Behind 700000 yuan of life-saving medicine: someone bought a cemetery for the child in advance

 Behind 700000 yuan of life-saving medicine: someone bought a cemetery for the child in advance

Lele was born in March 2017. Eight months later, her mother found that Leles crawling posture was a little strange. At the same time, accompanied by bursts of disturbing screams, if other babies can easily complete the movements, Lele has to work hard..

Shantou doctors suspected that Lele was cerebral palsy and muscular dystrophy. After a series of electromyography, magnetic resonance imaging and inadequate genetic testing, Lele was finally diagnosed with spinal muscular atrophy (SMA) type II.

At this time, Lele was 2 years old.

Spinal muscular atrophy, an autosomal recessive neuromuscular disease, is caused by mutations in the motor neuron survival gene. It is characterized by low muscle tone, progressive muscle weakness and loss of motor function. Earlier, the outside world mistakenly thought it was the same thing as ALS. In fact, the full name of progressive freezing disease is amyotrophic lateral sclerosis, which is characterized by the body gradually becoming dyskinesia, muscle weakness, as gradually frozen up, generally 30-60 years old onset; in contrast, most SMA patients in 6 months or 2 years old onset, the body gradually softened.

SMA is a strange and rare disease to the outside world. But in the eyes of professionals, the incidence is not low. The data show that the carrying rate of SMA in the global population is 1/40 - 1/60, the annual incidence rate is 5.1 - 16.6 cases of /100000 live babies. This means that if two gene carriers happen to get married, the probability of having a baby is one in four.

In my clinic alone, there are 60-70 SMA patients a year, which can be met almost every week. They come from all over the country, said Zhang Cheng, chief physician in the Department of Neurology of the First Affiliated Hospital of Sun Yat sen University According to the national population, there should be more than 30000 SMA patients in the mainland. According to the statistics of Meier SMA care center, there are about 1600 children registered in China. According to the data of Guangdong rare disease diagnosis and treatment cooperation network, 408 cases of SMA patients were registered in the province.

SMA is mainly divided into three types: the most dangerous type I, the patients life expectancy is less than 2 years old; if the diagnosis is late and the nursing is not in place, the children of type II will die of complications in early childhood; even the properly cared type III children will gradually lose their motor function day by day. Eventually, one day as an adult, you need to cut your trachea, plug in a ventilator, and spend the rest of your life in a wheelchair.

I bought a graveyard for my children

At the beginning of August, a news item 200 yuan per injection of imported medicine sold at home for 700000 yuan was on the hot search, and the SMA group swiped the screen again. The drug with a dose of 700000 yuan is called nosinogenase sodium. It was approved to be on the market in China in February last year. It is one of the special drugs for the treatment of SMA.

For SMA patients, the 700000 yuan is used to save lives.

It was the first spring festival that my family and I spent in our new house. Seeing that my son had really changed his fate, my parents were very proud. That night, my mother drank some wine, and at night she fell asleep. When she turned over, she accidentally lifted the quilt to my face. I tried to lift the quilt, but my arms were weak. Within a few minutes, I was suffering from a severe lack of oxygen and began to have a near death hallucination

At the critical moment of life and death, Luo Wangji has an idea. His voice calls for the smart Speaker: play Jay Chous double truncation sticks at the maximum volume.

His mother was awakened, and Luo Wangji finally escaped from death.

Luo Wangji is the lucky child among SMA patients, most of them are not so lucky.

SmaI patients are very difficult to break the so-called 2-year-old curse: a few patients with type I can live to be four or five years old or even teenagers, but most of them walk very suddenly and cant live for 2 years.

After diagnosis in February 2019, Lele began rehabilitation treatment immediately. However, the recovery speed can not catch up with the degradation speed. As can be seen by the naked eye, Leles physical function is degenerating. Some simple hand training movements, just diagnosed can still be done, after a few months, it must be on the chest pad support tools to do.

In a 279 person SMA communication group in Guangdong, five children have died since this year. Jingjing of Zhaoqing bought the ventilator online in February, but her condition deteriorated and left because of the epidemic situation. Weiting in Foshan and her favorite in Guangzhou had a sudden sputum blockage before her 4-year-old birthday. Jiajia in Changping was diagnosed on the 52nd day of her birth and left when she was 5 months old. At the end of August, Chaozhou patient more than one year old left the world simply because of pneumonia.

Children with SMA have the most expensive and not the colorful toys, but all kinds of treatment equipment.

SmaI children, but also to add more expensive ventilator. The process of changing these instruments is extremely dangerous, and many children stop breathing when they change the instruments.

In Guangdong SMA exchange group, we are used to the scene of farewell. As a parent, all you can do is stick to it.

Xiaoyi is a well-known child with SMA in Guangdong Province. On the one hand, she is over 8 years old, and Xiaoyis father has a lot of experience to share; on the other hand, Xiaoyis family is faced with the hell mode: her younger brother was diagnosed with autism shortly after birth, and her mother suffered from liver cirrhosis and liver failure. In order to take care of a family of three, my father quit his job and rely on the pension of Xiaoyis grandparents to maintain a basic living.

After this summer, Xiaoyi is in the third grade. She can write simple compositions, but the posture of holding the pen is more and more crooked. Ive bought her a place. Xiaoyis father said, I wont be reborn. Ill raise them well in my life.

The game of probability

When Xiaoyi was in the second grade of primary school, the teacher assigned a composition: my best friend. Xiaoyi wrote: my best friend is my brother. The younger brother with autism told her sister: when I grow up, I can take care of you.

Taking care of SMA children is full of adventure and unknown. Many families choose to have another one, which means another more adventurous gamble.

According to the probability, in the general population, there is one SMA carrier in every 40-50 people. If two carriers combine, there is a quarter chance of giving birth to SMA children each time.

This probability is not low, but as a rare disease, SMA recognition is still very low, routine premarital examination or pregnancy examination are not included.

After the screen of 700000 yuan high price medicine, many messages attacked the families of the children, believing that the illness was caused by the lack of prenatal screening, and that they needed to bear the responsibility themselves. In fact, conventional Downs screening can not screen the genes of rare diseases such as SMA, so it is necessary to pass the special screening of SMA gene.

Its just a game of probability.

Some families have lost the game completely. Most children with smaii develop at the age of 2 years. Before that, the weakness of children will be regarded as developmental retardation. The average time for diagnosis of SMA is months or even years. In a family in Chaozhou, the eldest daughter and the second daughter had symptoms of SMA, but the diagnosis was delayed. The doctor did not recommend prenatal screening for SMA. The family has a younger brother, who developed the same symptoms after 2 years old. Finally, all three children were diagnosed with SMA.

Some families rationally choose to do it again. Xiaoyis father did a complete set of SMA screening before he planned to have a second child, and confirmed that his brother was not a patient with SMA before giving birth to him.

Some families choose to divorce. As long as one of the husband and wife does not carry SMA gene, the probability of childrens disease is negligible. There is only one purpose in giving birth to a healthy baby: when you grow up, you can take care of the sick eldest - if you can live to that day.

The younger brother or sister became the only partner of SMA patients. Most children dare not approach SMA patients, let alone establish friendship between children, because they depend on wheelchairs all the year round and their posture is strange.

What bothers me most is not that the child has a short life span, but that she is trapped in a wheelchair and deprived of her most ordinary feelings. Apart from family affection, other feelings are luxury to her At the end of August, a mother of a patient with tears said, look at the video I took today. A three-year-old child watched a group of children walk past her hand in hand. What do you think she is thinking?

Money for life

How useful is the injection of sodium nosinogenase at 700000 yuan?

Zhang Cheng explained that the effect varies from person to person. Even if it works, patients who can no longer walk can not return to the state of being able to run and jump. The value of this drug lies in supporting the life of patients and improving their living conditions until better drug methods appear..

In the treatment of SMA, nosinogenase sodium is not the most expensive. In May 2019, zolgensma, a genetic drug developed by Novartis, a Swiss innovative drug company, was approved by the US Food and Drug Administration (FAD). Zolgensma, also used to treat SMA, is priced at $2.125 million, or about 14.48 million yuan - which many people describe as the most expensive candy..

At first glance, it seems that the cost of sodium nosign at a dose of 700000 yuan is cheaper than that of zolgensma, which costs 14.48 million yuan. But zolgensma is a one-time intravenous drug, while nosinogenase sodium needs to be injected every four months. Even in China, the annual cost of treatment is still more than 1 million yuan, and the treatment will last for life.

For most families with patients, this is an unbearable expense.

But in the face of the injection of sodium novolinate for 700000 yuan, they finally realized what is hard to get.

Both Leles mother and Xiaoyis father believe that the top priority is to work with the parents of other patients to promote the introduction of sodium nosinogenase into the health insurance and multi payment system.

But its not easy.

When zolgensma, which costs more than 14 million yuan, was launched in 2019, the response from the outside world was anger: do the poor have no right to live? But this year, when the sodium nosinogenase was given a dose of 700000 yuan, there were more voices of doubt: is it not depriving others of their right to life to spend so much money to save a patient with a rare disease?

I am against the inclusion of the drug in health insurance unless the health care sector can force pharmaceutical companies to reduce the price of drugs to ten thousand or twenty thousand yuan per yuan, said Ning Fang, a doctor in charge of burn department of Beijing Ji Shui Tan Hospital official account. The reason is simple: at the current level of Chinas economy, this medicine is too expensive.

Ning Fanggang calculated an account: even if the drug was reduced to lower than Australia through negotiation, the cost of each drug was 500000 yuan, and the cost in the first year was as high as 3 million yuan. After that, 1.5 million yuan a year. The cost of treatment in the first three years is as high as 6 million yuan. In China, 6 million yuan is enough to save the lives of hundreds of critically ill patients and ensure the normal treatment of thousands of patients with chronic diseases.

But most doctors disagree.

Hu called on 80% of the patients with liver cancer who had been diagnosed with rare diseases in Guangdong Province. Why cant 80% of the patients who have been diagnosed with liver cancer in Guangdong Province to take medicine for treatment are rare, because they cant get medical treatment because they dont have genetic diseases No matter how sophisticated an instrument or software is designed, there will also be defects; there will be mistakes in human evolution, and rare patients will bear the pain of evolutionary errors for us.

But the reality is bone feeling. Most people cant afford a life that can be bought with money.

Since the launch of nosilan sodium in February last year, only one injection has been carried out in the First Affiliated Hospital of Sun Yat sen University where Zhang Cheng works.

Zhang Cheng shared this case: in October 2019, a 1-year-old and 5-month-old boy came to see a doctor in October 2019 because he found that his motor development was backward in September. He could sit alone but could not crawl and turn over. After a complete evaluation process, Zhang Chengs team directly injected sodium nosignazon through lumbar puncture, including 4 loading doses and maintenance doses once every four months.

During the recent follow-up, the child was able to stand and walk in the water. One photo shows a child looking out on the railing. He wants to see the outside world, too. Zhang Cheng said.

Call for 1 + n multi payment

As a leader in the field of rare diseases in Guangdong and even in China, Zhang Cheng stressed on many occasions that the medicine that patients can use is the best medicine.

In view of rare diseases, many experts have proposed that it is not a perfect policy to enter the medical insurance list, and a 1 + n multi payment mechanism should be established: 1 refers to the gradual incorporation of the first batch of rare disease listed drugs into the medical insurance system; n includes: the establishment of national and provincial special relief projects for rare diseases; the overall arrangement of charitable funds into the payment mechanism for rare diseases; and the guidance of commercial insurance into the rare disease doctors Treatment payment mechanism; patients pay for themselves.

According to the data, in 2018, the general public budget revenue of Guangdong Province was 1.21 trillion yuan, ranking first in China for the 28th consecutive year, with the growth rate exceeding the national average. By the end of 2018, the accumulated balance of basic medical insurance fund in Guangdong Province was 283.26 billion yuan, accounting for 12% of the national basic medical insurance fund balance.

Wu Qingping, academician of the Chinese Academy of engineering and director of the Institute of Microbiology of Guangdong Province, wrote in his proposal on the medical security policy for rare diseases in Guangdong Province. The number of patients with rare diseases is small, and the overall expenditure is limited and controllable.

Zhang Cheng always believes that patients with rare diseases are like relatives with incurable diseases at home, will you give up your relatives because they have to spend a lot of money to treat them? cant. We should not give up any rare disease. Drugs have been on the market for such a long time. At present, there are only a single digit number of patients using drugs in Guangdong Province, and there is only one case in our large hospital. Obviously, we havent done well enough. .

Hope is ahead.

Every winter, Zhang Chengdu will send a message to Jiayi, who is studying in the National Peoples Congress: Beijing is cooling down. Remember to add clothes. At the same time, she was urged to practice blowing balloons to delay the deterioration of respiratory function.

Jiayi is a smaii patient in a wheelchair. She was admitted to Sun Yat sen University and Renmin University three years ago. She wants to go to Beijing to read the National Peoples Congress, but the whole family is against it. Zhang made a careful physical assessment on Jiayi and told Jiayi to support her going north after weighing a series of factors, such as the journey, weather and school support!

Everyone knows what the rush of the road and the cold in the North mean for SMA patients. But Zhang said he didnt want to leave any regrets for Jiayi.

Now, nearly every year, I can see a perfect report card.

There are always two sides to a coin. Patients with SMA are limited in body, but sometimes their knowledge and thoughts go faster and farther. Who can ignore the stubbornness of life?

Risdiplam has become a promising drug. At the end of April this year, risdipram of Roche company submitted an application for listing license in the State Drug Administration. This is a daily oral drug, the effect is equivalent to intrathecal injection of nosinogenase sodium, or it can promote the price reduction of nosinogenase sodium.

(function(){( window.slotbydup=window .slotbydup||[]).push({id:u5811557,container:ssp_ 5811557, async:true }Everyone is racing against time. The anxious parents in the group discussed whether to raise 700000 yuan first, and gave the first four injections. If you live one more year, there may be a turning point.. Some people also discussed whether or not to send a representative to talk with Indian pharmaceutical companies to develop a generic drug for children to use first. Leles mother, who has just turned 30 this year, firmly believes that before she grows old, both the national security system and drug treatment will usher in a turning point. But day by day, the strength was lost. At the age of three, he learned to make fun of himself: Im not a force, Im lazy. One day Lele couldnt move, so I bought him an eye tracker like Hawkings As for whether to cut the trachea and live longer, he decides for himself, said Leles mother Source: time weekly editor: Yuan Yijiao_ NB14956

Everyone is racing against time.

The anxious parents in the group discussed whether to raise 700000 yuan first, and gave the first four injections. If you live one more year, there may be a turning point.. Some people also discussed whether or not to send a representative to talk with Indian pharmaceutical companies to develop a generic drug for children to use first.

Leles mother, who has just turned 30 this year, firmly believes that before she grows old, both the national security system and drug treatment will usher in a turning point.

But day by day, the strength was lost. At the age of three, he learned to make fun of himself: Im not a force, Im lazy.

One day Lele couldnt move, so I bought him an eye tracker like Hawkings As for whether to cut the trachea and live longer, he decides for himself, said Leles mother