中国《鼓励研发申报儿童药品清单》的政策实施效果

doi:10.5246/jcps.2025.03.020

中国药学(英文版) ›› 2025, Vol. 34 ›› Issue (3): 260-268.DOI: 10.5246/jcps.2025.03.020

• 【药事管理与临床药学专栏】 • 上一篇

中国《鼓励研发申报儿童药品清单》的政策实施效果

李惟妙¹, 陈敬¹^,²^,^*(), 史录文¹^,²^,^*()

^1. 北京大学药学院临床药学与药事管理系, 北京 100191
^2. 北京大学国际医药管理中心, 北京 100191

收稿日期:2024-10-05 修回日期:2024-11-20 接受日期:2024-12-19 出版日期:2025-03-31 发布日期:2025-04-01
通讯作者: 陈敬, 史录文

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

Weimiao Li¹, Jing Chen¹^,²^,^*(), Luwen Shi¹^,²^,^*()

¹ Department of Pharmacy Administration and Clinical Pharmacy, School of Pharmaceutical Sciences, Peking University, Beijing 100191, China
² International Research Center for Medicinal Administration, Peking University, Beijing 100191, China

Received:2024-10-05 Revised:2024-11-20 Accepted:2024-12-19 Online:2025-03-31 Published:2025-04-01
Contact: Jing Chen, Luwen Shi
Supported by:
Noncommunicable Chronic Diseases-National Science and Technology Major Program (Grant No. 2023ZD0509600) and Researcher at the Chinese Academy of Sciences Think Tank on the Issues and Countermeasures in the Development of Innovative Drugs in China (Grant No. 2023-ZY06-B-032).

摘要/Abstract

摘要：

儿童用药是全球关注且亟待解决的重要问题。中国儿童基数大, 临床需求迫切。基于中国政府2016–2023年发布的四批《鼓励研发申报儿童药品清单》, 在公开网站中对所有清单包含的129种儿童急需药品的剂型、适应症、批文数、上市情况、纳入医保目录、罕见病用药等情况统计分析, 以评估中国儿童药物激励措施的实施效果。结果显示这129种药品剂型多为儿童友好型, 包括注射剂(28%)、溶液(20%)、悬浮液(15%)、局部制剂(6%)、喷雾剂(3%)等。主要针对常见的儿童疾病, 如神经系统、心血管系统、肿瘤、感染、消化和内分泌系统的疾病, 累计占比73%。此外, 清单中罕见病药品占比9%。政策实施效果方面, 有22种(17%)在清单发布前未在中国上市的药品获得上市销售批准, 其中有19种(86%)已被纳入国家或地方医保药品清单。但至今仍有64种药物(50%)未提交任何新药申请。该政策鼓励了儿童药物研发的创新, 引入了针对罕见病和儿童未满足临床需求的新疗法、新剂量和新配方。但本研究强调了这一领域仍存在的重大差距和挑战, 持续的政策监测和评估对于促进儿童药物的开发和可及性、解决各种问题至关重要。

关键词: 儿童药, 政策实施效果, 新药临床研究申请, 新药上市申请, 罕见病

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:

李惟妙, 陈敬, 史录文. 中国《鼓励研发申报儿童药品清单》的政策实施效果[J]. 中国药学(英文版), 2025, 34(3): 260-268.

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.

图/表 6

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

Figure 1. Formulations of listed drugs.

Figure 2. Therapeutic areas of listed drugs.

Table 2. Approval status for four lists of drugs.

Table 3. Details of approved drugs.

Figure 3. Launch delays of approved drugs (in months).

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中国《鼓励研发申报儿童药品清单》的政策实施效果

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

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