Abstract

coreHEM, a core outcome set (COS) for hemophilia gene therapy (GT) was identified by multi-stakeholder consensus (Iorio, Haemophilia, 2018). When consistently measured, the COS will inform a value-based approach to evaluating GT. First steps to determining appropriate measurements for each outcome were previously reported (ISPOR 2018). One outcome, mental health (MH) outlook, was regarded as highly important to capture the potential transformational impact of GT, however no instruments that measured MH when receiving a novel treatment were identified.

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