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The Reality of Rare Diseases in Canada

If you ask a Canadian what defines them as a citizen of Canada, there’s a high chance the answer is access to free universal healthcare. The response is legitimate, but “free” does not really explain the state of the current system. There are inconsistencies in the system, and that although Canadians are unified as one country, drug plans and healthcare policies vary from province to province, and even among identifiable groups. In fact, the government has no obligation to provide you with a drug you need outside of hospital or clinical grounds. Sadly, there’s a group of people in Canada who if they identify as ‘x’, they will receive little to none of ‘y’. These are the people who identify themselves as patients of rare diseases.

Despite a public healthcare system that is the envy of most countries, Canada continues to lag behind other developed countries who have already established and recognized the need for more research and support for rare diseases. Countries such as the USA, Australia and all European Union countries have orphan drug legislation in place for patients to access. Thankfully, Canadians diagnosed with rare diseases have a large advocacy group on their side called CORD, also known as the Canadian Organization for Rare Diseases. CORD works to serve as a voice on behalf of smaller focused patient groups and it addresses concerns of funding and other existential problems, such as why some provinces are willing to spend more money on expensive orphan drugs than others. And although gene mutations cause not only well-recognized rare diseases, such as cystic fibrosis and muscular dystrophy, they contribute to thousands of other lesser known diseases, such as aHUS (atypical hemolytic-uremic syndrome). While individually rare, the disorders and diseases are collectively common, affecting more than one million Canadian children and adults.

The policing of drugs, especially orphan drugs is a long and enduring process. Not to mention, those with rare diseases face increased anxiety as few medical experts exist and research is scattered. Drugs go through many years of research studies, clinical trials and much more, to gather the evidence required to support the efficacy. Two pan-Canadian governing bodies of drugs, the Common Drug Review (CDR) and the pan-Canadian Oncology Drug Review (pCODR), are responsible for investigating the mechanics of the drug and in the end, provide a positive, or negative recommendation for public drug plans, and decide what is going to be covered and what is not. The fate of public-funded access to drugs lies heavily in the hands of these two groups.

It’s quite frustrating when advocacy groups for rare diseases voice their concerns to government to publicly fund their orphan drug but get little to no response. When government denies Canadians publicly-funded access to the drug, it raises concerns of how much the life of a human really costs. For example, Soliris is an innovative orphan drug used to treat the rare disease known as aHUS (atypical hemolytic-uremic syndrome). The disease results in a condition that causes the immune system to attack the body, often resulting in extreme fatigue, nausea, organ damage and kidney failure. It’s estimated to affect only five in every one million people. According to CORD, Soliris is one of the most expensive drugs in the world, costing $450,000 per patient per year.

How far would the government go to publicly fund a life-saving drug for a tax-paying patient who desperately needs it? And will it be available for equal access across Canada? As of 2013, Soliris was approved for use by Health Canada, but the Common Drug Review (CDR) gave a negative recommendation for publicly funded access to the drug, even though evidence of the efficacy of the drug exists. Only time will tell when government publicly funds access to life-saving orphan drugs that treat Canadians with very real rare diseases, but when will they realize that the time is now.

The atypical hemolytic-uremic syndrome organization of Canada (aHUS Canada) is a not-for-profit organization with the intent to support patients and families living with atypical hemolytic uremic syndrome. To learn more about their platform and how you can help them convince the Canadian government to public fund their life-saving orphan drug, visit under the “Advocacy and Support” tab.

To learn more about CORD’s platform, visit

*Special thanks to the editorial content by MediaPlanet in their October 2013 issue in motivating me to write this article.


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