Professor Emeritus, School of Health Policy and Management, York University, Associate Professor, Faculty of Medicine, University of Toronto, Toronto, ON.
Clinical Professor, Faculty of Medicine, University of British Columbia, Vancouver, BC.
Healthc Policy. 2024 Aug;19(4):19-26. doi: 10.12927/hcpol.2024.27334.
The number of expensive drugs for rare diseases (EDRDs) approved by Health Canada and their contribution to healthcare costs have been rapidly increasing. The federal government has announced a three-year funding commitment of $1.4 billion for EDRDs, but principles need to be developed for how that funding will be allocated, especially in cases where insufficient data are available to guide decision making. Here, we review the role of evidence quality in making choices and draw on the experience from other countries to put forward five principles about how the money should be spent.
加拿大卫生部批准的昂贵罕见病药物（EDRDs）的数量及其对医疗保健成本的贡献一直在迅速增加。联邦政府已宣布为 EDRDs 提供为期三年的 14 亿加元资金承诺，但需要制定资金分配原则，特别是在缺乏数据来指导决策的情况下。在这里，我们回顾了证据质量在决策中的作用，并借鉴其他国家的经验，提出了关于如何使用这些资金的五项原则。
