2025.10. 13 本文字数：2498，阅读时长大约5分钟 作者 | 第一财经 吴斯旻 在当前优先审评审批等政策激励下，儿童药物研发已成为市场关注的焦点。 根据国家药监局数据，仅前5个月，我国批准上市57个儿童用药，远超去年同期。但儿童药占药品总 量的比例仍较低，从针对儿童特定疾病的专用药品到适合儿童剂型、规格的常见药品，均存在较大的 未满足需求。 同时，随着含儿童受试者的临床试验和仅在儿童人群中开展的临床试验数量不断增加，如何在鼓励创 新的同时，确保受试者安全和儿童用药安全，日益成为监管关切。 近日，国家药监局药审中心发布《儿童用药研发常见/共性问题及一般性答复》（下称"答复"），对 通过改进剂型、工艺、给药途径等，提升药物的安全性、有效性及儿童用药依从性的改良型新药，提 出更确定的支持信号。 该答复还明确，儿童新药研发需以保护患儿权益为前提，减少不必要的重复性研究，维护儿童受试者 知情权，与此同时，应合理利用真实世界证据及"成人数据外推至儿童"等方法，解决儿童受试者招 募困难的问题，加快药物研发。 儿童用药90%以上并非儿童专用药 根据答复，辅料选择需遵循安全性原则，同时还需遵循必要性原则。"在降低风 ...

在当前优先审评审批等政策激励下，儿童药物研发已成为市场关注的焦点。 根据国家药监局数据，仅前5个月，我国批准上市57个儿童用药，远超去年同期。但儿童药占药品总量 的比例仍较低，从针对儿童特定疾病的专用药品到适合儿童剂型、规格的常见药品，均存在较大的未满 足需求。 同时，随着含儿童受试者的临床试验和仅在儿童人群中开展的临床试验数量不断增加，如何在鼓励创新 的同时，确保受试者安全和儿童用药安全，日益成为监管关切。 近日，国家药监局药审中心发布《儿童用药研发常见/共性问题及一般性答复》（下称"答复"），对通 过改进剂型、工艺、给药途径等，提升药物的安全性、有效性及儿童用药依从性的改良型新药，提出更 确定的支持信号。 该答复还明确，儿童新药研发需以保护患儿权益为前提，减少不必要的重复性研究，维护儿童受试者知 情权，与此同时，应合理利用真实世界证据及"成人数据外推至儿童"等方法，解决儿童受试者招募困难 的问题，加快药物研发。 儿童用药90%以上并非儿童专用药 "在成人应用中可能不被认为有临床价值的改良（如片剂改为口服溶液），在低龄儿童中可能被视为具 有临床价值甚至临床优势。"答复提到。 儿童药品是指14岁及以下的未成年 ...

Group 1: Industry Overview - The production of pediatric medications has historically faced challenges, including a lack of manufacturers, limited varieties, and insufficient suitable formulations, referred to as the "three shortages" [1] - Recent government policies have been introduced to encourage and support the research and production of pediatric medications [1][9] - The approval of pediatric medications has been increasing, with 106 new pediatric drug varieties approved in 2024, along with 35 additional varieties expanding pediatric indications [9] Group 2: Challenges in Pediatric Medication - There are significant challenges in pediatric drug development, including difficulties in recruiting child patients for clinical trials and determining appropriate dosages and administration methods [8][10] - The issue of "disease waiting for drugs" and the prevalence of off-label use of adult medications for children pose risks to pediatric medication safety [8][10] Group 3: Importance of Education and Awareness - There is a need for enhanced public awareness and education regarding children's growth and development, as many parents still hold misconceptions that prevent timely medical consultations [2][3][4] - Strengthening educational efforts among healthcare professionals and the public is crucial for improving the diagnosis and treatment of growth-related issues in children [3][4][7] Group 4: Innovations in Pediatric Medication - Companies are increasingly focusing on the pediatric growth and development sector, with innovations such as long-acting growth hormones that reduce the frequency of administration from daily to weekly [5][6] - The introduction of user-friendly devices, such as smart electronic injection pens, aims to improve the safety and comfort of pediatric patients during treatment [6] Group 5: Future Directions - There is a call for further research into various causes of growth disorders and the development of specific treatment plans for rare syndromes and genetic defects [7] - Collaboration between enterprises and public organizations is essential to enhance the accessibility and precision of pediatric medications, ensuring that children receive timely and appropriate treatment [10]