policy has medicine but it is gone. The real version of "I am not a god of medicine" staged
text/Xinna Cao Yujie
edited by Wang Xiao
pic/pixabay
rare disease medicine will withdraw from China, affecting the lives of thousands of patients.
This medicine is sapropterin hydrochloride tablets (trade name "Kewang"), and is an indispensable medicine for some patients with rare diseases such as hyperphenylalanineemia (HPA). There is no generic drug available in the mainland Chinese market. Once the drug is withdrawn, it means that no drug will be available in these patients.
This rare disease is caused by abnormal phenylalanine metabolism in the human body. During the metabolism process, the enzyme is missing or completely missing, resulting in a higher concentration of phenylalanine in the blood in the body. This is a genetic mutation or genetic disease.
This drug is produced by BioMarin, a multinational company headquartered in California. The company's staff responded to the withdrawal of the Chinese market on the matter of withdrawing from the Chinese market. The reporter said that has been listed abroad, and the market for patients using "Science" has been lost in large quantities. From the overall consideration of the company, the value of continuing to retain the product line is not great, so it is considered to withdraw from the Chinese market.
A enterprise needs to consider economic accounts. However, on the other side of the balance is the current situation where patients urgently need medication.
The latest situation learned by the reporter of "Finance" is that the China-Japan Friendship Hospital will be the application for temporary import of drugs urgently needed in clinical practice. At present, the drug is being cleared and is expected to arrive at the hospital by the end of October, but it is not sure how many bottles of medicine are there.
Due to the long distance, different reimbursement ratios in other places, and the drugs themselves are susceptible to oxidation and other factors, most patients in other regions cannot come to Beijing Sino-Japanese Friendly Hospital to prescribe medicine, which means that there will be no medicine available for a long time. There is still a long way to go to how to solve the accessibility of rare diseases drugs.
There is no medicine to take
html In late June, Lu Wen (pseudonym) learned that "Ku Wang" no longer supplies the Chinese market. For a moment, she was anxious, "Patients cannot do without this medicine. This medicine helps children metabolize phenylalanine in their bodies, and they cannot be missed once a day."
Lu Wen lives in Beijing. Her three-year-old child suffers from tetrahydrobiopterin deficiency (BH4 deficiency, also known as malignant PKU), and is a "rare group" in the rare disease hyperphenylalanineemia. Since the child was diagnosed, Lu Wen and his family have been actively treating and taking medicine for the child, and now the child is developing more normally. "If you don't know that she suffers from this disease, you can't tell it just by looking at her appearance, because you are afraid that the child will be discriminated against, only the family knows about the child's situation." Lu Wen said.
Hyperphenylalanineemia is mainly children, especially newborns and infants. Once they become ill, they need lifelong treatment. When a child is diagnosed with hyperphenylalanineemia, he or she needs to determine his or her classification through layered screening: one is phenylalanine hydroxylase deficiency (PAH deficiency, also known as PKU); the other is tetrahydrobiopterin deficiency (BH4 deficiency, also known as malignant PKU).
Relevant person in charge of the Beijing HPA patient organization said that PKU patients account for more than 90% of the total patients and need to be treated with dietary control. 8% of patients with malignant PKU are treated with pure drug treatment. Another 2% of patients with reactive phenylketonuria can choose drug treatment or dietary control treatment.
Lu Wen's child is "unfortunate" belongs to 8%, malignant PKU, which can only be treated with drugs. The child started taking "Kuowang" since he was one month old. It has been more than three years since he was taking medicine. The price of the medicine has dropped from 9,000 yuan in the first bottle to 7,480 yuan in a bottle today. Because the child is still young, parents need to grind the medicine into powder every time and mix it with water before taking it to the child. The dosage of
is calculated based on the child's weight. It is recommended to take 5 mg per kilogram of body weight. "Kewang" has 30 capsules, each capsule is 100 mg. Lu Wen's child currently needs to take 60 mg a day, and he also needs to take medoba and serotonin. "Kewang" is to control blood value, and the other two target and in the nervous system. Any of these three drugs will affect the treatment of the child.
Beijing issued a new policy at the end of 2021, and "Kewang" was included in the scope of drug reimbursement, with a lifetime reimbursement of 60%. Lu Wen introduced that 40% of them are self-paid, and one bottle is worth 3,000 yuan. This stems from a cost subsidy policy issued by Beijing for special diseases, which is reimbursed. However, the other two are self-paid, and the monthly medicine fee is about 2,500 yuan, and the previous month's medicine fee is about 6,000 yuan.
However, the policy benefits have not been enjoyed for half a year. Lu Wen learned from the Beijing patient group that this drug will be withdrawn from China.
In the document "Request for Coordinating the Supply of this Drug" submitted by the Beijing Health Commission to the National Health Commission's Pharmaceutical Administration Department, "Ku Wang" is a rare disease drug that can reduce the risk of continuous brain damage and improve the quality of life of children. It requires lifelong use.
At Beijing Sino-Japanese Friendship Hospital, about 30 children use this medicine all year round every year. currently has no alternative drugs sold in China, and there are no alternative drugs similar to BH4. If "Kewang" is no longer available for the Chinese market, these rare children will face the situation where no medicine is available.
"Finance" reporter asked the Beijing Health Commission for confirmation and learned that the document was true.
"Take a risk" to use generic drugs
Many patients with rare diseases choose to use generic drugs, which can not only relieve the condition but also save money, and can also take over in time when there is no original drug. This is also the origin of India's "divine god".
A patient with multiple sclerosis had carefully calculated the calculations. On August 8, 2022, the domestic generic drug Terifomide was launched, with 30 tablets per box, and the drug price was 6,896 yuan. This drug can treat multiple sclerosis. The original medicine produced by Sanofi is 7,896 yuan per box of 28 pills, which is 1,000 yuan more expensive than a domestic box. The part of this generic drug
is at its own expense, which is about 65% of the original drug. The above-mentioned patient said, "I am at my age, I have elderly parents and young children, and I have to pay 15,000 yuan a month for mortgage loans. I save more than 20,000 yuan a year for domestic drugs, and I will have the money for tutoring for children."
However, patients with hyperphenylalanineemia are not so lucky. At present, there is no "Kewang" generic drugs on the market in the Chinese market.
Zhang Ruting lives in Chaozhou, Guangdong. Her child is a reactive patient who only accounts for 2% of the number of hyperphenylalanineemia patients. She can actively choose drug treatment or dietary control treatment. 90% of patients choose diet control, and the cost is relatively low, but Zhang Ruting "worried that children will be different from other children when they go to school and suffer from mental illnesses", so she gritted her teeth and chose drug treatment. In addition to imported sapropterin hydrochloride tablets, she also needs to take phenylalanine-free amino acid powder and phenylalanine-free complex amino acid powder for her children.
Beijing Medical Insurance can reimburse "Kuowang" ratio of 60%. It is fully self-paid in Guangzhou, and the price of medicine is more than Beijing. A bottle of Beijing is 7,480 yuan, and a bottle of Beijing is more than 1,000 yuan, reaching 8,815 yuan.
If the child is given medicine to control his condition, Zhang Ruting did the calculation: the child is 6 years old this year, 22 kilograms, and there are about eight cans of phenylalanine powder per month, each can of 258 yuan; "Kewang" requires 40 mg per day, and the monthly cost is about 5,500 yuan. Both drugs need to be used as the child increases in weight, and the cost will be higher and higher.
Zhang Ruting and her husband work in Chaozhou , with a monthly income of 10,000 yuan. They have to spend half of their children's medical expenses, which they can't afford. So she turned her attention to generic drugs sold by a Taiwanese company in China, which were relatively cheap.
generic drugs in a box of 100 pills, each pill of 10 mg. In the past year, the cost per box fluctuates between 890 and 950 yuan. Children need to take 120 pills per month, which costs about 1,100 yuan, and the price is only one-third of the "Kuowang". Zhang Ruting told the "Finance" reporter that although can still be purchased at present, since the generic drug needs to be frozen, the time of round trip, weather reasons, etc. will affect the efficacy of the drug.
More importantly, BH4 has a drug license in Taiwan, China and is compliant, but not in mainland China, so it is not compliant. Patients have to take many risks when buying generic drugs, such as buying fake drugs.
Patients who purchase generic drugs from Taiwan, China need to convert the price into the US dollar exchange rate before transferring the purchase, and the price of the drug also fluctuates with the fluctuation of the exchange rate . Zhang Ruting now wants to buy a generic drug from Taiwan, and needs to transfer money to a bank's personal corporate account. "It's more troublesome, and the bank's people are not willing to help us do this because they don't seem to be very consistent." But this is a "life-saving drug" for them and they can only take risks.
Under the influence of the epidemic, drugs are often detained at customs and there is no guarantee. This is also the reason why Lu Wen from Beijing would rather run around than give his children a generic drug.
Zhang Ruting is also worried about the efficacy stability and side effects of generic drugs. She said that it was not that she didn't want her children to eat "respect", but that she couldn't afford it without policy subsidies, so she chose imitations from Taiwan.
now doesn’t even have this opportunity to hesitate, and “Kuowang” is about to withdraw from the Chinese market.
There are about 600-700 hyperphenylalanineemia patients in Guangdong Province, of which about 200 malignant patients need to take medication. Zhang Ruting introduced that among the more than 200 patients in , less than 10% of them can really afford "Kuai" and "relatives and friends in some regions such as Beijing, , Qingdao, , and Sichuan eat more "Kuai" because they have subsidy policies." Even if you live in these areas with policy subsidies, "outsiders" cannot enjoy it because these policies will limit the birthplace, household registration, etc. of the drug buyer.
Patients who bought medicine by pills were waiting for the medicine
html In June, "Kewang"'s domestic market agent, Baiao Wanli (Shanghai) Biomedical Technology Consulting Co., Ltd. (hereinafter referred to as "Bao Wanli"), communicated with National Health Commission and local municipal health commissions, "no longer re-registration for 'Kewang' imported drug registration certificate ."
communication mentioned that this drug was initially approved by National Medical Products Administration on September 25, 2010, and then re-registered. The latest re-registration was approved in 2018, with a limited period until March 21, 2023.
Bai Ao Wanli file mentioned that based on internal evaluation, it is planned to no longer re-register this product.
"Finance" reporter contacted Baimalin Pharmaceutical staff and asked whether "Kewang" was delisted from China. The staff replied: "It is not very certain at the moment, but it is a plan."
The other party said that this is not because of whether the domestic market is attractive, but that generic drugs for treating BH4 diseases have been launched abroad, and patients who used "Kewang" have lost a large number of losses in the market. From an overall perspective, Baimalin Pharmaceutical believes that it is not worth it to continue to retain it.
This is also an economic account that pharmaceutical companies should consider. "If the number of patients taking the medicine can reach 1,000, we may have a certain profit." A relevant person in charge of a pharmaceutical company that produces a generic drug for a rare disease once said.
There are even rare diseases pharmaceutical companies that have "retreat" about whether to promote drugs into the national medical insurance. "We are also observing the rare disease drugs that are included in medical insurance. The dosage has not increased much. We have to consider the economic costs caused by a significant price reduction." A relevant person in charge of a pharmaceutical company admitted.
Of course, it is of commercial value to develop drugs for rare diseases. Many foreign pharmaceutical companies have made breakthroughs from the use of rare diseases, and then expanded their indications to other treatment areas. Because the identity of "orphan drug" can not only shorten the drug's market time, but also have a relatively high market success rate.
Through statistics on new drugs launched in the past decade, the success rate of "orphan drugs" development is much higher than that of ordinary drugs, about three times that of ordinary drugs. Making all parties profitable is also the basic logic for pharmaceutical companies to develop rare disease drugs, submit approval, pricing and sales.
generic drugs are hard to buy, and the original drug is "scientific" and there is another drug that is difficult to find. Patients who urgently need drugs cannot afford to wait. The "Work Plan for Temporary Import of Clinical Urgent Drugs" is a document released by the official website of the National Health Commission and the Drug Administration on June 29. It is suitable for small amounts of drugs that have been launched overseas in clinically urgently needing in clinically urgently needed by the National Health Commission and the Drug Administration on June 2.
This has also become the safest way to choose from for this patient group at present.
China-Japan Friendship Hospital is the leading import institution of "Scientific Victory". "Finance" reporter learned from the pharmacy of Beijing Sino-Japanese Friendship Hospital that drugs are being cleared and are expected to be implemented in Sino-Japanese Friendship Hospital by the end of October, but the staff said they were "unsure" about how many bottles of drugs can be obtained.
This is undoubtedly good news for patients in Beijing. Learning that the child's medicine has finally come to an end in the short term, Lu Wen was very grateful. "The Beijing Health Commission is the first to write a document to the National Health Commission in writing to submit instructions, and the Chinese and Japanese pharmacies have been trying to find ways to get inventory."
But what about patients in other regions?
Zhang Ruting told the "Finance" reporter that due to the distance too far, the restrictions on household registration conditions, and the characteristics of the drugs themselves being susceptible to oxidation, "even if there are medicines in Beijing, we will not buy them." "Kewang" used to be sold as a pill in Guangzhou Pharmacy and Shenzhen Hospital. The price of a pill in Guangzhou is about 293.83 yuan, and one pill in Shenzhen is about 249 yuan.
Zhang Ruting learned from her patients that some patients only register to buy more than a dozen pills each time, and her own child has only taken two pills of original pills since she was a child. She felt confused about the future and didn't know when she would end her waiting for medicine.
