{"id":1649,"date":"2024-08-19T15:49:12","date_gmt":"2024-08-19T15:49:12","guid":{"rendered":"https:\/\/lab.research.sickkids.ca\/enrich\/?page_id=1649"},"modified":"2026-02-06T15:07:08","modified_gmt":"2026-02-06T15:07:08","slug":"rarekids-can","status":"publish","type":"page","link":"https:\/\/lab.research.sickkids.ca\/enrich\/research-projects\/rarekids-can\/","title":{"rendered":"RareKids-CAN"},"content":{"rendered":"

\"\"<\/a>RareKids-CAN: The New Pediatric Rare Disease Clinical Trials and Treatment Network<\/span><\/strong><\/p>\n

To ensure that every child, adolescent, and young adult in Canada affected by rare diseases has access to effective and innovative treatments.<\/em><\/span><\/p>\n

Out of the 7,000 known rare diseases, only about 5% have specific\u00a0treatments available. Patients and families often need to go to other countries and pay themselves for experimental treatments. RareKids-CAN<\/strong> 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.<\/span><\/p>\n

Funded in early 2024 by the Canadian Institutes of Health Research Institute of Genetics, as part of the<\/span> Government of Canada’s National Strategy for Drugs for Rare Diseases investment<\/a><\/span>, 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<\/strong>.<\/span><\/p>\n

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

Find Out More About RareKids-CAN<\/a><\/span><\/p>\n

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Winter 2026 Update:<\/strong><\/span><\/p>\n

In 2025, RareKids-CAN and its Methods Sub-Platform expanded their national infrastructure to accelerate paediatric rare disease clinical trials.<\/span><\/p>\n

The Design and Methods Sub-Platform is leading the R<\/strong>are Disease Clinical Trial O<\/strong>utcome A<\/strong>ssessment and D<\/strong>esign (ROAD<\/strong>) Project, a national and international initiative focused on improving how outcomes are designed, selected, measured, analysed, and reported in paediatric rare disease trials. The goal is to ensure that future trials generate evidence that is meaningful to patients and families, while also meeting modern scientific standards, international regulatory expectations, and industry needs.<\/span><\/p>\n

What we are building<\/strong><\/span><\/p>\n

We are developing two ROAD Checklists <\/strong>to support best practices in outcome design for paediatric rare disease clinical trials:<\/span><\/p>\n