During the ID-EPTRI project, the EPTRI concept has been designed and tested and has allowed to create the base for research excellence that is the added value of the EPTRI proposal.
In particular, a large community has been identified during the Context analysis phase through several surveys, willing to cooperate in order to standardise models and approaches and to produce validated knowledge useful for drug development.
The EPTRI scientific community survey has allowed to identify a total of 259 Institutions, often grouping more than one research group, distributed across 29 countries in the European area and encompassing a total of 330 research units (RUs), which have provided description of scientific competences, human resources, tools, facilities, services they are available to provide in the future European Paediatric Translational Research Infrastructure.
The users’ survey, with 337 responders, allowed to identify the user community, that is widely distributed in the European area but also includes several extra-EU users. The survey showed that a total of 2207 paediatric research projects were planned for the next 3 years and identified 2780 paediatric projects performed over the past 5 years. Responders have been asked the number of times they have been blocked or delayed during the conduction of their projects. The following analytical tools and models have been identified as those with the highest incidence of blocks: PBPK Modelling and Simulation; Target identification; Preformulation; In vitro/in vivo pre-clinical test of the medical devices; Placental platforms.
A survey conducted within the existing Biomedical RIs helped to identify areas for collaboration and differentiate from the general description of standard analytical methods and the specific requirements. Some analytical approach resulted to be totally unique to EPTRI as the use of placenta to assess drug sensitivity and distribution, organoid for foetal to adult maturation and juvenile animal models.
EPTRI has been designed as a Hub and Spoke organisation with a Central Hub and several Spokes, represented by the Thematic Research Platforms (TRPs) and the national nodes in each member country participating of EPTRI.
The following four TRPs have been created grouping RUs in each specific scientific domain and able to act as providers of cutting-edge services that are unique for paediatric research on medicines setting.
• PAEDIATRIC MEDICINES DISCOVERY, dedicated to basic research and preclinical studies aimed to identify and validate druggable molecular targets specific to paediatric disease, as well as discover new drugs, using models reproducing specific paediatric ontogenetic stages.
• PAEDIATRIC BIOMARKERS AND BIOSAMPLES, focused on the identification, characterisation and validation of paediatric biomarkers and on the evaluation of the paediatric variability with respect to adult biomarkers.
• DEVELOPMENTAL PHARMACOLOGY, consisting on the identification and application of ad hoc innovative methods to study paediatric pharmacological variability due to ontogeny.
• PAEDIATRIC MEDICINES FORMULATIONS, mainly devoted to perform preformulation and formulation studies for preterm neonates, infants, toddlers, children and adolescents.
The the organisation of National Nodes in Italy, Belgium, Germany, The Netherlands, Spain, Hungary, Czech Republic, Cyprus, Ukraine and Israel has been started.
The need to establish a dedicated RI for paediatric research was recognized as a priority by the 17 EU and non-EU Ministries (Albania, Cyprus, Finland, Greece, Hungary, Ireland, Israel, Italy, Luxembourg, Malta, the Netherlands, Poland, Portugal, Romania, Russia, UK and Ukraine) and several Foundations, Patients Associations, Hospitals, Research Institutions and Universities providing their expression of political support letters.
