在研究历年来对医疗机构中药制剂监管要求的基础上，提炼出将医疗机构中药制剂转化为中药新药的现存问题，并结合人用经验以及真实世界证据的应用，对来源于医疗机构中药制剂转化为中药新药的有关问题进行思考，建议减免药效学试验，鼓励应用真实世界证据替代Ⅱ期临床试验，视情况决定是否减免单次及重复给药毒性试验；引导业界制定可量化的符合中药特点的诊断标准和疗效评价标准，从而认可中药对症状的治疗优势；推进真实世界证据的应用，特别是针对儿童用药的开发难点进行应用设计，为儿童用药的研发助力。

On the basis of studying the regulatory requirements for traditional Chinese medicine preparations in medical institutions over the years, the existing problems of transforming traditional Chinese medicine preparations in medical institutions into new Chinese medicines have been refined. Combined with human experience and the application of real-world evidence, consider the relevant issues of the transformation of traditional Chinese medicine preparations from medical institutions into new Chinese medicines. It is recommended to reduce or exempt pharmacodynamic trials, encourage the use of real-world evidence instead of phase II clinical trials, and decide whether to reduce or exempt single and repeated dose toxicity tests as appropriate. Guide the industry to formulate quantifiable diagnostic criteria and efficacy evaluation criteria that meet the characteristics of traditional Chinese medicine, thereby recognizing the advantages of traditional Chinese medicine in treating symptoms. Promote the application of real-world evidence, especially the application design for the development of children's medicines, and help the development of children's medicines.

R288.1;R283.6

国家自然科学基金资助项目（81503347，81503068）