Methods and Applications of Statistics in Clinical Trials, Volume 1 and Volume 2: Concepts, Principles, Trials, and Designs (Hardcover)

VOLUME 1

Contributors xxiii

Preface xxix

1 Absolute Risk Reduction 1

2 Accelerated Approval 14

3 AIDS Clinical Trials Group (ACTG) 27

4 Algorithm-Based Designs 40

5 Alpha-Spending Function 53

6 Application of New Designs in Phase I Trials 65

7 ASCOT Trial 74

8 Benefit/Risk Assessment in Prevention Trials 80

9 Biased Coin Randomization 90

10 Biological Assay, Overview 106

11 Block Randomization 125

12 Censored Data 139

13 Clinical Data Coordination 146

14 Clinical Data Management 164

15 Clinical Significance 170

16 Clinical Trial Misconduct 191

17 Clinical Trials, Early Cancer and Heart Disease 205

18 Cluster Randomization 216

19 Coherence in Phase I Clinical Trials 230

20 Compliance and Survival Analysis 240

21 Composite Endpoints in Clinical Trials 246

22 Confounding 252

23 Control Groups 263

24 Coronary Drug Project 273

25 Covariates 285

26 Crossover Design 300

27 Crossover Trials 310

28 Diagnostic Studies 320

29 DNA Bank 340

30 Up-and-Down and Escalation Designs 353

31 Dose Ranging Crossover Designs 362

32 Flexible Designs 383

33 Gene Therapy 399

34 Global Assessment Variables 423

35 Good Clinical Practice (GCP) 438

36 Group-Randomized Trials 448

37 Group Sequential Designs 467

38 Hazard Ratio 483

39 Large Simple Trials 500

40 Longitudinal Data 510

41 Maximum Duration and Information Trials 515

42 Missing Data 522

43 Mother to Child Human Immunodeficiency Virus Transmission Trials 536

44 Multiple Testing in Clinical Trials 550

45 Multicenter Trials 558

46 Multiple Endpoints 570

47 Multiple Risk Factor Intervention Trial 577

48 N-of-1 Randomized Trials 587

49 Noninferiority Trial 598

50 Nonrandomized Trials 609

51 Open-Labeled Trials 619

52 Optimizing Schedule of Administration in Phase I Clinical Trials 625

53 Partially Balanced Designs 635

54 Phase I/II Clinical Trials 658

55 Phase II/III Trials 667

56 Phase I Trials 682

57 Phase II Trials 692

58 Phase III Trials 700

59 Phase IV Trials 711

60 Phase I Trials in Oncology 719

61 Placebos 725

62 Planning a Group-Randomized Trial 736

63 Postmenopausal Estrogen/Progestin Interventions Trial (PEPI) 744

64 Preference Trials 759

65 Prevention Trials 770

66 Primary Efficacy Endpoint 779

67 Prognostic Variables in Clinical Trials 789

68 Randomization Procedures 799

69 Randomization Schedule 813

70 Repeated Measurements 827

71 Simple Randomization 841

72 Subgroups 850

73 Superiority Trials 867

74 Surrogate Endpoints 878

75 TNT Trial 887

76 UGDP Trial 894

77 Women's Health Initiative Hormone Therapy Trials 918

78 Women's Health Initiative Dietary Modification Trial 931

Index 945

VOLUME 2

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