Report

Teaser, summary, work performed and final results

Periodic Reporting for period 1 - PedCRIN (Paediatric Clinical Research Infrastructure Network)

Teaser

Children’s health is a major societal challenge for Europe and the world, over 50 % of the medicines used for children had not been tested in this specific age group. Conducting clinical trials in children requires specific competences and infrastructures. ECRIN-ERIC...

Summary

Children’s health is a major societal challenge for Europe and the world, over 50 % of the medicines used for children had not been tested in this specific age group. Conducting clinical trials in children requires specific competences and infrastructures. ECRIN-ERIC (www.ecrin.org) is a distributed infrastructure providing services for the management of multinational trials, in any disease area. Some of the ECRIN-supported clinical trials are in the paediatric area, however ECRIN does not specifically address the paediatric needs in terms of trial management capacity. The PedCRIN project was developed following a suggestion from the European Strategy Forum on Research Infrastructures (ESFRI). In its 2016 Roadmap, a project on paediatric clinical research was submitted to ESFRI (the European Paediatric Clinical Trial Research Infrastructure-EPCTRI) to build an infrastructure for paediatric trials. To avoid duplication and fragmentation, ESFRI suggested the paediatric community to propose a joint project with ECRIN, to further develop its capacity for the management of multinational investigator-initiated paediatric trials. At the same time the Innovative Medicine Initiative (IMI) decided to launch a call for the structuring of the paediatric clinical investigation capacity in Europe, which resulted in the IMI c4c (Connect for Children) project, to which all the PedCRIN consortium is participating. This is a very positive situation however requires careful distinction between the mission of PedCRIN (focusing on trial management) versus c4c (focusing on the investigation capacity). An excellent coordination, and continuous discussion between the ECRIN partners and the paediatric community is needed to avoid duplication and fragmentation. The PedCRIN project therefore aims to broaden the operational capabilities of ECRIN, by connecting to new clinical trial units with paediatric expertise, thus developing its capacity to manage multinational paediatric trials, and by attracting new users’ communities able to design multinational neonatal and paediatric trials. The PedCRIN project is also a unique opportunity for ECRIN to improve its business model and financial sustainability, attracting more industry-sponsored trials and more Member and Observer countries. Two other ESFRI-landmarks, BBMRI-ERIC and EATRIS-ERIC, are also contributing to PedCRIN. The PedCRIN project builds on six work packages: Project management & coordination (WP1), sustainability, strategy, governance, business plan (WP2), tools for paediatric and neonatal trials (WP3), pilot trials (WP4) & communication, dissemination, empowerment (WP5) and ethics requirements (WP6).

Work performed

During the reporting period the PedCRIN consortium achieved significant results and the project developed as planned. The transnational access activity started with the launch of a competitive call (WP4) to select pilot trials. A single stage call for applications was launched in February 2017 to provide operational and management support to the selected pilot clinical trials in the form of “transnational access”. In response to this call 13 applications were received from six different EU countries. Three trials (WE, POPART & OTBB3) were selected for PedCRIN funding. In order to optimize the forthcoming PedCRIN services and activities, a survey was launched (WP3) for the identification of the needs and expectations of paediatric medical research communities. Survey has shown that the most requested need for the support was the preparation of protocols for paediatric interventional clinical trials, including the application of innovative approaches in the design of studies. To meet this need, a methodology task force will be established through PedCRIN partners. Gap analysis was performed (WP3) to detect the existing and missing tools and services to support paediatric and neonatal clinical trials. In this context the paediatric specific contents currently available on ECRIN CAMPUS database was analysed and will be updated in line with the new regulations. Likewise “The Liverpool ADR Causality Assessment Tool” is identified and will be tested in PedCRIN funded pilot studies. Criteria for quality assessment of neonatal and paediatric Clinical Trials Units is underway. A working group is established by ECRIN for the certification of centres being able to perform pharmacovigilance activities, also taking paediatric specific requirements into consideration. For the communication and dissemination (WP5), a press release was published about the launch of the project and a factsheet was developed for the lay audience. The project website, logo, brochure, newsletter and social media profiles were created. The PedCRIN Advisory Board was established (WP1) to support the strategic decisions, quality and excellence of the project.

Final results

Due to the complexities associated with the paediatric clinical trials many researchers deter from designing and conducting multinational trials, which are not only important for the assessment of new drugs, but also for the optimal use of available treatments. Moreover, academic trials face an increasing number of challenges, including insufficient funding, divergent regulations and fragmented health and health research systems. PedCRIN will contribute to an integrated EU landscape of sustainable Research Infrastructures that respond to challenges in science, industry and society. Easy access to multinational studies through PedCRIN support will enhance the ability of academic and independent investigators to study novel approaches to improving child health or to optimise existing solutions. A major impact of PedCRIN will be the accelerated completion of the PedCRIN funded pilot trials. Once completed this data will reach the high quality level requested to support applications for marketing authorization and health technology assessment underpinning medicines availability. At the EU level, PedCRIN will promote studies that require cross-border collaboration, and thereby enhance the position of Europe as a place to conduct clinical studies. The removal of barriers to trial management through PedCRIN will speed up the evaluation of new and improved therapies, and by reducing time and cost of multinational studies will stimulate investigators and funders to conduct more multinational studies. The PedCRIN project is expected to result in the expansion of partnerships within ECRIN, through the connection of ECRIN European Correspondents and National Scientific Partners (in charge of coordinating and providing trials management services, respectively) with national EPCTRI paediatric experts. This will result in broadening the technical capabilities of ECRIN, in connecting new clinical trial units with paediatric expertise, and in attracting new users’ communities able to design multinational paediatric trials requesting ECRIN services. Development of tools for multinational clinical trials will promote better performances in paediatric research and will catalyse opportunities for the scientific community to adopt innovative tools and methodologies. Improving paediatric care therefore “supports the development of new, safer and more effective interventions”, will “improve understanding of the causes and mechanisms underlying health, healthy ageing and disease” and promotes “better health for all”. Paediatric research therefore impacts health care to a greater extent than any other age groups.

Website & more info

More info: http://www.pedcrin.org.