Difficulties behind the soaring price of 700,000 yuan for a "life-saving medicine"
Lei Lei is an SMA-I patient, one and a half years old this year
Recently, after seeing a piece of "seeking medicine" news, a mother from Guangdong, Ouyang Chunlan, submitted an information disclosure application to the National Medical Products Administration, hoping to learn about drugs for treating spinal muscular atrophy (hereinafter referred to as "SMA")—— The procurement method and domestic pricing basis of the 700,000 yuan-a-injection of Nosinagen Sodium Injection have attracted the attention and heated discussion of many netizens for a while.
The application showed that Ouyang Chunlan requested to make public the procurement contract for the introduction of Nosinassen Sodium Injection, the domestic sales price pricing basis and relevant instructions for pricing calculations, as well as information on the inventory quantity and distribution of Nosinassen Sodium Injection.
Spinal muscular atrophy is a rare hereditary neuromuscular disease, also known as the "killer of genetic diseases in infants and young children." According to the age of onset and the acquisition of exercise mileage, SMA is divided into SMA-I, II, III and IV. Without treatment, most children with SMA-I cannot survive to two years of age. At present, the only medicine used to treat this disease in China is Noxinat Sodium Injection.
Patient
"I can't afford it, and I can't afford to sell a house"
A doctor from Wuhan Children’s Hospital in related fields told reporters that SMA is also known as "the number one genetic disease that causes infant and child death". It is an autosomal recessive genetic disease. Usually, the parents of children are carriers. The child’s motor neurons located in the anterior horn of the spinal cord and the lower brain stem are lost and degenerated, resulting in severe muscle atrophy and weakness. The patient can hardly achieve even ordinary turning, kicking, and crawling, and may eventually lose the ability to walk and breathe , Swallowing disorders.
Leilei
Got the disease "not to live for two years"
I haven't used medicine after a year and a half
Leilei’s father joined the SMA Hubei patient group after Leilei became ill. Among the 34 families in the group, the most urgent need for treatment is Lei Lei. She is a patient with SMA-I. She is one and a half years old this year. She is only half a year away from what the doctor said "lives less than two years."
Lei Lei’s father said that Lei Lei not only has no strength in her legs, but now she has basically lost her swallowing function. She has difficulty breathing, has been ill for a year, has had a cold five times, and has to go to the intensive care unit every time she gets sick. It costs seven or eight thousand to live in the intensive care unit for one day. Leilei’s father said that he took the child’s medical expenses to the village committee to find a way, and finally took the subsistence allowance, saving a large amount of medical expenses, but seeing The child is about to be two years old, and he has not used Noxinat Sodium Injection. He has sought funds from charities such as the Qianjiang Red Cross Society, and currently raises less than 10,000 yuan.
Yingying
It costs 700,000 every eight months
To maintain the efficacy
In May of this year, Yingying was going to have the fifth injection, but the injection has disappeared. "I can't afford it, and I can't afford to sell a house... Do you guys understand that the kind of child who has finally recovered, and now you have to watch her not move day by day? If I delay my child's treatment, I will Regret for a lifetime." Yingying's mother said.
Yingying's parents work in a car factory, and their annual family income is less than 100,000. The house has been linked to an intermediary and plans to sell it at any time.
They asked if the village committee could provide some subsidies to the family. The village's response should be reported to the county. The county government stated that there is no relevant policy to help the disease. Then they contacted the Suizhou Charity Federation, but Yingying’s application for information was not passed because the disease was not covered by the organization's assistance. In the end, the charity organization subsidized more than 10,000 yuan, but for SMA families, It's just a drop in the bucket.
Since the efficacy of Noxinagen Sodium Injection will be slowly consumed in the body, patients need to use it for life. However, the injection of Noxinagen Sodium is 700,000 yuan per injection, which is currently not included in the National Medical Insurance Drug List and requires the patient to pay for it in full.
Xing Huanping, the relevant person in charge of the Beijing Meier Spinal Muscular Atrophy Care Center, the first domestic public welfare organization focusing on the rare disease SMA, told reporters that from the data held by the organization, there are currently more than 1,600 SMA patients across the country. It's just the tip of the iceberg, and the cost of SMA treatment is expensive. There is no substitute for imported precision targeted drugs. They need to be used for life, which would cost tens of millions."
In February 2019, the National Medical Products Administration officially approved "Noxinassen Sodium Injection" for the treatment of 5qSMA. On April 28 of the same year, Noxinassen Sodium Injection was officially launched in China. Intrathecal injection of Noxinassen Sodium Injection can increase the level of Survival Motor Neuron (SMN) in patients with SMA, thereby improving motor function, increasing survival rate, and changing the disease process of SMA.
In November 2019, Yingying, then 3-year-old, received the intrathecal injection of Noxinarine Sodium Injection at the Department of Neurology of Wuhan Children's Hospital, becoming the first child with SMA in Hubei Province to receive the drug.
"The child has changed a lot during the past six months." Yingying's mother said, the child has gained a few kilograms in weight, and his resistance has also increased. He has never caught a cold. "Now I can sit up, I can lift my legs up, and sometimes kick the quilt." Speaking of the child's changes, Yingying's mother's tone rose unconsciously.
Yingying's mother borrowed 600,000 yuan and almost sold the house, only to make up to 700,000 yuan, which was only the "threshold" for this medicine. After paying for the first injection, the China Primary Health Care Foundation presented the last three injections, and these four injections were given within two months. Starting from the fifth injection, it has become a free injection for one injection, and one injection every 4 months. In other words, it takes 700,000 at least every eight months to maintain the efficacy.
The above-mentioned doctor from Wuhan Children’s Hospital stated that he would come into contact with more than a dozen children with SMA every year. “99% of families with children cannot use this medicine. However, it has been more than a year since the introduction of this medicine and the relevant medical insurance cooperation departments are still discussing. Children with Type I and Type II drugs will have better effects, and the earlier it is used, the better for children."
Mr. Huang
I hope I have money to get an injection
Don't let parents get tired
According to the age of onset and clinical course, SMA is divided into 4 types from severe to light. The life span of patients with type III and IV will not be affected, but unfortunately, their lives are equally difficult. Unable to stand, lack of self-care ability, school and employment are successively affected, as if it is an endless loop that every SMA patient cannot escape.
Mr. Huang, who lives in the central city of Xiaogan, Hubei, is a type III SMA patient, 31 years old this year. At 1.8 meters tall, he weighed less than 100 jin, and only 70 jin when he was the thinnest. Mr. Huang said that his illness did not happen suddenly, but he graduated from elementary school. After that, climbing the four flights of stairs in the junior high school classroom became his "nightmare."
At first, it took him 8 minutes to climb the fourth floor with the handrails, a little slower than his peers, and then it took half an hour. This is not the most difficult. Since the break between classes is only ten minutes, he cannot go from the classroom to the bathroom and then back to the classroom within ten minutes. Because he is too late and has urinated his pants, he basically does not drink water during school. "If you don't drink water, you won't go to the toilet, and everyone won't realize that I'm strange."
Until, he couldn't climb one floor. Mr. Huang said, the feeling is like you stand for a long time, and when you reach the limit, you have exhausted your life's energy even raising your feet. Later, he went to school one hour earlier every day, and his parents carried him on the fourth floor. He arrived earlier than the classmate who opened the door. However, despite his persistence, he did not change his fate of being forced to drop out of school because his body was unable to bear the burden.
After dropping out of school, he stayed at home every day, but his parents were working. He couldn't go out every day. After sitting for three or four hours during the day, he was tired, and he lay down at other times. During the period, he would also sleep, which caused him to fall asleep until three or four in the morning. Occasionally, his parents would carry him down from the fourth floor and push him to stroll around the park. At the same time, he is also paying attention to the rescue policy related to SMA disease. When the reporter asked him to say something he most wanted to do, he said, "I dare not think about it or say it."
On December 26, 2019, an unprecedented earthquake occurred in Xiaogan, Hubei, with a magnitude of 4.9. At that time, Mr. Huang, who was sitting in a chair, looked at the shaking chandelier and noticed that something was wrong, but his father, who was able to move his back, was not at home at this time. He yelled at his mother to leave him alone and go out quickly.
Mother was scared and ran out. Mr. Huang stared at the continuously shaking roof. He felt that if he died in this earthquake, it would be a relief. But after a while, his mother returned home. In the next scene, the mother in her 50s supported her. With a thin body, he carried Mr. Huang, who was nearly 100 kilograms, down the fourth floor.
Mr. Huang said that the wish he was afraid to say was that he hoped that if he had money, he could get a shot and walk on the ground by himself, so that his parents would not be tired anymore.
Difficult negotiation
The high price of the drug has been included in the medical insurance negotiation schedule
On August 5, a staff member of the Letters and Visits Office of the National Medical Insurance Bureau said in an interview with reporters that the price of Noxinagen Sodium Injection is set by the pharmaceutical companies themselves, so the price of the drug in each country has certain discrepancies, "except Pharmaceutical companies will also consider profit issues for the raw materials and R&D costs of the drug. In addition, the drug is currently monopolized in the domestic market, and the price has remained high."
Only three treatments for SMA in the world
Only one stitch, price 15 million
It is reported that Noxinassen Sodium Injection was developed by Biogen Idec Ltd. It was first approved in the United States on December 23, 2016. It is the world’s first SMA precision targeted therapy drug. The drug has subsequently been launched in the European Union and Brazil. , Japan, South Korea, Canada and other regions and countries have been approved for the treatment of SMA. On April 28, 2019, Noxinassen Sodium Injection was launched in China for the treatment of 5qSMA and became the first drug in China to treat SMA. The other two drugs for the treatment of SMA, Roche's Risdiplam and Novartis's Zolgensma, are not on the domestic market.
The reporter learned that Nocinasheng Sodium Injection is worth 125,000 U.S. dollars (approximately RMB 870,000) in the United States. Compared with Noxinassen Sodium Injection, a drug that requires repeated injections every year, Zolgensma under Novartis can complete the treatment at one time, but the price of Zolgensma is also regarded as a "sky price", up to 2.1 million US dollars (about RMB 15 million yuan), and the drug is only listed in the European Union, the United States, Japan, etc.
Drug prices cannot come
Can't be included in the medical insurance catalog
The staff of the Letters and Visits Office of the National Medical Insurance Administration stated that since the launch of Nosinagen Sodium Injection in China in 2019, it has been included in the medical insurance negotiation schedule. The state hopes to negotiate with relevant pharmaceutical companies to lower the price of the drug to meet the needs of SMA patients . "The country started to negotiate with pharmaceutical companies last year, and the expert group will study the pricing. The specific pricing is not clear. However, the inclusion of medical insurance has not been discussed. Because the price of the drug cannot be lowered, it has never been included in the medical insurance catalog." The above-mentioned staff member said .
Why can't pharmaceutical companies be covered by medical insurance unless their prices drop? The staff member further explained to reporters that the medical insurance catalogue formulated by the National Medical Insurance Bureau is applicable to all parts of the country, so it is necessary to ensure that the medicines that enter the medical insurance catalogue can be used everywhere. "Once such rare disease drugs are included in the medical insurance list, for underdeveloped regions, after the fund is used to pay for high-priced rare disease drugs, other underlying diseases may not be covered, and the follow-up may also cause local economic pressure, so the most fundamental solution The way is for the state to negotiate with pharmaceutical companies to negotiate prices."
Difficulties for pharmaceutical companies
Rare disease drugs are expensive
High failure rate due to high R&D costs
On August 6, BioGen, the research and development and manufacturer of Noxinagen Sodium Injection, responded to reporters in response to the recently discussed online “low” price of A$41 in Australia.
Not eligible to participate in the 2019 national medical insurance negotiations
Bojian responded that according to relevant national regulations, drugs participating in the 2019 national medical insurance negotiations must be drugs approved before December 31, 2018. Noxinassen Sodium Injection was approved in February 2019. It did not meet the conditions for participating in the 2019 national medical insurance negotiations, so it did not participate in the 2019 national medical insurance negotiations.
Earlier, the relevant person in charge of the drug management department of the Hubei Provincial Medical Security Bureau said in an interview with reporters that in November 2019, the state adjusted a medical insurance drug list. The western medicine and Chinese patent medicines imported into the medical insurance drug list were on December 31, 2018. (Including) Drugs previously registered and marketed by the State Drug Administration. The Noxinasana injection was approved for listing in China in 2019, and it did not meet the relevant requirements.
The above-mentioned person in charge stated that the drugs imported from abroad, especially those for rare diseases, need a certain time to test whether they are really effective, and whether they can be included in the medical insurance catalogue is also determined by the National Medical Insurance Bureau.
AUD 41 is not
The price of the drug in Australia
According to a report from China Business News, in Australia, Nosinagen Sodium Injection was included in the local Pharmaceutical Benefits Program (PBS) in June 2018 for the treatment of patients under 18 years of age with SMA-1, 2 and 3a. And according to PBS regulations, patients need to pay a certain amount for the subsidized drugs in the plan.
Until January 1, 2020, patients with Australian health insurance cards will receive greater benefits. They only need to pay 41 Australian dollars (approximately RMB 205) for most PBS drugs. If they hold a discount card, they only need Pay 6.60 Australian dollars (about 33 yuan).
"We have to distinguish two basic concepts correctly: one is the price of medicines, and the other is the out-of-pocket expenses of patients after reimbursement." A related person from Bojian told reporters that taking the SMA drug Noxinagen Sodium Injection as an example, the drug was first introduced in the United States Approved, the single price in the US market is US$125,000. The annual treatment cost for the first year is about 750,000 US dollars. After that, the annual treatment cost is about 375,000 US dollars.
"Up to now, Nosinassen Sodium Injection has been approved in 50 countries and regions around the world, and has been fully or partially reimbursed in more than 40 countries and regions." Bojian told reporters that Nosinassen Sodium Injection has been Included in the Australian Pharmaceutical Benefits Program, the single government purchase price of the drug is 110,000 Australian dollars, and the patient’s out-of-pocket expenses after reimbursement are 41 Australian dollars, which is not the Australian price of the drug.
Bojian also introduced that in February 2019, Noxinassen Sodium Injection was approved in China through the priority review and approval process, becoming the first and only approved SMA treatment drug in China. The single price of the drug in the Chinese market is RMB 699,700, which is 20% lower than the US market.
Bojian said that the high cost of research and development of rare disease treatment drugs and the high rate of R&D failure are high-risk areas for global new drug development. Even if new drugs for rare diseases come out fortunately, because the applicable patient population is small, the commercial return of pharmaceutical companies is lower than that of non-rare disease drugs compared to the huge R&D investment and failure cost. Therefore, rare disease drug companies need to fully consider many factors when pricing, such as the R&D cost in the field of rare diseases, the value created by the drug for patients, the ability of the company to continue R&D and innovation, and its current and future contributions to society.
There are more than 80 SMA patients
Get treatment with the help of aid projects
According to Bojian, Nosinagen Sodium Injection is currently a self-funded drug in China. Bojian deeply understands the importance and urgency of dealing with payment challenges for the Chinese SMA patient group. "The company has always maintained active communication with relevant national and local government departments and called for the establishment of a multi-party co-payment mechanism to further improve the availability of medicines for SMA patients in China." Bojian said, May 31, 2019, China Primary Health Care Fund It was announced that the SMA patient assistance project was officially launched, and the assistance drugs were donated by Bojian Company.
This project helps SMA patients save about 2/3 of the cost of treatment in the first year compared to the full self-finance, and then save about half of the annual treatment cost compared with the full self-finance. Up to now, more than 80 SMA patients across the country have received drug treatment with the help of assistance projects.
According to the official website of the China Primary Health Care Foundation, on May 31, 2019, the spinal muscular atrophy (SMA) patient assistance project jointly prepared by the China Primary Health Care Foundation and Bojian Company was officially launched. It will be launched in 14 provinces and cities across the country. Launched, covering 25 central hospitals in the first phase.
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