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2022年2月 Journal of Nanjing Medical University（Social Sciences）of Nanjing Medical University（Social Sciences） · 5 ·
Journal
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Medical security mechanism for rare diseases:experience and

implication from some countries and regions

ZHU Kun
Chinese Academy of Fiscal Sciences，Beijing 100142，China
Abstract：Establishing and improving the medical security mechanism for rare diseases will not only help
to accelerate the process of universal health coverage and Healthy China Construction，but also help to promote
the transformation and upgrading of China’s biomedical industry. However，at present，the construction of rare
disease security mechanism in China lags behind. This paper reviewed the practices of some countries and
regions from the aspects of the definition of rare diseases，drug supply and cost sharing of rare diseases，as
well as the promotion of the development of the pharmaceutical industry，and summarized their experience，
implication and suggestions as follows. First，defining the scope of rare diseases is the premise and basis for
establishing the medical security mechanism of rare diseases. China needs to define rare diseases from a multi⁃
dimensional perspective to better promote the construction of the medical security mechanism of rare diseases.
Second，improving drug accessibility is an important part of establishing a rare disease medical security
mechanism. China needs to further strengthen its support for domestic enterprises，timely include the products
related to rare diseases of domestic enterprises into the medical insurance catalogue，and better improve the
drug accessibility of patients with rare diseases. Third，the medical security system combining inclusive and
preferential protection is the main means to alleviate the medical burden of patients with rare diseases. China
should give priority to establishing an inclusive system so that all patients with rare diseases can obtain the
benefit from the basic medical insurance system. On this basis，it is suggested to explore the establishment of a
preferential security system to alleviate the problem of poverty caused by illness. Fourth，establishing and
improving the medical security mechanism for rare diseases is an important booster to realize the transformation
and upgrading of China’s biomedical industry. China should strengthen the connection between medical security
policy and pharmaceutical industry policy，and further improve the security mechanism of rare diseases in
China，and promote the transformation，upgrading and high⁃quality development of the pharmaceutical industry
to better meet the drug needs of patients with rare diseases in China.
Key words：rare diseases；medical security；orphan drug；drug accessibility