Introduction
Background to Randomized Controlled Trials
Types of Clinical Trial
Assessing Evidence from Trials
Superiority Trials
Equivalence Trials
Non-Inferiority Trials
As-Good-as-or-Better Trials
Assessment of Bioequivalence
Estimation to a Given Precision
Seven Key Steps to Cook up a Sample Size
Introduction
Step 1: Deciding on the Trial Objective
Step 2: Deciding on the Endpoint
Step 3: Determining the Effect Size (or Margin)
Step 4: Assessing the Population Variability
Step 5: Type I Error
Step 6: Type II Error
Step 7: Other Factors
Sample Sizes for Parallel Group Superiority Trials with Normal Data
Introduction
Sample Sizes Estimated Assuming the Population Variance to Be Known
Design Considerations
Revisiting Worked Example
Sensitivity Analysis
Calculations Taking Account of the Imprecision of the Variance Used in the Sample Size Calculations
Interim Analyses and Sample Size Re-Estimation
Cluster Randomized Trials
Sample Size Calculations for Superiority Crossover Trials with Normal Data
Introduction
Sample Sizes Estimated Assuming the Population Variance to Be Known
Sensitivity Analysis about the Variance Used in the
Sample Size Calculations
Calculations Taking Account of the Imprecision of the Variance Used in the Sample Size Calculations
Sample Size Calculations for Equivalence Clinical Trials with Normal Data
Introduction
Parallel Group Trials
Crossover Trials
Sample Size Calculations for Non-Inferiority Clinical Trials with Normal Data
Introduction
Parallel Group Trials
Crossover Trials
As-Good-as-or-Better Trials
Sample Size Calculations for Bioequivalence Trials
Introduction
Crossover Trials
Parallel Group Studies
Sample Size Calculations for Precision-Based Clinical Trials with Normal Data
Introduction
Parallel Group Trials
Crossover Trials
Sample Size Calculations for Parallel Group Superiority Clinical Trials with Binary Data
Introduction
Inference and Analysis of Clinical Trials with Binary Data
Sample Sizes with the Population Effects Assumed Known
Inclusion of Baselines or Covariates
Sample Size Re-Estimation
Sensitivity Analysis about the Estimates of the Population Effects Used in the Sample Size Calculations
Calculations Taking Account of the Imprecision of the Estimates of the Population Effects Used in the Sample Size Calculations
Calculations Taking Account of the Imprecision of the Estimates Used in the Sample Size Calculations: Bayesian Methods
Sample Size Calculations for Superiority Crossover Clinical Trials with Binary Data
Introduction
Analysis of a Trial
Analysis of a Trial Revisited
Sensitivity Analysis about the Estimates of the Population Effects Used in the Sample Size Calculations
Calculations Taking Account of the Imprecision of the Estimates of the Population Effects Used in the Sample Size Calculations
Calculations Taking Account of the Imprecision of the Estimates Used in the Sample Size Calculations: Bayesian Methods
Sample Size Calculations for Non-Inferiority Trials with Binary Data
Introduction
Choice of Non-Inferiority Limit
Parallel Group Trials Sample Size with the Population Effects Assumed Known
As-Good-as-or-Better Trials
Sample Size Calculations for Equivalence Trials with Binary Data
Introduction
Parallel Group Trials
Crossover Trials
Sample Size Calculations for Precision-Based Trials with Binary Data
Introduction
Parallel Group Trials
Crossover Trials
Sample Size Calculations for Clinical Trials with Ordinal Data
Introduction
The Quality-of-Life Data
Superiority Trials
Non-Inferiority Trials
As-Good-as-or-Better Trials
Equivalence Trials
Estimation to a Given Precision
Sample Size Calculations for Clinical Trials with Survival Data
Introduction
Superiority Trials
Non-Inferiority Trials
Equivalence Trials
Precision-Based Trials
References
Appendix
