PedCRIN Assessment of outcomes of neonatal trials: Points to consider

This guidance document provides practical recommendations for integrating pharmacovigilance and risk management into the design of neonatal clinical trial protocols. Developed within the PedCRIN project, it highlights the specific safety considerations that should be addressed when planning interventional studies involving neonates, recognising that adverse events in this population may be influenced by developmental physiology, underlying disease, prematurity and routine neonatal care. The guidance supports researchers in developing protocol-specific pharmacovigilance strategies that are appropriate for the unique characteristics of neonatal clinical trials.

For protocol development, the guidance addresses key elements of safety planning, including identification of anticipated risks, definition and classification of adverse events, safety data collection, reporting procedures, risk management, safety oversight and the roles and responsibilities of investigators and sponsors. It encourages researchers to distinguish between expected neonatal clinical events and adverse reactions related to the investigational medicinal product, and to establish clear procedures for safety monitoring and documentation throughout the study.

Although developed for neonatal trials, many of the principles are applicable to paediatric rare disease studies involving vulnerable populations. By supporting prospective planning of pharmacovigilance activities, the guidance helps researchers strengthen protocol quality, improve participant safety and ensure that safety data are collected and reported in a consistent and scientifically robust manner. It complements broader paediatric regulatory guidance by providing practical recommendations that can be incorporated directly into clinical trial protocols.