PEI Strikes Deal on Rare Disease Drug Access
Today, the Governments of Canada and the province of Prince Edward Island (PEI) signed the National Strategy for Drugs for Rare Disease (DRD) agreement to invest over $10 million over three years to improve access to new drugs for rare diseases for PEI residents and to support enhanced access to existing drugs, early diagnosis, and screening for rare diseases.
In March 2023, the Government of Canada announced an investment of up to $1.5 billion over three years to support the National Strategy for Drugs for Rare Disease (the Strategy), including up to $1.4 billion for bilateral agreements with provinces and territories (PTs) to help patients with rare diseases have access to treatments as early as possible, for better quality of life.
These three-year agreements are part of the first phase of the Strategy, which is focused on building, testing, and learning in collaboration with governments and health system partners. Lessons learned from the initial three-year bilateral funding agreements with PTs as well as the Strategy-funded projects by pan-Canadian partners (e.g., drug pipeline work) will be incorporated into recommendations for the design of future phases of the Strategy.
In the lead-up to this bilateral agreement, PTs, except Quebec, worked together to develop a small common list of new drugs to be listed and cost-shared across the country, and initiated discussions on a collaborative approach to improve screening and diagnostics for rare diseases.
The common list of new drugs was developed with PTs to ensure that the National Strategy delivers the most possible benefits to all patients with rare diseases. Recognizing the unique challenges associated with decision making about rare diseases drugs, the common list is designed to further the development, collection, evaluation and use of real-world data and evidence in decision making about the listing and reimbursement of rare disease drugs within Canada’s existing pharmaceutical management system.
Through the signing of bilateral agreements, PTs, including PEI, are committing to work with the Government of Canada and other PTs who may sign bilateral agreements to design and implement evidence collection projects – complementary to related projects now underway through Canada’s Drug Agency (CDA-AMC) and the Canadian Institute of Health Information – that will inform the future collection and use of real-world data and evidence for decision making about all new rare disease drugs.
Following the conclusion of pan-Canadian Pharmaceutical Alliance (pCPA) price negotiations for each drug on the common list, they will be published online here on a drug-by-drug basis. PTs who choose to sign bilateral agreements may elect to make these drugs available to their residents.
As part of today’s announcement, the Government of PEI is confirming that it is initially electing to make the following three drugs available to its residents:
Health Canada has authorized Poteligeo, an intravenous chemotherapy drug, for the treatment of adult patients with relapsed or refractory mycosis fungoides (MF) or Sézary syndrome (SS) who have tried and failed at least one other treatment. For the estimated 6 to 9 people in PEI who will be diagnosed with this rare condition each year, public drug coverage will be in the order of $35,000 for the first treatment, and $17,000 for subsequent treatment cycles.
Health Canada has authorized Koselugo for pediatric patients aged 2 years and above, with neurofibromatosis type 1 (NF1) who have symptomatic, inoperable plexiform neurofibromas (PN). For the estimated 38-56 children in PEI who will be diagnosed with this rare condition each year, public drug coverage is expected to cost approximately $268,678 per patient per year.
Health Canda has authorized Epkinly for the treatment of adults with relapsed or refractory large B-cell lymphoma (DLBCL), a type of blood cancer which causes tumours in the lymph nodes or other organs including the spleen, liver or bone marrow. For the estimated 51 people diagnosed with this disease each year, public drug coverage for the first 28-day treatment cycle for each patient will be $14,000, with subsequent cycles varying in cost.
Epkinly is the first drug to receive a time-limited reimbursement recommendation from the CDA-AMC, which is a recommendation to publicly fund a drug for a certain period of time on the condition that the manufacturer will conduct ongoing clinical studies that address uncertainty in the evidence.
Additionally, it is the first drug to go through the pCPA Temporary Access Process, a new negotiation pathway developed by the pCPA to establish temporary access to drugs that have received a time-limited reimbursement recommendation from CDA-AMC.
Following submission of additional data, final recommendations and long-term pricing agreements can be completed and negotiated respectively. Such measures provide patients with earlier access to promising new drugs while creating mechanisms to revisit the clinical and economic evidence as it evolves.
In addition to improving access to new DRD on the common list, bilateral agreements aim to improve screening and diagnostics as well as access to other existing DRD. Through this agreement PEI is committing to work with Canada and other PTs who may sign bilateral agreements on developing and implementing a plan for improving screening and diagnostics for rare diseases. This planning work will take place over the first two years of the agreement period, with investments to begin no later than the third year.
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https://www.canada.ca/en/health-canada/news/2025/03/drugs-for-rare-diseases-prince-edward-island-agreement.html