title: Methods for Implementing and Reporting Patient-reported Outcome (PRO) Measures of Symptomatic Adverse Events in Cancer Clinical Trials.
abstract: There is increasing interest to use patient-reported outcome (PRO) measures to evaluate symptomatic adverse events (AEs) in cancer treatment trials. However, there are currently no standard recommended approaches for integrating patient-reported AE measures into trials.
  
  
  
  Approaches are identified from previous trials for selecting AEs for solicited patient reporting, administering patient-reported AE measures, and analyzing and reporting results.
  
  
  
  Approaches for integrating patient-reported AE measures into cancer trials generally combine current standard methods for clinician-reported AEs and established best practices for using PRO measures. Specific AEs can be selected for a PRO questionnaire based on common and expected reactions in a given trial context, derived from literature review and qualitative/mixed-methods evaluations and should be the same set administered across all arms of a trial. A mechanism for collecting unsolicited patient-reported AEs will also ideally be included. Patients will preferably report at baseline and at the end of active treatment as well as on a frequent standardized schedule during active treatment, such as weekly from home, with a recall period corresponding to the frequency of reporting (eg, past 7 days). Less frequent reporting may be considered after an initial intensive monitoring period for trials of prolonged treatments and during long-term follow-up. Electronic PRO data collection is preferred. Backup data collection for missed PRO reports is advisable to boost response rates. Analysis can use a combination of approaches to AE and PRO data. If a high proportion of patients is experiencing baseline symptoms, systematic subtraction of these from on-study AEs should be considered to improve reporting of symptoms related to treatment. More granular longitudinal analyses of individual symptoms can also be useful.
  
  
  
  Methods are evolving for integrating patient-reported symptomatic AEs into cancer trials. These methods are expected to further evolve as more data from trials become available.
authors.affiliation: Cancer Outcomes Research Program, Lineberger Comprehensive Cancer Center, University of North Carolina, Chapel Hill, North Carolina; Health Outcomes Research Group, Department of Epidemiology and Biostatistics, Memorial Sloan Kettering Cancer Center, New York, New York.
authors.forename: Ethan
authors.initials: E
authors.lastname: Basch
authors.affiliation: Health Outcomes Research Group, Department of Epidemiology and Biostatistics, Memorial Sloan Kettering Cancer Center, New York, New York.
authors.forename: Lauren J
authors.initials: LJ
authors.lastname: Rogak
authors.affiliation: Section of Biostatistics, Mayo Clinic, Scottsdale, Arizona. Electronic address: dueck@mayo.edu.
authors.forename: Amylou C
authors.initials: AC
authors.lastname: Dueck
mesh_terms: D016907:Adverse Drug Reaction Reporting Systems; D000970:Antineoplastic Agents / Q000009:adverse effects / Q000627:therapeutic use; D064420:Drug-Related Side Effects and Adverse Reactions; D006801:Humans; D009369:Neoplasms / Q000188:drug therapy*; D000071066:Patient Reported Outcome Measures; D011795:Surveys and Questionnaires
chemical_list: D000970:Antineoplastic Agents
keywords: Adverse event; Cancer; Common Terminology Criteria for Adverse Events; Patient-reported outcome; Symptom; Toxicity