ID-EPTRI project, coordinated by CVBF and funded within the H2020-INFRADEV-01-2017 programme, aims to create the framework for a new Research Infrastructure (RI) intended to enhance technology-driven paediatric research in drug discovery and early development 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 worldwide and to propose development models for paediatric medicines that integrates technology-driven aspects with clinical trials. The interest for Paediatrics was indeed mentioned in the ESFRI Roadmap 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.
EPTRI will be a complementary RI in the context of the existing RIs covering the current gaps in paediatric medicines. The new RI will represent a “paediatric common service” with three already established Research Infrastructures (BBMRI, EATRIS, ECRIN) to harness efficiency and delivery of paediatric research activities and services strengthening collaboration within the scientific paediatric community.
The final result of the project will be the Conceptual Design Report to realize EPTRI, the European Paediatric Translational Research Infrastructure, describing the scientific and technical requirements as well as the key components of the new RI.
To prepare the Conceptual Design Report (CDR), the project will encompass three phases.
During the Context Analysis phase, that will be performed in 5 technical and scientific domains (1-Paediatric Medicines Discovery, 2- Biomarkers, 3-Paediatric Pharmacology, 4-Formulation Science, 5-Underpinning Paediatric 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 components of the new RI will be organised, including governance model, strategies for interaction with national Authorities and the existing RIs, the IT-architecture model, services to be provided and a business plan.
In the second year of the project, a Feasibility phase is proposed to develop virtual exercises simulating the operations of the RI, that will work as a “one-stop-shop” for advice in paediatric drug development in order to:
- support researchers in many methodological areas,
- foster the technology innovation in all the academic and clinical settings,
- conduct research that effectively underpins the development of medicines for children,
- increase the global effectiveness of the European research area also in favour of children and young people.
The work plan of this proposal is based on 11 Work Packages:
- WP1 – Coordination and Project Management. WP1 will provide the organisational framework to conduct all the activities and until the end of the project.
- WP2 – Governance and Sustainability. Providing at least two possible alternative designs of the future RI;
- WP3 – Context Analysis. This is the key WP to conduct the First Phase of the (Operational and Feasibility phases);
- WP4 – Concept Design, IT Structure and feasibility assessment of the Infrastructure. WP4 will be in charge to work on the RI design, the integration of the thematic platforms, the definition of the IT technologies and the set-up of pilot IT tools if needed for pilot studies;
- WP5 – Thematic Platform supporting Paediatric Medicines Discovery and Early Drug Development. This platform is aimed to implement enabling technologies in order to accelerate discovery and early development in paediatrics such as the use of bioinformatics and computer modelling, the accelerated target identification and the proof of concept studies, and the imaging techniques in System Biology;
- WP6 – Thematic Platform supporting Biomarkers Use in Paediatric Medicines Development. This platform is aimed to generate optimal conditions for the development and use of paediatric biomarkers, with particular emphasis on enhancing the potential of -omics science in paediatrics;
- WP7 – Thematic Platform on Paediatric Pharmacology. This multidisciplinary platform on human development and in vivo/in vitro modelling will support cross-disciplinary research into paediatric pharmacology implementing age appropriated technologies in drug development including animal and cellular models for paediatric diseases, human developmental pharmacology, physiologically based PK (PBPK), pop-PK and PK/PD and modelling;
- WP8 – Thematic Platform on Formulation Science. This Platform will federate the necessary expertise and competences within pharmaceutical sciences to support drug development for the paediatric population and to facilitate drug evaluation in children by providing appropriate age related formulation;
- WP9 – Thematic Platform to relate work that underpins Medicines Development to Paediatric Clinical Studies. This platform will have a huge relevance in promoting the integration of innovative technologies in paediatric drug development with clinical trials;
- WP10 – Ethics. In cooperation with other WPs by providing advice on the context of the project documents under an ethical point of view;
- WP11 – Communication, Networking and Patients Involvement. WP11 will be aimed to develop a strategy and the tools for communication and dissemination in order to support the long-term goals of EPTRI and at the same time to contribute to generate a patient centric approach to the design of the infrastructure for the paediatric clinical research.
CVBF, OPBG and PENTA partners involve INCIPIT in the project activities and particularly in the WP9, which aims to promote the integration of innovative technologies in the development of pediatric drugs and clinical trials.
European Union’s Horizon 2020 programme
3.072 million euro
3.00 million euro
(1) Consorzio per Valutazioni Biologiche e Farmacologiche (CVBF), Italy; (2) Fondazione PENTA – For the treatment and care of children with HIV – ONLUS (PENTA), Italy; (3) European Infrastructure for Translational Medicine (EATRIS ERIC), The Netherlands; (4) University College London (UCL), United Kingdom; (5) ATHENA Research and innovation center in information communication & knowledge technologies (ATHENA), Greece; (6) Biobanks and BioMolecular resources Research Infrastructure Consortium (BBMRI-ERIC), Austria; (7) The Cyprus foundation for muscular dystrophy research (CING), Cyprus; (8) Assistance Publique – Hopitaux de Paris (AP-HP), France; (9) Stichting Katholieke Universiteit (RUMC), The Netherlands; (10) The University of Liverpool (ULIV), United Kingdom; (11) Romanian Angel Appeal Foundation (RAA), Romania; (12) Instytut Pomnik Centrum Zdrowia Dziecka (IPCZD), Poland; (13) Ospedale Pediatrico Bambino Gesù (OPBG), Italy; (14) Fyziologicky Ustav Akademie ved Ceske Republiky Verejna Vyzkumna Instituce (IPHYS), Czech Republic; (15) Fundacio Sant Joan de Deu (FSJD), Spain; (16) Nizhegorodskiy Gosudarstvenniy Universitet Im N.I. Lobachevskogo (UNN), Russia; (17) Servicio Madrileno de Salud (SERMAS-HULP), Spain; (18) European Clinical Research Infrastructure Network (ECRIN-ERIC), France; (19) Qendra Spitalore Universitare Nene Tereza Tirane (UHCT), Albania; (20) TECHNION – Israel Institute of Technology (TECHNION), Israel; (21) Tartu Ulikool (UTARTU), Estonia; (22) Universitatsklinikum Erlangen (UKER), Germany; (23) Swiss Clinical Trial Organisation Verein (SCTO), Switzerland; (24) Vastra Gotaland Lans Landsting (VGR), Sweden; (25) Universitair Medisch Centrum Utrecht (UMCU), The Netherlands; (26) State “Institute of Pediatrics Obstetrics and Gynecology National Academy of Medical Sciences of Ukraine” (UKR), Ukraine.
Other organizations linked to the project
Italian Network for Paediatric Clinical Trials (INCIPIT)
Third parties involved in the project
Fondazione per la Ricerca Farmacologica Gianni Benzi Onlus “FGB”, Italy (CVBF third party); TEDDY European Network of Excellence for Paediatric Clinical Research, Italy (CVBF third party); Azienda Ospedaliero Universitaria Consorziale Policlinico di Bari, Italy (CVBF third party); University of Bari Aldo Moro “UNIBA”, Italy (CVBF third party); Università degli Studi di Milano-Bicocca, Italy (BBMRI third party ); Fundación para la Investigación Biomédica del Hospital Universitario La Paz, Spain (SERMAS-HULP third party); Consorzio per Valutazioni Biologiche e Farmacologiche – Dege e shoqerise se huaj “CVBF-AL”, Albania (UHCT third party); Technion Research & Development Foundation Ltd. (“TRDF”), Israel, (TECHNION third party).
Arthritis and Rheumatism Association Malta (ARAM), Malta; Asociación Española de Pediatría (AEP), Spain; Associação Nacional de Displasias Ósseas (ANDO), Portugal; Childhood Cancer International (CCI), The Netherlands; Cyprus Institute of Neurology and Genetics, Cyprus; Department of Paediatric Neurology, University Children’s Hospital Ljubljana, Slovenia; European Network for Rare and Congenital Anaemias (ENERCA), Spain; Eurordis, Rare Disease Europe, France; Foundation MÁS QUE IDEAS, Spain; Foundation of child neurology, Slovenia; Fundaciòn ALPE acondroplasia, Spain; Greek Carers Network – EPIONI, Greece; LIFE (ZIVOT) – Organization for children rare diseases, Serbia; Norwegian Melanoma Patient Association (Foflekkreftforeningen), Norway.