For many sponsors, “engaging patient advocacy” still means an advisory board here or a one-off awareness campaign there. But in rare disease, advocacy organizations are playing a far more substantive role than most sponsors realize.

Organizations like the Alagille Syndrome Alliance (ALGSA) are quietly recruiting patients, providing protocol feedback, running their own observational studies, convening global scientific networks, and counseling families through every hurdle of participation – often with a fraction of the resources they actually need.

In this session, ALGSA leaders Roberta Smith and Cher Bork, together with Scientific Research Coordinator Karsten Baumgaertel, join moderator Jim Wahl to discuss how advocacy groups are “rewriting the playbook” on patient recruitment, education, and real-world study support across rare disease.

We’ll talk candidly about what sponsors don’t see, the roles advocacy groups already play in study success, and what stronger, more collaborative partnerships can unlock for patients and for rare disease research – with insights that extend well beyond any single therapeutic area.

What you’ll learn:

• Why families turn to advocacy groups first

• How ALGSA identifies recruitment barriers long before they appear in site metrics

• Where traditional sponsor-advocacy engagement breaks down

• Why some of advocacy’s most meaningful contributions remain invisible to patients and sponsors