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An Introduction to Statistics in Early Phase Trials



An Introduction to Statistics in Early Phase Trials

Steven Julious, Say Beng Tan, David Machin

ISBN: 978-0-470-31917-8 January 2010 264 Pages


All new medicines and devices undergo early phase trials to assess, interpret and better understand their efficacy, tolerability and safety. An Introduction to Statistics in Early Phase Trials describes the practical design and analysis of these important early phase clinical trials and provides the crucial statistical basis for their interpretation. It clearly and concisely provides an overview of the most common types of trials undertaken in early phase clinical research and explains the different methodologies used. The impact of statistical technologies on clinical development and the statistical and methodological basis for making clinical and investment decisions are also explained.
  • Conveys key ideas in a concise manner understandable by non-statisticians
  • Explains how to optimise designs in a constrained or fixed resource setting
  • Discusses decision making criteria at the end of Phase II trials
  • Highlights practical day-to-day issues and reporting of early phase trials

An Introduction to Statistics in Early Phase Trials is an essential guide for all researchers working in early phase clinical trial development, from clinical pharmacologists and pharmacokineticists through to clinical investigators and medical statisticians. It is also a valuable reference for teachers and students of pharmaceutical medicine learning about the design and analysis of clinical trials.

Chapter 1 Early phase trials

Chapter 2 Introduction to pharmacokinetics

Chapter 3 Sample size calculations for clinical trials

Chapter 4 Crossover trial basics

Chapter 5 Multi-period crossover trials

Chapter 6 First time into man

Chapter 7 Bayesian and frequentist methods

Chapter 8 First-time-into-new-population studies

Chapter 9 Bioequivalence studies

Chapter 10 Other Phase I trials

Chapter 11 Phase II trials: general issues

Chapter 12 Dose–response studies

Chapter 13 Phase II trials with toxic therapies

Chapter 14 Interpreting and applying early phase trial results

Chapter 15 Go/No-Go criteria




"An Introduction to Statistics in Early Phase Trials" is an admirably concise and practical guide to the pertinent context, principles and formulae for statisticians inexpert in the application of their discipline to Phase I and II clinical research". (Journal of Clinical Research Best Practices, 1 March 2011)

An Introduction to Statistics in Early Phase Trials provides concise descriptions of many early phase trial designs, along with the statistical equations necessary to gather and analyze the data”  (Annals of Pharmacotherapy, 2010)

"I enjoyed reading the work of Dr. Julious, Tan, and Machin,found it quite useful, and recommend it to others teaching about, working with, or considering work in the learning phase of drug development." (Journal of Biopharmaceutical Statistics, 2011)