Identifying patient subgroups that are most likely to benefit from medications used to treat alcohol use disorder

确定最有可能从用于治疗酒精使用障碍的药物中受益的患者亚组

• 批准号: 10555243
• 负责人: Joshua David Wallach
• 金额: $ 17.51万
• 依托单位: EMORY UNIVERSITY
• 依托单位国家: 美国
• 财政年份: 2021
• 资助国家: 美国
• 起止时间: 2021-02-01 至 2026-01-31
• 项目状态: 未结题
• 来源: https://reporter.nih.gov/project-details/10555243
• 关键词: Abstinence; Adoption; Affect; Alcohol consumption; Ambulatory Care; Award; Characteristics; Chronic Disease; Clinical Trials; Competence; Conduct Clinical Trials; Data; Data Sources; Databases; Development Plans; Disease; Effectiveness; Environment; Epidemiology; Evaluation; Exclusion Criteria; Frequencies; Goals; Health; Health Personnel; Health Services; Heavy Drinking; Individual; Intervention; Label; Literature; Measures; Medical; Medicare; Medicine; Mentored Research Scientist Development Award; Mentors; Mentorship; Methodology; Methods; National Institute on Alcohol Abuse and Alcoholism; Observational Study; Outcome; Outcome Measure; Patients; Persons; Pharmaceutical Preparations; Pharmacoepidemiology; Privatization; Public Health; Public Health Schools; Publishing; Recommendation; Research; Research Methodology; Research Personnel; Risk; Sample Size; Sampling; Statistical Data Interpretation; Strategic Planning; Subgroup; Techniques; Testing; Time; Training; Uncertainty; United States; United States Food and Drug Administration; Work; World Health Organization; addiction; alcohol response; alcohol use disorder; care outcomes; career development; clinical care; clinical decision-making; clinical practice; drinking; evidence base; hospital care; improved; innovation; multiple data sources; novel; patient subsets; personalized decision; professor; programs; psychosocial; statistical learning

PROJECT SUMMARY/ABSTRACT The objective of this K01 award is to provide Dr. Joshua Wallach, an Assistant Professor of Epidemiology at the Yale School of Public Health, with the protected time necessary to become an independent expert in synthesizing, evaluating, and establishing the best evidence to inform clinical practice in the field of alcohol use disorder (AUD). Although AUD is a common and morbid disease, for which medication treatment is available, uncertainties about whether and for whom treatments work may be causing AUD medication underutilization. To advance the evidence base to support AUD medication treatment, research is needed to identify patient subgroups that are most likely to benefit from each medication used to treat AUD. However, traditional subgroup analyses often consider only one patient characteristic at a time and are conducted in small clinical trials with strict exclusion criteria, short durations, and outcomes that may not reflect how patients feel or function. This proposed K01 will address these limitations by conducting subgroup analyses using multiple data sources, outcomes, and analytical techniques. In Aim 1, Dr. Wallach will identify and assess the validity of already published subgroup analyses to establish promising patient subgroups that are most likely to benefit from each medication used to treat AUD. In Aim 2, existing data from the largest AUD trial in the US (the COMBINE study) will be used to (a) corroborate promising patient subgroup differences identified in Aim 1 and (b) explore new differences between patient subgroups, using multiple methods (i.e. traditional and statistical learning, which can consider multiple characteristics) and outcome measures (abstinence, heavy drinking, and 2-shift reduction in World Health Organization drinking levels). In Aim 3, claims data from OptumLabs, a database of over 150 million privately insured and Medicare Advantage enrollees, will be used to determine whether the same, similar, or new differences between patient subgroups are observed in real-world settings when hospital and ambulatory care outcomes are considered. These aims will clarify uncertainties among patients and clinicians regarding the adoption of AUD medications, which can decrease the public health burden associated with AUD. To support these aims, Dr. Wallach’s career development plan will include training in: (1) AUD medications and addiction medicine, (2) novel statistical learning techniques that can be used to inform individualized clinical decision making, and (3) pharmacoepidemiology and observational research methods for real-world medical product evaluation. The candidate has identified an interdisciplinary team of mentors and advisors, including co-primary mentors Dr. Joseph Ross, a health services researcher and administrative data expert, and Dr. Stephanie O’Malley, an AUD treatment and addiction medicine expert. The Yale Schools of Public Health and Medicine provide the ideal environment for career development. This K01 will prepare Dr. Wallach for an R01 submission, and will facilitate his long-term goal of developing an innovative research program combining meta-research and real-world evaluation to improve AUD clinical care.

项目摘要/摘要 这一K01奖项的目的是为约书亚·沃拉赫博士提供流行病学助理教授 耶鲁大学公共卫生学院，拥有成为独立专家所需的保护时间 为酒精使用领域的临床实践提供信息的最佳证据的合成、评估和建立 精神障碍(澳元)。虽然AUD是一种常见和病态的疾病，药物治疗是可用的， 关于治疗是否有效以及对谁有效的不确定性可能导致AUD药物利用不足。 为了推进支持AUD药物治疗的证据基础，需要进行研究来识别患者 最有可能从治疗AUD的每种药物中受益的亚组。然而，传统的 亚组分析通常一次只考虑一个患者的特征，并在小型临床中进行 试验排除标准严格，持续时间短，结果可能不能反映患者的感受或 功能。建议的K01将通过使用多个 数据来源、结果和分析技术。在目标1中，Wallach博士将识别和评估 已经发表的分组分析，以建立最有可能受益的有前途的患者亚组 从每一种用于治疗澳门氏症的药物中。在AIM 2中，来自美国最大的AUD试验(The 联合研究)将用于(A)证实目标1和目标1中确定的有希望的患者亚组差异 (B)使用多种方法(即传统方法和统计方法)探索患者分组之间的新差异 学习，可以考虑多个特征)和结果衡量标准(禁欲、酗酒和 2--减少世界卫生组织饮酒水平)。在AIM 3中，声称来自OptomLabs的数据，一个 超过1.5亿私人保险和Medicare Advantage投保人的数据库将用于确定 无论患者亚组之间的差异是相同的、相似的还是新的，在现实世界中都可以观察到 当考虑到医院和门诊护理的结果时。这些目标将澄清以下方面的不确定性 患者和临床医生对使用AUD药物的态度，这可能会降低公共健康 与澳元相关的负担。为了支持这些目标，沃拉赫博士的职业发展计划将包括 培训：(1)AUD药物和成瘾药物，(2)新的统计学习技术 用于指导个体化临床决策，以及(3)药物流行病学和观察学 真实世界医疗产品评价的研究方法。候选人已经确定了一个跨学科的 导师和顾问团队，包括共同的小学导师约瑟夫·罗斯博士，一名卫生服务研究员 和行政数据专家，以及斯蒂芬妮·奥马利博士，AUD治疗和成瘾医学专家。 耶鲁大学公共卫生和医学院为职业发展提供了理想的环境。这 K01将为Wallach博士的R01提交做准备，并将促进他开发一种 结合元研究和真实世界评估的创新研究计划，以改善AUD临床护理。