Design Considerations for Two-Phase Studies

两阶段研究的设计注意事项

• 批准号: 8193351
• 负责人: SEBASTIEN HANEUSE
• 金额: $ 6.41万
• 依托单位: HARVARD SCHOOL OF PUBLIC HEALTH
• 依托单位国家: 美国
• 财政年份: 2009
• 资助国家: 美国
• 起止时间: 2009-09-01 至 2011-08-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8193351
• 关键词: Design; Considerations; Two; Phase; Studies

In identifying associations between exposures and diseases researchers have a wide variety of study designs at their disposal. Although two-phase studies have been shown to provide substantial efficiency gains over the traditional case-control design, they have not been widely adopted. A recent survey of 4,792 studies in five top-line epidemiological/medical journals, published since 2002, found just a single study having employed the two-phase design. This may be due, in part, to a lack of published guidance on how to design and plan a two-phase study and the practical difficulties of not having general-purpose software. To address these issues and broad the use of two-phase studies, this research proposes to develop a flexible framework for investigating design considerations in the context of planning a two-phase study. This will involve two steps; (i) development of a conceptual framework which encompasses the decisions required at the planning stage and (ii) developing an algorithmic simulation-based framework which facilitates the investigation of potential choices in a variety of settings. Building on this, we propose to conduct an extensive and comprehensive simulation study to explore the impact of various choices associated with the design of a two-phase study. This proposal is motivated by ongoing and future research conducted by the Breast Cancer Surveillance Consortium (BCSC). As new opportunities for scientific research within the BCSC arise it will be important to make the best possible use of the existing database/infrastructure. The two-phase design provides a framework to achieve this, and improved guidance on the planning and design of such studies will be crucial for future cost-effective and yet statistically powerful studies. A key component of this work, therefore, will be the dissemination of methods and results, and the delivery of software; algorithms/code, together with documentation, will be developed in commonly used statistical packages and made publicly available.

在确定暴露与疾病之间的关联时，研究人员可以使用多种研究设计。尽管两阶段研究已被证明可以比传统病例对照设计提供显着的效率提升，但它们尚未被广泛采用。最近对自 2002 年以来发表的五种顶级流行病学/医学期刊上的 4,792 项研究进行的调查发现，只有一项研究采用了两阶段设计。这可能部分是由于缺乏关于如何设计和规划两阶段研究的已发表指南以及没有通用软件的实际困难。为了解决这些问题并广泛使用两阶段研究，本研究建议开发一个灵活的框架，用于在规划两阶段研究的背景下调查设计考虑因素。这将涉及两个步骤； (i) 开发一个概念框架，其中包含规划阶段所需的决策；(ii) 开发一个基于算法模拟的框架，以方便调查各种环境中的潜在选择。在此基础上，我们建议进行广泛而全面的模拟研究，以探讨与两阶段研究设计相关的各种选择的影响。该提案是由乳腺癌监测联盟 (BCSC) 正在进行和未来进行的研究推动的。随着 BCSC 内科学研究的新机会出现，充分利用现有数据库/基础设施将变得非常重要。两阶段设计提供了实现这一目标的框架，并且改进对此类研究的规划和设计的指导对于未来具有成本效益且统计上强大的研究至关重要。因此，这项工作的一个关键组成部分将是方法和结果的传播以及软件的交付；算法/代码以及文档将在常用的统计包中开发并公开发布。