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尼达尼布治疗间质性肺疾病的价值:一种多准则决策分析。

A multi-criteria decision analysis on the value of nintedanib for interstitial lung diseases.

机构信息

Department of Health Economics, WEBER, Madrid, Spain.

Department of Quantitative Methods in Economics and Management, University of Las Palmas de Gran Canaria, Las Palmas, Spain.

出版信息

Int J Technol Assess Health Care. 2022 Aug 1;38(1):e64. doi: 10.1017/S0266462322000459.

Abstract

OBJECTIVES

Our aim was to assess the value of nintedanib for non-idiopathic progressive fibrosing interstitial lung disease (non-IPF PF-ILD) and systemic sclerosis-associated ILD (SSc-ILD) in the Spanish context, using a multi-criteria decision analysis (MCDA).

METHODS

Following an adaptation of the Evidence and Value: Impact on DEcision Making (EVIDEM) MCDA methodology, the estimated value of nintedanib was obtained by means of an additive linear model that combined individual weights (100-points distribution) of criteria with the individual scoring of nintedanib in each criterion for every indication, assigned by a multidisciplinary committee of twelve clinicians, patients, pharmacists, and decision-makers. To assess the reproducibility, an alternative weighting method was applied, as well as a re-test of weights and scores at a different moment of time.

RESULTS

The experts committee recognized nintedanib as an intervention with a positive value contribution in comparison to placebo for the treatment of non-IPF PF-ILD (0.50 ± 0.16, on a scale from -1 to 1) and SSc-ILD (0.40 ± 0.12), diseases which were considered as very severe and with high unmet needs. The drug was perceived as a treatment that provides an added therapeutic benefit for patients (0.06-0.07), given its proven clinical efficacy (0.05-0.06), slight improvements in patient-reported outcomes (0.01-0.02), and similar safety profile than placebo (-0.04-0.00), which will likely be positioned as a recommended therapy in the next clinical practice guidelines updates.

CONCLUSIONS

Under this increasingly used methodology, nintedanib has shown to provide a positive value estimate for non-IPF PF-ILD and SSc-ILD when compared to placebo in Spain.

摘要

目的

我们旨在使用多准则决策分析(MCDA)评估尼达尼布在西班牙非特发性进行性纤维化间质性肺疾病(非特发性纤维化性间质性肺病,非 IPF PF-ILD)和系统性硬化症相关间质性肺病(SSc-ILD)中的价值。

方法

在对证据和价值:对决策的影响(EVIDEM)MCDA 方法进行改编后,通过一种加性线性模型获得尼达尼布的估计值,该模型结合了由十二位临床医生、患者、药剂师和决策者组成的多学科委员会对每个适应证的每个标准的个人权重(100 分分布)与尼达尼布在每个标准中的个人评分进行组合。为了评估可重复性,应用了替代加权方法,以及在不同时间对权重和评分进行重新测试。

结果

专家委员会认为尼达尼布在治疗非特发性纤维化性间质性肺病(非 IPF PF-ILD)和系统性硬化症相关间质性肺病(SSc-ILD)方面与安慰剂相比具有积极的价值贡献(0.50±0.16,范围为-1 至 1),这些疾病被认为非常严重且有很高的未满足的需求。该药物被认为是为患者提供附加治疗益处的治疗方法(0.06-0.07),因为其具有已证明的临床疗效(0.05-0.06)、患者报告结局的轻微改善(0.01-0.02)和与安慰剂相似的安全性特征(-0.04-0.00),这可能会在未来的临床实践指南更新中被推荐为一种治疗方法。

结论

在这种越来越常用的方法下,尼达尼布在西班牙与安慰剂相比,为非特发性纤维化性间质性肺病和系统性硬化症相关间质性肺病提供了积极的价值估计。

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