Books in the Chapman & Hall/CRC Biostatistics Series series

Suitable for cancer clinicians and biostatisticians, this book explains how to properly select and accurately use diverse statistical methods for designing and analyzing phase II trials. The author first reviews the statistical methods for single-arm phase II trials since some methodologies for randomized phase II trials stem from single-arm pha

This book illustrates the use of effect size measures and corresponding confidence intervals as more informative alternatives to the most basic and widely used significance tests. It provides you with a deep understanding of what happens when these statistical methods are applied in situations far removed from the familiar Gaussian case. Requiri

Taking into account the International Conference Harmonisation E5 framework for bridging studies, this book covers the regulatory requirements, scientific and practical issues, and statistical methodology for designing and evaluating bridging studies and multiregional clinical trials. For bridging studies, the authors explore ethnic sensitivity,

A practical guide for biomedical researchers, clinicians, biostatisticians, and graduate students in biostatistics, this volume covers the latest developments in the analysis and modeling of interval-censored time-to-event data. Top researchers from academia, biopharmaceutical industries, and government agencies show how up-to-date statistical m

Nonpharmacological treatments include a wide variety of treatments such as surgery, technical procedures, implantable devices, non-implantable devices, rehabilitation, psychotherapy, and behavioral interventions. This book focuses on the methods of assessing nonpharmacological treatments, highlighting specific issues, and discussing trial design

From simple NLMs to complex GLMMs and beyond, this book describes how to use the GUI for WinBUGS-BugsXLA-an Excel add-in written by the author that allows a range of Bayesian models to be easily specified. With case studies throughout, the text shows how to routinely apply even the more complex aspects of model specification, such as GLMMs, outl

After a review of the usual measures, including specificity, sensitivity, positive predictive value, negative predictive value, and the area under the ROC curve, this book expands its scope to cover the more advanced topics of verification bias, diagnostic tests with imperfect gold standards, and medical tests where no gold standard is available

The increased use of non-inferiority analysis has been accompanied by a proliferation of research on the design and analysis of non-inferiority studies. Design and Analysis of Non-Inferiority Trials brings together this body of research and confronts the issues involved in the design of a non-inferiority trial. Using examples from real clinical tri

Written by one of the preeminent experts in the field, this book covers commonly encountered controversial statistical issues in clinical trials and, whenever possible, makes recommendations to resolve these problems. It focuses on issues occurring at various stages of clinical research and development, including early-phase clinical development

Exploring applications in medical research and clinical epidemiology, this text continues to cover important issues related to the design and analysis of reliability and agreement studies. This edition contains a new chapter that describes various models for methods comparison studies as well as a new chapter on the analysis of reproducibility u

Drawing from the authors' courses on the subject as well as the first author's more than 30 years working in the pharmaceutical industry, Clinical Trial Methodology emphasizes the importance of statistical thinking in clinical research and presents the methodology as a key component of clinical research. From ethical issues and

Illustrating how stability studies play an important role in drug safety and quality assurance, this book introduces the basic concepts of stability testing, focuses on short-term stability studies, and reviews several methods for estimating drug expiration dating periods. The author compares some commonly employed study designs and discusses both

Focuses on full analyses of real data sets based on the needs and requirements of end users. Fundamental theory is introduced with emphasis on the motivation by challenges coming from real problems with real data. Complete explanations of important concepts are provided, but the amount of mathematical derivation of formulation is minimized.

Drug development sponsors cannot run individual trials for all products in all indications. This results in missed opportunities. Clinical trial programs known as "basket" and "umbrella" have demonstrated that multi-product, multi-indication trials can be efficient and feasible.

This book focuses on clinical trial design and monitoring with time-to-event endpoints. Detail of subjects of the book are included in book contents. This book not only provides a comprehensive review of existing methods. The book also presents a general group sequential method for trial monitoring which has not been published in any book.

This book deals with randomization in clinical trials. Currently trials use permuted blocks randomization almost exclusively. Many researchers are aware of the drawbacks to this fatally flawed method, and while many are unaware of any better randomization methods, in fact several groups have proposed improvements, and also demonstrated the benef

This book provides the application of generalized linear mixed-effects models and its related models in the statistical design and analysis of repeated measures adopted in randomized controlled trials. With increasing concerns about intra-patient variability of treatment effects, the traditional ANCOVA-type methods can no longer cope with these

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.

The premise of Quality by Design (QbD) is that the quality of the pharmaceutical product should be based upon a thorough understanding of both the product and the manufacturing process. This state-of-the-art book provides a single source of information on emerging statistical approaches to QbD and risk-based pharmaceutical development. A compreh

This book shows how to apply biclustering methods to find local patterns in a big data matrix. It presents an overview of data analysis using biclustering methods from a practical point of view. Real case studies in drug discovery, genetics, marketing research, biology, toxicity, and sports illustrate the use of several biclustering methods. All

This book provides guidance on 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 submission. The

This book presents a comprehensive overview on the current status of conducting MRCTs in clinical development. It first provides a high-level introduction to the context, motivation, opportunities, and challenges in simultaneous global clinical development using MRCTs. It then focuses on the design, monitoring, and analysis/interpretation of MRC

This guide explains how to apply health economic evaluation techniques to both clinical trial and non-clinical trial data. Through a simple, applied approach using examples and SAS software, the book helps statisticians and researchers in health economics assess cost-effectiveness. It covers trial design, case report form design, quality of life

This book presents statistical models and methods for the analysis of longitudinal data. It focuses on models for analyzing repeated measures of quantitative and qualitative variables and events history, including survival and multistate models. The book also explores the possibility of unifying these models through a stochastic process point of

A critical issue in the development of biologics, immunogenicity can cause early termination or limited use of the products if not managed well. This book shows how to use robust statistical methods for detecting, quantifying, assessing, and mitigating immunogenicity risk. Along with covering regulatory requirements, the authors discuss statisti

This book explores a wide range of topics in exposure-response modeling, from traditional PKPD modeling to other areas in drug development and beyond. It incorporates numerous examples and software programs for implementing novel methods. The book emphasizes dose adjustment and treatment adaptation based on dynamic exposure-response models, illu

This practical and thorough book explains when and how to use models and techniques for the analysis of competing risks and intermediate states. It covers the most recent insights on estimation techniques and discusses in detail how to interpret the obtained results. Each chapter includes standard exercises; a software section on SAS, Stata, and