PedCRIN Protocol development for neonatal trials: Points to consider for pharmacovigilance

The ECRIN Paediatric Tools – Outcomes comprise a collection of practical guidance documents developed through the PedCRIN project to support the selection, definition and assessment of efficacy and safety outcomes in paediatric and neonatal clinical trials. The tools address a critical aspect of protocol development by providing structured recommendations for identifying appropriate outcomes and planning their measurement across different stages of a study. They include specific guidance on the assessment of short-term and long-term outcomes, recognising that many paediatric interventions require extended follow-up to evaluate developmental, functional and safety outcomes.

The collection supports researchers in defining clinically meaningful and measurable endpoints, identifying appropriate data items, and planning outcome collection strategies that are feasible within paediatric studies. It highlights the importance of capturing both efficacy and safety outcomes, taking into account the unique physiological and developmental characteristics of neonates and children. By encouraging systematic outcome assessment and harmonised data collection, the tools contribute to improving the quality, consistency and comparability of paediatric clinical trials.

For rare disease clinical trials, these resources are particularly valuable because they help researchers address common challenges related to small sample sizes, heterogeneous disease presentations and the need for meaningful long-term follow-up. The tools complement broader guidance on patient-reported outcomes, health-related quality of life and core outcome sets by providing practical recommendations for incorporating efficacy and safety outcome assessment directly into clinical trial protocols.