Geneva, Switzerland, 2025 – The World Health Organization (WHO) has released a new 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.
Commenting on the publication of the report, Dr Meg Doherty, Director of WHO’s Department of Science for Health, noted that the agenda provides a foundation for aligning research efforts with public health needs. “This research agenda offers governments, partners and research institutions a clear direction for investment. By identifying where evidence is most needed, it creates an opportunity to coordinate resources and foster collaboration to address the highest-burden areas affecting children today.”
Dr Pascale Allotey, Director of WHO’s Department of Sexual, Reproductive, Maternal, Child, Adolescent Health and Ageing, and UN Special Programme in Human Reproduction (HRP) emphasized the importance of inclusive and sustained investment. “Establishing research priorities is only the first step. Strategic and sustained funding will be critical to move these priorities forward. This agenda provides a shared roadmap that donors and partners can use to align investments, support national needs and ensure that research leads to real improvements in child health.”
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.
WHO will continue to work with Member States and partners to facilitate the application of this agenda, promote dialogue across sectors, and mobilize resources for priority research. WHO encourages Member States, research institutions, partners and funding agencies to use this agenda as a tool to guide strategic planning, mobilize resources and support collaborative action. By addressing these gaps, the evidence generated can be brought into much needed and potentially neglected areas for WHO guidelines and policies. By focusing on shared priorities and promoting equitable inclusion, the agenda aims to ensure that future research addresses the needs of all children, including those who have historically been left behind.