Last year, in Healthy Debate, we warned that Canadians were increasingly finding themselves in an impossible position: treatments could be approved yet remain inaccessible. One year later, the experience of Canadians living with Friedreich ataxia (FA) has become a powerful example of exactly that reality.
Health Canada approved SKYCLARYS (omaveloxolone) in March 2025 through a Priority Review.
Canada’s Drug Agency recommended reimbursement.
Quebec’s health technology assessment agency, INESSS, recognized its therapeutic value.
Researchers generated data. Patients and families shared their experiences. Clinicians provided evidence.
And yet Canadians living with FA still cannot access the only approved treatment for their disease.
If that sounds like a contradiction, it is.
And it should force all of us to ask a difficult question: Is Canada’s rare disease promise beginning to collapse?
In 2023, the federal government announced a National Strategy for Drugs for Rare Diseases, supported by up to $1.5 billion in funding and a commitment to improve access to promising therapies for Canadians living with rare conditions. Three years later, Canadians living with FA are asking a simple question: where is the fulfilment of that promise?
FA is a rare, inherited, progressive neurodegenerative disease that typically begins in childhood or adolescence. It affects balance, coordination, speech, mobility and heart function. Over time, individuals lose independence and opportunities. This is not a disease that pauses while policymakers deliberate or negotiations continue. Function lost today may never be recovered tomorrow.
The disease also carries particular significance in Canada. Due to founder effects, FA occurs at higher rates among certain French-Canadian and Acadian populations, particularly in Quebec and Atlantic Canada. For decades, treatment for FA focused largely on managing symptoms rather than altering the course of the disease itself. Families, clinicians, researchers and advocacy organizations worked tirelessly in the hope that science would deliver something more.
That day appeared to arrive on March 17, 2025.
Health Canada’s approval of SKYCLARYS marked a historic milestone. The therapy received Priority Review because it addressed a serious condition with a significant unmet medical need. For the first time, Canadians living with FA had access to a therapy specifically approved to treat their disease.
What followed should have represented a pathway toward access.
Patients and caregivers participated in submissions and consultations. Clinical experts provided evidence. Canada’s Drug Agency reviewed the evidence and issued a positive reimbursement recommendation. More recently, INESSS reassessed the therapy and recognized its therapeutic value for eligible patients. By any reasonable measure, the system appeared to be working.
Yet this past June 10, the pan-Canadian Pharmaceutical Alliance (pCPA) announced that negotiations with Biogen had ended without agreement.
Just like that, the pathway stopped.
The result is remarkable and troubling. After successfully navigating every major stage of Canada’s regulatory and reimbursement process, the first and only approved treatment for FA remains out of reach for the very people it was intended to help.
This is precisely the concern that led Muscular Dystrophy Canada to launch its What About Canada? initiative. Across the neuromuscular disease community, patients repeatedly tell the same story. Therapies become available in other countries while Canadians face years of uncertainty navigating regulatory reviews, health technology assessments, negotiations and reimbursement decisions. Even after a therapy receives regulatory approval, access remains unpredictable, prolonged and often dependent on where a person lives.
The experience of the FA community has become one of the clearest examples of this problem. There will be considerable debate about why negotiations failed. Some will point to pricing expectations. Others will cite affordability concerns, budget pressures or broader questions about the sustainability of public drug programs. These are legitimate discussions. But regardless of where one places the blame, the reality is that all parties involved – Biogen, the pCPA and participating governments – had a responsibility to find a path forward for patients.
Bringing the first approved FA therapy to Canada is a significant achievement. It represents decades of scientific research and offers hope to a community that has waited far too long for meaningful therapeutic progress. But innovation alone is not enough. When a company brings forward the first and only approved treatment for a progressive neurodegenerative disease, it also assumes a responsibility to pursue every reasonable opportunity to ensure patients can benefit from it. Canadians living with FA should not be left questioning whether commercial considerations ultimately took precedence over patient access. After years of waiting, patients and families deserved more than a failed negotiation and a closed file.
The pCPA and participating governments must also confront difficult questions about what patient-centred decision-making actually means. If a therapy can receive regulatory approval, a positive reimbursement recommendation and recognition of therapeutic value, yet still fail to reach patients, then the system owes Canadians more than a brief notice that negotiations have ended. It owes them transparency, accountability and a credible explanation of how patient interests were weighed.
Patient experience is repeatedly cited as a guiding principle of modern health technology assessment. Patients are invited to share their stories, describe the realities of living with disease and explain what access to treatment would mean for their lives. Yet when negotiations begin, those voices largely disappear from the room.
A system cannot claim to be patient-centred if patient perspectives help inform recommendations but have no meaningful role in the decisions that ultimately determine access.
Particularly troubling are reports that international pricing considerations, including “Most Favoured Nation” pricing expectations, may have contributed to the impasse, and the growing number of unsuccessful negotiations in 2026. Canadians deserve a transparent conversation about whether policies intended to achieve international pricing objectives are inadvertently creating barriers to access at home.
If Canada has reached a point where achieving international pricing objectives takes precedence over ensuring access to the first approved treatment for a progressive neurodegenerative disease, then something has gone profoundly wrong. Canadians should not become collateral damage in a pricing framework designed to influence markets beyond our borders.
Affordability matters. Stewardship of public resources matters. Sustainability matters.
But access matters, too.
A treatment that exists but cannot be accessed is not a success story. A treatment that is approved but unavailable is not a success story. And a health-care system that measures success primarily through cost containment while patients remain untreated risks losing sight of its purpose.
This is not an argument against responsible negotiation. Public resources must be managed carefully. Manufacturers must be accountable. Value matters.
But outcomes matter, too.
The purpose of Canada’s drug review and reimbursement system is not simply to review therapies, conduct assessments, facilitate consultations or negotiate prices. Its purpose is to ensure that Canadians can access treatments that are safe, effective and clinically meaningful. When that objective is not achieved, particularly after years of review, engagement and positive recommendations, the system must be willing to examine why.
The implications extend far beyond FA. Canadians living with Duchenne muscular dystrophy, myasthenia gravis, spinal muscular atrophy, Pompe disease and countless other rare conditions are watching closely. They are being asked to participate in research, share their experiences, contribute to evidence generation and place their trust in processes that promise to improve access. Each time a therapy successfully navigates years of review only to become stranded before reaching patients, confidence in that promise erodes.
Canada’s National Strategy for Drugs for Rare Diseases was intended to change this story. Yet the experience of the Friedreich ataxia community suggests that important structural barriers remain unresolved.
This situation can still be fixed: Biogen, the pCPA, participating governments, private payers and all parties involved should return to the table with urgency and a shared commitment to finding a solution. Canada should introduce greater transparency when negotiations fail, meaningfully incorporate patient experience into pricing and reimbursement discussions and develop pathways that prevent first-in-class therapies for serious rare diseases from becoming stranded indefinitely between approval and access.
Today, Canadians living with Friedreich ataxia are still waiting for access. What About Canada?
