Long-Acting Therapeutics in Pediatric Health: Bridging Innovation and Access.
This broad pediatric review discusses long-acting therapeutic platforms, developmental pharmacology, formulation, safety, acceptability, access, and implementation considerations across infectious diseases and selected noninfectious indications, including childhood cancer.
Open original publication →What the AI sees
This broad pediatric review discusses long-acting therapeutic platforms, developmental pharmacology, formulation, safety, acceptability, access, and implementation considerations across infectious diseases and selected noninfectious indications, including childhood cancer.
Research significance
The review supports the general premise that sustained drug exposure and reduced dosing frequency could improve adherence and implementation in selected pediatric settings; application to childhood cancer is only a prospective inference because the supplied record reports no cancer-specific agent, experiment, trial, or outcome.
Source abstract
Children have distinct therapeutic needs arising from age-dependent physiology, disease epidemiology, formulation requirements, dosing considerations, and safety vulnerabilities. Despite substantial reductions in childhood mortality over the past three decades, nearly 5 million children under five died in 2023, with infectious diseases continuing to contribute substantially to preventable morbidity and mortality. Long-acting therapeutics (LATs) may help address selected pediatric health priorities by sustaining therapeutic or prophylactic exposure, reducing dosing frequency, and improving adherence and implementation feasibility. This review synthesizes insights from the pediatric health session of the Community of Practice for Long-acting Therapeutics for Maternal and Paediatric Health workshop. The session considered priority pediatric conditions in which existing or emerging LAT approaches could have clinical or public health impact, including lower respiratory tract infections, malaria, tuberculosis, HIV, and syphilis, as well as selected noninfectious indications such as childhood cancer and growth hormone deficiency. We then examine clinical pharmacology and safety considerations central to pediatric LAT development, including developmental pharmacokinetics, absorption-limited disposition and flip-flop kinetics, pharmacokinetic tailing, dose selection, model-informed development, and injection-site tolerability. The review also discusses long-acting technology platforms with potential pediatric applications, route-of-administration considerations, excipient safety, formulation design, acceptability, and implementation challenges. Finally, we propose recommendations to support pediatric LAT development, including early integration of developmental pharmacology, age- and weight-informed dosing, standardized acceptability assessment, product-attribute frameworks, regulatory engagement, and equitable access planning. Realizing the potential of LATs for children will require coordinated, child-centered, and equity-driven approaches that address evidence generation, formulation suitability, implementation context, and affordability from the outset.