Pharmacotherapy is a powerful tool to improve the outcome of neonates. Unfortunately, the potential health impact of pharmacotherapy in neonates remains underexplored. This necessitates a structured approach to go beyond the current practice of trial and error, reflected in off-label prescription. The existing regulatory framework hereby provides a structure to reflect about aspects like pharmacokinetic models for dose selection and outcome assessment, including long-term safety. Future medicine development should also be driven by neonatal needs, diseases and pathophysiology, since surfactant is the latest product developed for preterm neonates. The potential impact is illustrated by ongoing repurposing (propranolol, allopurinol, erythropoietin, Insulin-like Growth Factor-1) projects.Clinical researchers will be crucial to close the knowledge gap by developing dose selection tools and outcome assessment tools and by exploring pathophysiological mechanisms. The final step of such a structured approach cycle is the subsequent translation of accumulated knowledge into improved prescribing.

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Early Human Development
Department of Pediatric Surgery

Allegaert, K. (Karel). (2017). Better medicines for neonates: Improving medicine development, testing, and prescribing. Early Human Development. doi:10.1016/j.earlhumdev.2017.09.007