Short- and longer-term goals for change – A report from the 2nd workshops of the EHC Think Tank Workstreams on Registries, the Hub and Spoke Model and Patient Agency

Abstract 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. Guiding Stars 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. Near Stars 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.