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Rare Disease Strategy

Nation-Wide strategy for rare disease drugs

For years, Cystic Fibrosis Canada and the Canadian CF community have advocated for access to life-changing CF drugs. Because we did not have a nation-wide approach to funding drugs for rare diseases, like so many other countries do, we have had to advocate relentlessly for access, every step of the way. Without a nation-wide strategy, access to potentially life-changing medications has been delayed, expensive and challenging.  

In 2019, the Federal government committed to fund a nation-wide drugs for rare diseases strategy. This strategy could help Canadians with rare diseases better access the medications they need and would mean that Canada would develop a drug review process that fairly assesses drugs for rare diseases, including CF drugs. It also means that Canada would become a more favourable country for access to rare disease medicines.  

It took four years with little movement on this promise, advocacy efforts from Cystic Fibrosis Canada the CF community and the broader rare disease community, until the federal government provided the funding it promised, but without a nation-wide framework.

Update from 2023 

On March 22, 2023, the federal government announced its approach to the 2019 commitment to improve drug access for Canadians with a rare disease. The government committed up to $1.5 billion over three years in support of the first ever National Strategy for Drugs for Rare Diseases. Much of the funding will go to the provinces and territories to support improved access to emerging and existing rare disease drugs through bi-lateral, individualized agreements.  

While this news appears promising to the broader rare disease community, it is unclear what this means specifically for the CF community, as some of the funding will be to create infrastructure for rare disease, infrastructure that the CF community has through Cystic Fibrosis Canada, including centres of expertise, registries, clinical trial network and research. For this to be a true success story, the provinces and territories will need to act swiftly and buy in from all levels of government will be required. That includes providing their own funding to advance this important work.  

Cystic Fibrosis Canada has done this difficult, but important work before, and will continue to work hard to ensure our community gets the fair and equitable access to CF drugs to live healthier lives. 

Read Cystic Fibrosis Canada’s response to the National Strategy for Drugs for Rare Diseases announcement here. 

History of delayed access for drugs for cystic fibrosis  

Kalydeco was approved by Health Canada for the G551D mutation in 2012; approval of the additional 9 mutations was received on June 6, 2014, and approval for R117H was received on March 13, 2015. While Kalydeco is accessible to some who need it, Canadians still don’t have public access to Kalydeco for all the mutations and age groups it can treat.

Orkambi, which treats the basic defect in CF, was approved for use in Canada in January 2016. Public access was extremely limited until 2021 when the criteria was expanded in many provinces. However, Canadians still don’t have unrestricted access in every province and territory in Canada.

Symdeko was approved by Health Canada on June 27, 2018 in people ages 12 and older who have two copies of the F508del mutation in the cystic fibrosis transmembrane (CFTR)  or those with one copy of the F508del mutation and have one of the following mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene: P67L, D110H, R117C, L206W, R352Q, A455E, D579G, 711+3A→G, S945L, S977F, R1070W, D1152H, 2789+5G→A, 3272-26A→G, and 3849+10kbC→T. Currently the drug is only available through private insurance as the manufacturer, Vertex Pharmaceuticals, has thus far declined to put the medicine through Canada’s drug reimbursement reviews.

The third-generation of modulators, Trikafta, is a new transformational drug that can treat up to 90% of Canadians with cystic fibrosis. As of September 2022, Trikafta is funded by every province and territory in Canada for children and adults aged six and older with at least one F508del gene mutation. While this is progress, unfortunately, many Canadians are experiencing access issues navigating between private and public insurance plans, some are having to remove themselves from private drug plans and others are experiencing high deductibles. Others may run into lifetime maximums quickly, among other issuesThe drug is not accessible to all who can benefit from it, and therefore our advocacy work continues.