In response to the US FDA's Critical Path Initiative, innovative adaptive designs are being used more and more in clinical trials due to their flexibility and efficiency, especially during early phase development. Handbook of Adaptive Designs in Pharmaceutical and Clinical Development provides a comprehensive and unified presentation of the principles and latest statistical methodologies used when modifying trial procedures based on accrued data of ongoing clinical trials. The book also gives a well-balanced summary of current regulatory perspectives. The first several chapters focus on the fundamental theory behind adaptive trial design, the application of the Bayesian approach to adaptive designs, and the impact of potential population shift due to protocol amendments. The book then presents a variety of statistical methods for group sequential design, classical design, dose-finding trials, Phase I/II and Phase II/III seamless adaptive designs, multiple stage seamless adaptive trial design, adaptive randomization trials, hypotheses-adaptive design, and treatment-adaptive design. It also covers predictive biomarker diagnostics for new drug development, clinical strategies for endpoint selection in translational research, the role of independent data monitoring committees in adaptive clinical trials, the enrichment process in targeted clinical trials for personalized medicine, applications of adaptive designs that use genomic or genetic information, adaptive trial simulation, and the efficiency of adaptive design. The final chapters discuss case studies as well as standard operating procedures for good adaptive practices. With contributions from leading clinical researchers in the pharmaceutical industry, academia, and regulatory agencies, this handbook offers an up-to-date, complete treatment of the principles and methods of adaptive design and analysis. Along with reviewing recent developments, it examines issues commonly encountered when applying adaptive design methods in clinical trials.
Front cover Contents Preface Editors Contributors Chapter 1. Overview of Adaptive Design Methods in Clinical Trials Chapter 2. Fundamental Theory of Adaptive Designs with Unplanned Design Change in Clinical Trials with Blinded Data Chapter 3. Bayesian Approach for Adaptive Design Chapter 4. The Impact of Protocol Amendments in Adaptive Trial Designs Chapter 5. From Group Sequential to Adaptive Designs Chapter 6. Determining Sample Size for Classical Designs Chapter 7. Sample Size Reestimation Design with Applications in Clinical Trials Chapter 8. Adaptive Interim Analyses in Clinical Trials Chapter 9. Classical Dose-Finding Trial Chapter 10. Improving Dose-Finding: A Philosophic View Chapter 11. Adaptive Dose-Ranging Studies Chapter 12. Seamless Phase I/II Designs Chapter 13. Phase II/III Seamless Designs Chapter 14. Sample Size Estimation/Allocation for Two-Stage Seamless Adaptive Trial Designs Chapter 15. Optimal Response-Adaptive Randomization for Clinical Trials Chapter 16. Hypothesis-Adaptive Design Chapter 17. Treatment Adaptive Allocations in Randomized Clinical Trials: An Overview Chapter 18. Integration of Predictive Biomarker Diagnostics into Clinical Trials for New Drug Development Chapter 19. Clinical Strategy for Study Endpoint Selection Chapter 20. Adaptive Infrastructure Chapter 21. Independent Data Monitoring Committees Chapter 22. Targeted Clinical Trials Chapter 23. Functional Genome-Wide Association Studies of Longitudinal Traits Chapter 24. Adaptive Trial Simulation Chapter 25. Efficiency of Adaptive Designs Chapter 26. Case Studies in Adaptive Design Chapter 27. Good Practices for Adaptive Clinical Trials Back cover.
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Electronic reproduction. Ann Arbor, Michigan : ProQuest Ebook Central, 2021. Available via World Wide Web. Access may be limited to ProQuest Ebook Central affiliated libraries.