On Nov. 6, 2025, the World Health Organization (WHO) released a technical report, The future of paediatric clinical trials – setting research priorities for child health, presenting a global research agenda designed to address critical gaps in evidence that inform policies, clinical guidelines and programmes for children aged 0–9 years.

This work follows the call from WHO’s Guidance for best practices for clinical trials (2024) for a greater focus on inclusion of under-represented populations in clinical trials, including children, to ensure that research better reflects the diversity of populations affected by health conditions globally. 

Despite major advances in child health over the past two decades, progress has not been equitable, and preventable illnesses continue to cause significant morbidity and mortality among children, particularly in low- and middle-income countries. Children remain under-represented in clinical trials, resulting in gaps in evidence that is directly applicable to their needs. This research agenda has been developed to help address these gaps by identifying priority questions where clinical trials can generate the greatest public health impact, support national decision-making and improve outcomes for children at risk. 

The agenda was developed through an inclusive and systematic process involving global experts, national programme leaders, researchers, and community representatives. More than 380 stakeholders contributed 653 research questions, which were refined through expert review and prioritization to produce a final list of 172 clinical research priorities spanning infectious diseases, noncommunicable diseases, newborn health, early childhood development and nutrition. The agenda is designed to be practical and action-oriented, with a strong emphasis on feasibility, scalability, and equitable impact.

In addition to identifying priority research questions, the report highlights the importance of regional collaboration, integration of research within national health systems, and the strengthening of institutional capacity to support multi-country trials. It also underscores the need for coordinated financing approaches to accelerate implementation and ensure that research investments are directed toward areas with the greatest potential for impact.