Abstract

ID-EPTRI project, coordinated by CVBF-TEDDY (Consorzio per Valutazioni Biologiche e Farmacologiche European Network of Excellence for Paediatric Clinical Research), has been submitted on March 29th, 2017, with-in the INFRADEV-01–2017 single-stage call for proposals with the aim to create the framework for a new Research Infrastructure (RI) intended to enhance technology-driven paediatric research in discovery and early develop-ment phases to be translated into clinical research and paediatric use of medicines.

The project arises from the need to find answers to the serious lack of medicines for children in EU and world-wide and to propose development models for paedi-atric medicines that integrates technology-driven as-pects with clinical trials. The interest for Paediatrics was indeed mentioned in the ESFRI Road Map 2016 (http://www.esfri.eu/sites/default/files/20160309_ROADMAP_browsable.pdf) where it was recognised that a similar RI should be included into the landscape of the research in Europe.

The main idea underpinning the project is to provide the European scientific community with a new Research

Infrastructure: EPTRI, the European Paediatric Trans-lational Research Infrastructure aimed to harnessing the research and services for the development of med-icines for children, as well as identify gaps in paediatric medicines research which prevent efficient use of re-search technologies across pertinent medicine research fields, from discovery and preclinical phase, all the way to ameliorate the therapeutic use of medicines in clini-cal practice. Sharing understanding of patients’ needs and concerted efforts in critical areas of research will end in further enhancing the health of children and will also have a positive impact on European competitiveness in the pharmaceutical sector.

EPTRI will be a complementary RI in the context of the existing RIs covering the current gaps in paediatric medi-cines. The new RI will represent a ‘one-stop-shop’ and will act as a paediatric common service with three already es-tablished Research Infrastructures (BBMRI, EATRIS, ECRIN) strengthening collaboration within the scientific paediat-ric community.

The project is aimed to prepare ‘on field’ a whole Con-ceptual Design Report (CDR) of EPTRI, describing the scientific and technical requirements as well as the key components of this new RI. To prepare the CDR, the proj-ect will encompass three phases.

During the Context Analysis phase, that will be per-formed in 5 technical and scientific domains (1-Paediatric Medicines Discovery, 2-Biomarkers, 3-Paediatric Pharma-cology, 4-Formulation Science, 5-Underpinning Paediat-ric Studies), the perceived value and the possible gaps to be covered will be estimated, by enquiring the scientific Communities, the concerned national Authorities and many other Stakeholders.

During the Operational phase, the different compo-nents of the new RI will be organised, including gover-nance model, strategies for interaction with national Au-thorities and the existing RIs, the IT-architecture model, services to be provided and a business plan.

Finally, a Feasibility phase is proposed to develop virtu-al exercises simulating the operations of the RI.