Specific, Fair and Transparent: A Canadian Process for Funding Drugs for Rare Diseases.

Abstract

In Canada, the focus on value for money and evaluating efficacy according to traditional ways has presented challenges to the funding of drugs for rare diseases (DRDs). This commentary validates and extends two worthy recommendations from the lead paper in this issue of Healthcare Papers (Sirrs et al. 2023). The paper's first recommendation, for a pan-Canadian approach for collecting evidentiary data, is critical. In the commentary, I add to this finding by suggesting that we enable patients to track and measure their response to treatment through data capture. The second recommendation is a pan-Canadian framework for funding DRDs. I extend the recommendation with an argument for public and private payer guidance as well as a fair and transparent funding framework solely for DRDs.