School of Healthcare Security, Shandong First Medical University & Shandong Academy of Medical Sciences, Jinan 250117, China.
Shandong Institute of Medicine and Health Information, Jinan 250117, China.
Int J Environ Res Public Health. 2022 Oct 15;19(20):13319. doi: 10.3390/ijerph192013319.
The affordability of rare disease drugs has become a social issue that cannot be ignored. This study aims to evaluate the current price and affordability of rare disease drugs in China, with evidence from Shandong province.
Data on prices and affordability of 50 drugs for 22 rare diseases were collected from secondary and tertiary public hospitals in Shandong Province, using an adaptation of the World Health Organization/Health Action International (WHO/HAI) methodology. Prices were measured as Median Price Ratios (MPRs). Affordability was measured as days of daily per capita disposable income required for the cost of one month's treatment.
Out of the 50 rare disease drugs, 11 drugs had MSH reference prices and 34 had PBS reference prices. Median prices of 11 drugs were higher than MSH reference prices (median 1.33), and median prices of 34 drugs were higher that Australian PBS prices (median 1.97). Thirty-six (72.00%) and forty-four (88.00%) drugs were unaffordable for urban and rural residents, respectively. Thirty-four (68.00%) and thirty-eight (76.00%) drugs were unaffordable for urban and rural residents even after reimbursement by the health insurance schemes of China, respectively.
The affordability of some rare disease drugs remained poor with their relatively high prices in Shandong Province. Sustainable mechanisms are needed to reduce the price of rare disease drugs and to improve the affordability of rare disease patients.
罕见病药物的可负担性已成为一个不容忽视的社会问题。本研究旨在评估中国罕见病药物的当前价格和可负担性，并以山东省为例。
从山东省二级和三级公立医院收集了 22 种罕见病 50 种药物的价格和可负担性数据，采用世界卫生组织/国际健康行动（WHO/HAI）方法进行了改编。价格以中位数价格比率（MPR）衡量。可负担性以一个月治疗费用所需的人均日可支配收入天数来衡量。
在 50 种罕见病药物中，有 11 种药物有 MSH 参考价格，34 种药物有 PBS 参考价格。11 种药物的中位数价格高于 MSH 参考价格（中位数为 1.33），34 种药物的中位数价格高于澳大利亚 PBS 价格（中位数为 1.97）。36 种（72.00%）和 44 种（88.00%）药物对城镇居民和农村居民来说都是不可负担的。即使在中国的医疗保险计划报销后，34 种（68.00%）和 38 种（76.00%）药物对城镇居民和农村居民来说仍然是不可负担的。
山东省一些罕见病药物的价格仍然较高，可负担性较差。需要建立可持续的机制来降低罕见病药物的价格，提高罕见病患者的可负担性。
