Especially in smaller provinces, it’s virtually impossible to predictably manage the affordability and accessibility of drugs necessary to treat rare disease

Around the world, rare diseases are becoming easier to diagnose and treat courtesy of the widespread adoption of personalized medicine, the growing capacity of genomics and the advent of “big data.” These developments are enabling our industry and governments to understand more accurately the incidence of rare diseases, their impact on patients, health care systems and society.


In Canada, patients with rare diseases face the same issues as elsewhere, but with a few added complications.  Unlike other countries, Canada has not agreed on a common definition of rare diseases.


In addition, there is a void of national information about the types of rare diseases and the size of the populations affected, which creates challenges for provinces and territories that deliver health care in Canada. Especially in smaller provinces, it’s virtually impossible to predictably manage the affordability and accessibility of drugs necessary to treat rare diseases without this knowledge.


Earlier this year, a federal parliamentary committee released a comprehensive report on the barriers faced by patients with rare diseases in accessing diagnosis, treatment and drugs. In Budget 2019, the federal government committed to working with provinces, territories and other partners to co-develop a plan to ensure that patients with rare diseases have better and more consistent coverage of treatments.


Canada’s innovative pharmaceutical industry recognizes that all governments, and people with rare diseases, are challenged by affordability and accessibility, and we want to see our pharmaceutical investments produce better results for patients. With this in mind, we created a working group of Canadian industry representatives to develop policy options to bring to the table.  We see this work as an important catalyst for industry collaboration with governments and other stakeholders.


To address the issue of affordability, we need an understanding of the scope of the rare disease landscape. This starts with a common definition of what constitutes a rare disease in Canada, as well as criteria for product eligibility. We can then use this common understanding to build critical knowledge infrastructure through a Rare Disease Patient Registry and Clinical Network -something in which our industry is ready to invest to help get off the ground.


A partnership among all players in this space is key to ensuring patients have access to the drugs they need to survive. If we can work together to establish a national risk-sharing model among the pharmaceutical industry, all governments and private insurers, we can help give Canadians with a rare disease consistent and timely coverage, regardless of their income, age or postal code.


Such an approach would support the measurement of fair value for patients and payers through ongoing evidence-based reviews of the contribution made by medicines that treat rare diseases.  Another important result would be fair and competitive prices for patients and payers through a risk-sharing model, and funding and greater budget predictability for both public drug plans and private insurers.


This pan-Canadian approach to market policy could also represent an important starting point for a future system of national pharmacare that could be scaled over time to include a broader set of medicines.


With nearly all federal political parties committing to a rare disease strategy in one form or another, it is clear that there is political will to address this long-time issue.  As an industry, we are eager to partner with governments at the federal, provincial and territorial levels to ensure Canadians get the medicines they need, when they need them, while supporting sustainability of our health care systems.