Strengthening reimbursement decision-making for rare disease drugs in Canada

A new article published in Healthcare Papers discusses the feasibility of using managed access agreements (MAAs) to mitigate the challenges associated with expensive drugs for rare diseases (EDRDs) in Canada. It can be difficult for reimbursement decision-makers to evaluate the cost-effectiveness of EDRDs, particularly given that rare disease drugs often have less available evidence than other pharmaceuticals.

MAAs have already been implemented in several jurisdictions in an attempt to bridge this evidence gap. These agreements are fixed-term contracts which fund EDRDs on an interim basis, allowing patients to access them while further information is collected as to their real-world effectiveness. This additional data can then be used to make a better-informed final reimbursement decision.

In the Canadian context, there are specific considerations which would need to be accounted for in the case of large-scale application of MAAs. Provisions would need to be made for information-sharing between provinces to ensure sufficient levels of data are collected through the agreements. As such, data infrastructure and governance systems would need to be strengthened to allow this while respecting differences in provincial privacy and drug legislation.

Read more about this here.