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EUROPEAN REFERENCE NETWORK ON RARE HEMATOLOGICAL DISEASES [ERN-EuroBloodNet] [769064] - Operating Grant
Project abstract

ERN-EuroBloodNet main goal is to improve the healthcare and overall quality of life of patients with a rare hematological disease (RHD) by facilitating best practice sharing for safe and high-quality cross-border healthcare and developing more evidence based clinical tools and cost-effective treatments. As a contribution to the 3rd Health Programme (objective 4-Facilitate access to better and safer healthcare for EU citizens), EuroBloodNet will aim at decreasing current cross-border health barriers. EuroBloodNet gathers 66 highly skilled multidisciplinary healthcare teams in 15 Member States, and advanced specialised medical equipment and infrastructures which will facilitate concentration of resources for the design, validation and implementation of high-quality and cost-effective services aimed at facing the challenges of RHD. Involvement from the outset of patient associations will contribute to patient empowerment, in keeping with EuroBloodNet patient-centred approach. EuroBloodNet’s objectives will be achieved through a) the implementation of a reliable repository of best healthcare services available across EU, guidelines for RHD, systematic assessment of clinical outcome indicators, assessment and promotion of ongoing clinical trials and collaborative research initiatives, and b) the development of inter-professional consultation systems and blended (on-site & on-line) educational programmes and short stays. Expected outcomes include reduction of healthcare inequalities for RHD in the EU by a)establishing a cross-border referral system allowing safe information, samples and patient mobility, b) provision of equal access to highly specialised procedures and innovative therapies resulting from best practice sharing, continuous medical education and virtual interprofessional consultation for complex RHD cases, and c)facilitation of a timely and efficient translation of research results into patient oriented strategy at the clinical and the public health level

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

As in other Rare Diseases, expertise in Rare Hematological Diseases (RHD) is scarce and distributed heterogeneously across the EU. There is no accessible repository of human and technical resources available to challenge the difficulties of RHD at the EU or national levels. As a result, healthcare professionals working in the field (i.e. hematologists, pediatricians and /or primary care general practitioners) feel isolated when dealing with a patient affected by a RHD, especially those with ultra-rare prevalence. Deficiencies in knowledge and information regarding health services for RHD are responsible for an increase in the time of diagnosis and the number of patients misdiagnosed, as well as a lack of decision-making support for best treatment options. This situation leads to patients and relatives, but also health professionals’ anxiousness. Similarly, although many scientific societies, at the Member State or European level, have produced a number of best practice guidelines in RHD, there is no comprehensive repository of reliable guidelines accessible to health professionals. Compounded by the fact that curricula for hematology medical training differs from one Member State to another and, in general, little time is dedicated to the rarest RHD, the delivery of highly specialised quality care across Europe for these patients is difficult to achieve.
Management of complex cases may also require inter-professional consultation with of experts from a multidisciplinary team who may not be physically in the same institution or even Member State, while, on the other hand, the scarce number of patients suffering from a RHD and the unawareness of their location frequently prevent the activation of clinical trials or collaborative research projects due to the lack of critical number of patients to obtain robust evidence-based results. In this situation many patients and relatives affected by RHD, especially the ultra-rare ones, have contributed investigate their condition and in some cases acquired good knowledge about therapeutic innovations.
The annual programme for the first year of ERN-EuroBloodNet was structured in two main branches:
a) launching a dynamic European repository of EuroBloodNet members profiles (HCP and experts), diseases covered (codification and related documentation), existing guidelines for RHD, patient registries implemented at the regional, national or EU level, on-going clinical trials and collaborative research initiatives.
b) establishing an educational programme identifying areas requiring continuous medical education in order to start contributing to solving inequalities among Member States in the delivery of best care, including short stays;
This annual programme also addresses legal issues concerning the setting-up of a virtual space for inter-professional consultation of complex cases and define the profile of the board of experts.
These activities are in line with EU initiatives that contribute to innovative, efficient and sustainable health systems and facilitate access to better and safer healthcare for EU citizens. Common principles in all EU health systems must ensure clarity and confidence with regard to authorities setting and monitoring healthcare standards. In order to ensure that the goals of ERN-EuroBloodNet support these objectives, a specific framework for facilitating RHD cross-border healthcare and European cooperation on health services is being implemented through the definition of five specific objectives:
Objective1. Improve equal access to highly specialised healthcare delivery for RHD across Europe.
Mapping of services in each Member State allows ERN-EuroBloodNet to identify where the best patient management care is taking place. In Member States where services are absent, ERN-EuroBloodNet will function as a focal point to provide information about possible cross-border options. Additionally, mapping of services will contribute to the shaping of policies at the national

Methods and means

The coverage of Rare Hematological Diseases (RHD) in ERN-EuroBloodNet has been assured through their organization in two main thematic groups: non-oncological and oncological diseases that have been divided into 4 and 2 sub-thematic areas, respectively i.e. 1) Rare red blood cell defects 2) Bone marrow failure (BMF) and hematopoietic disorders 3) Rare Bleeding-Coagulation disorders and related diseases and 4) Haemochromatosis and hereditary iron metabolism disorders; 1) Myeloid malignancies and 2) Lymphoid malignancies.

ERN-EuroBloodNet is constituted by a consortium of 66 members from 15 MS, shaping one of the largest ERNs officially approved by the EC in March 2017. During first year of implementation, important efforts from the coordination team have been focused on setting up the structure that a complex network like ERN-EuroBloodNet requires in order to ensure that all the stakeholders involved become active actors of the network.

Also in this context, the implementation of the different channels of communication and dissemination have been fundamental during this period in order to ensure the transmission of information from the central structure of the network to the members and to the third parties dealing with rare hematological disorders. Some of the initiatives undertaken have been the implementation of the ERN-EuroBloodNet website and production of dissemination material specially devoted for the members to spread the word of ERN-EuroBloodNet, including powerpoint, poster, leaflets, flyers…

Based on common unmet needs to all RHDs identified during the 1st year of the network, activities carried out were focussed to map the existing expertise, services and facilities available for RHD across ERN-EuroBloodNet Members as starting point for network objectives deployment in the consecutive phases. In addition, ERN-EuroBloodNet has also contributed to the consolidation of the Clinical Patient Management System, the telemedicine platform for interprofessional consultation across ERNs provided by the EC for the safe sharing of clinical data.

Methods and tasks aiming to achieve ERN-EuroBloodNet specific objectives were split into five categories of Transversal Field of action (TFA): 1) Cross border health 2) Best practices 3) Continuing medical education 4) Telemedicine and 5) Clinical trials and research.
ERN-EuroBloodNet methods and means have been split into 5 Core WPs or Transversal Fields of Action (TFA) with specific tasks linked to objectives.

TFA1 Cross border health: Linked to objective 1, TFA on cross-border health aims to establish a referral system for patients and samples in order to ensure the same level of access to healthcare across Europe.
TFA2 Best practices: Linked to objective 2, TFA on best practices aims to compile, create, and assess their implementation, alongside disseminating guidelines in RHD
TFA3 Continuing medical education (CME): Linked to objective 3, TFA on CME aims to spread cutting-edge knowledge and facilitate continuous medical education in the field of RHDs
TFA4 Telemedicine: Linked to objective 4, TFA on Tele-medicine aims to facilitate inter-professional consultation by sharing of expertise and safety exchange of clinical information
TFA5 Clinical Trials and Research: Linked to objective 5, TFA on Clinical Trials and Research aims to foster European cooperation for epidemiological surveillance, development of highly specialized procedures for diagnosis, innovative treatments and research

Work performed during the reporting period

TFA 1 – CROSS BORDER HEALTH
As starting point for the further mapping of clinical care services available at EU level for RHD, an inventory of ERN-EuroBloodNet members, Healthcare Providers, Departments and Experts of the multidisciplinary team has been implemented through the following actions:
- ORPHA classification for RHD revision and implementation in ERN-EuroBloodNet website back office
The diseases included in the RHD classification were classified according to the subnetworks established by the network and was circulated among subnetwork coordinators for its review and implementation at the website back office.
- ERN-EuroBloodNet members profiles
The dynamic inventory of ERN-EuroBloodNet members gathers information on the RHD covered and scope, data on HCPs and departments, facilities and experts in the multidisciplinary teams. For this, three different profiles were defined: Members, departments and experts profiles editable through three online applications endorsed at the ERN-EuroBloodNet website.
The skeleton and relations among the different profiles within the inventory was built based on general information extracted from the network’s proposal and gathered from the members representatives and substitutes.
In parallel, members, departments and members representatives and substitutes profiles as well as experts’ profiles were also implemented in order to be later completed directly by the members representatives and substitutes through the online applications forms.

TFA 2 – BEST PRACTICES
- Design of the protocol for the creation of the repository of reliable guidelines on RHD
The protocol for the creation of the repository of reliable guidelines on RHD ranging from prevention, diagnostic tests and treatments to the organisation of patient-centred care in multidisciplinary teams and patient safety has been designed and implemented based in two complimentary approaches: a) Creation of a list of international guidelines and b) Questionnaire conducted among ERN-EuroBloodNet members

TFA 3 – CONTINUING MEDICAL EDUCATION
- Collaboration with educational bodies
A collaboration has been established among ERN-EuroBloodNet and the educational bodies: the European Hematology Association (EHA) and the European School of Hematology (ESH)
- Definition of approaches for the identification of educational gaps
Three different approaches for the identification of educational gaps have already been presented by EHA, based on 1) the implementation of an online survey, 2) inclusion of RHDs in existing ESH and EHA meetings and 3) classify online learning material at the EHA online Learning Center as RHD material.
-Co-organization with the ePAGs of European symposia with interactive patient participation.
ePAGs representatives have agreed with the EHA to collaborate in order to reinforce the patients’ advocacy sessions at the EHA annual congresses.
-Identification of areas including highly specialized procedures requiring short stays for the acquisition of expertise.
An analysis was undertaken in order to identify the areas in which the organization of short stays of health professionals might have major impact.

TFA 4 – TELEMEDICINE
- Legal analysis of the General Data Protection Regulation
The European Commission DG SANTÉ has provided ERNs with the Clinical Patient Management System (CPMS), a secure web-based application to support the networks in two core tasks: inter-professional consultation of complex cases (telemedicine) and keep de-identified information on clinical data (registry).
A legal analysis and impact of the General Data Protection Regulation on the development of European registries and telemedicine platforms and in special, in the CPMS, has been performed.
- Constitution of an expert board
A board including experts from specific RHD fields has been identified in order to be officially invited to join the CPMS.
- Participation in the CPMS pilot phase
The CPMS was open to ERNs by November 2017,

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

The major outputs of first year of ERN-EuroBloodNet are:

The creation of the European inventory of RHD experts and facilities available at the EU and Member State levels based on the implementation of members, healthcare providers, departments and experts profiles. ERN-EuroBloodNet profiles’ edition is available through a set of applications forms allocated in the private area, that have been designed integrating the ORPHA classification for RHD in its back office, allowing the selection of health professionals’ specific area of expertise as well as for the diseases covered by each of the departments directly from the classification and through a user-friendly disease- level format.

Members, departments and experts’ database and public profiles have been designed as dynamic tool allowing both, the edition of the network members directly by the experts and the coordination team members, and ensuring the possibility of expansion of the ERN through the inclusion of new members and affiliated partners in the future stages. Moreover, it is in the internal structure where relies the real complexity of the inventory, envisaged to interrelate all the items (experts with departments with HCPs and members with the ORPHA classification), providing huge possibilities for the disease or group of diseases target data analysis while boosting search engines by either of the above items. Currently, 181 experts’ profiles have been already created including 122 members’ representatives and substitutes and 59 invited experts. 96 of them have fulfilled completely their profiles by linking their expertise with the RHD tackled, age coverage and area of expertise. 220 departments profiles have been completed. The implementation of ERN-EuroBloodNet members’ profiles provides as a result the mapping of resources available through members, setting the basis for patient pathways implementation and for establishing a model for cross border referral system for patients and samples based on patient pathways implementation and in accordance with Directive 2011/24/EU implementation. This will lead to a better use of resources at the MS level while arising gaps on services provided in a given MS or even at the EU level for the clinical management of a specific condition.

The protocol for the creation of the repository of reliable guidelines on RHD has been designed allowing the identification of a wide variety of guidelines through two different approaches: a) Identification of the most common used guidelines at EU and international level for the most frequent RHD – List of international guidelines, and b) Identification of the guidelines used for the less prevalent RHD (including very rare diseases) and national guidelines – Online questionnaire to ERN-EuroBloodNet members. The list of international guidelines has been produced with the feedback from subnetworks coordinators, gathering 69 guidelines for the six subnetworks. The list will be implemented in the back office of the website in order to provide experts fulfilling the online questionnaire the possibility of selecting those guidelines implemented in their healthcare providers, while if different ones are followed, they will be able to include the information related to the guidelines implemented. In result, the online questionnaire will provide with the information on the guidelines implemented a) for the coverage of very rare diseases or b) at national level, complementing the list of international guidelines. The implementation of the protocol will lead to an accurate and reliable source of information on the guidelines followed by the ERN-EuroBloodNet members that will allow the identification of valuable gaps at each Member State and EU level to be addressed in the coming years. In turn, this will promote the delivery of highly specialised procedures and treatments and the harmonisation of care delivery across the EU.

Different strategies for the gathering of educational gaps

Achieved outcomes compared to the expected outcomes

In general terms ERN-EuroBloodNet has achieved the expected outcomes for the first year of implementation following the working plan defined at the beginning of the network with the exception of few tasks related to the implementation of online questionnaires targeted to each TFA, which have been partially implemented, and thus, expected outcomes have not been fully achieved.
After an analysis undertaken by the ERN-EuroBloodNet coordination team, a common need across all TFAs was identified regarding the need for the gathering of available services/activities/resources within the network. In order to address this need, a transversal methodology was defined by the coordination team including a set of online questionnaires to be conducted among ERN-EuroBloodNet members encompassing the different key items of each of the TFAs.
TFA targeted online questionnaires were agreed to be endorsed in ERN-EuroBloodNet members profiles in order to facilitate the gathering of answers from the members and their analysis. As a result, all the different gaps within the TFAs will be identified and planned to be addressed in the coming annual work plans.
In order to get the maximum number of answers from the experts on RHD, the launch of TFAs questionnaires was agreed to be performed once the inventory of ERN-EuroBloodNet members and experts was finalized. However, and given the slight delay suffered in the early implementation of the ERN-EuroBloodNet inventory of members, some of the questionnaires foreseen have not been able to be conducted during this period of action.
Nevertheless, online questionnaires have been already discussed during the Teleconferences among the TFA coordinators and coordination team, as well as during the Scientific and Strategic Board Meetings and accordingly, questionnaires agreed will be will be carried out during the 2nd year of the network for the fully achievement of all expected outcomes.

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

DISSEMINATION
Dissemination has been one of the ERN-EuroBloodNet key transversal activities, becoming a priority for the whole consortium in order to increase the outreach of the outcomes achieved.
ERN-EuroBloodNet’s dissemination includes all the activities aiming to expand knowledge of the ERN in RHDs, its website and its activities and services in order to get the necessary critical mass to make the network fully successful and ensure its long-term sustainability. Specific objectives for the first year of implementation of ERN-EuroBloodNet were focused to:
- Define and establish a dissemination plan including creation of the dissemination material (ie Leaflets, slide presentations…) and creation of a Dynamic Stakeholders Directory
- Develop and stimulate ERN-EuroBloodNet social media channels as the main powerful tools for dissemination, including ERN-EuroBloodNet website and social networks.
- Promote relations with third parties: other ERNs, European Research Infrastructures Consortiums.
Major results have been:
- ERN-EuroBloodNet website
ERN-EuroBloodNet website provides the skeleton of the complex structure that ERN-EuroBloodNet represents by endorsing specific dedicated sections to the all the activities and tools developed by the netoworks while ensuring interoperability with other platforms.
In this context, ERN-EuroBloodNet website has been conceived as the on-line platform that provides not only the door of access to ERN-EuroBloodNet dynamic and public inventory of rare hematological diseases (RHD) members and services as key step for cross border pathways implementation, but also to the main tools developed and implemented during the running time of the network.
Furthermore, the design and implementation of a robust and dynamic ERN-EuroBloodNet website tool is cornerstone for creating a critical mass of interests and to boost public awareness of the network goals and achievements and to increase the impact of its results among target groups. In this sense, ERN-EuroBloodNet website becomes the bridge for the establishment of permanent contacts between ERN-EuroBloodNet coordination team, scientific and strategic board and board of the network with the different target groups of the platform including health professionals, scientific bodies, patients, health authorities and industries. It means that http://www.eurobloodnet.eu is increasing its importance within the network every day.
ERN-EuroBloodNet website can be understood as two-side online platform with two main objectives: a) ERN-EuroBloodNet website as the main tool for dissemination of the goals and achievements to boost public awareness of the network and b) ERN-EuroBloodNet website as the infrastructure (web portal and database) for accessing the eHealth tools developed and as main source of information for participation in such platforms.
- Dissemination material
ERN-EuroBloodNet leaflets and flyers have been produced to contextualize a long-term effort that is consolidating an endurable network across Europe and spread the existence of the ERNs, and specially of ERN-EuroBloodNet to the worldmby describing the rationale, objectives and expected results and outcomes of ERN-EuroBloodNet.
In addition, a poster and powerpoint presentation have been also produced specifically addressed to ERN-EuroBloodNet members to present the facilitate the dissemination of the network to a wide range of public by summarizing governance of the network, transversal fields of action and subnetworks, objectives most relevant challenges and achievements.
- Promotion of relations with third parties
ERN-EuroBloodNet during the first year has established relations with the key third parties related to: a) Transversal activities to all ERNs (EC, ERNs coordinators group, RD-Connect…), b) Rare Hematological Diseases (Orphanet…) and c) patients (EURORDIS, patients organizations…).

EVALUATION
Activities implemented during the first year of ERN-EuroBloodNet have been di