Importance of aligning the implementation of new payment models for innovative pharmaceuticals in European countries

References

• Malerba F, Orsenigo L. The evolution of the pharmaceutical industry. Bus Hist. 2015;57(5):664–687. doi: 10.1080/00076791.2014.975119
   Web of Science ®Google Scholar
• Schlander M, Hernandez-Villafuerte K, Cheng CY, et al. How much does it cost to research and develop a new drug? A systematic review and assessment a systematic review and assessment. PharmacoEconomics. 2021;39(11):1243–1269. doi: 10.1007/s40273-021-01065-y
   PubMed Web of Science ®Google Scholar
• Pontes C, Fontanet JM, Vives R, et al. Evidence supporting regulatory-decision making on orphan medicinal products authorisation in Europe: methodological uncertainties. Orphanet J Rare Dis. 2018;13(1):13. 206. doi: 10.1186/s13023-018-0926-z
   Google Scholar
• Tanveer S, Rowhani-Farid A, Hong K, et al. Transparency of COVID-19 vaccine trials: decisions without data. BMJ Evid Based Med. 2022;27(4):27. 199–205. doi: 10.1136/bmjebm-2021-111735
   PubMedGoogle Scholar
• Iglesias-Lopez C, Agustí A, Vallano A, et al. Current landscape of clinical development and approval of advanced therapies. Mol Ther Methods Clin Dev. 2021;23:11. 606–618. doi: 10.1016/j.omtm.2021.11.003
   Web of Science ®Google Scholar
• Dankó D, Blay JY, Garrison LP. Challenges in the value assessment, pricing and funding of targeted combination therapies in oncology. Health Policy. 2019;123(12):123. 1230–1236. doi: 10.1016/j.healthpol.2019.07.009
   PubMed Web of Science ®Google Scholar
• SiRM, L.E.K. Consulting & RAND Europe. The financial ecosystem of pharmaceutical R&D: an evidence base to inform further dialogue; 2022. www.sirm.nl/en/publications/the-financial-ecosystem-of-pharmaceutical-r-d
   Google Scholar
• Hughes DA, Poletti-Hughes J, Koomen JM. Profitability and market value of orphan drug companies: a retrospective, propensity-matched case-control study. PLoS One. 2016;1111(10):e0164681. doi: 10.1371/journal.pone.0164681
   PubMed Web of Science ®Google Scholar
• Marselis D, Hordijk L. From blockbuster to “nichebuster”: how a flawed legislation helped create a new profit model for the drug industry. BMJ. 2020;370:m2983. doi: 10.1136/bmj.m2983
   PubMed Web of Science ®Google Scholar
• Laverty H, Meulien P. The innovative medicines initiative -10 years of public-private collaboration. Front Med. 2019 3;6:275. doi: 10.3389/fmed.2019.00275
   Web of Science ®Google Scholar
• Suleman F, Low M, Moon S, et al. New business models for research and development with affordability requirements are needed to achieve fair pricing of medicines. BMJ. 2020;368:l4408. doi: 10.1136/bmj.l4408
   PubMedGoogle Scholar
• Moon S, Vieira M, Ruiz AA, et al. New Business models for pharmaceutical research and development as a global public good: considerations for the WHO European region. World Health Org. 2022. Available from: https://www.who.int/europe/publications/i/item/9789289058124
   Google Scholar
• Lemmens T, Ghimire KM, Perehudoff K, et al. The Social Contract and Human Rights Bases for promoting access to effective, novel, high-priced medicines. World Health Org. 2022. https://www.who.int/europe/publications/i/item/9789289058261
   Google Scholar
• Vreman RA, Broekhoff TF, Leufkens HG, et al. Application of managed entry agreements for innovative therapies in different settings and combinations: a feasibility analysis. Int J Environ Res Public Health. 2020;17(22):10. 8309. doi: 10.3390/ijerph17228309
   PubMed Web of Science ®Google Scholar
• Garrison LP Jr, Pauly MV, Willke RJ, et al. An overview of value, perspective, and decision context—A health economics approach: an ISPOR special task force report [2]. Value In Health. 2018;21(2):124–130. doi: 10.1016/j.jval.2017.12.006
   PubMed Web of Science ®Google Scholar
• Landon SN, Padikkala J, Horwitz LI. Defining value in health care: a scoping review of the literature. Int J Qual Health Care. 2021;33(4):mzab140. doi: 10.1093/intqhc/mzab140
   PubMed Web of Science ®Google Scholar
• Jakab I, Whittington MD, Franklin E, et al. Patient and payer preferences for additional value criteria. Front Pharmacol. 2021;24:690021. doi: 10.3389/fphar.2021.690021
   Google Scholar
• Kolasa K, Zwolinski KM, Zah V, et al. Revealed preferences towards the appraisal of orphan drugs in Poland - multi criteria decision analysis. Orphanet J Rare Dis. 2018;27(1):67. doi: 10.1186/s13023-018-0803-9
   Google Scholar
• Zamora B, Garrison LP, Unuigbe A, et al. Reconciling ACEA and MCDA: is there a way forward for measuring cost-effectiveness in the U.S. healthcare setting? Cost Eff Resour Alloc. 2021;19(1):13. doi: 10.1186/s12962-021-00266-8
   PubMed Web of Science ®Google Scholar
• Baltussen R, Marsh K, Thokala P, et al. Multicriteria decision analysis to support health technology assessment agencies: benefits, limitations, and the way forward. Value Health. 2019;22(11):1283–1288. doi: 10.1016/j.jval.2019.06.014
   PubMed Web of Science ®Google Scholar
• Garrison LP Jr, Towse A, Briggs A, et al. Performance-based risk-sharing arrangements—good practices for design, implementation, and evaluation: report of the ISPOR good practices for performance-based risk-sharing arrangements task force. Value In Health. 2013;16(5):703–719. doi: 10.1016/j.jval.2013.04.011
   PubMed Web of Science ®Google Scholar
• Ádám I, Callenbach M, Németh B, et al. Recommendations for implementing outcome-based reimbursement models for new technologies in Central and Eastern European and Middle-Eastern countries. Front Med. 2022;9:940886. doi: 10.3389/fmed.2022.940886
   Google Scholar
• Xoxi E, Facey KM, Cicchetti A. The evolution of AIFA registries to support managed entry agreements for orphan medicinal products in Italy. Front Pharmacol. 2021 10;12:699466. doi: 10.3389/fphar.2021.699466
   PubMed Web of Science ®Google Scholar
• Pauwels K, Huys I, Vogler S, et al. Managed entry agreements for oncology drugs: lessons from the European Experience to Inform the future. Front Pharmacol. 2017 4;8:171. doi: 10.3389/fphar.2017.00171
   PubMed Web of Science ®Google Scholar
• Callenbach MHE, Ádám L, Vreman RA, et al. Reimbursement and payment models in central and Eastern European as well as Middle Eastern countries: a survey of their current use and future outlook. Drug Discov Today. 2023;28(1):103433. doi: 10.1016/j.drudis.2022.103433
   PubMed Web of Science ®Google Scholar
• Vreman RA, Bloem LT, van Oirschot S, et al. The role of regulator-imposed post-approval studies in health technology assessments for conditionally approved drugs. Int J Health Policy Manag. 2022 1;11(5):642–650. doi: 10.34172/ijhpm.2020.198
   PubMed Web of Science ®Google Scholar
• Eichler HG, Adams R, Andreassen E, et al. Exploring the opportunities for alignment of regulatory post-authorization requirements and data required for performance-based managed entry agreements. Int J Technol Assess Health Care. 2021;23(1):e83. doi: 10.1017/S026646232100057X
   Google Scholar
• Inotai A, Tomek D, Niewada M, et al. Identifying patient access barriers for tumor necrosis factor alpha inhibitor treatments in rheumatoid arthritis in five Central Eastern European countries. Front Pharmacol. 2020;1111:845. doi: 10.3389/fphar.2020.00845
   PubMed Web of Science ®Google Scholar
• World Health Organization. Regional Office for Europe. Cross-country collaborations to improve access to medicines and vaccines in the WHO European Region. World Health Organization. Regional Office for Europe; 2020. Richardson E, Palm W, Mossialos E, editors. Copenhagen (Denmark): European Observatory on Health Systems and Policies; 2018 https://apps.who.int/iris/handle/10665/332933
   Google Scholar
• European Court of Auditors. Special report 19/2022: EU COVID-19 vaccine procurement; 2022. (https://www.eca.europa.eu/Lists/ECADocuments/SR22_19/SR_EU_COVID_vaccine_procurement_EN.pdf)
   Google Scholar
• Löblová O, Csanádi M, Ozieranski P, et al. Alternative access schemes for pharmaceuticals in Europe: towards an emerging typology. Health Policy. 2019;7123(7):630–634. doi: 10.1016/j.healthpol.2019.05.012
   Google Scholar
• Lukács G, Kovács Á, Csanádi M, et al. Benefits of timely care in pancreatic cancer: a systematic review to navigate through the contradictory evidence. Cancer Manag Res. 2019 19;11:9849–9861. doi: 10.2147/CMAR.S221427
   PubMed Web of Science ®Google Scholar
• Bucek Psenkova M, Visnansky M, Mackovicova S, et al. Drug policy in Slovakia. Value Health Reg Issues. 2017;13:44–49. doi: 10.1016/j.vhri.2017.07.002
   PubMedGoogle Scholar
• Inotai A, Kaló Z. How to solve financing gap to ensure patient access to patented pharmaceuticals in CEE countries? – the good, the bad, and the ugly ways. Expert Rev Pharmacoecon Outcomes Res. 2019;19(6):627–632. doi: 10.1080/14737167.2019.1702524
   PubMed Web of Science ®Google Scholar
• Salzman R, Cook F, Hunt T, et al. Addressing the value of gene therapy and enhancing patient access to transformative treatments. Mol Ther. 2018 5;26(12):2717–2726. doi: 10.1016/j.ymthe.2018.10.017
   PubMed Web of Science ®Google Scholar
• Vreman RA, Leufkens HGM, Kesselheim AS. Getting the right evidence after drug approval. Front Pharmacol. 2020 9;11:569535. doi: 10.3389/fphar.2020.569535
   PubMed Web of Science ®Google Scholar
• Michelsen S, Nachi S, Van Dyck W, et al. Barriers and opportunities for implementation of outcome-based spread payments for high-cost, one-shot curative therapies. Front Pharmacol. 2020;1:594446. doi: 10.3389/fphar.2020.594446
   Google Scholar
• Ádám I, Callenbach M, Németh B, et al. Delayed payment schemes in Central-Eastern Europe and Middle-East. Front Med (Lausanne). 2022;9:940371. doi: 10.3389/fmed.2022.940371
   PubMed Web of Science ®Google Scholar
• Simoens S, De Groote K, Boersma C. Critical reflections on reimbursement and access of advanced therapies. Front Pharmacol. 2022 18;13:771966. doi: 10.3389/fphar.2022.771966
   PubMed Web of Science ®Google Scholar
• Vogler S, Paris V, Panteli D Ensuring access to medicines: how to redesign pricing, reimbursement and procurement? Policy brief 30. European Observatory on Health Systems and Policies, WHO; 2018 https://www.euro.who.int/__data/assets/pdf_file/0009/379710/PolicyBrief_AUSTRIA_PB30_web_13082018.pdf
   Google Scholar
• Docteur E Towards a new vision for shared responsibility in pharmaceutical pricing, coverage and reimbursement: policy approaches building on principles of solidarity, transparency and sustainability. World Health Org; 2022. https://www.who.int/europe/publications/i/item/9789289058193
   Google Scholar