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美国肺动脉高压患者中前列环素的使用:一项大型医疗保健索赔数据库的回顾性分析。

Prostacyclin Use Among Patients with Pulmonary Arterial Hypertension in the United States: A Retrospective Analysis of a Large Health Care Claims Database.

机构信息

1 Department of Medicine, Mayo Clinic, Jacksonville, Florida.

2 Actelion Pharmaceuticals US, South San Francisco, California.

出版信息

J Manag Care Spec Pharm. 2018 Mar;24(3):291-302. doi: 10.18553/jmcp.2017.17228. Epub 2017 Dec 18.

Abstract

BACKGROUND

Prostacyclins play an important role in the management of pulmonary arterial hypertension (PAH). Intravenous prostacyclin was the first disease-specific treatment for patients with PAH. Subcutaneous and nonparenteral (oral or inhaled) formulations have subsequently become available. However, data are lacking on how these different prostacyclin formulations are being used in clinical practice.

OBJECTIVES

To (a) conduct retrospective analyses of a large U.S. health care claims database to describe the characteristics of patients with PAH initiating prostacyclin therapy, and (b) evaluate their treatment patterns, health care resource use, and associated costs.

METHODS

Truven Commercial and Medicare databases were used to define annual cohorts of adults with PAH between January 1, 2010, and October 31, 2015. These patients were identified based on claims with ICD-9-CM diagnoses indicative of PAH (codes 416.0 or 416.8) and claims for PAH-specific medications and PAH-related procedures. Patients with evidence of receiving a prostacyclin were identified, and prostacyclin use was categorized as parenteral versus nonparenteral. Health care costs were assessed alternatively employing an all-cause and PAH-related perspective.

RESULTS

Of 13,633 adults with identified PAH, 3,006 (22.0%) received a prostacyclin during at least 1 year of the study period, and annual prevalence of prostacyclin use ranged from 19.9% to 22.6%. Across calendar years, the median age of prostacyclin users ranged from 56 to 58 years, and 71.9%-75.8% were female. Among prostacyclin users, parenteral prostacyclin use declined from 63.2% in 2010 to 46.5% in 2015, while use of nonparenteral prostacyclins increased from 39.7% to 56.2% over the same period (both P < 0.001). Few patients (2.7%-4.1%) received both parenteral and nonparenteral formulations in a given calendar year. Among patients using prostacyclins, receipt of other PAH-specific medications increased from 62.1% in 2010 to 79.2% in 2015. Comparing the 6 months preceding the first prostacyclin prescription (any formulation) to the 6 months subsequent, mean overall health care costs rose from $61,243 to $119,283, and PAH-related health care costs increased from $58,815 to $116,661, driven mainly by PAH-specific medications, spending on which increased from $15,053 to $73,705 (all P < 0.001).

CONCLUSIONS

While overall use of prostacyclins was relatively constant from 2010 to 2015, our findings revealed a shift from parenteral to nonparenteral formulations, coupled with increased prescribing of PAH-related medications from other drug classes. Further research is needed to better understand how these changes in patterns of prostacyclin use affect levels of health care resource utilization and costs and patients' overall quality of life.

DISCLOSURES

This research was funded by Actelion Pharmaceuticals US, a Janssen pharmaceutical company of Johnson & Johnson. Burger has received grant funding from Actelion, Gilead Sciences, and United Therapeutics; personal fees from Actelion and Gilead Sciences; and nonfinancial support from Actelion. Pruett, Lickert, and Drake are employees of Actelion. Pruett and Lickert own shares in Actelion. Berger and Murphy are employees of Evidera, a consultancy that received payment from Actelion to conduct this research. Pruett, Lickert, Berger, and Drake contributed to study conception and participated with Burger in study design. Lickert and Murphy performed the data analyses. Burger, Pruett, Lickert, Murphy, and Drake interpreted the data. All authors participated in manuscript drafting and/or critical revision, approved the final manuscript, and agree to be accountable for all aspects of the work.

摘要

背景

前列环素在肺动脉高压(PAH)的治疗管理中发挥着重要作用。静脉内前列环素是 PAH 患者的第一种疾病特异性治疗药物。随后出现了皮下和非肠胃外(口服或吸入)制剂。然而,目前缺乏这些不同前列环素制剂在临床实践中如何使用的数据。

目的

(a)通过回顾性分析大型美国医疗保健索赔数据库,描述开始使用前列环素治疗的 PAH 患者的特征,(b)评估他们的治疗模式、医疗资源使用情况和相关成本。

方法

使用 Truven 商业和医疗保险数据库来定义 2010 年 1 月 1 日至 2015 年 10 月 31 日之间每年的成年 PAH 患者队列。这些患者是根据 ICD-9-CM 诊断代码 416.0 或 416.8 表明存在 PAH 和 PAH 特异性药物和 PAH 相关程序的索赔来确定的。识别出有证据表明接受前列环素的患者,并将前列环素的使用分类为肠胃外与非肠胃外。从全因和 PAH 相关的角度评估医疗保健成本。

结果

在确定的 13633 名成年 PAH 患者中,3006 名(22.0%)在研究期间至少使用了 1 年的前列环素,每年前列环素使用率范围为 19.9%至 22.6%。在历年中,前列环素使用者的中位年龄范围为 56 岁至 58 岁,71.9%-75.8%为女性。在前列环素使用者中,肠胃外前列环素的使用率从 2010 年的 63.2%下降到 2015 年的 46.5%,而同期非肠胃外前列环素的使用率从 39.7%上升到 56.2%(均 P <0.001)。在给定的日历年内,极少数患者(2.7%-4.1%)同时使用肠胃外和非肠胃外制剂。在使用前列环素的患者中,接受其他 PAH 特异性药物的比例从 2010 年的 62.1%上升到 2015 年的 79.2%。比较前列环素处方(任何制剂)前 6 个月和随后 6 个月的总医疗保健费用,从 61243 美元增加到 119283 美元,PAH 相关医疗保健费用从 58815 美元增加到 116661 美元,主要是由于 PAH 特异性药物的支出增加,从 15053 美元增加到 73705 美元(均 P <0.001)。

结论

虽然从 2010 年到 2015 年,整体前列环素的使用相对稳定,但我们的研究结果表明,从肠胃外制剂向非肠胃外制剂的转变,加上从其他药物类别开具更多的 PAH 相关药物,这一趋势表明,需要进一步研究以更好地了解前列环素使用模式的这些变化如何影响医疗资源利用和成本水平以及患者的整体生活质量。

披露

这项研究由 Actelion Pharmaceuticals US 资助,这是 Johnson & Johnson 的 Janssen 制药公司。Burger 曾收到过 Actelion、Gilead Sciences 和 United Therapeutics 的赠款;来自 Actelion 和 Gilead Sciences 的个人酬金;以及来自 Actelion 的非财务支持。Pruett、Lickert 和 Drake 是 Actelion 的员工。Pruett 和 Lickert 拥有 Actelion 的股份。Berger 和 Murphy 是咨询公司 Evidera 的员工,该公司因进行这项研究而从 Actelion 获得报酬。Pruett、Lickert、Berger 和 Drake 参与了研究构想和设计。Lickert 和 Murphy 进行了数据分析。Burger、Pruett、Lickert、Murphy 和 Drake 解释了数据。所有作者都参与了手稿的起草和/或关键修订,批准了最终手稿,并同意对工作的各个方面负责。

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