Ding Liming: research and development of orphan drugs should arouse the enthusiasm of domestic enterprises

Highlights at the two sessions in 2017, Ding liming, deputy to the National People's Congress and chairman of Zhejiang Beida Pharmaceutical Co., Ltd., said in an interview with minenet that the CPC Central Committee and the State Council have paid close attention to the drug use of patients with rare diseases, but a series of effective policies and measures need to be introduced and implemented The last day of February every year is the international day of rare diseases According to the definition of the World Health Organization (who), diseases with the number of patients accounting for 0.65 ‰ - 1 ‰ of the total population are collectively referred to as rare diseases Currently, nearly 7000 kinds of rare diseases have been confirmed, and about 80% of them are caused by genetic defects In order to let the patients with rare diseases get the attention of the whole society, some developed countries and regions have already legislated In 1983, the U.S Congress passed the orphan drug act, and the United States became the first country to legislate on rare diseases In 1993, Japan established the regulations on the administration of rare disease drugs in accordance with the pharmaceutical act In 1999, the European Union formulated the orphan drug regulations, which proposed that patients with rare diseases should be treated with the same quality as patients with other diseases The representative called on China to keep up with the pace of legislation On February 25, 2017, the first treatise on rare diseases, mainly clinical cases, was published in China It was compiled by Shanghai foundation for the prevention and control of rare diseases and 112 experts in various fields in China, which lasted more than a year In recent years, many representatives of the National People's Congress and members of the CPPCC in the field of medicine are actively calling for the state to support and encourage the research and development of orphan drugs through legislation At the two sessions in 2017, Ding liming, deputy to the National People's Congress and chairman of Zhejiang Beida Pharmaceutical Co., Ltd., said in an interview with minenet that in February this year, the general office of the State Council issued several opinions on further reform and improvement of drug production, circulation and use policies, which pointed out that "explore the classification of rare diseases, children, the elderly, emergency (emergency) rescue drugs and traditional Chinese medicine (classic formula)," Review and approval, to ensure the needs of children, the elderly and other groups and the prevention and treatment of major diseases "It shows that the CPC Central Committee and the State Council have paid close attention to the drug use of patients with rare diseases, but we still need to introduce and implement a series of effective policies and measures The government should give full support to orphan drug research and development "as soon as possible, formulate the national definition and catalogue of rare diseases, issue the corresponding orphan drug laws and regulations, and set up the orphan drug research and development management organization, increase the financial support for orphan drug research and development through the national science and technology special project, and adopt 50% tax preferential policy for the related expenses of orphan drug research and development." Ding Liming said that the research and development of orphan drugs in China is almost in a blank stage, and the treatment drugs for patients with rare diseases are basically imported from abroad, resulting in many patients can only choose expensive imported drugs or no drugs available The patients with rare diseases in China have become a vulnerable group that needs social attention The medication of patients with rare diseases has become a major livelihood issue in China There are data that show that in the development of new drugs, the cost of clinical trials is huge, but for patients with rare diseases, there are not many patients, so the number of clinical trials required is also a difficult problem for drug research and development institutions Some developed countries in foreign countries support the R & D, production and marketing of orphan drugs from the policy level The government provides R & D support for orphan drugs In the process of scientific research, it provides free consultation and help to reduce the amount of tax on enterprises For example, the U.S FDA has an orphan drug research and development office, which is responsible for approving whether to grant fund support The fund can be provided to pharmaceutical enterprises or academic institutions to support the clinical phase I-III trial process Japan's support for orphan drugs is reflected in the whole process of research and development, from preclinical trials to clinical trials, all of which can enjoy fund support Open a green channel for orphan drug approval "in the new drug approval stage, open a green evaluation channel for orphan drug approval, and it is recommended to adopt a filing system for orphan drug clinical trials." Ding Liming mentioned that due to the lack of demand for orphan drug monomer Market and high R & D cost, it will be difficult to solve this problem by relying solely on enterprises and market mechanism The government needs to guide it from the policy level, increase support for orphan drug R & D, and encourage domestic research institutions and enterprises to research and develop rare diseases and orphan drugs   Ding Liming said that since the promulgation of the orphan drugs act in 1983, the U.S government has established an incentive mechanism for research and development of orphan drugs based on qualification recognition, that is, the drugs developed by government agencies are first identified as orphan drugs, and when they are identified as orphan drugs, they can enjoy a series of incentive policies, including funding for research and development, opening a green registration approval channel, and exemption and approval Cost evaluation, tax relief, market monopoly, medical insurance reimbursement support, etc The enactment of the act has greatly promoted the research and development of orphan drugs in the United States As of March 2015, the U.S FDA has identified 3345 orphan drugs and approved 488 orphan drugs Adjust the market access and medical insurance reimbursement mechanism of orphan drugs "In terms of market access, the direct marketing mode is adopted for the sale of orphan drugs, which is directly sold by the manufacturer to the medical institution It is not necessary to purchase drugs through centralized bidding to reduce the circulation link At the same time, a special medical insurance reimbursement catalog is set up for orphan drugs, and a co payment mechanism funded by medical insurance fund, patients and third parties is established Orphan drugs will directly enter the reimbursement catalog after being listed And on the basis of the existing patent protection, we should establish an appropriate market monopoly period for orphan drugs " Ding Liming said that at present, after more than 30 years of reform and opening up, China has leaped to the second largest economy in the world, and the national economic strength has greatly increased Now we have the ability and need to help patients with rare diseases to solve their drug demand According to the reporter of mienei.com, at present, the medical expenses of patients with rare diseases in China mainly enjoy the corresponding compensation treatment according to the existing medical insurance reimbursement policy Due to the limitation of fund-raising level, the corresponding guarantee scope can only be determined by the local medical insurance (NCMS) fund payment ability and other actual situations, so that many special drugs and diagnosis and treatment projects needed by most patients with rare diseases can not be included in the reimbursement scope To create a favorable policy environment for orphan drug research and development "at present, China's drug supply security system and policy system are designed on the basis of common diseases and frequently occurring diseases, and there is no targeted and systematic incentive policy for research and development, pricing, reimbursement and talent training of special drugs for rare diseases." Ding stressed that he hoped to create a favorable policy environment for orphan drug research and development in China with the strong support of the state, mobilize the enthusiasm of domestic enterprises for orphan drug research and development, provide affordable good drugs for domestic patients with rare diseases, and save and improve the life and quality of life of patients with rare diseases.