Introduction: The key feature of the newborn is its fast age-dependent maturation, resulting in extensive variability in pharmacokinetics and -dynamics, further aggravated by newly emerging covariates like treatment modalities, environmental issues or pharmacogenetics. This makes clinical research in neonates relevant and needed, but also challenging.
Areas covered: To improve this knowledge, tailoring research tools as well as building research networks and clinical research skills for neonates are urgently needed. Tailoring of research tools is illustrated using the development of dried blood spot techniques and the introduction of micro-dosing and -tracer methodology in neonatal drug studies. Both techniques can be combined with sparse sampling techniques through population modeling. Building research networks and clinical research skills is illustrated by the initiatives of agencies to build and integrate knowledge on neonatal pharmacotherapy through dedicated working groups.
Expert opinion: Challenges relating to neonatal medicine research can largely be overcome. Tailored tools and legal initiatives, combined with clever trial design will result in more robust information on neonatal pharmacotherapy. This necessitates collaborative efforts between clinical researchers, sponsors, regulatory authorities, and last but not least patient representatives and society.
Keywords: agency initiatives; clinical pharmacology; covariates; dried blood spot; mechanism based models; microdosing; microtracer; neonatal networks; newborn.