RESUMEN
The aim was to assess the incremental costs of chimeric antigen receptor (CAR) T-cell therapy (axicabtagene ciloleucel, tisagenlecleucel) compared with standard of care in adult patients with relapsed or refractory diffuse large B-cell lymphoma (r/r DLBCL) from the German third-party payer perspective. A budget impact model was established over a 6-year period. Estimation of the third-line population: partitioned survival model based on outcome data from peer-reviewed literature, a top-down approach based on population forecasts, and age-standardized incidences. Cost data were derived from the controlling department of a tertiary hospital and a German cost-of-illness study. In the scenario analysis, the budget impact of treating second-line DLBCL patients was calculated. One-way deterministic sensitivity analyses were conducted to test the robustness of the model. For the period 2021-2026, 788-867 (minimum population, min) and 1,068-1,177 (maximum population, max) adult third-line r/r DLBCL patients were estimated. The budget impact ranged from 39,419,562; 53,426,514 (min; max) in year 0 to 122,104,097; 165,763,001 (min; max) in year 5. The scenario analysis resulted in a budget impact of 65,987,823; 89,558,611 (min; max) and 204,485,031; 277,567,601 (min; max) for years 0 and 5, respectively. This budget impact analysis showed a significant but reasonable financial burden associated with CAR T-cell therapy for a limited number of patients requiring individualized care. Further, this study presents challenges and future needs in data acquisition associated with cost analysis in personalized medicine. For comprehensive economic discussions, complementary cost-effectiveness analyses are required to determine the value of innovative therapies for r/r DLBCL.
RESUMEN
INTRODUCTION: Patients with relapsed or refractory diffuse large B-cell lymphoma (r/r DLBCL) require highly individualized therapies. Limited information exists regarding inpatient treatment patterns, outcomes, resource-use, and costs from the perspective of third-party payers in Germany. The aim of this study was to collect and evaluate routine inpatient care data to fill aforementioned gaps. METHODS: Retrospective single center observational study in a German tertiary teaching hospital. Data were collected from patient records, the hospital-pharmacy database, and claims data. RESULTS: Eighty-four patients (47 male; mean age at initial diagnosis, 59 years) were identified and grouped by treatment line (L): 2L (n = 78), 3L (n = 32), and >3L (n = 12). Prescribed treatments in 2L were chemotherapy 56%, auto-SCT 31%, allo-SCT 1%, other 12%; 3L: 50%, 16%, 6%, 28%, respectively, and >3L: 42%, 0%, 33%, 25%, respectively. Mean number of hospital admissions and length of inpatient stay (days) were: 2L (4, 44), 3L (2, 26), and >3L (5, 63). Average cost/patient: 2L = 44,750, 3L = 32,589 and >3L = 88,668. Mean treatment costs per patient for stem-cell-transplanted patients were 55,468 for autologous SCT (n = 28) and 131,264 for allogeneic SCT (n = 7). Documented death was 21%, 28%, and 41% for 2L, 3L, and >3L, respectively. CONCLUSION: Individualized DLBCL treatment in patients ≥ 2L is costly and results in a huge variability in resource consumption. The number of documented deaths and length of hospitalization signal a high economic burden on patients and families. A multicenter comprehensive evaluation of health and economic burdens of r/r DLBCL and linkage with other data sources (eg, registries, payers' claims data) is essential.
