Gharaibeh Mahdi, Bootman J Lyle, McBride Ali, Martin Jennifer, Abraham Ivo
Center for Health Outcomes and PharmacoEconomic Research, College of Pharmacy, University of Arizona, Drachman Hall B-306, 1295 N. Martin Ave, Tucson, AZ, 85721, USA.
Arizona Cancer Center, University of Arizona, Tucson, AZ, USA.
Pharmacoeconomics. 2017 Jan;35(1):83-95. doi: 10.1007/s40273-016-0452-6.
Effect sizes of efficacy of first-line treatments for (metastatic) pancreas cancer are constrained, underscoring the need for evaluations of the efficacy-to-cost relationship. We critically review economic evaluations of first-line chemotherapy regimens for pancreatic cancer since the 1997 introduction of gemcitabine. We searched PubMed/MEDLINE and EMBASE (1997-2015), and the websites of health technology assessment agencies. Two authors independently reviewed economic studies for eligibility in this review; evaluated peer-reviewed, journal-published studies in terms of the Drummond Checklist; and critiqued the technical and scientific merit of all studies. Sixteen pharmacoeconomic evaluations were included: ten published in nine peer-reviewed journals and six on three websites. Six were on single-agent therapies and ten on combination therapies. Analyses conducted included cost-effectiveness (three studies), cost-utility (one study), or combined cost-effectiveness and cost-utility (12 studies). Studies diverged in results, mainly because of different assumptions, methods, inputs, and country-specific guidelines. The two most recent regimens, nanoparticle albumin-bound paclitaxel plus gemcitabine (NAB-P + GEM) and the combination of fluorouracil, oxaliplatin, leucovorin, and irinotecan (FOLFIRINOX), were evaluated in an indirect comparison, yielding a statistically similar benefit in overall survival but superior progression-free survival for FOLFIRINOX. NAB-P + GEM showed greater economic benefit over FOLFIRINOX. In conclusion, the divergence in results observed across studies is attributable to economic drivers that are specific to countries and their healthcare (financing) systems. No recommendations regarding the relative economic benefit of treatment regimens, general or country-specific, are made as the purpose of pharmacoeconomic analysis is to inform policy decision-making and clinical practice, not set policy or define clinical practice.
（转移性）胰腺癌一线治疗的疗效效应大小有限，这突出了评估疗效与成本关系的必要性。我们严格审查了自1997年吉西他滨引入以来胰腺癌一线化疗方案的经济学评估。我们检索了PubMed/MEDLINE和EMBASE（1997 - 2015年）以及卫生技术评估机构的网站。两位作者独立审查经济研究以确定其是否符合本综述的纳入标准；根据德拉蒙德清单评估同行评审的期刊发表研究；并对所有研究的技术和科学价值进行评判。纳入了16项药物经济学评估：9项发表在9种同行评审期刊上，6项在3个网站上。6项是关于单药治疗，10项是关于联合治疗。进行的分析包括成本效益分析（3项研究）、成本效用分析（1项研究）或成本效益与成本效用综合分析（12项研究）。研究结果存在差异，主要是由于不同的假设、方法、投入和特定国家的指南。在一项间接比较中评估了两种最新的方案，纳米白蛋白结合型紫杉醇加吉西他滨（NAB - P + GEM）和氟尿嘧啶、奥沙利铂、亚叶酸钙和伊立替康的联合方案（FOLFIRINOX），结果显示总体生存获益在统计学上相似，但FOLFIRINOX的无进展生存期更优。NAB - P + GEM相对于FOLFIRINOX显示出更大的经济效益。总之，各研究结果的差异归因于特定国家及其医疗保健（融资）系统的经济驱动因素。由于药物经济学分析的目的是为政策决策和临床实践提供信息，而非制定政策或定义临床实践，因此未就治疗方案的相对经济效益给出一般性或特定国家的建议。
