What Happens When Medicine Meets Movement: Lessons from the Frontlines of Neuromuscular Advocacy
Suchandra Banerjee
Stakeholder Engagement, Patient Advocacy and Strategic Healthcare Communications | Storytelling for Policy Impact
When you work in healthcare communications and patient advocacy, you learn early that progress isn’t just about policies or protocols, it’s about people. It’s about the quiet shifts that happen when clinicians, researchers, and patient communities come together not just to treat disease, but to challenge the systems that shape healthcare itself.
Last week, I had the privilege of joining a remarkable gathering hosted by Muscular Dystrophy Canada A Seat at the Table: Where Neuromuscular Care Meets Policy (Thank you Homira Osman !). It wasn’t a typical conference. It was a space where various healthcare professionals came together, not to speak at one another, but to empower and think with one another.
What made it even more special for me, personally, was the opportunity to contribute as a speaker, not to the general public, but to a room full of clinicians committed to something bigger: moving the needle on advocacy for complex, often overlooked conditions like neuromuscular disorders.
When Advocacy is Not an Afterthought
In healthcare, advocacy is sometimes treated as an add-on, a well-meaning one, but sometimes (not always!) a secondary effort that comes in later to push the commercial process. However, for complex, often hard-to-diagnose conditions like neuromuscular disorders, that approach is HIGHLY inadequate.
Advocacy isn’t an accessory to care, it is a most important part of ensuring that patients aren’t left behind by systems that are slow to adapt or respond. This isn't a new concept, but it isn't as universal as you would like to think.
At A Seat at the Table, what struck me was the clear recognition of this reality. The workshop brought together physicians, researchers, and various advocates within the medical community not just to talk about the gaps in neuromuscular care, but to explore how the medical community could work more deliberately and more collaboratively to close them.
It was a reminder that true progress in healthcare demands more than evidence alone, especially in spaces where the margin for inertia is razor-thin and where patients, families, and communities carry the burden of that inertia EVERY SINGLE DAY.
Beyond the Clinic: Clinicians as Catalysts for Change
One of the workshop’s most powerful themes was the idea that the clinician’s role doesn’t end at the bedside. Dr. Jacqueline Dorothy Cochrane of the University of Ottawa , an important voice in the field, opened with a compelling reminder: health advocacy isn’t a separate skill set; it’s inextricably tied to good clinical care.
Dr. Cochrane emphasized that advocacy operates on a spectrum, from individual patient-level actions to broader system-level efforts. It can be as immediate as expediting critical test results or as complex as lobbying for expanded services like improved accessibility or mental health supports for neuromuscular patients. Drawing from her research (do check out her work on the Canadian Medical Education Journal) and frontline experience, she shared that up to 80% of a physician's ability to influence patient outcomes lies outside of traditional clinical interventions, rooted instead in the social determinants of health that advocacy seeks to address.
Perhaps more importantly, she underlined a shift in thinking: moving from advocating for patients to advocating with them. Real change, she reminded us, happens when lived experience is not just acknowledged, but invited into the advocacy process.
It’s not an optional add-on to clinical practice. It’s a professional responsibility, a way to extend care beyond what a prescription or a procedure can offer.
Yet, barriers persist. Lack of time, lack of training, and systemic inertia can make system-level advocacy feel daunting. However, as Dr. Cochrane noted, advocacy efforts don’t need to be solo ventures. Collaboration, she argued, is the key: working within multidisciplinary teams, partnering with patient organizations, and even reaching outside of medicine, to policymakers, city planners, and legal experts, to build the coalitions needed to push systemic change forward.
What resonated most for me was her humility. Dr. Cochrane didn’t pretend it was easy; she simply insisted it was necessary, and that conviction, shared by so many of the clinicians and advocates in the (virtual) room, set the tone for the discussions that followed that afternoon.
Up to 80% of a physician's ability to influence patient outcomes lies outside of traditional clinical interventions, rooted instead in the social determinants of health that advocacy seeks to address.
Systems in Motion: Rare Disease Policy and the Power of Collective Advocacy
If advocacy starts with the clinician, it gains force when it is anchored to systems. That idea came into sharp focus during the session on Canada’s evolving rare disease strategy.
In my talk, I traced the long road to Canada’s Rare Disease Drug Strategy, highlighting how Canada remained one of the few G7 countries without a national rare disease framework until very recently. I reflected on the community’s persistent efforts, from the failed 2012 orphan drug framework proposal to the 2023 launch of the national strategy, and the critical role patient advocacy groups like Muscular Dystrophy Canada have played along the way.
This wasn’t a story of linear progress. It was a reminder that for neuromuscular communities and rare disease patients more broadly, access to care has never been a guarantee. It’s been a hard-fought, fragile win built on storytelling, spreadsheets, and sheer willpower.
“The Rare Disease Strategy wasn’t built because Canada was ready. It was built because the pressure could no longer be ignored.”
The discussion deepened with insights from Sang Mi Lee, MBA, BSc Phm, RPh , an expert in drug reimbursement policy and former leader at Ontario’s public drug programs and the pan-Canadian Pharmaceutical Alliance (pCPA) from MORSE Consulting Inc. She pulled back the curtain on the HTA (Health Technology Assessment) process, offering a rare inside look at where and why rare disease therapies often stall after regulatory approval.
Sang Mi emphasized that while Health Canada | Santé Canada evaluates a drug’s safety and efficacy, the CDA ( Canada's Drug Agency ) and provincial payers are focused on cost-effectiveness, a calculus that rarely favours high-cost, low-evidence rare disease therapies. She showed that drugs for rare disease (DRDs), while receiving positive HTA recommendations at higher rates than other non-oncology drugs, still face longer timelines and complex negotiations before reaching patients, a process often stretching well beyond 16 to 20 months from initial submission to first provincial listing.
One chart she shared starkly captured the reality:
The bottleneck isn’t always science. It's systems, and for patients with progressive neuromuscular conditions, time isn’t an academic factor. It is a tangible, lived cost!
What made Sang Mi’s session especially powerful wasn’t just the data; it was her call to action. She stressed that clinician input during HTA reviews and drug plan negotiations can tip the scales. Evidence-based advocacy, delivered early and collaboratively, can shape reimbursement criteria, expedite negotiations, and ultimately get therapies to patients faster.
Her message was clear: advocacy doesn’t end after regulatory approval. It begins anew at every step of the access pathway.
Real-World Advocacy: When Clinicians and Communities Build Bridges
Theory and strategy are important, but what moved me most during the workshop were the stories, real-world examples of what happens when clinicians, patients, and advocates refuse to accept the status quo.
Dr. Colleen O’Connell: Building the Right to Breathe
Dr. Colleen O'Connell, a physiatrist from New Brunswick, shared a remarkable case study on the creation of a provincial ventilation program, something many of us take for granted but was once nonexistent in her province.
Faced with an alarming lack of access to respiratory support services - from CPAP machines to home ventilators - for individuals with neuromuscular disorders and spinal cord injuries, Dr. O’Connell and her colleagues did more than raise concerns. They organized.
They built a coalition of patients, clinicians, policymakers, and disability advocates, using the Convention on the Rights of Persons with Disabilities and Human Rights frameworks to argue that access to ventilation is not a luxury. It’s a fundamental right.
Through persistent advocacy, they presented a white paper to the provincial government, aligning patient needs with legal and ethical obligations. The result? New Brunswick’s first ventilator program, which not only provides essential equipment but also recognizes respiratory support as a human right, not a privilege.
Dr. O’Connell also shared another equally compelling case, the story of how a focused advocacy effort helped secure presumptive service connection for ALS among Canadian military veterans.
For years, scattered data and early cluster reports, such as the Shearwater naval base ALS cluster, suggested elevated ALS risks among veterans. However, without formal recognition, veterans had to fight individually to prove their conditions were linked to service.
Through a combination of clinician-driven advocacy, data gathering, and policy engagement, led by the ALS Society of Canada , advocates succeeded in securing a policy shift. In 2010, the Government of Canada introduced a Presumption of Service Connection policy for veterans with ALS. Veterans Affairs Canada / Anciens Combattants Canada recognized that veterans diagnosed with ALS could presume it was related to their service without having to prove it.
Veterans diagnosed with ALS are now automatically eligible for benefits and compensation, with no separate causality burden required. This also includes members of the Royal Canadian Mounted Police | Gendarmerie royale du Canada . This is a rare but significant example of how targeted advocacy, armed with evidence and persistence, can reshape national policy for high-need populations.
Dr. Alex MacKenzie: The Long Road to Genetic Therapies
Next, Dr. Alex MacKenzie, a senior clinician-scientist and pioneer in neuromuscular research from CHEO , took us through the decades-long journey to develop treatments for spinal muscular atrophy (SMA), a rare neuromuscular condition once considered untreatable.
Dr. MacKenzie spoke candidly about the evolution of SMA therapies, from the bleak landscape of the 1990s to today’s breakthroughs like nusinersen (Spinraza) and onasemnogene abeparvovec (Zolgensma). However, what stood out wasn’t just the science; it was the collaboration. The collaboration of patient organizations, clinicians, and scientific researchers to advocate under a single banner for expanded, standardized newborn screening that eventually led to provinces expanding their newborn screening panels to include SMA and other critical rare conditions
He emphasized that science alone didn’t get us here. It was the partnership between researchers, clinicians, patients, and advocates that kept momentum alive through failures, funding droughts, and regulatory hurdles.
“The engine of discovery is powerful,” he said, “but it needs the fuel of advocacy to keep running.”
Dr. Michelle Mezei: Closing the Diagnostic Divide
Finally, Dr. Michelle Mezei from the Division of Neurology at The University of British Columbia , a neurologist deeply engaged with underserved communities, spotlighted one of the most stubborn barriers in rare disease care: diagnostic inequity.
Dr. Mezei talked about the challenges faced by patients with neuromuscular disorders who require high-cost mobility aids, ventilators, or home modifications to maintain their independence and quality of life. While the New Brunswick ventilator program addressed critical respiratory needs, access to assistive technologies like powered wheelchairs and adaptive equipment remains inequitable and, too often, means-tested, requiring patients to prove need rather than guaranteeing access as a right.
She emphasized that without robust, universally accessible funding models, patients are left navigating a fragmented system, often stuck in diagnostic and funding deserts. Her call to action was clear: Assistive technologies are not conveniences; they are essential healthcare infrastructure.
Dr. Mezei, however, didn’t just describe the problem; she showed how clinician advocates are stepping up:
This echoed a theme running through the day:
Advocacy is not about grand gestures. It’s about small, sustained actions that remove barriers, one community, one policy, one patient at a time.
Advocacy IS the Work Now
If there’s one thing this workshop affirmed for me, it’s that advocacy isn’t something that happens after the fact. It’s not an extracurricular or an act of individual willpower, it’s becoming part of the work itself.
For patients living with neuromuscular disorders, and for the clinicians who walk alongside them, change will not be handed down. It has to be built: through partnerships, through data, through persistent, heart-centred advocacy. Clinicians, researchers, patient organizations, and policy thinkers MUST move together if we want to make systems not just work harder, but work differently.
A Seat at the Table was more than a workshop title. It was a quiet call to action. If healthcare is going to move forward, and if policies are going to reflect real patient needs, it will be because communities like this one showed up, stayed engaged, and kept pushing.
I’m grateful to have had the chance to be part of that conversation, and more determined than ever to help carry it forward.
Would love to hear how others are thinking about advocacy beyond the bedside. Let’s keep the conversation going.
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