RareKids-CAN:
Pediatric Rare Disease Clinical Trials and Treatment Network
Out of the 7,000 known rare diseases, only about 5% have specific treatments available. Patients and families often need to travel to other countries and pay out of pocket for experimental treatments. RareKids-CAN is working to change this by supporting national and international clinical trials. Our goal is to support groundbreaking research and advance access to therapies for children, adolescents, young adults, and their families here in Canada.
Who We Are
RareKids-CAN is funded by the Canadian Institutes of Health Research Institute of Genetics, as part of the Government of Canada’s National Strategy for Drugs for Rare Diseases investment.
We collaborate with:
Patient and family partners
Patient organizations
Investigators and research networks
Industry partners
Our focus is on supporting the high quality design, and execution of pediatric rare disease clinical trials in Canada and enabling access to treatments.
Our 3 Priorities
STRATEGIC PRIORITY 1: Strengthening Pediatric Rare Disease Clinical Trial Capacity and Advanced Therapy Medicinal Product Readiness
STRATEGIC PRIORITY 2: Optimizing Participant/Site/Trial Matching
STRATEGIC PRIORITY 3: Driving Regulatory Reform and System Innovation
Our Services
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FOR INVESTIGATORS
• Pre award consultations
• Regulatory submission support
• Ethics submission
• Database services
• Registry concierge service -
PATIENT & FAMILY RESOURCES
• Advocacy toolkits
• Engagement support -
BIOTECH & INDUSTRY
• Investigator and site identification
• Access to expertise and patient partner input
• Regulatory support services
Affiliated Institutions
We are partnered with 16 pediatric research institutions and their affiliated hospitals across Canada, uniquely positioning us to facilitate meaningful connections between investigators and collaborators. We have a database with over 180 investigators and experts from these institutions who are ready to collaborate on your project.
Featured Spotlight
RESEARCH SPOTLIGHT
Rewriting Rare Disease Care
May 25, 2026Canada is entering a new era in pediatric rare disease research, driven by advances in precision medicine and novel, innovative clinical trial approaches, including gene editing and gene therapies.
Read MoreClinical Trial Navigators
Strengthening Collaboration Across Canada
May 20, 2026As part of RareKids-CAN’s four-part Clinical Trials Day Q&A series, we spoke with Humaira Ahmed, Research Coordinator and Clinical Trial Navigator at McMaster Children’s Hospital.
Read the Q&APRESS RELEASE
RareKids-CAN supports Bill C-265 to improve access to treatments for children with rare diseases
May 8, 2026RareKids-CAN, Canada’s pediatric rare disease clinical trials and treatment network funded by the Canadian Institutes for Health Research, supports Bill C-265 which improves access pathways for medical therapies not commercially available in Canada.
Learn more about the bill