OBJECTIVES: In health technology assessment (HTA), disease models are used to mathematically represent the course of a disease in a patient cohort over time, including how risk factors of interest impact disease incidence, progression, and mortality. This study identified existing disease models in hereditary angioedema (HAE), a rare, chronic, and potentially life-threatening disorder, and assessed their utility for evaluating the health economic value of future HAE therapies.

METHODS: Literature searches were prespecified and conducted in PubMed and HTA databases to identify pertinent peer-review literature, congress proceedings, and agency assessments on HAE disease models. For each included study, we extracted detailed information on the 1) disease modeling approach, 2) impact of simulated interventions on disease progression, and 3) health economic analysis specifications and outcomes. We assessed reporting quality using the CHEERS II criteria.

RESULTS: We identified 5 publications and 5 HTA reports (6 cost-utility analyses, 3 cost-effectiveness analyses, and 1 cost-minimization analysis). State transition models were most frequently used (5 studies), followed by decision tree models (4 studies), with 1 cost-effectiveness study identified. Among reviewed models, 4 simulated a lifetime horizon, and of those, 2 simulated disease-related mortality. HAE attack characteristics were simulated in half of models reviewed (5 models simulated attack severity, duration, and/or location). However, none of the models reviewed comprehensively simulated all attack-related elements to robustly define the scope of disease progression, burden, and related costs. HTA reports from ICER, NICE, and CADTH demonstrated the highest reporting quality per CHEERS II criteria.

CONCLUSIONS: Current HAE disease models do not fully characterize the clinical disease process or scope of attack burden, including disease-related mortality and attack characteristics. Development of a new, comprehensive HAE disease model is important to capture the holistic burden of disease to support health economic evaluations for novel HAE therapies.