在社区中开展乳腺癌二十一基因检测的成本效益如何
根据既往研究结果和美国国家综合癌症网络（NCCN）指南推荐意见，基因表达谱检测（多基因检测）例如乳腺癌21基因检测（Oncotype DX）有助于早期、雌激素受体（ER）阳性、人表皮生长因子受体2（HER2）阴性乳腺癌患者的化疗决策。不过，其成本较高，既往主要在研究机构或大型医院开展，对于在社区医院开展多基因检测的成本效益（投入产出）尚不明确。
2018年1月8日，美国临床肿瘤学会《临床肿瘤学杂志》在线发表华盛顿乔治城大学医学中心和隆巴迪综合癌症中心、纽约爱因斯坦医学院和蒙蒂菲奥里医学中心、纽约罗彻斯特大学、北加利福尼亚凯泽永久医保集团、华盛顿大学弗雷德哈钦森癌症研究中心的研究报告，对40～79岁符合检测指征患者在社区医院开展乳腺癌21基因检测（Oncotype DX）的成本效益进行了评估。
凯泽永久：美国最大的医疗保险保健保障集团，创立于1945年，总部位于加利福尼亚州奥克兰，业务遍布美国8个州和首都华盛顿哥伦比亚特区，拥有自己的医疗保险基金、医院医生、研究机构。截至2017年10月，拥有1170万位医疗保险客户、20.8975万位员工、2.1275万位医生、5.4072万位护士、39个医学中心、720个医疗机构。由于需要为其医疗保险客户的医疗保健提供医疗保障，故其参与研究的现实意义相当大。
该研究通过计算机仿真模型，对社区医院开展乳腺癌21基因检测前（2000～2004年）后（2005～2012年）的25年社会成本增量和质量调整寿命年（根据生活质量进行校正的预期寿命，可理解为无病寿命，QALY）进行比较。输入数据包括来自综合医疗系统的乳腺癌21基因检测和化疗数据，全国和已经发表的乳腺癌21基因检测准确性、化疗有效性、使用率、生存和复发、联邦医疗保险和患者成本数据。
结果发现：
24%的符合指征患者接受了多基因检测。
年轻、I期（与IIA期相比）患者检测率较高。
复发风险评分高、低患者接受化疗率分别为75.3%、10.2%。
多基因检测与常规诊疗相比，每QALY的成本效益比为18.8125万美元。
如果考虑检测对心理的影响，每QALY的成本效益比减少为5.8431万美元。
如果检测的准确性非常完美，每QALY的成本效益比减少为2.8947万美元，理想条件下为3.9496万美元。
因此，根据社区实践模式推算，多基因检测成本效益比可能较高。然而，对于关键变量假设的实际变化，可能使多基因检测的成本效益比，大大减少至如同其他干预措施能够接受的范围。根据社区与理想条件相比相差的成本效益比，强调了评定新技术时应该对实际情况进行考虑的重要性。
J Clin Oncol. 2018 Jan 8. [Epub ahead of print]
Cost Effectiveness of Gene Expression Profile Testing in Community Practice.
Young Chandler, Clyde B. Schechter, Jinani Jayasekera, Aimee Near, Suzanne C. O'Neill, Claudine Isaacs, Charles E. Phelps, G. Thomas Ray, Tracy A. Lieu, Scott Ramsey, Jeanne S. Mandelblatt.
Georgetown University Medical Center, Lombardi Comprehensive Cancer Center, Washington, DC; Albert Einstein College of Medicine, Montefiore Medical Center, Bronx; University of Rochester, Rochester, NY; Kaiser Permanente Northern California, Oakland, CA; Fred Hutchinson Cancer Research Center, Seattle, WA.
PURPOSE: Gene expression profile (GEP) testing can support chemotherapy decision making for patients with early-stage, estrogen receptor-positive, human epidermal growth factor 2-negative breast cancers. This study evaluated the cost effectiveness of one GEP test, Oncotype DX (Genomic Health, Redwood City, CA), in community practice with test-eligible patients age 40 to 79 years.
METHODS: A simulation model compared 25-year societal incremental costs and quality-adjusted life-years (QALYs) of community Oncotype DX use from 2005 to 2012 versus usual care in the pretesting era (2000 to 2004). Inputs included Oncotype DX and chemotherapy data from an integrated health care system and national and published data on Oncotype DX accuracy, chemotherapy effectiveness, utilities, survival and recurrence, and Medicare and patient costs. Sensitivity analyses varied individual parameters; results were also estimated for ideal conditions (ie, 100% testing and adherence to test-suggested treatment, perfect test accuracy, considering test effects on reassurance or worry, and lowest costs).
RESULTS: Twenty-four percent of test-eligible patients had Oncotype DX testing. Testing was higher in younger patients and patients with stage I disease (v stage IIA), and 75.3% and 10.2% of patients with high and low recurrence risk scores received chemotherapy, respectively. The cost-effectiveness ratio for testing (v usual care) was $188,125 per QALY. Considering test effects on worry versus reassurance decreased the cost-effectiveness ratio to $58,431 per QALY. With perfect test accuracy, the cost-effectiveness ratio was $28,947 per QALY, and under ideal conditions, it was $39,496 per QALY.
CONCLUSION: GEP testing is likely to have a high cost-effectiveness ratio on the basis of community practice patterns. However, realistic variations in assumptions about key variables could result in GEP testing having cost-effectiveness ratios in the range of other accepted interventions. The differences in cost-effectiveness ratios on the basis of community versus ideal conditions underscore the importance of considering real-world implementation when assessing the new technology.
PMID: 29309250
DOI: 10.1200/JCO.2017.74.5034