The Center for the Evaluation of Value and Risk in Health, Institute for Clinical Research and Health Policy Studies, Tufts Medical Center, Boston, MA.
Janssen Scientific Affairs, LLC, Titusville, NJ.
J Manag Care Spec Pharm. 2024 Jun;30(6):541-548. doi: 10.18553/jmcp.2024.30.6.541.
Health plan coverage is central to patient access to care, especially for rare, chronic diseases. For specialty drugs, coverage varies, resulting in barriers to access. Pulmonary arterial hypertension (PAH) is a rare, progressive, and fatal disease. Guidelines suggest starting or rapidly escalating to combination therapy with drugs of differing classes (phosphodiesterase 5 inhibitors [PDE5is], soluble guanylate cyclase stimulators [sGC stimulators], endothelin receptor antagonists [ERAs], and prostacyclin pathway agents [PPAs]).
To assess the variation in commercial health plan coverage for PAH treatments and how coverage has evolved. To examine the frequency of coverage updates and evidence cited in plan policies.
We used the Tufts Medical Center Specialty Drug Evidence and Coverage database, which includes publicly available specialty drug coverage policies. Overall, and at the drug and treatment class level, we identified plan-imposed coverage restrictions beyond the drug's US Food and Drug Administration label, including step therapy protocols, clinical restrictions (eg, disease severity), and prescriber specialty requirements. We analyzed variation in coverage restrictiveness and how coverage has changed over time. We determined how often plans update their policies. Finally, we categorized the cited evidence into 6 different types.
Results reflected plan coverage policies for 13 PAH drugs active between August 2017 and August 2022 and issued by 17 large US commercial health plans, representing 70% of covered lives. Coverage restrictions varied mainly by step therapy protocols and prescriber restrictions. Seven plans had step therapy protocols for most drugs, 9 for at least one drug, and 1 had none. Ten plans required specialist (cardiologist or pulmonologist) prescribing for at least one drug, and 7 did not. Coverage restrictions increased over time: the proportion of policies with at least 1 restriction increased from 38% to 73%, and the proportion with step therapy protocols increased from 29% to 46%, with generics as the most common step. The proportion of policies with step therapy protocols increased for every therapy class with generic availability: 18% to 59% for ERAs, 33% to 77% for PDE5is, and 33% to 43% for PPAs. The proportion of policies with prescriber requirements increased from 24% to 48%. Plans updated their policies 58% of the time annually and most often cited the 2019 CHEST clinical guidelines, followed by randomized controlled trials.
Plan use of coverage restrictions for PAH therapies increased over time and varied across both drugs and plans. Inconsistency among health plans may complicate patient access and reduce the proportion who can persist on PAH treatments.
健康计划的覆盖范围是患者获得医疗服务的关键，特别是对于罕见的慢性病。对于专科药物，覆盖范围存在差异，从而导致获得药物的障碍。肺动脉高压（PAH）是一种罕见的、进行性的、致命的疾病。指南建议开始或快速升级到不同类别的药物联合治疗（磷酸二酯酶 5 抑制剂 [PDE5i]、可溶性鸟苷酸环化酶刺激剂 [sGC 刺激剂]、内皮素受体拮抗剂 [ERA] 和前列环素途径药物 [PPA]）。
评估商业健康计划对 PAH 治疗的覆盖范围的变化，以及覆盖范围的演变情况。检查计划政策中更新覆盖范围的频率和引用的证据。
我们使用了塔夫茨医疗中心专科药物证据和覆盖数据库，其中包括公开的专科药物覆盖政策。总体而言，在药物和治疗类别层面上，我们确定了计划实施的药物标签以外的覆盖范围限制，包括阶梯治疗方案、临床限制（例如疾病严重程度）和处方专家要求。我们分析了覆盖范围限制的变化情况，以及随着时间的推移覆盖范围的变化情况。我们确定了计划更新其政策的频率。最后，我们将引用的证据分为 6 种不同类型。
结果反映了 2017 年 8 月至 2022 年 8 月期间 17 家大型美国商业健康计划为 13 种活跃的 PAH 药物制定的覆盖政策，代表了 70%的参保人群。覆盖范围的限制主要取决于阶梯治疗方案和处方限制。7 个计划对大多数药物都有阶梯治疗方案，9 个计划对至少一种药物有阶梯治疗方案，1 个计划没有。10 个计划至少对一种药物要求专家（心脏病专家或肺病专家）处方，而 7 个计划没有。覆盖范围的限制随着时间的推移而增加：至少有 1 个限制的政策比例从 38%增加到 73%，有阶梯治疗方案的政策比例从 29%增加到 46%，最常见的是仿制药作为阶梯治疗方案。每个具有仿制药可用性的治疗类别中，具有阶梯治疗方案的政策比例都有所增加：ERA 从 18%增加到 59%，PDE5i 从 33%增加到 77%，PPA 从 33%增加到 43%。有处方要求的政策比例从 24%增加到 48%。计划每年有 58%的时间更新其政策，最常引用的是 2019 年 CHEST 临床指南，其次是随机对照试验。
随着时间的推移，计划对 PAH 治疗的覆盖范围限制的使用有所增加，并且在药物和计划之间存在差异。健康计划之间的不一致性可能会使患者获得药物的情况复杂化，并降低能够坚持 PAH 治疗的患者比例。
