial to the planning and data management of clinical trials
Table of Contents
Contributors xixPreface 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
