{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,5,8]],"date-time":"2026-05-08T09:11:24Z","timestamp":1778231484365,"version":"3.51.4"},"reference-count":25,"publisher":"Springer Science and Business Media LLC","issue":"5","license":[{"start":{"date-parts":[[2026,3,24]],"date-time":"2026-03-24T00:00:00Z","timestamp":1774310400000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by-nc\/4.0"},{"start":{"date-parts":[[2026,3,24]],"date-time":"2026-03-24T00:00:00Z","timestamp":1774310400000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by-nc\/4.0"}],"funder":[{"DOI":"10.13039\/100008484","name":"BioMarin Pharmaceutical","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100008484","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["link.springer.com"],"crossmark-restriction":false},"short-container-title":["Adv Ther"],"published-print":{"date-parts":[[2026,5]]},"DOI":"10.1007\/s12325-026-03556-w","type":"journal-article","created":{"date-parts":[[2026,3,24]],"date-time":"2026-03-24T06:53:45Z","timestamp":1774335225000},"page":"2254-2267","update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":0,"title":["Estimating the Impact of Hemophilia\u00a0A Treatment Administration on Patient Health Utility: Combining a Discrete Choice Experiment with Time Trade-Off Method"],"prefix":"10.1007","volume":"43","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-9904-039X","authenticated-orcid":false,"given":"Melody S.","family":"Benton","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Dongzhe","family":"Hong","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Mark W.","family":"Skinner","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Louis P.","family":"Garrison","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Milad","family":"Karimi","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Er","family":"Chen","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Henry","family":"Mead","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Lizheng","family":"Shi","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"297","published-online":{"date-parts":[[2026,3,24]]},"reference":[{"issue":"23","key":"3556_CR1","doi-asserted-by":"publisher","first-page":"1773","DOI":"10.1056\/NEJM200106073442307","volume":"344","author":"PM Mannucci","year":"2001","unstructured":"Mannucci PM, Tuddenham EG. The hemophilias - from royal genes to gene therapy. N Engl J Med. 2001;344(23):1773\u20139.","journal-title":"N Engl J Med"},{"issue":"6","key":"3556_CR2","doi-asserted-by":"publisher","first-page":"1114","DOI":"10.1160\/TH13-05-0363","volume":"110","author":"M Franchini","year":"2013","unstructured":"Franchini M, Mannucci PM. Acquired haemophilia a: a 2013 update. Thromb Haemost. 2013;110(6):1114\u201320.","journal-title":"Thromb Haemost"},{"key":"3556_CR3","unstructured":"Center for Disease Control and Prevention. What is hemophilia? Edition: National Center on Birth Defects and Developmental Disabilities. Last reviewed October 24, 2023. https:\/\/www.cdc.gov\/ncbddd\/hemophilia\/facts.html. Accessed 15 Feb 2021."},{"key":"3556_CR4","first-page":"33","volume":"7","author":"M Franchini","year":"2013","unstructured":"Franchini M, Frattini F, Crestani S, Sissa C, Bonfanti C. Treatment of hemophilia b: focus on recombinant factor IX. Biologics. 2013;7:33\u20138.","journal-title":"Biologics"},{"key":"3556_CR5","doi-asserted-by":"publisher","first-page":"1","DOI":"10.1111\/hae.14046","volume":"26","author":"A Srivastava","year":"2020","unstructured":"Srivastava A, Santagostino E, Dougall A, et al. WFH guidelines for the management of hemophilia, 3rd edition. Haemophilia. 2020;26:1\u2013158.","journal-title":"Haemophilia"},{"issue":"17-18","key":"3556_CR6","doi-asserted-by":"publisher","first-page":"782","DOI":"10.1089\/hum.2023.112","volume":"34","author":"D De Wolf","year":"2023","unstructured":"De Wolf D, Singh K, Chuah M, VandenDriessche T. Hemophilia gene therapy: the end of the beginning? Hum Gene Ther. 2023;34(17\u201318):782\u201392.","journal-title":"Hum Gene Ther"},{"key":"3556_CR7","unstructured":"US Food and Drug Administration. FDA approves first gene therapy for adults with severe hemophilia\u00a0A. June 29, 2023. https:\/\/www.fda.gov\/news-events\/press-announcements\/fda-approves-first-gene-therapy-adults-severe-hemophilia. Accessed 16 Aug 2023."},{"issue":"8","key":"3556_CR8","doi-asserted-by":"publisher","DOI":"10.1016\/j.rpth.2024.102615","volume":"8","author":"AD Leavitt","year":"2024","unstructured":"Leavitt AD, Mahlangu J, Raheja P, et al. Efficacy, safety, and quality of life 4\u00a0years after valoctocogene roxaparvovec gene transfer for severe hemophilia a in the phase\u00a03 GENEr8-1 trial. Res Pract Thromb Haemost. 2024;8(8):102615.","journal-title":"Res Pract Thromb Haemost"},{"issue":"5","key":"3556_CR9","doi-asserted-by":"publisher","first-page":"1138","DOI":"10.1111\/hae.15071","volume":"30","author":"E Symington","year":"2024","unstructured":"Symington E, Rangarajan S, Lester W, et al. Valoctocogene roxaparvovec gene therapy provides durable haemostatic control for up to 7\u00a0years for haemophilia\u00a0A. Haemophilia. 2024;30(5):1138\u201347.","journal-title":"Haemophilia"},{"issue":"4","key":"3556_CR10","doi-asserted-by":"publisher","first-page":"403","DOI":"10.1016\/j.jval.2010.11.013","volume":"14","author":"JF Bridges","year":"2011","unstructured":"Bridges JF, Hauber AB, Marshall D, et al. Conjoint analysis applications in health - a checklist: a report of the ISPOR Good Research Practices for Conjoint Analysis Task Force. Value Health. 2011;14(4):403\u201313.","journal-title":"Value Health"},{"issue":"4","key":"3556_CR11","doi-asserted-by":"publisher","first-page":"e167","DOI":"10.1111\/hae.13504","volume":"24","author":"A Iorio","year":"2018","unstructured":"Iorio A, Skinner MW, Clearfield E, et al. Core outcome set for gene therapy in haemophilia: results of the coreHEM multistakeholder project. Haemophilia. 2018;24(4):e167\u201372.","journal-title":"Haemophilia"},{"issue":"Suppl 1","key":"3556_CR12","doi-asserted-by":"publisher","first-page":"S53","DOI":"10.1007\/s10198-013-0508-x","volume":"14","author":"AE Attema","year":"2013","unstructured":"Attema AE, Edelaar-Peeters Y, Versteegh MM, Stolk EA. Time trade-off: one methodology, different methods. Eur J Health Econ. 2013;14(Suppl 1):S53\u201364.","journal-title":"Eur J Health Econ"},{"key":"3556_CR13","doi-asserted-by":"publisher","first-page":"1407","DOI":"10.2147\/PPA.S316276","volume":"15","author":"K Johnston","year":"2021","unstructured":"Johnston K, Stoffman JM, Mickle AT, et al. Preferences and health-related quality-of-life related to disease and treatment features for patients with hemophilia\u00a0A in a Canadian general population sample. Patient Prefer Adherence. 2021;15:1407\u201317.","journal-title":"Patient Prefer Adherence"},{"issue":"1","key":"3556_CR14","doi-asserted-by":"publisher","first-page":"17","DOI":"10.1186\/s41687-023-00550-6","volume":"7","author":"M Brod","year":"2023","unstructured":"Brod M, Bushnell DM, Neergaard JS, Saldman LT, Busk AK. Understanding treatment burden in hemophilia: development and validation of the Hemophilia Treatment Experience Measure (Hemo-TEM). J Patient Rep Outcomes. 2023;7(1):17.","journal-title":"J Patient Rep Outcomes"},{"issue":"5","key":"3556_CR15","doi-asserted-by":"publisher","first-page":"769","DOI":"10.1111\/hae.14383","volume":"27","author":"M Witkop","year":"2021","unstructured":"Witkop M, Morgan G, O\u2019Hara J, et al. Patient preferences and priorities for haemophilia gene therapy in the US: a discrete choice experiment. Haemophilia. 2021;27(5):769\u201382.","journal-title":"Haemophilia"},{"issue":"100","key":"3556_CR16","doi-asserted-by":"publisher","first-page":"38","DOI":"10.1016\/j.socscimed.2014.05.026","volume":"114","author":"N Bansback","year":"2014","unstructured":"Bansback N, Hole AR, Mulhern B, Tsuchiya A. Testing a discrete choice experiment including duration to value health states for large descriptive systems: addressing design and sampling issues. Soc Sci Med. 2014;114(100):38\u201348.","journal-title":"Soc Sci Med"},{"issue":"10","key":"3556_CR17","doi-asserted-by":"publisher","first-page":"1499","DOI":"10.1016\/j.jval.2021.03.007","volume":"24","author":"D Rowen","year":"2021","unstructured":"Rowen D, Powell P, Mukuria C, Carlton J, Norman R, Brazier J. Deriving a preference-based measure for people with Duchenne muscular dystrophy from the DMD-QoL. Value Health. 2021;24(10):1499\u2013510.","journal-title":"Value Health"},{"issue":"6","key":"3556_CR18","doi-asserted-by":"publisher","first-page":"812","DOI":"10.1016\/j.jval.2020.12.017","volume":"24","author":"LS Matza","year":"2021","unstructured":"Matza LS, Stewart KD, Lloyd AJ, Rowen D, Brazier JE. Vignette-based utilities: usefulness, limitations, and methodological recommendations. Value Health. 2021;24(6):812\u201321.","journal-title":"Value Health"},{"issue":"2","key":"3556_CR19","doi-asserted-by":"publisher","first-page":"245","DOI":"10.1111\/hae.14278","volume":"27","author":"J O'Hara","year":"2021","unstructured":"O\u2019Hara J, Martin AP, Nugent D, et al. Evidence of a disability paradox in patient-reported outcomes in haemophilia. Haemophilia. 2021;27(2):245\u201352.","journal-title":"Haemophilia"},{"key":"3556_CR20","unstructured":"Tice JA, Walton SM, Herce-Hagiwara B, et al. Gene therapy for hemophilia B and an update on gene therapy for hemophilia\u00a0A: effectiveness and value, Final Evidence Report. December 22, 2022. Institute for Clinical and Economic Review (ICER). https:\/\/icer.org\/assessment\/hemophilia-a-and-b-2022\/. Accessed 19 Mar 2024."},{"issue":"11","key":"3556_CR21","doi-asserted-by":"publisher","first-page":"1628","DOI":"10.1016\/j.jval.2021.05.008","volume":"24","author":"LP Garrison","year":"2021","unstructured":"Garrison LP, Pezalla E, Towse A, et al. Hemophilia gene therapy value assessment: methodological challenges and recommendations. Value Health. 2021;24(11):1628\u201333.","journal-title":"Value Health"},{"issue":"3","key":"3556_CR22","first-page":"316","volume":"27","author":"SE Croteau","year":"2021","unstructured":"Croteau SE, Cook K, Sheikh L, et al. Health care resource utilization and costs among adult patients with hemophilia\u00a0A on factor\u00a0VIII prophylaxis: an administrative claims analysis. J Manag Care Spec Pharm. 2021;27(3):316\u201326.","journal-title":"J Manag Care Spec Pharm"},{"issue":"4","key":"3556_CR23","first-page":"449","volume":"28","author":"CD Thornburg","year":"2022","unstructured":"Thornburg CD, Adamski K, Cook K, et al. Health care costs and resource utilization among commercially insured adult patients with hemophilia\u00a0A managed with FVIII prophylaxis in the United States. J Manag Care Spec Pharm. 2022;28(4):449\u201360.","journal-title":"J Manag Care Spec Pharm"},{"issue":"5","key":"3556_CR24","first-page":"674","volume":"27","author":"LP Garrison","year":"2021","unstructured":"Garrison LP, Juao B, Dabbous O. Gene therapy may not be as expensive as people think: challenges in assessing the value of single and short-term therapies. J Manag Care Spec Pharm. 2021;27(5):674\u201381.","journal-title":"J Manag Care Spec Pharm"},{"key":"3556_CR25","doi-asserted-by":"publisher","first-page":"5781","DOI":"10.1007\/s12325-025-03368-4","volume":"42","author":"S Santos","year":"2025","unstructured":"Santos S, Robinson TM, Trueman D. Estimated long-term durability of valoctocogene roxaparvovec treatment in male patients with severe hemophilia\u00a0A: an extrapolation of clinical data. Adv Ther. 2025;42:5781\u201393.","journal-title":"Adv Ther"}],"container-title":["Advances in Therapy"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/link.springer.com\/content\/pdf\/10.1007\/s12325-026-03556-w.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/link.springer.com\/article\/10.1007\/s12325-026-03556-w","content-type":"text\/html","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/link.springer.com\/content\/pdf\/10.1007\/s12325-026-03556-w.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2026,5,8]],"date-time":"2026-05-08T08:37:52Z","timestamp":1778229472000},"score":1,"resource":{"primary":{"URL":"https:\/\/link.springer.com\/10.1007\/s12325-026-03556-w"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2026,3,24]]},"references-count":25,"journal-issue":{"issue":"5","published-print":{"date-parts":[[2026,5]]}},"alternative-id":["3556"],"URL":"https:\/\/doi.org\/10.1007\/s12325-026-03556-w","relation":{},"ISSN":["0741-238X","1865-8652"],"issn-type":[{"value":"0741-238X","type":"print"},{"value":"1865-8652","type":"electronic"}],"subject":[],"published":{"date-parts":[[2026,3,24]]},"assertion":[{"value":"16 November 2025","order":1,"name":"received","label":"Received","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"27 February 2026","order":2,"name":"accepted","label":"Accepted","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"24 March 2026","order":3,"name":"first_online","label":"First Online","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"Melody S. Benton is principal investigator and lead researcher for the study; the project is sponsored\/funded by BioMarin. Dongzhe Hong declares that he has no competing interests. Mark W. Skinner\u2019s institution has received independent research funding from Bayer, BioMarin, Chugai, CSL Behring, Roche, Sanofi, Spark, and Takeda. His institution has received fees or honoraria from Bayer, BioMarin, Genentech, Novo Nordisk, Pfizer, Sanofi, Spark, and Takeda. Lizheng Shi is principal investigator for the project sponsored by BioMarin. Er Chen is an employee and stockholder of BioMarin. Louis P. Garrison did not receive funding for the research carried out in this work. However, he has received honoraria from BioMarin, serving on general advisory board on projects in development. He has also received funding from Pfizer and uniQure to speak on hemophilia, and he has been a consultant to numerous other life sciences companies, including Gilead, Roche GSK, BMS, Merck, and others. Milad Karimi is an employee and stockholder of BioMarin. Henry Mead was an employee and stockholder of BioMarin at the time of the study; he is now a consultant for BioBridges.","order":1,"name":"Ethics","group":{"name":"EthicsHeading","label":"Conflict of Interest"}},{"value":"Tulane University\u2019s Human Research Protection office conducted an expedited review and approved the study protocol in accordance with appropriate research regulations. The study was conducted in accordance with the ethical standards of the 1964 Declaration of Helsinki and its later amendments.","order":2,"name":"Ethics","group":{"name":"EthicsHeading","label":"Ethical Approval"}},{"value":"The authors would like to thank all patients who participated in this study. Their contributions were essential to the success of this research.","order":3,"name":"Ethics","group":{"name":"EthicsHeading","label":"Patient Participants"}}]}}