Strengthening the paediatric clinical trial ecosystem to better inform policy and programmes

The first WHO Global Clinical Trials Forum was convened in November, 2023 to develop a shared vision of an effective global clinical trial infrastructure. The Paediatric Clinical Trials Working Group was formed to provide perspectives, identify challenges, and propose solutions to strengthen the paediatric clinical trials ecosystem. Participants represented paediatric disciplines, including infectious diseases, nutrition, neonatology, pharmacology, oncology, neurodevelopment, public health, and policy. Childhood diseases have profound lifelong effects on health, livelihoods, and societies. Investment in early childhood results in highly cost-effective changes to lifelong health, productivity, and human capital returns. Yet, there remain substantial gaps in knowledge on the efficacy and safety of many paediatric interventions, which represents a failure to establish shared priorities and alignment across governments, researchers, communities, and funders. Children are frequently marginalised from clinical trials, which is an issue of equity. Challenges include mismatched priorities and funding, risk adversity, poor design, power imbalances, and inadequate infrastructure. Solutions include aligning on and tracking local and global child health priorities against funding and supporting regional consortia to pool resources for larger, more consequential trials. We propose actions and responsibilities for global, regional, and national institutions for prioritisation, coordination, enabling paediatric trials consortia, funding, and tracking progress.