Binary Data Analysis of Randomized Clinical

This book provides a systematic and organized approach to analyzing data for a RCT with noncompliance under the most frequently-encountered situations, including parallel sampling, stratified sampling, cluster sampling, parallel sampling with subsequent missing outcomes, and a series of dependent binomial sampling for repeated measurements. Consort-flow diagrams and numerical examples are used to illustrate the bias of commonly-used approaches such as AT analysis, AP analysis and ITT analysis for a RCT with noncompliance. The book also looks at testing the equality, non-inferiority, and equivalence between two treatments based on the different important indices, such as the proportion difference (PD), proportion ratio (PR) and odds ratio (OR). Sample size calculation formulae for testing equality, non-inferiority and equivalence based on different indices under various sampling designs are also featured. The Author presents an instructive and easily-understood approach by using contingency tables to lay down the latent probability structure of observed data so that readers can easily appreciate the logics and the ideas behind the development of test procedures and estimators.

Kung-Jong Lui, Department of Mathematics and Statistics, San Diego State University, USA.

Preface	p. xiii
About the Author	p. xvii
Randomized clinical trials with noncompliance: issues, definitions and problems of commonly used analyses	p. 1
Randomized encouragement design (RED)	p. 4
Randomized consent designs	p. 6
Single-consent randomized design (SCRD)	p. 7
Double-consent randomized design (DCRD)	p. 8
Treatment efficacy versus programmatic effectiveness	p. 9
Definitions of commonly used terms and assumptions	p. 10
Most commonly used analyses for a RCT with noncompliance	p. 13
Exercises	p. 20
Randomized clinical trials with noncompliance under parallel groups design	p. 21
Testing superiority	p. 26
Testing noninferiority	p. 35
Using the difference in proportions	p. 35
Using the ratio of proportions	p. 36
Using the odds ratio of proportions	p. 38
Testing equivalence	p. 41
Using the difference in proportions	p. 41
Using the ratio of proportions	p. 42
Using the odds ratio of proportions	p. 43
Interval estimation	p. 43
Estimation of the proportion difference	p. 44
Estimation of the proportion ratio	p. 47
Estimation of the odds ratio	p. 49
Sample size determination	p. 51
Sample size calculation for testing superiority	p. 52
Sample size calculation for testing noninferiority	p. 55
Sample size calculation for testing equivalence	p. 58
Risk model-based approach	p. 65
Constant risk additive model	p. 66
Constant risk multiplicative model	p. 68
Generalized risk additive model	p. 71
Generalized risk multiplicative model	p. 73
Exercises	p. 75
Appendix	p. 88
Randomized clinical trials with noncompliance in stratified sampling	p. 91
Testing superiority	p. 96
Testing noninferiority	p. 100
Using the difference in proportions	p. 100
Using the ratio of proportions	p. 102
Using the odds ratio of proportions	p. 104
Testing equivalence	p. 105
Using the difference in proportions	p. 106
Using the ratio of proportions	p. 107
Using the odds ratio of proportions	p. 107
Interval estimation	p. 108
Estimation of the proportion difference	p. 108
Estimation of the proportion ratio	p. 113
Estimation of the odds ratio	p. 115
Test homogeneity of index in large strata	p. 119
Testing homogeneity of the proportion difference	p. 120
Testing homogeneity of the proportion ratio	p. 122
Test homogeneity of the odds ratio	p. 125
Exercises	p. 128
Appendix	p. 133
Randomized clinical trials with noncompliance under cluster sampling	p. 137
Testing superiority	p. 143
Testing noninferiority	p. 146
Using the difference in proportions	p. 147
Using the ratio of proportions	p. 148
Using the odds ratio of proportions	p. 149
Testing equivalence	p. 151
Using the difference in proportions	p. 151
Using the ratio of proportions	p. 152
Using the odds ratio of proportions	p. 152
Interval estimation	p. 153
Estimation of the proportion difference	p. 153
Estimation of the proportion ratio	p. 154
Estimation of the odds ratio	p. 157
Sample size determination	p. 158
Sample size calculation for testing superiority	p. 159
Sample size calculation for testing noninferiority	p. 164
Sample size calculation for testing equivalence	p. 170
An alternative randomization-based approach	p. 172
Exercises	p. 176
Appendix	p. 181
Randomized clinical trials with both noncompliance and subsequent missing outcomes	p. 185
Testing superiority	p. 193
Testing noninferiority	p. 196
Using the difference in proportions	p. 196
Using the ratio of proportions	p. 197
Using the odds ratio of proportions	p. 198
Testing equivalence	p. 200
Using the difference in proportions	p. 200
Using the ratio of proportions	p. 201
Using the odds ratio of proportions	p. 201
Interval estimation	p. 202
Estimation of the proportion difference	p. 202
Estimation of the proportion ratio	p. 203
Estimation of the odds ratio	p. 205
Sample size determination	p. 206
Sample size calculation for testing superiority	p. 207
Sample size calculation for testing noninferiority	p. 212
Sample size calculation for testing equivalence	p. 213
An alternative missing at random (MAR) model	p. 221
Estimation of the proportion difference	p. 226
Estimation of the proportion ratio	p. 229
Estimation of the odds ratio	p. 230
Exercises	p. 233
Appendix	p. 238
Randomized clinical trials with noncompliance in repeated binary measurements	p. 247
Testing superiority	p. 253
Testing noninferiority	p. 256
Using the difference in proportions	p. 256
Using the ratio of proportions	p. 257
Testing equivalence	p. 260
Using the difference in proportions	p. 260
Using the ratio of proportions	p. 260
Interval estimation	p. 261
Estimation of the proportion difference	p. 261
Estimation of the proportion ratio	p. 268
Sample size determination	p. 273
Sample size calculation for testing superiority	p. 273
Sample size calculation for testing noninferiority	p. 275
Sample size calculation for testing equivalence	p. 278
Exercises	p. 281
References	p. 289
Index	p. 303
Table of Contents provided by Ingram. All Rights Reserved.

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Binary Data Analysis of Randomized Clinical Trials with Noncompliance

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