Hope of B.C. coverage for life-saving cystic fibrosis drug dashed

Liam Simpson, 16, is fighting for a drug he says helps him live a normal life with cystic fibrosis, but which the B.C government won't cover through PharmaCare. 

"My lung function has skyrocketed. It's a drastic change," said the teen who is no longer teased in gym class because he can't run.

"It makes me feel more normal. It's helped a lot."

More than that, the drug is saving and extending Simpson's life.

Experts say Orkambi is one of the few new cutting-edge drugs that targets the defect that causes cystic fibrosis, an inherited disorder which affects 4,100 Canadians.

The genetic mutation affects secretory glands, clogging lungs with mucus, affecting the pancreas and gut.

Orkambi is used in the treatment of people over 12 with a specific gene mutation, seen in about 80 per cent of CF patients.

But it's expensive — $250,000 per year per patient.

Despite Health Canada approval and coverage by some private insurance companies, the province won't fund the treatment of any of the estimated 150 people with the disorder in B.C.

"We understand that quality of life for people with cystic fibrosis can be a significant challenge and it is important that these patients get the treatment they need," the Ministry of Health said in an emailed statement.

However, the ministry says B.C. has decided "along with the other provinces and territories, not to list Orkambi as a PharmaCare benefit."

That's in part because the arms-length organization that advises provinces about drug coverage says there is "insufficient evidence" from drug trials to prove efficacy — a decision endorsed by a B.C. drug advisory committee.

Drug changed life

Tell that to Simpson who says taking four little pills a day has changed his life.

Simpson used to be hospitalized for every cold, struggling to breathe.

Since taking Orkambi he's had three years hospital-free with doubled lung function.

"It's clear that this drug can have a profound effect. It's about dollars," said Liam's father, Martin Dunphy, who describes the decision not to publicly fund the drug a "death sentence" for some.

"It's rather infuriating. People have had hope yanked out from underneath them."

Liam participated in trials of the drug and was offered access to it as part of that program as approvals were negotiated.

But others have no access.

It's heartbreaking to bump into parents at the hospital," said Dunphy.

Lung function lost

"Each time their kid is hospitalized they get more scar tissue on the lung. That is lung function lost that can never be regained."

While some private insurance companies cover it, some do not or cut it off after a certain amount of time or a certain number of claims.

The organization that recommended provinces do not cover the drug said its review of current science found it lacking, even though many world experts in CF applaud the drug.

Dr. John Wallenburg, the chief scientific officer at Cystic Fibrosis Canada, says trials have shown the drug to increase lung function and benefit more people with CF by improving lung function.

Health Canada agreed, approving the drug's safety and efficacy in January 2016.

But the Canadian Agency for Drugs and Technologies in Health (CADTH) gave the drug a negative recommendation in October 2016, urging provinces not to include it in their coverage.

Brent Fraser with the CADTH says cost was not the only factor.

He says the CADTH review was more extensive than most  private insurance companies and the efficacy of the drug was not proven in clinical trials.

Cost factor

Wallenburg disagrees saying the decision was "largely motivated by price."

Delays and demands for more clinical trials will be a death sentence for some, he said. "This is a dead-end decision."