Abstract：
OBJECTIVE: Ataluren and eteplirsen are orphan drugs that delay progression of Duchenne muscular dystrophy in mutation-specific subgroups. They have yet to be approved in Egypt but are expected to reach the market soon. This study describes 2 cost-utility models comparing the drugs with the standard of care.
METHODS: We used a partition-survival model with 5 states based on the ambulatory status to model a cohort of ambulatory patients at the age of 5 years. Baseline curves were obtained from a published model; then the ambulation loss curve was updated using the Kaplan-Meier curve of the standard of care from a study by McDonald et al. Other curves were updated by calibration to this curve. Costs and utilities were from a local study. Deterministic and probabilistic sensitivity analyses were conducted. Prices were estimated based on other orphan drugs\' prices.
RESULTS: In the base case, ataluren 1000 mg and eteplirsen 50 mg/mL resulted in an incremental cost-effectiveness ratio of EGP 51 745 605 and EGP 69 652 533/quality-adjusted life-year, respectively, at their hypothetical prices of EGP 308 600 for ataluren 30-sachet pack and EGP 62 800 for eteplirsen 10 mL vial. The incremental cost-effectiveness ratio was sensitive to health state utilities but not to state costs. At EGP 911 719/quality-adjusted life-year threshold, the value-based prices were EGP 4680 for ataluren 1000 mg and EGP 733 for eteplirsen 10 mL vial.
CONCLUSIONS: Based on these models, there is a huge gap between the prices of orphan drugs and their value-based prices, which highlights the need for major policy reforms in the assessment and pricing of orphan drugs.
METHODS: We used a partition-survival model with 5 states based on the ambulatory status to model a cohort of ambulatory patients at the age of 5 years. Baseline curves were obtained from a published model; then the ambulation loss curve was updated using the Kaplan-Meier curve of the standard of care from a study by McDonald et al. Other curves were updated by calibration to this curve. Costs and utilities were from a local study. Deterministic and probabilistic sensitivity analyses were conducted. Prices were estimated based on other orphan drugs\' prices.
RESULTS: In the base case, ataluren 1000 mg and eteplirsen 50 mg/mL resulted in an incremental cost-effectiveness ratio of EGP 51 745 605 and EGP 69 652 533/quality-adjusted life-year, respectively, at their hypothetical prices of EGP 308 600 for ataluren 30-sachet pack and EGP 62 800 for eteplirsen 10 mL vial. The incremental cost-effectiveness ratio was sensitive to health state utilities but not to state costs. At EGP 911 719/quality-adjusted life-year threshold, the value-based prices were EGP 4680 for ataluren 1000 mg and EGP 733 for eteplirsen 10 mL vial.
CONCLUSIONS: Based on these models, there is a huge gap between the prices of orphan drugs and their value-based prices, which highlights the need for major policy reforms in the assessment and pricing of orphan drugs.
摘要：
目的：Ataluren和eteplirsen是孤儿药，在突变特异性亚组中延迟Duchenne型肌营养不良的进展。它们尚未在埃及获得批准，但有望很快进入市场。这项研究描述了两种成本效用模型，将药物与护理标准进行了比较。
方法：我们使用了一个基于动态状态的5个状态的分区生存模型来模拟一个5岁的动态患者队列。从公开的模型获得基线曲线;然后使用来自McDonald等人的研究的护理标准的Kaplan-Meier曲线更新行走损失曲线。通过校准到该曲线来更新其他曲线。成本和公用事业来自当地的一项研究。进行确定性和概率敏感性分析。价格是根据其他孤儿药的价格估算的。
结果：在基本情况下，ataluren1000mg和eteplirsen50mg/mL导致EGP51745605和EGP69652533/质量调整寿命年的成本效益比增加，分别,他们的假设价格为阿塔利人30袋包装的EGP308600和eteplirsen10毫升小瓶的EGP62800。增量成本效益比对卫生国家公用事业敏感，但对国家成本不敏感。在EGP911719/质量调整寿命年阈值下，基于价值的价格为:Atalurein1000mg的EGP4680和eteplirsen10mL小瓶的EGP733.
结论：基于这些模型，孤儿药的价格与基于价值的价格之间存在巨大差距，这凸显了孤儿药评估和定价方面进行重大政策改革的必要性。
方法：我们使用了一个基于动态状态的5个状态的分区生存模型来模拟一个5岁的动态患者队列。从公开的模型获得基线曲线;然后使用来自McDonald等人的研究的护理标准的Kaplan-Meier曲线更新行走损失曲线。通过校准到该曲线来更新其他曲线。成本和公用事业来自当地的一项研究。进行确定性和概率敏感性分析。价格是根据其他孤儿药的价格估算的。
结果：在基本情况下，ataluren1000mg和eteplirsen50mg/mL导致EGP51745605和EGP69652533/质量调整寿命年的成本效益比增加，分别,他们的假设价格为阿塔利人30袋包装的EGP308600和eteplirsen10毫升小瓶的EGP62800。增量成本效益比对卫生国家公用事业敏感，但对国家成本不敏感。在EGP911719/质量调整寿命年阈值下，基于价值的价格为:Atalurein1000mg的EGP4680和eteplirsen10mL小瓶的EGP733.
结论：基于这些模型，孤儿药的价格与基于价值的价格之间存在巨大差距，这凸显了孤儿药评估和定价方面进行重大政策改革的必要性。
