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Cystic fibrosis Canada is committed to advocating for improved access to medicines for everyone living with cystic fibrosis in Canada. From symptom management drugs to CFTR modulators, we advocate for improved access to medicines for all Canadians living with CF. We also partner with other organizations to drive the system change needed to ensure future generations do not face similar challenges.
Cystic fibrosis is a complex disease caused by mutations in the gene for the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR). There are approximately 2000 known mutations. CFTR modulators target the cause of cystic fibrosis by correcting the defective CFTR protein, whereas other CF treatments and therapies manage the symptoms of CF.
All modulators can stabilize the health and reduce the number of pulmonary exacerbations experienced by Canadians with cystic fibrosis. Highly Effective Modulator Therapies (HEMTs) like Kalydeco and Trikafta can dramatically improve the health of people with cystic fibrosis.
The health benefits of CFTR modulators Kalydeco and Trikafta in particular have been nothing short of spectacular, with patients and physicians alike describing them as life-changing. There is no cure for cystic fibrosis and CFTR modulator drugs are the closest thing that many people living with cystic fibrosis have to a cure.
Yet Canada’s system of approving and reimbursing drugs for rare diseases is cumbersome and convoluted and many people living with cystic fibrosis in Canada have been unable to access these new, life-changing drugs. Cystic Fibrosis Canada is working to change this.
Kalydeco, the first generation modulator, was approved by Health Canada for treatment of a specific mutation in 2012 and for additional mutations in 2014. Altogether Kalydeco could treat about 4% of the cystic fibrosis population in Canada. While Kalydeco is accessible to some who need it across Canada, Canadians still don’t have public access to Kalydeco for all the mutations and age groups it can treat.
The second-generation modulators, Orkambi and Symdeko, were approved by Health Canada in 2016 and 2018, respectively. They could treat about 50% of the Canadian cystic fibrosis population that carry two copies of the most common CF mutation, delF508.
Orkambi is publicly available in several provinces, but access is extremely limited. Symdeko is only available through some private insurance plans, as the manufacturer has thus far declined to put the medicine through Canada’s Health Technology Assessment bodies, the Canadian Agency for Drugs and Technologies in Health (CADTH) and its Quebec counterpart, INESSS, which our public drug programs look to for non-binding recommendations to inform reimbursement decisions.
The third-generation of modulators, Trikafta, is a new transformational drug that can treat up to 90% of Canadians with cystic fibrosis. Trikafta was approved for sale in Canada on Friday, June 18th, 2021, by Health Canada for people aged 12 and up with cystic fibrosis and at least one F508del mutation. Every province and territory in Canada has since indicated they have or will fund Trikafta for eligible people living with cystic fibrosis. Unfortunately, it is not accessible to all who can benefit from it, and therefore our advocacy work continues
An important part of Cystic Fibrosis Canada’s advocacy work is navigating and working with different parts of Canada’s complex drug review and reimbursement system to improve access to medicines for future cystic fibrosis therapies. Regulatory bodies our work involves:
Our efforts to remove barriers to access can include influencing policy through government relations efforts and driving system change alongside partners. Ongoing work includes pushing for the implementation of a national Rare Disease Strategy.
Approximately 5% of the Canadian CF population does not have genetic mutations addressed by modulator therapies, meaning there are no modulators that can treat their disease. A number of other therapies that might help those who do not benefit from modulators are in early stages of development; these drugs use other, often genetic, approaches to correcting the defect in the CF protein. Cystic Fibrosis Canada continues to advance research into innovative, therapeutic treatments to ensure no person is left behind when it comes to life-changing therapies and treatments.
Additionally, while new modulators will help a large portion of the Canadian CF population, modulators do not constitute a cure. Even for people on modulators, the disease will continue to progress in ways that remain largely unpredictable. Until we find a cure, all individuals with CF will still require access to symptom management medicines and specialized clinical care.
Cystic Fibrosis Canada remains committed to ending CF for everyone. Find out more about our work.