El-Kersh Karim, Zawadzki Nadine, Watzker Anna, Zhang Shurui, Dalal Dhruv, Lautsch Dominik, Shafrin Jason
University of Arizona College of Medicine, Phoenix.
FTI Consulting, Los Angeles, CA.
J Manag Care Spec Pharm. 2025 Sep;31(9):909-921. doi: 10.18553/jmcp.2025.24354. Epub 2025 Jul 31.
Drug manufacturers often use disease analogs to describe diseases-particularly rare diseases-to payers, policymakers, and stakeholders. However, these comparisons are typically anecdotal. We propose a 4-step, systematic approach to identify disease analogs based on prespecified metrics and apply it to a rare disease, pulmonary arterial hypertension (PAH), as a case study. When there is limited knowledge of a rare disease, such as PAH, the application of the systematic disease analog approach presented can help managed care pharmacists leverage their existing knowledge and perspectives applied to other conditions to inform coverage, tier placement, and pricing decisions.
药品制造商经常使用疾病类似物向支付方、政策制定者和利益相关者描述疾病，尤其是罕见病。然而，这些比较通常只是 anecdotal（此处原文有误，anecdotal 应为 anecdotal，意为轶事性的、传闻的）。我们提出一种基于预先设定的指标来识别疾病类似物的四步系统方法，并将其应用于一种罕见病——肺动脉高压（PAH）作为案例研究。当对一种罕见病（如 PAH）的了解有限时，所提出的系统性疾病类似物方法的应用可以帮助管理式医疗药剂师利用他们应用于其他病症的现有知识和观点，为保险范围、分级定位和定价决策提供信息。
