儿科中药新药临床研究安全性考虑

Safety evaluation of traditional Chinese medicine in pediatric populations

由于在儿童人群中开展临床研究的特殊性,目前各监管机构均致力于利用已知数据外推至儿科人群,以减少不必要的临床研究。但外推的原则目前仅限于有效性数据,安全性数据并不适用。中药由于其独特的理论体系、复杂的药物成分及多数情况下不明确的量效关系,使得开展儿科人群临床研究的风险评估需要特殊的考量,对儿科中药品种的安全性评价需贯穿儿科药物研发的始终。本文从临床审评角度,对儿科中药新药研发从立项之初到临床研究及上市后的全生命周期中,如何充分利用已知信息尽可能地识别潜在的安全性风险,有针对性地进行观察、评价和监测的相关考虑进行了详细阐述;并对当前监管环境下如何权衡儿童临床研究的风险和获益,以推动儿科中药的研发提出了思考,以期为儿科中药研发提供参考。

Along with the development of pediatric drugs,drug administration agencies are commit⁃ted to extrapolating known data to the pediatric population in order to reduce unnecessary clinical studies.However,the principle of extrapolation is limited to efficacy,and safety analysis currently does not apply.Because of its unique theoretical system,complex drug ingredients and unclear dose-response relationship in most cases,traditional Chinese medicine(TCM)requires special consideration in the risk assessment of pediatric clinical studies.Safety assessment of pediatric varieties of traditional Chinese medicine should run through the development of pediatric drugs.From the perspective of clinical review,this paper was intended to elaborate how to make full use of the known information to identify potential safety risks,observe the right things and to evaluate and monitor safety issues in the whole life cycle of drug development for pediatric TCM products.Furthermore,some suggestions were raised on how to balance the risks and benefits of clinical research for children in the current regulatory environment in order to promote the research and development of TCM products for pediatric populations.