06/17/2026
How do therapies perform in the real world, beyond the controlled environment of clinical trials? For rare diseases like Niemann-Pick disease type C (NPC), answering that question is critical.
In collaboration with IntraBio, Curant Rare developed a first-of-its-kind real-world evidence (RWE) system that is generating new insights into the lived experience of individuals with NPC. At the World Orphan Drug Congress, our teams presented a poster demonstrating how structured, patient-centered data can enhance clinical understanding and support treatment discussions.
Using the validated WeeFIM® outcomes management tool, caregivers and individuals with NPC contributed data on key functional domains including self-care, mobility, and cognition. By leveraging a standardized framework grounded in the World Health Organization's bio-psycho-social model, this approach enables consistent measurement of functional change over time while strengthening the credibility of real-world findings.
The poster highlights how transforming everyday patient experiences into meaningful, validated evidence can help inform clinical decision-making, support ongoing research, and deepen our understanding of therapeutic impact in NPC.
Read the poster to explore the methodology, key findings, and why real-world evidence is becoming an increasingly important part of rare disease research: https://curantrare.com/project/curant-rare-and-intrabio-bring-real-world-evidence-to-niemann-pick-d…
