Design and Analysis of Clinical Trials
Design and Analysis of Clinical Trials
Design and Analysis of Clinical Trials
Copyright
Welcome
Preface
Conventions
How to use this book
Weeks 1-2: Foundations
1
The Trial as an Experiment
2
The Protocol, the Estimand, and the Endpoint
Weeks 3-5: Design
3
Randomization and Allocation Concealment
4
Blinding, Bias, and Trial Conduct
5
Sample Size and Power
Weeks 6-7: Early-Phase Trials
6
Phase I: Dose-Finding Designs
7
Phase II: Screening Designs
Weeks 8-10: Confirmatory Trials
8
Phase III: The Primary Analysis
9
Interim Monitoring and Adaptive Designs
10
Missing Data, Sensitivity, and Reporting
Advanced Topics I: Design
11
Run-In Designs and Repeated Baselines
12
Optimal Visit Placement
13
N-of-1 and Hybrid Within-Patient Designs
14
Covariate-Adaptive Allocation and Its Analysis
15
Multicenter Trials and the Site Effect
16
Baseline Covariate Adjustment
17
Factorial Designs and Matched-Tuple Randomization
Advanced Topics II: Longitudinal Analysis
18
MMRM in Practice: Linearity, Equivalence, and Fit
19
Continuous or Time-to-Event? Choosing the Endpoint Model
20
Power and Sample Size for Longitudinal Trials
21
Sample-Size Adjustment for Anticipated Dropout
Advanced Topics III: Small Samples and Monitoring
22
Exact Tests for Small and Sparse Trials
23
Exact Conditional Power and Stochastic Curtailment
24
Fully Sequential Analysis Revisited
25
Maintaining the Size of Tests
26
Repowering Trials: The Ethical Argument
Advanced Topics IV: Estimation from Imperfect Data
27
Summary Statistics for Treatment Efficacy
28
Imputation in Practice: Machine Learning and Detection Limits
References
Appendices
Credits
Colophon
Design and Analysis of Clinical Trials
Code
A ten-week graduate course.
Copyright