Data Sheet 1_“We are ambassadors, we are advocates”: rare disease patient advocacy groups as knowledge brokers across health and social systems—a qualitative study from Poland.pdf

BackgroundRare diseases (RDs) pose a significant public health concern, particularly in Poland, where awareness among healthcare professionals, educators, and the general public remains low. Rare disease patient advocacy groups (RDPAGs), often led by caregivers, play a vital yet under recognized role in addressing these gaps through informal education and advocacy. This study explores how RDPAGs help fill systemic gaps in RD-related education.
MethodsThis qualitative descriptive study draws on 11 interviews with leaders of Polish RDPAGs engaged in advocacy, education, and public engagement. Interviews were analysed using Reflexive Thematic Analysis, following Braun and Clarke’s six-phase framework, and reported in line with COREQ guidelines.
ResultsThe analysis identified four interrelated themes describing the educational and advocacy practices of RDPAGs. Participants portrayed RDPAGs as: (1) acting as ambassadors of knowledge across sectors by translating scientific and experiential expertise into accessible information; (2) supporting families through lived experience by providing curated resources, peer mentoring, and practical guidance, particularly following diagnosis; (3) engaging with and sometimes challenging professional expertise by sharing caregiver-informed knowledge, co-developing materials, and initiating dialogue with healthcare and education professionals; and (4) raising public awareness and engaging in policy-oriented advocacy through campaigns and inter-organizational collaboration. Collectively, participants described these practices as addressing systemic knowledge gaps and supporting cross-sectoral care navigation.
ConclusionThe findings indicate that healthcare, education, and policy systems in Poland already rely substantially on the informal educational and advocacy work of RDPAGs. Formal recognition and support may help stabilize these contributions, provided that issues of sustainability, role boundaries, and uneven organizational burdens are taken into account.