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Canadians demand access to ‘miracle’ cystic fibrosis drug already approved in U.S. and U.K.

Imagine having a life threatening disease, knowing a treatment exists that could greatly improve your chances of survival, but that you can't get it. Even worse, you can't get it because you live in Canada. As Dan Spector explains, that's what thousands of cystic fibrosis sufferers in this country are living through right now – Jul 6, 2020

Montreal’s Chelsea Gagnon may look like a healthy 31-year-old woman at first glance, but every single day she’s fighting just to breathe.

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“Last month, I actually started coughing up blood, which is a pretty common thing as your lungs get more scarred,” Gagnon said.

She lives with cystic fibrosis, a genetic disorder that attacks the respiratory system. She takes dozens of pills a day, and twice daily, she uses a machine to help loosen up the mucus that accumulates in her lungs. Each year, she says her condition gets worse.

“I am deteriorating,” she said.

There is no cure, but treatments are emerging. Drugs like Orkambi have improved quality of life for CF patients during trials in Ontario.

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Another medication, however, is creating even more optimism in the cystic fibrosis community. It’s called Trikafta, a combination of three drugs created by American pharma company Vertex.

READ MORE: Ontario brothers fight Cystic Fibrosis, family pleads for equal access to drug treatment

“It’s really as close to a miracle drug as you could get,” said Gagnon.

In the United States, Trikafta was green-lit by the FDA last fall. Last week, it was lauded in the United Kingdom.

“NHS has just landed a deal to deliver the lifesaving cystic fibrosis drug Trikafta here in the U.K.,” British Secretary of State for Health and Social Care Matt Hancock announced in a video posted on Twitter.

Cystic fibrosis patients there were overjoyed, some posting videos of themselves crying happy tears on social media.

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Gagnon and thousands of other Canadians have not experienced that joy. The drug is not on the market here.

“I’m scared at this point that I’ll pass away before I get it,” worries Gagnon.

Cystic Fibrosis Canada says she is not alone.

“Behind one Chelsea, you have thousands of CF families that are really frustrated, because this drug Trikafta, really it is game-changing,” said Olivier Jerome, a regional director with Cystic Fibrosis Canada.

Like other cystic fibrosis drugs, Trikafta can cost hundreds of thousands of dollars per year per patient. Health Canada does allow Canadian patients to apply for medications like Trikafta via the Special Access Program, and to date 122 Canadians have been given access. Not everyone is approved, however, and to Gagnon, that’s not enough.

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“Really we’re in the middle of this tug of war, and we’re the ones who are suffering,” she said.

READ MORE: ‘It’s unfair’: Cystic fibrosis patient calls on Quebec to cover costs of $200K ‘life-changing’ drug

Health Canada says that it has been in talks with Vertex since 2019 but the company has not applied to market the drug in Canada yet.

Advocates say new government regulations are scaring Vertex away. “You need to create that fine balance, between attracting innovative treatments and respecting your payment capacity,” Jerome said.

Health Canada told Global News there are many steps involved in the regulation and reimbursement of drugs in Canada, and recognizes delays can be frustrating for families.

“To improve access to these medicines for Canadians, we’ve made changes to the rules regulating the maximum prices of patented medicines,” explained Cole Davidson, press secretary to Health Minister Patty Hajdu.

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“They put all these hurdles in, which are harmful to the rare disease community,” said Gagnon.

Vertex told Global News it is urging Canadian regulators to reconsider their new regulations.

“New Canadian medicine pricing reforms will limit the ability of Vertex and other manufacturers to launch future medicines in Canada and we have made every effort to communicate our concerns about these pricing reforms to the Canadian Government and communicated several potential alternative solutions to the Patented Medicine Prices Review Board (PMPRB),” said a Vertex spokesperson.

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Gagnon just hopes everyone who needs Trikafta will be able to access it sooner, rather than later.

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