28 kinds of accelerated approval of children's drugs have been included in the priority review

The concept of using children's medicine for children's illness is being recognized by more parents, but many parents find that the commonly used medicine often has no dosage form for children, and children's difficult and severe illness is even more useless On October 18, the media learned from the drug review center of the State Food and drug administration that up to now, 28 tasks of children's drug use have been included in the priority review plan, among which most of the endocrine system drugs for children's diabetes are related to the treatment of children's leukemia, hemangioma, neonatal apnea and other critical diseases There are few kinds, specifications and dosage forms of children's drugs The white paper of 2016 investigation report on children's drug safety issued by South Institute of pharmaceutical economics, State Food and Drug Administration shows that children's drug dosage forms account for less than 2% of China's existing more than 3500 drug dosage forms In 2015, 2690 drugs passed the examination and approval, while only 19 drugs for children, accounting for 0.7% "At present, there are few specifications and dosage forms for children's medicine in China, which leads to the lack of medicine for children with many diseases, or for adults and children, which increases the risk of drug safety for children." Wang Xiaoling, director of Pharmacy Department of Beijing Children's Hospital, said that in 2016, Beijing Children's Hospital conducted a questionnaire survey on drug safety of children in Beijing, Shanghai, Guangzhou and Wuhan The survey found that 8% of parents used adult drugs for children The reason is that the parents said they know that children should not use adult drugs, but the drugs lack the dosage form of children, there is no medicine available, so they have to use adult drugs "Children's organs are not yet mature, and the digestion and excretion ability of liver and kidney are weak If children are allowed to use drugs together without permission, it will increase the burden of liver and kidney functions, and even lead to hepatorenal poisoning." Wang Xiaoling pointed out that, compared with adults, the drug-induced damage caused by unreasonable use of drugs and wrong use of drugs in children is more serious, and children should be particularly careful in their choice of drug use It is pointed out that one of the reasons for the lack of children's drugs is that there are few manufacturers of children's drugs in China According to a survey conducted by the pharmaceutical chamber of Commerce of the all China Federation of industry and commerce, of the more than 6000 pharmaceutical factories in the country, only more than 10 specialized in children's drug use, and only 30 enterprises with children's drug production departments "Due to the limited market share of children's drug use, higher technical requirements and stricter index requirements for children's drug use, higher human, material and time costs invested in the R & D process, higher R & D costs than ordinary new drugs, and corresponding pharmaceutical research on different dosage forms, specifications and tastes for children of different ages, which is costly and complicated Therefore, the R & D investment risk is large, and the profit space is small Most drug manufacturers are reluctant to research and develop children's drugs Subject to these factors, the enthusiasm of children's drug R & D is not high " The head of the drug Audit Center pointed out that children's drug research and development costs are high and profits are low, and many drugs are expected to make progress In fact, "at present, the biggest difficulty in the research and development of children's drug use in China is the lack of clinical trial basis." The person in charge further pointed out that due to the small number of clinical trial institutions for children's drug use and the lack of guarantee mechanism for subjects, and the majority of children in China are only children, the vast majority of parents are unwilling to treat children as clinical subjects, in addition, the risk of children's drug use is too high, leading to the lack of enthusiasm of clinical trial institutions and doctors participating in clinical trials, which led to the lack of support mechanism for children's drug use clinical trials in China Difficult problem "Government departments, medical departments, pharmaceutical enterprises and so on should perform their respective duties and make contributions." Wang Xiaoling believes that the solution to the problem of children's drug use needs cooperation and joint efforts The approval of children's drugs is speeding up, and the guiding principles for research and development of children's drugs are expected to be promulgated On June 25 this year, the state new office held a press conference on the implementation opinions on reform and improvement of the mechanism for ensuring the supply of drugs in short supply Zeng Yixin, deputy director of the national health and Family Planning Commission, said that the issue of children's drug use has attracted great attention from all parties During the 13th Five Year Plan period, the Ministry of science and technology, the Ministry of finance, the State Food and Drug Administration and the health and Family Planning Commission will jointly speed up the R & D and approval procedures for drugs specifically suitable for children in China According to the media, 28 drug use tasks for children have been included in the priority review plan so far There are 12 indications involved in the drug use for children included in the priority review, most of which are endocrine system drugs for children's diabetes, and other critical diseases such as childhood leukemia, hemangioma and neonatal apnea In addition, some pediatric drugs with clinical value and urgent need of patients have completed the pre marketing technical review, such as: the approval of megestat capsule for the treatment of rare C-type Niemann peak disease, which is the only drug for the treatment of the disease in the world; Tuozhumab injection approved for the treatment of systemic juvenile idiopathic arthritis (sJIA) is the first effective treatment drug for this indication in the world, and it is also a priority drug for children The recombinant human thrombopoietin injection, dulaglutide injection, lidocaine hydrochloride powder needle free percutaneous delivery system, which have important clinical value, have been approved to carry out new drug clinical trials in pediatric patients through priority review In terms of the review time of the 16 priority review varieties that have completed the review, the shortest time is 13 working days, with an average of 64 working days, and the review speed is significantly improved "In the future, we will speed up the formulation of guiding principles for drug research and development for children, establish a systematic and perfect guiding principle system, and provide guidance for enterprises to carry out drug research and development Study the evaluation standard system of children's drug use, and fully consider the special requirements of children's safety and adaptability for children's drug use in pharmacy, especially for dosage form selection, specifications, packaging materials, accessories, etc " Said the person in charge of the drug audit center.