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elranatamab 与医生选择治疗方案(非 BCMA 靶向方案)用于日本三类暴露多发性骨髓瘤患者的成本效果分析

英文原题:Cost-effectiveness analysis of elranatamab versus physician's choice of treatment (non-BCMA-directed regimens) in patients with triple class exposed multiple myeloma in Japan.

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Cost-effectiveness analysis of elranatamab versus physician's choice of treatment (non-BCMA-directed regimens) in patients with triple class exposed multiple myeloma in Japan.

PubMed 2026/09/08(内容时间) J Med Econ Q1 · IF 3.1(JCR 2025)

研究概要

从医疗支付方角度来看,在基础案例和情景分析中,elranatamab 相较于 PCT 具有成本效益,其 ICER 始终低于日本通常接受的阈值;但鉴于非锚定 MAIC 和长期外推的不确定性,应谨慎解读这一结果。

研究思路结论见上方概要

评估elranatamab与医生选择治疗方案(PCT)用于日本三类暴露多发性骨髓瘤患者的成本效果。

从日本公共医疗支付方视角,采用三健康状态的分区生存模型,对elranatamab与PCT进行了成本效果分析。应用每周周期长度和25年终身时间范围,根据日本HTA指南,成本和健康产出按每年2%贴现。elranatamab的临床输入来自2期MagnetisMM-3试验。与PCT的比较效果通过非锚定匹配调整间接比较(MAIC)与前瞻性真实世界LocoMMotion研究进行估计。效用值通过将日本价值集应用于MagnetisMM-3中收集的EQ-5D-5L数据计算得出。情景分析使用基于MAIC的比较 versus teclistamab和idecabtagene vicleucel(ide-cel)进行。

在基础案例分析中,与PCT相比,elranatamab提高了质量调整生命年(QALYs)(2.59 vs 0.82),但总成本更高(JPY 36,901,695(USD 246,570)vs 30,899,696(USD 206,466)),导致增量成本为JPY 6,001,999(USD 40,104),增量QALY增益为1.77。增量成本效果比(ICER)为JPY 3,394,966(USD 22,684)每QALY,仍处于日本的成本效果阈值之内。敏感性分析证明了基础案例结果的稳健性。在情景分析中,elranatamab相对于teclistamab和ide-cel占优。局限性:局限性包括依赖单一血液学专家对日本临床实践模式的临床意见、超出观察到的试验随访进行外推,以及由于缺乏头对头试验而导致的MAIC固有局限。

展开英文摘要原文

AIM: To evaluate the cost-effectiveness of elranatamab versus physician's choice of treatment (PCT) for patients with triple class exposed multiple myeloma in Japan. MATERIALS AND METHODS: A cost-effectiveness analysis was conducted comparing elranatamab with PCT using a partitioned survival model with three health states from the Japanese public healthcare payer perspective. A weekly cycle length and a 25-year lifetime horizon were applied, with costs and health outcomes discounted at 2% annually in accordance with Japanese HTA guidelines. Clinical inputs for elranatamab were derived from the phase 2 MagnetisMM-3 trial. Comparative effectiveness versus PCT was estimated using an unanchored matching-adjusted indirect comparison (MAIC) with the prospective real-world LocoMMotion study. Utility values were calculated by applying the Japanese value set to EQ-5D-5L data collected in MagnetisMM-3. Scenario analyses were performed using MAIC-based comparisons versus teclistamab and idecabtagene vicleucel (ide-cel). RESULTS: In the base-case analysis, elranatamab increased quality-adjusted life-years (QALYs) compared with PCT (2.59 vs 0.82) at a higher total cost (JPY 36,901,695 (USD 246,570) vs 30,899,696 (USD 206,466)), resulting in an incremental cost of JPY 6,001,999 (USD 40,104) and an incremental QALY gain of 1.77. The incremental cost-effectiveness ratio (ICER) was JPY 3,394,966 (USD 22,684) per QALY, remaining within cost-effectiveness thresholds in Japan. Sensitivity analyses demonstrated the robustness of the base-case results. In scenario analyses, elranatamab was dominant versus teclistamab and ide-cel. LIMITATIONS: Limitations include reliance on the clinical opinion of a single hematology expert for Japanese clinical practice patterns, extrapolation beyond observed trial follow-up, and inherent constraints of MAIC due to the lack of head-to-head trials. CONCLUSION: From the healthcare payer perspective, elranatamab was cost-effective versus PCT in the base case and scenario analyses, with ICERs consistently below commonly accepted thresholds in Japan; contingent on the unanchored MAIC and long-term extrapolation, this should be interpreted with caution.

论文信息

作者
Yuasa A、Nagano M、Kamei Y、Hatsuyama K、Matsuda H、Hlavacek P、DiBonaventura M、Suzuki K
第一作者单位
Japan Access & Value, Pfizer Japan Inc, Tokyo, Japan.Japan
通讯作者单位
Myeloma/AL Amyloid Unit, Japanese Red Cross Medical Center, Tokyo, Japan.Japan
期刊
Journal of medical economics2026 Dec
原文标识
PubMed 42709039 · DOI 10.1080/13696998.2026.2730069