Medicines have made a major contribution to population health over many decades. An ageing population in much of the developed world means that, despite the challenge for publicly funded health systems, maintaining provision of care in a dynamically efficient and effective way is important to avoiding great economic and societal costs. Medicines expenditure is kept efficient by ensuring rational use of cheaper generic medicines on the one hand and scrutinizing value for money of new medicines on the other. A variety of tools exist to help assess the value of medicines, increasingly involving the use of real-world data in various forms. There needs to be continuous improvement in assessment methods – through a multistakeholder effort – in order to keep pace with new science and to correct for any unintended biases.