{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,26]],"date-time":"2026-10-26T16:53:16Z","timestamp":1793033596399,"version":"4.5.0"},"reference-count":73,"publisher":"BMJ","issue":"9","license":[{"start":{"date-parts":[[2025,9,9]],"date-time":"2025-09-09T00:00:00Z","timestamp":1757376000000},"content-version":"unspecified","delay-in-days":8,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"}],"funder":[{"DOI":"10.13039\/100004328","name":"Genentech Inc.","doi-asserted-by":"crossref","award":["N\/A"],"award-info":[{"award-number":["N\/A"]}],"id":[{"id":"10.13039\/100004328","id-type":"DOI","asserted-by":"crossref"}]}],"content-domain":{"domain":["bmj.com"],"crossmark-restriction":true},"short-container-title":["BMJ Open"],"accepted":{"date-parts":[[2025,8,14]]},"published-print":{"date-parts":[[2025,9]]},"abstract":"<jats:sec>\n                  <jats:title>Objectives<\/jats:title>\n                  <jats:p>Emicizumab is the first bispecific antibody approved for prophylaxis in people with haemophilia A with or without factor VIII inhibitors. Aggregate distributional cost-effectiveness analysis assesses health equity impacts by evaluating how health effects and costs from funding an intervention are distributed among population subgroups. The objective was to evaluate how funding emicizumab for people with severe haemophilia A (PwSHA) impacts population health and health disparities in the USA.<\/jats:p>\n               <\/jats:sec>\n               <jats:sec>\n                  <jats:title>Design<\/jats:title>\n                  <jats:p>Population-level model of PwSHA from the perspective of the US healthcare system, using published sources and considering a lifetime time horizon.<\/jats:p>\n               <\/jats:sec>\n               <jats:sec>\n                  <jats:title>Intervention<\/jats:title>\n                  <jats:p>Emicizumab versus other haemophilia A prophylaxis treatments.<\/jats:p>\n               <\/jats:sec>\n               <jats:sec>\n                  <jats:title>Primary outcome measures<\/jats:title>\n                  <jats:p>Quality-adjusted life-years (QALYs) gained and change in Atkinson index of inequality in quality-adjusted life expectancy.<\/jats:p>\n               <\/jats:sec>\n               <jats:sec>\n                  <jats:title>Results<\/jats:title>\n                  <jats:p>When an estimated 6512 PwSHA in the USA were treated with emicizumab, the US healthcare system would save US$160\u2009billion over those individuals\u2019 lifetimes. If these cost savings fund additional healthcare interventions in the overall population, funding emicizumab would improve overall US population health (1\u2009068\u2009903 QALYs gained, using a threshold of US$150 000\/QALY) and reduce existing overall US inequities (\u22120.01% on the Atkinson index).<\/jats:p>\n                  <jats:p>In all scenarios tested for sensitivity, increased emicizumab and prophylaxis utilisation led to further reductions in health disparities and greater increases in population health. Results were robust to deterministic variations in the allocation of cost savings due to emicizumab use.<\/jats:p>\n               <\/jats:sec>\n               <jats:sec>\n                  <jats:title>Conclusion<\/jats:title>\n                  <jats:p>Funding emicizumab treatments for PwSHA improves overall population health and reduces overall health inequities in the USA. Cost savings from the use of emicizumab free up important resources that can be leveraged to support other healthcare interventions, but decisions on how these funds are used have large consequences for equity.<\/jats:p>\n               <\/jats:sec>","DOI":"10.1136\/bmjopen-2025-103112","type":"journal-article","created":{"date-parts":[[2025,9,10]],"date-time":"2025-09-10T05:10:41Z","timestamp":1757481041000},"page":"e103112","update-policy":"https:\/\/doi.org\/10.1136\/crossmarkpolicy","source":"Crossref","is-referenced-by-count":3,"title":["Modelling US health equity impacts of emicizumab for severe haemophilia A: aggregate distributional cost-effectiveness analysis"],"prefix":"10.1136","volume":"15","author":[{"ORCID":"https:\/\/orcid.org\/0009-0008-6331-0416","authenticated-orcid":false,"given":"Thomas","family":"Majda","sequence":"first","affiliation":[{"name":"Genentech, South San Francisco, California, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Janet S.","family":"Lee","sequence":"additional","affiliation":[{"name":"Genentech, South San Francisco, California, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Randall","family":"Curtis","sequence":"additional","affiliation":[{"name":"Factor VIII Computing, Berkeley, California, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Stacey L.","family":"Kowal","sequence":"additional","affiliation":[{"name":"Genentech, South San Francisco, California, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"239","published-online":{"date-parts":[[2025,9,9]]},"reference":[{"key":"2025090922103124000_15.9.e103112.1","doi-asserted-by":"crossref","first-page":"130","DOI":"10.3390\/hematolrep15010014","article-title":"Haemophilia A: A Review of Clinical Manifestations, Treatment, Mutations, and the Development of Inhibitors","volume":"15","author":"Sarmiento Doncel","year":"2023","journal-title":"Hematol Rep"},{"key":"2025090922103124000_15.9.e103112.2","doi-asserted-by":"crossref","first-page":"487","DOI":"10.1111\/hae.13998","article-title":"Occurrence rates of haemophilia among males in the United States based on surveillance conducted in specialized haemophilia treatment centres","volume":"26","author":"Soucie","year":"2020","journal-title":"Haemophilia"},{"key":"2025090922103124000_15.9.e103112.3","unstructured":"US Centers for Disease Control and Prevention . 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