Innovative Designs and Analyses for Small Population Clinical Trials : Development Strategies and Operational Engagement for Pediatric and Rare Diseases (ICSA Book Series in Statistics) （2024. xix, 473 S. XIX, 473 p. 50 illus., 48 illus. in color. 235 mm）

Ye, Jingjing/Nie, Lei

SPRINGER, BERLIN; SPRINGER NATURE SWITZERLAND; SPRING（2024発売）

SPRINGER, BERLIN; SPRINGER NATURE SWITZERLAND; SPRING（2024発売）

• 製本 Hardcover:ハードカバー版
• 商品コード 9783031608421

Full 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.

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.