Context is Everything: Using Real-World Data to Redefine Evidence for Ultra-Rare Diseases

In ultra-rare diseases, where traditional control arms are often impractical, regulators still require meaningful context to interpret trial outcomes. Here’s how a biotech company partnered with Panalgo, leveraging NorstellaLinQ to build a regulatory-grade real-world control dataset that combined deep unstructured clinical notes, AI-enabled data curation, and rigorous propensity score matching. By creating a highly comparable real-world cohort, the company provided critical context for survival outcomes, supporting its FDA submission and strengthening stakeholder confidence.