Access equity: key questions and challenges - A report from the 1st workshop of the European Haemophilia Consortium (EHC) Think Tank Workstream on Access Equity

1. Lewandowska M, Nasr S, Shapiro AD. Therapeutic and technological advancements in haemophilia care: Quantum leaps forward. Haemophilia 2022;28 Suppl 4: 77-92. doi: 10.1111/hae.14531. Lewandowska M Nasr S Shapiro AD . Therapeutic and technological advancements in haemophilia care: Quantum leaps forward . Haemophilia 2022 ; 28 Suppl 4 : 77 - 92 . doi: 10.1111/hae.14531 . Open DOISearch in Google Scholar

2. European Union. Regulation (EU) 2021/2282 of the European Parliament and of the Council of 15 December 2021 on health technology assessment and amending Directive 2011/24/EU. PE/80/2021/INIT. 15 December 2021. Available from https://eur-lex.europa.eu/legal-content/EN/ALL/?uri=CELEX:32021R2282 (accessed 26 June 2023). European Union . Regulation (EU) 2021/2282 of the European Parliament and of the Council of 15 December 2021 on health technology assessment and amending Directive 2011/24/EU . PE/80/2021/INIT. 15 December 2021 . Available from https://eur-lex.europa.eu/legal-content/EN/ALL/?uri=CELEX:32021R2282 ( accessed 26 June 2023 ). Search in Google Scholar

3. Srivastava A. The case for equitable haemophilia care. Lancet Haematol 2021; 8(9): e626. doi: 10.1016/S2352-3026(21)00132-0. Srivastava A . The case for equitable haemophilia care . Lancet Haematol 2021 ; 8 ( 9 ): e626 . doi: 10.1016/S2352-3026(21)00132-0 . Open DOISearch in Google Scholar

4. Pierce GF, Adediran M, Diop S, et al. Achieving access to haemophilia care in low-income and lower-middle-income countries: expanded Humanitarian Aid Program of the World Federation of Hemophilia after 5 years. Lancet Haematol 2022; 9(9): e689-e697. doi: 10.1016/S2352-3026(22)00209-5. Pierce GF Adediran M Diop S . Achieving access to haemophilia care in low-income and lower-middle-income countries: expanded Humanitarian Aid Program of the World Federation of Hemophilia after 5 years . Lancet Haematol 2022 ; 9 ( 9 ): e689 - e697 . doi: 10.1016/S2352-3026(22)00209-5 . Open DOISearch in Google Scholar

5. Peluso NL, Ribot J. Postscript: A theory of access revisited. Society and Natural Resources 2020; 33(2): 300-306. doi: 10.1080/08941920.2019.1709929. Peluso NL Ribot J . Postscript: A theory of access revisited . Society and Natural Resources 2020 ; 33 ( 2 ): 300 - 306 . doi: 10.1080/08941920.2019.1709929 . Open DOISearch in Google Scholar

6. Clark J. How to balance equity, equality and fairness. 24 September 2019. Concordia University. Available from https://www.cui.edu/academicprograms/education/servant-leadership-institute/perfecting-the-practice/blog/post/how-to-balance-equity-equality-and-fairness (accessed 2 May 2023). Clark J . How to balance equity, equality and fairness . 24 September 2019 . Concordia University . Available from https://www.cui.edu/academicprograms/education/servant-leadership-institute/perfecting-the-practice/blog/post/how-to-balance-equity-equality-and-fairness ( accessed 2 May 2023 ). Search in Google Scholar

7. Bok A, Noone D, Skouwe-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. Bok A Noone D Skouwe-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 . Open DOISearch in Google Scholar

8. Goodman M. Systems thinking: what, why, when, where and how? The Systems Thinker. Available from https://thesystemsthinker.com/systems-thinking-what-why-when-where-and-how/ (accessed 2 May 2023). Goodman M . Systems thinking: what, why, when, where and how? The Systems Thinker . Available from https://thesystemsthinker.com/systems-thinking-what-why-when-where-and-how/ ( accessed 2 May 2023 ). Search in Google Scholar

9. Carayon P, Wooldridge A, Hoonakker P, Schoofs Hundt A, Kelly MM. SEIPS 3.0: Human-centered design of the patient journey for patient safety. Appl Ergon 2020; 84: 103033. doi: 10.1016/j.apergo.2019.103033. Carayon P Wooldridge A Hoonakker P Schoofs Hundt A Kelly MM . SEIPS 3.0: Human-centered design of the patient journey for patient safety . Appl Ergon 2020 ; 84 : 103033 . doi: 10.1016/j.apergo.2019.103033 . Open DOISearch in Google Scholar

10. Schrijvers G, van Hoorn A, Huiskes N. The care pathway: concepts and theories. Int J Integr Care 2012; 12 (Special Edition Integrated Care Pathways): e192. doi: 10.5334/ijic.812. Schrijvers G van Hoorn A Huiskes N . The care pathway: concepts and theories . Int J Integr Care 2012 ; 12 (Special Edition Integrated Care Pathways): e192 . doi: 10.5334/ijic.812 . Open DOISearch in Google Scholar

11. European Commission. European Reference Networks. Available from https://health.ec.europa.eu/european-reference-networks_en (accessed 19 May 2023). European Commission . European Reference Networks . Available from https://health.ec.europa.eu/european-reference-networks_en ( accessed 19 May 2023 ). Search in Google Scholar

12. Valentino LA, Blanchette V, Negrier C, et al. Personalising haemophilia management with shared decision making. J Haem Pract 2021; 8(1): 69-79. doi: 10.17225/jhp0078. Valentino LA Blanchette V Negrier C . Personalising haemophilia management with shared decision making . J Haem Pract 2021 ; 8 ( 1 ): 69 - 79 . doi: 10.17225/jhp0078 . Open DOISearch in Google Scholar

13. Wang M, Negrier C, Driessler F, Skinner M. The hemophilia gene therapy patient journey: questions and answers for shared decision-making. Patient Prefer Adherence 2022; 16: 1439-1447. doi: 10.2147/PPA.S355627. Wang M Negrier C Driessler F Skinner M . The hemophilia gene therapy patient journey: questions and answers for shared decision-making . Patient Prefer Adherence 2022 ; 16 : 1439 - 1447 . doi: 10.2147/PPA.S355627 . Open DOISearch in Google Scholar

14. Ostlin P, Eckermann E, Shankar Mishra U, Nkowane M, Wallstam E. Gender and health promotion: a multisectoral policy approach. Health Promot Int 2006; 21 Suppl 1:25-35. doi: 10.1093/heapro/dal048. Ostlin P Eckermann E Shankar Mishra U Nkowane M Wallstam E . Gender and health promotion: a multisectoral policy approach . Health Promot Int 2006 ; 21 Suppl 1 : 25 - 35 . doi: 10.1093/heapro/dal048 . Open DOISearch in Google Scholar

15. Weyand AC, James PD. Sexism in the management of bleeding disorders. Res Pract Thromb Haemost 2020; 5(1): 51-54. doi: 10.1002/rth2.12468. Weyand AC James PD . Sexism in the management of bleeding disorders . Res Pract Thromb Haemost 2020 ; 5 ( 1 ): 51 - 54 . doi: 10.1002/rth2.12468 . Open DOISearch in Google Scholar

16. Sanigorska A, Chaplin S, Holland M, et al. The lived experience of women with a bleeding disorder: A systematic review. Res Pract Thromb Haemost 2022; 6(1): e12652. doi: 10.1002/rth2.12652. Sanigorska A Chaplin S Holland M . The lived experience of women with a bleeding disorder: A systematic review . Res Pract Thromb Haemost 2022 ; 6 ( 1 ): e12652 . doi: 10.1002/rth2.12652 . Open DOISearch in Google Scholar

17. EURORDIS Rare Diseases Europe. Newborn screening. Available from https://www.eurordis.org/our-priorities/diagnosis/newborn-screening/ (accessed 19 June 2023). EURORDIS Rare Diseases Europe . Newborn screening . Available from https://www.eurordis.org/our-priorities/diagnosis/newborn-screening/ ( accessed 19 June 2023 ). Search in Google Scholar

18. Loeber G, Platis D, Zetterström RH, et al. Neonatal screening in Europe revisited: An ISNS perspective on the current state and developments since 2010. J Neonatal Screen 2021; 7(1): 15. doi: 10.3390/ijns7010015. Loeber G Platis D Zetterström RH . Neonatal screening in Europe revisited: An ISNS perspective on the current state and developments since 2010 . J Neonatal Screen 2021 ; 7 ( 1 ): 15 . doi: 10.3390/ijns7010015 . Open DOISearch in Google Scholar

19. DiMichele D, Chuansumrit A, London AJ, et al. Ethical issues in haemophilia. Haemophilia 2006; 12(s3): 30-35. doi: 10.1111/j.1365-2516.2006.01258.x. DiMichele D Chuansumrit A London AJ . Ethical issues in haemophilia . Haemophilia 2006 ; 12 (s3): 30 - 35 . doi: 10.1111/j.1365-2516.2006.01258.x . Open DOISearch in Google Scholar

20. Noone D, Coffin D, Pierce G. Reimbursing the value of gene therapy care in an era of uncertainty. Haemophilia 2021; 27(1): 12-18. doi: 10.1111/hae.14218. Noone D Coffin D Pierce G . Reimbursing the value of gene therapy care in an era of uncertainty . Haemophilia 2021 ; 27 ( 1 ): 12 - 18 . doi: 10.1111/hae.14218 . Open DOISearch in Google Scholar

21. O’Hara J, Neumann PJ, International Haemophilia Access Strategy Council. Health technology assessment for gene therapies in haemophilia. Haemophilia 2022; 28(S2): 19-26. doi: 10.1111/hae.14413. O’Hara J Neumann PJ International Haemophilia Access Strategy Council . Health technology assessment for gene therapies in haemophilia . Haemophilia 2022 ; 28 (S2): 19 - 26 . doi: 10.1111/hae.14413 . Open DOISearch in Google Scholar

22. Fox T, Bueren J, Candotti F, et al. Access to gene therapy for rare diseases when commercialization is not firt for purpose. Nat Med 2023; 29(3): 518-519. doi: 10.1038/s41591-023-02208-8. Fox T Bueren J Candotti F . Access to gene therapy for rare diseases when commercialization is not firt for purpose . Nat Med 2023 ; 29 ( 3 ): 518 - 519 . doi: 10.1038/s41591-023-02208-8 . Open DOISearch in Google Scholar

23. Oliver A, Mossialos E. Equity of access to health care: outlining the foundations for action. J Epidemiol Community Health 2004; 58(8): 655-658. doi: 10.1136/jech.2003.017731. Oliver A Mossialos E . Equity of access to health care: outlining the foundations for action . J Epidemiol Community Health 2004 ; 58 ( 8 ): 655 - 658 . doi: 10.1136/jech.2003.017731 . Open DOISearch in Google Scholar

24. Abdallah K, Claes K, Huys I, et al. Exploring alternative financing models and early access schemes for orphan drugs: a Belgian case study. Orphanet J Rare Dis 2022; 17: 429. doi: 10.1186/s13023-022-02571-8. Abdallah K Claes K Huys I . Exploring alternative financing models and early access schemes for orphan drugs: a Belgian case study . Orphanet J Rare Dis 2022 ; 17 : 429 . doi: 10.1186/s13023-022-02571-8 . Open DOISearch in Google Scholar

25. Doetsch JN, Schlösser C, Barros H, et al. A scoping review on the impact of austerity on healthcare access in the European Union: rethinking austerity for the most vulnerable. Int J Equity Health 2023; 22(1): 3. doi: 10.11868/s12939-022-01806-1. Doetsch JN Schlösser C Barros H . A scoping review on the impact of austerity on healthcare access in the European Union: rethinking austerity for the most vulnerable . Int J Equity Health 2023 ; 22 ( 1 ): 3 . doi: 10.11868/s12939-022-01806-1 . Open DOISearch in Google Scholar

26. Rifat A. Health systems, systems thinking and innovation. Health Policy Plan 2012; 27 Suppl 4: 4-8. doi: 10.1093/heapol/czs088. Rifat A . Health systems, systems thinking and innovation . Health Policy Plan 2012 ; 27 Suppl 4 : 4 - 8 . doi: 10.1093/heapol/czs088 . Open DOISearch in Google Scholar

27. Ledley FD, McCoy SS, Vaughan G, Cleary EG. Profitability of large pharmaceutical companies compared with other large public companies. JAMA 2020; 323(9): 834-843. doi: 10.1001/jama.2020.0442. Ledley FD McCoy SS Vaughan G Cleary EG . Profitability of large pharmaceutical companies compared with other large public companies . JAMA 2020 ; 323 ( 9 ): 834 - 843 . doi: 10.1001/jama.2020.0442 . Open DOISearch in Google Scholar

28. Tomeo F, Mariz S, Brunei AL, et al. Haemophilia, state of the art and new therapeutic opportunities: a regulatory perspective. Br J Clin Pharmacol 2021; 87: 4183-4196. doi: 10.1111/bcp.14838. Tomeo F Mariz S Brunei AL . Haemophilia, state of the art and new therapeutic opportunities: a regulatory perspective . Br J Clin Pharmacol 2021 ; 87 : 4183 - 4196 . doi: 10.1111/bcp.14838 . Open DOISearch in Google Scholar

29. van der Gronde T, Uyl-de Groot CA, Pieters T. Addressing the challenge of high-price prescription drugs in the era of precision medicine: A systematic review. PLoS One 2017; 12(8): e0182613. doi: 10.1371/journal.pone.0182613. van der Gronde T Uyl-de Groot CA Pieters T . Addressing the challenge of high-price prescription drugs in the era of precision medicine: A systematic review . PLoS One 2017 ; 12 ( 8 ): e0182613 . doi: 10.1371/journal.pone.0182613 . Open DOISearch in Google Scholar

30. Devlin NJ, Brooks R. EQ-5D and the EuroQol Group: past, present and future. Appl Health Econ Health Policy 2017; 15(2): 127-137. doi: 10.1007/s40258-017-0310-5. Devlin NJ Brooks R . EQ-5D and the EuroQol Group: past, present and future . Appl Health Econ Health Policy 2017 ; 15 ( 2 ): 127 - 137 . doi: 10.1007/s40258-017-0310-5 . Open DOISearch in Google Scholar

31. Grut L, Kvam MH. Facing ignorance: people with rare disorders and their experiences with public health and welfare services. Scand J Disabil Res 2013; 15(1): 20-32. doi: 10.1080/15017419.2011.645870. Grut L Kvam MH . Facing ignorance: people with rare disorders and their experiences with public health and welfare services . Scand J Disabil Res 2013 ; 15 ( 1 ): 20 - 32 . doi: 10.1080/15017419.2011.645870 . Open DOISearch in Google Scholar

32. Potter BK, Khangura SD, Tingley K, et al. Translating rare-disease therapies into improved care for patients and families: what are the right outcomes, designs, and engagement approaches in health-systems research? Genet Med 2016; 18: 117-123. doi: 10.1038/gim.2015.42. Potter BK Khangura SD Tingley K . Translating rare-disease therapies into improved care for patients and families: what are the right outcomes, designs, and engagement approaches in health-systems research? Genet Med 2016 ; 18 : 117 - 123 . doi: 10.1038/gim.2015.42 . Open DOISearch in Google Scholar

33. Vogler S. Prices of new medicines: International analysis and policy options. Z Evid Fortbild Qual Gesundhwes 2022; 175: 96-102. doi: 10.1016/j.zefq.2022.09.009. Vogler S . Prices of new medicines: International analysis and policy options . Z Evid Fortbild Qual Gesundhwes 2022 ; 175 : 96 - 102 . doi: 10.1016/j.zefq.2022.09.009 . Open DOISearch in Google Scholar

34. Vogler S, Paris V, Ferrario A, et al. How can pricing and reimbursement policies improve affordable access to medicines? Lessons learned from European countries. Appl Health Econ Health Policy 2017; 15(3): 307-321. doi: 10.1007/s40258-016-0300-z. Vogler S Paris V Ferrario A . How can pricing and reimbursement policies improve affordable access to medicines? Lessons learned from European countries . Appl Health Econ Health Policy 2017 ; 15 ( 3 ): 307 - 321 . doi: 10.1007/s40258-016-0300-z . Open DOISearch in Google Scholar

35. Adachi T, El-Hattab AW, Jain R, et al. Enhancing equitable access to rare disease diagnosis and treatment around the world: a review of evidence, policies, and challenges. Int J Environ Res Public Health 2023; 20(6): 4732. doi: 10.3390/ijerph20064732. Adachi T El-Hattab AW Jain R . Enhancing equitable access to rare disease diagnosis and treatment around the world: a review of evidence, policies, and challenges . Int J Environ Res Public Health 2023 ; 20 ( 6 ): 4732 . doi: 10.3390/ijerph20064732 . Open DOISearch in Google Scholar

36. Moon S, Mariat S, Kamae I, Bak Pedersen H. Defining the concept of fair pricing for medicines. BMJ 2020; 368: 14276. doi: 10.1136/bmj.14726. Moon S Mariat S Kamae I Bak Pedersen H . Defining the concept of fair pricing for medicines . BMJ 2020 ; 368 : 14276 . doi: 10.1136/bmj.14726 . Open DOISearch in Google Scholar

37. Rémuzat C, Urbaniti D, Mzoughi O, et al. Overview of external reference pricing systems in Europe. J Mark Access Health Policy 2015; 3: 10.3402/jmahp.v3.27675. doi: 10.3402/jmahp.v3.27675. Rémuzat C Urbaniti D Mzoughi O . Overview of external reference pricing systems in Europe . J Mark Access Health Policy 2015 ; 3 : 10.3402/jmahp.v3.27675. doi: 10.3402/jmahp.v3.27675 . Open DOISearch in Google Scholar

38. Miesbach W, Chowdary P, Coppens M, et al. Delivery of AAV-based gene therapy through haemophilia centres – A need for re-evaluation of infrastructure and comprehensive care: A joint publication of EAHAD and EHC. Haemophilia 2021; 27(6): 967-973. doi: 10.1111/hae.14420. Miesbach W Chowdary P Coppens M . Delivery of AAV-based gene therapy through haemophilia centres – A need for re-evaluation of infrastructure and comprehensive care: A joint publication of EAHAD and EHC . Haemophilia 2021 ; 27 ( 6 ): 967 - 973 . doi: 10.1111/hae.14420 . Open DOISearch in Google Scholar

39. Bok A, Noone D, Skouw-Rasmussen N. 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 . 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 . Open DOISearch in Google Scholar

40. Hedley V, Castro R, Rodwell C, Simon F. Rare 2030 Knowledge Base Summary on Integrated Social and Holistic Care for Rare Diseases. 2019. Available from https://www.rare2030.eu/our-work/ (accessed 19 June 2023). Hedley V Castro R Rodwell C Simon F . Rare 2030 Knowledge Base Summary on Integrated Social and Holistic Care for Rare Diseases . 2019 . Available from https://www.rare2030.eu/our-work/ ( accessed 19 June 2023 ). Search in Google Scholar

41. EHC. Think Tank. Available from https://ehcthinktank.eu (accessed 26 June 2023). EHC . Think Tank . Available from https://ehcthinktank.eu ( accessed 26 June 2023 ). Search in Google Scholar

42. Chatham House. Chatham House rule. Available from https://www.chathamhouse.org/about-us/chatham-house-rule (accessed 26 June 2023). Chatham House . Chatham House rule . Available from https://www.chathamhouse.org/about-us/chatham-house-rule ( accessed 26 June 2023 ). Search in Google Scholar