The Value of Patient Registries: Common Needs for Rare Diseases

Key Messages

• Rare disease registries platforms serve an important function in assembling and linking real-world data for a broader population of patients—they collect real-world data to evaluate natural histories of disease, outcome measures, standards of care, and drug safety and effectiveness.
• Several barriers remain that impede the widespread use of rare disease registries for real-world data, including concerns over data quality, governance, and implementation of the assembled real-world evidence for decision-making purposes.
• A report published in this issue of the journal is the first in a series of case studies that CADTH is pursuing that focus on registries for rare diseases, with the goal of leveraging the power of registries to improve the portrait of care for several rare diseases and support the decision-makers responsible for enacting these changes.