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Classical and Adaptive Clinical Trial Designs with ExpDesign Studio [Other digital carrier]

  • Formatas: Other digital carrier, 280 pages
  • Išleidimo metai: 11-Jul-2008
  • Leidėjas: Wiley-Blackwell
  • ISBN-10: 0470368713
  • ISBN-13: 9780470368718
Kitos knygos pagal šią temą:
Classical and Adaptive Clinical Trial Designs with ExpDesign Studio
  • Formatas: Other digital carrier, 280 pages
  • Išleidimo metai: 11-Jul-2008
  • Leidėjas: Wiley-Blackwell
  • ISBN-10: 0470368713
  • ISBN-13: 9780470368718
Kitos knygos pagal šią temą:
Pastovi nuoroda: https://www.kriso.lt/db/9780470368718.html
Raktažodžiai:
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.

Recenzijos

"This book has been selected for The First Clinical Research Bookshelf. Essential reading for clinical research professionals" ( Journal of Clinical Research Best Practices , June 2009)

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 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. 7.4.2 Two-Stage Design. 7.4.3 Three-Stage Design. 7.5
Mathematical Notes on the CRM. 7.5.1 Probability Model for Dose-Response.
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 Dose-Response 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.
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 .