The Research Pulse

Canada Needs Orphan Diseases Policy

I read with much interest an article on orphan diseases in the GLOBE AND MAIL on April 5.  This article followed a CBC Radio segment of The Current addressing the ordeals patients with rare diseases have, firstly, in obtaining a diagnosis, and then in trying to get insurance coverage for extremely expensive medications to treat their disease.

We recently completed a growth disorder study for a leading pharma client.  Most growth disorders are so rare that there are no patient advocacies or charities dedicated to them in Canada.  Fortunately there is a foundation called the Magic Foundation in the US that is dedicated to advocacy for patients with rare growth disorders and they were kind enough to confidentially invite their Canadian members to participate in our study.

Obviously lack of awareness is partly a function of the rarity of these disorders but there are countries with smaller populations than Canada doing far better at supporting patients with orphan diseases. It’s ironic that we Canadians believe we are a compassionate people and, despite its well publicized challenges, we still firmly wear our universal health care system as a badge of honour, yet we allow these most vulnerable individuals to slip through the cracks.  It is essentially a perfect storm for these individuals and their families.  First they must suffer with often debilitating symptoms, then it can take months or even years for a correct diagnosis – often after enduring several misdiagnoses and finally they have to do to an exhaustive battle trying to source funding for unaffordable medicines and treatments.  I can’t imagine the shock in learning my child had a lifelong incurable disease and then subsequently learning that there is no funding for her medication that would help her lead a normal life but I couldn’t possibly afford it.  

The Globe article accurately points out that Canada is one of the few developed countries with no federal policy or funding for orphan diseases … something that continues to be lacking due to political wrangling between provinces and the federal government.   It is mind boggling the difference in orphan drug funding between provinces.

Slow progress is being made in some provinces but this has dragged on for too long.  It’s time for a federal orphan drug policy in Canada and the only way it will happen is if all stakeholders including federal and provincial governments, pharmas, patient advocacies and the medical community collaborate.