Duke Clinical Research Institute, Durham, North Carolina.
Amgen Inc, Thousand Oaks, California.
JAMA Cardiol. 2017 Nov 1;2(11):1217-1225. doi: 10.1001/jamacardio.2017.3451.
Although PCSK9 inhibitors (PCSK9i) were approved in 2015, their high cost has led to strict prior authorization practices and high copays, and use of PSCK9i in clinical practice has been low.
To evaluate patient access to PCSK9i among those prescribed therapy.
DESIGN, SETTING, AND PARTICIPANTS: Using pharmacy transaction data, we evaluated 45 029 patients who were newly prescribed PCSK9i in the United States between August 1, 2015, and July 31, 2016.
The proportion of PCSK9i prescriptions approved and abandoned (approved but unfilled); multivariable analyses examined factors associated with approval/abandonment including payor, prescriber specialty, pharmacy benefit manager, out-of-pocket cost (copay), clinical diagnoses, lipid-lowering medication use, and low-density lipoprotein cholesterol levels.
Of patients given an incident PCSK9i prescription, 51.2% were women, 56.6% were 65 years or older, and 52.5% had governmental insurance. Of the patients given a prescription, 20.8% received approval on the first day, and 47.2% ever received approval. Of those approved, 65.3% filled the prescription, resulting in 30.9% of those prescribed PCSK9i ever receiving therapy. After adjustment, patients who were older, male, and had atherosclerotic cardiovascular disease were more likely to be approved, but approval rates did not vary by patient low-density lipoprotein cholesterol level nor statin use. Other factors associated with drug approval included having government vs commercial insurance (odds ratio [OR], 3.3; 95% CI, 2.8-3.8), and those filled at a specialty vs retail pharmacy (OR, 1.96; 95% CI, 1.66-2.33). Approval rates varied nearly 3-fold among the top 10 largest pharmacy benefit managers. Prescription abandonment by patients was most associated with copay costs (C statistic, 0.86); with abandonment rates ranging from 7.5% for those with $0 copay to more than 75% for copays greater than $350.
In the first year of availability, only half of patients prescribed a PCSK9i received approval, and one-third of approved prescriptions were never filled owing to copay.
尽管 PCSK9 抑制剂（PCSK9i）于 2015 年获得批准，但由于其高昂的价格，导致了严格的事先授权规定和高额的共付额，因此 PCSK9i 在临床实践中的应用率很低。
评估开出治疗处方的患者获得 PCSK9i 的机会。
设计、地点和参与者：我们使用药房交易数据，评估了 2015 年 8 月 1 日至 2016 年 7 月 31 日期间在美国新开出 PCSK9i 处方的 45029 名患者。
PCSK9i 处方获得批准和放弃（批准但未填写）的比例；多变量分析检查了与批准/放弃相关的因素，包括支付方、开处方医生的专业、药房福利经理、自付费用（共付额）、临床诊断、降脂药物使用和低密度脂蛋白胆固醇水平。
在接受 PCSK9i 治疗的患者中，51.2%为女性，56.6%为 65 岁或以上，52.5%有政府保险。在开出处方的患者中，20.8%在第一天获得批准，47.2%曾获得批准。在获得批准的患者中，65.3%填写了处方，导致 30.9%的患者接受了治疗。调整后，年龄较大、男性和患有动脉粥样硬化性心血管疾病的患者更有可能获得批准，但批准率与患者的低密度脂蛋白胆固醇水平或他汀类药物的使用无关。其他与药物批准相关的因素包括有政府保险与商业保险（比值比[OR]，3.3；95%置信区间[CI]，2.8-3.8），以及在专科药房与零售药房配药（OR，1.96；95%CI，1.66-2.33）。在最大的 10 家药房福利管理公司中，批准率相差近 3 倍。患者放弃处方最主要与共付额有关（C 统计量，0.86）；对于共付额为 0 美元的患者，放弃率为 7.5%，而对于共付额超过 350 美元的患者，放弃率则超过 75%。
在可获得的第一年，只有一半的开出 PCSK9i 处方的患者获得了批准，而批准的处方中有三分之一从未填写，原因是共付额过高。
