Focusing on an integral part of pharmaceutical development, Sample Size Calculations in Clinical Research, Second Edition presents statistical procedures for performing sample size calculations during various phases of clinical research and development. It provides sample size formulas and procedures for testing equality, noninferiority/superiority, and equivalence. A comprehensive and unified presentation of statistical concepts and practical applications, this book highlights the interactions between clinicians and biostatisticians, includes a well-balanced summary of current and emerging clinical issues, and explores recently developed statistical methodologies for sample size calculation. Whenever possible, each chapter provides a brief history or background, regulatory requirements, statistical designs and methods for data analysis, real-world examples, future research developments, and related references. One of the few books to systematically summarize clinical research procedures, this edition contains new chapters that focus on three key areas of this field. Incorporating the material of this book in your work will help ensure the validity and, ultimately, the success of your clinical studies.

Table of Contents:
Preface Introduction Regulatory Requirement Basic Considerations Procedures for Sample Size Calculation Aims and Structure of the Book Considerations Prior to Sample Size Calculation Confounding and Interaction One-Sided Test versus Two-Sided Test Crossover Design versus Parallel Design Subgroup/Interim Analyses Data Transformation Practical Issues Comparing Means One-Sample Design Two-Sample Parallel Design Two-Sample Crossover Design Multiple-Sample One-Way ANOVA Multiple-Sample Williams Design Practical Issues Large Sample Tests for Proportions One-Sample Design Two-Sample Parallel Design Two-Sample Crossover Design One-Way Analysis of Variance Williams Design Relative Risk—Parallel Design Relative Risk—Crossover Design Practical Issues Exact Tests for Proportions Binomial Test Fisher’s Exact Test Optimal Multiple-Stage Designs for Single Arm Trials Flexible Designs for Multiple-Arm Trials Remarks Tests for Goodness-of-Fit and Contingency Tables Tests for Goodness-of-Fit Test for Independence—Single Stratum Test for Independence—Multiple Strata Test for Categorical Shift Carry-Over Effect Test Practical Issues Comparing Time-to-Event Data Basic Concepts Exponential Model Cox’s Proportional Hazards Model Weighted Log-Rank Test Practical Issues Group Sequential Methods Pocock’s Test O’Brien and Fleming’s Test Wang and Tsiatis’ Test Inner Wedge Test Binary Variables Time-to-Event Data Alpha Spending Function Sample Size Re-Estimation Conditional Power Practical Issues Comparing Variabilities Comparing Intra-Subject Variabilities Comparing Intra-Subject CVs Comparing Inter-Subject Variabilities Comparing Total Variabilities Practical Issues Bioequivalence Testing Bioequivalence Criteria Average Bioequivalence Population Bioequivalence Individual Bioequivalence In Vitro Bioequivalence Dose Response Studies Continuous Response Binary Response Time-to-Event Endpoint Williams’ Test for Minimum Effective Dose (MED) Cochran–Armitage’s Test for Trend Dose Escalation Trials Concluding Remarks Microarray Studies Literature Review False Discovery Rate (FRD) Control Family-Wise Error Rate (FWER) Control Concluding Remarks Bayesian Sample Size Calculation Posterior Credible Interval Approach Posterior Error Approach The Bootstrap-Median Approach Concluding Remarks Nonparametrics Violation of Assumptions One-Sample Location Problem Two-Sample Location Problem Test for Independence Practical Issues Sample Size Calculation in Other Areas QT/QTc Studies with Time-Dependent Replicates Propensity Analysis in Nonrandomized Studies ANOVA with Repeated Measures Quality of Life Bridging Studies Vaccine Clinical Trials INDEX