The CF Community and Canada's Rare Disease Strategy - 2022
After a long wait, in June 2021 the cystic fibrosis community celebrated a huge victory. Health Canada had approved a new “miracle drug” called Trikafta.
Cystic fibrosis is a progressive, genetic disease caused by mutations in the CFTR gene. Symptoms generally include a cough with thick mucus, shortness of breath, chest infections, bowel disturbances, failure to gain weight, and salty tasting sweat. The average life expectancy for individuals in Canada with CF is 54 years old.
Trikafta has shown life-changing benefits for those living with cystic fibrosis, slowing the disease's progression and significantly easing symptoms, sometimes within days of starting the medication. With Trikafta access, the estimated lifespan for a child born with CF increases by about 9 years.
But Trikafta had already been approved in the U.S. in 2019 and in Europe in 2020. In addition to the slower Health Canada approval, issues with both restrictive criteria from drug review bodies and the processes of public and private payers still kept some from accessing Trikafta. With a disease like CF, time is of the essence. Why has it taken so long to get the medication to those who need it? How can Canada do better?
Canada is one of a few developed nations without a national rare disease framework. According to the Canadian Organization for Rare Disorders (CORD), “Right now, only 60% of treatments for rare disorders make it into Canada and most get approved up to six years later than in the USA and Europe.”
Canada having a rare disease strategy would improve access to drugs for rare diseases and ensure that access is consistent in every province and territory. A national strategy would also create a consistent definition of rare diseases and a framework to address problems with access, which can vary depending on the drug plan and the province. It could also result in a coordinating body for decisions on topics like pricing.
In May 2023, the federal government finally announced a total investment of up to $1.5 billion over three years in support of Canada's first-ever national rare disease strategy.
For years, members of the cystic fibrosis community have been navigating a complex, disjointed system in order to access and afford the medication they need to live. When the next cystic fibrosis medical breakthrough occurs, a rare disease strategy in place may lift that burden.