Advancing Rare Disease Research Through AI Innovation and Patient Trust

Artificial intelligence is rapidly reshaping the landscape of rare disease research -- but how close are we to realizing its full potential?

In this episode, Tanya Binette (Director of Therapeutic Expertise, Rare Disease at ICON plc) and Roz Round (SVP of Operational Strategy, Patient and Site Engagement at Precision for Medicine) explore how AI is being applied across the rare disease clinical trial lifecycle, from drug discovery and protocol design to patient identification and engagement.

The discussion highlights both the promise and complexity of using AI in a research space defined by small patient populations, fragmented data, and unique ethical considerations. Guests emphasize the importance of patient trust, regulatory alignment, and responsible innovation, while also identifying opportunities to accelerate trials, improve access, and empower patients through AI-enabled tools.