Provinces reject price negotiations for Orkambi cystic fibrosis drug

The provinces are maintaining a united front in denying a drug manufacturer's bid to negotiate the price for a costly drug, used to treat patients with cystic fibrosis, that has not been approved for public funding in Canada.

Vertex Pharmaceuticals Inc. is demanding negotiations with the provinces on a bulk deal for Orkambi, which costs just under $250,000 a patient each year. But the national agency that evaluates new medicines to determine if they should be publicly funded in Canada has twice rejected the drug, concluding there is not enough evidence it is effective.

Advocates for patients with the life-threatening lung disease say the drug can be "life-altering" and are calling for the provinces to approve funding. It's expected fewer than 2,000 Canadians would be in line for the treatment.

B.C. Health Minister Adrian Dix said on Thursday he pushed to change the review process so that the company can resubmit its application for federal approval, but he said the provinces will not bargain with the company until it clears that hurdle.

"They have to stop playing games and follow the rules," Mr. Dix told reporters in Victoria.

The Canadian Agency for Drugs and Technologies in Health (CADTH) has recommended, on two occasions, against public funding for Orkambi, saying there is not enough evidence of a significant clinical benefit weighed against the cost of the twice-a-day tablet regime.

Mr. Dix asked health officials in August to look at changes to ensure that the company could submit additional clinical data that it says will demonstrate the drug's benefits. New rules to allow that evidence were adopted on Nov. 13. "We've responded to concerns of patients," he said. "That doesn't mean this manufacturer gets to do an end-run around the rules."

Vertex spokesperson Christina Cunningham said the decision is disappointing and the company will only resubmit its application if it is granted an expedited hearing and if the provinces, through the pan-Canadian Pharmaceutical Alliance, will enter into price negotiations at the same time.

"Patients have already waited two years to access medications," she said, "and it is our belief there is no reason why government can't enter into parallel negotiations … on the pricing structure." A full review would take up to six months.

John Wallenburg, chief scientific officer for Cystic Fibrosis Canada, a charitable advocacy organization, said Canada's review process is flawed and patients should not be forced to wait. "When you purely evaluate drugs based on statistics without looking at the benefit to people's lives, I think you are missing the boat. People aren't statistics," he said in an interview.

The advocacy group, which says less than 2 per cent of its funding comes from the pharmaceutical industry (including from Vertex), is planning to hold a rally in Victoria next week seeking to apply pressure to the B.C. government.

"I would like them to sit down with the companies and negotiate an appropriate and an affordable price so that individuals living with cystic fibrosis have equitable and affordable access to the drugs that they need," Dr. Wallenburg said.

Mr. Dix said the Pharmaceutical Alliance, which negotiates group discounts on behalf of Canada's public drug plans, will stand firm and wait for the clinical evidence to be evaluated before discussing pricing.

The Health Minister said patients should be encouraging the drug manufacturer to make its application. Instead, he said, the company is using patients as pawns in an attempt to exert political pressure on health ministers.

Earlier this week, Mr. Dix announced that B.C. has joined four other Canadian provinces in agreeing to publicly fund – in exceptional circumstances – one of the world's most expensive drugs, Soliris. which is used to treat a rare blood disease.

On Thursday, Mr. Dix maintained that the circumstances around Soliris and Okambi are different – other provinces had already provided two years' worth of clinical experience with Soliris.

B.C.'s public drug program, PharmaCare, currently lists 15 high-cost drugs for rare diseases. The addition of Orkambi to coverage would add an estimated $29-million a year in costs to the program.