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

 

Winter 2026 Update:

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

The Design and Methods Sub-Platform is running ROAD (Rare Disease Clinical Trial Outcome Assessment and Design) Project. ROAD initiative aims to strengthen trial outcome design so that future paediatric rare disease trials reflect patient and family perspectives, while meeting modern scientific standards, international regulatory expectations, and industry needs.

We are developing a checklist and manual to support the highest standards for outcome design in paediatric rare disease clinical trials. A checklist draft has been built through:

  • findings from our needs survey of trialists;
  • a workshop with rare disease patient & family partners;
  • consultation with methodological and clinical experts;
  • a review of relevant regulatory guidance (FDA and EMA);
  • alignment with international trial reporting guidelines (SPIRIT-Outcomes, CONSORT-Outcomes, SPIRIT-PRO).

Upcoming activities: PPI workshops, International Advisory Board meetings to refine and finalize the ROAD checklist, followed by a structured expert voting process, piloting of the checklist, and knowledge translation through publications and a 6-episode podcast series.

Stay tuned for more announcements and opportunities to get involved.

 

Updated December 2025