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Journal of Market Access & Health Policy Volume 8, 2020 - Issue 1
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Guideline

Health economic evaluation of gene replacement therapies: methodological issues and recommendations

Samuel Aballéaa Creativ-Ceutical, HEOR, Rotterdam, NetherlandsCorrespondence[email protected]
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Katia Thokagevistkb Creativ-Ceutical, HEOR, Paris, FranceView further author information
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Rimma Velikanovac Groningen Research Institute of Pharmacy, PharmacoTherapy, Epidemiology & Economics, University of Groningen, Groningen, NetherlandsView further author information
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Steven Simoensd Department of Pharmaceutical and Pharmacological Sciences, KU Leuven, Leuven, BelgiumView further author information
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Lieven Annemanse Department of Public Health, Ghent University, Ghent, BelgiumView further author information
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Fernando Antonanzasf Department of Economics, University of La Rioja, Logroño, SpainView further author information
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Pascal Auquierg CEReSS - Health Service Research and Quality of Life Center, Aix-Marseille University, Marseille, FranceView further author information
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Clément Françoisb Creativ-Ceutical, HEOR, Paris, France;h Public Health Department - Research Unit, Aix-Marseille University, Marseille, FranceView further author information
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Frank-Ulrich Frickei Technische Hochschule Nürnberg, Nürnberg, GermanyView further author information
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Daniel Malonej Pharmacotherapy Faculty, College of Pharmacy, University of Utah, Salt Lake City, USAView further author information
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Aurélie Millierb Creativ-Ceutical, HEOR, Paris, FranceView further author information
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Ulf Perssonk The Swedish Institute for Health Economics (IHE), Lund, SwedenView further author information
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Stavros Petroul Nuffield Department of Primary Care Health Sciences, University of Oxford, Oxford, UKView further author information
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Omar Dabbousm Global Health Economics and Outcomes Research and Real World EvidenceAveXis Inc, Novartis Gene Therapies, Bannockburn, IL, USAView further author information
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Maarten Postmac Groningen Research Institute of Pharmacy, PharmacoTherapy, Epidemiology & Economics, University of Groningen, Groningen, NetherlandsORCID Iconhttps://orcid.org/0000-0002-6306-3653View further author information
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Mondher Toumib Creativ-Ceutical, HEOR, Paris, France;h Public Health Department - Research Unit, Aix-Marseille University, Marseille, FranceORCID Iconhttps://orcid.org/0000-0001-7939-7204View further author information
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Article: 1822666 | Received 20 Mar 2020, Accepted 04 Sep 2020, Published online: 11 Oct 2020

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Alexa C. Klimchak, Lauren E. Sedita, Katherine L. Gooch & Daniel C. Malone. (2023) Assessing the impact of single or short-term administration on a therapy’s cost-effectiveness: a hypothetical disease-agnostic model. Journal of Medical Economics 26:1, pages 594-602.
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Mondher Toumi, Omar Dabbous, Samuel Aballéa, Michael F. Drummond, Johann-Matthias Graf von der Schulenburg, Daniel C. Malone, Peter J. Neumann, Sean D. Sullivan & Sean Tunis. (2023) Recommendations for economic evaluations of cell and gene therapies: a systematic literature review with critical appraisal. Expert Review of Pharmacoeconomics & Outcomes Research 23:5, pages 483-497.
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Hugh J. McMillan, Crystal M. Proud, Michelle A. Farrar, Ian E. Alexander, Francesco Muntoni & Laurent Servais. (2022) Onasemnogene abeparvovec for the treatment of spinal muscular atrophy. Expert Opinion on Biological Therapy 22:9, pages 1075-1090.
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Michał Pochopień, Tingting Qiu, Samuel Aballea, Emilie Clay & Mondher Toumi. (2021) Considering potential solutions for limitations and challenges in the health economic evaluation of gene therapies. Expert Review of Pharmacoeconomics & Outcomes Research 21:6, pages 1145-1158.
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Articles from other publishers (13)

Buthainah Ghanem, Enrique Seoane-Vazquez, Lawrence Brown & Rosa Rodriguez-Monguio. (2023) Analysis of the Gene Therapies Authorized by the United States Food and Drug Administration and the European Medicines Agency. Medical Care 61:7, pages 438-447.
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Sean P. Gavan, Stuart J. Wright, Fiona Thistlethwaite & Katherine Payne. (2023) Capturing the Impact of Constraints on the Cost-Effectiveness of Cell and Gene Therapies: A Systematic Review. PharmacoEconomics 41:6, pages 675-692.
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Michael Drummond, Oriana Ciani, Giulia Fornaro, Claudio Jommi, Eva Susanne Dietrich, Jaime Espin, Jean Mossman & Gerard de Pouvourville. (2023) How are health technology assessment bodies responding to the assessment challenges posed by cell and gene therapy?. BMC Health Services Research 23:1.
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Louis P. GarrisonJrJr, Andrew W. Lo, Richard S. Finkel & Patricia A. Deverka. (2023) A review of economic issues for gene‐targeted therapies: Value, affordability, and access . American Journal of Medical Genetics Part C: Seminars in Medical Genetics 193:1, pages 64-76.
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Erik Landfeldt. (2022) Gene Therapy for Neuromuscular Diseases: Health Economic Challenges and Future Perspectives. Journal of Neuromuscular Diseases 9:6, pages 675-688.
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Steven Simoens, Katrien De Groote & Cornelis Boersma. (2022) Critical Reflections on Reimbursement and Access of Advanced Therapies. Frontiers in Pharmacology 13.
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Min Yang, Hiroyuki Awano, Satoru Tanaka, Walter Toro, Su Zhang, Omar Dabbous & Ataru Igarashi. (2022) Systematic Literature Review of Clinical and Economic Evidence for Spinal Muscular Atrophy. Advances in Therapy 39:5, pages 1915-1958.
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Tingting Qiu, Michał Pochopień, Eve Hanna, Shuyao Liang, Yitong Wang, Ru Han, Mondher Toumi & Samuel Aballéa. (2022) Challenges in the market access of regenerative medicines, and implications for manufacturers and decision-makers: a systematic review. Regenerative Medicine 17:3, pages 119-139.
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Tingting Qiu, Michal Pochopien, Shuyao Liang, Gauri Saal, Ewelina Paterak, Justyna Janik & Mondher Toumi. (2022) Gene Therapy Evidence Generation and Economic Analysis: Pragmatic Considerations to Facilitate Fit-for-Purpose Health Technology Assessment. Frontiers in Public Health 10.
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Shunsuke Takada & Mamoru Narukawa. (2021) Medicine characteristics affecting the time to guidance publication by National Institute for Health and Care Excellence in the single technology appraisal process. International Journal of Technology Assessment in Health Care 38:1.
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Tingting Qiu, Yitong Wang, Shuyao Liang, Ru Han & Mondher Toumi. (2021) The impact of COVID-19 on the cell and gene therapies industry: Disruptions, opportunities, and future prospects. Drug Discovery Today 26:10, pages 2269-2281.
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Maria Kamusheva, Adina Turcu-Stiolica, Jakub Gierczyński, Mihaela-Simona Subtirelu, Marcin Czech & Guenka Petrova. (2021) Do Advanced Therapies Have a Future in the Low- and Middle-Income Countries - The Case of Bulgaria, Romania, and Poland. Frontiers in Public Health 9.
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Tingting Qiu, Yitong Wang, Shuyao Liang, Ru Han & Mondher Toumi. (2021) Partnership agreements for regenerative medicines: a database analysis and implications for future innovation. Regenerative Medicine 16:8, pages 733-755.
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