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If Canada refuses to cover CFTR Modulators, Their Standard of Care will Suffer



Canadian cystic fibrosis (CF) care has long been a worldwide standard in the fight against the fatal illness. The effects of the disease differ from person to person; it ravages the body from the inside out, leading to premature death amongst its sufferers. The disease is most often seen in European descendants, although it does also affect people of color. Due to migratory and immigration patterns, the illness has spread to all corners of the globe. For this reason cystic fibrosis care centers are present all over, from Australia to Germany, and from the United States and it’s northern neighbor – Canada. Certainly many countries do lack in care for CF, due to low prevalence of the CF genes in their populations, and for these sufferers it is difficult to access care. However, due to many factors, CF patients in Canada survive longer than in any other country. A beacon in socialized medicine for many decades, Canada boasts an impressive median age of survival for CF patients. In 2017, it was 52.3 years old. However, at the moment a great discrepancy exists between Canada and 18 other countries, including the US; they do not fund the disease modulators that are shown to extend survival and improve quality of life. Modulators are the first treatment to target the root cause of cystic fibrosis. If this disparity is not rectified soon, Canada is doomed to fall behind in CF care, leaving thousands of patients to live out their shortened life-spans in suffering while the rest of the world’s patients get a new lease on life.

A beacon in socialized medicine for many decades, Canada boasts an impressive median age of survival for CF patients. In 2017, it was 52.3 years old. However, at the moment a great discrepancy exists between Canada and 18 other countries, including the US; they do not fund the disease modulators that are shown to extend survival and improve quality of life.

It must be noted, before assumptions are made, that the issue in Canada prohibiting funding life-saving modulators is not one based on socialized (or single-payer) healthcare. Over 17 countries with socialized healthcare fund modulators, notably in Europe and also Australia; The issue is solely a Canadian one. The first modulator to come to market, Kalydeco, which works on the G551D gene mutation, is funded in this country. The successors, Orkambi and Symdeko, however, are not. Orkambi has been approved in Canada since early 2017, and Symdeko since early 2019. This means that the drug may be used in Canada by patients. Despite that, it does not mean that every single eligible patient is able to access and use it. This unfortunate circumstance is because there are several steps that must be followed to allow a drug to be taken by Canadian patients. The first is a submission by the pharmaceutical company to Canada, to pass Health Canada’s safety tests. Once a drug passes, it is licensed to be used in the country. However, this does not mean it is automatically funded on the public drug benefit program. For that to happen, another governing body must review the drug’s efficacy and cost-benefit. This is where Canadians are being robbed of years of their lives. 


These governing bodies (there is one for the province of Quebec, and another for the rest of the country) have, several times, decided that funding Orkambi and Symdeko is not in the best interest of the governments.  There are no CF physicians on these boards, and so the importance of getting these medications to patients is not properly understood by those making the decision. If it were, the fact that stability can be granted in an otherwise complete degenerative disease would most certainly be sufficient evidence to grant approval for funding. Medicine seeks to find the best outcome for patients, and in CF that would mean, at the least, slowing the degeneration that characterizes the lungs of patients. 

However disappointing these decisions may be (for they have been consulted upon several times), the devastation of not allowing the newest and most effective modulator into this country would be much worse. As previously mentioned, Canada’s survival rate for CF patients currently sits at a decade longer than their American counterparts. The disparity will most definitely swing in the other direction soon if Canadian patients are still denied access to life-saving modulators. 


Legislation has recently changed in Canada that has created barriers for pharmaceutical companies trying to bring novel therapies into the country. The process of creating an application for Health Canada (similar to the FDA in the US) has become riddled with hurdles, that some say have been put into place solely to discourage pharmaceutical companies from submitting. The change has discouraged companies like Vertex from wanting to submit new drugs, such as Trikfata, through the approval process. It is apparently now more likely than before to have a drug denied after the application process. So not only do the companies have to submit more paperwork, evidence, and pay more to get drugs into the “door” for approval, after all this the likelihood of getting a medication approved is less than it has ever been. 


Even if Vertex did submit Trikafta for Health Canada approval, if it was accepted, this would not be the final step in getting the life-changing medication into the hands of those who need it. The same governing bodies that have continuously denied Orkambi and Symdeko will have to review the medication’s efficacy versus it’s cost, deciding whether to fund it on the public formulary or not. When a medication is funded on the formulary this means that anyone can access it (when prescribed by a doctor), no matter their income, insurance coverage, or job. 

This public drug coverage is part of what makes Canada such an appealing and happy place to live. The country is continually ranked among the best countries around the world to call home, yet for CF patients that could very well change within the next few years. If these modulators do not get funded, then patients in this country will no longer be amongst those who live the longest and fare the best given their fatal diagnosis.

This public drug coverage is part of what makes Canada such an appealing and happy place to live. The country is continually ranked among the best countries around the world to call home, yet for CF patients that could very well change within the next few years. If these modulators do not get funded, then patients in this country will no longer be amongst those who live the longest and fare the best given their fatal diagnosis. Such a disparate future for Canadian children and young adults should bring shame to anyone involved in the process. 


Canadian lawmakers, politicians, and Vertex must come together to work for the 4300 patients in this country who could benefit from taking these modulators, and even live longer lives. Each day that these medications are denied to Canadian sufferers is a day that irreversible damage is occurring in the lungs of 4300 people who are being held hostage by forces out of their control. 


If you want to help Canadians access these modulators, please use the hashtag #CFCantWait and contact Chelsea Gagnon who can help you send letters to lawmakers to help plead this vital case. 


 

About the Author Chelsea Gagnon is a 30-year-old CF patient from Montreal, Canada. She graduated from elementary education at McGill University in 2014, after dropping several semesters due to her failing health. Her full-time job is keeping herself alive, and she supplements her time by being a cat mom to 2 beautiful cats. She spends as much time as she can advocating for CF patients, and tends to fight for general human rights in her downtime. You can find her standing up to systematic oppression, or laying down to take daily naps.

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