More drugs for rare diseases are expected to be included in medical insurance to solve the payment problem of patients

[industry trends of pharmaceutical network] in late December 2019, the state health insurance bureau intensively released responses to the proposal, including 4 for rare diseases In addition to the two responses on December 3, that is to say, in December 2019, the National Health Insurance Bureau issued six responses to the rare disease proposal In response to the proposal to establish a "national special fund for rare diseases" and other suggestions, the wording "inappropriate" was used However, the health insurance bureau also proposed that the next step would be to continue to promote the inclusion of drugs for rare diseases in health insurance, and actively study the innovative security model for rare diseases Rare diseases are incidence rate is very low, the total number of patients is very low, the quality of life of patients is far lower than that of the general population, and may be life-threatening diseases At present, there are about 20 million patients with rare diseases in China In order to solve the pain point problem of difficult diagnosis and no medicine available for the patients with rare diseases, more and more attention has been paid to the patients with rare diseases and the prevention and treatment of rare diseases in China in recent years For example, in May 2018, China released a list of rare diseases, including 121 kinds of rare diseases such as hemophilia, albinism, Gaucher's disease and Fabre's disease, affecting about 3 million patients, which represents the substantial progress of the national level in the prevention and control of rare diseases Among the 121 rare diseases, 83 drugs are available for 53 diseases in the mainland, 29 of which are included in the medical insurance, involving 18 rare diseases In addition, 21 kinds of rare diseases are faced with the situation that there are drugs abroad and there are no drugs in China In November 2018, the two departments selected and published a batch of "list of new drug varieties urgently needed to be listed abroad in clinical practice", with a total of 48 varieties, including 20 drugs for rare diseases In February 2019, the state established a diagnosis and treatment network for rare diseases, and 324 hospitals across the country joined in the diagnosis and treatment network for rare diseases Patients with rare diseases can find a hospital suitable for diagnosis and treatment according to their condition In addition, in order to solve the problem of the diagnosis of rare diseases, the national health and Health Commission issued the guide for diagnosis and treatment of rare diseases (2019 version) According to the drug administration law, which was formally implemented on December 1, 2019, the State encourages the development and production of drugs in short supply, and gives priority to the review and approval of new drugs urgently needed in clinical practice for the prevention and treatment of major infectious diseases, rare diseases and other diseases This can encourage and support the rare disease drug R & D enterprises It can be seen that with the promotion of these measures, although it is still far away from truly solving the problem of rare diseases in China, there may be some improvements in the situation that rare diseases in China cannot be diagnosed and there is no medicine to treat In addition to difficult diagnosis, expensive treatment is a big problem It is understood that some rare diseases require one to two million yuan a year for treatment, and patients are facing a serious burden of treatment According to the 2019 comprehensive social survey of Chinese rare disease patients launched by China rare disease alliance and Beijing Union Medical College Hospital, less than two-thirds of the 12601 effective responses are currently being treated More than 40% of the respondents who stopped treatment said that they had stopped treatment because of "too high medical expenses to afford" In order to reduce the burden of drug use for patients with rare diseases, the 2019 version of the national health insurance regular directory includes 32 kinds of 55 kinds of drugs for rare diseases, which are applicable to 19 kinds of rare diseases, including 5 kinds of new access directories in 2019, which are applicable to primary carnitine deficiency, early-onset Parkinson's disease, etc In November 2019, seven more rare drugs were added to the health care negotiation catalogue In addition, 33 varieties of the second batch of national organization drugs, which will be opened on January 17, 2020, have been selected for centralized collection, covering the treatment fields of diabetes, hypertension, anti-tumor and rare diseases In the reply, the medical insurance bureau proposed that in the next step, the National Medical Insurance Bureau will gradually include the rare disease drugs with exact curative effect and the medical insurance fund can undertake into the scope of medical insurance payment through strict expert review in combination with the drug demand of the insured, medical insurance financing ability and other factors And actively work with relevant departments to carry out research on the feasibility of the innovative guarantee mode of rare diseases, do as much as you can, and gradually improve the guarantee level of patients with rare diseases The industry believes that as more rare disease drugs are included in the medical insurance, the burden of drug costs will be reduced for patients with rare diseases who need lifelong medication, which will further enhance the willingness of patients with rare diseases to receive treatment