■这项研究旨在通过跨环境和条件的应用类型来量化精准医学(PM)的物有所值的异质性,并随着PM领域的发展,将异质性的来源量化到特定承诺或关注的领域。
■在Embase中进行了系统搜索,Medline,EconLit,和CRD数据库,用于2011年至2021年之间发表的关于PM干预措施的成本效益分析(CEA)的研究。根据每个研究国家一次性人均GDP的支付意愿阈值,PM的净货币收益(NMB)采用随机效应meta分析进行汇总.异质性和研究偏差的来源使用随机效应元回归进行了检查,刀刀灵敏度分析,以及经济研究清单中的偏见。
■在PM的275个独特CEA中,公开赞助的研究发现,基因检测和基因治疗总体上都没有成本效益,这与商业实体资助的研究和早期评估相矛盾。PM具有成本效益的证据集中在筛查的基因测试中,诊断,或作为伴随诊断(汇集的NMB,$48,152,$8,869,$5,693,p<0.001),以多基因小组测试的形式(合并的NMB=31,026美元,p<0.001),仅适用于癌症和高收入国家等少数疾病领域。增量有效性是各种基因测试的重要价值驱动因素,而不是基因治疗。
■精准医学在不同应用类型和背景下的物有所值很难从已发表的研究中得出结论,这可能会受到系统性偏见的影响。PM的CEA的进行和报告应基于本地并标准化以进行有意义的比较。
This study aimed to quantify heterogeneity in the value for money of precision medicine (PM) by application types across contexts and conditions and to quantify sources of heterogeneity to areas of particular promises or concerns as the field of PM moves forward.
A systemic search was performed in Embase, Medline, EconLit, and CRD databases for studies published between 2011 and 2021 on cost-effectiveness analysis (CEA) of PM interventions. Based on a willingness-to-pay threshold of one-time GDP per capita of each study country, the net monetary benefit (NMB) of PM was pooled using random-effects meta-analyses. Sources of heterogeneity and study biases were examined using random-effects meta-regressions, jackknife sensitivity analysis, and the biases in economic studies checklist.
Among the 275 unique CEAs of PM, publicly sponsored studies found neither genetic testing nor gene therapy cost-effective in general, which was contradictory to studies funded by commercial entities and early stage evaluations. Evidence of PM being cost-effective was concentrated in a genetic test for screening, diagnosis, or as companion diagnostics (pooled NMBs, $48,152, $8,869, $5,693, p < 0.001), in the form of multigene panel testing (pooled NMBs = $31,026, p < 0.001), which only applied to a few disease areas such as cancer and high-income countries. Incremental effectiveness was an essential value driver for varied genetic tests but not gene therapy.
Precision medicine\'s value for money across application types and contexts was difficult to conclude from published studies, which might be subject to systematic bias. The conducting and reporting of CEA of PM should be locally based and standardized for meaningful comparisons.