CRA Insights

Identifying rare disease patient populations via integrated claims data

November 18, 2025
Blue pipettes filling test tubes

Rare diseases are increasingly driving drug development due to scientific advancements, government incentives, and a growing understanding of genetic predisposition.

Given the complexity of diagnosing rare disease patients, identifying real-world prevalence rates is difficult, as ranges from academic literature are often wide with varying methodologies. As a result, biopharma companies often encounter unique challenges when looking to better understand the epidemiology and addressable market opportunity of a rare disease population to inform drug development and launch strategies.

In this Insight, CRA Life Sciences consultant Cliff Li emphasizes how beginning with a general framework for sizing difficult-to-identify markets and then customizing it to meet specific objectives can facilitate the use of integrated data in rare disease patient populations to inform demand forecasts, clinical trial design, and commercial launch planning.

Read more about identifying rare disease patient populations via integrated claims data in the US here.

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