China News Agency, Beijing, August 8th (Reporter Li Yanan) Recently, a message about "seeking medicine" circulated on the Internet, because it contains "the treatment of rare diseases" spinal muscular atrophy" specific drugs require 700,000 yuan (RMB, below) The same) A "sky price" and "price disparity in different countries" and other contents have aroused social attention to the plight of rare diseases.
Spinal muscular atrophy (SMA) is characterized by the degeneration and loss of motor neurons in the spinal cord and lower brain stem. It is a rare disease of hereditary neuromuscular diseases. According to Lu Junlan, chief physician of the Department of Neurology at the National Children's Medical Center and Beijing Children's Hospital affiliated to Capital Medical University, the incidence of SMA in newborns in China is about 1/6000 to 1/10000, and there are currently more than 30,000 SMA patients.
The World Health Organization defines a rare disease as a disease in which the number of patients accounts for 0.65‰ to 1‰ of the total population. Currently, there are nearly 7,000 rare diseases identified globally. Experts estimate that China currently has tens of millions of people suffering from the disease. In 2018, SMA was included in the "First List of Rare Diseases" jointly issued by China's National Health Commission and other departments. According to incomplete statistics, of the 121 diseases published in the "First List of Rare Diseases", only more than 40 diseases have related drugs on the global market.
The reporter's investigation found that the SMA drug Nosinagen Sodium Injection, which is on the cusp of the storm, is also a "high-priced drug" in the world. The "price disparity in different countries" that has aroused concern is because some countries or regions have given local residents different degrees Medical insurance reimbursement, but generally strict requirements, limited places, and some countries include mass purchase or medical insurance to achieve price reduction process is difficult and long.
According to media reports, the staff of the Letters and Visits Office of the National Medical Insurance Administration of China recently revealed that since the launch of Nosinagen Sodium Injection in China in 2019, it has been included in the medical insurance negotiation agenda. The state hopes to negotiate with relevant pharmaceutical companies to lower the price of the drug. So as to meet the needs of patients.
In addition, in 2019, the China Primary Health Care Foundation launched the "Spine Live Newborn-Spinal Muscular Atrophy SMA Patient Assistance Program" to provide assistance drugs to patients who meet the requirements of the project. The project is carried out in 14 provinces and cities across the country, and the first phase covers 25 central hospitals. Up to now, more than 80 SMA patients across the country have received drug treatment with the help of assistance projects.
In recent years, in order to solve the problems of difficult and expensive drugs for rare diseases, the relevant departments have taken many measures in the innovative research and development, review and approval, and medical insurance of rare disease drugs. The "Pharmaceutical Administration Law of the People's Republic of China", which was implemented at the end of 2019, clearly stipulates the strengthening of the research and development, production and supply of rare disease drugs. The Announcement on Issues Concerning Clinical Urgently Needed Overseas New Drug Review and Approval issued by multiple departments jointly reviewed and approved the establishment of special channels for rare disease treatment drugs and other clinically urgently needed overseas new drugs, and approved rare disease treatment drugs listed in the special channels. Ended within months.
“Of the 55 rare disease treatment drugs that have been approved for marketing in China, 32 have been included in the National Medical Insurance Drug List and are applicable to 19 rare diseases. Among them, 5 are newly added in the 2019 catalogue adjustment, and are still Some drugs for the treatment of rare diseases have entered the negotiation stage, and those successfully negotiated will be included in the catalog according to procedures." Li Tao, deputy director of the National Medical Security Administration of China, said earlier.
Although many parties are currently actively exploring the rare disease medical insurance model with Chinese characteristics, how to fundamentally reduce the pressure on the medical expenses of patients with rare diseases is still a global problem. Experts pointed out that the deep-seated reason that rare diseases are frequently paid in medical insurance is due to the low incidence of rare diseases, the very few medicines used, and the high prices. The types of rare diseases that can be covered by current medical insurance types are limited, and China’s current commercial medical insurance is still waiting Perfect, the medical needs of patients with rare diseases cannot be fully satisfied.
The relevant person in charge of the health department of Hunan Province stated that some rare disease medication issues were successfully resolved because of China’s large population base, and rare disease patients with certain diseases are not “rare”. The medical insurance department can use volume for price and other price negotiations. Means, to promote enterprises to reduce prices, and to form a common interest with the current security system. But in the long run, there are still many new drugs for rare diseases that have a small clinical total, and it is difficult to negotiate prices. Therefore, to truly solve the problems of difficult and expensive medications for rare diseases, it is necessary to actively develop commercial insurance and comprehensively utilize social resources such as charity and assistance to reduce the burden on patients. Relevant departments should also speed up the research and development and consistency evaluation of generic drugs to fundamentally improve related drugs. Accessibility. (Finish)