医保准入视角下罕见病用药经济价值评估的难点、方法与建议

Difficulties,Methods,and Suggestions for Assessing the Economic Value of Rare Disease Medications from the Perspective of Health Insurance Access

罕见病用药的经济价值评估在临床试验、经济学评价方法、标准及价值维度等方面存在着诸多不足,各国对此积极探索,比如基于自然史数据库构建对照臂、基于健康状态偏好测量健康状态效用、根据健康获益水平调整支付阈值、纳入更多价值维度。借鉴国外经验,我国可建立高质量和高效率的罕见病真实世界数据库,推动效用和偏好研究,引导罕见病单独阈值和多维价值研究,加快构建罕见病多层次医保体系,从而促进罕见病用药经济价值评估的高质量发展。

In the economic value evaluation of rare disease drugs,there are many shortcomings in clinical trials,economic evaluation methods,standards,and value dimensions.Various countries are actively exploring this,such as building control arms based on natural history databases,measuring health status utility based on health status preferences,adjusting payment thresholds based on health benefit levels,and incorporating more value dimensions.Drawing on foreign experience,China can establish a high-quality and efficient real-world database for rare diseases,promote utility and preference studies,guide the research on individual thresholds and multidimensional value for rare diseases,and accelerate the construction of a multilevel health insurance system for rare diseases,so as to promote the high-quality development of the economic value evaluation of medication for rare diseases.