Accelerating access to paediatric medicines: lessons learned from the Global Accelerator for Paediatric Formulations, a WHO-hosted network

Abstract

Despite progress in reducing neonatal and child mortality, access to age-appropriate medicines remains inequitable, particularly in low-income and middle-income countries. The Global Accelerator for Paediatric Formulations (GAP-f), a WHO-hosted network established in 2020, addresses these gaps by uniting 33 partners to promote innovation and access to child-friendly formulations. Phase 2 (2022-24) of GAP-f's work focused on therapeutic areas where innovation and access efforts often did not have stakeholder alignment and coordination of designing and implementing innovative clinical trial methodology, engaging with regulators to address systemic barriers, identifying novel technologies for safe and effective delivery, and collaborating across stakeholders for product roll out. Learnings from GAP-f work include the need to adapt prioritisation processes to diverse therapeutic areas and focus on high-impact areas based on unmet needs. Platform trials emerged as a promising tool to accelerate evidence generation but require guidance from regulators, collaboration among pharmaceutical companies, and operational challenges and funding constraints to be overcome. Incentivising paediatric formulation development remains crucial for small volume paediatric markets, and efforts are also needed to more proactively enhance, accelerate, and effectively match innovations to deliver medicines to children more efficiently. Even when products are successfully developed, political will and community engagement are essential to creating demand, supporting roll out, and ensuring equitable access and appropriate use in children. Dedicated funding and targeted programmes are crucial to drive systemic and sustainable change. GAP-f implementation lessons discussed in this Personal View will inform the future of this needed initiative, accelerating progress towards improved medicines for children.