Drugs for Rare Diseases

CADTH is working to improve the collection and use of evidence to help improve access to drugs for rare diseases in Canada

In March 2023, the Government of Canada announced the National Strategy for Drugs for Rare Diseases (DRDs). The national strategy aims to increase the accessibility and affordability of DRDs for patients across Canada by taking action across 4 broad pillars to, first, seek national consistency in coverage for drugs for rare diseases; second, support patient outcomes and sustainability; third, collect and use evidence; and fourth, invest in innovation. The first phase of the national strategy will be implemented over 3 years, from 2023 to 2026, and involves coordinated actions by Health Canada, provinces and territories, and various health system partners.  

As such, part of the national strategy investment includes funding for CADTH-led initiatives to improve the collection and use of evidence to inform and support decision-making. Some of these initiatives include:

  • building on existing work and developing pan-Canadian guidance to support newborn screening programs, which play an important role in the early diagnosis of rare diseases
  • supporting efforts to improve the generation of, and access to, real-world data from rare disease-based registries, which are important sources of data for the evaluation of treatments for rare diseases.