Pan- Canadian Rare Disease Registries

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Project Status:
Completed
Project Line:
Health Technology Review
Project Sub Line:
Environmental Scan
Project Number:
ES0369-000
Effective finish date:

Optimal use of real-world evidence (RWE) to inform health care decision-making requires timely access to data that are fit for purpose. RWE is particularly important for rare diseases due to smaller numbers of patients and the associated challenges with conducting randomized clinical trials.

With the support of Health Canada, CADTH has launched a learning period to better understand how to optimize the integration of RWE into decision-making processes for drugs for rare diseases.

To achieve this goal, CADTH researched sources of real-world data for rare disease in Canada.

As part of this learning period, using publicly available information and the Registry Evaluation and Quality Standards Tool (REQueST) developed by the European Network for Health Technology Assessment (EUnetHTA), a preliminary assessment of Canadian rare disease registries was completed.