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 aimed at addressing critical gaps in evidence for children aged 0–9 years. This initiative responds to WHO’s 2024 guidance on best practices for clinical trials, which calls for greater inclusion of under-represented populations, including children, to ensure that research better reflects the diversity of populations affected by health conditions worldwide.
Despite significant advances in child health over the past two decades, progress has been uneven, and preventable illnesses continue to cause high morbidity and mortality, particularly in low- and middle-income countries. Children remain under-represented in clinical trials, leading to gaps in evidence that are directly applicable to their needs. The new research agenda seeks to 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.
Developed through an inclusive and systematic process, the agenda involved global experts, national programme leaders, researchers, and community representatives. More than 380 stakeholders submitted 653 research questions, which were refined through expert review and prioritization to produce a final list of 172 clinical research priorities. These priorities span infectious diseases, non-communicable diseases, newborn health, early childhood development, and nutrition. The agenda emphasizes practicality, feasibility, scalability, and equitable impact to ensure that research investments translate into meaningful outcomes.
Commenting on the report, Dr Meg Doherty, Director of WHO’s Department of Science for Health, said, “This research agenda offers governments, partners, and research institutions a clear direction for investment. By identifying where evidence is most needed, it creates opportunities 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), highlighted the need for sustained investment. “Establishing research priorities is only the first step. Strategic and sustained funding will be critical to advance these priorities. This agenda provides a shared roadmap that donors and partners can use to align investments, support national needs, and ensure that research translates into real improvements in child health.”
The report also stresses the importance of regional collaboration, integration of research within national health systems, and the strengthening of institutional capacity to support multi-country trials. Coordinated financing approaches are emphasized to accelerate implementation and ensure that research funding targets areas with the greatest potential for impact.
WHO will continue to work with Member States and partners to facilitate the agenda’s application, promote cross-sector dialogue, and mobilize resources for priority research. By focusing on shared priorities and equitable inclusion, the agenda aims to ensure that future research addresses the needs of all children, including those historically left behind, ultimately strengthening evidence-based policies and guidelines to improve child health globally.
