PT -期刊文章AU -埃文·l·雷诺兹AU -加里·加拉格尔盟盟克洛伊·希尔Mousumi Banerjee AU -亚里士多德Mante盟Gregory j .灵异少女AU -布莱恩·c·卡拉汉TI -成本和利用援助- 10.1212 / WNL New-to-Market神经药物。0000000000201627 DP - 2023年2月28日TA -神经病半岛投注体育官网学PG - e884 e898 VI - 100 IP - 9 4099 - //www.ebmtp.com/content/100/9/e884.short 4100 - //www.ebmtp.com/content/100/9/e884.full所以Neurology2023 2月28日;100 AB -背景和目的本研究的目的是比较的利用率和成本(总和付现)new-to-market神经药物与现有guideline-supported神经药物。方法我们使用医疗制药声称数据库(从2001年到2019年)识别患者诊断为1的11个独立的神经条件和现有guideline-supported new-to-market药物或药物治疗的条件。神经条件包括直立性低血压,脊髓性肌萎缩,杜氏营养不良症疾病、帕金森病、多发性硬化症、肌萎缩性脊髓侧索硬化症,重症肌无力,亨廷顿疾病、迟发性运动障碍,转体基因淀粉样变,偏头痛。New-to-market药物被定义为所有神经药物通过了美国食品和药物管理局(FDA)在2014年和2018年之间。每年,我们确定中位数的和标准化的总成本为30天的每个药物的供应。我们也接受new-to-market药物的患者所占的比例,而所有相关药物的具体条件。结果我们发现大多数new-to-market药物的利用率很小(除了1条件& lt; 20%),与现有相比,guideline-supported药物。现款支付的和标准化的总成本是大大大new-to-market药物。中位数(第25百分位,第75个百分位)付现成本为30天的最大供应2019年药物不良反应(712.8美元(59.8 - 802.0美元))和eculizumab(91.1美元(3.0 - 3216美元。4))。 For new-to-market medications, the distribution of out-of-pocket costs was highly variable and the trends over time were unpredictable compared with existing guideline-supported medications.Discussion Despite the increasing number of FDA-approved neurologic medications, utilization of newly approved medications in the privately insured population remains small. Given the high costs and similar efficacy for most of the new medications, limited utilization may be appropriate. However, for new medications with greater efficacy, future studies are needed to determine whether high costs are a barrier to utilization.ALS=amyotrophic lateral sclerosis; ATTR=TTR amyloidosis; CADTH=Canadian Agency for Drugs and Technologies in Health; CDSS=clinical decision support system; CGRP=calcitonin gene-related peptide; DMD=Duchenne disease; EHR=electronic health record; FDA=Food and Drug Administration; HD=Huntington disease; ICD=International Classification of Diseases; ICER=Institute for Clinical and Economic Review; MAO-B=monoamine oxidase type B; MG=myasthenia gravis; MS=multiple sclerosis; OH=orthostatic hypotension; PD=Parkinson disease; RTBT=real-time benefit tool; SMA=spinal muscular atrophy; TD=tardive dyskinesia; TTR=transthyretin

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