Craig Mallinckrodt – 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 data

Together, the authors have offered the readers their ample expertise in clinical trial design and analysis, from an industrial and academic perspective.

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 data

Together, the authors have offered the readers their ample expertise in clinical trial design and analysis, from an industrial and academic perspective.

This book is intended for anyone who is considering a career in statistics or a related field, or those at any point in their career with sufficient work time remaining such that investing in additional learning could be beneficial. As such, the book would be suitable for anyone pursing an MS or PhD in statistics or those already working in statistics. The book focuses on the non-statistical aspects of being a statistician that are crucial for success. These factors include 1) productivity and prioritization, 2) innovation and creativity, 3) communication, 4) critical thinking and decisions under uncertainty, 5) influence and leadership, 6) working relationships, and 7) career planning and continued learning. Each of these chapters includes sections on foundational principles and a section on putting those principles into practice. Connections between these individual skills are emphasized such that the reader can appreciate how the skills build upon each other leading to a whole that is greater than the sum of its parts. By including the individual perspectives from other experts on the fundamental principles and their application, readers will have a well-rounded view on how to build upon and fully leverage their technical skills in statistics. The primary audience for the book is large and diverse. It will be useful for self-study by virtually any statistician, but could also be used as a text in a graduate program that includes a course on careers and career development.

Key Features:

This book is intended for anyone who is considering a career in statistics or a related field, or those at any point in their career with sufficient work time remaining such that investing in additional learning could be beneficial. As such, the book would be suitable for anyone pursing an MS or PhD in statistics or those already working in statistics. The book focuses on the non-statistical aspects of being a statistician that are crucial for success. These factors include 1) productivity and prioritization, 2) innovation and creativity, 3) communication, 4) critical thinking and decisions under uncertainty, 5) influence and leadership, 6) working relationships, and 7) career planning and continued learning. Each of these chapters includes sections on foundational principles and a section on putting those principles into practice. Connections between these individual skills are emphasized such that the reader can appreciate how the skills build upon each other leading to a whole that is greater than the sum of its parts. By including the individual perspectives from other experts on the fundamental principles and their application, readers will have a well-rounded view on how to build upon and fully leverage their technical skills in statistics. The primary audience for the book is large and diverse. It will be useful for self-study by virtually any statistician, but could also be used as a text in a graduate program that includes a course on careers and career development.

Key Features:

Analyzing Longitudinal Clinical Trial Data: A Practical Guide provides practical and easy to implement approaches for bringing the latest theory on analysis of longitudinal clinical trial data into routine practice.The 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 endpoint and statistical test; modeling means and the correlations between repeated measurements; accounting for covariates; modeling categorical data; model verification; methods for incomplete (missing) data that includes the latest developments in sensitivity analyses, along with approaches for and issues in choosing estimands; and means for preventing missing data. Each chapter stands alone in its coverage of a topic. The concluding chapters provide detailed advice on how to integrate these independent topics into an over-arching study development process and statistical analysis plan.