Cystic fibrosis drug 'a relief' for family in North Bay, Ont., but restrictions on cost coverage remain

A new cystic fibrosis drug is a game-changer for a North Bay family.

Ontario is one of three provinces that announced last month it would provide eligible patients with Trikafta, which costs roughly $300,000 a year at its list price, under the Ontario Health Insurance Plan (OHIP).

Brayden Perron, 15, has lived with cystic fibrosis his entire life. The rare genetic disease affects organs like the lungs, pancreas and liver with mucus that lines those organs and can damage them. In severe cases, cystic fibrosis patients can require a lung transplant.

Cystic Fibrosis Canada estimates about 4,300 Canadians have the disease.

"Luckily, I'm still very healthy," Brayden said. "So I get to participate in all the activities that are presented to me and I don't show real struggle. But people who are less fortunate than me, who have more problems, definitely show a little bit more of a struggle in day-to-day activities." 

Brayden and his 11-year-old sister Keira, who also has cystic fibrosis, each take about 40 pills per day to manage the disease. 

On Sept. 24, Brayden started taking Trikafta, and says he has already noticed a positive change.

"I've definitely found that my appetite has significantly increased," he said. "I've just been eating so much and I am able and I feel like I never get full anymore. And my quality of sleep has significantly increased. Maybe it's because it's easier to breathe."

Trikafta is the latest in a new family of precision medicine designed to treat the underlying defect that causes cystic fibrosis. It has been found to prevent the progression of the disease in some patients and has helped others restore their lung function, which can avoid the need for a transplant.

"The drug is what they're calling the single greatest advancement in cystic fibrosis history," said Macrina Perron, Brayden and Keira's mother. 

Perron has been a strong advocate for her children, and has fought along with Cystic Fibrosis Canada for the drug to be approved and covered in Canada.

The European Union approved the drug for medical use in August 2020. In Canada, it was approved in June 2021.

"It still feels really surreal," Perron said. "As a mom, when you get that diagnosis, you automatically go to the worst case scenario. 

"You wonder what life's going to look like for your child. But the fact that this drug is now here, it was once a dream. It was once something that we dreamed about as parents. And now it's a reality. It's just so exciting. It's such a relief that it's here."

Not everyone eligible for the drug

But while Brayden qualifies for Trikafta through OHIP, his little sister Keira does not.

That's because the province has followed a draft recommendation from the Canadian Agency for Drugs and Technologies in Health to restrict the therapy to patients with less than 90 per cent lung function.

Perron said Keira had 80 per cent lung capacity at one point, but they worked hard to help her improve past 90 per cent.

She compared the restriction to withholding cancer treatments from patients who are in the early stages of the disease. 

Kim Steele, Cystic Fibrosis Canada's director of government and community relations, said the organization is petitioning the government to amend those restrictions.

Cystic Fibrosis Canada would like to see clinicians have final say on who qualifies for Trikafta, she said.

"For those patients who fall out of that 90 per cent, but might have a rapid decline or something else happening in them, it doesn't allow the clinicians to intervene in those cases directly or as directly as we would like," Steele said.