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法国特发性肺纤维化的卫生经济评价。

Health economic evaluation in idiopathic pulmonary fibrosis in France.

机构信息

a Stève Consultants , Oullins , France.

b Hospices Civils de Lyon, Hôpital Louis Pradel, Service de pneumologie Centre de référence national des maladies pulmonaires rares, Université Claude Bernard Lyon 1, Université de Lyon , Lyon , France.

出版信息

Curr Med Res Opin. 2018 Oct;34(10):1731-1740. doi: 10.1080/03007995.2018.1433143. Epub 2018 Mar 9.

Abstract

BACKGROUND

Idiopathic pulmonary fibrosis (IPF) is a specific form of chronic, progressive, fibrosing interstitial pneumonia of unknown cause. To date, there is no specific cure for IPF, and only two treatments (pirfenidone and nintedanib) have marketing authorizations and recommendations in international and French guidelines.

OBJECTIVES

A cost-utility analysis (CUA) has been conducted to evaluate the efficiency of nintedanib, in comparison to all available alternatives, in a French setting using the official methodological guidelines.

METHODS

A previously developed lifetime Markov model was adapted to the French setting by simulating the progression of IPF patients in terms of lung function decline, incidence of acute exacerbations, and death. Considering the effect of IPF on patients' quality-of-life, a CUA integrating quality adjusted life years (QALY) was chosen as the primary outcome measure in the main analysis. One-way, probabilistic, and scenario sensitivity analyses were performed to evaluate the robustness of the model.

RESULTS

Treatment with nintedanib resulted in an estimated total cost of €76,414 (vs €82,665 for pirfenidone). In comparison with all other available options, nintedanib was predicted to provide the most QALY gained (3.34 vs 3.29). This analysis suggests that nintedanib has a 59.0% chance of being more effective than pirfenidone and s 77.3% chance of being cheaper than pirfenidone. Sensitivity analyses showed the results of the CUA to be robust.

CONCLUSIONS

In conclusion, this CUA has found that nintedanib appears to be a more cost-effective therapeutic option than pirfenidone in a French setting, due to fewer acute exacerbations and a better tolerability profile.

摘要

背景

特发性肺纤维化(IPF)是一种原因不明的慢性、进行性、纤维性间质性肺炎的特殊形式。迄今为止,IPF 尚无特效治疗方法,国际和法国指南中仅推荐了两种治疗药物(吡非尼酮和尼达尼布)。

目的

本研究采用官方方法学指南,在法国开展了一项成本-效用分析(CUA),以评估尼达尼布相对于所有其他可用替代药物的治疗效果。

方法

通过模拟 IPF 患者的肺功能下降、急性加重发作和死亡情况,对先前开发的终生马尔可夫模型进行了调整,以适应法国的情况。考虑到 IPF 对患者生活质量的影响,本研究选择纳入健康调整生命年(QALY)的 CUA 作为主要分析的主要结局指标。此外,还进行了单因素、概率和情景敏感性分析,以评估模型的稳健性。

结果

尼达尼布治疗的总费用估计为 76414 欧元(与吡非尼酮的 82665 欧元相比)。与所有其他可用方案相比,尼达尼布预计能获得更多的 QALY(3.34 比 3.29)。该分析表明,尼达尼布的有效性比吡非尼酮高 59.0%,而成本比吡非尼酮低 77.3%。敏感性分析表明,CUA 的结果具有稳健性。

结论

总之,本研究发现,在法国,尼达尼布似乎比吡非尼酮更具成本效益,因为尼达尼布可减少急性加重发作且具有更好的耐受性。

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