Some Thoughts on Precision Medicine

[摘要] In clinical trials, a typical approach for evaluation of safety andefficacy of a test treatment under investigation is to first test for thenull hypothesis of no treatment difference in efficacy based on clinicaldata collected under a valid trial design. If significant, the investigatorwould reject the null hypothesis of no treatment difference and thenconclude the alternative hypothesis that there is a difference in favor ofthe test treatment. If there is a sufficient power for correctly detecting aclinically meaningful difference (improvement) when such a differencetruly exists, we claim that the test treatment is efficacious. The testtreatment will then be reviewed and approved by the regulatory agencysuch as the United States Food and Drug Administration (FDA) ifthe test treatment is well tolerated and there appears to be no safetyconcerns. We will refer to medicine developed based on this typicalapproach as traditional medicine.