Instrumental Variable Methods for Censored Cost Data and an Application in Prosta

用于审查成本数据的工具变量方法及其在 Prosta 中的应用

• 批准号: 8028216
• 负责人: ANIRBAN BASU
• 金额: $ 45.67万
• 依托单位: UNIVERSITY OF WASHINGTON
• 依托单位国家: 美国
• 财政年份: 2011
• 资助国家: 美国
• 起止时间: 2011-07-21 至 2014-03-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8028216
• 关键词: Acceleration; Accounting; Address; Adverse effects; Affect; Area; Cessation of life; Characteristics; Chronic Disease; Clinical; Cost Analysis; Cost of Illness; Costs and Benefits; Data; Databases; Disease; Evaluation; Expenditure; Health; Healthcare; Heterogeneity; Individual; Institute of Medicine (U.S.); Link; Literature; Malignant Neoplasms; Malignant neoplasm of prostate; Medical Surveillance; Medicare; Methods; Modeling; Patient Preferences; Patients; Policies; Policy Analysis; Probability; Reporting; Risk; Role; Severity of illness; Time; Weight; Work; alternative treatment; base; common treatment; comparative; cost; cost effectiveness; design; economic evaluation; end of life; expectation; innovation; interest; life time cost; novel; simulation; surveillance data; treatment effect

DESCRIPTION (provided by applicant): There are many studies where the analytical task is to estimate the total costs over a defined period of time for a treatment group with a particular condition or chronic disease. These include cost of illness studies such as incremental or total lifetime costs for a disease, the components of cost effectiveness, and other forms of economic evaluation in health care. However, answering such questions requires addressing a range of statistical and econometric issues that include variable time periods of observation for individual patients, fluctuating costs over the course of the disease or treatment, the escalation of costs when the patient is dying and administrative censoring of data prior to the endpoint of interest (e.g. cure, death). These analytical obstacles are compounded by skewed cost data and the endogeneity of treatment decisions caused by self-selection into treatment based on expected idiosyncratic benefits, costs and risks of treatments. Our proposal will develop innovative methods to address each of these problems in a consolidated manner. The proposed work will advance four broad areas in the fields of cost analysis and economic evaluation in health studies. First, it will provide and validate a consistent method of estimating costs in a setting characterized by varying rates of spending over time, right censoring, and the escalation of expenditures commonly observed just before death. Second, it will bridge two large literatures on methods, one on modeling skewed cost data (with right censoring) and the other on the use of IV in the presence of treatment heterogeneity. Third, it can highlight the role that unobserved patient preferences and disease severity may have in generating treatment effect heterogeneity in the context of prostate cancer. Lastly, this work has a high likelihood of generating new information about the comparative cost estimates in prostate cancer which can serve as important inputs into cost-effectiveness and other policy analyses. PUBLIC HEALTH RELEVANCE: The proposed work will develop innovative methods that will advance four broad areas in the fields of cost analysis and economic evaluation in health studies. First, it will provide and validate a consistent method of estimating costs in a setting characterized by varying rates of spending over time, right censoring, and the escalation of expenditures commonly observed just before death. Second, it will bridge two large literatures on methods, one on modeling skewed cost data (with right censoring) and the other on the use of IV in the presence of treatment heterogeneity. Third, it can highlight the role that unobserved patient preferences and disease severity may have in generating treatment effect heterogeneity in the context of prostate cancer. Lastly, this work has a high likelihood of generating new information about the comparative cost estimates in prostate cancer which can serve as important inputs into cost-effectiveness and other policy analyses.

描述（由申请人提供）：在许多研究中，分析任务是为患有特定疾病或慢性病的治疗组估算定义时间内的总成本。其中包括疾病研究的成本，例如疾病的增量或终生成本，成本效益的组成部分以及医疗保健中其他形式的经济评估。但是，回答此类问题需要解决一系列统计和计量经济学问题，其中包括各个患者的观察时间段，在疾病过程中或治疗过程中的成本波动，当患者垂死并在终点之前进行数据审查时成本升级（例如，治疗，例如治疗，死亡）。这些分析障碍是由偏斜的成本数据以及基于预期的特质福利，成本和治疗风险而引起的治疗造成的治疗决策的内生性的复杂性。 我们的建议将开发创新的方法，以合并的方式解决这些问题。拟议的工作将在健康研究中的成本分析和经济评估领域中推进四个广泛的领域。首先，它将提供并验证一种一致的方法来估计成本的方式，其特征是随着时间的流逝，正确的审查率以及通常在死亡前观察到的支出的升级。其次，它将桥接有关方法的两种大文献，一个关于对偏斜的成本数据进行建模（具有正确的审查），另一个在存在治疗异质性的情况下使用IV。第三，它可以强调未观察到的患者偏好和疾病严重程度在前列腺癌中产生治疗效应异质性的作用。最后，这项工作很有可能产生有关前列腺癌比较成本估算的新信息，这些信息可以作为成本效益和其他政策分析的重要意见。 公共卫生相关性：拟议中的工作将开发创新的方法，这些方法将在健康研究中的成本分析和经济评估领域推进四个广泛领域。首先，它将提供并验证一种一致的方法来估计成本的方式，其特征是随着时间的流逝，正确的审查率以及通常在死亡前观察到的支出的升级。其次，它将桥接有关方法的两种大文献，一个关于对偏斜的成本数据进行建模（具有正确的审查），另一个在存在治疗异质性的情况下使用IV。第三，它可以强调未观察到的患者偏好和疾病严重程度在前列腺癌中产生治疗效应异质性的作用。最后，这项工作很有可能产生有关前列腺癌比较成本估算的新信息，这些信息可以作为成本效益和其他政策分析的重要意见。