Boehringer Ingelheim, Paris, France.
Putnam, Paris, France.
Clin Drug Investig. 2024 Nov;44(11):811-828. doi: 10.1007/s40261-024-01398-4. Epub 2024 Oct 24.
The efficacy and safety of empagliflozin in the treatment of chronic kidney disease (CKD) were demonstrated in the EMPA-KIDNEY trial, which showed a 28% reduction in combined risks of kidney disease or death from cardiovascular causes (hazard ratio, 0.72; 95% confidence interval, 0.64-0.82; p < 0.001) compared with placebo. Based on these results, the present study aimed to assess the cost-effectiveness of empagliflozin + standard of care (SoC) compared with SoC alone in the treatment of CKD in France.
A Markov state microsimulation model was adapted to compare the health and economic outcomes in France, considering a healthcare system perspective, in patients treated with empagliflozin in addition to SoC versus patients treated with SoC alone. The model simulated the intention-to-treat population of the trial, transitioning between 18 mutually exclusive and collectively exhaustive health states defined based on the Kidney Disease: Improving Global Outcomes classification. For each arm, the model estimated (over a 25-year time horizon) the number of events and deaths, and the costs associated with these events, to calculate the incremental cost-effectiveness ratio. The resources used were derived using French authorities reports, literature, and French CKD guidelines. Both economic and health outcomes were discounted at a 2.5% annual rate according to French guidelines.
The model predicted that using empagliflozin + SoC to treat patients with CKD would prevent CKD-related complications and deaths associated with a cardiovascular event or all-cause deaths while in kidney replacement therapy, resulting on average in a discounted gain of 1.29 years in overall survival (9.48 years vs. 8.19 with SoC alone). Empagliflozin costs (treatment, events, and disease management) were completely offset by the cost savings from avoided kidney failure events. Overall, empagliflozin + SoC would be more effective and less costly than SoC alone and would therefore be the dominant treatment strategy. The sensitivity analyses conducted support the results' robustness in showing the dominance of empagliflozin + SoC over SoC alone.
The base-case results indicate that empagliflozin + SoC is a dominant strategy compared with the current SoC for the management of CKD in France. Empagliflozin + SoC would have a positive impact on patients with CKD by slowing CKD progression and leading to the prevention of kidney failure events on top of all-stages CKD complications.
在 EMPA-KIDNEY 试验中证实了恩格列净治疗慢性肾脏病（CKD）的疗效和安全性，与安慰剂相比，该药使肾脏病或心血管原因导致的死亡的联合风险降低了 28%（风险比，0.72；95%置信区间，0.64-0.82；p<0.001）。基于这些结果，本研究旨在评估法国恩格列净+标准治疗（SoC）与单独 SoC 治疗 CKD 的成本效益。
采用 Markov 状态微观模拟模型，从法国医疗保健系统的角度，比较加用恩格列净治疗与单独 SoC 治疗的患者的健康和经济结局。该模型模拟了试验的意向治疗人群，在基于肾脏病：改善全球结局（KDIGO）分类的 18 个相互排斥且完全穷尽的健康状态之间进行转换。对于每个治疗组，模型估计（25 年时间范围内）事件和死亡的数量，以及与这些事件相关的成本，以计算增量成本效益比。所使用的资源来源于法国当局报告、文献和法国 CKD 指南。根据法国指南，以 2.5%的年贴现率对经济和健康结局进行贴现。
模型预测，使用恩格列净+SoC 治疗 CKD 患者可预防与 CKD 相关的并发症以及与心血管事件或全因死亡相关的接受肾脏替代治疗的死亡，从而使总体生存的贴现获益平均增加 1.29 年（9.48 年 vs. 单独使用 SoC 时的 8.19 年）。恩格列净的成本（治疗、事件和疾病管理）完全被避免的肾衰竭事件的成本节约所抵消。总体而言，恩格列净+SoC 比单独 SoC 更有效且成本更低，因此是更优的治疗策略。进行的敏感性分析结果支持了恩格列净+SoC 比单独 SoC 更优的结果稳健性，表明恩格列净+SoC 具有优势。
基于现有数据，本研究结果表明，与当前的 SoC 相比，法国 CKD 的管理中，恩格列净+SoC 是一种主导策略。恩格列净+SoC 将通过减缓 CKD 进展和预防 CKD 各阶段并发症之外的肾衰竭事件，对 CKD 患者产生积极影响。
