Cornwall, Ont. woman fighting for access to life-changing drug

Chanelle LaFlèche has been waiting her entire life for a drug that can treat her rare form of cystic fibrosis.

Finally, it exists, but LaFlèche cannot access it, and neither can any of the 10 per cent of Canadians living with cystic fibrosis who do not have the most common gene mutation.

Doctors diagnosed LaFlèche at age seven, but it was after she turned 18 that the disease worsened dramatically.

Air and blood flow in her right lung dropped to just five per cent and in January surgeons removed it in a pneumonectomy. Her remaining lung only functions at roughly 30 per cent and is at risk of dropping further, but the disease isn’t confined to her lungs.

“Most people think it’s just lungs. It’s digestive, it’s sinus it’s lungs, my hearts been affected, my growth, my liver is being affected from all the medications,” LaFlèche said.

In 2019, the American government approved a drug called Trikafta, which targets the specific genetic mutations that cause cystic fibrosis and attempts to eliminate the buildup of mucus that infects and damages organs.

The American Federal Drug Administration approved the drug for those living with at least one F580del mutation, roughly 90 per cent of CF patients based on clinical studies, and extended it to those living with a number of other mutations, against which the medication proved effective through in vitro data.

Health Canada has only approved its use for those with at least one F580del mutation.

“The fact that people a few kilometres away are on it, if I was a few kilometres across the bridge I would be on it, but – it’s ridiculous,” LaFlèche said.

Health Canada is reviewing expanding the use of Trikafta and clinical trials are currently underway, but LaFlèche’s doctor says she needs treatment now.

They appealed to Health Canada under a Special Access request but a spokesperson says the request doesn’t apply, writing “this would not apply to individual indications of an authorized medication as it only applied to unapproved products.”

Health Canada recommends her doctor prescribe the medication ‘off-label,’ or outside of the authorized indicated use, something experts say is common when treating rare diseases but frequently prohibitively expensive and unlikely to be covered by provincial insurance.

“Most provincial insurance, most private insurance will say if it’s not ‘on-label’ – there’s not a clear indication – we will not cover it. Even if we know it works, even if it’s covered in other countries, even if it’s just an extension,” Durhane Wong-Reiger, President and CEO of the Canadian Organization for Rare Disorders said.

“We think it’s an obstacle in many drug plans, they’re just looking for a way of getting out of it.”

For LaFlèche and her family it would mean covering the more than $308,000-per-year drug out-of-pocket.

“If I need to sell my house, I will do it. Powerless for me, I’m getting there but I’m not there yet because I’m going to do what I need to do, I’ll make it friggin’ happen. I will,” LaFlèche’s mother Céline said.

In a statement, the Ontario Health Ministry says, “coverage for Trikafta is consistent with Health Canada product labelling,” meaning LaFlèche would not be covered.

She can make an Exceptional Access Program request but the province says those requests are reviewed against “established clinical criteria.”

A list of the drug criteria for Trikafta on the province’s website cites the main F508del genetic mutation as a requirement.

Wong-Reiger says Ontario needs to do more when considering exceptional access requests, particularly when the people involved belong to small patient populations that can easily fall outside the guidelines due to the relative rarity of their diseases.

“Quebec makes it, other countries make it; to be able to look at the individual patient, how well do you fit the existing guidelines, what is the likelihood this will work for you,” Wong-Reiger said. “It’s a matter of being able to provide some flexibility, especially when we’re talking about life-changing, life-saving kind of therapies.”

“It’s such an easy fix for them, it’s such an easy fix that allows her to live,” Céline LaFlèche said.