Thursday, November 20, 2014

Deal reached on costly CF drug

TORONTO—It’s been a long time coming but some Canadians with cystic fibrosis soon should 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 yesterday 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 percent.
A year-and-a-half later, his lung function has rised to 56 percent—he’s aiming for 65.
He’s also able to climb four storeys worth of stairs to his office, where he’s putting in 60-70 hours of work a week.
“This truly is a remarkable treatment,” MacLeod said.
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 percent 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 only is 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.

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