Canada's Strategy for Drugs for Rare Diseases
In March 2023, the Government of Canada launched the National Strategy for Drugs for Rare Diseases (2023–2027), committing $1.5 billion to improve access to new and existing rare disease drugs. It represents a significant step forward for the rare disease community. Here’s a look at what this strategy entails and its implications for those affected by rare disease.
Key Components of the Strategy
Support patient outcomes and sustainability
The Government of Canada is investing $1.4 billion through bilateral agreements with all 13 provinces and territories to make sure people across the country can get better access to rare disease drugs, as well as improved screening and diagnostic services. Extra funding is also provided to Indigenous Services Canada’s Non-Insured Health Benefits Program to support eligible First Nations and Inuit patients with rare diseases.
Invest in innovation
The federal government has allocated $32 million for rare disease research through the Canadian Institutes of Health Research (CIHR). This funding supports work to improve diagnostic tools, prepare Canada for future gene therapy clinical trials, and create a new national rare disease clinical trial network (called RareKids-CAN) to help bring innovative treatments to patients faster.
Seek national consistency
An Implementation Advisory Group (IAG) has been established to ensure that the strategy is patient-centered and includes patients in decision-making processes. Other new structures, such as a Health Canada Secretariat and a Federal–Provincial–Territorial working group, are helping coordinate efforts and ensure that rare disease programs are implemented effectively across the country.
Collect and use evidence
Through the Canadian Drug Agency (CDA) and the Canadian Institute for Health Information (CIHI), Canada is improving how information on rare diseases is collected and used. This includes building national guidance for rare disease screening and data collection, improving how patient registries capture information about treatments and outcomes, and using real-world evidence to guide decisions about which drugs and treatments should be made available.
By improving access to medications, supporting research and development, and enhancing patient care, this strategy promises to make a profound difference in the lives of those affected by rare diseases.