Personalised medicine, also known as ‘precision’ or ‘stratified’ medicine, targets treatment by using genetics and/or biomarkers to identify patients or subgroups of patients who have distinct mechanisms of disease or who may respond to treatment in a particular way. The goal is to provide more effective care, which potentially not only benefits the patient but also improves the efficiency of the healthcare system. This comes at the cost of adapting the regulatory environment, so it is fit for purpose, and raising some difficult issues with product pricing.

This more tailored approach is producing important changes in drug development, regulation, and use. Mullard (2020; see Figure 1) suggests the growing importance of personalised medicine. The lighter blue portion of the bars are traditional chemical entities; the darker blue are biologics, and an increasing proportion of those are personalised medicines. By 2019, more than 260 drugs approved by the FDA included genomic information in the official labelling for use (Mehta, 2020).