PCORI intends to pursue this limited competition opportunity, open to all PCORnet^® Clinical Research Networks (CRNs)/Health Plan Research Networks (HPRNs) and their partner organizations to perform an observational research study that will answer one or more important research questions about the care of patients with rare diseases. A secondary goal is to enhance the capabilities for the conduct of multi-site rare disease research by creating partnerships, methods, tools, and linkages that will facilitate future comparative effectiveness studies for the rare disease community.

Through this initiative, PCORI seeks to fund high-quality observational cohort studies of people with rare diseases or conditions. This initiative will require broad use of PCORnet resources for research, collaboration between a minimum of two CRNs/HPRNs, and the establishment of partnerships with rare disease patient organizations, with attention to representing the diversity of the population affected by the disease.