1 Mayo Clinic, Jacksonville, Florida, New York.
3 Gilead Sciences, Foster City, California.
J Manag Care Spec Pharm. 2018 Aug;24(8):834-842. doi: 10.18553/jmcp.2018.17391. Epub 2018 Feb 13.
Despite multiple treatment options, the prognosis of pulmonary arterial hypertension (PAH) remains poor. PAH patients experience a high economic burden due to comorbidities, hospitalizations, and medication costs. Although combination therapy has been shown to reduce hospitalizations, the relationship between treatment, health care utilization, and costs remains unclear.
To provide a characterization of health care utilization and costs in real-world settings by comparing periods before and after initiating PAH-specific treatment.
This retrospective study identified PAH patients in the Truven Health MarketScan Commercial and Medicare Supplemental Databases between 2010 and 2014 who initiated treatment with endothelin receptor antagonists (ERAs), phosphodiesterase-5 inhibitors (PDE-5Is), or soluble guanylate cyclase (sGC) stimulators. The index date was the date of the first PAH pharmacy claim. We included patients with ≥ 2 medical claims with diagnoses for PAH (ICD-9-CM: 416.0, 416.8) or PAH-related conditions and continuous enrollment in medical and pharmacy benefits for the 6 months before and after the index date. Treatment patterns were assessed at the drug class level (ERAs, PDE-5Is, sGC stimulators, and prostacyclins) from outpatient pharmacy claims during the 6-month post-index period. All-cause and PAH-related utilization and costs were measured. McNemar's and paired t-tests were used to compare patients' health care resource utilization and costs in the 6-month pre- and posttreatment periods.
A total of 3,908 patients met the selection criteria. The study sample was 63% female with a mean age of 63 ± 15 years. Only 5% of patients began initial combination therapy for PAH, defined as claims for ≥ 2 medication classes within the first 30 days of treatment. Treatment interruption (≥ 30-day gap in days supply) of any PAH-specific medication was observed in 38% of patients. Compared with the 6-month pre-index period, the proportion of patients in the 6-month post-index period with any inpatient admission decreased, 42% versus 30% (P < 0.001). In addition, PAH-related inpatient admissions decreased in the 6-month post-index period from 7% to 3% (P < 0.001). After treatment initiation, patients' nonpharmacy medical costs decreased from $48,200 (SD = $117,686) to $33,962 (SD = $90,294; P < 0.001), mainly attributable to reduced inpatient costs. However, total average medical costs including pharmacy costs remained comparable after treatment initiation (pre-index period = $51,455 vs. post-index period = $53,923; P = 0.213).
This study found that while patients' PAH-related pharmacy costs increased after treatment initiation, the increase was offset by reduced inpatient utilization; therefore, total health care costs remained constant. While the majority of patients in this study were treated with monotherapy, the recently completed AMBITION study indicated that initial combination therapy with ambrisentan plus tadalafil reduced PAH-related hospitalizations compared with initial monotherapy with either of these agents. Future cost analyses of patients treated with combination therapy will be required to determine the economic effect of initial combination therapy.
This study was sponsored and funded by Gilead Sciences. Ozbay is an employee of Gilead Sciences. At the time that this project and manuscript were developed, Lazarus was an employee of Gilead Sciences and may own stock/stock options. Riehle, Montejano, and Lenhart are employees of Truven Health Analytics, an IBM company, which received funding from Gilead Sciences to conduct this study. Burger and White do research with, and are paid consultants for, Gilead Sciences; they do not own equity and received no personal compensation for the work here. Burger also reports consultancy and advisory board work for Actelion Pharmaceuticals and grants from Gilead Sciences, Actelion Pharmaceuticals, Bayer, and United Therapeutics.
尽管有多种治疗选择，肺动脉高压（PAH）的预后仍然很差。由于合并症、住院和药物费用，PAH 患者的经济负担很高。尽管联合治疗已被证明可降低住院率，但治疗、医疗保健利用和成本之间的关系仍不清楚。
通过比较开始 PAH 特异性治疗前后的时期，提供真实世界环境中医疗保健利用和成本的特征描述。
这项回顾性研究在 2010 年至 2014 年间，从 Truven Health MarketScan 商业和医疗保险补充数据库中确定了开始内皮素受体拮抗剂（ERA）、磷酸二酯酶-5 抑制剂（PDE-5I）或可溶性鸟苷酸环化酶（sGC）刺激剂治疗的 PAH 患者。索引日期是 PAH 药房索赔的日期。我们纳入了至少有 2 次医疗索赔，诊断为 PAH（ICD-9-CM：416.0、416.8）或 PAH 相关疾病，并在索引日期前后连续 6 个月参加医疗和药房福利的患者。在索引后 6 个月内，从门诊药房索赔中评估药物类别水平（ERA、PDE-5I、sGC 刺激剂和前列环素）的治疗模式。测量全因和 PAH 相关的利用和成本。使用 McNemar 和配对 t 检验比较患者在治疗前和治疗后 6 个月的医疗保健资源利用和成本。
共有 3908 名患者符合选择标准。研究样本中 63%为女性，平均年龄为 63±15 岁。只有 5%的患者开始了 PAH 的初始联合治疗，定义为在治疗的前 30 天内至少有 2 种药物类别的索赔。观察到 38%的患者中断了任何 PAH 特异性药物的治疗（供应天数中断≥30 天）。与 6 个月前索引期相比，索引后 6 个月内任何住院治疗的患者比例下降，分别为 42%和 30%（P <0.001）。此外，在索引后 6 个月内，PAH 相关住院治疗从 7%降至 3%（P <0.001）。在开始治疗后，患者的非药房医疗费用从 48200 美元（SD=117686 美元）降至 33962 美元（SD=90294 美元；P <0.001），主要归因于住院费用减少。然而，包括药房费用在内的总平均医疗费用在开始治疗后仍保持可比（前索引期=51455 美元，后索引期=53923 美元；P=0.213）。
这项研究发现，虽然患者开始治疗后 PAH 相关的药房费用增加，但由于减少了住院治疗，因此增加的费用被抵消；因此，总医疗保健费用保持不变。虽然这项研究中的大多数患者接受了单药治疗，但最近完成的 AMBITION 研究表明，与最初的单药治疗相比，安立生坦联合他达拉非的初始联合治疗降低了 PAH 相关的住院率。需要对接受联合治疗的患者进行进一步的成本分析，以确定初始联合治疗的经济效果。
这项研究由吉利德科学公司赞助和资助。Ozbay 是吉利德科学公司的员工。在开展这个项目和撰写本论文时，Lazarus 是吉利德科学公司的员工，可能拥有公司的股票/股票期权。Riehle、Montejano 和 Lenhart 是 IBM 旗下 Truven Health Analytics 的员工，该公司收到了吉利德科学公司的资金来开展这项研究。Burger 和 White 与吉利德科学公司进行研究，并担任其顾问；他们没有股权，也没有因这项工作而获得个人报酬。Burger 还报告了与 Actelion Pharmaceuticals 的顾问和咨询委员会工作，并从吉利德科学公司、Actelion Pharmaceuticals、Bayer 和 United Therapeutics 获得了资助。
