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Infradev - European Paediatric Translational Research Infrastructure

Periodic Reporting for period 2 - ID-EPTRI (Infradev - European Paediatric Translational Research Infrastructure)

Reporting period: 2019-07-01 to 2019-12-31

Nowadays most of the medicines addressed to children and young patients have not been tested specifically for them, after being developed for adult use. Rarely medicines are specifically developed for children use.

In Europe and worldwide only 30% of marketed drugs include a paediatric authorization with serious risks of lack of cure and/or inappropriate ‘off-label’ medicines’ use. Children and young patients cannot be compared with adults as they are growing up and their metabolism is different. This “special” population is indeed made of several different age groups with different and variable characteristics.

Minors represent 20% of the European population and their care is one of the most important priority and challenge for Europe. For this reason, it is important to develop better medicines for children and young patients by studying them specifically.

EPTRI (European Paediatric Translational Research Infrastructure) has the aim to enhance technology-driven paediatric research in discovery and early development phases to be translated into clinical research and paediatric use of medicines. It will be the result of the integration of many existing (but currently disaggregated) paediatric competences in Europe in the field of basic, preclinical and translational research aimed to foster the development of new and innovative health products for children in a structured cooperative framework.

EPTRI already involves 29 partners from EU and non-EU countries, including established RIs, non-profit research organisations, top-level universities, scientific and clinical centers of excellence, while more than 300 research units provided their availability to participate to the delivery of services and paediatric expertise in the EPTRI context.

EPTRI key objectives are:
o to extend the knowledge of the normal and pathological human development process in order to move from the current approach based on adult drugs that are administered off label to children to a new approach characterised by medicines studied and marketed for children, and consequently to reduce the existing gap on medicines availability for children
o to involve multiple experts and competences to create multidisciplinary scientific and technology groups working to integrate basic science with key technologies (from biological, technological and data science advancements) to drive innovative medicines and health products discovery and development
o to enhance clinical research and paediatric medicines availability contributing to creating a unique paediatric research framework
o to accelerate the paediatric drug development processes from medicines discovery, biomarkers identification and preclinical research to developmental pharmacology, age tailored formulations and medical devices, with the final goal to facilitate the translation of the acquired new knowledge and scientific innovation into paediatric clinical studies and medical use.

EPTRI is based on five technical and scientific domains:
Paediatric Medicines Discovery
Paediatric Biomarkers and Biosamples
Developmental Pharmacology
Paediatric Medicines Formulations
Underpinning paediatric clinical studies.
The main output of the project is the preparation of the Conceptual Design Report (CDR), for which several phases have been foreseen: context analysis, operational and feasibility phases.
Context analysis allowed to scan competencies, available services and technologies to be included in EPTRI. At the time of the report a total of 329 research units provided their availability.
Moreover, an online survey of users has been delivered with the aim to identify the nature and extent of the needs for integrated approaches to paediatric drug development. The users’ survey has been crucial to acquire preliminary ideas and feelings from 337 users, as well as information on their interests and needs in terms of access, costs, barriers. The survey has also investigated the willingness of some users to be engaged in running pilot activities intended to test EPTRI’s feasibility, and has highlighted the strong recommendation to develop new and complementary areas of research that could lead to the creation of other thematic platforms grouping research units working on advanced therapies, medical devices, artificial intelligence for child health.

In addition, dedicated initiatives were addressed to other relevant stakeholders such as:
- National and European Authorities (European Commission, the National and Regional Governments, the Health and Research Institutions)
- Existing ESFRI biomedical research infrastructures
- Networks and other relevant paediatric initiatives
- Academia, Universities and Scientific Associations
- Pharma and Biotech Industry, Contract Research Organizations (CROs), SMEs and start-ups
- Children, young patients and patients’ organisations.

In the operational phase the results from the context analysis have been used to draft the EPTRI Technical Design and the Conceptual Design Report (CDR), that provides a first draft of the future EPTRI integrating aims and scientific goals, organisation, operation, governance, the IT-architecture model, services to be provided and business plan as preliminary elements.

Finally, several feasibility studies are currently running and would provide information about services/technologies that EPTRI could offer to support the researchers in developing their ideas.
EPTRI will bring a positive impact on the scientific community, by integrating the different research units within the new establishing RI, so to increase the scientific relevance of the paediatric research at international level.

EPTRI will bring together scientists, researchers, regulators and industry to support smarter paediatric medicines development in an atmosphere of innovation and collaboration for the benefit of society, the economy and ultimately patients. It will benefit also children’s health as it will establish the framework to speed-up the drug development process in paediatrics including the top-level research innovations.
In particular, EPTRI will have the following expected impacts:
• enhancing and widely implementing the Key Enabling Technologies (KET) for drug development that have been poorly used in the paediatric field;
• preparing the ground to increase paediatric research and reduce the serious delay to provide children with new and innovative therapies as resulting from the advancement of biological and medical sciences;
• map the paediatric research needs as well as the opportunities to access to high-tech research facilities.