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Petition asks for support for national rare disease medication strategy

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Canada is one of the few developed countries that doesn’t have a rare disease medication strategy.

That’s according to Kim Steele, Director of Government and Community Relations for Cystic Fibrosis Canada. She says this is why they’ve put a petition out, asking Canadians to support their pursuit of a new – and better – rare disease medication strategy, including cystic fibrosis. 

“What that means is when a rare disease drug comes to Canada, it’s treated the same as any other drug when it comes to burden of evidence, so the type of evidence and the amount of evidence that has to be provided to our government for them to cover the cost of those drugs,” Steele said.

She adds the problem with Canada’s current strategy is there can’t be big trials for new drugs for rare diseases, which means the government can’t then justify covering the costs like they can easily justify it for new drugs for common diseases that have bigger trial output. 

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Steele says there will never be enough evidence, especially with the case of cystic fibrosis, as it only affects approximately 4, 300 Canadians. 

“We need to think about new models, new strategies, to assess the evidence that we can provide. That might mean aftermarket studies or if I go on a drug, the government and the company are going to follow me for a while to make sure that drug is working for me,” she said. 

“There are all kinds of ways to get different types of evidence. It’s just that our government has not done it to date.”

“With the rise of national pharmacare, we now have the opportunity to say ‘we need a fair process to asses these drugs for rare diseases so that people can actually get them’. Because right now, it’s taking between two to five or even more years for these types to come to market and get approved,” Steele added. 

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Campbell River mother Kim Wood says she’s been lucky to be part of a trial group that has given her access to life-saving medication. 

“Once I started on this study medication, my whole life changed. I started to be able to be a mom that I didn’t think I would be able to be. I had energy and I could breathe completely unrestricted for the first time ever,” Wood said.

But because it’s still in the trial stage, it’s not widely available yet. And with how difficult it is to access rare disease medication, Wood may not be able to access it again. 

“That’s how we get better, is through research and being introduced to new medication.”

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“Because it’s not laid out in a fair way, me potentially being taken off this medication and having my health decline, it’s pretty heartbreaking. And also for youngsters with CF nowadays, one of the things that this medication does is it prevents your lungs from getting worse. So for these youngsters to get on this medication, if they can access it at a younger age, they’ll have an even brighter future.” (8:50)

“It’s heartbreaking thinking about trying to tell my son that things are going to get worse, and also thinking about other kids with CF and thinking about their future just because they can’t access a medication that could make them so much better.” 

You can sign the petition through this link.

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