PRO-CTCAE in Oncology Clinical Trials: a U.S. Regulatory Perspective

• Activity Number: 393 - Using Patient-Reported Outcomes to Capture Symptomatic Adverse Events in Cancer Clinical Trials: Challenges and Opportunities
• Type: Topic Contributed
• Date/Time: Tuesday, August 1, 2017 : 2:00 PM to 3:50 PM
• Sponsor: Health Policy Statistics Section
• Author(s): Paul Kluetz*
• Companies: U.S. Food and Drug Administration
• Keywords: patient-reported outcome ; PRO ; drug development ; tolerability ; oncology ; regulatory

Abstract

Assessment of safety and tolerability is an important trial objective across clinical stages of drug development. The US National Cancer Institute (NCI) has developed a patient-reported outcome (PRO) version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE) that is being incorporated into academic and commercial cancer trials. Work is ongoing to identify thoughtful approaches to trial design, conduct, and data analysis in order to maximize the value of patient-centered data generated from PRO-CTCAE and other PRO measures. In this presentation, a US regulatory perspective will be provided to review trial design considerations including unbiased methods to select items, appropriate assessment frequency, and to what degree PRO scores should be monitored during trials. As data matures, agreement on standard analysis and presentation methods taking advantage of this longitudinal data is needed that can complement existing clinician reported safety data. Sustained collaborative effort is underway to successfully incorporate PRO-CTCAE and other PRO measures in clinical trials that hold promise to further our understanding of a cancer treatment's effect on the patient.