Periodic Report Summary 1 - ECRIN-IA (European Clinical Research Infrastructures Network - Integrating Activity)
Project Context and Objectives:
Clinical research is critical for the development of innovative preventive, diagnostic and therapeutic procedures and tools, and for the optimisation of their use in healthcare, with a major impact on science, on health, and on health economy at the national, European and global levels. Access to patients is a key success factor for clinical research, requiring international cooperation. However fragmentation of health and legislative systems raises major obstacles to multinational trials in Europe. For this reason, the European Clinical Research Infrastructures Network (ECRIN, www.ecrin.org) was designed to support multinational clinical research, with the objective of making Europe a single area for clinical trials. ECRIN applied for an ERIC status, and the ECRIN-ERIC budget will cover the core activities required to enable operational provision of services to multinational trials in Europe. In turn, the ECRIN IA project is designed structure the users communities, to develop common tools, and to expand ECRIN partnerships and impact.
Through the networking and joint research activities and the transnational access, the objectives of this ECRIN IA project are to:
- Expand the network and strengthen the national partners and partnership with other world regions
- Develop pan-European investigation networks in three strategic areas (rare diseases, medical devices, nutrition)
- Develop a common and de-fragmented culture and common tools for multinational projects.
As part of the networking activities:
a) The WP2 promotes pan-European expansion and the capacity of ECRIN to support multinational clinical studies in Europe, and develops partnerships with other world regions.
b) The WP3 uses material collected by WP4, 5 and 6, and previous ECRIN material to develop e-services, and education material and toolkits to train the trainers for professionals and patients associations. It also fosters communication with users, patients, citizens and policymakers.
c) The WP4 (rare disease), WP5 (medical devices) and WP6 (nutrition) implement parallel strategies to support the structuring and connection to ECRIN of pan-European networks and hubs in the three specific areas. These WPs also develop and adapt common tools for clinical research in these specific areas. These infrastructures will be used to design and run multinational clinical studies in their respective medical fields, with the support provided by WP7.
As regards of transnational access activities, the WP7 provides access to, and support the cost of, services for the multinational implementation of clinical trials on rare diseases, medical devices and nutrition selected by the ECRIN scientific board.
The objectives of the joint research activities are to improve the quality and enlarge the spectrum of services proposed by ECRIN through the:
- Development of a toolkit of validated strategies for cost-effective, risk-based monitoring (WP8)
- Upgrade the VISTA data management tool, to make a professional data management software available at a not-for-profit cost for the academic community.
During the first 18 months of the project, the ECRIN-IA consortium achieved significant results and the project developed as planned :
- The transnational access activity started with the successful launch of a competitive call (WP7) resulting in the selection, by the ECRIN scientific board, of 8 clinical trials that will benefit from multinational support provided by ECRIN-IA partners over 21 countries.
- The WP4 (rare disease), WP5 (medical devices) and WP6 (nutrition) performed the mapping and an inventory of the research capacities in Europe, and collected the national regulatory and ethical requirements specific for those three areas. For the nutrition area this resulted in the establishment of a network of 61 nutrition research centres across 18 European countries, with a hub located in Lyon (France) acting as a single entry and contact point for the nutrition clinical research community, and coordinating the development of common procedures and tools. A similar process is ongoing for the medical device and rare diseases clinical research communities. In addition, a joint methodology workshop was held in February 2013, with experts in rare diseases, medical devices and nutrition, to address the specific methodological issues raised by clinical trials in these areas (including small sample size, blinding, comparator, control group), and resulting in an article in preparation.
- The ECRIN regulatory and ethical know-how was updated and upgraded with the collection of regulatory and ethical requirements for clinical research in the 9 new countries joining ECRIN-IA and for rare diseases, medical devices and nutrition areas.
- Communication targeting the ECRIN community, the users communities, and the patients representatives was carried-out through various channels: organisation of the ECRIN IA summer school dedicated to train the ECRIN European correspondents; development of the users networks within WP4, -5 and -6; organisation of the International Clinical Trials Day (18 May 2012 and 17 May 2013), and participation in the Eurordis summer school and the development of on line interactive tools for patient advocates..
- Partnerships with other world regions were successfully established, numerous contacts for global collaboration in clinical research were established outside the European Union; following its involvement in the OECD initiative to facilitate international collaboration in clinical trials, ECRIN is now responsible for the global implementation group on infrastructure and funding, under the umbrella of WHO.
- the new European Correspondents were integrated within the network and involved in the whole spectrum of ECRIN activities – structuring activities and operations. The transnational access (WP7 competitive call) plays a critical role in the project as it involves in the operations not only the three networks under development (rare diseases, medical device, nutrition), but also almost all the countries participating in the project (21 countries), acting as a pilot for most of the European Correspondents in supporting multinational clinical trials.
- The upgrade of the VISTA data management tool (WP9), following the ECRIN specifications, resulted in the release of the first VISTA module for internal testing.
- A systematic review on monitoring practices and recommendations in clinical trials was performed by the WP8 allowing the identification of the key elements of monitoring activities and tools for risk-based monitoring strategies. This activity has a global impact on the implementation of the OECD Council Recommendation on the risk-based governance of clinical trials.
- In spite of the size of the ECRIN-IA consortium, the scientific activities and general project management have been working efficiently, taking advantage of a robust and simple management structure and well-defined decision making processes, regular communication and meetings.
Clinical research supports evidence-based decision in clinical practice , addressing the question of what is the best treatment option for a disease condition / group of patients. Depending on the disease and on the products available, this may correspond to the development of innovative health products, to the exploration of new indications for already marketed products, or to the comparative assessment of efficacy and safety of existing healthcare (or nutrition) strategies.
As a consequence, clinical research has a major societal impact as it represents a key instrument for treatment optimisation and healthcare cost containment, for the benefit of health authorities, of healthcare professionals, and of course for the benefit of patients.
However, due to the cost and complexity of clinical research, and to the need to access patients and relevant expertise, an efficient organisation of clinical research requires international cooperation. The long-term objective of ECRIN-IA is to contribute to the structuring, at the pan-European level, of a clinical research capacity strenghtening the scientific competitiveness and attractiveness for clinical trials. For this purpose, ECRIN-IA adresses four aspects :
- Structuring pan-European investigator communities in rare diseases, medical device, and nutrition (WP4-5-6), is a critical element for the design of relevant clinical trials : clinical questions have to be discussed across a broad expert community before designing a trial, taking into account systematic review of curent evidence and uncertainty. In addition, the ECRIN-IA methodology task force provides additonal expertise to optimise trial design. Finally, the role of the ECRIN Scientific Board is to improve the methodological standards and the clinical relevance of the supported trials (WP7). This is also the reason why ECRIN-IA includes in its scientific board patients representatives for the selection of the supported projets.
- Conducting these trials requires appropriate infrastructure, both at the national and international level, to ensure efficient patient recruitment, data quality and credibility of the results. In this perspective, ECRIN-IA contributes to the development or upgrade of national infrastructure through its capacity building programme (WP2), and of the pan-European integration of these infrastructures through the structuring of pan-European investigation networks in rare diseases, medical devices and nutrition (WP4-5-6), through the development of education programmes, regulatory databases and e-services (WP3), through the development of common tools for data management (WP9) and monitoring (WP8), and through the experience derived from the conduct of WP7-funded trials.
- Funding for multinational investigator-driven trials is a major bottleneck for academic research. ECRIN-IA makes an attempt to close this funding gap in a model where trials already funded in the coordinating countries recieve additional funding through the ‘transnational access’ scheme (WP7) for implementation in other countries. However other funding mechanisms have to be discussed, based on H2020 cooperation projects, but also based on national funding : availability for foreign investigation sites, mutual recognition in the selection of projects… This will be discussed during the WP2 funding workshop, and in the context of the ERIC with the Ministries of the relevant countries.
- Finally interoperability is a key element to enable international collaboration and to boost the scientific competitiveness of Europe in clinical research. Fostering interoperability includes the development of common monitoring (WP8) and data management (WP9) tools for international trials, made available to the EU scientific community, the common regulatory database and e-service tool, the shared training syllabus for clinical investigators (WP3), the lobbying for a better harmonised regulation at the EU and the global levels. Moreover, the pan-European rare diseases, medical device and nutrition (WP4-5-6) networks will share common sets of outcome measures and various specific tools and methodologies for clinical trials. Finally the WP2 capacity building programme allows newly connected countries to adopt the already delevoped tools and to take into account, for the development of their own national infrastructure, the best adapted models in the other countries, thus fostering interoperability.
List of Websites: