Methods for studying treatment heterogeneity using large observational databases

使用大型观察数据库研究治疗异质性的方法

• 批准号: 9038278
• 负责人: Shirley Wang
• 金额: $ 24.9万
• 依托单位: BRIGHAM AND WOMEN'S HOSPITAL
• 依托单位国家: 美国
• 财政年份: 2015
• 资助国家: 美国
• 起止时间: 2015-03-01 至 2018-02-28
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/9038278
• 关键词: Methods; studying; treatment; heterogeneity; using

We propose a multi-phase program of research to develop, validate, apply and disseminate methods for estimating and communicating individual level heterogeneity in effectiveness and safety (for a range of patient/clinician identified outcomes of interest) for non-randomized comparative effectiveness research. The research program will translate theory to practice, and produce a template for clinical decision support tools that could be used to facilitate personalized medicine and informed decision making while addressing the personal characteristics, conditions and preferences of individual patients. Recent innovations in statistical methods for analyzing randomized clinical trials involve prediction of individual patient probabilities of experiencing benefit and/or harm. During the K99 phase of the award, I will work with my mentors and collaborators as we develop methods for non-randomized comparative effectiveness research which allow us to predict heterogeneous individual level treatment effects based on a constellation of patient characteristics. During this time, I will participate in the Postdoctoral Medical Informatics Research Training program at Harvard Medical School with a focus on training related to clinical and population health informatics. I will also be an active participant in education and training on topics such as patient/stakeholder engagement through the Brigham and Women's Hospital (BWH) Center for Patient Centered Comparative Effectiveness Research (PCERC). During the R00 phase of the award, I will solicit input from patient and physician members of the Patient and Family Advisory Councils (PFAC) at BWH PCERC regarding choices between alternative lipid lowering therapeutic strategies. I will work with them to identify safety and effectiveness outcomes that are priority concerns for the stakeholder groups they represent and understand what they expect from personalized treatment information. We will apply validated methods for estimating individual level treatment heterogeneity in studies using large, diverse healthcare databases. These studies will be designed to address the patient and provider identified priority questions. There will be continuous input from patients and providers as I work on developing clinical decision support tools designed to communicate relevant evidence, using metrics that healthcare consumers understand. I will use the skills developed through the Medical Informatics Training Program and collaborate with members of the Clinical Informatics Department at BWH to implement and test an evidence-based clinical decision support tool that is compatible with the hospital informatics infrastructure. This tool could be used during the office visit to help physicians and patients discuss the expected risks and benefits that are particular to each patient at a “critical moment”, during the office visit when they the treatment decision is being made.

我们提出了一项多相研究计划，以开发，验证，应用和传播方法 估计和交流有效性和安全性的个人水平异质性（一系列 患者/临床医生确定了非随机比较有效性研究的感兴趣的结果。这 研究计划将把理论转化为实践，并生成用于临床决策支持工具的模板 可以用来促进个性化医学和明智的决策，同时解决 个别患者的个人特征，条件和偏好。 分析随机临床试验的统计方法的最新创新涉及对个体的预测 患者经历福利和/或伤害的可能性。在奖项的K99阶段，我将与 当我们开发非随机比较有效性研究的方法时，我的导师和合作者 这使我们能够根据患者星座来预测异质的个体治疗效果 特征。在此期间，我将参加博士后医学信息学研究培训 哈佛医学院的课程，重点是与临床和人口健康有关的培训 信息。我还将成为患者/利益相关者等主题的教育和培训的积极参与者 通过Brigham and妇女医院（BWH）中心进行比较 有效性研究（PCERC）。在奖励的R00阶段，我将征集患者的意见和 BWH PCERC的患者和家庭咨询委员会（PFAC）的身体成员有关选择 在替代脂质降低治疗策略之间。我将与他们一起确定安全性和 有效性结果是他们所代表和理解的利益相关者群体的优先问题 他们对个性化治疗信息的期望。我们将应用经过验证的方法估算 使用大型，潜水的医疗保健数据库的研究中的个人水平治疗异质性。这些研究会 旨在解决患者和提供者确定的优先问题。将继续输入 从患者和提供者开始开发旨在交流的临床决策支持工具时 相关证据，使用医疗消费者理解的指标。我将使用通过 医学信息学培训计划并与临床信息学部门的成员合作 在BWH以实施和测试与与之兼容的基于证据的临床决策支持工具 医院信息基础设施。可以在办公室访问期间使用此工具来帮助医生和患者 在“关键时刻”中讨论每位患者所带来的预期风险和益处 当他们做出治疗决定时，办公室访问。