Canadian rare disease patients and direct caregivers experience major hurdles for timely diagnosis, care and access to medicines

Toronto, ON, February 28, 2023 — More than 3 million Canadians living with a rare disease have the added heartache of knowing that they, or their child, were diagnosed too late or didn’t get the specialist care they needed to avoid irreparable harm or disability. These were among the findings from a recent Ipsos poll carried out on behalf of the Canadian Organization for Rare Disorders (CORD), conducted among 528 Canadian caregivers and patients living with rare disease. It found that significant delays and issues arise for patients and their families in all key areas – including long delays in diagnosis, disease and knowledge information gaps, lack of access to drugs, and lack of coordinated and accessible care.

 Canada is the only major developed nation without a comprehensive rare diseases strategy to help patients and families. Canadians have been waiting for years for one to be finalized and implemented. The federal government in 2019 committed new spending of $500 million a year for a national plan for drugs for rare disorders, starting in the current government year. With just one month left, the plan has not been finalized and no money made available to help improve healthcare services, access to drug, and patient group support.

“We know what has to be done, health leaders are ready to move, and we even have the funding allocated but while governments procrastinate, patients and families continue to suffer,” said Durhane Wong-Rieger, President and CEO of CORD. “These new survey results confirm that rare disease patients and families are being left behind, even as governments and health systems prepare to recover from the devastation of COVID.”

The new Ipsos survey featured input from over 500 Canadian patients and their direct caregivers, highlighting their challenges and their perspectives on what emerging government strategies should focus on. The overwhelming majority support the creation of specialized centres networked to local healthcare professionals to reach all patients wherever they live in Canada.

 

Challenges with diagnosis

According to the survey, patients and caregivers felt that their healthcare professionals lacked knowledge of rare diseases, which delayed diagnosis and specialist care:

• Four out of five (80%) do not think family physicians are aware and informed about rare diseases and 62% say the same about pediatricians
• Almost half (47%) do not believe specialists have up-to-date knowledge about rare diseases
• Respondents said they waited an average of 3.7 years to get an accurate diagnosis
• In the process, they saw an average of 5.9 different healthcare professionals and received an average of 3.2 wrong diagnoses

Information and knowledge gaps

Canadians impacted by rare diseases can often feel lost and in need of connection with others with rare diseases, but face obstacles in doing so:

• Almost two-thirds (63 %) say they do not have access to a care coordinator
• There is a large lack of counselling support for patients with rare diseases, with almost three out of four (74%) saying they did not have counselling support at the time of diagnosis and 70% did not receive appropriate emotional and psychosocial support
• Less than six out of 10 (58%) feel they have access to all of the appropriate information they need and only 43% say they were given a contact person from whom they could get additional information
• Less than one in 10 agree that educational, disability and employment services are aware and informed about rare diseases

Access to treatments

The large number of Canadians with rare diseases have issues related to their access to appropriate drug therapy for their condition:

• Only six out of 10 (60%) say they have access to appropriate drugs for their condition and 63% say they must pay out-of-pocket expenses
• More than a third (36%) say they are unable to access prescribed medicines because their share of the costs is too high
• Two-thirds (68%) of Canadians with rare diseases are aware of Health Canada-approved medicines for their condition but 43% cannot easily access prescribed approved medicines
• A third (32%) are aware of off-label medicines that could help their condition but almost two-thirds (63%) cannot access them

Need for rare disease centres

Survey respondents were offered two alternatives that have been proposed as key attributes of a national rare disease strategy using the committed federal funding. One is the development of a new national list of drugs for rare diseases and the other is the creation of rare disease centres to specialize in rare disease treatment.

• Four out of five (80%) respondents prefer the development of rare disease centres over a new national list of drugs (20%)
• The most cited reason supporting rare disease centres was that they would offer “comprehensive support, a research centre with access to clinical trials, and more efficient and accessible for patients”
• Among the minority (20%) who supported a new national list of rare disease drugs was that it would provide better drug coverage and lower costs and that drugs would be more accessible to all, regardless of location
• A large majority (84%) favour federal oversight of a rare disease strategy to set standards, facilitate inter-provincial care and improve access to treatment and care because such a policy would provide “more equal access across Canada” and establish “sets of standards and consistent policies.” If the provinces maintain oversight, respondents said the federal government should set pre-conditions tied to federal funding
• “These survey results provide important guidance for Canadian policy makers in shaping Canada’s rare diseases strategy which is so urgently needed,” said Stephen Parrott, Board Chair of Canadian VHL Alliance which advocates for Canadians with VHL, a rare inherited disease that causes cysts and tumours which can be cancerous. “My two daughters have VHL and both lost their adrenal glands. They are now taking steroids for the rest of their lives. They are the lucky ones with VHL, many have it much worse. Canadians with rare diseases and their families have waited far too long and with severe consequences that in many cases could be prevented. We need this strategy implemented without further delay.”

 

About Rare Disease Day

Rare Disease Day is the globally-coordinated movement on rare diseases, working towards equity in social opportunity, healthcare and access to diagnosis and therapies for people living with a rare disease. Since its creation in 2008, Rare Disease Day has played a critical part in building an international rare disease community that is multi-disease, global and diverse – but united in purpose. Rare Disease Day is observed every year on Feb. 28 or during leap years on Feb. 29 – the rarest day on the calendar. Rare Disease Day was set up and is coordinated by EURORDIS and more than 65 national alliance patient organization partners, including CORD. For details, visit www.rarediseaseday.org/

 

About the Study

These are some of the findings of an Ipsos poll conducted between Oct 26 – Nov 28, 2022 and interviews between Jan 25 - Feb 10, 2023, on behalf of the Canadian Organization for Rare Disorders (CORD). For this study, a sample of 528 caregivers and patients living with rare disease was surveyed and 10 were interviewed. The survey was administered via an open link to CORD’s network, utilizing a convenience sampling approach. The sample is not representative of all patients with rare diseases. However, our approach allowed us to access a large, valid sample size of patients with rare diseases/caregivers that would have otherwise been very challenging to obtain. By the nature of this sample, the data was not weighted. A credibility interval/margin of error cannot be calculated when an open link is used and when the population universe is unknown. Convenience samples are at risk for both sampling bias and selection bias. All sample surveys and polls may be subject to other sources of error, including, but not limited to coverage error, and measurement error.

For more information on this news release, please contact:

Grace Tong
Vice President, Public Affairs, Ipsos Canada
+1 647 328-1033
[email protected]

Hyejin Park
Research Analyst, Public Affairs, Ipsos Canada
+1 437 246-2681
[email protected]

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