This article originally appeared in The Hills Times on May 6, 2024
Have you ever tried to fix something without the right tool? If you use a hammer when you really need a screwdriver, it’s hard and the result won’t be the best.
That’s the type of feeling doctors have when they treat a disease, and they don’t have access to the right tools. This is especially true in treating rare diseases, where treatment options are often more limited and can be difficult to access given how long governments take to reimburse them.
Consider a rare disease I know very well in my practice: pulmonary arterial hypertension, or PAH. It’s a serious, progressive, and often fatal rare lung disease characterized by high blood pressure in the blood vessels of the lungs. This causes stress on the right side of the heart, specifically the right ventricle, resulting in heart failure.
One patient I have treated for PAH is Nicole, who was diagnosed when she was an otherwise healthy 39-year-old primary school teacher and mother of two young daughters. The disease and side effects from the medicines she had to take meant she could no longer do many of the things she once enjoyed, including having to stop teaching after 12 years in the profession.
Nicole is not alone. A new survey by the Pulmonary Hypertension Association of Canada (PHA Canada) shows that just one quarter (28 per cent) of people with PAH are still employed, and many of those have had to reduce their work hours. Some family caregivers devote more than 40 hours a week to care for someone with PAH. Strikingly, nine out of 10 people with PAH reported having impaired mental or emotional well-being, all of which results in much worse quality of life.
PAH used to be a rapidly fatal illness, with patients living just two to three years after diagnosis. We are fortunate that significant advances have been made in the diagnosis and treatment of PAH, with many patients living better and longer lives. However, Canadians with PAH still suffer daily with physical symptoms, psychosocial impacts, poor quality of life and a very uncertain future.
World Pulmonary Hypertension Day on May 5 emphasized the global importance of raising awareness of the burden of this condition with the goal of improving care and treatments for Canadians living with PAH.
I and my medical colleagues are waiting for the many new treatments in development to give us additional tools to treat Nicole and others with rare conditions. But we can only do that if our health system makes them available to patients through our public drug plans soon after they have been approved by Health Canada.
Most industrialized nations have recognized the seriousness and special aspects of the more than 7,000 known rare diseases, like PAH, and are clearly ahead of Canada in directly supporting initiatives to improve access to care, including diagnosis and medical treatment.
That’s why the federal government five years ago promised funding for rare disease treatments. It formalized this commitment in March last year, committing to providing $1.5-billion over three years to help improve access to these medicines. However, no new money has been spent yet so patients can actually benefit from the new treatments.
I join PHA Canada in calling on the federal government to prioritize the health of Canadians affected by PAH and other rare diseases, starting with the release of the promised federal funds to the provinces and territories for medicines to treat these conditions.
Canadian doctors need access to the right tools to help rare disease patients.
Sanjay Mehta, MD, FRCPC, FCCP, is a professor of medicine at the University of Western Ontario and director of the Southwest Ontario Pulmonary Hypertension Clinic at the London Health Sciences Centre in London, Ont. He is also a founding member of PHA Canada, having served on the board of directors since 2008.
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