title: Integrating the patient's perspective into device evaluation trials.
abstract: Innovations in medical device technology have greatly expanded the range of therapeutic options available to physicians and their patients. The understanding of treatment effects from the patient's perspective is an essential component of a comprehensive assessment of any new therapy, including medical devices. The term "patient-reported outcomes" (PROs) has been growing in use to refer to a cluster of variables such as health-related quality of life, symptoms, physical functioning, psychological well-being, treatment satisfaction, and treatment preferences. As in drug trials, the use of PROs in device evaluation has several methodological challenges, ranging from general concerns about interpretation, to more specific issues related to study design and regulatory approval (use of PROs as primary end points, incorporation in labeling, and product promotion). Successful approaches for integrating PROs into device evaluation trials include the careful selection of appropriate, interpretable PRO end points, accounting for possible confounding factors, and the use of alternatives to placebo-controlled trial designs, such as single-arm pre-post, observational, and registry studies, when the use of placebo control groups is not feasible. This article discusses the potential value and difficulties in measuring PROs in device studies.
authors.affiliation: Center for Health Outcomes Research at United BioSource Corporation, Bethesda, MD 20814, USA. nancy.leidy@unitedbiosource.com
authors.forename: Nancy Kline
authors.initials: NK
authors.lastname: Leidy
authors.forename: Kathleen
authors.initials: K
authors.lastname: Beusterien
authors.forename: Erin
authors.initials: E
authors.lastname: Sullivan
authors.forename: Randel
authors.initials: R
authors.lastname: Richner
authors.forename: Neal I
authors.initials: NI
authors.lastname: Muni
mesh_terms: D002986:Clinical Trials as Topic / Q000379:methods*; D003657:Decision Making; D018795:Device Approval; D019317:Evidence-Based Medicine; D006801:Humans; D017063:Outcome Assessment, Health Care / Q000379:methods*; D017060:Patient Satisfaction; D011788:Quality of Life; D020377:Self Efficacy; D013673:Technology Assessment, Biomedical / Q000379:methods*