Innovative Designs and Analyses for Small Population Clinical Trials
Development Strategies and Operational Engagement for Pediatric and Rare Diseases
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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.
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.
Offers coverage and discussion on a relatively new topic in the clinical trial space Features successful and precedent-setting examples Promotes the importance of interdisciplinary collaboration
Autor*in
Jingjing Ye
Themen in »Innovative Designs and Analyses for Small Population Clinical Trials«
rare disease pediatric patient registry enrichment design master protocol Bayesian analysis adaptive design external and historic control Clinical Trials