Carolinas HealthCare System Neurosciences Institute. Email:
Am J Manag Care. 2018 Apr;24(9 Suppl):S175-S186.
Amyotrophic lateral sclerosis (ALS) is a progressive and fatal neuromuscular disease affecting approximately 5 out of every 100,000 individuals living in the United States. ALS is associated with 50% mortality within 30 months of initial symptom onset. The rarity of the disease, along with the significant inter- and intra-patient variability in clinical course and a lack of reliable biomarkers, have rendered the development of effective agents to treat ALS a challenge. Because oxidative stress is considered a contributing factor to ALS onset and progression, drugs that eliminate free radicals may protect motor neurons from damage potentially caused by free-radical and oxidative stress. Edaravone is an antioxidant free-radical scavenger approved by the FDA in 2017 for the treatment of ALS. A review of the edaravone clinical development program offers a clearer view of the clinical utility of this agent. Broader treatment success is also influenced by factors such as limited patient access and the restrictive payer environment. Cooperation within the healthcare community, among clinicians, patient advocacy groups, pharmaceutical companies, and managed care payers, must occur to advance ALS management and treatment and improve patient access. Moreover, collaborative discussions are useful in identifying potential solutions to problems currently surrounding patient access.
肌萎缩侧索硬化症（ALS）是一种进行性和致命的神经肌肉疾病，影响大约每 10 万名居住在美国的人中的 5 人。ALS 与初始症状出现后 30 个月内 50%的死亡率相关。该疾病的罕见性，以及临床病程中显著的个体间和个体内变异性，以及缺乏可靠的生物标志物，使得开发治疗 ALS 的有效药物成为一项挑战。由于氧化应激被认为是 ALS 发病和进展的一个促成因素，因此消除自由基的药物可能会保护运动神经元免受自由基和氧化应激潜在损伤。依达拉奉是一种抗氧化自由基清除剂，于 2017 年获得 FDA 批准用于治疗 ALS。对依达拉奉临床开发项目的回顾提供了更清晰的视角，了解该药物的临床应用。更广泛的治疗成功还受到患者可及性有限和支付方环境受限等因素的影响。医疗保健界内的合作，包括临床医生、患者权益组织、制药公司和管理式医疗支付方之间的合作，必须开展以推进 ALS 的管理和治疗并改善患者可及性。此外，协作性讨论有助于确定当前围绕患者可及性的问题的潜在解决方案。
