The Governments of Canada and British Columbia (BC) signed the National Strategy for Drugs for Rare Diseases (DRD) agreement to invest a total amount of $194 million over three years to improve access to new drugs for rare diseases for its 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 Diseases (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 funding and design of future phases of the Strategy, for which the Government of Canada has confirmed ongoing funding of up to $500 million per year to help Canadians with rare diseases access the drugs they need.
In the lead-up to this first bilateral agreement, PTs, except Quebec, worked together to develop a small common list of new drugs to be funded and cost-shared across the country, and began discussions on a collaborative approach to improve screening and diagnostics for rare diseases.
Common List
The common list of new drugs is a result of over a year of development with PTs to ensure that the National Strategy delivers the most benefits possible to patients with rare diseases. Recognizing the unique challenges associated with decision making about drugs to treat rare diseases, 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, such as BC, are committing to work with 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 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 other new rare disease drugs.
As the pan-Canadian Pharmaceutical Alliance concludes 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.
Types of Drugs Being Covered at time of BC agreement signature
The first two drugs on the common list to be announced are Poteligeo, for the treatment of mycosis fungoides or Sézary syndrome, and Oxlumo, for the treatment of primary hyperoxaluria type 1.
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. MF and SS are the most common forms of a rare type of cancer in the blood called cutaneous T-cell lymphoma (CTCL), which can result in painful, itchy, and infected skin, and problems with the functioning of the circulatory system and lymph nodes. For the estimated 20 people in British Columbia who will be diagnosed with these rare conditions each year, based on the list prices, Poteligeo costs approximately $35K thousand for the first treatment, and $17K thousand for subsequent treatment cycles.
Health Canada has authorized Oxlumo for use in pediatric and adult patients with primary hyperoxaluria type 1 (PH1), a condition which leads to crystals forming in the body. These crystals can cause permanent damage to tissues, specifically the kidneys, leading to loss of function. Oxlumo may help to prevent the formation of crystals. For the estimated 5-15 people with this rare disease in British Columbia at any one time, the costs of treatment, based on public pricing, can be as high as $1.7M for the first treatment as adults, and over $500K thousand as children. Every year afterwards adult patients face costs of over $1M million, and up to $400K thousand for children.
Funding through the National Strategy will support the Province of British Columbia to provide access to the drug these patients need to treat their rare disease and reduce the cost burden on their families.
By entering into this bilateral agreement, the Government of BC is confirming that it is electing to make these two drugs available to its residents. Poteligeo will be subject to eligibility and rules of BC Cancer drug funding. Funding of Oxlumo will be subject to patient eligibility and the rules of the Expensive Drugs for Rare Diseases process in BC.
Screening and Diagnostics and Other DRD
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 BC 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.
