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Kelsa Hague, 21,  spends up to three hours a day treating her disease.The Globe and Mail

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.

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."

Cystic Fibrosis Canada says that without provincial coverage, eligible patients can only access Kalydeco through private health care or involvement in clinical trials. Patients such as Kelsa, whose employer does not provide extended health care, cannot begin to pay the price.

"There's no way we can afford it," Ms. Craik says. "We'd have to sell everything we own, and that would last for about a year [of Kalydeco]. And then we would be back to square one."

Vertex first began researching CF in 1998, when the Cystic Fibrosis Foundation of America approached the company in search of a drug that would treat the underlying cause instead of the symptoms. To date, the CF Foundation has given more than $100-million to Vertex for the development of treatments like Kalydeco.

Yet Vertex spokesman Zach Barber insists the majority of funds used to develop Kalydeco has come from Vertex itself. "The price of Kalydeco reflects well over a decade of research and development work," he says. "And Kalydeco treats such a small number of patients right now. Ideally our goal with CF is to treat many more patients."

Kalydeco is currently used by more than 2,000 patients in 15 different countries, including the U.S. Vertex's net revenues in 2013 amounted to $1.21-billion, with more than 30 per cent coming from increasing Kalydeco sales – which are projected to continue growing in 2014. Mr. Barber says the company isn't driven primarily by the profit motive.

"For us this is more than just developing a medicine," he says. "We've had a number of people with CF that have come in over the years, that our employees have watched grow up. People are absolutely, personally invested in this."

Still, some critics question why the cost of Kalydeco remains so high, given the CF Foundation's investments. Bob Nakagawa, former deputy minister of pharmaceutical services in the B.C. Ministry of Health, argues that the pharmaceutical companies are ultimately setting the price of drugs for rare diseases like CF. The government can be made out to be the villains, he says, when really they are stewards of taxpayer dollars.

"The price is entirely, 100 per cent determined by the manufacturer," he says. "It's only unaffordable because they made it that way."

Patients such as Kelsa also ask how a tiny blue pill can cost hundreds of thousands of dollars a year.

"What was the point of even making a cure if nobody can have it?" she asks. "I understand if it's still a fee that you've got to pay because it takes money to make the drug. But $300,000 a year? It's like they're trying to make a profit off our health."

It's 8:30 on a Saturday morning and Shan Rée is well into her swimming training with the West Vancouver Otters. The 10-year-old glides effortlessly through the water with her teammates – making it hard to believe that, just three years ago, she was admitted to hospital numerous times. Both Shan and her older brother Carter were born with cystic fibrosis.

"It's your worst nightmare when something's wrong with your kids," says their mother, Wendy Rée. "We dreamed of normal."

Carter was 14 months old when he was diagnosed with cystic fibrosis. By then Wendy was pregnant with Shan, the couple's third child. She and her husband were told they were both carriers of the CF gene, and there was a 25-per-cent chance the baby would also have CF. Shan was diagnosed shortly after her birth.

"You realize how vulnerable they are," says Robert Rée, the children's grandfather. "That their lungs could be damaged to the point that they might survive it, but they might eventually need a lung transplant."

Having two children with CF meant constant hospital admissions, lung infections and endless bouts of intravenous antibiotics. It meant hours spent on physical therapy and medical treatments each day. Wendy had to quit her job to care for the children, and the family moved several times to be closer to a hospital.

Then came Kalydeco. Within weeks, Shan's constant congestion cleared up. She had more energy and wanted to take swimming lessons. Neither child has been in hospital for CF since they began taking Kalydeco.

"As a grandparent, it's like having witnessed a miracle," Robert Rée says. "[Before] it was terrible to think that I might actually outlive them. Now with Kalydeco, I like to think that they will live forever. The future looks a lot brighter."

Private work insurance covers the more than $600,000 annual cost of both children's medication. Without it, they could never afford Kalydeco.

"Some people have said that the cost is unconscionable to charge people, given the difference it could make to someone's life," Mr. Rée says. "We're very fortunate that it's currently being paid for."

Alberta has been leading the Pan-Canadian Pricing Alliance (PCPA) in negotiations with Vertex for the past year and a half. Composed of provincial and territorial health ministers, the PCPA combines the drug purchasing power of individual provinces with the hopes of saving Canada millions of dollars on bulk drug purchases.

The process has been slow. In March, Alberta's Minister of Health Fred Horne expressed frustration, saying "Vertex so far has not been willing to come to the table with a reasonable price."

By June, news broke across Canada's CF community that, at last, Vertex and the PCPA had settled on a tentative price.

Mr. Barber says that while discussions between Vertex and provincial officials are encouraging, their work will not be done until all the provinces and territories agree to provide Kalydeco for patients who need it. Ontario has now done just that.

The Alberta Ministry of Health confirms that a tentative price has been reached with Vertex, and Mr. Horne is encouraging his provincial counterparts to accept the deal. As of this week, however, the B.C. Ministry of Health said it was going to take some time to review agreements before making any decisions.

This could create a situation in which CF patients in one province have Kalydeco covered, while those in a neighbouring jurisdiction do not. Mr. Rée, young Shan's grandfather, believes things could get complicated.

"I think we need to get our act together as a country when it comes to health care," he says. "We're broken in that respect."

But his 10-year-old granddaughter, who dreams of swimming in the Olympics one day, sees the dilemma in a simpler way. "What if you had cystic fibrosis, and then you suddenly had a miracle pill, and no one else did?" she says. "Would you give half of it away, or would you be greedy?" Fidgeting on her chair, the little girl thinks for a moment. "I would give half of it away. But I'd probably be a little bit greedy too."

What cystic fibrosis is

Approximately 4,000 Canadians live with cystic fibrosis, or CF. The disease primarily affects the lungs and digestive system, causing thick mucus to clog the linings of vital organs. CF patients can suffer from a variety of symptoms that include persistent coughing, chest infections and bowel disturbance. There's no known cure for the disease, and half of CF patients die before they turn 34. A rare mutation of the CFTR gene, called G551D, is found in 4 per cent of patients.

How Kalydeco works

Kalydeco (Ivacaftor) is a drug that treats a rare form of cystic fibrosis triggered by a specific mutation (G551D) of the gene that causes CF. The drug is the first to treat the causes of CF as opposed to the symptoms. It allows electrolytes to enter the cell membranes, reducing the thick, sticky mucus that clogs the lungs and other organs. Kalydeco was first approved in Canada in 2012, and is used by more than 2,000 patients in 15 countries. The drug costs $300,000 a year and is not covered by the Canadian public health-care system.

This work was supported by the Canadian Institutes of Health Research, and led by UBC's Graduate School of Journalism and the Faculty of Pharmaceutical Science:,

Editor's note: A previous version of this article said Kalydeco reduces the thick, sticky mucus that blocks the flow of salt across cell membranes in lungs and other organs. In fact, Kalydeco allows electrolytes to enter the cell membranes, reducing the thick, sticky mucus that clogs the lungs and other organs.