Sample Sizes for Clinical Trials (Hardcover)

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