Ontario OK’s ‘transformational’ cystic fibrosis drug for patients six and older

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One by one, Sara Aldrich’s dreams are coming true.

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Eight months ago, the mere thought of climbing stairs without throwing up and vomiting seemed incredibly over the top for the now 24-year-old Stittsville resident with cystic fibrosis, not to mention becoming a teacher, running a marathon or the one of the other notions that might enter a young woman’s head with her whole life stretched out before her.

At the time, with his lung function at around 40 percent and declining, Aldrich’s life was uncertain in length and quality, and planning anything beyond a lung transplant seemed overly optimistic.

All of that changed thanks to Trikafta, a drug described as “transformative” and the biggest development in the treatment of cystic fibrosis, allowing people with the disease to lead lives that previously would have been impossible.

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The drug is not a cure – cystic fibrosis has no known cure – but it does significantly reduce the severity of the disease and slow its progression.

Aldrich, who started taking Trikafta last November, isn’t ready to run a marathon just yet, but since taking the drug she’s walked three miles without any problems and, with her lung function in the years 60, a figure she says she can expect to improve for another year and a half before plateauing, almost daily escalations. This summer, she is director of Camps Canada at Stephen Leacock Public School, the same position she had to leave three summers ago because of her failing lungs.

This fall, she plans to take up a teaching position with the Ottawa-Carleton District School Board.

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« Honestly, I can do pretty much anything now, » she says. “I just feel unstoppable. They told me I probably couldn’t even finish college, and I just finished my second degree.

« It’s good to no longer have a fixed expiration date in my life. »

The enthusiasm for life that Aldrich now enjoys is set to spread across Ontario and beyond following the province’s announcement last week that it would make Trikafta available to CF patients as soon as six years of age – recipients previously had to be at least 12 years old – and to relax restrictions on qualifying criteria, eliminating the previous requirement that a patient’s lung function had to be at 90% or less, a constraint that prevented about a quarter of Canadian CF patients from accessing Trikafta. According to Kim Steele, director of government and community relations for Cystic Fibrosis Canada, the end of the restriction will allow many cystic fibrosis patients to request the drug for the first time.

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« That’s huge for anyone who may have high lung function but may have severe digestive issues or many exacerbations, » Steele said. “It also opens the door for children aged 6-11 in a way that will slow down progression. They may not even feel any symptoms of the disease for a while.

« Being able to start children at age six is ​​going to mean a very different reality for them than people who are currently living with cystic fibrosis. »

Cystic fibrosis is a progressive, degenerative multisystem disease that primarily affects the lungs and digestive system by creating a buildup of thick mucus, causing breathing problems and making it difficult to digest and absorb nutrients. In 2018, half of CF deaths in Canada were in people under the age of 33.

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Figures from the Canadian Cystic Fibrosis Registry indicate that there are approximately 1,450 people with CF in Ontario, 930 of whom are 12 years of age or older and could benefit or could already benefit from Trikafta. Another 157 are between the ages of 6 and 11 and could potentially benefit from the drug, provided they can access it through public or private insurance.

In 2020, a study by researchers from Dalhousie University, The Hospital for Sick Children and St Michael’s Hospital concluded that widespread access to Trikafta would lead to numerous health benefits, including an increase in median age estimated survival of a child born with cystic fibrosis of 9.2 years, a 60% drop in the number of people living with severe lung disease, and 15% fewer cystic fibrosis-related deaths.

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About 10 percent of patients with cystic fibrosis show no improvement in their condition after taking Trikafta.

With an annual list price of $300,000, however, the drug is extremely expensive and not all insurers will cover it. The provinces and territories have negotiated a lower price, but one of the conditions of the negotiation is that they cannot reveal the lower price.

Ontario is the first province to lower the age of availability and eliminate the lung function requirement, announcing the changes last Friday. Alberta followed suit on Monday, and Steele hopes other provinces and territories will soon follow suit.

But Steele notes that drug coverage can still be an issue in Ontario, the only province, she says, that doesn’t have a safety net for those who can’t get coverage through other means.

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But the benefits that more widespread availability of Trikafta can bring to the overall healthcare system are enormous, she says, including fewer hospitalizations and transplants.

“There are downstream impacts,” she says, “not just on individual health, but on the system that this decision is going to have. Later, we can envision a world where people with cystic fibrosis may not need to go to clinics four times a year, for example. So we’re really, really excited about what this could mean for easing some of the burden on the healthcare system and for people who are pursuing lives they might never have thought they would have.

Like the one Sara Aldrich likes.

« It’s nice to hear my family and friends say, ‘Oh, we got the you back that we had before you got so sick, » Aldrich says. « Because I’ve been sick my whole life. That’s how I identified myself, as someone who was sick and couldn’t live a normal life. But now I can hang out with my friends and be a normal 24 year old.

bdeachman@postmedia.com