Periodic Reporting for period 1 - ERICA (European Rare dIsease research Coordination and support Action)
Reporting period: 2021-03-01 to 2022-08-31
Rare diseases affect no more than 1 person in 2000, however collectively they affect 30 million people in the EU. In the area of rare diseases, knowledge exchange and research within the EU and international collaboration is indispensable.
For this purpose, under the EU directive 2011/24/EU on cross-border healthcare the European Commission and the EU Member States have jointly installed European Reference Networks (ERNs) for rare diseases. The mission of the ERNs includes the better sharing across borders of existing knowledge on rare diseases and generation of new knowledge on rare diseases and complex procedures, as stated in the EU directive 2011/24/EU.
The aim of the European Rare Disease Research Coordination and Support Action consortium (ERICA), in which all 24 European Reference Networks (ERNs) take part, is to build on the strength of the individual ERNs and create a platform that integrates all ERNs research and innovation capacity.
The overall objective of ERICA is to increase the research and innovation potential of ERNs through facilitating inter-ERN collaboration and increase the visibility and impact of ERNs through outreach to and collaboration with multiple stakeholders.
Through knowledge sharing, engagement with stakeholders in the rare disease domain and assembly of transdisciplinary research groups working across the global health spectrum, ERICA strives to reach the following goals:
• new intra- and inter-ERN rare disease competitive networks;
• effective data collection strategies;
• better patient involvement;
• enhanced quality and impact of clinical trials;
• increased awareness of ERNs innovation potential.
ERICA will strengthen research and innovation capacity by integration of ERN research activities, outreach to European research infrastructures to synergistically increase impact, and innovation. This will result in efficient access and safe therapies for the benefit of patients suffering from rare diseases and complex conditions.
The overall objective of ERICA is to increase the research and innovation potential of ERNs through facilitating inter-ERN collaboration and increase the visibility and impact of ERNs through outreach to and collaboration with multiple stakeholders. All endeavors will be in close agreement and collaboration with all other European initiatives on rare diseases where the focus will be to enhance synergy.
The following specific objectives are followed and designed, based on the results of the survey and to support the overall objective:
• Objective 1: Record, coordinate, and disseminate ERN clinical research activities in line with ERICA’s strategic positioning (WP1, WP6)
• Objective 2: Facilitate collection, sharing and analysis of clinical research data within and between ERNs (WP2, WP4)
• Objective 3: Advance patient-centred research and outcomes monitoring by promoting the identification and application of patient-centred outcome measures for rare diseases (WP3)
• Objective 4: Map, integrate and link ongoing activities with regards to facilitating Europe-wide multicentre clinical trials (WP4)
• Objective 5: Encourage methodology sharing and increase the innovation potential of ERNs (WP4, WP5)
For this purpose, under the EU directive 2011/24/EU on cross-border healthcare the European Commission and the EU Member States have jointly installed European Reference Networks (ERNs) for rare diseases. The mission of the ERNs includes the better sharing across borders of existing knowledge on rare diseases and generation of new knowledge on rare diseases and complex procedures, as stated in the EU directive 2011/24/EU.
The aim of the European Rare Disease Research Coordination and Support Action consortium (ERICA), in which all 24 European Reference Networks (ERNs) take part, is to build on the strength of the individual ERNs and create a platform that integrates all ERNs research and innovation capacity.
The overall objective of ERICA is to increase the research and innovation potential of ERNs through facilitating inter-ERN collaboration and increase the visibility and impact of ERNs through outreach to and collaboration with multiple stakeholders.
Through knowledge sharing, engagement with stakeholders in the rare disease domain and assembly of transdisciplinary research groups working across the global health spectrum, ERICA strives to reach the following goals:
• new intra- and inter-ERN rare disease competitive networks;
• effective data collection strategies;
• better patient involvement;
• enhanced quality and impact of clinical trials;
• increased awareness of ERNs innovation potential.
ERICA will strengthen research and innovation capacity by integration of ERN research activities, outreach to European research infrastructures to synergistically increase impact, and innovation. This will result in efficient access and safe therapies for the benefit of patients suffering from rare diseases and complex conditions.
The overall objective of ERICA is to increase the research and innovation potential of ERNs through facilitating inter-ERN collaboration and increase the visibility and impact of ERNs through outreach to and collaboration with multiple stakeholders. All endeavors will be in close agreement and collaboration with all other European initiatives on rare diseases where the focus will be to enhance synergy.
The following specific objectives are followed and designed, based on the results of the survey and to support the overall objective:
• Objective 1: Record, coordinate, and disseminate ERN clinical research activities in line with ERICA’s strategic positioning (WP1, WP6)
• Objective 2: Facilitate collection, sharing and analysis of clinical research data within and between ERNs (WP2, WP4)
• Objective 3: Advance patient-centred research and outcomes monitoring by promoting the identification and application of patient-centred outcome measures for rare diseases (WP3)
• Objective 4: Map, integrate and link ongoing activities with regards to facilitating Europe-wide multicentre clinical trials (WP4)
• Objective 5: Encourage methodology sharing and increase the innovation potential of ERNs (WP4, WP5)
Work performed from the beginning of the report and main results achieved so far are:
- The ERICA kick-off meeting / 1st General Assembly (GA) was held 27-28th May 2021 and 2nd GA was held 20-22 June in Bologna, Italy.
- Installation of the Managerial bodies and Advisory Boards, introduction to the Consortium members, and integration within the executive board meeting every six months and in the WP specific expert working groups.
- The ERN Research Roadmap is developed as living document. Integration of ERN research efforts and alignment with the important partnerships are the primary goals of this task and are formulated into the ERICA common research roadmap.
- Common Data access policy documents, Common Informed consent policy and forms have been developed and available to customize according to the needs of the ERNs.
- Recommendations report on registry data collection have been composed and available for all the ERNs.
- The development and launch of the first version of the ERICA PROMs Repository at ERICA web-platform.
- Report on the state-of-the-art of available PCOMs/PROMs and overlap/gap analysis.
- Procedure to provide ERN expert availability to EMA for expert opinion on Rare Diseases.
- Procedure for producing factsheets on the theme: "You know of ..." and YouTube videos have been defined.
- To help the ERN community in understanding the process of translation and to support their collective ability to develop knowledge into patient benefit, a catalogue of current translational research services has been developed, which is now available to the rare diseases research community.
- The established Innovation Expert Working Group (I-EWG) worked together in writing the Innovation roadmap and strategic plan.
- Development of the Dissemination plan & toolkit.
- The ERICA web-platform has been launched and is under continuous development.
- The ERICA kick-off meeting / 1st General Assembly (GA) was held 27-28th May 2021 and 2nd GA was held 20-22 June in Bologna, Italy.
- Installation of the Managerial bodies and Advisory Boards, introduction to the Consortium members, and integration within the executive board meeting every six months and in the WP specific expert working groups.
- The ERN Research Roadmap is developed as living document. Integration of ERN research efforts and alignment with the important partnerships are the primary goals of this task and are formulated into the ERICA common research roadmap.
- Common Data access policy documents, Common Informed consent policy and forms have been developed and available to customize according to the needs of the ERNs.
- Recommendations report on registry data collection have been composed and available for all the ERNs.
- The development and launch of the first version of the ERICA PROMs Repository at ERICA web-platform.
- Report on the state-of-the-art of available PCOMs/PROMs and overlap/gap analysis.
- Procedure to provide ERN expert availability to EMA for expert opinion on Rare Diseases.
- Procedure for producing factsheets on the theme: "You know of ..." and YouTube videos have been defined.
- To help the ERN community in understanding the process of translation and to support their collective ability to develop knowledge into patient benefit, a catalogue of current translational research services has been developed, which is now available to the rare diseases research community.
- The established Innovation Expert Working Group (I-EWG) worked together in writing the Innovation roadmap and strategic plan.
- Development of the Dissemination plan & toolkit.
- The ERICA web-platform has been launched and is under continuous development.
The project is well-progressing in line with the general goals and significant progress has already also been made towards the specific objectives through the work carried out under the distinctive ERICA work packages. As a result of resumed in person networking events and dissemination activities increased involvement of stakeholders is expected, which helps to accelerate the expected impacts even further.
The individual ERNs are in continuous development and well organised. Although there is still noticeable differences among ERNs, the Europe wide networks for diagnosis and care of patients with rare and low prevalent diseases are in place for the vast majority of rare diseases in 24 different ERNs. We envision capacity building through knowledge sharing by coordinated support of collaborative research activities of the ERNs. Through networking and the organisation of thematic workshops related to the defined ERN research priorities we intend to display knowledge present within a particular ERN and make this assessable for the benefit of other ERNs that lack this knowledge.
As such, ERICA will also contribute and lay a solid foundation for the new RD Partnership.
The individual ERNs are in continuous development and well organised. Although there is still noticeable differences among ERNs, the Europe wide networks for diagnosis and care of patients with rare and low prevalent diseases are in place for the vast majority of rare diseases in 24 different ERNs. We envision capacity building through knowledge sharing by coordinated support of collaborative research activities of the ERNs. Through networking and the organisation of thematic workshops related to the defined ERN research priorities we intend to display knowledge present within a particular ERN and make this assessable for the benefit of other ERNs that lack this knowledge.
As such, ERICA will also contribute and lay a solid foundation for the new RD Partnership.