title: Integrating patient-reported outcomes into cancer symptom management clinical trials supported by the National Cancer Institute-sponsored clinical trials networks.
abstract: Patient-reported outcomes (PROs) are often the primary end point in symptom management trials. The scientific field of PROs is evolving, as evidenced by the US Food and Drug Administration's February 2007 release of a draft guidance for using PROs in effectiveness claims for drug labeling. This article presents issues encountered during use of PROs in National Cancer Institute-sponsored symptom management trials. Selected trials are presented that exemplify the challenges often seen in symptom management trials, and solutions are described. The examples presented include defining the appropriate end point, selecting and validating assessments, and answering the research questions through statistical analysis and interpretation. Progress has been made in addressing some of the unique challenges of PRO-based symptom management research. Many challenges still remain, but a foundational body of work now exists for more consistent and rigorous application of PROs into symptom management trials. There remains a need for more research in several methodologic aspects of design, analysis, and interpretation of symptom management trials.
authors.affiliation: Department of Health Sciences Research, Mayo Clinic and Mayo Foundation, Rochester, MN 55905, USA. jsloan@mayo.edu
authors.forename: Jeff A
authors.initials: JA
authors.lastname: Sloan
authors.forename: Lawrence
authors.initials: L
authors.lastname: Berk
authors.forename: Joseph
authors.initials: J
authors.lastname: Roscoe
authors.forename: Michael J
authors.initials: MJ
authors.lastname: Fisch
authors.forename: Edward G
authors.initials: EG
authors.lastname: Shaw
authors.forename: Gwen
authors.initials: G
authors.lastname: Wyatt
authors.forename: Gary R
authors.initials: GR
authors.lastname: Morrow
authors.forename: Amylou C
authors.initials: AC
authors.lastname: Dueck
mesh_terms: D000203:Activities of Daily Living; D002986:Clinical Trials as Topic; D006801:Humans; D054547:National Cancer Institute (U.S.); D009369:Neoplasms / Q000628:therapy*; D017060:Patient Satisfaction; D019984:Quality Indicators, Health Care; D011788:Quality of Life; D018434:Sickness Impact Profile; D016896:Treatment Outcome; D014481:United States