Schechter Michael S, Sabater-Anaya Natalia, Oster Gerry, Weycker Derek, Wu Hongsheng, Arteaga-Solis Emilio, Bagal Sukirti, McGarry Lisa J, Van Brunt Kate, Geiger Jessica Morlando
Children's Hospital of Richmond at Virginia Commonwealth University, Children's Pavilion, Room 5-544, 1000 East Broad Street, PO Box 980315, Richmond, VA, 23298, USA.
Policy Analysis Inc, Boston, MA, USA.
Pulm Ther. 2023 Dec;9(4):479-498. doi: 10.1007/s41030-023-00241-z. Epub 2023 Oct 24.
Cystic fibrosis (CF) is a life-limiting genetic disease caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) is a CFTR modulator (CFTRm) that targets the underlying cause of CF. Based on safety and efficacy demonstrated in clinical trials, ELX/TEZ/IVA is approved in the US for the treatment of CF in people aged ≥ 2 years who have ≥ 1 F508del-CFTR mutation or a CFTR mutation that is responsive to ELX/TEZ/IVA based on in vitro data. While ELX/TEZ/IVA demonstrated unprecedented improvements in lung function and dramatic reductions in pulmonary exacerbations (PEx) and associated hospitalizations in clinical trials, a limited number of studies have examined the impact of ELX/TEZ/IVA on healthcare resource utilization (HCRU) and associated costs in a real-world setting. The aim of this retrospective study was to evaluate changes in PEx, HCRU, and associated non-CFTRm healthcare costs following initiation of ELX/TEZ/IVA among people with CF aged ≥ 12 years in the US.
We evaluated the rates of PEx, HCRU, and associated costs before and after initiation of ELX/TEZ/IVA in people with CF aged ≥ 12 years using data from the Merative MarketScan® Commercial Claims and Encounters Database and the Merative Multi-State Medicaid Database from April 21, 2019 to December 31, 2020. Because the study period included time following the onset of the COVID-19 pandemic, we limited our primary analysis to the period prior to the pandemic (October 21, 2019 to March 12, 2020). Outcomes following the onset of the pandemic (March 13 to December 31, 2020) were examined in an exploratory analysis.
In both commercially insured and Medicaid-insured people with CF, ELX/TEZ/IVA was associated with reductions in PEx, hospitalizations, and associated costs prior to the COVID-19 pandemic, and these reductions were maintained following the onset of the pandemic.
These findings suggest that ELX/TEZ/IVA reduces the burden and costs associated with PEx and hospitalizations in people with CF.
囊性纤维化（CF）是一种由囊性纤维化跨膜传导调节因子（CFTR）基因突变引起的危及生命的遗传性疾病。依列卡福/替扎卡福/依伐卡福（ELX/TEZ/IVA）是一种针对CF根本病因的CFTR调节剂（CFTRm）。基于临床试验中证明的安全性和有效性，ELX/TEZ/IVA在美国被批准用于治疗年龄≥2岁、具有≥1个F508del-CFTR突变或基于体外数据对ELX/TEZ/IVA有反应的CFTR突变的CF患者。虽然ELX/TEZ/IVA在临床试验中显示出肺功能前所未有的改善以及肺部加重（PEx）和相关住院次数的显著减少，但在真实世界环境中，仅有少数研究考察了ELX/TEZ/IVA对医疗资源利用（HCRU）和相关成本的影响。这项回顾性研究的目的是评估美国年龄≥12岁的CF患者开始使用ELX/TEZ/IVA后PEx、HCRU及相关非CFTRm医疗成本的变化。
我们使用来自默克多市场扫描®商业索赔和诊疗数据库以及默克多州医疗补助数据库的数据，评估了年龄≥12岁的CF患者开始使用ELX/TEZ/IVA前后的PEx发生率、HCRU及相关成本，研究时间段为2019年4月21日至2020年12月31日。由于研究期间包括了COVID-19大流行开始后的时间，我们将主要分析限制在大流行之前的时间段（2019年10月21日至2020年3月12日）。在探索性分析中考察了大流行开始后（2020年3月13日至12月31日）的结果。
在商业保险和医疗补助保险的CF患者中，ELX/TEZ/IVA与COVID-19大流行之前PEx、住院次数及相关成本的降低相关，并且在大流行开始后这些降低仍得以维持。
这些发现表明，ELX/TEZ/IVA减轻了CF患者PEx和住院相关的负担及成本。
