Rare Disease
Canadian Rare Disease Patients Are Getting Impatient — Here’s Why
The Canadian Organization for Rare Disorders is urging the Canadian government to accelerate its National Strategy for Drugs for Rare Diseases implementation.
Durhane Wong-Rieger, President & CEO, Canadian Organization for Rare Disorders
In 2005, Ontario ombudsman Andre Marin issued a clarion call on behalf of a rare disease community that was struggling to make its voice heard. The issue was the Ontario government’s delay in implementing newborn screening. Ombudsman Marin legitimized our patient “right to be impatient.” He unflinchingly denounced the “consequence of politicians and bureaucrats failing to sense the urgency and […] to remedy the situation peremptorily.”
Fast forward to 2023, days after Rare Disease Day. The federal government announced $1.5 billion to make good on a 2019 promise to implement a national rare disease drug strategy to
“increase access to promising and effective drugs for rare diseases.”
The Canadian Organization for Rare Disorders (CORD) and the entire rare disease community cheered. We proposed an initial list of drugs that save or extend patient lives. They reduce the risk of disability, catastrophic harm, or life-threatening surgery. Already approved and recommended, these drugs are stalled by bureaucratic inertia: in price negotiations with the pan-Canadian Pharmaceutical Alliance, in budget impact assessments with provincial Health Ministries, or with drug plan managers who claim no more monies to allocate. Meanwhile, the $1.4 billion designated for drug funding appears to be hopelessly stalled in feder-
al-provincial negotiations.
Urgent action is needed
Patients whose lives and well-being are at risk have the right to be impatient. The governments can and must act to make urgently needed drugs immediately available. The CORD has proposed an implementation plan in which rare disease therapies that are approved and recommended can be started immediately with a defined cohort of patients along with a plan for ongoing monitoring, data collection, and assessment of benefit and potential harm.
Ombudsman Marin’s opinion is worth repeating: “Government responsibilities to citizens are owed at large, not in isolation
Durhane Wong-Rieger President & CEO, Canadian Organization for Rare Disorders
by divisions of government. The right thing to do […] is to discharge those responsibilities faithfully and with commitment, while trying to achieve reorganization. It is never appropriate to abdicate those responsibilities while waiting for reorganization to occur.” This was true in 2005, and it’s true in 2024.
To learn more, visit raredisorders.ca
Advancements in Research Help Patients With SMA Push New Boundaries
People with SMA once faced a bleak future. Today they’re not only maintaining ability but achieving more than ever before.
Anne Papmehl
Few people, including his doctor, could’ve predicted what Ty Durant would be capable of today. At age four, Ty was diagnosed with spinal muscular atrophy (SMA), a rare neuromuscular disease. “It was devastating,” his mother, Angie, recalls of his diagnosis. “I was told that over time he’d lose his ability to walk, stand, or live independently, but to take comfort in knowing he should live ‘a normal life expectancy.’”
But today, the sixteen-year-old from Olds, Alberta is not just surviving — he’s thriving. Defying expectations, Ty does rigorous daily weightlifting workouts at the gym and is passionate about physical fitness. Ty is also a strong swimmer and can hold his own in any singles badminton match.
SMA is a disease that affects infants, children, and adults, and leads to progressive motor function loss and muscle atrophy. Only five years ago it caused Ty’s life to look very different, as he faced a diminishing quality of life and bleak future.
New therapeutic approaches lead to a new outlook on life Ty has Type 3 SMA and he remembers how the progression of the disease accelerated at around the age of 11. “The muscles in my lower extremities were getting quite weak and I was not able to walk as fast and as far as I could before,” he says. “I was at my
lowest point and feeling very unmotivated.” He eventually needed to use a forearm crutch when walking and a manual wheelchair for moving longer distances.
It was painful for Angie to watch, but she was determined to give her son the best life possible. She learned through Ty’s neurologist about advancements in research that were enabling SMA patients to achieve more developmental milestones, and to preserve and potentially regain function.
Setting and achieving ambitious goals
Now I feel a lot better physically and mentally, and am extremely motivated.
For the Durant family, these new therapeutic approaches would be life-changing, and have made it possible for Ty to build up his physical strength. “I’m close to being able to do 10 pounds on the leg extension machine, when a couple of months ago, I could barely lift it at all,” he says.
— Ty Durant
Ty is like most teenagers, enjoying a rich social life with his peers and helping out with the household chores. “He cleans his own bedroom and bathroom, vacuums, unloads the dishwasher, dusts, and is a great cook and baker,” says Angie. Adds Ty: “My friends think I must have it pretty easy around the house, and they’re always shocked when they learn about how much I do. It really puts into perspective that I, and others living with SMA, can exceed the limitations that people may place on us.”
Ty Durant & his mother, Angie. Sixteen-yearold Ty lives with Type 3 SMA and is determined to defy expectations.
As Ty’s strength and stamina have improved, so has his outlook on life.
“It’s been a big break for me because my mindset wasn’t in a good place before. Now I feel a lot better physically and mentally, and am extremely motivated. I devote most of my time to school and the gym, and push myself more and more,” says Ty.
Angie too has noticed significant changes in her son, especially with his mood and energy. “He’s also becoming such a wonderful advocate for himself and speaking his truth about his experiences,” she says.
Resilience epitomizes the SMA community’s spirit, and Ty is no exception as he continues to set goals for himself and chart out his future. He loves mathematics and plans to pursue an engineering career. He also wants to learn to drive and one day have a partner and children. “I’m a big family guy,” he says.
Although Ty still relies on support for mobility, his ambition to push past barriers continues: He is determined to walk across the stage independently by the time of his high-school graduation, and Angie has no doubt her son will succeed. “I believe he’s going to make that happen,” she says.
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community at
How Patient Support Services Bolster Up Canada’s Rare Disease Strategy
Bayshore Specialty Rx is a strategic partner in the implementation of Canada’s new National Strategy for Drugs for Rare Disease.
For the 1 in 12 Canadians living with a rare disease and their families, access to groundbreaking therapeutic innovations comes with a high cost. In addition to the time and financial burden of travelling to specialists, drugs used in the treatment of rare diseases can range from $100,000 to more than $2 million per year1. As most rare diseases result from genetic mutations, the drugs used to treat these individuals are often needed for life. In March 2023, the Canadian government made a pivotal step in announcing the first National Strategy for Drugs for Rare Diseases, which is designed to improve the equitable access and affordability of drugs used to treat rare diseases2
A strategic ally
Successful implementation of the strategy necessitates engagement among numerous stakeholders. Bayshore Specialty Rx acts as a strategic ally for the implementation of the strategy by providing comprehensive patient services through manufacturer-supported patient support programs (PSPs) and special access programs (SAPs).
to alleviate several challenges faced by rare disease patients and their caregivers through the navigation of public and private benefits and the coordination of clinical requirements related to drug administration. Nationwide infrastructures for the distribution of drugs and patient care are important considerations for equitable access to rare disease therapies. Bayshore Specialty Pharmacy and Nursing Services are equipped to provide national product-specific distribution of these drugs and flexible nursing services provided in alignment to individualized treatment schedules to support patients in their unique needs. The coordinated approach ensures timely access and contributes to better drug adherence, promoting the best patient outcomes and health care system sustainability.
The delivery of health care remotely and the collection of patient-caregiver feedback through digital innovations such as Bayshore Digital Gateway™ and MyBayshoreCare™ will be critical in obtaining key health and qualityof-life outcomes.
Bayshore PSPs, in collaboration with industry partners, are designed
RESOURCES
Embracing digital innovation
Digital health solutions need to be adopted, as a strategic effort must be made to monitor successful patient outcomes to ensure these drugs remain a viable option in the Canadian market. The delivery of health care remotely and the collection of patient-caregiver feedback through digital innovations such as Bayshore Digital Gateway™ and
MyBayshoreCare™ will be critical in obtaining key health and quality-oflife outcomes. Bayshore has made significant advancements to integrate data across multiple platforms to provide a comprehensive overview of the patient journey. Crucial insights gathered from the PSP real-world setting can drive evidence-based outcomes, payer and regulatory conversations, and submissions to expand access to life-changing drugs.
Facilitating
access to innovative therapies
SAPs also facilitate access to innovative therapies for rare disease patients, even when a drug is not yet approved for use in Canada. Although SAPs are approved on a case-by-case basis, manufacturers must be able to provide access to the drug and adhere to strict reporting regulations. Pharmaceutical partners have already begun to leverage the existing Bayshore support services, digital platforms, and national infrastructure to support rare disease SAP patients. Concurrently, the high-quality data capture by Bayshore allows manufacturers to meet regulations for SAPs. Information garnered from the SAP can be used to drive equitable access should the treatment receive approval for use in Canada.
With key stakeholders, Bayshore Specialty Rx will be an instrumental partner in providing equitable access and improving patient outcomes for rare disease patients.
1 A Prescription for Canada: Achieving Pharmacare for All. Health Canada. June (2019). https://www.canada.ca/en/health-canada/corporate/about-health-canada/ public-engagement/external-advisory-bodies/implementation-national-pharmacare/final-report.html#2
2 Government of Canada improves access to affordable and effective drugs for rare disease. Health Canada. March (2023). https://www.canada.ca/en/health-canada/ news/2023/03/government-of-canada-improves-access-to-affordable-and-effective-drugs-for-rare-diseases.html
To learn more, visit bayshore.ca/bsrx
article was
Nicole Bellefontaine
Nicole Bellefontaine, PhD Senior Clinical Research Program Manager, Bayshore Specialty Rx
This
sponsored by Bayshore Specialty Rx