我国罕见病药品价格水平和可负担性研究

目的:评估我国罕见病药品价格水平及可负担性情况,提出改善罕见病治疗药品可负担性的政策建议,以期为政府主管部门的相关决策提供依据.方法:基于《第一批罕见病目录》和《罕见病诊疗指南(2019年版)》选择罕见病药品,采用中位价格比值法(MPR)评估药品价格水平,用药品年治疗费用和城乡居民年均可支配收入的倍数评估药品可负担性以...

Study on the Price and Affordability of Rare Disease Drugs in China

OBJECTIVE:To evaluate the price and affordability of rare disease drugs in China and provide the suggestions for the improvement of rare disease drug affordability in order to provide reference for the relevant decision-making of government departments.METHODS:According to the List of the First Batch of Rare Diseasesand Diagnosis and Treatment Guideline for Rare Diseases(2019 edition),rare disease drugs were selected.The median price ratio(MPR)was used to evaluate the drug price level,and the ratio of the annual drug costs to the annual disposable income of urban or rural residents was used to evaluate the affordability,and the impact of price management policieson drug prices and affordability.RESULTS and CONCLUSIONS:A total of 71 kinds of rare disease drugs were included,and the median MPR of them was 0.83;among them,the median MPR of the original drugs was 1.13,and the median MPR of the generic drugs was 0.37.The annual cost of 71 rare disease drugs was 0.001-178.43 times the average annual income of urban residents,and 0.003-456.57 times the average annual income of rural residents.There were 21 and 28 drugs whose annual cost exceeded the annual income of urban and rural residents.After the implementation of national medical insurance negotiation or volume based procurement of 14 drugs,the median MPR of these drugs decreased from 1.71 to 0.46.For urban residents,the number of unaffordable drugs had been reduced from 8 to 0;for rural residents,it had been reduced from 10 to 5.In summary,the price level of orphan drugs in China was slightly lower than international reference price,but the price level of original drugs was higher than the international reference price,and the cost of some drugs far exceeded the residents’ability to pay.It is suggested that the government should strengthen the price management of original drugs,promote the substitution of high-quality generic drugs for original drugs,and improve the payment system for high-value rare disease drugs.