Statistical Methods for Partially Controlled Studies

部分对照研究的统计方法

• 批准号: 6464181
• 负责人: CONSTANTINE E FRANGAKIS
• 金额: $ 25.75万
• 依托单位: JOHNS HOPKINS UNIVERSITY
• 依托单位国家: 美国
• 财政年份: 2002
• 资助国家: 美国
• 起止时间: 2002-05-01 至 2006-04-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/6464181
• 关键词: analytical method; clinical research; clinical trials; data collection methodology /evaluation; data management; health science research; health science research analysis /evaluation; human data; longitudinal human study; mathematical model; meta analysis; method development; public health; statistics /biometry

DESCRIPTION (provided by applicant): The objective of this project is to develop new statistical designs and methods to better evaluate partially controlled studies in public health. There are three specific aims. The first aim is to develop methods to evaluate treatments in studies that: do not directly control which subjects get which treatment, or which subjects provide outcomes; but do control the location of the sites that provide the treatments. Because existing methods, including standard instrumental variables, cannot handle the coexistence of the uncontrolled factors of taking treatment and of providing outcomes, the work under aim 1 is essential for proper treatment evaluation. This work will be applied to ongoing needle exchange programs in Baltimore, for preventing HIV transmission. The second aim is to develop new methods for clinical trials where treatment received is controlled, and outcomes are measured; a post-treatment surrogate endpoint is measured; and the goal is to estimate the treatments effects on the outcome that are concurrent and not concurrent with the surrogate. The methods under aim 2 use a new type of stratification for the uncontrolled surrogates that avoid the complications of standard stratifications on post-treatment variables. This work is essential for proper evaluation of early post-treatment symptoms as measures of later treatment effect, and we will apply it to cancer and ophthalmology trials. The third aim is to develop methods for study designs where: some persons drop out of a longitudinal follow-up, and where we can find (double-sample) a representative subset of the dropouts. This work is essential for preserving generalizability of results from long follow-up studies, and we will apply it to better evaluate time to potential drug dependence in youths.

描述（由申请人提供）：该项目的目的是 开发新的统计设计和方法来更好地评估 公共卫生的对照研究。 有三个特定的目标。第一个目的是开发评估的方法 研究中的治疗方法：不直接控制哪些受试者得到哪些 治疗或哪些受试者提供结果；但请控制 提供治疗的站点。因为现有方法，包括 标准仪器变量，无法处理 接受治疗和提供结果的不受控制的因素 在AIM 1下，对于适当的治疗评估至关重要。这项工作将是 适用于巴尔的摩正在进行的针交易程序，以防止艾滋病毒 传播。第二个目的是开发用于临床试验的新方法 控制接受治疗的地方并测量结果；一个 测量治疗后替代端点；目标是估计 治疗对结果的影响 代理。 AIM 2下的方法使用新类型的分层 避免标准并发症的不受控制的替代物 治疗后变量的分层。这项工作对于适当至关重要 评估早期治疗后症状作为治疗的措施 效果，我们将其应用于癌症和眼科试验。第三个目标 是开发研究设计的方法：有些人退出 纵向随访，我们可以找到（双样本）代表 辍学子集。这项工作对于保持普遍性至关重要 长期随访研究的结果，我们将其应用于更好地评估 是在青年人中潜在毒品依赖的时间。