About RareKids-CAN

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A scientist search for record in the cabinet

What is RareKids-CAN?

RareKids-CAN is a diverse coalition of partners focused on simplifying and speeding up clinical trials and access to innovative treatments for pediatric (children and youth) rare diseases across Canada.

Objectives

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A consultant presenting and training

Attract international PRDCTs, increasing investments in Canada and providing earlier access to innovative therapies to people living in Canada​

Foster diversity and inclusion in and improve access to pediatric rare disease clinical trials

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Drugs safely authorized with ticks

Implement processes to acquire data from clinical trials, real-world registries, health economics evaluation, and post-marketing surveillance to inform assessment reviews throughout the whole drug/therapy cycle​

Increase the number of new rare disease drug submissions for authorization of commercialization to Health Canada​

Training

Infrastructure

Increase the capacity to perform pediatric rare disease clinical trials in Canada by offering training and mentorship opportunities​

Develop a platform to support pediatric rare disease clinical trials in Canada​ 

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Whole of Canada map

Inclusivity

Attraction

Authorization

Data

RareKids-CAN operates through five main thematic pillars that follow the life cycle of a research project to drug market access:

  • We have established a Clinical Trial Operations and Coordinating Hub (CTOCH) aimed at uniting the rare disease research community. This central hub will link children, adolescents, young adults and their families with registry opportunities for the collection of natural history data, expedite the identification of eligible participants, facilitate patient access to pediatric rare disease clinical trials, connect sponsors with research teams, and attract international trials.

  • We translate therapies developed by private and public sectors into efficient clinical trials. It involves developing the knowledge base required for successful clinical trial design and execution. This theme also emphasizes mentorship and training to build capacity for the next generation of researchers and patient/family partners.

  • Addresses the operational setup of pediatric rare disease clinical trials in Canada, aiming to overcome delays and barriers that often hinder efficient launches and deter Canadian participation. The focus is on streamlining multi-jurisdictional processes involving data sharing, ethics, contracts, patient enrolment, and budgeting.

  • In partnership with the Maternal Infant Child and Youth Research Network (MICYRN), we offer Academic Research Organization (ARO) services to ensure efficient trials. We strive to elevate local site standards; diversify participation; address barriers, such as age transitions during trials; and ensure the inclusion of individuals in remote or rural regions, such as through de-centralization approaches.

  • Translating evidence from clinical trials into real-world application and care advancement. It goes beyond clinical trials; involving real-world evidence, post-market surveillance, health economics, and increased rare disease drug submissions for authorization of commercialization to Health Canada. RareKids-CAN aims to bridge the gap between knowledge and action, ensuring innovations benefit patients, and ultimately improving the lives of children with rare diseases.

These five themes are underpinned by five cross-cutting platforms (15 sub-platforms):

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1

Expertise and Innovation

  • Biostatistical Methods
  • Methods and Design
  • Pharmacology

2

Capacity Building and Knowledge Sharing

  • Training and Mentorship
  • Knowledge Synthesis and Mobilization

3

Information Technology and Data Science

  • Real World Data
  • Registry
  • Data Coordinating Centre

4

Inclusivity and Lifespan Engagement

  • Patient and Family Engagement
  • EDI+ Indigeneity
  • Sex and Gender
  • Transition to Adulthood

5

Regulatory Affairs and Strategic Partnerships

  • Health Canada Relations and Advocacy
  • Health Economics, HTA and Reimbursement
  • Sustainability and Business Development