Reliably optimizing a new treatment in humans is a critical first step in clinical evaluation since choosing a suboptimal dose or schedule may lead to failure in later trials. At the same time, if promising preclinical results do not translate into a real treatment advance, it is important to determine this quickly and terminate the clinical evaluation process to avoid wasting resources.

Bayesian Designs for Phase I II Clinical Trials describes how phase I II designs can serve as a bridge or protective barrier between preclinical studies and large confirmatory...

The aim of this book is to equip biostatisticians and other quantitative scientists with the necessary skills, knowledge, and habits to collaborate effectively with clinicians in the healthcare field. The book provides valuable insight on where to look for information and material on sample size and statistical techniques commonly used in clinical research, and on how best to communicate with clinicians. It also covers the best practices to adopt in terms of project, time, and data management; relationship with collaborators; etc.

Healthcare is important to everyone, yet large variations in its quality have been well documented both between and within many countries. With demand and expenditure rising, it s more crucial than ever to know how well the healthcare system and all its components from staff member to regional network are performing. This requires data, which inevitably differ in form and quality. It also requires statistical methods, the output of which needs to be presented so that it can be understood by whoever needs it to make decisions.

Guides You on the Development and Implementation of B R Evaluations

Benefit Risk Assessment Methods in Medical Product Development: Bridging Qualitative and Quantitative Assessments provides general guidance and case studies to aid practitioners in selecting specific benefit risk (B R) frameworks and quantitative methods. Leading experts from industry, regulatory agencies, and academia present practical examples, lessons learned, and best practices that illustrate how to conduct structured B R assessment in clinical development and regulatory...

Maintaining a practical perspective, Bioequivalence and Statistics in Clinical Pharmacology, Second Edition explores statistics used in day-to-day clinical pharmacology work. The book is a starting point for those involved in such research and covers the methods needed to design, analyze, and interpret bioequivalence trials; explores when, how, and why these studies are performed as part of drug development; and demonstrates the methods using real world examples.

Drawing on knowledge gained directly from working in the pharmaceutical industry, the authors set...

Praise for the first edition:

"Given the author's years of experience as a statistician and as a founder of the first DMC in pharmaceutical industry trials, I highly recommend this book--not only for experts because of its cogent and organized presentation, but more importantly for young investigators who are seeking information about the logistical and philosophical aspects of a DMC."

-S. T. Ounpraseuth, The American Statistician

In the first edition of this well-regarded book, the author provided a...

Analyzing Longitudinal Clinical Trial Data: A Practical Guide provide practical and easy to implement approaches for bringing the latest theory on analysis of longitudinal clinical trial data into routine practice. This book, with its example-oriented approach that includes numerous SAS and R code fragments, is an essential resource for statisticians and graduate students specializing in medical research.

The authors provide clear descriptions of the relevant statistical theory and illustrate practical considerations for modeling longitudinal data. Topics covered include choice of...

An important factor that affects the duration, complexity and cost of a clinical trial is the endpoint used to study the treatment s efficacy. When a true endpoint is difficult to use because of such factors as long follow-up times or prohibitive cost, it is sometimes possible to use a surrogate endpoint that can be measured in a more convenient or cost-effective way. This book focuses on the use of surrogate endpoint evaluation methods in practice, using SAS and R.

Cluster Randomised Trials, Second Edition discusses the design, conduct, and analysis of trials that randomise groups of individuals to different treatments. It explores the advantages of cluster randomisation, with special attention given to evaluating the effects of interventions against infectious diseases. Avoiding unnecessary mathematical detail, the book covers basic concepts underlying the use of cluster randomisation, such as direct, indirect, and total effects.

In the time since the publication of the first edition, the use of cluster...

Repeated Measures Design with Generalized Linear Mixed Models for Randomized Controlled Trials is the first book focused on the application of generalized linear mixed models and its related models in the statistical design and analysis of repeated measures from randomized controlled trials. The book introduces a new repeated measures design called S: T design combined with mixed models as a practical and useful framework of parallel group RCT design because of easy handling of missing data and sample size reduction. The book emphasizes practical, rather than theoretical, aspects of...