Innovative Designs and Analyses for Small Population Clinical Trials: Development Strategies and Operational Engagement for Pediatric and Rare Diseases

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 determinatio...