Treatments

There are many types of treatments for rare diseases: medication, medical devices, specialty foods, surgery, physiotherapy, etc. Drugs for rare diseases may be those used to treat common conditions or they may be developed for a specific rare disease. The latter are called “orphan drugs “.

What is an orphan drug?

An « orphan » drug is intended for the treatment of a rare disease. In some countries (e.g. United States, European Union, etc.), a pharmaceutical company can apply for its experimental drug to obtain an « orphan designation » and thus benefit from certain advantages (financial and others) to continue the research and development of this therapy. If, following clinical trials, the drug is found to be safe and effective and it is authorized for sale, it is then called an “orphan drug.”

In Canada:

  • There is no special program for the development of orphan drugs like the Orphan Drug Act in the United States or the « Orphan Drug Products » program in the European Union.
  • The term “orphan drug” is not officially used to designate a drug for a rare disease.
  • All drugs go through an evaluation by Health Canada to determine if they can be sold in Canada (authorized).
  • Approximately 40% of orphan drugs authorized elsewhere in the world are not available in Canada.

To find out if a drug is authorized in Canada for your rare disease, consult the Drug Product Database (DPDB).

To find out if there is an orphan drug on the market or in development elsewhere in the world, consult the following sites:

Drug reimbursement

Public insurance plans: when a drug is authorized for sale, it must go through an evaluation process to determine if it should be paid by public insurance plans. This is done by the Canadian Drug Agency for all provinces except Quebec, in which case INESSS does this evaluation. These agencies make recommendations, but each provincial government decides if a drug is paid through their public insurance plan. Private insurance companies usually pay for a drug included on a provincial formulary and may pay for other drugs.

Patients, patient organizations, and health professionals can give feedback during these evaluations.

At the Canadian Drug Agency:

At INESSS in Quebec:

Patient organizations who advocate for access to drugs

Do you want to know if there is an orphan drug in development or on the market for your rare disease? Contact us.
Or do you want us to contact you when a drug is being evaluated by the government for your disease? Register in our contact registry.

The National Strategy for Drugs for Rare Diseases

In March 2023, the Minister of Health of the Government of Canada announced the launch of the “National Strategy for Drugs for Rare Diseases”.

Read the press release

Excerpt from the press release: “As part of this overall investment, the Government of Canada will make available up to $1.4 billion to provinces and territories through bilateral agreements. This funding will help provinces and territories improve access to new and emerging drugs, as well as support enhanced access to existing drugs, early diagnosis, and screening for rare diseases. This will help patients with rare diseases, including children, have access to treatments as early as possible, for better quality of life.”

As part of this Strategy, a Rare Disease Drug Secretariat was created within Health Canada.

See the following page for the bilaterail agreements signed between the federal government and provinces/territories in 2024-2025: Bilateral Agreements for Drugs for Rare Diseases

See the composition and mandate of the “Implementation Advisory Group” (IAG) for the Strategy.

Visit the Canadian Organization for Rare Disorders website for more information and activities around the Strategy.

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