Miquel-Cases Anna, Steuten Lotte M G, Retèl Valesca P, van Harten Wim H
Netherlands Cancer Institute-Antoni van Leeuwenhoek Hospital (NKI-AVL), Department of Psychosocial Research and Epidemiology, Plesmanlaan 121, 1066 CX, Amsterdam, The Netherlands.
Hutchinson Institute for Cancer Outcomes Research, Fred Hutchinson Cancer Research Center, Seattle, USA.
Breast. 2015 Aug;24(4):397-405. doi: 10.1016/j.breast.2015.03.002. Epub 2015 Apr 28.
Triple negative breast cancers (TNBC) with a BRCA1-like profile may benefit from high dose alkylating chemotherapy (HDAC). This study examines whether BRCA1-like testing to target effective HDAC in TNBC patients can be more cost-effective than treating all patients with standard chemotherapy. Additionally, we estimated the minimum required prevalence of BRCA1-like and the required positive predictive value (PPV) for a BRCA1-like test to become cost-effective.
Our Markov model compared 1) the incremental costs; 2) the incremental number of respondents; 3) the incremental number of Quality Adjusted Life Years (QALYs); and 4) the incremental cost-effectiveness ratio (ICER) of treating TNBC women with personalized HDAC based on BRCA1-like testing vs. standard chemotherapy, from a Dutch societal perspective and a 20-year time horizon, using probabilistic sensitivity analysis. Furthermore, we performed one-way sensitivity analysis (SA) to all model parameters, and two-way SA to prevalence and PPV. Data were obtained from a current trial (NCT01057069), published literature and expert opinions.
BRCA1-like testing to target effective HDAC would presently not be cost-effective at a willingness-to-pay threshold of €80.000/QALY (€81.981/QALY). SAs show that PPV drives the ICER changes. Lower bounds for the prevalence and the PPV were found to be 58.5% and 73.0% respectively.
BRCA1-like testing to target effective HDAC treatment in TNBC patients is currently not cost-effective at a willingness-to-pay of €80.000/QALY, but it can be when a minimum PPV of 73% is obtained in clinical practice. This information can help test developers and clinicians in decisions on further research and development of BRCA1-like tests.
具有BRCA1样特征的三阴性乳腺癌(TNBC)患者可能从高剂量烷化剂化疗(HDAC)中获益。本研究旨在探讨针对TNBC患者进行BRCA1样检测以确定有效的HDAC治疗是否比用标准化疗治疗所有患者更具成本效益。此外,我们估计了BRCA1样特征的最低所需患病率以及BRCA1样检测具有成本效益所需的阳性预测值(PPV)。
我们的马尔可夫模型从荷兰社会视角和20年时间范围,使用概率敏感性分析,比较了以下方面:1)增量成本;2)应答者增量数量;3)质量调整生命年(QALY)增量数量;4)基于BRCA1样检测的个性化HDAC治疗TNBC女性与标准化疗的增量成本效益比(ICER)。此外,我们对所有模型参数进行了单向敏感性分析(SA),并对患病率和PPV进行了双向SA。数据来自当前试验(NCT01057069)、已发表的文献和专家意见。
在支付意愿阈值为80,000欧元/QALY(81,981欧元/QALY)时,针对有效HDAC的BRCA1样检测目前不具有成本效益。敏感性分析表明PPV驱动ICER变化。发现患病率和PPV的下限分别为58.5%和73.0%。
在支付意愿为80,000欧元/QALY时,针对TNBC患者进行BRCA1样检测以确定有效的HDAC治疗目前不具有成本效益,但在临床实践中获得至少73%的PPV时则可能具有成本效益。该信息可帮助检测开发者和临床医生在BRCA1样检测的进一步研发决策中提供参考。