Emerging roles and opportunities for rare disease patient advocacy groups.

Background: Patient advocacy groups (PAGs) serve a vital role for rare disease patients and families by providing educational resources, support, and a sense of community. Motivated by patient need, PAGs are increasingly at the forefront of policy, research, and drug development for their disease of interest.

Objectives: The study explored the current landscape of PAGs in order to guide new and existing PAGs on available resources and challenges to research engagement.