Assessing methods to specify the targeted difference for a randomised controlled trial

评估指定随机对照试验目标差异的方法

• 批准号: G0902147/1
• 负责人: Jonathan Cook
• 金额: $ 19.48万
• 依托单位: University of Aberdeen
• 依托单位国家: 英国
• 项目类别: Research Grant
• 财政年份: 2010
• 资助国家: 英国
• 起止时间: 2010 至 无数据
• 项目状态: 已结题
• 来源: https://gtr.ukri.org/projects?ref=G0902147%2F1
• 关键词: Assessing; methods; specify; targeted; difference

Increasingly it is acknowledged that decisions about what health care to provide should be evidence based. One part of the evidence needed is whether the intervention is a beneficial and worthwhile use of the limited money available to provide health care. Arguably the best way of providing this evidence is by conducting a randomised controlled trial. In a randomised controlled trial individuals, or groups of individuals, are typically randomly assigned to receive either the new treatment (which might be the way in which care is provided) or an alternative treatment. A sufficient number of people is needed so that we can be confident that if a difference between treatments exists we can detect it. The size of difference between the two treatments desired to be identified (the target difference) affects the number of people needed to be recruited and the cost of the study. If the target difference is not correctly identified then there is a chance that time, effort (by patients, health care practitioners and researchers) and money will be wasted or poorly used. It is also possible that the health of patients may be adverse affected because the target difference was too small and we carry on randomising people to a treatment that we should already be able to say is inferior. Alternatively, if the target is too large we may fail to detect a truly important difference and as a result delay or prevent the introduction of a worthwhile treatment.In this study we will systematically identify methods of determining the target difference. Scientific papers will be identified that described different methods, experts in the field will be approached, and we will survey organisations and individuals who are actively involved in the design and conduct of randomised controlled tiles. The different methods that we identify will be described, strengths and weaknesses appraised and potential scope for their use assessed. We will develop draft guidance for researchers and funders of randomised clinical trials. This will include separate sections for different types of trials and on different ways in which the outcomes of a treatment might be measured. This guidance will first be discussed amongst the study applicants and an expert advisory group. The recommendations will then presented at a symposium to which those with an interest in the design and conduct of trials and will help further refine recommendations and act as a first method of dissemination.

越来越多的人认识到，关于提供什么样的医疗保健的决定应该以证据为基础。所需证据的一部分是，干预是否是对提供医疗保健的有限资金的有益和值得的使用。可以说，提供这一证据的最佳方法是进行随机对照试验。在随机对照试验中，个体或个体组通常被随机分配接受新治疗（可能是提供护理的方式）或替代治疗。需要有足够数量的研究对象，这样我们才能确信，如果两种治疗方法之间存在差异，我们可以检测到它。需要确定的两种治疗方法之间差异的大小（目标差异）会影响需要招募的人数和研究成本。如果目标差异没有被正确识别，那么时间、精力（患者、医疗保健从业者和研究人员）和金钱就有可能被浪费或利用不当。患者的健康也可能受到不利影响，因为目标差异太小，我们继续将人们随机分配到我们应该已经能够说是劣效的治疗。或者，如果目标太大，我们可能无法检测到一个真正重要的差异，因此延迟或阻止引入一个有价值的treatment.In这项研究中，我们将系统地确定方法的目标差异。将确定描述不同方法的科学论文，将与该领域的专家联系，我们将调查积极参与随机控制瓷砖设计和实施的组织和个人。我们确定的不同方法将被描述，优点和缺点的评价和潜在的使用范围进行评估。我们将为随机临床试验的研究人员和资助者制定指南草案。这将包括不同类型试验的单独章节，以及衡量治疗结果的不同方法。本指南将首先在研究申请人和专家咨询小组之间进行讨论。这些建议随后将在一次专题讨论会上提出，对试验的设计和进行感兴趣的人将在会上发言，这将有助于进一步完善建议，并作为第一个传播方法。