Frequently Asked Questions

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General

  • There is currently no definition of a rare disease in Canada like there is the EU and the United States. Our network has defined a rare disease to be a condition that has an ORPHA code on orpha.net or a prevalence of＜1 in 2,000.

  • We support clinical trial projects that meet the following criteria:

    1. Must be an interventional clinical trial (does not need to be regulated by Health Canada, includes behavioural interventions such as mindfulness and music therapy)

    2. The condition must have an ORPHA code or has a prevalence of＜1 in 2,000

    3. Fetal or pediatric population (the clinical trial may involve pregnant individuals as long as the intervention is on the fetus and not the mother)

    4. Non-oncology

    Registries:

    If the project is requesting services from the registry sub-platform, we do offer a consultation/concierge service for longitudinal registries t with one or more of the following potential objectives:

    1. Enhancing readiness for interventional trials (therapies under development

    2. Serving as a source of external controls for single arm trials

    3. Registry based trials

    4. Post trial Real World Evidence generation for Health Technology Assessment processes

I am an investigator:

  • To find out more about our services, please visit our services page, or check out our brochure for investigators. Additionally, we are happy to meet with investigators to discuss how our services can be tailored to your needs. Please complete the form on the contact us page, and we will contact you to schedule a meeting.

  • We offer a range of services to support your research from basic science to clinical trials in human. Examples of our services include:

    • Connecting you with our pharmacology team for dosing and formulation

    • Grant application support

    • Protocol design with expertise from our design and methods team as well as our biostats and methods team

    • Patient and family engagement

    To find out what services are available to support your clinical trial idea, visit our services page, or complete the form on the contact us page, and we will and we will reach out to discuss your ideas further and how we may help.

  • We have a team of experts, from biostatisticians, pharmacologists, patient and family partners as well as the clinical trial coordinating hub who can provide guidance and feedback on the design of your project. 

    To find out what services are available to support your clinical trial idea, visit our services page, or complete the form on the contact us page, and we will and we will reach out to discuss your ideas further and how we may help.

  • We provide Regulatory Support and Submissions to Health Canada services by assisting study teams in the preparation and submission of clinical trial applications, investigational testing authorizations, and single-patient study applications for regulated clinical trials to Health Canada. We also assist in adverse event reporting.

  • We offer Monitoring and Quality Assurance services, and have a dedicated monitoring team to perform risk-based remote and on-site monitoring to meet the Health Canada required sponsor obligations. Please complete the form on the contact us page, and we will and we will reach out to discuss further.

  • To access our validated REDCap and other tools for your project, please submit a project request using our project request form. We will then schedule a meeting to discuss your project needs.

  • We have multiple avenues to advertise your research project that fits within the RareKids-CAN scope. We advertise clinical trials through our newsletter that is distributed to network members and patient/family partners. We can also advertise your clinical trial through our social media platforms and website.

    Additionally, we have 16 Clinical Trial Navigators based at our affiliated institutions. Our clinical trial navigators are greatly positioned to contact their local investigators for potential collaboration for your clinical trial.

  • We have an expertise database with over 120 investigators ready to be contacted for clinical trial opportunities at our 17 affiliated pediatric research institutions and affiliated hospitals across  Canada. We are uniquely positioned to facilitate connections and collaborations between investigators across Canada. We have seen success with our unique approach and have connected several investigators already.

    We also provide networking and learning opportunities through our webinars. Upcoming webinar events are listed on our events page as well as our monthly newsletter. To join our newsletter, please submit your request via the form on the contact us page

  • We are always excited to see experts interested in contributing to RareKids-CAN! Whether you’re a physician, dentist, nurse practitioner, methodologist, psychologist, physiotherapist, dietician, or other healthcare professional, your expertise is vital to supporting a range of pediatric rare disease clinical trials and access to treatment in Canada.

    Some of the ways you can contribute is to join our expertise database, where you can indicate whether you are interested in being a:

    • Clinical expert

    • Methodology expert

    • Willing to be contact to be a DSMB member

    • Willing to be contacted as an investigator in your area of expertise

    • Willing to be contacted as a clinical expert to advise Canadian Drug Agency (CDA) on a product within your area of expertise

    To join the database, please complete the form on the contact us page, and we will send you the form to be a member of the expertise database which will take no longer than 2 minutes to complete.

I am from industry/biotech:

  • RareKids-CAN is embedded within MICYRN which is a standalone non-for profit legal entity. Additionally, we can serve as the sponsor for your clinical trial.

    We also have an expertise database with over 100 investigators ready to be contacted for clinical trial opportunities at our 17 affiliated institutions in Canada. We are uniquely positioned to facilitate site and investigator identification. We have already seen success through our personal and tailored approach whereby industry have received a more positive outcome and response from investigators.

    For more information, please complete the form on the contact us page, and we will contact you to schedule a meeting.

I am a patient/ family of a child with a rare condition:

  • We welcome children, adolescents, young adults with lived or living experience and their families to join the network, your insights are highly valuable. If you are interested in getting involved, please complete the form on the contact us page, and we will contact you.

    Opportunities may include joining our Patient and Family Engagement sub-platform to provide feedback on network initiatives or specific projects, such as reviewing study protocols, consent forms, and communications shared within the network.

  • Ongoing research opportunities are currently shared via our website as well as our social media platforms such as LinkedIn, and Instagram. Follow us to keep up to date!

    We are also working very hard behind the scenes to bring more research opportunities to you, so stay tuned!

  • We appreciate your feedback and suggestions. Please submit the form on the contact us page. We may reach out to you for additional information.

Research Training and Events:

  • We believe that every researcher should have the opportunity to grow and gain confidence in their role by continuously expanding their research knowledge. Through our Training and Mentorship program, we fund 2 training award per year through the IMPaCT Training Program. IMPACT is a clinical trial training platform funded by the Canadian Institutes of Health Research. Their goal is to increase clinical trial capacity in perinatal and child health in Canada.

    We also share training opportunities with network members through our newsletter, and via our social media platforms such as LinkedIn, and Instagram, as well as our events page.

  • To discuss sponsorship opportunities, please submit the form on the contact us page to share details about yourself, and we will reach out to discuss further.

  • We encourage knowledge sharing and collaboration within the network and offer opportunities for guest speakers and experts to present at our meetings and events. If you would like to present, please submit the form on the contact us page to share details about your topic, and we will reach out to discuss further.

  • After an event, you should receive an email with instructions how to submit your expenses for reimbursement. If you did not receive an email with instructions or have additional questions, please submit the form on the contact us page form and we will reach out to discuss further.