Jeremie Saunders, who lives with cystic fibrosis and co-hosts of a long-running podcast about chronic illness called Sickboy, poses in his studio in Halifax on Nov. 20. Within two months of taking Trikafta, Mr. Saunders’s lung function was up to 83 per cent.Darren Calabrese /The Globe and Mail
In the three years before he started taking an expensive new drug for his cystic fibrosis, Jeremie Saunders could feel his health failing in ways he knew would cut his life short.
The Halifax podcaster, now 36, was in and out of hospital with pneumonia and chest infections. He coughed up blood. His lung function dropped to 50 per cent.
All that began to change in November, 2021, when he swallowed his first dose of Trikafta, a life-altering medication for people with cystic fibrosis, an inherited genetic disease that afflicts about 4,500 Canadians.
Within two months, Mr. Saunders’s lung function was up to 83 per cent. He hasn’t been admitted to hospital since.
Mr. Saunders’s experience is like that of many cystic fibrosis patients taking Trikafta, according to a new report released Thursday by the Canadian Institute for Health Information (CIHI.) It found that, in the year after patients started taking the daily pills, their admissions to hospital fell by an average of 65 per cent, their visits to the emergency room dropped by 20 per cent, and their use of antibiotics to cure infections decreased by 58 per cent.
Measuring Trikafta’s downstream effects on the Canadian health system – and on health care spending – is particularly important because of the drug’s upfront cost. The sticker price for Trikafta is just over $300,000 per patient per year, making it one of the more expensive drugs in the world. Nearly all Canadians taking Trikafta have at least 90 per cent of the cost of the drug covered by taxpayers, according to CIHI.
“It would appear that [Trikafta] is going to have an effect on decreasing hospitalizations for this group of people, which is, on the one hand, good for the health care system, because it frees up beds,” said Tracy Johnson, the director of pharmaceuticals at CIHI. “On the other hand, it improves quality of life for these folks.”
Cystic fibrosis is an inherited disease in which the most common genetic mutation causes a critical protein to malfunction, filling some organs with a thick, sticky mucus. The mucus clogs patients’ lungs, causing relentless coughs, severe chest infections and, eventually, an early death. Trikafta doesn’t fix the underlying genetic defect that causes CF, rather it makes the faulty protein work well enough to dramatically improve health.
The new CIHI report attempts to put a partial dollar figure on the savings Trikafta produced for hospitals. An analysis of 683 patients in six provinces found that the total costs of their days in hospital dropped by 74.5 per cent, from $6.97-million in the year prior to starting Trikafta to $1.75-million in the year after.
But as Michael Law, the Canada Research Chair in Access to Medicines at the University of British Columbia, points out, if Canadian public drug plans were paying the list price of $306,000 a year for Trikafta, buying a one-year supply of the drug for those 683 patients would cost nearly $210-million.
When the organization that is now Canada’s Drug Agency evaluated Trikafta for patients 12 and older in 2021, it concluded that the list price would have to be reduced by at least 90 per cent to be considered cost effective.
“When we’re considering the value of this drug, I think it’s really important that we think about the clinical benefit that it’s providing for patients, which is pretty clear,” said Dr. Law, who is also a professor in the School of Population and Public Health. “But there’s been a promise for a long time that this drug would offset a ton of expenditure in the health system, and we’re seeing a bit of it here, but we’re certainly not seeing it on the same order of magnitude as we’re seeing in the price of the drug.”
There are many complicating factors when trying to judge whether an expensive new drug is worth the money. One is that publicly available list prices are something of a mirage because they don’t reflect the confidential discounts that pharmaceutical companies negotiate with public drug plans, Dr. Law said.
Provincial and territorial public drug plans spent $285-million on Trikafta in 2022, the most recent year for which CIHI has released data. That figure reflects spending before confidential discounts are factored in; the real financial outlay isn’t publicly available.
The drug, which is made by Boston-based Vertex Pharmaceuticals, accounted for 25 per cent of the growth in public spending on medications in Canada that year. (Sarah Wilkie, the communications manager for Vertex Canada, told The Globe and Mail by e-mail that the company’s pricing philosophy reflects the benefits of its medications to “patients, caregivers and health care systems.”)
Another complicating factor is that some savings to the health care system aren’t necessarily tallied soon after a drug hits the market. The new CIHI report, for example, does not assess savings from the reduction in lung and other organ transplants that the Canadian Cystic Fibrosis Registry has recorded since Health Canada approved Trikafta in 2021.
There were seven organ transplants in CF patients in 2022, down from 62 in 2018, according to registry data. Fourteen CF patients were removed from the transplant waiting list.
For Mr. Saunders, who hosts Sickboy, a podcast that uses humour to demystify illness and disease, Trikafta has given him the possibility of a longer life after defining himself in his teens and 20s as someone who lived for the moment because he had no other choice.
The depth of his health transformation sunk in on a day when, not long after starting Trikafta, he walked his dog, Donut, up Citadel Hill in Halifax without getting winded. “That was the moment,” he said, “where I could conceptualize how wild this transformation had been.”