Strengthening pediatric rare disease research 

We are committed to helping researchers build high-quality, impactful studies. We provide consultations with experts in methods and design, pharmacology, biostatistics, knowledge mobilization, and regulatory affairs, we can help refine your protocol, strengthen your grant application, and position your project for success.

This service is free for all investigator initiated pediatric rare disease clinical trials in Canada.

Whether you’re launching a new study or managing an ongoing trial, our team provides project specific electronic case report form (eCRF) development and database management in a validated REDCap database hosted by the Women and Children’s Health Research Institute.

We work with researchers to build secure, high-quality databases that support efficient data collection and study management throughout the clinical trial lifecycle.

This service is available to eligible clinical trial projects involving one of the following therapeutic approaches:

• Antisense oligonucleotide (ASO) therapy
• Cell therapy
• Gene therapy

For eligible projects, database build is provided at no cost. Ongoing data management services are available on a cost-recovery basis. 

Simplify ethics approvals for multi-jurisdictional clinical trials. We facilitate centralized ethics submissions to help streamline the review process, reduce administrative burden, and support more efficient study start-up across participating sites.

This service is free for investigator initiated pediatric rare disease clinical trials.

Whether you’re establishing a new registry or enhancing an existing one, our experts provide free strategic guidance on registry design, ethics, governance, and best practices. We help ensure your registry is designed to support high-quality clinical research and is optimized to meet your research objectives.

We offer support to project teams looking to develop longitudinal registries with one or more of the following objectives: 

• Support post-trial real-world evidence generation for Health Technology Assessment (HTA) purposes
• Enhance trial readiness by identifying participants for therapies in development
• Serve as a source of external control data for single-arm clinical trials
• Enable registry-based clinical trials

Access practical tools, templates, guidance documents, and shared resources developed by experts across our network to support pediatric rare disease clinical trials.

Designed to reflect best practices and real-world experience, these resources help researchers navigate trial planning, implementation, and delivery.

Access expert-led training and knowledge sharing opportunities designed to strengthen pediatric rare disease clinical trial capacity across Canada.

RareKids-CAN supports learning and capacity-building through webinars, training opportunities, and educational resources for researchers, clinicians and healthcare providers. Our workshops and educational initiatives bring together experts from across the country to share knowledge, best practices, and practical insights to support clinical trial development and delivery.