在研究歷年來對醫(yī)療機(jī)構(gòu)中藥制劑監(jiān)管要求的基礎(chǔ)上，提煉出將醫(yī)療機(jī)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(yàn)以及真實(shí)世界證據(jù)的應(yīng)用，對來源于醫(yī)療機(jī)構(gòu)中藥制劑轉(zhuǎn)化為中藥新藥的有關(guān)問題進(jìn)行思考，建議減免藥效學(xué)試驗(yàn)，鼓勵(lì)應(yīng)用真實(shí)世界證據(jù)替代Ⅱ期臨床試驗(yàn)，視情況決定是否減免單次及重復(fù)給藥毒性試驗(yàn)；引導(dǎo)業(yè)界制定可量化的符合中藥特點(diǎn)的診斷標(biāo)準(zhǔn)和療效評(píng)價(jià)標(biāo)準(zhǔn)，從而認(rèn)可中藥對癥狀的治療優(yōu)勢；推進(jìn)真實(shí)世界證據(jù)的應(yīng)用，特別是針對兒童用藥的開發(fā)難點(diǎn)進(jìn)行應(yīng)用設(shè)計(jì)，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(xiàng)目（81503347，81503068）