Analysis of Costs per Responder in US Adults with Paroxysmal Nocturnal Hemoglobinuria with a Suboptimal Response to Prior Eculizumab Treatment.

ABSTRACT: European Society for Blood and Marrow Transplantation (EBMT) hematologic response categories comprehensively assess complement inhibitor responses in patients with paroxysmal nocturnal hemoglobinuria (PNH). Using data from the 16-week randomized controlled period of the phase 3 PEGASUS trial (N = 80), we estimated the treatment cost per responder by the EBMT response category for pegcetacoplan and eculizumab in adults with PNH and a suboptimal response to eculizumab. Average drug costs per responder, number needed to treat, and incremental drug costs per responder were estimated using dosages administered during the trial (base case). A US payer perspective (2020 US dollars) was used. Scenario analyses were conducted for various costs, dosages, treatment durations, patient populations, and

SUBMITTER: Fishman J 

PROVIDER: S-EPMC10594490 | biostudies-literature | 2023 Oct

REPOSITORIES: biostudies-literature