Sample Sizes for Clinical, Laboratory and Epidemiology Studies

An authoritative resource that offers the statistical tools and software needed to design and plan valid clinical studies

Now in its fourth and extended edition, Sample Sizes for Clinical, Laboratory and Epidemiology Studiesincludes 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 when 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 EpidemiologyStudies offers an updated fourth edition of the important guide for designing and planning reliable and evidence based clinical studies.

Ein fundiertes Referenzwerk zu den statistischen Instrumenten und Softwareprogrammen, die für das Design und die Planung klinischer Studien erforderlich sind.

Die erweiterte 4. Auflage von Sample Sizes for Clinical, Laboratory and Epidemiology Studies beinhaltet eine Sample-Size-Software (SSS), Formeln und nummerische Tabellen für die Gestaltung valider klinischer Studien. Das Fachbuch behandelt ebenfalls Labor- und epidemiologische Studien und stellt die Informationen zur Verfügung, damit Studien einen wesentlichen Beitrag zur medizinischen Forschung leisten.
Die Autoren, allesamt anerkannte Experten des Fachgebiets, erläutern und untersuchen Schritt für Schritt die vielfältigen Überlegungen bei der Festlegung geeigneter Stichprobengrößen und helfen so bei der Planung von Studien. Bereitgestellt werden ebenfalls Stichprobentabellen mit Erläuterungen und aussagekräftige Beispiele auf der Basis von Echtdaten. Darüber hinaus enthält das Fachbuch ein Literaturverweise und Angaben zu weiterführenden Referenzen zur Unterstützung der vorgestellten Prinzipien.

Diese überarbeitete 4. Auflage
- ist das bislang einzige Referenzwerk mit einem Softwareprogramm für die Gestaltung und Planung klinischer Studien.
- enthält neue und erweiterte Kapitel mit einer Vielzahl neuer und aktualisierter Beispiele.
- erläutert verständlich die angewandten Prinzipien und Methoden anhand von Beispielen aus der Praxis.
- präsentiert auf nachvollziehbare Weise ein komplexes und dennoch wichtiges Thema, damit die richtigen Methoden verwendet werden und fundierte Ergebnisse veröffentlicht werden können.
- bietet Hilfestellung von einem Team international anerkannter Statistikexperten im medizinischen Bereich.

Sample Sizes for Clinical, Laboratory and Epidemiology Studies richtet sich an medizinische Forscher aller Disziplinen sowie an Medizinstatistiker. Die aktualisierte 4. Auflage ist ein wichtiges Referenzwerk für die Gestaltung und Planung verlässlicher, evidenzbasierter klinischer Studien.

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 83

7 Survival Time Outcomes 99

8 Paired Binary, Ordered Categorical and Continuous Outcomes 117

9 Confidence Intervals 137

10 Repeated Outcome Measures 157

11 Non‐Inferiority and Equivalence 169

12 Cluster Designs 195

13 Stepped Wedge Designs 215

14 More than Two Groups Designs 229

15 Genomic Targets and Dose‐Finding 239

16 Feasibility and Pilot Studies 251

17 Therapeutic Exploratory Trials: Single Arm with Binary Outcomes 269

18 Therapeutic Exploratory Trials: Survival, Dual Endpoints, Randomised and Genomic Targets 283

19 The Correlation Coefficient 305

20 Observer Agreement Studies 317

21 Reference Intervals and Receiver Operating Curves 339

22 Sample Size Software ^SS_S 361

Cumulative References 363

Index 381

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.

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.