Classical and Adaptive Clinical Trial Designs Using Expdesign Studio - Mark Chang

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Résumé :
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 Dose-Response 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 McNemar's 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 Mantel-Haenszel Test for an Odds Ratio with Two Strata.

3.2.13 Pearson's Chi-Square Test for Rate Difference.

3.2.14 One-Way ANOVA for Parallel Groups.

3.2.15 Dose-Response 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 Sequenti...

Biographie:
Mark Chang, PHD, is a Scientific Fellow at Millennium Pharmaceuticals. He has extensive experience with IND and NDA submissions. His involvement in drug development includes both strategic and methodological aspects. Dr. Chang is an Associate Editor for the Journal of Biopharmaceutical Statistics....

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