健康效用值测量中的映射法及其相关模型概述

目的:为将非效用量表测量结果转化为健康效用值提供方法学参考。方法:查阅国内外文献,总结健康效用值测量中的映射法及其相关模型,并以生存质量量表测量结果转换成欧洲五维健康量表效用值为例,介绍各模型在概率映射中的应用。结果:映射法可通过建立非效用量表和效用量表之间的映射关系,进而得到健康效用值;其常用模型包括普通最小二乘法(OLS)模型、最小绝对离差法(CLAD)模型、Tobit模型、多元Logistic回归(MNL)模型、贝叶斯网络(BN)模型等。其中,OLS模型相对简单,预测效度较好,但会受到天花板效应的限制;Tobit模型不受天花板或地板效应的限制,当误差项满足方差齐性及正态性时,其预测结果优于OLS模型;CLAD模型可用于误差项非方差齐性不适用Tobit模型的情况;MNL模型先通过回归分析确定一种健康状态,再确定其效用值;BN模型预测效度较好,且不涉及计量经济学中的诸多假设和限制条件,但其构建过程受领域专家的影响较大。使用MNL或BN模式计算健康效用值的方法主要有蒙特卡洛模拟法、期望效用值法和最大可能概率法等。利用拟合优度、调整拟合优度、平均误差、均方误差、平均绝对误差等指标进行模型性能评价,可选出最优模型,进而计算健康效用值。结论:由于各映射模型各有优缺点,在临床研究中需要根据实际情况选择不同的映射模型。     

效用量表在中国人群中的应用研究综述

目的：对通用量表在中国人群中的应用研究进行文献综述,以期为我国药物经济学评价中健康效用测量工具的完善提供参考。方法：在中文期刊全文数据库(CNKI)、万方数据库、维普数据库和PubMed数据库中进行系统文献检索,筛选其中在中国人群中采用通用健康效用量表测量的研究。对研究中通用健康效用量表在中国人群中应用的可行性、信度、效度、天花板及地板效应进行综述。结果：目前中国人群相关研究中使用最多的通用健康效用量表为EQ-5D,其次为SF-6D。在各项研究中,这两个量表均具有较好的可行性、信度及结构效度,但没有校标效度的检验,也没有研究对量表的内容效度进行考察,因此无法做出全面评估。此外,文献中报告的EQ-5D的天花板效应较严重,SF-6D的地板效应较严重。结论：EQ-5D和SF-6D量表在在我国药物经济学研究中被广泛使用,但仍存在一定限制。关键词：健康效用；通用量表；EQ-5D；SF-6D     

欧洲五维健康量表效用值积分体系构建方法及结果的国际比较

文中系统地介绍了英国、美国、日本等7个国家在发展本国欧洲五维健康量表(EQ-5D)效用值积分体系时所采用的方法,并比较各研究结果间的差异及其原因,旨在为建立适合我国人群的EQ-5D效用值积分体系提供参考。不同人群对于健康相关生命质量的偏好具有一些共同点,但表现出来的差异也很大,我国应及早进行研究,这有助于推动国内成本效用分析方法在药物经济学领域中的应用与发展。     

关节炎健康效用值映射法转换的研究进展

目的 系统梳理关节炎健康效用值映射函数式,以期为国内关节炎健康效用值领域的发展研究提供可供借鉴的科学参考.方法 以2007～2019年为检索年限,收集国内外关于关节炎相关疾病量表和健康效用值映射法转化的研究文献,汇总并整理.结果 国外映射法研究较为成熟,国内尚处于起步阶段.关节炎映射法研究多为叙述性文献,实证类文献较少...     

药物经济学评价中采用映射法获取健康效用值的文献计量研究

目的:了解药物经济学评价中采用映射法获取健康效用值的研究现状,为药物经济学评价中映射研究的开展提供参考。方法:以"映射法""健康效用值""成本-效用""效用积分体系""Mapping""Health utility value""Cost-utility""Utivity point system""Value set"为中英文关键词检索中国知网、万方数据库、PubMed、Medline、Ebsco、Ovid、Wiley数据库中自建库起至2018年12月31日发表的有关映射法获取健康效用值的实证类期刊文献,采用文献计量法对纳入文献的基本信息、模型的构建与检验、最佳模型的类型等内容进行统计分析。结果:纳入研究的124篇文献均发表在英文期刊;在映射模型的构建与检验中,应用频次最多的计量经济学方法、性能评价指标和模型检验方法分别是普通最小二乘法(OLS)、平均绝对误差(MAE)和残差正态性检验,应用频次分别为97次(31.60%)、89次(24.93%)和62次(21.09%);明确最佳映射模型的有117篇,其中,直接映射效果最佳的有101篇(86.32%),非效用量表中大多采用的是特异性量...     

国外通用量表比较分析

目的:为我国健康相关生命质量的研究提供参考。方法:比较分析常用的健康相关生命质量通用量表。结果:SF-36健康调查是健康相关生命质量结果测量中最常用量表;NHP反映的是业外人士对健康的认知情况;SIP衡量的是疾病对行为的影响;QWB、HUI和EQ-5D都是基于偏好的测量。结论:目前并无统一的量表,要根据测量目的、人口特征和使用环境等选择量表。     

调整有限因变量混合模型在药物经济学健康效用量表映射中的运用

目的 介绍调整有限因变量混合模型(ALDVMM)及其在映射研究中的运用情况,为需要通过映射获取健康效用值的药物经济学评价提供参考.方法 采用文献研究的方法,分别从ALDVMM的开发背景、模型原理、模型确定与检验、模型优势以及目前该模型在实证研究中的运用现状等方面进行介绍.结果 与结论 ALDVMM是国外学者针对欧洲五维...     

国槐枝炭和复方新诺明合用治疗脓疱疮39例的报道

本文介绍了与健康相关的生活质量的概念和评价方法，以及生活质量评价在药物经济学中的应用趋势。近10年来，生活质量评价方法的研究不断深入和完善。生活质量指标作为药物治疗结果的衡量标准逐步进入实用阶段，在药物经济学的研究中处于愈益重要的地位。本文系统地论述了常用的生活质量评价方法，如通用量表和特殊量表、健康问卷和健康效用测量方法、健康效用指数等，以及这些方法的特点和应用。     

肝移植儿童生命质量评估量表的系统综述

摘要：目的:探索更适合肝移植儿童的健康相关生命质量（HRQoL）评估量表，为药物经济学研究提供参考。方法:计算机检索PubMed、Embase、Cochrane Library、CNKI、WanFang Data、VIP、SinoMed数据库，搜集使用标准HRQoL评估量表对肝移植儿童生命质量进行评估的文献，检索时限为2011年1月～2021年5月，比较纳入文献涉及量表的适用情况、异同点、优缺点。结果:共检索到919篇文献，纳入37篇，包括13种标准HRQoL评估量表，最常用的普适性量表为儿童生命质量评估模型核心量表（PedsQL4.0）,最常用的疾病特异性量表为PedsQL移植模块（PedsQL3.0TM）,对肝移植儿童针对性最强的量表为儿童肝移植生命质量（PeLTQL）,最便于得到健康效用值的量表为9维儿童健康效用量表（CHU9D）。结论:推荐使用PedsQL3.0TM评估肝移植儿童生命质量，使用CHU9D得到健康效用值。PeLTQL仍需进一步翻译和验证。     

药物经济学中效用概念浅析

目的:加深对药物经济学中效用、效用测量工具及其在健康领域中运用的理解,促进健康效用的实际运用。方法:通过查阅、综合总结国、内外相关文献,给出效用定义、效用的3个基本公理、效用的各类测量方法及区别,以及本文对测量得出效用值的理解。结果与结论:健康效用测量法中仅标准概率法将人群的风险态度考虑在内;健康效用值属于等区间基数刻度。     

Assessing modified risk tobacco and nicotine products: Description of the scientific framework and assessment of a closed modular electronic cigarette

Cigarette smoking causes many human diseases including cardiovascular disease, lung disease and cancer. Novel tobacco products with reduced yields of toxicants compared to cigarettes, such as tobacco-heating products, snus and electronic cigarettes, hold great potential for reducing the harms associated with tobacco use. In the UK several public health agencies have advocated a potential role for novel products in tobacco harm reduction. Public Health England has stated that “The current best estimate is that e-cigarettes are around 95% less harmful than smoking” and the Royal College of Physicians has urged public health to “Promote e-cigarettes widely as substitute for smoking”.Health related claims on novel products such as ‘reduced exposure’ and ‘reduced risk’ should be substantiated using a weight of evidence approach based on a comprehensive scientific assessment. The US FDA, has provided draft guidance outlining a framework to assess novel products as Modified Risk Tobacco Products (MRTP). Based on this, we now propose a framework comprising pre-clinical, clinical, and population studies to assess the risk profile of novel tobacco products.Additionally, the utility of this framework is assessed through the pre-clinical and part of the clinical comparison of a commercial e-cigarette (Vype ePen) with a scientific reference cigarette (3R4F) and the results of these studies suggest that ePen has the potential to be a reduced risk product.     

A review of health-utility data for osteoarthritis: implications for clinical trial-based evaluation

The objective of this review was to describe the performance of health-utility measures in valuing the quality-of-life (QOL) impact of changes in osteoarthritis (OA)-related chronic pain when administered within a clinical trial setting. Because the collection of utility data within a clinical trial is not always feasible in the development of health economic models, utility data from prior non-randomised studies conducted among patients with OA were also summarized.We conducted a literature review using the MEDLINE, EMBASE and PsycINFO databases. We selected studies employing validated direct and multi-attribute measures of health utility: the standard gamble, time trade-off, EuroQol index, Health Utilities Index, SF-6D, 15D and the Assessment of Quality of Life measure.We identified four randomized controlled trials and 17 observational studies. The results of prior clinical trials in which these health utility measures were used in evaluating OA are summarized and attributes of the utility measures such as the clinical importance and statistical significance of the results obtained are noted. Furthermore, the sensitivity of the utility measure to changes in co-administered non-utility based measures of health-related quality of life (e.g. visual analogue scale for pain, WOMACtrade mark) are also reported. Five findings emerged.First, the EQ-5D system was the most widely used metric to derive utilities. Second, for whatever utility measure was used, reported mean utilities for patient groups spanned a rather wide range of values across studies, potentially reflecting variation in illness severity, patient co-morbidities and/or patient treatment. Third, when studies reported more than one utility-based statistic, the utility valuations frequently differed by measure, suggesting that the choice of metric can potentially have an effect on QALY calculations. However, there was no consistent pattern as to which measure yielded the highest and lowest utility valuations. Fourth, changes in health-related QOL (HR-QOL) and utility measures displayed the expected relationships. When HR-QOL declined, the utility values also moved in this direction. The reverse was also true. In some instances, statistically significant changes in QOL measures were not mirrored by statistically significant changes in utility measures, suggesting that some studies may have been underpowered for the latter purpose. Finally, the body of clinical trial-based utility literature in OA was found to be relatively modest, with considerably more observational studies collecting utility data.Based on the limited number of trial-based health-utility evaluations in OA to date, there can potentially be divergent findings with respect to clinical and statistical significance of changes in utility measures and corresponding measures of health status. Analysts should carefully evaluate issues of statistical power and clinical sensitivity in utilizing these measures in clinical trials of OA interventions.     

Multi-attribute preference functions. Health Utilities Index

Multi-attribute utility theory, an extension of conventional utility theory, can be applied to model preference scores for health states defined by multi-attribute health status classification systems. The type of preference independence among the attributes determines the type of preference function required: additive, multiplicative or multilinear. In addition, the type of measurement instrument used determines the type of preference score obtained: value or utility. Multi-attribute utility theory has been applied to 2 recently developed multi-attribute health status classification systems, the Health Utilities Index (HUI) Mark II and Mark III systems. Results are presented for the Mark II system, and ongoing research is described for the Mark III system. The theory is also discussed in the context of other well known multi-attribute systems. The HUI system is an efficient method of determining a general public-based utility score for a specified health outcome or for the health status of an individual. In clinical populations, the scores can be used to provide a single summary measure of health-related quality of life. In cost-utility analyses, the scores can be used as quality weights for calculating quality-adjusted life years. In general populations, the measure can be used as quality weights for determining population health expectancy.     

阿法替尼和吉非替尼一线治疗EGFR突变阳性非小细胞肺癌的成本效用分析

目的 从中国医疗保健支付者的角度,评价阿法替尼与吉非替尼在表皮生长因子受体（epidermal growth factor receptor,EGFR）突变阳性非小细胞肺癌一线治疗中的成本效用。方法 基于一项高质量、多中心的二期随机临床试验（LUNG7）,依据疾病发展进程建立三状态Markov模型（无进展生存状态、疾病进展状态、死亡状态）,模型各状态转移概率与不良反应发生率通过临床试验数据提取并计算,效用值取自研究文献中的中国人群效用值,直接医疗成本取自本地收费或相关文献。对总人群Markov模型进行为期10年的成本效用评估,并对模型分析结果的稳定性进行确定敏感性和概率敏感性分析。结果 在基础分析中,10年间阿法替尼组相对于吉非替尼组需多花费$16 499.77,但同时可多获得0.29个质量调整生命年（quality-adjusted life years,QALYs）,其增量成本效果比（incremental cost-effectiveness ratio,ICER）为$57 428.17/QALY。此时ICER值高于中国支付意愿阈值（willingness to pay,WTP）$26 331/QALY,表明阿法替尼目前相对于吉非替尼不具经济优势。一维敏感性分析结果显示疾病进展阶段的效用值、吉非替尼和阿法替尼的价格以及无进展生存期效用值对结果的稳定性影响较大,但除吉非替尼价格外,其他变量均不能使ICER值降至WTP之下,表明模型结果稳定。结论 对于中国EFGR突变阳性非小细胞肺癌患者,阿法替尼在一线治疗中相对于吉非替尼当前没有表现出经济性。     

Eliciting social preference weights for Functional Assessment of Cancer Therapy-Lung health states

BACKGROUND: The Functional Assessment of Cancer Therapy-Lung (FACT-L) is a multidimensional measure of quality of life developed for use in the evaluation of interventions in lung cancer. OBJECTIVE: To develop a set of utility weights that could be used to convert FACT-L into a single index capable of being used in the economic analysis of clinical trial data. METHOD: A core set of FACT-L items were valued in two versions of a 14-page postal survey of over 400 members of the UK general population. Respondents valued hypothetical FACT-L health states using a scale from 0 to 100 (worst to best health state). Respondents also valued their own health using the standard form of the EuroQol EQ-5D. Data were entered into an ordinary least squares regression model. RESULTS: Item weights estimated in regression analysis yielded values for 10 items from the FACT-L. The summary index based on this selected set of FACT-L items has a maximum value of 0.703 and a minimum value of 0.111. CONCLUSION: This study demonstrates a practical method of converting a standard condition-specific measure into a form that has the requisite properties to legitimise its use in cost-utility analysis. The methodology used here is not unique to FACT-L and might be considered appropriate for use in converting similar instruments.     

The effect of clinical pharmacy services on family practice residents' attitudes: a nationwide study

A nationwide study investigated the attitudes of family practice residents toward: interdisciplinary health care teams in family practice; the clinical pharmacist as a member of the health care team; and the utility of clinical pharmacist involvement in private family practice offices. A random sample of 174 family practice residency programs was selected for study. First-year residents comprised the sample population. Based on the response of the directors, programs were assigned to experimental (programs offering clinical pharmacy services) or control (no clinical pharmacy services) groups. Completed attitudinal instruments were received from 158 resident respondents in the experimental group and 153 resident respondents from the control group. The reliability coefficient of the returned questionnaires was 0.901 by the split-halves method. Residents in the experimental group had significantly more favorable attitudes than those residents in the control group on several scales. These scales included the clinical pharmacist's participation on the health care team, utility of a clinical pharmacist in a private practice setting, desirability of hiring a clinical pharmacist, and the desirability of practicing with a multidisciplinary health care team. These results support the hypothesis that exposure to clinical pharmacy services can significantly affect physician resident's attitudes toward clinical pharmacy.