7 Tips For Designing And Operationalizing Rare Disease Natural History Studies

By Kris O'Brien

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 offer seven tips on designing and operationalizing natural history studies to support successful rare disease therapeutic development.