Abstract: 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.