Going beyond statistical significance: Is the estimate for the PRO findings clinically meaningful?

Music City Center 

Currently, the terminology around thresholds used to interpret PRO data is heterogeneous and often unclear. This poses challenges when selecting thresholds for a specific purpose and may lead to their inappropriate or misleading application and interpretation. SISAQOL-IMI has, therefore, established a harmonised terminology that differentiates different types of PRO score interpretation thresholds, in particular focusing on thresholds for individual- and group-level data.

The analysis of PRO scores requires various different statistical methods depending on the research question. Some methods compare mean scores between two or more treatment groups for example, or compare how mean scores change from baseline over time within a group. Other methods are more focussed on individual patient changes, such as the proportion of individuals who achieve an improvement (or deterioration) in their PRO score over time or the time to improvement (or deterioration). Commonly, the same interpretation threshold has been applied regardless of whether groups or individual scores are of interest. Our work demonstrates this can lead to invalid interpretation of results and conclusions and the SISAQOL-IMI recommendations ensure adequate consideration of the choice of threshold and improved reporting of how thresholds have been applied. This will allow adequate assessment of the robustness of conclusions from the PRO analyses.

SISAQOL-IMI recommendations provide key criteria for the selection of appropriate thresholds based on the analysis of interest and also for assessment of robustness of how a published threshold has been derived. The recommendations emphasize a preference for thresholds derived from anchor-based methods over distribution-based methods in order to utilize an assessment of meaning to the interpretation over and above the measurement properties of the PRO. The need for patient-centred anchors is highlighted and consideration of the population that thresholds are derived in compared to the study population where interpretation is required is also emphasized.
This session will cover these key recommendations from SISAQOL-IMI on the interpretation of PRO scores. The established key criteria aim to support the application of valid, high-quality thresholds as a cornerstone of PRO data analysis and interpretation.