Canada Expands Access to Rare Disease Medications Through Provincial Agreements
Key Insights
The Government of Canada (search) has signed bilateral agreements with Yukon and Nova Scotia, investing over $8.5 million and $39 million respectively over three years to improve access to drugs for rare diseases.
The agreements will provide funding for several specialized medications including Yescarta, a CAR T-cell therapy for B-cell lymphomas (search), and five additional drugs in Nova Scotia for conditions including mycosis fungoides (search) and von Hippel-Lindau disease (search).
These initiatives are part of Canada's National Strategy for Drugs for Rare Diseases, which aims to help the one in 12 Canadians living with rare diseases access treatments that can cost between $100,000 and $4 million annually.
The Canadian government has taken significant steps to address the healthcare needs of citizens with rare diseases by signing bilateral agreements with Yukon and Nova Scotia. These agreements, announced in March 2025, will provide substantial funding to improve access to specialized medications that are often prohibitively expensive for patients.
In Yukon, the federal government will invest over $8.5 million over three years, while Nova Scotia will receive more than $39 million under similar terms. These funds are part of Canada's National Strategy for Drugs for Rare Diseases, which includes a total investment of up to $1.5 billion over three years announced in March 2023.
Addressing a Critical Healthcare Gap
Approximately one in 12 Canadians lives with a rare disease (search), and for most affected individuals, treatment costs are unaffordable without assistance. Innovative treatments for rare diseases can range from $100,000 to more than $4 million per year, creating significant financial barriers to care.
"Reaching this agreement is another step in our collaborative efforts to build a stronger public health system for the Yukon," said the Honourable Mark Holland, Canada's Minister of Health. "Investing in drugs for rare diseases will improve affordability and access to medications, early diagnosis, and screening, so that people in the territory with rare diseases have the best health outcomes possible."
Specific Medications Covered
The initial phase of the Yukon agreement will provide funding for Yescarta, a chimeric antigen receptor (search) (CAR) T-cell therapy used to treat several forms of relapsed or refractory B-cell lymphomas (search) in adults. This immunotherapy uses a patient's own immune cells to fight cancer, specifically targeting follicular lymphoma (search), large B-cell lymphoma (search) (LBCL), diffuse large B-cell lymphoma (search) (DLBCL), and high-grade B-cell lymphoma (search) (HGBL).
The Nova Scotia agreement is more extensive, covering six specific medications:
- Poteligeo, for the treatment of mycosis fungoides or Sézary syndrome
- Oxlumo, for the treatment of hyperoxaluria type 1
- Epkinly for relapsed or refractory diffuse large B-cell lymphoma
- Welireg for the treatment of von Hippel-Lindau (VHL) disease
- Yescarta for B-cell lymphomas (same indications as in Yukon)
- Koselugo, for the treatment of neurofibromatosis type 1
"Nova Scotia is pleased to partner with the federal government to better support Nova Scotians living with rare diseases," said the Honourable Michelle Thompson, Nova Scotia's Minister of Health and Wellness. "This agreement helps support access to medications for rare diseases, improvements to screening and diagnostics, and work to strengthen data and evidence for decision making."
Beyond Medication Access
Both agreements extend beyond simply providing access to medications. They also include commitments to work on improving early diagnosis, screening, and enhanced access to existing drugs. The territories and provinces will collaborate with the federal government to develop and implement plans for improving screening and diagnostics specifically for rare diseases.
The Honourable Kamal Khera, Canada's Minister of Health who signed the Nova Scotia agreement, emphasized that this initiative is "another step toward building a stronger public health system for the people of Nova Scotia."
Transparent Implementation Process
The federal government has committed to a transparent process for implementing these agreements. Additional drugs on the common list will be published online on a drug-by-drug basis following the conclusion of price negotiations through the pan-Canadian Pharmaceutical Alliance (search).
"This significant investment of over $39 million, through the Government of Canada (search)'s bilateral agreement with Nova Scotia, represents a crucial step forward in ensuring Canadians with rare diseases have access to medications they need," said the Honourable Kody Blois, Minister of Agriculture and Agri-Food and Rural Economic Development. "We are committed to building a healthcare system where cost and location are not barriers to essential treatment."
A National Approach to Rare Disease Treatment
These bilateral agreements with Yukon and Nova Scotia represent part of a broader national strategy to address the challenges faced by Canadians with rare diseases. The federal government has indicated its commitment to working with all provinces and territories to improve healthcare access, including affordable prescription medications.
For patients with rare diseases who have often faced limited treatment options and financial hardship, these agreements signal a meaningful shift toward more equitable healthcare access regardless of geographic location or financial means. As the National Strategy for Drugs for Rare Diseases continues to roll out across Canada, more patients are expected to benefit from improved access to these specialized treatments.