Frequently Asked Questions

There is currently no formal definition of a rare disease in Canada. RareKids-CAN defines a rare disease as a condition with an ORPHA code on Orphanet or a prevalence of fewer than 1 in 2,000 people. 

RareKids-CAN supports pediatric rare disease clinical trial projects that meet the following criteria: 

• Interventional clinical trials, including behavioural interventions 
• Conditions with an ORPHA code or prevalence under 1 in 2,000 
• Fetal, pediatric, adolescent, or young adult populations 
• Non-oncology studies 

For pediatric rare disease registries, complimentary consultation and concierge support are available for registries being developed with a clear clinical trial or evidence-generation purpose. 

To qualify for this service, the registry must be intended to support one or more of the following objectives: 

• Readiness for future interventional trials  
• Use as an external control for single-arm trials  
• Registry-based clinical trials  
• Real-world evidence generation and health technology assessment activities  

Consultation support may include guidance on registry governance, core data elements, platform and hosting considerations, and alignment with future clinical trial and evidence-generation needs. 

We offer flexible support across the clinical trial lifecycle — from early planning through implementation and execution. Visit our Services pages or submit a consultation request to discuss how our team can support your project. 

Yes. RareKids-CAN brings together experts across methods and design, biostatistics, pharmacology, patient and family engagement, and our Clinical Trial Operations and Coordinating Hub (CTOCH) to help move ideas into action. 

Whether you’re developing a protocol, preparing a grant application, or refining a study concept, our team can help identify the next steps and connect you with the right expertise. 

RareKids-CAN supports projects across the clinical trial journey. Services may include: 

• Study design and methodological guidance 
• Pharmacology expertise 
• Biostatistical consultation 
• Patient and family engagement 
• Knowledge mobilization support 
• Grant and protocol development support 

We recognize that every project is different and tailor support to your needs. 

Does RareKids-CAN provide support for regulatory submissions? 

Yes. RareKids-CAN provides support for Canadian regulatory submissions for pediatric rare disease clinical trials. This includes guidance and support for pre-Clinical Trial Application (CTA) meetings with Health Canada, preparation of CTA materials, including quality and manufacturing sections, and support throughout the submission process. We also have a cell and gene therapy consultant that provides guidance to study teams during the pre-clinical stages of their project development 

We also provide support for ongoing regulatory activities after approval, including amendments, notifications, and other required communications with Health Canada. 

In addition to drug clinical trials, RareKids-CAN offers tailored regulatory support for medical device clinical trials through the Investigational Testing Authorization (ITA) pathway, as well as support for natural health product clinical trials. 

Please note that RareKids-CAN’s regulatory support is focused on submissions and regulatory processes within Canada. 

Yes. RareKids-CAN provides support for Canadian regulatory submissions for pediatric rare disease clinical trials. This includes guidance and support for pre-Clinical Trial Application (CTA) meetings with Health Canada, preparation of CTA materials, including quality and manufacturing sections, and support throughout the submission process. We also have a cell and gene therapy consultant that provides guidance to study teams during the pre-clinical stages of their project development 

We also provide support for ongoing regulatory activities after approval, including amendments, notifications, and other required communications with Health Canada. 

In addition to drug clinical trials, RareKids-CAN offers tailored regulatory support for medical device clinical trials through the Investigational Testing Authorization (ITA) pathway, as well as support for natural health product clinical trials. 

Please note that RareKids-CAN’s regulatory support is focused on submissions and regulatory processes within Canada. 

Yes. Through MICYRN, our monitoring and quality assurance teams provide risk-based support to help study teams meet sponsor and regulatory requirements in Canada. 

Yes. We offer a validated REDCap database through the Women and Children’s Health Research Institute at the University of Alberta including the database build and management for ATMP and N of 1/few pediatirc rare disease clinical trials.  

Yes. Through our national network, RareKids-CAN helps increase visibility for eligible studies and connect investigators with collaborators across Canada. 

Our network includes: 

• 16 pediatric research institutions and affiliated hospitals 
• Clinical Trial Navigators across Canada 

A National Expertise Database with more than 180 clinical and methodological experts 

We are always looking to grow our community of experts and collaborators. You can join our National Expertise Database and indicate your interest in: 

• Clinical expertise 
• Methodology expertise 
• Data Safety Monitoring Boards 
• Clinical trial opportunities 

Advisory opportunities 

RareKids-CAN offers a national point of engagement for investigators, biotech and industry partners looking to conduct pediatric rare disease clinical trials in Canada. 

Support may include: 

• Site and investigator identification 
• Access to clinical and methodological expertise 
• Trial feasibility support 
• Regulatory guidance 
• Clinical trial operations support 
• Connections with patient and family partners 

Yes. Through our national network and expertise database, we help connect partners with investigators and institutions aligned with project needs. Our goal is to simplify collaboration and reduce duplication across trial start-up activities. 

We welcome conversations about collaboration and partnership opportunities.  

Clinical trials are research studies that help determine whether treatments, therapies, or approaches are safe and effective. Participation helps researchers better understand rare diseases and improve care and treatment options. 

Patients and families are central partners in RareKids-CAN. We provide access to educational resources, engagement opportunities, clinical trial information, and tools that support meaningful participation in research. 

RareKids-CAN works to connect patients and families with research opportunities and improve awareness of pediatric clinical trials through our national network and Clinical Trial Operations and Coordinating Hub (CTOCH). 

We believe patient and family voices strengthen research and improve outcomes. 

There are many ways to get involved, including: 

• Participating in research studies 
• Joining patient and family partnership activities 
• Sharing lived experiences 
• Participating in events and educational opportunities 

RareKids-CAN is not a diagnostic service and does not provide medical diagnosis or clinical care. If you are looking for help with diagnosis, genetic testing, treatment, or other medical questions, your child’s healthcare team is the best place to start.

RareKids-CAN’s role is focused on pediatric rare disease clinical trial research, helping to strengthen research opportunities and bringing patients, families, researchers, and other partners together.

RareKids-CAN brings together people with lived experience of pediatric rare disease and the research community to help advance pediatric rare disease clinical trials in Canada. RareKids-CAN also offers resources to explore, including a glossary.

People with lived experience of pediatric rare diseases can become involved as partners in research, sharing their experiences and perspectives to help make pediatric rare disease clinical trials more meaningful, relevant, accessible, and responsive to the needs of families.

RareKids-CAN provides a searchable clinical trial database where patients and families can discover ongoing pediatric rare disease clinical trials in Canada. Additionally, RareKids-CAN shares information about relevant research opportunities through its website, social media, newsletter, and other network channels. 

However, RareKids-CAN is not a replacement for your child’s healthcare team. Eligibility for a particular clinical trial is determined by the research team based on the study’s requirements.

No. You can become involved in RareKids-CAN as a patient or family partner even if you are not currently participating in a pediatric rare disease clinical trial.

There may be opportunities to provide feedback on research priorities, study materials, communications, protocols, consent forms, or other network activities.

Yes. RareKids-CAN regularly offers webinars, training opportunities, and educational events designed for researchers, clinicians, patients, families, and partners.  

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