"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"-- Provided by publisher.
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, Group Sequential and Adaptive Methods for Clinical Trials, Second Edition provides the comprehensive understanding needed to design more efficient, ethical, and scientifically rigorous clinical trials, ultimately accelerating the delivery of effective treatments to patients.
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.
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.
Christopher Jennison is Professor of Statistics in the Department of Mathematical Sciences, University of Bath, UK. He has worked on the analysis of clinical trials at the Dana-Farber Cancer Institute, Boston and collaborated with medical researchers in areas such as cancer research, cardiology, gynaecology, and complementary medicine.
Bruce Turnbull is Emeritus Professor of Statistics in the School of Operations Research & Industrial Engineering and in the Department of Statistical Science, Cornell University, USA. He has a long experience of designing, monitoring, and analyzing clinical trials and has served on the Data Safety Monitoring Boards for international, multi-center trials for the treatment or prevention of cancer, heart disease and AIDS. He has also served as a consultant to pharmaceutical companies and health-related government review panels.
