Development of a single‑item patient‑reported measure of treatment side‑effect burden in clinical trials of rheumatic and musculoskeletal diseases using stakeholder input and large language model refinement.

OBJECTIVES: To build on existing evidence regarding single-item measurement instruments of patient-reported bother or trouble from medical side effects in individuals with rheumatic and musculoskeletal diseases (RMDs). Further, to collect input from the OMERACT community through a structured survey that rated and ranked available options and to seek agreement to advance one or more of these measures for use as exploratory outcomes in future clinical trials.

METHODS: At OMERACT 2025 we presented and discussed survey results for domain match, feasibility and ranking of six candidate instruments of bother or trouble from side effects. Collaborator feedback - including comments from patients, clinicians, and researchers - was synthesized with a large-language-model (LLM) to identify key concerns and guide refinement of the instrument's relevance, clarity, and acceptability. The LLM-assisted synthesis of participant comments resulted in a new, single-item instrument designed to improve patient safety reporting from the patient's perspective.

RESULTS: The merged and modified version of the instrument was presented at the OMERACT 2025 meeting, where 33 participants approved it as a reasonable approach to incorporate collaborator input. The proposed instrument is feasible (32 [97%]) and voting supported advancing its further assessment (30 [91%]) as an exploratory outcome measurement instrument in coming RMD trials.

CONCLUSION: We developed a novel single-item instrument. This is the first known application of LLMs in refining a patient-reported outcome instrument for clinical trials. It is designed to capture the patient perspective on symptomatic treatment-related side effects in RMDs and is supported for exploratory use in trials.