Methods and Applications of Statistics in Clinical Trials, Volume 1 and Volume 2
Concepts, Principles, Trials, and Designs
1st Edition
Will be published approx. on 11. July 2014
Book
Hardback
1952 pages
978-1-118-79078-6 (ISBN)
Description
This set includes Methods and Applications of Statistics in Clinical Trials, Volume 1: Concepts, Principles, Trials, and Designs & Methods and Applications of Statistics in Clinical Trials, Volume 2: Planning, Analysis, and Inferential Methods.
Volume 1
Methods and Applications of Statistics in Clinical Trials, Volume 1: Concepts, Principles, Trials, and Designs successfully upholds the goals of the Wiley Encyclopedia of Clinical Trials by combining both previously-published and newly developed contributions written by over 100 leading academics, researchers, and practitioners in a comprehensive, approachable format. The result is a succinct reference that unveils modern, cutting-edge approaches to acquiring and understanding data throughout the various stages of clinical trial design and analysis.
Volume 2
Featuring newly-written material as well as established literature from the Wiley Encyclopedia of Clinical Trials, this book provides a timely and authoritative review of techniques for planning clinical trials as well as the necessary inferential methods for analyzing collected data.
This comprehensive volume features established and newly-written literature on the key statistical principles and concepts for designing modern-day clinical trials, such as hazard ratio, flexible designs, confounding, covariates, missing data, and longitudinal data. Examples of ongoing, cutting-edge clinical trials from today's research such as early cancer & heart disease, mother to child human immunodeficiency virus transmission, women's health initiative dietary, and AIDS clinical trials are also explored.
Volume 1
Methods and Applications of Statistics in Clinical Trials, Volume 1: Concepts, Principles, Trials, and Designs successfully upholds the goals of the Wiley Encyclopedia of Clinical Trials by combining both previously-published and newly developed contributions written by over 100 leading academics, researchers, and practitioners in a comprehensive, approachable format. The result is a succinct reference that unveils modern, cutting-edge approaches to acquiring and understanding data throughout the various stages of clinical trial design and analysis.
Volume 2
Featuring newly-written material as well as established literature from the Wiley Encyclopedia of Clinical Trials, this book provides a timely and authoritative review of techniques for planning clinical trials as well as the necessary inferential methods for analyzing collected data.
This comprehensive volume features established and newly-written literature on the key statistical principles and concepts for designing modern-day clinical trials, such as hazard ratio, flexible designs, confounding, covariates, missing data, and longitudinal data. Examples of ongoing, cutting-edge clinical trials from today's research such as early cancer & heart disease, mother to child human immunodeficiency virus transmission, women's health initiative dietary, and AIDS clinical trials are also explored.
More details
Series
Language
English
Place of publication
New York
United States
Target group
Professional and scholarly
Product notice
sewn/stitched
Cloth over boards
Dimensions
Height: 267 mm
Width: 188 mm
Thickness: 97 mm
Weight
3583 gr
ISBN-13
978-1-118-79078-6 (9781118790786)
Copyright in bibliographic data and cover images is held by Nielsen Book Services Limited or by the publishers or by their respective licensors: all rights reserved.
Schweitzer Classification
Person
N. BALAKRISHNAN is Professor in the Department of Mathematics and Statistics at McMaster University, Canada. He is a Fellow of the American Statistical Association. His publications include other Wiley books: A First Course in Order Statistics, Continuous Univariate Distributions - 1 & 2, and Discrete Multivariate Distributions.
Content
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
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