Criner Gerard, Martinez Fernando, Gandhi Hitesh, Pyenson Bruce, Feigler Norbert, Emery Matthew, Gupta Umang, Vaduganathan Muthiah
Temple University, Philadelphia, Pennsylvania.
Weill Cornell Medicine, New York, New York.
J Health Econ Outcomes Res. 2023 Jan 24;10(1):20-27. doi: 10.36469/001c.55635. eCollection 2023.
The US population includes 24 million to 29 million people with diagnosed and undiagnosed chronic obstructive pulmonary disease (COPD). Studies have demonstrated the safety and efficacy of single-inhaler triple therapy (SITT) in reducing COPD exacerbations. Long-term population implications of SITT use have not been quantified. This simulation-based projection aimed to estimate the potential impact of widespread SITT use on the US COPD population. Exacerbation and all-cause mortality reductions reported in the Efficacy and Safety of Triple Therapy in Obstructive Lung Disease trial (ETHOS; NCT02465567) were used to project clinical outcomes in US patients meeting ETHOS trial eligibility criteria (ETHOS-Eligible) and patients meeting a practical definition of SITT eligibility (Expanded ETHOS-Eligible). The US COPD population was modeled with 1000 simulations of patient progression over 10 years. Agent characteristics were based on literature and claims analysis of the 2016-2018 Medicare 100% fee-for-service and IBM MarketScan databases. Agent annual characteristics reflected incident cases, changes in COPD severity, treatment, mortality, and exacerbations under status quo treatment patterns and scenarios for the adoption of SITT. The scenarios assumed the reduced exacerbation and mortality rates associated with SITT according to ETHOS trial outcomes mean values. Higher than current SITT adoption over 10 years would be expected to substantially reduce COPD exacerbation-associated hospitalizations by 2 million. Applying mean improvements reported in ETHOS for SITT would extend average patient life expectancy 2.2 years for ETHOS-Eligible patients and 1.7 years for Expanded ETHOS-Eligible patients. The number needed to treat to extend the average patient life by 1 year was 8 for the ETHOS-Eligible population and 10 for the Expanded ETHOS-Eligible population. Widespread SITT adoption may be impeded by competitive pressures from generic treatments and nonadherence, and efficacy observed in clinical trials may not occur in real-world populations. Assuming ETHOS treatment effects and adherence translate to clinical practice, higher than current use of SITT can substantially reduce COPD exacerbations and hospitalizations and extend survival. These results should be viewed cautiously, because the improved outcomes for SITT in the ETHOS final retrieved vital statistics data were not statistically significant for all comparator therapy groups.
美国有2400万至2900万人患有已确诊和未确诊的慢性阻塞性肺疾病（COPD）。研究已证明单吸入器三联疗法（SITT）在减少COPD急性加重方面的安全性和有效性。SITT使用对长期人群的影响尚未量化。这项基于模拟的预测旨在估计广泛使用SITT对美国COPD人群的潜在影响。阻塞性肺病三联疗法的疗效和安全性试验（ETHOS；NCT02465567）中报告的急性加重和全因死亡率降低情况，被用于预测符合ETHOS试验资格标准的美国患者（符合ETHOS标准）以及符合SITT资格实际定义的患者（扩展符合ETHOS标准）的临床结局。对美国COPD人群进行了建模，模拟了1000次患者在10年中的病情进展。个体特征基于对2016 - 2018年医疗保险100%按服务收费和IBM MarketScan数据库的文献及索赔分析。个体年度特征反映了在现状治疗模式和采用SITT的情况下的新发病例、COPD严重程度变化、治疗、死亡率和急性加重情况。这些情景假设根据ETHOS试验结果均值，SITT可降低急性加重和死亡率。预计在10年中采用高于当前水平的SITT将使与COPD急性加重相关的住院人数大幅减少200万。应用ETHOS中报告的SITT平均改善情况，对于符合ETHOS标准的患者，平均患者预期寿命将延长2.2年，对于扩展符合ETHOS标准的患者将延长1.7年。对于符合ETHOS标准的人群，使平均患者寿命延长1年所需治疗的人数为8人，对于扩展符合ETHOS标准的人群为10人。广泛采用SITT可能会受到仿制药治疗的竞争压力和不依从性的阻碍，并且临床试验中观察到的疗效可能在真实人群中不会出现。假设ETHOS治疗效果和依从性可转化为临床实践，高于当前水平使用SITT可大幅减少COPD急性加重和住院人数，并延长生存期。这些结果应谨慎看待，因为在ETHOS最终检索到的生命统计数据中，SITT的改善结局对所有对照治疗组而言在统计学上并不显著。
