When Sandy Stevens heard that a life-saving drug had been approved by Health Canada to treat cystic fibrosis, she first told her 15-year-old daughter, Laura.
Laura Stevens has been in and out of hospitals for most of her life after being diagnosed with cystic fibrosis as a child. At the age of eight, she lost more than half her lung capacity after contracting an infection. Then, in 2019, came a period when she was close to death, her mother says.
“I can’t laugh with him anymore because if I do, he gets short of breath and it’s too hard,” Stevens said.
When this happens, “everything feels serious and not fun,” she said.
Cystic fibrosis (CF) is a fatal and progressive genetic disease that affects more than 4,300 Canadians. This disease causes a build-up of mucus in the body which leads to severe respiratory problems and other related complications.
Last month, Health Canada issued a compliance notice for certain people with cystic fibrosis, Tricafta, a prescription drug developed by Toronto-based Vertex Pharmaceuticals.
The notice means that the drug can be launched in the market. It was approved for use in people 12 years of age and older with the F508del mutation – the most common cause of cystic fibrosis.
The drug is the “greatest innovation in the history of cystic fibrosis” and could potentially increase the life expectancy of children with CF by more than nine years, says Cystic Fibrosis Canada, a national nonprofit dedicated to finding a cure for the disease. said in a news release.
Trikafta can reduce the number of people suffering from severe lung disease by 60 percent and the number of deaths by 15 percent, the release said.
Laura Stevens has educated herself about the drug and has spoken with others who have used it. He now has a new hope for life and plans for his future, said Stevens.
“She talks about being over 50, which she didn’t think of,” Stevens said.
“It gives a lot of hope.”
Next steps to access
There are still several things that need to happen before Canadians with CF can access Trikafta.
The Pan-Canadian Pharmaceutical Alliance, which aims to create cost-effective drug plans, sets the price and terms of coverage on behalf of provincial and regional governments.
Those governments would then have to decide whether to publicly list the drug in their formulary—essentially a list of drugs paid for by a health plan.
Some people may choose to pay for medication through private insurance. But that’s not an option for other Canadians, including Stevens and his daughter.
Stevens joined forces with Sharon Stepanuk, a mother of two with cystic fibrosis, in 2019 to advocate for the Alberta government to incorporate trikafta and other treatments into its public formula.
“Alberta has in the past been a leader in adding these modulators to the provincial funding formulary,” Stepaniuk said, adding that the province is having a “really good” ongoing relationship.
“I expect Alberta to lead the way in listing this as part of the public formulary.”
Alberta is awaiting final recommendations regarding coverage from the Canadian Agency for Drugs and Technologies in Health – a nonprofit that provides information to health care leaders about optimal drug use and medical devices. A provincial spokesman told the News in a statement that the province would review the information and then decide whether to add the vaccine to the formulary.
The spokesperson said Alberta is taking steps to “streamline its listing processes” so that people can access the drug as quickly as possible.
“Access to this drug is important for Albertans with cystic fibrosis and we look forward to providing this drug to all eligible patients,” he said.
For Laura Stevens, the drug means she can laugh more.
“I just want to laugh. I want to laugh without coughing and try to catch my breath,” recalls her mother, Laura.