(People’s Daily Health Client Reporter Tan Qixin) On March 26, the two-week supply-cut crisis of the “Angels”‘s life-saving drug “clonazepam” was finally lifted.
“As the family members of children with rare diseases, we no longer expect our children to have a normal life like normal children. Our wish is very simple: keep taking medicine and save lives.” Angel Syndrome Patient Zhang Xue, the head of the organization, said in an interview with a reporter from the People’s Daily Health Client.
Angelman syndrome is one of the rare diseases included in the “First Batch of Rare Diseases List” in my country. For severe symptoms, according to the international incidence rate of 1/15,000, it is estimated that there are 50,000 to 100,000 patients in China. Currently, there are only about 1,000 patients who have been registered and diagnosed.
The patient family members who are boiling after receiving the reply from the pharmaceutical factory temporarily restore the supply, the interviewee For picture.
There is currently no medicine to treat, and the “life-saving medicine” for controlling epilepsy is cut off
“The child was diagnosed at the age of 1, and it has been 11 years since the diagnosis. The cognitive level is fixed at 15 months, and the language level is fixed at 11 months. In the thousands of nights since the diagnosis, The child has not had a complete and peaceful sleep, and neither have I.” Zhang Xue told reporters.
July 6, 2012 was Lei Lei’s first birthday. According to regulations, Lei Lei had to undergo a routine intelligence examination on that day. This examination completely broke the peace of Zhang Xue’s family. life. “On that day, the doctor tested the child’s intelligence several times and told us that the child’s intelligence was only ten and a half months old,” Zhang Xue recalled. After being transferred to a higher-level hospital, the final diagnosis was that Lei Lei’s cognitive level may have been frozen.
As a mother, Zhang Xue was not reconciled to this result. “What caused the delay?” “Is there any medicine to cure it?” In order to find the answer, Zhang Xue took Lei Lei to the hospital’s pediatrics department, neurology department, and rehabilitation department to seek medical treatment, and registered doctors one by one. Go out the door and look at that door. Three months later, Lei Lei was diagnosed with “Angel Syndrome”.
“The doctor told me that there is no cure for this disease, and it cannot be cured.” Zhang Xue said. In addition to developmental delay and slow movement, almost all children with Angelman syndrome are accompanied by severe epileptic seizures. In order to control the condition, “angels” often need to take 3-4 drugs at the same time, and clonazepam is one of them. . Clonazepam, also known as the “strongest sleeping pill”, is a second-class psychoactive drug. Under the guidance of a professional doctor, it can treat epilepsy and convulsions.
The reporter checked the official website of the State Food and Drug Administration and found that there are currently 18 drug production approvals for clonazepam, involving 11 pharmaceutical companies. However, currently available clonazepam on the market is mainly produced by two pharmaceutical companies, namely Jiangsu Enhua Pharmaceutical and Shanghai Shanghai Pharmaceutical Xinyi Pharmaceutical Factory.
In the past ten years, families of children with Angelman syndrome have been able to purchase the corresponding amount of clonazepam after a prescription from the “neurologist” doctor of the Children’s Hospital. However, starting from March 10, Zhang Xue discovered that the supply of clonazepam had been cut off, and the difficulty of purchasing clonazepam across the country gradually became apparent.
More than 1,000 families of children send letters of help
Children’s life-saving medicine, Clonazepam. Photo courtesy of the interviewee.
In order to find a way out, nearly 100 family members of the children reported to the 12345 hotline within a week of the problem of discontinuing the “clonazepam” drug for children with Angelman syndrome to control epilepsy daily . But as time passed, the medicines still failed to be put on the shelves for normal sales…
“The reply we got is that because of the control of psychotropic drugs and the application of clonazepam production pharmaceutical companies to increase drug prices has not been approved by the relevant departments, only the Shanghai Mental Health Center has Can supply. But Angel Syndrome is a rare disease, and there are very few doctors in the world who understand this rare disease. We need to go to the “neurology department” instead of “psychiatry” for daily medical treatment. Going to the Shanghai Mental Health Center, the doctor could not prescribe clonazepam for our children,” Zhang Xue told reporters.
The overwhelmed parents thought of sending a letter of help to the whole society in order to help their children find “life-saving medicine” as soon as possible. At 8:00 a.m. on March 25th, an article titled “Who Moved My Child’s Medicine-A Letter of Help from the Family of More than 1,000 Rare Disease Angel Syndrome Children” was released on major platforms.
In less than an hour after the letter of help was sent, it got the attention of the Koder Rare Disease Center, and under the active coordination of the latter, the family of the child with Angelman Syndrome 4 hours later We received a reply from the pharmaceutical factory: It is expected that on March 28, the supply of clonazepam to the Pediatric Hospital Affiliated to Fudan University and Shanghai Children’s Medical Center will be temporarily restored.
Who wants to develop special medicines for our children
“Clonazepam is a life-saving medicine for children. As long as the medicine is available and life-saving, we can accept price increases within a reasonable range. The producers and demanders are raising the prices of medicines. Under the circumstance of reaching an agreement with Cocoa, our children will still face the dilemma of drug withdrawal, which link is the problem?” After a short excitement, Zhang Xue’s mood was not easy.
The reporter learned that the current price of clonazepam is about 32 yuan per bottle (100 tablets), and one piece is only about 3 cents. This price has not changed for 10 years. After reimbursement, the patient will pay about 28 yuan/Bottle, for a 10-year-old child with Angelman syndrome, one bottle can be taken for half a year.
In response to the tight supply of “life-saving drugs” that has frequently occurred in various places in recent years, since 2019, the regulatory measures of national policies have continued to be relaxed. According to the “Opinions on Doing a Good Job in the Management of Current Drug Prices” issued by the National Medical Insurance Administration, for the varieties in the national and provincial lists of drugs that are prone to shortage, enterprises are allowed to set prices independently and reasonably, and directly purchase through online bidding. Procurement or further negotiation with the operator to negotiate the purchase. However, an industry insider who did not want to be named told reporters that there are still many restrictions behind this “autonomy”.
“In the past, it was bidding and pricing, and the bargaining power of enterprises was limited. Now, independent pricing can relatively protect their own profits and report a reasonable price, but enterprises still face a situation of passive price reduction in the end. The price still needs to be further verified and negotiated by the relevant departments. Often after a series of links, the result is still the one with the lower price. To solve the difficulty of purchasing drugs in short supply, the relevant supporting policies need to be further improved.” said the above-mentioned industry insiders.
For the families of more than 1,000 children with Angelman syndrome, they have further concerns, “If there is no reasonable incentive mechanism, who will be willing to develop special drugs for our children? “
