CDE Yang Zhimin, Xie Songmei: Drug review promotes the development of pediatric drugs through multiple channels, and product reviews are gradually increasing!

The issue of medicine for children is a livelihood issue that the party and the state attach great importance to.
General Secretary Xi Jinping has issued important instructions on this issue on August 25th
.
Recently, a reporter from the "Pharmaceutical Economic News" learned from the National Medical Products Administration that in recent years, in order to solve the problem of difficult drug development for children, the drug review and approval agency has launched a series of encouraging research and development innovation, improving review efficiency, and improving work >
.

 

During the interview, the reporter learned that the drug risporan oral solution for the treatment of rare diseases was launched in the first half of 2021 and was approved for the treatment of spinal muscular atrophy (SMA) in patients aged 2 months and older
.
According to Xie Songmei, the deputy director of the Second Clinical Department of the Drug Approval Center, in order to promote the early listing of risporan oral solution in China, it will provide Chinese patients with a clear curative effect, and it is safer than the only existing intraspinal intrathecal injection.
Convenient new mechanism for oral treatment options, the Drug Approval Center has conducted multiple rounds of communication with the applicant companies
.

 

She said: “Through early communication in clinical research and development, we recommend that the original research company conduct clinical trials in China as soon as possible to provide data support for scientific decision-making in drug review
.
Companies adopt our suggestions to carry out two key phase II clinical trials on a global scale.
A certain proportion of Chinese subjects were included in the trials
.
In the end, based on the data of global and Chinese clinical trials, we made recommendations for approval of listing in China on the basis of ensuring that the benefits of patients’ medications outweigh the risks
.
This is A case where China has joined the global early simultaneous research and development based on regulatory encouragement, and promoted the simultaneous registration and listing of new mechanism drugs in China and the world, so that Chinese patients with rare diseases can get valuable new drugs as soon as possible
.
"

 

　　Publish a list of encouraged R&D

 

　　The development and production of medicines for children is a worldwide technical problem, which is closely related to the pathophysiological characteristics of children and their tolerance to the safety risks of medicines
.

 

　　Yang Zhimin, director of the Department of Clinical Medicine of the Center for Drug Appraisal, pointed out: "To promote the solution of children's drug use problems, regulatory agencies, clinical institutions and drug manufacturers need to work together in the same direction
.
"

 

　　In recent years, with the support of major national science and technology projects, a number of children's medical institutions in China have established a relatively complete research and development technology platform for children's drugs, which provides a strong guarantee for the innovative research and development of children's drugs in China
.
Yang Zhimin explained that, as the main body of research and development, enterprises are the main force to solve the problem of children's medicine in China.
The drug review department maintains good communication with research and development enterprises, and gives specific guidance on the actual difficulties in the enterprise's research and development process, which is crucial to speeding up the process of children's medicine research and development.
Important
.

 

　　In order to implement Document No.
44 of the State Council and the newly revised "Drug Administration Law", the Center for Drug Evaluation will give priority review and approval to children's drugs that are urgently needed clinically and have obvious clinical advantages
.

 

　　 "The review of children-first and urgently-needed varieties has its own particularities.
It requires tacit cooperation and efficient interaction in various links such as professional review, business management, verification, inspection, and scientific comprehensive decision-making
.
" Xie Songmei introduced that the current drug review center has opened children's drugs.
The green channel of review adheres to patient-centered, specific analysis based on clinical urgent needs and specific types of research data, and continuously rationalizes the priority processing procedures and requirements, and achieves high standards, strict requirements and strong services
.

 

　　In order to improve the efficiency of variety review, minimize review time, and accelerate the launch of children's drugs, the Center for Drug Evaluation has also set up a special label for "Children's Drugs", which will be connected by a project manager to supervise the review progress and ensure progress in time
.
At the same time, strengthen early intervention and proactively serve in the communication, acceptance, review and other links of the enterprise
.

 

　　Data shows that among the three batches of clinically urgently needed overseas new drug catalogues released by the National Health and Construction Commission, 16 new drugs that are urgently needed for clinical use in children have been approved for marketing
.
From 2016 drug trial center will be the same National Health Health Commission, the State Ministry of Industry issued three batches of medicines for children are encouraged to develop a list of declarations, the first involving the list of 32 varieties, the second batch of the list involves 39 varieties, the third installment of the list Involving 34 varieties, promote the research and development, innovation and application review of children's suitable varieties, dosage forms and specifications
.

 

　　As of June 30, 2021, 14 varieties on the list have completed technical review and been approved for marketing, 9 varieties on the list are under review for marketing applications, and 5 varieties on the list have been approved for clinical trials
.

 

　　Improve the review standard system

 

　　 " Children's drug development is different from general drugs.
We can start from several aspects at the moment
.
" Yang Zhimin suggested that first, children's drug development needs to be based on clinical urgent needs, mainly in the areas of common and frequently-occurring diseases, and the drug should be strictly defined.
Dosage measurement standards; Second, the development of children's drugs can be combined with the actual clinical situation.
Many drugs have been used in clinical practice for many years and have been accepted by clinical guidelines and expert consensus.
These drugs can supplement children's indications by exploring real-world research data; Third, the most difficult part is the research and development of rare disease drugs, and the research and development of these drugs should involve more international cooperation
.

 

　　The formulation of technical guidelines is an important supervision method for regulating drug research and development and improving the quality and efficiency of drug research
.
Accelerating the improvement of the evaluation standard system and evaluation methods for children's medications can effectively support and guide scientific and orderly research and development, improve the quality of declarations, enhance the vitality of children's medications, and provide important technical support and evaluation for standardizing the development and evaluation of children's medications in China Basis
.

 

　　It is understood that before 2020, the Center for Drug Evaluation only issued three specific guidelines for children's medication
.
After 2020, the Center for Drug Evaluation has increased its ability to build technical guidelines for children's medications, and in accordance with the principle of emergency use first, successively released the "Guidelines for the Development of Children's Drugs (Chemicals) Pharmacy Development (Trial)" and "Clinical Pharmacology Research on Pediatric Drugs" "Technical Guiding Principles" and "Technical Guiding Principles for the Development and Evaluation of Drugs for Children Supported by Real World Research (Trial)" and other 4 technical guiding principles
.
At the same time, in order to promote the international level of the guidelines for children’s medication, the Center for Drug Evaluation has fully implemented "S11: Non-clinical Safety Evaluation to Support the Development of Pediatric Drugs" and "E11 (R1): Drugs for Medical Products for Pediatrics" There are 2 ICH guidelines in Clinical Research
.

 

　　This year, the Center for Drug Evaluation also published the "Technical Guidelines for the Clinical Trials of Modified New Drugs for Children's Chemicals", "Technical Guidelines for the Writing of Children's Drug-related Information in the Instructions for Chemicals and Biological Products for Treatment", and "Clinical Trials of Attention Deficit Hyperactivity Disorder Drugs" A total of 4 technical guidelines are included in the annual plan
.
It is expected that by the end of this year, a total of 13 guidelines for children's medication will be issued
.

 

　　In addition, in order to standardize the standardization and supplementation of children's drug information in the labeling of listed drugs, the drug review center makes full use of the data resources of children's medical institutions, adopts real-world research methods, follows review technical standards, organizes expert demonstrations, and publicly solicits opinions.
The review screened out two batches of species lists and specific revisions for the proposed revision of the specification
.
At present, the first batch of revised lists has been released, and the second and third batches will also be released to the public
.

　　The issue of medicine for children is a livelihood issue that the party and the state attach great importance to.
General Secretary Xi Jinping has issued important instructions on this issue on August 25th
.
Recently, a reporter from the "Pharmaceutical Economic News" learned from the National Medical Products Administration that in recent years, in order to solve the problem of difficult drug development for children, the drug review and approval agency has launched a series of encouraging research and development innovation, improving review efficiency, and improving work >
.

 

　　During the interview, the reporter learned that the drug risporan oral solution for the treatment of rare diseases was launched in the first half of 2021 and was approved for the treatment of spinal muscular atrophy (SMA) in patients aged 2 months and older
.
According to Xie Songmei, the deputy director of the Second Clinical Department of the Drug Approval Center, in order to promote the early listing of risporan oral solution in China, it will provide Chinese patients with a clear curative effect, and it is safer than the only existing intraspinal intrathecal injection.
Convenient new mechanism for oral treatment options, the Drug Approval Center has conducted multiple rounds of communication with the applicant companies
.

 

　　She said: “Through early communication in clinical research and development, we recommend that the original research company conduct clinical trials in China as soon as possible to provide data support for scientific decision-making in drug review
.
Companies adopt our suggestions to carry out two key phase II clinical trials on a global scale.
A certain proportion of Chinese subjects were included in the trials
.
In the end, based on the data of global and Chinese clinical trials, we made recommendations for approval of listing in China on the basis of ensuring that the benefits of patients’ medications outweigh the risks
.
This is A case where China has joined the global early simultaneous research and development based on regulatory encouragement, and promoted the simultaneous registration and listing of new mechanism drugs in China and the world, so that Chinese patients with rare diseases can get valuable new drugs as soon as possible
.
"

 

　　Publish a list of encouraged R&D

 

　　The development and production of medicines for children is a worldwide technical problem, which is closely related to the pathophysiological characteristics of children and their tolerance to the safety risks of medicines
.

 

　　Yang Zhimin, director of the Department of Clinical Medicine of the Center for Drug Appraisal, pointed out: "To promote the solution of children's drug use problems, regulatory agencies, clinical institutions and drug manufacturers need to work together in the same direction
.
"

 

　　In recent years, with the support of major national science and technology projects, a number of children's medical institutions in China have established a relatively complete research and development technology platform for children's drugs, which provides a strong guarantee for the innovative research and development of children's drugs in China
.
Yang Zhimin explained that, as the main body of research and development, enterprises are the main force to solve the problem of children's medicine in China.
The drug review department maintains good communication with research and development enterprises, and gives specific guidance on the actual difficulties in the enterprise's research and development process, which is crucial to speeding up the process of children's medicine research and development.
Important
.

 

　　In order to implement Document No.
44 of the State Council and the newly revised "Drug Administration Law", the Center for Drug Evaluation will give priority review and approval to children's drugs that are urgently needed clinically and have obvious clinical advantages
.

 

　　 "The review of children-first and urgently-needed varieties has its own particularities.
It requires tacit cooperation and efficient interaction in various links such as professional review, business management, verification, inspection, and scientific comprehensive decision-making
.
" Xie Songmei introduced that the current drug review center has opened children's drugs.
The green channel of review adheres to patient-centered, specific analysis based on clinical urgent needs and specific types of research data, and continuously rationalizes the priority processing procedures and requirements, and achieves high standards, strict requirements and strong services
.

 

　　In order to improve the efficiency of variety review, minimize review time, and accelerate the launch of children's drugs, the Center for Drug Evaluation has also set up a special label for "Children's Drugs", which will be connected by a project manager to supervise the review progress and ensure progress in time
.
At the same time, strengthen early intervention and proactively serve in the communication, acceptance, review and other links of the enterprise
.

 

　　Data shows that among the three batches of clinically urgently needed overseas new drug catalogues released by the National Health and Construction Commission, 16 new drugs that are urgently needed for clinical use in children have been approved for marketing
.
From 2016 drug trial center will be the same National Health Health Commission, the State Ministry of Industry issued three batches of medicines for children are encouraged to develop a list of declarations, the first involving the list of 32 varieties, the second batch of the list involves 39 varieties, the third installment of the list Involving 34 varieties, promote the research and development, innovation and application review of children's suitable varieties, dosage forms and specifications
.

 

　　As of June 30, 2021, 14 varieties on the list have completed technical review and been approved for marketing, 9 varieties on the list are under review for marketing applications, and 5 varieties on the list have been approved for clinical trials
.

 

　　Improve the review standard system

 

　　 " Children's drug development is different from general drugs.
We can start from several aspects at the moment
.
" Yang Zhimin suggested that first, children's drug development needs to be based on clinical urgent needs, mainly in the areas of common and frequently-occurring diseases, and the drug should be strictly defined.
Dosage measurement standards; Second, the development of children's drugs can be combined with the actual clinical situation.
Many drugs have been used in clinical practice for many years and have been accepted by clinical guidelines and expert consensus.
These drugs can supplement children's indications by exploring real-world research data; Third, the most difficult part is the research and development of rare disease drugs, and the research and development of these drugs should involve more international cooperation
.

 

　　The formulation of technical guidelines is an important supervision method for regulating drug research and development and improving the quality and efficiency of drug research
.
Accelerating the improvement of the evaluation standard system and evaluation methods for children's medications can effectively support and guide scientific and orderly research and development, improve the quality of declarations, enhance the vitality of children's medications, and provide important technical support and evaluation for standardizing the development and evaluation of children's medications in China Basis
.

 

　　It is understood that before 2020, the Center for Drug Evaluation only issued three specific guidelines for children's medication
.
After 2020, the Center for Drug Evaluation has increased its ability to build technical guidelines for children's medications, and in accordance with the principle of emergency use first, successively released the "Guidelines for the Development of Children's Drugs (Chemicals) Pharmacy Development (Trial)" and "Clinical Pharmacology Research on Pediatric Drugs" "Technical Guiding Principles" and "Technical Guiding Principles for the Development and Evaluation of Drugs for Children Supported by Real World Research (Trial)" and other 4 technical guiding principles
.
At the same time, in order to promote the international level of the guidelines for children’s medication, the Center for Drug Evaluation has fully implemented "S11: Non-clinical Safety Evaluation to Support the Development of Pediatric Drugs" and "E11 (R1): Drugs for Medical Products for Pediatrics" There are 2 ICH guidelines in Clinical Research
.

 

　　This year, the Center for Drug Evaluation also published the "Technical Guidelines for the Clinical Trials of Modified New Drugs for Children's Chemicals", "Technical Guidelines for the Writing of Children's Drug-related Information in the Instructions for Chemicals and Biological Products for Treatment", and "Clinical Trials of Attention Deficit Hyperactivity Disorder Drugs" A total of 4 technical guidelines are included in the annual plan
.
It is expected that by the end of this year, a total of 13 guidelines for children's medication will be issued
.

 

　　In addition, in order to standardize the standardization and supplementation of children's drug information in the labeling of listed drugs, the drug review center makes full use of the data resources of children's medical institutions, adopts real-world research methods, follows review technical standards, organizes expert demonstrations, and publicly solicits opinions.
The review screened out two batches of species lists and specific revisions for the proposed revision of the specification
.
At present, the first batch of revised lists has been released, and the second and third batches will also be released to the public
.