Design and Analysis of Pragmatic Trials (Chapman &amp Hall CRC Biostatistics Series) (Original PDF from Publisher)

Design and Analysis of Pragmatic Trials (Chapman & Hall/CRC Biostatistics Series) (Original PDF from Publisher)

By Song Zhang, Chul Ahn, and Hong Zhu

This book begins with an introduction to pragmatic cluster randomized trials (PCTs) and explores the key practical considerations statisticians must address during the study design phase.

It provides detailed guidance on sample size determination, sensitivity analyses, and worked examples, while also examining the strengths and limitations of different types of PCTs. Sample size formulas are derived using the generalized estimating equation (GEE) approach for a variety of outcome types within the exponential family, including continuous, binary, and count data.

The book reviews a wide range of experimental designs commonly applied in PCTs, such as cluster randomized designs, matched-pair and stratified cluster designs, stepped-wedge designs, longitudinal cluster designs, and crossover cluster randomized designs. It emphasizes the flexibility of the GEE framework in handling real-world challenges, including hierarchical correlation structures, missing data patterns, and variable cluster sizes. The authors also address the issue of variance underestimation when the number of clusters is small and examine potential solutions to this limitation in PCT design.

By outlining the advantages and disadvantages of various PCT designs and presenting practical sample size formulas that account for common methodological challenges, this book supports practitioners in designing and implementing effective pragmatic trials aimed at improving healthcare outcomes. It is also suitable as a graduate-level textbook for students seeking a deeper understanding of clinical trial design and biostatistical methods.

Key features include:

• Comprehensive discussion of PCT types, with corresponding benefits and limitations

• Sample size formulas, sensitivity analyses, and practical examples

• Coverage of a wide range of experimental designs used in pragmatic trials

• Easily applicable solutions that accommodate real-world design complexities

• A valuable reference for biostatisticians in both academic and industry settings

• Suitable as a textbook for graduate students in statistics and biostatistics

Design and Analysis of Pragmatic Trials
(Chapman & Hall/CRC Biostatistics Series)