‘Like turning back the clock’: Windsor dad with cystic fibrosis among patients seeking access to new therapy – CBC.ca

Rian Murphy was diagnosed with cystic fibrosis as a child andnever expected to live into his 30s, but Health Canada's recent approval of a new breakthrough drug treatmenthas theWindsor, Ont., dad hopefulhe'll be spending many more years with hisson.

"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, Trikaftawasapproved for use in patientsage 12 and over who have aminimum of one of the CF F508del gene mutations.

Cystic Fibrosis Canada (CFC) calls Trikaftaa "transformational" therapy that couldtreat up to 90 per centof Canadianswith theprogressive, genetic disease, whichaffects the lungs and digestive system, and is the most common fatal genetic disease in children. TheCFCestimates one in every 3,600 children is born with the disease, and over4,370 Canadiansattend specialized clinics.

Over time, the CFC says, Trikafta could reduce severe lung disease by 60 per cent andthe 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.

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

"I never thought about retirement. Inever 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 andwas hospitalized for threeweeks at St. Michael's Hospital in Toronto. He hadbeen on and off intravenousantibiotics 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."

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 aswith a couple of other drugs for CF, provincial insurance coverage for Trikaftaremains a concern for patients, Kelly Grover, president and chief executive officer of CFC, saidin a release.

"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. Provincesend the wait and save lives."

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

In astatement released shortly after Health Canada announcedapproval of Trikafta, thepCPAsaid it has agreed to negotiate prices for the CF drugsOrkambi 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 hecan eventually access the drug.

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

His wife Diane, who'sactively 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.

Shehas hopes of her husband "watching our child grow up."

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