Opportunities for Pragmatic Design Elements in Surgical Trials.

IMPORTANCE

Randomized clinical trials (RCTs), the gold standard of medical evidence, remain relatively rare in surgery. In part, this is the result of challenges that are unique to surgical trials, including surgeon concerns about lack of equipoise, slow recruitment, difficulty standardizing interventions, and a lack of funding and infrastructure for surgical trials. Here, it is argued that increasing the use of pragmatic clinical trials (PCTs) could help overcome some of the challenges of surgical RCTs. The study perspective is informed by work at the US Food and Drug Administration (FDA), which regulates many clinical trials.

OBSERVATIONS

While exploratory trials aim to maximize the chance that an intervention demonstrates a hypothesized benefit in a narrow population and indication, PCTs are designed to evaluate an intervention in conditions that resemble routine clinical care. The FDA has been interested in increasing the use of pragmatic design elements in clinical trials because these trials can lower the cost and complexity of evidence generation and result in improved generalizability. In surgical trials, pragmatic design elements could include giving surgeon-investigators flexibility in deciding which patients to enroll and which surgical technique to use, a flexibility that helps to ensure that the resulting evidence is applicable to these decisions in routine practice. To streamline data collection, PCTs could leverage existing surgical registries or use data routinely collected in the electronic health record to measure outcomes.

CONCLUSIONS AND RELEVANCE

Increasing the use of PCTs presents an opportunity to overcome the challenges of surgical RCTs. However, there is a need to prioritize questions that matter most to patients and health care professionals and to develop improved training, funding, and institutional support for PCTs in surgery.