Sample Size Calculations in Clinical Research, Second Edition N-Solution bundle version
Purchasing Options
Features
- Explains how the formulas 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, to illustrate clinical and statistical concepts, interpretations, and interactions
- Compares the relative merits and disadvantages of statistical methods for sample size calculations
- Provides sample size calculations for three major areas of clinical research: dose response studies, microarray studies, and Bayesian approaches
- Explores emerging topics in pharmaceutical research and development, including QT/QTc studies with time-dependent replicates and the use of propensity score analysis in nonrandomized or observational studies
- Includes a basic version of N Solution 2007, which is the leading software for sample size calculations and power analysis
Summary
This book explains how the formulas and procedures for sample size calculations can be used in a variety of clinical research and development stages. It presents real-world examples from several therapeutic areas and explores emerging topics in pharmaceutical research and development. This edition now provides sample size calculations for dose response studies, microarray studies, and Bayesian approaches. It also includes a basic version of PharmaSoftware Solutions, Inc.’s N Solution 2007. Along with sensitivity analyses for each procedure, the software contains all procedures for testing equality, superiority/noninferiority, and equivalence for comparing means, proportions, and time-to-event data.
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
