□ Our reporter Zhang Wei
In a healthy China, no one must be left behind.
Up to now, more than 60 kinds of drugs for rare diseases have been approved for marketing in China, and more than 40 kinds of drugs have been included in the national medical insurance drug list, involving 25 kinds of diseases.
Through the negotiation of rare disease drugs, the price of rare disease drugs has been greatly reduced. In 2021, a total of 7 rare disease drugs have been successfully negotiated, with an average reduction of 65%.
At the 2021 China Rare Disease Conference held recently, Li Tao, deputy director of the National Medical Security Administration, revealed the above information.
This is undoubtedly a warm message.
In the medical insurance negotiation in early November last year, 7 rare disease drugs were finally negotiated into the medical insurance, especially the medullary muscular atrophy drug Nosinagen sodium, which was originally priced at 700,000 yuan per injection, and was shortlisted at a very low price.
This makes "rare diseases" a hot spot of social concern for a while.
Every recent move on "rare diseases" has particularly touched people's hearts.
On New Year's Day, nearly 20 patients in 11 provinces and cities including Guangdong, Beijing, and Shanghai took the life-saving drugs newly included in the medical insurance drug list, which also aroused strong social concern.
"Compared with major common diseases, the number of rare diseases is relatively small, but the absolute number is not large." Chen Kaixian, academician of the Chinese Academy of Sciences, pointed out that it is very urgent to promote the research and development of rare disease drugs, and it is necessary to strengthen the basic research and clinical translation research of rare disease drugs. Advocate pharmaceutical companies to actively invest in the research and development of rare disease drugs.
The problem of rare diseases requires the joint efforts of many parties.
Beyond pharmaceutical companies, the government is playing an active role.
It is understood that in recent years, my country has implemented priority review and approval for rare disease treatment drugs.
The newly revised "Drug Registration Management Measures" in 2020 clarifies that innovative drugs and improved new drugs for the prevention and treatment of rare diseases with obvious clinical value will be included in the priority review and approval process. Closed within 70 days.
It is understood that at present, 26 rare disease drugs have been approved for marketing through the special channel for overseas new drugs that are urgently needed in clinical practice.
The power of the people cannot be ignored.
Recently, the reporter learned from Boao Lecheng Weijian Clinical Medical Center for Rare Diseases that under the efficient relay between Ali Health and the center, a rare disease drug named TRIENTINE HYDROCHLORIDE has been successfully introduced into China. The drug will provide more treatment options for patients with hepatolenticular degeneration in China.
Hepatolenticular degeneration is a rare autosomal recessive disease of copper dysfunction, and patients suffering from this disease are commonly known as "copper dolls".
The disease leads to obstacles in the transport and excretion of copper ions in the body, which can easily cause liver function and brain damage after the onset, and patients need lifelong drug maintenance.
The request for the use of trientine came from the mother of Xiaojiang (pseudonym), a patient in Wuhu, Anhui.
Xiao Jiang was intolerant of penicillamine, the traditional medicine for the disease, and could not take it normally.
Xiaojiang's mother learned that the drug trientine for penicillamine intolerance had been approved for marketing in the United States in 1982, but there was no such drug or generic drugs on the market in China.
With the attitude of giving it a try, Xiao Jiang's mother logged in to the "Global Drug Information Platform for Rare Diseases" and submitted the drug search information.
The platform was launched by Ali Health Public Welfare and Boao Lecheng Rare Disease Clinical Medical Center in September last year. It aims to provide support for the availability of drugs for domestic rare disease groups by building integrated online and offline services.
After receiving the demand, the platform immediately mobilized global business resources, launched a drug search campaign, and successfully introduced the drug exclusively in December.
At present, the drug has been accessible in Hainan Boao Lecheng International Medical Tourism Pilot Zone.
Although the disease is rare, caring is not "rare", and even one patient is worth the effort.
I hope that all rare disease patients can have medicines and use good medicines.