The State Food and Drug Administration took multiple measures to encourage innovation, and the review and approval of children's drugs continued to maintain a growth momentum

At present, the "Regulations on the Implementation of the Drug Administration Law of the People's Republic of China (Draft for Comments)" organized by the State Food and Drug Administration is being posted on the Internet for public comments, which proposes "the first approved new varieties, dosage forms and specifications for childr.

The consultation draft provides substantial incentives for companies to develop children's medicines, but it is not the only incentive poli.

It is reported that as of April 30, 2022, the Center for Drug Evaluation of the State Food and Drug Administration has completed 30 technical review tasks for children's drug use, a total of 21 varieties, including 8 varieties for priority review and approval and 3 "Encouraging R&D applications for children" Drug List" varieti.

Generally, children's medicines refer to a class of medicines suitable for minors under the age of 1 Due to the special physiological functions of children, especially the hypoplasia of liver, kidney, nerve and endocrine functions, they have special laws in drug absorption, distribution, metabolism, e.

With the improvement of the economic level and the liberalization of the national fertility policy, the number of children has continued to increase, and the demand for children's medicines has also continued to increa.

Specifically, first, from the perspective of new drug research and development, due to the particularity of the pediatric population, there are difficulties in formulation design, specification selection, design of clinical trials for safety evaluation, e.

Previously, the "Annual Report on the Status of Clinical Trials of New Drug Registration in China (2020)" issued by the Center for Drug Evaluation of the State Food and Drug Administration showed that there were 129 clinical trials on children subjects, accounting for 8% of the total number of registered drug clinical trials that ye.

"The recruitment of pediatric drug subjects is difficult and the safety risk is high, which leads to a long clinical trial period, high R&D risks, and high cos.

In the above report, among the 129 registered pediatric clinical trials, according to the drug classification analysis, biological products have the largest number of registrations, 70, followed by chemical drugs and traditional Chinese medicines; For preventive vaccines, dermatology and ENT, e.

According to reports, in the field of children's medicine, there are still too many unmet clinical nee.

In 2011, China began to explicitly encourage the development and production of special medicines for children and improve the catalogue of medicines for childr.

In 2016, the Center for Drug Evaluation issued the "Announcement on the Basic Principles of Variety Evaluation for Priority Review and Approval of Clinical Urgently Needed Pediatric Drug Applications and the First Batch of Priority Review Varieties", and the first batch of 5 pediatric drug registration applications received priority review and approv.

In 2019, the newly revised Drug Administration Law clearly encourages the development and innovation of children's medicines, supports the development of new varieties, dosage forms and specifications of children's medicines that meet the physiological characteristics of children, and gives priority review and approval to children's medicin.

The newly revised "Administrative Measures for Drug Registration" in 2020 established a priority review channel in the accelerated marketing registration process, and included children's drugs into the priority review and approval proce.

Data source: 2021 annual drug review report

So how fast can this priority review be?

According to the Center for Drug Evaluation, compared with the general application route, the priority review and approval process is shortened to 130 working days, and the review time limit for overseas new drugs urgently needed in clinical practice is 70 working da.

In recent years, China has accelerated the approval of 16 pediatric medicines, including Nosinagen Sodium Injection for the treatment of 5q Spinal Muscular Atrophy, and Agalsidase α Injection Concentrated Solution for the treatment of Fabry disea.

Data source: 2021 annual drug review report

In response to the pain points and difficulties in the research and development of children's medicines by enterprises, the State Food and Drug Administration has strengthened research, prescribe the right medicines, conducted in-depth investigations on the applicable specifications and lack of dosage forms for children's medicines, and established the extrapolation of adult medicines data in accordance with the principle of "emergency first" and combined with clinical practi.

Evidence system for pediatric drug R&D review including guiding principles such as real-world data suppo.

By the end of 2021, a total of 12 special guidelines for children's medicine, including the "Guidelines for the Pharmaceutical Development of Children's Medicines (Chemicals) (Trial)", have been issued, which have improved the clinical trials and safety evaluation standards for children's medicines, and provided important information for research and development and revi.

The technical support and review basis have stimulated the R&D vitality of the enterpri.

In 2022, four special guidelines for children's drug use have been included in the annual work pl.

In order to speed up the entry of overseas marketed drugs into China and meet China's clinical needs, the State Food and Drug Administration, in conjunction with the National Health Commission, organized experts to select and publish a list of overseas new drug varieties urgently needed for clinical u.

A special channel was established for the review of the declared varieties, and a total of three batches of overseas new drug varieties in urgent clinical need were releas.

In addition, in order to effectively understand the clinical needs of children's medicines and strengthen the support for the research and development of enterprises, the State Food and Drug Administration has strengthened the cooperation with relevant departments, the National Children's Medical Center and the industry in the field of children's medicine research and development, and convened policies and procedures related to children's medicine research and developme.

Technical Guiding Principles Seminar, as a guiding unit, dispatched staff to participate in the relevant seminars of the China Children's Drug Research and Industrialization Alliance, and went to the National Children's Medical Center to conduct field research to further meet clinical needs, provide guidance for the actual difficulties in enterprise research and development, and establish solutions to children The collaborative mechanism of drug research and development will jointly promote the innovative research and development of children's dru.

In response to the problem of off-label use of drugs in pediatric patients reported by clinical institutions, the Drug Evaluation Center of the State Food and Drug Administration signed a strategic cooperation agreement with the National Children's Medical Center to set up the "China Children's Instructions Standardization Project" to standardize the information of children's medication in the package inserts of marketed dru.

Supplementary wo.

The State Food and Drug Administration has innovated the means of supervision, guided by the clinical needs of paediatrics, adopted cutting-edge research methods on the basis of sufficient demonstration, based on the actual clinical drug data of paediatrics, and made rational use of real-world evidence on the premise of abiding by science and ensuring safe.

Add children's medication information to the package inserts of listed drugs to guide clinical rational and safe medicati.

Under the incentives of multiple policies, in 2021, a total of 47 children's medicines have been approved for marketing in China, including pediatric inhalation preparations, rotavirus vaccines, Chinese medicine granules and other important varieties and dosage forms, and the application and approval of children's medicines The number is showing a steady upward tre.