The Economic Value of Innovative Treatments over the Product Life Cycle: The Case of Targeted Trastuzumab Therapy for Breast Cancer
The Economic Value of Innovative Treatments over the Product Life Cycle: The Case of Targeted Trastuzumab Therapy for Breast Cancer
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DOI：
10.1111/j.1524-4733.2009.00572.x
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发表时间：
2009-11-01
期刊：
影响因子：
4.5
通讯作者：
Veenstra, David L.
中科院分区：
文献类型：
--
作者：
Garrison, Louis P., Jr.;Veenstra, David L.
目的：药物经济学分析通常预测一种新产品在特定适应症下的预期成本效益。这项分析开发了一个动态生命周期模型，利用2006年和1998年分别批准的早期和转移(或晚期)人表皮生长因子受体2(HER2)阳性乳腺癌治疗(早期乳腺癌[EBC]；转移性乳腺癌[MBC])在美国获得许可的曲妥珠单抗案例进行多适应症评估。方法：该动态模型结合了1998年至2016年特定适应症预期增量成本效用比的信息和按适应症在产品生命周期内的流行病学使用预测。净经济价值估计为在生命周期中获得的累积质量调整寿命年(QALY)乘以健康收益的社会估值(美元/QALY)减去累积的直接治疗净成本。在一系列假设下进行了敏感性分析。结果：我们预测到2016年，接受曲妥珠单抗治疗的EBC患者的年度数量将是MBC的三倍以上，部分原因是辅助治疗降低了MBC的未来发病率。在这个生命周期中，估计的总体增量成本效益比为35,590美元/QALY，总共获得了432,547个折扣QALY。根据敏感性分析，总体ICER在21,000美元/QALY到53,000美元/QALY之间，曲妥珠单抗治疗产生的净经济价值在62亿美元到495亿美元之间。结论：多适应症化合物的平均ICER在产品生命周期中可以增加或减少。在这个例子中，曲妥珠单抗预计的整个生命周期ICER不到最初适应症的一半。这种动态的观点--而不是通常的静态观点--突显了药物开发决策和投资激励之间的相互依存关系，从而提出了重要的补偿政策问题。
Objective:Pharmacoeconomic analyses typically project the expected cost-effectiveness of a new product for a specific indication. This analysis develops a dynamic life-cycle model to conduct a multiindication evaluation using the case of trastuzumab licensed in the United States for both early-stage and metastatic (or late-stage) human epidermal growth factor receptor 2 (HER2)-positive breast cancer therapy (early breast cancer [EBC]; metastatic breast cancer [MBC]), approved in 2006 and 1998, respectively.Methods:This dynamic model combined information on expected incremental cost-utility ratios for specific indications with an epidemiologically based projection of utilization by indication over the product life cycle-from 1998 to 2016. Net economic value was estimated as the cumulative quality-adjusted life years (QALYs) gained over the life cycle multiplied by a societal valuation of health gains ($/QALY) minus cumulative net direct treatment costs. Sensitivity analyses were performed under a range of assumptions.Results:We projected that the annual number of EBC patients receiving trastuzumab will be more than three times that of MBC by 2016, in part because adjuvant treatment reduces the future incidence of MBC. Over this life cycle, the estimated overall incremental cost-effectiveness ratio (ICER) was $35,590/QALY with a total of 432,547 discounted QALYs gained. Under sensitivity analyses, the overall ICER varied from $21,000 to $53,000/QALY, and the projected net economic value resulting from trastuzumab treatment ranged from $6.2 billion to $49.5 billion.Conclusions:Average ICERs for multiindication compounds can increase or decrease over the product life cycle. In this example, the projected overall life-cycle ICER for trastuzumab was less than one half of that in the initial indication. This dynamic perspective-versus the usual static one-highlights the interdependence of drug development decisions and investment incentives, raising important reimbursement policy issues.