What challenges to care do cystic fibrosis patients still face in wake of new drug approval?

Families across Canada breathed a deep sigh of relief last month when Health Canada finally green-lit a new miracle drug for cystic fibrosis, but there are still more than a few hoops to jump through.

Trikafta is the latest in a class of drugs called CFTR modulators that correct deficiencies caused by one or more of the 178 gene mutations related to the disease.

Unlike the others, which have limited success, Trikafta can literally halt the progression of lung and other organ failure in about 90% of patients.

"It's not a cure, of course, but it helps treat the underlying causes of CF,'' said John Bennett, regional director of Cystic Fibrosis Canada (CF Canada) and the father of an 11-year-old boy with cystic fibrosis in St. John's.

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"It can be a life-changing drug.''

But its approval by Health Canada has been tied up in red tape, sparking a loud lobby from parents and advocates ever since the drug was approved in the U.S. in 2019.

Now that it's approved, draft guidelines drawn up by the independent Canadian Agency for Drugs and Technologies in Health (CADTH) has many parents still scratching their heads.

"It's been a whirlwind of emotion, there's no doubt about it,'' said Bennett.

"We're still having to fight in terms of what is covered from both a provincial perspective and private insurer perspective, and now you've got these restrictive guidelines that essentially say you've got to get sicker in order to get the drug.''

The draft guidelines he's talking about are the CADTH recommendations that are adopted by most provinces, including Newfoundland and Labrador, once a drug is approved.

In this case, they include not giving the drug to anyone younger than 12, and not giving it to anyone whose lung function is greater than 90%.

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John Wallenburg says those guidelines are out of sync with the needs of cystic fibrosis patients.

"The people at CADTH have a difficult job to do,'' Wallenburg, chief science officer with CF Canada, said in a phone interview. "They're generalists, typically pharmacoeconomics. They don't necessarily understand the disease, and that's painfully obvious when you look at the draft recommendations. They simply do not understand this disease.''

Wallenburg says cystic fibrosis is progressive. Once lung and other organ damage occurs, it does not come back. Lung damage should be prevented, not treated, he says.

"This is saying, 'Well, we're not going to prevent the disease. We have to wait until we already have demonstrable damage to the lungs before we're going to start treating people.' I mean, it's analogous to a woman finding a small lump in her breast going in to see her doctor, and her doctorconfirming, 'Yes, you have breast cancer, but we're not going to start treatment until you start showing more symptoms.' It's absurd.''

Wallenburg says the agency only looked at data from drug trials, which would only have been conducted on people who have already lost part of their lung capacity.

He said scientists and advocates who contributed to CADTH's research will now respond to the guidelines and, hopefully, correct its deficiencies.

Bennett says his son, John, doesn't qualify under the guidelines because he's under 12 and his lung function is still strong.

But that doesn't mean he doesn't have major issues.

Since he was diagnosed as a baby, after not being able to gain weight, he's been taking enzymes and other treatments to make up for pancreatic involvement.

And he does various physiotherapies and lung exercises daily.

It's kept him involved in sports such as hockey and soccer, even though it hasn't kept him out of hospital on occasion.

"I don't think he'll ever have perfect attendance from a school perspective, because typically if he does get a cough, especially in the wintertime, it takes him a little bit longer to recover,'' Bennett said.

"We've been fortunate that we've never had to kind of push him to do physical activity.''

His younger brother, Noah, doesn't have the disease.

For Bennett and others, there are still hurdles other than the CADTH guidelines.

Provinces and private insurers have to wrangle over a price tag for it to be covered, and that won't be easy given it costs about US$300,000 per year across the border.

CF Canada has an online petition that anyone can sign. It can be found at https://action.cysticfibrosis.ca/.