Seven drugs for rare diseases, including sodium nosinogenase injection, were included in the national medical insurance. In recent years, the inclusion of high-value rare disease drugs into medical insurance has achieved a zero breakthrough.
On the first day of the new year in 2022, the national catalogue of drugs for basic medical insurance, industrial injury insurance and maternity insurance (2021) was officially implemented. Surging news reporters learned that on January 1, nearly 20 patients with spinal muscular atrophy (SMA) in hospitals in 11 provinces and cities such as Beijing, Shanghai, Guangdong, Zhejiang, Sichuan, Shandong, Hunan, Hubei, Fujian, Jiangxi and Henan were treated with nosinasheng sodium. The drug price has dropped from nearly 70 yuan a needle to nearly 33000 yuan a needle.
That morning, the surging news reporter came to Binjiang Hospital of children's Hospital Affiliated to Medical College of Zhejiang University (hereinafter referred to as "Zhejiang University Children's Hospital"). In the Department of Neurology on the 18th floor of the inpatient department of the hospital, Xinxin, a 4-year-old SMA child, was waiting for the injection of nosinasheng sodium.
Mao Shanshan, chief physician of the Department of Neurology of the children's Hospital Affiliated to the Medical College of Zhejiang University, leader of the multi scientific diagnosis and treatment team of spinal muscular atrophy (sma-mdt) and leader of the SMA diagnosis and treatment expert group of Zhejiang Province, introduced to surging news reporters that Xinxin was diagnosed with SMA type II when she was 1 year old and 9 months old. Previously, she had been under the management and rehabilitation treatment of the multi scientific diagnosis and treatment team, "I haven't received drug treatment and have been waiting for drugs to reach."
Xinxin's mother told the surging news reporter that her family decided to take Xinxin for inspection at about the age of 1. "Basically, children learn to walk at about the age of 1, but Xinxin can't climb and sit stably when grasping the week. She always falls back and forth." After 9 months of consultation, Xinxin was diagnosed at the Capital Institute of Pediatrics in Beijing at the age of 1 year and 9 months.
SMA is an autosomal recessive disease caused by motor neuron survival gene 1 (SMN1) mutation. Due to the degeneration of motor neurons in the anterior horn of the spinal cord and medulla oblongata, SMA leads to progressive and symmetrical muscle weakness and muscle atrophy in the proximal limbs and trunk, ranking the first among the fatal genetic diseases in children under 2 years old. The incidence rate of rare diseases in the survival population of the European and American populations is about 1/10000, and the carrier frequency is 1/40-1/50. It is worth mentioning that there is no exact data on the incidence rate in China. The frequency of carriers in Chinese population is about 1/42.
At 10:36 a.m., Xinxin was carried into the injection room; At 10:48, the doctor team opened the door and the whole process was over. In the injection room, after the completion of local anesthesia, a dose of nosinasheng sodium injection was injected into Xinxin's sheath through lumbar puncture. Mao Shanshan told Xinxin before the injection, "aunt gave you a gift. It's an invisible gift, but it will make a magical change on you."
After the injection, Mao Shanshan's team gave Xinxin a second gift, a little warrior gold medal. Mao Shanshan explained the moral of this visible gift, "one side of the gold medal is Zhejiang University Children's Hospital, and the other side reads' smart marvelous angel '. Its acronym is the same as spinal muscular atrophy, which is' SMA', but the meaning is completely different."
The number on this little warrior gold medal will also change with the increase of injection times. "At present, among the patients managed by our team, the largest number on this medal is 10." Mao Shanshan believes that two gifts, one visible and the other invisible, also represent the intersection of "science" and "humanities", which are also the two forces we need to pay attention to rare disease groups.
In addition to the children's Hospital of Zhejiang University, the Second Affiliated Hospital of Medical College of Zhejiang University (hereinafter referred to as "the Second Affiliated Hospital of Zhejiang University") and Yuying children's Hospital of the Second Affiliated Hospital of Wenzhou Medical University (hereinafter referred to as "the Second Affiliated Hospital of Wenzhou Medical University") of the other two core units of SMA diagnosis and treatment expert cooperation group of Zhejiang Province have also completed the injection of the first batch of sodium nosinasheng on New Year's day.
Professor Wu Zhiying, head of the Department of medical genetics / rare disease diagnosis and treatment center of the second hospital of Zhejiang University and director of the Zhejiang cooperation group of China rare disease alliance, told surging journalists that a middle-aged male patient who received sodium nosinogenase injection in the second hospital of Zhejiang University had a long misdiagnosis time and a late diagnosis time, "It was diagnosed only 10 years ago. The price has been relatively expensive since there are drugs to treat it, and there has been no drug treatment."
It should be noted that the inclusion of sodium nosinogenase injection in the medical insurance catalogue will benefit more SMA patients. "At the same time, it will also be a great challenge for our doctors." Mao Shan believes that the most important thing for us to do in the future is to provide more and more accurate medical resources for patients with SMA
For example, she told surging journalists, "the clinical manifestation of SMA patients is progressive aggravation. For example, because they have no strength, the patient's spinal deformation will gradually aggravate." Long term untreated patients may also come to see a doctor in the future, "if some patients have very serious spinal deformation, it will be more difficult for us to inject intrathecal injection into patients."
Wu Zhiying also talked about the above problems. She also stressed that "there will be more patients, and our workload will naturally increase. We need to better manage these patients." Wu Zhiying also mentioned that at present, the drug delivery method of nosinogenase sodium is temporary procurement according to the special clinical needs. "The drug price is expensive and the storage requirements are high, but after the number of patients increases in the future, I hope the drug supply channel can be further smooth."
Wu Zhiying said that the research and development of drugs for rare diseases is difficult, high range and long cycle. It has always been difficult to meet the clinical needs of patients. Timely research and development and listing are often extremely expensive, and the accessibility of drugs is facing severe challenges. This adjustment of the medical insurance catalogue shows that the state attaches great importance to the group of patients with rare diseases, The newly added drugs for rare diseases have well filled the gap in the treatment of rare diseases in China, provided a choice for clinical drugs, and brought hope for more patients with rare diseases to delay the development of the disease and improve the quality of life.
Nosinasheng sodium injection is the world's first disease correction treatment drug for the pathogenic cause of SMA. With accurately designed antisense oligonucleotide (ASO), it directly acts on spinal cord motor neurons through intrathecal injection, opening the era of SMA disease correction treatment. It has won the Galen prize for best biotechnology product, known as the "Nobel Prize in the pharmaceutical industry". In December 2016, sodium nosinogenase injection was approved in the United States. In 2018, sodium nosinogenase injection entered the list of the first batch of overseas new drugs in urgent clinical need in China, and was approved for listing in China in February 2019.
Previously, due to the high price of nearly 70 thousand injections, nosinasheng sodium injection was difficult for patients to reach. In May 2019, China primary health care foundation announced the official launch of the first SMA patient assistance project, and the aid drugs were donated by Bojian company.
In January 2021, the "spinal cord living newborn - assistance project for patients with spinal muscular atrophy" was upgraded again, further reducing the self payment proportion of patients. The data show that with the help of the previous new assistance program, the out of pocket cost of drug treatment for patients using nosinoxazole sodium injection in the first year decreased from about 1.4 million yuan to 550000 yuan, a decrease of about 60% in the first year; After that, the annual out of pocket cost of drug treatment was reduced from the original average annual 1.05 million yuan to 550000 yuan, a decrease of about 50%.
Thanks to the medical insurance negotiation in 2021, the injection of sodium nosinogenase has been reduced to nearly 33000 yuan per injection. It is reported that patients only need thousands of yuan at their own expense.
(surging News)
