Abstract
Gene therapies (GTs) often lack comparators in their pivotal clinical trials when they first seek market access, especially when targeting rare diseases where only symptomatic treatment exists. Furthermore, rare diseases represent a challenge in terms of patient population and clinical trial sample size. Therefore, GT manufacturers resort to single-arm trials and develop indirect treatment comparisons (ITCs) for health technology assessment (HTA). Our aim is to understand HTA bodies’ evidence requirements for ITCs for GTs, and identify key drivers and limitations.
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