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Advancing safer pediatric medicine through trusted genomic research
How a leading researcher in Canada is using deeply phenotyped pediatric data to better understand and enable safer and more individualized treatment decisions
For more than three decades, Professor Bruce C. Carleton in the Department of Pediatrics at the University of British Columbia and his team have worked to make medicines safer for children by uncovering why some patients experience severe adverse drug reactions to therapies intended to help them.
In pediatric care, the stakes are especially high as children with cancer and other serious conditions often receive potent therapies that must balance efficacy with long-term safety. For families and clinicians, the question goes beyond whether a drug works. They also need to know how to use it in the safest and most informed way for each child.
That question drives Dr. Carleton’s longstanding work in pediatric pharmacogenomics, the study of how genetic variation influences drug response, focusing on adverse drug reactions. Dr. Carleton founded the Canadian Pharmacogenomics Network for Drug Safety, a national research network focused on identifying genomic biomarkers that can predict which children face greater risk from specific treatments, understand the underlying mechanistic basis of adverse drug reactions, and implement testing programs to identify children at risk before therapies begin.
This is where his collaboration with BC Platforms becomes central: giving researchers a trusted environment where sensitive clinical and genomic data can be analyzed at scale with the control, consistency, and confidence this work requires.
Optimizing drug therapy in children means ensuring effective responses without causing harm. Unfortunately, adverse drug reactions to prescription drugs are a leading cause of death and disability in North America.
Professor Bruce C. Carleton, Department of Pediatrics, University of British Columbia
A national network built around deeply phenotyped pediatric data
Since 2004, Dr. Carleton’s team has developed a drug safety and effectiveness network of 32 academic health centers across Canada, supporting patient recruitment and enrollment, informed consent and contracting, data collection, and day-to-day program operations.
Over time, the program has become a pre-eminent resource for pediatric drug safety research, combining clinical data, follow-up and longitudinal information, biological samples, and deep genomic data. This pan-Canadian collaboration has enrolled more than 12,000 participants to date and continues to add approximately 1,000 participants annually to expand a cohort that can help researchers understand the genetic contributors to drug outcomes.
The program stands out for both scale and depth. The team captures detailed clinical variables such as diagnoses, drug exposures, dosing, comorbidities, treatment response, follow-up outcomes, and other longitudinal measures that place genomic findings in meaningful clinical context.
That depth matters because adverse drug reactions are difficult to study, particularly in pediatric populations where diseases may be rare, patient numbers limited, and clinical context highly complex. Genomic data becomes far more powerful and useful when connected to well-curated, longitudinal, and clinically meaningful information.
Deeply phenotyped clinical data is essential for understanding pediatric adverse drug reactions. Without a valid phenotype, there is no valid genotype,
Professor Bruce C. Carleton, Department of Pediatrics, University of British Columbia
From data collection to clinically meaningful insight
One example from the program illustrates the potential impact. In some pediatric cancer treatments, a drug regimen may be effective against the disease but also carries an associated high risk of hearing loss. By studying the genomic and clinical factors that influence this risk, researchers can identify patterns that may point to safer approaches, such as modifying dosing schedules for children with specific genetic risk profiles to mitigate or reduce risk of adverse drug reactions.
The goal is to bring those insights closer to care by leveraging genomic information that could help clinicians understand which therapeutic approach may be most appropriate and how to deliver it in a way that may reduce preventable harm. In this model, pharmacogenomics supports a more individualized assessment of benefit and risk. As whole genome sequencing becomes more accessible globally, connecting genomic variation with longitudinal outcomes as seen in this work will become increasingly important for drug safety, effectiveness, and precision medicine.
Why more than a secure data environment was needed
As the program expanded, Dr. Carleton and his collaborating investigators needed an infrastructure that could support complex data, specialized workflows, and rigorous oversight without slowing the research. Programs like this often unwittingly rely on patchwork systems, manual access processes, site-specific workflows, and locally managed tools. That fragmentation makes it harder to govern sensitive data consistently, reproduce analyses across users or sites, and maintain a clear record of who did what, when, and under which analytical conditions.
BC Platforms’ trusted research environment (TRE) provides the governed environment for this work. Dr. Carleton and collaborators manage access, support appropriate analytical workflows, track activity, and promote consistency in how analyses are performed. This common environment also helps participating sites work from the same governed methods and analytical conditions, increasing the likelihood that teams can reach the same answer from the same data. For research where replicability is integral to validity, that consistency is not merely operational; it is a scientific imperative.
That distinction matters in genomic research, where an unsuitable tool, inconsistent workflow, or poorly governed analytical step can produce results that appear compelling but lack scientific validity. BC Platforms helps reduce that risk by bringing permissions, data access, approved tools, auditability, governance, and reproducible workflows into one environment designed for sensitive, multi-site research.
In this kind of research, the technical environment matters because the science depends on it. We need to know who accessed the data, understand how analyses were performed, ensure the correct analysis set, and know that results can be reproduced under the right conditions. That level of control gives us greater confidence in the evidence we generate.
Professor Bruce C. Carleton, Department of Pediatrics, University of British Columbia
Reducing the burden of building and maintaining research infrastructure
For this national research network, a trusted research environment must do far more than protect sensitive data. It needs to support multiple institutions, users with different roles and permissions, specialized data types, evolving analytical tools, governance requirements, audit trails, and reproducible workflows. Building that capability internally requires sustained investment in cloud infrastructure, cybersecurity, identity and access management, data engineering, scientific computing, compliance, user support, and ongoing platform maintenance.
Much of that burden comes after implementation to remain current, compliant, and usable as privacy expectations, cybersecurity requirements, software, analytical tools, and support needs evolve. For scientific teams, those obligations can consume scarce technical and operational resources that could otherwise remain focused on research.
Dr. Carleton and collaborators focus on conducting sensitive research within a setting designed to keep pace with evolving security, privacy, and compliance expectations. That gives research teams confidence that the infrastructure supporting their work is secure at launch and actively managed over time.
Many academic and research organizations can build elements of this environment. The harder task is turning those elements into a reliable operating model researchers can use every day. Without a mature TRE, investigators may end up stitching together fragmented systems, managing inconsistent access processes, recreating analytical workflows, or spending valuable time solving infrastructure problems instead of advancing the science.
BC Platforms gives research organizations a secure, governed foundation they can adapt to specific programs without carrying the full operational burden alone. The model helps reduce infrastructure complexity, accelerate readiness, and support collaboration across sites, data stewards, analysts, and investigators.
For other multi-site, multi-user research initiatives, Dr. Carleton’s experience offers a practical lesson: as data complexity grows, infrastructure becomes part of the research strategy. A trusted research environment at one site or federated across several sites can help teams scale collaboration, protect sensitive data, standardize workflows, and maintain confidence in how analyses are performed across users, sites, and studies.
Building toward a future of safer, more individualized treatment
The next phase of Dr. Carleton’s work will make that foundation even more important. As the program expands whole genome sequencing, explores additional genomic methods, and continues to recruit and reconnect with participants, the dataset will become larger and more powerful for understanding adverse drug responses across diverse pediatric populations.
This research demonstrates the value of trusted research environments in one of precision medicine’s most demanding settings. Pediatric pharmacogenomics requires secure access, deep clinical context, controlled analysis, consistent methods, and a clear path from discovery to application. BC Platforms provides the mature, trusted research environment on AWS to bring those requirements together in a governed environment that helps complex, multi-site research programs generate evidence that is protected, reproducible, credible, and ready to move closer to care.
At the heart of Dr. Carleton’s work is a simple but urgent goal: to understand why children suffer adverse drug reactions and work to reduce the risk of being harmed by medicines meant to help them. We’re proud to support that mission by giving researchers a secure, reliable environment where they can turn complex data into evidence that may help save lives.
Timo Kanninen, Chief Scientific Officer, BC Platforms
Dr. Carleton and his collaborators are turning decades of carefully collected pediatric data into evidence that may help clinicians choose and manage treatment more safely for each child. As a technology partner, BC Platforms supports that mission with a scalable, governed, research-ready environment that protects sensitive data, aligns researchers around consistent methods, and turns complex genomic and clinical information into evidence that can be trusted.
In a field where the right decision can shape both survival and lifelong quality of life, that foundation is central to making precision medicine meaningful for children and families.
Bruce C. Carleton, BSc, PharmD, FCP, FISPE is Chair, Division of Translational Therapeutics, Department of Pediatrics, Faculty of Medicine, and Professor of Pediatrics, Medical Genetics, Population and Public Health at the University of British Columbia. He is also Professor in the School of Health Information Science at the University of Victoria and a Senior Clinician Scientist at the BC Children’s Hospital Research Institute.
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