Authors :
Vanessa Vogel-Farley, BA – Rare Epilepsy Network
Presenting Author: Christina Saninocencio, PhD – Fairfield University
Ilene Miller, JD, LLM – Rare Epilepsy Network
Yssa DeWoody, PhD – Lundbeck Pharmaceuticals
Priya Balasubramanian, PhD – CURE Epilepsy
Dennis Lal, PhD – Department of Neurology, McGovern Medical School, The University of Texas Health Science Center at Houston, Houston, TX, USA
Caitlin Grzeskowiak, PhD – Epilepsy Foundation
Rationale:
Patient advocacy organizations are increasingly essential partners in rare disease research, yet little is known about their capacity to engage in multisite studies or drive research readiness across diverse conditions. The Rare Epilepsy Network (REN), founded in 2013, is a coalition of more than 150 rare epilepsy advocacy organizations that has tripled in size in the last four years. REN's longevity and experience position it uniquely to observe trends across this rapidly changing landscape. REN launched a membership-wide survey to identify research priorities, assess operational challenges, and guide community-informed strategy. This abstract reports findings from the 2024 REN Membership Survey, highlighting common needs and gaps that shape how rare epilepsy organizations contribute to collaborative science.
Methods:
The REN conducted an electronic survey of its membership organizations in 2024 to assess current research collaboration activities, interest levels, and barriers to participation. The survey captured organizational characteristics, research engagement patterns, resource availability, and perceived obstacles to multi-site research initiatives.
Results:
A total of 73 organizations completed the survey, representing 71 unique rare epilepsy disorders. While the vast majority expressed strong interest in research collaboration, only 38% reported current active participation in multi-site research initiatives, revealing a substantial gap between interest and actual engagement. This disparity was consistent across all research areas surveyed, including natural history studies, clinical assessments, electronic health record research, biosample research, and patient-reported outcome data. Three main obstacles prevented organizations from translating research interest into participation: limited staffing capacity, unclear pathways for establishing research partnerships, and funding constraints.
Conclusions:
The 2024 REN Membership Survey reveals a highly committed and motivated rare epilepsy community with strong enthusiasm for research collaboration. However, significant structural barriers, particularly related to organizational capacity, partnership facilitation, and financial resources, prevent many organizations from translating their interest into active participation. The Survey highlights a committed, motivated rare epilepsy community that is eager to participate in research but constrained by bandwidth, funding, and infrastructure. Addressing these infrastructure and resource gaps will be essential for maximizing the research potential of the rare epilepsy community and advancing therapeutic development for these underserved patient populations. Bridging this gap will require tailored support such as partnership matching and resource sharing initiatives with shared infrastructure and training programs, where establishing sustainable funding mechanisms and standardized research frameworks can transform REN from a networking organization into an active research enabler, directly addressing the gap between the community's strong interest and current participation levels.
Funding: N/A