Protecting seriously ill populations during pragmatic clinical trials

Pragmatic clinical trials (PCTs) emphasize real-world effectiveness methodology to address the limitations of results from explanatory randomized clinical trials (RCTs), which often fail to translate to real-world medical practice. An inherent tension in the conduct of PCTs is that the research must impose a minimal burden on patients and health care institutions. PCTs prioritize outcome measures from existing data sources to minimize data collection burden; however, a lack of patient-reported outcomes may result in gaps in safety for vulnerable populations, such as those with serious illnesses. One proposed standard for judging the readiness of a study for a pragmatic trial is a ranking system that assigns PCTs a lower rank if they impose additional data collection burdens. However, this results in the wide use of measures of health care utilization and costs while patient experience measures, which could capture adverse unintended consequences, are omitted. In this article, we make the case for a risk-based approach to imposing additional data collection in PCTs to capture potential safety and patient experience outcomes, using examples from "real life" implemented interventions to improve end-of-life care through the Liverpool Pathway and through the implementation of Physician Orders for Life Sustaining Treatment (POLST) in Oregon.