Analytic Considerations in Applying a General Economic Evaluation Reference Case to Gene Therapy

• Published in: Value in health Vol. 22; no. 6; pp. 661 - 668
• Main Authors: Drummond, Michael F., Neumann, Peter J., Sullivan, Sean D., Fricke, Frank-Ulrich, Tunis, Sean, Dabbous, Omar, Toumi, Mondher
• Format: Journal Article
• Language: English
• Published: United States Elsevier Inc 01.06.2019; Elsevier Science Ltd
• Subjects: Cost analysis; cost-effectiveness analysis; Decision makers; drug pricing; Gene therapy; Health care; Internal Medicine; Methods; reimbursement; specialty drugs; United States > US; specialty drugs; reimbursement; cost-effectiveness analysis; drug pricing
• ISSN: 1098-3015; 1524-4733; 1524-4733
• DOI: 10.1016/j.jval.2019.03.012

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 healthcare interventions. In the case of gene therapy, there is a difference of opinion on whether a specific methodological reference case is required. The aim of this article was 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. •Gene therapy is a novel approach that uses specific genetic material to treat or prevent disease. It has been argued that these therapies have special characteristics that pose challenges for economic evaluation and may require changes in methods.•The article provides a detailed analysis of the special characteristics of gene therapy, discusses what changes in methods may be necessary, and outlines the particular factors to which analysts and decision makers should pay particular attention in the economic evaluation of these therapies.•The guidance given should help decision makers undertake a more informed assessment of the costs and benefits of gene therapies.