Careful care for more than 20 million patients with rare diseases -- China accelerates the exploration of the "Chinese model" for the diagnosis, treatment and protection of rare diseases

2022-11-04

This is good news for patients with rare diseases - up to now, 45 kinds of drugs for rare diseases have been included in the national medical insurance drug directory, covering 26 kinds of rare diseases. China has a large population base. Although the proportion of patients with various rare diseases is small, the absolute number has reached more than 20 million. In recent years, patients with rare diseases have received more and more attention from all walks of life. On the road of building a healthy China, none can be left without. We have formulated a rare disease directory, established a national rare disease diagnosis and treatment collaboration network, and established the China Rare Disease Alliance. More than 60 rare disease drugs have been approved for marketing, and rare disease drugs have been continuously included in the national medical insurance drug directory... China has accelerated the exploration of the "China model" of rare disease diagnosis and treatment and protection. At the 2022 China Rare Diseases Conference being held in Beijing, heads of relevant national departments, medical experts and others conducted in-depth discussions on this topic. Multiple measures were taken to promote the improvement of rare disease prevention and treatment capacity. In the 2021 adjustment of the national medical insurance drug catalog, the once "priceless" life-saving drug, Nostradamus sodium injection, was included in the medical insurance with more than 30000 yuan after the medical insurance negotiation. Xiao Hou, a child with spinal muscular atrophy, became an unfortunate "lucky child". "It is still a challenge to promote the construction of a healthy China to accelerate the construction of rare disease prevention and treatment capacity, so that patients can receive timely and effective diagnosis and treatment." Li Linkang, executive director of the China Rare Diseases Alliance, introduced that, taking mucopolysaccharide storage disease and Gaucher disease as examples, the annual treatment costs often exceed one million yuan, which is a real problem that many families cannot afford. In recent years, relevant departments have strongly supported the prevention and protection of rare diseases, strengthened the research, diagnosis and treatment of rare diseases, and drug research and development and supply, and improved the medical security system for rare diseases. Huang Guo, deputy director of the State Food and Drug Administration, said that the State Food and Drug Administration has continued to deepen the reform of the drug evaluation and approval system, supported the research and innovation of rare disease drugs, and has approved more than 60 rare disease drugs for marketing, with the number and speed increasing rapidly. The National Natural Science Foundation of China has set up a special project for rare diseases, the Ministry of Science and Technology has approved the construction of a national key laboratory for difficult and severe diseases and rare diseases, the publication of a rare disease research journal, and the launch of the China Red Cross Foundation's rare disease co aid fund... China is making every effort to promote the research and prevention of rare diseases, and lighting up new hope for patients with rare diseases and their families. Yu Xuejun, deputy director of the National Health Commission, said that the National Health Commission has strengthened the linkage of medical treatment, medical insurance and medicine, constantly established and improved relevant policies and measures, improved the ability to diagnose and treat rare diseases, and ensured drug supply. Multidisciplinary consultation improves the ability of precise diagnosis and treatment of rare diseases. At noon every Thursday, the National Rare Diseases Consultation Center of Peking Union Medical College Hospital brings together top experts from different disciplines. Also attending and consulting online were doctors from 323 hospitals of the rare disease diagnosis and treatment collaboration network nationwide. They jointly provide a diagnosis and treatment program for a rare disease patient and family. As the national leading hospital of the national rare disease diagnosis and treatment collaboration network, Peking Union Medical College Hospital has established the China rare disease diagnosis and treatment service information system, led the preparation of the rare disease diagnosis and treatment guide, and established a "one-stop" diagnosis and treatment model for rare diseases in multiple disciplines. President of Peking Union Medical College Hospital

Edit:He Chuanning    Responsible editor:Su Suiyue

Source:Xinhua

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