Common Daily Practices of Gene Therapy Clinical Research

描述(由申请人提供)：对基因治疗研究的安全性和科学价值的高度乐观和关注。与其在临床实践出错时耸人听闻地报道个别病例、机构或个人投资者违背诚信，不如发现如何防止意外事故。研究人员的目标是非常详细地阐明与基因治疗临床试验和监管文件工作有关的真实世界、被视为理所当然的日常操作和决定。没有人真正了解工作是如何完成的，谁做了什么，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确定和描述常见的做法、系统和工具，以及使基因治疗在多种环境中同时进行具有挑战性和困难的障碍。另一个目标是描述促进、保护或保护研究诚信的不同类型的机构文化以及那些不促进、保护或保护研究诚信的机构文化。三种文化人种学将在三个州、三种类型的基因疗法临床研究合作伙伴关系中进行。田野笔记是由3名人种学家进行的非参与者观察的主要数据来源，他们断断续续地在环境中观察，但有规律地观察，长达18个月。民族志专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。访谈被逐字转录，实地笔记被重写和翻译。这两种类型的数据都将使用传统的文化人种学方法进行分析。分析将由3名联合调查员和3名专家顾问进行。所有数据都是汇总报告的；没有单一的背景或机构被确定或突出显示。将制作两种类型的手稿。对于公众来说，这将是对日常临床实践中发生的所有复杂性、挑战和限制的清晰而厚实的描述，这些都是联邦政策制定者没有注意到的。二是对基因治疗临床研究规范和实践的学术解读。通过了解每个利益相关者在多个机构(研究、大学、临床、实验室和监管机构)中的角色；可以看到进行基因治疗研究到底有多复杂。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，并突出协作临床实践的示范模式。这项研究对改变国家政策建议以及为设计和实施所有参与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.