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European Reference Network (ERN) on Rare Multisystemic Vascular Diseases (VASCERN), SGA Proposal 2017 [VASCERN] [769036] - Operating Grant
Project abstract

This is the VASCERN SGA Proposal for 2017, under the ERN Framework Partnership Agreement (FPA) 2017-2021.
The ERN is coordinated by the ERN Network Coordinator, Pr. Guillaume JONDEAU - AP-HP, H�pital Bichat-Claude
Bernard, CRMR Marfan Diseases and related disorders (Centre de R�f�rence Maladies Rares Syndrome de Marfan et
VASCERN aims to facilitate and improve diagnosis, treatment and care for ALL patients suffering from Rare
Multisystemic Vascular Diseases, thus enhancing access to care, and improving quantity and quality of life of these patients.
Networking, sharing and spreading our expertise, promoting best practices, guidelines and clinical outcomes, patient empowerment, improving knowledge through clinical and basic research are among our objectives.
The European Reference Network on Rare Multisystemic Vascular Diseases gathers European highly specialized
multidisciplinary Healthcare Providers (HCPs) in this thematic area of expertise. The Network includes 5 Rare
Diseases Working Groups: Heritable Thoracic Aortic Diseases (HTAD-WG), HHT-WG, Medium Sized Arteries (vascular Ehlers Danlos) (MSA-WG) ,Pediatric and Primary Lymphedemas (PPL-WG) and Vascular Anomalies (VASCA-WG). The specific Patient Group enables Patient representatives to work on common issues and to be involved in all activities. In addition, several Thematic Working Groups are established to better tackle transversal issues which are planned in the Multiannual Work Programme on: eHealth, Training & Education, Patient Registry, Ethics, and Communication / Dissemination.

Summary of context, overal objectives, strategic, relevance and contribution of the action

In order to implement the European Union’s Directive 2011/24/EU on the application of patients’ rights in cross-border healthcare, the European Commission (DG SANTE) has launched the first call for proposal for the creation of the European Reference Networks for rare, low-prevalence and complex diseases on March, 2016. VASCERN, a network of highly specialized experts in rare multisystemic vascular diseases, has been constituted and applied in June 2016 to this first ERN call for proposals. Simultaneously, VASCERN has applied to the call for EU co-funding from the 3rd Health Programme of the European Union, managed by CHAFEA (The Consumers, Health, Agriculture and Food Executive Agency).
European Reference Networks (ERN) are virtual networks involving healthcare providers across Europe. They aim to tackle complex or rare medical diseases or conditions that require highly specialised treatment and a concentration of knowledge and resources. To review a patient’s diagnosis and treatment, ERN coordinators convene a “virtual” advisory board of medical specialists across different disciplines, using a dedicated IT platform and telemedicine tools. This way it is the medical knowledge and expertise that travel rather than the patients.
The first 24 European Reference Networks (ERNs) on Rare Diseases have been approved by the ERN Board of Member States (BoMS) in December 2016, following this first EU Call and an assessment conducted by an Independant Assessment Body mandated by the European Commission. The ERNs have received the ERN label for their first 5 years of operation, and have been officially launched in March 2017.
According to Vytenis Andriukaitis, the European Commissioner for Health and Food Safety : “The value of EU collaboration is particularly clear in the case of rare and complex diseases. […] How can ERNs improve the lives of Europeans? Patients with rare and complex conditions will be able to benefit from the best treatment and advice available for their specific condition. Their doctors will have access to a highly-specialised pool of colleagues from all over Europe. In the first phase, over 900 healthcare units will work together in 24 thematic networks, ranging from childhood cancer to immunodeficiency.”
VASCERN, coordinated at the Hospital Bichat (Assistance Publique-Hôpitaux de Paris) has signed a 5 years Framework Partnership Agreement (FPA) with the European Commission in order to be able to receive annual grant agreement to support VASCERN’s coordination and networking activities. Under this FPA, VASCERN has signed a Specific Grant Agreement (SGA) for its first year of official activities, running from March, 1st 2017 to February, 28th, 2018.
VASCERN, the European Reference Network on Rare Multisystemic Vascular Diseases, is dedicated to gathering the best expertise in Europe in order to provide accessible cross-border healthcare to patients with rare vascular diseases (an estimated 1.3 Million concerned). These include arterial disease (affecting aorta to small arteries), arterio-venous anomalies, venous malformations, and lymphatic diseases. These rare and complex diseases require a multidisciplinary approach to patient care, which involves many different specialists belonging to the HCPs Members’ teams.
VASCERN currently consists of 31 highly specialized multidisciplinary Healthcare Providers (HCPs) from 11 EU Member States and of various European Patient Organisations (ePAG) and is coordinated in Paris, France.
Through our 5 Rare Disease Working Groups (RDWGs) as well as several Thematic WGs and the ePAG – European Patient Advocacy Group, we aim to improve care, promote best practices and guidelines, reinforce research, empower patients, provide training for healthcare professionals and realise the full potential of European cooperation for specialised healthcare by exploiting the latest innovations in medical science and health technologies.
More information available

Methods and means

Overall method: Network Coordination and Conduction of various virtual and Face-to-Face VASCERN Meetings
The importance of people meeting each other’s is crucial when a network is set up; the number of meetings held during the first year illustrates the interactions within VASCERN between the different members. During this first year of operation, the first VASCERN Annual Seminar was held during two days in October 2017, Paris (the VASCERN Days 2017 Full Report (one of this year deliverable) is available at this link: This event enabled our Board to meet formally once this year, and an Assembly meeting to take place with more Healthcare Providers and Patient Representatives. Our Council has met 3 times during the year (one face-to-face and 2 virtual meetings), as planned in our Terms of Reference. In addition, our Working Groups (5 Rare Disease Working Groups and 2 active Transversal Working Groups) have held in total an average of 6.75 monthly meetings during the year (a total of 81 meetings during the year).
Means: Networking & Coordination activities
1/ Virtual tools: Both the WebEx system and the ECP are provided by the European Commission to all of the 24 European Reference Networks on rare diseases.
One of the core principles of the ERNs being that the knowledge should travel rather than the patients; we are therefore also very dependent on these IT platforms as we are indeed a European “virtual” network. The ERN Collaborative Platform (ECP) is provided by the European Commission (ECP) as a Core Service in order to share our documents, a common agenda, a forum for discussion between experts, and to lower the amount of emails and ease communication between all partners. This is our internal communication tool. With more than 500 posts, VASCERN is very active and actually the most active ERN user of the ECP.
2/ Organisation of Networking activities, virtual and face-to-face meetings: The second important mean is the organisation of the networking activities (both face-to-face and virtual) using the co-funding provided by the European Union 3rd Health Programme.
Methods and means to implement our 11 Work Packages
We have detailed in our technical periodic report the specific methods and means for our of our 11 Work Packages.
To summarize, VASCERN’s framework offers the opportunity to share expertise between medical experts, which is one of the main added value of the ERN. Conducting monthly meetings for each of the Rare Disease Working Groups (RDWGs) and for the transversal Working Group and the European Patient Advocacy Group (ePAG) is the overall method to discuss the points necessary to advance on the implementation of our action plan. In addition to our 1st Annual Seminar, these monthly meetings enable to progress and discuss our work packages, including the cases discussions (WP1) patient pathways (WP2), the Pills of Knowledge (WP4), the registries (WP5), the clinical trials and research studies (WP6), the clinical outcome measures (WP8), the clinical guidelines and the do’s and don’ts recommendations (WP9 and WP10). The eHealth and Training/ Education WG meetings were important milestones to implement the WP3 on the creation and development of the VASCERN App and our eLearning tools (WP4 Pills of Knowledge, WP7 Availability of conferences on YouTube), as well as to advice on the WP11 Communication, implemented by the VASCERN Coordination team. The Patient Registry WG have held meetings, allowing lots of progress with regard to our WP5 Registries.
The Coordination team provides VASCERN project management, coordination, communication and the organisation of meetings, among others. In addition, using the co-funding provided by this grant agreement, we are working with service providers for the creation and development of our mobile application (developer), our website (webmaster), design of our communication t

Work performed during the reporting period

During the VASCERN Specific Grant Agreement for Y1, covering the period between March, 1st 2017 and February 28th, 2018, VASCERN’s main activities was to implement our action plan’s 11 work packages thanks to the networking and coordination activities outlined in the above part and to the very high commitment of the experts involved without funding.
WP1: Sharing of experience: discussion of difficult cases
Description of activities: VASCERN’s experts have started to share their experience, expertise and knowledge regarding complex clinical cases

WP2: Definition of patient pathways
Description of activities: Development of a patient pathway within each RDWG, indicating the optimal mode of evaluation of a patient for diagnosis, the optimal evaluation of the patient once the diagnosis is made, the optimal follow-up and the optimal therapy.
WP3: Development and implementation of Cross-border pathways Mobile Application
Description of activities: Development of a free of charge VASCERN cross-border Mobile App for smartphone IOS & Android, sharing information on HCPs and Patient Organisations (services offered, how to reach HCPs, contacts, etc.).
WP4: Pills of knowledge
Description of activities: Development of the VASCERN “Pills of Knowledge”, which are series of short single video-lessons (3 to 10mins maximum) in which experts talk about a single brief topic.
WP5: Registries
Description of activities: Important work conducted by the Registry WG and its technical task force towards the implementation of a VASCERN registry and a FAIR pilot project.
WP6: Clinical trials
Description of activities: Communication on the clinical trials and research projects, scientific publication. Research studies and clinical trials conducted within the network.
WP7: Availability of conferences on YouTube
Description of activities: A specific VASCERN YouTube Channel has been set up in order to share scientific meetings held by our Members, VASCERN conferences, scientific congress events, patient organisation meetings and the Pills of Knowledge.
WP8: Definition of clinical outcomes
Description of activities: VASCERN RDWGs have worked on the definition of clinical outcomes (simple and disease specific).
WP9: Writing Recommendations: Clinical Guidelines
Description of activities: VASCERN five RDWGs have started to work on clinical guidelines (identification, collection, selection, assessment of the existing the existing and writing first guidelines).
WP10: Writing Recommendations: Do’s and Don’ts Factsheets
Description of activities: Writing and validation of the Do’s and Don’ts Factsheets for Rare Vascular Disease Patients facing common situation.
WP11: Communication
Description of activities: VASCERN has created and developed several important communication tools during this first year of activities (website; monthly newsletters; social media pages: Twitter, Facebook, YouTube; communication tools with a graphic designer; translation of content for patient; internal communication on the ECP).

The main output achieved so far and their potential impact and use by target group (including benefits)

A) Structure & Functioning: a new ERN
A major result of this first year is actually the setting up of VASCERN as a network: functioning as an institutionalised ERN, with a structure, a governance, a functional coordination, a coordination team, monthly meetings, an efficient communication and dissemination strategy with newly established tools. Illustrations are the numbers of meetings held over the year and the great success of the VASCERN Days 2017 and all of the outcomes outlined in this report.
This is a major result and has enabled to set up a very strong basis for the following years under the Framework Partnership Agreement.
B) Major results linked to the Work Packages activities and outcomes
WP1: starting to share knowledge and expertise with regard to complex cases between highly specialised experts within the framework of the ERN in order to improve care. More cases proposed, CPMS tested.
WP2: defining and finalising the first VASCERN patient pathways available for testing, publication and dissemination
WP3: creating and developing a VASCERN Mobile application as a tool for patients to easily find and reach out the expert centers they need and the patient organisations in the European Union, therefore facilitating cross-border healthcare
WP4: Online upload of the first 5 Pills of Knowledge videos available on YouTube with one reaching already more than 600 views in 3 months, first content translated for patients (translation of patient information material)
WP5: Work towards the implementation of a VASCERN Registry complying with the FAIR principles. Decision made to support a VASCA FAIR registry pilot project. RDWG specific registry projects ongoing, including the MSA-WG European cohort project enabled by VASCERN.
WP6: Lots of research projects, studies underway within VASCERN, many collaborative. Encouraging and fostering new scientific cooperation in the field of rare vascular diseases. A Comprehensive annual report on clinical trials and research studies including collaborative and HCPs publications available for publication will be published (deliverable).
WP7: Launch of the YouTube Channel, populated with 75 videos
WP8: Definition of the first simple and disease specific outcome measures, ongoing work. Paper submitted by the HHT-WG on how these 5 Outcome Measures provide metrics to identify healthcare providers of good care, and encourage care improvement by all healthcare providers is discussed and explained further within VASCERN HHT’s first manuscript, currently in review: Shovlin, Buscarini et al: European Reference Network for Rare Vascular Diseases (VASCERN) Outcome Measures for Hereditary Haemorrhagic Telangiectasia (HHT) in review Feb 2018.
PPL-WG Quality of Life face to face meeting, working on outcome measures for QoL
WP9: Identification, collection, selection, appraisal of the existing national, European and international guidelines in order to anticipate the writing of new recommendations. Writing of the 2 first VASCERN expert consensus statements, work in progress for a second HHT expert consensus statement.
1/ Shovlin, Buscarini et al, European Reference Network for Rare Vascular Diseases (VASCERN) Outcome Measures for Hereditary Haemorrhagic Telangiectasia (HHT) submitted 12.02.2019.
(As the paper remains in the journal review process, it is not yet available for public release.) + In progress: a second statement on NOACs (new oral anticoagulants). the first draft has been circulated and reviewed, and it will be developed to publication in Year 2.
2/ HTAD-WG expert consensus statement on ACTA2 related HTAD: work in progress
WP10: First 3 RDWGs Do’s and Don’ts Factsheets are ready for official publications
WP11: Establishment of an efficient communication and of new tools (website, monthly newsletters, active communication on social media: Twitter, Facebook, YouTube and now LinkedIn) with good results and outreach.
The benefits and potential use are important for our target group

Achieved outcomes compared to the expected outcomes

Although some of the targets set up in the initial plan were not fully reached, overall we have achieved most of the outcomes expected for this first year of actities as outlined in the previous section. The expected outcomes which are not fully reached are mainly due to the delay with regard to the availability of the CPMS for our WP1, and due to the lack of expert time and funding to fully reached some of the objectives (Pills of Knowledge targets have been reviewed and there are now 5 Pills of Knowledge videos on our YouTube Channel, providing eLearning to clinicians and patients). Processes to produce clinical guidelines and to define the consolidated patient pathways, the clinical outcomes and create a registry need more times. However, there are already 2 expert consensus statements generated, consolidated and draft patient pathways produced and do’s and don’ts factsheets for rare disease patient care ready for publication. Several registry projects are launched, incluiding a VASCA Registry FAIR pilot project. VASCERN has encouraged and fostered scientific cooperation, and new scientific research projects have been launched. Not all the clinical outcomes measures were defined, nevertheless a HHT-WG paper about outcome measures in under journal review for publication. Our VASCERN App is underway to be publicly launched in the upcoming weeks, as the collection of data and development took more time than expected. Our communication activities and results are meeting our expected outcomes.

Dissemination and evaluation activities carried out so far and their major results

The dissemination activities towards our identified target groups have been very important during the course of the project and also after, as the project is running under a 5 years framework partnership agreement, the dissemination activities are not stopping.
Main dissemination activities are:
1/ VASCERN’s Website, diffusing all information about the network and its activities
2/ VASCERN’s Social Media pages (Twitter, Facebook, YouTube Channel and now the LinkedIn (established in March, 2018))
3/ VASCERN’s Monthly Newsletters, which are sent to a broad audience (large diffusion list and publication on our website) including all the patient organisations registered in the EURORDIS database for the VASCERN European Patient Advocacy Group, so that the patients and their families are widely targeted. These Newsletters include all of our results, outputs, news so it is a very important way for VASCERN to disseminate information.
4/ Internal dissemination of information on our ERN Collaborative Platform (as underlined in the first part of this report, VASCERN is the most active ERN user of the ECP with more than 500 posts)
5/ Dissemination via our partners’ websites and social media (Healthcare Providers and Patient Organisations)
6/ Work on the diffusion list with our members to increase our communication impact
7/ Publications in in medical and paramedical journals involving VASCERN members and therefore spreading information about our network
8/ Discussions held with public policy makers
9/ Patient organisations involved: as they are fully involved within VASCERN structure and activities, patients are empowered and disseminate information, educate patients and families within the EU
10/ Translation of patient information material
11/ eHealth tools, including the mobile application under final development
12/ Common answers to research calls and other calls for proposals
13/ Dissemination of results during communication in conferences and scientific congresses, where our Members have been promoting VASCERN: our members are indeed very active in Europe and abroad, participating in scientific congresses, meetings and other events.

Overall, our communication and dissemination activity have good results for this first year (increase of users/ visits on our website and good geographical outreach, numbers of followers on our social media pages and rapid progress regarding these results, implementation of new tools and design of improved dissemination strategies in order to improve results with regard to our outcomes’ outreach).
We have received very good feedback from several stakeholders about our communication activities, including EURORDIS, the European Commission ERN policy team and other ERNs.
Evaluation activities
We have conducted several evaluation activities during the course of the project :
• At the beginning of the project, we sent out a survey to all VASCERN members, both HCP and ePAG representatives, to ascertain their motivation to join one of our transversal working groups (and if so, which one) as well as to receive their feedback on the ERN project in general (i.e. the proposal process, the proposal submitted, areas for improvement, ideas for project implementation). The majority of respondants were very happy with objectives outlined in the project and the overall process lead by the Coordination team.
• At the VASCERN days 2017, all participants (including external stakeholders) were consulted and we requested any feedback on the project during this important annual event. In addition, a satisfaction survey was sent out to all participants following our first annual seminar. The results were very positive (see our report of the Annual seminar, available online).
• As the Advisory Board was not yet constituted, the external evaluation we had during the first year is limited to the feedback received from the external stakeholders in particular those who were attending the first annual sem