'A price on your life': Toronto cystic fibrosis patients slam review process while waiting for access to life-saving treatment

Mikayla and Madison Bianchin. (Provided by Madison Bianchin)

TORONTO -

Hamilton sisters Mikayla and Madison Bianchin are looking ahead to bright futures.

Mikayla, 20, is in her last year of illustration at Sheridan College. Madison, 18, has just graduated high school and is heading to university for nursing in the fall.

However, both sisters are living with cystic fibrosis (CF), the most common fatal genetic disease affecting Canadian children and young adults.

While there is no known cure for cystic fibrosis, a relatively new drug — a genetic modulator called Trikafta — has seen significant improvement in lung function and overall quality of life in those who live with the condition. Cystic Fibrosis Canada describes Trikafta as "game-changing" for patients.

While Health Canada approved Trikafta for patient use on June 18, 2021, it is still not approved to be covered under Canadian health insurance plans.

A year’s worth of Trikafta for one person can cost more than US$ 300,000 a year, so having the drug insured is necessary for most Canadians living with cystic fibrosis to access it.

Trikafta will not be covered in Canada until The Canadian Agency for Drugs and Technologies in Health (CADTH) completes a review of the drug and provides guidance for coverage.

A draft of that review arrived this week. However, Cystic Fibrosis Canada called the draft “a short-sighted and narrow guidance ..., that, if adopted by the provinces, could effectively exclude over 25 per cent of eligible Canadians with cystic fibrosis.”

The two recommendations flagged by Cystic Fibrosis Canada as exclusionary state that coverage will be limited to:

• those with a measure of lung function of 90 per cent or less; and
• those who see at least a 5 per cent increase in lung function within six months of starting on the drug.

If these guidelines are implemented, Madison would be one of the many Canadian’s excluded from coverage, as her lung function is currently above 90 per cent.

“I'm fairly healthy, but I still have to do three hours of insulation treatments every day to feel healthy enough to do everything that I need to do. My lung function is above 90 percent, but it's scary because I know how fast that can decline,” Bianchin told CTV News Toronto on Monday.

“I don't see the point in getting more sick, just so I can get on Trikafta. I just want to stay healthy.”

Mikayla, on the other hand, would qualify for coverage under CADTH’s proposed guidelines, as her lung function is currently below 90 per cent.

“I've literally been waiting 20 years for some kind of treatment option that actually helps the root cause of cystic fibrosis,” she said. “I do hours of daily treatments. I take over 50 pills every day. I have multiple puffers. I cough all day long. I feel pain all day long,” she said.

“If I could just have even a day of being on Trikafta, it would be a miracle.”

Dr. Anne Stephenson, Toronto respirologist and cystic fibrosis physician, told CTV News Toronto on Monday that the proposed guidelines could “discriminate mostly against children,” although not exclusively. 

“Within youth, lungs can have significant deterioration or damage within them, but lung function can often still be within the normal range,” she explained.

Some of her patients are currently taking Trikafta through a compassionate care program offered by manufacturer Vertex. She says her patients feel “generally much better” when on the drug.

“We've seen a significant weight gain in patients, a significant reduction in hospitalizations and exacerbations … They don't cough as much anymore or they don't feel short of breath,” Stephenson said.

She says the proposed guidelines could encourage patients to wait until their health declines enough to be considered for coverage.

“With a drug like Trikafta and a disease like CF, you don't want to have to wait until lung function has been reduced and damage to the lungs has already occurred,” Stephenson argued.

“It sort of defeats the purpose.”

Madison is feeling the effects of waiting to know whether she will qualify for the life-saving drug.

“It's just so upsetting to have someone put a price on your life. They decide whether you're going to live a good quality life or if you're going to just decline and not live as long as everyone else,” Bianchin said.

 

 

 

 

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Ontario Health Minister Christine Elliot has made verbal promises that Ontario will provide provincial coverage to all those who live with CF, despite CADTH’s final guidelines and review, but has yet to act on those promises.

When reached for comment, the Ministry of Health said that “Ontario welcomes the news that Health Canada has recently approved Trikafta for sale in Canada.”

“Once the final CADTH recommendation is available, Ontario looks forward to continuing existing discussions with Vertex through the pan-Canadian Pharmaceutical Alliance (pCPA),” Bill Campbell, media relations for the Ministry, told CTV News Toronto on Monday.

“Ontario recognizes that the cystic fibrosis community is anxious for access to new and effective treatments such as Trikafta. Ontario will continue to work quickly through the established review and pCPA processes,” Campbell said.

Meanwhile, the Bianchin family has a message to others living with cystic fibrosis, waiting for their chance to get on Trikafta.

“To everyone in the community that's waiting for Trikafta: I want them to know that nobody fights cystic fibrosis alone,” Mikayla and Madison’s mother, Tammy Strong, said.

“There's a whole team of volunteers, parents, CF advocates, and patients that are helping us get the word out. They're not alone and we're all going to fight and help them get the medication that they deserve.”