Bayesian Methods in Pharmaceutical Research
This edited book finds its origin in a series of Bayesian conferences, the "Bayes2000" meetings, started by the third editor in 2010. The aim of these meetings is to promote the use of applied Bayesian biostatistics in life sciences, pharmaceutical research & development and public hea...
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| Main Authors: | , , |
|---|---|
| Format: | eBook Book |
| Language: | English |
| Published: |
Boca Raton
CRC Press
2020
CRC Press LLC Chapman & Hall |
| Edition: | 1 |
| Series: | Chapman & Hall/CRC Biostatistics Series |
| Subjects: | |
| ISBN: | 9781138748484, 113874848X, 9781032241524, 1032241527 |
| Online Access: | Get full text |
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Table of Contents:
- 8. Bayesian Adaptive Designs in Drug Development -- 8.1 Introduction -- 8.2 Brief history of adaptive designs -- 8.3 What is an adaptive clinical trial? -- 8.4 Types of adaptation -- 8.5 Reasons we might consider adaptive designs -- 8.6 Example of an adaptive design -- 8.7 Adaptive enrichment designs -- 8.8 Some criticisms of adaptive designs -- 8.9 Summary -- 9. Bayesian Methods for Longitudinal Data with Missingness -- 9.1 Introduction -- 9.2 Common frequentist approaches -- 9.3 Bayesian approaches -- 9.4 Ignorable and nonignorable missingness -- 9.5 Posterior inference -- 9.6 Model selection -- 9.7 Model checking and assessment -- 9.8 Practical example: Growth hormone trial -- 9.9 Wrap-up and open problems -- 10. Survival Analysis and Censored Data -- 10.1 Introduction -- 10.2 Review of survival analysis -- 10.3 Software -- 10.4 Applications -- 10.5 Reporting -- 10.6 Other comments -- 11. Benefit of Bayesian Clustering of Longitudinal Data: Study of Cognitive Decline for Precision Medicine -- 11.1 Introduction -- 11.2 Motivating example -- 11.3 Nonparametric Bayesian models -- 11.4 Standard frequentist analysis: Latent class mixed models -- 11.5 Profile regression analysis -- 11.6 Conclusion -- 12. Bayesian Frameworks for Rare Disease Clinical Development Programs -- 12.1 Introduction -- 12.2 Natural history studies -- 12.3 Long-term safety evaluation with Real-World Data -- 12.4 Bayesian approaches in rare diseases -- 12.5 Case study -- 12.6 Conclusions and future directions -- 13. Bayesian Hierarchical Models for Data Extrapolation and Analysis in Pediatric Disease Clinical Trials -- 13.1 Introduction -- 13.2 Classical statistical approaches to data extrapolation -- 13.3 Current Bayesian approaches -- 13.4 Practical example -- 13.5 Outlook -- Part III: Post-marketing -- 14. Bayesian Methods for Meta-Analysis -- 14.1 Introduction
- 14.2 Pairwise meta-analysis -- 14.3 Network meta-analysis -- 14.4 Bias modeling in pairwise and network meta-analysis -- 14.5 Using meta-analysis to inform study design -- 14.6 Further reading -- 15. Economic Evaluation and Cost-Effectiveness of Health Care Interventions -- 15.1 Introduction -- 15.2 Economic evaluation: A Bayesian decision theoretic analysis -- 15.3 Trial-based economic evaluation -- 15.4 Model-based economic evaluation -- 15.5 Value of information -- 15.6 Conclusion / outlook -- 16. Bayesian Modeling for Economic Evaluation Using "Real-World Evidence -- 16.1 Introduction -- 16.2 Real World Evidence -- 16.3 Economic modeling and survival analysis -- 16.4 Case study: ICDs in cardiac arrhythmia -- 16.5 Conclusions and further developments -- 17. Bayesian Benefit-Risk Evaluation in Pharmaceutical Research -- 17.1 Introduction -- 17.2 Classical approaches to quantitative benefit-risk -- 17.3 Bayesian approaches to quantitative benefit-risk -- 17.4 Outlook for Bayesian benefit-risk -- 17.5 Discussion -- Part IV: Product development and manufacturing -- 18. Product Development and Manufacturing -- 18.1 Introduction -- 18.2 What is the question in manufacturing? -- 18.3 Bayesian statistics for comparability and analytical similarity -- 18.4 Bayesian approach to comparability and biosimilarity -- 18.5 Conclusions -- 19. Process Development and Validation -- 19.1 Introduction -- 19.2 ICH Q8 design space -- 19.3 Assay robustness -- 19.4 Challenges for the Bayesian approach -- 20. Analytical Method and Assay -- 20.1 Introduction -- 20.2 Analytical quality by design -- 20.3 Assay development -- 20.4 Analytical validation and transfer -- 20.5 Routine -- 20.6 Conclusion -- 21. Bayesian Methods for the Design and Analysis of Stability Studies -- 21.1 Introduction -- 21.2 New perspectives on stability data analysis
- Cover -- Half Title -- Series Page -- Title Page -- Copyright Page -- Dedication -- Contents -- Preface -- Editors -- Contributors -- List of abbreviations -- Part I: Introduction -- 1. Bayesian Background -- 1.1 Introduction -- 1.2 The frequentist approach to inference -- 1.3 Bayesian concepts -- 1.4 More than one parameter -- 1.5 Choosing the prior distribution -- 1.6 Determining the posterior distribution numerically -- 1.7 Hierarchical models and data augmentation -- 1.8 Model selection and model checking -- 1.9 Bayesian nonparametric methods -- 1.10 Bayesian software -- 1.11 Further reading -- 2. FDA Regulatory Acceptance of Bayesian Statistics -- 2.1 Introduction -- 2.2 Medical devices -- 2.3 Pharmaceutical products -- 2.4 Differences between devices and drugs -- 2.5 Some promising opportunities in pharmaceutical drugs -- 2.6 The future -- 2.7 Conclusion -- 3. Bayesian Tail Probabilities for Decision Making -- 3.1 Introduction -- 3.2 Posterior tail probabilities -- 3.3 Predictive tail probabilities -- 3.4 Discussion -- Part II: Clinical development -- 4. Clinical Development in the Light of Bayesian Statistics -- 4.1 Introduction -- 4.2 Introduction to drug development -- 4.3 Quantitative decision making in drug development -- 4.4 Bayesian thinking -- 4.5 Applications of Bayesian methods in drug development -- 4.6 Conclusion -- 5. Prior Elicitation -- 5.1 Introduction -- 5.2 Methods for prior elicitation -- 5.3 Examples -- 5.4 Impact and outlook -- 6. Use of Historical Data -- 6.1 Introduction -- 6.2 Identifying historical or co-data -- 6.3 An example: Guillain-Barré syndrome in children -- 6.4 Methods -- 6.5 Application: Non-inferiority trials -- 6.6 Discussion -- 6.7 Code -- 7. Dose Ranging Studies and Dose Determination -- 7.1 Introduction -- 7.2 Dose-response studies -- 7.3 Dose escalation trials in oncology -- 7.4 Conclusions
- 21.3 Stability designs, models and assumptions -- 21.4 Overview of frequentist methods in stability data -- 21.5 Bayesian methods of analysis of stability data -- 21.6 Conclusions -- 22. Content Uniformity Testing -- 22.1 Introduction -- 22.2 Classical procedures for testing content uniformity -- 22.3 Bayesian procedures for testing content uniformity and risk -- 22.4 Challenges for the Bayesian procedures -- 23. Bayesian Methods for In Vitro Dissolution Drug Testing and Similarity Comparisons -- 23.1 Introduction -- 23.2 Current statistical practices in IV dissolution and their limitations -- 23.3 The value of adopting Bayesian paradigms -- 23.4 Applying Bayesian approaches: Two examples -- 23.5 Conclusions -- 24. Bayesian Statistics for Manufacturing -- 24.1 Introduction -- 24.2 Manufacturing situation 1: Revalidation/transfer -- 24.3 Manufacturing situation 2: Evaluating process capability -- 24.4 Manufacturing situation 3: Bayesian modeling of complex testing schemes -- 24.5 Discussion -- Part V: Additional topics -- 25. Bayesian Statistical Methodology in the Medical Device Industry -- 25.1 Introduction -- 25.2 Use of stochastic engineering models in the medical device design stage -- 25.3 Bayesian design and analysis of medical device trials -- 25.4 Challenges -- 26. Program and Portfolio Decision-Making -- 26.1 Introduction -- 26.2 Classical approaches -- 26.3 Current Bayesian approaches to program design -- 26.4 Program and portfolio-level Bayesian decision analysis -- 26.5 Research opportunities -- Index