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

In 2021, we 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, we 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: