Xinhua News Agency, Beijing, August 6 - Title: "a medicine 700,000 yuan," "cheap foreign domestic expensive"? ——Focus on "sky-priced" rare disease drugs

  Xinhua News Agency reporters Shuai Cai and Qu Ting

  A few days ago, the Internet circulated that "the treatment of rare disease, spinal muscular atrophy, requires a high price of 700,000 yuan, and the domestic price is much higher than abroad", which has aroused social concern.

  What is "spinal muscular atrophy"? What kind of medicine is Noxinasheng Sodium Injection? Why are medicines so expensive? Is the price of the drug really different in the domestic and foreign markets? Is the price of medication for domestic patients expected to fall? Xinhua News Agency reporters launched an investigation on this.

Focus 1: What is spinal muscular atrophy?

  According to Lu Junlan, chief physician of the Department of Neurology at the National Children’s Medical Center and Beijing Children’s Hospital, spinal muscular atrophy (SMA) is a fatal disease for children. Usually 80% of type I patients cannot live beyond 2 years; type II patients walk Difficult and often accompanied by severe complications of pneumonia; although type III does not affect life span, the patient will be weakened, causing a huge burden of disease and mental suffering to him and his family.

  Wu Liwen, deputy chief physician of the Department of Neurology, Hunan Children's Hospital, said that this disease is characterized by the degeneration and loss of motor neurons in the spinal cord and lower brainstem and is a rare disease of hereditary neuromuscular diseases.

  Experts said that 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, but the pathogenic gene carrying rate in newborns is about 1/50, which is very dangerous. High, so it is very important to carry out large-scale prenatal screening.

Focus 2: What kind of medicine is Noxinasheng Sodium Injection?

  According to experts, Noxinnasheng Sodium Injection is an antisense nucleotide therapeutic drug for infants, children and adults with 5q spinal muscular atrophy, and it is also the first imported drug approved for the treatment of spinal muscular atrophy in China. The doctor administers medicine to the patient through intrathecal injection (injecting the medicine into the body through a lumbar puncture).

  Wu Liwen told reporters that the child should be given medication as soon as possible after diagnosis: 6 injections in the first year, 4 injections in the first 2 months, and 1 injection every 4 months thereafter.

  Wu Liwen said that some children have recovered their athletic ability to a certain extent after the injection. A 2-year-old and 11-month-old child in Hunan started to inject the drug in May this year and has already injected 4 times. After treatment, Nono's athletic ability has been better restored.

  "From a therapeutic point of view, Nocinasheng Sodium Injection is indeed useful, but it cannot completely reverse the condition." Lu Junlan reminded.

Focus 3: Why are medicines so expensive?

  The reporter learned that Nosinagen Sodium Injection was approved for import in 2019. It is currently not covered by medical insurance and is borne by patients at their own expense. In China, the price per unit of the drug is 699.7 thousand yuan.

  According to the newly revised Drug Administration Law in 2019, drug prices are no longer set by the government, but are mainly adjusted by the market, and they play a role through centralized bidding and procurement of medical insurance. Song Hualin, deputy dean of the School of Law of Nankai University and an expert in drug law, said that the number of rare disease patients is small and there are clinical uncertainties. Therefore, drug development costs are high and drugs are expensive. Individual pharmaceutical companies often obtain pricing advantages due to technological leadership, which does not necessarily violate my country's anti-monopoly law or drug regulatory laws and regulations.

  He believes that the deep-seated reason why rare diseases have repeatedly caused concern in medical insurance is that the types of rare diseases that can be covered by current medical insurance are limited, and the current commercial medical insurance in my country still needs to be improved, and the medical needs of rare disease patients cannot be fully satisfied.

Focus 4: Does the drug have a huge price gap in the domestic and foreign markets?

  Internet rumors that the drug "prices only US$41 in Australia, or about RMB 280", "nearly free in Japan", and said that "our hospitals are so profitable to such an appalling level" and so on.

  The reporter's investigation found that according to the Australian Pharmaceutical Benefits Program (PBS) website, ordinary patients pay $41 per unit for medication, but the government purchase price of the drug is 110,000 Australian dollars per unit.

  According to the reporter's understanding, Japan recently included related drugs in the scope of medical insurance, and patients only need to bear relatively low expenses in accordance with the country's overall medical insurance policy. But the drug itself is still a high-priced drug.

  The reporter also found that there are only a few countries in the world that include the drug in public health insurance. In the United States, the drug sells for $125,000 each. In Canada, a local resident told reporters that after the inaugural company purchased commercial insurance for him, he had to pay more than US$5,700 per unit for the drug, which was not cheap. It is reported that most European countries have not included it in the scope of public medical insurance.

Focus 5: Is the price of drugs for domestic patients expected to fall?

  Experts said that due to the low incidence of rare diseases, the very small amount of medicines, and the high prices, it is not easy to bring them into mass purchases to achieve price reductions. How to reduce the pressure on medical expenses of such patients is still a problem.

  The reporter learned that in 2019, the China Primary Health Care Foundation launched the "Spine Live Newborn-Spinal Muscular Atrophy SMA Patient Assistance Project" to provide assistance drugs to patients who meet the project application conditions. Family members of the affected children can apply to participate in the assistance program, and in the first year they can inject 6 medicines with the original price of 700,000 yuan each at a price of 1.4 million yuan.

  Experts such as Lu Junlan and Wu Liwen expressed the hope that relevant departments can study methods to effectively reduce the medical burden of patients with the disease through comprehensive measures such as medical insurance. To improve the treatment and survival of patients with rare diseases requires the joint efforts of the state, social organizations, medical institutions and patients.

  Song Hualin believes that for rare diseases with extremely low incidence and high diagnosis and treatment costs, multi-channel assistance should be adopted, such as considering medical assistance and special assistance, and establishing special funds. He suggested that provinces can determine the rare diseases that the province includes in the scope of medical insurance based on their own economic and social development level, medical insurance fund balance, and diagnosis and treatment of rare diseases.

  Ms. Yang, who lives and works in Japan, told reporters that some local residents and experts expressed concern and dissatisfaction with including the extremely expensive drug in medical insurance, believing that this would threaten the balance and safety of guarantee funds, and squeeze out other diseases and demand. Opportunities for more urgent diseases to be included in the security system.

  Many experts believe that the production of high-priced rare disease drugs has its own particularities. At present, whether it is through centralized procurement negotiations or included in medical insurance, there are practical difficulties in pushing their prices down.

  The relevant person in charge of the health department of Hunan Province said that some rare disease medication problems were successfully resolved because of the large population of our country, and the rare disease patients of some diseases are not "rare". The medical insurance department can use the quantity to exchange 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.