Ong Jian Chun Matthew, Than Hein, Tripathi Sandeep, Gkitzia Christina, Wang Xiaojun
Novartis Singapore Pte. Ltd., Mapletree Business City, Singapore, Singapore.
Department of Haematology, Singapore General Hospital, Singapore, Singapore.
Cost Eff Resour Alloc. 2023 May 31;21(1):34. doi: 10.1186/s12962-023-00444-w.
Approximately 30-70% of patients who have undergone allogeneic (allo) hematopoietic stem cell transplantation (HSCT) eventually experience chronic graft-versus-host disease (cGVHD). Patients who develop steroid-refractory (SR)-cGVHD are the most severely impacted due to significant disease and financial burden. There remains an unmet need for safe, efficacious, and accessible treatments for these patients. The objective of this study was to determine the cost effectiveness of ruxolitinib for treatment of SR-cGvHD from the Singapore healthcare system perspective.
Based on data from the REACH3 randomized open-label trial, a semi-Markov model was developed to evaluate cost-effectiveness of ruxolitinib compared with investigators' choice of best alternative therapy (BAT) for treatment of patients > 12 years of age with SR-cGVHD in Singapore over a 40-year time horizon. The model only considered direct medical-care costs related to the treatment of SR-cGVHD and reported them in Singapore Dollars (SGD). Half-cycle correction was applied to all costs and outcomes, which were discounted at 3%. Probabilistic sensitivity analysis (PSA), one-way sensitivity analysis (OWSA), and scenario analysis were conducted to explore the drivers of uncertainty in the model.
In the deterministic base case, more life years (LY; 10.28 vs. 9.42) and quality-adjusted life years (QALYs; 7.31 vs. 6.51) were gained with ruxolitinib than BAT at higher costs (SGD 303,214 vs. SGD 302,673) leading to an incremental cost-effectiveness ratio (ICER) of SGD 677/QALY. At a willingness-to-pay threshold of SGD 75,000/QALY gained, PSA found that ruxolitinib had a 78.52% probability of being cost-effective. Findings were sensitive to variations in non-responder utilities in the BAT arm and duration of BAT treatment in the OWSA, or comparison to either methotrexate (MTX) or mycophenolic acid as a single comparator in the scenario analysis. ICERs remained lower than SGD 75,000/QALY in all other tested variations and scenarios.
Ruxolitinib is likely to be cost-effective from Singapore healthcare system's perspective for patients with SR-cGVHD, which is promising in the management of patients with unmet clinical needs.
接受异基因造血干细胞移植（allo-HSCT）的患者中，约30%-70%最终会发生慢性移植物抗宿主病（cGVHD）。发生激素难治性（SR）-cGVHD的患者受到的影响最为严重，因为疾病负担和经济负担都很大。对于这些患者，仍然需要安全、有效且可及的治疗方法。本研究的目的是从新加坡医疗保健系统的角度确定芦可替尼治疗SR-cGVHD的成本效益。
基于REACH3随机开放标签试验的数据，开发了一个半马尔可夫模型，以评估在40年的时间范围内，与研究者选择的最佳替代疗法（BAT）相比，芦可替尼治疗新加坡12岁以上SR-cGVHD患者的成本效益。该模型仅考虑与SR-cGVHD治疗相关的直接医疗费用，并以新加坡元（SGD）报告。对所有成本和结果应用半周期校正，并按3%进行贴现。进行概率敏感性分析（PSA）、单因素敏感性分析（OWSA）和情景分析，以探索模型中不确定性的驱动因素。
在确定性基础案例中，与BAT相比，芦可替尼获得了更多的生命年（LY；10.28对9.42）和质量调整生命年（QALY；7.31对6.51），但成本更高（303,214新元对302,673新元），导致增量成本效益比（ICER）为677新元/QALY。在每获得一个QALY的支付意愿阈值为75,000新元时，PSA发现芦可替尼具有成本效益的概率为78.52%。研究结果对BAT组中无反应者效用的变化、OWSA中BAT治疗持续时间的变化敏感，或在情景分析中与甲氨蝶呤（MTX）或霉酚酸作为单一对照物进行比较敏感。在所有其他测试的变化和情景中，ICER均低于75,000新元/QALY。
从新加坡医疗保健系统的角度来看，芦可替尼对于SR-cGVHD患者可能具有成本效益，这在管理有未满足临床需求的患者方面很有前景。
