Scoping review of the recommendations and guidance for improving the quality of rare disease registries

BackgroundRare disease registries (RDRs) are valuable tools for improving clinical care and advancing research. However, they often vary qualitatively, structurally, and operationally in ways that can determine their potential utility as a source of evidence to support decision-making regarding the approval and funding of new treatments for rare diseases.ObjectivesThe goal of this research project was to review the literature on rare disease …