Establish a national diagnosis and treatment collaboration network and open a green channel for drug listing
China is taking action in response to rare diseases (towards national health in the past five years)
Rare diseases are a type of disease with extremely low incidence and prevalence. However, due to the large population base in China, rare diseases still have a large population in China, with an estimated number of more than 20 million.
Rare diseases are medical problems as well as social problems.
Since the 18th National Congress of the Communist Party of China, under the strong leadership of the Party Central Committee and the State Council, relevant departments such as the National Health Commission, the National Medical Products Administration, and the National Medical Security Administration have introduced the diagnosis and treatment of rare diseases, drug access and medical security. A series of policies and measures all reveal China's determination to promote the prevention and treatment of rare diseases and implement the national strategic goal of national health.
"In terms of the diagnosis and treatment of rare diseases, the work China has done is really amazing, and its contribution is also amazing." Du Han, chairman of the International Federation of Rare Diseases, said at the "2020 China Rare Disease Conference" held recently.
The conference was guided by the National Health Commission and the State Drug Administration, and hosted by the China Rare Disease Alliance.
Participants conducted in-depth discussions on China's effectiveness and experience in dealing with rare diseases in recent years.
Academician Zhao Yupei, president of Peking Union Medical College Hospital and chairman of the China Rare Disease Alliance, said that in recent years, under the guidance of the Healthy China Strategy, the prevention and treatment of rare diseases has received great attention from the party and the state, and various policies have promoted the development of the prevention and treatment of rare diseases. Intensive releases, China's rare disease related work has entered a new stage.
About 20 million people in China are plagued by rare diseases
According to Zhang Shuyang, chairman of the National Health Commission’s Rare Disease Diagnosis, Treatment and Protection Expert Committee and vice president of Peking Union Medical College Hospital, rare diseases refer to diseases that "rarely or rarely occur in the general population", usually chronic and degenerative. , Life-threatening, often seriously affect people's physical, sensory, mental or intellectual abilities.
Among the countries that have formulated rare disease policies, the United States defines “rare” as the affected population less than 200,000.
The threshold set by the EU is 5 for every 10,000 citizens.
Japan and South Korea stipulate that the prevalence of rare diseases is 0.4‰.
There are more than 7000 rare diseases in the world, and the number is constantly increasing. Approximately 250 new diseases are added to the list every year.
It is estimated that there are 263 million to 446 million people affected by rare diseases in the world, and there are about 20 million in China.
Zhang Shuyang said that rare diseases are an emerging social problem, which not only affects people's health and quality of life, but also makes individuals and families affected by them encounter many aspects such as medical care, education, employment, social integration and social security. There are many obstacles, and even the desperate situation of life.
The social inequality caused by disease has severely deprived people of the right to live and develop with dignity.
To deal with rare diseases, we are faced with the dilemma of lack of medical treatment and medicine.
"There are fewer doctors who can see rare diseases than rare disease patients." Said Shang Huifang, deputy director of the Department of Neurology, West China Hospital of Sichuan University. "Many rare diseases are not included in the courses of medical colleges and universities, and we need to accumulate experience at work. But if you enter a hospital that is not very big after graduation, and you haven’t seen this disease in a few years, how can you learn from experience in diagnosis and treatment?"
According to Zhang Shuyang, almost no rare diseases can be cured at present, and only 5%-10% of known rare diseases can be treated with drugs.
Illness is rare, love is always
Illness is rare, love is always there.
The care for rare patients from all walks of life is first reflected in the importance of the country, and secondly reflected in the establishment of various organizations.
In May 2018, the National Health Commission and the National Medical Products Administration jointly issued the "China's First List of Rare Diseases", including 121 rare diseases.
And proposed that the catalog will continue to be adjusted and expanded in the future.
After that, a series of policies and measures related to medical insurance for rare diseases were introduced.
In October 2018, the China Rare Disease Alliance was established.
The alliance is composed of more than 50 medical institutions, universities, scientific research institutions, and enterprises. Since its establishment, it has carried out fruitful work in gathering research talents, integrating disciplinary advantages, strengthening collaborative innovation, and connecting all walks of life to promote the development of rare diseases in China. .
Promote multidisciplinary diagnosis and treatment, carry out rare disease diagnosis and treatment training, carry out rare disease registration and promote rare disease direct reporting; carry out theoretical and policy research, and explore rare disease drugs and medical security programs with Chinese characteristics.
In February 2019, the National Health Commission announced the establishment of a national rare disease diagnosis and treatment collaboration network. The first batch of hospitals included 324 hospitals in all provinces across the country.
China has also established China's National Rare Disease Registration System, with a view to forming a unified technical specification and standard for rare disease registration statistics, so as to unite with superior units in rare disease research across the country to form a collaborative network.
"China has established a national rare disease network. I think this is a very good job." said Matt, consultant of the World Health Organization Rare Disease Global Collaboration Network. "These hospitals can unite to form the hub of the global rare disease network, forming a hospital and The ties of patient organizations can cooperate with each other to support rare disease patients. These hubs should eventually become beacons for rare disease research, education, and technological development on a global scale."
Under the vigorous promotion of China Rare Diseases Alliance and Peking Union Medical College Hospital, a series of clinically significant medical guidelines have been published, including the Interpretation of China's First Batch of Rare Disease Catalogues in 2018 and the Guidelines for Diagnosis and Treatment of Rare Diseases (2019). Year Edition)".
36 drugs are included in the National Medical Insurance List
Rare diseases are difficult to diagnose because many cases involve multiple disciplines.
“Sometimes this patient is not only affected by one system. If the doctor’s knowledge is very limited, it will cause delay in diagnosis. Therefore, multidisciplinary cooperation is very important in the diagnosis and treatment of rare diseases. This is why many hospitals have established many Disciplinary cooperation clinics and referral teams work together to serve rare patients. This is also an aspect of improving diagnosis and treatment capabilities." Shang Huifang said.
In terms of drugs, according to Zhang Shuyang, there is a clear green channel for the marketing of rare disease drugs.
In 2019, the newly revised "Pharmaceutical Administration Law of the People's Republic of China" clearly stipulates that the state encourages the research and creation of new drugs, and opens green channels for new drugs for rare diseases and children's drugs that are urgently needed in clinical practice, and prioritizes review and approval; New drugs for diseases without effective treatments can be subject to conditional approval under the condition that the existing data in clinical trials show curative effects and the clinical value can be predicted, so as to improve the availability of urgently needed drugs in clinical practice.
"This shortens the development time of clinical trials, so that patients who are in urgent need of treatment can use new drugs as soon as possible. For patients with rare diseases, it is undoubtedly an extremely favorable policy." Zhang Shuyang said that since 2018, there have been 38 An urgently needed drug for rare diseases was approved for marketing because it met the requirements of the State Drug Administration for rapid and simplified approval.
On the whole, 61 drugs that can treat diseases listed in China's "First Batch of Rare Diseases List" have been on the market in China, of which 36 drugs have been included in the National Medical Insurance List.
Chen Shifei, deputy director of the State Food and Drug Administration, pointed out that the State Food and Drug Administration has established a priority review and approval procedure for rare disease drugs, and established a fast track for the clinically urgently needed rare disease drugs on the market overseas, and the approval will be completed within 60 days.
In terms of meeting urgent clinical needs, two batches of clinically urgently needed overseas new drug evaluation lists have been selected and released, of which rare disease treatment drugs account for more than 50% of the total.
Wang Jiangping, Vice Minister of the Ministry of Industry and Information Technology, pointed out that the Ministry of Industry and Information Technology has issued rare disease drugs as key support targets in the implementation plan for the development of major innovations in protein biopharmaceuticals and vaccines and the development planning guide for the pharmaceutical industry.
The industrialization of rare disease drugs will be included in the scope of special support for industrial transformation and technological transformation, and rare disease drugs that are urgently needed clinically will be included in the key tasks of engineering research.
Xiong Jian