The hopes of more than 20 million patients in China are even brighter.
After 2019, this year’s government work report has once again proposed that "strengthening rare medical drug protection".
Seeing this content, Yang Linhua, deputy to the National People’s Congress and director of the Institute of Hematology of Shanxi Medical University, couldn’t help but praise: "The government work report involves the affairs of our country. Easy. "According to the definition of the World Health Organization, rare diseases refer to diseases that have a total population of -1 ‰ of the total population. According to the data released by the National Health and Health Commission, more than 60 rare diseases have been approved to be listed in China, of which more than 40 are included in the national medical insurance directory, involving 25 diseases.
After the national medical insurance negotiations in December last year, there were 7 kinds of rare medical drug negotiations successfully, and they were included in the latest version of the national medical insurance catalog, with an average price of 65%.
More and more rare patients are not only seen, but also can use "life -saving medicine" to see the hope of life.
"Any small group should not be abandoned." Since serving as a representative of the National People’s Congress, Yang Linhua has been providing suggestions for the treatment of rare diseases.
As a hematological expert, she had encountered some patients with hemophilia.
Data show that the incidence of social crowds of hemophilia is 5-10/100,000, and in May 2018, it was included in the "First Batch of Rare Diseases" in my country. The blood of patients with hemophilia lacks a kind of coagulation factor and requires repeated injection of coagulation factors to ensure normal coagulation function. Last year, the drug "human coagulation factor IX" used to treat type B hemophilia (coagulation factor IX deficiency) was included in the latest adjustment of the national medical insurance drug directory. There are 6 rare diseases that are included in the scope of medical insurance, including Nosna sodium injection for the treatment of spinal muscle atrophy (SMA). The annual treatment costs exceeded 1 million yuan, and the price was reduced to more than 30,000 yuan/needle after being included in medical insurance. At that time, a sentence that the national medical insurance negotiator said became a "golden sentence": "No small group should be abandoned.
Du Liqun, a member of the CPPCC National Committee and the nurses of the Fourth People’s Hospital of Nanning, Guangxi, talked about a detail in an interview with the media: Guangxi Maternal and Child Health Hospital is the only China SMA Diagnosis and Treatment Center Unit in Guangxi. There are only two patients who injected this drug through the hospital. After incorporating the "big price reduction" in the medical insurance, more than 20 patients have begun to use, and more patients are registering for medication.
Behind the medicine that treats rare diseases, there is a tortuous story of a family.
As a doctor, Yang Linhua has seen the pain of many rare patients with his own eyes, and he can understand their demands. Although the supply of drugs has improved greatly today, for patients, treatment costs are always a heavy burden.
As a doctor, what he is most looking forward to is to allow more patients with rare diseases to take medicine. "If we can take medicine for patients, he will contribute to the society like a normal person in the future.
"Most of the patients with rare diseases are facing difficulties in diagnosis and no medicine. In addition, because of the small scale of patients, the cost of rare diseases is difficult to cover. Huang Rufang, the founder of Rare Disease Center, is also a rare disease patient with pseudo -cartilage development. Since 2008, he has been trying to promote changes in the system and policy level of rare diseases.
In his opinion, since the 2019 government work report first mentioned rare diseases, the whole society has comprehensively increased the attention of rare disease groups. This year’s government work report once again mentioned "strengthening rare medical drug protection", which shows that the country pays more and more attention to this. "The country must have made great determination to promote the use of medication for patients with rare diseases.
"Multi -channel reduces the burden of rare disease medicine. Huang Rufang felt that last year was a" milestone "type of a rare medical treatment. Because the latest version of the national medical insurance catalog was updated at the end of last year, 7 rare diseases were added, including Nosina sodium injection Equal high -value drugs.
In addition, the State Drug Administration is also accelerating the review and approval of rare diseases. Hainan Boao also opened a "special channel" to allow some rare diseases to "test first" here. However, most of the rare diseases that have not yet been included in the scope of medical insurance are still most.
Therefore, many representative members also appealed at the two sessions this year to reduce the burden of medication for patients with rare diseases through various methods. "It is not realistic to rely on national medical insurance.
"Fang Laiying, member of the CPPCC National Committee and vice chairman of the Chinese Hospital Association, said that in general, the use of rare diseases is very low, and large -scale production and sales may not be formed in a certain area.
He suggested that social forces can be mobilized, considering the overall design of national medical insurance, commercial insurance, various charity funds, and public welfare organizations, and supplemented from different perspectives.
However, he also emphasized: "This requires a new policy design, hoping to have a department to study it.
"Mao Zongfu, deputy to the National People’s Congress and director of the Global Health Research Center of Wuhan University, analyzed that there are many types of rare diseases, fewer people, and less available medicines. Common and extraordinary basic disease treatment space for treatment.
Therefore, he suggested that in addition to national medical insurance, inclusive insurance and other innovative payment methods can also be used to solve the problem of "no medicine or medicine" for patients with rare diseases.
In recent years, in Zhejiang, Guangdong, Shandong and other provinces, Shanghai, Chengdu and other cities have been carrying out new attempts to include inclusive commercial health supplementary insurance (hereinafter referred to as "inclusive insurance") into the rare diseases. Mao Zongfu believes that while clarifying the "basic, subject, and pocket" government responsibility, through innovative social business cooperation, HP insurance has become a "mass medical health insurance" that facilitates millions of households. Ding Jie, a member of the National Committee of the Chinese People’s Political Consultative Conference and chief physician of Peking University First Hospital, suggested that the work system and mechanism of provincial -level rare medical assistance funds for rare medical assistance funds at the provincial -level rare diseases. The workflow is comfortable to avoid unclear responsibilities and the policy cannot be implemented; the social charity institutions and social mutual assistance organizations are relative to the basic medical insurance system of the government to ensure that the good funds and rescue funds enter the medical insurance payment mechanism in an orderly manner; Formulate the operation process to clarify specific operation methods and details such as "where the money comes from, where to go, and whether to enter the patient’s medical payment account". As a rare patient, Huang Rufang is looking forward to the addition of commercial insurance and charitable organizations, but he also admits that he is even more looking forward to the national medical insurance or local medical insurance departments "one step forward."
"After all, inclusive commercial insurance is not the main force of solving rare diseases.
"He believes that it should be allowed to try first, and try to test some of the rare diseases that are special and urgent.