# FAQs Frequently Asked Questions General How does RareKids-CAN define a rare disease? + 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.  What types of projects are eligible for RareKids-CAN support? + 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.  Request a consultation For Researchers  How can I learn more about RareKids-CAN services? + 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.  I have an idea for a clinical trial but don't know where to start. Can RareKids-CAN help? + 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.  Please submit a Consultation Request How does RareKids-CAN support research beyond trial implementation? + 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.  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.  Can RareKids-CAN support compliance and quality assurance? + Yes. Through MICYRN, our monitoring and quality assurance teams provide risk-based support to help study teams meet sponsor and regulatory requirements in Canada.  Can RareKids-CAN help with a clinical trial database?+ 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.   Can RareKids-CAN help  recruit study participants or connect with collaborators? + 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  Get to know the team How can I contribute my expertise? + 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  Learn more about our Expertise Database For Biotech & Industry I am planning a pediatric rare disease clinical trial in Canada. How can RareKids-CAN help? + 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  Learn more about our Biotech & Industry Services Can RareKids-CAN help connect us with investigators and sites? + 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.  Are sponsorship opportunities available? + We welcome conversations about collaboration and partnership opportunities.   Reach out for more information For Patients & Families What is a pediatric rare disease clinical trial? + 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.  How can RareKids-CAN support patients and families? + 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.  Learn more here Can RareKids-CAN help me find research opportunities? + 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).  Learn more about current Clinical Trials How can patients and families get involved? + 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  Learn more here Can RareKids-CAN help my child get a rare disease diagnosis?+ 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. What does RareKids-CAN do for patients and families?+ 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. Can RareKids-CAN help me find a clinical trial for my child? + 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. If I want to engage as a partner with RareKids-CAN in helping advance pediatric rare disease clinical trials, does my child or family need to be enrolled in a clinical trial?+ 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. Research, Training & Events Does RareKids-CAN offer webinars or training? + Yes. RareKids-CAN regularly offers webinars, training opportunities, and educational events designed for researchers, clinicians, patients, families, and partners.   Learn more about our current events How can I stay informed? + You can stay up to date by subscribing to our newsletter, following us on social media, and visiting our Events and News pages.  Subscribe here