'Like turning back the clock': Windsor dad with cystic fibrosis among patients seeking access to new therapy

·4 min read
Windsor, Ont., dad Rian Murphy has had cystic fibrosis since childhood, and wants to live to see his year-old son grow up. Murphy has hope he and other people with the respiratory disease will get access to a new drug, Trikafta, which was approved Friday by Health Canada. (Submitted by Rian Murphy - image credit)
Windsor, Ont., dad Rian Murphy has had cystic fibrosis since childhood, and wants to live to see his year-old son grow up. Murphy has hope he and other people with the respiratory disease will get access to a new drug, Trikafta, which was approved Friday by Health Canada. (Submitted by Rian Murphy - image credit)

Rian Murphy was diagnosed with cystic fibrosis as a child and never expected to live into his 30s, but Health Canada's recent approval of a new breakthrough drug treatment has the Windsor, Ont., dad hopeful he'll be spending many more years with his son.

"It's a massive step going forward for cystic fibrosis patients such as myself," said Murphy about the triple-combination therapy Trikafta. "At the end of the day, it's a big, big window of opportunity for us to look down the road, future-wise."

On Friday, Trikafta was approved for use in patients age 12 and over who have a minimum of one of the CF F508del gene mutations.

Cystic Fibrosis Canada (CFC) calls Trikafta a "transformational" therapy that could treat up to 90 per cent of Canadians with the progressive, genetic disease, which affects the lungs and digestive system, and is the most common fatal genetic disease in children. The CFC estimates one in every 3,600 children is born with the disease, and over 4,370 Canadians attend specialized clinics.

Over time, the CFC says, Trikafta could reduce severe lung disease by 60 per cent and the number of deaths by 15 per cent, and increase life expectancy by several years, the CFC says in quoting research. Clearing the airways from mucus buildup is important in CF care.

Submitted by Rian Murphy
Submitted by Rian Murphy

In the last three years, half of Canadians who died of cystic fibrosis were under age 34.

"I never thought about retirement. I never thought about those things because my whole life I was told you're never going to make it until you're 20, you're 30," said Murphy, who with wife Diane are parents to their year-old son Logan.

Three years ago, Murphy lost significant lung function and was hospitalized for three weeks at St. Michael's Hospital in Toronto. He had been on and off intravenous antibiotics for months.

"On an average day, I'm doing about two to three hours of masks, and vests and physiotherapy, not including all the pills I take," he said.

"I'm 34 years old. If I can obtain this drug [Trikafta] and take it for the recommended period of time to get the results, it would be like me turning back the clock."

'Fight hard for access'

There's no cure for CF. While other therapies work to address the symptoms, Trikafta helps the defective protein function more effectively.

With Health Canada's approval, doctors can now prescribe Trikafta.

But as with a couple of other drugs for CF, provincial insurance coverage for Trikafta remains a concern for patients, Kelly Grover, president and chief executive officer of CFC, said in a release.

Submitted by Rian Murphy
Submitted by Rian Murphy

"Together we have been fighting hard for access to these medications," he said. "Thousands of letters and signatures and hundreds of meetings later, we are finally seeing the progress our community deserves.

"We turn to the provinces next. They must immediately fund Kalydeco and Orkambi, which have been in negotiations for more than a year, and fund Trikafta as soon as possible. Provinces — end the wait and save lives."

Ontario awaiting review processes

The pan-Canadian Pharmaceutical Alliance (pCPA) is a regulatory body that negotiates drug prices on behalf of the provinces.

In a statement released shortly after Health Canada announced approval of Trikafta, the pCPA said it has agreed to negotiate prices for the CF drugs Orkambi and Kalydeco, and Trikafta might be added to the agreements, pending a positive regulatory and health technology assessment recommendation.

CBC reached out to the Ontario government for comment, and in an email, the Ministry of Health said the province "recognizes that the cystic fibrosis community is anxious for access to new and effective treatments such as Trikafta," and "will continue to work productively through the established review and pCPA processes."

In the meantime, Murphy and his wife have started a fundraiser, hoping to raise enough money so he can eventually access the drug.

"If I can get a couple months, that's huge," he said.

His wife Diane, who's actively involved with CFC, as well as a petition and Instagram groups calling for the Ontario government to fund Trikafta, encourages the public to send letters to the province.

She has hopes of her husband "watching our child grow up."

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