Common Daily Practices of Gene Therapy Clinical Research

描述（由申请人提供）：人们对基因治疗研究的安全性和科学价值高度乐观和关注。当临床实践出现问题时，不应该对个别病例、机构或pi违反诚信进行耸人听闻的报道，而应该有可能发现如何防止意外事故的发生。研究人员的目标是非常详细地阐明现实世界中被视为理所当然的日常操作和决定，这些操作和决定与基因治疗临床试验和监管文书工作有关。没有人真正看到工作如何完成的全貌，谁做什么，他们如何相互作用，或者组织系统或卫生保健政策如何影响研究诚信。这项制度人种学研究的目的之一是确定和描述共同的做法、系统和工具，这些做法、系统和工具同时在多种环境中起作用，同时也存在使基因治疗的实施具有挑战性和困难的障碍。另一个目的是描述促进、保护或保护研究诚信的不同类型的机构文化，以及那些不这样做的机构文化。三种文化人种学将在三个州进行，在三种类型的基因治疗临床研究伙伴关系。实地记录是3位民族志学家的非参与性观察的主要数据来源，他们在这个环境中观察，断断续续，但定期，长达18个月。人种学家将采访尽可能多的利益相关者，包括员工和病人。采访被逐字记录，现场记录被重写和解释。这两种类型的数据将使用传统的文化人种学方法进行分析。分析将由3名共同调查人员和3名专家顾问进行。所有数据以汇总方式报告；没有一个单一的背景或机构被确定或突出。将制作两种类型的手稿。面向公众的一份将是对日常临床实践中发生的所有复杂性、挑战和制约因素的清晰、详尽的描述，而这些都是联邦政策制定者所忽视的。二是对基因治疗临床研究规范和实践的学术解读。通过了解多个机构位置（研究、大学、临床、实验室和监管机构）中每个利益相关者的角色；我们有可能看到进行基因治疗研究到底有多复杂。这项研究的结果将引起人们对最佳实践标准和教育的关注。其目的是建立公众信任，促进患者参与未来的研究，并聚焦合作临床实践的典范。这项研究对改变国家政策建议具有巨大的影响力，并为设计和实施对所有涉及基因治疗的卫生保健专业人员的教育奠定了基础。

DESCRIPTION (provided by applicant): There is heightened optimism and attention to safety and the scientific merit of gene therapy studies. Instead of sensationalizing individual cases, or institutions or PIs for violating integrity when clinical practices go awry, it should be possible to discover how to prevent unintended mishaps. The researchers aim to elucidate in great detail the real world, taken-for-granted, day-to-day operations and decisions that bear on gene therapy clinical trials and regulatory paper work. Nobody really sees the whole picture of how the work gets done, who does what, how they interact, or how organizational systems, or health care policies affect research integrity. One aim of this institutional ethnography study is to identify and describe common practices, systems, and tools that work as well as barriers that make the conduct of gene therapy challenging and difficult in multiple settings concurrently. Another aim is to describe different types of institutional cultures that promote, safe guard, or protect research integrity as well as those that don't. Three cultural ethnographies will be conducted in three states, in three types of gene therapy clinical research partnerships. Field notes are the main source of data from non-participant observation by 3 ethnographers who observe in the setting, off and on, but regularly, for as long as 18 months. The ethnographers will interview as many different stakeholders as possible including staff and patients. Interviews are transcribed verbatim, and field notes are rewritten and interpreted. Both types of data will be analyzed using traditional cultural ethnography methods. Analysis will be conducted by 3 co-investigators and 3 expert consultants. All data are reported in the aggregate; no single setting or institution is identified or spotlighted. Two types of manuscripts will be produced. The one for the public will be a clear thick description of all complexities, challenges, and constraints that happen in daily clinical practice that go unnoticed by federal policy makers. The second is an academic interpretation of norms and practices of gene therapy clinical research. By understanding the role of each stakeholder in multiple institutional locations (research, university, clinical, laboratory, and regulatory); it is possible to see just how complex conducting gene therapy research really is. Results of this study will draw attention to best practice standards and education. The aim is to build public trust, promote patient participation in future research and spotlight exemplary models of collaborative clinical practices. This study has enormous power for changing national policy recommendations as well as laying the ground work to design and implement education for all health care professionals involved in gene therapy.