RareKids-CAN: The New Pediatric Rare Disease Clinical Trials and Treatment Network

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

Out of the 7,000 known rare diseases, only about 5% have specific treatments available. Patients and families often need to go to other countries and pay themselves for experimental treatments. RareKids-CAN wants to change this by supporting national and international clinical trials to make new discoveries and advance treatments for children, adolescents, young adults and their families here in Canada.

Funded in early 2024 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, RareKids-CAN collaborates with patient/family partners, patient organizations, investigators, research networks, and industry partners to design, develop, set up, manage, and execute pediatric rare disease clinical trials in Canada.

To help develop and execute these cutting-edge clinical trials, the EnRICH Lab is involved and Dr. Offringa leads the RareKids-CAN Design and Methods Sub-Platform: https://www.rarekidscan.com/sub-platform-leads

Find Out More About RareKids-CAN

Summer 2025 Update:

In 2025, RareKids-CAN and its Methods Sub-Platform continue to expand their national infrastructure to accelerate paediatric rare disease clinical trials.

The Design and Methods Sub-Platform is developing tailored guidance for trial design in paediatric rare diseases, focusing on trial outcome definition, measurement and best-practice tools to support high-quality, patient-centred data collection. This includes collaborations with family advisors and patient advocacy groups to help ensure that trial designs reflect the priorities and lived experiences of paediatric rare disease communities.

Based on this guidance, a training program for clinical trialists is in development. The program will feature modules on trial outcome co-design with patient partners, alignment with regulatory standards, research ethics, real-world implementation of outcome data capture, and trial reporting.

Activities planned for the coming months:

  • Design of a consensus process with international RD trial experts is ongoing
  • A series of working meetings on trial outcome design for paediatric rare disease trials will take place in fall 2025
  • RareKids-CAN investigators will present their work in progress to a new international Rare Disease Outcome Club during the 27th Society for the Study of Behavioural Phenotypes (SSBP) Meeting in Amsterdam, the Netherlands, in September 2025

Stay tuned for more announcements and opportunities to get involved.

Updated August 2025