Access equity: key questions and challenges - A report from the 1st workshop of the European Haemophilia Consortium (EHC) Think Tank Workstream on Access Equity
This work is licensed under the Creative Commons Attribution-NonCommercial-NoDerivatives 3.0 License.
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.LewandowskaMNasrSShapiroAD.Therapeutic and technological advancements in haemophilia care: Quantum leaps forward.2022;28Suppl 4:77-92. doi:10.1111/hae.14531.Open DOISearch in Google Scholar
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.. PE/80/2021/INIT.15December2021. Available from https://eur-lex.europa.eu/legal-content/EN/ALL/?uri=CELEX:32021R2282(accessed 26 June 2023).Search in Google Scholar
Srivastava A. The case for equitable haemophilia care. Lancet Haematol 2021; 8(9): e626. doi: 10.1016/S2352-3026(21)00132-0.SrivastavaA.The case for equitable haemophilia care.2021;8(9):e626. doi:10.1016/S2352-3026(21)00132-0.Open DOISearch in Google Scholar
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.PierceGFAdediranMDiopS.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.2022;9(9):e689-e697. doi:10.1016/S2352-3026(22)00209-5.Open DOISearch in Google Scholar
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.PelusoNLRibotJ.Postscript: A theory of access revisited.2020;33(2):300-306. doi:10.1080/08941920.2019.1709929.Open DOISearch in Google Scholar
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).ClarkJ..24September2019.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
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.BokANooneDSkouwe-RasmussenNEHC Think Tank.Patient agency: key questions and challenges – A report from the 1st workshop of the EHC Think Tank Workstream on Patient Agency.2022;9(1):27-35. doi:10.2478/jhp-2022-0004.Open DOISearch in Google Scholar
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).GoodmanM.. Available from https://thesystemsthinker.com/systems-thinking-what-why-when-where-and-how/(accessed 2 May 2023).Search in Google Scholar
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.CarayonPWooldridgeAHoonakkerPSchoofs HundtAKellyMM.SEIPS 3.0: Human-centered design of the patient journey for patient safety.2020;84:103033. doi:10.1016/j.apergo.2019.103033.Open DOISearch in Google Scholar
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.SchrijversGvan HoornAHuiskesN.The care pathway: concepts and theories.2012;12(Special Edition Integrated Care Pathways):e192. doi:10.5334/ijic.812.Open DOISearch in Google Scholar
European Commission. European Reference Networks. Available from https://health.ec.europa.eu/european-reference-networks_en (accessed 19 May 2023).European Commission..Available from https://health.ec.europa.eu/european-reference-networks_en(accessed 19 May 2023).Search in Google Scholar
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.ValentinoLABlanchetteVNegrierC.Personalising haemophilia management with shared decision making.2021;8(1):69-79. doi:10.17225/jhp0078.Open DOISearch in Google Scholar
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.WangMNegrierCDriesslerFSkinnerM.The hemophilia gene therapy patient journey: questions and answers for shared decision-making.2022;16:1439-1447. doi:10.2147/PPA.S355627.Open DOISearch in Google Scholar
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.OstlinPEckermannEShankar MishraUNkowaneMWallstamE.Gender and health promotion: a multisectoral policy approach.2006;21Suppl 1:25-35. doi:10.1093/heapro/dal048.Open DOISearch in Google Scholar
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.WeyandACJamesPD.Sexism in the management of bleeding disorders.2020;5(1):51-54. doi:10.1002/rth2.12468.Open DOISearch in Google Scholar
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.SanigorskaAChaplinSHollandM.The lived experience of women with a bleeding disorder: A systematic review.2022;6(1):e12652. doi:10.1002/rth2.12652.Open DOISearch in Google Scholar
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.. Available from https://www.eurordis.org/our-priorities/diagnosis/newborn-screening/(accessed 19 June 2023).Search in Google Scholar
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.LoeberGPlatisDZetterströmRH.Neonatal screening in Europe revisited: An ISNS perspective on the current state and developments since 2010.2021;7(1):15. doi:10.3390/ijns7010015.Open DOISearch in Google Scholar
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.DiMicheleDChuansumritALondonAJ.Ethical issues in haemophilia.2006;12(s3):30-35. doi:10.1111/j.1365-2516.2006.01258.x.Open DOISearch in Google Scholar
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.NooneDCoffinDPierceG.Reimbursing the value of gene therapy care in an era of uncertainty.2021;27(1):12-18. doi:10.1111/hae.14218.Open DOISearch in Google Scholar
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’HaraJNeumannPJInternational Haemophilia Access Strategy Council.Health technology assessment for gene therapies in haemophilia.2022;28(S2):19-26. doi:10.1111/hae.14413.Open DOISearch in Google Scholar
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.FoxTBuerenJCandottiF.Access to gene therapy for rare diseases when commercialization is not firt for purpose.2023;29(3):518-519. doi:10.1038/s41591-023-02208-8.Open DOISearch in Google Scholar
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.OliverAMossialosE.Equity of access to health care: outlining the foundations for action.2004;58(8):655-658. doi:10.1136/jech.2003.017731.Open DOISearch in Google Scholar
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.AbdallahKClaesKHuysI.Exploring alternative financing models and early access schemes for orphan drugs: a Belgian case study.2022;17:429. doi:10.1186/s13023-022-02571-8.Open DOISearch in Google Scholar
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.DoetschJNSchlösserCBarrosH.A scoping review on the impact of austerity on healthcare access in the European Union: rethinking austerity for the most vulnerable.2023;22(1):3. doi:10.11868/s12939-022-01806-1.Open DOISearch in Google Scholar
Rifat A. Health systems, systems thinking and innovation. Health Policy Plan 2012; 27 Suppl 4: 4-8. doi: 10.1093/heapol/czs088.RifatA.Health systems, systems thinking and innovation.2012;27Suppl 4:4-8. doi:10.1093/heapol/czs088.Open DOISearch in Google Scholar
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.LedleyFDMcCoySSVaughanGClearyEG.Profitability of large pharmaceutical companies compared with other large public companies.2020;323(9):834-843. doi:10.1001/jama.2020.0442.Open DOISearch in Google Scholar
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.TomeoFMarizSBruneiAL.Haemophilia, state of the art and new therapeutic opportunities: a regulatory perspective.2021;87:4183-4196. doi:10.1111/bcp.14838.Open DOISearch in Google Scholar
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 GrondeTUyl-de GrootCAPietersT.Addressing the challenge of high-price prescription drugs in the era of precision medicine: A systematic review.2017;12(8):e0182613. doi:10.1371/journal.pone.0182613.Open DOISearch in Google Scholar
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.DevlinNJBrooksR.EQ-5D and the EuroQol Group: past, present and future.2017;15(2):127-137. doi:10.1007/s40258-017-0310-5.Open DOISearch in Google Scholar
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.GrutLKvamMH.Facing ignorance: people with rare disorders and their experiences with public health and welfare services.2013;15(1):20-32. doi:10.1080/15017419.2011.645870.Open DOISearch in Google Scholar
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.PotterBKKhanguraSDTingleyK.Translating rare-disease therapies into improved care for patients and families: what are the right outcomes, designs, and engagement approaches in health-systems research?2016;18:117-123. doi:10.1038/gim.2015.42.Open DOISearch in Google Scholar
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.VoglerS.Prices of new medicines: International analysis and policy options.2022;175:96-102. doi:10.1016/j.zefq.2022.09.009.Open DOISearch in Google Scholar
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.VoglerSParisVFerrarioA.How can pricing and reimbursement policies improve affordable access to medicines? Lessons learned from European countries.2017;15(3):307-321. doi:10.1007/s40258-016-0300-z.Open DOISearch in Google Scholar
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.AdachiTEl-HattabAWJainR.Enhancing equitable access to rare disease diagnosis and treatment around the world: a review of evidence, policies, and challenges.2023;20(6):4732. doi:10.3390/ijerph20064732.Open DOISearch in Google Scholar
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.MoonSMariatSKamaeIBak PedersenH.Defining the concept of fair pricing for medicines.2020;368:14276. doi:10.1136/bmj.14726.Open DOISearch in Google Scholar
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.RémuzatCUrbanitiDMzoughiO.Overview of external reference pricing systems in Europe.2015;3: 10.3402/jmahp.v3.27675. doi:10.3402/jmahp.v3.27675.Open DOISearch in Google Scholar
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.MiesbachWChowdaryPCoppensM.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.2021;27(6):967-973. doi:10.1111/hae.14420.Open DOISearch in Google Scholar
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.BokANooneDSkouw-RasmussenN.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.2022;9(1):20-26. doi:10.2478/jhp-2022-0003.Open DOISearch in Google Scholar
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).HedleyVCastroRRodwellCSimonF..2019. Available from https://www.rare2030.eu/our-work/(accessed 19 June 2023).Search in Google Scholar
EHC. Think Tank. Available from https://ehcthinktank.eu (accessed 26 June 2023).EHC.. Available from https://ehcthinktank.eu(accessed 26 June 2023).Search in Google Scholar
Chatham House. Chatham House rule. Available from https://www.chathamhouse.org/about-us/chatham-house-rule (accessed 26 June 2023).Chatham House.. Available from https://www.chathamhouse.org/about-us/chatham-house-rule(accessed 26 June 2023).Search in Google Scholar