A drug that has been lauded as the "single biggest innovation" in the treatment of cystic fibrosis has yet to be funded in Manitoba — and the national organization that informs how provinces fund medications will limit who will be eligible for the drug, advocates say.
The Canadian Agency for Drugs and Technologies in Health has drafted a recommendation that the cystic fibrosis drug Trikafta be rationed to limit coverage to only those with a lung function of 90 per cent or less.
This means Canadians with cystic fibrosis, a progressive genetic disease that damages the respiratory and digestive system with no known cure, with healthier lungs will be forced to wait for their health to decline before being able to access a drug that could help prevent further disease progression, said Dr. Nancy Porhownik, a respirologist and director of the Winnipeg adult cystic fibrosis clinic.
"I think our concern as the population of CF physicians is that the recommendations that came out really have kind of missed the boat on some of the ways that this drug is really vital for the population," she said.
Cystic Fibrosis Canada estimates that under the current suggested restrictions, more than 25 per cent of Canadians with cystic fibrosis, many of whom are young children, will be excluded or need to become more sick in order to access Trikafta.
Trikafta can be taken by people with cystic fibrosis who have a specific type of genetic mutation that causes the disease, and is the first treatment that deals with the underlying causes of cystic fibrosis, rather than trying to mitigate the damage caused by it, Porhownik said.
"The difference that this medication has made in their lives is honestly just breathtaking," she said. "I have patients who were on oxygen, who were able to come off oxygen. I’ve had patients who were on the wait-list for lung transplant who have been able to come off the wait-list for lung transplant."
Trikafta was approved for sale in Canada in June, but isn’t accessible to Canadians until it is funded by provincial drug plans. Those plans are decided by provinces independently, and take the review from the Canadian Agency for Drugs and Technologies in Health into account.
"I think (the agency) is sort of trying to balance it. They’re wanting to ensure there is robust data to justify costs. I think that is part of it," Porhownik said.
While advocates consider widening the pool of people who can take Trikafta in Canada the first hurdle, getting it approved in Manitoba is the second. Ontario, Alberta, Prince Edward Island and Newfoundland and Labrador have agreed to fund Trikafta treatment, but Manitoba has yet to do the same.
"We’d like to see that our patients have the same access as other parts of Canada and other parts of the world, for sure," Porhownik said. "That’s a frustration that I’ll admit that I have as well. And I can’t speak to why they’re slower to decide to fund the medication."
Malak Abas is a reporter for the Winnipeg Free Press.