Department of Pharmacy, The First Affifiliated Hospital of Fujian Medical University, 20 Cha Zhong Road, Taijiang, Fuzhou 350005, China; People...s Republic of China and department of Pharmacy, National Regional Medical Center, Binhai Campus of the First Affiliated Hospital, Fujian Medical University, 999 Huashan Road, Changle District, Fuzhou, China.
Department of Oncology, The First Affiliated Hospital of Fujian Medical University, Fuzhou, China; Department of Oncology, National Regional Medical Center, Binhai Campus of the First Affiliated Hospital, Fujian Medical University, Fuzhou, China.
Clin Breast Cancer. 2023 Jul;23(5):508-518. doi: 10.1016/j.clbc.2023.03.013. Epub 2023 Mar 31.
BACKGROUND: Recently, the DESTINY-Breast04 trial revealed that trastuzumab deruxtecan (T-DXd) significantly prolonged overall survival in patients with human epidermal growth factor receptor 2 (HER2)-low metastatic breast cancer (MBC). Considering the extraexpensive price of the new drug, a cost-effectiveness analysis of T-DXd is necessary to perform in the United States. In addition, because T-DXd has not been marketed in China, the pricing is a very important driver for the cost-effectiveness of T-DXd. The range of drug costs for which T-DXd could be considered cost-effective from a Chinese healthcare system perspective was explored. METHODS: We developed a Markov model to evaluate the cost-effectiveness of T-DXd versus physician's choice of chemotherapy (PCC). The simulation time horizon for this model was the life-time of patients. Transition probabilities were based on data from the DESTINY-Breast04 trial. Health utility data were derived from published studies. Outcome measures were costs (in 2022 US$), life-years (LYs), quality-adjusted LYs (QALYs), and the incremental cost-effectiveness ratio (ICER). One-way and probabilistic sensitivity analyses assessed the uncertainty of key model parameters and their joint impact on the base-case results. RESULTS: The model predicted that T-DXd provided an improvement of 0.84 LYs and 0.58 QALYs compared to PCC, with an ICER of $259,452.05 per QALY in the United States and $87,646.40 per QALY in China. The one-way sensitivity analysis demonstrated that the price of T-DXd had the greatest impact on ICERs. Probabilistic sensitivity analysis predicted that the probabilities of T-DXd being cost-effective compared to PCC were 7.2% and 0% at a willingness-to-pay of $150,000 per QALY in the United States and $36,475 per QALY (3 times the per capita gross domestic product) in China, respectively. Subgroup analyses showed that T-DXd was more effective for patients without visceral disease at baseline, followed by patients with Asian ethnic, patients without prior CDK 4/6 inhibitors therapy, and patients with HER2-1+ (IHC detection) status. CONCLUSION: T-DXd was unlikely to offer a reasonable value for the money spent compared to PCC for patients with HER2-low MBC in the United States. A value-based price for T-DXd was reduced by 51% in the United States and less than $1950 per cycle in China.
背景：最近，DESTINY-Breast04 试验表明，曲妥珠单抗-德鲁替康（T-DXd）可显著延长人表皮生长因子受体 2（HER2）低转移性乳腺癌（MBC）患者的总生存期。鉴于新药价格昂贵，有必要在美国进行 T-DXd 的成本效益分析。此外，由于 T-DXd 尚未在中国上市，因此定价是 T-DXd 成本效益的一个非常重要的驱动因素。本研究旨在探讨从中国医疗保健系统的角度来看，T-DXd 药物成本的范围，使其具有成本效益。
方法：我们开发了一个马尔可夫模型来评估 T-DXd 与医生选择的化疗（PCC）相比的成本效益。该模型的模拟时间范围是患者的终生。转移概率基于 DESTINY-Breast04 试验的数据。健康效用数据来自已发表的研究。结果衡量标准为成本（2022 年以美元计）、生命年（LY）、质量调整生命年（QALY）和增量成本效益比（ICER）。单因素敏感性分析和概率敏感性分析评估了关键模型参数的不确定性及其对基础案例结果的联合影响。
结果：该模型预测，与 PCC 相比，T-DXd 可提高 0.84 LY 和 0.58 QALY，在美国的 ICER 为每 QALY 259,452.05 美元，在中国的 ICER 为每 QALY 87,646.40 美元。单因素敏感性分析表明，T-DXd 的价格对 ICER 的影响最大。概率敏感性分析预测，在美国，当意愿支付每 QALY 150,000 美元时，T-DXd 比 PCC 更具成本效益的概率为 7.2%，在中国，当意愿支付每 QALY 36,475 美元（人均国内生产总值的 3 倍）时，T-DXd 比 PCC 更具成本效益的概率为 0%。亚组分析表明，对于基线时无内脏疾病的患者，T-DXd 的效果更好，其次是亚洲人种的患者、无先前 CDK4/6 抑制剂治疗的患者以及 HER2-1+（免疫组化检测）状态的患者。
结论：在美国，与 PCC 相比，对于 HER2 低转移性乳腺癌患者，T-DXd 的花费不太可能获得合理的价值。在美国，T-DXd 的基于价值的价格降低了 51%，在中国，每个周期的价格低于 1950 美元。
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