Advocacy for rare diseases in Canada

For many years, patient organizations and other stakeholders have worked to improve the lives of Canadians living with a rare disease and their families.

The Canadian Organization for Rare Disorders (CORD) has advocated for a Canadian Rare Disease Strategy (May 2015). However, it was not adopted by the Canadian government as such. Read about CORD’s work here.

In 2023, the Canadian government announced a National Strategy for Drugs for Rare Diseases which aims to give better access to drugs for rare diseases and also advance newborn screening and diagnosis of rare diseases. This followed consultations done by Health Canada (read report).

For more information about this Strategy for drugs, consult our page on Treatments.

In Quebec, the Regroupement québécois des maladies orphelines (RQMO)/Quebec Coalition of Orphan Diseases advocated since its creation in 2010 for a Quebec Strategy for Rare Diseases (written in 2025, revised in 2019). After consultations with the RQMO and rare disease patient organizations, the Quebec government published a Policy for rare diseases in 2022 and an Action Plan in 2023. Read about RQMO’s work here.

In Ontario, a Rare Diseases Working Group (Critical Care Services Ontario) published a report in 2017, and a private bill was presented in 1st reading in Ontario’s Legislative Assembly in June 2023. Bill 129, “Rare Disease Strategy Act” is “An Act to amend the Health Protection and Promotion Act to implement the recommendations of the Rare Diseases Working Group Report”.

(For initiatives in other countries, consult Rare Disease National Plans in the European Union)

Some rare disease initiatives in Canada

Canadian Rare Disease Network (research, support, and care)

On Rare Disease Day, February 29, 2024, the Canadian Rare Disease Network (CRDN) was launched to “connect Canada’s rare disease community to advance rare disease care, research, and innovation both in Canada and worldwide.” It brings together the expertise of clinicians, researchers, patients, and patient organizations in the field of rare diseases. 

The Canadian iRARE Centres are a component of Pillar #3 of the CRDN: Care, Support & Empowerment.

Canadian Institutes of Health Research (CIHR) Rare Disease Research Initiative

In 2024, through the investment of the federal government in the National Strategy for Drugs for Rare Diseases, the CIHR funded four initiatives for research on: diagnostics, gene therapy, pediatric clinical trials, and collection of data for rare diseases.

RareKids-CAN is a diverse coalition of partners focused on simplifying and speeding up clinical trials and access to innovative treatments for pediatric (children and youth) rare diseases across Canada.

Rare.Qc:  A network to advance research on rare diseases in Quebec funded by the Fonds de recherche du Québec.

Canadian Drug Agency (CDA)

• Newborn Screening Guidance
• Rare Disease-Based Registries

Canadian Institute of Health Information (CIHI): Improving Data and Information for Rare Diseases

Health Canada’s Pediatric Drug Action Plan aims to improve access to safe and effective medicine for children in Canada, including drugs for rare diseases.

First Nations and Inuit patients 

Through the National Strategy for Drugs for Rare Diseases, funding is going to Indigenous Services Canada’s Non-Insured Health Benefits Program to support eligible First Nations and Inuit patients living with rare diseases.