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.

临床试验被认为是国家卫生服务（NHS）提供哪些治疗方法的最佳测试方法。进行试验以使患者受益，因此患者应该是其设计的核心。即使认为患者和公众参与（PPI）很重要，但患者和公众在关键试验数值方面很少有发言权（例如，测试治疗时我们要寻找的差异多大）。我们旨在共同设计新方法和工具，以在试验设计和解释 - 目标差异中改善患者和公众的参与。 “正常”（优越性）临床试验试图找出新治疗是否比当前治疗更好。一项非效率试验试图表明新疗法并不比当前的治疗差（但它可能具有其他优势，例如成本较低或安全性更好）。为了提出治疗建议，新的治疗需要在一定余量中变得更好或足够好。这称为目标差。大多数试验旨在检测对相关利益相关者很重要的目标差异，但他们经常将患者排除在决策之外。例如，我们可能会进行试验测试药物对药物B的测试，以治疗患者的疼痛。为了确定药物A是否比药物B更好，我们将不得不定义患者报告的结果（疼痛）的目标差异。在此示例中，疼痛是通过问患者“您今天感觉到多少疼痛？”来衡量疼痛。”刻度为1（根本没有疼痛）至10（很多疼痛）。该试验可能旨在在进行试验后10天后检测疼痛量表的差异。这意味着，如果药物的参与者A组在疼痛量表中的得分为3分，而药物B组的参与者的得分为5.5，我们认为药物A是更好的治疗方法。但这对患者有价值吗？目前，目标差异决策通常基于基于小团队的判断（通常不包括公共合作伙伴）的猜测（在此定义为研究小组的患者或公共部位）） 。包括患者和公共合作伙伴将确保在设计时考虑患者的重要差异。这将为目标差异以及其他考虑因素提供信息，例如试验的成本和临床医生的观点。它还将支持对试验结果的解释，以帮助告知NHS中哪些治疗方法。为了实现这一目标，我们建议： - 与患者和公共合作伙伴一起开发包容性工具，以支持（a）对研究团队中目标差异的共同理解； （b）在试验设计阶段有效地识别患者的重要差异； （c）在决定试验的最终目标差异之前，对与公共合作伙伴的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。在达成目标差异达成共识的情况下 - 评估与患者和公共合作伙伴在确定针对试验者和公共合作伙伴的目标差异和草案草案的可行性和影响的情况参与的统计数据。这些工具将被开发为具有不同算术水平的人可以理解和访问。我们的项目已开发，并将由包括三个经验丰富的公共合作伙伴在内的咨询小组告知。最终，该项目中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。