Guideline on good pharmacovigilance practices (GVP) Product- or Population-Specific Considerations IV: Paediatric population

The EMA Good Pharmacovigilance Practices (GVP) Product- or Population-Specific Considerations IV guideline provides recommendations for the assessment, monitoring and reporting of safety information for medicinal products used in paediatric populations. It complements general pharmacovigilance requirements by addressing specific challenges associated with children and adolescents, including differences in development, age-dependent responses to medicines, difficulties in identifying and interpreting adverse events, and the need for appropriate long-term safety monitoring.

The guideline highlights important considerations for safety reporting during paediatric clinical development, including the design of pharmacovigilance strategies, collection and evaluation of adverse events, risk management planning, identification of potential safety signals and communication of safety information. It recognises that safety profiles observed in adults cannot always be directly extrapolated to children, and that dedicated approaches are needed to appropriately evaluate benefit–risk throughout paediatric development.

This resource is particularly relevant for rare disease clinical trials because a large proportion of rare diseases affect children, and many investigational therapies are developed for paediatric populations with limited available safety data. Rare disease trials often involve small numbers of participants, heterogeneous disease manifestations and prolonged treatment exposure, making systematic safety monitoring and appropriate interpretation of adverse events essential. The principles described in this guideline can support researchers in developing robust safety monitoring plans adapted to vulnerable and data-limited populations.

Although developed within the European regulatory framework, the concepts and recommendations are broadly applicable internationally to paediatric and rare disease clinical trials. The guidance provides a valuable reference for investigators, sponsors and clinical trial teams seeking to implement appropriate pharmacovigilance strategies and ensure high-quality safety reporting throughout the lifecycle of rare disease therapies.