Jakobs Florian, Jeck Julia, Ahmadi Paymon, Kron Anna, Kron Florian
Department of Hematology and Stem Cell Transplantation, University Hospital Essen, University of Duisburg-Essen, Essen, Germany.
VITIS Healthcare Group, Cologne, Germany.
Cost Eff Resour Alloc. 2022 Dec 12;20(1):67. doi: 10.1186/s12962-022-00400-0.
BACKGROUND: In the past decades, highly innovative treatments in the field of diffuse large B-cell lymphoma (DLBCL) became available in clinical practice. The aim of this study was to assess the cost-benefit relation of third-line interventions in DLBCL from a German payer perspective. METHODS: Clinical benefit of allogeneic stem cell transplantation (alloSCT), chimeric antigen receptor T cells therapy (CAR T) [tisagenlecleucel (tisa-cel) and axicabtagene ciloleucel (axi-cel)] and best supportive care (BSC) was assessed in terms of median overall survival (median OS) derived from a systematic literature review in PubMed. Real-world treatment costs were retrieved from the university hospitals Cologne and Hamburg-Eppendorf. The cost-benefit relation was analysed using the efficiency frontier concept. RESULTS: Median OS varied from 6.3 months in BSC to 23.5 months in CAR T (axi-cel), while median real-world treatment costs ranged likewise widely from €26,918 in BSC to €340,458 in CAR T (axi-cel). Shown by the efficiency frontier, alloSCT and axi-cel were found as most efficient interventions. CONCLUSION: The efficiency frontier supports the pricing of innovative therapies, such as third-line interventions in DLBCL, in relation to appropriate comparators. Yet, studies with longer follow-up periods are needed to include studies with unreached median OS and to reflect experiences gained with CAR T in clinical practice.
背景：在过去几十年中，弥漫性大B细胞淋巴瘤（DLBCL）领域出现了高度创新的治疗方法并应用于临床实践。本研究旨在从德国医保支付方的角度评估DLBCL三线干预措施的成本效益关系。 方法：通过对PubMed系统文献综述得出的中位总生存期（中位OS）来评估异基因干细胞移植（alloSCT）、嵌合抗原受体T细胞疗法（CAR T）[替沙仑赛（tisagenlecleucel，tisa-cel）和阿基仑赛（axicabtagene ciloleucel，axi-cel）]以及最佳支持治疗（BSC）的临床获益。实际治疗成本从科隆大学医院和汉堡-埃彭多夫大学医院获取。使用效率前沿概念分析成本效益关系。 结果：中位OS从BSC组的6.3个月到CAR T（axi-cel）组的23.5个月不等，而实际治疗成本中位数同样差异很大，从BSC组的26,918欧元到CAR T（axi-cel）组的340,458欧元。效率前沿分析显示，alloSCT和axi-cel是最有效的干预措施。 结论：效率前沿分析有助于确定创新疗法（如DLBCL的三线干预措施）相对于适当对照的定价。然而，需要进行更长随访期的研究，以纳入未达到中位OS的研究，并反映CAR T在临床实践中的经验。
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