Common Daily Practices of Gene Therapy Clinical Research

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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.