Completion Rates and Considerations for Analyses of Patient-Reported Outcomes in Open-Label Cancer Trials: FDA Review of Trials, 2007 - 2017

• Activity Number: 264 - Health Policy Statistics Section A.M. Roundtable Discussion (Added Fee)
• Type: Roundtables
• Date/Time: Tuesday, July 31, 2018 : 7:00 AM to 8:15 AM
• Sponsor: Health Policy Statistics Section
• Author(s): Jessica K. Roydhouse* and Mallorie H. Fiero and Bellinda King-Kallimanis and Paul G. Kluetz
• Companies: Office of Hematology and Oncology Products, US Food and Drug Administration and Office of Biostatistics, US Food and Drug Administration and Office of Hematology and Oncology Products, US Food and Drug Administration and Oncology Center of Excellence, US Food and Drug Administration
• Keywords: cancer; trial; open-label; missing data; sensitivity

Abstract

BACKGROUND: Open-label designs are used frequently in cancer trials. Patient-reported outcomes (PROs) are commonly assessed as part of cancer trials, and can provide important information about the patient experience while on therapy. However, the cancer trial setting poses numerous challenges, including high patient attrition and missing data. As part of the FDA's commitment to analyze and communicate patient experience data, sustained effort is being exerted to explore and address these challenges. We conducted a review of cancer trials submitted to the FDA from 2007 - 2017 using FDA databases and sponsor documents. Of the 51 RCTs that provided PRO completion information to the FDA during this 10-year period, 25 were open-label RCTs. Among the analytic issues encountered were gaps of >=10% in PRO completion rates between study arms for at least one PRO instruments as well as the infrequent use of sensitivity analyses for missing PRO data.

ISSUES FOR DISCUSSION: PROs in cancer trials pose numerous challenges and carefully planned endpoint definitions, primary analyses and sensitivity analyses for missing data are important.