Stella & Rose's Books

Contents

• Preface to the Second Edition
• Prefaced to the First Edition
• Acknowledgements
• 1 Introduction
• 1.1 Drug Development
• 1.2 The role of statistics in drug development
• 1.3 The object of this book
• 1.4 The author's knowledge of statistics in drug development
• 1.5 The reader and his or her knowledge of statistics
• 1.6 How to use this book
• References
• Part 1 Four Views of Statistics in Drug Development: Historical, Methodological, Technical and Professional
• 2 A Brief and Superficial History of Statistics for Drug Developers
• 2.1 Introduction
• 2.2 Early Probabilists
• 2.3 James Bernoulli (1654-1705)
• 2.4 John Arbuthnott (1667-1753)
• 2.5 The mathematics of probability in the late 17th, the 18th and early 19th centuries
• 2.6 Thomas Bayes (1701-1761)
• 2.7 Adolphe Quetelet (1796-1874)
• 2.8 Francis Galton (1822-1911)
• 2.9 Karl Pearson (1857-1936)
• 2.10 'Student' (1876-1937)
• 2.11 RA Fisher (1890-1962)
• 2.12 Modern Mathematical statistics
• 2.13 Medical Statistics
• 2.14 Statistics in clinical trials today
• 2.15 The current debate
• 2.16 A living science
• 2.17 Further Reading
• References
• 3 Design and Interpretation of Clinical Trials as Seen by a Statistician
• 3.1 Prefatory Warning
• 3.2 Introduction
• 3.3 Defining effects
• 3.4 Practical problems in using the counterfactual argument
• 3.5 Regression to the mean
• 3.6 Control in clinical trials
• 3.7 Randomization
• 3.8 Blinding
• 3.9 Using concomitant observations
• 3.10 Measuring treatment effects
• 3.11 Data generation models
• 3.12 In conclusion
• 3.13 Further reading
• References
• 4 Probability, Bayes, P-values, Test of Hypotheses and Confidence Intervals
• 4.1 Introduction
• 4.2 An example
• 4.3 Odds and Sods
• 4.4 The Bayesian solution to the example
• 4.5 Why don't we regularly use the Bayesian approach in clinical trials?
• 4.6 A frequentish approach
• 4.7 Hypothesis testing in controlled clinical trials
• 4.8 Significance tests and p-values
• 4.9 Confidence intervals and limits and credible intervals
• 4.10 Some Bayesian criticism of the frequentist approach
• 4.11 Decision theory
• 4.12 Conclusion
• 4.13 Further Reading
• References
• 5 The Work of the Pharmaceutical Statistician
• 5.1 Prefatory remarks
• 5.2 Introduction
• 5.3 In the beginning
• 5.4 The trial protocol
• 5.5 The statistician's role in planning the protocol
• 5.6 Sample size determination
• 5.7 Other important design issues
• 5.8 Randomization
• 5.9 Data collection preview
• 5.10 Performing the trial
• 5.11 Data analysis preview
• 5.12 Analysis and Reporting
• 5.13 Other activities
• 5.14 Statistical research
• 5.15 Further reading
• References
• Part 2 Statistical Issues: Debatable and Controversial Topics in Drug Development
• 6 Allocating Treatments to Patients in Clinical Trials
• 6.1 Background
• 6.2 Issues
• References
• 6A Technical Appendix
• 7 Baselines and Covariate Information
• 7.1 Background
• 7.2 Issues
• References
• 7A Technical appendix
• 8 The Measurement of Treatment Effects
• 8.1 Background
• 8.2 Issues
• References
• 8A Technical Appendix
• 9 Demographic Subgroups: Representation and Analysis
• 9.1 Background
• 9.2 Issues
• References
• 9A Technical Appendix
• 10 Multiplicity
• 10.1 Background
• 10.2 Issues
• References
• 10A Technical Appendix
• 11 Intention to Treat, Missing Data and Related Matters
• 11.1 Background
• 11.2 Issues
• References
• 11A Technical Appendix
• 12 One-sided and Two-sided Tests and other issues to do with Significance and p-values
• 12.1 Background
• 12.2 Issues
• References
• 13 Determining the Sample Size
• 13.1 Background
• 13.2 Issues
• References
• 14. Multicentre Trials
• 14.1 Background
• 14.2 Issues
• References
• 14A Technical Appendix
• 15 Active Control Equivalence Studies
• 15.1 Background
• 15.2 Issues
• References
• 15A Technical Appendix
• 16 Meta-Analysis
• 16.1 Background
• 16.2 Issues
• References
• 16A Technical Appendix
• 17 Cross-over Trials
• 17.1 Background
• 17.2 Issues
• References
• 18 n-of-1 Trials
• 18.1 Background
• 18.2 Issues
• References
• 19 Sequential Trials
• 19.1 Background
• 19.2 Issues
• References
• 20 Dose-Finding
• 20.1 Background
• 20.2 Issues
• References
• 21 Concerning Pharmacokinetics and Pharmacodynamics
• 21.1 Background
• 21.2 Issues
• References
• 22 Bioequivalence Studies
• 22.1 Background
• 22.2 Issues
• References
• 23 Safety Data, Harms, Drug Monitoring and Pharmaco-epidemiology
• 23.1 Background
• 23.2 Issues
• References
• 24 Pharmaco-economics and Portfolio Management
• 24.1 Background
• 24.2 Issues
• References
• 25 Concerning Pharmacogenetics, Pharmacogenomics and Related Matters
• 25.1 Background
• 25.2 Issues
• References
• 25A Technical Appendix
• Glosary
• Index