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Kelsa Hague, 21,  spends up to three hours a day treating her disease. (Katelyn Verstraten/The Globe and Mail)

Kelsa Hague, 21,  spends up to three hours a day treating her disease.

(Katelyn Verstraten/The Globe and Mail)

Kalydeco: A ‘miracle drug,’ with a catch Add to ...

Like many 21-year-olds, Kelsa Hague is trying to figure out her career, hang out with friends and care for her assorted pets. But unlike other people her age, she is also waiting for a drug that could save her life.

It’s called Kalydeco (trade name for ivacaftor), and it’s being heralded as a “miracle drug” by cystic fibrosis patients who have experienced its life-changing effects. But this miracle comes at a cost of more than $300,000 a year, and while Kalydeco was approved for use in Canada in 2012, it has not been covered by the health-care system.

Globe and Mail Update Jul. 07 2014, 4:02 PM EDT

Video: A $300,000-a-year drug is helping this young cystic fibrosis patient live

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That is changing, at least for some Canadians. After more than a year of negotiations with a group representing the provinces and territories, Vertex Pharmaceuticals, which makes Kalydeco, announced this week that it had reached agreement to enable public plans to cover the cost of the drug. And on Friday, Cystic Fibrosis Canada announced that CF patients in Ontario now have access to Kalydeco through the provincial drug program, effective immediately.

But each province and territory must decide whether it will cover the cost of the drug at the negotiated price – raising the possibility that coverage for Canadian patients may depend on where they live.

Kelsa’s mother, Kathy Craik, says she is not surprised at this caveat. “It figures,” Ms. Craik says, adding that even if the price has been lowered, unless the province of British Columbia decides to cover the drug, it will remain out of reach for her daughter. “I guess we’ll just have to wait and see.”

Kelsa is one of about 4,000 Canadians living with cystic fibrosis. Half of CF patients die before the age of 34 – and there is no cure. However, for the 4 per cent of CF patients with the G551D mutation to the CF gene – a rare mutation that Kelsa has – Kalydeco can markedly improve their lives. It has been approved for eight additional mutations in the United States, although Health Canada has yet to approve this addition.

Kalydeco is the first drug that addresses the cause of the disease instead of just the symptoms. Various mutations of the CF gene block the “gates” that allow the flow of salt across the cell membranes. Without these electrolytes, mucus becomes thick and sticky, clogging such organs as the lungs. Kalydeco reduces the mucus, helping the gates stay open.

The question, even with the new agreement with Vertex, is whether Canada can pay for that relief. Rare diseases such as cystic fibrosis have a relatively small patient population, and the drugs to treat them can cost millions of dollars a year. Vertex did not disclose details of the newly negotiated Kalydeco price tag, but providing the drug at the original price would cost the health-care system more than $36-million a year – roughly the cost of 36,000 MRIs.

Why does Kalydeco cost so much? And what are Canadians willing to pay to improve a life?

Living with a fatal genetic disease never used to slow Kelsa down. She grew up in Nanaimo, B.C., chasing after her older brother. She played soccer and hockey, rode horses and camped every summer. But unlike other children, her life has been punctuated by unexpected and lengthy hospital stays. While as a child her disease was manageable, things have begun to change.

“I can tell that I’ve slowly deteriorated,” Kelsa admits. “I used to play hockey – if I was on the ice today I’d probably die in five minutes. I can tell that I am getting worse. It’s not going to get better on its own.”

Kelsa takes nine different medications and spends up to three hours a day on treatment to reduce the deadly mucus. What Kalydeco would offer Kelsa now, she says, is the promise of a future.

“I don’t really plan on living past 50,” she says, her voice breaking. “I’m not even going to be old enough to retire, so screw setting up a retirement fund. It would be nice to be able to plan for your future instead of just living with what you’ve got. And hoping.”

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