基于FDA“动物法则”外推超常规抗毒药物人用剂量的方法解读和思考

Analysis on extrapolation of“humanized dose”toward extraordinary use new drugs under Animal Rule of FDA

超常规使用药物在应对诸如新发突发烈性传染病、大规模中毒事件等突发公共卫生事件时具有重要的战略意义,但也面临明显的研发难度。针对该类药物的特殊性和难点,该文调研了国际通用的“动物法则”,回顾总结基于“动物法则”批准上市的药品,分析解读基于动物药效数据外推临床药效和给药剂量的原理和关键技术要点,为促进我国超常规使用药物以及应急防控药物研发提供借鉴和思路。

Effective extraordinary use new drugs(EUND)and medical countermeasures(MCM)are of strategic importance under the current situation.Traditional drug development pathway toward approval is not possible for these drugs.International Animal Rule formulated for the drugs when human efficacy studies are not ethical was reviewed in this research.All the FDA-approved MCM and EUND under Animal Rule are systematically summarized.Approaches to the translation of animal efficacy findings to acquire“humanized”dose have been accomplished.The principles and technical points are analyzed.Animal Rule may provide a new registration strategy for domestic MCM and EUND development.