Song Ci, Kunovszki Peter, Beaudet Amélie
Janssen Global Commercial Strategy Organization.
Actelion Pharmaceuticals Ltd.
J Health Econ Outcomes Res. 2022 Jun 8;9(1):151-160. doi: 10.36469/001c.35246. eCollection 2022.
Agents targeting the prostacyclin (PGI) pathway are important in managing pulmonary arterial hypertension (PAH). No head-to-head clinical trials have compared outcomes between the 3 different PGI-pathway drugs most commonly available in countries with advanced healthcare: oral selexipag, inhaled iloprost, and parenteral (subcutaneous or intravenous) treprostinil. To conduct retrospective database analyses to describe characteristics of patients with PAH initiating therapy with these agents and compare the rate and risk of healthcare facility encounters and drug persistence. Data were obtained from the Optum™ Clinformatics® Data Mart and Truven™ Health Analytics® MarketScan® Commercial Claims and Encounters databases from July 1, 2008, to September 30, 2020 (Optum™), or October 31, 2020 (Truven™). Patients were categorized into index-drug cohorts based on first pharmacy claims for selexipag, inhaled iloprost, or parenteral treprostinil. Eligible patients were ≥18 years of age with ≥1 ICD-9-CM or ICD-10-CM diagnosis code indicating pulmonary hypertension and no diagnosis code suggesting Group 3-5 pulmonary hypertension. Rates of hospitalization (inpatient admissions), emergency room visits, or outpatient visits per person-year were calculated. Drug persistence was measured as time to discontinuation of index drug. Multivariable analyses were performed to compare outcomes with selexipag vs inhaled iloprost and parenteral treprostinil, adjusting for baseline characteristics using inverse probability of treatment weighting. Overall, 583 patients were included in the Optum™ sample and 482 in the Truven™ sample. Mean (SD) age was 61.7 (14.5) and 49.3 (11.3) years, respectively; 74.4% and 75.7% of patients, respectively, were women. In the pooled samples, after adjustment for baseline characteristics, selexipag had a lower risk than inhaled iloprost or parenteral treprostinil for hospitalization (relative rate ratio [95% CI], 0.40 [0.22, 0.75], and 0.26 [0.17, 0.39]) and outpatient visits (0.66 [0.56, 0.78] and 0.76 [0.66, 0.88]). Trends toward lower risk of emergency room visits did not attain statistical significance. Drug discontinuation risk was 16% and 36% lower with selexipag vs parenteral treprostinil and inhaled iloprost, respectively. In real-world use, selexipag appears to be associated with lower rates of hospitalization and outpatient visits than inhaled iloprost or parenteral treprostinil. Further research is required to identify factors underlying these differences.
靶向前列环素(PGI)通路的药物在肺动脉高压(PAH)的治疗中具有重要作用。在医疗水平先进的国家,最常用的3种不同的PGI通路药物(口服司来帕格、吸入用伊洛前列素和肠外(皮下或静脉注射)曲前列尼尔)之间尚未进行过直接比较疗效的临床试验。开展回顾性数据库分析,以描述开始使用这些药物治疗的PAH患者的特征,并比较医疗机构就诊率和药物持续使用情况及风险。数据来自Optum™ Clinformatics® Data Mart和Truven™ Health Analytics® MarketScan® Commercial Claims and Encounters数据库,时间范围为2008年7月1日至2020年9月30日(Optum™)或2020年10月31日(Truven™)。根据首次开具司来帕格、吸入用伊洛前列素或肠外曲前列尼尔的药房处方,将患者分为索引药物队列。符合条件的患者年龄≥18岁,有≥1个ICD - 9 - CM或ICD - 10 - CM诊断代码表明患有肺动脉高压,且无诊断代码提示3 - 5组肺动脉高压。计算每人每年的住院率(住院入院)、急诊室就诊率或门诊就诊率。药物持续使用情况以停用索引药物的时间来衡量。进行多变量分析,比较司来帕格与吸入用伊洛前列素和肠外曲前列尼尔的疗效,并使用治疗权重的逆概率对基线特征进行调整。总体而言,Optum™样本纳入了583例患者,Truven™样本纳入了482例患者。平均(标准差)年龄分别为61.7(14.5)岁和49.3(11.3)岁;患者中女性分别占74.4%和75.7%。在汇总样本中,调整基线特征后,司来帕格相比吸入用伊洛前列素或肠外曲前列尼尔,住院风险更低(相对率比[95%置信区间],分别为0.40[0.22, 0.75]和0.26[0.17, 0.39]),门诊就诊风险也更低(分别为0.66[0.56, 0.78]和0.76[0.66, 0.88])。急诊室就诊风险较低的趋势未达到统计学显著性。与肠外曲前列尼尔和吸入用伊洛前列素相比,司来帕格的药物停用风险分别低16%和36%。在实际应用中,司来帕格相比吸入用伊洛前列素或肠外曲前列尼尔,住院率和门诊就诊率似乎更低。需要进一步研究以确定这些差异背后的因素。