Bohdana Ratitch – författare

The concepts of estimands, analyses (estimators), and sensitivity are interrelated. Therefore, great need exists for an integrated approach to these topics. This book acts as a practical guide to developing and implementing statistical analysis plans by explaining fundamental concepts using accessible language, providing technical details, real-world examples, and SAS and R code to implement analyses. The updated ICH guideline raises new analytic and cross-functional challenges for statisticians. Gaps between different communities have come to surface, such as between causal inference and clinical trialists, as well as among clinicians, statisticians, and regulators when it comes to communicating decision-making objectives, assumptions, and interpretations of evidence.This book lays out a path toward bridging some of these gaps. It offers¿ A common language and unifying framework along with the technical details and practical guidance to help statisticians meet the challenges¿ A thorough treatment of intercurrent events (ICEs), i.e., postrandomization events that confound interpretation of outcomes and five strategies for ICEs in ICH E9 (R1)¿ Details on how estimands, integrated into a principled study development process, lay a foundation for coherent specification of trial design, conduct, and analysis needed to overcome the issues caused by ICEs:¿ A perspective on the role of the intention-to-treat principle¿ Examples and case studies from various areas¿ Example code in SAS and R¿ A connection with causal inference¿ Implications and methods for analysis of longitudinal trials with missing dataTogether, the authors have offered the readers their ample expertise in clinical trial design and analysis, from an industrial and academic perspective.

This book provides practical guidance for statisticians, clinicians, and researchers involved in clinical trials in the biopharmaceutical industry, medical and public health organisations. Academics and students needing an introduction to handling missing data will also find this book invaluable. The authors describe how missing data can affect the outcome and credibility of a clinical trial, show by examples how a clinical team can work to prevent missing data, and present the reader with approaches to address missing data effectively. The book is illustrated throughout with realistic case studies and worked examples, and presents clear and concise guidelines to enable good planning for missing data. The authors show how to handle missing data in a way that is transparent and easy to understand for clinicians, regulators and patients. New developments are presented to improve the choice and implementation of primary and sensitivity analyses for missing data. Many SAS code examples are included – the reader is given a toolbox for implementing analyses under a variety of assumptions.