Making the full impact of a rare disease asset clear

Situation

A client needed to establish the value of a pipeline asset in rare disease, where the impact on patients and caregivers can extend far beyond clinical outcomes and direct healthcare costs.

Solution

Avalere connected patient and caregiver experience with claims-based analysis to quantify the total burden of disease. By bringing direct and indirect impacts together, we built an evidence base that reflected not only what disease costs the healthcare system, but also what it takes from the people living with it.

This was a departure from traditional burden-of-illness assessments, which often prioritize direct medical costs and clinical measures. This can leave the indirect impact of illness—on caregivers, work, daily life, and communities—less visible in the value narrative.

Success

Our expanded analysis enabled us to produce a fuller, evidence-based value narrative that better reflected patient and caregiver experiences of the pipeline asset’s value proposition. It also established a stronger  foundation for communicating how the treatment could reduce the total burden of disease. Findings were credibly disseminated through a white paper, congress, and scientific channels.