Yang Min, Mittal Manish, Fendrick A Mark, Brixner Diana, Sherman Bruce W, Liu Yifei, Patel Pankaj, Clewell Jerry, Liu Qing, Garrison Louis P
Analysis Group, Boston, MA, USA.
University of Texas, Austin, TX, USA.
Adv Ther. 2025 Feb;42(2):568-578. doi: 10.1007/s12325-024-03076-5. Epub 2024 Dec 20.
The healthcare system in the United States (US) is complex and often fragmented across national and regional health plans which exhibit substantial variability in benefit design and formulary policies for accessing medications. We propose an access-focused value assessment framework for formulary decision-making for medications to manage immune-mediated inflammatory diseases (IMIDs), where patients are at the center of this framework. Formulary decision-making for IMID medications can be a challenging, even daunting, task with continuously evolving and enhanced treat-to-target goals. Given the complexity of the US healthcare system, patients and their caregivers need assurance from formulary decision-makers that rapid, predictable, and sustained access to both well-established treatments and innovative therapies will be a priority, with a particular emphasis on continuity of effective care. This access-focused patient-centric (APAC) value assessment approach encompasses three "value components"-higher therapeutic goals, better health-related quality of life, and improved work productivity-the monetization of which can be derived using data from clinical trials when real-world data are yet to become available. Measures and assessment approaches are outlined to serve as a pragmatic tool for decision-makers in the US to ensure timely delivery and sustained access of clinically indicated therapies aimed to improve patient outcomes, enhance equity, and increase efficiency.
美国的医疗保健系统复杂,且在国家和地区医疗计划之间常常支离破碎,这些计划在药物获取的福利设计和处方政策方面存在很大差异。我们提出了一个以获取为重点的价值评估框架,用于免疫介导的炎症性疾病(IMID)药物的处方决策,该框架以患者为中心。对于IMID药物进行处方决策可能是一项具有挑战性甚至令人生畏的任务,因为治疗目标在不断演变和提高。鉴于美国医疗保健系统的复杂性,患者及其护理人员需要得到处方决策者的保证,即快速、可预测和持续获取成熟治疗方法和创新疗法将是优先事项,尤其要强调有效护理的连续性。这种以获取为重点的以患者为中心(APAC)的价值评估方法包含三个“价值组成部分”——更高的治疗目标、更好的健康相关生活质量和更高的工作效率,在实际数据尚不可用时,可以使用临床试验数据对这些价值进行货币化。文中概述了相关措施和评估方法,作为美国决策者的实用工具,以确保及时提供并持续获取旨在改善患者预后、提高公平性和提升效率的临床指定疗法。