Drugs for rare diseases bilateral agreements: Results
On this page
- Reporting highlights
- Overall spending
- Consistency in access to drugs: Drugs on the common list newly covered by provinces and territories in 2024–25
- Improvements in access to drugs: Other drugs for rare diseases with new or enhanced coverage
- Improving access to drugs through system improvements
- Improving screening and diagnostics
The Government of Canada has signed 3-year bilateral funding agreements with all 13 provinces and territories, for a total of $1.4 billion, to:
- make drugs for rare diseases more accessible and affordable
- improve the health of patients across Canada
These agreements commit provinces and territories, except Quebec, to investing in improving:
- coverage and access to drugs
- screening and diagnostics services for rare diseases
Coverage and access to drugs includes funding for:
- drugs on the common list developed by Health Canada and the provinces and territories
- other rare disease drugs outside of this common list, based on the context of each jurisdiction
As part of these agreements, all provinces and territories, except Quebec, must submit annual financial statements and report on outcomes and results achieved collaboratively with the Government of Canada. This supports transparent use of strategy funds.
Quebec's funding agreement supports its Plan d'action québécois sur les maladies rares (Quebec action plan for rare diseases 2023–2027) and other related activities. Quebec will continue to make reports available to the public.
Learn more:
The first year of agreements were from April 2024 to March 2025.
This summary shows progress made during this first year of the strategy, and was jointly developed with provinces and territories who were fully engaged as required under the agreements. Activities will build over the 3 years, so system investments made now will support more patients over time.
Reporting highlights
- Over 632 people living in Canada got new access to drugs for their rare condition due to strategy funding.
- Provinces and territories spent 88% of agreement funding, carrying the remainder forward to spend in year 2 (2025–26).
- Of the money spent in year 1:
- 10% was spent on drugs on the common list. This proportion will grow as implementation of this part of the Strategy ramps up, reaching a minimum of 50% in the third year of the agreements.
- 87% was spent on access to drugs outside of the common list, where new or expanded coverage for patients was implemented as determined by jurisdictions.
- Investments were also made to:
- build system capacity
- address barriers to access
- improve coordination and monitoring of patient outcomes
Overall spending
Out of all funding spent by provinces and territories under the strategy:
- 10% supported consistent access to drugs on the common list
- 87% went to improved coverage for other drugs for rare diseases, either to:
- provide access to new drugs, or
- enhance coverage for drugs that were already available to patients
- 3% was invested in improving screening and diagnostics for rare diseases
Consistency in access to drugs: Drugs on the common list newly covered by provinces and territories in 2024–25
In total, provinces and territories spent $31.8 million of strategy funds on common list drugs in 2024–25. Over the course of the 3-year agreements, the list will improve consistency of drug coverage across Canada, as more provinces and territories cover more of these drugs. To ensure that consistency, provinces and territories (except Quebec) must spend 50% of strategy dollars on common list drugs by year 3.
Coverage in table 1 will continue to grow over the 3 years, based on decisions made by drug plans and patients requesting the drugs.
Learn more:
| Drug | Condition | Number of provinces and territories except Quebec covering in 2024–25 |
|---|---|---|
| Poteligeo | Mycosis fungoides and Sézary syndrome | 11 |
| Epkinly | Relapsed or refractory diffuse large B-cell lymphoma | 10 |
| Oxlumo | Primary hyperoxaluria type 1 | 8 |
| Welireg | von Hippel-Lindau disease | 8 |
| Yescarta | Relapsed or refractory diffuse large B-cell lymphoma and follicular lymphoma | 7 |
| Koselugo | Neurofibromatosis type 1 | 3 |
| Sohonos | Fibrodysplasia ossificans progressiva | 1 |
Improvements in access to drugs: Other drugs for rare diseases with new or enhanced coverage
In year 1, provinces and territories spent $280.2 million of strategy funds on improving coverage for other drugs for rare diseases not included on the common list. The agreements allow them to make these improvements based on their unique circumstances and on the needs of their population living with rare diseases. Improvements included:
- newly covered drugs
- expanding coverage eligibility to more patients
- faster approvals for coverage due to certainty of funding
For example, multiple jurisdictions began to cover Zolgensma for spinal muscular atrophy. Through a new screening program for newborns for this condition, health care providers in these jurisdictions can identify and treat it as early as possible.
The drugs listed in tables 2 and 3 are based on information provided by provinces and territories where strategy funds were used to make improvements. Cancer and non-cancer drugs are listed separately for ease of reference.
| Type of condition | Condition | Drugs | Improvement in some provinces and territories |
|---|---|---|---|
| Metabolic | Cystic fibrosis | Trikafta | Newly covered, covered for more patients |
| Hypophosphatasia | Strensiq | Newly covered at 100% | |
| Fabry disease | Galafold | Covered for more patients | |
| Phenylketonuria | Kuvan | Continued coverage | |
| Nervous system | Spinal muscular atrophy | Zolgensma, Spinraza, Evrysdi | Newly covered, covered for more patients, continued coverage |
| Hereditary transthyretin-mediated amyloidosis | Amvuttra, Vyndamax/Vyndaqel | Newly covered, newly covered at 100%, covered for more patients | |
| Neuromyelitis optica spectrum disorder | Enspryng | Continued coverage, faster access | |
| Blood and cardiac system | Obstructive hypertrophic cardiomyopathy | Camzyos | Newly covered, newly covered at 100% |
| Paroxysmal nocturnal hemoglobinuria | Empaveli | Newly covered, newly covered at 100% | |
| Paroxysmal nocturnal hemoglobinuria, hemolytic uremic syndrome | Ultomiris | Newly covered, continued coverage, faster access | |
| Hereditary angioedema | Orladeyo | Newly covered, covered for more patients | |
| Eye conditions | Cystinosis | Cystadrops | Newly covered |
| Multiple rare disorders | Paroxysmal nocturnal hemoglobinuria, hemolytic uremic syndrome, myasthenia gravis, Neuromyelitis optica spectrum disorder | Soliris | Continued coverage |
| Type of cancer | Condition | Drugs | Improvement in some provinces and territories |
|---|---|---|---|
| Carcinomas | Breast cancer | Tukysa | Continued coverage, faster access |
| Epithelial ovarian, fallopian tube, and peritoneal cancer | Zejula | Newly covered | |
| Non-small cell lung cancer | Rozlytrek | Continued coverage, faster access | |
| Hepatocellular carcinoma | Imjudo, Tecentriq with Avastin | Newly covered, continued coverage, faster access | |
| Leukemias | Acute lymphocytic leukemia | Asparlas | Newly covered |
| Leukemia, mastocytosis | Rydapt | Continued coverage | |
| Lymphomas | Relapsed or refractory diffuse large B-cell lymphoma | Columvi | Newly covered |
| Multiple conditions | Yescarta | Newly covered, newly covered at 100% | |
| Other blood cancer | Multiple myeloma | Darzalex | Continued coverage |
| Melanomas | Melanoma | Opdualag | Newly covered |
| Melanoma | Braftovi with Mektovi | Continued coverage, faster access | |
| Melanoma | Yervoy | Continued coverage | |
| Melanoma | Opdivo | Continued coverage | |
| Uveal melanoma | Kimmtrak | Newly covered | |
| Multiple rare cancers | Multiple rare cancers | Avastin | Covered for more patients |
| Multiple rare cancers | Calquence | Newly covered, continued coverage, faster access | |
| Multiple rare cancers | Cabometyx | Continued coverage, faster access | |
| Multiple rare cancers | Imbruvica | Covered for more patients | |
| Multiple rare cancers | Lynparza | Covered for more patients | |
| Multiple rare cancers | Libtayo | Continued coverage, faster access | |
| Multiple rare cancers | Mekinist | Covered for more patients | |
| Multiple rare cancers | Tecartus | Newly covered | |
| Multiple rare cancers | Keytruda | Continued coverage | |
| Multiple rare cancers | Imfinzi | Continued coverage |
Improving access to drugs through system improvements
Investments in drug coverage also supported infrastructure and system changes that will benefit all people living with rare diseases, not just those newly receiving drugs. Here are some examples of what different provinces and territories have been doing to improve access.
Collaborative and coordinated decision-making
A province is coordinating a national panel for Sohonos funding applications for many provinces and territories. This increases consistency of drug reimbursement across the country.
Supporting better patient monitoring and experience
A province is now using electronic forms for coverage requests for high-cost drugs, including drugs for rare diseases. This removes administrative steps and speeds up response time when applying for funding.
Other provinces and territories are using new software to manage records and coverage requests for drugs for rare diseases.
Some provinces and territories are also supporting coordinated care and monitoring of patient outcomes and harms that may have been caused by treatment.
Addressing geographic barriers
Provinces and territories are paying for more patient travel and transportation costs. They're also working to improve access across more of their jurisdictions.
Increasing capacity
Provinces and territories are hiring more people to reduce the time it takes to list a drug and to generally increase their ability to deliver more drugs for rare diseases to patients.
Improving screening and diagnostics
Provinces and territories spent $9.7 million of strategy funds on screening and diagnostics improvements in year 1, such as:
- lab networks
- training and education
- equipment and infrastructure
For example:
- A province expanded a newborn screening program to newly screen babies for:
- spinal muscular atrophy
- tyrosinemia type I
- A province invested in sequencers to build the infrastructure for whole genome sequencing in Canada, so testing will no longer need to be sent outside the country.
- This will help doctors increase the number of rare conditions they screen and help identify the best drug for the patient.
- Other provinces and territories are also bringing diagnostic testing panels into Canada instead of sending testing outside the country.
- Some provinces and territories are facilitating training and expanding the types of specialists who can order genetic testing.
Other activities supporting improved access
The Government of Canada is also providing funding to Indigenous Services Canada's Non-Insured Health Benefits program. This will support eligible First Nations and Inuit patients living with rare diseases.