Zhang said to Xinhua, "what are social organizations and hospitals doing for patients with rare diseases?"

On February 13, the National Health Commission held a press conference with the theme of targeted poverty alleviation, and introduced the work related to rare diseases In particular, Zhang Shuyang, vice president of Beijing Union Medical College Hospital, vice chairman of National Health Commission Expert Committee on diagnosis and treatment of rare diseases, and vice president and Secretary General of China Union of rare diseases, was invited to attend the conference and answered questions from reporters Jiao Yahui, deputy director of the medical administration bureau of the national health and Health Commission, introduced to reporters the relevant work of the national health and Health Commission in the management of rare diseases As a clinical expert who has paid close attention to patients with rare diseases for many years, president Zhang expressed his feelings and introduced the current situation of patients with rare diseases in China Zhang said that rare diseases in various countries have gone through the process from focusing on major diseases, frequently occurring diseases and common diseases to focusing on rare diseases The incidence rate of rare diseases is low, but there are many diseases, and there are nearly 7000 rare diseases worldwide For rare diseases, the government has issued a series of policies How to implement this policy and what social organizations, hospitals and doctors are doing are introduced to reporters Zhang Shuyang said: on October 24, 2018, under the leadership of Zhao Yupei, President of Beijing Union Medical College Hospital, China Association of hospitals, China Association for the promotion of medicine and other medical institutions, academic groups, pharmaceutical enterprises, patient organizations, etc., China rare diseases alliance was established, which has great social significance and influence Under the guidance and support of the government, the Alliance for rare diseases will promote the implementation of policies on rare diseases, promote the improvement of the ability of standardized diagnosis and treatment of rare diseases, stimulate and promote the innovation vitality of research institutions and pharmaceutical enterprises, and develop effective drugs, so as to fundamentally reduce the cost of drugs At present, the biggest problem of patients with rare diseases is lack of medicine Less than 5% of the rare diseases in the world have effective drug treatment, usually the drug is expensive, and the patients with rare diseases in China can not afford it On February 11, the executive meeting of the State Council proposed to give preferential policies for the import tariff of 21 kinds of drugs with rare diseases and 4 APIs, promote the simultaneous listing of overseas new drugs in China, and guarantee the medication for more than 20 million patients with rare diseases This is good news for patients and doctors Doctors will have more drugs for patients with rare diseases and improve their prognosis In the future, China rare diseases alliance will actively carry out training, promote the improvement of doctors' ability nationwide, and standardize diagnosis and treatment, especially early diagnosis Around the world, including developed countries, in the past, it usually took 5 or 10 years for patients with rare diseases to have a clear diagnosis Beijing Union Medical College Hospital is the national guidance center for the diagnosis and treatment of difficult and severe rare diseases, and also the national leading Hospital of the national cooperation network for the diagnosis and treatment of rare diseases As a hospital, according to the policy requirements of the National Health Committee, the national rare disease diagnosis and treatment cooperation network should be well done, and through two-way referral, remote consultation, expert tour and other mechanisms to further facilitate patients' medical treatment, early diagnosis and early treatment From prevention to rehabilitation, improve the quality of life of patients It is an indisputable fact that patients with rare diseases are rare, and doctors with the ability to diagnose and treat rare diseases are even rarer For such a situation, Beijing Union Medical College Hospital and China rare diseases alliance actively carried out training work, from disease knowledge to diagnosis technology to treatment methods, and carried out standardized diagnosis and treatment training to play a leading role In addition, it is of great significance to report and register rare disease cases and understand the epidemic situation of rare disease in China It not only provides advice and suggestions for the government to formulate policies, but also has an important impact on improving the medical and health security system and accelerating the research and development of new drugs We call on all hospitals to complete the registration of patients with rare diseases according to the requirements of the health and Safety Commission, and hope that media friends can help to promote Beijing Union Medical College Hospital also took the lead in the national 13th five year precision medicine key research and development project "rare disease cohort research", established the national database of rare diseases in China, and carried out disease diagnosis and pathogenesis research through the second-generation sequencing method Finally, president Zhang affectionately called out for the patients with rare diseases, "the patients with rare diseases also need your help", everyone in the society will give a little help and care, more patients with rare diseases and families will feel more warmth, and their lives will be better Jiao Yahui, deputy director of the conference, also mentioned rare diseases for many times On February 11, the executive meeting of the State Council presided over by Premier Li Keqiang decided to give value-added tax preference to 21 drugs with rare diseases and 4 APIs from March 1, which is a major measure taken by the party and the state to guarantee the basic drug demand of patients with rare diseases, as well as a concrete embodiment of caring for patients with rare diseases and safeguarding basic rights and interests Jiao also introduced the current situation of rare diseases to reporters The management of rare diseases is not only a medical problem, but also a social problem For patients with rare diseases, we need to give more care than medical help The problem of rare diseases is a common problem in the world Many rare diseases lack effective diagnosis and treatment Due to the small number of users, the comprehensive medical security department, and the pharmaceutical companies with research and development capabilities, they have not paid enough attention to the research and development of orphan drugs, or even do not consider copying orphan drugs Therefore, the price of drugs for rare diseases is expensive, which brings great economic burden to patients At the same time, many patients have no drugs available, which makes patients struggle in despair Therefore, in recent years, the party and the government have attached great importance to the groups with rare diseases and issued a series of preferential policies The national health and Health Commission, we have carried out a number of work In the next step, the National Health Committee will follow the principles of prevention first, classified policy implementation and steady progress On the one hand, it is necessary to further reduce the incidence of birth defects of newborns to take some measures to reduce the incidence of rare diseases At the same time, it is necessary to further increase diagnosis and treatment and play the role of network At the same time, we hope to promote the improvement of the rare disease registration system as soon as possible, play the role of big data, and promote the research and development of rare disease drugs At the same time, we hope to gather all forces to care for patients with rare diseases Related news: related news link: Health Care Commission: five aspects of work on rare disease prevention and management https://www.cn-healthcare.com/article/20190213/content-514586.html