Updates on Trikafta for 2-5 Years olds, Rare Mutations and the Drugs for Rare Disease Strategy  

JULY 20, 2023

Last month Cystic Fibrosis Canada held a National Advocacy Briefing to discuss access to Trikafta for 2–5-year-olds and for those with rare mutations, as well as the latest on Canada’s strategy for drugs for rare diseases. Here is what you need to know. 

Trikafta: 

In Canada Trikafta is currently accessible to those with at least one F508del gene mutation, the most common CF mutation, aged six and older. However, we know that there are some people who are still unable to access Trikafta due to access issues around affordability and insurance.  Access for the 2–5-year age group is currently under review. There are also some people in Canada with rare mutations that could benefit from the drug, but do not yet have access.  

Affordability issues:

There are inequities across the country when it comes to the cost of accessing therapies. Currently, six jurisdictions in Canada require individuals to pay a deducible to access Trikafta. Manitoba, Ontario and New Brunswick have the highest public drug program deductibles – while there are some provinces in which residents can access the drug for no cost. Recently, the Cystic Fibrosis Canada team held events in Manitoba and Ontario to discuss the financial burden individuals face while accessing the medications they need in these provinces and shine a light on the inequities. We continue to advocate for fair access to rare disease medications, the federal government is aware of these inequities and some of the rare disease funding has been indicated to fix the postal code lottery in the cost of medications. 

Updates on Trikafta 2-5 years old:

In April 2023, the United States approved Trikafta for children ages two through five years with certain CF mutations. Health Canada is still in the process of reviewing the safety and efficacy for 2–5-year-olds. 

In May, Cystic Fibrosis Canada held focus groups with several parents to 2-5 year olds with cystic fibrosis who stand to benefit from Trikafta. Using information gathered during these sessions, Cystic Fibrosis Canada sent a submission to the Canadian Agency for Drugs and Technologies in Health (CADTH) and Institut national d’excellence en santé et services sociaux (INESSS). Along with Cystic Fibrosis Canada’s submission, our Health Advisory Council, Clinical Trials Network and an independent group of cystic fibrosis clinicians also provided submissions to CADTH recommending that Canada’s public drug programs fund Trikafta for 2-5 year olds. Cystic Fibrosis Canada and Quebec clinicians provided a submission to the  Institut national d’excellence en santé et services sociaux INESSS to support the recommendation of funding for Trikafta for 2-5year olds.  Cystic Fibrosis Canada continues to monitor for a timeline for BC Pharmacare’s review. 

A challenge for this age group is the inability to test children for a response to this medication. Given these limitations, the Health Canada review will be focused on safety, and clinicians will be looking for delayed disease progression and improved quality of life for children and caregivers. These are the key areas that CF Canada’s submissions focused on. We are grateful to the families who participated in feedback sessions to help formulate these submissions.  

Our anticipated timeline: 

Mid-August – mid-September: Health Canada approval 

Late Sept/late October – CADTH draft recommendation 

Late October – Patient group, clinician and researcher feedback due to CADTH 

Early to mid-November – CADTH final recommendation / INESSS (Quebec) recommendation to Health Minister  

Late November and on – Jurisdictional reimbursement  

Access to Trikafta for those with rare mutations:

In the United States, Trikafta has been approved for 177 CF mutations, in Canada, only 1 mutation (at least one copy of delF508) has been approved. Cystic Fibrosis Canada has long advocated for jurisdictions to make Trikafta available for all who stand to benefit, and we continue to advocate for broader access to this potentially life-changing drug. We are calling on all drug plans to work with the manufacturer to provide access to those with rare mutations that respond to Trikafta.