OBJECTIVES: Patient-reported treatment experiences are increasingly important in oncology drug development and clinical practice. The Patient Reported Outcomes – Common Terminology Criteria for Adverse Events (PRO-CTCAE^TM) was developed to explore patient perceptions of symptomatic effects. Although analyzing and presenting data consistently across drugs/clinical programs/sponsors is beneficial for decision making, there is no definitive guidance on how to analyze or present PRO-CTCAE™ data. While the Food and Drug Administration (FDA) has presented a framework for describing and visualizing PRO-CTCAE™ data, numerous questions remain, including: “What is the appropriate denominator in calculating incidence?”; “How should missing data be summarized?”; and “Which graphical presentation is easiest to interpret?”.

METHODS: Based on a review of the literature; the authors own experiences in analyzing, interpreting, and presenting PRO-CTCAE™ data; and post-hoc exploration of trial data, key questions to ask of PRO-CTCAE™ data have been considered.

RESULTS: We recommend a standard set of 26 questions related to completion rates, evolution of symptom experiences, and treatment effects, as well as associated analyses and graphical presentations to inform regulatory/payer decision making and support clinical interactions. Three levels of recommended analyses are specified to answer these key questions: (1) Core analyses to inform messaging to regulators, payers, and sponsor teams; (2) Contextual/supportive analyses to facilitate messaging derived from core analyses; (3) Additional analyses for communicating with patients and clinicians. Each analysis should produce a combination of tables and graphical presentations. A variety of presentation modalities, including stacked bar charts, line graphs, pie charts and Kaplan Meier curves, are recommended depending on the key question.

CONCLUSIONS: An iterative approach was taken to propose a list of PRO-CTCAE™ questions, analyses and data presentations that should resonate with regulators and payers and support interactions between patients and clinicians. We propose consistency between sponsors in analyzing and presenting PRO-CTCAE™ data to facilitate review and comparison.