The PRO-CTCAE in oncology clinical trials: Insights from a targeted literature review.

Abstract

e23193 Background: The Patient-Reported Outcomes Common Terminology Criteria for Adverse Events (PRO-CTCAE) is a tool used to collect information on symptomatic adverse events (AEs) directly from patients in oncology clinical trials. It has been designated as a core measure by the FDA. Despite its increased use, there is limited guidance on item selection or how to score and analyze PRO-CTCAE data. The study aimed to conduct a targeted literature review to understand how the PRO-CTCAE has been used in oncology clinical trials, identify implementation challenges, and summarize recommendations for optimal use. Methods: We conducted a search of PubMed and ClinicalTrials.gov from 2014 to 2024. The results were categorized into three areas: use of PRO-CTCAE in clinical trials, methods for item selection, and scoring and data analysis. We synthesized the findings, critically evaluated the PRO-CTCAE, and identified persistent variabilities regarding its optimal use. Results: We screened 198 PubMed articles and 59 ClinicalTrials.gov records and identified 50 relevant sources; 31 clinical trials using the PRO-CTCAE, 12 articles detailing PRO-CTCAE item selection methods, 4 articles on strategies to score and analyze data from the PRO-CTCAE, and 3 articles providing information covering all areas of interest. Clinical trials: The PRO-CTCAE was most commonly used in phase 2 trials as a secondary endpoint (42%). The majority (84%) of studies assessed gastrointestinal symptoms, most commonly nausea, decreased appetite, and vomiting; item selection was often based only on investigator judgement. Item selection: Recommended strategies included using multiple sources of information (e.g., literature reviews, patient interviews, medical chart audits) augmented by open-ended AE assessments throughout studies. Scoring and data analysis: No standard scoring algorithm was identified. While some articles have been published recommending strategies to analyze the PRO-CTCAE, no consistency was identified in how the PRO-CTCAE was scored or analyzed across the reviewed trials. Conclusions: This review found heterogeneity in the implementation of the PRO-CTCAE with no standard approach to item selection, analysis, or reporting. However, published recommendations suggest using multiple methods to select items, including open-ended questions and systematic approaches for analysis. A standardized implementation approach would enable cross trial comparisons and ensure that patient experiences of AEs are captured, with selected items based on relevance to patient experience rather than investigator judgement alone.