OBJECTIVES: Understanding the meaning of change scores produced by PRO questionnaires is important to support clinical trial endpoints. While quantitative, anchor-based methods are a common method to quantify MWPC on a target PRO assessment, the FDA has demonstrated interest in collecting additional supportive qualitative evidence from patient interviews. This research describes ways that qualitative data associated with single-item patient global assessments (PGAs) can support MWPC estimates on PRO scores.

METHODS: Published regulatory guidance and literature related to qualitative approaches for collecting data on PGAs to support MWPC estimates were summarized alongside recommendations for data utilization.

RESULTS: Patient interviews to evaluate MWPC can be conducted either independent from a clinical trial (e.g., concept elicitation or cognitive interviews) or during/after a clinical trial (e.g., exit interviews). For example, interviews conducted independent from trials provide justification for how patients define meaningful changes in their health status (e.g., what change on a PGA categorizes a patient as improved), which could inform conduct of quantitative anchor-based analyses. One limitation to outside-of-trial interviews is that patients’ feedback on change is hypothetical; however, a benefit is that interview timing is not contingent on clinical trial timelines. In-trial interviews can evaluate change on PGAs and contextualize what the change on a PRO score means to patients using a mixed-methods approach. A limitation of in-trial interviews is the risk that information reported during the interview potentially conflicts with clinical trial data but provides real-world context to quantitative trial results. For either approach, multiple PGAs can be evaluated, patients can speak to either true or hypothetical change, and data can be presented alongside the in-trial data for PRO scores.

CONCLUSIONS: Qualitative data via patient interviews can add richness to quantitative analyses and further support the clinical meaning of drug efficacy by providing patient interpretations of the meaningfulness of changes.