Health-economic evaluation of precision medicine in cancer care – literature review and analysis of methodological considerations

Precision medicine is often cited for its expected potential to revolutionise health care. The rapid evolution of diagnostics, treatments, and methods to deliver care based on the individual’s unique characteristics promises to increase health gains. These advances also bring new challenges on how to determine whether new and innovative methods are cost-effective in relation to the current standard of health care delivery. Cost-effectiveness information of new medicines is required in many countries across Europe and globally to be included on lists of accepted therapies. The reimbursement decisions then rely in part on the information on costs in relation to health outcomes provided from a cost-effectiveness analysis but also other aspects including equity, affordability, and health care infrastructure. This report reviews challenges to standard methods for cost-effectiveness analyses raised in health-economic and clinical oncology literature. It also discusses further considerations and challenges when estimating the value of precision medicine interventions. The report concludes that the methods used in health economics are not dependent on the availability of rigorous phase 3 data to be able to assess cost-effectiveness and support decision-makers by sorting out the consequences of uncertainty in the evidence. Indeed, cost-effectiveness analysis and other economic evaluation methods have several tools to deal with uncertainty about effect sizes such as sensitivity analysis and scenario analyses. In that sense, the challenges of precision medicine in terms of greater statistical uncertainty of results can be dealt with using already available tools.