Case Study: Using Real World Data to Estimate the Annual Incidence of a Rare Disease
This case study explores how a small pharmaceutical company utilized
real-world evidence (RWE) to calculate the national incidence of a rare disease.
Key takeaways include:
- Challenge: Understand how many cases of a rare disease occur annually in the United States to understand market size for a new drug.
- Approach: Magnolia Market Access used a claims-based algorithm to identify patients with disease and stratified by age and sex. Incidence rates were projected to the US population using age and sex weighting.
- Outcome: Annual incidence rates were calculated. Findings were used in a conference abstract and peer-reviewed manuscript submission.
