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 reviews 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 summarizes 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, explore the establishment of a preferential security system to alleviate the problem of poverty caused by illness. Fourth, establish and improve the medical security mechanism for rare diseases, which 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.