Fishman Jesse, Anderson Seri, Talbird Sandra E, Dingli David
Apellis Pharmaceuticals, Inc., Waltham, MA 02451, USA.
RTI Health Solutions, Research Triangle Park, NC 27709, USA.
Hematol Rep. 2023 Oct 13;15(4):578-591. doi: 10.3390/hematolrep15040060.
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 settings. In total, 30 of 41 (73%) who switched to pegcetacoplan and 2 of 39 (5%) patients who continued eculizumab had a good, major, or complete response (good-to-complete responders) at Week 16. Average weekly drug costs per good-to-complete responder were USD 15,923 with pegcetacoplan and USD 216,100 with eculizumab; average weekly drug costs per patient were USD 11,651 and USD 11,082, respectively. Average drug costs per good-to-complete responder with pegcetacoplan were similar across complement inhibitor-naïve populations and were consistently lower than with eculizumab. Switching from eculizumab to pegcetacoplan allowed more patients with a suboptimal response to attain a good-to-complete response at lower costs. These results apply to patients with a suboptimal response to prior eculizumab treatment only.
欧洲血液和骨髓移植学会（EBMT）血液学反应类别全面评估阵发性睡眠性血红蛋白尿（PNH）患者的补体抑制剂反应。利用3期PEGASUS试验16周随机对照期的数据（N = 80），我们按EBMT反应类别估算了培塞妥单抗和依库珠单抗在接受依库珠单抗治疗反应欠佳的成人PNH患者中的每例反应者治疗成本。使用试验期间给药剂量（基础病例）估算每例反应者的平均药物成本、治疗所需人数和每例反应者的增量药物成本。采用美国支付方视角（2020年美元）。针对各种成本、剂量、治疗持续时间、患者群体和环境进行了情景分析。在第16周时，改用培塞妥单抗的41例患者中有30例（73%）、继续使用依库珠单抗的39例患者中有2例（5%）获得良好、主要或完全反应（良好至完全反应者）。培塞妥单抗组每例良好至完全反应者的平均每周药物成本为15,923美元，依库珠单抗组为216,100美元；每组患者的平均每周药物成本分别为11,651美元和11,082美元。培塞妥单抗组每例良好至完全反应者的平均药物成本在未使用过补体抑制剂的人群中相似，且始终低于依库珠单抗组。从依库珠单抗改用培塞妥单抗可使更多反应欠佳的患者以更低成本获得良好至完全反应。这些结果仅适用于既往依库珠单抗治疗反应欠佳的患者。
