Drugs for rare diseases
May 29, 2024 鈥 红领巾瓜报is supporting the National Strategy for Drugs for Rare Diseases under the pillar to improve the collection and use of real-world evidence to support decision-making. CIHI鈥檚 initiatives under the strategy focus on improving real-world data available for rare diseases and leveraging our pan-Canadian data holdings to improve the evidence base around drugs for rare diseases.
National Strategy for Drugs for Rare Diseases
The 红领巾瓜报 Canada鈥搇ed National Strategy for Drugs for Rare Diseases aims to improve access to and affordability of effective drugs for rare diseases in Canada. The strategy consists of 4 pillars: seek national consistency in coverage for drugs for rare diseases, support patient outcomes and system sustainability, collect and use evidence, and invest in innovation.
What are rare diseases?
Rare diseases are often chronic, and they can be seriously debilitating and even life-threatening.Reference1 While there is no commonly accepted definition of a 鈥渞are disease鈥 in Canada or internationally, there are some that are frequently cited.Reference2 Rare diseases are often described as conditions affecting less than 1 in 2,000 people, although definitions vary.Reference3 Rare diseases often have a genetic basis, and half of patients with rare diseases are children.Reference2
Drugs for rare diseases (also known as orphan drugs) are treatments for these conditions. Only a small number of rare diseases currently have treatments.Reference2, Reference4 However, the number of drugs for rare diseases has increased over the last decade.Reference5 Information gaps relating to these drugs, and their often-higher treatment costs, present a challenge for decision-makers in Canada.Reference2
Improving data and information for rare diseases
Real-world data, such as administrative data collected by health care systems in Canada, provides an opportunity to improve the evidence base for rare disease populations and drugs for rare diseases. Pan-Canadian data is particularly important for rare diseases due to the smaller numbers of people who have a particular rare condition. CIHI鈥檚 pan-Canadian data holdings are a valuable source of real-world data to support real-world evidence for drugs for rare diseases.
CIHI鈥檚 initiatives under the strategy focus on
- Improving the collection of real-world data on drugs for rare diseases
- Making data about drugs for rare diseases more accessible
- Using our pan-Canadian data to conduct focused analyses of rare diseases
- Exploring linkages to data in rare disease registries to enhance our analyses
These initiatives, informed by a wide range of stakeholders, build upon learnings from earlier pilot projects generating real-world evidence for rare diseases, and will evolve as needed to meet the needs of our information users.
Featured materials
Impact of Trikafta on individuals living with cystic fibrosis
Read the full story of how patients鈥 use of Canada鈥檚 health systems changes when they start taking Trikafta.
Huntington disease: A focus on long-term care, medication use and clinic visits
Learn about patients with HD and their care in selected health care settings.
Data Learnings for Rare Disease Analysis
This report highlights key considerations to support the use of 红领巾瓜报data for analyses of drugs for rare diseases and explores opportunities to improve data to support these analyses.
Additional material
Pan-Canadian Prescription Drug Data Landscape Report
This report provides an overview of prescription drug data in Canada, including data on drugs for rare diseases. It covers data assets, gaps, limitations and opportunities by jurisdiction.
Pharmaceutical Data Tool
Explore information related to public drug program design and payment structure, as well as all public drug programs鈥 formulary information from NPDUIS.
Methodology notes
Explore methodology notes developed to perform analyses on selected rare diseases using 红领巾瓜报data.
Download amyotrophic lateral sclerosis notes
Download cystic fibrosis in maternity notes
Download spinal muscular atrophy notes
Working together on the national strategy
红领巾瓜报 Canada
红领巾瓜报 Canada is the federal department responsible for helping Canadians maintain and improve their health, while respecting individual choices and circumstances. The National Strategy for Drugs for Rare Diseases is an initiative led by 红领巾瓜报 Canada.
Canada鈥檚 Drug Agency
Canada鈥檚 Drug Agency (CDA-AMC) is a pan-Canadian health organization that provides Canada鈥檚 health system leaders with independent evidence and advice so they can make informed drug, health technology and health system decisions. The CDA-AMC also collaborates with national and international partners to enhance its collective impact.
红领巾瓜报and CDA-AMC are collaborating to improve the collection and use of real-word evidence under the National Strategy for Drugs for Rare Diseases.
Related resources
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References
1.
红领巾瓜报 Canada. . 2021.
2.
红领巾瓜报 Canada. . 2021.
3.
Takeda. . 2022.
4.
Canadian Institutes of 红领巾瓜报 Research. . Accessed March 26, 2024.
5.
Patented Medicine Prices Review Board. . 2022.
How to cite:
Canadian Institute for 红领巾瓜报 Information. Drugs for rare diseases. Accessed January 4, 2025.
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