● New drug research and development has always been considered a high-risk and high-yield activity. Targeted drugs for rare diseases are more expensive. In addition to the small population of rare disease patients, it is very likely that losses will occur. Pharmaceutical companies are often "daunted", so rare diseases Once the new drug with special effects comes out, it will basically be in a monopoly state, and the price will be higher
● For some rare disease drugs with definite therapeutic effects, it can be considered to be included in the medical insurance gradually, but which rare disease treatment drugs can be included in the medical insurance scope need to be further evaluated and discussed
● The burden of high-priced rare disease drugs should not only rely on medical insurance, but should also explore the "multi-party co-payment" medical insurance model involving the government, public welfare institutions, enterprises and individual patients. However, the current social support and help system in China is not yet perfect. Allow some social forces to participate, and the state can also encourage enterprises to subsidize through tax adjustments and other methods
□ Our reporter Zhao Li
A small 5ml injection costs 700,000 yuan. For children with SMA (Spinal Muscular Atrophy), every drop of Noxinat is as precious as gold. It means that the child has hope of survival, but it also means that a family may go bankrupt.
Public information shows that SMA is a rare chromosomal recessive genetic disease characterized by the loss of motor neurons in the spinal cord and lower brain stem, leading to severe and progressive muscle atrophy and weakness. The incidence in newborns is about One thousandth to one thousandth. SMA is divided into 4 types according to the age of onset and clinical course of patients, from mild to severe. The most severe type usually develops within 6 months. Without treatment, most children cannot survive to two years of age.
Not long ago, a mother of a child with SMA in Guangdong submitted an application for disclosure of information to the State Food and Drug Administration, hoping to learn about the purchasing method of Noxinagen Sodium Injection and the basis for domestic pricing.
This incident has aroused public concern about the price of 700,000 yuan per injection of Nosinagen Sodium Injection. Whether it should be included in medical insurance, whether it can be "co-paid", whether to encourage the development of new drugs or to make special drugs is the main point of dispute. .
Highly priced new drugs
How to compress profit margins
Noxinassen Sodium Injection is the world's first SMA precision targeted therapy drug. It was developed by Bojian Company. It was approved for the first time in the United States on December 23, 2016, and was successively approved for use in the European Union, Brazil, Japan, South Korea, and Canada. For the treatment of SMA. On February 22, 2019, Noxinassen Sodium Injection was officially approved by the National Medical Products Administration, becoming China's first drug to treat SMA.
According to the China Times, at present, children with SMA in China can only hope to survive if they use this expensive drug. In the pre-symptomatic dosing experiment, 100% of the children can sit alone, 92% can assist walking, and 88% can walk independently.
A reporter from the "Rule of Law" noted that the high price of Nosinagen Sodium Injection has been widely discussed.
At present, the domestic price of Noxinagen Sodium Injection is 697,000 yuan per bottle. It is a completely self-funded drug. Patients need to inject 6 bottles in the first year, and then 1 injection every 4 months, every year.
A neurologist said that in terms of unit price, my country’s public price is already the lowest level in the world. In the United States, according to the previous public statement of Bojian Biotechnology, the single price of Nosina Sodium is US$125,000 (equivalent to approximately RMB 870,000), which is about 20% higher than the single price of nearly 700,000 RMB in China. %. In Australia, the government purchases the drug at a price of 110,000 Australian dollars (equivalent to approximately 550,000 yuan), which is also very high.
According to Liu Xin, director of the Medical Law and Ethics Research Center of China University of Political Science and Law, the Chinese government has made many efforts and reforms in cutting intermediate links and reducing drug costs, such as proposing and implementing the "two-vote system."
"Two invoices" refer to the fact that the pharmaceutical production company issues an invoice to the circulation company, and the circulation company issues an invoice to the medical institution. The purpose is to reduce the circulation of drugs, make the intermediate price increase transparent, and further reduce the false high prices of drugs and reduce the burden on the people. . From the pilot in April 2016 to the further deepening in November 2016, and then the policy was finally finalized on January 9, 2017, eight ministries including the former National Health and Family Planning Commission and the former State Food and Drug Administration jointly issued the Implementation Opinions on the Implementation of the "Two Invoice System" in Institutional Drug Purchase (Trial).
On January 17, 2020, the National Medical Insurance Administration issued the "Notice of the State Organizing the Centralized Procurement and Use of Drugs Pilot Program", which means that the "4+7 volume procurement" policy is officially implemented.
"The profit margin of medicines has been greatly compressed, and the prices of many medicines have dropped significantly." Liu Xin said.
According to Zheng Xueqian, executive vice chairman and secretary general of the Medical Legal Professional Committee of the Chinese Hospital Association, the state has been working hard to reduce the tariffs on imported drugs to reduce patient expenditures. For example, according to the relevant provisions of the "Regulations on Import and Export Tariffs of the People's Republic of China", since 2018 From May 1, 2005, the import tariffs on all common drugs including anti-cancer drugs, alkaloid drugs with anti-cancer effects and actually imported Chinese patent medicines will be reduced to zero by means of temporary tariff rates. However, high-priced imported drugs for rare diseases, such as Noxinagen Sodium Injection, can not greatly reduce the burden on patients' families by reducing tariffs.
A professional who studies the domestic medical insurance system said that the price of a drug is determined by the enterprise itself during the listing stage. After the National Development and Reform Commission abolished the maximum retail price of drugs in 2014, if drugs are not included in medical insurance, pricing decisions are basically market-oriented.
The industry generally believes that the development of new drugs has always been regarded as a high-risk and high-yield activity. Targeted drugs for rare diseases are more expensive. In addition to the small group of rare disease patients, losses are very likely to occur, and pharmaceutical companies are often "daunted." . Therefore, once specific new drugs for rare diseases come out, they will basically be in a monopolistic state, and the prices will be higher.
Can it be included in the medical insurance system
It is still unknown at present
Whether or not to include Noxinasheng Sodium Injection into medical insurance is also the focus of public opinion.
According to Bojian’s statement, as of June 30, 2020, Noxinassen Sodium Injection has been approved in 50 countries and regions around the world, and has been reimbursed in more than 40 countries and regions.
The public information on the Australian Drug Benefits Program website shows that Nocinasheng Sodium Injection has been included in the Drug Benefits Program. The single government purchase price of the drug is 110,000 Australian dollars, and the patient’s out-of-pocket expenses are 41 Australian dollars, which is equivalent to RMB 206.
Liu Xin believes that Australia will include SMA in the medical insurance system, but this is only a case of disease. Australia does not and cannot have sufficient financial support to include all rare diseases in the medical insurance coverage. "Even countries like the United Kingdom that are leading the implementation of a free medical system do not include all drugs in the free medical drug list. Drugs not in the list still need to be paid by the patient. The economic level of all countries has not reached the level of free medical care. The degree of medical treatment."
In fact, the National Medical Insurance Bureau is already considering the inclusion of Noxinagen Sodium Injection in medical insurance.
A staff member of the Letters and Visits Office of the National Medical Insurance Bureau said in an interview with the media that since the launch of Nosinagen Sodium Injection in China in 2019, it has been included in the medical insurance negotiation agenda. The country hopes to negotiate with relevant pharmaceutical companies to lower the price of the drug. Then meet the needs of SMA patients. "The state started to negotiate with pharmaceutical companies last year, and the expert group will study the pricing. The specific pricing is not clear. However, the inclusion of medical insurance has not been discussed. Because the price of the drug cannot be lowered, there has been no way to enter the medical insurance catalog."
According to the "Report on the Access to Drugs for Rare Diseases in China (2019)" jointly issued by the Center for Rare Disease Development and Ai Kunwei China, there are only 55 drugs that are clearly registered as rare disease indications in China, involving only 31 rare diseases. Among these 55 kinds of drugs, only 29 kinds of drugs are included in the national basic medical insurance, work injury insurance and maternity insurance drug catalog, involving 18 kinds of rare diseases. According to the "First List of Rare Diseases" issued by five ministries and commissions including the National Health Commission and the Ministry of Science and Technology on May 22, 2018, 121 rare diseases are involved. There are more than 7,000 rare diseases recognized internationally.
On August 5, Bojian Company replied to the media that the company is actively preparing to submit the information of Nocinagen Sodium to the National Medical Insurance Bureau. If the drug passes the expert review, it will enter the medical insurance negotiation link. If it is determined to be included in the 2020 National Medical Insurance Drug List , Patients will be expected to buy at a lower price.
However, according to the analysis of many interviewed experts, the inclusion of rare disease drugs into medical insurance is a very complicated process. Due to the uncertainty in the number of patients, condition, and drug efficacy of rare diseases, and the high price of rare disease drugs, the benefits are small. If covered by the medical insurance fund, whether it will impose a heavier burden on the medical insurance fund, whether it is unfair to others, etc., are issues that need to be considered in reality. Therefore, it is still unknown whether Noxinasheng Sodium Injection will be included in medical insurance.
On July 31, the National Medical Insurance Bureau deliberated and promulgated the "Interim Measures for the Administration of Medications for Basic Medical Insurance", in which Article 4 pointed out that the functional positioning of "basic protection" should be adhered to, and the use of medication should be guaranteed. The level is compatible with the basic medical insurance fund and the affordability of the insured.
In Liu Xin’s view, a country’s medical insurance system is compatible with the country’s national strength and economic development. China’s current basic medical insurance system has limited medical funds. "If it is similar to Nocina Sodium Injection, etc. All of the high-priced drugs are included in medical insurance, which will affect the basic medical insurance for most people."
"The cost of such imported drugs cannot be directly reduced through negotiations between pharmaceutical companies by reducing intermediate links. The manufacturer’s original price is very high. Even through negotiations, it is difficult to reduce the drug price to a very low level. Level." Liu Xin said.
However, Wang Yue, deputy dean of the School of Medical Humanities of Peking University, believes that some rare disease drugs with definite therapeutic effects can be considered gradually included in medical insurance. However, which rare disease treatment drugs can be included in the medical insurance scope needs further evaluation and discussion.
Medical insurance can't fully cover
Explore the multi-party co-payment model
Although my country has not yet included Nosinagen Sodium Injection in the medical insurance list, in many provinces and cities, some public welfare foundations have launched assistance projects to reduce the burden on SMA patients.
In November 2019, Zhezhe, a child with SMA-Ⅱ, was injected with Nocinagen sodium in the Children's Hospital of Soochow University, becoming the first child in Jiangsu Province to receive the drug. This assistance project for SMA patients accepted by Zhezhe, the first 4 injections are “one get three free”, that is, the patient only needs to pay 700,000 yuan for the first injection; in the subsequent maintenance phase, one injection every 4 months , "One shot gets one shot" is equivalent to 350,000 yuan per shot.
According to Bojian Company, the above-mentioned project is an SMA patient assistance project launched by the China Primary Health Care Foundation on May 31, 2019. The first-year treatment cost of the patients receiving assistance is about 1.4 million yuan, which can save about two-thirds compared with full self-finance; after that, the annual treatment cost is about 1.05 million yuan, which can save about half compared with full self-finance. Up to now, more than 80 SMA patients across the country have received medication with the help of this assistance project.
Public information shows that the China Primary Health Care Foundation was established in 1996 with the approval of the Ministry of Civil Affairs. It is a national public fundraising foundation sponsored by the China Peasants and Workers Democratic Party and managed by the National Health Commission. According to its official website, the foundation has cooperated with Bojian to prepare patient assistance projects to help SMA patients reduce the payment burden and improve the accessibility of treatment. It has been launched in 14 provinces and cities across the country, and the first phase covers 25 central hospitals.
In addition to receiving assistance from the China Primary Health Care Foundation, Zhezhe also received assistance from the Jiangsu Charity Federation. The injection cost nearly 700,000 yuan, which is about 150,000 yuan.
According to industry insiders, the burden of high-priced rare disease drugs should not only rely on medical insurance. The society should also explore the "multi-party co-payment" medical insurance model with the participation of the government, public welfare institutions, enterprises and individual patients. This will be the establishment of rare disease medical care. The only way to guarantee the system.
For example, the Pain Challenge Foundation, a public welfare foundation that focuses on supporting groups of rare diseases, also jointly established rare disease special aid funds in Shanxi and Zhejiang provinces this year. The fund will serve as a provincial-level social charity force to participate in the supplementary attempt of the rare disease medical security system, support Gaucher disease, Pompe disease and other patient groups in the national rare disease catalog, and promote the continuous and effective treatment of patients.
Wang Yue also believes that the "multi-party co-payment" medical security model is indeed the only way to establish a rare disease medical security system, but the current social support and help system in China is not yet perfect. Some social forces should be allowed to participate, and the state can also adjust through taxation. Encourage corporate funding.
In Zheng Xueqian's view, it is necessary to help rare disease patients and their families through multiple channels: one is social donations, which can call for the establishment and supervision of rare disease foundations, and raise rare disease funds through social donations and corporate funding; second, the state Relief, outside the scope of medical insurance, can provide hardship subsidies to patients with rare diseases and their families; third, individuals purchase commercial insurance.
Encourage domestic drug development
Special drug imitation follow-up
In 2019, Zhejiang Province established a mechanism for guaranteeing drugs for rare diseases. According to local policies, the annual self-finance limit for patients with rare diseases in Zhejiang will not exceed 100,000 yuan.
Liu Xin explained that there are two prerequisites for this mechanism. One is that the drug must be available, and the other is that the drug must be negotiated and included in the scope of the Zhejiang Fund. "Otherwise, it will not be possible to achieve the annual self-finance limit for rare disease patients not exceeding 100,000 yuan. ".
Can this mechanism be replicated and promoted in other regions? A staff member of the Letters and Visits Office of the National Medical Insurance Bureau said that it is still difficult to achieve at the national level.
"This is related to the economic level of various regions, and regional policies are not necessarily suitable for national promotion. The actual conditions of economically underdeveloped regions must be taken into consideration." Wang Yue said.
According to the "China Rare Disease Medical Insurance City Report 2020" jointly released by the Center for Rare Diseases and Ai Kunwei Management Consulting Team in May this year, there are seven modes of supplementary protection for rare diseases in addition to medical insurance in China: special fund model , Major illness negotiation model, financial contribution model, policy-based commercial insurance model, medical assistance model, medical insurance sporadic supplement model, independent declaration model.
Li Yangyang, the lead researcher of the report, said that the rare disease protection plan that all walks of life are looking forward to is the implementation of the "N+1" model. "1" refers to the reimbursement of rare diseases by basic medical insurance, and "N" refers to the supplementary assistance policy jointly cooperated by the government, pharmaceutical companies, insurance companies, charity organizations and other parties. At present, these policies are being carried out experimentally in various parts of the country. In the future, it is expected to realize a multi-pilot and multi-level rare disease treatment guarantee network at the national level.
A reporter from the Rule of Law Daily noted that Article 60 of the Basic Medical and Health Promotion Law states that the state shall establish and improve a clinical demand-oriented drug review and approval system to support urgently needed drugs, drugs for children, and the prevention and treatment of rare diseases and major diseases. The development and production of medicines for diseases and other drugs meet the needs of disease prevention and treatment.
Liu Xin also mentioned to a reporter from the Rule of Law Daily that the newly revised Drug Administration Law clearly stipulates in the general provisions that the state encourages research and creation of new drugs, adds and improves more than 10 clauses, and adds a number of institutional measures.
"This is to encourage innovation and speed up the launch of new drugs, releasing a series of institutional dividends. This includes focusing on supporting clinical value-oriented drug innovation with clear curative effects on human diseases. Encouraging new treatment mechanisms that are seriously life-threatening. The development of new drugs for severe diseases, rare diseases and children’s drugs." Liu Xin said.
Wang Yue mentioned that the legal system for the treatment of rare diseases should be improved, and relevant regulations and action regulations should be promulgated from the State Council level. The government has the responsibility to help these rare disease patients and their families obtain effective information about relevant drugs and treatments on a global scale. Reduce information asymmetry.
He also suggested that an office for rare diseases could be established. On the one hand, a green channel for rare diseases could be set up, allowing family members to "buy" some drugs that have been marketed in Europe and the United States but have not yet been approved for marketing in China, and the policies should be appropriately relaxed; on the other hand, drugs Supervision and management departments have the responsibility to understand the global rare disease drugs and the current market situation in China, which will help speed up the approval and entry process of rare disease drugs.
In addition, Zheng Xueqian suggested that the two methods should go hand in hand to encourage the research and development of domestic drugs and the imitation of special drugs.
Regarding the above two methods, Wang Yue believes that in terms of China’s current R&D capabilities, it is more efficient to imitate rare disease treatment drugs, but legal issues need to be considered. “Maybe it may be possible to consider the adoption of the “TRIPS Agreement Amendment” by the WTO in 2005. The strong imitation system in the Protocol is used to encourage domestic companies to imitate, in order to achieve the goal of drastically reducing drug prices, and even force the original manufacturer to reduce prices."
Cartography/Gao Yue