Original title: Increase research and development efforts, optimize approval procedures, and improve the level of children's drug supply security
Take multiple measures to protect children's health
Core reading
In the first five months of this year, 34 pediatric drugs were approved for marketing; In March this year, the new version of the national medical insurance drug catalogue was officially implemented, and the new drugs covered 22 children's drugs; In January this year, the General Office of the National Health Commission issued a notice to encourage medical institutions to develop new technologies and methods that can flexibly adjust the dosage for children's medication, and strengthen the standardized management and quality control of personalized drug delivery...
The mainland has adopted a series of measures to encourage the development and production of medicines for children, and continuously improve the level of drug supply security.
According to data from the National Bureau of Statistics, at the end of 2022, the population aged 0-15 (including under 16 years old) in the mainland was 256.15 million, accounting for 18.1% of the total population.
Compared with adult drugs, the R&D and production of children's drugs face problems such as late start, difficulty and weak foundation, which require the joint efforts of medical institutions, scientific research institutes and pharmaceutical enterprises.
In January this year, the General Office of the National Health Commission issued the Notice on Further Strengthening the Management of Clinical Medication for Children, encouraging medical institutions to develop new technologies and methods that can flexibly adjust the dosage for children's medication; In March, the new version of the national medical insurance drug catalogue was officially implemented, and the new drugs covered 22 children's drugs; In the first five months of this year, 34 children's drugs have been approved for marketing, exceeding the same period last year... The mainland has adopted a series of measures to encourage the development and production of drugs for children, and continuously improve the level of drug supply security.
Break through key technologies and fill market gaps
"Taking medicine depends on breaking, and the dose depends on guessing" used to be a problem faced by some children and parents when using medicine. "Children are not the epitome of adults, and their medication needs to be 'tailored'." Ni Xin, director of the National Children's Medical Center and president of Beijing Children's Hospital, said.
Take mercaptopurine, an important drug for the treatment of acute lymphoblastic leukemia in children, as an example. Currently, the only available size of mercaptopurine on the market is a 50 mg tablet, about 1 cm in diameter, all of which are adult tablets. Children taking mercaptopurine requires a dose calculated in kilograms of body weight and adult tablets cut or milled, which is inconvenient and inaccurate.
In order to solve this problem, the National Children's Medical Center and Beijing Children's Hospital took the lead in breaking through key technologies and developing an improved dosage form of mercaptopurine that can be accurately measured. The tablets are 2 to 3 mm in diameter and the size of rice grains, which is convenient for swallowing. This achievement has been unveiled at the 2022 China International Fair for Trade in Services.
"The variety, dosage form and specification of children's drugs in mainland China are generally insufficient compared with adult drugs, and about 45% of the drug inserts in pediatric clinical drugs lack children's dosage, resulting in a much higher risk of medication errors in children than adults." Wang Xiaoling, director of the Department of Pharmacy of Beijing Children's Hospital, said that integrating resources, breaking through a series of core key technologies, and rationally carrying out pediatric clinical research are the keys to achieving efficient research and development and clinical transformation of pediatric drugs.
In recent years, Beijing Children's Hospital has been committed to the research and development of pediatric drugs. Wang Xiaoling introduced that Beijing Children's Hospital took the lead in undertaking the "13th Five-Year Plan" major special project of new drug creation science and technology - "research and development of children's drugs and key technologies", and united the research and development strength of 59 scientific research institutes, universities and enterprises across the country to strengthen scientific and technological innovation and develop children's drugs. At present, 65 relevant approvals, acceptance numbers or signature numbers have been obtained, and the relevant results have filled the gap in the mainland children's drug market and will alleviate the current shortage of children's drugs in the market.
In addition, the National Health Commission has invested more than 600 million yuan in research on children's drugs through major projects on the creation of new drugs; The National Children's Medical Center was established, 23 national pediatric related units were organized, and the "China Pediatric Population Drug Clinical Trial Collaboration Network" was established to promote clinical research on pediatric drugs.
Accelerate review and approval and stimulate innovation momentum
"My son finally used domestic chlorbazhan!" Ms. Zhang from Henan said excitedly, "The child was diagnosed with epilepsy when he was two years old and needed to take the therapeutic drug clobazam, and the listing of domestic chlorbazam solved a big problem for us. ”
In May 2022, the National Center for Drug Evaluation included the marketing application of the generic drug clobazam tablets in the priority review for the treatment of epilepsy patients aged 2 years and older. In September, the first domestic generic drug of clobazam in mainland China was approved for marketing. Soon after, a batch of clobazam tablets went off the production line and entered hospitals in more than 20 provinces, effectively alleviating the lack of medication for children with epilepsy.
Clobazam tablets are a microcosm of the results of the review and approval of drugs for children in mainland China in 2022. As a drug technical review institution, the National Center for Drug Review optimizes the review and approval mechanism for children's drugs to stimulate the vitality of industrial innovation. Since 2021, the National Drug Review Center has set up a special leading group and working group for children's medication, set up a special label for "children's medication", shortened the approval time by 35%, and accelerated the marketing of children's drugs.
According to data from the State Food and Drug Administration, a total of 66 children's drugs passed the technical review in 2022, a significant increase from 47 in 2021, including 21 priority review and approval varieties and 11 varieties that encouraged R&D to apply for children's drug lists. These products have accelerated the market launch and further met the medication needs of children.
To solve the problems of fewer types of drugs and fewer dosage forms for children, it is also necessary to stimulate the research and development power of enterprises. In accordance with the principle of "urgent use first", the National Drug Review Center has established an evidence system for the review of children's drug research and development, and guided enterprises to carry out research and development smoothly. At present, more than ten special guidelines for pediatric drugs have been issued, improving the clinical trial and safety evaluation standards for pediatric drugs, and providing important technical support and review basis for R&D and review.
Accelerating the introduction of overseas marketed drugs is also an important way to meet the drug needs of mainland children. In recent years, the State Food and Drug Administration, together with the National Health Commission, has organized experts to select and release a list of new overseas drugs urgently needed for clinical use, encouraged overseas pharmaceutical companies to come to apply, and established a special channel for the review of declared varieties. Mainland China has accelerated the approval of 16 pediatric drugs, including nocinaxen sodium injection for the treatment of spinal muscular atrophy, which has brought new hope for improving the survival rate and quality of life of children.
Strengthen multi-party cooperation to promote R&D and production
"The mainland still lags behind the international leading level in the research and development and independent production of suitable preparations for children, and the research and development of drugs for major diseases and rare diseases." Wang Xiaoling said frankly.
How to overcome the problem of medication in children? Wang Xiaoling suggested that a special project for the research and development of children's drugs should be set up, focusing on cutting-edge issues and key technologies, integrating basic research and transformation application resources, and realizing independent innovation and variety transformation of characteristic technologies for children's medicine.
"It is necessary to encourage medical institutions to cooperate with scientific research institutes and enterprises to develop new technologies and methods that can flexibly adjust drug doses, establish a personalized adjustment platform for young children, and promote personalized medication for children." For the preparation of pediatric medical institutions with precise efficacy, obvious characteristic advantages and few adverse reactions, the inter-provincial transfer process is simplified to meet the needs of clinical medication. Wang Xiaoling said.
Zhang Shuyang, president of Peking Union Medical College Hospital, proposed to further improve the R&D catalogue of pediatric drugs on the basis of the existing list of children's drugs declared by R&D, and guide priority R&D creation to meet the needs of pediatric clinical drugs. Mobilize the enthusiasm of enterprises to participate in the research and development of children's drugs through tax exemptions, targeted subsidies, and designated production. Strengthen basic and clinical research on children's drugs, promote the improvement of children's medication information in adult drug labels, and encourage international certification of children's drugs. Continue to strengthen technical guidance for standardizing pediatric research, and improve the safety and security of pediatric drug clinical trials.
"Different disease areas in pediatrics face different medication problems." Ni Xin believes that the treatment of some common and frequent diseases in children lacks drug dosage forms and specifications suitable for young children, the treatment of major diseases such as hematological oncology lacks pediatric drug data, and the lack of available drugs for children with rare diseases exists to a certain extent.
Ni Xin suggested that on the one hand, we should increase the research and development of new drugs, and pediatric experts and drug manufacturers should strengthen cooperation and carry out scientific research; On the other hand, for the latest effective innovative drugs abroad, it is necessary to carry out timely indication research and introduce them into China in a timely manner, so that children can use drugs as soon as possible.
"The research and development of children's drugs is difficult, the process is relatively complex, the production cost is higher, coupled with the small dosage, some pharmaceutical companies are not enthusiastic about R&D and production." Yang Jie, head of Dyne Pharmaceutical, suggested that relevant departments should formulate children's drug access and pricing incentive policies to allow enterprises to have certain profit margins, so as to encourage enterprises to invest more human and financial resources in the research and development and production of children's drugs.