Part I Basics
1 Introduction
1.1 Background and outline
1.2 Examples
1.2.1 The ChroPac trial
1.2.2 The Parkinson trial
1.3 General considerations when calculating sample sizes
2 Statistical test and sample size calculation
2.1 The main principle of statistical testing
2.2 The main principle of sample size calculation
Part II Sample size calculation
3 Comparison of two groups for normally distributed outcomes and test for difference or superiority
3.1 Background and notation
3.2 z-test
3.3 t-test
3.4 Analysis of covariance
3.5 Bayesian approach
3.5.1 Background
3.5.2 Methods
4 Comparison of two groups for continuous and ordered categorical outcomes and test for difference or superiority
4.1 Background and notation
4.2 Continuous outcomes
4.3 Ordered categorical outcomes
4.3.1 Assumption-free approach
4.3.2 Assuming proportional odds
5 Comparison of two groups for binary outcomes and test for difference and superiority
5.1 Background and notation 5.2 Asymptotic tests
5.2.1 Difference of rates as effect measure
5.2.2 Risk ratio as effect measure 5.2.3 Odds ratio as effect measure
5.2.4 Logistic regression
5.3 Exact unconditional tests 5.3.1 Background
5.3.2 Fisher-Boschloo test
6 Comparison of two groups for time-to-event outcomes and test for differences or superiority
6.1 Background and notation
6.1.1 Time-to-event data
6.1.2 Sample size calculation for time-to-event data
6.2 Exponentially distributed time-to-event data
6.3 Time-to-event data with proportional hazards
6.3.1 Approach of Schoenfeld
6.3.2 Approach of Freedman
7 Comparison of more than two groups and test for difference
7.1 Background and notation
7.2 Normally distributed outcomes
7.3 C
About the Author:
Prof. Dr. Meinhard Kieser is a Professor of Medical Biometry and Director of the Institute of Medical Biometry and Informatics at the University of Heidelberg. He studied Mathematics and received his PhD in Medical Biometry in 1992. He then worked for more than 15 years as a biostatistician and Head of Biometrics in the pharmaceutical industry. Professor Kieser has comprehensive experience in the planning and analysis of clinical trials and has been a member of numerous independent data monitoring committees. He serves as an associate editor for Pharmaceutical Statistics and the Journal of Biopharmaceutical Statistics. His main research areas are biostatistical methods for clinical trials, including sample size calculation and recalculation, and methods for evidence synthesis.
