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Personalized Medicine in the Context of Comparative Effectiveness Research 比较有效性研究背景下的个体化医疗
Q3 Economics, Econometrics and Finance Pub Date : 2013-09-01 DOI: 10.1515/fhep-2013-0009
A. Basu
Abstract The world of patient-centered outcomes research (PCOR) seems to bridge the previously disjointed worlds of comparative effectiveness research (CER) and personalized medicine (PM). Indeed, theoretical reasoning on how information on medical quality should inform decision making, both at the individual and the policy level, reveals that personalized information on the value of medical products is critical for improving decision making at all levels. However, challenges to generating, evaluating and translating evidence that might lead to personalization need to be critically assessed. In this paper, I discuss two different concepts of personalized medicine – passive personalization (PPM) and active personalization (APM) that are important to distinguish in order to invest efficiently in PCOR and develop objective evidence on the value of personalization that will aid in its translation. APM constitutes the process of actively seeking identifiers, which can be genotypical, phenotypical or even environmental, that can be used to differentiate between the marginal benefits of treatment across patients. In contrast, PPM involves a passive approach to personalization where, in the absence of explicit research to discover identifiers, patients and physicians “learn by doing” mostly due to the repeated use of similar products on similar patients. Benchmarking the current state of PPM sets the bar to which the expected value of any new APM agenda should be evaluated. Exploring processes that enable PPM in practice can help discover new APM agendas, such as those based on developing predictive algorithms based on clinical, phenotypical and preference data, which may be more efficient that trying to develop expensive genetic tests. It can also identify scenarios or subgroups of patients where genomic research would be most valuable since alternative prediction algorithms were difficult to develop in those settings. Two clinical scenarios are discussed where PPM was explored through novel econometric methods. Related discussions around exploring PPM processes, multi-dimensionality of outcomes, and a balanced agenda for future research on personalization follow.
以患者为中心的结果研究(PCOR)的世界似乎连接了以前脱节的比较有效性研究(CER)和个性化医疗(PM)的世界。事实上,关于医疗质量信息应如何在个人和政策层面为决策提供信息的理论推理表明,关于医疗产品价值的个性化信息对于改善各级决策至关重要。然而,需要对可能导致个性化的证据的生成、评估和翻译所面临的挑战进行批判性评估。在本文中,我讨论了个性化医疗的两个不同概念-被动个性化(PPM)和主动个性化(APM),这对于区分有效地投资于PCOR和开发个性化价值的客观证据非常重要,这将有助于其翻译。APM构成了积极寻找标识符的过程,这些标识符可以是基因型的、表型的,甚至是环境的,可以用来区分不同患者治疗的边际效益。相比之下,PPM涉及一种被动的个性化方法,在缺乏明确的研究来发现标识符的情况下,患者和医生“边做边学”,主要是因为在类似的患者身上重复使用类似的产品。对PPM的当前状态进行基准测试为任何新的APM议程的期望值设定了标准。探索在实践中实现PPM的流程可以帮助发现新的APM议程,例如基于临床、表型和偏好数据开发预测算法的议程,这可能比开发昂贵的基因测试更有效。它还可以确定基因组研究最有价值的情景或患者亚组,因为在这些情况下很难开发替代预测算法。讨论了两种临床情况,其中PPM通过新颖的计量经济学方法进行了探索。接下来将围绕探索PPM过程、结果的多维性以及个性化未来研究的平衡议程进行相关讨论。
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引用次数: 3
The Economics of Personalization in Prevention and Public Health 预防和公共卫生中的个性化经济学
Q3 Economics, Econometrics and Finance Pub Date : 2013-09-01 DOI: 10.1515/fhep-2013-0011
D. Kenkel, Hua Wang
Abstract Personalized prevention uses family history and predictive genetic testing to identify people at high risk of serious diseases. The availability of predictive genetic tests is a newer and still-developing phenomenon. Many observers see tremendous potential for personalized prevention to improve public health. At the same time, the emergence of these new markets raises familiar health policy concerns about costs, cost-effectiveness, and health disparities. This paper first discusses an economic framework for the analysis of personalized prevention. On the demand side, consumers use personalized prevention as a form of information that allows them to make better choices about prevention, including medical care and health behaviors like diet and exercise. On the supply side, an interplay of complex market forces and regulations will determine the prices, advertising, and insurance coverage of predictive genetic tests. Beyond the question of whether health insurance will cover the costs of predictive genetic tests, there is a great deal of concern about whether consumers’ use of genetic tests might place them at risk of genetic discrimination or might lead to adverse selection. The paper also reports descriptive analysis of data from the 2000, 2005, and 2010 National Health Interview Surveys on the use of predictive genetic tests. The empirical analysis documents large socioeconomic status-related disparities in consumers having heard of genetic tests: for example, consumers with less schooling, Blacks, and Hispanics were substantially less likely to have heard of genetic tests. Evidence from other empirical studies provides little evidence that genetic testing leads to genetic discrimination in insurance markets. There is more evidence suggesting adverse selection, where genetic testing leads consumers to purchase long-term care insurance. The paper concludes with some preliminary thoughts about important directions for future research. The goal of the paper is to review relevant research to help develop an economic approach and social science research agenda into the determinants and consequences of genetic tests for prevention.
个性化预防利用家族史和预测性基因检测来识别严重疾病的高危人群。预测性基因测试的可用性是一个较新的和仍在发展的现象。许多观察人士看到了个性化预防改善公共卫生的巨大潜力。与此同时,这些新市场的出现引起了人们对成本、成本效益和卫生差距的熟悉的卫生政策关切。本文首先讨论了个性化预防分析的经济框架。在需求方,消费者使用个性化预防作为一种信息形式,使他们能够更好地选择预防,包括医疗保健和健康行为,如饮食和锻炼。在供应方面,复杂的市场力量和法规的相互作用将决定预测性基因测试的价格、广告和保险范围。除了健康保险是否会支付预测性基因检测费用的问题之外,人们还非常关注消费者使用基因检测是否会使他们面临基因歧视的风险或可能导致逆向选择。该论文还报告了对2000年、2005年和2010年全国健康访谈调查中使用预测性基因测试的数据的描述性分析。实证分析表明,在听说过基因检测的消费者中,与社会经济地位相关的巨大差异:例如,受教育程度较低的消费者、黑人和西班牙裔人听说过基因检测的可能性大大降低。来自其他实证研究的证据几乎没有证明基因检测导致保险市场的基因歧视。有更多的证据表明存在逆向选择,基因检测导致消费者购买长期护理保险。最后,对今后的重要研究方向提出了一些初步的思考。这篇论文的目标是回顾相关的研究,以帮助制定一种经济方法和社会科学研究议程,以研究基因检测用于预防的决定因素和后果。
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引用次数: 3
Economics of Personalized Health Care and Prevention: Introduction 个性化卫生保健和预防经济学:导论
Q3 Economics, Econometrics and Finance Pub Date : 2013-09-01 DOI: 10.1515/fhep-2013-0018
Gregory Bloss, J. Haaga
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引用次数: 3
Economic Perspectives on Personalized Health Care and Prevention 个性化医疗保健和预防的经济学观点
Q3 Economics, Econometrics and Finance Pub Date : 2013-09-01 DOI: 10.1515/fhep-2013-0010
K. Phillips, J. Sakowski, S. Liang, N. Ponce
Abstract The objective of this paper is to provide an overview of economic evaluation of personalized medicine, focusing particularly on the use of cost-effectiveness analysis and other methods of valuation. We draw on insights from the literature and our work at the University of California, San Francisco Center for Translational and Policy Research on Personalized Medicine (TRANSPERS). We begin with a discussion of why personalized medicine is of interest and challenges to adoption, whether personalized medicine is different enough to require different evaluation approaches, and what is known about the economics of personalized medicine. We then discuss insights from TRANSPERS research and six areas for future research: Develop and Apply Multiple Methods of Assessing Value Identify Key Factors in Determining the Value of Personalized Medicine Use Real World Perspectives in Economic Analyses Consider Patient Heterogeneity and Diverse Populations in Economic Analyses Prepare for Upcoming Challenges of Assessing Value of Emerging Technologies Incorporate Behavioral Economics into Value Assessments
摘要本文的目的是提供个性化医疗的经济评估概述,特别侧重于使用成本效益分析和其他评估方法。我们从文献和我们在加州大学旧金山分校个性化医疗转化和政策研究中心(TRANSPERS)的工作中吸取了见解。我们首先讨论了为什么个性化医疗引起人们的兴趣,以及采用个性化医疗面临的挑战,个性化医疗是否足够不同,需要不同的评估方法,以及个性化医疗的经济学知识。然后,我们讨论了来自TRANSPERS研究的见解和未来研究的六个领域:开发和应用多种评估价值的方法确定确定个性化医疗价值的关键因素在经济分析中使用现实世界的视角在经济分析中考虑患者异质性和多样化的人群为即将到来的新兴技术价值评估的挑战做好准备将行为经济学纳入价值评估
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引用次数: 15
Better Quality of Care or Healthier Patients? Hospital Utilization by Medicare Advantage and Fee-for-Service Enrollees. 更好的护理质量还是更健康的患者?医疗保险优势和按服务收费的参保人对医院的利用。
Q3 Economics, Econometrics and Finance Pub Date : 2013-05-15 DOI: 10.1515/fhep-2012-0037
Lauren Hersch Nicholas

Do differences in rates of use among managed care and Fee-for-Service Medicare beneficiaries reflect selection bias or successful care management by insurers? I demonstrate a new method to estimate the treatment effect of insurance status on health care utilization. Using clinical information and risk-adjustment techniques on data on acute admission that are unrelated to recent medical care, I create a proxy measure of unobserved health status. I find that positive selection accounts for between one-quarter and one-third of the risk-adjusted differences in rates of hospitalization for ambulatory care sensitive conditions and elective procedures among Medicare managed care and Fee-for-Service enrollees in 7 years of Healthcare Cost and Utilization Project State Inpatient Databases from Arizona, Florida, New Jersey and New York matched to Medicare enrollment data. Beyond selection effects, I find that managed care plans reduce rates of potentially preventable hospitalizations by 12.5 per 1,000 enrollees (compared to mean of 46 per 1,000) and reduce annual rates of elective admissions by 4 per 1,000 enrollees (mean 18.6 per 1,000).

管理式医疗和按服务收费的医疗保险受益人之间使用率的差异是否反映了保险公司的选择偏差或成功的医疗管理?本文提出了一种新的方法来估计保险状况对医疗保健利用的治疗效果。利用临床信息和风险调整技术对与近期医疗护理无关的急性入院数据,我创建了一个未观察到的健康状况的代理度量。我发现积极的选择占了四分之一到三分之一的风险调整后的住院率的差异,在流动护理敏感条件和选择性程序中,医疗保险管理护理和按服务收费的注册者在7年的医疗成本和利用项目中,来自亚利桑那州,佛罗里达州,新泽西州和纽约州的住院患者数据库与医疗保险注册数据相匹配。除了选择效应,我发现管理式医疗计划将潜在可预防的住院率降低了12.5 / 1000(平均为46 / 1000),将选择性住院率降低了4 / 1000(平均为18.6 / 1000)。
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引用次数: 20
AIDS and Conflict: Micro Evidence from Burundi1) 艾滋病与冲突:来自布隆迪的微观证据
Q3 Economics, Econometrics and Finance Pub Date : 2013-01-01 DOI: 10.1515/fhep-2012-0035
Matthias Rieger
Abstract This paper studies the relationship between civil war and HIV/AIDS in Burundi at the micro level. The case of Burundi provides interesting grounds of analysis, as seroprevalence rates are heterogeneous across the country, the serological and conflict data for Burundi are of good quality and conclusions can inform HIV/AIDS policies in Burundi and other fragile states. Ordinary least squares and instrumental variable results indicate that there is no empirical relationship between seroprevalence at the general population level and three measures of local conflict intensity within provinces. This evidence could imply that areas that are relatively more conflict affected do not need to be prioritized over others in terms of HIV/AIDS policies. Further research should focus on individual rather than geographical exposure to conflict. There are likely certain groups and individuals at risk in the general population that need special attention after conflict. Furthermore, violence changes societies, in particular gender relations, thereby indirectly feeding and possibly fueling the dynamics of the epidemic.
本文从微观层面研究布隆迪内战与艾滋病的关系。布隆迪的情况提供了有趣的分析基础,因为全国各地的血清患病率各不相同,布隆迪的血清学和冲突数据质量良好,结论可以为布隆迪和其他脆弱国家的艾滋病毒/艾滋病政策提供信息。普通最小二乘和工具变量结果表明,一般人群水平的血清患病率与省内地方冲突强度的三个指标之间没有经验关系。这一证据可能意味着,在艾滋病毒/艾滋病政策方面,受冲突影响相对较大的地区不需要优先于其他地区。进一步的研究应侧重于个人而不是地理上受冲突影响的程度。在一般人群中,可能有某些处于危险中的群体和个人在冲突后需要特别关注。此外,暴力改变社会,特别是改变两性关系,从而间接助长并可能助长这一流行病的动态。
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引用次数: 0
Price-Shopping in Consumer-Directed Health Plans. 消费者导向健康计划中的价格购物
Q3 Economics, Econometrics and Finance Pub Date : 2013-01-01 DOI: 10.1515/thep-2012-0028
Neeraj Sood, Zachary Wagner, Peter Huckfeldt, Amelia Haviland

We use health insurance claims data from 63 large employers to estimate the extent of price shopping for nine common outpatient services in consumer-directed health plans (CDHPs) compared to traditional health plans. The main measures of price-shopping include: (1) the total price paid on the claim, (2) the share of claims from low and high cost providers and (3) the savings from price shopping relative to choosing prices randomly. All analyses control for individual and zip code level demographics and plan characteristics. We also estimate differences in price shopping within CDHPs depending on expected health care costs and whether the service was bought before or after reaching the deductible. For 8 out of 9 services analyzed, prices paid by CDHP and traditional plan enrollees did not differ significantly; CDHP enrollees paid 2.3% less for office visits. Similarly, office visits was the only service where CDHP enrollment resulted in a significantly larger share of claims from low cost providers and greater savings from price shopping relative to traditional plans. There was also no evidence that, within CDHP plans, consumers with lower expected medical expenses exhibited more price-shopping or that consumers exhibited more price-shopping before reaching the deductible.

我们使用来自63家大型雇主的健康保险索赔数据来估计与传统健康计划相比,消费者导向健康计划(CDHPs)中9种常见门诊服务的价格购物程度。价格购物的主要衡量标准包括:(1)索赔支付的总价;(2)低成本和高成本供应商的索赔份额;(3)相对于随机选择价格,价格购物节省的费用。所有的分析都控制了个人和邮政编码水平的人口统计和计划特征。我们还估计了cdhp内价格购物的差异,这取决于预期的医疗保健成本,以及服务是在达到免赔额之前还是之后购买的。在分析的9项服务中,CDHP和传统计划参保人支付的价格没有显著差异;CDHP参保者的办公室就诊费用减少2.3%。同样,诊所就诊是唯一一项CDHP注册导致低成本供应商索赔比例显著增加的服务,并且与传统计划相比,价格购物节省了更多费用。也没有证据表明,在CDHP计划中,预期医疗费用较低的消费者表现出更多的价格购物,或者消费者在达到免赔额之前表现出更多的价格购物。
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引用次数: 25
Measuring the Financial Exposure from Medical Care Spending Among Families with Employer Sponsored Insurance 衡量雇主赞助保险家庭医疗保健支出的财务风险
Q3 Economics, Econometrics and Finance Pub Date : 2013-01-01 DOI: 10.1515/fhep-2012-0012
J. Abraham, A. Royalty, T. DeLeire
Abstract We develop an empirical method to assess the degree of financial exposure associated with medical care spending among non-elderly US families with employer-sponsored insurance. A key feature of this method is its simplicity – it only requires data on out-of-pocket (OOP) health care spending and total health care spending and does not require detailed knowledge of health insurance benefit design. We apply our method to assess whether families with a chronically ill member face more financial exposure given their level of total spending relative to families with no chronically ill members. We find that the insured chronically ill face more financial exposure than the insured non-chronically ill. Additional analyses suggest that the reason for this additional financial exposure is not that families with a chronically ill member are in different, less generous plans, on average. Rather, families with a chronically ill member have higher spending on certain types of medical services (e.g., pharmaceuticals) that face higher levels of coinsurance. Given recent work on value-based insurance design and coinsurance as an obstacle to medication adherence, our findings suggest that the current design of health plans could jeopardize both the health and the financial well-being of the chronically ill.
摘要:我们开发了一种实证方法来评估与医疗保健支出相关的非老年美国家庭与雇主赞助的保险的财务风险程度。这种方法的一个关键特点是它的简单性——它只需要自付(OOP)医疗保健支出和总医疗保健支出的数据,而不需要详细了解医疗保险福利设计。我们应用我们的方法来评估是否有慢性病成员的家庭面临更多的财务风险,因为他们的总支出水平相对于没有慢性病成员的家庭。我们发现,被保险的慢性病患者比被保险的非慢性病患者面临更多的财务风险。其他分析表明,造成这种额外财务风险的原因,并不是平均而言,有慢性病成员的家庭在不同的、不那么慷慨的计划中。相反,有慢性病成员的家庭在某些类型的医疗服务(如药品)上的支出更高,面临更高的共同保险水平。鉴于最近关于基于价值的保险设计和共同保险作为药物依从性障碍的工作,我们的研究结果表明,目前的健康计划设计可能会危及慢性病患者的健康和财务状况。
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引用次数: 1
Price Shopping in Consumer-Directed Health Plans 消费者导向健康计划中的价格购物
Q3 Economics, Econometrics and Finance Pub Date : 2013-01-01 DOI: 10.1515/fhep-2012-0028
N. Sood, Z. Wagner, P. Huckfeldt, A. Haviland
Abstract We use health insurance claims data from 63 large employers to estimate the extent of price shopping for nine common outpatient services in consumer-directed health plans (CDHPs) compared to traditional health plans. The main measures of price shopping include (1) the total price paid on the claim, (2) the share of claims from low- and high-cost providers, and (3) the savings from price shopping relative to choosing prices randomly. All analyses control for individual and zip code level demographics and plan characteristics. We also estimate differences in price shopping within CDHPs depending on expected health care costs and whether the service was bought before or after reaching the deductible. For eight out of nine services analyzed, prices paid by CDHP and traditional plan enrollees did not differ significantly; CDHP enrollees paid 2.3% less for office visits. Similarly, office visits was the only service where CDHP enrollment resulted in a significantly larger share of claims from low-cost providers and greater savings from price shopping relative to traditional plans. There was also no evidence that, within CDHP plans, consumers with lower expected medical expenses exhibited more price shopping or that consumers exhibited more price shopping before reaching the deductible.
摘要:我们使用来自63家大型雇主的健康保险索赔数据来估计消费者导向健康计划(CDHPs)中9种常见门诊服务的价格购物程度与传统健康计划相比。价格购物的主要度量包括(1)索赔支付的总价,(2)低成本和高成本供应商的索赔份额,以及(3)相对于随机选择价格的价格购物节省的费用。所有的分析都控制了个人和邮政编码水平的人口统计和计划特征。我们还估计了cdhp内价格购物的差异,这取决于预期的医疗保健成本,以及服务是在达到免赔额之前还是之后购买的。在分析的9项服务中,CDHP和传统计划参保人支付的价格没有显著差异;CDHP参保者的办公室就诊费用减少2.3%。同样,诊所就诊是唯一一项CDHP注册导致低成本供应商索赔比例显著增加的服务,与传统计划相比,价格购物节省了更多费用。也没有证据表明,在CDHP计划中,预期医疗费用较低的消费者表现出更多的价格购物,或者消费者在达到免赔额之前表现出更多的价格购物。
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引用次数: 11
Should Global Health be Tailored Toward the Rich? Altruism and Efficient R&D for Neglected Diseases 全球健康应该为富人量身定制吗?被忽视疾病的利他主义与高效研发
Q3 Economics, Econometrics and Finance Pub Date : 2013-01-01 DOI: 10.1515/fhep-2012-0036
A. Jena, Stéphane Mechoulan, T. Philipson
Abstract We analyze the problem of incentivizing research and development (R&D) into developing world disease from an economic efficiency perspective. We view the problem as how to best promote R&D into goods with positive external effects in the sense that medicines that directly affect the health of the poor also indirectly affect the utility of the altruistic “rich.” We demonstrate why existing policy proposals – such as price concessions by manufacturers – adversely impact the poor by placing the burden of R&D only on innovators rather than all altruists in the rich world. We offer policy solutions that are based on economic efficiency and therefore rely on a broad sense of how the world values the treatment of developing world disease. We estimate that global altruism toward those with malaria is, at a minimum, valued between $835 million and $2.4 billion annually and for HIV/AIDS, between $9.1 billion and $26.6 billion annually. We argue that future policies toward neglected diseases need to better incorporate how efficient R&D meets the need of this global altruism.
摘要本文从经济效率的角度分析了发展中国家疾病研究与开发的激励问题。我们认为,问题在于如何最好地促进研发成为具有积极外部效应的产品,因为直接影响穷人健康的药物也会间接影响利他的“富人”的效用。我们论证了为什么现有的政策建议——比如制造商的价格让步——把研发的负担只放在创新者身上,而不是富国的所有利他主义者身上,从而对穷人产生了不利影响。我们提供的政策解决方案以经济效率为基础,因此依赖于对世界如何重视发展中国家疾病治疗的广泛认识。我们估计,全球对疟疾患者的利他主义每年至少价值8.35亿至24亿美元,对艾滋病毒/艾滋病的利他主义每年价值91亿至266亿美元。我们认为,未来针对被忽视疾病的政策需要更好地纳入研发效率如何满足这种全球利他主义的需求。
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引用次数: 0
期刊
Forum for Health Economics and Policy
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