Key initiatives
Our strategic plan is anchored in three strategic priorities:
- Capacity: Strengthening pediatric rare disease clinical trial capacity and advanced therapy medicinal product (ATMP) readiness
- Matching: Optimizing participant, site, and trial matching
- Reform: Driving regulatory reform and system innovation
This balanced approach ensures continuity, strengthens capacity, and promotes long-term impact for children and families affected by rare diseases across Canada.
Reform: Driving regulatory reform and system innovation
Goal: To drive regulatory reform and system innovation to enable timely access to pediatric rare disease therapies in Canada. The network leads efforts to reduce regulatory barriers to pediatric rare disease clinical trial (PRDCT) development, conduct and treatment access by actively engaging with Health Canada and international partners.
Key Initiative 3.1: Regulatory pathways
Identify policy objectives designed to:
- Ensure Canada’s regulatory environment is competitive and aligned with contemporary international best practice
- Decrease barriers to the launch of drug and device clinical trials
- Increase access to life-saving and life-sustaining pediatric rare disease therapies
RareKids-CAN has developed and is executing a multi-faceted advocacy strategy to advance the implementation of a policy reform agenda designed to modernize Health Canada’s regulatory environment in the development and conduct of PRDCTs and facilitate timely access to pediatric rare disease treatments in Canada.
Key Initiative 3.2: Global engagement
The profound impact of rare diseases on affected children, adolescents, young adults, and their families cannot be overstated. Despite the complexity and rarity of these conditions, a unified international effort in PRDCTs and treatment holds immense potential to improve quality of life and clinical outcomes for these patient populations.
To enhance access to and increase the number of innovative PRDCTs coming to Canada, we will build on international collaborations established by MICYRN and RareKids-CAN. The network, together with the Canadian Rare Disease Network (CRDN), co-leads the Canadian National Mirror Group of the European Rare Diseases Research Alliance (ERDERA).
Key Initiative 3.3: Access pathways for therapies unfit for commercialization
Therapies unfit for commercialization are promising rare disease treatments for which a viable or sustainable traditional commercial pathway does not exist, due to factors such as very small patient populations, limited commercial incentives, intellectual property constraints, manufacturing complexity, or the need for academic or hospital-based development and delivery.
Advancing these therapies requires alternative approaches to regulation, manufacturing, evidence generation, reimbursement, and long-term delivery. RareKids-CAN is working to address these system-level barriers and support sustainable Canadian pathways for equitable access to appropriate therapies.