Methods and Applications of Sample Size Calculation and Recalculation in Clinical Trials

This book provides an extensive overview of sample size calculation and recalculation in clinical trials, covering fixed and adaptive designs, multiple comparisons, safety assessment, and multi-regional trials. It is suitable for beginners, experienced applied statisticians, and practitioners, and has been used for courses on the topic.

Format: Hardback
Length: 396 pages
Publication date: 20 November 2020
Publisher: Springer Nature Switzerland AG

Clinical researchers involved in designing and analyzing clinical trials. It provides an extensive overview of the principles and methods of sample size calculation and recalculation in clinical trials. Appropriate calculation of the required sample size is crucial for the success of clinical trials. At the same time, a sample size that is too small or too large is problematic due to ethical, scientific, and economic reasons. Therefore, state-of-the-art methods are required when planning clinical trials.

Part I describes a general framework for deriving sample size calculation procedures. This enables an understanding of the common principles underlying the numerous methods presented in the following chapters. Part II addresses the fixed sample size design, where the required sample size is determined in the planning stage and is not changed afterwards. It covers sample size calculation methods for superiority, non-inferiority, and equivalence trials, as well as comparisons between two and more than two groups. A wide range of further topics is discussed, including sample size calculation for multiple comparisons, safety assessment, and multi-regional trials. There is often some uncertainty about the assumptions to be made when calculating the sample size upfront. Part III presents methods that allow to modify the initially specified sample size based on new information that becomes available during the ongoing trial. Blinded sample size recalculation procedures for internal pilot study designs are considered, as well as methods for sample size reassessment in adaptive designs that use unblinded data from interim analyses. The application is illustrated using numerous clinical trial examples, and software code implementing the methods is provided.

The book offers theoretical background and practical advice for biostatisticians and clinical researchers involved in designing and analyzing clinical trials. It provides a comprehensive guide to the field, covering both theoretical concepts and practical applications, and is an essential resource for anyone working in clinical research.

Weight: 756g
Dimension: 162 x 241 x 32 (mm)
ISBN-13: 9783030495275
Edition number: 1st ed. 2020