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At the second series of workshops for the EHC Think Tank Workstreams on Registries, Hub and Spoke Model and Patient Agency, stakeholder participants worked towards consensus on addressing challenges to progress in areas identified in the first series of workshops. Each workshop identified a 'guiding star' determining the direction of ongoing focus, defined achievable 'near star' milestones, and explored the enablers and 'constraints' to achieving these. The Registries Workstream recommended establishing rights- and responsibility-based international guidance to ensure accountability from all stakeholders contributing, collecting, handling and registry data. The Hub and Spoke Model Workstream proposed the development of a dynamic and agile health provision system to meet individual treatment, care and quality of life goals for people with rare disorders as they evolve. The Patient Agency Workstream recommended achieving a new cultural norm for patient agency embedded at all systemic levels, whereby health care is collaborative and based on patients' ability to make choices and take ownership of decisions relating to their care and quality of life. Four common themes emerged in near star milestones across all workstreams: 1. Mapping the system; 2. Collaborating and sharing; 3. Education and training; 4. Organisational change and good governance. Enablers include stakeholder experience in other specialties undergoing similar change; relevant examples of best practice; tapping into existing policy-making networks; adherence to government, regulatory, and inter-/intra-institutional quality standards; leveraging frustration in current systems to challenge mindsets and demonstrate the benefit of patient-centred insights to improve outcomes; and application of technologies (e.g. distributed analytics, algorithms, telemedicine, remote monitoring). Constraints include limited understanding of national and cross-border legal and regulatory requirements; a lack of awareness of and reluctance to accept the need for change or to take responsibility for making it happen, or a misunderstanding of whose responsibility it is; time limitations; a lack of meaningful outcome measures; a lack of understanding of key factors for success; and financial issues. [ABSTRACT FROM AUTHOR]

Introduction: Despite increasing awareness of issues faced by women and girls with inherited BDs (WGBD), standards of care are lacking, with disparities in diagnosis and treatment for WGBD across Europe. We aimed to develop practical principles of care (PoC) to promote standardization of care for WGBD within European Haemophilia Treatment and Comprehensive Care Centres (HTC/CCCs). Methods: The co‐creation process, supported by the European Association for Haemophilia and Allied Disorders, consisted of four multidisciplinary meetings with health care providers (HCPs) experienced in WGBD care, and European Haemophilia Consortium representatives, combined with broad patient and HCP consultations in the European haemophilia community. Relevant medical societies outside Europe were contacted for confirmation. Results: We developed ten PoC for WGBD, stressing the importance and benefits of a centralized, multidisciplinary, comprehensive, family‐centred approach to support and manage WGBD during all life stages. These PoC emphasise the right to equitable access and quality of care for all people with BDs, irrespective of gender. Multiple medical societies outside Europe also confirmed their support for endorsement. Conclusions: Ten PoC for WGBD evolved from an iterative process among stakeholders, supported by relevant medical societies worldwide. These PoC can serve as a benchmark for diagnosis and comprehensive multidisciplinary management of WGBD, and improve awareness of their unique challenges. They offer a framework to guide HTC/CCCs in providing equitable care for all WGBD, both in their own services and in other healthcare settings. Implementation of these principles aims to positively impact the health, wellbeing and quality of life for WGBD. [ABSTRACT FROM AUTHOR]

Introduction: The impact of bleeding for women with bleeding disorders (WBD) is of increasing focus and importance. Despite this, optimal management strategies are unclear and knowledge gaps persist. Aim: To examine practices and define research priorities on diagnosis and management of WBD in Europe. Methods: An electronic survey on clinical management of WBD was sent to 136 European haemophilia treatment centres (HTCs), including open questions on knowledge gaps and research priorities. Results: Fifty‐nine HTCs from 12 Western (WE) and 13 Central/Eastern European (CEE) countries completed the survey. Less than half runs a joint clinic (24 HTCs, 42%). Most centres without a joint clinic have a named obstetrician (81%) and/or gynaecologist (75%) available for collaboration. Overall 18/54 (33%) European HTCs do not offer preimplantation genetic diagnosis. Third trimester amniocentesis to guide obstetric management is available 28/54 HTCs (52%), less frequent in CEE compared to WE countries (5/17 vs 23/37, P =.03). 53% of HTCs (28/53) reported that only 0%‐25% of WBD seek medical advice for heavy menstrual bleeding (HMB). An algorithm managing acute HMB in WBD is lacking in 22/53 (42%) HTCs. The main reported knowledge and research gaps are lack of awareness & education on WBD among patients and caregivers, optimal diagnostic strategies and effective multidisciplinary management of pregnancy & HMB. Conclusion: Joint clinics, prenatal diagnostics and algorithms for managing acute HMB are lacking in many European HTCs. HMB may be an underestimated issue. This survey highlights the need to prioritize improvement of knowledge and patient care for WBD across Europe. [ABSTRACT FROM AUTHOR]