Exploratory trials of complex public health interventions: development of guidance for researchers and funders

复杂公共卫生干预措施的探索性试验：为研究人员和资助者制定指南

• 批准号: MR/N015843/1
• 负责人: Laurence Moore
• 金额: $ 31.15万
• 依托单位: University of Glasgow
• 依托单位国家: 英国
• 项目类别: Research Grant
• 财政年份: 2016
• 资助国家: 英国
• 起止时间: 2016 至 无数据
• 项目状态: 已结题
• 来源: https://gtr.ukri.org/projects?ref=MR%2FN015843%2F1
• 关键词: Exploratory; trials; complex; public; health

Recognition is growing of the potential public health value of interventions to improve health behaviours such as diet, physical activity and substance use. Given the complex causality of these behaviours, improving public health often requires complex interventions which comprise multiple components interacting to produce change, with their actions constrained and facilitated by their interaction with the settings into which they are introduced. Given the cost of such interventions and the potential for unintended harms, drawing on evidence of what works and rigorously evaluating effectiveness is crucial.Where feasible, randomised controlled trials (RCTs) represent the most robust means of examining effectiveness. However, complex interventions are often difficult to implement in a manner consistent with programme theory across multiple settings and target groups, whilst conducting and interpreting RCTs in real word settings is challenging. Hence, prior to committing to definitive evaluation, randomised or otherwise, attention is needed to establishing the feasibility and acceptability of intervention plans and intended evaluation designs.The need to carefully examine these issues is increasingly recognised, with an increasing number of studies describing an objective of establishing the feasibility of full scale trials. Such studies are described under terms including 'exploratory trials', 'phase 2 trials' or 'piloting and feasibility' studies. However, there is currently a lack of consistent guidance for researchers preparing bids for such work, or for peer reviewers or funders, on the most important questions to address in such studies, key methodological considerations in addressing these questions, when it is more or less appropriate to use randomisation during this phase, and the criteria on which decisions should be made regarding whether and how to progress to a full scale definitive evaluation. Hence, as reflected by the range of terms under which such studies are described, their conduct and reporting is inconsistent, and the basis on which conclusions regarding whether or not to progress to a full-scale evaluation are made often unclear.The proposed study will develop guidance for the conduct of exploratory trials through reviewing current practice and eliciting views of expert funders and researchers, beginning by using systematic review methodology to identify studies describing an intention to investigate the potential for a full scale trial (including phase 2 trials, exploratory trials or feasibility and pilot studies) and existing guidance on their conduct. We will also conduct a horizon-scanning exercise to identify recent methodological developments in the design of phase 2 trials and pilot and feasibility studies in other areas of clinical research, to ensure that the guidance learns from recent developments in these related areas.A web-based Delphi process and consensus meeting will then be used to develop guidance on when exploratory trials should be conducted, what research questions they should address, key methodological considerations in the conduct of such studies and the range of criteria that should inform a decision to proceed to a full scale definitive trial. The guidance will then be actively disseminated to research funders, researchers and journal editors to maximise its impact on the commissioning, conduct and reporting of exploratory trials of complex public health interventions.

人们日益认识到改善饮食、身体活动和药物使用等健康行为的干预措施的潜在公共卫生价值。鉴于这些行为的复杂因果关系，改善公共卫生往往需要复杂的干预措施，这些干预措施包括相互作用以产生变化的多个组成部分，其行动受到它们与引入它们的环境的相互作用的制约和促进。考虑到此类干预措施的成本和潜在的意外危害，收集有效措施的证据并严格评估有效性至关重要。在可行的情况下，随机对照试验（RCTs）是检验有效性的最可靠的方法。然而，复杂的干预措施往往难以在多种环境和目标群体中以与项目理论一致的方式实施，而在现实世界环境中进行和解释随机对照试验是具有挑战性的。因此，在进行最终评估（随机或非随机）之前，需要注意确定干预计划和预期评估设计的可行性和可接受性。随着越来越多的研究描述了建立全面试验可行性的目标，人们越来越认识到需要仔细检查这些问题。这些研究被描述为“探索性试验”、“第二阶段试验”或“试点和可行性”研究。然而，对于准备投标这类工作的研究人员、同行评审人员或资助者，目前缺乏一致的指导，包括在这类研究中需要解决的最重要问题、解决这些问题时的关键方法学考虑、在这一阶段使用随机化是否合适以及如何进行全面确定评估的决策标准。因此，正如描述这些研究所用的各种术语所反映的那样，它们的行为和报告是不一致的，关于是否进展到全面评价的结论所依据的基础往往不明确。拟议的研究将通过审查目前的实践和征求专家资助者和研究人员的意见，为探索性试验的开展制定指导方针，首先使用系统的审查方法来确定那些描述了调查全面试验潜力的意图的研究（包括2期试验、探索性试验或可行性和试点研究），以及对其开展的现有指导。我们还将开展一项横向扫描工作，以确定第二阶段试验设计的最新方法学发展，以及其他临床研究领域的试点和可行性研究，以确保指南借鉴这些相关领域的最新发展。然后，基于网络的德尔菲过程和共识会议将用于制定指导意见，包括何时进行探索性试验，应解决哪些研究问题，进行此类研究时的关键方法学考虑因素，以及应决定进行全面确定试验的标准范围。然后，该指南将积极传播给研究资助者、研究人员和期刊编辑，以最大限度地影响复杂公共卫生干预措施探索性试验的委托、实施和报告。

项目成果

Exploratory studies to decide whether and how to proceed with full-scale evaluations of public health interventions: a systematic review of guidance.

• DOI: 10.1186/s40814-018-0290-8
• 发表时间: 2018
• 期刊: Pilot and feasibility studies
• 影响因子: 1.7
• 作者: Hallingberg B;Turley R;Segrott J;Wight D;Craig P;Moore L;Murphy S;Robling M;Simpson SA;Moore G
• 通讯作者: Moore G