McMaster Pediatrics to play key role in new $20M rare disease research network

In Canada, 14,000 children under the age of 15 die each year from a rare disease. With over 7,000 recognized rare diseases, a new collaborative effort is underway, as researchers from McMaster University and across the country join forces to enhance health outcomes for children and teens grappling with these conditions.

On February 28, 2024, the Government of Canada announced it is providing $20 million over the next five years to the Maternal Infant Child and Youth Research Network (MICYRN) for the establishment of RareKIDS-CAN: the Pediatric Rare Disease Clinical Trails and Treatment Network.

The national Network will foster collaboration among researchers, patients, caregivers, healthcare providers, and policymakers. Its objectives include streamlining clinical research processes, supporting national and international clinical trials, and advancing discoveries for the prevention, diagnosis, and treatment of rare diseases in children and adolescents.

Researchers from McMaster University’s Department of Pediatrics, in collaboration with colleagues and families from CanChild, will play pivotal roles on various platforms and working groups within the newly funded network:

• Andrea Cross (Pediatrics) and Sara Pot (CanChild) will co-lead the Patient and Family Engagement sub-platform.
• Michelle Batthish will lead the Transition to Adulthood sub-platform.
• Anne Klassen is a member of the Design and Methods working group.
• Tania Cellucci is a member of the Training and Mentorship and Knowledge Mobilization sub-platform working groups.
• Liane Heale is a member of the Registry sub-platform working group.
• Samantha Micsinszki (CanChild) is a member of the Training and Mentorship and Patient and Family Engagement sub-platform working group.
• Anthony Chan and Lindsay Akrong are co-applicants as members of MICYRN’s Clinical Trials Consortium.

Sara Pot from CanChild, along with Andrea Cross from Pediatrics, who co-leads the Network’s new Patient and Family Engagement platform, expressed, “As a parent of two children who live with a rare diagnosis, I’m encouraged by the work and development of a Canadian rare disease clinical trial research network. Our family, like many with lived experience in the rare disease community, has faced uncommon challenges. I am hopeful this network will create partnership and engagement between families and researchers, as we work together to weave hope amidst uncertainty.”

This investment was made possible through the Canadian Institutes of Health Research Rare Disease Research Initiative.