References
- Hilton K, Anderson A. IHI Psychology of Change Framework to Advance and Sustain Improvement. IHI White Paper. Boston, Massachusetts: Institute for Healthcare Improvement; 2018. Available from https://www.ihi.org/resources/white-papers (accessed May 2024).
- Atun R. Health systems, systems thinking and innovation. Health Policy Plan 2012; 27 Suppl 4: iv4–iv8. doi: 10.1093/heapol/czs088.
- Bok A, Noone D, Skouw-Rasmussen N; EHC Think Tank. Key challenges for patient registries – A report from the 1st workshop of the EHC Think Tank Workstream on Registries. J Haem Pract 2022; 9 (1): 14–19. doi: 10.2478/jhp-2022-0002.
- Bok A, Noone D, Skouw-Rasmussen N; EHC Think Tank. Key challenges for hub and spoke models of care – A report from the 1st workshop of the EHC Think Tank on Hub and Spoke Treatment Models. J Haem Pract 2022; 9 (1): 20–26. doi: 10.2478/jhp-2022-0003.
- Bok A, Noone D, Skouw-Rasmussen N; EHC Think Tank. Patient agency: key questions and challenges – A report from the 1st workshop of the EHC Think Tank Workstream on Patient Agency. J Haem Pract 2022; 9 (1): 27–35. doi: 10.2478/jhp-2022-0004.
- Skouw-Rasmussen N, Savini L; EHC Think Tank. Access equity: key questions and challenges – A report from the 1st workshop of the European Haemophilia Consortium (EHC) Think Tank Workstream on Access Equity. J Haem Pract 2023; 10(1) 56–61 doi: 10.2478/jhp-2023-0011.
- Skouw-Rasmussen N, Savini L; EHC Think Tank. Future Care Pathways – A report from the 1st workshop of the EHC Think Tank Workstream on Future Care Pathways. J Haem Pract 2023; 10(1) 74–81. doi: 10.2478/jhp-2023-0013.
- Bok A, Noone D, Skouw-Rasmussen N, on behalf of the EHC Think Tank. 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. J Haem Pract 2023; 10(1). doi: 10.2478/jhp-2023-0001.
- Gacser Z, Skouw-Rasmussen N, Bourke S, ten Ham R, Hosszú D; EHC Think Tank. Short- and longer-term goals for change – A report from the 2nd workshops of the EHC Think Tank Workstreams on Access Equity and Future Care Pathways. J Haem Pract 2023; 10(1) 155–163. doi: 10.2478/jhp-2023-0023.
- Gacser Z, Skouw-Rasmussen N, on behalf of the EHC Think Tank. System change in practice: A report from the EHC Think Tank workstreams on Registries and Patient Agency. J Haem Pract 2024; 11(1) 1-10. doi: 10.2478/jhp-2024-0005.
- EHC Think Tank. Access Equity | System map of the underlying challenges. Available from https://ehcthinktank.eu/workstream/workstream-four-access-equity/ (accessed May 2024).
- EHC Think Tank. Future Care Pathways | System map of the underlying challenges. Available from https://ehcthinktank.eu/workstream/workstream-five-future-care-pathways/ (accessed May 2024).
- LUCID. What is the lotus blossom technique? Available from https://www.lucidmeetings.com/glossary/lotus-blossom-technique#:~:text=The%20Lotus%20Blossom%20Technique%20is,the%208%20boxes%20surrounding%20it (accessed May 2024).
- Teisberg E, Wallace S, O’Hara S. Defining and implementing value-based health care: a strategic framework. Acad Med 2020; 95(5): 682–685. doi: 10.1097/ACM.0000000000003122.
- McQueen RB, Mendola ND, Jakab I, et al. Framework for patient experience value elements in rare disease: a case study demonstrating the applicability of combined qualitative and quantitative methods. PharmacoEconomics Open 2023; 7: 217–228. doi: 10.1007/s41669-022-00376-w.
- De Santis M, Hervás C, Weinman A, Bosi G, Bottarelli V. Patient empowerment of people living with rare diseases: its contribution to sustainable and resilient healthcare systems. Ann Ist Super Sanita 2019; 55(3): 283–291. doi: 10.4415/ANN_19_03_15.
- Chhetri D, Zacarias F. Advocacy for evidence-based policy-making in public health: experiences and the way forward. J Health Management 2021; 23(1): 85–94. doi: 10.1177/0972063421994948.
- Kalkman S, van Delden J, Banerjee A, et al. Patients’ and public views and attitudes towards the sharing of health data for research: a narrative review of the empirical evidence. J Med Ethics 2022; 48: 3–13. doi: 10.1136/medethics-2019-105651.
- Koster F, Kok MR, Barreto DL, Weel-Koenders AEAM. Capturing patient value in an economic evaluation. Arthritis Care Res 2024; 76(2): 191–199. doi: 10.1002/acr.25229.
- EURORDIS-Rare Diseases Europe. Process on Corporate Social Responsibility in the Field of Pharmaceuticals Platform on Access to Medicines in Europe Working Group on Mechanism of Coordinated Access to Orphan Medicinal Products (MoCA-OMP). Ref. Ares(2014)3857202 - 19/11/2014. Available from http://download2.eurordis.org.s3.amazonaws.com/moca/history/WG%20MoCA-OMP%20Transparent%20Value%20Framework.pdf (accessed May 2024).
- Andreoletti M, Haller L, Vayena E, Blassime A. Mapping the ethical landscape of digital biomarkers: a scoping review. PLOS Digit Health 2024; 3(5): e0000519.
- Bergier H, Duron L, Sordet C, et al. Digital health, big data and smart technologies for the care of patients with systemic autoimmune diseases: Where do we stand? Autoimmun Rev 2021; 20(8): 102864. doi: 10.1016/j.autrev.2021.102864.
- Mikk KA, Sleeper HA, Topol EJ. The pathway to patient data ownership and better health. JAMA 2017; 318(15): 1433–1434. doi: 10.1001/jama.2017.12145.
- Heijsters F, Santema J, Mullender M, et al. Stakeholders barriers and facilitators for the implementation of a personalised digital care pathway: a qualitative study. BMJ Open 2022; 12: e065778. doi: 10.1146/bmjopen-2022-065778.
- Solebo AL, Hysi P, Horvat-Gitsels LA, Rahi JS. Data saves lives: optimising routinely collected clinical data for rare disease research. Orphanet J Rare Dis 2023; 18: 285. doi: 10.1186/s13023-023-02912-1.
- Jacob C, Bourke S, Heuss S. From testers to cocreators – the value of and approaches to successful patient engagement in the development of eHealth solutions: qualitative expert interview study. JMIR Hum Factors 2022; 9(4): e41481. doi: 10.2196/41481.
- Brasier N, Sempionatto JR, Bourke S, et al. Towards on-skin analysis of sweat for managing disorders of substance abuse. Nat Biomed Eng 2024. doi: 10.1038/s41551-024-01187-6.
- Jobin A, Ienca M, Vayena E. The global landscape of AI ethics guidelines. Nat Mach Intell 2019; 1: 389–399. doi: 10.1038/s42256-019-0088-2.
- O’Mahony B, Dolan G, Nugent D, Goodman C; International Haemophilia Access Strategy Council. Patient-centred value framework for haemophilia. Haemophilia 2018; 24(6): 873–879. doi: 10.1111/hae.13456.
- Fantini B, Vaccaro CM. Value based healthcare for rare diseases: efficiency, efficacy, equity. Ann Ist Super Sanità 2019; 55(3): 251–257. doi: 10.4415/ANN_19_03_10.
- Schlieter H, Marsch LA, Whitehouse D, et al. Scale-up of digital innovations in health care: expert commentary on enablers and barriers. J Med Internet Res 2022; 24(3): e24582. doi: 10.2196/24582.
- European Commission. Commission welcomes European Parliament’s adoption of the European Health Data Space and regulation on substances of human origin. 24 April 2024. Available from https://ec.europa.eu/commission/presscorner/detail/en/IP_24_2250 (accessed May 2024).
- European Haemophilia Consortium. Think Tank. Available from https://ehcthinktank.eu (accessed May 2024).
- Chatham House. Chatham House rule. Available from https://www.chathamhouse.org/about-us/chatham-house-rule (accessed May 2024).