Presentation (CTI)

OBJECTIVES: Single-arm trials (SATs) have been increasingly used to support oncology health economic assessments (HTAs) worldwide. Due to the lack of head-to-head comparisons in SATs, real-world data (RWD) serve as a pragmatic option to inform comparative efficacy and are accepted by the Center for Outcomes Research and Economic Evaluation for Health (C2H) guidelines as a potential source of evidence. This study assessed SAT-based oncology submissions in past C2H evaluations, their approaches to generating comparative evidence, and the use of RWD.
METHODS: C2H cost-effectiveness evaluations from April 2019 to March 2025 were reviewed to identify oncology submissions based solely on SATs. Full-text screening of the Summary Reports was performed by one investigator, and data extraction was validated by a second investigator. Information on the comparator data source, indirect treatment comparison (ITC) approach, and review commentaries was collected for the analysis.
RESULTS: Of the 59 evaluations identified, 34 were completed, and 7 focused on oncology treatments across 14 distinct indications. Of these, 36% (5/14) relied on SATs for clinical evidence, and all used suboptimal external trials to derive comparative efficacy. Among SAT-based evaluations, ITCs using individual patient-level data (IPD) or pseudo-IPD from external trials were generally preferred (88%) over naive comparisons against aggregated data. Only one evaluation adopted RWD from a domestic registry through an unanchored matching-adjusted indirect comparison (MAIC). ITC approaches, including MAIC, naïve comparison, and network meta-analysis, were employed to generate comparative efficacy. Although the review committee generally accepted ITC evidence submitted in SAT-based evaluations, there were concerns regarding the high uncertainty associated with comparative evidence derived from external trials, given the nature of the SAT design.
CONCLUSIONS: External trials were used as primary evidence for SAT-based C2H oncology submissions but were subject to uncertainty for comparative efficacy outcomes. Potentially, RWD can serve as an alternative to suboptimal trials in future evaluations.