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Drug development in neonates

Work Package 6 - Drug development in neonates


GRiP Drug development in neonates

Conducting clinical drug trials in neonates raises many questions and problematic concerns that need to be addressed on a global unified basis. Work Package 6 addresses these questions and concerns by undertaking the following:

  • Identify neonatal networks in developing and developed countries in order to highlight key questions and problems in conducting clinical drug trials in neonates and set up a common plan of action.
  • Organize a training module in neonatal clinical pharmacology
  • Identify common elements and standardized definitions for risk factors, interventions, complications and short- and long-term outcomes in neonates. 
  • Make an inventory of unadapted formulations / presentations, therapeutic protocols and strategies in neonatal diseases and identify unmet medical needs. 
  • Define standards for biological banking (including tissue and DNA samples) 
  • Organize international consensus conferences to validate and disseminate common agreements for high-quality clinical drug development in neonates (including ethics, methodology, pharmacokinetic studies, sampling, normal biological values, definitions of standard of care and reference treatments, etc.). 



Please see the latest results delivered by the GRiP Network under Work Package 6. Where possible, abstracts of the reports have been provided on this page. To obtain more detailed information, you can download the available materials by clicking on the links at the bottom of the page (only public documents will be made available).


WP6 – Drug development in neonates


Report on existing neonatal networks outside EU


Lead Beneficiary – BIOEF


Objective – A report on the identified neonatal networks in developed and developing countries that have databases including information on their characteristics and activities with respect to  neonatal clinical studies (and updates during the project).


First Report on standardised and validated definitions 


Lead Beneficiary - INSERM


Description: this document describes the Delphi study aimed to optimize quality and performance of drug evaluation in neonates and the identification of elements / definitions common to European neonatal networks


Reports on preclinical and clinical studies 


Lead Beneficiary - BIOEF


Description: This document represents a reference for preclinical studies, including information obtained by juvenile animal studies, implementation of new methodologies, including evaluation of pharmacokinetics, efficacy and safety of drugs in neonates as well as for clinical studies


Final report on standardised and validated definitions


Lead Beneficiary  INSERM


Description: This document describes the final report on the Delphi study.


Training module in neonatal clinical pharmacology set up


Lead Beneficiary – INSERM


Description: Neonates is a high risk heterogeneous age group, from extremely preterm to post mature neonates. Understanding developmental pharmacology, taking into account immaturity and very rapid physiological changes that occur in the first days – weeks after birth is essential to treat patients in an effective way, while avoiding short and long term adverse events. Neonatal pharmacology is one of the modules developed for the Master’s program in paediatric clinical pharmacology as well as for separate programs for clinical investigators and support staff around the globe. This module provides the students with the basics of neonatal developmental pharmacology and provide also practical examples of drugs specifically evaluated for neonates.


Report to identify difficulties to perform clinical trials in pregnant women


Lead Beneficiary – INSERM


DescriptionIt is recognised that pharmacokinetics, efficacy and safety of medications may change during pregnancy. Historically pregnant women have been excluded from clinical trials due to lack of data on the potential effects of in-utero drug exposure and the ethical concerns this implies. However, certain medical conditions, such as hypertension, may require drug treatment during pregnancy to prevent or reduce maternal and foetal morbidity or mortality. The lack of data for many drugs makes the benefit-risk assessment of treatment options for this vulnerable patient population a difficult, if not impossible, task. Over the past 20 years, regulatory guidance documents have been published concerning the general conduct of clinical trials and those in pregnant women in particular. Good quality publications of clinical trials contribute to much needed evidence for clinicians and patients. It is currently unclear whether recent publications of clinical trials in pregnant women have taken regulatory guidance and recommendations on how to publish clinical trial data into consideration. The aim of this review is, therefore, to provide an assessment on the type of data included in these publications.


Report on ethical standard procedures (Blood, DNA, RNA banking and for sample collections)


Lead Beneficiary – INSERM


DescriptionWithin this deliverable, we reviewed the regulatory documents and position papers by experts in Ethics and Research. We then discuss the following aspects of biobanking on genetic materials: 1) Ethical principles for biobanks, 2) Informed consent, 3) Privacy protection, 4) Returning research results to participants, 5) Research Ethics Committees. We also focussed in the last part aspects specific biobanking in paediatric research