ICSA Book Series in Statistics: Development Strategies and Operational Engagement for Pediatric and Rare Diseases

Ye, Jingjing; Nie, Lei

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.

Details

Published by: Springer

Publication Date: 2025-11-20

Format: Paperback

ISBN-13: 9783031608452

DOI: 10.1007/978-3-031-60843-8

Dimensions: 235cm x155cm

Pages: 473