Ball Graeme, Levine Mitch, Thabane Lehana, Tarride Jean-Eric
Department of Health Research Methods, Evidence, and Impact, Faculty of Health Sciences, McMaster University, Hamilton, ON, Canada.
The Research Institute of St. Joe's Hamilton, St. Joseph's Healthcare Hamilton, Hamilton, ON, Canada.
Pharmacoecon Open. 2021 Sep;5(3):397-410. doi: 10.1007/s41669-021-00263-w. Epub 2021 Apr 24.
The type of methods used in economic evaluations of health technology can lead to results that may influence decisions. Despite the potential impact on decision making, there is very little documentation of methods used in economic evaluation in oncology pertaining to key assumptions and extrapolation methods of survival benefits, especially in terms of survival analysis techniques and methods for extrapolation.
The primary objectives of this study were to identify, examine, and describe the methods used in economic evaluations in oncology over a 10-year period, while secondary objectives included examining the use of identified methods across different geographic regions.
A systematic search of the published oncology literature was conducted to identify economic evaluations of advanced or metastatic cancers published between 2010 and 2019 using the PUBMED, Ovid MEDLINE, and EMBASE databases. A random sample was taken, and information on type of study, data source, modeling techniques, and survival analysis methods were abstracted and descriptively summarized.
A total of 8481 abstracts were identified and 76 economic evaluations were abstracted and assessed. Most identified studies were from North America (38%), East Asia (21%), continental Europe (18%), or the UK (16%), and most commonly focused on lung cancer (18%), colorectal cancer (16%), or breast cancer (13%). A large majority of studies were based on data from randomized controlled trials (82%), utilized a cost-utility approach (82%), and took a public healthcare system perspective (83%). Common model structures included Markov (49%) and partitioned survival (17%). Fitted parametric curves were the most commonly used extrapolation method (89%) for overall survival and most often utilized the Weibull distribution (64%). Secondary assessments showed modest regional variation in the use of identified methods, including the use of fitted parametric curves, testing of the proportional hazards assumption, and validation of results.
A majority of papers in the study sample reported basic characteristics of study type, data source used, modeling techniques, and utilization of survival analysis methods. However, greater detail in reporting extrapolation methods, statistical analyses, and validation of results could be potential improvements, especially across regions, in order to support greater consistency in decision making. Future research could document the diffusion of novel modeling techniques into economic evaluation.
卫生技术经济评估中所使用的方法类型可能会导致影响决策的结果。尽管对决策制定有潜在影响，但关于肿瘤学经济评估中使用的方法，特别是在生存分析技术和外推方法方面，与生存获益的关键假设和外推方法相关的文献却非常少。
本研究的主要目的是识别、审查和描述肿瘤学经济评估在10年期间所使用的方法，次要目的包括研究在不同地理区域所识别方法的使用情况。
通过对已发表的肿瘤学文献进行系统检索，以使用PUBMED、Ovid MEDLINE和EMBASE数据库识别2010年至2019年期间发表的晚期或转移性癌症的经济评估。抽取随机样本，并提取和描述性总结有关研究类型、数据来源、建模技术和生存分析方法的信息。
共识别出8481篇摘要，提取并评估了76项经济评估。大多数已识别的研究来自北美（38%）、东亚（21%）、欧洲大陆（18%）或英国（16%），最常关注的是肺癌（18%）、结直肠癌（16%）或乳腺癌（13%）。绝大多数研究基于随机对照试验的数据（82%），采用成本效用方法（82%），并从公共医疗系统角度出发（83%）。常见的模型结构包括马尔可夫模型（49%）和分段生存模型（17%）。拟合参数曲线是总生存最常用的外推方法（89%），并且最常使用威布尔分布（64%）。二次评估显示，在所识别方法的使用方面存在适度的区域差异，包括拟合参数曲线的使用、比例风险假设的检验以及结果的验证。
研究样本中的大多数论文报告了研究类型、所用数据来源、建模技术和生存分析方法的使用等基本特征。然而，在报告外推方法、统计分析和结果验证方面提供更详细的信息可能会有所改进，特别是在不同区域，以便在决策制定中支持更高的一致性。未来的研究可以记录新型建模技术在经济评估中的传播情况。
