Westerink Lotte, Nicolai Jelmer Lennart Jens, Postma Maarten Jacobus, van Boven Job Frank Martien, Boersma Cornelis
Department of Health Sciences, University of Groningen, University Medical Center Groningen, Groningen, The Netherlands.
Asc Academics Inc., 12 East 49th Street, New York, NY, 10017, USA.
Pharmacoecon Open. 2022 Sep;6(5):647-656. doi: 10.1007/s41669-022-00354-2. Epub 2022 Aug 4.
Progressive fibrosing interstitial lung disease (PF-ILD) is characterised by increased pulmonary fibrosis, lung function decline, acute exacerbations, decreased quality of life and increased mortality. Nintedanib may slow down disease progression, but long-term outcomes are unknown. We aimed to assess the cost-effectiveness of nintedanib in comparison to placebo, both on top of usual care in patients with PF-ILD.
An individual PF-ILD patient simulation model was created, using data and extrapolations from the nintedanib and placebo arms of the INBUILD trial. Clinical outcomes (mortality, exacerbations, lung transplants), economic outcomes (direct and indirect costs) and the cost-effectiveness of nintedanib over a 10-year time horizon were forecasted using the Netherlands as a case example. Disease progression was driven by lung function decline, with forced vital capacity (FVC) health states ranging from < 40 to ≥ 110 FVC of % predicted. Sensitivity and scenario analyses were performed to assess the impact of parameter assumptions on the cost-effectiveness and to test model robustness.
Over a 10-year follow-up, nintedanib gained an average of 1.31 discounted life years and an average of 0.87 discounted quality-adjusted life years (QALYs), resulting in an incremental cost-effectiveness ratio (ICER) of €60,690 per QALY. Sensitivity analyses showed cost variations had a minor impact on the ICER. Results were mainly driven by mortality probabilities and disease-related utilities. Scenario analyses indicated most sensitivity to the time horizon and lung transplantation costs.
Long-term treatment with nintedanib could result in considerable health gains for patients with PF-ILD and can be considered cost-effective under the common willingness-to-pay threshold.
进行性纤维化间质性肺疾病(PF-ILD)的特征是肺纤维化加重、肺功能下降、急性加重、生活质量降低和死亡率增加。尼达尼布可能会减缓疾病进展,但长期结果尚不清楚。我们旨在评估尼达尼布与安慰剂相比的成本效益,二者均用于PF-ILD患者的常规治疗之上。
利用INBUILD试验中尼达尼布组和安慰剂组的数据及外推法,建立了PF-ILD患者个体模拟模型。以荷兰为例,预测了10年时间范围内的临床结局(死亡率、急性加重、肺移植)、经济结局(直接和间接成本)以及尼达尼布的成本效益。疾病进展由肺功能下降驱动,用力肺活量(FVC)健康状态范围为预测值的<40%至≥110%。进行了敏感性和情景分析,以评估参数假设对成本效益的影响并测试模型稳健性。
在10年的随访中,尼达尼布平均获得1.31个贴现生命年和0.87个贴现质量调整生命年(QALY),增量成本效益比(ICER)为每QALY 60,690欧元。敏感性分析表明成本变化对ICER影响较小。结果主要由死亡概率和疾病相关效用驱动。情景分析表明对时间范围和肺移植成本最为敏感。
尼达尼布的长期治疗可为PF-ILD患者带来可观的健康收益,在常见的支付意愿阈值下可被认为具有成本效益。