With the gradual improvement of the national medical security system, the treatment and security of rare diseases have attracted more and more attention. At the recently held China rare diseases conference in 2021, Li Tao, deputy director of the State Medical Security Bureau, introduced that up to now, more than 60 kinds of drugs for rare diseases have been approved for listing in China, of which more than 40 kinds have been included in the national medical insurance drug catalogue. In 2021, a total of 7 rare disease drugs were successfully negotiated, with an average price decrease of 65%. The current China rare diseases conference is guided by the National Health Commission and the Red Cross Society of China, and hosted by China rare diseases alliance, China Red Cross foundation, China pharmaceutical innovation promotion association and China Hospital Association. At the meeting, Chen Zhu, vice chairman of the Standing Committee of the National People's Congress and President of the Red Cross Society of China, said that since the 18th CPC National Congress, the special field of rare diseases has increasingly attracted the attention of the state and all sectors of society. On the basis of strengthening policy research and system construction, the national health, science and technology, drug administration, medical insurance and other relevant departments have worked together to explore a "Chinese model" to solve the problems of rare disease prevention and control by formulating a catalogue of rare diseases, establishing a national rare disease diagnosis and treatment cooperation network, strengthening regional cooperation and remote support, Jointly promote the cause of prevention and protection of rare diseases to a new level. Focus on rare diseases Embody the supremacy of life The research on the prevention and treatment of rare diseases is of great significance. Academician Zhong Nanshan, consultant of the National Committee of experts on the diagnosis, treatment and protection of rare diseases, pointed out that first, although rare diseases are rare, there are a large number of patients with any kind of rare diseases in China, which is a vulnerable group that is easy to be ignored. The concern for rare diseases reflects the national policy of "life first" of the party and the state. Second, in the analysis and synthesis of many diseases by clinicians, rare diseases are often found or diagnosed finally, which actually drives our understanding level of diagnosis and differential diagnosis of many diseases. To understand a rare disease, we must have more understanding of many so-called common or rare diseases. After elimination, we can get a correct conclusion through some special diagnosis, and our understanding of many diseases will be greatly improved. Third, China's in-depth research on rare diseases will provide new ideas for international prevention and control in this field. R & D platform Improve supply capacity During the 13th Five Year Plan period, the five departments of health, industry and information technology jointly issued the catalogue of the first batch of rare diseases, which contains 121 kinds of rare diseases. At present, 83 kinds of therapeutic drugs have been listed in China. Wang Jiangping, Vice Minister of the Ministry of industry and information technology, said that the Ministry of industry and information technology paid close attention to the drug demand of patients with rare diseases, guided enterprises to strengthen the research and development of specific drugs for the treatment of rare diseases through the implementation of the guidelines for the development of pharmaceutical industry and the 14th five year plan for the development of pharmaceutical industry, and included the industrialization project of rare disease drugs into the key tasks of the project, Promote the establishment of rare disease drug R & D platform, support and encourage enterprises to give full play to their main role in rare disease drug R & D. At the same time, rare disease drugs were included in the management of minor drugs and shortage drugs, and centralized production base construction and production supply monitoring were carried out to promote the continuous improvement of the supply guarantee capacity of rare disease drugs. Wang Jiangping said that the Ministry of industry and information technology will strive to create a good industrial policy and market environment and continue to promote the high-quality development of the pharmaceutical industry; Strive to mobilize the enthusiasm of scientific research institutions and production enterprises to develop drugs for rare diseases, and realize the R & D and production of drugs for the treatment of rare diseases as soon as possible; We will strive to strengthen the monitoring of the production and supply of drugs for rare diseases and the connection between supply and demand, promote the establishment of a shortage drug supply guarantee alliance, give full play to the role of the national pharmaceutical reserve reservoir, constantly make new contributions to meeting the people's health needs and aspirations for a better life, and fully support the prevention, control and guarantee of rare diseases as always. Approval in optimization Accelerate the speed of listing "This year, the State Food and Drug Administration newly approved 10 rare diseases drugs such as lisipram oral solution powder and brosuzumab injection for the treatment of spinal muscular atrophy (SMA), young Parkinson's disease, hyperammonemia and other rare diseases." Chen Shifei, deputy director of the State Food and drug administration, said, "these drugs fill the gap in the treatment of related rare diseases in China and bring hope for more patients with rare diseases to delay the development of the disease and improve the quality of life." In recent years, the State Food and drug administration has continuously optimized the review and approval procedures to speed up the listing of new drugs for rare diseases. In 2020, the newly revised Measures for the administration of drug registration made it clear that innovative drugs and improved new drugs for the prevention and treatment of rare diseases with obvious clinical value should be included in the priority review and approval procedure, and the drugs for rare diseases that have been listed abroad and are not listed in China that are urgently needed should be audited within 70 days. Among all drug marketing applications, the time limit for review and approval of drugs for rare diseases is the shortest. At the same time, new drugs for rare diseases that have been listed abroad will be included in the special channel for review and approval. At present, three batches of overseas new drug varieties in urgent clinical need have been selected and released, of which rare disease treatment drugs exceed 50% of the total selected drugs. The drugs for the treatment of rare diseases listed in the special channel shall be reviewed and concluded within 3 months. Chen Shifei said that the State Food and drug administration has actively built a communication platform and successively issued a series of technical guiding principles, such as the administrative measures for communication and exchange of drug R & D and technical review, and the technical guiding principles for accepting overseas clinical trial data of drugs, to guide the R & D of drugs for the treatment of rare diseases. At the same time, strengthen the linkage with other departments and cooperate with the Ministry of finance to implement tax preference for rare disease treatment drugs. The first batch of 21 and the second batch of 14 varieties of rare disease drugs shall be calculated and paid value-added tax according to the simple method and the collection rate of 3%, so as to encourage the development of rare disease pharmaceutical industry and reduce the drug cost of patients. Good medicine into medical insurance Prices have fallen sharply Since its establishment in 2018, the National Medical Security Bureau has dynamically adjusted the Medicare drug catalog once a year for four consecutive years, and a total of 507 new and good drugs have been included in the catalog. Through the catalog adjustment, the scope of drug security has been continuously expanded and the structure has been optimized. Li Tao pointed out that so far, more than 60 drugs for rare diseases have been approved for listing in China, of which more than 40 have been included in the national medical insurance catalogue, involving 25 diseases. At the same time, through the negotiation and access of drugs for rare diseases, the price of drugs for rare diseases was greatly reduced. A total of 7 drugs for rare diseases were successfully negotiated this year, with an average decrease of 65%. In particular, spinal muscular atrophy injection was included in the catalogue at a substantial price reduction, from the sky high price of 700000 yuan per year to the civilian price of more than 30000 yuan. In addition, it was included in the expense reimbursement of medical insurance, which basically solved the drug problem of spinal muscular atrophy patients, greatly reduced the economic burden of patients and saved the lives of many patients with rare diseases. "But at the same time, we should also see that the current positioning of basic medical insurance is still basic, and we must adhere to doing our best and acting according to our ability. In the next step, the National Medical Insurance Bureau will work with all parties to jointly take a series of measures to continuously promote the guarantee of medication for patients with rare diseases." Li Tao said that on the one hand, we will continue to promote the adjustment of the catalogue of Medicare drugs, and give priority to qualified rare disease drugs into the catalogue of Medicare drugs; On the other hand, actively promote and improve the establishment of multiple medical insurance system, give play to the role of basic medical insurance, serious illness insurance, charity assistance and other security functions, and further improve the medical security level of rare diseases. (Xinhua News Agency)