Out Of The Shadows: How Patient Advocacy Groups Are Rewriting The Clinical Trial Playbook

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 many sponsors realize.

Organizations like the Alagille Syndrome Alliance (ALGSA) are quietly recruiting patients, providing protocol feedback, running observational studies, convening scientific networks, and counseling families through the real-world hurdles of participation, often with a fraction of the resources they actually need.

This panel discussion covers:

• 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