Full Description
In clinical trials, monitoring accumulating data at regular intervals is essential for balancing ethical and financial considerations against scientific rigor. This comprehensive second edition reflects the remarkable evolution in adaptive clinical trial methodology over the past two decades. Since publication of the first edition, these approaches have transformed from theoretical concepts to widely accepted practices, now endorsed by regulatory authorities, including formal FDA guidance.
Features:
Eleven entirely new chapters, including optimal design construction, delayed "pipeline" data accommodation, and multiple comparison procedures
Eight dedicated chapters on adaptive methods including sample size re-assessment, seamless Phase II/III trials, multi-arm multi-stage trials, and enrichment designs
Exact methods for binary and Poisson data with applications to vaccine trials
Expanded discussion of binding versus non-binding futility boundaries for enhanced decision-making
A chapter with a thoroughly updated review of Bayesian approaches to sequential trial design and analysis
Incorporates response-adaptive treatment assignment in group sequential designs
Practical computational methods and software guidance for implementing the techniques
This definitive resource serves statisticians, clinical researchers, pharmaceutical scientists, and regulatory professionals involved in clinical trial design and analysis. The text balances theoretical foundations with practical implementation guidance, making it invaluable for both experienced practitioners and those new to adaptive trial methodology. With twice the content of the first edition, this volume provides the comprehensive understanding needed to design more efficient, ethical, and scientifically rigorous clinical trials, ultimately accelerating the delivery of effective treatments to patients.
Contents
1. Introduction. 2. Two-sided Tests: Introduction. 3. A Unified Formulation and the General Calculation. 4. Two-sided Tests: General Applications. 5. One-Sided Tests. 6. Two-Sided Tests with Early Stopping Under the Null Hypothesis. 7. Tests for Equivalence and Non-inferiority. 8. Information Monitoring: The Error Spending Approach. 9. Analysis Following a Sequential Test. 10. Repeated Coincidence Intervals. 11. Stochastic Curtailment. 12. Optimal Group Sequential Tests. 13. Bayesian Approaches. 14. Group Sequential Tests for Delayed Observations. 15. General Group Sequential Distribution Theory. 16. Binary Data. 17. Survival Data. 18. Nuisance Parameters: Internal Pilot Studies, Sample Size Re-estimation and Information Monitoring. 19. Multivariate Endpoints. 20. Multiple Hypotheses: Primary and Secondary Endpoints. 21. Multi-arm Multi-stage Trials. 22. From Group Sequential to Adaptive Designs: Flexible Sample Size Re-assessment. 23. Adaptive Combination Tests. 24. Sample Size Re-assessment given an Interim Estimate of Treatment Effect . 25. Adaptive Trials Testing Multiple Hypotheses: Closed Testing Procedures with Combination Tests. 26. Treatment Selection and Testing: Seamless Phase II/III Trials. 27. Adaptive Multi-arm Multi-stage Phase III Trials. 28. Enrichment Designs: Targeting Therapies to Selected Sub-populations. 29. Adaptive Seamless Design: A Case Study with Intermediate and Long- Term Survival Endpoints. 30. Response Adaptive Treatment Assignment. 31. Numerical Computations for Group Sequential and Adaptive Tests.
