In cancer research, a traditional phase II trial is designed as a single-arm trial that compares the experimental therapy to a historical control. This simple trial design has led to several adverse issues, including increased false positivity of phase II trial results and negative phase III trials. To rectify these problems, oncologists and biostatisticians have begun to use a randomized phase II trial that compares an experimental therapy with a prospective control therapy.

Randomized Phase II Cancer Clinical Trials explains how to properly select and accurately...

Using real data sets throughout, Survival Analysis in Medicine and Genetics introduces the latest methods for analyzing high-dimensional survival data. It provides thorough coverage of recent statistical developments in the medical and genetics fields.

The text mainly addresses special concerns of the survival model. After covering the fundamentals, it discusses interval censoring, nonparametric and semiparametric hazard regression, multivariate survival data analysis, the sub-distribution method for competing risks data, the cure rate model, and Bayesian...

Theory of Drug Development presents a formal quantitative framework for understanding drug development that goes beyond simply describing the properties of the statistics in individual studies. It examines the drug development process from the perspectives of drug companies and regulatory agencies.

By quantifying various ideas underlying drug development, the book shows how to systematically address problems, such as:

• Sizing a phase 2 trial and choosing the range of p-values that will trigger a follow-up phase 3 trial
• Deciding...

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 comprehensive resource, it combines in-depth explanations of advanced statistical methods with real-life case studies that illustrate practical applications of these methods in QbD implementation.

As many biological products face losing their patents in the next decade, the pharmaceutical industry needs an abbreviated regulatory pathway for approval of biosimilar drug products, which are cost-effective, follow-on/subsequent versions of the innovator's biologic products. But scientific challenges remain due to the complexity of both the manufacturing process and the structures of biosimilar products.

Written by a top biostatistics researcher, Biosimilars: Design and Analysis of Follow-on Biologics is the first book entirely devoted to the statistical...

Many practitioners in the pharmaceutical industry are still largely unfamiliar with benefit-risk assessment, despite its growing prominence in drug development and commercialization. Helping to alleviate this knowledge gap, Benefit-Risk Assessment in Pharmaceutical Research and Development provides a succinct overview of the key considerations relevant to benefit-risk assessment across the pharmaceutical R&D spectrum, from early clinical development to late-stage development to regulatory review to post-launch assessment.

The book first presents...

Statistical Analysis of Human Growth and Development is an accessible and practical guide to a wide range of basic and advanced statistical methods that are useful for studying human growth and development. Designed for nonstatisticians and statisticians new to the analysis of growth and development data, the book collects methods scattered throughout the literature and explains how to use them to solve common research problems. It also discusses how well a method addresses a specific scientific question and how to interpret and present the analytic results. Stata is used...

In a global clinical development strategy, multiregional clinical trials (MRCTs) are vital in the development of innovative medicines. Multiregional Clinical Trials for Simultaneous Global New Drug Development presents a comprehensive overview on the current status of conducting MRCTs in clinical development. International experts from academia, industry, and health organizations address various aspects of the important problems in global clinical development and MRCTs.

The book first provides a high-level introduction to the context, motivation,...

Written by a biostatistics expert with over 20 years of experience in the field, Bayesian Methods in Epidemiology presents statistical methods used in epidemiology from a Bayesian viewpoint. It employs the software package WinBUGS to carry out the analyses and offers the code in the text and for download online.

The book examines study designs that investigate the association between exposure to risk factors and the occurrence of disease. It covers introductory adjustment techniques to compare mortality between states and regression methods to study the...

The Future of Clinical Research and Health Care: From Empirical to Precision Medicine
Clinical and Statistical Considerations in Personalized Medicine explores recent advances related to biomarkers and their translation into clinical development. Leading clinicians, biostatisticians, regulators, commercial professionals, and researchers address the opportunities and challenges in successfully applying biomarkers in drug discovery and preclinical and clinical development.

Robust Biomarkers for Drug Development and Disease Treatment
The first...