title: Patient-Reported Outcomes Labeling for Products Approved by the Office of Hematology and Oncology Products of the US Food and Drug Administration (2010-2014).
abstract: To review the use of patient-reported outcome (PRO) data in medical product labeling granted by the US Food and Drug Administration (FDA) for new molecular entities and biologic license applications by the FDA Office of Hematology and Oncology Products (OHOP) between January 2010 and December 2014, to elucidate challenges faced by OHOP for approving PRO labeling, and to understand challenges faced by drug manufacturers to include PRO end points in oncology clinical trials.
  
  
  
  FDA Drug Approval Reports by Month were reviewed to obtain the number of new molecular entities and biologic license applications approved from 2010 to 2014. Drugs approved by the FDA OHOP during this period were selected for further review, focusing on brand and generic name; approval date; applicant; indication; PRO labeling describing treatment benefit, measures, end point status, and significant results; FDA reviewer feedback on PRO end points; and study design of registration trials. First in class, priority review, fast track, orphan drug, or accelerated approval status was retrieved for selected oncology drugs from 2011 to 2014. Descriptive analyses were performed by using Microsoft Excel 2010.
  
  
  
  Of 160 drugs approved by the FDA (2010-2014), 40 were approved by OHOP. Three (7.5%) of the 40 received PRO-related labeling (abiraterone acetate, ruxolitinib phosphate, and crizotinib). Compared with nononcology drugs (2011-2014), oncology drugs were more likely to be orphan and first in class. The majority of oncology drug reviews by FDA were fast track, priority, or accelerated.
  
  
  
  Although symptoms and functional decrements are common among patients with cancer, PRO labeling is rare in the United States, likely because of logistical hurdles and oncology study design. Recent developments within the FDA OHOP to capture PROs in oncology studies for the purpose of product labeling are encouraging.
authors.affiliation: Ari Gnanasakthy, Carla DeMuro, Marci Clark, and Emily Haydysch, RTI Health Solutions, Research Triangle Park, NC; and Esprit Ma and Vijayveer Bonthapally, Millennium Pharmaceuticals, Cambridge, MA.
authors.forename: Ari
authors.initials: A
authors.lastname: Gnanasakthy
authors.affiliation: Ari Gnanasakthy, Carla DeMuro, Marci Clark, and Emily Haydysch, RTI Health Solutions, Research Triangle Park, NC; and Esprit Ma and Vijayveer Bonthapally, Millennium Pharmaceuticals, Cambridge, MA. demuromercon@rti.org.
authors.forename: Carla
authors.initials: C
authors.lastname: DeMuro
authors.affiliation: Ari Gnanasakthy, Carla DeMuro, Marci Clark, and Emily Haydysch, RTI Health Solutions, Research Triangle Park, NC; and Esprit Ma and Vijayveer Bonthapally, Millennium Pharmaceuticals, Cambridge, MA.
authors.forename: Marci
authors.initials: M
authors.lastname: Clark
authors.affiliation: Ari Gnanasakthy, Carla DeMuro, Marci Clark, and Emily Haydysch, RTI Health Solutions, Research Triangle Park, NC; and Esprit Ma and Vijayveer Bonthapally, Millennium Pharmaceuticals, Cambridge, MA.
authors.forename: Emily
authors.initials: E
authors.lastname: Haydysch
authors.affiliation: Ari Gnanasakthy, Carla DeMuro, Marci Clark, and Emily Haydysch, RTI Health Solutions, Research Triangle Park, NC; and Esprit Ma and Vijayveer Bonthapally, Millennium Pharmaceuticals, Cambridge, MA.
authors.forename: Esprit
authors.initials: E
authors.lastname: Ma
authors.affiliation: Ari Gnanasakthy, Carla DeMuro, Marci Clark, and Emily Haydysch, RTI Health Solutions, Research Triangle Park, NC; and Esprit Ma and Vijayveer Bonthapally, Millennium Pharmaceuticals, Cambridge, MA.
authors.forename: Vijayveer
authors.initials: V
authors.lastname: Bonthapally
mesh_terms: D017277:Drug Approval; D004348:Drug Labeling; D006405:Hematology; D006801:Humans; D008495:Medical Oncology; D000071066:Patient Reported Outcome Measures; D014481:United States; D014486:United States Food and Drug Administration