The Governments of Canada and Ontario have signed an agreement under the National Strategy for Drugs for Rare Diseases.
This partnership includes an investment of over $535 million over three years to enhance access to new and existing drugs for rare diseases in Ontario, as well as support for early diagnosis and screening efforts, Federal officials say.
In March 2023, the Government of Canada announced a commitment of up to $1.5 billion over three years to the National Strategy, including $1.4 billion for agreements with provinces and territories. These agreements aim to provide patients with earlier access to treatments, improving their quality of life.
The initial phase of the National Strategy focuses on collaboration with governments and health system partners to test and refine approaches. Insights from these agreements and related projects will inform the design of future phases of the Strategy.
In preparation for this agreement, provinces and territories, excluding Quebec, developed a common list of new drugs to be shared and cost-managed across the country. Discussions were also initiated to enhance screening and diagnostics for rare diseases.
The common list was designed to maximize benefits for patients while addressing challenges in decision-making about rare disease drugs. It supports the collection and evaluation of real-world data within Canada’s pharmaceutical system. Ontario and other participating provinces and territories have committed to evidence collection projects to improve the use of real-world data in decision-making for new drugs.
Drugs on the common list will be made available after pricing negotiations with the pan-Canadian Pharmaceutical Alliance. Ontario has confirmed that it will cover five drugs under the agreement. These include treatments for conditions such as mycosis fungoides, Sézary syndrome, primary hyperoxaluria type 1, von Hippel-Lindau disease, B-cell lymphomas, and large B-cell lymphoma. Treatment costs vary significantly, with some drugs exceeding $1 million per year for adult patients.
One notable example is Epkinly, a drug for relapsed or refractory large B-cell lymphoma. It is the first treatment to receive a time-limited reimbursement recommendation from Canada’s Drug Agency, allowing public funding on the condition that further clinical studies address evidence uncertainties. This drug also went through the Temporary Access Process, a new negotiation pathway that allows earlier patient access while additional data is collected.
In addition to improving access to drugs, the agreement includes a commitment to enhance screening and diagnostics for rare diseases. Ontario will work with Canada and other provinces and territories to develop a plan for better diagnostics within the first two years of the agreement. Investments in this area will begin no later than the third year.
This agreement represents an important step toward addressing the challenges of rare diseases, improving treatment access, and fostering collaboration across Canada’s healthcare systems, Federal officials added.
