School of Health and Related Research, University of Sheffield, Sheffield, UK.
Centre for Health Economics and Medicines Evaluation, Bangor University, Bangor, UK.
Trials. 2021 Jan 19;22(1):68. doi: 10.1186/s13063-021-05022-0.
Depending on the treatment to be investigated, a clinical trial could be designed to assess objectives of superiority, equivalence or non-inferiority. The design of the study is affected by many different elements including the control treatment, the primary outcome and associated relationships. In some studies, there could be more than one outcome of interest. In these situations, benefit-risk methodologies could be used to assess the outcomes simultaneously and consider the trade-off between the benefits against the risks of a treatment. Benefit-risk is used within the regulatory industry but seldom included within publicly funded clinical trials within the UK. This project aims to gain an expert consensus on how to select the appropriate trial design (e.g. superiority) and when to consider including benefit-risk methods.
The project will consist of four work packages: 1. A web-based survey to elicit current experiences and opinions, 2. A rapid literature review to assess any current recommendations, 3. A two-day consensus workshop to gain agreement on the recommendations, and 4. Production of a guidance document.
The aim of the project is to provide a guideline for clinical researchers, grant funding bodies and reviewers for grant bodies for how to select the most appropriate trial design and when it is appropriate to consider using benefit-risk methods. The focus of the guideline will be on publicly funded trials however, the vision is that the work will be applicable across research settings and we will connect with other organisations and committees as appropriate.
根据待研究的治疗方法，临床试验可以设计为评估优越性、等效性或非劣效性的目标。研究设计受到许多不同因素的影响，包括对照治疗、主要结局和相关关系。在某些研究中，可能有多个感兴趣的结局。在这些情况下，可以使用获益-风险方法来同时评估结局，并考虑治疗的获益与风险之间的权衡。获益-风险在监管行业中使用，但在英国，很少在公共资助的临床试验中包含。该项目旨在就如何选择适当的试验设计（例如优越性）以及何时考虑纳入获益-风险方法达成专家共识。
该项目将包括四个工作包：1. 基于网络的调查，以征求当前的经验和意见；2. 快速文献综述，以评估任何现有建议；3. 为期两天的共识研讨会，以就建议达成一致；4. 编写指南文件。
该项目的目的是为临床研究人员、资助机构和资助机构的评审人员提供如何选择最合适的试验设计以及何时考虑使用获益-风险方法的指南。该指南的重点将是公共资助的试验，但我们的愿景是该工作将适用于所有研究环境，并将与其他组织和委员会进行适当的联系。
