Classical and adaptive clinical trial designs using ExpDesign Studio [trademark symbol] /
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Author / Creator: | Chang, Mark. |
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Imprint: | Hoboken, N.J. : John Wiley, ©2008. |
Description: | 1 online resource (xviii, 260 pages) : illustrations |
Language: | English |
Subject: | |
Format: | E-Resource Book |
URL for this record: | http://pi.lib.uchicago.edu/1001/cat/bib/11219459 |
Table of Contents:
- Preface.
- Self-Study and Practice Guide.
- 1. ExpDesign Studio.
- 1.1. Introduction
- 1.2. How to Design a Trial Using ExpDesign Studio
- 1.2.1. How to Design a Classical Trial
- 1.2.2. How to Design a Group Sequential Trial
- 1.2.3. How to Design an Adaptive Trial
- 1.2.4. How to Run Adaptive Trial Simulations
- 1.2.5. How to Design a Multistage Trial
- 1.2.6. How to Design a Dose-Escalation Trial
- 1.3. ExpDesign Menus
- 2. Clinical Trial Design.
- 2.1. Introduction
- 2.2. Classical Clinical Trial Design
- 2.2.1. Substantial Evidence
- 2.2.2. Clinical Trial Endpoint
- 2.2.3. Confirmatory Trials
- 2.2.4. Exploratory Trials
- 2.2.5. Multicenter Trials
- 2.2.6. Trials to Show Superiority
- 2.2.7. Trials to Show Equivalence or Noninferiority
- 2.2.8. Trials to Show a DoseûResponse Relationship
- 2.2.9. Parallel Design
- 2.2.10. Crossover Design
- 2.2.11. Factorial Design
- 2.3. Selection of a Trial Design
- 2.3.1. Balanced Versus Unbalanced Designs
- 2.3.2. Crossover Versus Parallel Designs
- 2.3.3. Dose Escalation Versus Titration Designs
- 2.3.4. Bioavailability Versus Bioequivalence Designs
- 2.3.5. Equivalence Versus Bioequivalence
- 2.3.6. Sample-Size Determination
- 2.4. Adaptive Clinical Trial Design
- 2.4.1. Group Sequential Design
- 2.4.2. Sample-Size Reestimation Design
- 2.4.3. Drop-Loser Design
- 2.4.4. Response-Adaptive Randomization Design
- 2.4.5. Adaptive Dose-Escalation Design
- 2.4.6. Biomarker-Adaptive Design
- 2.4.7. Multistage Design of Single-Arm Trials
- 3. Classical Trial Design.
- 3.1. Introduction
- 3.1.1. Hypothesis Test
- 3.1.2. Importance of Sample-Size Calculation
- 3.1.3. Factors Affecting Sample Size
- 3.1.4. Avoiding Under- or Overpowered Designs
- 3.2. How to Calculate Sample Size Using ExpDesign
- 3.2.1. Testing the Mean Difference Between Two Groups
- 3.2.2. Testing the Proportion Difference Between Two Groups
- 3.2.3. Testing the Survival Difference Between Two Groups
- 3.2.4. Testing the Survival Difference with a Follow-up Period
- 3.2.5. Exact Test for a One-Sample Proportion
- 3.2.6. McNemarÆs Test for Paired Data
- 3.2.7. Noninferiority Test for Two Means
- 3.2.8. Bioequivalence Test for Two Means
- 3.2.9. Bioequivalence Test for Two Means of Lognormal Data
- 3.2.10. Equivalence Test Based on the Ratio of Two Means
- 3.2.11. Precision Method for the Mean Difference for a Paired Sample
- 3.2.12. MantelûHaenszel Test for an Odds Ratio with Two Strata
- 3.2.13. PearsonÆs Chi-Square Test for Rate Difference
- 3.2.14. One-Way ANOVA for Parallel Groups
- 3.2.15. DoseûResponse Trial for a Myocardial Infarction
- 3.3. Mathematical Notes on Classical Design
- 3.3.1. Large-Sample-Size Calculation for Classical Design
- 3.3.2. Commonly Used Terms and Their Mathematical Expressions
- 3.3.3. Relationship Between Enrollment Rate and Number of Events
- 4. Group Sequential Trial Design.
- 4.1. Introduction
- 4.2. Basics of Group Sequential Design
- 4.3. How to Design Sequential Trials Using ExpDesign
- 4.3.1. Design Featuring Early Efficacy Stopping for Two Means
- 4.3.2. Design Featuring Early Futility Stopping for a Proportion
- 4.3.3. Design Featuring Early Stopping for a Survival Endpoint
- 4.3.4. Design Featuring Early Stopping for Paired Proportions
- 4.4. How to Monitor a Group Sequential Trial Using ExpDesign
- 4.4.1. Need for Trial Monitoring
- 4.4.2. Techniques for Monitoring a Sequential Trial
- 4.4.3. How to Monitor a Trial Using ExpDesign
- 4.5. Mathematical Notes on Sequential Trial Design
- 4.5.1. Unified Formulation for Sequential Trial Design
- 4.5.2. Calculation of Conditional Probability
- 4.5.3. Conditional and Predictive Power and RCI for Trial Monitoring
- 4.5.4. Bias-Adjusted Estimates
- 5. Adaptive Trial Design.
- 5.1. Introduction
- 5.2. Basics of Adaptive Design Methods
- 5.3. How To Design a Sample-Size Reestimation Trial Using ExpDesign
- 5.3.1. Sample-Size Adjustment Based on the Effect-Size Ratio
- 5.3.2. Sample-Size Adjustment Based on Conditional Power
- 5.3.3. Adaptive Design for an Acute Ischemic Stroke Trial
- 5.3.4. Adaptive Design for an Asthma Study
- 5.3.5. Adaptive Design for an Oncology Trial
- 5.3.6. Noninferiority Design with a Binary Endpoint
- 5.4. How to Design a Drop-Loser Trial Using ExpDesign
- 5.4.1. Drop-Loser Mechanism
- 5.4.2. Seamless Design of an Asthma Trial
- 5.5. How to Design a Trial Using a Classifier Biomarker
- 5.5.1. Biomarker Classifications
- 5.5.2. Biomarker-Adaptive Design
- 5.6. How to Design a Play-the-Winner Trail Using ExpDesign
- 5.6.1. Randomized Play-the-Winner Design
- 5.6.2. Adaptive Randomization with a Normal Endpoint
- 6. Adaptive Trial Monitoring.
- 6.1. Introduction
- 6.2. Error-Spending Approach
- 6.3. How to Recalculate Stopping Boundaries Using ExpDesign
- 6.4. Conditional Power and the Futility Index
- 6.5. How to Reestimate Sample Size Using ExpDesign
- 6.5.1. Calculating Conditional Power Using ExpDesign
- 6.5.2. Reestimating Sample Size Using ExpDesign
- 6.6. Trial Examples
- 6.6.1. Changes in Number and Timing of the Analyses
- 6.6.2. Recursive Two-Stage Adaptive Design
- 6.6.3. Conditional Power and Sample-Size Reestimation
- 7. Oncology Adaptive Trial Design.
- 7.1. Multistage Trial Design
- 7.1.1. Introduction
- 7.1.2. How to Design a Multistage Design Using ExpDesign
- 7.2. Dose-Escalation Trial Design
- 7.2.1. Introduction
- 7.2.2. Bayesian Continual Reassessment Method
- 7.2.3. How to Design a Dose-Escalation Trial Using ExpDesign
- 7.3. Dose-Escalation Trial Monitoring Using CRM
- 7.4. Mathematical Notes on Multistage Design
- 7.4.1. Decision Tree for a Multistage Trial
- 7.4.1. Two-Stage Design
- 7.4.3. Three-Stage Design
- 7.5. Mathematical Notes on the CRM
- 7.5.1. Probability Model for DoseûResponse
- 7.5.2. Prior Distribution of a Parameter
- 7.5.3. Likelihood Function
- 7.5.4. Reassessment of a Parameter
- 7.5.5. Assignment of the Next Patient
- 8. Adaptive Trial Simulator.
- 8.1. Adjusting the Critical Region Method
- 8.2. Classical Design with Two Parallel Treatment Groups
- 8.3. Flexible Design with Sample-Size Reestimation
- 8.4. Design with Random-Play-the-Winner Randomization
- 8.5. Group Sequential Design with One Interim Analysis
- 8.6. Design Permitting Early Stopping and Sample-Size Reestimation
- 8.7. Classical Design with Multiple Treatment Groups
- 8.8. Multigroup Trial with Response-Adaptive Randomization
- 8.9. Adaptive Design Featuring Dropping Losers
- 8.10. DoseûResponse Trial Design
- 8.11. Dose-Escalation Design for an Oncology Trial
- 9. Further Assistance from ExpDesign Studio.
- 9.1. ExpDesign Probability Functions
- 9.2. Virtual Trial Data Generation Using ExpDesign Randomizor
- 9.2.1. Random Number Generation Using ExpDesign
- 9.2.2. How to Generate a Random Univariate Using ExpDesign
- 9.2.3. How to Generate a Random Multivariate Using ExpDesign
- 9.2.4. How to Generate a Random Multibinomial Using ExpDesign
- 9.3. ExpDesign Toolkits
- 9.3.1. Graphic Calculator
- 9.3.2. Statistical Calculator
- 9.3.3. Confidence Interval Calculator
- 10. Classical Design Method Reference.
- 10.1. Single-Group Design
- 10.1.1. One/Paired-Sample Hypothesis Test for the Mean
- 10.1.2. One/Paired-Sample Hypothesis Test for the Proportion
- 10.1.3. One/Paired-Sample Hypothesis Test for Others
- 10.1.4. Paired-Sample Equivalence Test for the Mean
- 10.1.5. Paired-Sample Equivalence Test for the Proportion
- 10.1.6. One-Sample Confidence Interval for the Mean
- 10.1.7. One-Sample Confidence Interval for the Proportion
- 10.1.8. One-Sample Confidence Interval for Others
- 10.2. Two-Group Design
- 10.2.1. Two-Sample Hypothesis Test for the Mean
- 10.2.2. Two-Sample Hypothesis Test for the Proportion
- 10.2.3. Two-Sample Hypothesis Test for Others
- 10.2.4. Two-Sample Equivalence/Noninferiority Test for the Mean
- 10.2.5. Two-Sample Equivalence/Noninferiority Test for the Proportion
- 10.2.6. Two-Sample Equivalence/Noninferiority Test for Survival
- 10.2.7. Two-Sample Confidence Interval for the Mean
- 10.2.8. Two-Sample Confidence Interval for the Proportion
- 10.3. Multigroup Trial Design
- 10.3.1. Multisample Hypothesis Test for the Mean
- 10.3.2. Multisample Hypothesis Test for the Proportion
- 10.3.3. Multisample Hypothesis Test for Others
- 10.3.4. Multisample Confidence Interval for Others
- Afterword.
- Appendix A: Validation of ExpDesign Studio..
- A.1. Validation Process for ExpDesign Studio
- A.1.1. Algorithm Validation
- A.1.2. Statistical Outcome Validation
- A.1.3. Criteria for Passing Validation
- A.1.4. Input and GUI Validation
- A.2. Validation of the Classical Design Module
- A.3. Validation of the Group Sequential Design Module
- A.3.1. Stopping Boundary and Type I Error Rate Validation
- A.3.2. Power and Sample-Size Validation
- A.4. Validation of the Adaptive Design Module
- A.4.1. Stopping Boundary and Type I Error Rate Validation
- A.4.2. Validation of Adaptive Design Monitoring
- A.5. Validation of the Multistage Design Module
- A.6. Validation of the Traditional Dose-Escalation Design Module
- A.6.1. Validation of the Traditional Escalation Rule
- A.6.2. Validation of the Bayesian Continual Reassessment Method
- A.7. Validation of the Trial Simulation Module
- A.8. Validation of the Randomizor
- A.9. Validation of the ExpDesign Toolkits
- A.10. Computer Programs for Validations
- A.10.1. SAS Macro for Three-Stage Design Validation
- A.10.2. Traditional 3 + 3 Escalation Design Validation
- A.10.3. SAS Program for CRM Validation
- Appendix B. Sample-Size Calculation Methods: Classical Design.
- References.
- Index.
- System Requirements, Software Installation, and Software License Agreement.