Methods and Applications of Statistics in Clinical Trials, Volume 1 Concepts, Principles, Trials, and Designs

A complete guide to the key statistical concepts essential for the design and construction of clinical trials

As the newest major resource in the field of medical research, Methods and Applications of Statistics in Clinical Trials, Volume 1: Concepts, Principles, Trials, and Designs presents a timely and authoritative reviewof the central statistical concepts used to build clinical trials that obtain the best results. The referenceunveils modern approaches vital to understanding, creating, and evaluating data obtained throughoutthe various stages of clinical trial design and analysis.

Accessible and comprehensive, the first volume in a two-part set includes newly-written articles as well as established literature from the Wiley Encyclopedia of Clinical Trials. Illustrating a variety of statistical concepts and principles such as longitudinal data, missing data, covariates, biased-coin randomization, repeated measurements, and simple randomization, the book also provides in-depth coverage of the various trial designs found within phase I-IV trials. Methods and Applications of Statistics in Clinical Trials, Volume 1: Concepts, Principles, Trials, and Designs also features:

• Detailed chapters on the type of trial designs, such as adaptive, crossover, group-randomized, multicenter, non-inferiority, non-randomized, open-labeled, preference, prevention, and superiority trials
• Over 100 contributions from leading academics, researchers, and practitioners
• An exploration of ongoing, cutting-edge clinical trials on early cancer and heart disease, mother-to-child human immunodeficiency virus transmission trials, and the AIDS Clinical Trials Group

Methods and Applications of Statistics in Clinical Trials, Volume 1: Concepts, Principles, Trials, and Designs is an excellent reference for researchers, practitioners, and students in the fields of clinicaltrials, pharmaceutics, biostatistics, medical research design, biology, biomedicine, epidemiology,and public health.

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.

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

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