Exploring Therapeutic Misconception and Increasing Wellbeing Related to Clinical

描述（由申请人提供）：治疗误解（TM）可能导致对肌营养不良临床试验结果的不切实际的期望。无论是否获得知情同意，希望、承诺和期望都会影响信息的解释。目前尚不清楚TM是否代表违反同意程序和/或它们是否可能代表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以及如何修改TM，以及对参与者的健康有多大影响。该项目将参与“杜氏/贝克尔肌营养不良社区”（父母，受影响的青少年和成人，临床研究人员和生物制药领导人），以探索在考虑和参与临床试验时治疗误解的适应性和适应不良因素。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是根据各种经验和看法确定的。其结果将是一个相当大的贡献，有关TM的文献，包括努力制定措施，以评估TM;和干预目标的发展是：关注福祉，而不是消灭TM，是可以接受的社区，并可以在下游干预研究进行测试。具体而言，本研究旨在采用定性方法，从家庭和研究人员获得TM的概念，范围和来源的深入评估，以及TM的适应性和适应不良的元素。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。这些研究的数据将用于创建一个定量调查，以获得有关TM的一般信息。我们会进一步邀请社会各界参与，在评估和参与临床试验时，利用这三项分析所得的数据，制订一套可行的目的，并设计干预措施，以减少TM的适应不良和负面后果，并改善家庭福祉

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