The cost-effectiveness, of pegcetacoplan compared with ravulizumab for the treatment of paroxysmal nocturnal hemoglobinuria, in a UK setting.

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  • Additional Information
    • Source:
      Publisher: Becaris Publishing Country of Publication: England NLM ID: 101577308 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 2042-6313 (Electronic) Linking ISSN: 20426305 NLM ISO Abbreviation: J Comp Eff Res Subsets: MEDLINE
    • Publication Information:
      Publication: 2023- : Royston, UK : Becaris Publishing
      Original Publication: London : Future Medicine
    • Subject Terms:
      Hemoglobinuria, Paroxysmal*/drug therapy; Antibodies, Monoclonal, Humanized ; Clinical Trials as Topic ; Cost-Benefit Analysis ; Hemoglobins ; Humans ; Peptides, Cyclic ; United Kingdom
    • Abstract:
      Aim: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare blood disorder characterized by hemolytic anemia, bone marrow failure and thrombosis. We evaluated, the cost-effectiveness of pegcetacoplan, a novel proximal C3 inhibitor, versus ravulizumab in patients with PNH and hemoglobin levels <10.5 g/dl despite eculizumab treatment in the UK healthcare and social services setting. Materials & methods: A Markov cohort framework model, based on the data from the pivotal trial of pegcetacoplan (PEGASUS/NCT03500549), evaluated lifetime costs and outcomes. Patients transitioned through 3 PNH hemoglobin level/red blood cell transfusion health states. Results: Pegcetacoplan provides lower lifetime costs/greater quality-adjusted life years (£6,409,166/14.694QALYs, respectively) versus ravulizumab (£6,660,676/12.942QALYs). Conclusion: Pegcetacoplan is associated with enhanced anemia control, greater QALYs and reduced healthcare costs versus ravulizumab in the UK healthcare and social services setting.
    • Grant Information:
      Swedish Orphan Biovitrum AB
    • Contributed Indexing:
      Keywords: cost–effectiveness; hemolytic anemia; paroxysmal nocturnal hemoglobinuria; pegcetacoplan; red blood cell transfusion
    • Molecular Sequence:
      ClinicalTrials.gov NCT03500549
    • Accession Number:
      0 (Antibodies, Monoclonal, Humanized)
      0 (Hemoglobins)
      0 (Peptides, Cyclic)
      C3VX249T6L (ravulizumab)
      TO3JYR3BOU (pegcetacoplan)
    • Publication Date:
      Date Created: 20220707 Date Completed: 20220805 Latest Revision: 20220927
    • Publication Date:
      20221213
    • Accession Number:
      10.2217/cer-2022-0076
    • Accession Number:
      35796199