Optimizing the Use of Real-World Evidence as Part of Decision-Making for Drugs for Rare Diseases


( Last Updated : August 11, 2022)

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In 2021, CADTH launched a learning period to better understand how to optimize the use of real-world evidence to inform decision-making for drugs for rare diseases. As part of the learning period, CADTH co-hosted a Best Brains Exchange with the Canadian Institutes of Health Research (CIHR) and the Canadian Organization for Rare Disorders (CORD).

The event was held over 2 days in the fall of 2021 with 137 participants from Canadian stakeholder groups that collect, hold, analyze, and use data for regulatory and health technology assessment purposes. The discussions identified 2 overarching themes for consideration relating to the use of real-world data and real-world evidence for decision-making pertaining to rare diseases in Canada: