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

Abstract

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.

Health economic evaluation of gene replacement therapies: methodological issues and recommendations

“…In addition, we identified three recommendation or discussion papers about the economic evaluation of gene therapies [ 6 , 7 , 15 ]. This included an article by Drummond et al, providing a checklist for analysts and decision-makers to determine which aspects of economic evaluation should be considered further, given the unique nature of gene therapy[ 15 ].…”

“…The ability to sit was chosen as one of the primary outcomes in phase 3 clinical trial of Zolgensma® because it is an important goal for patients to achieve, and also because it is expected that this milestone correlates with many other symptoms[ 29 ]. The use of surrogate outcomes makes it difficult to use clinical research results in economic evaluation, especially in rare and understudied conditions where validation of surrogacy is challenging[ 15 ].…”

“…In the context of gene therapy, Drummond et al [ 15 , 37 ] also consider scientific spillovers as a relevant broader element of value. Scientific spillovers relate to the impact of a new technology on future generations of patients.…”

“…Drummond et al [ 15 , 37 ] highlight that in practice, HTA agencies consider those elements of value through a deliberative process, and it would be important to identify all relevant elements when presenting evaluations of GRT. Multiple Criteria Decision Analysis (MCDA) would be another possible approach to include broader elements of value associated with GRT, in a more transparent manner than through deliberation.…”

A Beginner's Guide to Understanding Curative Therapies

“…3 To aid evaluators and address challenges with clinical evidence and social values that may need to be considered, Drummond and colleagues provide a checklist that groups these additional considerations into curative therapy-specific domains. 9 One such factor is spillover effects from cures. Research conducted by Julia Snider and colleagues 10 further describes one such spillover effect-the potential to reduce wait times for liver transplants from current use of antiviral medications and its associated social value.…”