关于精准医学时代转变药物监管技术体系的思考

Considerations on the Transformation of Drug Regulatory Technology System in the Era of Precision Medicine

随着基因组学与蛋白质组学研究的进展,人类对生命和疾病有了更加深刻的认识,推动医学进入到精准医学时代。为少数患者研制的"孤儿药"和为单一患者制备的"定制药"开始显现,在这种情况下新药审批和监管部门遇到了前所未有的挑战,是坚持工业化时代形成的新药研发审批和监管体系,还是根据新形势的要求制定新的指导原则?这对"孤儿药"和"定制药"的发展,乃至精准医学的发展具有重要的意义。

With the development of genomics and proteomics research, people have a deeper understanding of life and diseases, which promotes medicine into the era of precision medicine. For a tiny part of patients developed “orphan drug” and preparation for single patient “customized pharmaceutical” began to emerge, in this case the new drug approval and regulatory encountered unprecedented challenges: we should stick to the system of approval and supervision for research and development of new drugs established in the industrial era, or formulate new guideline according to the requirements of the new situation?which is of great significance for the “orphan drug” and the development of the “customized drug”, and even the precision medicine.