Department of Health Policy and Management, The Johns Hopkins University Bloomberg School of Public Health, Hampton House 496, 624 N Broadway St, Baltimore, MD 21205. Email:
Am J Manag Care. 2020 Sep 1;26(9):e289-e294. doi: 10.37765/ajmc.2020.88494.
It is unclear on what basis Medicare drug plans impose coverage restrictions on orphan drugs. This study aims to investigate the factors associated with utilization controls in Medicare fee-for-service Part D formularies.
Cross-sectional analysis.
We used multivariate logistic regression to assess the association between orphan drug characteristics and use of formulary utilization controls in 2016. We controlled for number of beneficiaries per drug, exclusivity expiration, and the number of plans and beneficiaries per formulary. We conducted sensitivity analyses using fixed and random effects.
On average, 85% of orphan drugs on a formulary were placed on its highest cost-sharing tier and 76% were subject to prior authorization (PA). Orphan drugs with annual costs of $50,000 or more had twice the odds of having PA requirements compared with less expensive ones. Relative to orphan drugs with a single indication, drugs with multiple indications were more likely to have restrictions. Less effective drugs had 1.5 times the odds of highest tier placement relative to more effective drugs. The presence of black box warnings and patient assistance programs were associated with more restricted access. Orphan drugs with generics were less likely to undergo restrictions than those without generics (all P < .05).
Plans are making evidence-based decisions by rewarding more clinically effective and safer orphan drugs. They are penalizing drugs with multiple indications. Surprisingly, plans place fewer restrictions on orphan drugs that have a generic equivalent, which may further discourage generic entry into the orphan space, where competition is already sparse.
尚不清楚医疗保险药品计划对孤儿药施加覆盖限制的依据是什么。本研究旨在调查医疗保险按服务收费计划 D 部分处方集与利用控制相关的因素。
横断面分析。
我们使用多变量逻辑回归来评估孤儿药特征与 2016 年处方集利用控制使用之间的关联。我们控制了每一种药物的受益人数、专有权到期以及每个处方集的计划和受益人数。我们使用固定效应和随机效应进行了敏感性分析。
平均而言，处方集中 85%的孤儿药被置于其最高自付额级别，76%的孤儿药受到事先授权（PA）的限制。年费用为 50000 美元或以上的孤儿药与费用较低的孤儿药相比，有两倍的可能性需要 PA 要求。与单一适应症的孤儿药相比，具有多种适应症的孤儿药更有可能受到限制。与更有效的药物相比，效果较差的药物具有 1.5 倍的可能性被置于最高级别。存在黑框警告和患者援助计划与更受限的准入相关。与没有仿制药的孤儿药相比，有仿制药的孤儿药进行限制的可能性较小（均 P < 0.05）。
计划通过奖励更具临床疗效和更安全的孤儿药来做出基于证据的决策。他们对具有多种适应症的药物进行处罚。令人惊讶的是，计划对具有通用等效药物的孤儿药施加的限制较少，这可能会进一步阻碍仿制药进入孤儿药领域，而孤儿药领域的竞争已经很少。
