我国儿童药研制的制约因素分析及政策建议——基于医药企业的视角

目的：为完善我国儿童药供应保障政策提供依据。方法：基于医药企业视角通过文献综述、问卷调研梳理药品全生命周期中儿童药研制的制约因素，分析其集中和迫切程度。结果：儿童药的主要制约因素包括：儿童临床试验开展难度大；注册审批规则中缺乏对儿童药特殊性的考虑和专门的材料要求；生产激励政策落地缺乏实施细则和措施；市场利益机制尚不完善等。其中，研发和使用支付是问题相对集中的环节。讨论及建议：建议我国开拓并合理使用儿童临床试验资源，多举措增加儿童药研发投入；制定儿童药申报的专项指南，鼓励成人药品注册时提交儿童研究计划；基于典型案例探索儿童药生产供应优化路径；在药品使用与支付规则中给予儿童药更多空间。

Analysis of constraints and policy recommendations for the development of pediatric drugs in China: Based on the perspective of pharmaceutical enterprises

Objective: To provide a basis for improving the design and implementation of policies for ensuring the supply of pediatric drugs in China. Method: Based on the perspective of pharmaceutical enterprises, reviewed literature and conducts questionnaire surveys to identify the constraints in the development of pediatrict drugs throughout the entire drug lifecycle, and analyzes the constraints'' concentration and urgency. Result: The main constraints include: difficulty in conducting clinical trials for children; the current registration and approval rules lack consideration for the specificity of pediatric drugs and specific requirements for application materials; lack of implementation rules and measures in the implementation process of incentive policies for pediatric drug production; The market interest mechanism of pediatric drugs is not yet perfect. Among them, research and development and payment for use are currently relatively concentrated issues. Discussion and suggestions: It is recommended that China fully utilize existing clinical trial data of pediatric and broaden sources, take multiple measures to increase investment in pediatric drug R&D; Develop special guidelines for pediatric drug application and encourage adult drug registration to submit pediatric research plans; Explore the optimization path of pediatrict drug production and supply based on typical cases; Provide more space for pediatric drugs in the rules of drug use and payment.