Advancing pediatric drug development in China: implementation of the national priority R&D encouragement list

doi:10.5246/jcps.2025.03.020

Abstract

Abstract:

Access to medicines for children is a critical global issue that demands urgent attention. In China, the need is particularly acute due to its large pediatric population and corresponding clinical demand. This study aimed to analyze the characteristics of drugs included in the Encouraging R&D and Declaring the Children Medicine Lists issued by the Chinese government from 2016 to 2023, and to assess the accessibility and challenges associated with these priority medicines. Data were collected from the official websites of the Health, Industry, and Drug Regulatory Departments of the People’s Republic of China. By organizing information on 129 drugs included in all the lists, a systematic analysis was conducted to evaluate the effectiveness of China’s pediatric medication policies. The 129 listed medications featured a variety of child-friendly formulations, including injections (28%), solutions (20%), suspensions (15%), topical preparations (6%), sprays (3%), and other forms. These drugs primarily target common childhood illnesses, such as diseases affecting the nervous system, cardiovascular system, tumors, infections, digestive system, and endocrine system, which collectively account for 73% of the total. In contrast, treatments for rare diseases represented only 9% of the listed medications. Among these drugs, 22 (17%) were previously unavailable on the market but have since been approved for sale, with 19 (86%) of them included in national or local reimbursement drug lists. However, half of the drugs (64, 50%) have yet to be submitted for any investigational new drug applications, indicating areas where progress remains slow. While the policy has successfully incentivized innovation in pediatric drug development, leading to the introduction of novel therapies and new dosage forms for rare diseases and unmet medical needs in children, significant challenges persist. The findings highlighted ongoing gaps in pediatric drug development and accessibility. Continuous monitoring and evaluation are crucial to sustaining progress, addressing obstacles, and ensuring equitable access to essential pediatric medicines.

Key words: Children, Policy implementation, Investigational new drug, New drug application, Rare diseases

Supporting:

Weimiao Li, Jing Chen, Luwen Shi. Advancing pediatric drug development in China: implementation of the national priority R&D encouragement list[J]. Journal of Chinese Pharmaceutical Sciences, 2025, 34(3): 260-268.

Figures/Tables 6

Table 1. Regulations for pediatric drugs issued in China in 2011.

Table 2. Approval status for four lists of drugs.

Table 3. Details of approved drugs.

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