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Media attention | Save 20000 yuan a year, and make careful budget for patients with rare diseases

  • Categories:Company News
  • Author:Finance and Economics Great Health, written by Ling Xin
  • Origin:
  • Time of issue:2022-09-24
  • Views:0

(Summary description)Seeing a report on a rare disease published by Caijing, Zhang Xiao immediately added a WeChat message from Caijing reporter to make the media pay attention to his disease, mucopolysaccharide storage disease. This is a rare disease. Zhang Xiao has been diagnosed for more than 20 years.

Media attention | Save 20000 yuan a year, and make careful budget for patients with rare diseases

(Summary description)Seeing a report on a rare disease published by Caijing, Zhang Xiao immediately added a WeChat message from Caijing reporter to make the media pay attention to his disease, mucopolysaccharide storage disease. This is a rare disease. Zhang Xiao has been diagnosed for more than 20 years.

  • Categories:Company News
  • Author:Finance and Economics Great Health, written by Ling Xin
  • Origin:
  • Time of issue:2022-09-24
  • Views:0

Since June 2020, when the specific drug laronidase for mucopolysaccharide storage syndrome type I was approved in China, Zhang Xiao started to call for the drug to be included in medical insurance. However, by August 23, 2022, she had not used this drug.

Gui, in the final analysis, is just one word.

Zhang Xiao calculated a bill. According to the current selling price of Laronidase, an adult like her weighing 41 kg would have the lowest annual treatment cost of 3.79 million yuan, and would need lifelong medication. The retail price of the drug in China is 9114 yuan/bottle. It is recommended to use the drug once a week. The dosage is calculated according to the patient's weight, and one bottle is added for every 5 kg of weight increase. In all, she needs to use at least 416 bottles a year.

Zhang Xiao, 29, suffered from mucopolysaccharide storage syndrome type I, which can cause systemic symptoms such as elevated intraocular pressure, otitis media, muscle stiffness and pain. She has now lost the ability to walk independently, and her daily life is sitting or standing against the wall.

Zhang Xiao hopes that the medical insurance can include this medicine in the payment scope, so that she can have the opportunity to use the medicine first and "maintain her life". In the adjustment of the national medical insurance catalogue in 2021, Nostradamus sodium injection, which was once used to treat spinal muscular atrophy for 700000 yuan per injection, was included in the medical insurance with more than 30000 yuan after medical insurance negotiations.

However, things are not as simple as Zhang Xiao thought. Medical insurance is the basic insurance. For some drugs for special rare diseases, which are particularly expensive, "they are not included in the payment scope of basic medical insurance because they are far beyond the funds and patients' affordability." At the end of 2020, the State Medical Insurance Bureau replied to the proposal of "including the use of drugs for rare diseases in the medical insurance catalogue" put forward by the National People's Congress.

What's more, R&D costs are high and patients are few. Even if drugs for rare diseases are included in medical insurance, they are also "expensive". The key is that patients often need lifelong medication. To further reduce the total cost of medication is the common appeal of patients with rare diseases, even if their drugs have been included in the medical insurance.

first-tier cities

Only then can we diagnose and treat rare diseases

Compared with many rare patients without available drugs, Su Jian (not his real name) is the lucky one.

When Su Jian was in his 30s, he had a stable job, a good income, and lived in a city with a high medical level, such as Suzhou. Two years after he was diagnosed with multiple sclerosis, he caught up with the original drug terifluoramide, which was used to treat the disease. It was listed in China in 2018 and included in the medical insurance list in 2020.

The better news is that a cheaper domestic generic drug will be launched at the beginning of August 2022. "It is important to calculate that the annual self paid drug cost will be 20000 yuan cheaper." Su Jian has settled the account.

To say "lucky" is only relative. Before the first diagnosis in 2016, Su Jian went back and forth among the major departments of the hospital, saw ophthalmology, orthopaedics, neurology, and also did an EMG. In the end, he checked his own papers and consulted with insiders to find a doctor who specializes in neuroimmune diseases, and was diagnosed with multiple sclerosis.

This is an autoimmune disease of unknown etiology, which will damage the central nervous system of people. Its symptoms include numbness of hands and feet, instability of gait, impaired vision, etc. In severe cases, it will paralyze. Many patients are young people in their twenties and thirties. Most of Su Jian's patients are young girls.

After the diagnosis, Su Jian found that only one type of interferon could alleviate the disease, and only several cities in Beijing and Chengdu were available. The source of goods was unstable, and refrigeration was also needed. "All at their own expense, tens of thousands of yuan per month".

Su Jian can neither buy nor use such medicine.

After passing the acute attack period in the hospital, Su Jian started to take an immunosuppressive drug "tacrolimus", which is mainly aimed at the rejection of allografts, under the recommendation of the doctor. By 2018, "I obviously felt that tacrolimus was useless to me", and Su Jian, who was hospitalized after a relapse, was waiting for terifluoramine to be listed. In August of that year, 12900 yuan/box of the original drug was officially sold. He was not willing to pay for it.

 Su Jian's second "luck" appeared in 2020. In January that year, terifluoramine was officially included in the national medical insurance directory and soon landed in Suzhou. In addition to the benefit of the people and the charity drug plan, Su Jian pays more than 40000 yuan per year, which is more than 100000 yuan lower than his own expense.

Su Jian recalled to the reporter of Caijing that throughout Jiangsu Province, Suzhou and Nantong were the first to incorporate terifluoramine into medical insurance, and patients in other cities were envious. A girl from northern Jiangsu even found a job in Suzhou remotely in order to use medical insurance.

Li Tao, Deputy Director of the National Medical Security Bureau, once introduced at the 2021 China Rare Diseases Conference that in 2021, there were 7 rare diseases drugs successfully negotiated, with an average price drop of 65%.

"Some places give more than 30000 yuan of reimbursement per year to patients with multiple sclerosis. After calculation, 80000 yuan will be paid at one's own expense, and many people give up." As far as Su Jian knows, even though the medical insurance policy has been implemented in more cities in different forms, many patients still have no money to stick to long-term medication. Some people have gradually disappeared from the group of sick friends.

Even in Suzhou, in the past many years, only one pharmacy can buy terifluoramine, which is the economic trade-off of many parties. Now, hospital drug sales are "zero mark up", and drug sales do not bring income. The number of patients with rare diseases is small. Adding one drug will bring few patients, and the management cost will increase. In addition, the high unit price of drugs for rare diseases can easily increase the "proportion of drugs" of hospitals and lower the hospital's score in the year-end assessment of health departments (see the related report in the "Hard to Buy Heavily Priced Drugs: Economic Game among Hospitals, Patients, Pharmaceutical Enterprises and Medical Insurance" on August 11).

According to the information on the website of the Chinese government, up to now, more than 60 rare disease drugs have been approved for marketing in China, of which more than 40 have entered the national medical insurance directory, involving 25 diseases.

The patients with rare diseases can't think about that much. They are always calculating the medicine fees and examination fees, which are calculated in 10000 yuan every year. Zhang Xiao spent 50000 yuan on symptomatic treatment every year because he didn't use effective drugs.

If Zhang Xiao wants to live on his own, he needs to continue treatment and rehabilitation training. "With the rehabilitation costs, the annual cost is more than 100000 yuan, which is not included in the rent in Beijing.".

Before October 2021, Zhang Xiao insisted on working in Beijing, calling attention to the accessibility of medication for about 100 known patients with mucopolysaccharide storage syndrome type I.

Only the super large tertiary hospitals in the first tier cities have the ability to diagnose and symptomatically treat patients with rare diseases, can they introduce the specific drugs they need. "Ordinary hospitals are relatively deficient in the diagnosis and medication of rare diseases, which limits the diagnosis and treatment of patients with rare diseases in China." Yang Xuehua, sales director of Shengshi Tyco Biomedical Technology Co., Ltd. (hereinafter referred to as "Shengshi Tyco"), told Caijing.

Only through research and development can drug prices be expected to fall

On August 8, 2022, the domestic imitation of terifluoramine will be put on the market, 30 tablets in a box, and the drug price will be 6896 yuan. The original research drug produced by Sanofi is 7896 yuan for a box of 28 pills, 1000 yuan more expensive than a box made in China.

"There must be a discount compared with the original drug". When Yang Xuehua and the company's management sat down to price their own terifluoramine, they also suggested adding two more pills to each box, so that patients only need to prepare one box of drugs every month.

Su Jian is very concerned about the two extra pills given in each box. After careful calculation, he can buy one box less medicine every year. The self paid portion of generic drugs is about 65% of the original drug. "At my age, there are old people and young people, and the mortgage is still 15000 yuan a month. Domestic drugs save more than 20000 yuan a year, and children will have the money for tuition".

Domestic specific fluoroamines have been on the market for two weeks, and they are being publicized on the provincial medical insurance procurement platforms, and have just begun to be sold in some "dual channel" pharmacies. "Double channel" is a policy issued by the National Medical Insurance Bureau and the Health Commission, which allows patients to purchase drugs in the medical insurance catalog at designated hospitals and designated pharmacies at the same time and apply for reimbursement.

However, the main reason why drugs for rare diseases are scarce is that the return on investment is not sweet enough. Yu Qiang, founder of Shengshi Tyco, said frankly that as a biopharmaceutical company, the company needs to have products to launch into the market as soon as possible. In 2018, the State Food and Drug Administration gave priority to the review of the use of rare diseases, including terifluoramine, and a series of policies, which prompted them to select this drug in the research and development pipeline.

According to Frost Sullivan's calculation, by 2021, there will be only 49700 multiple sclerosis patients in China, and even fewer people know, are willing to use and can afford terifluoramine. According to Yang Xuehua, in the First Affiliated Hospital of Suzhou University, only five or six patients may need medication within a month, mainly through the "dual channel" pharmacy.

On August 8, Shengshi Tyco sold terifluoramine at the head office of a large pharmacy in Suzhou, as well as hospitals in Sichuan, Fujian and other places and "dual channel" pharmacies. Those who come here to buy are those who have long been concerned about and waiting for the listing of domestic drugs.

"If the number of patients taking drugs reaches 1000, we may have some profits." Yang Xuehua estimated that it would take at least one year. At the initial stage of the product launch, he would not pursue profits excessively.

Yang Xuehua pays more attention to the establishment of sales channels and the characteristics of ensuring that patients with rare diseases need lifelong medication.

There are more than 2.3 million multiple sclerosis patients in the world, and the incidence rate is about eight times that of China, which may mean that there are still patients in China who have not been found or diagnosed. "The reason is still limited by the diagnosis of patients with rare diseases. At present, it is still concentrated in the super top three hospitals in the first tier cities. This situation may change in the future." Yang Xuehua said.

In the second quarter of 2022, the sales volume of Sanofi's original terifluoramine was 526 million euros.

Not only Sanofi, but also multinational pharmaceutical enterprises such as Novartis, Pfizer and GlaxoSmithKline have been successful in the development of rare disease drugs. Especially Gleevec, when pharmaceutical enterprises developed this drug, the total number of patients was less than 10000. The development of this drug is very fast. It has been less than three years from clinical use to marketing, and it has shown good efficacy in a very short time after marketing. This drug can make the five-year survival rate of leukemia patients reach 90%.

Such a drug originally used to treat leukemia has finally turned into a drug for treating chronic diseases, and later more clinical indications have been developed. Therefore, the sales volume of this drug in 2015 before the expiry of the patent was close to 4.8 billion dollars.

Zheng Weiyi, founder of China Orphan Drug Innovation Alliance and professor of the School of Pharmacy of Nanjing University of Technology, once wrote an article to Caijing that rare diseases are not rare, and about 5% to 10% of people have some form of rare diseases. At present, more than 7000 cases have been diagnosed in the world, and the number is still increasing.

It is of great commercial value to develop drugs for rare diseases. Many foreign pharmaceutical factories have made breakthroughs in the distribution of drugs for rare diseases, and then expanded the indications to other therapeutic fields. Because the identity of "orphan drug" can not only shorten the time of drug listing, but also has a high success rate. According to the statistics on the marketing of new drugs in the past ten years, the success rate of the development of "orphan drugs" is far higher than that of ordinary drugs, about three times more than that of ordinary drugs.

In China, previous generic drugs for rare diseases, like new drugs, need to go through a series of clinical trials, and the cost may be tens of millions of yuan. After the introduction of relevant policies in 2018, foreign clinical data can be directly used for drug use for rare diseases, and only bioequivalence tests (equivalent to drug consistency evaluation) are required.

Under this policy, the R&D of several rare disease drugs has been launched one after another. For example, the late solid tumor research and development drug FCN-159 of Fosun Pharmaceuticals, and the anti melanoma drug HL-085 of Shanghai Kezhou Drug Research and Development Co., Ltd. are simultaneously conducting clinical research on related rare diseases. This may not only add an indication to the drugs under research, but also is expected to speed up the listing process, which is a real benefit for enterprises.

The State Medical Insurance Bureau recently issued a document stating that, before June 30, 2022, the drugs for treating rare diseases approved by the State Food and Drug Administration for marketing can be included in the adjustment scope of the national medical insurance catalog in 2022. This also means that more rare disease drugs are expected to be included in the medical insurance.

Making all parties profitable is the basic logic for pharmaceutical enterprises to develop rare disease drugs, submit for approval, price and sell them.

About Shengshi Tyco

Founded in Suzhou Industrial Park in 2010, Shengshi Tyco is committed to the research, development and industrialization of small molecule innovative drugs for breakthrough therapy. Relying on the integrated drug R&D technology platform and diversified business vision, the company has built a rich pipeline of innovative drugs, covering many disease fields such as hypoglycemic, anti-cancer and rare diseases. In addition to terifluoramine, an original new hypoglycemic drug, Shenggliptin Phosphate, has entered the final stage of clinical phase III, which is expected to fill the blank of domestic original DPP-4 inhibitors; Three anti-cancer drugs under research have entered the clinical stage, and the FGFR/VEGFR double target inhibitor CGT-6321 has been carried out in the phase I clinical trial in advanced solid tumors, which is expected to become a potential first in class drug; CXCR4 antagonist CGT-1881 is used for hematopoietic stem cell mobilization. It is the only original small molecule oral CXCR4 antagonist in China, and the administration is more convenient; CGT-9475, a new generation of ALK inhibitor, is expected to solve several major clinical pain points in the treatment of lung cancer, such as drug resistance and brain metastasis. The latter two products have both obtained the clinical license of the US FDA, which is a solid step for the company to launch innovative drugs.

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