Introduction

Funding decisions for many health technologies occur without undergoing health technology assessment (HTA), in particular, without assessment of cost effectiveness (CE). Immunoglobulins in Australia are an interesting case study because they have been used for a long time for various rare disorders and their price is publicly available. Undertaking an HTA enables us to assess CE for an intervention for which there is limited clinical and economic evidence. This study presents a post-market review to assess the CE of immunoglobulins for the treatment of multifocal motor neuropathy (MMN) compared with best supportive care.

Methods

A Markov model was used to estimate costs and quality-adjusted life-years (QALYs). Input sources included randomised controlled trials, single-arm studies, the Australian clinical criteria for MMN, clinical guidelines, previous Medical Services Advisory Committee (MSAC) reports and inputs from clinical experts. Sensitivity analyses were conducted to assess the uncertainty and robustness of the CE results.

Results

The cost per patient of treating MMN with immunoglobulin was AU$275,853 versus AU$26,191when no treatment was provided, with accrued QALYs of 6.83 versus 6.04, respectively. The latter translated into a high incremental cost-effectiveness ratio (ICER) of AU$317,552/QALY. The ICER was most sensitive to the utility weights and the price of immunoglobulins. MSAC advised to continue funding of immunoglobulins on the grounds of efficacy, despite the high and uncertain ICER.

Conclusions

Beyond the ICER framework, other factors were acknowledged, including the high clinical need in a patient population for which there are no other active treatments available. This case study highlights the challenges of conducting HTA for already funded interventions, and the efficiency trade-offs required to fund effective high-cost therapies in rare conditions.

中文翻译：

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使用卫生技术评估来持续资助卫生技术：免疫球蛋白治疗多灶性运动神经病的案例

介绍

许多卫生技术的资助决策是在没有经过卫生技术评估（HTA）的情况下做出的，特别是没有进行成本效益（CE）评估。澳大利亚的免疫球蛋白是一个有趣的案例研究，因为它们长期以来一直用于治疗各种罕见疾病，并且其价格是公开的。进行 HTA 使我们能够评估临床和经济证据有限的干预措施的 CE。本研究进行了一项上市后审查，旨在评估免疫球蛋白与最佳支持治疗相比治疗多灶性运动神经病 (MMN) 的CE。

方法

马尔可夫模型用于估计成本和质量调整生命年 (QALY)。输入来源包括随机对照试验、单组研究、澳大利亚 MMN 临床标准、临床指南、之前的医疗服务咨询委员会 (MSAC) 报告以及临床专家的意见。进行敏感性分析以评估 CE 结果的不确定性和稳健性。

结果

使用免疫球蛋白治疗 MMN 的每位患者的费用为 AU$275,853，而未提供治疗时则为 AU$26,191，累计 QALY 分别为 6.83 和 6.04。后者转化为高增量成本效益比 (ICER)，达到 AU$317,552/QALY。 ICER 对免疫球蛋白的效用权重和价格最敏感。尽管 ICER 较高且不确定，但 MSAC 建议以功效为由继续资助免疫球蛋白。

结论

除了 ICER 框架之外，还承认了其他因素，包括没有其他有效治疗方法的患者群体的高临床需求。本案例研究强调了对已经资助的干预措施进行 HTA 的挑战，以及在罕见情况下资助有效的高成本疗法所需的效率权衡。