Hoogendoorn Martine, Feenstra Talitha L, Asukai Yumi, Briggs Andrew H, Hansen Ryan N, Leidl Reiner, Risebrough Nancy, Samyshkin Yevgeniy, Wacker Margarethe, Rutten-van Mölken Maureen P M H
Institute for Medical Technology Assessment (iMTA), Erasmus University Rotterdam, Rotterdam, The Netherlands.
Department for Prevention and Health Services Research, National Institute for Public Health and the Environment (RIVM), Bilthoven, The Netherlands; Department of Epidemiology, University Medical Centre Groningen, Groningen, The Netherlands.
Value Health. 2017 Mar;20(3):397-403. doi: 10.1016/j.jval.2016.10.016. Epub 2017 Jan 3.
To validate outcomes of presently available chronic obstructive pulmonary disease (COPD) cost-effectiveness models against results of two large COPD trials-the 3-year TOwards a Revolution in COPD Health (TORCH) trial and the 4-year Understanding Potential Long-term Impacts on Function with Tiotropium (UPLIFT) trial.
Participating COPD modeling groups simulated the outcomes for the placebo-treated groups of the TORCH and UPLIFT trials using baseline characteristics of the trial populations as input. Groups then simulated treatment effectiveness by using relative reductions in annual decline in lung function and exacerbation frequency observed in the most intensively treated group compared with placebo as input for the models. Main outcomes were (change in) total/severe exacerbations and mortality. Furthermore, the absolute differences in total exacerbations and quality-adjusted life-years (QALYs) were used to approximate the cost per exacerbation avoided and the cost per QALY gained.
Of the six participating models, three models reported higher total exacerbation rates than observed in the TORCH trial (1.13/patient-year) (models: 1.22-1.48). Four models reported higher rates than observed in the UPLIFT trial (0.85/patient-year) (models: 1.13-1.52). Two models reported higher mortality rates than in the TORCH trial (15.2%) (models: 20.0% and 30.6%) and the UPLIFT trial (16.3%) (models: 24.8% and 36.0%), whereas one model reported lower rates (9.8% and 12.1%, respectively). Simulation of treatment effectiveness showed that the absolute reduction in total exacerbations, the gain in QALYs, and the cost-effectiveness ratios did not differ from the trials, except for one model.
Although most of the participating COPD cost-effectiveness models reported higher total exacerbation rates than observed in the trials, estimates of the absolute treatment effect and cost-effectiveness ratios do not seem different from the trials in most models.
对照两项大型慢性阻塞性肺疾病(COPD)试验——为期3年的COPD健康改善全球策略(TORCH)试验和为期4年的噻托溴铵对功能影响的潜在长期效应研究(UPLIFT)试验的结果,验证目前可用的COPD成本效益模型的结果。
参与研究的COPD建模小组以试验人群的基线特征为输入,模拟TORCH试验和UPLIFT试验中安慰剂治疗组的结果。然后,各小组以与安慰剂相比在最强化治疗组中观察到的肺功能年下降率和急性加重频率的相对降低为模型输入,模拟治疗效果。主要结局为总/重度急性加重次数(变化)和死亡率。此外,总急性加重次数和质量调整生命年(QALY)的绝对差异用于估算避免每次急性加重的成本和每获得一个QALY的成本。
在参与研究的六个模型中,三个模型报告的总急性加重率高于TORCH试验中观察到的率(1.13次/患者年)(模型:1.22 - 1.48次)。四个模型报告的率高于UPLIFT试验中观察到的率(0.85次/患者年)(模型:1.13 - 1.52次)。两个模型报告的死亡率高于TORCH试验(15.2%)(模型:20.0%和30.6%)和UPLIFT试验(16.3%)(模型:24.8%和36.0%),而一个模型报告的率较低(分别为9.8%和12.1%)。治疗效果模拟显示,除一个模型外,总急性加重次数的绝对减少、QALY的增加以及成本效益比与试验结果无差异。
尽管大多数参与研究的COPD成本效益模型报告的总急性加重率高于试验中观察到的率,但在大多数模型中,绝对治疗效果和成本效益比的估计似乎与试验结果并无不同。