In recent years, the US FDA has become more critical of instruments used to measure patient-reported outcomes (PROs) in clinical trials. To facilitate decisions related to the approval of drugs, labels and promotional claims based on PROs, the FDA created the Study Endpoints and Label Development (SEALD) group. SEALD has developed a PRO guidance related to the use of PRO measures used to support drug approvals and label claims, including recommendations for establishing thresholds for meaningful change at the individual level (i.e., defining a responder). This article examines in detail the FDA-recommended methodology for defining a responder and analyzing responder-based PRO measure results. We also present other responder analysis approaches for consideration in furthering the precision and interpretation of this methodology.

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The FDA continues to approve PRO claims, with 24% of new molecular entities and biologic license applications being granted. Successful PRO label claims over the past 5 years have generally supported treatment benefit for symptoms specified as primary endpoints.

The results of this investigation indicate that the DFS-SF has good psychometric properties and replicability.

The Menopause Epidemiology Study builds upon existing literature by providing data on daily frequency and severity of vasomotor symptoms. There are many women with frequent and severe vasomotor symptoms who may benefit from treatment.

SUMMARY BackgroundGastrointestinal side effects are commonly associated with opioid treatment for pain.

Shorter recall periods may underestimate symptom burden when symptoms have diurnal or day-to-day fluctuation and may place undue burden on patients. On the other hand, recall intervals that are too long may either over- or underestimate the health state. Therefore, appropriate criteria should be considered given attributes of the disease when selecting an adequate recall period.

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