Hiding in Plain Sight: Leveraging AI to Redefine Patient Finding & Outcomes Prediction

Across therapeutic areas and development stages, the ability to identify the right patient population, and understand what will happen to them, remains one of the most consequential and most consistently underestimated challenges in clinical research and real-world evidence generation.

This is particularly true in rare disease research which faces a fundamental paradox: the patients who most need to be found are the hardest to find, and the outcomes that matter most are the least studied.

In this eBook, we present results from PhenOM® across six rare diseases in the United States. The diseases profiled in this report were selected precisely because they stress-test each of these questions at the extreme end of rarity. Some have fewer than a dozen known patients globally, while others have thousands of patients hiding in plain sight. All of them represent a meaningful frontier for patient finding and outcomes prediction and illustrate what AI can do when applied at scale.