Table Of Content European Rare Kidney Diseases Reference Network... 2 Summary... 3 Work Package... 9 Network Management... 9 Promote cross-border expert healthcare... 9 Harmonize specialized care... 9 Review and advance patient management... 9 Propagate specialized knowledge... 9 Advance research... 9 Coordinator, Leader contact and partners... 11 Outputs... 12 Newsletter... 12 2 international educational events on focused rare kidney topics... 12 Performance & outcome indicators... 12 Annual Research Report... 12 Network Website... 12 Page 1/12
European Rare Kidney Diseases Reference Network JA2015 - GPSD [705038] START DATE: 01/03/2017 END DATE: 28/02/2018 DURATION: 12 month(s) CURRENT STATUS: Finalised PROGRAMME TITLE: 3rd Health Programme (2014-2020) PROGRAMME PRIORITY: - CALL: EUROPEAN REFERENCE NETWORKS SPECIFIC GRANT AGREEMENTS COVERING YEAR 2017 TOPIC: ERN Specific Grant Agreements Year 1 EC CONTRIBUTION: 199645 EUR KEYWORDS: Cakut, Ciliopathies, Cystinosis, Dialysis, Econsult, Genetic Diagnostics, Glomerulopathies, Guidelines, Hus, Hyperoxaluria, Outcome Measures, Rare Kidney Diseases, Transplantation., Tubulopathies PORTFOLIO: Rare diseases Page 2/12
SUMMARY Project abstract The European Reference Network for Rare Kidney Diseases (ERKNet) will offer highly specalised healthcare to children and adults suffering from congenital and acquired glomerulopathies, tubulopathies, metabolic and stone forming nephropathies, thrombotic microangiopathies, renal ciliopathies and other anomalies of kidney and urinary tract development. The Network comprises almost 40 centres in 12 European countries with specific expertise documented by large patient numbers (collectively more than 45,000), state-of-art diagnostic services and therapeutic equipment and professional multidisciplinary care. The Members will share best practices by offering physical and virtual cross border consultation, series of educational workshops and webinars, e-learning modules on focused rare kidney disease topics, short-term training opportunities, and by endorsing existing and developing new clinical practice guidelines and recommendations wherever needed. In collaboration with the patient organisations, the Network will collect informational materials on all rare kidney diseases and their treatments and make the documents available in multiple languages to affected families throughout Europe. Furthermore, ERKNet will strive to harmonize and further optimize patient care across the Member centres by regularly monitoring and benchmarking guideline adherence, disease-specific performance and outcomes measures, as well as patient safety and satisfaction. Another important goal of the Network will be to actively support clinical research into risk factors and biomarkers of early and severe disease phenotypes, and provide a platform for innovative interventional clinical trials aimed at improving or stabilizing kidney functions and preventing progressive renal failure in adults and children with rare kidney diseases. Summary of context, overal objectives,strategic, relevance and contribution of the action ERKNet is a Consortium of 38 adult and pediatric nephrology centers from 12 EU countries which offer highly specialized clinical services to patients suffering from rare and complex inherited or acquired kidney diseases. Furthermore, the centers are key providers of training and education and perform high-end translational, clinical and epidemiological research in the field of rare kidney disease. The overarching mission of the Network is to promote optimal healthcare for all European patients suffering from rare kidney diseases. In concordance with the delegated and implementing decision for European Reference Networks, ERKNet pursues the following objectives: to establish molecular diagnoses for all patients early in the course of kidney disease, allowing rational and cost-effective therapy with minimized adverse effects and optimized quality of life. to uniformly provide top quality healthcare according to international Page 3/12
standards and guidelines, using evidence based approaches whenever available. To systematically assess patient outcomes by developing and monitoring quality and safety benchmarks To provide expertise and support to clinicians throughout Europe who need help with challenging patients with a rare kidney disease, using novel digital health technologies to facilitate the mobility of expertise. To reinforce epidemiological surveillance and clinical research by promoting joint patient registries throughout the Network. To advance the development of new therapies for rare kidney disorders With these activities our programme is clearly committed to contribute to the 4th major objective of the 3rd Health Programme, i.e. to facilitate access to better and safer healthcare for Union citizens. ERKNet brings together the largest, most experienced and best renowned centers for rare kidney diseases in Europe, who are willing to harmonize their patient management from diagnostic procedures to therapies and long-term rehabilitation. The use of uniform clinical practice guidelines and recommendations, common training activities for healthcare professionals, the implementation of an EUwide econsulting service for cases from both within and outside the Network, the public benchmarking of management performance and outcomes, the creation of a common thesaurus of patient information documents on rare kidney diseases and their treatment, and the use of a common tool to measure patient satisfaction will transform the management of these disorders in the EU. The Network s activities help setting new standards of care, not only in the Member centers but throughout the EU as a growing number of centers is expected to start following the algorithms and workflows recommended by the Network. Finally, taking advantage of its mixed composition of paediatric and adult nephrology services, the Network will develop strategies for a structured transition of patients with rare kidney diseases and/or renal replacement therapy from pediatric to adult care, a long-standing challenge in the longterm management of these life-long diseases. Methods and means In Year 1 of the project, the initial activities subserving the implementation of the work plan included the creation of the Network s executive structures, i.e. the formation of the Network Secretariat, Workgroups and Task Forces. Next, the required communication and information infrastructures were established (telephone, web and video conferencing platforms, list of contact information of all active member representatives) and the channels for external communication were set up (Website, social media accounts). The Workgroups are primarily responsible for the structure, process and data reviewing Page 4/12
activities in pursuit of the objectives scheduled for year 1. They are guided in their work by the topic specific Task Forces. The Secretariat, Workgroups and Task Forces are operating under constant supervision by the Executive Board. Work performed during the reportingperiod The secretariat and communication infrastructures were established within the first two months of the first project year. In parallel, the Network website was set up and went live in May 2017. The kick-off meeting of the Network was held in May 2017 in Heidelberg. The meeting was attended by 115 participants including representatives of each member center as well as patient representatives. During this meeting the executive board was formed and met for the first time. Furthermore the workgroups and task forces were initiated and their work programmes were defined. On occasion of the annual meeting of the European Society for Pediatric Nephrology (ESPN) in Glasgow in September 2017 meeting of the pediatric participants of all workgroups were organized to discuss face-to-face the progress of the guideline review and evaluation, the online research database and the setup of the basic patient registry with the plan to integrate specific clinical outcome indicators for each disease group. In November 2017 the Network s adult nephrologist experts met on occasion of the American Society of Nephrology annual meeting in San Diego. The meeting was attended by 35 ERKNet members, who discussed the status of the various activities of the Network. ERKNet has initiated a close cooperation with OrphaNet and supports this organisation by identifying missing and modifying outdated rare kidney disease codes. To that end two coding workshops were organized, one covering the rare glomerular diseases and the other one renal malformative disorders. With the help of the local ERKNet partners and patient advocacy groups in the different countries covered by ERKNet, a repository of rare kidney disease information documents was compiled and the documents and online links were published on the network website. An educational program comprising CME courses and webinars on rare kidney disease topics was started and guided by the task force for educations and training. In collaboration with the European Commission the CPMS (clinical patient management system) was tested and evaluated. A team of experts was set up who will start online consultations in the second year of the program. Page 5/12
The main output achieved so far and their potential impact and use by target group (including benefits) The secretariat and communication infrastructures are fully functional. The Executive Board, workgroups and taskforces communicate via regular telephone conferences and face-to-face meetings as required. Information is quickly disseminated across the Network via the Newsletters and social media. All ERKNet members have access to mailing lists and the telephone conference system. Via the ERKNet website (www.erknet.org) patients and families have the possibility to identify expert centers specialized in the disease of their interest across Europe. Also, external experts find helpful guidance for their patients and can easily contact an ERKNet reference center for consultation. In addition to the public pages with general information about a large number of rare kidney diseases, the Network s structure and expertise of the reference centers, there is a password-protected domain where ERKNet members can correspond with each other and post relevant materials. Furthermore, anyone signing up for the ERKNet newsletter will be informed about upcoming events such as webinars, conferences and other activities. The repository for patient information materials, online on the website (public) domain helping patients and families to be informed and find the nearest expert for their specific disease. To date the repository contains more than 70 documents in German, French, Portuguese, Italian, Dutch, Spanish and English. The website also includes an online research database listing all on-going clinical and translational research projects carried out in the ERKNet centers. In addition, the workgroup members recent scientific work can be viewed in continuously updated online publication lists. During the kick-off meeting in May 2017, a project plan for systematic screening of existing guidelines was developed by the task force for Guidelines and Pathways, which was adopted by the thematic workgroups. To date, 169 guidelines were screened by the workgroup chairs and 81 of these were evaluated by two reviewers each To ensure a valid evaluation process the AGREE (Appraisal and Guidelines for Research and Evaluation) platform was used, an instrument that assesses the methodological rigour and transparency of clinical guidelines which has undergone validity and reliability testing. In case of a great inconsistency in the evaluation scores a third reviewer was consulted. To date, more than 20 guidance documents were endorsed following positive evaluation and the key recommendations of the endorsed documents have been posted on the ERKNet website. The endorsed guidelines were used to draft the list of the clinical outcome indicators, which will be used in project year 2 for inclusion as monitoring and benchmarking indicators in the ERKNet Core Registry. During two workshops for disease coding in November in Leuven and December in Heidelberg an interdisciplinary team of clinicians, pathologists and geneticists worked on the formation of missing Orphanet codes in rare Page 6/12
kidney diseases. Associated with each new code was the formulation of disease descriptions for integration in the correct disease families. The workshop results are currently being included in the Orphanet catalogue. Two international CME courses were co-organized with ERKNet and held in Madrid and Leuven in June and December 2017, each attended by 80-100 participants. Furthermore, the Taskforce for Education and Training started a bi-weekly webinar series in February 2018. While the Webinars are held by ERKNet experts, invitations to join the webinars are extended to all members of the European professional societies for pediatric and adult nephrology. So far, the Webinars had excellent attendance by both ERKNet members and members of the wider nephrology community, with approximately 100 registrations per webinar. The active participation in the Q&A sessions and very positive feedback from the online event evaluations confirm that the pediatric and adults renal community is Achieved outcomes compared to the expected outcomes All milestones and deliverables planned for the first year were achieved, some with a few weeks of delay. The list of performance and outcome indicators is still in preliminary form, awaiting final consentation by the work groups. The delivery of a regular webinar series, foreseen for year 2, was already started in the last month of year 1. Dissemination and evaluation activitiescarried out so far and their major results The Network s activities are disseminated via multiple channels: The Website is a comprehensive, frequently updated source of information on rare kidney diseases, the Network structure and the services offered by the expert centers. In the last month of year 1 the Website was used in 800 sessions by 600 (thereof 400 new) users. The most frequently visited sites are the ones with information about rare diseases, the expert centers, and the downloadable Webinar presentations. Online newsletters reporting the Network s most recent activities are produced and disseminated on a regular basis. The Newsletters are currently received by 330 active subscribers including healthcare professionals within and outside ERKNet, the patient community, policymakers and other stakeholders. The Network is also active in the social media, including Twitter and Facebook. On average, 30 original tweets per month are currently sent to more than 200 followers. The tweets cover news related to the Network, social events and Page 7/12
activities as well as new publications and research findings in the rare kidney disease domain. Page 8/12
Work package Work Package 1: Network Management Start month: 1 End month: 12 Work Package Leader: UKL-HD Hire staff for secretariat Set up telephone, video- and web conferencing systems Set up member and partner address directory Appoint Workgroup chairs Appoint Task Force members Establish Network website Develop a plan for social media outreach Provide regular newsletters on ERKNet activities using both email and website posting Review and provide opinion on new membership applications to BoMS Define Roles of Affiliated and Collaborative Partners and develop Collaboration Agreements Work Package 2: Promote cross-border expert healthcare Start month: 4 End month: 12 Work Package Leader: UKL-HD Identify disease experts set up for international consulting Provide referral contact information on website Inform Affiliated Partners and public about international referral system Develop ERN IT infrastructure Work Package 3: Harmonize specialized care Start month: 7 End month: 12 Work Package Leader: UKL-HD Review existing clinical practice recommendations and guidelines Endorse existing recommendations and guidelines for use throughout Network Page 9/12
Work Package 4: Review and advance patient management Start month: 5 End month: 12 Work Package Leader: UKL-HD Review existing registries and databases for currently monitored performance, safety and outcome indicators Develop common list of performance, safety and outcome indicators Review locally used patient satisfaction survey tools Work Package 5: Propagate specialized knowledge Start month: 5 End month: 12 Work Package Leader: UKL-HD Organize regular CME courses on rare disease topics Develop online repository of rare kidney disease information documents Identify gaps and produce information documents for uncovered rare kidney diseases Translate documents into all ERKNet Member state languages Work Package 6: Advance research Start month: 8 End month: 12 Work Package Leader: UKL-HD Review present research activities throughout the Network Develop online rare kidney disease patient registry Page 10/12
COORDINATOR, LEADER CONTACT AND PARTNERS COORDINATOR UNIVERSITAETSKLINIKUM HEIDELBERG (UKL-HD) IM NEUENHEIMER FELD 672 69120 HEIDELBERG Germany Project leader contact Name: SCHAEFER Franz Email: franz.schaefer@med.uni-heidelberg.de Phone: PARTNERS No partners related to the current project Page 11/12
Powered by TCPDF (www.tcpdf.org) OUTPUTS Newsletter UKL-HD European Rare Kidney Diseases Reference Network (ERKNet) Published on: 03/04/2018 1st Network Newsletter edited and circulated 2 international educational events on focused rare kidney topics UKL-HD European Rare Kidney Diseases Reference Network (ERKNet) Published on: 12/04/2018 First two CME courses held The content of both CME courses will be documented and will be available afterwards. Planned CME courses are an Update on rare Kidney Disease and Multidisciplinary Management of Inherited Kidney Diseases. Performance & outcome indicators UKL-HD European Rare Kidney Diseases Reference Network (ERKNet) Published on: 10/04/2018 List of performance and outcome indicators List of performance and outcome indicators consented by Workgroups Annual Research Report UKL-HD European Rare Kidney Diseases Reference Network (ERKNet) Published on: 10/04/2018 First Annual Research Report composed and submitted Network Website UKL-HD European Rare Kidney Diseases Reference Network (ERKNet) Published on: 03/04/2018 Website fully functional and online Page 12/12