Tag: natural history studies 3 results

Rare Disease

7 Tips for Designing and Operationalizing Rare Disease Natural History Studies

Natural history studies play a crucial role in rare disease drug development, providing insights that advance discovery and shape clinical trial design. The benefits of these studies also extend to understanding patient journeys, clarifying the standard of care, identifying disease-specific centers of excellence, and surfacing opportunities to improve patient care. In this blog post, we…

Rare Disease

How to Use Patient Registries and Natural History Studies in Rare Disease Development

Successful drug development requires a deep understanding of the disease of interest—its etiology, epidemiology, presentation, manifestations, and progression. In rare diseases, however, much of this information may be unknown. Patient populations are small and historical data are collected inconsistently and dispersed across treating physicians practicing in diverse geographies. Patient registries and natural history studies are…