WHO Launches Global Research Agenda to Strengthen Paediatric Clinical Trials

Geneva, Switzerland — November 6, 2025 — The World Health Organization (WHO) has announced a new global research agenda designed to close the evidence gap in children’s health and strengthen paediatric clinical trials worldwide.

Outlined in the report “The Future of Paediatric Clinical Trials – Setting Research Priorities for Child Health,” the initiative sets a coordinated roadmap to ensure that medical interventions for children are supported by robust, age-appropriate scientific data.

Closing the Evidence Gap for Children

Despite major advances in global child health over the past two decades, children—particularly those in low- and middle-income countries—remain under-represented in clinical research. Many paediatric treatments continue to be adapted from adult data, creating uncertainty about their safety and effectiveness for younger patients.

The new WHO agenda seeks to address this imbalance by identifying the most urgent research priorities for children aged 0–9 years and by providing a framework for countries, research institutions, and funding bodies to align their efforts toward evidence-based solutions.

Defining the Priorities

Developed through an extensive global consultation involving more than 380 experts from across regions and disciplines, the agenda reviewed over 650 research questions. From this process, 172 clinical research priorities were identified—spanning infectious diseases, non-communicable diseases, newborn health, early childhood development, and nutrition.

Each priority was selected based on its feasibility, scalability, and potential to create equitable health outcomes, especially for children historically left behind in global health progress.

A Coordinated Call to Action

WHO leaders emphasized that the new framework provides governments and research partners with a clear direction for investment and collaboration.

“This research agenda offers governments, partners, and research institutions a clear direction for investment,” said Meg Doherty, Director of WHO’s Department of Science for Health. “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.”

Pascale Allotey, Director of WHO’s Department of Sexual, Reproductive, Maternal, Child, Adolescent Health and Ageing, added that funding and sustained commitment will be critical:

“Setting priorities is only the first step—strategic and sustained funding is needed to move these priorities forward.”

Building Stronger Research Systems

The agenda also outlines the key structural enablers required to drive progress: enhancing regional collaboration, integrating trials within national health systems, strengthening institutional capacity for multi-country studies, and ensuring long-term financing for child-focused research.

It stresses the importance of designing trials that are ethical, inclusive, and age-appropriate—considering children’s unique physiological, developmental, and social contexts. Likewise, it calls for stronger regulatory frameworks and data-sharing systems to support transparent, high-quality paediatric research.

Global Relevance and Regional Implications

For regions around the world—particularly those with diverse child health challenges such as infectious diseases and rising non-communicable conditions—the WHO framework provides a vital opportunity to prioritize locally relevant research while fostering international collaboration.

By aligning national research agendas with WHO’s global priorities, countries can strengthen their evidence base for child health policies and ensure more equitable access to effective, evidence-driven care.

Challenges and the Road Ahead

While the agenda marks a milestone in global child health, significant challenges remain. Funding limitations, inadequate infrastructure for large-scale trials, and ethical complexities unique to paediatric research continue to pose barriers. Ensuring equitable participation from under-resourced regions will also require targeted investment and political commitment.

Still, WHO underscores that this is not merely a policy paper—it is a call to action. The organization aims to work closely with member states, research networks, and funding agencies to transform these priorities into tangible programmes that improve children’s lives.

A Global Step Forward for Child Health

The new research agenda represents a renewed commitment to closing the long-standing evidence gap in paediatric medicine. For governments, scientists, and healthcare systems, it offers a shared vision to ensure children are no longer an afterthought in clinical research but a central focus of global health innovation.

As the world continues to face complex public health challenges, WHO’s initiative reinforces a simple but powerful principle: no child should be treated based on assumptions—only on evidence.

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