为新诊断、不符合移植条件的多发性骨髓瘤一线使用达拉单抗建模:医疗支付方的成本效益和风险分析。

IF 2 Q2 ECONOMICS
PharmacoEconomics Open Pub Date : 2024-09-01 Epub Date: 2024-06-20 DOI:10.1007/s41669-024-00503-9
Diana Beatriz Bayani, Yihao Clement Lin, Chandramouli Nagarajan, Melissa G Ooi, Allison Ching Yee Tso, John Cairns, Hwee Lin Wee
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引用次数: 0

摘要

背景和目的:本研究旨在评估两种被视为治疗新加坡新诊断、不符合移植条件的多发性骨髓瘤的标准方案的成本效益:(1)达拉单抗、来那度胺和地塞米松;(2)硼替佐米、来那度胺和地塞米松。此外,该研究还旨在探索管理决策不确定性和降低财务风险的潜在策略:采用分区生存模型从医疗保健系统的角度进行了成本效益分析,以估算与基于达拉单抗的治疗和基于硼替佐米的治疗方案相关的终生成本和质量调整生命年(QALYs)。分析采用了 MAIA 和 SWOG S0777 试验的数据,并纳入了当地的实际数据(如有)。进行了敏感性分析以评估研究结果的稳健性,并进行了风险分析,从支付方策略和不确定性负担(P-SUB)的角度分析了各种支付方策略,其中考虑了决策的不确定性和选择次优干预方案的额外成本:达拉单抗、来那度胺和地塞米松(DRd)与硼替佐米、来那度胺和地塞米松(VRd)相比,每获得1 QALY的增量成本效益比(ICER)为90,364美元。研究结果对 DRd 的生存期、进展后治疗费用、临终关怀费用和无进展生存期危险比的变化非常敏感。所探讨的方案表明,由于不成熟的试验数据和治疗效果随时间推移而产生的不确定性,结构性假设(如分析的时间跨度)对结果有很大影响。在比较的各种支付方策略中,达拉曲单抗的预付价格折扣是最佳方法,P-SUB最低,为14,708美元:总之,本研究发现达拉土单抗作为骨髓瘤的一线治疗超出了本评估所考虑的成本效益阈值。为管理不确定性和降低财务风险,建议采取先期降价的策略。这些发现凸显了有针对性的支付方策略对于解决特定类型和来源的不确定性的重要性。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Modeling First-Line Daratumumab Use for Newly Diagnosed, Transplant-Ineligible, Multiple Myeloma: A Cost-Effectiveness and Risk Analysis for Healthcare Payers.

Background and objective: This study aimed to assess the cost-effectiveness of two regimens regarded as the standard of care for the treatment of newly diagnosed, transplant-ineligible multiple myeloma in Singapore: (1) daratumumab, lenalidomide, and dexamethasone and (2) bortezomib, lenalidomide, and dexamethasone. Additionally, it aimed to explore potential strategies to manage decision uncertainty and mitigate financial risk.

Methods: A cost-effectiveness analysis from the healthcare system perspective was conducted using a partitioned survival model to estimate lifetime costs and quality-adjusted life years (QALYs) associated with daratumumab-based treatment and the bortezomib-based regimen. The analysis used data from the MAIA and SWOG S0777 trials and incorporated local real-world data where available. Sensitivity analyses were performed to evaluate the robustness of the findings, and a risk analysis was conducted to analyze various payer strategies in terms of their payer strategy and uncertainty burden (P-SUB), which account for the decision uncertainty and the additional cost of choosing a suboptimal intervention.

Results: The incremental cost-effectiveness ratio (ICER) for daratumumab, lenalidomide, and dexamethasone (DRd) compared with bortezomib, lenalidomide, and dexamethasone (VRd) was US $90,364 per QALY gained. The results were sensitive to variations in survival for DRd, postprogression treatment costs, cost of hospice care, and hazard ratio for progression-free survival. The scenarios explored indicated that structural assumptions, such as the time horizon of the analysis, significantly influenced the results due to uncertainties arising from immature trial data and treatment efficacy over time. Among the various payer strategies compared, an upfront price discount for daratumumab emerged as the best approach with the lowest P-SUB at US $14,708.

Conclusion: In conclusion, this study finds that daratumumab as a first-line treatment for myeloma exceeds the cost-effectiveness threshold considered in this evaluation. An upfront price reduction is the recommended strategy to manage uncertainties and mitigate financial risks. These findings highlight the importance of targeted payer strategies to address specific types and sources of uncertainty.

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来源期刊
CiteScore
3.50
自引率
0.00%
发文量
64
审稿时长
8 weeks
期刊介绍: PharmacoEconomics - Open focuses on applied research on the economic implications and health outcomes associated with drugs, devices and other healthcare interventions. The journal includes, but is not limited to, the following research areas:Economic analysis of healthcare interventionsHealth outcomes researchCost-of-illness studiesQuality-of-life studiesAdditional digital features (including animated abstracts, video abstracts, slide decks, audio slides, instructional videos, infographics, podcasts and animations) can be published with articles; these are designed to increase the visibility, readership and educational value of the journal’s content. In addition, articles published in PharmacoEconomics -Open may be accompanied by plain language summaries to assist readers who have some knowledge of, but not in-depth expertise in, the area to understand important medical advances.All manuscripts are subject to peer review by international experts. Letters to the Editor are welcomed and will be considered for publication.
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