Moving from a pilot study to large pragmatic trial in primary care settings: A study on acute rhinosinusitis

Acute rhinosinusitis is one of the most common conditions seen in primary care. One in seven adults are diagnosed with ARS annually, resulting in one in five of all antibiotic prescriptions. Yet there has been limited research comparing the effectiveness of widely used treatments such as antibiotics and nasal steroids. Conducting such a trial in the context of decades of established practice poses unique challenges.

A feasibility phase was conducted with continuing feedback to provide refinement and guidance regarding the design of a large-scale, pragmatic randomized controlled trial. The pilot trial assessed the ability to enroll, retain, and evaluate adherence to the intervention and assessment protocols.

The feasibility phase allowed us to seek input from patients and experts. This resulted in changes pre and post pilot that will impact the full study. A priori enrollment targets for the pilot were achieved, and with high adherence rates. In total, 373 patients were pre-screened and 140 patients were enrolled participants. Adherence to data collection via the daily diary was 93% throughout the study, with 95% completing their diary on the day of the primary outcome, 3 post-randomization.

Expert panels and a patient advisory committee recommended critical changes to our study design. Stakeholder engagement is a key component of this funding source and was widely used throughout the 18-months. An achieved primary goal of the feasibility phase was to evaluate recruitment and study methods prior to implementing a large clinical trial that requires significant resources.