NIRG: Patient and public INvolvement In target differeNces in Trials (The PINpoINT Study)

NIRG：患者和公众参与试验中的目标差异（PINPOINT 研究）

• 批准号: MR/X011461/1
• 负责人: Beatriz Goulao
• 金额: $ 55.34万
• 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: 2023
• 资助国家: 英国
• 起止时间: 2023 至 无数据
• 项目状态: 未结题
• 来源: https://gtr.ukri.org/projects?ref=MR%2FX011461%2F1
• 关键词: NIRG; Patient; public; INvolvement; target

Clinical trials are considered the best way of testing which treatments are made available in the National Health Service (NHS). Trials are run to benefit patients and so patients should be central to their design. Even though patient and public involvement (PPI) is considered important, patients and the public rarely have a say in key trial numerical aspects (for example, how big of a difference we look for when testing treatments). We aim to co-design new methods and tools to improve the involvement of patients and the public in a key numerical aspect in trial design and interpretation - the target difference. A "normal" (superiority) clinical trial tries to find out whether a new treatment is better than the current treatment. A non-inferiority trial tries to show the new treatment is not worse than the current treatment (but it may have other advantages, like lower cost or better safety). To make a treatment recommendation, new treatments need to be better or good enough by a certain margin. This is called the target difference. Most trials aim to detect a target difference that is important to relevant stakeholders, but they often exclude patients from decision making. For example, we may have a trial testing drug A against drug B to treat pain in patients. To decide if drug A is better than drug B, we will have to define a target difference in the patient reported outcome (pain). In this example pain is measured by asking patients "How much pain do you feel today?" in a scale of 1 (no pain at all) to 10 (a lot of pain). The trial may aim to detect a difference of 2 points in the pain scale after 10 days of being in the trial. This means that if participants in the drug A group had a score of 3 points in the pain scale and participants in the drug B group had a 5.5 score, we would decide that drug A is a better treatment. But is that a worthwhile difference to patients?Currently, target difference decision making is usually based on (informed) guesswork based on a small team's judgement which often excludes public partners (defined here as patients or members of the public part of the research team). Including patients and public partners would ensure trials consider patient important differences when they are designed. This would inform the target difference along with other considerations, like the trial's costs and clinician's views. It would also support the interpretation of trial results that help inform which treatments are available in the NHS. To achieve this, we propose to:- Work with patients and public partners to develop inclusive tools to (a) support a common understanding of target differences in the research team; (b) enable an efficient identification of patient important differences at the trial design stage; and (c) enable a meaningful discussion of existing target differences with public partners ahead of deciding the final target difference for the trial. We will identify and use current practice to support the design of these tools.- Pilot the full process - from identification of available target differences at the start of trial design, to a final meeting with all relevant stakeholders in the trial team including public partners, where consensus about the target difference is reached - Assess the feasibility, and impact of involving patients and public partners in deciding the target difference and draft guidance for trialists and public partners considering the findingsPatients and public partners do not need to have a prior understanding of medical statistics to get involved. The tools will be developed to be understandable and accessible to people with different numeracy levels. Our project has been developed and will be informed by an advisory group including three experienced public partners. Ultimately, the tools developed in the project aim to improve the relevance of trials and reduce research waste by making patients the foundation of clinical research.

临床试验被认为是测试国民保健服务（NHS）提供哪些治疗的最佳方式。试验是为了使患者受益，因此患者应该是试验设计的核心。尽管患者和公众参与（PPI）被认为是重要的，但患者和公众很少在关键的试验数字方面有发言权（例如，我们在测试治疗时寻找多大的差异）。我们的目标是共同设计新的方法和工具，以提高患者和公众在试验设计和解释的关键数字方面的参与-目标差异。“正常”（优效）临床试验试图找出一种新的治疗方法是否比目前的治疗方法更好。非劣效性试验试图证明新的治疗方法并不比目前的治疗方法差（但它可能有其他优势，如成本更低或安全性更好）。为了提出治疗建议，新的治疗方法需要在一定程度上更好或足够好。这就是所谓的目标差异。大多数试验旨在检测对相关利益相关者重要的目标差异，但它们通常将患者排除在决策之外。例如，我们可能有一个试验测试药物A与药物B治疗患者的疼痛。为了确定药物A是否优于药物B，我们必须定义患者报告结局（疼痛）的目标差异。在这个例子中，疼痛是通过询问患者“你今天感觉有多痛？“从1（一点也不疼）到10（很疼）。试验的目的可能是在试验10天后检测疼痛量表中2分的差异。这意味着，如果药物A组的参与者在疼痛量表中得分为3分，药物B组的参与者得分为5.5分，我们将决定药物A是一种更好的治疗方法。但这对患者来说是值得的区别吗？目前，目标差异决策通常是基于（知情的）猜测，基于一个小团队的判断，往往排除公共合作伙伴（定义为患者或研究团队的公共部分的成员）。包括患者和公共合作伙伴将确保试验在设计时考虑患者的重要差异。这将告知目标差异沿着其他考虑因素，如试验成本和临床医生的观点。它还将支持对试验结果的解释，帮助告知NHS提供哪些治疗。为了实现这一目标，我们建议：-与患者和公共合作伙伴合作开发包容性工具，以（a）支持研究团队对目标差异的共同理解;（B）在试验设计阶段有效识别患者重要差异;（c）在决定试验的最终目标差异之前，与公共合作伙伴对现有目标差异进行有意义的讨论。我们将确定并使用当前的实践来支持这些工具的设计。试点整个过程-从试验设计开始时确定可用的目标差异，到与试验团队中的所有相关利益相关者（包括公共合作伙伴）举行最终会议，就目标差异达成共识-评估可行性，以及让患者和公共合作伙伴参与决定目标差异的影响，并为考虑结果的试验者和公共合作伙伴起草指南。公共合作伙伴不需要事先了解医疗统计就可以参与。这些工具将被开发为不同算术水平的人可以理解和使用。我们的项目已经开发，并将由一个咨询小组，包括三个经验丰富的公共合作伙伴提供信息。最终，该项目开发的工具旨在通过使患者成为临床研究的基础来提高试验的相关性并减少研究浪费。