Unmet Medical Needs in Pediatric Endocrinology: A Position Statement from the European Society for Paediatric...

Children affected by complex endocrine disorders continue to face substantial and persistent barriers in diagnosis, treatment, and long-term care across Europe and worldwide. Diagnostic uncertainty remains common due to non-standardized criteria, limited validation of biomarkers, and unequal access to advanced testing. Fragmented care pathways and insufficient multidisciplinary coordination further undermine optimal management, particularly during the transition from pediatric to adult services. Pediatric drug development is constrained by small patient populations, ethical and regulatory challenges, and limited commercial incentives, resulting in therapeutic gaps. Families frequently shoulder considerable psychosocial and financial burdens, often without adequate educational, psychological, or social support. Addressing these inequalities requires strategic action to stimulate research innovation, harmonize diagnostic and training standards, improve equitable access to care and medicines, and strengthen patient and family empowerment through collaborative networks and advocacy. This document aimed to highlight these unmet medical needs and outline a path forward. By identifying key barriers and proposing strategic directions, ESPE seeks to foster collaboration among clinicians, researchers, policymakers, patient communities, and pharmaceutical partners. Only through coordinated action can we ensure that every child with an endocrine disorder receives equitable, timely, and high-quality care - regardless of where they live.