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.
Table of Contents:
Preface. A bout the author. 1 Randomized clinical trials with noncompliance: issues, definitions and problems of commonly used analyses.
1.1 Randomized encouragement design (RED).
1.2 Randomized consent designs.
1.3 Treatment efficacy versus programmatic effectiveness.
1.4 Definitions of commonly used terms and assumptions.
1.5 Mmost commonly used analyses for a RCT with noncompliance.
Exercises.
2 Randomized clinical trials with noncompliance under parallel groups design.
2.1 Testing superiority.
2.2 Testing noninferiority.
2.3 Testing equivalence.
2.4 Interval estimation.
2.5 Sample size determination.
2.6 Risk model-based approach.
Exercises.
Appendix.
3 Randomized clinical trials with noncompliance in stratified sampling.
3.1 Testing superiority.
3.2 Testing noninferiority.
3.3 Testing equivalence .
3.4 Interval estimation.
3.5 Test homogeneity of index in large strata.
Exercises.
Appendix.
4 Randomized clinical trials with noncompliance under cluster sampling.
4.1 Testing superiority.
4.2 Testing noninferiority.
4.3 Testing equivalence.
4.4 Interval estimation.
4.5 Sample size determination.
4.6 An alternative randomization-based approach.
Exercises.
Appendix.
5 Randomized clinical trials with both noncompliance and subsequent missing outcomes.
5.1 Testing superiority.
5.2 Testing noninferiority.
5.3 Testing equivalence.
5.4 Interval estimation.
5.5 Sample size determination.
5.6 An alternative missing at random (MAR) model.
Exercises.
Appendix.
6 Randomized clinical trials with noncompliance in repeated binary measurements.
6.1 Testing superiority.
6.2 Testing noninferiority.
6.3 Testing equivalence.
6.4 Interval estimation.
6.5 Sample size determination.
Exercises.
References.
Index.
