1 RTI Health Solutions, Research Triangle Park, North Carolina, and Division of Management, Policy, and Community Health, School of Public Health, University of Texas Health Science Center at Houston.
2 Division of Management, Policy, and Community Health and Division of Epidemiology, Human Genetics, and Environmental Sciences, School of Public Health, University of Texas Health Science Center at Houston.
J Manag Care Spec Pharm. 2017 Jan;23(1):64-73. doi: 10.18553/jmcp.2017.23.1.64.
Treatment patterns for metastatic colorectal cancer (mCRC) patients have changed considerably over the last decade with the introduction of new chemotherapies and targeted biologics. These treatments are often administered in various sequences with limited evidence regarding their cost-effectiveness.
To conduct a pharmacoeconomic evaluation of commonly administered treatment sequences among elderly mCRC patients.
A probabilistic discrete event simulation model assuming Weibull distribution was developed to evaluate the cost-effectiveness of the following common treatment sequences: (a) first-line oxaliplatin/irinotecan followed by second-line oxaliplatin/irinotecan + bevacizumab (OI-OIB); (b) first-line oxaliplatin/irinotecan + bevacizumab followed by second-line oxaliplatin/irinotecan + bevacizumab (OIB-OIB); (c) OI-OIB followed by a third-line targeted biologic (OI-OIB-TB); and (d) OIB-OIB followed by a third-line targeted biologic (OIB-OIB-TB). Input parameters for the model were primarily obtained from the Surveillance, Epidemiology, and End Results-Medicare linked dataset for incident mCRC patients aged 65 years and older diagnosed from January 2004 through December 2009. A probabilistic sensitivity analysis was performed to account for parameter uncertainty. Costs (2014 U.S. dollars) and effectiveness were discounted at an annual rate of 3%.
In the base case analyses, at the willingness-to-pay (WTP) threshold of $100,000/quality-adjusted life-year (QALY) gained, the treatment sequence OIB-OIB (vs. OI-OIB) was not cost-effective with an incremental cost-effectiveness ratio (ICER) per patient of $119,007/QALY; OI-OIB-TB (vs. OIB-OIB) was dominated; and OIB-OIB-TB (vs. OIB-OIB) was not cost-effective with an ICER of $405,857/QALY. Results similar to the base case analysis were obtained assuming log-normal distribution. Cost-effectiveness acceptability curves derived from a probabilistic sensitivity analysis showed that at a WTP of $100,000/QALY gained, sequence OI-OIB was 34% cost-effective, followed by OIB-OIB (31%), OI-OIB-TB (20%), and OIB-OIB-TB (15%).
Overall, survival increases marginally with the addition of targeted biologics, such as bevacizumab, at first line and third line at substantial costs. Treatment sequences with bevacizumab at first line and targeted biologics at third line may not be cost-effective at the commonly used threshold of $100,000/QALY gained, but a marginal decrease in the cost of bevacizumab may make treatment sequences with first-line bevacizumab cost-effective. Future economic evaluations should validate the study results using parameters from ongoing clinical trials.
This study was supported in part by a grant from the Agency for Healthcare Research and Quality (R01-HS018956) and in part by a grant from the Cancer Prevention and Research Institute of Texas (RP130051), which were obtained by Du. The authors report no conflicts of interest. Study concept and design were primarily contributed by Parikh, along with the other authors. All authors participated in data collection, and Parikh took the lead in data interpretation and analysis, along with Lairson and Morgan, with assistance from Du. The manuscript was written primarily by Parikh, along with Lairson, Morgan, and Du, and revised by Parikh.
在过去十年中,随着新的化疗药物和靶向生物制剂的引入,转移性结直肠癌(mCRC)患者的治疗模式发生了很大变化。这些治疗方法通常以不同的顺序进行,关于其成本效益的证据有限。
对老年 mCRC 患者常用治疗方案的成本效益进行评价。
假设威布尔分布,建立概率离散事件模拟模型,评估以下常见治疗方案的成本效益:(a)一线奥沙利铂/伊立替康加贝伐珠单抗(OI-OIB);(b)一线奥沙利铂/伊立替康加贝伐珠单抗(OIB-OIB);(c)OI-OIB 后加三线靶向生物制剂(OI-OIB-TB);(d)OIB-OIB 后加三线靶向生物制剂(OIB-OIB-TB)。模型的输入参数主要来自监测、流行病学和最终结果-医疗保险链接数据集,用于 2004 年 1 月至 2009 年 12 月期间诊断的 65 岁及以上的新发病例 mCRC 患者。进行概率敏感性分析以考虑参数不确定性。成本(2014 年美元)和效果按每年 3%贴现。
在基线分析中,在意愿支付(WTP)阈值为 10 万美元/QALY 的情况下,治疗方案 OIB-OIB(与 OI-OIB 相比)没有成本效益,每个患者的增量成本效益比(ICER)为 119,007 美元/QALY;OI-OIB-TB(与 OIB-OIB 相比)被排除;OIB-OIB-TB(与 OIB-OIB 相比)无成本效益,ICER 为 405,857 美元/QALY。在假设对数正态分布的情况下,得到了与基线分析相似的结果。从概率敏感性分析得出的成本效益接受曲线表明,在 WTP 为 10 万美元/QALY 的情况下,治疗方案 OI-OIB 的成本效益为 34%,其次是 OIB-OIB(31%)、OI-OIB-TB(20%)和 OIB-OIB-TB(15%)。
总的来说,在一线和三线增加贝伐珠单抗等靶向生物制剂的治疗方案在显著增加成本的情况下,略微提高了生存率。在一线使用贝伐珠单抗和三线使用靶向生物制剂的治疗方案在通常使用的 10 万美元/QALY 阈值下可能没有成本效益,但贝伐珠单抗成本的微小降低可能会使一线使用贝伐珠单抗的治疗方案具有成本效益。未来的经济评估应使用正在进行的临床试验中的参数验证研究结果。
本研究部分由美国医疗保健研究与质量局(R01-HS018956)和德克萨斯州癌症预防与研究协会(RP130051)资助,这两个资助由 Du 获得。作者没有利益冲突。概念和设计主要由 Parikh 提出,其他作者也参与了研究。所有作者都参与了数据收集,Parikh 主要负责数据解释和分析,Lairson 和 Morgan 提供了帮助,Du 也参与了研究。Parikh 主要负责撰写手稿,Lairson、Morgan 和 Du 对其进行了修订。