ExpDesign Studio facilitates more efficient clinical trial design This book introduces pharmaceutical statisticians, scientists, researchers, and others to ExpDesign Studio software for classical and adaptive designs of clinical trials. It includes the Professional Version 5.0 of ExpDesign Studio software that frees pharmaceutical professionals to focus on drug development and related challenges while the software handles the essential calculations and computations. After a hands-on introduction to the software and an overview of clinical trial designs encompassing numerous variations, Classical and Adaptive Clinical Trial Designs Using ExpDesign Studio: * Covers both classical and adaptive clinical trial designs, monitoring, and analyses * Explains various classical and adaptive designs including groupsequential, sample-size reestimation, dropping-loser, biomarker-adaptive, and response-adaptive randomization designs * Includes instructions for over 100 design methods that have been implemented in ExpDesign Studio and step-by-step demos as well as real-world examples * Emphasizes applications, yet covers key mathematical formulations * Introduces readers to additional toolkits in ExpDesign Studio that help in designing, monitoring, and analyzing trials, such as the adaptive monitor, graphical calculator, the probability calculator, the confidence interval calculator, and more * Presents comprehensive technique notes for sample-size calculation methods, grouped by the number of arms, the trial endpoint, and the analysis basis Written with practitioners in mind, this is an ideal self-study guide for not only statisticians, but also scientists, researchers, and professionals in the pharmaceutical industry, contract research organizations (CROs), and regulatory bodies. It's also a go-to reference for biostatisticians, pharmacokinetic specialists, and principal investigators involved in clinical trials. ERRATUM Classical and Adaptive Clinical Trial Designs Using ExpDesign Studio By Mark Chang The license for the ExpDesign Studio software on the CD included with this book is good for one-year after installation of the software. Prior to the expiration of this period, the software will generate a reminder about renewal for the license. The user should contact CTriSoft International (the owners of ExpDesign Studio) at www.CTriSoft.net or by email at license@ctrisoft.net, about renewal for the license. This should have been made clear in the first printing of this book. We apologize for this error.
Autorentext
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.
Klappentext
ExpDesign Studio facilitates more efficient clinical trial design
This book introduces pharmaceutical statisticians, scientists, researchers, and others to ExpDesign Studio software for classical and adaptive designs of clinical trials. It includes the Professional Version 5.0 of ExpDesign Studio software that frees pharmaceutical professionals to focus on drug development and related challenges while the software handles the essential calculations and computations. After a hands-on introduction to the software and an overview of clinical trial designs encompassing numerous variations, Classical and Adaptive Clinical Trial Designs Using ExpDesign Studio:
-
Covers both classical and adaptive clinical trial designs, monitoring, and analyses
-
Explains various classical and adaptive designs including group sequential, sample-size reestimation, dropping-loser, biomarker-adaptive, and response-adaptive randomization designs
-
Includes instructions for over 100 design methods that have been implemented in ExpDesign Studio and step-by-step demos as well as real-world examples
-
Emphasizes applications, yet covers key mathematical formulations
-
Introduces readers to additional toolkits in ExpDesign Studio that help in designing, monitoring, and analyzing trials, such as the adaptive monitor, graphical calculator, the probability calculator, the confidence interval calculator, and more
-
Presents comprehensive technique notes for sample-size calculation methods, grouped by the number of arms, the trial endpoint, and the analysis basis
Written with practitioners in mind, this is an ideal self-study guide for not only statisticians, but also scientists, researchers, and professionals in the pharmaceutical industry, contract research organizations (CROs), and regulatory bodies. It's also a go-to reference for biostatisticians, pharmacokinetic specialists, and principal investigators involved in clinical trials.
Inhalt
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 DoseResponse 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 MantelHaenszel 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 DoseResponse Trial for a Myocardial Infarction.
3.3 Mathematical Notes on Classical Design.
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