What is RareKids-CAN?

RareKids-CAN is a diverse coalition of partners focused on simplifying and speeding up clinical trials and access to innovative treatments for pediatric (children and youth) rare diseases across Canada.

RareKids-CAN operates through five main thematic pillars that follow the life cycle of a research project to drug market access.

  • We have established a Clinical Trial Operations and Coordinating Hub (CTOCH) aimed at uniting the rare disease research community. This central hub will link children, adolescents, young adults and their families with registry opportunities for the collection of natural history data, expedite the identification of eligible participants, facilitate patient access to pediatric rare disease clinical trials, connect sponsors with research teams, and attract international trials.  

  • We translate therapies developed by private and public sectors into efficient clinical trials. It involves developing the knowledge base required for successful clinical trial design and execution. This theme also emphasizes mentorship and training to build capacity for the next generation of researchers and patient/family partners. 

  • Addresses the operational setup of pediatric rare disease clinical trials in Canada, aiming to overcome delays and barriers that often hinder efficient launches and deter Canadian participation. The focus is on streamlining multi-jurisdictional processes involving data sharing, ethics, contracts, patient enrolment, and budgeting.  

  • In partnership with the Maternal Infant Child and Youth Research Network (MICYRN), we offer Academic Research Organization (ARO) services to ensure efficient trials. We strive to elevate local site standards; diversify participation; address barriers, such as age transitions during trials; and ensure the inclusion of individuals in remote or rural regions, such as through de-centralization approaches.   

  • Translating evidence from clinical trials into real-world application and care advancement. It goes beyond clinical trials; involving real-world evidence, post-market surveillance, health economics, and increased rare disease drug submissions for authorization of commercialization to Health Canada. RareKids-CAN aims to bridge the gap between knowledge and action, ensuring innovations benefit patients, and ultimately improving the lives of children with rare diseases. 

These five themes are underpinned by five cross-cutting platforms (15 sub-platforms):  

1. Expertise and Innovation 

2. Capacity Building and Knowledge Exchange 

3. Information Technology and Integrated Data Science 

4. Inclusivity and Lifespan Engagement 

5. Regulatory Affairs and Strategic Partnership 

RareKids-CAN operates as an Academic Research Organization, bringing advanced expertise and comprehensive support to develop and conduct top-quality clinical trials for children and youth affected by rare diseases. 

  • • Biostatistical Methods
    • Methods and Design
    • Pharmacology

  • • Training and Mentorship
    • Knowledge Synthesis and Mobilization

  • • Real World Data
    • Registry
    • Data Coordinating Centre

  • • Patient and Family Engagement
    • EDI+ Indigeneity
    • Sex and Gender
    • Transition to Adulthood

  • • Health Canada Relations and Advocacy
    • Health Economics, HTA and Reimbursement
    • Sustainability and Business Development

Mission

Establish a cross-jurisdictional platform that develops, manages, and executes cutting-edge clinical trials, providing treatment options and support for children, adolescents, young adults, and their families affected by rare diseases in Canada.

Vision

To ensure that every child, adolescent, and young adult in Canada affected by rare diseases has access to effective and innovative treatments.

Objectives 

Infrastructure:

Develop a platform to support pediatric rare disease clinical trials (PRDCT) in Canada​ 

Training:

Increase the capacity to perform PRDCTs in Canada by offering training and mentorship opportunities​ 

Attraction:

Attract international PRDCTs, increasing investments in Canada and providing earlier access to innovative therapies to people living in Canada​ 

Inclusivity:

Foster diversity and inclusion in and improve access to PRDCTs​ 

Data:

Implement processes to acquire data from clinical trials, real-world registries, health economics evaluation, and post-marketing surveillance to inform assessment reviews throughout the whole drug/therapy cycle​ 

Authorization:

Increase the number of new rare disease drug submissions for authorization of commercialization to Health Canada​