Extract

Pragmatic randomized trials are increasingly used for the study of novel interventions as well as for comparative effectiveness research of existing treatments and therapies. A pragmatic trial uses 1 or more “pragmatic” design elements to more closely reflect real-world settings, such as broad inclusion criteria, recruitment integrated into clinical care procedures, and group- or cluster-level interventions. The book Design and Analysis of Pragmatic Trials provides a toolkit for trialists, particularly for statisticians, who are working on or planning studies with this design.

The book is organized into 6 chapters, with the first chapter providing an overview of pragmatic trials and describing several common cluster-randomized trial designs used in this area. The second chapter focuses on standard parallel-group cluster-randomized trials and provides detailed descriptions of analysis methods and sample size formulas for a variety of scenarios. The third chapter expands the discussion to similar topics in matched-pair cluster-randomized trials. The fourth chapter focuses on stratified cluster-randomized designs. The fifth and sixth chapters describe methods and sample size calculations for stepped-wedge trials, with the former leveraging the generalized estimating equations framework and the latter using mixed-effects models.

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