Vertex and provinces reach agreement on Kalydeco

It's been a long time coming, but some Canadians with cystic fibrosis should soon be able to begin treatment with an expensive drug that is considered a real game changer for their disease.

Game-changing drug costs $306,600 a year

It's been a long time coming, but some Canadians with cystic fibrosis should soon be able to begin treatment with an expensive drug that is considered a real game changer for their disease.

Vertex Pharmaceuticals Inc., which makes the drug Kalydeco, announced late Monday that it had reached an agreement with provincial and territorial governments to enable public drug plans to cover the cost of the drug.

A group called the pan-Canadian Pricing Alliance — representing the three territories and all provinces except Quebec — has been involved in negotiations with Vertex for more than a year, with price believed to be the key sticking point.

The company will not reveal the negotiated price, but a previous submission to the Common Drug Review, which assesses new drugs for provincial and territorial drug plans, listed the price at $306,600 per year.

Chris MacLeod, a Toronto lawyer who has been on Kalydeco since October 2012, was delighted by the news.

"It's a real day of celebration," said MacLeod, 44, who first received the drug under a compassionate release program when his lung function plummeted to about 30 per cent.

A year-and-a-half later, his lung function has rised to 56 per cent — he's aiming for 65 — and he's able to climb four storeys worth of stairs to his office, where he's putting in 60 to 70 hours of work a week.

"This truly is a remarkable treatment," he said.

Drug a 'breakthrough' for some patients

The drug only works for a small subset of CF patients — those whose disease is caused by a mutation on the CFTR gene called G551D. There are several other mutations for which the drug is useful and is licensed, but people with those mutations make up a tiny fraction of Canadian CF cases.

In total only about 2.9 per cent of the just under 4,000 Canadians with CF have one of these mutations, said Dr. Elizabeth Tullis, director of North America's largest CF clinic, at St. Michael's Hospital in Toronto.

The drug is only approved for patients six and older who have those mutations.

But for these patients, Kalydeco is a breakthrough, Tullis suggested, explaining it is the first drug that corrects the faulty mechanism that causes the disease process rather than trying to mitigate the downstream effects of unchecked disease.

"Doctors and CF researchers were really just kind of dumbfounded," she said of the drug. "It was beyond our expectations."

Tullis said the lung function of patients who take the drug improves, even adult CF patients who already have extensive lung damage. The drug also reduces the number of times CF patients get sick — a common problem. As well it helps them gain weight, something they struggle to do.

Tullis's clinic was one of a number that were involved in the clinical trials that led to the licensing of the drug. While she was not paid for participating in the trials — compensation went to her hospital — she has done some consulting for the Boston-based Vertex, and was paid for that work.

The company's statement said that each province and territory still must decide to reimburse the drug through its own drug plan before the oral medication will become available. Ontario Premier Kathleen Wynne has said her province will pay for Kalydeco.

Quebec not part of agreement

The agreement does not cover Quebec, which does not take part in the pan-Canadian Pricing Alliance process. Nikki Levy, senior director of corporate communications for Vertex, said in an email that CF patients with private insurance in Quebec can get Kalydeco covered through their insurance plans. For people with public insurance, coverage may be possible through an "exceptional patient" measure of the provincial drug program.

That divide between private insurance and public insurance has been the state of play in Canada since the drug was first approved here in late 2012.

Tullis said watching some patients flourish while others have had to wait for the drug has been difficult; her clinic literally has siblings on its roster, one of whom was able to get the drug because of private insurance while the other was not.

"For the last year, I've really felt the most uncomfortable that I've ever felt looking after people with CF," she said. "I really felt for the first time ever that I was in very much a two-tier health-care system."

"Most of the time in medicine in Canada, usually it goes by medical need. And you can usually get drugs through one mechanism or another for patients who need it. And this was one time where that wasn't the case. That was really tough."

Cystic fibrosis is caused by a defective or missing CFTR protein, the result of a mutation in the CFTR gene. Only children who inherit two faulty CFTR genes — one from each parent — have the disease. There are more than 1,900 known mutations in the CFTR gene.

People with CF have a poor flow of salt and water into and out of the cells of a number of organs, including the lungs. This results in a buildup of abnormally thick, sticky mucus that can cause chronic lung infections and progressive lung damage.