Department of Oncology, Xiangya Hospital, Central South University, Changsha, 410008, Hunan, China.
Department of Oncology, Enshi Central Hospital, Wuhan University, Hubei, 445000, China.
Adv Ther. 2022 Jun;39(6):2614-2629. doi: 10.1007/s12325-022-02101-9. Epub 2022 Apr 8.
In 2021, KEYNOTE-590 (NCT03189719) showed that pembrolizumab plus 5-fluorouracil and cisplatin (PPF) has more benefits than 5-fluorouracil and cisplatin (PF) as a first-line regimen to treat individuals with advanced esophageal cancer. However, given that it is expensive, controversies over the value of using this compared to competitive strategies remain. Hence, we conducted a cost-effectiveness evaluation of pembrolizumab plus chemotherapy.
A Markov model was applied in evaluating the efficacy and cost of PPF and PF over a 7-year horizon and measured the health outcomes in life-years (LYs), quality-adjusted life-years (QALYs), and incremental cost-effectiveness ratio (ICER). The economic data included were relevant to patients in the USA and China. We also performed one-way and probabilistic sensitivity analyses to determine the uncertainties relevant to the model. Willingness to pay thresholds (WTP) of $150,000/QALY (USA) and $35,673/QALY (China) were used to calculate a probability for the cost-effectiveness of PPF.
PPF yielded 0.386-0.607 QALYs (0.781-1.195 LYs) compared with PF. In our analysis, compared with receiving PF, patients with advanced esophageal cancer receiving PPF had an ICER of $577,461/QALY in the USA and $258,261/QALY in China, those for esophageal squamous cell carcinoma were $550,211/QALY in the USA and $244,580/QALY in China, and a programmed cell death ligand 1 combined positive score (PD-L1 CPS) ≥ 10 was associated with a cost of $479,119/QALY in the USA and $201,355/QALY in China. Sensitivity analysis found the price of pembrolizumab to be the biggest influence.
From the economic perspectives of the USA and China, a first-line regimen of PPF for esophageal cancer therapy may not be as cost-effective as PF. However, patients with esophageal cancer and PD-L1 CPS ≥ 10 may gain the most LYs from initial PPF treatment.
2021 年，KEYNOTE-590（NCT03189719）研究表明，与氟尿嘧啶和顺铂（PF）相比，帕博利珠单抗联合氟尿嘧啶和顺铂（PPF）作为一线治疗方案，可为晚期食管癌患者带来更多获益。然而，鉴于其价格昂贵，与竞争策略相比，使用该方案的价值仍存在争议。因此，我们开展了帕博利珠单抗联合化疗的成本效益评估。
采用马尔可夫模型评估 7 年内 PPF 和 PF 的疗效和成本，并以生命年（LY）、质量调整生命年（QALY）和增量成本效益比（ICER）衡量健康结果。经济数据与美国和中国的患者相关。我们还进行了单因素和概率敏感性分析，以确定与模型相关的不确定性。使用 150000 美元/QALY（美国）和 35673 美元/QALY（中国）的意愿支付阈值（WTP）计算 PPF 的成本效益概率。
与 PF 相比，PPF 可获得 0.386-0.607 QALY（0.781-1.195 LY）。在我们的分析中，与接受 PF 相比，美国接受 PPF 的晚期食管癌患者的 ICER 为 577461 美元/QALY，中国为 258261 美元/QALY；美国接受 PPF 的食管鳞癌患者的 ICER 为 550211 美元/QALY，中国为 244580 美元/QALY；程序性死亡配体 1 联合阳性评分（PD-L1 CPS）≥10 的患者的 ICER 为 479119 美元/QALY，中国为 201355 美元/QALY。敏感性分析发现，帕博利珠单抗的价格是最大的影响因素。
从美国和中国的经济角度来看，PPF 作为食管癌一线治疗方案的成本效益可能不如 PF。然而，PD-L1 CPS≥10 的食管癌患者可能从初始 PPF 治疗中获得最多的 LY。
