Department of Pharmacy, The Second Xiangya Hospital of Central South University, Changsha, Hunan, People's Republic of China.
Department of Nuclear Medicine/PET Image Center, The Second Xiangya Hospital of Central South University, Changsha, Hunan, People's Republic of China.
PLoS One. 2021 Nov 15;16(11):e0258605. doi: 10.1371/journal.pone.0258605. eCollection 2021.
The phase III KEYNOTE-604 study confirmed the benefit of pembrolizumab combined with chemotherapy in the first-line treatment of extensive-stage small-cell lung cancer (ES-SCLC). Taken into account the clinical benefits of pembrolizumab and its high cost, this study aimed to assess the cost-effectiveness of adding pembrolizumab to standard first-line etoposide-platinum (EP) for patients with ES-SCLC from the US payer perspective.
A Markov model was developed to compare the cost and quality-adjusted life-year (QALY) of pembrolizumab plus EP and placebo plus EP over a 10-year time horizon. Clinical efficacy and safety data were pooled from the KEYNOTE-604 trial. Utilities were obtained from published resources. Costs were mainly collected from Medicare in 2020. Sensitivity analyses were performed to examine the robustness of our model.
Adding pembrolizumab to standard first-line EP resulted in the better effectiveness than EP chemotherapy alone for ES-SCLC by 0.22 QALYs. Pembrolizumab plus EP was dominated economically by placebo plus EP, leading to an incremental cost-effectiveness ratio (ICER) of $334,373/ QALY. Deterministic sensitivity analyses indicated that the uncertainty in model parameters exerted no substantial effect on our results. Probability sensitivity analysis indicated that probabilities for pembrolizumab plus EP being cost-effective within a wide range of willingness to pay were modest.
From the US payer perspective, the first-line treatment for ES-SCLC with pembrolizumab plus EP was not cost-effective compared with placebo plus EP. Although pembrolizumab combination chemotherapy was beneficial to the survival of ES-SCLC, price reduction may be the necessary to improve its cost-effectiveness.
III 期 KEYNOTE-604 研究证实了帕博利珠单抗联合化疗在广泛期小细胞肺癌（ES-SCLC）一线治疗中的获益。考虑到帕博利珠单抗的临床获益及其高昂的成本，本研究旨在从美国支付者的角度评估帕博利珠单抗联合标准一线依托泊苷-铂类（EP）方案用于 ES-SCLC 患者的成本效果。
我们开发了一个 Markov 模型，以比较帕博利珠单抗联合 EP 与安慰剂联合 EP 在 10 年时间内的成本和质量调整生命年（QALY）。临床疗效和安全性数据来自 KEYNOTE-604 试验。效用值来自已发表的资源。成本主要来自 2020 年的 Medicare。进行了敏感性分析以检验模型的稳健性。
与 EP 化疗相比，在 ES-SCLC 中添加帕博利珠单抗到标准一线 EP 治疗方案可提高 0.22 个 QALY 的有效性。帕博利珠单抗联合 EP 在经济上优于安慰剂联合 EP，导致增量成本效果比（ICER）为 334,373 美元/QALY。确定性敏感性分析表明，模型参数的不确定性对我们的结果没有实质性影响。概率敏感性分析表明，在广泛的支付意愿范围内，帕博利珠单抗联合 EP 具有成本效果的概率适中。
从美国支付者的角度来看，与安慰剂联合 EP 相比，帕博利珠单抗联合 EP 一线治疗 ES-SCLC 并不具有成本效果。尽管帕博利珠单抗联合化疗对 ES-SCLC 的生存有益，但降价可能是提高其成本效果的必要条件。
