Conducting Successful Pragmatic Clinical and Transactional Trial

By Douglas A. Colquhoun, MB ChB, from the IARS, AUA and SOCCA 2019 Annual Meetings*

The Scholars’ Program day continued with its second session: Navigating and Conducting Pragmatic Clinical and Translational Trials for Research. Moderated by Kristin L. Schreiber, MD, PhD, Instructor of Anesthesiology, Harvard Medical School, and included presentations from Vafi Salmasi, MD, Instructor of Anesthesiology, Stanford University School of Medicine, Hance Clarke, MD, PhD, Assistant Professor of Anesthesia, University of Toronto, Daniel King-Wai Low, MD, MB BS, Associate Professor, Seattle Children’s Hospital.

Dr. Schreiber set the stage for the panel with a discussion of key tradeoffs in the conduct of clinical research. She laid out the main topics for the session, including the idea of control of study variables set in tension with the generalizability of the results, challenges in assuring that the study intervention is in fact the only intervention, and finally variability between and within subjects. This is often thought about under the domain of personalized medicine.

In his presentation, Dr. Salmasi described the lifecycle of medical discovery and then noted that very high proportions of patients do not even come close to the study inclusion criteria from which their treatment is being extrapolated. In explicitly contrasting the extremes of pragmatic vs. explanatory clinical trials, he highlighted the challenges and benefits of a pragmatic trial approach. He was able, using examples, which included his own research, to analyze some of the necessary underpinnings of pragmatic research, specifically support of institutional culture and in many projects very sophisticated utilization of the electronic health record. He indicated some of the challenges, which may arise, particularly as the regulatory framework for this type of work is continuing to evolve.

Dr. Hance Clarke described issues with engagement with the institution, government and community in the context of pain medicine. He described his work in pain management and the development of a translational pain service to limit ramp up of opioid therapy, prevention of transition to chronic opioid use patterns and achieve de-escalation of therapy in a complex postsurgical pain population. He described the benefits of partnering with institutional resources to craft a message around the work being performed in the recruitment of further patients and resources. He also described the essential work of measuring outcomes in the development of a new clinical program to ensure that its goals are being realized and appropriate utilization is being achieved.

Dr. Daniel Low described his work on the use of real world data in medical research. He argued that clinicians have extremely limited insight into patient outcomes. Despite the near universal digitization of medical data, this has not improved the availability of granular medical information. He presented an example of how emerging technologies may allow rapid reporting of aggregated data and empower clinicians to understand short- and long-term outcomes of their care. Referring to this self-serve model as a democratization of data access, he showed examples of how near time feedback of practice information was able to drive changes in clinical practice while ensuring patient outcomes were not negatively impacted.

*Coverage from the Scholars’ Program: Navigating and Conducting Pragmatic Clinical and Translational Trials for Research during the IARS 2019 Annual Meeting