title: Documenting the rationale and psychometric characteristics of patient reported outcomes for labeling and promotional claims: the PRO Evidence Dossier.
abstract: The Food and Drug Administration (FDA) and European Medicines Agency (EMEA) are willing to consider including information on patient reported outcomes (PROs) in product labeling and advertising. Pharmaceutical industry researchers must provide sufficient evidence supporting PRO benefit before an approval may be granted. This report describes the purpose and content of a PRO Evidence Dossier, which consists of important information supporting PRO claims. The dossier should be completed by pharmaceutical industry or other researchers to document the planning of the PRO assessment strategy, psychometric evidence, desired target labeling statements, and the clinical trial evidence of PRO benefits. The systematic reporting and documentation of information on the rationale for including PROs, rationale for the selection of specific PRO instruments, evidence on the psychometric qualities of the PRO measures, and guidelines for interpreting PRO findings will facilitate achieving a PRO labeling or promotional claim. Combining all the relevant information into a single document will facilitate the review and evaluation process for clinical and regulatory reviewers. The PRO Evidence Dossier may also be helpful to industry and academic researchers in identifying further information that will need to be developed to support the clinical development program and the PRO endpoints.
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: Ari
authors.initials: A
authors.lastname: Gnanasakthy
authors.forename: Kevin
authors.initials: K
authors.lastname: Weinfurt
mesh_terms: D000329:Advertising; D002986:Clinical Trials as Topic / Q000379:methods / Q000592:standards*; D003657:Decision Making; D004282:Documentation; D017277:Drug Approval / Q000379:methods*; D004345:Drug Industry; D004348:Drug Labeling / Q000592:standards*; D006801:Humans; D017063:Outcome Assessment, Health Care / Q000379:methods*; D011594:Psychometrics / Q000379:methods / Q000592:standards*; D011788:Quality of Life; D014481:United States; D014486:United States Food and Drug Administration