There is a new way to pay for this kind of disease

Recently, the report on strengthening the research and policy recommendations on drug use guarantee industry for patients with rare diseases in China (hereinafter referred to as the report) was released by the health finance research office of Fudan University School of management and aisekang (Shanghai) Health Consulting Co., Ltd For the first time, the report focuses on the problem of "difficult drug use" of rare disease groups in China, comprehensively analyzes the current situation of drug use accessibility of rare disease groups in China, and points out that the accessibility of "orphan drugs" is not only a medical problem, but also has a unique social attribute Attaching importance to rare disease groups is an indispensable part of building a harmonious society At the same time, the report pointed out that under the current situation of some "orphan drugs" supported by national and local medical insurance, the introduction of commercial insurance to improve the drug security system for rare diseases with innovative mechanisms provided a new idea ▍ rare diseases, which bring a fatal blow to the family, are rare, but rare diseases are enough to bring a fatal blow to an ordinary family As the new year's Day approaches, many families will take advantage of the "small and long vacation" to prepare a short trip, while Liu Lu, 35, has to take advantage of this rare holiday to take the 2-year-old children to Beijing by high-speed rail for further consultation At the age of 8 months, the children had high fever and red rash all over the body Then the joints began to be inflamed After several times of turnover, they were diagnosed as systemic juvenile idiopathic arthritis (sJIA) It is a rare disease that seriously affects the growth and development of children and causes high mortality rate Liu Lu sighed: "compared with those" incurable "children, we are still lucky Yamelo will be on the market soon after children's diagnosis After treatment, it is no different from normal children Our only expectation is that special medicine will be included in the medical insurance as soon as possible, so that we can take a breath in the economy " It is reported that children have been treated with topiramab injection (trade name: yamelo) since their diagnosis, and their condition has been well controlled However, the cost of each period of treatment still makes it difficult for the family with a solid foundation to maintain In this large group of patients, about 50% of the patients with rare diseases in childhood, need long-term treatment Compared with chronic diseases and tumors, the patients with rare diseases and their families have to face not only the physical and mental load from the disease itself and the caregivers, but also the pressure from school, employment and social participation, which causes more disastrous expenditure on the families Guan Tao, founder of hemophilia family, said: "although rare diseases are not easy to treat, some rare diseases can significantly improve the quality of life of patients and make them return to social life through the standardized use of special drugs." Therefore, to improve the accessibility of drugs for rare diseases and strengthen the medication security of patients can not only improve the quality of life of patients, but also effectively reduce the economic burden of patients with rare diseases and their families, and prevent catastrophic medical expenditure of families, so as to reduce the families who are poor or return to poverty due to illness ▍ only six kinds of special drugs included in the medical insurance report show that at present, there is a large room for improvement in the key indicators of "orphan drugs" in China, such as the types of drugs on the market, the coverage of national medical insurance and the overall expenditure level At present, among the 121 diseases included in the first list of rare diseases in China, 44 rare diseases have 88 kinds of treatment drugs in the world, while only 35 are listed in China Many innovative rare disease drugs have not entered China, and many patients are still "without drugs" At the same time, according to the calculation of iqvia, 24 of the 35 orphan drugs in China belong to the special drugs with high monthly average treatment cost However, only 6 of the 24 special drugs are listed in the national medical insurance catalog, 3 of which are negotiated by the state in 2017 Most of the other special drugs for rare diseases have not yet entered the coverage of the national medical insurance, and the long-term standardized treatment of patients is difficult to maintain In the face of the economic burden and realistic pressure, most patients can't receive enough treatment, even have to give up At present, the role of health technology assessment is increasing day by day, and it has become a universal tool for health decision-making in the world The United States, Britain, Canada, Australia and other developed countries have passed legislation to determine the legal status and value of health technology assessment, and become a strong gatekeeper for innovative technology, drugs, and equipment to enter clinical practice With the deepening of medical and health system reform, the establishment and improvement of system and mechanism, resource allocation, technology selection and utilization strategy become more and more important In October 2018, the national comprehensive assessment center for drugs and health technology was officially established In the face of the health technology evaluation of rare diseases, Zhao Kunze, director of the Health Technology Evaluation Research Office of the health research and development center of the national health and Family Planning Commission, said: rare diseases are not suitable for traditional pharmacoeconomic evaluation, and there is no need to discuss the "value or not" aspect, and no need to make comparative cost-effectiveness analysis Looking around the world, many developed countries will exempt and recommend innovative drugs with breakthrough treatment mechanisms, clinical treatment gaps and huge benefits for patients Fairness is one of the important factors that affect the overall health insurance expenditure of rare diseases In recent years, the medical insurance departments in some provinces and cities have begun to take the initiative to include "orphan drugs" in the local medical insurance catalogue, but their coverage and security efforts are still limited It is believed that the number of rare disease population is small and the cost of single person is high The cost of medical insurance for a rare disease patient can cover more chronic disease patients Director Zhao Kun said: "patients with rare diseases and their relatives are also social taxpayers As long as they pay medical insurance, they have the right to enjoy the help of public funds Although the unit price of rare diseases is high, the number of patients is small, and the total amount will not be very high This part of the cost is very small for the overall public funds expenditure " ▍ China has successively issued rare disease safeguard policies to improve the accessibility of rare disease drugs in China, which involves many links, including the research and development of new drugs, the rapid approval and listing of new drugs, and the discussion of more innovative financing and payment models Although China's policies really started late, in recent years, under the environment of encouraging innovative drug development and accelerating the review and approval, some encouraging policies involving drugs with rare diseases have also been introduced In 2015, the State Food and Drug Administration (CFDA) issued the announcement on several policies for drug registration review and approval, which clearly proposed that the application for innovative orphan drugs should be queued separately to speed up the review and approval In October 2016, the product "yamelo" of Roche pharmaceutical, which was used to treat systemic juvenile idiopathic arthritis (sJIA), was approved by the former State Food and Drug Administration for exemption from phase III clinical trials, and was listed in China four years in advance, becoming the first biological agent for treatment of sJIA in China Recently, at the same time when the report was released, there was also good news that the new hemophilia drug hemlibra shuyoulile (aimesizumab) was approved In addition to the policy guarantee, the payment system for rare diseases is gradually improving On the one hand, the National Health Insurance Bureau improves the security system from the top-level design through the "top-down" mechanism Chen Jinfu, deputy director of the state health insurance bureau, once said that in the future, a normalized and dynamic access mechanism for medical insurance drugs will be established, and innovative drugs, high-value drugs and clinically urgent drugs will be included in the scope of drug bidding and purchase preferentially With the accelerated approval of orphan drugs by the State Drug Administration and the continuous listing of innovative orphan drugs, the drug type selection mechanism in the future national health insurance negotiation will inevitably take into account the clinical emergency needs of rare diseases On the other hand, local medical insurance promotes the innovation and upgrading of the top-level design of national medical insurance Many provinces and regions, including Qingdao and Zhejiang Province, have made different explorations and initially established a guarantee system for the use of drugs for rare diseases, which provides valuable experience for other regions ▍ it is not only a problem in the field of medical research, but also a problem in the whole society to promote the use of drugs to protect rare diseases In the face of the drug use of rare diseases, in addition to the strong support and active promotion of national and local medical insurance, more financing channels and payers are still needed to participate in the formation of multi-party cooperation innovation mechanism and sustainable development guarantee system The report pioneered the introduction of multiple forms of financing such as commercial insurance and special funds into the payment model for rare diseases, which also became a major innovation highlight of the report In recent years, with the development of commercial health insurance, some experts began to explore the feasibility of using commercial health insurance to protect the treatment of rare diseases Commercial health insurance can provide supplementary protection for the public, provide patients with the right of choice, and reduce the pressure of medical insurance fund In 2015, Shenzhen Social Security Bureau and Ping An Insurance Co launched an insurance product for supplementary medical treatment of major and serious diseases, and encouraged citizens to purchase it through individual account funds After several years of operation, the project has received good social repercussions and provided useful reference for system design in other regions With more local medical insurance exploring the mode of individual account activation, the cooperation between commercial insurance and government departments will be further strengthened, and the commercial insurance products for rare diseases may be promoted to some extent Because the awareness rate of rare diseases is low and the patients are scattered, the landing and long-term development of rare disease commercial insurance products need the active participation of the government to improve the awareness and participation rate of the target population The intervention of commercial insurance mode may open a door for the rare disease insurance system, and this innovation mode still needs more support and promotion.