Today is the 17th International Day of Rare Diseases. The World Health Organization places the number of cases at 0065%—0.Between 1% of diseases are defined as rare diseases. According to China's established rare disease registration system, more than 780,000 rare disease cases have been registered in more than 480 hospitals across the country so far.
The new version of the NRDL includes 15 drugs for rare diseases.
Due to the high cost of research and development and the small number of drug users, the average annual cost of some rare disease drugs can reach millions of yuan, or even millions of yuan, which is astronomical for ordinary families. In recent years, many rare disease drugs that have been approved for marketing have been negotiated and included in medical insurance, significantly reducing the burden of family medication for rare disease patients.
Recently, the results of the adjustment of the 2023 medical insurance drug list have been implemented. A total of 126 new drugs have been added in this adjustment, including 15 drugs for rare diseases, filling the gap of 10 drugs for rare diseases in the catalog. The price reduction and medical insurance reimbursement have significantly reduced the burden on patients in related fields.
Huang Xinyu, Director of the Department of Pharmaceutical Administration of the National Health Insurance Administration: **Agamod injection for myasthenia gravis, and eculizumab for paroxysmal nocturnal hemoglobinuria, these diseases are actually some rare disease drugs that have plagued us for many years, and the original ** cost is very high. After negotiation, the company also gave a more rational ** and was able to successfully be included in the catalog. There will be significant benefit for these patients in the future.
Since its establishment, the National Health Insurance Administration has carried out the adjustment of the national medical insurance drug list for six consecutive years. According to statistics, more than 80 of the more than 100 drugs for rare diseases that have been approved for marketing in China have been included in the list of drugs covered by the National Medical Insurance List. Among them, 51 rare disease drugs included in the medical insurance negotiation have an average price reduction of more than 50%.
Huang Xinyu, Director of the Department of Pharmaceutical Administration of the National Health Insurance Administration: It is true that rare diseases have received the attention of the whole society, and everyone's awareness of rare diseases has been continuously improved, and the clinical diagnosis and treatment ability of rare diseases has also been continuously improved. In recent years, from the perspective of new drug research and development, rare disease drugs are also a hot spot in research and development, so based on these circumstances, there are relatively more and more rare disease drugs included in the catalog.
From drug-free to sky-high to affordable medicine.
Spinal muscular atrophy, also known as "SMA", is a rare condition in which motor neurons in the anterior horn of the spinal cord degeneration lead to muscle weakness and muscle atrophy. Through the adjustment of the medical insurance drug list in recent years, two drugs for SMA have been included in the medical insurance with significant price reductions, and patients have gone from no drugs available, to sky-high prices, to affordable and affordable drugs.
In a ** center in Qingdao, Shandong Province, children and their families are participating in the game of pitching pots and Tuofu characters, and the children are having a lot of fun.
Sun Haiying, director of a ** center in Qingdao: Today, 15 SMA children also participated, and after the first training, they recovered a certain motor function and were able to participate in similar activities moderately.
Patient Zhang Jianuo: I made it myself, and my mother held me to do it.
Zhang Jianuo's mother Li Hui: When he couldn't lift his arm before, he didn't have the strength to press it down at all, but today I held him, and he dipped his arm in that thing to make a blessing character, which was very happy.
The four-and-a-half-year-old little Jianuo had a lot of fun at the event, and he didn't look much different from ordinary children. Li Hui said that when Xiao Jianuo was seven or eight months old, it was normal to kick his legs, raise his head, and turn over, but slowly something was wrong.
Zhang Jianuo's mother Li Hui: When it was time to climb, he couldn't climb at four o'clock, so we thought about going to other places to check, and when the doctor saw it, he knocked the child on the knee, and there was no knee jerk reflex, so he wrote SMA-II on the paper, put a question mark, and let us do genetic testing.
After various tests, the child was finally diagnosed with SMA. Although the special drug has been approved to enter the country, however, this drug is as high as 69 in one shot970,000 yuan, a year ** fee needs millions of yuan, which is undoubtedly an astronomical amount for ordinary families.
Zhang Jianuo's mother Li Hui: In the first year, I needed 6 injections, and 6 injections was more than 4 million, and in 2020, I wanted to sell everything in my family's house, but at our local economic level, it was not enough for the child's expenses for a year. I felt that although there was medicine, I couldn't save the child, and I felt very uncomfortable.
The special medicine can't afford it, so Li Hui can only choose to give the child ** to delay the development of the disease. In November 2021, children and families with SMA finally waited for a turnaround. After negotiations, the special drug was reduced from 700,000 injections of "sky-high drug" to 3About 30,000 yuan, included in the list of medical insurance drugs. Coupled with medical insurance reimbursement and subsidies, the burden on patients is even lower.
Zhang Jianuo's mother Li Hui: At that time, my father and I cried. I am really grateful to the country for letting us see that the child is saved and there is hope.
The sooner you take the medication, the better the effect. On January 9, 2022, two-and-a-half-year-old Xiao Jianuo received his first shot. Subsequently, with the special effect medicine and ** training, Xiao Jianuo's condition is getting better and better.
Xu Dan, the limb leader of a ** center in Qingdao: Before he used to maintain head control with elbow support on the flat ground, he could only maintain it for 5 seconds. Now, he can move forward on this elbow support, and he can also make the transition from elbow support to hand support on the ball.
Xiao Jianuo's mother calculated that the original annual drug burden was one million, and now with various reimbursements and policy support, the personal burden is about 30,000 yuan. "Normal life", for this SMA family, suddenly changed from an unattainable dream to a new New Year's wish that can be touched.
Zhang Jianuo's mother Li Hui: Dad does a few more jobs for the child, so that he can gradually get better.
Patient Zhang Jianuo: I want to walk up quickly, help my mother with her work, and go out on my mother's back.
Reporter: Can you carry your mother?
Patient Zhang Jianuo: When I am older and taller than her, I can carry it on my back.
At present, the National Medical Insurance Catalogue contains two SMA specific drugs.
Now, there are two SMA drugs in the national medical insurance list, and more and more families are seeing hope.
When the SMA department of this ** center was first established half a year ago, there were only 7 families working on **, and now it has increased to 22.
Sun Haiying, president of a ** center in Qingdao: I can clearly feel that more and more children are coming, and we should improve the children's ** effect as soon as possible, so that they can return to society as soon as possible.
Zhang Jianuo's mother Li Hui: This morning he also got up and said, Mom, I am a strong man, and I am brave and not afraid of difficulties. I said okay, then you will soon walk up and down to your happy childhood like everyone else.