Steven A. Julious "Sample Sizes for Clinical Trials"
Chapman & Hall | English | 2009-08-26 | ISBN: 1584887397 | 317 pages | PDF | 1,6 MB


With worked examples and problems based on real-world scenarios, this book takes readers through the process of calculating the sample size for many types of clinical trials. It covers the most common types of clinical trials across all phases. The author discusses how assumptions made in a sample size calculation can impact the calculation. He provides calculations for many of the trials and offers hints and tips on how to optimize the calculations. He also illustrates the steps involved in determining sample size, from defining the trial objective to selecting an appropriate endpoint.

From the Back Cover
Drawing on various real-world applications, Sample Sizes for Clinical Trials takes readers through the process of calculating sample sizes for many types of clinical trials. It provides descriptions of the calculations with a practical emphasis.

Focusing on normal, binary, ordinal, and survival data, the book explores a range of trials, including superiority, equivalence, non-inferiority, bioequivalence, and precision for both parallel group and crossover designs. The author discusses how trial objectives impact the study design with respect to the derivation of formulae for sample size calculations. He uses real-life studies throughout to show how the concepts and calculations can be employed.

This work underscores the importance of sample size calculation in the design of a clinical trial. With useful calculation tables throughout, it enables readers to quickly find an appropriate formula, formula application, and associated worked example.

Contents
Preface .................................................................................................................. xvii
List of Figures ...................................................................................................... xix
List of Tables ........................................................................................................ xxi
1 Introduction .....................................................................................................1

2 Seven Key Steps to Cook Up a Sample Size ...........................................27

3 Sample Sizes for Parallel Group Superiority Trials with Normal Data .............45

4 Sample Size Calculations for Superiority Cross-over Trials with Normal Data ...........73

5 Sample Size Calculations for Equivalence Clinical Trials with Normal Data .............83

6 Sample Size Calculations for Non-Inferiority Clinical Trials with Normal Data .............95

7 Sample Size Calculations for Bioequivalence Trials .......................... 107

8 Sample Size Calculations for Precision-Based Clinical Trials with Normal Data ........123

9 Sample Size Calculations for Parallel Group Superiority Clinical Trials with Binary Data .............139

10 Sample Size Calculations for Superiority Cross-over Clinical Trials with Binary Data .........167

11 Sample Size Calculations for Non-inferiority Trials with Binary Data .............179

12 Sample Size Calculations for Equivalence Trials with Binary Data........... 197

13 Sample Size Calculations for Precision-Based Trials with Binary Data ............... 209

14 Sample Size Calculations for Clinical Trials with Ordinal Data ................215

15 Sample Size Calculations for Clinical Trials with Survival Data .....................247
15.1 Introduction ....................................................................................... 247
15.2 Superiority Trials............................................................................... 248
15.2.1 Primary Endpoint Is Negative ........................................... 248
15.2.1.1 Sample Size Calculations:
Number of Events ................................................. 249
15.2.1.2 Worked Example 15.1 ........................................... 251
15.2.1.3 Sample Size Calculations:
Total Number of Subjects .................................... 252
15.2.1.4 Loss to Follow-up ................................................. 252
15.2.1.5 Worked Example 15.2 ........................................... 252
15.2.1.6 Total Sample Size Revisited ................................253
15.2.1.7 Worked Example 15.3: Uniform
Recruitment ...........................................................256
15.2.1.8 Worked Example 15.4: Truncated
Exponential Recruitment .................................... 257
15.2.1.9 Worked Example 15.5:
Uniform and Truncated Exponential
Recruitment Revisited .........................................258
15.2.1.10 Comment on the Results .....................................260
15.2.2 Primary Endpoint Is Positive .............................................260
15.2.2.1 Sample Size Calculations: Number of
Events and Total Sample Size ............................. 262
15.2.2.2 Worked Example 15.6: Noether Approach ....... 262
15.2.2.3 Worked Example 15.7: Normal Approach ........263
15.3 Non-inferiority Trials .......................................................................264
15.3.1 If Primary Endpoint Is Negative .......................................264
15.3.2 If Primary Endpoint Is Positive .........................................266
15.4 Equivalence Trials .............................................................................266
15.4.1 If Primary Endpoint Is Negative .......................................266
15.5 Precision Trials .................................................................................. 267
15.5.1 If Primary Endpoint Is Negative ....................................... 267
References ........................................................................................................... 269
Bibliography ........................................................................................................277
Appendix .............................................................................................................285
Index ..................................................................................................................... 291