Imagine waking up one morning and discovering that swallowing, speaking or even taking a breath feels like climbing a mountain. That is a reality for Canadians living with Myasthenia Gravis, a life-altering autoimmune neuromuscular disease that progressively weakens the muscles required for walking, speaking, swallowing and breathing.
Myasthenia Gravis turns routine activities into daily challenges and transforms independence into a constant struggle for stability and strength. Each day brings uncertainty, as even simple tasks can become exhausting or impossible.
Despite the recent approval of targeted biologics that can significantly improve muscle strength, quality of life and address long-standing unmet needs, Canadians living with Myasthenia Gravis continue to face a policy barrier that is both medically unjust and ethically unacceptable.
In several jurisdictions, including Alberta and Saskatchewan, individuals with generalized Myasthenia Gravis must try and then fail with a drug called rituximab before being allowed access to efgartigimod, a treatment that has been rigorously evaluated, approved by Health Canada, and recognized by the Institut national d’excellence en santé et en services sociaux (INESSS) and Canada’s Drug Agency (CDA) for both its clinical results and cost effectiveness.
Rituximab (Rituxan) is approved by Health Canada for certain cancers – non-Hodgkin’s lymphoma, chronic lymphocytic leukemia, rheumatoid arthritis and ANCA-associated vasculitis – but not for the treatment of Myasthenia Gravis. Its use for this condition is entirely off-label and supported only by limited retrospective evidence, primarily in rare MuSK-positive cases that represent a small minority of patients. The 2021 International Consensus Guidance for Management of Myasthenia Gravis is explicit on this point: “Rituximab is recommended only for MuSK-positive MG and not for acetylcholine receptor (AChR)-positive disease – which accounts for approximately 85 per cent of patients, where its efficacy remains unproven.”
Yet patients are told they must fail rituximab before receiving efgartigimod – a drug specifically designed, studied and approved for their disease. It forces patients into a clinical dead end, compelling them to use a therapy never approved for their condition before granting access to one backed by modern evidence and regulatory review.
Requiring individuals to fail a drug that was never intended or approved for their condition contradicts the core principles of patient-centred care and undermines public trust in the health system. It also ignores the progress made in precision medicine, where treatments are now developed for specific disease subtypes based on strong scientific evidence, and the significant investment Canada has already made in reviewing a new drug’s safety, efficacy and cost-effectiveness.
Requiring individuals to fail a drug that was never intended or approved for their condition contradicts the core principles of patient-centred care.
In practice, rituximab carries risks that make its mandated use even more troubling. It can cause hypogammaglobulinemia, a condition that lowers immune protection and increases vulnerability to infections. Ironically, patients who develop this complication often require immunoglobulin replacement therapy at a time when Canada is already facing sustainability challenges and supply pressures for these life-saving products. This policy not only delays effective care but also risks worsening the very system pressures it seeks to manage.
Precedent from other disease areas shows that such requirements can and should be changed. In rheumatoid arthritis, inflammatory bowel disease and multiple sclerosis, provinces and insurers have modified or removed step-therapy protocols when they caused treatment delays or compromised patient outcomes. In the United States, several states have passed legislation limiting “fail-first” rules for serious or rare diseases after recognizing that these policies often harm patients more than they help.
Imagine telling a parent of a child with a rare progressive disease like spinal muscular atrophy that they must first try and fail a drug not approved for their child’s condition before receiving disease-modifying therapies like Spinraza, Risdiplam or Zolgensma – treatments known to preserve mobility and extend life. We would consider that unconscionable. Yet this is precisely what is happening to adults living with Myasthenia Gravis.
Within Canada, the federal government’s Strategy for Drugs for Rare Diseases, announced with great optimism in 2023, acknowledges that people living with rare and complex conditions face inequities in access and require accelerated, flexible pathways for reimbursement. Yet, two years later, tangible progress remains limited.
The experience of the Myasthenia Gravis community illustrates exactly why this strategy is necessary and why it must move beyond promises to policy change. Adults living with rare diseases continue to face inconsistent, slow and inequitable access to therapies already proven to be safe, effective and life changing.
This policy for efgartigimod is not an isolated problem. If left unchallenged, Muscular Dystrophy Canada is concerned that the same “fail-first” logic could apply to other emerging targeted therapies for Myasthenia Gravis, such as ravulizumab, rozanolixizumab, nipocalimab, and zilucoplan. Instead of building a responsive system that embraces innovation, Canada risks creating a patchwork of restrictive rules that deny patients timely access to the very treatments designed to keep them out of crisis.
We believe that clinicians, in partnership with their patients, should always have the discretion to consider and prescribe off-label therapies when evidence, experience or individual circumstances warrant it. This shared decision-making respects both medical expertise and patient choice. That flexibility is essential. But clinical discretion and patient partnership are very different from policy coercion. When an unapproved drug becomes a mandatory first step, choice is no longer clinical or collaborative, it becomes bureaucratic.
Myasthenia Gravis is not a disease that can wait for bureaucratic cycles or administrative hesitation. Every week of delay increases the risk of crisis, hospitalization and irreversible harm. Behind every policy delay is a person fighting to breathe, to walk, to work and to live with dignity. We urge provinces that require Rituxan use before granting access to new, targeted biologics to remove this criterion immediately.
Together as patients, family members, clinicians, policymakers and advocates, we can build a system guided by science, compassion and partnership, ensuring that access to life-changing treatment is never determined by outdated policy.
