title: The use of patient-reported outcomes in advanced breast cancer clinical trials: a review of the published literature.
abstract: As a means to measure quantifiable signs, symptoms, and impacts of a disease or its treatment, patient-reported outcome (PRO) instruments can be applied to numerous settings, including use in drug development to support labeling claims. This research summarizes the use of PROs in trials for 16 commonly used regulatory approved treatments for advanced or metastatic breast cancer.
  
  
  
  For each treatment (n = 16), a literature search was conducted in MEDLINE, Embase, and PsycINFO. The primary criterion for selection was the report of studies that used PROs to evaluate treatment benefit and/or toxicity in advanced or metastatic breast cancer. From this, a sub-set of articles for each treatment were selected for full-text review where PRO-related information was extracted and summarized.
  
  
  
  The searches yielded 1727 publications. Following abstract review, 1702 were excluded because they failed to meet criteria, or were duplicates or less relevant for PRO information reported. Thus, 25 articles were reviewed in detail for this evaluation. Eleven PRO instruments were identified from these publications. The European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire - Core (EORTC QLQ-C30) was utilized the most frequently (n = 13, 52.0%). Most publications reported PROs positioned as secondary endpoints (n = 20, 80.0%); described some of the statistical analyses applied to PRO data (n = 21, 84.0%); and specified PRO results (n = 23, 92.0%).
  
  
  
  While several of the publications provided some information on how PROs were utilized, many did not describe details for PRO administration, scoring, analyses, and results interpretation. While it is encouraging that PROs are often used in clinical trials for patients with metastatic breast cancer, they are not commonly used to support endpoints that establish the basis for label claims. Because they yield direct insight into the patient experience of a condition, PROs may be used to provide a more comprehensive perspective of the benefits and risks from treatment.
authors.affiliation: a Adelphi Values , Boston , MA , USA ;
authors.forename: Diane M
authors.initials: DM
authors.lastname: Turner-Bowker
authors.affiliation: b Novartis Pharmaceuticals Corporation , East Hanover , NJ , USA.
authors.forename: Yanni
authors.initials: Y
authors.lastname: Hao
authors.affiliation: a Adelphi Values , Boston , MA , USA ;
authors.forename: Catherine
authors.initials: C
authors.lastname: Foley
authors.affiliation: a Adelphi Values , Boston , MA , USA ;
authors.forename: Nina
authors.initials: N
authors.lastname: Galipeau
authors.affiliation: a Adelphi Values , Boston , MA , USA ;
authors.forename: Iyar
authors.initials: I
authors.lastname: Mazar
authors.affiliation: a Adelphi Values , Boston , MA , USA ;
authors.forename: Meaghan
authors.initials: M
authors.lastname: Krohe
authors.affiliation: b Novartis Pharmaceuticals Corporation , East Hanover , NJ , USA.
authors.forename: Denise
authors.initials: D
authors.lastname: Globe
authors.affiliation: a Adelphi Values , Boston , MA , USA ;
authors.forename: Alan L
authors.initials: AL
authors.lastname: Shields
mesh_terms: D001943:Breast Neoplasms / Q000628:therapy*; D002986:Clinical Trials as Topic / Q000379:methods*; D005260:Female; D006801:Humans; D017063:Outcome Assessment, Health Care / Q000379:methods*; D000071066:Patient Reported Outcome Measures; D011788:Quality of Life; D011795:Surveys and Questionnaires
keywords: Breast cancer; Clinical trial; Drug labeling; Food and Drug Administration; Oncology; Patient-reported outcomes