Table 3.

Steps to developing outcome measures in SLE clinical trials

Review of the data from SLE RCTs to determine whether a unique combination of outcome measures distinguishes responders to active therapy plus standard of care from those receiving placebo plus standard of care. Detailed statistical review of data from RCTs reporting both significant benefit and those that failed to achieve their primary outcome. Establish whether changes in background therapy confound response by analyzing changes over time within individuals. Determine if organ-specific versus global measures are more appropriate depending the study design. Ascertain if individual components of response correlate with outcomes. Proposed new response measures will be reviewed, discussed and agreed upon by the participants as applied to one data set will be tested in subsequent data sets for confirmation Review of the utility of patient-reported outcomes will be included (ex: are patient-reported outcomes useful in assessing responder status; what do patient-reported outcomes add to the responder assessment; when should patient-reported outcomes be included—always, specific to study, other?). Review of patient global assessment of disease activity if included in trial. Review of patient-reported HRQOL including specific domains of SF-36 and transition question. Review of patient-reported fatigue. Review of physician-reported measures of global disease activity and disease flare as well as time to flare. Review of glucocorticoid doses and whether attainment of a “clinically meaningful” definition of taper, such as prednisone ≤7.5mg/day, corresponds with a response.

SLE = systemic lupus erythematosus; RCT = randomized clinical trial; HRQOL = health-related quality of life.