This volume provides formulas and procedures for determination of sample size required not only for testing equality, but also for testing non-inferiority/superiority, and equivalence (similarity) based on both untransformed (raw) data and log-transformed data under a parallel-group design or a crossover design with equal or unequal ratio of treatment allocations. It contains a comprehensive and unified presentation of statistical procedures for sample size calculation that are commonly employed at various phases of clinical development. Each chapter includes, whenever possible, real examples of clinical studies from therapeutic areas such as cardiovascular, central nervous system, anti-infective, oncology, and women's health to demonstrate the clinical and statistical concepts, interpretations, and their relationships and interactions.
The book highlights statistical procedures for sample size calculation and justification that are commonly employed in clinical research and development. It provides clear, illustrated explanations of how the derived formulas and/or statistical procedures can be used.
Suitable for graduate students and researchers in statistics, the book presents thorough treatments of:
Statistical theories of likelihood-based inference with missing data Computational techniques and theories on imputation Methods involving propensity score weighting, nonignorable missing data, longitudinal missing data, survey sampling, and statistical matching
Assuming prior experience with statistical theory and linear models, the text uses the frequentist framework with less emphasis on Bayesian methods and nonparametric methods. It includes many examples to help readers understand the methodologies. Some of the research ideas introduced can be developed further for specific applications.
"... this is a useful, comprehensive compendium of almost every possible sample size formula. The strong organization and carefully defined formulae will aid any researcher designing a study."-Biometrics
"This impressive book contains formulae for computing sample size in a wide range of settings. One-sample studies and two-sample comparisons for quantitative, binary, and time-to-event outcomes are covered comprehensively, with separate sample size formulae for testing equality, non-inferiority, and equivalence. Many less familiar topics are also covered ..." – Journal of the Royal Statistical Society
Sample Size Calculations in Clinical Research, Third Edition presents statistical procedures for performing sample size calculations during various phases of clinical research and development. A comprehensive and unified presentation of statistical concepts and practical applications, this book includes a well-balanced summary of current and emerging clinical issues, regulatory requirements, and recently developed statistical methodologies for sample size calculation.
Compares the relative merits and disadvantages of statistical methods for sample size calculations
Explains how the formulae and procedures for sample size calculations can be used in a variety of clinical research and development stages
Presents real-world examples from several therapeutic areas, including cardiovascular medicine, the central nervous system, anti-infective medicine, oncology, and women’s health
Provides sample size calculations for dose response studies, microarray studies, and Bayesian approaches
This new edition is updated throughout, includes many new sections, and five new chapters on emerging topics: two stage seamless adaptive designs, cluster randomized trial design, zero-inflated Poisson distribution, clinical trials with extremely low incidence rates, and clinical trial simulation.