Building Capacity for Pediatric Rare Disease Research: Q&A with Cara Grobbecker, RareKids-CAN Clinical Trial Navigator

RareKids-CAN’s Clinical Trial Navigators help strengthen pediatric rare disease clinical trial capacity across Canada by serving as a local contact for investigators, sponsors and clinical trial opportunities.

They also liaise with investigators and study teams to support timely, efficient study start up, coordinate local metrics collection, identify barriers, improve coordination, and support families and researchers navigating complex clinical trial environments.

As part of RareKids-CAN’s four-part Clinical Trials Day Q&A series, we spoke with Cara Grobbecker, Research Coordinator and Clinical Trial Navigator at Children’s Hospital, London Health Sciences Centre.

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

Cara Grobbecker: I work with several investigators across pediatric specialties, primarily neurology and endocrinology, working on Phase I–IV investigator-initiated and industry-sponsored trials.

In this role, I support all aspects of the clinical trial lifecycle, including study start-up, ethics and regulatory submissions, participant recruitment and study visits, biological specimen collection and processing, data collection, monitoring and audit preparation, through study close-out.

Locally, we are also developing a Southwestern Ontario Rare Disease (SWORD) program to better understand and support the needs of patients affected by rare disease. Through this work, we aim to improve patient identification, connect families with clinical trial opportunities, and strengthen links to community resources.

“By improving coordination and reducing delays, Clinical Trial Navigators enable more efficient trial delivery, strengthen Canada’s ability to attract industry-sponsored studies, and expand access to clinical trials for Canadians affected by rare disease.”

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

CG: A Clinical Trial Navigator serves as a central point of contact to connect patients, investigators, and industry sponsors with clinical trial opportunities while supporting the growth of pediatric rare disease research.

At the local level, we complete feasibility assessments, identify barriers, connect teams with resources, and help facilitate efficient study start-up. Nationally, we collect trial metrics, collaborate to identify system-level challenges, share best practices, and build capacity across institutions.

This role is particularly important in rare disease research, where trials are often limited and highly specialized, and where many patients rely on clinical trials for potential treatment options. As advanced therapy medicinal products (ATMPs) such as gene and cell therapies continue to emerge, it is critical that we are equipped locally and nationally to support these trials.

By improving coordination and reducing delays, Clinical Trial Navigators enable more efficient trial delivery, strengthen Canada’s ability to attract industry-sponsored studies, and expand access to clinical trials for Canadians affected by rare disease.

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

CG: Through my work in pediatric clinical research, I became increasingly interested in the challenges of designing and delivering trials in rare diseases, where patient populations are small and timelines are critical.

“I became increasingly interested in the challenges of designing and delivering trials in rare diseases, where patient populations are small and timelines are critical.”

While individual rare diseases affect relatively small numbers of patients, collectively they represent a significant portion of our community. I was drawn to the opportunity to contribute not only at the study level, but also at a systemic level, helping to build the infrastructure and connections needed to support sustainable, high-quality research programs for children and families affected by rare disease.

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?

CG: In practice, being the “boots on the ground” of RareKids-CAN means being closely involved in both the day-to-day operations and the broader strategic aspects of clinical trials.

At my institution, this includes supporting feasibility assessments, identifying and addressing potential barriers to study start-up, coordinating across departments, and working directly with investigators to move projects forward. It also means serving as a local point of contact who understands the institutional landscape and the needs of patients and families, while staying connected to national efforts through RareKids-CAN.

I also work closely with our Rare Disease Patient Health Facilitator to support the development of the SWORD program. This includes building a local rare disease patient registry, supporting initiatives such as Coffee Connections, contributing to events like Rare Disease Day, as well as raising awareness through local and national conferences.

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

CG: Pediatric rare disease clinical trials often involve very small patient populations, complex protocols, and limited existing data. There are also important logistical, ethical, and safety considerations, particularly for advanced therapies such as gene and cell therapies. These challenges make strong coordination, collaboration, and infrastructure essential to successful trial delivery.

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

CG: It’s important to recognize that families affected by rare disease often have very limited treatment options, and in some cases, a clinical trial may be their only treatment option. Many families travel long distances, sometimes across provinces or even internationally, to access treatment through clinical trials. Behind each clinical trial is a high level of coordination across multiple teams and institutions, all working towards the shared goal of improving outcomes for patients with rare conditions and limited therapeutic options.

“Many families travel long distances, sometimes across provinces or even internationally, to access treatment through clinical trials.”

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

CG: I’m excited about the continued advancement of gene and cell therapies (also known as advanced therapy medicinal products or ATMPs) and their potential to transform treatment options for rare diseases.

As these therapies evolve, there is an increasing need for strong collaboration and infrastructure to support ATMP trials. Through the RareKids-CAN network, we can expand local capacity across institutions, ensuring that all sites in Canada are equipped to deliver these trials and that patients and families have equitable access to them.