On March 22, 2023, the Government of Canada announced up to $1.5 billion in funding as part of its National Strategy for Drugs for Rare Diseases (the "National Strategy"). The announcement represents a major step towards a long-awaited national approach to improving access to drugs for rare diseases, and follows a July 2021 report on a public consultation held between January and March 2021.

Breakdown of Funding

Much of the funding under the National Strategy (up to $1.4 billion) will be diverted to the provinces and territories through bilateral agreements that will help these jurisdictions improve access to new and emerging drugs, and support greater access to existing drugs, early diagnosis and screening for rare diseases. Notably, as a next step, the federal government will engage with the provinces and territories to determine a small set of new and emerging drugs for which coverage would be cost-shared and consistent across the country.

The rest of the funding is earmarked as follows:

  • $33 million to Indigenous Services Canada's Non-Insured Health Benefits Program, for First Nations and Inuit patients living with rare diseases;
  • $20 million to the Canadian Agency for Drugs and Technologies in Health (CADTH) and the Canadian Institute for Health Information (CIHI) to improve the collection and use of evidence to support decision-making;
  • $32 million to the Canadian Institutes of Health Research (CIHR) to advance rare disease research, with a focus on developing diagnostic tools and establishing a rare disease clinical trials network; and
  • $16 million to support national governance structures for implementation of the National Strategy, such as a Health Canada secretariat and a stakeholder Implementation Advisory Group.

Provinces' Approaches to Rare Diseases

Individual provinces have taken steps towards improving care for rare diseases. For example:

  • As previously reported, the Quebec Rare Disease Policy was published in June 2022. The Policy focuses on awareness and training, easy and equitable access to diagnosis, care and services, and promotion of research, innovation and data collection.
  • The British Columbia PharmaCare Program may provide exceptional funding, on a case-by-case basis, for "expensive drugs for rare diseases".
  • The Alberta government administers a Rare Diseases Drug Coverage Program which may cover drugs approved for the treatment of a short list of rare diseases (see details in Section 4 of the Alberta Drug Benefit List).
  • In December 2017, the previous Ontario government established a Rare Disease Implementation Plan Steering Committee to implement certain recommendations from a Rare Diseases Working Group Report.

It remains to be seen whether federal funding under the National Strategy may go towards supporting existing provincial initiatives such as these.

Background on Drugs for Rare Diseases

A rare disease is defined as a disease which affects fewer than 1 in 2,000 people. It is estimated that about 1 in 12 Canadians will be affected by a rare disease in their lifetime, and rare diseases disproportionately affect children. Drugs for rare diseases, also known as orphan drugs, are typically more costly to develop and can be more difficult for patients to access. Patient advocacy groups in Canada have for years called on the federal government to take steps towards facilitating market access for drugs for rare diseases.

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