Innovative Designs and Analyses for Small Population Clinical Trials: Development Strategies and Operational Engagement for Pediatric and Rare Diseases (ICSA Book Series in Statistics)

By Jingjing Ye, Lei Nie 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. Product Details Publisher ‏ : ‎ Springer; 2024th edition (November 20, 2024) Language ‏ : ‎ English Hardcover ‏ : ‎ 492 pages ISBN-10 ‏ : ‎ 3031608429 ISBN-13 ‏ : ‎ 978-3031608421