Epidemiology and access to research for participants in a rare disease real-world data research platform
Natural history studies are often complicated by small and geographically dispersed patient populations, especially in the case of rare diseases. Here, we characterize the geographic distribution of research participants across 35 rare conditions using our real-world data (RWD) platform. We also characterize the distance these patients would need to travel to participate in observational or interventional studies in their condition. Our results underscore the importance of an improved understanding of patient geography to inform study site selection and recruitment efforts. This poster was presented at the National Organization for Rare Disorders (NORD) Breakthrough Summit in October 2021.