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OPEN LETTER to elected officials and policymakers in Canada’s drug review and reimbursement system from cystic fibrosis clinicians

September 17, 2020

More than 70 Canadian cystic fibrosis clinicians are imploring the government to fix Canada’s broken drug review and reimbursement system that is impacting access to life-changing drugs, specifically Trikafta. This show of public support from the medical community, facilitated by Cystic Fibrosis Canada, follows the recent release of a Canadian study demonstrating that early access to Trikafta would result in dramatic health benefits for up to 90% of Canadians living with CF and that further delays to access will tragically result in preventable deaths. Read on for the open letter.

Dear Canadian Government officials,

Based on the Canadian Cystic Fibrosis Registry, a baby born with cystic fibrosis (CF) in Canada in 2018 has only a 50% chance of living to 52.1 years and will endure significant health-related disabilities.

The impact for people living with cystic fibrosis is significant. They must spend at least two hours daily doing treatment, taking medication, wearing masks, using inhalers, sanitizing constantly and frequently missing school and work while spending time in the hospital. There is also the prospect of requiring a future lung transplant.

There is a way that we could alleviate this suffering, however, Canada’s drug review and reimbursement system stands in the way.

Trikafta is a breakthrough drug, with the potential to treat 90% of people living with cystic fibrosis. Canadian research recently published in the Journal of Cystic Fibrosis demonstrates that access to this medication in 2021 would result in profound health benefits for people living with cystic fibrosis. By 2030, Trikafta could reduce the number of people living with severe lung disease by 60% and reduce the number of deaths by 15%. The research projects a significantly slower progression with an 18% increase in people with mild lung disease and 19% fewer hospitalizations or home intravenous antibiotics for pulmonary infection exacerbations. The estimated median age of survival for a child born with cystic fibrosis would increase by 9.2 years.

Instead, we wait. Trikafta was fast-tracked for access in the U.S. and Europe. It is not available in Canada.

The disease is devastating and difficult to treat. Since the Canadian discovery of the cystic fibrosis gene, cystic fibrosis clinicians and scientists have made a significant difference in our ability to help people with this disease. But nothing has come close in effectiveness to the breakthrough drug Trikafta.

It is heartbreaking to have to tell patients, again and again, that the drug that could dramatically improve and extend their lives is not available in Canada. It’s even worse when these people discover that a major hurdle is a broken system that does not prioritize precision medicines for rare diseases like cystic fibrosis -- a system so complicated and unfavorable to innovation that this drug’s manufacturer has not even submitted it for approval in Canada.

Time is of the essence. As the study’s senior author Dr. Anne Stephenson said, “delays in access to Trikafta will come at significant cost, in terms of health outcomes, health care costs and ultimately lost lives.” As clinicians, we call on you today to fix Canada’s broken drug review and reimbursement system. Federal policies have so far impeded access to precision medicines like Trikafta.

• Ensure that the new guidelines for the Patented Medicine Prices Review Board include a pathway for precision medicines like Trikafta.
• Urge the pan-Canadian Pharmaceutical Alliance to negotiate openly and fairly with manufacturers.
• Establish a “fast-track” approval mechanism for orphan medications for rare diseases, specifically for Trikafta.

The research is conclusive. Our patients desperately need this drug. Canada, what are you waiting for?

Yours sincerely,

Find out more about actions you can take to help advocate for Trikafta.