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.
Table of Contents
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
