Islander hopes for broad access to new cystic fibrosis drug

An Island man is hopeful Health Canada's recent approval of a drug to treat cystic fibrosis will mean easier access for Islanders with the disease.

Hunter Guindon, 25,spends hours every daytaking a collection of medications to help lessen his symptoms of cystic fibrosis.

There are vitamins, digestive pills, an antibiotic, another antibiotic, an inhaled antibiotic and a device used for chest physiotherapy, among others.

"Cystic fibrosis is something that you want to ignore, but you can't ignore because it affects every single thing that you do," he said.

"I have a full-time job and my part-time job is taking care of my health."

But Guindon says there could be another solution.

"Trikafta is three pills a day that's it."

The drug was approved by Health Canada last month for those aged 12 and up with at least one F508del mutation the most common gene mutation that results in cystic fibrosis.

Getting to the source of the problem

Cystic fibrosis (CF) is a genetic disease that impacts almost every organ in the body, according to Dr. Nancy Morrison, the medical director of the Adult Cystic Fibrosis Clinic in Halifax.

In the lungs, it makes the mucus thicker than usual leading to infections, damaged airways, lung transplants and in some cases, death.

It is estimated thatmore than4,300 Canadians live with the condition and there is currently no cure.

"We have many, many treatments for CF and most of the treatments are directed at treating the infections, trying to get the mucus out. So treating what's been caused," Morrison said.

"Trikafta works to correct the underlying problem."

Morrison recounts stories of patientsno longer requiring transplants, being able to return to work full time, having more energy and spending less time in hospital when they have to go in for treatment.

"One gentleman said that he ... went out and chopped wood and he said what amazed him, his arms and legs got tired before his lungs caused him to stop because of shortness of breath," Morrison said.

"So it's just, you know, remarkable."

'A sad misunderstanding'

But Trikaftacan cost up to $300,000 USfor a year of treatment.

TheP.E.I. government has verballycommitted to adding Trikafta to the provincial formulary so the cost of the drug can be covered, according to a release from Cystic Fibrosis Canada, but draft recommendations from theCanadian Agency for Drugs and Technologies in Health (CADTH) could make that access more complicated.

CADTHis an independent, not-for-profit organization that gives advice to health-care decision makers, including provincial bodies, to help make informed choices.

It's absolutely terribly frustrating.-Dr. John Wallenburg, Cystic Fibrosis Canada

Its recommendations include restricting access for those with more than 90 per cent lung function and those who see less than a five per cent increase in lung function during their first six months on Trikafta.

"[Trifakta is] as close to a cure as we could get at this point in time, and it's really a phenomenal innovation," said Dr. John Wallenburg, the chief scientific officer at Cystic Fibrosis Canada.

"And sadly, [CADTH's] draft recommendations that were put forward display, I would say, a narrow and selective interpretation of the science and a sad misunderstanding of the disease."

Wallenburg's daughter died of cystic fibrosis in 2014, and he says there's "no question" the drug would have helped.

"It's easy for me to put myself in the shoes of the people alive today and the parents with kids today," he said.

"I mean, it's absolutely terribly frustrating."

'Totally revolutionary'

In an email to CBC News, a spokesperson for CADTH said the organization is unable to comment until it formally receives stakeholder feedback.

However, the email did say the final recommendations are non-binding and a number of CF patient groups, clinical experts and specialists provided input during the process.

As for the province, it sent an email saying it "recognizes that the cystic fibrosis community would like access to new and promising treatments such as Trikafta," adding that working through the CADTH review is a "vital part of the overall process to ensure that drug treatments entering the Canadian market are safe, effective and provide value."

In the meantime,Guindon is crossing his fingersthe drug becomes available toall cystic fibrosis patients on the Island.

"It would be totally revolutionary. It would completely flip my life 360. I wouldn't even dream of starting a family or owning a business or planning for retirement at this point with that now doom and gloom of a 30, maybe 40-year-old life expectancy," he said.

"But if I was able to be on Trikafta ... I would be able to plan for a real life. I would be able to plan to start a family, plan what I'm going to do when I retire it would completely change my life."

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