Neonatal medicines research: challenges and opportunities

Abstract

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

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Erratum

Acknowledgments

We thank the European Society for Pediatric Research (ESPR) for the opportunity provided to discuss neonatal medicines research in Europe at the latest EAPS meeting (October 2014) in a workshop. We are grateful to Pam Simpkins, Janssen, for supplying the data about projected recruitment from clinicaltrials.gov cited in the text.

Declaration of interest

J Samardzic is supported by ERAWEB II scholarship for postdoctoral program at the KU Leuven, Belgium (2014 – 2015). MA Turner is supported by the Medical Research Council grant G1100158, European Commission Network of Excellence Grant Agreement 261060, (Global Research in Paediatrics), and FP7 grants TINN (223614), TINN2 (260908), NeoCirc (282533) and NeoVanc (602401). K Allegaert is supported by the Fund for Scientific Research, Flanders (fundamental clinical investigatorship 1800214N) and the research activities are further facilitated by the agency for innovation by Science and Technology in Flanders (IWT) through the SAFEPEDRUG project (IWT/SBO 130033). The views expressed in this article are the personal views of the authors and may not be understood or quoted as being made on behalf of or reflecting the position of the European Medicines Agency or one of its committees or working parties. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.

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