WHO Releases Global Research Agenda to Address Critical Gaps in Pediatric Clinical Trials
Key Insights
The World Health Organization has published a comprehensive global research agenda identifying 172 clinical research priorities for children aged 0-9 years to address critical evidence gaps in pediatric healthcare.
The agenda was developed through an inclusive process involving over 380 global stakeholders who contributed 653 research questions, covering infectious diseases (search), noncommunicable diseases (search), newborn health, early childhood development and nutrition.
Despite major advances in child health over two decades, children remain significantly under-represented in clinical trials, particularly affecting outcomes in low- and middle-income countries.
The World Health Organization has released a landmark technical report establishing a global research agenda designed to strengthen pediatric clinical trials and address critical evidence gaps affecting children worldwide. The report, titled "The future of paediatric clinical trials – setting research priorities for child health," focuses specifically on children aged 0-9 years and aims to guide strategic investment in pediatric research.
Addressing Persistent Healthcare Inequities
Despite significant advances in child health over the past two decades, progress has not been equitable across global populations. Preventable illnesses (search) continue to cause substantial morbidity and mortality among children, particularly in low- and middle-income countries. The persistent under-representation of children in clinical trials has created significant evidence gaps that directly impact their healthcare needs.
The new research agenda follows WHO's 2024 "Guidance for best practices for clinical trials," which called for greater inclusion of under-represented populations, including children, to ensure research better reflects the diversity of populations affected by health conditions globally.
Comprehensive Stakeholder Engagement Process
The agenda was developed through an extensive and systematic consultation process that engaged more than 380 stakeholders from around the world. These participants, including global experts, national programme leaders, researchers, and community representatives, contributed a total of 653 research questions across multiple domains of child health.
Through expert review and prioritization processes, these contributions were refined into a final list of 172 clinical research priorities. The priorities span five critical areas: infectious diseases (search), noncommunicable diseases (search), newborn health, early childhood development, and nutrition.
Strategic Focus on Public Health Impact
The research agenda is designed to be both practical and action-oriented, with strong emphasis on feasibility, scalability, and equitable impact. The priority questions were specifically selected based on their potential to generate the greatest public health impact and support national decision-making processes.
Dr. Meg Doherty, Director of WHO's Department of Science for Health, emphasized the agenda's strategic value: "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."
Implementation Framework and Collaboration
The report highlights several key elements necessary for successful implementation, including the importance of regional collaboration, integration of research within national health systems, and strengthening of institutional capacity to support multi-country trials. The agenda also underscores the need for coordinated financing approaches to accelerate implementation and ensure research investments target areas with the greatest potential for impact.
Dr. Pascale Allotey, Director of WHO's Department of Sexual, Reproductive, Maternal, Child, Adolescent Health and Ageing, stressed the importance of 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."
Future Directions and Global Coordination
WHO will continue working with Member States and partners to facilitate the application of this agenda, promote cross-sector dialogue, and mobilize resources for priority research. The organization encourages Member States, research institutions, partners, and funding agencies to use the agenda as a strategic planning tool to guide resource mobilization and support collaborative action.
By addressing these identified gaps, the evidence generated can inform WHO guidelines and policies in areas that have been potentially neglected. The agenda aims to ensure that future research addresses the needs of all children, including those who have historically been left behind, through its focus on shared priorities and promotion of equitable inclusion in clinical trials.