Sample Size Calculations in Clinical Research, Second Edition N-Solution bundle version

Duke University School of Medicine, Durham, North Carolina, USA University of Wisconsin, Madison, USA

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