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

基本信息

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
项目类别：
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

项目摘要

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.

项目概要/摘要该 K01 奖项的目的是提供约书亚·瓦拉赫 (Joshua Wallach) 博士，他是该大学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受保护时间综合、评估和建立最佳证据，为酒精使用领域的临床实践提供信息尽管 AUD 是一种常见的病态疾病，可以进行药物治疗，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，需要进行研究以确定患者最有可能从用于治疗 AUD 的每种药物中受益的亚组。亚组分析通常一次仅考虑一名患者的特征，并且在小型临床中进行具有严格标准排除、持续时间短且结果可能无法反映患者感受或感受的试验所提出的 K01 函数将通过使用多个进行亚组分析来解决这些限制。在目标 1 中，Wallach 博士将确定并评估数据来源、结果和分析技术的有效性。已经发表的亚组分析，以确定最有可能受益的有希望的患者亚组在目标 2 中，来自美国最大的 AUD 试验的现有数据。 COMBINE 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）探索患者亚组之间的新差异学习，可以考虑多种特征）和结果测量（禁欲、酗酒和在目标 3 中，OptumLabs 的数据声称，世界卫生组织的饮酒水平降低了 2 个班次。超过 1.5 亿私人保险和 Medicare Advantage 参保者的数据库将用于确定在现实世界中是否观察到患者亚组之间存在相同、相似或新的差异当考虑医院和门诊护理结果时，这些目标将澄清之间的不确定性。患者和关于采用 AUD 药物，这可能会降低公众健康为了支持这些目标，Wallach 博士的职业发展计划将包括培训内容：(1) AUD 药物和成瘾医学，(2) 新颖的统计学习技术用于为个体化临床决策提供信息，以及（3）药物流行病学和观察候选人已经确定了一种跨学科的现实世界医疗产品评估的研究方法。导师和顾问团队，包括共同主要导师 Joseph Ross 博士，一位健康服务研究员行政数据专家，以及 AUD 治疗和成瘾医学专家 Stephanie O’Malley 博士。耶鲁大学公共卫生与医学院为职业发展提供了理想的环境。 K01 将为 Wallach 博士准备 R01 提交，并将促进他开发一个结合元研究和现实世界评估的创新研究计划，以改善 AUD 临床护理。