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Gerardus WJ Frederix & Renske MT ten Ham. (2023) Gene therapies, uncertainty, and decision-making: thinking about the last mile at the first step. Expert Review of Pharmacoeconomics & Outcomes Research 23:8, pages 853-856.
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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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Articles from other publishers (28)
Deborah A. Marshall, Brittany Gerber, Diane L. Lorenzetti, Karen V. MacDonald, Riley Jewel Bohach & Gillian R. Currie. (2023) Are We Capturing the Socioeconomic Burden of Rare Genetic Disease? A Scoping Review of Economic Evaluations and Cost-of-Illness Studies. PharmacoEconomics 41:12, pages 1563-1588.
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Antal Zemplenyi, Jim Leonard, Michael J. DiStefano, Kelly E. Anderson, Garth C. Wright, Nicholas D. Mendola, Kavita Nair & R. Brett McQueen. (2023) Using Real-World Data to Inform Value-Based Contracts for Cell and Gene Therapies in Medicaid. PharmacoEconomics.
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Chi Heem Wong, Dexin Li, Nina Wang, Jonathan Gruber, Andrew W. Lo & Rena M. Conti. (2023) The estimated annual financial impact of gene therapy in the United States. Gene Therapy 30:10-11, pages 761-773.
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Giovanni Di Minno, Gaia Spadarella, Nelson Mauro Maldonato, Natascia De Lucia, Giancarlo Castaman, Raimondo De Cristofaro, Cristina Santoro, Flora Peyvandi, Anna Borrelli, Angelo Lupi, Marco Follino, Gerardo Guerrino, Filomena Morisco & Matteo Di Minno. (2023) Awareness of individual goals, preferences, and priorities of persons with severe congenital haemophilia A for a tailored shared decision-making approach to liver-directed gene therapy. A practical guideline. Blood Reviews 62, pages 101118.
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Amitava Mitra, Mariam A. Ahmed, Rajesh Krishna, Kefeng Sun, Francis D. Gibbons, Olivia Campagne, Noha Rayad, Youssef M. Roman, Salwa Albusaysi, Maria Burian & Islam R. Younis. (2023) Model‐Informed Approaches and Innovative Clinical Trial Design for Adeno‐Associated Viral Vector‐Based Gene Therapy Product Development: A White Paper. Clinical Pharmacology & Therapeutics 114:3, pages 515-529.
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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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Kazuya Sakai & Masanori Matsumoto. (2023) Clinical Manifestations, Current and Future Therapy, and Long-Term Outcomes in Congenital Thrombotic Thrombocytopenic Purpura. Journal of Clinical Medicine 12:10, pages 3365.
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Antonio Olry de Labry-Lima, Angela Ponce-Polo, Leticia García-Mochón, Marta Ortega-Ortega, Daniel Pérez-Troncoso & David Epstein. (2023) Challenges for Economic Evaluations of Advanced Therapy Medicinal Products: A Systematic Review. Value in Health 26:1, pages 138-150.
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Gabriela G Yamaguti-Hayakawa & Margareth C Ozelo. (2023) Gene therapy for hemophilia: looking beyond factor expression. Experimental Biology and Medicine 247:24, pages 2223-2232.
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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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Ulrike M. Reiss, Johnny Mahlangu, Tsukasa Ohmori, Margareth C. Ozelo, Alok Srivastava & Lei Zhang. (2022) Haemophilia gene therapy—Update on new country initiatives. Haemophilia 28:S4, pages 61-67.
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Courtney D ThornburgKelly AdamskiKeziah CookMohini VembusubramanianSelvam R SendhilDavid HindsEr ChenJoshua SammonPaul SolariLouis P GarrisonJrJrStacy E Croteau. (2022) Health care costs and resource utilization among commercially insured adult patients with hemophilia A managed with FVIII prophylaxis in the United States. Journal of Managed Care & Specialty Pharmacy 28:4, pages 449-460.
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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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Louis P. Garrison & Diane Kleinermans. (2022) Is the world ready for gene therapy?. Haemophilia 28:S2, pages 5-8.
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Jamie O'Hara & Peter J. Neumann. (2022) Health technology assessment for gene therapies in haemophilia. Haemophilia 28:S2, pages 19-26.
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Chunla HeDavid HindsEdmund PezallaDunlei ChengEr ChenJoshua SammonPaul G SolariMichael Recht. (2022) Health insurance coverage and switching among people with hemophilia A in the United States. Journal of Managed Care & Specialty Pharmacy 28:2, pages 232-243.
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Renske M.T. ten Ham, Simon M. Walker, Marta O. Soares, Geert W.J. Frederix, Frank W.G. Leebeek, Kathelijn Fischer, Michiel Coppens & Stephen J. Palmer. (2022) Modeling Benefits, Costs, and Affordability of a Novel Gene Therapy in Hemophilia A. HemaSphere 6:2, pages e679.
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Ash Bullement, Emma S. Knowles, Pronabesh DasMahapatra, Talaha Ali & Ron Preblick. (2021) Cost-Effectiveness Analysis of rFVIIIFc Versus Contemporary rFVIII Treatments for Patients with Severe Hemophilia A Without Inhibitors in the United States. PharmacoEconomics - Open 5:4, pages 625-633.
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Nancy S. Bolous, Yichen Chen, Huiqi Wang, Andrew M. Davidoff, Meenakshi Devidas, Timothy W. Jacobs, Michael M. MeagherAmit C. Nathwani, Ellis J. Neufeld, Bryan A. Piras, Carlos Rodriguez-Galindo, Ulrike M. Reiss & Nickhill Bhakta. (2021) The cost-effectiveness of gene therapy for severe hemophilia B: a microsimulation study from the United States perspective. Blood 138:18, pages 1677-1690.
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Joseph Khoa Ho, Kennedy Borle, Nick Dragojlovic, Manrubby Dhillon, Vanessa Kitchin, Nicola Kopac, Colin Ross & Larry D. Lynd. (2021) Economic Evidence on Potentially Curative Gene Therapy Products: A Systematic Literature Review. PharmacoEconomics 39:9, pages 995-1019.
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Inmaculada Hernandez, Deanna Rowe, Walid F Gellad & Chester B. Good. (2021) Trends in the Use of Conventional and New Pharmaceuticals for Hemophilia Treatments Among Medicaid Enrollees, 2005-2020. JAMA Network Open 4:6, pages e2112044.
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Louis P GarrisonJrJrBoshen JiaoOmar Dabbous. (2021) Gene therapy may not be as expensive as people think: challenges in assessing the value of single and short-term therapies. Journal of Managed Care & Specialty Pharmacy 27:5, pages 674-681.
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Tyler W. Buckner, Iryna Bocharova, Kaitlin Hagan, Arielle G. Bensimon, Hongbo Yang, Eric Q. Wu, Eileen K. Sawyer & Nanxin Li. (2021) Health care resource utilization and cost burden of hemophilia B in the United States. Blood Advances 5:7, pages 1954-1962.
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Saskia Hendriks & Steven D Pearson. (2021) Assessing potential cures: are there distinctive elements of value beyond health gain?. Journal of Comparative Effectiveness Research 10:4, pages 255-265.
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Paul BattyDavid Lillicrap. (2021) Hemophilia Gene Therapy: Approaching the First Licensed Product. HemaSphere 5:3, pages e540.
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Ulrike M. Reiss, Lei Zhang & Tsukasa Ohmori. (2020) Hemophilia gene therapy—New country initiatives. Haemophilia 27:S3, pages 132-141.
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Declan Noone, Donna Coffin & Glenn F. Pierce. (2020) Reimbursing the value of gene therapy care in an era of uncertainty. Haemophilia 27:1, pages 12-18.
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