Development Strategies and Operational Engagement for Pediatric and Rare Diseases Ye, Jingjing. creator author. aut http://id.loc.gov/vocabulary/relators/aut Nie, Lei. author. aut http://id.loc.gov/vocabulary/relators/aut SpringerLink (Online service) text sz 2024 1st ed. 2024. monographic eng

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electronic resource

access XIX, 473 p. 50 illus., 48 illus. in color. online resource. 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. 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. by Jingjing Ye, Lei Nie. Biometry Clinical medicine Research Statistics  Artificial intelligence Data processing Machine learning Biostatistics Clinical Research Statistics in Engineering, Physics, Computer Science, Chemistry and Earth Sciences Applied Statistics Data Science Machine Learning QH323.5 9783031608438 https://doi.org/10.1007/978-3-031-60843-8 https://doi.org/10.1007/978-3-031-60843-8 241120 20251030113322.0 978-3-031-60843-8