The National Strategy for Drugs for Rare Diseases
On this page
- Overview
- Working with provinces and territories
- Research partners and initiatives
- Resources for patients and practitioners
Overview
The Government of Canada's National Strategy for Drugs for Rare Diseases provides $500 million in ongoing annual funding to help increase access to effective drugs for rare diseases and make them more affordable for patients across Canada. The strategy focuses on 4 key areas:
- Support patient outcomes and sustainability
- Invest in innovation
- Seek national consistency
- Collect and use evidence
As part of the strategy, the Government of Canada signed bilateral funding agreements with all 13 provinces and territories. We're also providing funding to Indigenous Services Canada's Non-Insured Health Benefits Program to support eligible First Nations and Inuit patients living with rare diseases.
We've also set up the Implementation Advisory Group, which will support the rollout of the strategy. This will ensure a patient-centered approach towards our goals for accessible and affordable drugs for rare diseases.
We're also working with key research partners to improve how we collect, use and research evidence.
During the strategy's consultation phase, we heard about:
- the need for faster and easier access to drugs for rare diseases
- the challenges of high drug costs
- the disparity in access across the country
We'll continue to adapt the strategy as we go so that we can address these challenges effectively. The first phase of the strategy focuses on building, testing and learning with governments and health system partners. We'll use lessons learned from agreements and other partner activities when we make recommendations for future phases of the strategy.
Learn more:
- Non-insured health benefits for First Nations and Inuit
- Implementation Advisory Group for the National Strategy for Drugs for Rare Diseases
Working with provinces and territories
We're making up to $1.4 billion in funding available to provinces and territories through 3-year agreements, to help them provide better coverage and access to:
- elected new drugs for rare diseases on the common list
- other new and existing drugs for rare diseases
- screening and diagnostics services
Funding will be available between April 1, 2024 and March 31, 2027.
These agreements between federal, provincial and territorial governments will aim to improve access to treatments and drugs for rare diseases by focusing on:
- collecting data
- making informed decisions
- expanding coverage of drugs for rare diseases
- improving screening and diagnostics for rare diseases
- exploring how to improve availability of drugs for rare diseases
Learn more:
We worked with provinces and territories to develop a common drug list for rare diseases. This list helps decision-makers gather and evaluate data relevant for drug listing and reimbursement. This informs future rare disease drug approvals to help increase benefits for all rare disease patients.
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We worked collaboratively with provinces and territories to report finances and results for each year of these agreements.
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Research partners and initiatives
We're working with partners to advance rare disease research, and to collect and use evidence that supports decision-making. These partners include:
- Canada's Drug Agency
- the Canadian Institutes of Health Research
- the Canadian Institute for Health Information
The Canadian Institutes of Health Research
The Canadian Institutes of Health Research provide 4 different funding opportunities, for projects focused on:
- developing gene therapies for rare diseases
- developing better diagnostic tools for rare diseases
- improving administrative data and monitoring for rare diseases
- creating a network for pediatric rare disease clinical trials and treatment
Learn more:
- CIHR Rare Disease Research Initiative
- Projects funded through the CIHR Rare Disease Research Initiative
Canada's Drug Agency and the Canadian Institute for Health Information
The agency and the institute have both launched data and evidence collection projects that focus on all stages of the drug lifecycle.
Projects from Canada's Drug Agency include:
- customized drug reviews to advise decision-makers
- a panel to develop guidance on newborn screening across Canada
- standards and tools to improve the data from rare disease registries
- tools with information on emerging drugs for rare diseases to better prepare health systems
The Canadian Institute for Health Information is:
- reviewing and reporting on:
- existing prescription drug data across Canada
- how to improve data collection in the future
- creating an interactive tool for users to compare public prescription coverage across Canada
- analyzing data from different sources to help answer questions about rare diseases and related treatments
This work will help to fill in gaps in information that decision-makers need related to drug reviews and drug coverage.
Learn more:
- Drugs for rare diseases (Canada's Drug Agency)
- Drugs for rare diseases (Canadian Institute for Health Information)
Real-world evidence work plan
Rare disease drug assessments often rely on limited data due to very small patient populations. This can lead to uncertainties in the evidence used to make decisions.
By signing bilateral agreements under the strategy, provinces and territories have committed to working with Health Canada. Together, we will look at practical ways to generate and use real-world evidence to resolve uncertainties about drugs for rare diseases.
We have designed 2 pilot projects to test how we identify, collect and analyze real-world evidence for rare disease drugs in Canada. If successful, the pilot projects will show policy makers how real-world evidence can be used in decision-making in the future.
These pilot projects will use the drugs Oxlumo and Yescarta as test cases because:
- During their drug reimbursement reviews, reviewers found evidence uncertainties common in drugs for rare diseases coming to market in Canada
- We have available data sources that could support generation of more evidence for these drugs
As such, the purpose of these pilot projects is to:
- evaluate the quality of real-world evidence that we can generate in Canada
- understand the institutional and authority issues in sharing data, conducting analysis, and using results
- evaluate how to apply lessons learned in making decisions about future drugs that are not yet publicly funded in Canada
The pilot projects will not revisit specific decisions around approvals or reimbursement for any drugs.
These pilot projects are a collaborative effort between:
- Health Canada
- provinces and territories
- Canada's Drug Agency
- including project leads from the Post-Market Drug Evaluation program, through its network of external expert researchers
- Canadian Institute for Health Information.
Learn more:
Resources for patients and practitioners
Find out more about a rare disease
Orphanet is a directory of specialized information for people with rare diseases and health care providers. Services include directories of:
- rare diseases
- orphan drugs
- patient organizations
- professionals and expert centres
- ongoing research projects and clinical trials
Clinical trials
We publish authorized trials in Canada on our Clinical Trials Database.
Find a clinical trial in Canada
International clinical trial registries include:
- ClinicalTrials.gov
- World Health Organization International Clinical Trials Registry Platform
- Current Controlled Trials International Standard Randomized Controlled Trials Number Register
Special access to a drug
If you're a health care provider and need to use an unapproved medication to address a medical emergency, you can submit a request to our special access program for drugs.
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Drug decisions
You can find out about the safety, effectiveness and quality of a drug by looking it up in our drug product database. We review every new or updated drug to assess its benefits and risks before allowing it to be sold in Canada. We also provide summaries to explain our decisions in more detail on our drug and health products portal.
Once we authorize a drug for use in Canada, provinces and territories decide if their drug plans will cover it or not. When making their decision, they follow the recommendations of 1 of 2 organizations:
- l'Institut national d'excellence en sante et en services sociaux (Quebec)
- Canada's Drug Agency: Reimbursement Review (all other provinces and territories)
The pan-Canadian Pharmaceutical Alliance is an independent organization that negotiates drug prices based on these recommendations. It negotiates lower prices on brand name drugs for all public plans and sets prices for many generic drugs.
Learn more:
- Drug product database
- Drug and health products portal
- Provincial and territorial public drug benefit programs
- Brand name drug negotiations status (pan-Canadian Pharmaceutical Alliance)
Ongoing drug monitoring
Health Canada lists recalls, advisories and safety alerts for products including drugs.
Canada's Drug Agency's Post-Market Drug Evaluation program helps decision-makers by responding to their concerns about approved drugs.
Learn more:
Related links
- Prescription drug insurance coverage
- Regulatory innovation for health products
- Canada's regulatory approach to drugs for rare diseases: Orphan drugs
- Backgrounder: Investments to Support Access to Drugs for Rare Diseases
- Government of Canada signs two bilateral agreements with British Columbia
- Canada's approach to drugs for children and youth: Pediatric Drug Action Plan
- Final Report of the Advisory Council on the Implementation of National Pharmacare
- What We Heard Report: Public Engagement on the National Strategy for Drugs for Rare Diseases
- Drugs for Rare Diseases Strategy News Release: Government of Canada improves access to affordable and effective drugs for rare diseases