Innovative Designs and Analyses for Small Population Clinical Trials

This book provides a comprehensive guide to conducting clinical trials in small populations, emphasizing a collaborative, multidisciplinary approach. It presents a systematic framework for integrating the efforts of patient communities, advocacy groups, researchers, pharmaceutical companies, global regulatory agencies, and other stakeholders. The text highlights essential considerations in small population trials, including natural history studies, patient registries, establishing diagnostic criteria, selecting clinically meaningful endpoints, and navigating global regulatory guidance. Drawing from real-world examples, the book details the unique challenges and strategies for designing, implementing, and interpreting clinical trials in rare and specialized patient populations.

Key Features:

• Focuses on collaborative, multi-stakeholder frameworks for small population clinical trials.
• Discusses patient registries, natural history studies, and diagnostic criteria development.
• Explains identification and validation of clinically meaningful endpoints.
• Provides guidance on global regulatory requirements and compliance.
• Includes real-world examples illustrating trial design, implementation, and interpretation.
• Serves as a practical reference for researchers, clinicians, pharmaceutical professionals, and patient advocacy groups involved in rare disease research.