Economics of stratified medicine: Challenges and opportunities in adapting stratified medicine approaches to enhance patient access, population health, cost-effectiveness and return on investment

Stratified medicine (SM) is an innovative treatment concept which has drawn major attention in the pharmaceutical research community due to improved diagnostic technologies and a better understanding of diseases´ heterogeneity. The ability to target patient subpopulations has the potential to improve treatment efficacy and minimize side effects, enabling physician to more selectively deploy therapeutics in those patient groups. Several SM interventions have proven beneficial in a number of cancers and genetic diseases and researchers are working to identify more and more biomarkers that could be used to refine treatments in the future. At the same time, the process of stratification of patients and the implementation of this “disruptive innovation” has raised economic questions for payers, the healthcare system and for the manufacturer. Decision makers at all levels want value for money and require economic evaluations on the opportunity costs before implementing such approaches into clinical practice. This thesis investigates these economic challenges and opportunities in adapting SM approaches to enhance patient access, population health, cost effectiveness and return on investment.