中国罕见病药物经济学评估适用模型与支付阈值参考标准探讨  

Consideration of pharmacoeconomic evaluation model and standard of payment threshold for rare diseases in China

作　　者：宣建伟 孙巧 XUAN Jian-wei;SUN Qiao(Health Economic Research Institute,School of Pharmacy,SunYat-sen University,Guangzhou 200032,China)

机构地区：[1]中山大学药学院医药经济研究所

出　　处：《国际药学研究杂志》2019年第9期659-665,共7页Journal of International Pharmaceutical Research

基　　金：国家重点研发计划精准医学研究重点专项“罕见病临床队列研究”资助项目（2016YFC0901500）

摘　　要：罕见病通常定义为人群中发病率较低的疾病。根据报道,2016年全球发现的罕见病有6084种,涉及到3715个有关的基因。80%罕见病是由遗传引起的,通过显性或隐性遗传或是由于突变而造成基因缺陷。关于罕见病的确定标准各国均不相同,如欧盟定为患病率<1/2000,美国定为患病率<1/2500。用于预防、治疗、诊断罕见病的药物被称为孤儿药。由于研发难度大、目标人群少、投资风险大、生产成本高等特点,使得罕见病药物价格高昂的问题十分突出。在进行成本-效果评价时,其结果所生成的增量成本-效果比很可能会超过目前大多数国家和地区所采用的成本-效果支付阈值,导致许多国家提高支付阈值参考标准(3~25倍),或直接对罕见病豁免卫生技术评估(HTA)或仅对其基金影响进行评估。本文对国际上通用的罕见病HTA方法进行了系统梳理,并根据罕见病特征对罕见病药物HTA适用模型的建立及多维度价值评估框架进行了讨论。同时,根据国际通用方法对中国孤儿药HTA支付阈值的确定进行了探讨。Rare diseases are usually defined as diseases with a low incidence in the population.According to reports,there were 6084 rare diseases found worldwide in 2016,involving 3715 related genes.80%of rare diseases are caused by heredity,dominant or recessive inheritance or mutations.The definition of rare diseases by“incidence”in the population varies from country to country,like the EU<1/2000 and the United States<1/2500.A drug used to prevent,treat,or diagnose a rare disease is called an orphan drug.Due to the difficulty of research and development in this small target population,the high investment risk and the high R&D cost,the problem associated with high price of orphan drugs is very prominent.In the cost-effectiveness evaluation,the resulting incremental cost-effectiveness ratio(ICER)is likely to exceed the willingness to pay threshold adopted by most countries and regions,resulting in multiple countries raising its willingness to pay threshold reference standard(3-25 times GDP per capital)for orphan drugs,or simply exempting health technology assessment(HTA)assessment for orphan drugs and only evaluating the impact on medical insurance fund.This paper systematically reviewed the various internationally accepted orphan drug HTA assessment methodologies.Based on the characteristics of rare diseases,the paper discusses the establishment of a model for the evaluation of orphan drugs and the multi-dimensional value assessment framework for HTA.At the same time,according to the internationally accepted norm,the paper also explores the way to determine the willingness to pay threshold in HTA assessment for orphan drugs in China.

关 键 词：罕见病 孤儿药 卫生技术评估(HTA) 药物经济学模型 支付阈值 

分 类 号：R954.4[医药卫生—药学] R956