School of Pharmaceutical Science and Technology, Tianjin University, Tianjin 300072, China.
Center for Social Science Survey and Data, Tianjin University, Tianjin 300072, China.
Int J Environ Res Public Health. 2021 Jun 8;18(12):6194. doi: 10.3390/ijerph18126194.
To evaluate the cost-effectiveness of dabrafenib plus trametinib combination therapy versus vemurafenib as first-line treatment in patients with BRAF V600 mutation-positive unresectable or metastatic melanoma from a healthcare system perspective in China. This study employed a partitioned survival model with three health states (progression-free survival, post-progression survival and dead) to parameterize the data derived from Combi-v trial and extrapolated to 30 years. Health states' utilities were measured by EQ-5D-3L, also sourced from the Combi-v trial. Costs including drug acquisition costs, disease management costs and adverse event costs were based on the Chinese Drug Bidding Database and physician survey in China. The primary outcomes of the model were lifetime costs, life-years (LYs), quality-adjusted life-years (QALYs) and incremental cost-effectiveness ratio (ICER). Deterministic and probabilistic sensitivity analyses were conducted, respectively. Dabrafenib plus trametinib is projected to increase a patient's life expectancy by 0.95 life-years over vemurafenib (3.03 vs. 2.08) and 1.09 QALY gains (2.48 vs. 1.39) with an incremental cost of $3833. The incremental cost-effectiveness ratio (ICER) was $3511 per QALY. In the probabilistic sensitivity analyses, at a threshold of $33,357 per QALY (three times the gross domestic product (GDP) per capita in China in 2020), the probability of dabrafenib plus trametinib being cost-effective was 90%. In the deterministic sensitivity analyses, the results were most sensitive to the dabrafenib plus trametinib drug costs, vemurafenib drug costs and discount rate of cost. Dabrafenib plus trametinib therapy yields more clinical benefits than vemurafenib. Using a threshold of $33,357 per QALY, dabrafenib plus trametinib is very cost-effective as compared with vemurafenib in China.
从中国医疗体系的角度出发，评估达拉非尼联合曲美替尼组合疗法与维莫非尼作为 BRAF V600 突变阳性不可切除或转移性黑色素瘤患者一线治疗的成本效益。本研究采用分割生存模型，有三个健康状态（无进展生存期、进展后生存期和死亡），对来自 Combi-v 试验的数据进行参数化，并外推至 30 年。健康状态的效用通过 EQ-5D-3L 进行衡量，也来自 Combi-v 试验。成本包括药物采购成本、疾病管理成本和不良事件成本，基于中国药品招标数据库和中国医生调查。模型的主要结果是终身成本、生命年（LY）、质量调整生命年（QALY）和增量成本效益比（ICER）。分别进行了确定性和概率敏感性分析。达拉非尼联合曲美替尼与维莫非尼相比，预计将使患者的预期寿命延长 0.95 个生命年（3.03 年对 2.08 年）和 1.09 个 QALY 获益（2.48 年对 1.39 年），增量成本为 3833 美元。增量成本效益比（ICER）为每 QALY3511 美元。在概率敏感性分析中，当 QALY 阈值为 33357 美元（2020 年中国人均国内生产总值（GDP）的三倍）时，达拉非尼联合曲美替尼具有成本效益的概率为 90%。在确定性敏感性分析中，结果对达拉非尼联合曲美替尼药物成本、维莫非尼药物成本和成本折扣率最为敏感。达拉非尼联合曲美替尼治疗比维莫非尼产生更多的临床获益。使用 33357 美元/QALY 的阈值，与维莫非尼相比，达拉非尼联合曲美替尼在中国非常具有成本效益。
