Hospital de Clínicas da Universidade Federal de Uberlândia, Uberlândia, Brazil.
Oncocentro (Grupo Oncoclinicas do Brasil), Uberlândia, Brazil.
J Med Econ. 2021 Jan-Dec;24(1):1185-1193. doi: 10.1080/13696998.2021.1993864.
Considering that healthcare systems' financial resources are limited, we aimed to analyze the number needed to treat (NNT) and cost of preventing an event (COPE) related to drug use from Supplementary Health System (SSS) perspective.
Data from KEYNOTE-189 (NCT02578680) were considered, comparing pembrolizumab + chemotherapy to chemotherapy alone. A cost-per-responder model was developed considering the 24- and 12-month time horizons for overall survival (OS) and progression-free survival (PFS) endpoints, respectively. Restricted mean survival time (RMST) and restricted mean time-on-treatment (ToT) were determined for NNT and COPE calculation. Costs were reported in American dollars (USD) and reflect those related to drug use. The analysis was conducted for the total indicated population, and an exploratory assessment was carried out for subgroups with different programmed death-ligand 1 (PD-L1) expression levels.
Considering PFS data, the overall population NNT to prevent a progression event with pembrolizumab + chemotherapy versus chemotherapy was 2.63 (95%CI: 1.90-4.02) with an estimated COPE of 251,038 USD (95%CI: 181,359-383,717) in the 12-months follow-up. Regarding OS endpoint, overall NNT and COPE were 3.18 (95%CI: 2.20-5.31) and 414,163 (95%CI: 286,528-691,573) USD respectively, in the 24 months follow-up. The PFS NNT was lower with higher levels of PD-L1 expression (1.71, 3.22 and 5.53 for PD-L1 ≥ 50%, PD-L1 1%-49%, and PD-L1 < 1% groups, respectively), while there was no such apparent relationship for OS (3.23, 4.37 and 2.80 for PD-L1 ≥ 50%, PD-L1 1%-49%, and PD-L1 < 1% groups, respectively). The 95%CIs overlapped for PFS and OS NNT across the PD-L1 subgroups.
The magnitude of benefit of the pembrolizumab combination used for first-line non-small cell lung cancer (NSCLC) treatment to improve survival compared to chemotherapy alone was confirmed. The exploratory analysis from the SSS perspective suggests no differences among the PDL-1 subgroups in terms of clinical benefit or economic impact.
考虑到医疗保健系统的财务资源有限，我们旨在从补充健康保险系统（SSS）的角度分析与药物使用相关的预防事件所需人数（NNT）和预防成本（COPE）。
本研究分析了 KEYNOTE-189（NCT02578680）的数据，比较了帕博利珠单抗联合化疗与单纯化疗。针对总生存期（OS）和无进展生存期（PFS）终点，分别建立了 24 个月和 12 个月的成本-应答模型。使用受限平均生存时间（RMST）和受限平均治疗时间（ToT）来计算 NNT 和 COPE。成本以美元（USD）表示，反映了与药物使用相关的成本。分析针对总适应证人群进行，并且对不同程序性死亡配体 1（PD-L1）表达水平的亚组进行了探索性评估。
考虑到 PFS 数据，与单纯化疗相比，帕博利珠单抗联合化疗预防进展事件的总体人群 NNT 为 2.63（95%CI：1.90-4.02），在 12 个月的随访中，预计 COPE 为 251038 美元（95%CI：181359-383717）。关于 OS 终点，在 24 个月的随访中，总 NNT 和 COPE 分别为 3.18（95%CI：2.20-5.31）和 414163 美元（95%CI：286528-691573）。在较高 PD-L1 表达水平下，PFS 的 NNT 更低（PD-L1≥50%、PD-L1 1%-49%和 PD-L1<1%组分别为 1.71、3.22 和 5.53），而 OS 中则没有明显的相关性（PD-L1≥50%、PD-L1 1%-49%和 PD-L1<1%组分别为 3.23、4.37 和 2.80）。在 PD-L1 亚组中，PFS 和 OS 的 NNT 95%CI 有重叠。
从补充健康保险系统角度证实了，与单纯化疗相比，帕博利珠单抗联合化疗用于一线非小细胞肺癌（NSCLC）治疗以改善生存的获益程度。探索性分析表明，在临床获益或经济影响方面，PD-L1 亚组之间没有差异。
