{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,2]],"date-time":"2026-09-02T22:24:12Z","timestamp":1788387852772,"version":"build-2803163510"},"reference-count":105,"publisher":"Becaris Publishing Limited","issue":"10","content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["J. Comp. Eff. Res."],"published-print":{"date-parts":[[2023,10]]},"abstract":"<jats:p>\n            <jats:bold>Aim:<\/jats:bold>\n            Paroxysmal nocturnal hemoglobinuria (PNH) is a rare blood disorder characterized by hemolytic anemia, bone marrow failure and thrombosis, and is associated with high healthcare burden. We evaluated the cost\u2013effectiveness of pegcetacoplan, a proximal complement-3 inhibitor (C3i), compared with the C5i, eculizumab and ravulizumab, in complement treatment-naive adults with PNH, from the US\u00a0healthcare payer perspective.\n            <jats:bold>Materials &amp; methods:<\/jats:bold>\n            A\n            <jats:italic>de novo<\/jats:italic>\n            cost\u2013effectiveness model based on a Markov cohort structure evaluated lifetime (55-year) PNH costs and outcomes. The 6-month cycles of the model reflected the follow-up period of PRINCE (NCT04085601), an open-label trial of pegcetacoplan compared with eculizumab in C5i-naive patients. Data from PRINCE informed the clinical, safety and health-related quality of life outcomes in the model.\n            <jats:bold>Results:<\/jats:bold>\n            Pegcetacoplan was associated with lifetime cost savings of USD1,176,808 and USD213,062 relative to eculizumab and ravulizumab, respectively (largely attributed to reduced drug costs and blood transfusions), and additional quality-adjusted life years (QALYs) of 0.25 and 0.24.\n            <jats:bold>Conclusion:<\/jats:bold>\n            In patients with PNH who are treatment-naive, the base-case cost\u2013effectiveness analysis, scenario analysis and sensitivity analysis showed both lifetime cost savings and increased QALYs associated with pegcetacoplan compared with eculizumab or ravulizumab in the USA.\n          <\/jats:p>","DOI":"10.57264\/cer-2023-0055","type":"journal-article","created":{"date-parts":[[2023,9,1]],"date-time":"2023-09-01T10:51:42Z","timestamp":1693565502000},"source":"Crossref","is-referenced-by-count":6,"title":["The cost\u2013effectiveness of pegcetacoplan in complement treatment-na\u00efve adults with paroxysmal nocturnal hemoglobinuria in the USA"],"prefix":"10.57264","volume":"12","author":[{"given":"Jesse","family":"Fishman","sequence":"first","affiliation":[{"name":"Apellis Pharmaceuticals, Waltham, MA 02451, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Koo","family":"Wilson","sequence":"additional","affiliation":[{"name":"Swedish Orphan Biovitrum AB, Stockholm, Sweden"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Aleksandra","family":"Drzewiecka","sequence":"additional","affiliation":[{"name":"Putnam PHMR, Krakow, Poland (previously Creativ-Ceutical, Krakow, Poland during conduct of study)"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Micha\u0142","family":"Pochopie\u0144","sequence":"additional","affiliation":[{"name":"Assignity, Krakow, Poland (previously of Creativ-Ceutical, Krakow, Poland during conduct of study)"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"David","family":"Dingli","sequence":"additional","affiliation":[{"name":"Mayo Clinic, Rochester, MN 55902, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"36897","reference":[{"issue":"19","key":"e_1_3_7_2_2","doi-asserted-by":"crossref","first-page":"1253","DOI":"10.1056\/NEJM199511093331904","article-title":"Natural history of paroxysmal nocturnal hemoglobinuria","volume":"333","author":"Hillmen P","year":"1995","unstructured":"Hillmen P, Lewis SM, Bessler M, Luzzatto L, Dacie JV. Natural history of paroxysmal nocturnal hemoglobinuria. N. Engl. J. Med. 333(19), 1253\u20131258 (1995).","journal-title":"N. Engl. J. Med."},{"issue":"8","key":"e_1_3_7_3_2","doi-asserted-by":"crossref","first-page":"4461","DOI":"10.1007\/s12325-021-01825-4","article-title":"Real-world healthcare resource utilization (HRU) and costs of patients with paroxysmal nocturnal hemoglobinuria (PNH) receiving eculizumab in a US population","volume":"38","author":"Cheng WY","year":"2021","unstructured":"Cheng WY, Sarda SP, Mody-Patel N Real-world healthcare resource utilization (HRU) and costs of patients with paroxysmal nocturnal hemoglobinuria (PNH) receiving eculizumab in a US population. Adv. Ther. 38(8), 4461\u20134479 (2021).","journal-title":"Adv. Ther."},{"key":"e_1_3_7_4_2","unstructured":"National Organization for Rare Disorders (NORD). 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