The white paper discusses the significance of registry and natural history studies in rare disease research, emphasizing the need for flexible study designs. Observational studies, including registry and natural history studies, are crucial for understanding disease progression, especially in rare diseases with limited data. Sponsors face challenges such as scarce data, small patient populations, and disease heterogeneity. Natural history studies provide essential insights into disease subtypes, study design, and meaningful endpoints, thereby aiding drug development. Key considerations for these studies include careful planning, data collection standardization, and regulatory compliance.

Join for free to read