Why Children's Medicines are Still Playing Catch Up

Written by Lucy Nguyen

Children receive medicines for everything from common infections to life-threatening illnesses. Yet many treatments have historically been studied primarily in adults, leaving doctors with limited evidence for prescribing them to younger patients. Although regulations have improved pediatric research, developing safe, effective and accessible medicines for children remains a global challenge.

The problem, of course, begins with biology. Children's bodies change throughout development, affecting how drugs are absorbed, distributed, metabolised and eliminated. Consequently, simply reducing an adult's dose according to body weight may not produce a safe or effective result. Researchers must investigate age-specific pharmacokinetics and pharmacodynamics, using clinical studies and, where appropriate, mathematical modelling to establish suitable doses.

Formulation presents another obstacle in pediatric pharmaceutical science. Young children may struggle to swallow tablets, while unpleasant tastes can make medicines difficult to administer consistently. Researchers must therefore consider alternatives such as dispersible tablets and oral liquids. These formulations must also remain stable under specified storage conditions, retain their quality and allow accurate dosing. Such requirements become particularly important where refrigeration and reliable supply chains are limited.

Regulators have introduced measures to address these gaps. In the United States, the Pediatric Research Equity Act enables the FDA to require certain pediatric assessments, while the Best Pharmaceuticals for Children Act incentivises eligible studies. In the European Union, companies generally need an agreed pediatric Investigation Plan for relevant medicines unless a waiver applies. These measures have strengthened the framework for pediatric research, but they cannot eliminate every scientific or commercial barrier. In 2025, the World Health Organization announced its Global Accelerator for Paediatric Formulations roadmap for 2025–2030, with the goals of prioritising10 high-burden diseases, accelerating 10 priority medicines and strengthening pediatric medicine systems in 10 countries. In 2026, the WHO also reported work to improve child-friendly formulations of six cancer medicines, addressing requirements such as stability, appropriate dosing, affordability and safe handling.

These initiatives acknowledge that scientific progress alone is insufficient. Small patient populations can complicate trials, while development costs and limited commercial incentives may discourage investment in medicines for rare childhood diseases. Even when a suitable medicine exists, affordability and availability can prevent children from benefiting from it.

In my view, protecting children should mean neither excluding them from necessary research nor exposing them to avoidable risks. It means conducting carefully regulated studies, designing medicines around children's developmental needs and ensuring that effective treatments can reach those who need them. Progress should ultimately be measured not only by how many medicines are developed, but by whether children everywhere can access treatments that are safe, appropriate and supported by reliable evidence.