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生物改善病情抗风湿药物(bDMARDs)、靶向合成 DMARDs(tsDMARDs)和生物类似物 DMARDs(bsDMARDs)联合甲氨蝶呤治疗泰国高疾病活动度类风湿关节炎患者的成本-效用分析。

Cost-utility analysis of biologic disease-modifying antirheumatic drugs (bDMARDs), targeted synthetic DMARDs (tsDMARDs) and biosimilar DMARDs (bsDMARDs) combined with methotrexate for Thai rheumatoid arthritis patients with high disease activity.

机构信息

Siriraj Health Policy Unit, Faculty of Medicine Siriraj Hospital, Mahidol University, Bangkok, Thailand.

Health Intervention and Technology Assessment Program (HITAP), Ministry of Public Health, Nonthaburi, Thailand.

出版信息

BMC Health Serv Res. 2023 May 31;23(1):561. doi: 10.1186/s12913-023-09595-1.

Abstract

BACKGROUND

New biologic disease-modifying antirheumatic drugs (bDMARDs), targeted synthetic DMARDs (tsDMARDs) and biosimilar DMARDs (bsDMARDs) all showed greater clinical benefits in the treatment of patients with rheumatoid arthritis (RA) with high disease activity, but imposed higher costs than standard treatment. This study evaluated the cost-effectiveness of 11 alternative treatment strategies for RA patients with high disease activity whose treatment with three conventional synthetic DMARDs (csDMARDs) failed.

METHODS

A Markov model was constructed using a societal perspective to estimate relevant costs and health outcomes in terms of quality-adjusted life years (QALYs) for a lifetime horizon (100 years), given a 3% annual discount. Alternative treatment strategies including five bDMARDs, two tsDMARDs, and four bsDMARDs in combination with methotrexate (MTX) were compared with the standard of care (SoC), i.e., cyclosporine and azathioprine. Direct and non-medical care costs were estimated by identifying the resources used, then multiplied by the standard costing menu in the year 2022. Utility and transitional probabilities were collected in three advanced tertiary hospitals. A network meta-analysis was used to estimate the efficacy of each treatment. Lifetime cost, QALYs and an incremental cost-effectiveness ratio were calculated and compared to the cost-effectiveness threshold of 160,000 THB per QALY gained (US $4,634, where 1 USD = 34.53 THB in 2022). Probabilistic and one-way sensitivity analyses were performed to estimate parameter uncertainties.

RESULTS

The bDMARDs, tsDMARDs or bsDMARDs combined with MTX provided 0.09 to 0.33 QALYs gained with additional costs of 550,986 to 2,096,744 THB (US $15,957 to $60,722) compared to the SoC. The ICER ranged from 2.3 to 8.1 million THB per QALY (US $65,935 to $234,996) compared to the SoC. None of these combinations was cost-effective in the Thai context. The results were sensitive to the mortality hazard ratio of patients with high disease activity.

CONCLUSIONS

Combinations of MTX with either bDMARDs, tsDMARDs or bsDMARDs were not economically attractive compared to the standard practice. However, they reduced disease activity and improved patient quality of life. The price negotiation process for these treatments must be conducted to ensure their financial value and affordability before they are included in the pharmaceutical reimbursement list.

摘要

背景

新型生物改善病情抗风湿药物(bDMARDs)、靶向合成改善病情抗风湿药物(tsDMARDs)和生物类似物改善病情抗风湿药物(bsDMARDs)在治疗高疾病活动度的类风湿关节炎(RA)患者方面均显示出更大的临床获益,但治疗费用高于标准治疗。本研究评估了 11 种替代治疗策略的成本效益,这些策略适用于三种常规合成改善病情抗风湿药物(csDMARDs)治疗失败的高疾病活动度 RA 患者。

方法

采用社会视角构建 Markov 模型,以质量调整生命年(QALYs)为指标,从终生(100 年)的角度估算相关成本和健康结果,贴现率为 3%。将五种 bDMARDs、两种 tsDMARDs 和四种 bsDMARDs 联合甲氨蝶呤(MTX)与标准治疗(SoC),即环孢素和硫唑嘌呤进行比较。直接和非医疗护理成本通过确定使用的资源来估算,然后乘以 2022 年的标准成本菜单。效用和过渡概率在三家三级先进医院收集。采用网络荟萃分析估计每种治疗的疗效。计算并比较了终生成本、QALYs 和增量成本效益比与获得每 QALY 160,000 泰铢(4634 美元,2022 年 1 美元=34.53 泰铢)的成本效益阈值。进行概率和单向敏感性分析以估计参数不确定性。

结果

与 SoC 相比,bDMARDs、tsDMARDs 或 bsDMARDs 联合 MTX 可获得 0.09 至 0.33 个 QALYs,额外费用为 550,986 至 2,096,744 泰铢(65,935 至 234,996 美元)。与 SoC 相比,增量成本效益比(ICER)范围为 2.3 至 8.1 百万泰铢/QALY(65,935 至 234,996 美元)。在泰国背景下,这些组合均不具有成本效益。结果对高疾病活动度患者的死亡率风险比敏感。

结论

与标准治疗相比,MTX 联合 bDMARDs、tsDMARDs 或 bsDMARDs 的组合在经济上并不具有吸引力。然而,它们降低了疾病活动度并改善了患者的生活质量。在将这些治疗方法纳入药品报销清单之前,必须进行价格谈判,以确保其经济价值和可负担性。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/75ba/10230705/159223368896/12913_2023_9595_Fig1_HTML.jpg

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