On January 1, 2022, in the children's ICU ward of Zaozhuang Maternal and Child Health Hospital in Shandong Province, medical staff cheered for a 4-year-old child with spinal muscular atrophy (SMA) who completed the targeted treatment drug Nocinasin sodium injection. (Xinhua News Agency reporter Guo Xulei/photo)
Sugar Heart is 3 years old. She was finally able to pinch the little toy with her little finger and make a little "mm-hmm" sound.
Before being injected with "700,000 shots of sky-high drug" - Nocina raw sodium injection, she could not move, did not laugh, breathed on a ventilator, ate by gastrostomy, and the home was full of medical instruments, like a small ICU.
Everything is due to sugar heart spinal muscular atrophy (SMA). This is a rare autosomal recessive neurodegenerative disease, there are 20,000 to 30,000 patients in China, muscle weakness is the most typical symptom.
According to the degree of deterioration of exercise ability, it is divided into four types, and in the absence of medical intervention, some seriously ill patients cannot survive the 2nd birthday, so SMA is also regarded as the "number one genetic disease killer" for infants and young children under 2 years of age.
Sugar heart is the most serious and common type I SMA, but Hu Yue refused to give up her daughter sugar heart, and through the help of donating medicine, she was injected with 7 doses of Northinacine sodium injection.
At that time, Nosinasin sodium injection had been approved for marketing in the Chinese market, and the treatment cost was 550,000 yuan / year. Even if the price of nearly 700,000 yuan / needle has been greatly reduced from the beginning of the listing, it is not a figure that an ordinary family can afford.
Fortunately, in the 2021 national medical insurance negotiations, after eight rounds of "soul bargaining" by the medical insurance department, the price of this drug was reduced to about 33,000 yuan / needle, successfully entered the national medical insurance directory, and it is estimated that patients will pay about 10,000 yuan / needle.
From January 1, 2022, with the landing of the new version of the national medical insurance directory, the good news of the first injection has been reported from all over the world. On January 4, southern weekend reporters learned from Biogen, a research and development enterprise of Nosinasson sodium, that the company has completed the hanging network work of 34 procurement platforms across the country, and only on the first day of medical insurance landing, nearly 30 patients in 11 provinces such as Guangdong and Fujian were treated.
"I wish I could hit it on January 1st"
Watching the negotiators of the national medical insurance directory on TV finally bargain successfully, Liu Li was excited, "Every small group should not be abandoned."
Liu Li, director of the Department of Genetics and Endocrinology of Guangzhou Women and Children's Medical Center, is also the leader of the expert group of the Guangdong Rare Disease Diagnosis and Treatment Collaboration Network, and many SMA patients in and around Guangdong Province have been diagnosed and treated in her unit, and more than 200 patients have been registered and diagnosed over the years.
At less than 9:00 a.m. on January 1, 2022, Liu Li's hospital injected the first injection of Northinasson sodium after the medical insurance landed into the body of a sixteen- or seventeen-year-old teenager.
Fortunately, this teenager is a type III SMA patient, the condition is not so serious, when he was a child, he was able to walk independently, but his steps were unstable and easy to fall, and he had deteriorated to the point of not being able to walk in junior high school, and it was difficult to sit, and he needed to sit next to each other or lean on.
"He is a very sunny and smart big kid, very much looking forward to being able to get treatment, to be able to walk, to be able to go to school like a normal person, but before the price of medicine was expensive, his family could not afford it." Liu Li remembers that after The Naushina Sodium Injection entered the Chinese market on national day in 2019, her medical center "also treated about 20 patients" in two years, and it was very rare to be able to pay all of them.
It can be compared that after the price reduction of sodium in Northinas entered the medical insurance, Liu Li's medical center was ready to treat more than twenty cases of SMA patients, "type II, type III families requiring treatment have increased significantly, most of them are type III, and they are very eager, and they are eager to hit it on January 1."
Liu Li and her colleagues stepped up the arrangement of doctors and beds, coordinated the medical insurance departments in Guangzhou and various cities, and ensured that the hospital computer system could issue medical orders, and the pharmacy department had prepared the drugs.
From the treatment of pediatric diseases for more than thirty years, Liu Li has seen too many misdiagnoses and mistreatments, ruined family and parting lives and deaths. Children with type I like sugar hearts, if they do not have ventilator support, may die by the age of two, but if they have ventilator support, they can maintain life without injecting sodium norcinol, but the quality of life is not good, they cannot move, and they can only rely on gastrostomy to eat.
Liu Li knows that after pulling out the ventilator, these children with normal intelligence and clear minds, even if the desire to survive is strong, will either slowly be unable to breathe and die, or bid farewell to the world in their sleep under the action of sedatives, which is "very cruel".
Although life is full of helplessness, from the 20 cases of patients clinically treated with nocinasone sodium in the past two years, Liu Li's team found that after treatment, patients do feel that muscle strength has increased, appetite will increase, and weight will increase.
"Most children have improved in various exercise scores, and not all children have improved, but I think it takes more time and a larger sample size to observe and objectively evaluate the effects and side effects of this drug." Liu Li told Southern Weekend reporters.
At present, there are three SMA drugs on the market worldwide, in addition to nocinazine sodium, there are also Risdiplam oral solution and Zolgensma gene therapy drugs, all of which are expensive.
"We were lucky"
On December 3, 2021, the new version of the medical insurance catalogue was officially announced, and it was implemented on January 1, 2022, compared with the previous year, the preparation time left for local medical insurance departments and medical institutions is very tight.
Seven patients in Fujian Province received injections on New Year's Day, the largest number in the country. Southern Weekend reporter learned from the Fujian Provincial Medical Insurance Bureau that all newly negotiated drugs are included in the dual-channel catalog management (referring to the two channels of designated medical institutions and designated retail pharmacies to meet the guarantee of negotiated drug supply and clinical use, and improve the accessibility of negotiated drugs), and on December 29, 2021, the platform hanging work has been completed, and the selection of Nocinasine sodium injection is included in the third batch of medical insurance drugs paid by separate outpatient clinics.
"We are speeding up all aspects of the entire supply chain, including imports, customs clearance, local distribution, stockpiling, etc., to ensure the orderly supply of follow-up drugs." Bo Jian wrote in a written reply to southern weekend reporters, "It can be said that this is the best New Year's gift we have given to the SMA patient group." ”
This New Year's gift also brings a "sweet burden" to Mei'er SMA Care Center (hereinafter referred to as Mei'er).
"The overall progress of medical insurance this year is very smooth, the local rules will come out soon, and we have been doing one thing in the past month, that is, to help SMA patients everywhere interpret the policy, and the situation of each place and everyone is different." Since the beginning of the establishment of mei'er patient organization, Xing Huanping has been running for SMA patients, and as of now, there are nearly 2400 real-name registered SMA patients.
Xing Huanping and colleagues help patients around the world to study which hospital to go to register, hang pediatrics or adult departments, how much medical insurance can reimburse, how much commercial insurance can reimburse, what materials are needed for off-site referral, etc. "When you are not with patients, you can never think of where the problem will be."
"It's only been a few days on the job, and I've seen 11 provinces get their first shots, and the patients who received the injection have also completed direct settlement in the hospital system, like creating a new miracle." When Xing Huanping was interviewed by a reporter from Southern Weekend, it was the third working day after the New Year's Day holiday.
Many SMA patients and patients' families invariably mentioned that without Mei'er, Nosina sodium would not be so fast into medical insurance. "We're just like shadows always moving throughout the process." Xing Huanping said that when a drug is listed, patients are often outside the closed loop, and patient tissues are only "always there.".
In the more than six years since Meier was founded, the mood of Xing Huanping and her colleagues is like a roller coaster, every year there are new breakthroughs and new difficulties, "I hope that patients will have a good year every year and can hear the good news of the stage."
In December 2016, the situation that SMA had been incurable for many years was finally broken, and the pharmaceutical giant Bojian's Nocinacon sodium was approved for listing in the United States.
In 2017, a number of heavy policies to accelerate drug marketing and review and approval were introduced, and a year later, SMA was recognized by the National Health Commission as included in the "First Rare Disease Catalog", and Nocinasin sodium, as a new drug for the treatment of rare diseases that has been listed overseas and is urgently needed clinically, has been included in the priority review and approval procedure by the drug regulatory department.
"The work after 2019 will be much easier to do." In 2019, Nocinasin sodium officially entered the Chinese market, the main promotion and popularization work was completed by pharmaceutical companies, and Xing Huanping said many times in the interview that "we are lucky".
"Talk about price first, then talk about protection"
The luck of the rare disease group is inseparable from the continuous development of the whole social and economic level and the optimization and innovation of the national drug policy.
Before 2017, the public was more concerned about the drug registration declaration and review and approval system that needed to be reformed. If an overseas marketed drug wants to enter the Chinese market, the process is cumbersome and long, and organizations of rare disease patients like Mei'er are waiting for the opportunity.
In 2017, the former State Food and Drug Administration issued the Decision on Matters Related to the Adjustment of the Registration and Administration of Imported Drugs, the General Office of the CPC Central Committee and the General Office of the State Council issued the Opinions on Deepening the Reform of the Review and Approval System and Encouraging the Innovation of Drugs and Medical Devices, and the dynamic adjustment of the medical insurance catalogue every year after the establishment of the National Medical Insurance Bureau in 2018, the society is paying more and more attention to the price and medical insurance policy of drugs for rare diseases.
"With the gradual improvement of the national medical security system, the vast majority of rare disease drugs have been included in the scope of medical security, and a very small number of drugs have not been included in medical insurance for the time being due to their expensive prices." Xiong Xianjun, former director of the Department of Pharmaceutical Service Management of the National Medical Security Bureau, was introduced at the China Rare Disease Conference in December 2021.
Xiong Xianjun pointed out that high research and development costs are the common characteristics of all innovative drugs, and the income from the research and development of rare disease drugs and the acquisition of research and development expenses are in Europe and the United States, so the sharing of research and development costs should also be in Europe and the United States. At the same time, rare disease drugs are generally listed in Europe and the United States first, and then listed in developing countries, and may have already recovered research and development costs in the first few years before they are listed in the European and American markets, so it is unreasonable to require developing countries to share research and development costs.
In the case of SMA, for example, patients need to inject sodium northenasone for life, 6 injections in the first year, and one injection every quarter after the second year. Southern Weekend reporter combed the third quarter of 2021 financial report of Bojian Company and found that only one sodium, Nosinasin sodium, contributed $494 million in revenue in 2020, and it is expected to continue to bring $444 million in revenue in 2021, of which the global market outside the United States accounted for more than 60%.
Considering that the number of rare disease patients in China is higher than that of developed countries in Europe and the United States, measured by the size of the market, Xiong Xianjun believes that the price of rare disease drugs should be much lower than that of developed countries in Europe and the United States.
Zheng Jie, head of the 2021 National Medical Insurance Negotiation Fund Calculation Team, once told southern weekend reporters that through statistics, it was found that among the nearly 3,000 drugs in the current medical insurance directory, the most expensive annual treatment cost was less than 300,000 yuan, about 260,000 yuan. "This represents the upper limit of the socially acceptable cost of drugs in the catalogue now, so 300,000 yuan / year is an important reference value, not a line drawn by the experts themselves."
Xiong Xianjun also pointed out that 300,000 yuan is equivalent to 4 times the level of Chinese average GDP, and many regions in the world also give 4 times the per capita GDP price for the annual treatment cost of rare disease drugs.
"We especially want to pull rare diseases (drugs) into more, especially those that have a clear effect and are exactly clinical gaps." This year has been good, with a particularly large price drop, lower than in the world (in other regions). On December 3, 2021, Huang Huabo, director of the Department of Fund Supervision of the National Medical Security Bureau, said in an interview with Southern Weekend reporters.
"I hope to stand up slowly"
"Then fight, hurry up and fight!" Nosina's raw sodium medical insurance landed, and the mother was obviously more excited than Xiaoqing, urging her daughter to hurry up and fight. Since Xiao Qing's diagnosis of SMA at the age of 4, her family has been waiting for more than 7,000 days and nights for this day.
When the 25-year-old Xiaoqing heard the news that Nosina had entered the medical insurance, she was still studying the first-year classwork of the master's degree, and the patients could not hold back the joy in the group: "After waiting for many years, I finally waited!" ”
Xiaoqing is a type III SMA patient who used to be able to "walk independently like a normal person." But growing up, this progressive disease quietly stole her ability to walk, bent her spine, and now Xiaoqing can't go out without a bulky electric wheelchair.
"After being in a wheelchair, I found that people would look at me a lot, and I was treated as an 'other.'" Xiao Qing did not want to be treated like this, "They thought that I would not have psychological ups and downs, so I looked at them with the same eyes, and when they found that they were also objectified (stared), they knew that this look was an offense, and they would feel ashamed." ”
As the American writer Susan Kane put it, the way we treat the most special groups determines who we are in nature, and in this intertwined history of failure and success, we are able to gradually see our souls.
It turns out that SMA did not steal the optimism and tenacity of this young girl, she went out in a wheelchair with friends to watch movies, went to Badaling to climb the Great Wall, measured the mutilation or thoughtfulness of barrier-free facilities with her body, and resented that people always had to add a "pity" to her.
Xiaoqing told the Southern Weekend reporter that she has made an appointment registration in the hospital, and after the final exam and the Spring Festival with her family, she will receive injections of sodium Nosinasone at the First Affiliated Hospital of Sun Yat-sen University, "The good hope is that it can no longer deteriorate, and she can slowly stand up after cooperating with auxiliary means."
Like many of her peers, Xiaoqing's biggest annoyance is her future work.
"Originally, I wanted to go to the university as a teacher, and if I entered the lifelong appointment system, it would be safer, and I could do the research I wanted to do, but then I found that it was not, and I may become a contract worker in the future, and the pressure of non-promotion and departure is very high." Xiao Qing worried.
Some of the adult patients of SMA have a bright educational background like Xiaoqing, and even have overseas education experience, but the work is more limited and can only work from home. After there is a cure for drugs and the availability of drugs has improved, how to live a good life has become a topic of communication among many SMA patients.
Xiaoqing hopes to engage in "more social" work in the future, and has a lot of communication with others offline. "My academic qualifications are not much different from others, but I need help to go to the bathroom myself, and I don't know if the employer will have additional concerns."
In the past few days, Mei'er has also received good news from some patients, and medical institutions will also guarantee medication during the Spring Festival, so that SMA patients who need to receive injections on time have no worries.
"We are such an ordinary family, hard work can also fight, no longer like before to fight this year do not know what to do next year." Sugar heart mother Hu Yue told the Southern Weekend reporter that it was originally planned to be able to inject nocinna raw sodium in late January, and a sudden Omicron epidemic delayed the treatment plan of the Tianjin patient family, but after all, it was not as anxious and out of reach as in the past.
Southern Weekend reporter Huang Sizhuo Southern Weekend intern Guo Xuan
![From 700,000 to 33,000 per needle! The price of sky-high drugs has been reduced, benefiting patients in Jiangxi[fig]](data:image/gif;base64,R0lGODlhAQABAIAAAP///wAAACwAAAAAAQABAAACAkQBADs=)