Hofmarcher Thomas, Malmberg Chiara, Lindgren Peter
IHE-The Swedish Institute for Health Economics, Lund, Sweden.
Karolinska Institutet, Solna, Sweden.
Front Med (Lausanne). 2023 Feb 20;10:1119506. doi: 10.3389/fmed.2023.1119506. eCollection 2023.
Biomarker testing is indispensable for the implementation of precision medicine (PM) in oncology. The aim of this study was to assess the value of biomarker testing from a holistic perspective based on the example of advanced non-small cell lung cancer (aNSCLC).
A partitioned survival model was populated with data from pivotal clinical trials of first-line treatments in aNSCLC. Three testing scenarios were considered; "no biomarker testing" encompassing chemotherapy treatment, "sequential testing" for EGFR and ALK encompassing treatment with targeted- or chemotherapy, and "multigene testing" covering EGFR, ALK, ROS1, BRAF, NTRK, MET, RET and encompassing treatment with targeted- or immuno(chemo)therapy. Analyses of health outcomes and costs were run for nine countries (Australia, Brazil, China, Germany, Japan, Poland, South Africa, Turkey, United States). A 1-year and 5-year time horizon was applied. Information on test accuracy was combined with country-specific information on epidemiology and unit costs.
Compared to the no-testing scenario, survival improved and treatment-related adverse events decreased with increased testing. Five-year survival increased from 2% to 5-7% and to 13-19% with sequential testing and multigene testing, respectively. The highest survival gains were observed in East Asia due to a higher local prevalence of targetable mutations. Overall costs increased with increased testing in all countries. Although costs for testing and medicines increased, costs for treatment of adverse events and end-of-life care decreased throughout all years. Non-health care costs (sick leave and disability pension payments) decreased during the first year but increased over a 5-year horizon.
The broad use of biomarker testing and PM in aNSCLC leads to more efficient treatment assignment and improves health outcomes for patients globally, in particular prolonged progression-free disease phase and overall survival. These health gains require investment in biomarker testing and medicines. While costs for testing and medicines would initially increase, cost decreases for other medical services and non-health care costs may partly offset the cost increases.
生物标志物检测对于肿瘤学中精准医学(PM)的实施不可或缺。本研究旨在以晚期非小细胞肺癌(aNSCLC)为例,从整体角度评估生物标志物检测的价值。
利用aNSCLC一线治疗关键临床试验的数据构建了一个分区生存模型。考虑了三种检测方案;“不进行生物标志物检测”包括化疗治疗,“序贯检测”针对EGFR和ALK,包括靶向治疗或化疗,以及“多基因检测”涵盖EGFR、ALK、ROS1、BRAF、NTRK、MET、RET,包括靶向治疗或免疫(化疗)治疗。对九个国家(澳大利亚、巴西、中国、德国、日本、波兰、南非、土耳其、美国)进行了健康结果和成本分析。采用了1年和5年的时间范围。将检测准确性信息与特定国家的流行病学和单位成本信息相结合。
与不检测方案相比,随着检测增加,生存率提高,治疗相关不良事件减少。序贯检测和多基因检测的五年生存率分别从2%提高到5 - 7%和13 - 19%。由于可靶向突变的当地患病率较高,东亚地区观察到的生存获益最高。所有国家的总体成本随着检测增加而增加。尽管检测和药物成本增加,但所有年份中不良事件治疗和临终关怀的成本均下降。非医疗保健成本(病假和残疾抚恤金支付)在第一年下降,但在5年期间有所增加。
在aNSCLC中广泛使用生物标志物检测和精准医学可实现更有效的治疗分配,并改善全球患者的健康结果,特别是延长无进展疾病期和总生存期。这些健康获益需要对生物标志物检测和药物进行投资。虽然检测和药物成本最初会增加,但其他医疗服务和非医疗保健成本的降低可能会部分抵消成本的增加。