A Meta-Epidemiological Assessment of the Role of Pilot Studies in the Design of Well-Powered Trials - the Pilot Project

对试点研究在设计有力试验中的作用进行元流行病学评估 - 试点项目

• 批准号: 10213827
• 负责人: MICHAEL W BEETS
• 金额: $ 58.7万
• 依托单位: UNIVERSITY OF SOUTH CAROLINA AT COLUMBIA
• 依托单位国家: 美国
• 财政年份: 2019
• 资助国家: 美国
• 起止时间: 2019-07-15 至 2023-06-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/10213827
• 关键词: AIDS/HIV problem; Address; Applications Grants; Attention; Catalogs; Complex; Data; Development; Epidemiology; Funding; Funding Mechanisms; Future; Government; Guidelines; Health; Institutes; Intervention; Intervention Studies; Intervention Trial; Interview; Journals; Lead; Literature; Medical Research; Methodology; Methods; Phase; Pilot Projects; Play; Probability; Process; Public Health; Randomized; Reporting; Research; Research Design; Role; Sampling; Social Sciences; Surveys; Testing; Tobacco; Treatment Efficacy; United Kingdom; United States National Institutes of Health; Validation; adult obesity; base; behavioral health intervention; design; efficacy testing; improved; innovation; obesity in children; pilot trial; public health intervention; randomized trial; success; systematic review; therapy design

Pilot intervention studies (aka feasibility or preliminary studies) play an indispensable, fundamental role in the development, refinement, and dissemination of social science/public health interventions. In pilot studies, preliminary evidence on important processes and potential efficacy of an intervention are collected. Despite their prominence in the development and funding of almost every well-powered randomized intervention, pilot studies have received very little attention regarding how they should be designed so that the study’s findings will provide information to inform decisions about further testing and refining of an intervention. Other guidelines, such as CONSORT, TREND, SPIRIT, or TIDeR, focus on factors associated with internal validity or transparency/replication, and fail to address substantive issues interventionists need to consider during the early stages of testing an intervention, such as what is delivered, who its delivered to, and the intensity of support for delivery and whether these can be scaled in a larger, well-powered trial. Based on our preliminary findings, these issues, which we refer to as “generalizability biases,” are a few of the hypothesized emerging factors that lead to “false-positive or exaggerated early discoveries” and subsequent failed well-powered trials. The identification and avoidance of generalizability biases can guide intervention decisions during the early testing so that, according to the NIH, a pilot study’s results can “…enhance the probability of obtaining meaningful results in subsequent well-powered trials.” In the proposed study, we will conduct a comprehensive systematic review and meta-epidemiological assessment of the role pilot studies play in the testing and scaling of interventions. This information will be used to inform the field of scientific practice on how to design more informative pilot studies that can increase the probability of success of interventions in well-powered trials. For this study, we will use an innovative multi-phase, cross-validation approach to develop a working set of generalizability biases interventionists should avoid when conducting preliminary tests of an intervention. In Phase 1 we will identify, define, and catalogue candidate generalizability biases within a large body of pilot intervention studies (N = 740) on the topic of child obesity. In Phase 2 we will cross-validate the biases, refine them, and identify new ones in a sample of pilot intervention studies on the topic of adult obesity (anticipated sample of 800 studies). In Phase 3 we will perform a double cross-validation of the biases in a new sample of pilot interventions on the topics of tobacco and HIV/AIDS (anticipated sample of 400 studies each). In Phase 4, a working draft of the principles will be refined via Delphi survey with leading Journal Editors. This study is significant because it will be the first to systematically develop guidance for intervention pilot studies. This study is innovative because it will address a topic that has largely been ignored (pilot studies) and will synthesize both quantitative and qualitative data to understand how pilot studies can be improved – an essential, yet overlooked aspect of developing high-quality interventions.

试点干预研究(又名可行性或初步研究)在以下方面发挥着不可或缺的基础作用 发展、完善和传播社会科学/公共卫生干预措施。在试点研究中， 收集关于干预的重要过程和潜在效果的初步证据。尽管 他们在开发和资助几乎每一项有效的随机干预、试点方面的突出地位 关于研究应该如何设计，以便研究结果，很少受到关注 将提供信息，为进一步测试和完善干预措施的决策提供信息。其他 指导方针，如配偶、趋势、精神或等级，侧重于与内部有效性或 透明度/可复制性，未能解决干预者在 测试干预的早期阶段，例如提供什么、提供给谁以及干预的强度 对交付的支持，以及这些支持是否可以在更大、功能强大的试验中进行扩展。根据我们的初步调查 发现，这些问题，我们称之为“泛化偏差”，是假想出现的几个问题 导致“假阳性或夸大早期发现”以及随后的动力充足的试验失败的因素。 识别和避免概括性偏差可以指导早期的干预决策 根据美国国立卫生研究院的说法，测试这样一项先导研究的结果就可以“…提高获得 在随后的强有力的试验中取得了有意义的结果。在建议的研究中，我们会全面研究 对试点研究在测试和评估中所起作用的系统审查和元流行病学评估 干预的结果。这些信息将被用来向科学实践领域提供如何设计更多 信息丰富的先导性研究，可以增加在动力充足的试验中干预成功的可能性。为 在这项研究中，我们将使用创新的多阶段交叉验证方法来开发一套工作集 干预者在进行干预的初步测试时应避免泛化偏差。在……里面 第1阶段，我们将识别、定义和分类大量试点中的候选概括性偏差 以儿童肥胖为主题的干预研究(N=740)。在阶段2中，我们将交叉验证偏差、细化 并在以成人肥胖(预期)为主题的先导干预研究样本中确定新的 800项研究的样本)。在第三阶段，我们将对新样本中的偏差进行双重交叉验证 关于烟草和艾滋病毒/艾滋病专题的试点干预措施(预期抽样各400项研究)。在第四阶段， 原则的工作草案将通过德尔福与主要期刊编辑的调查进行完善。这项研究是 这具有重大意义，因为它将是第一个系统地制定干预试点研究指南的项目。这 这项研究具有创新性，因为它将解决一个在很大程度上被忽视的主题(试点研究)，并将 综合定量和定性数据，以了解如何改进试点研究- 制定高质量干预措施的关键但被忽视的方面。