Paediatric Medicines Development Services
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Human cell-based in vitro approaches for paediatric medicine testing, disease modelling and formulation development
This set of services provides a comprehensive human cell-based platform, covering genome editing in immortalised cells, complex 3D and patient-derived disease models, thus providing an essential bridge for understanding paediatric disease and developing preventive, diagnostic, and therapeutic strategies tailored for children. The platform generates mechanistic insights, candidate biomarkers, and prioritised compounds.
Juvenile in vivo model development and non-GLP toxicity assessment
This set of services providing access to juvenile animal models, including rodents (mice, rats) and large animals (piglets, preterm rabbits), for non-clinical testing essential to drug development. Studies encompass PK, maximum tolerated dose determination, biodistribution, and comprehensive phenotyping, supporting evaluation of new therapies, administration routes, organ-targeted delivery, and preliminary toxicology.
GLP-compliant juvenile mouse toxicity, efficacy, and safety studies
These services provide GLP-compliant pharmacological testing to support robust non-clinical safety evaluation. Services include safety and toxicology studies, proof-of-concept and long-term efficacy assessments, and lead compound selection using GLP-certified juvenile mouse models that combine advanced phenotyping and efficacy testing, reducing timelines and variability, particularly for rare paediatric indications.
Human and juvenile animal sample biobanking, processing and analysis
This service offers the operational handling and access to paediatric-relevant biological samples from humans and juvenile animal models to ensure they are ready for research use. It covers efficient solutions for the processing, collection, storage and distribution of biological samples along with their associated data, supporting reproducibility, integrated data analysis, and translational research workflows.
In silico ADME and toxicity prediction
This set of services provides virtual access to advanced computational services for predicting ADME properties and toxicological risks of candidate compounds, supporting early-stage decision-making in paediatric drug development. Services include developing QSAR models for paediatric PK, computational toxicology assessments (QSTR, read-across, dose–response, toxicogenomics), compound library filtering, off-target and reactive species prediction, and interspecies extrapolation via PBPK modelling using specialised software platforms.
Scientific and regulatory advice and support
These services provide regulatory advice and support for planning R&D of medicines of paediatric interest and preparing for GLP- and Directive 2010/63/EU–compliant non-clinical studies and EMA regulatory procedures. It oversees ethical compliance and regulatory readiness for all studies, ensuring alignment with legal and scientific standards for paediatric drug development. Services include consulting on preparation of documentation, application and follow-up of PIPs or partial/full waivers, EMA voluntary procedures (Scientific Advice and Protocol Assistance), covering the qualification of novel methodologies for medicine development such as non-clinical models, biomarkers, endpoints, registries, data management tools, and statistical methods, orphan designations for rare disease products, Innovation Task Force (ITF) briefings for innovative products, PRIME applications for paediatric unmet needs, and the classification of Advanced Therapy Medicinal Products (ATMPs), and Paediatric Marketing Authorisation Applications.