Ethical considerations for clinical trials on medicinal products conducted with minors

This guidance from the European Commission provides recommendations on the ethical considerations that should be addressed when conducting clinical trials involving minors, from birth to the age of legal competence to provide informed consent. Developed by the expert group on clinical trials supporting the implementation of Regulation (EU) No 536/2014, the document aims to promote the protection of children participating in clinical research while ensuring that they are not excluded from the potential benefits of research and access to evidence-based treatments.

The guidance addresses key ethical aspects that should be considered during protocol development and trial management, including justification for involving minors in research, assessment of risks and burdens, potential benefits, informed consent processes, parental permission, child assent, age-appropriate communication, and the involvement of children and families in clinical research decisions. It also highlights the importance of considering the developmental characteristics, vulnerabilities and specific needs of paediatric participants when designing and conducting clinical trials.

This resource is particularly relevant for rare disease clinical trials because a substantial proportion of rare diseases have a paediatric onset, and many investigational therapies are evaluated in children due to unmet medical need. Rare disease trials often involve vulnerable populations, small patient numbers and complex decision-making processes involving children, parents and healthcare professionals. The ethical principles described in this guidance can therefore support researchers in designing participant-centred protocols, developing appropriate consent and assent procedures, and ensuring that trial participation respects the rights and needs of children and families.

Although developed within the European regulatory context, the ethical principles and recommendations are broadly applicable internationally and can inform the planning and conduct of paediatric and rare disease clinical trials across different healthcare and regulatory settings.