Exploring patient and public perspectives on recruitment and consent in cluster randomised trials involving children

临床试验为改善患者护理提供了重要依据。有两种主要类型的试验，患者随机对照试验（RCT）和集群随机试验（CRT）。两者之间的主要区别在于，在随机对照试验中，个体被随机分配接受干预（例如药物A或药物B）。然而，在随机对照试验中，整个人群（如医院病房）是随机分配的，例如，一个病房接受药物a，另一个病房接受药物b。在随机对照试验中，向患者提供有关试验的信息，包括潜在风险，并在参加试验前征求他们的知情同意。现有的CRT指引订明，在不可能取得同意且对参与者的风险不超过最低限度的试验中，可豁免同意。在crt中获得同意的方法取决于正在调查的内容和在什么水平上。例如，如果干预措施是在媒体形式（如电视、广播、报纸广告、社交媒体）上播放的公共卫生信息，则无需征求知情同意，因为无论如何，社区所有成员都会接受干预措施。因此使用放弃同意。向个人提供的卫生保健干预措施（例如药物或疫苗）原则上可以在征求知情同意的情况下使用随机对照试验进行评估。然而，由于实际情况或成本方面的考虑，可能会设计涉及放弃同意的CRT。虽然放弃同意的CRT似乎是一个适当的设计，但选择整群随机设计和放弃同意都必须是合理的；特别是当crt涉及比随机对照试验更大的患者样本时，效率更低，更容易产生偏倚。从历史上看，CRT的指导来自“专家”的专业意见，而没有深入了解患者和公众的观点。这是一种疏忽，因为如果没有这种认识，目前的招募和同意程序可能不符合患者和家属的需求和观点。例如，我们不知道病人是否能接受在未经他们同意的情况下进入单独的CRT。在涉及弱势群体（如儿童和新生儿）的crt中，深入了解患者和公众的观点尤为重要。这些人群中的crt相对较新，并且具有与患者和父母决策能力相关的特定伦理和同意问题，这可能因环境（例如外科或紧急crt）而异。有必要探索涉及这些弱势群体的患者/家庭和公众对crt的看法，以确保未来的crt在道德上是适当的，并以考虑儿童及其家庭需求的方式进行。这项研究是新颖的。在涉及儿童和新生儿的crt中，我们迫切需要知道：1。在crt中最合适的招募和同意方法是什么？2. 研究人员应该如何与患者和公众就crt的招募和同意（或放弃同意）进行协商，并根据不同类型的试验或患者群体调整这些过程？鉴于这些观点，如何制定crt的指导方针？我们为期30个月的项目将使用既定的社会科学研究方法，包括范围审查，访谈，焦点小组和在线调查，以帮助回答这些重要问题。以儿科和新生儿为重点，我们的研究将包括那些有和没有试验参与经验的人。我们将在考虑伦理原则（如尊重人、尊重人、仁慈和正义）和研究伦理文献的情况下分析收集到的数据，并就这些人群中未来crt的招募和同意方法提出建议。为了帮助确保这项工作具有国际相关性，最后阶段将包括对有关crt的公众和国际专家参与的指导草案进行评估。

Clinical trials provide important evidence to improve patient care. There are two main types of trials, patient randomised controlled trials (RCT) and cluster randomised trials (CRT). The main difference between the two is that in an RCT individuals are randomly allocated to receive an intervention (e.g. drug A or drug B). However, in a CRT a whole group of people (e.g. hospital ward) are randomised, for example, one ward receives drug A and another receives drug B. In an RCT a patient is provided with information about the trial, including potential risks, with their informed consent being sought before taking part. Existing CRT guidance make provision for waiver of consent for the intervention in trials where consent would not be feasible and pose no more than minimal risk to participants.Approaches to consent in CRTs depend upon what is being investigated and at what level. For example, when the intervention is a public health message shown on media formats (e.g. TV, Radio, newspaper adverts, social media) informed consent is not sought because as all members of the community would receive the intervention anyway. A waiver of consent is therefore used. Health care interventions given to an individual person (e.g. a drugs or vaccine) could in principle be evaluated using a RCT with informed consent sought. However, a CRT involving a waiver of consent may be designed due practical, or cost concerns. Although a CRT with a waiver of consent may seem an appropriate design, both the choice of a cluster randomised design and waiver of consent must be justified; particularly as CRTs involve larger patient samples than RCTs, are less efficient and more prone to bias.Historically CRT guidance has been derived from 'expert' professional opinion without insight into patient and public perspectives. This is an oversight as without such insight there is a risk that current recruitment and consent processes may not be in line with the needs and perspectives of patients and family members. For example, we don't know if patients find it acceptable to be entered into an individual CRT without their consent.The need for insight into patient and public perspectives is particularly important in CRTs involving vulnerable populations, such as children and neonates. CRTs in these populations are relatively new and have specific ethical and consent issues related to patient and parental decision making capacity, which may vary by setting (e.g. surgical or emergency CRTs). There is a need to explore patient/family and public perspectives on CRTs involving such vulnerable populations to ensure future CRTs are ethically appropriate and conducted in a way that considers the needs of children and their families. The proposed study is novel. In CRTs involving children and neonates we urgently need to know:1. What are the most appropriate approaches to recruitment and consent in CRTs? 2. How should researchers consult with patients and the public about recruitment and consent (or waiver of consent) in CRTs and adapt such processes for different types of trials or patient populations?3. Given these views, how should guidance for CRTs be formulated?Our 30 month project will use established social science research methods, including scoping review, interviews, focus groups and an online survey to help answer these important questions. Focussing on paediatric and neonatal settings, our research will include those who do and do not have trial participation experience. We will analyse data collected with consideration of ethical principles (such as respect for persons, respect for persons, beneficence and justice) and the research ethics literature to make recommendations on approaches to recruitment and consent in future CRTs in these populations. To help ensure this work is internationally relevant, the final stages will include an evaluation of draft guidance involving the public and international experts on CRTs.