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Innovative Designs and Analyses for Small Population Clinical Trials

Development Strategies and Operational Engagement for Pediatric and Rare Diseases

Lei Nie, Jingjing Ye

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ca. 139,09

Springer Nature Switzerland img Link Publisher

Naturwissenschaften, Medizin, Informatik, Technik / Wahrscheinlichkeitstheorie, Stochastik, Mathematische Statistik

Beschreibung

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.

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Schlagwörter

pediatric, Clinical Trials, external and historic control, Biostatistics, patient registry, enrichment design, adaptive design, rare disease, Bayesian analysis, master protocol