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Why Ontario Needs a Rare Disease Strategy


Canada’s rare disease patients face many challenges, including long diagnosis journeys and barriers to treatment access — and it’s time to change that. 

Rare diseases affect 1 in 12 Canadians — around three million Canadians. Yet patients with rare diseases are often unable to receive timely and accurate diagnoses and equitable access to drugs. A rare disease strategy for Ontario would not only improve the health and quality of life for patients with rare diseases and their families, it would also support Ontario’s diverse and vibrant life sciences sector. To get there, we need to realize the importance of investing in innovation and research to secure our economic growth and improved health for years to come.

Envisioning Ontario’s rare disease strategy 

In 2021, Life Sciences Ontario outlined our vision for Ontario’s rare disease strategy in our report, “Cast Our Pebble Into the Pond.” Our vision begins with an understanding that rare diseases cannot be managed under the same policies and regulations that currently govern our health care system, which includes clinical trials, regulatory approvals, emergency drug access, and health technology assessments. The federal patented drug pricing policy also creates barriers for access to drugs that treat rare diseases, especially for new and innovative drugs.  

To address these barriers, Ontario’s rare disease strategy must be based on a flexible, patient-centered system that will accelerate both diagnosis and patient access to new treatments. Increased funding for health research and innovation is a key pillar of this strategy. Canada is already home to several success stories in health innovation, such as the discoveries of insulin and the gene that causes cystic fibrosis. We must make a collective and deliberate decision to invest in innovation and research in rare diseases as an economic development strategy for the long term. 

Next steps for building Ontario’s rare disease strategy

As Canada’s most populous province, Ontario must align its strategy with other rare disease initiatives, such as Quebec’s rare disease policy, as well as the federal government’s commitment toward $500 million in funding per year for drugs that treat rare diseases from 2022 to 2023. Ontario’s rare disease strategy should also build upon work completed by previous governments, such as the Rare Diseases Working Group Report from 2017. The Ontario government should engage leading stakeholders in the rare disease space, including RAREi, advocacy organizations (like CORD), patient groups, companies, and key federal and provincial government ministries. Now is the time to build a rare disease strategy that will improve the lives of millions of Canadians living with rare diseases and support our economic growth. 

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