Objective: As a grantee of the Chan Zuckerberg Initiative's Rare as One Project, Cure HHT created a research network (CHRN) to develop a research roadmap for the next 3-5 years. CHRN is led by HHT patients working in collaboration with researchers and clinicians to prioritize research initiatives by order of feasibility, impact, importance, and logical sequence. Methods: Comprehensive surveys were sent to the HHT patient and professional community to identify perceived gaps in research and treatments. 1,204 patients answered 49 questions, 42 HHT scientists answered 17 questions, and 96 HHT clinicians answered 25 questions. Based on the analyzed results of patient needs, broad-topic work streams were established which included patients, clinicians, and researchers. Work streams performed a comprehensive literature review on their respective topic, discussed gaps in research, and proposed recommendations to fill each knowledge gap. Finally, a 2-day convening was held with all working groups to present recommendations and vote on a consensus for a final research roadmap to meet patient needs over the next 3-5 years. Results: 8 work streams identified 31 recommendations to address patient needs. Recommendations were selected by anonymous vote after their presentation to the group and ranked by feasibility, impact, and importance. Additionally, resources and tools were identified to achieve all recommendations. Conclusion: CHRN is poised with specific, actionable goals to address patient priorities. The research roadmap will serve as a guide for next steps, research funding opportunities, and collaboration between patients, clinicians, and researchers at an unprecedented level for the HHT community.
