Strategic Priorities
RareKids-CAN is anchored in three strategic priorities designed to enable meaningful and lasting impact beyond the five-year funding period. These priorities are:
- 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
Capacity: Strengthening pediatric rare disease clinical trial capacity and advanced therapy medicinal product (ATMP) readiness
Strengthening the capacity of Canadian pediatric research institutions and affiliated hospitals to lead and deliver high-quality, innovative pediatric rare disease clinical trials while building readiness for the safe and effective administration of ATMPs (ex-vivo and in-vivo gene therapies, genome editing, antisense oligonucleotides therapies, and cell therapies) across research and clinical settings.
Matching: Optimizing participant, site, and trial matching
Optimizing participant, site, and trial matching to improve the efficiency of pediatric rare disease clinical trial initiation and delivery, while increasing Canada’s attractiveness for investment.
This priority aims to create an integrated ecosystem that supports patient identification, recruitment, feasibility assessment, site and investigator identification, and registry use.
Reform: Driving regulatory reform and system innovation
Driving regulatory reform and system innovation to enable timely access to pediatric rare disease therapies in Canada. Through active engagement with Health Canada and international partners, this priority aims to reduce regulatory barriers across clinical trial development, conduct, and treatment access
Looking Ahead
This strategic plan positions RareKids-CAN to deliver meaningful long-term impact by supporting today’s clinical trials while building a stronger and more connected future for pediatric rare disease research in Canada. By strengthening national coordination, advancing regulatory and system innovation, and centering the needs of children and families, RareKids-CAN is helping ensure that promising therapies are delivered more equitably and efficiently.