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JAMA Oncol. 2015 Jun;1(3):375-9. doi: 10.1001/jamaoncol.2015.0530.

Patient-Reported Outcomes in Cancer Drug Development and US Regulatory Review: Perspectives From Industry, the Food and Drug Administration, and the Patient.

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Lineberger Comprehensive Cancer Center, University of North Carolina, Chapel Hill.
Patient and Partners LLC, Madison, Connecticut.
Patient-Reported Outcome Consortium, Critical Path Institute, Tucson, Arizona.
Novartis Pharmaceuticals, East Hanover, New Jersey.
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.
Office of Hematology and Oncology Products, Office of New Drugs, Center for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, Maryland.


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.

[Indexed for MEDLINE]

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