April 17 is the 34th World Hemophilia Day.

There are coagulation factors in the blood of normal people, and when there is a wound, the blood will coagulate quickly.

But for hemophiliacs, because there are not enough coagulation factors in the blood, the patient is as fragile as glass, and a little bump that is insignificant to ordinary people may cause the hemophiliac to bleed continuously. Therefore, it has a common name - "glass man".

　　The incidence of the disease is about 1 in 10,000, and every 10,000 newborns will have one person with hemophilia. It is estimated that there are about 140,000 people with hemophilia in China, most of whom are male.

At present, there is no cure for the disease, and patients need lifelong medication.

　　The Beginning of "Glass Life"

　　Xiaoshu (pseudonym), who lives in Guangdong, is a 12-year-old patient with hemophilia A antibody.

Shortly after birth, Xiaoshu was diagnosed with hemophilia.

Immediately after being diagnosed, there is no local condition to inject coagulation factors, and only plasma can be transfused.

Later, with the coagulation factor, one injection can stop the bleeding.

But this situation did not last long.

In 2018, Xiaoshu accidentally fell and his knee kept bleeding.

At that time, blood clotting factors alone could not stop the bleeding.

The doctor's diagnosis is that Xiaoshu has produced antibodies.

This means that the clotting factor is losing its effect on him.

　　There are not a few patients with hemophilia A antibodies like Xiaoshu.

According to the different coagulation factors lacking, hemophilia is divided into type A (deficiency of coagulation factor VIII), type B and type C. Among them, patients with hemophilia type A antibodies are "rare diseases among rare diseases".

For such patients, the previously applicable coagulation factors can no longer play a role in rapid coagulation due to the production of antibodies in the body.

　　But it's not "drug-free" either.

In the middle of last year, Xiaoshu began to try Roche's innovative drug Emicizumab Injection (trade name "Shuyou Lile").

"Doctors have long suggested using this drug, but the price is too expensive." Xiaoshu's mother said that emicizumab is too expensive, and now it is only used in small doses (30mg/time).

Even a small dose costs nearly 400,000 yuan a year.

　　Dosage is linked to body weight,

　　The annual drug cost of antibody patients may exceed one million

　　Guan Tao, president of the Beijing Hemophilia Home Rare Disease Care Center (hereinafter referred to as "Hemophilia Home"), a non-profit organization, said that emicizumab is the first FDA-approved drug in recent years for the treatment of inhibitors containing factor VIII in the body. The new drug for hemophilia A is currently the only preventive and therapeutic drug approved for hemophilia A antibody patients in China.

"This is a drug that can give patients a 'healing sense'," Guan Tao said.

　　However, high drug prices have made it difficult for antibody patients to have access to drugs.

Guan Tao told reporters that, as a life-long drug, the dosage of Emicizumab is directly linked to the patient's weight.

He calculated the account: an adult hemophilia patient weighing 60 kilograms, the drug cost for a year is about 1.3 million yuan; even a lighter child patient, at least a year Need more than 600,000 yuan.

　　"This price is unbearable for the vast majority of patients." Guan Tao said that since the launch of Emicizumab in China for three years, the price has never been adjusted. "The high drug price has made antibody patients from The original 'no medicine can be cured' has helplessly changed to 'the medicine is difficult to reach'."

　　High-priced "life-saving drugs" lingered outside the medical insurance negotiation room for three consecutive years

　　In January this year, the new version of the National Medical Insurance Drug List was officially implemented, and the average price of 67 drugs was reduced by 61.7%.

"Also as a drug for rare diseases, the news that Nosinagen Sodium Injection was included in the medical insurance gave hemophilia patients hope, but it is still unknown when Emicizumab will be included in the medical insurance and achieve a substantial price reduction. Guan Tao said that Roche lingered outside the medical insurance negotiation room for three consecutive years.

　　The relevant departments of Roche Pharmaceuticals in China have responded.

They believe that price is indeed one of the important reasons that restrict many rare disease patients from obtaining urgently needed treatment, but the resistance of high-value drugs to the Internet and hospital admission, as well as the reimbursement problem after infusion in out-of-hospital pharmacies are all restricting patients. Access to medicines.

None of these jobs can be solved by one business, one organization, or one disease area.

　　From a policy perspective, in order to encourage the development of the pharmaceutical industry for rare diseases and reduce the cost of medication for patients, the Ministry of Finance, the General Administration of Customs, the State Administration of Taxation, and the Food and Drug Administration jointly issued the "VAT Policy on Rare Disease Drugs" on February 20, 2019 Notice (Cai Shui [2019] No. 24), which stipulates that from March 1, 2019, the import value-added tax rate levied on imported rare disease drugs will be reduced from the original 13% to 3%, and the tax reduction rate will be as high as 10%. .

In September of the same year, emicelizumab injection was included in the second batch of anti-cancer drugs and rare disease drugs subject to the VAT policy.

　　"Can the reduced 10% import-link VAT rate be reflected in the reduction of drug prices?" Guan Tao suggested that it is not easy for enterprises to develop innovative drugs, but he hopes that enterprises can also see the difficulties of patients. The preferential tax policies are truly enjoyed by patients.

　　/ industry advice /

　　Expand the limit of medical insurance and increase support for innovation at the policy level

　　Why is it difficult to reduce the price of innovative drugs for rare diseases?

　　An industry insider, Li Ming (pseudonym), told reporters that companies need to invest a lot of money to develop an innovative drug.

An innovative drug, after 10 to 15 years, can cost billions of dollars.

　　At the same time, the development of innovative drugs is extremely difficult.

"Maybe only one of the ten R&D products is really successful." And this one product that was successfully launched has to bear all the R&D expenses of the failed product, "the price will naturally be high."

　　In addition, for rare diseases, the number of people in this group itself is not large. In addition, the process of sinking drugs to some grassroots cities costs a lot, which is not economical from the perspective of enterprises.

　　From the perspective of medical insurance, as a payer of drugs, Zheng Jie, head of the expert group for the calculation of the National Medical Insurance Negotiation Drug Fund in 2021, once said that according to the limited payment scope, the current annual treatment cost of all drugs in the national medical insurance catalogue does not exceed 30 million.

　　"That is to say, the annual per capita expenditure cannot exceed 300,000 yuan in order to enter the scope of medical insurance reimbursement. But not all pharmaceutical companies are like Bojian, willing to reduce the price of Nosinasheng Sodium Injection by 95%." Li Ming revealed that he learned that , Some multinational pharmaceutical companies producing innovative drugs for rare diseases are planning to withdraw from the Chinese market.

The core problem is that if the price is reduced to the required price for medical insurance in China, it means that the price system in developed countries will be lost, and thus the global market will be lost.

Li Ming believes that rare diseases cannot be solved by a single company, and they need the support of pharmaceutical companies, academia, and the government.

　　In addition to pushing companies to cut prices, he suggested that, on the one hand, medical insurance can give appropriate preference to the payment of drugs for rare disease patients, and expand the limit on the basis of the average annual cost of 300,000 yuan for common patients to achieve fairness to the niche.

On the other hand, the state can increase support at the policy level to support original innovation.

For example, subsidy support is provided at the front end of enterprise research and development, and a "green channel" is opened for innovative drugs in the administrative approval process to shorten their time to market.

　　Guan Tao suggested that at the institutional level, encourage policies for commercial insurance and private charitable assistance; government departments, pharmaceutical companies, commercial insurance companies, private charitable organizations and individuals constitute the multi-party participants in the protection of drugs for rare diseases; medical insurance funds, government special projects Funds, social assistance funds, corporate individual donations, etc. are funded to jointly solve the problem of rare disease medical insurance and medication, and different participants jointly explore and improve the rare disease payment guarantee system through innovative payment models.

　　Chengdu Business Daily-Red Star News reporter Peng Xiangping