Abstract

Background:

Gene therapies are an innovative strategy for treating genetic disorders; however, they are still emerging on the market access and in the healthcare system. Significant differences can be seen during HTAs carried out in various countries since Health technology assessment (HTA) agencies have not yet elaborated any standardized approach for assessing gene therapies. We focused on submitted economic models of gene therapies approved for use by the US FDA and EMA with the aim to provide a comprehensive summary of how selected HTA bodies assessed the cost-effectiveness of gene therapies.