After 15 days of denial, the "life-saving medicine" for children with angel syndrome continued!

(People's Daily health client reporter Tan Qixin) On March 26, the 2-week supply crisis of "angels" life-saving drug "clonazepam" was finally lifted.

"As a family member of a child with a rare disease, we no longer expect our children to have a normal life like a normal child, and our wish is very simple: to keep taking medicine and saving our lives." Zhang Xue, head of the Angel Syndrome Patient Organization, said in an interview with the People's Daily health client reporter.

Angelman syndrome (Angelman syndrome) is one of the rare diseases included in the mainland "the first batch of rare disease catalog", patients have epilepsy, developmental delay, language development disorders and movement disorders and other serious symptoms, according to the international incidence of 1/15,000 calculations, it is expected that there are 5-100,000 patients in China, the current statistics are on the register, only about 1,000 patients have been confirmed.

The patient's family group that boiled after receiving a reply from the temporary resumption of supply by the pharmaceutical factory, the interviewee provided the picture.

So far, there is no medicine to cure, and the "life-saving drug" to control epilepsy has been cut off

"The child was diagnosed more than 1 year old, it has been 11 years, the cognitive level is fixed at fifteen months, and the language level is fixed at 11 months. In the thousands of nights since the diagnosis, the child has not been able to sleep a full and stable sleep, nor have I. Zhang Xue told reporters.

July 6, 2012, is Lei Lei's first birthday, according to the regulations, Lei Lei must undergo a routine intelligence examination on the same day, this investigation completely broke the peaceful life of Zhang Xue's family. "On the same day, the doctor measured the child's intelligence several times and told us that the child's intelligence was only ten and a half months," Zhang Xue recalled, after referral to a higher-level hospital, the final diagnosis was that Lei Lei's cognitive level may be frozen.

As a mother, Zhang Xue was not reconciled to this result. "What causes the sluggishness?" "Is there any medicine that can be cured?" In order to find the answer, Zhang Xue took Lei Lei to seek medical treatment in multiple departments of the hospital's pediatric protection department, neurology department, and rehabilitation department, and registered one doctor after another, and this door came out to see that door. After three months of running like this, Lei Lei was diagnosed with "angel syndrome".

"The doctor told me that there is no cure for this disease at present, and there is no cure." Zhang Xue said. In addition to developmental delay and slow movement, almost all children with angel syndrome are accompanied by severe seizures, in order to control the disease, "angels" often need to take 3-4 drugs at the same time, clonazepam is one of them. Clonazepam, also known as "the strongest sleeping pill", is a class II psychotropic drug, which can treat epilepsy and convulsions under the guidance of professional doctors.

The reporter inquired into 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, at present, the purchasable clonazepam on the market is mainly produced by two pharmaceutical companies, namely Jiangsu Enhua Pharmaceutical and Shanghai Shangyao Xinyi Pharmaceutical Factory.

In the past decade, the families of children with angel syndrome have been able to purchase the corresponding amount of clonazepam after the "neurology" doctor of the children's hospital prescribes it. However, from March 10, Zhang Xue found that the supply of clonazepam was cut off, and the difficulty of purchasing clonazepam across the country gradually appeared.

More than 1,000 family members sent letters for help

The children's life-saving drug "clonazepam". Courtesy of respondents.

In order to find a way out for themselves, nearly 100 family members of the children have reported to the 12345 hotline within a week that the "clonazepam" drug for the daily control of epilepsy in children with angel syndrome has been discontinued. But as the days passed, the drugs still failed to be sold normally...

"The response we received was that only the Shanghai Mental Health Center could supply the control of psychotropic drugs and the application of clonazepam pharmaceutical companies to raise the price of drugs that had not yet been approved by the relevant authorities. However, angel syndrome is a rare disease, and there are very few doctors who understand this rare disease around the world, and we need to go to "neurology" in our daily medical treatment, not "psychiatry". If we go to the Shanghai Mental Health Center, doctors will not be able to prescribe clonazepam for our children. Zhang Xue told reporters.

Overwhelmed parents thought of sending out letters of help to the whole society to help their children find "life-saving medicines" as soon as possible. At 8:00 a.m. on March 25, an article entitled "Who Moved My Child's Medicine - A Letter of Help from More Than 1,000 Families of Children with Rare Disease Angel Syndrome" was released on major platforms.

Less than an hour after the letter of help was sent, it received the attention of the Corde Rare Disease Center, and under the active coordination of the latter, four hours later, the families of the children with Angel Syndrome received a reply from the pharmaceutical company: it is expected that on March 28, the supply of clonazepam will be temporarily restored at the Children's Hospital affiliated to Fudan University and the Shanghai Children's Medical Center.

Who wants to develop special medicines for our children

"Clonazepam is a life-saving medicine for children, as long as we ensure that there is a medicine available and a medicine to save life, we can accept a reasonable range of price increases." In the case that the producer and the demand side have reached an agreement on the price increase of the drug, our children still have to face the dilemma of drug discontinuation, which link is the problem? After a short period of excitement, Zhang Xue's mood was not relaxed.

The reporter learned that the current price of clonazepam is about 32 yuan / bottle (100 pieces), a piece of only about 3 cents, this price has not changed for 10 years, after medical insurance reimbursement, the patient pays about 28 yuan / bottle, for a 10-year-old child with angel syndrome, a bottle can be taken for half a year.

In view of the tight supply of "life-saving drugs" that have frequently appeared in various places in recent years, since 2019, the regulatory measures of national policies have continued to relax. According to the "Opinions on Doing a Good Job in the Management of Current Drug Prices" issued by the National Medical Insurance Bureau, for the varieties in the list of drugs that are prone to shortage at the national and provincial levels, enterprises are allowed to set their own reasonable pricing, directly bid for procurement on the network, and medical institutions to purchase at the price of the network or further negotiate with operators for procurement. 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 pricing, and the bargaining power of enterprises was limited, and now independent pricing can relatively guarantee their own profits while reporting reasonable prices, but enterprises eventually face the situation of passive price reduction, and the price declared by enterprises still needs to be further verified and negotiated by relevant departments. It is not difficult to solve the shortage of drug procurement, and the relevant supporting policies must be further improved. The above-mentioned industry insiders said.

For the families of more than 1,000 children with Angel Syndrome, they have further concerns, "If there is no reasonable incentive mechanism, who would want to develop a special drug for our children?" ”