Today, the Governments of Canada and Newfoundland and Labrador signed the National Strategy for Drugs for Rare Disease (DRD) agreement to invest a total amount of over $22 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 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.
Common List
The common list of new drugs is a result of over a year of development with provinces and territories 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, such as Newfoundland and Labrador, 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 (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 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 Newfoundland and Labrador agreement signature
As part of today's announcement, the Government of Newfoundland and Labrador is confirming that it is initially electing to make these two 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 2 to 3 people in Newfoundland and Labrador who will be diagnosed with this rare condition each year, public drug coverage will be in the order of $35K for the first treatment, and $17K 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 1 to 2 people with this rare disease in Newfoundland and Labrador at any one time, the costs of treatment can be as high as $1.7 million for the first treatment as adults, and over $500K as children. Every year afterwards adult patients face costs of over $1 million, and up to $400K for children.
Screening and Diagnostics and Other DRDs
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 Newfoundland and Labrador is committing to work with Canada and other provinces and territories 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.
