Trikafta funding recommendation for rare cystic fibrosis mutations brings hope to some, but falls short for others, says Cystic Fibrosis Canada
FOR IMMEDIATE RELEASE
Toronto, November 6, 2024 – Cystic Fibrosis Canada acknowledges a new significant step forward in access to the life-changing cystic fibrosis drug Trikafta. Canada’s Drug Agency (CDA) has recommended that public drug programs should fund Trikafta for Canadians who have at least one of 152 rare mutations that cause cystic fibrosis. Cystic Fibrosis Canada urges all public and private drug plans to provide immediate access to Trikafta for these 240 Canadians. But we can’t stop there. There are still 180 Canadians living with rare mutations that may respond to the drug who have not been included in this latest recommendation. They cannot be left behind.
“Canada’s regulatory and reimbursement systems must act now to keep up with evolving science and ensure equitable access to life changing treatments,” shared Kelly Grover, President and CEO, Cystic Fibrosis Canada. “This recommendation is a vital step forward, but almost 200 Canadians who could potentially benefit from Trikafta are still excluded. We cannot sit by when others in our community could benefit. Our advocacy efforts will continue, not only for these individuals but also for those who may benefit from other innovative therapies that are in the pipeline.”
Following Health Canada’s July 2024 decision, Trikafta is now approved for Canadians aged two years and older with mutations that are responsive to the drug, based on clinical and/or in vitro evidence. In its decision, Health Canada refers to 152 mutations that are known to respond to Trikafta, and Canada’s Drug Agency has followed suit, recommending funding for the included mutations.
While CDA has recommended funding for the mutations acknowledged by Health Canada, this recommendation is non-binding and it is now up to Canada’s public and private drug plans to take it at face value or determine their own reimbursement strategies. Cystic Fibrosis Canada urges decision makers to provide immediate and broad access to ensure that no one is left behind, including specifically giving the ability to try Trikafta to 180 Canadians living with rare mutations that could respond to the drug but were not acknowledged by Health Canada.
“This is an advancement for nearly 240 Canadians living with one of the 152 mutations recommended for funding, offering hope of accessing a drug that can dramatically improve their lives,” said Dr. Paul Eckford, Chief Scientific Officer, Cystic Fibrosis Canada. “While we applaud this progress, it is crucial that drug plans provide immediate coverage for Canadians with these 152 mutations and provide a pathway to access for the many other Canadians who may benefit from Trikafta but are still being left behind.”
Cystic Fibrosis Canada is calling on public and private drug programs to implement approaches that could ensure all people who could benefit from Trikafta have access. By exploring emerging models such as France’s right-to-try compassionate use program and personalized assessments for individuals who cannot participate in clinical trials due to the ultra-rarity of their mutations, we can ensure that more people with cystic fibrosis who could benefit from Trikafta get the chance to.
“Every day, I fight for my health, my breath, and a chance at a normal life. Trikafta isn’t just a treatment; it’s my path back to what cystic fibrosis has taken from me. It’s incredibly painful to know there’s a treatment that could give me and others that chance, yet it remains out of reach for some,” says Lauren Clift, who is living with cystic fibrosis. “I ask those making these decisions to consider what it’s like to lose so much to this disease and prioritize access to a treatment that could give all of us a chance at a life we’ve only dreamed of.”
This recommendation follows the CDA’s 2023 endorsement of Trikafta for those aged two and older living with the most common cystic fibrosis mutation, F508del, which is now covered across all public drug plans for this demographic.
While most of Canada’s drug plans are guided by CDA to inform their reimbursement decisions, Quebec has its own process. The Institut national d'excellence en santé et en services sociaux (INESSS) provides a recommendation to Quebec’s Minister of Health and Social Services, after which the Minister has 30 days to make a reimbursement decision. Cystic Fibrosis Canada is hopeful this recommendation will come soon and be inclusive of all Quebec residents who will or could benefit from Trikafta.
Cystic Fibrosis Canada will keep the community informed as updates become available regarding public coverage for Trikafta and encourages everyone to follow the latest developments through its social media channels.
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