Improving Rare Disease Recruitment With Data And AI

Rare diseases affect 300 million people worldwide, yet many individuals still lack adequate treatment options despite these conditions. Citeline’s latest research reveals that recruiting for these studies is often hindered by extreme diagnostic delays—with 28% of patients waiting seven years or more for a correct diagnosis—and a significant gap in trial awareness. While 88% of patients are willing to consider investigational treatments, over half remain only "somewhat aware" of available clinical trials.

To bridge this gap, sponsors must prioritize building long-term community trust and involving patients in trial codesign. By leveraging deep data and natural language processing to identify clinical signals beyond standard codes, sponsors can find patients much earlier in their journey. Moving from episodic engagement to sustained community partnerships ensures that trial designs reflect the day-to-day realities of patients, reducing barriers to participation and accelerating the delivery of life-changing therapies.