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

Rare diseases (RDs) affect almost 5% of the population. One in 15 children is born with a RD. Patients with these conditions occupy a quarter of pediatric hospital beds. The journey of diagnosis of RDs and access to treatment, when available, can be long and often distressing. Canada is currently behind other countries in providing access to specific treatments for children with a RD. There is an urgent need to bring together stakeholders in the RD community. The goal is to increase access to therapies for all Canadian children with a RD. The initiative we propose is called RareKids-CAN: A Pediatric Rare Disease Clinical Trials and Treatment Network. RareKids-CAN aims to build a platform with experts from across Canada. This will include RD experts, pediatric healthcare centres, patient/family partners and organizations, and decision makers. RareKids-CAN will provide services through 15 Sub-Platforms coordinated by an Operations Hub. RareKids-CAN will enable the development, conduct and execution of high-quality clinical trials (CT) for children with RDs. RareKids-CAN consists of 5 main themes. Theme 1 is "Creating Connections." We will create a one-shop stop for easy access to CT opportunities. We will connect patients and families with researchers, and industry sponsors. Theme 2 is "Clinical Trial Preparedness" and aims to support CT and patient registry designs. Theme 2 also includes mentorship and training opportunities to build capacity for the next generation of researchers and patient/family partners. Theme 3 aims to accelerate CT start-up processes. We will work to reduce delays and barriers that impact participation in CT. Theme 4 facilitates the enrolment of children in CT. Theme 4 will break down participation barriers, address inequity, and support the inclusion of children in remote locations. Finally, theme 5 will take evidence generated from CT into real-world application. Patient engagement and advocacy play a crucial role in all themes.