CURE ID: A Platform to Collect Real-World Treatment Data for Drug Repurposing in Rare Genetic Disorders

Rare diseases collectively affect millions of Americans, but less than 5% have approved treatments, and new drug development remains limited. For such diseases, drug repurposing may be an effective strategy to find new treatment options. In the rare genetic disorder community, drugs are frequently prescribed off-label. This information is rarely available for research, but if captured, could be leveraged to accelerate the identification of candidate drugs to be evaluated for safety and efficacy of the treatment of rare diseases. CURE ID is a publicly available treatment registry that collects real-world treatment data directly from healthcare providers, patients, and care partners in a consistent format. By aggregating this information, CURE ID can generate hypotheses for follow-up targeted research of repurposed drugs, potentially leading to the approval of these drugs for new indications. The success of the platform is predicated on its adoption in the rare disease community and routinely reporting treatment experiences to CURE ID.