Imperial Clinical Trials Unit, Imperial College London, London, UK.
MRC Clinical Trials Unit at UCL, London, UK.
Trials. 2023 Jul 6;24(1):443. doi: 10.1186/s13063-023-07469-9.
Clinical trials aim to draw conclusions about the effects of treatments, but a trial can address many different potential questions. For example, does the treatment work well for patients who take it as prescribed? Or does it work regardless of whether patients take it exactly as prescribed? Since different questions can lead to different conclusions on treatment benefit, it is important to clearly understand what treatment effect a trial aims to investigate-this is called the 'estimand'. Using estimands helps to ensure trials are designed and analysed to answer the questions of interest to different stakeholders, including patients and public. However, there is uncertainty about whether patients and public would like to be involved in defining estimands and how to do so. Public partners are patients and/or members of the public who are part of, or advise, the research team. We aimed to (i) co-develop a tool with public partners that helps explain what an estimand is and (ii) explore public partner's perspectives on the importance of discussing estimands during trial design.
An online consultation meeting was held with 5 public partners of mixed age, gender and ethnicities, from various regions of the UK. Public partner opinions were collected and a practical tool describing estimands, drafted before the meeting by the research team, was developed. Afterwards, the tool was refined, and additional feedback sought via email.
Public partners want to be involved in estimand discussions. They found an introductory tool, to be presented and described to them by a researcher, helpful for starting a discussion about estimands in a trial design context. They recommended storytelling, analogies and visual aids within the tool. Four topics related to public partners' involvement in defining estimands were identified: (i) the importance of addressing questions that are relevant to patients and public in trials, (ii) involving public partners early on, (iii) a need for education and communication for all stakeholders and (iv) public partners and researchers working together.
We co-developed a tool for researchers and public partners to use to facilitate the involvement of public partners in estimand discussions.
临床试验旨在得出关于治疗效果的结论，但一项试验可以解决许多不同的潜在问题。例如，该治疗方法对按规定服用的患者效果如何？或者，无论患者是否完全按规定服用，它是否有效？由于不同的问题可能会导致治疗效果的不同结论，因此，明确了解试验旨在研究哪种治疗效果非常重要——这被称为“目标治疗效应”。使用目标治疗效应有助于确保试验的设计和分析能够回答不同利益相关者（包括患者和公众）感兴趣的问题。然而，目前尚不确定患者和公众是否希望参与定义目标治疗效应以及如何参与。公众合作伙伴是参与或为研究团队提供建议的患者和/或公众成员。我们的目标是：（i）与公众合作伙伴共同开发一种工具，帮助解释目标治疗效应是什么；（ii）探索公众合作伙伴对在试验设计过程中讨论目标治疗效应的重要性的看法。
我们与来自英国不同地区、具有不同年龄、性别和种族的 5 名公众合作伙伴举行了一次在线咨询会议。收集了公众合作伙伴的意见，并由研究团队在会前起草了一份描述目标治疗效应的实用工具。之后，对该工具进行了完善，并通过电子邮件征求了额外的反馈意见。
公众合作伙伴希望参与目标治疗效应的讨论。他们发现，研究人员展示和描述的一个入门工具有助于在临床试验设计背景下开始讨论目标治疗效应。他们建议在工具中使用讲故事、类比和视觉辅助手段。确定了与公众合作伙伴参与定义目标治疗效应相关的四个主题：（i）在试验中解决与患者和公众相关的问题的重要性，（ii）尽早让公众合作伙伴参与，（iii）需要对所有利益相关者进行教育和沟通，以及（iv）公众合作伙伴和研究人员共同合作。
我们共同开发了一种研究人员和公众合作伙伴都可以使用的工具，以促进公众合作伙伴参与目标治疗效应的讨论。
