Source: Hangzhou Traffic 918
At the beginning of December 2021, Zhang Jinni, a negotiator of the National Medical Insurance Bureau and director of the Pharmaceutical equipment Procurement Supervision Department of the Fujian Provincial Medical Insurance Bureau, brushed the screen. In the video, after 8 rounds of price negotiations by the medical insurance department, the price of the rare disease drug "Northinalsan Sodium Injection", which was originally 700,000 yuan a shot, was finally sold at a price of 33,000 yuan / needle.
On the first day of the New Year in 2022, the National Basic Medical Insurance, Work Injury Insurance and Maternity Insurance Drug Catalogue (2021) was officially implemented. The reporter 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 received sodium treatment for nocinasine.
On the morning of the 1st, the reporter came to the Binjiang Branch of the Children's Hospital Affiliated to Zhejiang University School of Medicine (hereinafter referred to as "Zhejiang University Children's Hospital"), and on the 18th floor of the neurology department of the hospital's inpatient department, Xinxin (pseudonym), a 4-year-old child with SMA, was waiting for injection of Nocinazine sodium.
Mao Shanshan, chief physician of the Department of Neurology of the Children's Hospital Affiliated to Zhejiang University School of Medicine, leader of the multiscientific diagnosis and treatment team for spinal muscular atrophy (SMA-MDT), and leader of the SMA diagnosis and treatment expert group in Zhejiang Province, said that Xinxin was diagnosed with SMA TYPE II at the age of 1 year and 9 months, and had been receiving management and rehabilitation treatment from the multi-scientific diagnosis and treatment team. ”
Xinxin's parents are anxiously waiting, and the sister of the hospital bed next door who has recently met is holding Xinxin's favorite doll.
Xinxin's mother told reporters that the family decided to take Xinxin to check on it at about 1 year old, "Basically the child learned to walk at the age of 1, but Xinxin could not climb when she grabbed the week, nor could she sit steadily, and always fell backwards." After 9 months of seeking medical treatment, at the age of 1 year and 9 months, Xinxin was diagnosed at the Capital Pediatric Research Institute in Beijing.
SMA is an autosomal recessive disorder caused by mutations in the motor neuron survival gene 1 (SMN1), which leads to progressive, symmetrical muscle weakness and muscle atrophy in the proximal limb and trunk due to degeneration of the anterior horn of the spinal cord and bulbar motor neurons, ranking first among the fatal genetic diseases in children under 2 years of age. The incidence of this rare disease in surviving newborns in European and American populations is about 1 in 10,000, and the frequency of carriers is 1/40-1/50. It is worth mentioning that there is no exact data on the incidence in China, and the frequency of carriers in Chinese group is about 1/42.
Xinxin completed the injection, and the team of Mao Shanshan of the Children's Hospital Affiliated to Zhejiang University School of Medicine presented the little warrior gold medal
At 10:36 a.m., Xinxin was carried into the injection room; at 10:48 a.m., the doctor's team pushed open the door and the whole process ended. In the injection chamber, after completing local anesthesia, a dose of Nosinasin sodium injection was injected into Xinxin's sheath by lumbar puncture. Mao Shanshan told Xinxin before the injection, "Auntie gives you a gift, which is an invisible gift, but it will change very magically in you." ”
After the injection was completed, Mao Shanshan's team gave Xinxin a second gift, a small warrior gold medal. Mao Shanshan explained the meaning of this visible gift, "On one side of the gold medal is Zhejiang University Children's Hospital, on the other side is written 'Smart Marvelous Angel', its acronym is the same as spinal muscular atrophy, it is 'SMA', but the meaning is completely different. ”
The number on this little warrior gold medal will also change with the increase in the number of injections, "At present, among the patients managed by our team, the largest number on this medal is already 10. Mao Shanshan believes that two gifts, one visible and one invisible, also represent the intersection of "science" and "humanities", which is also the two forces we need when focusing on rare disease groups.
Little Warrior Gold Medal
In addition to Zhejiang University Children's Hospital, the second affiliated hospital of Zhejiang University School of Medicine (hereinafter referred to as "Zhejiang University Second Hospital") and the Yuying Children's Hospital (hereinafter referred to as "Wenzhou Medical Second Hospital"), two other core units of the Zhejiang SMA Diagnosis and Treatment Expert Collaboration Group, have also completed the first batch of injections of sodium Northinalsen 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 Provincial Collaborative Group of the China Rare Disease Alliance, introduced that the middle-aged male patient who received sodium injection from Nocinasine in the Second Hospital of Zhejiang University had a long misdiagnosis time and a late diagnosis time. ”
It should be noted that the inclusion of Nocinasine sodium injection in the medical insurance directory will benefit more SMA patients. "At the same time, it will be a big challenge for our doctors." Mao Shanshan believes, "What we have to do at this time is to build a better MDT team and provide the most accurate and comprehensive multidisciplinary medical resources for more and more SMA patients who can use drugs in the future." ”
For example, she said, "The clinical manifestation of SMA patients is progressive exacerbation, such as because there is no strength, the patient's spinal deformation will slowly worsen. "Long-term untreated patients may also come to see the doctor in the future," if some patients have spinal deformations that are already very serious, then it is more difficult for us to inject patients intrathecally. ”
Wu Zhiying also talked about the above problems, she also stressed, "There will be more patients, our workload will naturally increase, we need to better manage these patients." Wu Zhiying also mentioned that the current drug intake method of Sodium Northinal is based on temporary procurement according to special clinical needs, "The price of drugs is expensive and the storage requirements are high, but after the increase in patients in the future, I hope that the drug supply channel can be further smoothed." ”
Wu Zhiying said that the research and development of rare disease drugs is difficult, the amplitude is high, the cycle is long, it has been difficult to meet the clinical needs of patients, timely research and development, the price is often extremely expensive, the availability of drugs is facing severe challenges, this medical insurance catalog adjustment, indicating that the country pays great attention to the rare disease patients this group, the newly added rare disease drugs into the catalog are well filled in the gap of domestic related rare disease treatment drugs, providing options for clinical drugs, for more rare disease patients to delay the development of the disease, improve the quality of life has brought hope.
Northinal sodium injection is the world's first disease correction treatment drug for the cause of SMA disease, with a precise design of antisense oligonucleotide (ASO), through intrathecal injection directly on spinal cord motor neurons, opening the era of SMA disease correction treatment. It has won the Galen Prize for Best Biotechnology Product, known as the "Nobel Prize of the Pharmaceutical Industry". In December 2016, Northinalson sodium injection was approved in the United States. In 2018, Nocinasin sodium injection entered China's "List of the First Batch of Clinically Urgently Needed Overseas New Drugs" and was approved for listing in China in February 2019.
Previously, The high price of nearly 700,000 injections made it difficult for patients to reach. In May 2019, the China Primary Health Care Foundation announced the official launch of the first SMA Patient Assistance Program, with the aid drugs donated by Bojian.
In January 2021, the "Spinal Renewal – Spinal Muscular Atrophy Patient Assistance Program" was upgraded again, further reducing the out-of-pocket ratio of patients. The data shows that with the help of the previous new assistance program, the out-of-pocket cost of drug treatment for patients using Nocinasin sodium injection in the first year decreased from about 1.4 million yuan to 550,000 yuan in the first year, a decrease of about 60%; after that, the annual out-of-pocket cost for drug treatment was reduced from the original average of 1.05 million yuan per year to 550,000 yuan, a decrease of about 50%.
Thanks to the 2021 health insurance negotiations, the current Nocinasine sodium injection has dropped to nearly 33,000 yuan per injection. It is reported that patients only need a few thousand yuan at their own expense.
Source: Hangzhou Wenguang Group - Hangzhou Comprehensive Channel (ID: htvweiyi), The Paper (ID: thepapernews)