Abstract
BackgroundChina is actively promoting regulation of rare diseases, rare disease and orphan drugs have been formally incorporated into the national planning. However, few studies have been done to evaluate the affordability of rare disease patients in China. This study aims to provide policy recommendations for the establishment of social security mechanism for rare diseases in China, so as to address the problem of poverty caused by these diseases.MethodsA total of 7 rare diseases were selected by Delphi method. Affordability of treatment for the 7 rare diseases was assessed through annual per capital income, catastrophic expenditure and impoverishment expenditure among urban and rural residents in China.ResultsAssessed through annual per capital income, health expenditure for the 7 rare diseases are all rather high. The highest health expenditure is equivalent to income of 69.34 years of one urban resident, and the burden is heavier for rural residents. Through catastrophic expenditure assessment, proportions of the population experiencing catastrophic expenditure caused by the 7 rare diseases are all under 0.167 ‰. However, once one is ill and taking medications, he will suffer from catastrophic health expenditure. Through impoverishment expenditure assessment, the proportions of impoverishment payment are low among both urban and rural residents, but the 7 rare diseases could lead nearly 4.6 million people into poverty on a national scale.ConclusionThe affordability of treatment for rare disease as well as orphan drugs is rather poor. Residents of different income levels all have difficulties to afford the treatment for rare diseases, so poverty caused by rare diseases is quite widespread. Therefore, social security mechanism for rare disease patients should be established and specific payment pattern for orphan drugs should be set up.Electronic supplementary materialThe online version of this article (doi:10.1186/s13023-016-0454-7) contains supplementary material, which is available to authorized users.
Highlights
China is actively promoting regulation of rare diseases, rare disease and orphan drugs have been formally incorporated into the national planning
In February of the same year, Chinese Premier Li Keqiang proposed to promote the development and update of pharmaceutical industry at State Council executive meeting, emphasizing improving the industrial development of orphan drugs. It symbolised that rare disease and orphan drugs have been formally incorporated into the national planning
The average annual health expenditure of Gaucher Disease (GD) is equivalent to 69.34 times of average annual income of urban residents and 32 times of high-income groups of urban residents whose average annual disposable income are 61,615 yuan
Summary
China is actively promoting regulation of rare diseases, rare disease and orphan drugs have been formally incorporated into the national planning. Since 1980s, the legislation on rare diseases and polices towards orphan drugs have been gradually implemented in developed countries and regions [3]. In February of the same year, Chinese Premier Li Keqiang proposed to promote the development and update of pharmaceutical industry at State Council executive meeting, emphasizing improving the industrial development of orphan drugs. It symbolised that rare disease and orphan drugs have been formally incorporated into the national planning
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