Innovative Designs and Analyses for Small Population Clinical Trials

Development Strategies and Operational Engagement for Pediatric and Rare Diseases

Jingjing Ye, Lei Nie (Autoren)

Buch | Hardcover

XIX, 477 Seiten

2024 | 2024
Springer International Publishing (Verlag)
978-3-031-60842-1 (ISBN)

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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.

Jingjing Ye is Senior Director, Head of Data Science of Operational Excellence (DSOE) in Global Statistics and Data Science (GSDS). She leads a team to promote innovations in various organizations within BeiGene. She has been active in the scientific community, serving as an FDA representative between industry leaders, academic institutions, and the FDA on various scientific working groups, including pediatric, external controls in oncology, simulation in adaptive designs, and quantitative imaging biomarkers. She currently leads several cross-disciplinary working groups, including ASA Biopharmaceutical (BIOP) Section Pediatric Working Group, ASA-DIA Master Protocol and DIA Data Sciences.
Lei Nie is the Associate Division Director for DB II, in the US Food & Drug Administraion. He is an elected fellow of the American Statistical Association and has authored a total of 99 peer-reviewed journal publications. Passionate about multidisciplinary collaboration and the important work performed at the FDA, he always looks for ways to improve efficiencies and to maintain a good work-family balance.

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.

Erscheint lt. Verlag	14.11.2024
Reihe/Serie	ICSA Book Series in Statistics
Zusatzinfo	Approx. 350 p. 40 illus.
Verlagsort	Cham
Sprache	englisch
Maße	155 x 235 mm
Themenwelt	Mathematik / Informatik ► Mathematik ► Wahrscheinlichkeit / Kombinatorik
Schlagworte	adaptive design • Bayesian analysis • Biostatistics • Clinical Trials • enrichment design • external and historic control • master protocol • patient registry • Pediatric • Rare Disease
ISBN-10	3-031-60842-9 / 3031608429
ISBN-13	978-3-031-60842-1 / 9783031608421
Zustand	Neuware