This book addresses the most important aspects of how to plan and evaluate clinical trials with a composite primary endpoint to guarantee a clinically meaningful and valid interpretation of the results. Composite endpoints are often used as primary efficacy variables for clinical trials, particularly in the fields of oncology and cardiology. These endpoints combine several variables of interest within a single composite measure, and as a result, all variables that are of major clinical relevance can be considered in the primary analysis without the need to adjust for multiplicity. Moreover, composite endpoints are intended to increase the size of the expected effects thus making clinical trials more powerful.
The book offers practical advice for statisticians and medical experts involved in the planning and analysis of clinical trials. For readers who are mainly interested in the application of the methods, all the approaches are illustrated with real-world clinical trial examples, and the software codes required for fast and easy implementation are provided. The book also discusses all the methods in the context of relevant guidelines related to the topic. To benefit most from the book, readers should be familiar with the principles of clinical trials and basic statistical methods.

Discusses methods for planning and evaluating clinical trials with a composite primary endpoint

Preface.- Contents.- Part I General Introduction to Composite Endpoints.- 1 Definition and Rationale.- 2 Challenges of Composite Endpoints.- 3 Guideline View - Recommendations and Requirements.- 4 Clinical Trial Examples.- Part II Confirmatory Test Problem for a Single (Composite) Endpoint.- 5 The Single-Stage Design.- 6 Group-Sequential and Adaptive Designs.- 7 Related Software Code.- Part III Confirmatory Multiple Test Problem.- 8 Correlation Between Test Statistics.- 9 The Single-Stage Design.- 10 Group-Sequential and Adaptive Designs.- 11 Related Software Code.- Part IV Confirmatory Test Problem for a Weighted Composite Endpoint.- 12 Weighted Binary Composite Endpoint.- Weighted Time-to-Event Composite Endpoint.- 14 OtherWeighted Effect Measures.- 15 Related Software Code.- Part V Descriptive and Confirmatory Evaluation of the Components.- 16 Descriptive Analysis of the Components.- 17 Supplementary Confirmatory Analyses of the Components.- 18 Related Software Code.- Part VI Illustrating Clinical Trial Examples.- 19 Clinical Trial Examples with Binary (Composite) Endpoints.- 20 Clinical Trial Examples with (Composite) Time-to-Event Endpoints.