Methods and Applications of Statistics in Clinical Trials, Volume 2 Planning, Analysis, and Inferential Methods

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

N. BALAKRISHNAN, PHD, is Professor in the Department of Mathematics and Statistics at McMaster University, Canada. He is the author of over twenty books and is the coeditor of Encyclopedia of Statistical Sciences, Second Edition, also published by Wiley.

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

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