Table 2.
Evaluation criteria used to prioritize patient-reported outcome measures mapped to the conceptual framework
| Criteriaa,b | 'Type | Definition (Example Criteria) |
|---|---|---|
| Burden | Item number, complexity, time to completion | |
| Validity | ||
| Content | Evidence that instrument measures the concept of interest in the intended population (qualitative interviews, focus groups with target population) | |
| Construct | Evidence that relationships among items, domains, and concepts conform to a priori hypotheses about relationships that should exist with measures of related concepts (discriminant and convergent validity, known group validity)c | |
| Reliability | ||
| Test-retest | Stability of scores over time when no change in the measurement concept is expected (intraclass correlation coefficient) | |
| Internal consistency | Extent to which items comprising a scale measure the same concept (Cronbach alpha, item-total correlations) | |
| Responsiveness | Evidence that instrument can identify differences in scores over time in individuals who have changed with respect to the measurement concept (within-person change over time, effect size statistic) | |
| Scoring | Scores easily interpretable versus not | |
| Availability | Public domain versus not | |
| Translation | Translation with relevant language and cultural validation versus not | |
PROM, patient-reported outcome measure; FDA, US Food and Drug Administration.
Meeting participants considered these criteria (in listed order) when evaluating the PROMs selected for in-depth review and in prioritizing the PROMs for framework mapping.
The presented criteria reflect some, but not all, of the PROM properties evaluated when using PROMs in clinical trials reviewed by the FDA. Please see the FDA’s Guidance for Industry on Patient-Reported Outcome Measures: Use in Medical Product Development to Support Labeling Claims (issued by the Center for Drug Evaluation and Research, the Center for Biologics Evaluation and Research, and the Center for Devices and Radiologic Health) for more information (10).
Discriminant and convergent validity measure the strength of correlation testing a priori hypotheses, and known group validity measures the degree to which the instrument can distinguish among groups hypothesized a priori to be different.