Rare and Complex Urology

Rare and Complex Urology combines information on clinical aspects, network developments and patient journeys in specific rare and complex urological diseases within the ERN (European Reference Network) template structure, allowing for better diagnosis, treatment and patient outcome. Each chapter contains an introduction on specific diseases with representative clinical images followed by a clinical view on treatment options (non-surgical and surgical) with clear schema and drawings; nurse specialist views on current care, patient views on the lifelong journey and expected development and research innovations for the future. Diseases covered include those of the bladder, stone and kidney diseases, malformations, pelvic floor disorders, the female urethra, reconstruction, surgery, cancers and rare tumors. This is a must have reference for basic scientists interested in engaging in translational urology and surgery and well as urologists seeking guidance on how to implement novel research.

• Provides a foundation for many rare urogenital diseases and complex conditions with contributions from high-volume centres monitored by ERN eUROGEN
• Combines in-depth research findings with clinical information to supply state-of-the-art care solutions for rare and complex aspects of urology
• Removes the uncertainty of managing rare and complex urogenital cases and variations in practice

• Language: English
• Publisher: Academic Press
• Release date: Feb 12, 2024
• ISBN: 9780323999359

Book preview

Chapter 1 Introduction: Rare urology, ERN eUROGEN and knowledge exchange

Wouter F.J. Feitz    Division of Paediatric Urology, Department of Urology, Radboudumc Amalia Children's Hospital, Nijmegen, The Netherlands

Abstract

Rare Urology presents the latest developments of ERN eUROGEN, the European Reference Network for rare urogenital diseases and complex conditions, with the shared views of the multiple experts currently actively involved in providing these new knowledge exchanges for you.

Keywords

Urology; Rare urology; Paediatric urology; Functional urology; Urooncology; Rare disease; Complex condition; Healthcare provider; European Reference Network; Patients; Multidisciplinary team; Quality of life; European Commission

Dear Reader,

Rare Urology involves patients with rare uro-recto-genital anomalies, rare and complex functional urogenital conditions, and rare and complex oncological urology.

It involves the lifelong care of our patients with rare diseases or complex conditions and knowledge exchange between all stakeholders.

This means especially the patients and their caretakers, experts in the field, all members of a specific multidisciplinary team, fellows, medical and nursing students, researchers in rare diseases, healthcare providers (HCPs) and their connected education and research institutes. It involves innovations in healthcare at national, European and international levels, patient organisations, scientific societies and new collaborations for healthcare innovation and much-needed improvements in the quality of life for patients living with rare diseases or complex conditions.

The central theme ‘Share. Care. Cure.’ (when possible) was established for the large European initiative of the European Reference Networks (ERNs) for patients with rare diseases or complex conditions. This involves around 30 million European patients and an estimated 300 million globally. Of these patients, approximately 10% are directly connected to experts in the field of rare urology. The ERN programme is an innovation in European healthcare, initiated, supported and sustained by the European Commission through the EU4Health Programme (2023–2027).

Rare Urology presents the latest developments of ERN eUROGEN, the European Reference Network for rare urogenital diseases and complex conditions, with the shared views of the multiple experts currently actively involved in providing these new knowledge exchanges for you.

All involved in this process wish you an interesting and stimulating time reading this newly provided information. If you want to be more involved in the care of and innovations for your patients with rare urological diseases or complex conditions, additional knowledge can be found in the different sources related to Rare Urology, the 24 ERNs, and the knowledge exchange channels of ERN eUROGEN.

Chapter 2 The European Association of Urology (EAU) and ERN eUROGEN

Christopher R. Chapple    Department of Urology, Sheffield Teaching Hospitals NHS Foundation Trust, Sheffield, United Kingdom

Abstract

It is evident that the expansion of scientific knowledge and the variety of surgical and medical techniques available have made it difficult for individual clinicians to stay abreast of all the latest advances in knowledge. This rapid increase in information poses a challenge to the most effective management of rare and complex conditions in any speciality, which by their nature are not encountered commonly by any individual in their clinical practice. Consequently, subspecialisation has proven essential in order to provide the most effective clinical outcomes for patients. In fact, many suggest that rather than subspecialisation, one should talk about supraspecialisation, over and above the basic skill set.

Keywords

ERNs; MDT; Supraspecialisation; Urology

It is evident that the expansion of scientific knowledge and the variety of surgical and medical techniques available have made it difficult for individual clinicians to stay abreast of all the latest advances in knowledge. This rapid increase in information poses a challenge to the most effective management of rare and complex conditions in any speciality, which by their nature are not encountered commonly by any individual in their clinical practice. Consequently, subspecialisation has proven essential in order to provide the most effective clinical outcomes for patients. In fact, many suggest that rather than subspecialisation, one should talk about supraspecialisation, over and above the basic skill set. In line with the aphorisms: ‘There is no such thing as brave surgeons, just brave patients’ and ‘A jack of all trades and master of none’, Hippocrates said: ‘Whenever the art of medicine is loved, there is also a love of humanity’, and Osler said: ‘The good physician treats the disease; the great physician treats the patient who has the disease’. In this context, to effectively treat a patient with a rare clinical disorder, we need to pool expertise and call upon the advice of colleagues skilled in managing a particular disorder, using the principle of the multidisciplinary team approach, thereby utilising their understanding of the latest knowledge on clinical developments and the appropriate investigations and treatment (whether medical and/or surgical skills) to treat patients with rare diseases and complex conditions most effectively.

It is clear that with the uncommon clinical scenarios presented by rare diseases and complex conditions, pooling experience will lead to more rapid advances in knowledge and hence to improved clinical outcomes for individual patients, which only this approach can effectively achieve. The logic of the European Reference Networks (ERNs) is apparent; if a clinician only sees one patient every year or two, despite their expertise and undoubted technical ability, it is very unlikely that they will have the adequate knowledge and personal experience of dealing with a rare condition. In particular, paediatric urology remains an area within urology where the practising clinician must deal with many diverse and rare conditions, many of which are congenital disorders where the patient will often need lifelong care by an experienced extended multidisciplinary team. However, diverse rare clinical scenarios are also seen in adult urology. ‘Medicine is the science of uncertainty and an art of probability’. This probability can only be achieved in rare diseases and complex conditions through sharing knowledge via ERNs such as ERN eUROGEN.

In 2016, whilst I was the Secretary General (2015–2023) of the European Association of Urology (EAU), I became aware of the European Commission’s plans to develop ERNs across all medical disciplines, and I was convinced this was a crucial initiative for urology. With Professor Wout Feitz at Radboudumc in the Netherlands and some active patient representatives, we co-developed the original ERN eUROGEN proposal, which was submitted to the European Commission. Initially, this brought together 29 key expert healthcare provider hospitals across Europe. This resulted in the acceptance and launch in March 2017 of the ERN for rare urogenital diseases and complex conditions, ERN eUROGEN—one of 24 ERNs covering different medical fields.

ERN eUROGEN is a clinical reference network that provides a secure Internet-based clinical platform, which allows the discussion of rare and complex cases. This allows the confidential sharing of clinical information on individual patients and creates a forum to bring together all current knowledge and experts in any particular supraspecialist field within urology, thereby allowing detailed discussion of that case. Whilst the fundamental premise of the ERN networks was to empower clinicians by cross-referral to provide the best expert management of rare congenital disorders, we were fortunate that the EC accepted our proposal to expand the remit of ERN eUROGEN to become a comprehensive reference network dealing with rare conditions (both benign and malignant in nature), in patients of all ages, thereby providing expert advice throughout the lifetime of the patients affected.

ERN eUROGEN comprises three workstreams, namely,

•Rare congenital uro-recto-genital anomalies (Workstream 1),

•Rare functional urogenital conditions requiring highly specialised surgery (Workstream 2) and

•Rare urogenital tumours (Workstream 3).

I was privileged to be ERN eUROGEN’s first coordinator. I remained in this role until 2019, when I was delighted to pass on the coordination role of the network to the skilled and effective leadership of Prof. Wout Feitz at Radboudumc, Nijmegen (NL). Under his leadership, ERN eUROGEN has expanded to encompass more specialist centres across all EU/EEA member states (currently 56 healthcare providers and their specialist teams).

ERN eUROGEN has several work packages covering its ongoing activities and developments:

(1)Coordination, including governance, the three workstreams and the expertise areas (EAs) contained within these, and collaboration with scientific, academic, and other organisations or bodies across Europe and the world,

(2)Dissemination, including the communication and sharing of knowledge and outcomes,

(3)Evaluation, including monitoring to maintain the effectiveness of the network,

(4)Healthcare and the Clinical Patient Management System (CPMS), including facilitating virtual multidisciplinary team (MDT) consultations advise on the diagnosis and treatment of patients,

(5)Registries, data management, and analysis,

(6)Training and education, including webinars, courses, exchange programmes and this book,

(7)Clinical Practice Guidelines (CPGs) and Clinical Decision Support Tools (CDSTs),

(8)Capacity building, best practice sharing, and potentially other activities for Ukraine.

The European Association of Urology (EAU) is committed to the field of urology at an educational and scientific level and, in this context, works closely with ERN eUROGEN as an official supporting partner as the two organisations are complementary at many different levels, as evidenced by the EAU’s mission statement:

•To act as the representative body for European urologists and facilitate the continued development of urology and all its supraspecialties.

•To foster the highest standards of urological care throughout Europe.

•To encourage urological research and enable the broadcasting of its results.

•To promote contributions to the medical and scientific literature by its members.

•To promote European urological achievements worldwide.

•To establish European standards for training and urological practice.

•To contribute to the determination of European urological healthcare policies.

•To disseminate high-quality urological information to patients and public.

In this context, from the very beginning, I promoted the strategic alignment between the two organisations as the objectives of both were perfectly complementary. The EAU includes several activities.

•Four journals amongst the highest ranked in the field of urology (European Urology, European Urology Oncology, European Urology Focus and European Urology Open Science);

•The education office (the European School of Urology) provides a highly comprehensive and wide-ranging international programme of education across Europe and beyond;

•The section offices comprehensively cover all aspects of specialised urological practice;

•The guidelines office provides the most up-to-date and widely used evidence-based approach to the management of urological conditions, which we believe will dovetail very effectively with the ERN eUROGEN guidelines as they become available and the close collaboration between the two organisations will ensure there is no duplication of effort;

•The annual EAU congress, the largest international urological congress in Europe, at which ERN eUROGEN has a prominent role.

It is a great pleasure to see the enormous dedication and selfless, often voluntary, contributions made by so many colleagues to support ERN eUROGEN. I am deeply honoured to have been invited to introduce this very comprehensive overview of the latest developments in the field of rare urogenital diseases and complex conditions. Long may this initiative and its enormous contribution to the effective management of rare and complex urological conditions continue!

Part II

Overview chapters

Chapter 3 ERN eUROGEN: Impact and innovations for rare and complex urology

Wouter F.J. Feitza; Loes Oomena; Darren Shilhanb; Michelle Battyeb; Jen Tidmanb    a Division of Paediatric Urology, Department of Urology, Radboudumc Amalia Children's Hospital, Nijmegen, The Netherlands

b ERN eUROGEN, Department of Urology, Radboud University Medical Center, Nijmegen, The Netherlands

Abstract

ERN eUROGEN is the European Reference Network (ERN) for rare urogenital diseases and complex conditions. It carries out activities to improve the healthcare management of patients throughout Europe by facilitating knowledge generation and sharing, exchanging of best practices concerning diagnosis, establishing new ways to reduce unfavourable outcomes for patients, improving and innovating the quality of care provided by expert healthcare professionals and their teams, and subsequently diminishing the cumulative cost for society. Its work packages cover coordination, dissemination and collaboration, monitoring and evaluation, virtual multidisciplinary team meetings, a patient registry, training and education, and guidelines. ERN eUROGEN welcomes any enquiries from healthcare professionals regarding rare urogenital diseases or complex conditions.

Keywords

European reference network; ERN eUROGEN; European Union; European Commission; European Patient Advocacy Group; Board of member states; Urology; Rare diseases; Rare urogenital diseases; Complex functional urology; Surgery; Highly specialised surgery; Paediatric surgery; Paediatric urology

Introduction

Rare or low-prevalence complex diseases in Europe

Half a million people in Europe are diagnosed with a rare disease every year, and rare or low-prevalence complex diseases affect the daily lives of around 30 million European Union (EU) citizens. Health systems in the EU aim to provide high-quality, cost-effective care, but this is particularly challenging for rare or low-prevalence complex diseases and conditions. Clinicians face challenges in diagnosing and treating patients due to limited knowledge, information and exposure to uncommon symptoms and disease mechanisms, meaning many patients have faced a diagnostic odyssey when searching for answers.

Highly specialised care and surgery for rare conditions should occur in centres of excellence with highly trained and experienced surgeons using multidisciplinary teams (MDTs) [1]. This centralised approach is cost-effective and provides the best possible outcomes, improving survival and quality of life.

European reference networks

European Reference Networks (ERNs)a are virtual networks connecting healthcare providers (HCPs), professionals and patients across the EU and Norway (Fig. 1). They aim to facilitate discussion on rare diseases and complex conditions requiring highly specialised treatment by pooling knowledge and resources and thereby improving diagnoses and treatment for patientsb with a ‘Share. Care. Cure.’ approach.c Using technology and telemedicine, the networks join up the information and expertise scattered across the EU, ensuring that information travels to the patient.

Fig. 1

Fig. 1 The relationship between European reference networks, national healthcare providers and patients.

On 9 March 2017, 24 ERNs covering a variety of medical disciplinesd and involving more than 900 highly specialised healthcare units from over 300 hospitals were formally approved, accredited and launched by the European Commission (EC) and the Board of Member States for ERNs (BoMS).e The BoMS involves (mainly government) representatives from the health ministries of all EU member states and specific experts who oversee the governance of the whole ERN system at the European level.

As required by the legislation, in 2018, HCPs in countries where an ERN has no full member were appointed as affiliated partners to work with the ERNs to expand the geographical coverage of the ERNs to involve all EU member states.f These HCPs are usually located in member states with smaller populations that benefit the most from the knowledge transfer from interacting with the high-volume HCPs and expert clinicians in the networks.

The UK withdrawal from the European Union took effect on 31 January 2020, with the transition period ending on 31 December 2020. This meant that UK HCPs ceased to be part of the ERNs.

In January 2022, following a 2019 EC call for proposals for new members to join existing ERNs,g the BoMS approved 620 new HCP members assessed by the EC, ERNs and Independent Assessment Body,h meaning the ERNs now include more than 1400 healthcare units from around 400 hospitals across all 27 EU member states and Norway.

The ERNs are not a time-limited project; instead, they are an EU initiative. They create a new infrastructure with interactions on a scale never attempted before to improve care for patients with rare diseases and/or complex conditions. They are based on the 2011 Directive on Patients’ Rights in Cross-border Healthcare, were built on a robust policy and legal framework,i and benefit from the previous work done by EU-funded projects, programmes and networks over the decade before their launch.

Full financial support for the ERNs has been agreed upon between the member states, the European Parliament and the EC, with all ERN grant management, funding, support actions and innovations currently taken care of by the Health and Digital Executive Agency (HaDEA). Current funding is through the EU4Health Programme, which will be followed by the EC’s next health programme that, according to the EU vision on ERN Developments (ERN2030), will include funding calls for the 24 ERNs. In the future, collaboration with the medical industry may be another source of funding for the ERNS, and policy developments relating to this are under discussion by the EC, ERN Coordinators Group and the BoMS.

While there is still much work to do, the ‘birth’ of 24 ERNs has been a landmark moment in the history of European voluntary cooperation in the field of health with the potential to change millions of lives. They have already improved the healthcare management of patients with rare diseases and complex conditions throughout Europe by facilitating knowledge generation and sharing, care coordination and exchanging of best practices concerning diagnosis, establishing new ways to reduce unfavourable outcomes for patients, improving and innovating the quality of care provided by expert healthcare professionals and their teams and subsequently diminishing the cumulative cost for society.

The future success of ERNs will be stimulated by creating synergies with other European funding programmes: a Joint Action on the integration of the ERNs into national healthcare systems (JARDIN), which is in progress and will be enhanced by a Joint Action Towards the European Health Data Spacej,k. A Joint Programme is also underway to align member states’ research strategies through the European Rare Diseases Research Alliance (ERDERA) l (following on from the European Joint Programme on Rare Diseasesm). In the meantime, the ERNs and member states will continue to work to raise awareness of the networks among patients/caregivers, health professionals, hospitals, managers and national agencies.

ERN eUROGEN: The European reference network for rare urogenital diseases and complex conditions

ERN eUROGEN and rare urogenital diseases and complex conditions

ERN eUROGEN is the ERN for rare urogenital diseases and complex conditions affecting the reproductive organs or the urinary system.

Millions of people suffer from urogenital conditions annually, but only a small fraction suffer from urogenital conditions that are rare. Generally, the EU considers a condition rare when it affects fewer than 1 in 2000 people (<0.05%).n However, in the case of some highly complex urogenital conditions, the prevalence rate is significantly lower than the EU definition; for example, urethral diverticulum, a localised outpouching of the urethra into the anterior vaginal wall, has an estimated annual incidence of 17.9 per 1,000,000 (0.02%) per year [2].

Urogenital conditions significantly impact paediatric, adolescent and adult patients, their families and their quality of life. The little available knowledge is often scattered among a small number of expert specialists and unique centres located in disparate countries and regions, which impedes diagnosis, satisfactory advice and treatment and encumbers healthcare systems and society with a significant cumulative economic burden.

This is why ERN eUROGEN’s activities are essential to advance innovation in medicine, address gaps and collect evidence at the European level, improve diagnostics, treatment and outcomes and ultimately create more equitable access for as many patients as possible efficiently and cost-effectively.

Full and up-to-date information about ERN eUROGEN and its recent developments can be found on its website: www.eurogen-ern.eu.

Target groups

The following target groups all benefit from ERN eUROGEN’s activities:

•Patients living with a rare disease or complex condition and their families

•European patient advisory group (ePAG) advocates

•Associate/supporting patient partners

•Nonpartner patient, family and advisory organisations

•Healthcare providers (HCPs)

•Full members and affiliated partners

•Nonmember HCPs

•Public health authorities and policymakers

•EU institutions

•(Sub)national authorities

•Regulatory agencies

•Researchers in the scientific and health community related to rare diseases and/or complex conditions

•Scientific, academic and professional societies

•Supporting partners

•Nonpartner scientific, academic and professional societies

•Other ERNs

•New member states, European countries outside the EU and non-EU countries

•Industry: pharmaceutical and medical device organisations

Patients

For the patient view on the ERN programme, see Chapter 4 of this book.

Patients are at the centre of ERNs, and this has been the EC’s goal from the outset—a fundamental principle is that they and their representatives should actively participate in the advisory bodies of the networks as key partners. Patients, parents and families often endure difficult times while seeking a final diagnosis and prognosis for their diseases. By being involved in a pan-European effort, these individuals are empowered and able to make a difference.

ERN eUROGEN has worked closely with EURORDIS (a unique, nonprofit alliance of over 1000 rare disease patient organisations from 74 countries that work together to improve the lives of over 300 million people living with rare diseases globally) to increase engagement with urogenital patient organisations and establish the network’s European Patient Advocacy Group (ePAG).o The ePAG comprises associate patient partners, also known as ePAG advocates, who are patients from European patient organisations representing those affected by rare diseases or complex conditions under ERN eUROGEN’s scope. When associate requirements are not met, patients can also be involved in ERN eUROGEN as supporting patient partners. Finding patient organisations and representatives in this medical area can be difficult, so new applications from patients to work with ERN eUROGEN are warmly welcomed.

ERN eUROGEN’s ePAG has two seats on the network’s strategic board. The ePAG is included in all activities, for example, giving input on disease coding, setting the research agenda, completing patient surveys in the registry, advising on training and educational activities and being trained on how to be involved in developing ERN clinical practice guidelines and clinical decision support tools so they can be involved in their production. They are encouraged to join initiatives from the network’s supporting partners so that they can make connections and links.

Members

At the time of writing in 2023, ERN eUROGEN involves 56 healthcare providers (HCPs) comprising 51 full members and 5 affiliated partners, including associated national centres (ANCs) and national coordination hubs (HUBs), in 20 EU member states (Fig. 2). Each year, these HCPs care for around 12,500 patients diagnosed under the rare urological disease codes included within the network (see Table 1).

Fig. 2

Fig. 2 A map showing the geographical spread of ERN eUROGEN’s full member and affiliated partner healthcare providers: Full members =  blue /National Coordination Hubs =  red /Associated National Centre =  green .

Table 1

These full members and affiliated partners use a hub-and-spoke model at the national level, referring rare or complex cases from a primary/secondary local centre to a tertiary centre, broadening coverage and reaching more HCPs and patients depending on national healthcare systems and policies.

New memberships of ERN eUROGEN are not dependent on the network but on calls for applications and designations by the BoMS and national ministries of health. Further expansion of geographical coverage is expected when the BoMS appoints additional affiliated partners.

ERN eUROGEN has a noncompliance and termination procedure for HCPs, which may start if they do not maintain relevant expertise in any workstreams and expertise areas, do not contribute to or participate in ERN activities, or face ethical, legal, privacy, conflict of interest or transparency issues. The coordination team will ask the strategic board to vote on termination if no resolution can be reached. Loss of membership will follow EC regulations.

Workstreams

The network covers the following three workstreams (WS) based on age, functional disorder or malignancy:

Rare congenital uro-recto-genital anomalies (WS1): These diseases and complex conditions require specific diagnoses and surgical correction, often in both an acute and staged approach during the neonatal period or childhood. High expertise in techniques and clinical problems is necessary because failed initial surgery and potential secondary surgery are costly and damaging. These patients require lifelong care provided by multidisciplinary teams (MDTs) of experts who plan and perform surgery and provide postoperative physiotherapy and psychological support during follow-up to improve their quality of life. There has previously been a gap between paediatric treatment and adult outcomes, which ERN eUROGEN addresses through a crossover between workstreams and transfer from WS1 to WS2. Improving transitional care increases positive outcomes and the self-management of rare diseases, ensuring continuity of care throughout the lifespan.

Functional urogenital conditions requiring highly specialised surgery (WS2): As discussed earlier, optimal outcomes are reached if a surgeon has considerable experience in techniques and clinical problems. Follow-up data are available in very few areas, and more is needed. Therefore, ERN eUROGEN prioritises and facilitates data collection through the ERN eUROGEN registry.

Rare urogenital cancers (WS3): A key part of EU health strategy is Europe’s Beating Cancer Plan, which aims to improve the entire cancer pathway, including prevention, treatment and care, and quality of life for cancer patients and survivors.p The importance of a centralised approach and the increased reach to patients with penile, testicular, adrenal and soft tissue cancers have already been evidenced by the literature generated within the network [3]. By promoting and conducting highly specialised surgical training, organ preservation rates for penile cancer may increase 10% within participating HCPs over 5 years. Increasing training and surgical expertise will also improve testis cancer mortality rates in Eastern and Central Europe.

Expertise areas

Each WS is further subdivided into 19 different expertise areas (EAs) covering over 60 distinct ORPHAcodes (both group and disorder level) and supported by ICD-10 codes for areas covering complex surgery where ORPHAcodes cannot be applied (Table 1). Each expertise area is covered by a clinical chapter within this book (see Chapters 8–26).

ERN eUROGEN prefers to refer to EAs rather than disease areas (respecting the EU’s principles of equality, diversity and inclusivenessq) because the network provides expertise relating to complex cases involving differences in sex development (DSDs) (EA 1.1) and surgery for transgender patients (EA 2.7). These areas involve the identities of individuals and are not diseases or disorders, so it would be disrespectful to the patients involved to refer to their requirements as such.

ERN eUROGEN’s full member and affiliated partner HCPs must show evidence that they cover particular EAs and that they meet the network-specific criteria related to these when they apply to join the network to ensure they have the required standards of expertise and resources to treat patients appropriately and effectively. The network has a protocol for filling in gaps and reviewing existing criteria using data from annual monitoring exercises and input from network experts and the ePAG.

Because the expertise of an HCP can change due to new staff appointments or new endorsements of expertise by national health ministries, HCPs can apply to expand their expertise. ERNs can also apply to extend their EAs and include additional rare diseases and complex conditions. The BoMS must approve these applications. ERN eUROGEN is currently awaiting a decision about expanding its EA coverage to include male infertility, paediatric renal transplantation in urology, paediatric oncological urology and rare bladder tumour surgery.

Work packages

From October 2023 to October 2027 (using a direct operational grant covering the remainder of the EU4Health Programme), ERN eUROGEN will organise its activities using nine work packages (WPs), which will be expanded upon in the rest of this chapter:

•Coordination (WP1)

•Dissemination (WP2)

•Evaluation (WP3)

•Healthcare and CPMS (WP4)

•Registries, data management and analysis (WP5) (see also Chapter 6)

•Training and education (WP6)

•Clinical practice guidelines and clinical decision support tools (WP7) (see also Chapter 7)

•Capacity building and best practice sharing/other activities for Ukraine (WP8 and WP9) (see also Chapter 5)

Coordination

Objectives

•To progress ERN development, cohesion and engagement through ERN eUROGEN’s coordination, management and operational activities.

•To progress coordination, management, collaboration and support activities for the long-term sustainability of the ERN system and better integration of the ERNs into national systems.

Management and operational activities

Since its launch in 2017, ERN eUROGEN has fostered a culture of openness and trust among clinicians and patients by establishing a comprehensive and robust governance and organisational structure with vertical and horizontal aspects, which provides top-down and bottom-up guidance on strategy and activities (see Fig. 3).

Fig. 3Fig. 3

Fig. 3 ERN eUROGEN’s organisational structure (2023).

ERN eUROGEN’s original full members signed a consortium agreement, and original affiliated partners signed a bilateral cooperation agreement. The network’s governance statutes were reviewed and updated in 2022 to ensure they were up-to-date for the new full members joining from January 2022. They were sent to all full members, affiliated partners, supporting partners, associate/supporting patient partners, and the ePAG for signature and agreement. The new governance statutes were discussed at the network’s annual strategic board meeting in September 2022, and no changes were requested.

The governance statutes outline clear tasks and actions for full members and affiliated partners; changes in HCP representatives, the HCP network coordinator and/or the HCP network coordinator representative; a process for the noncompliance and termination of HCPs; data privacy; personal conflicts of interest; requirements for supporting partnership for organisations and individual experts; memorandums of understanding for global collaboration and rules of engagement for patients.

HCP network coordinator

Radboud University Medical Center (Radboudumc) is a very large HCP in the Netherlands that acts as the HCP network coordinator, meaning it is the sole beneficiary of EC funds provided to support the network. Radboudumc is very familiar with running large EC research proposals and has extensive in-house resources, including experienced legal and ethics departments, a privacy office, an EU grants team, financial support teams, a clinical trials office, data entry support, data stewards and data protection support. Radboudumc employees (both clinical and nonclinical, across multiple departments in the HCP) support the HCP network coordinator representative and the coordination team. This streamlined process and the significant contributions of the healthcare professionals working there facilitate the management of ERN eUROGEN.

Coordination team

The HCP network coordinator representative is an individual healthcare professional with experience in the EAs covered by ERN eUROGEN and a proven ability to coordinate and lead a network. They have overall responsibility for managing the network’s participants, overseeing all aspects of strategic plans, work packages, objectives and activities relating to the network’s work packages. They chair the strategic board, manage the coordination team, assign work, ensure objectives are met and delivered on time and within budget and identify and resolve issues and risks.

The coordination team comprises individuals with expertise in project management, business support management, data performance analysis, IT support and clinical knowledge of the EAs covered by ERN eUROGEN, who support the HCP network coordinator representative with all aspects of network coordination relating to the work packages. The team meets weekly to make sure work is kept on track.

Strategic board

In accordance with EC legal regulations and the network’s governance statutes, the strategic board comprises the HCP network coordinator representative, representatives of the full member and affiliated partners, and the ePAG chair.

It is the main governing body of the network responsible for making major decisions related to changes to the HCP network coordinator and HCP network coordinator representative, accepting/terminating board members and network members, approving EC grant proposals, creating additional workstreams and/or expertise areas, approving amendments to the governance statutes and dissolving the ePAG, the strategic board and/or the network if required.

The strategic board is also responsible for adopting annual and multiannual specific objectives, strategic plans, work packages and network activities, monitoring and approving all activities, outcomes and initiatives in accordance with the monitoring and assessment system for the ERNs, empowering patient organisations, setting up ad hoc working groups as required, authorising requests for grants from third-party entities and adopting relevant network policies.

The strategic board meets at least once annually for a full meeting and may have other ad hoc partial meetings. Each member and affiliated partner HCP has a single vote that their representative or proxy can cast. Two votes are cast by the ePAG chair, or their proxy, for the whole ePAG. In the case of a tie, the HCP network coordinator representative casts the deciding vote. Supporting partners, associate/supporting patient partners and ePAG advocates are invited to attend strategic board meetings with an informative/advisory role but have no voting