OBJECTIVES: Hereditary angioedema (HAE) is a rare and potentially life-threatening genetic disease characterised by recurrent swelling attacks affecting various body parts. Treatments aim to reduce morbidity associated with attack manifestations (on-demand treatment, ODT) and to prevent occurrence of recurrent attacks (long-term prophylaxis, LTP). Despite availability of approved ODT and LTP therapies, an unmet need remains for additional efficacious, well-tolerated, and easily administered options.

METHODS: A systematic literature review (SLR) was performed to identify economic evidence and appraise its suitability for incorporation into de novo economic modelling of ODT and LTP for HAE encompassing three economic components: economic evaluations, healthcare cost and resource use (HCRU), and health-related quality of life (HRQoL), including utility studies.

RESULTS: The literature review was conducted in October 2023, resulting in 128 records meeting the PICOS eligibility criteria. Of these, 18 economic evaluations, 11 HRQoL studies, and 3 HCRU studies formed the narrative synthesis. There was a sparsity of economic evaluation evidence, particularly for ODT, where limited details of model structures and data inputs were reported. Evaluations of LTP reported clearer, consistent models accepted by HTA bodies. Utility data were limited across ODT and LTP, and no mapping algorithms were identified. Only two sources were identified reporting HRQoL inputs potentially suitable for cost-effectiveness modelling, although these were limited by small patient numbers and generalisability caveats: a UK-based study including attack location related utilities and a study with HAE health state utilities in Sweden. Two HCRU studies reported estimates for hospitalisations and productivity impacts.

CONCLUSIONS: This review highlights a sparsity of economic evidence in HAE. Economic modelling will require long term assessment of disease-specific HRQoL, including utility and healthcare resource use estimates. This data is critical for both healthcare providers and payers to understand the financial implications of treatment decisions made to improve patient outcomes.