Edmonton

Local family hopeful after Health Canada approves drug that treats common cause of cystic fibrosis

Local families and advocates reflect on the recent approval of a drug to treat cystic fibrosis — and what needs to happen before they can access it.

Trikafta approved for people aged 12 and up with the fatal genetic disease

Laura Stevens, left, poses with her mother Sandy Stevens, right. Laura was diagnosed with cystic fibrosis as a baby, but is excited about a new drug on the market to treat the illness. (Submitted by Sandy Stevens)

When Sandy Stevens heard that a life-saving drug to treat cystic fibrosis was approved by Health Canada, the first person she told was 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 baby. At eight years old, she lost over half of her lung capacity after contracting an infection. Then, in 2019, there was a period where she was near death, her mother says.

"I can't laugh with her anymore because if I do, she gets so short of breath and it's so hard," said Stevens.

When that happens, "everything kind of feels serious and not fun," she added.

Cystic fibrosis (CF) is a fatal and progressive genetic disease that affects over 4,300 Canadians. The illness causes a buildup of mucus in the body that leads to severe respiratory issues and other related complications.

Last month, Health Canada issued a notice of compliance for Trikafta, a prescription drug developed by Toronto-based Vertex Pharmaceuticals for some people with cystic fibrosis.

Laura, left, and Stevens, right, are shown here speaking at a fundraiser. (Submitted by Sandy Stevens)

The notice means the drug can be put on the market. It was approved for use in people aged 12 and older with the F508del mutation — the most common to cause cystic fibrosis. 

The drug is the "single greatest innovation in the history of cystic fibrosis" and could potentially increase the life expectancy of children with CF by over nine years, Cystic Fibrosis Canada, a national non-profit devoted to finding a cure for the disease, said in a news release.

Trikafta could reduce the number of people living with severe lung disease by 60 per cent, and the number of deaths by 15 per cent, the release adds.

Laura Stevens has educated herself on the drug and spoken with people who have used it. She now has a new hope for life and plans for her future, said Stevens.

"She talks about being over 50, which is not what she thought," Stevens said.

"It gives so much hope."

The next steps to access

There are still several things that must happen before Canadians with CF can access Trikafta. 

The pan-Canadian Pharmaceutical Alliance, which aims to create cost-effective drug plans, determines the price and conditions of coverage on behalf of provincial and territorial governments.

Those governments then must decide whether to publicly list the drug in their formulary — essentially a list of prescription drugs that are paid for by a health plan.

Some people may choose to pay for the drug through private insurance. But that is not an option for other Canadians, including Stevens and her daughter.

Stevens joined forces with Sharon Stepaniuk, a mother with two children with cystic fibrosis, in 2019 to advocate for the Alberta government to include Trikafta and other treatments on its public formulary.

Sharon Stepaniuk, a mother with two children who have cystic fibrosis, teamed up with Stevens to advocate for the Alberta government to include Trikafta on its drug formulary. (Submitted by Sharon Stepaniuk)

"Alberta has been a leader for adding these modulators to the provincial funding formulary in the past," said Stepaniuk.

"I do feel hopeful that Alberta will lead the way when it comes to listing this as part of the public formulary."

Alberta is waiting on final recommendations regarding coverage from the Canadian Agency for Drugs and Technologies in Health — a non-profit that gives information to health-care leaders about optimal drug use and medical devices. The province will review the information before deciding whether to add Tikafta to the formulary, a provincial spokesperson told CBC News in a statement.

Alberta is taking steps to "streamline its listing processes" so people can access the drug as soon as possible, the spokesperson said.

"Having access to this medication is important to Albertans with cystic fibrosis and we look forward to providing this medication to all eligible patients."

For Laura Stevens, the medication means she can laugh more.

"I just want to laugh. I want to be able to laugh without coughing and trying to catch my breath," her mother recalls Laura saying.

ABOUT THE AUTHOR

Anusha Kav is an intern at CBC Edmonton. She is completing a Master of Journalism at the University of British Columbia where she specializes in gender issues. Contact Anusha at anusha.kav@cbc.ca.

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