Incredible scientific and technological advances in recent years have improved our collective understanding of many rare diseases. This is a very exciting time for the rare disease community. These diseases are often severe conditions that affect a very small population – fewer than one in 2,000 Canadians.
However, too many Canadians cannot benefit from these technologies. Rare disease therapies are launched in Canada later than in the United States and/or Europe – a median of 340 days later[1] – and, in many cases, are not even available for patients in Canada.
That is because the lengthy Canadian pharmaceutical review and approval system is not organized appropriately to account for the distinct nature of how medications designed to treat small patient populations are developed. Canada is virtually alone in not using tailored review processes and incentives to encourage manufacturers to bring rare disease treatments to patients.
In recent reports, both the House of Commons Standing Committee on Health,[2] and the federal government’s Advisory Council on the Implementation of National Pharmacare[3] have recognized the need for a distinct approach to reviewing and approving rare disease treatments. RAREi will continue to foster a dialogue between rare disease stakeholders and policy makers to ensure that people affected by rare diseases can access important new therapies.
[1] Rawson, N., Fraser Institute, Regulatory, Reimbursement, and Pricing Barriers to Accessing Drugs for Rare Disorders in Canada, 2018: https://www.fraserinstitute.org/sites/default/files/barriers-to-accessing-drugs-for-rare-disorders-in-canada.pdf
[2] RAREi – The Canadian Forum for Rare Disease Innovators, Unique approach needed: Addressing barriers to accessing rare disease treatments, October 31, 2018: https://www.linkedin.com/feed/update/urn:li:activity:6556579888877363200. See also Standing Committee on Health, Canadians Affected by Rare Diseases and Disorders: Improving Access to Treatment, February 2019: https://www.ourcommons.ca/DocumentViewer/en/42-1/HESA/report-22/.
[3] Advisory Council on the Implementation of National Pharmacare, A Prescription for Canada: Achieving Pharmacare for All, June 12, 2019: https://www.canada.ca/en/health-canada/corporate/about-health-canada/public-engagement/external-advisory-bodies/implementation-national-pharmacare/final-report.html.
The Canadian Forum for Rare Disease Innovators is a Sponsor of Research Canada’s Your Candidates, Your Health 2019 Federal Election Campaign. To learn more, visit yourcandidatesyourhealth.ca.