BACKGROUND Systemic chemotherapy is crucial for pancreatic adenocarcinoma (PAC). It serves as a palliative chemotherapy for unresectable PAC and as a (neo-)adjuvant chemotherapy for (borderline) resectable PAC. Survival benefits were found with the use of liposomal irinotecan combined with 5-fluorouracil and leucovorin (nal-IRI/5-FU/LV) in the global phase III NAPOLI-1 trial. However, limited evidence supports the cost-effectiveness of liposomal irinotecan throughout the PAC treatment course.

OBJECTIVES We investigated the real-world efficacy and cost implications of nal-IRI/5-FU/LV in PAC management.

DESIGN A multicenter real-world study.

METHODS We analyzed real-world data from electronic health records to evaluate outcomes of nal-IRI/5-FU/LV exposure in this retrospective study. Effectiveness was assessed using overall survival (OS). Medical costs were those incurred from imaging or laboratory tests, chemotherapy, surgery, radiotherapy, and supportive care during inpatient, outpatient, and emergency department visits. Propensity score matching was used to adjust for potential differences in patient characteristics between the nal-IRI and non-liposomal irinotecan groups. The incremental cost-effectiveness ratio (ICER) was calculated by dividing cost differences by survival gains.

RESULTS Overall, 1734 patients diagnosed with PAC were screened, and 705 who received at least one cycle of chemotherapy were included in the analysis. The median OS was significantly longer in the nal-IRI group than in the non-liposomal irinotecan group (28.0 vs 18.3 months, p = 0.011). Patients in the nal-IRI group had more hospital visits and admissions during follow-up and incurred higher costs ($61,430 vs $39,129 per patient), which resulted in an ICER of $2787 per month of survival gained ($33,285 per life-year gained).

CONCLUSION Despite its higher cost, nal-IRI/5-FU/LV significantly improved the survival of patients with PAC compared to the conventional chemotherapies. Our findings suggest that it may be a cost-effective treatment option for gemcitabine failure despite the high cost of liposomal irinotecan, considering the poor prognosis of PAC.