In recent years, the whole society has gradually paid more attention to rare diseases. During the two sessions this year, Professor Mao Zongfu, deputy to the National People's Congress and director of the global health research center of Wuhan University, and Wu Ming, member of the National Committee of the Chinese people's Political Consultative Conference and director of the Department of health policy and management of the school of public health of Peking University, all put forward suggestions related to the drug guarantee mechanism for rare diseases. Recently, in an interview with surging news reporters, Wu Ming said that he attached importance to the scientific evaluation of the impact of rare disease drugs on the medical insurance fund, explored the establishment of a medical insurance access mechanism suitable for rare disease drugs, and paid attention to the volume price balance to ensure the innovation power of Medicine in the field of rare diseases. Mao Zongfu proposed that, in addition to the coverage of medical insurance fund, we should also strengthen the connection between urban customized commercial medical insurance (universal benefit insurance) and basic insurance, strengthen the guiding role of the government, improve macro policies, standardize market behavior, strengthen the scientific and technological support of the new business form of universal benefit insurance, and promote the accessibility of medical security for rare disease drugs. The inclusion of rare disease drugs in medical insurance depends on the total amount Rare diseases, also known as orphan diseases, refer to diseases with very low incidence rate and very few total population. They are characterized by many species, heredity, difficult diagnosis, heavy illness, low treatment and high cost. According to the statistics of the national rare diseases organization of the United States, of the more than 7000 rare diseases that have been found, only 10% have corresponding treatment methods, and only 1% have effective treatment drugs for the cause. From the perspective of pharmaceutical enterprises, the R & D cost of rare drugs is higher, the return on investment is lower and the risk is higher than that of ordinary drugs. However, at present, more and more pharmaceutical enterprises have launched drug listing in the field of overweight rare diseases. Another problem follows. Drugs for rare diseases are often expensive and are called "sky high price drugs", which makes many patients out of reach of drugs. There are more than 20 million patients with rare diseases in China. How to solve the problem of drug accessibility for patients with rare diseases has long been made at the national medical insurance level. Taking the new catalogue of drugs for national basic medical insurance, work-related injury insurance and maternity insurance implemented on January 1, 2022 as an example, a total of seven rare diseases drugs were included, including nosinasheng sodium injection, a drug for spinal muscular atrophy. Previously, the annual treatment cost of the drug exceeded 1 million yuan. According to the data released by the National Health Commission in October 2021, there are more than 50 kinds of drugs for rare diseases listed in China, of which more than 40 kinds have been included in the national medical insurance drug catalogue. In an interview with surging journalists, Wu Ming said that promoting the inclusion of rare disease drugs in medical insurance mainly depends on the space of medical insurance fund in addition to the clinical value and price of drugs themselves. In the past, the medical insurance fund overspent in many places and it was difficult to include drugs for rare diseases. However, at present, through comprehensive means such as the centralized purchase of drug consumables and the reform of medical insurance payment method, some medical insurance space has been vacated and the use efficiency of medical insurance funds has been improved. In addition, with the continuous increase of medical insurance financing level in China, the prospect of rare diseases being included in medical insurance is relatively good. It is worth mentioning that Wu Ming emphasized in his proposal that the inclusion of rare diseases in medical insurance "depends on the total amount, not the unit price". Wu Ming explained that drugs for rare diseases are expensive, and inclusion in medical insurance can easily lead to a significant increase in medical insurance expenditure or even overspending, which is a misunderstanding. For example, for imisidase, which has a good curative effect on Gaucher's disease, the annual per capita drug cost is 1.63 million yuan. There are more than 400 registered patients with a total cost of 688 million a year. Even if the number of drug users will increase after being included in medical insurance, because it is a rare disease, the number of patients will not be large, and the total cost is not large, so it has little impact on the fund space of medical insurance. Through the medical insurance negotiation, many rare disease enterprises take the initiative to reduce prices and enter the medical insurance catalogue. Will this affect the R & D and innovation of pharmaceutical enterprises in the field of rare diseases? Wu Ming believes that for enterprises, not only rare disease drugs, but also many innovative drugs are facing the practical problems of high price and small quantity, low price and large quantity. Although the sales volume of medical insurance has increased significantly, the price of medical insurance negotiation has been greatly reduced. When the return rate of enterprise investment is reduced or even there may be no return, it may indeed affect the innovation. "In the evaluation and negotiation of pharmacoeconomics, we should consider the relationship between price and quantity and the impact on profits as much as possible. There is no such number as how much can be returned. I hope we can find such a balance in the future." Wu Ming told surging news reporters that the first thing to do is to look at the curative effect. If the curative effect of orphan drugs is very good, we can consider leaving a certain profit space for enterprises in pharmacoeconomic evaluation, so that enterprises can get a certain R & D return, rather than just considering the price or even pursuing a low price. Otherwise, in the long run, it will reduce the confidence of the capital market, Affect the enthusiasm of enterprise R & D. Strengthen the close connection between basic medical insurance and innovative payment In addition to being included in the national medical insurance catalogue, innovative payment means such as inclusive insurance are also regarded as an important starting point to solve the accessibility of drugs for rare diseases. Mao Zongfu's proposal this year is about the sustainable development of the connection between urban customized commercial medical insurance and basic medical insurance. According to Mao Zongfu's analysis, there are many kinds of rare diseases, a small number of people and relatively few drugs available. Considering the current level of economic development, the whole society and the basic medical security system, if rare disease drugs occupy too many financial resources in the basic medical insurance, they will inevitably occupy more space for the treatment of common and frequently occurring basic diseases, This may not be the best in terms of social benefits. "Even for the rare disease drugs that enter the medical insurance, in different overall planning areas, the proportion of medical insurance payment in some overall planning areas with tight funds is still relatively low, and the personal burden is still relatively heavy." Mao Zongfu said that the rise of Pratt & Whitney insurance in recent years has reduced the insurance threshold of ordinary people to a certain extent, and China's Zhejiang, Chengdu, Guangdong, Shandong, Shanghai and other places have made preliminary achievements in relevant practice. As a livelihood project, Pratt Whitney insurance should clarify the government responsibility of "basic, main body and bottom-up", give full play to the role of the market in resource allocation, and make HP insurance a "mass medical and health insurance" for thousands of families through innovative social and business cooperation. However, Mao Zongfu also told surging news reporters that the problem of rare diseases can not be completely solved by inclusive insurance alone. According to its research data, at present, the proportion of inclusive insurance products that can develop sustainably is not high, one of the reasons is low premium and high insurance amount. "There must be a law of large numbers, that is, more people pay premiums to ensure the operation of inclusive insurance." Mao Zongfu introduced that some places have good experience and practices. For example, the experience of Zhejiang shows that government guidance can improve the insured's confidence in inclusive insurance. Through the sharing of public resources, information platforms and policy tools, the commercial operation cost of inclusive insurance can be reduced, and resources can be more effectively invested in the joint relief of serious diseases and serious diseases. (Xinhua News Agency)