Challenges for gene therapy in the financial sustainability of health systems: a scoping review

Han Q, Fu H, Chu X, Wen R, Zhang M, You T, et al. Research advances in treatment methods and Drug Development for Rare diseases. Front Pharmacol. 2022;13:971541.

Article  CAS  PubMed  PubMed Central  Google Scholar 

National Cancer Institute. Gene Therapy. https://www.cancer.gov/publications/dictionaries/cancer-terms/def/gene-therapy. Accesed 31 May 2023.

Shahryari A, Saghaeian Jazi M, Mohammadi S, Razavi Nikoo H, Nazari Z, Hosseini ES, et al. Development and clinical translation of approved gene Therapy products for Genetic disorders. Front Genet. 2019;10:868.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Kerpel-Fronius S, Baroutsou V, Becker S, Carlesi R, Collia L, Franke-Bray B, et al. Development and Use of Gene Therapy Orphan drugs-ethical needs for a broader Cooperation between the Pharmaceutical Industry and Society. Front Med (Lausanne). 2020;7:608249.

Article  PubMed  Google Scholar 

Jørgensen J, Kefalas P. Annuity payments can increase Patient Access to innovative cell and Gene Therapies under England’s Net Budget Impact Test. J Mark Access Health Policy. 2017;5(1):1355203.

Article  PubMed  PubMed Central  Google Scholar 

Muigai AWT. Expanding Global Access to genetic therapies. Nat Biotechnol. 2022;40(1):20–1.

Article  CAS  PubMed  Google Scholar 

Hanna E, Rémuzat C, Auquier P, Toumi M. Advanced Therapy Medicinal products: current and future perspectives. J Mark Access Health Policy. 2016;4.

Abou-El-Enein M, Hey SP. Cell and Gene Therapy Trials: Are We Facing an ‘Evidence Crisis’? EClinicalMedicine. 2019;7:13 – 4.

Sharpe M, Barry J, Kefalas P. Clinical adoption of Advanced therapies: challenges and opportunities. J Pharm Sci. 2021;110(5):1877–84.

Article  CAS  PubMed  Google Scholar 

Coyle D, Durand-Zaleski I, Farrington J, Garrison L, Graf von der Schulenburg J-M, Greiner W, et al. Hta Methodology and Value frameworks for evaluation and policy making for cell and gene therapies. Eur J Health Econ. 2020;21(9):1421–37.

Article  PubMed  Google Scholar 

Peters M, Godfrey C, McInerney P, Soares C, Khalil H, Parker D. Methodology for Jbi Scoping Reviews. 2015. pp. 1–24.

Tricco AC, Lillie E, Zarin W, O’Brien KK, Colquhoun H, Levac D, et al. Prisma Extension for scoping reviews (Prisma-Scr): Checklist and Explanation. Ann Intern Med. 2018;169(7):467–73.

Article  PubMed  Google Scholar 

Carr DR, Bradshaw SE. Gene therapies: the challenge of Super-high-cost treatments and how to pay for them. Regen Med. 2016;11(4):381–93.

Article  CAS  PubMed  Google Scholar 

Lee S, Lee JH. Cell and Gene Therapy Regulatory, Pricing, and Reimbursement Framework: With a Focus on South Korea and the Eu. 2023;11.

Barlow JF, Yang M, Teagarden JR. Are payers ready, willing, and able to provide Access to new durable gene therapies? Value Health. 2019;22(6):642–7.

Article  PubMed  Google Scholar 

Ballreich J, Ezebilo I, Sharfstein J. Affording Genetic therapies in the Medicaid Program. JAMA Pediatr. 2020;174(6):523–4.

Article  PubMed  Google Scholar 

Jørgensen J, Hanna E, Kefalas P. Outcomes-based reimbursement for Gene therapies in Practice: the experience of recently launched Car-T cell therapies in major European countries. J Mark Access Health Policy. 2020;8(1):1715536.

Article  PubMed  PubMed Central  Google Scholar 

van Overbeeke E, Michelsen S, Toumi M, Stevens H, Trusheim M, Huys I, et al. Market Access of Gene therapies across Europe, USA, and Canada: challenges, trends, and solutions. Drug Discov Today. 2021;26(2):399–415.

Article  PubMed  Google Scholar 

Jørgensen J, Kefalas P. The use of innovative payment mechanisms for Gene Therapies in Europe and the USA. Regen Med. 2021;16(4):405–22.

Article  PubMed  Google Scholar 

Dabbous M, Toumi M, Simoens S, Wasem J, Saal G, Wang Y, et al. Amortization of gene replacement therapies: a Health Policy Analysis exploring a mechanism for Mitigating Budget Impact of high-cost treatments. Health Policy. 2022;126(1):49–59.

Article  PubMed  Google Scholar 

Koleva-Kolarova R, Buchanan J, Vellekoop H, Huygens S, Versteegh M, Mölken MR, et al. Financing and reimbursement models for Personalised Medicine: a systematic review to identify current models and future options. Appl Health Econ Health Policy. 2022;20(4):501–24.

Article  PubMed  PubMed Central  Google Scholar 

Hampson G, Towse A, Pearson SD, Dreitlein WB, Henshall C. Gene Therapy: evidence, Value and Affordability in the us Health Care System. J Comp Eff Res. 2018;7(1):15–28.

Article  PubMed  Google Scholar 

Mueller J, Amortization of Certain Intangible Assets Journal of Accountancy. 2004. https://www.journalofaccountancy.com/issues/2004/dec/amortizationofcertainintangibleassets.html. Accesed 04 April 2023.

Papanikolaou E, Bosio A. The Promise and the Hope of Gene Therapy. Front Genome Ed. 2021;3:618346.

Article  PubMed  PubMed Central  Google Scholar 

Moon S, Erickson E. Universal Medicine Access through Lump-Sum Remuneration — Australia’s Approach to Hepatitis C. 2019;380(7):607–10.

Matthews DW, Coleman S, Razavi H, Izaret J-M. The payer license agreement, or Netflix Model, for Hepatitis C Virus therapies enables Universal Treatment Access. Lowers Costs Incentivizes Innov Competition. 2022;42(7):1503–16.

Google Scholar 

Gene Therapy’s Next Installment. Nat Biotechnol. 2019;37(7):697.

Article  Google Scholar 

Mahase E. Uk launches subscription style model for antibiotics to encourage New Development. J BMJ. 2020;369:m2468.

Article  Google Scholar 

Barlow E, Morton A, Megiddo I, Colson A. Optimal subscription models to pay for antibiotics. Soc Sci Med. 2022;298:114818.

Article  PubMed  PubMed Central  Google Scholar 

Espinoza MA, Manca A, Claxton K, Sculpher MJ. The value of heterogeneity for cost-effectiveness subgroup analysis: conceptual Framework and Application. Med Decis Mak. 2014;34(8):951–64.

Article  Google Scholar 

Espinoza M, Manca A, Sculpher M, Claxton K. Co1 individual decisions and Social Value: a conceptual Framework to explore alternative decision making approaches and the value of heterogeneity in the era of Individualized Care. Value Health. 2012;15:A2.

Article  Google Scholar 

Espinoza MA, Sculpher MJ, Manca A, Basu A. Analysing heterogeneity to support decision making. In: Culyer AJ, editor. Encyclopedia of Health Economics. San Diego: Elsevier; 2014. pp. 71–6.

Chapter  Google Scholar 

Dabbous M, Chachoua L, Caban A, Toumi M. Managed Entry agreements: policy analysis from the European perspective. Value Health. 2020;23(4):425–33.

Article  PubMed  Google Scholar 

Faulkner A, Mahalatchimy A. The politics of valuation and payment for Regenerative Medicine products in the Uk. New Genet Soc. 2018;37(3):227–47.

Article  Google Scholar 

Michelsen S, Nachi S, Van Dyck W, Simoens S, Huys I. Barriers and opportunities for implementation of outcome-based spread payments for High-Cost, one-shot curative therapies. Front Pharmacol. 2020;11:594446.

Article  PubMed  PubMed Central  Google Scholar 

Simoens S, De Groote K, Boersma C. Critical reflections on reimbursement and Access of Advanced therapies. Front Pharmacol. 2022;13:771966.

Article  PubMed  PubMed Central  Google Scholar 

Clopés Estela A, Soler Rotllant F, Germà Lluch JR, Calle Rodríguez C. Pay-for-performance schemes: 10 years’ experience in a Comprehensive Cancer Center. Med Clínica (English Edition). 2022;158(10):488–92.

Article  Google Scholar 

Ministerio de Sanidad, Preguntas Y. Secretaría general de Sanidad y Consumo. Dirección general de cartera básica de servicios del SNS y farmacia: Ministerio de Sanidad; 2019. Respuestas Frecuentes Sobre El Sistema De Información Para Determinar El Valor Terapéutico En La Práctica Clínica Real De Los Medicamentos De Alto Impacto Sanitario Y Económico En El Sistema Nacional De Salud (Valtermed).

Diario Farma, Valtermed. La Conexión Y El Registro De Resultados Clínicos Ya Es Posible Diario Farma; 2019. https://diariofarma.com/2019/07/22/valtermed-la-conexion-y-el-registro-de-resultados-clinicos-ya-es-posible. Accesed 05 April 2023.

Garattini L, Curto A, van de Vooren K. Italian risk-sharing agreements on drugs: are they worthwhile? Eur J Health Econ. 2015;16(1):1–3.

Article  PubMed  Google Scholar 

Navarria A, Drago V, Gozzo L, Longo L, Mansueto S, Pignataro G, et al. Do the current performance-based schemes in Italy really work? Success Fee: a Novel measure for cost-Containment of Drug Expenditure. Value Health. 2015;18(1):131–6.

Article  PubMed  Google Scholar 

Boletín Oficial de la República Argentina. Legislación Y Avisos Oficiales. Ministerio De Salud Subsecretaría De Medicamentos E Información Estratégica Argentina Presidencia. 2023. https://www.boletinoficial.gob.ar/detalleAviso/primera/279782/20230116. Accesed 02 May 2023.

Ministerio de Salud. El

留言 (0)

沒有登入
gif