Presentation (CTI)

OBJECTIVES: Managed access agreements (MAA) allow patients interim access to therapies where data is considered too naive to inform long-term outcomes. Using an example from Chimeric antigen receptor T-cell (CAR-T) for treating relapsed/refractory mantle cell Lymphoma (r/r MCL), we estimate the impact of early access to therapy, and explore the impact of potential interruptions to access, on the life expectancy of r/r MCL patients.
METHODS: CAR-T has been available to NHS patients with r/r MCL from 2021. CAR-T cohort life years (LYs) were modelled against historical control (HC) cohort LYs (r/r MCL patients managed on Rituximab, Bendamustine and Cytarabine [R-BAC]). LYs were estimated based on overall survival reported in ZUMA-2 (CAR-T) and published evidence (R-BAC) with extrapolations performed according to best practice. Cohort gains were estimated based on patients receiving CAR-T in an England NHS setting (n=193 to June 2025). Exploratory analysis estimated annual patient numbers (n=41) and assumed a 2-year interruption in CAR-T, based on an estimated timeframe for access to alternate, comparable interventions in this setting. Sensitivity analysis assessed uncertainty.
RESULTS: Across the lifetime of patients, an estimated 1,597 (95% CI: 1,132 to 1,936 ) LYs were gained by the CAR-T cohort, when compared against the outcomes of the HC cohort. Impact of access interruption across 2-years was estimated at 678 LY lost (95% CI: 481 to 822) equivalent to an 81.9% reduction in LYs (95% CI: 67.5% to 86.8%).
CONCLUSIONS: We estimate that early access to CAR-T therapy could generate an additional 1,597 LYs for r/r MCL patients. The impact of interruptions to treatment access through unsuccessful MAA exit is not well explored, however we highlight the substantial patient impact of potential interruptions in access to treatment, using the CAR-T r/r MCL example.