A systematic review of the methodological quality of economic evaluations in genetic screening and testing for monogenic disorders

PurposeUnderstanding the value of genetic screening and testing for monogenic disorders requires high-quality, methodologically robust economic evaluations. This systematic review sought to assess the methodological quality among such studies and examined opportunities for improvement.MethodsWe searched PubMed, Cochrane, Embase, and Web of Science for economic evaluations of genetic screening/testing (2013-2019). Methodological rigor and adherence to best practices were systematically assessed using the British Medical Journal checklist.ResultsAcross the 47 identified studies, there were substantial variations in modeling approaches, reporting detail, and sophistication. Models ranged from simple decision trees to individual-level microsimulations that compared between 2 and >20 alternative interventions. Many studies failed to report sufficient detail to enable replication or did not justify modeling assumptions, especially for costing methods and utility values. Meta-analyses, systematic reviews, or calibration were rarely used to derive parameter estimates. Nearly all studies conducted some sensitivity analysis, and more sophisticated studies implemented probabilistic sensitivity/uncertainty analysis, threshold analysis, and value of information analysis.ConclusionWe describe a heterogeneous body of work and present recommendations and exemplar studies across the methodological domains of (1) perspective, scope, and parameter selection; (2) use of uncertainty/sensitivity analyses; and (3) reporting transparency for improvement in the economic evaluation of genetic screening/testing.     

Health technology assessment with risk aversion in health

Standard cost-effectiveness models compare incremental cost increases to incremental average gains in health, commonly expressed in Quality-Adjusted Life Years (QALYs). Our research generalizes earlier models in several ways. We introduce risk aversion in Quality of Life (QoL), which leads to “willingness-to-pay” thresholds that rise with illness severity, potentially by an order of magnitude. Unlike traditional CEA analyses, which discriminate against persons with disabilities, our analysis implies that the marginal value of improving QoL rises for disabled individuals. Our model can also value the uncertain benefits of medical interventions by employing well-established analytic methods from finance. Finally, we show that traditional QALYs no longer serve as a single index of health, when consumers are risk-averse. To address this problem, we derive a generalized single-index of health outcomes—the Generalized Risk-Adjusted QALY (GRA-QALY). Earlier models of CEA that abstract from risk-aversion nest as special cases of our more general model.     

早期糖尿病视网膜病变药物治疗的药物经济学分析

目的：探讨不同方案治疗早期糖尿病视网膜病变的经济效果,找出既经济又有效的方案。方法：对目前临床常用的4种不同治疗方案（导升明、胰激肽原酶、复方血栓通胶囊、复方丹参滴丸,治疗时间均为90d）运用药物经济学方法进行成本-效果分析。结果：4种方案所需费用分别为1417.50元、810.00元、891.00元、656.10元;治疗有效率分别为94.25%、85.09%、85.17%、83.53%;成本-效果比分别为15.04、9.52、10.46、7.85。结论：复方丹参滴丸治疗效果较好,不良反应少,成本低廉。     

国产促动力药物治疗功能性消化不良的成本-效果分析

目的　评价不同药物治疗功能性消化不良的经济效果。方法　运用经济学中的成本 -效果分析方法 ,对莫沙必利、伊托必利和吗丁啉治疗功能性消化不良方案进行分析。结果　莫沙必利与吗丁啉成本效果比相当 ,但所需增长的成本效果比高 ;伊托必利的成本效果比和增长的成本效果比均高于吗丁啉。结论　吗丁啉是治疗功能性消化不良的最佳用药方案     

Economic appraisal of laser applications in health care. Report of a project

This article reports on the results of a project evaluating effectiveness and cost-effectiveness of laser applications in health care. The literature was collected, emphasizing well-designed clinical trials and cost-effectiveness analyses. Few randomized clinical trials (RCTs) have been done outside the specialty of ophthalmology. Even fewer cost-effectiveness studies have been done, despite the frequent claims of cost-effectiveness in the medical literature. Review papers were commissioned, and a group of laser experts, economists, and policy-analysts met for 2 days to discuss the policy implications of lasers. The main conclusion from the conference is that inappropriate and unskilled use of lasers is presently the greatest problem with their application in health care.     

遗传毒理学试验预测致癌物的成本效益分析

本文对检测致癌物的遗传毒理试验与动物致癌试验相结合的序贯判别方法进行成本-效益分析。结果表明,这种方法具有显著的社会经济效益。如忽略各遗传毒理试验间的不独立性,并设致癌性先验概率为0.1,则遗传毒理学试验次数可限制在5次左右。当受试物致癌性的后验概率小于0.03时判为非致癌物,大于0.7时判为致癌物。     

国产与进口头孢他啶治疗小儿细菌性下呼吸道感染的成本-效果分析

目的对国产与进口头孢他啶治疗小儿细菌性下呼吸道感染进行成本—效果分析。方法对我院2008年10月-2009年10月间患小儿细菌性下呼吸道感染且单用头孢他啶治疗的住院患者136例进行回顾性分析,根据药品不同产地将其分为A、B两组,A组给与头孢他啶（国产）60mg/（kg·d）,分2次静脉滴注;B组给与头孢他啶（进口）60mg/（kg·d）,分2次静脉滴注,两组疗程均为7d,分别计算两组病人治疗后的细菌清除率、不良反应发生率,两组的治疗有效率和总成本,计算成本-效果比。结果疗程结束后A、B两组的药物治疗成本分别为：A组236.4元、B组982.5元,组间比较存在显著性差异（P〈0.05）;治疗有效率分别为：91.30%、94.03%;A、B两组的细菌清除率分别为：86.36%、90.70%;两组不良反应发生率分别为：5.80%、2.99%,组间比较差异均无显著性（P〈0.05）;两组成本-效果比分别为：2.59、10.45。结论国产头孢他啶的成本-效果比优于进口头孢他啶,为较佳的治疗方案。     

Vitamin a cost-effectiveness model

To assist in the selection of a preferable vitamin A deficiency control policy, a model has been developed to organize information on program costs and program-related effects. The model was designed to compare three approaches: (1) diet modification; (2) fortification of processed foods; and (3) periodic large doses. Health effects projected are rates of specific eye pathologies associated with vitamin A deficiency (xerophthalmia), and mortalities within age cohorts. Effectiveness is calculated as a function of coverage, biological efficacy, and incidence of vitamin A deficiency. The model was applied to data from the Province of West Java in Indonesia. The results of this application suggest that funding level considerations are an important factor in selecting a preferred control strategy. In addition to determining the relative resource requirements of alternative interventions aimed at reducing the morbidity and mortality effects of vitamin A deficiencies, the model, using marginal cost and marginal effectiveness information, can serve as a guide to the most efficient allocation of resources for each type of intervention.