Collection, storage and use of biological samples and related data in paediatric trials

The PedCRIN Biosample Management tool provides practical support for investigators, sponsors and clinical trial teams involved in the collection, processing, storage and management of biological samples and related data in paediatric clinical trials. Developed within the PedCRIN project, the tool addresses the lack of specific guidance for biospecimen management in paediatric research and provides a structured approach to ensure that samples are collected and managed according to ethical, regulatory and quality requirements.

The tool highlights key considerations for biosample management in children, including obtaining appropriate consent and assent, minimising risks and participant burden, reducing sample volumes where possible, ensuring appropriate staff expertise and facilities, and establishing procedures for long-term storage of biological materials. These considerations are particularly important in paediatric trials because children may have limited capacity for repeated sampling and because biological material collected during childhood may have long-term scientific value.

This resource is particularly relevant for rare disease clinical trials, where limited patient availability makes efficient and responsible management of biosamples essential. In rare diseases, biological samples may support diagnosis, molecular characterisation, biomarker discovery, pharmacodynamic assessments and evaluation of treatment response. Harmonised biosample procedures can also facilitate collaboration across centres and countries, enabling data and sample integration from multiple small studies.

The tool is especially valuable for rare disease trials involving precision medicine approaches, genomic analyses, biomarkers, natural history studies or advanced therapies, where high-quality biological samples and associated clinical data are often central to successful therapeutic development.

Although developed within the European PedCRIN framework, the principles are broadly applicable internationally. The tool provides practical guidance for researchers designing paediatric and rare disease clinical trials that involve biosample collection, helping to ensure scientific value while protecting participant welfare and respecting ethical requirements.