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持高度乐观和关注的态度。当临床实践出错时，不要耸人听闻地报道个别病例、机构或PI违反诚信，应该有可能发现如何防止意外事故。研究人员的目标是详细阐明真实的世界，理所当然的，日常运作和决定，对基因治疗临床试验和监管文件工作。没有人真正看到工作是如何完成的，谁做什么，他们如何互动，或者组织系统或医疗保健政策如何影响研究的完整性。这项机构人种学研究的目的之一是识别和描述共同的做法，系统和工具，以及使基因治疗的挑战和困难，在多个设置同时进行的障碍。另一个目的是描述不同类型的机构文化，促进，安全警卫，或保护研究的完整性，以及那些不。三个文化人种志将在三个州进行，在三种类型的基因治疗临床研究伙伴关系。田野记录是3名民族志学者非参与性观察的主要数据来源，他们断断续续地、但定期地观察，长达18个月。人种学家将采访尽可能多的不同利益相关者，包括工作人员和患者。访谈是逐字记录的，现场记录是重写和翻译的。这两种类型的数据将使用传统的文化民族志方法进行分析。分析将由3名共同研究者和3名专家顾问进行。所有数据都是汇总报告的;没有确定或突出单一的环境或机构。将制作两种类型的手稿。面向公众的将是对日常临床实践中发生的联邦政策制定者未注意到的所有复杂性、挑战和限制的清晰而厚重的描述。二是对基因治疗临床研究规范和实践的学术解读。通过了解每个利益相关者在多个机构（研究，大学，临床，实验室和监管）中的作用，可以看到进行基因治疗研究的复杂性。这项研究的结果将提请注意最佳做法标准和教育。其目的是建立公众信任，促进患者参与未来的研究，并突出合作临床实践的示范模式。这项研究对于改变国家政策建议以及为所有参与基因治疗的医疗保健专业人员设计和实施教育奠定基础具有巨大的力量。

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.