title: Patient-Reported Outcomes in Cancer Drug Development and US Regulatory Review: Perspectives From Industry, the Food and Drug Administration, and the Patient.
abstract: Data reported directly by patients about how they feel and function are rarely included in oncology drug labeling in the United States, in contrast to Europe and to nononcology labeling in the United States, where this practice is more common. Multiple barriers exist, including challenges unique to oncology trials, and industry's concerns regarding cost, logistical complexities, and the Food and Drug Administration's (FDA's) rigorous application of its 2009 guidance on the use of patient-reported outcome (PRO) measures. A panel consisting of representatives of industry, FDA, the PRO Consortium, clinicians, and patients was assembled at a 2014 workshop cosponsored by FDA to identify practical recommendations for overcoming these barriers. Key recommendations included increasing proactive encouragement by FDA to clinical trial sponsors for including PROs in drug development programs; provision of comprehensive PRO plans by sponsors to FDA early in drug development; promotion of an oncology-specific PRO research agenda; development of an approach to existing ("legacy") PRO measures, when appropriate (focused initially on symptoms and functional status); and increased FDA and industry training in PRO methodology. FDA has begun implementing several of these recommendations.
authors.affiliation: Lineberger Comprehensive Cancer Center, University of North Carolina, Chapel Hill.
authors.forename: Ethan
authors.initials: E
authors.lastname: Basch
authors.affiliation: Patient and Partners LLC, Madison, Connecticut.
authors.forename: Cindy
authors.initials: C
authors.lastname: Geoghegan
authors.affiliation: Patient-Reported Outcome Consortium, Critical Path Institute, Tucson, Arizona.
authors.forename: Stephen Joel
authors.initials: SJ
authors.lastname: Coons
authors.affiliation: Novartis Pharmaceuticals, East Hanover, New Jersey.
authors.forename: Ari
authors.initials: A
authors.lastname: Gnanasakthy
authors.affiliation: Study Endpoints Team, Study Endpoints and Labeling Development, Office of New Drugs, Immediate Office, Center for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, Maryland.
authors.forename: Ashley F
authors.initials: AF
authors.lastname: Slagle
authors.affiliation: Study Endpoints Team, Study Endpoints and Labeling Development, Office of New Drugs, Immediate Office, Center for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, Maryland.
authors.forename: Elektra J
authors.initials: EJ
authors.lastname: Papadopoulos
authors.affiliation: Office of Hematology and Oncology Products, Office of New Drugs, Center for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, Maryland.
authors.forename: Paul G
authors.initials: PG
authors.lastname: Kluetz
mesh_terms: D000970:Antineoplastic Agents / Q000627:therapeutic use*; D003299:Cooperative Behavior; D017277:Drug Approval / Q000331:legislation & jurisprudence; D055808:Drug Discovery / Q000331:legislation & jurisprudence; D004345:Drug Industry / Q000331:legislation & jurisprudence; D007722:Health Knowledge, Attitudes, Practice; D006801:Humans; D033183:Interdisciplinary Communication; D009369:Neoplasms / Q000188:drug therapy* / Q000473:pathology; D010358:Patient Participation; D017060:Patient Satisfaction; D010361:Patients / Q000523:psychology*; D010465:Perception; D016896:Treatment Outcome; D014481:United States; D014486:United States Food and Drug Administration / Q000331:legislation & jurisprudence
chemical_list: D000970:Antineoplastic Agents