Belgium participates in the EPTRI framework through an active National Node, which brings together Antwerp University Hospital (UZA), Katholieke Universiteit Leuven (KUL), Ghent University (UGent) and Hôpital Universitaire des Enfants Reine Fabiola (HUDERF). These institutions collectively contribute across complementary domains of paediatric medicines discovery, biomarker and biosample research, and developmental pharmacology. Together, they form a highly integrated translational ecosystem spanning preclinical modelling, clinical research, advanced omics technologies, biobanking, and quantitative pharmacology, with strong links to European research networks and regulatory initiatives.
- Antwerp University Hospital: UZA hosts integrated clinical and research programmes with strong expertise in paediatric and neonatal translational medicine. Key clinical domains include pneumology (sleep disorders, bronchopulmonary dysplasia, cystic fibrosis using advanced imaging), cardiology (including Down syndrome and obesity-related disease), infectious diseases (notably RSV with biomarker-based severity prediction), immunology (e.g. asthma), neurology (traumatic brain injury and neuroinflammation), and paediatric metabolic and rare diseases. Research activities cover a wide spectrum of inherited and acquired metabolic disorders, including mucopolysaccharidosis, phenylketonuria, Fabry disease, urea cycle disorders, and Crigler–Najjar syndrome. UZA integrates three core infrastructures:
- a paediatric biobanking platform with LIMS-based sample tracking and high-quality biospecimen management
- a Clinical Trial Center ensuring ICH-GCP compliant paediatric and neonatal clinical research
- a Data Innovation Lab enabling FAIR, GDPR-compliant secondary use of clinical data and AI-driven analytics (FHIR/OMOP interoperability)
Together, these components form a unified translational ecosystem linking biospecimens, clinical trials, and real-world health data.
- Hôpital Universitaire des Enfants Reine Fabiola: HUDERF, in collaboration with ULB, delivers integrated translational research focused on inflammatory, infectious, metabolic, and genetic paediatric diseases. Its research platform includes primary cell culture, immune stimulation assays, gene expression profiling (RT-qPCR, RNA/microRNA), flow cytometry, and metabolic and biochemical analyses supported by mass spectrometry technologies. These activities are embedded in a clinical-research environment with dedicated biobanking and data systems, enabling biomarker discovery and mechanistic studies in diseases such as sepsis, sickle cell disease, and metabolic disorders. HUDERF also provides integrated deep phenotyping and genotyping services for rare and complex diseases, combining structured clinical cohorts (REDCap-based phenotyping) with multi-omics approaches including whole-exome sequencing, long-read sequencing, and methylome analysis. Supported by accredited sequencing pipelines and European research networks, this enables full genotype–phenotype integration for precision medicine and rare disease research.
- Katholieke Universiteit Leuven: KUL provides a highly integrated biomedical research and clinical innovation ecosystem with strong links between academic institutes, university hospitals, and European research networks. Its activities span translational medicine with particular strengths in rare diseases, immunology, developmental biology, cardiovascular and metabolic diseases, neurodevelopmental and neurodegenerative disorders, and precision oncology. A key strength is the integration of clinical cohorts, deep phenotyping, and multi-omics technologies, including genomics (whole genome and exome sequencing), transcriptomics, proteomics, and epigenetic profiling. These are supported by robust biobanking infrastructures and longitudinal patient registries. KU Leuven also operates advanced data-driven platforms enabling secure integration of clinical, imaging, and molecular datasets. These systems support AI-based analytics, biomarker discovery, and predictive modelling in compliance with GDPR and international data governance standards. Overall, KU Leuven provides a unified translational ecosystem bridging fundamental research, clinical expertise, and computational innovation to advance precision medicine across a wide range of paediatric and adult diseases.
- Ghent University: UGent, in close collaboration with UZ Gent, provides a fully integrated translational paediatric research ecosystem covering the entire pipeline from preclinical modelling to clinical trials and quantitative pharmacology. On the preclinical side, the Laboratory of Pharmacology and Toxicology offers juvenile piglet models for paediatric PK/PD research, including disease models such as sepsis and augmented renal clearance. These studies are supported by advanced techniques including microdialysis for tissue distribution and portal catheterisation for presystemic drug disposition. A high-end bioanalytical platform (UHPLC-MS/MS and LC-HRMS) enables ultra-sensitive quantification in microsamples such as dried blood spots and volumetric absorptive microsampling. In the clinical domain, the Safepedrug Clinical Trial Unit (SPD-CTU), embedded within UZ Gent and the Belgian Paediatric Clinical Research Network (BPCRN), provides full clinical trial support including feasibility assessment, protocol development, regulatory and ethics submissions, patient recruitment, monitoring, pharmacovigilance, and data management. The unit has strong expertise in vulnerable paediatric populations and advanced therapies (gene and cell therapy, ATMPs), supported by GMP pharmacy services and biobanking infrastructure. Complementing these activities, Ghent University hosts strong pharmacometrics and PBPK modelling expertise, using tools such as NONMEM, Monolix, R, Simcyp, GastroPlus, and PK-Sim. These approaches support study design, exposure–response analysis, dose optimisation, and paediatric extrapolation, enabling model-informed drug development across all paediatric age groups. Overall, Ghent University represents a fully integrated translational ecosystem combining experimental models, clinical research infrastructure, and quantitative pharmacology within a strong European collaborative framework.