Statistical Methods for Partially Controlled Studies

部分对照研究的统计方法

基本信息

批准号：
6889868
负责人：
CONSTANTINE E FRANGAKIS
金额：
$ 23.84万
依托单位：
JOHNS HOPKINS UNIVERSITY
依托单位国家：
美国
项目类别：
财政年份：
2002
资助国家：
美国
起止时间：
2002-05-01 至 2008-04-30
项目状态：
已结题

来源：
https://reporter.nih.gov/project-details/6889868
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

项目摘要

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）：该项目的目标是开发新的统计设计和方法以更好地评估部分公共卫生方面的对照研究。具体目标有三个。第一个目标是开发评估方法研究中的治疗方法：不直接控制哪些受试者接受哪些治疗治疗，或哪些受试者提供结果；但要控制的位置提供治疗的网站。因为现有的方法，包括标准工具变量，无法处理共存接受治疗和提供结果的不受控制因素、工作目标 1 对于正确的治疗评估至关重要。这项工作将是应用于巴尔的摩正在进行的针头交换项目，以预防艾滋病毒传播。第二个目标是开发临床试验的新方法控制所接受的治疗并衡量结果；一个测量治疗后替代终点；目标是估计治疗对结果的影响是同时发生的和不同时发生的代理人。目标 2 下的方法使用一种新型的分层方法不受控制的替代品，避免了标准的并发症治疗后变量的分层。这项工作对于正确开展工作至关重要评估早期治疗后症状作为后期治疗的措施效果，我们将把它应用到癌症和眼科试验中。第三个目标是开发研究设计方法，其中：一些人退出纵向随访，以及我们在哪里可以找到（双样本）代表辍学的子集。这项工作对于保持普遍性至关重要长期跟踪研究的结果，我们将应用它来更好地评估青少年产生潜在药物依赖的时间。