This article originally appeared in Maclean’s print and healthinsights.ca on February 16, 2023
Canada desperately needs a rare disease strategy that’ll enable better patient care and access to treatment for those living with rare diseases.
The start of a new calendar year signifies hope and new beginnings for many, but for the 1 in 12 Canadians affected by a rare disease, it marks another year without news of a national rare disease strategy.
The federal government offered a glimmer of hope for rare disease patients in 2019 by announcing a $1 billion investment over two years, and up to $500 million per year on an ongoing basis, to support a rare disease strategy. The strategy was supposed to be implemented in 2022 to 2023 but, as we near the end of that fiscal year, patients are still waiting to see the investments and benefits.
Addressing gaps and challenges
Unfortunately, most rare disease patients don’t have the luxury of time. Any delay in patient access to diagnostics and treatments has the potential to significantly impact health outcomes and disease progression, and to place additional strain on already challenged health systems. Currently, only 60 percent of available rare disease treatments make it into Canada and most get approved up to six years later than in the U.S. or Europe, according to the Canadian Organization for Rare Disorders.
Canada is also the only G7 country without an existing national approach to rare diseases. A comprehensive Canadian strategy should include an accelerated drug review, approval, and access pathway, a standard definition for what consititutes a rare disease in line with our international peers, the use of real-world data, and rare disease centres of excellence. These elements within a national rare disease strategy would help address the persistent gaps and challenges across the country in diagnosing, monitoring, and treating rare disease patients — two thirds of whom are children — in an efficient and effective way.
It’s time for the government to work with the provinces and other stakeholders to translate promising announcements into meaningful impact for patients and their loved ones — because the 2023 clock has already started ticking.
Translating announcements into impact
Additionally, the government needs to consider how other policy and regulatory changes might affect the success of a national strategy for drugs for rare diseases. For example, the lack of predictability and clarity in the most recent version of the proposed changes to the Patented Medicine Prices Review Board’s guidelines will discourage businesses from launching new medicines in Canada, including those for rare diseases.
Health Canada concluded its stakeholder consultations on a pan-Canadian rare disease strategy almost two years ago. The funding is available and there’s a real opportunity for the federal government to lead the way with a rare disease strategy that prioritizes patient access to rare disease treatments.
It’s time for the government to work with the provinces and other stakeholders to translate promising announcements into meaningful impact for patients and their loved ones — because the 2023 clock has already started ticking.
