title: Patient-Reported Outcomes in Cancer Clinical Trials: Measuring Symptomatic Adverse Events With the National Cancer Institute's Patient-Reported Outcomes Version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE).
abstract: Systematic capture of the patient perspective can inform the development of new cancer therapies. Patient-reported outcomes (PROs) are commonly included in cancer clinical trials; however, there is heterogeneity in the constructs, measures, and analytic approaches that have been used making these endpoints challenging to interpret. There is renewed effort to identify rigorous methods to obtain high-quality and informative PRO data from cancer clinical trials. In this setting, PROs are used to address specific research objectives, and an important objective that spans the product development life cycle is the assessment of safety and tolerability. The U.S. Food and Drug Administration's (FDA) Office of Hematology and Oncology Products (OHOP) has identified symptomatic adverse events (AEs) as a central PRO concept, and a systematic assessment of patient-reported symptomatic AEs can provide data to complement clinician reporting. The National Cancer Institute's Patient-Reported Outcomes version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE) is being evaluated by multiple stakeholders, including the FDA, and is considered a promising tool to provide a standard yet flexible method to assess symptomatic AEs from the patient perspective. In this article, we briefly review the FDA OHOP's perspective on PROs in cancer trials submitted to the FDA and focus on the assessment of symptomatic AEs using PRO-CTCAE. We conclude by discussing further work that must be done to broaden the use of PRO-CTCAE as a method to provide patient-centered data that can complement existing safety and tolerability assessments across cancer clinical trials.
authors.affiliation: From the Office of Hematology and Oncology Products, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, MD; Independent Cancer Patient Advocate, Los Angeles, CA; Lineberger Comprehensive Cancer Center, University of North Carolina, Chapel Hill, NC; Outcomes Research Branch, National Cancer Institute, Rockville, MD.
authors.forename: Paul G
authors.initials: PG
authors.lastname: Kluetz
authors.affiliation: From the Office of Hematology and Oncology Products, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, MD; Independent Cancer Patient Advocate, Los Angeles, CA; Lineberger Comprehensive Cancer Center, University of North Carolina, Chapel Hill, NC; Outcomes Research Branch, National Cancer Institute, Rockville, MD.
authors.forename: Diana T
authors.initials: DT
authors.lastname: Chingos
authors.affiliation: From the Office of Hematology and Oncology Products, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, MD; Independent Cancer Patient Advocate, Los Angeles, CA; Lineberger Comprehensive Cancer Center, University of North Carolina, Chapel Hill, NC; Outcomes Research Branch, National Cancer Institute, Rockville, MD.
authors.forename: Ethan M
authors.initials: EM
authors.lastname: Basch
authors.affiliation: From the Office of Hematology and Oncology Products, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, MD; Independent Cancer Patient Advocate, Los Angeles, CA; Lineberger Comprehensive Cancer Center, University of North Carolina, Chapel Hill, NC; Outcomes Research Branch, National Cancer Institute, Rockville, MD.
authors.forename: Sandra A
authors.initials: SA
authors.lastname: Mitchell
mesh_terms: D000970:Antineoplastic Agents / Q000009:adverse effects*; D064420:Drug-Related Side Effects and Adverse Reactions / Q000453:epidemiology* / Q000473:pathology; D006801:Humans; D054547:National Cancer Institute (U.S.); D009369:Neoplasms / Q000188:drug therapy* / Q000453:epidemiology; D000071066:Patient Reported Outcome Measures; D057566:Self Report; D014481:United States; D014486:United States Food and Drug Administration
chemical_list: D000970:Antineoplastic Agents