Presentation (CTI)

OBJECTIVES: To evaluate how single-arm trial (SAT) evidence influences Health Technology Assessment (HTA) decisions across four jurisdictions—Canada (CADTH), France (HAS), England (NICE), and Australia (PBAC)—with a focus on uncertainties, social value judgments, and the use of external comparators.
METHODS: A mixed-methods design was used. Conditionally approved oncology drugs (2014-2021) based on SATs were identified from EMA, Health Canada, TGA, and MHRA records. Corresponding HTA reports were sourced from CADTH, HAS, NICE, and PBAC. A content analysis framework was used to systematically code clinical, economic, and social decision factors. Nominal and continuous variables were analyzed using multinomial logistic regression and survival analysis to examine factors influencing HTA outcomes and duration.
RESULTS: Among 113 SAT-based HTA submissions, 47% were conditionally listed, 30% were not listed, and only 21% received full reimbursement. Decision patterns varied significantly by country: HAS leaned toward ASMR V (61%), NICE had the highest positive listings (59%), while PBAC showed the highest rejection rate (58%). Factors associated with negative decisions included uncertainties in study design (RRR=4.16, p=0.003), comparability of efficacy (RRR=2.74, p=0.033), and patient population in external comparators (RRR=0.24, p=0.001). Use of real-world data and price discounts were positively associated with favorable outcomes. Median HTA duration ranged from 3.7 to 11 months depending on the agency.
CONCLUSIONS: SATs introduce significant variability in HTA outcomes due to methodological and contextual uncertainties. While agencies increasingly rely on real-world evidence and social value judgments, the lack of harmonized standards hinders replicability. Findings underscore the need for standardized frameworks to evaluate SATs and external comparator use, enabling transparent and equitable access to high-cost oncology treatments across jurisdictions.