HALIFAX -- A new study by researchers at Halifax's Dalhousie University suggests the lives of people with cystic fibrosis would improve dramatically with access to a new CF drug.
The drug, Trikafta, has not been submitted for approval for use in Canada yet, with the drug's manufacturer suggesting earlier this year that proposed changes to the guidelines used to regulate drug prices in Canada.
The study, primarily funded by CF Canada, found that if patients could get the drug by 2021, they would experience "profound health improvements" by 2030.
Researchers used data from CF Canada's patient registry to predict the impact of Trikafta on the CF population. There are more than 4,370 cystic fibrosis patients in Canada.
The goal of the study was to look at "what would happen to the population and what would the population look like in 10 years' time," says lead author and Dalhousie University associate professor Sanja Stanojevic.
Some of the improvements in the study include: 60 per cent fewer patients living with severe lung disease; 18 per cent more people living with mild (as opposed to severe) lung disease; and 19 per cent fewer hospitalizations.
The study also projected a change in the life expectancy of CF patients over a nine-year period.
"Today, (life expectancy) is 52 years. With no new therapies it's 58 years (by 2030), and with Trikafta, it's 67 years," says Stanojevic.
The chief scientific officer for CF Canada, John Wallenburg, says the study illustrates just how much the drug would improve the lives of Canadians diagnosed with the fatal genetic disease.
"There's marked reduction in the number of people who are suffering from severe disease and an improvement in people suffering from mild disease," he says.
For those living with cystic fibrosis, which gradually degrades lung function, the study is encouraging, but the drug still remains out of reach for most Canadians.
Like most CF patients, 27-year-old Stefan Strecko was diagnosed with the disease as an infant.
"It's very frustrating, being someone here in Canada who can't have access to it," says Strecko.
Trikafta is designed to target one of the genetic mutations that can cause CF, which about 90 per cent of patients -- including Strecko -- possess.
He says that makes it imperative to have the drug approved in Canada.
"We haven't heard much from the Canadian government, or from the pharmaceutical company, or anyone that can shed light on the status of it right now."
Tim Vallilee agrees.
The 52-year-old knows the difference the right treatment can make. Eight years ago, he and his wife fought for publicly-funded access to another drug made by Vertex -- Kalydeco. That fight took three years.
For some, the wait for Trikafta has been too long. Vallilee thinks often of Chantelle Lindsay, the 23-year-old who died in February in a Halifax hospital, after her family's application for the drug through the manufacturer's compassionate access program was denied.
Vallilee says, for those patients who have managed to access Trikafta through that program, the medication has been life-changing.
"Their results are astounding," he says. "When I got my drug, it happened overnight, literally."
His wife, Agatha Bourassa, says Canada's health minister, Patty Hajdu, needs to make Trikafta a priority.
"It just takes two people to sit at a table and have a discussion about the most amazing medication that's ever come out for cystic fibrosis," she says.
