Imagine having a family member with a debilitating condition that slowly affects their ability to walk, speak and live independently. Now imagine learning there’s a treatment. It’s safe, effective and approved by Health Canada. Your hopes lift. Relief washes over you. Finally, something.
Then you’re told: Not so fast. Not in your province. Not yet. Maybe never. You qualify, but geography disqualifies you. It’s a cruel twist, a gut punch that turns hope into heartbreak.
This is the lived reality of families across Canada affected by neuromuscular disorders. Friedreich Ataxia (FA) is one such rare and progressive condition. The first and only treatment for FA, Skyclarys (omaveloxolone), was approved by Health Canada in early 2025. Canada’s Drug Agency (CDA) recommended it for public reimbursement. But in Quebec, the Institut national d’excellence en santé et en services sociaux (INESSS) said no, saying the treatment lacks long-term proof and cost-effectiveness – despite also having been approved in the U.S. and Europe.
We are told health care in Canada is equal for everyone. But it is not. What you get depends on where you live. Some provinces say yes. Others say no. It is a system full of gaps, delays and unfair rules. And Canadians pay the price.
The injustice is even more glaring when you consider that FA disproportionately affects French Acadian communities rooted in Quebec and Eastern Canada. A treatment exists, and yet the very people most affected are being told to wait. Or worse, that their health is not worth the cost.
Let’s call this what it is: A national failure.
Canada’s process for reviewing and reimbursing new treatments is fractured, sluggish and often contradictory. A drug can be deemed safe and necessary by Health Canada and endorsed by national reviewers, but unless each province agrees using its own budget, process and priorities, Canadians affected by neuromuscular disorders are left in limbo. Some families may pay out of pocket. Others cannot. Most are left watching their loved ones decline, knowing help exists but is just out of reach.
Let’s call this what it is: A national failure.
We were told the federal Rare Disease Drug Strategy would fix this. That it would create a coordinated and equitable system to ensure access to life-changing therapies for people with rare diseases. But for those living with FA, and many others, the strategy has yet to make any real or meaningful difference. It promised change. Instead, we have seen more delay, more confusion and more divergence between drug regulator decisions and provincial realities.
Canada is falling behind, not just compared to other countries, but within its own borders. We love to say we have a universal health-care system in Canada. But what does “universal” really mean if your access depends on your postal code? Why do people in Quebec affected by FA face denial while those in Ontario might soon access treatment? And on the flip side, why can adults with spinal muscular atrophy (SMA) access disease-modifying therapies in Quebec, while in most other provinces they remain excluded entirely? These inconsistencies are not theoretical. They are daily, painful reminders that where you live determines whether you get to live well or at all.
For families, this is not policy. It is personal. While our systems debate cost-effectiveness, people lose mobility. Time is muscle. While ministries negotiate budgets, people lose hope. For rare disease communities, the cost of delay is irreversible. As one mother recently shared with us: “It is unbearable to know a drug exists that could help your child and still be told you can’t have it.”
Muscular Dystrophy Canada (MDC) stands firmly with the FA community and with all Canadians affected by neuromuscular and rare diseases. We are agnostic to any specific treatment. Our goal is simple and unwavering: To ensure that all people have timely, fair and informed access to the treatments they need and to preserve treatment choice based on patient values and clinical judgment.
Skyclarys should have marked a turning point for Canadians with FA. Instead, it has become a case study in the systemic barriers that continue to curb innovation and delay access to life-changing treatments. We need real reform. We need timely, equitable and transparent access to therapies, especially for rare diseases. The Rare Disease Drug Strategy must become more than a promise.
What we want is simple. A Canada where no family has to beg for a treatment their loved one qualifies for. A Canada where access does not depend on your postal code, but on your needs. Together, we can build a system that truly lives up to our values of fairness, dignity and care for all.
This article has been updated.
