title: Responsiveness and minimal important differences for patient reported outcomes.
abstract: Patient reported outcomes provide the patient's perspective on the effectiveness of treatment. The draft Food and Drug Administration guidance on patient reported outcomes for labeling and promotional claims raises a number of method and measurement issues that require further clarification, including methods of determining responsiveness and minimal important differences. For clinical trials, instruments need to be based on a clear conceptual framework, have evidence supporting content validity and acceptable psychometric qualities. The measures must also have evidence documenting responsiveness and interpretation guidelines (i.e., minimal important difference) to be most useful as effectiveness endpoints in clinical trials. The recommended approach is to estimate the minimal important difference based on several anchor-based methods, with relevant clinical or patient-based indicators, and to examine various distribution-based estimates (i.e., effect size, standardized response mean, standard error of measurement) as supportive information, and then to triangulate on a single value or small range of values for the MID. Confidence in a specific MID value evolves over time and is confirmed by additional research evidence, including clinical trial experience. The MID may vary by population and context, and no one MID will be valid for all study applications involving a PRO instrument. Responsiveness and MID must be demonstrated and documented for the particular study population, and these measurement characteristics are needed for PRO labeling and promotional claims.
authors.affiliation: Center for Health Outcomes Research, United Biosource Corporation, 7101 Wisconsin Ave., Suite 600, Bethesda, MD 20814, USA. dennis.revicki@unitedbiosource.com
authors.forename: Dennis A
authors.initials: DA
authors.lastname: Revicki
authors.forename: David
authors.initials: D
authors.lastname: Cella
authors.forename: Ron D
authors.initials: RD
authors.lastname: Hays
authors.forename: Jeff A
authors.initials: JA
authors.lastname: Sloan
authors.forename: William R
authors.initials: WR
authors.lastname: Lenderking
authors.forename: Neil K
authors.initials: NK
authors.lastname: Aaronson
mesh_terms: D002986:Clinical Trials as Topic / Q000379:methods* / Q000592:standards; D004348:Drug Labeling; D023381:Endpoint Determination; D019317:Evidence-Based Medicine; D006801:Humans; D017060:Patient Satisfaction; D011356:Product Labeling; D011594:Psychometrics / Q000295:instrumentation* / Q000379:methods; D011788:Quality of Life / Q000523:psychology; D015203:Reproducibility of Results; D011795:Surveys and Questionnaires; D016896:Treatment Outcome; D014481:United States; D014486:United States Food and Drug Administration