03740nam a22006135i 4500 978-3-031-60843-8 DE-He213 20251030113322.0 cr nn 008mamaa 241120s2024 sz | s |||| 0|eng d 9783031608438 978-3-031-60843-8 10.1007/978-3-031-60843-8 doi QH323.5 PBT bicssc MED090000 bisacsh PBT thema Ye, Jingjing. author. (orcid)ZORCID;HTTP://ORCID.ORG/0000-0003-0086-2228 https://orcid.org/ZORCID;HTTP://ORCID.ORG/0000-0003-0086-2228 aut http://id.loc.gov/vocabulary/relators/aut 87873 Innovative Designs and Analyses for Small Population Clinical Trials [electronic resource] : Development Strategies and Operational Engagement for Pediatric and Rare Diseases / by Jingjing Ye, Lei Nie. 1st ed. 2024. Cham : Springer Nature Switzerland : Imprint: Springer, 2024. XIX, 473 p. 50 illus., 48 illus. in color. online resource. text txt rdacontent computer c rdamedia online resource cr rdacarrier text file PDF rda ICSA Book Series in Statistics, 2199-0999 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. 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. Biometry. 65981 Clinical medicine Research. 74123 Statistics . 65982 Artificial intelligence Data processing. Machine learning. 55059 Biostatistics. 65985 Clinical Research. 74124 Statistics in Engineering, Physics, Computer Science, Chemistry and Earth Sciences. 66911 Applied Statistics. 65986 Data Science. Machine Learning. 55059 Nie, Lei. author. aut http://id.loc.gov/vocabulary/relators/aut 87874 SpringerLink (Online service) Springer Nature eBook Printed edition: 9783031608421 Printed edition: 9783031608445 Printed edition: 9783031608452 ICSA Book Series in Statistics, 2199-0999 80221 https://doi.org/10.1007/978-3-031-60843-8 ZDB-2-SMA ZDB-2-SXMS 65124 65124