{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,3,17]],"date-time":"2026-03-17T05:57:48Z","timestamp":1773727068769,"version":"3.50.1"},"reference-count":29,"publisher":"Wiley","issue":"6","license":[{"start":{"date-parts":[[2023,2,23]],"date-time":"2023-02-23T00:00:00Z","timestamp":1677110400000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Pediatric Blood &amp; Cancer"],"published-print":{"date-parts":[[2023,6]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Objective<\/jats:title><jats:p>To evaluate the lifetime cost\u2010effectiveness of recombinant factor IX Fc fusion protein (rFIXFc) and recombinant factor IX (rFIX) for the treatment of hemophilia B (HB) in China.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>We developed a decision\u2010analytic Markov model including three health states: alive, requiring surgery, and dead. This model estimated the lifetime cost and quality\u2010adjusted life\u2010years (QALYs) of prophylaxis in childhood, followed by on\u2010demand treatment in adulthood for moderate\u2013severe to severe HB patients from China's healthcare system perspective. Efficacy data derived from pivotal clinical trials, clinical guideline recommendations, and expert consultation were applied to two scenarios (full dose and low dose). One\u2010way sensitivity analysis and probabilistic sensitivity analysis (PSA) were performed to assess the robustness of the model.<\/jats:p><\/jats:sec><jats:sec><jats:title>Outcomes<\/jats:title><jats:p>Lifetime cost, QALYs, and the incremental cost\u2010effectiveness ratio were calculated, and the results were compared with willingness\u2010to\u2010pay (WTP) thresholds of one to three times the gross domestic product per capita of China in 2021 ($12,551\u2013$37,653).<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>RFIXFc was associated with lower cost and more QALYs than rFIX in both scenarios, which suggested that it is a dominant strategy (more effective and cheaper) for moderate\u2013severe to severe HB in China. In the full\u2010dose scenario, rFIXFc saved more money and yielded more QALYs than in the low\u2010dose scenario (low doses are the typical clinical reality in China). PSA demonstrated that rFIXFc had an over 90% probability of being cost\u2010effective with full\u2010dose and low\u2010dose treatment at WTP thresholds of $12,551\u2013$37,653.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusions<\/jats:title><jats:p>Compared with rFIX, rFIXFc appears to be a cost\u2010effective option for the lifetime management of moderate\u2013severe to severe HB patients in China.<\/jats:p><\/jats:sec>","DOI":"10.1002\/pbc.30264","type":"journal-article","created":{"date-parts":[[2023,2,23]],"date-time":"2023-02-23T11:13:18Z","timestamp":1677150798000},"update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":7,"title":["Cost\u2010effectiveness analysis of recombinant factor IX Fc fusion protein compared with recombinant factor IX for the treatment of moderate\u2013severe to severe hemophilia B in China"],"prefix":"10.1002","volume":"70","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-0049-4595","authenticated-orcid":false,"given":"Ting","family":"Zhou","sequence":"first","affiliation":[{"name":"School of International Pharmaceutical Business China Pharmaceutical University  Nanjing China"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Shuyue","family":"Wang","sequence":"additional","affiliation":[{"name":"Public Affairs, Market Access, and Trade, Sanofi China Inc.  Beijing China"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Yao","family":"Zhang","sequence":"additional","affiliation":[{"name":"School of International Pharmaceutical Business China Pharmaceutical University  Nanjing China"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-4030-209X","authenticated-orcid":false,"given":"Runhui","family":"Wu","sequence":"additional","affiliation":[{"name":"Hematology Oncology Center Beijing Children's Hospital, Capital Medical University, National Children's Health Center  Beijing China"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Hongchao","family":"Li","sequence":"additional","affiliation":[{"name":"School of International Pharmaceutical Business China Pharmaceutical University  Nanjing China"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"311","published-online":{"date-parts":[[2023,2,23]]},"reference":[{"key":"e_1_2_11_2_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.14046"},{"key":"e_1_2_11_3_1","unstructured":"European Medicines Agency.Guideline on clinical investigation of recombinant and human plasma\u2010derived factor IX products.EMA. Accessed August 2 2021.https:\/\/www.ema.europa.eu\/en\/documents\/scientific\u2010guideline\/guideline\u2010clinical\u2010investigation\u2010recombinant\u2010human\u2010plasma\u2010derived\u2010factor\u2010ix\u2010products_en.pdf"},{"issue":"4","key":"e_1_2_11_4_1","first-page":"265","article-title":"Chinese guidelines on the treatment of hemophilia (version 2020)","volume":"41","author":"Thrombosis and Hemostasis Group","year":"2020","journal-title":"Chin J Hematol"},{"key":"e_1_2_11_5_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1365\u20102516.2010.02348.x"},{"issue":"5","key":"e_1_2_11_6_1","first-page":"364","article-title":"Consensus of Chinese expert on the diagnosis and treatment of hemophilia (version 2017)","volume":"38","author":"Thrombosis and Hemostasis Group","year":"2017","journal-title":"Chin J Hematol"},{"key":"e_1_2_11_7_1","unstructured":"National Reimbursement Drug List (version 2017).Ministry of Human Resources and Social Security of the People's Republic of China. Accessed August 3 2021.http:\/\/www.mohrss.gov.cn\/wap\/zc\/zcwj\/201702\/t20170223_266775.html"},{"issue":"3","key":"e_1_2_11_8_1","first-page":"A230","article-title":"Indirect comparison of the efficacy of recombinant factor IX Fc fusion protein and other factor IX products for prophylaxis: Simulating the effect of compliance on real\u2010world effectiveness","volume":"17","author":"Iorio A","year":"2014","journal-title":"Value Health"},{"key":"e_1_2_11_9_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1305074"},{"key":"e_1_2_11_10_1","doi-asserted-by":"publisher","DOI":"10.1016\/S2352\u20103026(16)30193\u20104"},{"key":"e_1_2_11_11_1","unstructured":"World Health Organization.China mortality.WHO. 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