Exploring Therapeutic Misconception and Increasing Wellbeing Related to Clinical

描述(由申请人提供)：治疗误区(TM)可能导致在肌肉营养不良的临床试验中对结果的不切实际的期望。无论是否实现了知情同意，希望、承诺和期望都会影响对信息的解读。目前尚不清楚多发性硬化症是否违反了同意程序，和/或它们是否可能是应对严重危及生命的疾病预后的有效方法。我们不知道我们可以在多大程度上和如何修改TM，也不知道以什么代价来改变参与者的福祉。该项目将邀请“Duchenne/Becker肌营养不良症社区”(父母、受影响的青少年和成年人、临床医生研究人员和生物制药领导者)在考虑和参与临床试验时探索治疗误解中的适应性和非适应性因素。这项研究将采用以社区为基础的参与性研究方法，以帮助确保问题和解释的范围由一系列不同的经验和看法提供信息。这一结果将对TM的文献做出相当大的贡献，包括努力制定评估TM的措施；以及制定干预目标(S)：关注TM的福祉而不是消灭TM，社区可以接受，并且可以在下游干预研究中进行测试。具体地说，这项研究的目的是从家庭和研究人员那里获得对TM的概念、范围和来源的深入评估，以及TM的适应性和非适应性因素。同时，我们将分析有关临床试验的书面信息来源，以确定治疗误解的潜在来源。这些研究的数据将被用来创建一项量化调查，以获得关于TM的概括性信息。我们将进一步动员社会各界利用三项分析的数据，制定一套可行的目标，并设计一种干预措施，在评估和参与临床试验时减少TM的不良适应和负面后果，并改善家庭福祉。 与公共卫生相关：治疗误区(TM)可能导致对肌营养不良症未来临床试验结果的不切实际的期望。目前尚不清楚经皮肾移植在多大程度上违反了知情同意程序，和/或经皮移植是否可能是应对严重、危及生命的疾病预后的有效方法。该项目包括定性和定量研究，以了解TM的范围、来源和性质，并将对TM的文献和干预目标(S)的开发做出相当大的贡献，这些目标是：专注于福祉而不是消灭TM，社区可以接受，并且可以在下游干预研究中进行测试。

DESCRIPTION (provided by applicant): The therapeutic misconception (TM) may lead to unrealistic expectations of outcomes in clinical trials for muscular dystrophies. Regardless of whether informed consent is achieved, hope, promise and expectations all influence how information is interpreted. It is unclear whether TMs represent a breach of the consent process and/or whether they may represent effective ways of coping with the prognosis of serious, life-threatening conditions. We do not know to what extent and how we can modify TM, and at what cost to participant wellbeing. This project will engage the "Duchenne/Becker muscular dystrophy community" (parents, affected adolescents and adults, clinician investigators, and biopharmaceutical leaders) to explore adaptive and maladaptive elements of the therapeutic misconception when considering and participating in a clinical trial. The study will use a community-based participatory research approach to help ensure that the scope of questions and interpretation is informed by a diverse set of experiences and perceptions. The result will be a considerable contribution to the literature about TM, including efforts to develop measures to evaluate TM; and the development of target(s) for intervention that are: focused on wellbeing rather than extinguishing TM, are acceptable to the community, and can be tested in a downstream intervention study. Specifically, the study aims to use qualitative approaches to obtain from families and researchers an in-depth assessment of the concept, scope, and sources of TM, as well as adaptive and maladaptive elements of TM. Concurrently we will analyze written sources of information about clinical trials to identify potential sources of therapeutic misconceptions. The data from those studies will be used to create a quantitative survey to obtain generalizable information about TM. We will further engage the community to use the data from the three analyses to develop a set of feasible targets and design an intervention that would reduce maladaptive and negative outcomes of TM and improve family wellbeing when evaluating and participating in clinical trials PUBLIC HEALTH RELEVANCE: The therapeutic misconception (TM) may lead to unrealistic expectations of outcomes in future clinical trials for muscular dystrophies. It is unclear to what extent TMs represent a breach of the informed consent process, and/or whether TM may represent an effective way of coping with the prognosis of serious, life-threatening conditions. This project includes qualitative and quantitative studies to inform about the scope, sources, and nature of TM and will result in a considerable contribution to the literature about TM and the development of target(s) for intervention that are: focused on wellbeing rather than extinguishing TM, acceptable to the community, and can be tested in a downstream intervention study.