Department of Dermatology, Liverpool Hospital, Liverpool, New South Wales, Australia.
South West Sydney Clinical Campuses, University of New South Wales, Liverpool, New South Wales, Australia.
JAMA Dermatol. 2022 Sep 1;158(9):1013-1021. doi: 10.1001/jamadermatol.2022.2878.
There is an increasing body of literature that supports the use of rituximab as a first-line steroid-sparing agent in pemphigus vulgaris. However, the cost of rituximab is substantial compared with conventional agents, and there are limited health economic data to justify its use.
To evaluate the cost-effectiveness of rituximab biosimilars relative to mycophenolate mofetil as a first-line steroid-sparing agent for moderate to severe pemphigus vulgaris.
DESIGN, SETTING, AND PARTICIPANTS: A cost-utility analysis over a 24-month time horizon was conducted from the perspective of the Australian health care sector using a modeled cohort of treatment-naive adult patients with moderate to severe pemphigus vulgaris. A Markov cohort model was constructed to simulate disease progression following first-line treatment with rituximab biosimilars or mycophenolate mofetil. The simulated cohort transitioned between controlled disease, uncontrolled disease, and death. Efficacy and utility data were obtained from available published literature. Cost data were primarily obtained from published government data. One-way and probabilistic sensitivity analyses were performed to assess uncertainty. Primary outcomes were the changes in cost and quality-adjusted life-years (QALYs), and incremental cost-effectiveness ratio (ICER) over the 24 months.
Rituximab biosimilars and mycophenolate mofetil.
The simulated cohort of treatment-naive patients had a mean age of 50.8 years, a female-to-male ratio of 1.24, and moderate to severe disease as classified by the Harman criteria. First-line rituximab biosimilars were associated with a cost reduction of AU$639 and an improvement of 0.07 QALYs compared with mycophenolate mofetil, resulting in an ICER of -AU$8818/QALY. Rituximab biosimilars were therefore more effective and less costly compared with mycophenolate mofetil. Sensitivity analyses demonstrated that rituximab biosimilars remained cost-effective across a range of values for cost, utility, and transition probability input parameters and willingness-to-pay thresholds.
In this cost-utility analysis, rituximab biosimilars were cost-effective compared with mycophenolate mofetil for moderate to severe pemphigus vulgaris. Further investigation into its cost-effectiveness over a longer time horizon is necessary, but the favorable results of this study suggest that the high acquisition costs of rituximab biosimilars may be offset by its effectiveness and provide economic evidence in support of its listing on the Pharmaceutical Benefits Scheme for pemphigus vulgaris.
越来越多的文献支持将利妥昔单抗作为天疱疮的一线类固醇节约药物。然而，与传统药物相比，利妥昔单抗的成本很高，并且没有足够的健康经济学数据来证明其使用的合理性。
评估利妥昔单抗生物类似药相对于吗替麦考酚酯作为中重度天疱疮一线类固醇节约药物的成本效益。
设计、设置和参与者：在 24 个月的时间范围内，从澳大利亚医疗保健部门的角度，对未接受治疗的中重度天疱疮成年患者的治疗队列进行了成本-效用分析。构建了一个马尔可夫队列模型，以模拟利妥昔单抗生物类似药或吗替麦考酚酯一线治疗后的疾病进展。模拟队列在疾病控制、疾病未控制和死亡之间进行转换。疗效和效用数据来自已发表的文献。成本数据主要来自已发表的政府数据。进行了单因素和概率敏感性分析以评估不确定性。主要结果是 24 个月内成本和质量调整生命年（QALY）的变化以及增量成本效益比（ICER）。
利妥昔单抗生物类似药和吗替麦考酚酯。
未经治疗的患者队列的平均年龄为 50.8 岁，女性与男性的比例为 1.24，并且根据 Harman 标准，疾病程度为中度至重度。与吗替麦考酚酯相比，一线利妥昔单抗生物类似药可降低 639 澳元的成本，并提高 0.07 个 QALY，因此 ICER 为-8818 澳元/QALY。因此，与吗替麦考酚酯相比，利妥昔单抗生物类似药更有效且成本更低。敏感性分析表明，在成本、效用和转换概率输入参数以及支付意愿阈值的一系列值下，利妥昔单抗生物类似药仍然具有成本效益。
在这项成本-效用分析中，与吗替麦考酚酯相比，利妥昔单抗生物类似药在治疗中重度天疱疮方面具有成本效益。还需要对其在更长时间内的成本效益进行进一步研究，但这项研究的有利结果表明，利妥昔单抗生物类似药的高获得成本可能会被其疗效所抵消，并为支持其在天疱疮药物福利计划中的上市提供经济证据。
