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Innovative Designs and Analyses for Small Population Clinical Trials : Development Strategies and Operational Engagement for Pediatric and Rare Diseases / by Jingjing Ye, Lei Nie.
Springer Nature - Springer Mathematics and Statistics eBooks 2024 English International Available online
View online- Format:
- Book
- Author/Creator:
- Ye, Jingjing.
- Series:
- ICSA Book Series in Statistics, 2199-0999
- Language:
- English
- Subjects (All):
- Biometry.
- Clinical medicine--Research.
- Clinical medicine.
- Statistics.
- Artificial intelligence--Data processing.
- Artificial intelligence.
- Machine learning.
- Biostatistics.
- Clinical Research.
- Statistics in Engineering, Physics, Computer Science, Chemistry and Earth Sciences.
- Applied Statistics.
- Data Science.
- Machine Learning.
- Local Subjects:
- Biostatistics.
- Clinical Research.
- Statistics in Engineering, Physics, Computer Science, Chemistry and Earth Sciences.
- Applied Statistics.
- Data Science.
- Machine Learning.
- Physical Description:
- 1 online resource (486 pages)
- Edition:
- 1st ed. 2024.
- Place of Publication:
- Cham : Springer Nature Switzerland : Imprint: Springer, 2024.
- Summary:
- This book discusses a multidisciplinary and multi-stakeholder collaborative approach to small population clinical trials. It provides and illustrates a systematic framework that depends upon a collaborative infrastructure between the patient community, patient advocacy groups, researchers and investigators, pharmaceutical companies, global regulatory agencies, and customers. The book also highlights the considerations of natural history and patient registry, diagnostic criteria determination, identification of clinically meaningful endpoints, and global regulatory guidance. The authors detail the nuances of clinical trial design as specific to small population disease, drawing from real examples.
- Contents:
- 1. Introduction
- 2. The Case Study of NCI-COG Pediatric Match Trials
- 3. The Case Study of Non-oncology (Lonafarnib)
- 4. Natural History and Patient Registry
- 5. Diagnostic Criteria and Endpoints
- 6. Enrichment Designs
- 7. Pediatric Extrapolation
- 8. Cross-over Designs
- 9. Adaptive Designs
- 10. Master Protocols
- 11. Other Designs
- 12. Modeling and Simulations
- 13. Frequentist Approaches
- 14. Bayesian Approaches
- 15. Operation and Other Considerations
- 16. Case Studies.
- Notes:
- Description based on publisher supplied metadata and other sources.
- ISBN:
- 9783031608438
- OCLC:
- 1472980426
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