A new cystic fibrosis drug could reduce deaths by 15% – if it comes to Canada. The drug is already available in the US and UK, and could be life-changing for Canadians with cystic fibrosis.
Cystic fibrosis (CF) is a fatal genetic disease that affects around 4,300 Canadian children and young adults. It mainly affects the lungs and digestive system, with a buildup of thick, sticky mucus causing breathing difficulties and lung infections that worsen over time. For some, this can make everyday activities almost impossible, as kids struggle to breathe and absorb adequate nutrients to grow and thrive.
Ultimately, lung damage leads to a transplant or premature death. While the outlook for people with CF has improved dramatically in recent years, the life expectancy of a child born in 2018 is still only 44.
There is no cure for CF, but Trikafta could treat 90% of people living with the disease. Its component parts – the drugs elexacaftor, ivacaftor and tezacaftor – bind to, and fix, the faulty protein that causes CF. This could prevent the mucus buildup and its associated complications, keeping patients out of hospitals, off transplant waitlists and feeling better.
While it’s not currently available in Canada, researchers wanted to know what would happen if Trikafta was introduced, and the impact of delaying access.
Led from Dalhousie University, St Michael’s Hospital, and The Hospital for Sick Children, the research team used a microsimulation model to forecast three scenarios: no Trikafta in Canada, early access (2021), and delayed access (2025).
To make the forecasts as realistic as possible, the team used data from Trikafta clinical trials. The results are both exciting and frustrating.
The model showed that making Trikafta available in 2021 would mean 15% fewer deaths by 2030 compared to no drug. It could also mean 60% fewer people with severe lung disease and add nine years to the estimated survival age for newborns.
“I’ve spent my entire life struggling to stave off the deterioration of my lungs. Over 40 pills a day, multiple hours of daily treatments – it’s no small feat,” says Jeremie Saunders, who lives with CF and hosts the Sickboy podcast. “To know that there is a drug like Trikafta that could very well have a life-altering effect on my life is beyond exciting.”
The drug could also cut hospitalizations and at-home intravenous treatment courses, and reduce the need for transplants.
So, what’s the catch?
According to Cystic Fibrosis Canada, who funded the study, Trikafta costs USD $311,000 per year. While it is available to some Canadians through Health Canada’s Special Access Programme, there are some big regulatory hoops to jump through if it’s going to be more widely available.
While Canada waits, the researchers stress that the expected benefits are cumulative, and delayed access will result in preventable deaths.
Sanja Stanojevic, the study’s lead author and an assistant professor of community health and epidemiology at Dalhousie University, hopes these results will support calls for treatment access.
“This study illustrates the profound impact Trikafta can have on the Canadian CF population,” says Stanojevic. “Availability of this treatment will translate to people with CF living longer and more productive lives, and accessing healthcare far less frequently.”