Innovative Designs and Analyses for Small Population Clinical Trials
Development Strategies and Operational Engagement for Pediatric and Rare Diseases
Series: ICSA Book Series in Statistics;
- Publisher's listprice EUR 160.49
- Prospero’s price EUR 112.49
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62 687 Ft (59 702 Ft + 5% VAT)
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62 687 Ft
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Product details:
- Publisher Springer Nature Switzerland
- Date of Publication 20 November 2025
- ISBN 9783031608452
- Binding Paperback
- See also 9783031608421
- No. of pages473 pages
- Size 235x155 mm
- Language English
- Illustrations XIX, 473 p. 50 illus., 48 illus. in color. 639
Categories
Long description:
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.
MoreTable of 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.
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