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Canadian governments should not pay for a $250,000-a-year cystic fibrosis medication because it is not clear the drug actually improves patients’ health, according to a fresh review that has devastated some with the potentially fatal lung disease.

For the second time in as many years, the expert committee that advises the provinces and territories on drug coverage has said no to Orkambi, a pill designed to help correct the most common underlying genetic cause of CF.

The committee’s rejection is not binding, but the provinces and territories almost never break ranks with the Canadian Agency for Drugs and Technologies in Health (CADTH), the independent organization that oversees the expert committee.

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“We’re beyond disappointment,” said Kim Steele, the director of government and community relations at Cystic Fibrosis Canada, a charity and patient organization. “Our community is devastated. This is a medicine that we know works for some people.”

Orkambi is one of the new medications at the centre of a fight over how the cash-strapped Canadian health-care system should treat expensive drugs for rare diseases.

Unlike many other well-off countries, Canada does not do special evaluations for drugs that relatively few people need. There is not even a national definition of what constitutes a rare disease.

A proposed class-action lawsuit has already been launched against the federal government and British Columbia, which is the home province of the two lead plaintiffs, over the lack of public coverage for Orkambi.

About 2,000 Canadians have the genetic mutation that Orkambi is intended to help correct.

In a report released on Thursday, the CADTH’s expert panel said its review of the Orkambi clinical trials and other studies found the drug produced only slight improvements for about one in four patients.

The report also said Orkambi’s Boston-based maker, Vertex Pharmaceuticals, would have to slash the drug’s price by more than 98 per cent to satisfy the agency’s value-for-money analysis.

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“[Orkambi] is better than nothing. It’s better than placebo,” said Trevor Richter, director of the CADTH’s Common Drug Review, which oversees the review process for non-cancer medications. “But the benefit is really small. Not only is it small, but there’s a huge amount of uncertainty about what the actual size of that benefit is.”

Vertex called the agency’s recommendation “deeply disappointing."

A company spokeswoman said eight other countries, including Australia, Germany, Ireland and the United States, have made Orkambi eligible for public drug coverage.

Vertex will continue to provide the drug for free to Canadians who are on its compassionate-use plan, the spokeswoman added.

That is some solace for Lilia Zaharieva of Victoria, who has cystic fibrosis and said she was crushed by CADTH’s decision.

Lilia Zaharieva said her health has improved dramatically since she started taking Orkambi two years ago.

CHAD HIPOLITO/The Globe and Mail

Ms. Zaharieva said she “moved from deflated to indignant” as she pored over the report.

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The 31-year-old said her health has improved dramatically since she started taking Orkambi two years ago – initially paid for by private insurance, now under Vertex’s compassionate-use program.

In the year before she started the twice-daily pill regime, Ms. Zaharieva spent 45 days on intravenous antibiotics, fighting infections. She was weak, struggled for breath and had coughing fits that sometimes led to vomiting.

“Before Orkambi, I felt like I was starved for air all the time," she said. “That’s a terrifying way to exist.”

Ms. Zaharieva is one of the lead plaintiffs in the proposed class-action lawsuit, which was launched over the summer with the pro-bono help of Chris Macleod, a Toronto lawyer and CF patient who runs the Cystic Fibrosis Treatment Society, a small group that advocates for access to promising drugs for CF patients.

(The CF Treatment Society does not receive money from Vertex, Mr. Macleod said. Cystic Fibrosis Canada receives funding from drug makers, including Vertex, but Ms. Steele said the pharmaceutical industry accounts for 2 per cent to 6 per cent of the group’s annual budget, and does not influence its positions.)

Mr. Macleod takes another Vertex drug, Kalydeco, which targets a different gene mutation. CADTH gave Kalydeco a positive recommendation; it is now covered by public drug plans across the country.

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Mr. Macleod urged Ontario Health Minister Christine Elliott to set the CADTH recommendation aside.

“The reality,” he said, "is they’re not bound by CADTH. … So sit down and negotiate in good faith with the company. If you can’t get a good price, then you don’t do the deal.”

Ms. Elliott’s office referred questions to the pan-Canadian Pharmaceutical Alliance (pCPA), the group that usually negotiates confidential drug discounts on behalf of the provincial, territorial and federal governments.

The pCPA is meeting with Vertex next week to discuss next steps, but a spokeswoman for the group would not say if it intends to negotiate with the company. So far, the pCPA has refused to come to the table, citing the CADTH’s first negative recommendation on Orkambi.

Mr. Richter of the CADTH said it is difficult but necessary sometimes to disappoint patients.

“We don’t enjoy delivering recommendations that are seen as negative," he said. “In the end, we are an evidence-based review process. It’s rigorous and it’s transparent.”

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