在研究歷年來對醫(yī)療機構(gòu)中藥制劑監(jiān)管要求的基礎(chǔ)上，提煉出將醫(yī)療機構(gòu)中藥制劑轉(zhuǎn)化為中藥新藥的現(xiàn)存問題，并結(jié)合人用經(jīng)驗以及真實世界證據(jù)的應(yīng)用，對來源于醫(yī)療機構(gòu)中藥制劑轉(zhuǎn)化為中藥新藥的有關(guān)問題進行思考，建議減免藥效學(xué)試驗，鼓勵應(yīng)用真實世界證據(jù)替代Ⅱ期臨床試驗，視情況決定是否減免單次及重復(fù)給藥毒性試驗；引導(dǎo)業(yè)界制定可量化的符合中藥特點的診斷標準和療效評價標準，從而認可中藥對癥狀的治療優(yōu)勢；推進真實世界證據(jù)的應(yīng)用，特別是針對兒童用藥的開發(fā)難點進行應(yīng)用設(shè)計，為兒童用藥的研發(fā)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

國家自然科學(xué)基金資助項目（81503347，81503068）