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European Reference Network (ERN) on Rare Multisystemic Vascular Diseases (VASCERN), SGA Proposal 2017 [VASCERN] [769036] - Operating Grant
A- Sharing of experience: discussion of difficult cases by lead APHP

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VASCERN offers the opportunity for sharing experience between experts - one of the main added
values of the ERN. Sharing experience at European level will be made possible using:
- Web-based platforms...
VASCERN offers the opportunity for sharing experience between experts - one of the main added
values of the ERN. Sharing experience at European level will be made possible using:
- Web-based platforms and teleconferencing during the monthly RDWG teleconference: difficult
clinical cases will be discussed. A summary of the case, discussion and conclusions will be made
available on the website and on the intranet, as decided by Chairs.
- Through e-mail via a secured mailing list including all the HCPs of the RDWG, specific question
will be discussed. Similarly, a brief summary will made available on the website or on intranet, as
decided by the RDWG Chairs.

B- Definition of patients pathways by lead APHP

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It will be discussed with the
respective HCPs of each WG and completed during the first 6 months of VASCERN (priority). It will
enable derivation of quality of care measures and outcome measures. It w...
It will be discussed with the
respective HCPs of each WG and completed during the first 6 months of VASCERN (priority). It will
enable derivation of quality of care measures and outcome measures. It will then be diffused as an
educational tool, and to help new centers (in others countries).
This measure is necessary before choosing relevant and consensual outcomes in the different diseases
of the network. It is essential to be able to respond to the EU expectations from the ERN.
This measure is specific, measurable (number of pathways), acceptable (definition of mode of care for
the rare diseases is the responsibility of VASCERN), realistic (as each HCP has already more or less
clearly defined such a pathway) and time bound (this measure is going to be implemented rapidly after
VASCERN approval, because it is the necessary step to derive the first outcome measures specific for
a disease).
C- Cross-border pathways Mobile Application by lead APHP

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share information about the HCP from VASCERN and broader network characteristics (services
offered, how to reach each HCP), an application for smart-phone IOS & Android has been developed
by a Rare Di...
share information about the HCP from VASCERN and broader network characteristics (services
offered, how to reach each HCP), an application for smart-phone IOS & Android has been developed
by a Rare Disease Center in Lombardy. It is free of charge and VASCERN will adapt this program to
be implemented on wider scale and in a larger set of languages so to cover the whole ERN and further
national networks and HCP partners. It will allow patients throughout Europe to be able to choose the
relevant HCP according to the rare disease they face (or MD to accurately choose for their patients).
This needs funding for software development (application already available, so it will only needs
further development/updates), and update of data is critical for its reliability. This will be the
responsibility of the ERN Project Team.
D – Pills of knowledge by lead APHP

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Professional upgrade may result difficult for clinicians, in terms of time available. We plan to make it
easier by the development of single video-lessons (each 7-12 minutes of duration) in which an e...
Professional upgrade may result difficult for clinicians, in terms of time available. We plan to make it
easier by the development of single video-lessons (each 7-12 minutes of duration) in which an expert
talks about a single brief topic. Evaluation is included, a downloadable certificate of attendance will be
available and accreditation at term. It is expected to mix specialists from different Centers/Countries
and create specific modules to cover every aspect of every disease related to VASCERN, as well every
tone of voice (i.e. levels of training/learning, from simplest to most difficult). These “pills” of rare
disease knowledge will be available on a mobile-friendly platform and so one does not need a PC, a
desk and can have a learning-moment virtually everywhere.
Actually, this concept will also be used without video by Cross European training of Professionals
initially in HHT. It will be developed through a web based online course (MOOC, or University
based), based on recent experience (2015-2016) setting up all new modules for a new MSc in
Genomic Medicine. Modules will be part face-to-face, part online, with individual items available as
Short Courses or standalone lectures for lesser credit. It may be feasible to expand to a full MSc as the
Late Pr. Pierre Lasjaunias set up through ESNR.
This action will begin during the first year of VASCERN for the MOOC without video (HHT WG
starting) and during the second year of VASCERN with the videos. The possibilities for adaptation
and improvements are multiple
E- Registries by lead APHP

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Registries are ongoing in numerous HCPs: local, national and international registries. The use of
European registries faces specific problems, both technical and legal. The Registry WG will be
respons...
Registries are ongoing in numerous HCPs: local, national and international registries. The use of
European registries faces specific problems, both technical and legal. The Registry WG will be
responsible for these legal and technical issues (gathering the information from the different
countries). A member of each country is expected to participate in this WG to indicate the specificity
faced nationally. The registry WG is also expected to make the inventory of all the existing registries
within the network.
A single VASCERN registry for all diseases covered by the network should be built, with the capacity
of evaluation of activity and quality control (first outcome measures, to be implemented rapidly). This
registry will progressively be implemented to analyse all the development in the patient’s disease
(follow-up and analysis). A common minimum dataset conceivable for all RD will initially be used in
accordance with the ERN IT platform and the JRC EU RD registration platform criteria currently
under development. Links with the EU infrastructures, once set up, will be implemented. The RDWGs
will have the task to determine the disease specific datasets to be implemented in this registry. A
reflexion is already taking part in the framework of the 2016 Call for RD Registry for ERNs. Links
with biobank data will be also studied. A reliable unique identifier must be adopted by VASCERN.
F - Clinical trials by lead APHP

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Clinical trials are the only way to demonstrate the benefit of a new treatment, mode of follow-up and
diagnostic strategy. VASCERN has therefore the ambition to promote the design and help in the
real...
Clinical trials are the only way to demonstrate the benefit of a new treatment, mode of follow-up and
diagnostic strategy. VASCERN has therefore the ambition to promote the design and help in the
realisation of international clinical trials in as many diseases included in VASCERN as possible.
Therefore, VASCERN HCP Members will have to make every effort to participate in clinical trials
that have been validated by the Board. All the clinical trials running through VASCERN will be listed
on the website, with contact information, so that additional HCPs outside VASCERN can participate
as appropriate (including cooperating and affiliated HCPs not yet included as full members). Some
clinical trials are already ongoing in VASCERN. Information on publication, research and clinical
trials will be regularly updated in the website.
VASCERN plans include the implementation of a newsletter for calls for research collaboration or
clinical trials in order to promote clinical trials. Monthly meeting of RDWG (including patient
representative co-chair) will discuss new protocols, research projects and clinical trials within the area
of the RDWG. Patient representatives will also have the role to promote clinical trials among the
patient community.VASCERN also plan to include in its Annual Report a report on VASCERN
Research Projects and Clinical Trials as requested in the ERN call, as well as publications report as
appendix.
G- Availability of conferences on YouTube by lead APHP

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A specific VASCERN YouTube Channel will be prepared in order to share scientific meetings held by
HCPs of the ERN or congress events or single traditional lessons for professionals. An important part
...
A specific VASCERN YouTube Channel will be prepared in order to share scientific meetings held by
HCPs of the ERN or congress events or single traditional lessons for professionals. An important part
of the work will be to collect the congress events and the traditional lessons to be able to record them,
or collect the recording if this is already organized.
This measure is specific, measurable (number of videos available), acceptable (a public diffusion of
conferences is expected), realistic (VASCERN experts are giving a lot of conferences, often recorded)
and time bound (this is a continuous process, with ongoing collection of the conferences).
I - Definition of clinical outcomes by lead APHP

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A first set of simple outcome measures will rapidly be followed in VASCERN. A specific disease will
be chosen for each RDWG. This set will include simple parameters such as number of patient
screened,...
A first set of simple outcome measures will rapidly be followed in VASCERN. A specific disease will
be chosen for each RDWG. This set will include simple parameters such as number of patient
screened, number of patient with a given diagnosis (clinical and molecular), number of patient with a
follow-up, number of patient undergoing surgery, number of patient recruited in a research study).
This should be obtained rapidly (M12). A second set of outcomes will be derived after patient
pathways have been implemented, and will require meetings for discussion.
J.1- Development of guidelines for optimal care of the rare disease by lead APHP

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This process implies many steps and requires organizational skills, numerous interactions between
HCPs and patient representatives, meetings between experts: 1) Collect the guidelines, consensus
state...
This process implies many steps and requires organizational skills, numerous interactions between
HCPs and patient representatives, meetings between experts: 1) Collect the guidelines, consensus
statements, and expert position papers on the disease of interest: 3 months M1-M3; 2) Define the gap
in knowledge for each disease (questions not included, guidelines not updated): M3-M4; 3) Define the
area for the new guidelines to be developed (M4-M5): face to face meeting 1. Who writes what?; 4)
Centralization of the manuscript by a specific person (HCP representative or another HCP member)
who will be responsible for the guidelines (M5-M7); 5) Circulating the first draft among members of
the disease WG and patients WG (M7-M9); 6) Making of a second draft (M9-M11); 7) Face to face
meeting 2 for comments (M11); 8) Making a third draft (M11-M12); 9) Face to face meeting 3 if
necessary (M12); 10) Last draft (M12-M13); 11) Circulating last draft (M13-M14); 12) Agreement on
a text (M14); 13) Circulation of the text among others expert groups throughout the world (USA
included) M14-M15; 14) M15: agreement (or not) on a common text on the disease studied, which
will be published to appropriate journal.
The whole process should take around 1 year, including 2 face to face meetings between experts and
circulation of many drafts, after definition of the scope of the guidelines which depends on the
available literature. This work has to be done for each disease (e.g. Kippel Trenaunay), and sometimes
independently for different aspect of a disease (e.g. treatment of HHT). Caution will be taken to not duplicate work of scientific society and to work in concert with them. This is timely bound as roughly one new document should be issued every year for each WG:
J.2- Development of guidelines for optimal care of these patients facing common problems not related to their rare diseases: Do and Don’t” factsheets in common situations by lead APHP

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Patients with rare diseases face frequent situations including medical situations such as abdominal
surgery for any reason, dentist intervention, pregnancy. The question then arises of how to deal wit...
Patients with rare diseases face frequent situations including medical situations such as abdominal
surgery for any reason, dentist intervention, pregnancy. The question then arises of how to deal with
frequent diseases in rare patients. The aim of this project is to propose some simple guides available to
the medical community, which would indicate the particularities of care due to the fact that the patient
is affected by a rare disease included in VASCERN. This has been done in France and the idea is to
propose a similar service at a European level. The process should include 1) choosing the relevant
situation 2) writing the simple factsheets 3) diffusion for comments 4) edition in a booklet (to be
shown during meetings and disseminated through patient association), translation in several EU
languagespublication on the website.
L- Communication by lead APHP

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Dissemination and awareness-raising are of major importance to share information related to health: among healthcare workers (clinicians, students, and researchers), patients, caregivers, and all targ...
Dissemination and awareness-raising are of major importance to share information related to health: among healthcare workers (clinicians, students, and researchers), patients, caregivers, and all target groups identified. e-Health in communication includes:
A website, mobile-friendly (L.1). A website has already been setup. It will be improved, specific pages
/ deliverables will be translated in different languages of EU (education material for patients) (L.2)
Internal and external communication will be set up allowing forum for professionals (reserved area).
This will be used for the clinical case discussion (see above) and set up as soon as possible, depending
of the ERN IT platform proposed.
Forum for patients and caregivers (L.3). This will be set up after 1 year,
Newsletter and periodical minutes/deliverables/mid-term (L.4). A VASCERN monthly newsletter has
already been issued, and a monthly newsletter is foreseen. Communication on news, events as well as
relevant call for proposals (H2020, etc.) on the website to support circulation of knowledge,
awareness-raising as well as to support European collaborative research projects, within and outside
the Network. It will also include calls for research/collaborations/patient cohorts (A specific newsletter
will be issued when necessary); calls for instant pool, surveys among health operators or among
patients (A specific newsletter will be issued when necessary).
Social network (L.5): Facebook/Twitter VASCERN pages (community management, Q&A for
patients, promotion VASCERN and health messages (social networks) as part of health promotion
campaigns…), promotion of VASCERN towards the general public and stakeholders, including public
entities as well as private companies so to enhance the Network action.
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