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.
The book provides sample size formulas and procedures for testing equality, noninferiority/superiority, and equivalence.
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
Contents
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
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
Bayesian Sample Size Calculation
- Posterior Credible Interval Approach
- Posterior Error Approach
- The Bootstrap-Median Approach
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
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