Wang Yaqun, Zhou Na, Li Baoxin, Lv Zixuan, Duan Shengnan, Li Xin, Yuan Ni
School of Public Health, Dalian Medical University, Dalian, Liaoning, China.
Department of Health Policy and Management, Peking University, Beijing, China.
Int J Equity Health. 2024 Jul 31;23(1):151. doi: 10.1186/s12939-024-02225-0.
The accessibility issue of orphan drugs in China is prominent. Based on real-world data from a tier-one city in Northeast China, this study aims to analyze the current usage and affordability of orphan drugs for rare diseases.
The data was sourced from the health insurance claims data of a certain city from 2018 to 2021, including a total of 16 orphan drugs. The utilization of orphan drugs is assessed using four indicators: frequency of medical insurance claims, medication cost, defined daily doses (DDDs), and defined daily drug cost (DDDc). Affordability is measured using the concept of catastrophic health expenditure (CHE).
Between January 2018 and December 2021, there were a total of 2,851 medical insurance claims in the city, with a total medication costs of $3.08 million. Overall, during the study, there was a year-on-year increase in the utilization frequency of individual rare disease drugs in the city, with DDDs rising from 140.22 in 2018 to 3983.63 in 2021. Additionally, the annual medication costs of individual drugs showed a consistent upward trend, increasing from $10,953.53 in 2018 to $120,491.36 in 2021. However, the DDDc of individual drugs decreased from $398.12 in 2018 to $96.65 in 2021.The number of sales and the amount of sales for orphan drugs in community pharmacies have significantly increased. Prior to medical insurance coverage, out of the 16 orphan drugs, 9 drugs had annual treatment costs exceeding CHE for urban residents, and 15 drugs had annual treatment costs exceeding CHE for rural residents. After medical insurance coverage, there were no drugs with out-of-pocket costs exceeding CHE for urban residents, while 8 drugs had out-of-pocket costs exceeding CHE for rural residents. Furthermore, both before and after medical insurance coverage, the four treatment drugs for idiopathic pulmonary arterial hypertension were more affordable compared to the four treatment drugs for multiple sclerosis.
The usage frequency of orphan drugs in a certain city increased gradually, but the disease burden remained heavy. More policy support should be provided to the priority rare disease populations, and the rare disease medical security and diagnosis and treatment systems should be improved.
中国孤儿药的可及性问题突出。基于中国东北某一线城市的真实世界数据，本研究旨在分析罕见病孤儿药的当前使用情况和可负担性。
数据来源于某城市2018年至2021年的医疗保险理赔数据，共包括16种孤儿药。使用四个指标评估孤儿药的使用情况：医疗保险理赔频率、用药费用、限定日剂量（DDD）和限定日费用（DDDc）。使用灾难性卫生支出（CHE）的概念衡量可负担性。
2018年1月至2021年12月，该市共有2851笔医疗保险理赔，用药总费用为308万美元。总体而言，在研究期间，该市个别罕见病药物的使用频率逐年增加，DDD从2018年的140.22增加到2021年的3983.63。此外，个别药物的年度用药费用呈持续上升趋势，从2018年的10953.53美元增加到2021年的120491.36美元。然而，个别药物的DDDc从2018年的398.12美元降至2021年的96.65美元。社区药房中孤儿药的销售数量和销售额显著增加。在医疗保险覆盖之前，16种孤儿药中，有9种药物的年度治疗费用超过城市居民的CHE，15种药物的年度治疗费用超过农村居民的CHE。医疗保险覆盖后，没有城市居民自付费用超过CHE的药物，而有8种药物的农村居民自付费用超过CHE。此外，在医疗保险覆盖前后，特发性肺动脉高压的四种治疗药物比多发性硬化症的四种治疗药物更具可负担性。
某城市孤儿药的使用频率逐渐增加，但疾病负担仍然较重。应给予优先罕见病群体更多政策支持，完善罕见病医疗保障和诊疗体系。
