Building National Readiness for Pediatric Rare Disease Trials: Q&A with Davy Eng, RareKids-CAN Clinical Trial Navigator

RareKids-CAN’s Clinical Trial Navigators help strengthen pediatric rare disease clinical trial readiness across Canada by supporting study start-up, facilitating collaboration between institutions, and helping connect investigators and families to research opportunities.

Clinical Trial Navigators work within institutions while contributing to a coordinated national network focused on improving access to clinical trials and treatment for children and youth living with rare diseases.

For the final installment of RareKids-CAN’s four-part Clinical Trials Day Q&A series, we spoke with Davy Eng, Manager of the Pediatric Clinical Research Unit at CHU de Québec–Université Laval and Clinical Trial Navigator for RareKids-CAN.

RareKids-CAN: Can you describe your role alongside your work as a Clinical Trial Navigator at your institution?

Davy Eng: Since April 2022, I have served as the Manager of the Pediatric Clinical Research Unit (PCRU) at CHU de Québec–Université Laval, where my team and I support more than 20 investigators.

In my current role, I lead the negotiation of confidentiality agreements and budgets, evaluate study feasibility, coordinate site selection visits, and oversee ethical and regulatory processes from project initiation through close-out. I also participate in Clinical Trial Agreement (CTA) negotiations in close collaboration with our legal team.

In parallel, I support pediatric residents in the development of their research projects when needed.

I have served as a Clinical Trial Navigator for the RareKids-CAN network since April 2024.

“What motivates me most is contributing to greater access to clinical trials and advancing research for children and families affected by rare diseases communities that often lack visibility, advocacy, and equitable access to research opportunities.”

RKC: What is a Clinical Trial Navigator, and why is this important?

DE: A Clinical Trial Navigator (CTN) is a dedicated professional who serves as a central point of contact to support the planning, initiation, and coordination of clinical trials, particularly in complex fields such as pediatric rare diseases.

In the RareKids-CAN network, the CTN acts as the local liaison for all pediatric rare disease clinical trial inquiries, working closely with investigators and study teams to ensure timely and efficient study start-up. The CTN also supports the feasibility assessments, coordinates local data collection for performance metrics and quality improvement, and participates in regular meetings to share best practices.

This role is important because pediatric rare disease trials face significant challenges, including small patient populations, complex protocols, and limited resources. By streamlining communication, improving study feasibility and start-up timelines, and strengthening local and national collaboration, the CTN helps increase access to clinical trials, enhances trial quality and efficiency, and ultimately accelerates research and innovation for children with rare diseases.

RKC: What drew you to working in pediatric rare disease clinical trials?

DE: What motivates me most is contributing to greater access to clinical trials and advancing research for children and families affected by rare diseases communities that often lack visibility, advocacy, and equitable access to research opportunities.

RKC: Clinical Trial Navigators are often described as the “boots on the ground” of RareKids-CAN. What does that look like in practice at your institution?

DE: At CHU de Québec–Université Laval, being a “boots on the ground” Clinical Trial Navigator means serving as the local point of contact for pediatric rare disease clinical trials, supporting feasibility assessments, and working directly with investigators and study teams to ensure timely and efficient study start-up.

I also contribute to the recruitment of investigators for the Expertise Database and support performance measurement and quality improvement initiatives, while remaining actively engaged with the national RareKids-CAN network to share and implement best practices.

RKC: What makes the RareKids-CAN Clinical Trial Navigators network unique compared to working in a single institution?

DE: What makes the RareKids-CAN Clinical Trial Navigators (CTN) network unique is that it connects locally embedded expertise across institutions into a coordinated national system dedicated to pediatric rare disease clinical trials.

“Rather than working in isolation, CTNs share best practices, receive mentorship, and collaborate through a common infrastructure that helps streamline study start-up, align processes across jurisdictions, and build national capacity.”

Rather than working in isolation, CTNs share best practices, receive mentorship, and collaborate through a common infrastructure that helps streamline study start-up, align processes across jurisdictions, and build national capacity. This networked approach strengthens site readiness, improves trial efficiency, and expands access to pediatric rare disease clinical trials across Canada beyond what a single institution could achieve alone.

RKC: How does being part of a national network improve access to or quality of clinical trials for rare disease patients?

DE: Being part of a national network like RareKids-CAN improves access to and quality of clinical trials for rare disease patients by connecting sites, investigators, and patients through a shared, pan-Canadian infrastructure.

This coordination helps streamline study start-up across jurisdictions, identify eligible participants more efficiently, and attract international pediatric rare disease trials to Canada.

By harmonizing processes and supporting collaboration and capacity building, the network enables faster, higher-quality trials and earlier access to innovative therapies for children and youth with rare diseases.

RKC: What challenges are unique to pediatric rare disease clinical trials?

DE: Pediatric rare disease clinical trials face unique challenges, including very small and geographically dispersed patient populations that make recruitment difficult.

With fewer sites available, patients may need to travel long distances, increasing logistical complexity. These trials often involve multiple vendors and extensive staff training, as well as complex, heterogeneous diseases with limited natural history data. Stricter pediatric ethical and regulatory requirements, combined with the burden placed on children and families, further add to the complexity of planning and conducting these studies.

RKC: What would you want people outside the research world to better understand about pediatric rare disease clinical trials?

DE: I would want people to understand that pediatric rare disease clinical trials are often the only path to diagnosis, treatment, or hope for many children and families.

These trials are much more complex than they may appear because patient populations are very small, studies are highly specialized, and families often face significant travel and time burdens. Progress can seem slow, but each trial represents an enormous collaborative effort aimed at improving care where few or no options currently exist.

RKC: What excites you most about the future of rare disease clinical trials?

DE: What excites me most is the growing pan-Canadian collaboration in rare disease research. Networks like RareKids-CAN are helping streamline processes, share expertise, and attract more pediatric rare disease clinical trials to Canada.

These initiatives will significantly improve access to trials and accelerate the development of much-needed treatments for children living with rare diseases in Canada.