'Lives are on the line:' Cystic fibrosis patients file class action lawsuit for $250K-a-year drug

For a B.C. mother who suffers from cystic fibrosis, taking four pink pills a day made it possible to get out of bed, feed her kidsand take them to school.

"For the first time in a long time, I got to be a mother to them," said Melissa Verleg, 34, of Vernon, B.C., whose children are seven and nine years old.

"Life was fantastic."

The pill was Orkambi, a medication designed to treat cystic fibrosis in patients with a specific mutation.

Patients like Verleg call it life-changing, but B.C. and other provinces don't cover its hefty cost: about $250,000 per patient per yearor $170.54 per pill.

Since Verleg's extended health plan also ruled it too expensive in January, she says her lungfunction has become "unstable," and she spendshalf her days on IV antibiotics.

Now, she is one of two lead plaintiffs in a class action lawsuit against the federal ministerof health and others for $60 million in damages for denying the coverage.

The suit alleges that failing to cover a "medically necessary treatment" is a violation of the charter rights ofcystic fibrosis patients with the mutationand hopes to change how Canada handles rare diseases.

'Insufficient evidence' says province

Orkambi is approved by Health Canada, but it's up to individual provinces what they pay for under provincial health plans, and they take guidance from a federal drug review body called the Canadian Agency for Drugs and Technologies in Health.

B.C. decided in March 2017 not to cover Orkambi, saying it was "not cost-effective," given the high price tag and what the government characterized as a "modest improvement in lung function."

CADTH's earlier review made the same call, noting that while two double-blind randomized controlled trials had shown "statistically significant absolute improvements" in lung function, the magnitude of improvement was of "uncertain clinical significance."

In a statement, the B.C. Ministry of Health said it won't comment on the lawsuit, but said there is "insufficient evidence" to pay the drug's high costand defended the decision.

"This process ensures that important drug listing decisions are based on evidence-based, clinical analysis involving patient groups and are not the result of political lobbying," said the statement.

Problem of rare conditions

The lawsuit disputes those rulings, saying the medication is medically necessary for the approximately200 patients in B.C. who have that type of cystic fibrosis.

The second lead plaintiff, Lilia Zaharieva of Victoria, B.C., says Orkambi stopped years of steep decline that had her heading for a lung transplant.

"In the year before I took Orkambi, I could hardly walk between my classes without feeling short of breath," said the 31-year-old, who now has access to the drug from the company on compassionate grounds.

The Toronto lawyer spearheading the case, who himself has cystic fibrosis with a different rare mutation, says Canada's drug evaluation system isn't fair to people suffering from rare diseases, where markets are small and large clinical trials are hard to come by.

"The system doesn't work for rare conditions," said Chris MacLeod of Cambridge LLP. "Lives are on the line."

"We all agree it's too much money. No one wants to defend a high-priced drug. Therefore, you sit down at a table and negotiate."

Beyond financial relief, the suit asks for a more "transparent, objective and evidence-based system" for drug approval in Canada, and for B.C. to negotiate with the drug manufacturer, Vertex Pharmaceuticals.

Cambridge LLP is taking on the case on a contingency basis, with neither firm nor plaintiffs getting money from Vertex, said MacLeod.

MacLeod said they chose B.C. though all provinces "are in the same boat" because of successful charter challenges here in the past, including on medically-assisted death.

CADTH is currently re-reviewing Orkambi, after the manufacturer submitted new evidence that the B.C. government said it recommendedbe considered.

The federal minister of health declined to comment.