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Comparative and Relative Effectiveness: A Challenge for Health Systems, Regulators, or Pharmaceutical Companies? 比较和相对有效性:对卫生系统、监管机构或制药公司的挑战?
Pub Date : 2014-12-01 DOI: 10.2139/ssrn.2637910
A. Towse
This OHE Occasional Paper by Adrian Towse discusses Professor Bengt Jonsson's observations in a 2011 publication about the role of relative effectiveness research (RE) in the European medicines market and how this could lead to improved efficiency in the development of medicines, the pricing and use of medicines, and of health systems. To ensure value for money, understanding actual use of medicines in the health care system is an essential supplement to information from clinical trials. To move in this direction, the author sets out the efficiency criteria that would need to be met. He defines a deceptively simple challenge - creating an environment where choice of therapy is based on an optimal amount of evidence from relative effectiveness research that is generated and used efficiently across health care systems both within Europe and as between the US and Europe. He identifies three sets of changes that must occur to meet the challenge - 1. A new drug development paradigm supported by regulatory authorities, HTA agencies and pharmaceutical companies that encourages the efficient collection of RE data before and after a medicine reaches the market. 2. Concerted efforts to eliminate the currently costly duplication across countries in infrastructure and RE-based assessments. 3. Understanding how and how well individual health systems use and benefit from new medicines, both to identify when expected differences mean country-specific RE may be needed and help identify instances where health systems use treatments inefficiently. The author notes that 'appraisal of RE evidence should edge both parties towards efficient pricing and use of a new drug on the assumption that pricing and use will reflect value and, as evidence of value changes, so will price and use.' He believes EU reforms can encourage improved efficiency in various ways. He is somewhat less optimistic about trans-Atlantic convergence, although he argues that, ironically, convergence on the mutual use of CER/RE evidence could occur before convergence of FDA and EMA approaches to efficacy and relative efficacy.
这篇由Adrian Towse撰写的OHE不定期论文讨论了Bengt Jonsson教授在2011年发表的关于相对有效性研究(RE)在欧洲药品市场中的作用的观察,以及这如何导致提高药品开发、药品定价和使用以及卫生系统的效率。为确保物有所值,了解卫生保健系统中药物的实际使用情况是对临床试验信息的重要补充。为了朝这个方向发展,作者列出了需要满足的效率标准。他定义了一个看似简单的挑战——创造一种环境,在这种环境中,治疗的选择是基于来自相对有效性研究的最佳数量的证据,这些证据是在欧洲内部以及美欧之间的医疗保健系统中产生和有效使用的。他确定了三种必须发生的变化来迎接挑战:1。一种新的药物开发模式,由监管当局、卫生保健管理局机构和制药公司支持,鼓励在药物进入市场之前和之后有效收集可再生能源数据。2. 共同努力消除目前各国在基础设施和基于re的评估方面代价高昂的重复工作。3.了解各个卫生系统如何以及如何使用和受益于新药,既可以确定预期差异何时意味着可能需要针对具体国家的可再生资源,也有助于确定卫生系统使用治疗方法效率低下的情况。作者指出,“在定价和使用将反映价值的假设下,对可再生证据的评估应该使双方都倾向于有效定价和使用新药,作为价值变化的证据,价格和使用也将反映价值变化。”他认为,欧盟的改革可以鼓励以各种方式提高效率。他对跨大西洋趋同不太乐观,尽管他认为,具有讽刺意味的是,在FDA和EMA的疗效和相对疗效方法趋同之前,相互使用CER/RE证据的趋同可能会发生。
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引用次数: 1
Correlates of Depression and Anxiety Among Older Public Housing Residents 老年公屋居民抑郁和焦虑的相关因素
Pub Date : 2014-11-16 DOI: 10.16935/EJSS.2014.30.2.004
Sheery Cummings, S. Trecartin, Lyle Cooper
Older adults represent the fastest growing segment of the worldwide population. Mental health disorders present a major challenge to older individuals. Depression and anxiety are two of the most common mental health problems experienced by the older population and give rise to high impact adverse consequences, such as decreased quality of life and increased mortality. Poverty level older adults suffer from higher rates of mental health disorders than do their more highly resourced counterparts. Given worldwide growth of the older population an increasing number of low-income elderly live in public housing. This study examined the prevalence of depression and anxiety in older public housing residents and explored factors related to these disorders within context of the Social Antecedents Model of Psychopathology (SAMP). The SAMP posits a multi-stage model of cumulative demographic, behavioral and social factors that represent mental health risk correlates, with more proximal factors representing greater risk (George, 1989). Study participants included 187 older adults residing in two public housing facilities located in a mid-size city in the southeast United States. Data was gathered concerning residents’ demographic characteristics, psychiatric symptomatology, substance usage, health conditions, and social support via face-to-face interviews. The majority of the residents were male and the average age was 66.2 years (SD= 7.6). Residents reported high levels of clinically significant depression (20.1%) and anxiety (10%). Generalized linear models (GZLM) were used to identify variables that significantly predicted depression and anxiety among the residents. Social support, self-rated health, pain, years smoking, and opioid misuse were significant predictors of depression scores (LR x2 (11) = 90.4, p
老年人是世界人口中增长最快的部分。精神健康障碍是老年人面临的一个重大挑战。抑郁和焦虑是老年人最常见的两种心理健康问题,并会造成严重的不良后果,如生活质量下降和死亡率上升。处于贫困水平的老年人患精神疾病的比率高于资源更丰富的老年人。鉴于全球老年人口的增长,越来越多的低收入老年人住在公共住房中。本研究探讨了老年公屋居民抑郁和焦虑的患病率,并在精神病理学社会前因模型(SAMP)的背景下探讨了与这些疾病相关的因素。SAMP假设了一个多阶段的累积人口、行为和社会因素模型,这些因素代表着心理健康风险的相关性,更近的因素代表着更大的风险(George, 1989)。研究参与者包括187名老年人,他们居住在位于美国东南部一个中等城市的两个公共住房设施中。通过面对面访谈收集有关居民人口统计学特征、精神症状、物质使用、健康状况和社会支持的数据。男性居多,平均年龄66.2岁(SD= 7.6)。居民报告了高水平的临床显著抑郁(20.1%)和焦虑(10%)。采用广义线性模型(GZLM)识别显著预测居民抑郁和焦虑的变量。社会支持、自评健康、疼痛、吸烟年数和阿片类药物滥用是抑郁评分的显著预测因子(LR x2 (11) = 90.4, p
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引用次数: 0
Eliminating Aggregation Bias When Estimating Treatment Effects on Combined Outcomes with Applications to Quality of Life Assessment 在评估治疗效果对综合结果的影响并应用于生活质量评估时消除聚集偏倚
Pub Date : 2014-11-04 DOI: 10.2139/ssrn.2536521
Ian Paul McCarthy
Researchers are often interested in combined measures such as overall ratings, indices of physical or mental health, or health-related quality-of-life (HRQoL) outcomes. Such measures are typically composed of two or more underlying discrete variables. I show that estimating the effect of a treatment on the combined measure is biased with non-random treatment selection. I provide a solution to this problem by adopting an alternative estimator that first estimates treatment effects on the underlying variables and then combines these effects into an overall effect on the combined outcome of interest.
研究人员通常对综合测量感兴趣,如总体评分、身体或心理健康指数或与健康相关的生活质量(HRQoL)结果。这种测量通常由两个或多个潜在的离散变量组成。我表明,估计一种治疗对综合测量的影响与非随机治疗选择有偏差。我提供了一个解决这个问题的方法,通过采用一种替代估计器,首先估计对潜在变量的治疗效果,然后将这些效果组合成对感兴趣的综合结果的总体效果。
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引用次数: 0
Survival Analysis of Lung and Bronchus Cancer Patients Segmented by Demographic Characteristics 按人口统计学特征分类的肺癌和支气管癌患者的生存分析
Pub Date : 2014-11-01 DOI: 10.2139/ssrn.2527780
D. Jun, Kyung-Hoon Kim
We propose a Weibull mixture model considering both covariates and unobserved heterogeneity to examine how demographic variables affect individual survival times and to derive the annual number of deaths. We analyze the records of patients diagnosed with lung and bronchus cancer, the most common cancer in the United States. The result shows that the diagnosis year as well as age, gender, race, and registry significantly affect individual survival times. including unobserved heterogeneity, we remove a bias in hazard rates and provide better performance in forecasting the annual number of cancer deaths than other benchmarks. Furthermore, from segmenting patients into several groups, we specify the difference between groups and assess their group-specific survival probabilities within a given period. Our study is distinctive in that a bottom-up strategy is adopted to predict aggregate-level units. This makes health forecasting available in two sides: public and private sector. For the public sector, our study enables a more precise allocation of the government’s health and welfare budget. Also for the private sector, our segmentation results provide guidance to the insurance industry for targeting customers more specifically.
我们提出了一个威布尔混合模型,考虑协变量和未观察到的异质性,以检验人口变量如何影响个体生存时间,并得出年死亡人数。我们分析了诊断为肺癌和支气管癌的患者的记录,这是美国最常见的癌症。结果表明,诊断年份以及年龄、性别、种族和登记对个体生存时间有显著影响。包括未观察到的异质性,我们消除了危险率的偏差,并在预测癌症年死亡人数方面提供了比其他基准更好的表现。此外,通过将患者分为几个组,我们指定组之间的差异,并评估他们在给定时期内的组特异性生存概率。我们的研究的独特之处在于采用了自下而上的策略来预测聚合级单位。这使得卫生预测可以在两个方面使用:公共部门和私营部门。对公共部门而言,我们的研究可以更精确地分配政府的健康和福利预算。此外,对于私营部门,我们的细分结果为保险行业更具体地定位客户提供了指导。
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引用次数: 1
The Economics of an HIV/AIDS Vaccine 艾滋病毒/艾滋病疫苗经济学
Pub Date : 2014-10-28 DOI: 10.2139/ssrn.2515834
P. Leoni
We survey the main economic aspects of the creation of an AIDS vaccine. We first describe the market conditions for this vaccine, and we show the critical importance of patents to start the costly R&D process. We then describe the main economic impediments to this venture, and the main financial mechanisms in place to alleviate them. We pinpoint their pitfalls, and we finally present novel mechanisms capable of restoring economic efficiency, and of providing greater incentives for investors toundertake R&D in this vaccine.
我们调查了研制艾滋病疫苗的主要经济方面。我们首先描述了这种疫苗的市场状况,并展示了专利对于启动昂贵的研发过程的至关重要性。然后,我们描述了这项事业的主要经济障碍,以及缓解这些障碍的主要金融机制。我们指出了它们的缺陷,并最终提出了能够恢复经济效率的新机制,并为投资者参与这种疫苗的研发提供了更大的激励。
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引用次数: 0
Estimating Heterogeneous Treatment Effects in Randomized Control Trials 估计随机对照试验中的异质性治疗效果
Pub Date : 2014-10-07 DOI: 10.2139/ssrn.2433064
C. Adams
It is not possible to directly observe the distribution of individual treatment effects. However, this paper presents new results showing that the distribution may be inferred with data usually available from randomized control trials. Developing upon recent results presented in the computer science, signal engineering and economics literature, the paper presents necessary and sufficient conditions for non-parametric identification of mixture distributions. The identification results provide a natural analogy to a general method of moments estimator. The paper uses the procedure to estimate the distribution in survival benefits from chemotherapy treatments of colon cancer patients. For approximately 80% of patients the addition of adjuvant chemotherapy has little effect on survival, while for 20% of patients the proportion who survive past 4 years increase thirty five percentage points. However, these treatment effects are not estimated with precision
直接观察个别治疗效果的分布是不可能的。然而,本文提出了新的结果,表明可以用随机对照试验的数据推断出分布。在计算机科学、信号工程和经济学文献的基础上,提出了混合分布非参数辨识的充分必要条件。辨识结果与一般矩估计方法有很好的相似之处。本文使用该程序来估计结肠癌患者化疗的生存收益分布。对于大约80%的患者,辅助化疗的增加对生存几乎没有影响,而对于20%的患者,存活超过4年的比例增加了35个百分点。然而,这些治疗效果不能精确估计
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引用次数: 1
Testimony of Kevin Outterson (Boston University School of Law) to the House Energy and Commerce Committee, September 19, 2014 凯文·奥特森(波士顿大学法学院)在众议院能源和商业委员会的证词,2014年9月19日
Pub Date : 2014-09-24 DOI: 10.2139/SSRN.2500799
K. Outterson
We must act decisively to fix the broken business model for antibiotics and other methods to prevent and treat bacterial diseases. These other methods include vaccines, diagnostics, infection control, and devices. Last year, the CDC issued the first national threat assessment on antimicrobial resistance. The media reported that 23,000 Americans die each year from antibacterial resistance, but the CDC estimated an additional 14,000 deaths per year from a horrible intestinal disease related to antibiotic use, Clostridium difficile. These calculations are conservative and likely undercount the true impact in the US, the equivalent of a 100-passenger jet crashing every day.Antibiotic resistance deaths in Europe are in the same range, but the situation in poorer countries is also dire. Resistant pathogens in low-income countries cause several hundred thousand neonatal sepsis deaths each year. Similar numbers of people die in low-income countries from susceptible bacteria, so we face an antibiotic access crisis in addition to the global problem of resistance. Much of our world lives in a pre-antibiotic era.Future projections are much worse. If we lose antibiotics as a drug class, the social cost may be more than a trillion dollars, shaving several years off life expectancy and making many modern medical procedures either impossible or much more dangerous.The ability to prevent and treat bacterial diseases is a global common pool resource of immense value, akin to fisheries. Exhausting this resource is cheap and lazy; preserving it will take concerted effort and substantial resources. These future expenditures are an investment in the continued effectiveness of one of the greatest classes of drugs ever discovered. Consider this as an “insurance premium,” protecting us against the post-antibiotic era.
我们必须果断采取行动,修复抗生素和其他预防和治疗细菌性疾病的方法的破碎商业模式。这些其他方法包括疫苗、诊断、感染控制和设备。去年,美国疾病控制与预防中心发布了第一份关于抗菌素耐药性的国家威胁评估。据媒体报道,每年有2.3万美国人死于抗生素耐药性,但疾病预防控制中心估计,每年还有1.4万人死于与抗生素使用有关的可怕的肠道疾病——艰难梭菌。这些计算过于保守,可能低估了美国的真实影响——相当于每天有一架载有100名乘客的客机坠毁。欧洲因抗生素耐药性而死亡的人数也在同一范围内,但较贫穷国家的情况也很糟糕。在低收入国家,耐药病原体每年造成数十万新生儿败血症死亡。低收入国家死于易感细菌的人数也差不多,因此,除了全球耐药性问题外,我们还面临着获得抗生素的危机。世界上大部分地区都生活在前抗生素时代。对未来的预测要糟糕得多。如果我们不再把抗生素作为一种药物,社会成本可能会超过一万亿美元,预期寿命将缩短数年,许多现代医疗程序要么无法进行,要么更加危险。预防和治疗细菌性疾病的能力是一种具有巨大价值的全球共同资源,类似于渔业。耗尽这种资源既便宜又懒惰;保存它需要共同努力和大量资源。这些未来的支出是对迄今为止发现的最伟大的一类药物之一的持续有效性的投资。把它看作是“保险费”,保护我们免受后抗生素时代的侵害。
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引用次数: 3
Do Labor Market Institutions Influcence Suicide Mortality? An International Panel Data Analysis 劳动力市场制度是否影响自杀死亡率?国际面板数据分析
Pub Date : 2014-07-22 DOI: 10.2139/ssrn.2469743
Christian Breuer, H. Rottmann
We examine the economic and social determinants of suicide mortality in a panel of 25 OECD countries over the period 1970 – 2011 and explicitly analyze the effects of unemployment and labor market institutions on suicide rates. In line with a large body of literature our results suggest that unemployment increases suicide mortality, while real economic growth tends to decrease suicides. The results also indicate that unemployment benefits decrease suicides of males, while relatively strict employment protection regulations have a positive influence on suicide mortality. These findings indicate that labor market institutions may influence job satisfaction and the quality of life in industrial countries. We suggest taking into account the role of labor market institutions when analyzing the effects of institutional and economic determinants on health.
在1970年至2011年期间,我们对25个经合组织国家的自杀死亡率的经济和社会决定因素进行了研究,并明确分析了失业和劳动力市场制度对自杀率的影响。与大量文献一致,我们的研究结果表明,失业增加了自杀死亡率,而实际经济增长往往会降低自杀率。结果还表明,失业救济降低了男性自杀率,而相对严格的就业保护法规对自杀死亡率有积极影响。这些发现表明,劳动力市场制度可能会影响工业国家的工作满意度和生活质量。我们建议在分析制度和经济决定因素对健康的影响时,考虑到劳动力市场制度的作用。
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引用次数: 6
Social Factors Affecting Women's Susceptibility to HIV in India 影响印度妇女艾滋病毒易感性的社会因素
Pub Date : 2014-06-17 DOI: 10.2139/ssrn.2454257
P. Lall
India is the global epicentre of the HIV/AIDS epidemic in Asia. Previous research indicates that the majority of HIV-positive women in India were infected by their husbands, their only sexual partner, which makes them difficult to identify as a high-risk population. This paper assesses social factors associated with the transmission of HIV based on demographic determinants, such as age; sexual risk behavior; and gendered discrimination, such as domestic violence. Bivariate and multivariate analysis of the National Family Health Survey yields the result that women's socioeconomic status could have an association with their serostatus, as HIV-positive women were significantly more likely to have a low level of education than their HIV-negative counterparts. In contrast, female HIV-positive respondents displayed low tendencies toward high-risk sexual behavior, as less than 10% had two or more sexual partners in their lifetime. Finally, they were significantly more likely to have previously experienced domestic violence (45%) and sexual violence (22%) than the general population. Overall, results illustrate that Indian women's vulnerability toward HIV infection may not be the product of their own sexual risk behavior. The most prominent social factors are their socioeconomic status, such as their level of education, and other sociodemographic determinants, including their region of residence.
印度是亚洲艾滋病毒/艾滋病流行病的全球中心。先前的研究表明,印度大多数艾滋病毒阳性妇女是被她们唯一的性伴侣丈夫感染的,因此很难将她们确定为高危人群。本文基于人口统计学决定因素(如年龄)评估了与艾滋病毒传播相关的社会因素;性危险行为;以及性别歧视,比如家庭暴力。对全国家庭健康调查的双变量和多变量分析得出的结果是,妇女的社会经济地位可能与她们的血清状况有关,因为艾滋病毒阳性的妇女比艾滋病毒阴性的妇女受教育程度低的可能性要大得多。相比之下,女性hiv阳性应答者表现出较低的高危性行为倾向,只有不到10%的人一生中有两个或两个以上的性伴侣。最后,与一般人群相比,他们以前经历过家庭暴力(45%)和性暴力(22%)的可能性要大得多。总的来说,研究结果表明,印度妇女容易感染艾滋病毒可能不是她们自己的性危险行为的产物。最突出的社会因素是他们的社会经济地位,例如他们的教育水平,以及其他社会人口统计学决定因素,包括他们居住的地区。
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引用次数: 2
Social Return on Investment: Health Promotion Programs 投资的社会回报:健康促进计划
Pub Date : 2014-06-01 DOI: 10.2139/ssrn.2497017
Worawan Chandoevwit, K. Thampanishvong, Rattanakarun Rojjananukulpong
This study uses the social return on investment (SROI) method to evaluate selected health promotion programs supported by Thai Health Promotion Foundation. The selected programs include one issue-based program, i.e., food and nutrition, and three target-based programs, namely disabled persons, the elderly, and children and youth. SROI is a useful method for measuring the social impacts of projects or programs. It enables monetizing the social values of projects through the use of financial proxies. The method is transparent and based on cost-benefit analysis. It was found that social investment provides positive returns to the general public. Programs focusing on food and nutrition can cover a wide range of the population with low investment. The return is about 10 baht for each baht of investment. Programs on children and youth provide about half that of programs on food and nutrition. Health promotion programs for disabled persons and the elderly provide much lower return on investment than the other two programs. One reason is because programs for disabled persons and the elderly were started off later than the other two programs.
本研究采用社会投资回报(SROI)方法评估泰国健康促进基金会所支持的健康促进计划。被选定的项目包括一个以问题为基础的项目,即食品和营养,以及三个以目标为基础的项目,即残疾人、老年人和儿童和青年。SROI是衡量项目或计划的社会影响的有用方法。它可以通过使用金融代理将项目的社会价值货币化。该方法是透明的,并基于成本效益分析。研究发现,社会投资为公众提供了正回报。以食品和营养为重点的项目可以以较少的投资覆盖广泛的人口。每投资泰铢,回报约为10泰铢。儿童和青少年项目提供的资金大约是食品和营养项目的一半。残疾人和老年人健康促进方案的投资回报率远低于其他两个方案。原因之一是残疾人事业和老年人事业起步晚于其他两个事业。
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引用次数: 4
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Health Economics Evaluation Methods eJournal
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