Analytic considerations in applying a general economic evaluation reference case to gene therapy

The concept of a reference case, first proposed by the US Panel on Cost-Effectiveness in Health and Medicine, has been used to specify the required methodological features of economic evaluations of health care interventions. In the case of gene therapy, there is a difference of opinion on whether a specific methodologic reference case is required. The aim of this paper is to provide a more detailed analysis of the characteristics of gene therapy and the extent to which these characteristics warrant modifications to the methods suggested in general reference cases for economic evaluation. We argue that a completely new reference case is not required, but propose a tailored checklist that can be used by analysts and decision-makers to determine which aspects of economic evaluation should be considered further, given the unique nature of gene therapy.