Sample Sizes for Clinical, Laboratory and Epidemiology Studies

Sample Sizes for Clinical, Laboratory and Epidemiology Studies

Now in its fourth and extended edition, Sample Sizes for Clinical, Laboratory and Epidemiology Studies includes the sample size software (SSS) and formulae and numerical tables needed to design valid clinical studies. The text covers clinical as well as laboratory and epidemiology studies and contains the information needed to ensure a study will form a valid contribution to medical research.

The authors, noted experts in the field, explain step–by–step and explore the wide range of considerations necessary to assist investigational teams when deriving an appropriate sample size for their planned study. The book contains sets of sample size tables with companion explanations and clear worked out examples based on real data. In addition, the text offers bibliography and references sections that are designed to be helpful with guidance on the principles discussed.

This revised fourth edition:

Offers the only text available to include sample size software for use in designing and planning clinical studies Presents new and extended chapters with many additional and refreshed examples Includes clear explanations of the principles and methodologies involved with relevant practical examples Makes clear a complex but vital topic that is designed to ensure valid methodology and publishable results Contains guidance from an internationally recognised team of medical statistics experts

Written for medical researchers from all specialities and medical statisticians, Sample Sizes for Clinical, Laboratory and Epidemiology Studies offers an updated fourth edition of this important guide for designing and planning reliable and evidence based clinical studies.

Preface vii

1 Basic Design Considerations 1

2 Further Design Considerations 29

3 Binary Outcomes 41

4 Ordered Categorical Outcomes 55

5 Continuous Outcomes 67

6 Rate Outcomes 81

7 Survival Time Outcomes 97

8 Paired Binary, Ordered Categorical and Continuous Outcomes 115

9 Confidence Intervals 135

10 Repeated Outcome Measures 155

11 Non ]Inferiority and Equivalence 167

12 Cluster Designs 193

13 Stepped Wedge Designs 211

14 More than Two Groups Designs 225

15 Genomic Targets and Dose ]Finding 235

16 Feasibility and Pilot Studies 247

17 Therapeutic Exploratory Trials: Single Arm with Binary Outcomes 265

18 Therapeutic Exploratory Trials: Survival, Dual Endpoints, Randomised and Genomic Targets 281

19 The Correlation Coefficient 303

20 Observer Agreement Studies 315

21 Reference Intervals and Receiver Operating Curves 337

22 Sample Size Software 359

Cumulative References 361

Index

David Machin, Leicester Cancer Research Centre, University of Leicester, Leicester and Medical Statistics Group, School of Health and Related Research, University of Sheffield, Sheffield, UK.

Michael J. Campbell, Medical Statistics Group, School of Health and Related Research, University of Sheffield, Sheffield, UK.

Say Beng Tan, SingHealth Duke–NUS Academic Medical Centre, Singapore.

Sze Huey Tan, Division of Clinical Trials and Epidemiological Sciences, National Cancer Centre, Singapore.