Global Demand for Better Pediatric Treatments Grows

On World Children’s Day, the Global Accelerator for Paediatric Formulations Network (GAP-f) renewed its call to expand access to age-appropriate medicines, outlining progress made this year and highlighting ongoing gaps that continue to limit treatment options for children worldwide. The initiative comes as several global health actors report rising demand for pediatric-specific therapies.

GAP-f, hosted by the World Health Organization (WHO), aims to align stakeholders around priority formulations and support development pathways that ensure children receive medicines suited to their clinical and physiological needs. “The PADO-Epilepsy report charts a shared path to accelerate access to child-friendly medicines for the treatment of epilepsy. By aligning funders, developers, regulators, and procurement partners around clear priorities, the PADO report will be key to translating science into access to paediatric antiseizure medicines,” says Dévora Kestel, Director a.i. of Noncommunicable Diseases and Mental Health Department, WHO. 

The organization’s renewed push follows the launch of its Strategic Roadmap 2025–2030 during the 77th World Health Assembly. The plan sets out three shifts: broadening the disease focus to conditions such as malaria, dengue, sickle cell disease, respiratory syncytial virus (RSV), and epilepsy; strengthening national and regional engagement; and driving innovation through new formulation technologies. WHO and partners argue these steps are necessary to close longstanding gaps in research, policy alignment, and investment that impede medicine availability in many low-resource settings.

GAP-f and WHO technical teams completed several prioritization exercises this year through the Paediatric Drug Optimization (PADO) process, identifying formulations needed across RSV, malaria, epilepsy, dengue, and sickle cell disease. The result is a set of documented product priorities intended to guide research and development and coordinate funding decisions. These exercises build on an evidence-driven approach that aims to direct industry and public health partners toward formulations most likely to improve treatment outcomes.

WHO also released six target product profiles (TPPs) for child-friendly formulations of essential cancer medicines. Developed through an expert consultation process in late 2024 and refined with input from GAP-f members, the profiles serve as detailed technical guidance for manufacturers. WHO says that these documents can support future inclusion of new formulations in the organization’s prequalification program and, eventually, the Model List of Essential Medicines for Children (EMLc). The cancer-related profiles follow earlier TPPs issued for nitrofurantoin and azithromycin to encourage development of age-appropriate antibiotics.

Work to improve the EMLc is another area where GAP-f has contributed. Over the past four years, WHO reviewed global needs for pediatric formulations, assessed market availability, and evaluated therapeutic suitability. The process informed the ninth and tenth updates of the Model List, resulting in the addition of 163 formulations and the removal of 92 outdated products. WHO identified 79 medicines with gaps in child-appropriate versions, establishing a priority list for future PADO exercises. Efforts to support national adoption of the updated list are underway, beginning in the South-East Asia region.

The push to strengthen pediatric treatment options coincides with advances in clinical research across the pharmaceutical sector. Eli Lilly and Co. recently reported results from the first Phase 3 trial of tirzepatide, marketed as Mounjaro, in children and adolescents with type 2 diabetes. The SURPASS-PEDS study included 99 participants aged 10 to under 18 across eight countries. At 30 weeks, patients receiving Mounjaro recorded an average A1C reduction of 2.2 percentage points from a baseline of 8.05%, while the placebo group saw no significant change. The 10mg dose group also experienced an average BMI decrease of 11.2%.

“Youth living with type 2 diabetes often face a more aggressive disease course, and in many instances, first-line treatments like metformin and basal insulin fail to control their A1C adequately,” says Tamara Hannon, Director of the Clinical Diabetes Program, Indiana University School of Medicine. 

Eli Lilly has submitted the pediatric data to regulators seeking an expanded indication. If authorized, tirzepatide would become one of the first new treatment options for youth-onset type 2 diabetes in more than a decade. The development aligns with GAP-f’s call for greater investment in child-specific research, as most pediatric therapeutic areas continue to face limited drug pipelines.

As GAP-f marks World Children’s Day, WHO said it will continue building partnerships across industry, academia, civil society, and global health agencies to advance access to formulations that meet children’s needs. The organization plans to expand country-level engagement and monitor adoption of the EMLc to support procurement decisions and strengthen national pediatric medicine ecosystems.