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Trikafta is a triple combination precision medicine (ivacaftor, tezacaftor and elexacaftor). A disease-modifying drug, Trikafta treats the most common cystic fibrosis (CF) mutation, meaning it could potentially treat 90% of people living with CF. It is not yet available in Canada.
In the United States of America, Trikafta was approved in October 2019 after the FDA designated it a breakthrough therapy and fast-tracked its application.
Access to life-changing medicines, like Trikafta, is one of Cystic Fibrosis Canada’s most important priorities and we have been working hard to bring this medication to Canada.
An August 2020 study demonstrates that early access to Trikafta would result in life-changing health improvements. The study found that introduction of Trikafta in 2021 would result in an increase of 9.2 years in the estimated median age of survival for a child born with CF in 2020. By 2030, there would be 60% fewer people living with severe lung disease due to cystic fibrosis and 15% fewer deaths. Read more about this research.
Why isn’t Trikafta available in Canada?
The manufacturer of Trikafta, Vertex Pharmaceuticals, has not yet submitted this medicine to Health Canada for approval. Cystic Fibrosis Canada has been advocating hard for them to do so, but the company has pointed to concerns about the regulatory changes Canada is poised to adopt. As of today* Trikafta is only available in Canada through Health Canada’s Special Access Programme.
Who can potentially benefit from Trikafta?
In the U.S., Trikafta is approved for use in anyone over the age of 12 with one copy of the F508del mutation, regardless of their second mutation. In Canada, that would represent approximately 66% of people with cystic fibrosis. The hope is that it will also be indicated for younger children if the necessary clinical trials prove it safe and effective in this age group – meaning that it could potentially benefit 90% of people living with CF in Canada.
Is Trikafta more effective than existing cystic fibrosis medications?
Yes, and significantly so. In clinical trials of the triple combination therapy, people with two copies of the F508del mutation had a 10% increase in lung function compared to treatment with Symdeko (that already provides a 4% increase over baseline), and people with a single copy of F508del had more than a 14% increase in lung function compared to treatment with the placebo. On average, Trikafta use leads to a 14% increase in lung function over the untreated baseline.
How much does it cost?
In the U.S., the list price is USD $311,000 per year. In order to get to a Canadian price, the manufacturer must first apply for Health Canada approval of the drug, after which Canada’s drug review and reimbursement processes kick in. The PMPRB (Patented Medicine Prices Review Board) will determine the maximum price a company can charge for a new patented sold in Canada. What public and private insurers pay is negotiated with the company. A body called the pCPA (pan-Canadian Pharmaceutical Alliance) negotiates on behalf of the provincial, territorial and federal programs the price that will actually be paid. Learn more about Canada’s drug review process.
Cystic Fibrosis Canada is advocating strongly for a drug pricing system that enables faster access to Trikafta, and broader, less restrictive access to other cystic fibrosis treatments. Find out more about our advocacy work and how you can help.
Are there any other therapies like this?
Trikafta is the first triple combination therapy to be approved in the U.S. At this time at least three companies (AbbVie, Proteostasis Therapeutics and Vertex Pharmaceuticals) are developing other potential triple combination therapies.
What can I do to advocate for access to Trikafta?
Stand up and make your voice heard. Meet with your elected officials. Demand that the federal government halt the changes to the PMPRB. Join our National Advocacy Network. Share your access stories on social media using #cfcantwait, and tag your elected officials.
Write to and meet with your provincial and federal elected officials. CF Canada’s advocacy toolkit will help you have these conversations.
Visit our ‘How to Take Action’ webpage for ways to support our current advocacy work.
What is Cystic Fibrosis Canada doing to advocate for access to Trikafta?
Trikafta represents the single biggest advancement in treating cystic fibrosis in the history of the disease and has been proven to significantly improve health outcomes for 90% of CF patients. At this critical time, access to life-changing CF medications, like Trikafta, is one of our most important priorities.
Our CEO, our Chief Scientific Officer, our program team, volunteers and Board members have been and are working hard to ensure people living with CF in Canada have access to these medications.
Since the fall of 2019, we have advocated for changes to the Patented Medicine Prices Review Board (PMPRB) and met with their senior leadership, encouraged Health Canada and Vertex to create a Special Access Programme for Trikafta, appealed to Vertex to submit Trikafta for Health Canada approval and launched a media and social media campaign #cfcantwait. Our advocacy work continues and you can find the latest updates on the CF Canada blog. Please see a timeline of these and other advocacy activities.
Find out more about Cystic Fibrosis Canada’s work to gain better access to life-changing medications in Canada, including an advocacy toolkit that you can use.
*Last updated March 2020