“十三五”规划以来，国家在罕见病领域多方面布局，积极推进罕见病药物临床研发和引进，提高患者临床治疗有效性和可及性，其中一项重要工作就是开展罕见病登记注册系统建设。大部分罕见病患者在出生时或儿童期发病，临床治疗需求集中，因此针对这类患者开展数据库建设意义重大。文章就儿童罕见病数据库建设的必要性和重要性、策略选择以及现阶段遇到的挑战进行总结，旨在为我国儿童罕见病数据库建设探索最佳实践路径。

Since the 13th Five-Year Plan, China has made various arrangements in the field of rare diseases, such as actively promoting the clinical research and introduction of rare disease drugs, improving the effectiveness and accessibility of patient clinical treatment. One important task is to carry out the construction of the rare disease registration system. Most rare disease patients develop symptoms at birth or during childhood, with a concentrated demand for clinical treatment. Therefore, it is of great significance to carry out database construction for such patients. This paper summarizes the necessity and importance of the construction of rare disease database in children, strategy selection and challenges encountered at the present stage, aiming to explore the best practice path for the construction of rare disease database in children in China.