The value of personalised medicine from a health economics perspective

Personalised medicine (PM) aims to use genetic information to better target healthcare to each individual. PM has been met with excitement and anticipation. Indeed, a better tailoring of healthcare may have many positive consequences – such as improved treatment effectiveness, less adverse drug reactions, more diseases prevented – that in turn could lead to better population health and a reduction in wasteful healthcare spending. But there are also concerns about the potentially high costs of PM. Some of the PM treatments that have come onto the market have come with large price tags. And more widespread implementation of genetic testing in clinical practice may be expensive.

This PhD thesis looks at PM from a health economics perspective. We investigate the added value of PM. Have the PM interventions that have become available so far been able to deliver on the high expectations? And are certain types of PM more valuable than others? We also explore the characteristics of PM that may make it more difficult to assess (cost-)effectiveness, and we offer guidance for economic evaluations of PM interventions. Finally, we perform a case study. We investigate the cost-effectiveness of entrectinib. Entrectinib is a cancer treatment that is prescribed for patients with NTRK gene fusions in the tumour DNA and is a so-called “histology-independent” (or “tumour-agnostic”) therapy. This marks an innovative approach to cancer care, as most cancer treatments to date are prescribed based on primary site and histology. The thesis concludes by reflecting on the policy changes that may be needed for a (more) successful implementation of PM.