Neonatal trials and informed consent: Points to consider

This PedCRIN guidance provides practical recommendations for designing and implementing informed consent processes in neonatal clinical trials. It addresses the specific ethical and operational challenges associated with enrolling newborns into research, recognising that parents or legal representatives are often required to make decisions during a period of significant emotional stress and uncertainty. The guidance considers key aspects of consent procedures, including timing of discussions, communication strategies, parental understanding, information provision, documentation of consent and the balance between protecting participants and enabling access to potentially beneficial research.

Neonatal research presents unique challenges for informed consent because interventions may need to be initiated rapidly, parents may have limited time to consider participation, and newborns cannot provide consent themselves. This guidance supports researchers in developing consent approaches that are respectful, transparent and adapted to the needs of families while ensuring compliance with ethical principles for research involving vulnerable populations.

The resource is particularly relevant to rare disease clinical trials because many rare diseases present during the neonatal period or early childhood, and early diagnosis and intervention can be critical for improving outcomes. Rare disease trials often involve small populations, complex decision-making and families facing uncertainty regarding disease progression and available treatments. The principles described in this guidance—such as clear communication, appropriate timing of consent, family-centred approaches and attention to participant vulnerability—are therefore transferable to many paediatric rare disease research settings.

Although developed within the European PedCRIN framework, the recommendations address universal ethical challenges in neonatal and paediatric research and are broadly applicable internationally. By supporting the development of appropriate informed consent processes, this guidance contributes to participant-centred, ethical and feasible clinical trial conduct in neonatal, paediatric and rare disease research.