Analysis Group, Inc., Boston, MA, USA.
Merck & Co., Inc., Kenilworth, NJ, USA.
Pharmacoeconomics. 2020 Nov;38(11):1201-1218. doi: 10.1007/s40273-020-00949-9.
Ovarian cancer is the fifth leading cause of cancer death in women in the US. With poly(ADP-ribose) polymerase (PARP) inhibitors having shown promising results in ongoing trials, there is interest in better understanding their economic value.
This study aimed to review and evaluate the quality of published cost-effectiveness analyses (CEAs), and provide recommendations for CEAs in this setting.
A systematic literature review of the MEDLINE and EMBASE databases was conducted in June 2019 to identify CEAs of PARP inhibitors in treating advanced ovarian cancer from peer-reviewed journals and conferences. Key information from the identified publications were extracted and reviewed. The quality of full-text studies was assessed using the Quality of Health Economic Studies instrument. Recommendations for future CEAs were developed based on the findings from the literature review.
Eighteen CEAs (five in full texts) met the inclusion criteria. Most adopted a US healthcare or societal perspective. The majority of the studies did not clearly display the economic model structure. No studies reported the validation of model projections based on internal or external data. Surrogate outcomes such as incremental costs per progression-free life-year gained were the most common outcomes reported. The majority of studies drew their conclusions based on surrogate outcomes, even with no theoretical or empirical threshold for cost effectiveness. All five full-text studies included some type of sensitivity or scenario analyses. The key drivers of the incremental cost-effectiveness ratio were treatment duration, effects, and costs, health utility, and prevalence of BRCA mutations.
In the existing CEAs for PARP inhibitors, there were uncertainties and challenges leading to variation in quality. We provided recommendations to improve consistency and quality of CEAs in this setting, which will help to better understand the value of PARP inhibitors, improve decision making, and reduce potential misallocation of resources.
卵巢癌是美国女性癌症死亡的第五大主要原因。由于聚（ADP-核糖）聚合酶（PARP）抑制剂在正在进行的试验中显示出良好的效果，因此人们对更好地了解其经济价值产生了兴趣。
本研究旨在回顾和评估已发表的成本效益分析（CEA）的质量，并为该领域的 CEA 提供建议。
2019 年 6 月，我们对 MEDLINE 和 EMBASE 数据库进行了系统的文献回顾，以确定来自同行评议期刊和会议的治疗晚期卵巢癌的 PARP 抑制剂的 CEA。从已确定的出版物中提取并审查了关键信息。使用健康经济研究质量工具评估全文研究的质量。根据文献综述的结果，提出了未来 CEA 的建议。
符合纳入标准的 CEA 有 18 项（5 项为全文）。大多数研究采用了美国医疗保健或社会视角。大多数研究没有清楚地展示经济模型结构。没有研究报告根据内部或外部数据验证模型预测。增量成本每无进展生存期是最常见的报告结果。大多数研究基于替代结果得出结论，即使没有成本效益的理论或经验阈值。所有五项全文研究都包括某种类型的敏感性或情景分析。增量成本效益比的主要驱动因素是治疗持续时间、效果和成本、健康效用和 BRCA 突变的流行率。
在 PARP 抑制剂的现有 CEA 中，存在不确定性和挑战，导致质量存在差异。我们提供了改善该领域 CEA 一致性和质量的建议，这将有助于更好地了解 PARP 抑制剂的价值，改善决策，并减少潜在的资源错配。
