A man with cystic fibrosis says a drug that could potentially help him live longer is unaffordable for those who need it.

Kalydeco was approved by Health Canada in 2012. But the treatment comes at a steep cost — $400 a pill. Taken twice a day, it costs patients $306,000 a year.

Cystic Fibrosis Canada calls the drug a breakthrough "because it is the first drug to address the underlying cause of cystic fibrosis." It said Kalydeco helps improve lung function and lower sweat chloride levels. 

Tim Vallillee of Wilmot, near Greenwood, N.S., said Kalydeco could help him live healthier and longer.

“To see my life change the way this drug says it will change is unfathomable,” he said. “Because I’ve lived my whole life knowing what’s going to happen to me the older I get, and it’s not good.”

The 45-year-old's health is deteriorating. For the last year and a half, he’s required an oxygen tank while he sleeps.

“I’m one bout of pneumonia away from being really ill or not here.”

That’s why it’s so frustrating that he doesn’t have access to the medication, he said.

“These drugs are costing so much per person, it’s ridiculous.”

Vallillee is one of only about 100 Canadians with a rare mutation of cystic fibrosis — G551D — that responds to the drug. About 24 of them are getting the drug through private insurance or because they were part of clinical trials. 

The provinces, with the exception of Quebec, have banded together to negotiate a lower price, but after a year there’s still no deal with Vertex, the drug maker.

Only a handful of Canadians who have private insurance covering the drug are receiving the medication.

“To come to find out that this drug was not going to be given to me because of the price tag involved is extremely frustrating to me and very depressing — knowing that it’s not the Holy Grail, it’s not a cure, but it’s pretty darn close.”

More widely available in U.S., U.K.

Ken Chan, a vice-president at Cystic Fibrosis Canada, said that more patients in the U.S. have access to the pills.

"We're so quick as Canadians to judge the U.S. health-care system, but right here, in our backyards, we have our own version of a two-tiered health-care system."

Chan said Kalydeco has been available in the States since it was approved in January 2012. It's provided to nearly all 1,200 people who need it either through private insurance or government programs.

'We cannot put a price tag on the health and well-being of any Canadian'— Ken Chan, VP at Cystic Fibrosis Canada

The drug is also available to the 400 people who require it in the United Kingdom.

Chan said just a few people in Canada are taking Kalydeco right now because they have generous private insurance programs.

"Meanwhile, dozens of others – children and adults – with cystic fibrosis still wait patiently for access to the drug."

He's quick to add their patience is wearing thin as governments slowly debate with the drug manufacturer for a new price.

"If they can't get a deal, my fear is that Canadians with cystic fibrosis who need access to Kalydeco, in essence, their luck will run out."

Chan is calling on provincial governments and drug companies to strike a balance and come up with a long-term solution to the drug price problem for rare illnesses. He points out that there are other drugs that are going through a similar negotiation process.

"My submission to the provincial governments across the country is focus on the clinical benefits of this product, not so much on the price, because we cannot put a price tag on the health and well-being of any Canadian."

Chan said nearly every person who needs Kalydeco and takes it ends up leading a relatively normal life. He said patients return to work, and children can play freely. 

Family support

Meanwhile, Vallillee said he feels as if he’s on an emotional roller-coaster. The potentially life-changing drug he’s wished for is out of reach, and he's increasingly angry about the situation.

“Don’t make a profit off me and my life," he said. "That’s not fair.”

Fake Kalydeco

Vallillee made this fake bottle of Kalydeco, in hopes that it may one day contain the actual pills. (CBC)

Vallillee’s wife, Agatha Bourassa, said the family has been aggressively fundraising to try to pay for the pills.

“But we know that fundraising $300,000 a year would be literally impossible,” Bourassa said. “Obviously it’s not an amount that we would ever be able to come up with.”

Still, the family remains optimistic. Vallillee is determined to watch his young son grow up.

Vallillee made his own fake bottle of Kalydeco, which remains empty on the table next to the stacks of pills he’s taking now. He said he pretends to take a Kalydeco pill every day, in hopes of it one day being a reality.

The Nova Scotia Health Department said it's considering covering the medication. But a key factor is whether the price comes down.

His wife has a message for the politicians who are considering the plan.

“I wish that they would really consider that it is the life of a person that’s at stake here.”

Vertex, the company that makes Kalydeco, said its goal was to help as many people with cystic fibrosis as possible.

"The price of Kalydeco reflects how well it works, the time and cost to develop the medicine and our commitment to help more people with CF in the coming years," said spokesman Zachary Barber. 

"We are in active discussions in Canada that we hope will — similar to other countries around the world — result in eligible patients being able to receive this medicine as soon as possible."