Report

Teaser, summary, work performed and final results

Periodic Reporting for period 1 - ID-EPTRI (Infradev - European Paediatric Translational Research Infrastructure)

Teaser

Minors represent 20% of the European population and their care is one of the most important priority and challenge for Europe. Nowadays most of the medicines addressed to children and young patients have not been tested specifically for them, after being developed for adult...

Summary

Minors represent 20% of the European population and their care is one of the most important priority and challenge for Europe. 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.

Children and young patients are not little adults and they 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. For this reason, it is important to develop better medicines for children and young patients by studying them specifically and/or using extrapolation methods whenever possible.

Moreover, there is the need to enhance in the paediatric sectors the many innovative technologies currently used in drug development covering different phases from medicines discovery and preclinical research to age tailored formulations and medical devices.

In this context EPTRI (European Paediatric Translational Research Infrastructure) has been set up as an EU project coordinated by Consorzio per Valutazioni Biologiche e Farmacolo¬giche (CVBF) with 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. Two main scientific approaches are relevant for EPTRI to reach the stated aim: better understanding of the molecular basis of human development from the foetal to the adult age, and adaptation of recently developed novel technologies in drug development and new therapies to the paediatric population.

The new paediatric RI will be the result of the integration of many existing (but currently disaggregated) paediatric competences in Europe in the field of basic and preclinical science aimed to foster the development of innovative health products for children in a structured cooperative framework.

EPTRI already involves about 30 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:
I. to support and stimulate excellent science in the field of human development, in order to establish novel knowledge and standards for the development of drug and therapies dedicated primarily to children health (scientific goal);
II. to aggregate the research capacities existing in the EU, supporting them to enhance research and innovative technologies in the sector of paediatric drug development (structural goal);
III. to bridge the scientific advancements in human development and early medicines research in the whole drug developmental process and to underpin paediatric clinical research (strategic goal);
IV. to take advantage of the existing RIs that can provide tools and resources to reduce the gap by avoiding duplications (operational goal).

EPTRI will be composed of five technical and scientific domains including:
- 4 thematic research platforms:
Paediatric Medicines Discovery
Paediatric Biomarkers and Biosamples
Developmental Pharmacology
Paediatric Medicines Formulations and Medical Devices
- the scientific domain “Underpinning medicines development to paediatric studies” integrating Key Technologies into medicines development process in children and acting as a bridge between EPTRI and other paediatric initiatives and clinical trials networks.

Work performed

EPTRI officially started with the Kick-off meeting in Rome at the Italian Ministry of Education, University and Research (MIUR) on January 15th-16th, 2018.

As first step of the project, a context analysis has been conducted using on line questionnaires and direct contacts for review of the responder contents and to get confirmation of interest. Context analysis allowed to scan competencies, available services and technologies to be included in EPTRI. Data collection is still ongoing. At the time of the report a total of 308 responses have been collected.
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 drug development and to acquire preliminary ideas and feelings from the future users, as well as information on their interests and needs in terms of access, costs, barriers.
The higher rates of responses for both surveys are from Germany, Italy, the Netherlands, Spain and UK.

In addition, during the context analysis, dedicated initiatives were addressed to other relevant stakeholders such as:
- National and European Authorities (European Commission, the National and Regional Governments, the Health Governing Bodies)
- Existing ESFRI biomedical research infrastructures
- Networks and other relevant paediatric initiatives
- Academia, Universities and Scientific Associations
- Pharma and Biotech Industry, Contract Research Organizations (CROs)
- 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.

The feasibility phase is ongoing since June 2019 with a call for application to EPTRI feasibility studies open both to the project partners and to external providers/users. Feasibility studies will be aimed to test the process, the scientific interest, the availability of requested services and facilities that could be needed to conduct paediatric research in the areas of competence of EPTRI.

Final results

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, innovative and advanced therapies as resulting from the advancement of biological and medical sciences;
• link advancements in paediatric research to the rapid development of regulatory provisions;
• map the paediatric research needs as well as the opportunities to access to high-tech research facilities.

Website & more info

More info: https://eptri.eu/.