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Volunteer Advocate
cf insights cf insights

Advocating for Cystic Fibrosis

Cystic fibrosis (CF) is the most common fatal genetic disease affecting Canadian children and young adults. Currently, there is no cure. Approximately, one in every 3,600 children born in Canada has CF, making CF a rare – or orphan – disease, yet Canada doesn’t formally recognize rare diseases. So what does this mean?

In Europe, a disease is considered rare when it affects less than 1 in 2000 people. The USA considers diseases rare when they affect fewer than 200,000. There are over 6000 known rare diseases and 80% of rare diseases have identified genetic origins. Yet, after over a decade of trying, Canadian governments have yet to adopt a definition – let alone an approach – to improve access to medications for Canadians with rare diseases.

Unlike many rare diseases, Canadians with CF have access to high-quality care: more than 4,100 Canadian children, adolescents, and adults attend specialized CF clinics. We also understand a lot of the science behind CF and have a body of high quality information about CF, which helps clinicians provide world-class care and helps researchers develop cutting-edge treatments. In fact, Canadians with CF live 10 years longer than their American counterparts.

Never has the future looked so bright for CF: with 121 treatments in the drug development pipeline, 52 of which target the basic defect of CF, most people with CF can expect to have some kind of disease modifying therapy targeted to their genetic make-up in the next decade. However, these therapies are only good if people can access them. Therapies like Kalydeco and Orkambi are publicly available in the USA, but it took battles at legislatures across the country to get Kalydeco publicly covered. We continue to face an uphill battle to get just one province to cover Orkambi, let alone all provinces and territories.

Without a strategy for rare diseases, CF meds will continue to be evaluated like drugs for non-rare diseases, even though we have a smaller population, which makes it difficult to conduct large-scale, clinical trials that are considered the standard of evidence by Canada’s bodies that make recommendations on whether to publicly reimburse or not reimburse medicines. These bodies – CADTH and INESSS – will not accept any other data, even real world data collected after people have been on the drug for some time.

There has been a lot of talk about how best to manage medications for rare diseases, but not as much action. We can learn from the work that Alberta and Ontario has done, but the problem starts with our pan-Canadian health technology evaluators and ends with the provinces and territories, not the other way around. It’s time for CADTH and INESSS to put their money where their mouth is, to develop and implement a strategy that will make drugs for rare diseases accessible.