Aguiar P M, Lima T M, Storpirtis S
Department of Pharmacy, Faculty of Pharmaceutical Sciences, University of São Paulo, São Paulo, Brazil.
J Clin Pharm Ther. 2016 Apr;41(2):189-97. doi: 10.1111/jcpt.12384. Epub 2016 Mar 23.
Given the increasing healthcare costs and the recent introduction of novel agents in the treatment for multiple myeloma (MM), an incurable haematologic malignancy, more efficient use of existing resources is fundamental. The objective of this study was to systematically review economic evaluations of the use of novel agents in MM and assess their reporting quality.
A literature search was performed in PubMed/Medline, Latin American and Caribbean Health Sciences Literature, Cost-Effectiveness Analysis Registry and the National Health Services Economic Evaluation Database for economic evaluations up to June 2015. The search strategy included Medical Subject Headings terms or text words related to MM, economic evaluations and drugs. Full economic evaluations of bortezomib, thalidomide or lenalidomide in patients with MM that were published in English, Portuguese or Spanish were included. Two independent authors performed study selection, data extraction and quality assessment using 24 items from the Consolidated Health Economic Evaluation Reporting Standards (CHEERS) statement.
Of the 132 potentially relevant records identified, eight satisfied the inclusion criteria. Most studies were cost-effectiveness analyses combined with cost-utility analyses (n = 6) from the public payer perspective (n = 4) and were performed in Europe (n = 6) on patients with refractory or relapsed MM (n = 5). All studies were based on economic models, with four of them using discrete event simulation. We found bortezomib-based therapies to be one of the more commonly selected treatment strategies for comparison (n = 7). Overall, the intervention was more effective and costlier than the alternative strategy (average of $54 630 per life year; $68 261 per quality-adjusted life year-QALY). The CHEERS' total score was 14·6 (SD = 2·6) with the most frequent problems being the lack of precision measures for all model parameters, no evaluation of heterogeneity of the results by subgroup analyses and no description of the role the funder in the identification, design, conduct and reporting of the analysis.
Most analyses of the novel therapeutic agents determined that they were cost-effective in MM at a threshold of up to $100 000/QALY. Nevertheless, the poor reporting quality of the economic studies requires improvement to ensure greater transparency.
鉴于医疗成本不断增加,且近期针对多发性骨髓瘤(MM,一种无法治愈的血液系统恶性肿瘤)的治疗引入了新型药物,更有效地利用现有资源至关重要。本研究的目的是系统回顾新型药物在MM治疗中的经济学评价,并评估其报告质量。
在PubMed/Medline、拉丁美洲和加勒比健康科学文献数据库、成本效益分析注册库以及国家卫生服务经济评价数据库中进行文献检索,以获取截至2015年6月的经济学评价。检索策略包括与MM、经济学评价和药物相关的医学主题词或文本词。纳入以英文、葡萄牙文或西班牙文发表的关于硼替佐米、沙利度胺或来那度胺在MM患者中的全面经济学评价。两名独立作者使用《卫生经济评价报告标准合并声明》(CHEERS)中的24项内容进行研究筛选、数据提取和质量评估。
在识别出的132条潜在相关记录中,8条符合纳入标准。大多数研究是从公共支付者角度(n = 4)进行的成本效益分析与成本效用分析相结合(n = 6),在欧洲进行(n = 6),针对难治性或复发性MM患者(n = 5)。所有研究均基于经济模型,其中4项使用离散事件模拟。我们发现基于硼替佐米的疗法是比较中较常选择的治疗策略之一(n = 7)。总体而言,干预措施比替代策略更有效且成本更高(每生命年平均54630美元;每质量调整生命年-QALY为68261美元)。CHEERS的总分是14.6(标准差 = 2.6),最常见的问题是所有模型参数缺乏精确测量、未通过亚组分析评估结果的异质性以及未描述资助者在分析的识别、设计、实施和报告中的作用。
大多数新型治疗药物的分析确定,在每QALY高达100000美元的阈值下,它们在MM治疗中具有成本效益。然而,经济学研究报告质量较差,需要改进以确保更高的透明度。