The challenges and opportunities for equitable global access to medications for paediatric rheumatology in low-to middle-income countries: a review on behalf of the Paediatric Global Task Force for Musculoskeletal Health

Abstract

Abstract Advances in medications to treat paediatric rheumatic conditions including juvenile idiopathic arthritis (JIA) and systemic lupus erythematosus (SLE) have transformed outcomes in well-resourced settings. Access to therapeutic agents that are an accepted standard of care in many regions of the world remains unequal. Here we review access to medications through a global paediatric rheumatology lens, examining challenges from drug development to delivery. The high cost of novel medications, combined with complex research and regulatory environments, market forces, the rarity of conditions and policy gaps, drive disparities in low- and middle-income countries (LMICs) where outcomes in children with JIA and SLE are closely linked to socioeconomic status and geography. Data concerning the prevalence of rheumatic conditions in LMICs are sparse, contributing to the under-recognition of disease burden. Clinical trials are disproportionately conducted in high-income countries, limiting global representation and knowledge of safety and efficacy across diverse populations. Additionally, medicines may technically be available, but with barriers that undermine true access. Global and regional organizations, including the Paediatric Task Force for Global Musculoskeletal Health, have advanced education, advocacy, and alignment of medicines with the WHO Essential Medicines List for children (EMLc). However, implementation at the country level remains inconsistent, and essential medicines for treating rheumatic diseases are often absent from national formularies. Addressing these inequities requires coordinated strategies: expanding and harmonizing EMLs, accelerating biosimilar approval and uptake, building a workforce and research capacity in LMICs, and embedding paediatric rheumatology within broader noncommunicable disease and universal health coverage frameworks. We summarize some strategic directions and aspirations within these 5 described arenas, that may help to address the global inequity of drug access.