Abstract
AAV-mediated gene therapy (AAVGT) is a novel treatment offering curative effects for several genetic diseases. The high upfront cost of AAVGT and the uncertainty around durability of its effectiveness are posing challenges for health economists. The ISPOR taskforce compiled a list of special considerations, including exploring different discounting rates (DRs). We examined the impact of different DRs when comparing AAVGT to alternative treatments in persons with hemophilia A.
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