Recommended methods for determining responsiveness and minimally important differences for patient-reported outcomes.

OBJECTIVE

The objective of this review is to summarize recommendations on methods for evaluating responsiveness and minimal important difference (MID) for patient-reported outcome (PRO) measures.

STUDY DESIGN AND SETTING

We review, summarize, and integrate information on issues and methods for evaluating responsiveness and determining MID estimates for PRO measures. Recommendations are made on best-practice methods for evaluating responsiveness and MID.

RESULTS

The MID for a PRO instrument is not an immutable characteristic, but may vary by population and context, and no one MID may be valid for all study applications. MID estimates should be based on multiple approaches and triangulation of methods. Anchor-based methods applying various relevant patient-rated, clinician-rated, and disease-specific variables provide primary and meaningful estimates of an instrument's MID. Results for the PRO measures from clinical trials can also provide insight into observed effects based on treatment comparisons and should be used to help determine MID. Distribution-based methods can support estimates from anchor-based approaches and can be used in situations where anchor-based estimates are unavailable.

CONCLUSION

We recommend that the MID is based primarily on relevant patient-based and clinical anchors, with clinical trial experience used to further inform understanding of MID.