System change in practice: A report from the EHC Think Tank workstreams on Registries and Patient Agency

Abstract

Abstract Introduction System change addresses the fundamental causes of persistent, complex problems to achieve large-scale, sustainable solutions at multiple levels. Recognising the need for system change to ensure equitable access to healthcare for people with bleeding disorders and other rare diseases, the European Haemophilia Consortium (EHC) established a Think Tank to work alongside its traditional advocacy initiatives. The Think Tank has mobilised a broad range of healthcare stakeholders to identify challenges and co-create potential solutions through a series of thematic workstreams exploring specific aspects of the healthcare system. This paper reports on outcomes and learnings from the Registries and Patient Agency workstreams. Methodology/Process During a series of online meetings and face-to-face discussions, workstream stakeholders contributed to a three-phase process: 1. Discovery; 2. Strategy; 3. Innovation. Having identified key challenges to system change for Registries and Patient Agency, stakeholders mapped the system in which they were working to refine the challenges, recognise enablers and constraints to progress, and use leverage points to co-create strategies for change. Results The Registries workstream prioritised actions to address challenges around lack of common purpose and data quality, and agreed to move forward with developing a consensus statement to facilitate buy-in from key stakeholders, working on good governance for registries, hosting a network for registry owners, and creating a model for patient data input and feedback. A Registries Roadmap was completed for 2025 and 2030, and a project initiated to align registries in Europe. The Patient Agency workstream agreed actions should focus on challenges related to the role of the patient, recognising the need to elevate patient influence in all aspects of the healthcare system. Actions aimed to address the current stakeholder hierarchy and gaps in patient health literacy, and to optimise the potential of digital tools to enable patient contributions to patient-reported outcome and experience measures (PROMs and PREMs). Projects include developing a patient agency guidebook and a patient experience data (PED) dossier on von Willebrand disease, to provide a one-stop repository for regulators, researchers, clinicians and patients. Conclusions There is a clear need for system change to ensure equitable access to healthcare for people with rare diseases such as bleeding disorders. Bringing together multiple stakeholders with different and complementary knowledge and approaches has facilitated the development of innovative strategies for system change in relation to Registries and Patient Agency. Work has started on pilot projects to move these strategies forward.