The National Strategy for Drugs for Rare Diseases

On this page

Overview

On March 22, 2023, the Government of Canada announced the launch of the first phase of the National Strategy for Drugs for Rare Diseases. This will 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:

  1. Support patient outcomes and sustainability
  2. Invest in innovation
  3. Seek national consistency
  4. Collect and use evidence

As part of the strategy, we're working with provinces and territories to develop bilateral funding agreements. 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:

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. We've also confirmed ongoing funding of up to $500 million per year for future phases to help people with rare diseases access the drugs they need.

Learn more:

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:

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:

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.

Learn more:

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:

The Canadian Institutes of Health Research

The Canadian Institutes of Health Research launched 4 different funding opportunities, supporting projects focused on:

Learn more:

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:

The Canadian Institute for Health Information is:

This work will help to fill in gaps in information that decision-makers need related to drug reviews and drug coverage.

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:

Find a directory on Orphanet

Clinical trials

We publish authorized trials in Canada on our Clinical Trials Database.

Find a clinical trial in Canada

International clinical trial registries include:

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.

Learn more:

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:

  1. l'Institut national d'excellence en sante et en services sociaux (Quebec)
  2. 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:

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

Page details

Date modified: