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Volunteer Advocate

Rare Disease Strategy

Canada Needs a Rare Disease Strategy

People in Canada living with rare diseases, like cystic fibrosis, are all too familiar with the ongoing issue of access to life changing medicines. Cystic Fibrosis Canada has long pushed and advocated for better access to medicines like Orkambi and Kalydeco, and we’ve determined enough is enough – we’re going to the heart of the issue. We’re calling for the creation of a rare disease strategy in which all Canadian provinces and territories work with the federal government (pan-Canadian) to fairly assess and improve access to drugs for rare diseases, including cystic fibrosis (CF).

Canada is one of the few developed countries without a rare disease strategy that guides decisions about access to drugs for rare diseases. Canadian drug review bodies such as the Canadian Agency for Drugs Technologies in Health (CADTH) and the Québec Institut national d'excellence en santé et services sociaux (INESSS), do not have processes to fairly assess drugs for rare diseases, nor do they have a common definition of rare diseases.

As a result, there has been limited and delayed access to some rare disease drugs in Canada, which is the case for CF disease-modifying drugs such as Kalydeco, Orkambi, Symdeko and Trikafta. These are examples of how our drug review and reimbursement systems fail Canadians with CF.

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 people with the G551D mutation have had limited public access in most provinces since 2014, those with the additional mutations still wait for access, four to five years after approval.

Orkambi, which treats the basic defect in CF, was approved for use in Canada in January 2016. Almost four years later only three provinces – Saskatchewan, Alberta, and Ontario – have extremely limited public access mechanisms, which apply to children with CF only. Since the implementation of these criteria in February 2019, one child with CF has accessed the drug in Canada, to the best of CF Canada’s knowledge. Meanwhile, the drug is publicly covered in: U.S.A, Australia, England, Scotland, Ireland, Sweden, Denmark, France, Germany, Austria, Italy, Netherlands, and Luxemborg.

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.

This is both a promising and heartbreaking time for Canadians with CF and their loved ones. 

To that point, on October 21,2019 the Food and Drug Administration (FDA) in the U.S. approved Trikafta, a highly effective disease-modifying medication that can treat up to 90% of the CF population, for sale in the U.S. As a first step in gaining access in Canada, the manufacturer, Vertex Pharmaceuticals, must first apply for Health Canada approval of the drug.

Cystic Fibrosis Canada has asked the manufacturer to do what they can to expedite access, starting with an application to fast-track approval by Health Canada. Until the manufacturer applies for Health Canada approval, the drug cannot be sold in Canada, which means our governments can’t provide public coverage through common access channels.

Additionally, Cystic Fibrosis Canada does not lose sight of that fact that, while new medications have the potential to improve the outcomes for many, they will unfortunately not be beneficial for the entire CF population. While 90% of the CF population has potential to be treated with newly developed disease-modifying medications, that is not a solution for the remaining 10% who are not able/eligible to receive these treatments. CF Canada represents all those living with cystic fibrosis and continues to advocate for and fund ongoing research into new treatments, medications, therapies and ultimately – to find a cure or control for all people living with CF.

Creating a rare disease strategy would mean that Canada would develop a drug review process that fairly assesses drugs for rare diseases, including some CF drugs.It also means that Canada will become a more favourable country for access to rare disease medicines.

Cystic Fibrosis Canada is calling for the creation of a pan-Canadian rare disease strategy, a strategy that is developed and built in collaboration between the federal and provincial governments.  This is different than suggesting the development of a national strategy where responsibility only lies with the federal government. We need all governments to work together to build a better system to improve access to drugs for rare diseases.

Cystic Fibrosis Canada believes that people with CF and their loved ones have waited long enough for equitable access to medicine. CF is a progressive disease and people with CF do not have time to wait. We need a pan-Canadian rare disease strategy now.

For information contact

The Canadian Government’s 2019 budget included implementing a strategy for high cost drugs for rare diseases. In order to meet their proposed timeline, Health Canada is currently seeking input from the rare disease community on their proposal. The Cystic Fibrosis Canada advocacy team, members of the National Advocacy Network and members of the CF community participated in the government’s consultations for the development of a rare disease strategy, recently renamed to a strategy for high cost drugs. We also provided a submission to their consultations.

View our infographic to understand what is at stake for rare diseases in the 2019 Canadian federal election: