Modelling Considerations for Evaluating the Cost-Effectiveness of Biologics in Eosinophilic-Associated Diseases

OBJECTIVES: To identify and summarise challenges in designing conceptual models and modelling approaches for currently available and emerging biologics in eosinophilic-associated diseases (EADs) and to provide recommendations on economic modelling and evidence generation.

METHODS: A targeted search of the Embase database and the websites of health technology assessment (HTA) agencies was performed to identify cost-effectiveness models for treatments in chronic rhinitis with nasal polyps (CRSwNP), eosinophilic granulomatosis with polyangiitis, hypereosinophilic syndrome, and eosinophilic esophagitis (EoE).

RESULTS: A total of 13 studies from the Embase database (11 studies for surgical treatments, nasal budesonide and dupilumab in CRSwNP; 2 studies for budesonide in EoE) and 5 HTA submissions (4 submissions for budesonide in EoE and 1 submission for mepolizumab in CRSwNP) were eligible for inclusion. Model structures often include health states defined based on levels of disease activity (controlled vs. uncontrolled disease, remission vs. recurrence/relapse) and treatment response (response vs. no response). However, ambiguous and vague definitions of disease control in the clinical practice and a lack of consensus on biologics’ treatment response pose a challenge in evaluating the comparative treatment efficacy. Other challenges include the uncertainty in the long-term extrapolation of the treatment efficacy/response for biologics, the difficulty in implementing appropriate treatment stopping rules for biologics, the lack of publications reporting cost and utility estimates stratified by levels of disease control/treatment response, and the difficulty in quantifying the costs and disutility of long-term steroids use.

CONCLUSIONS: Given the challenges in the economic modelling for biologics, early engagement with clinical experts and payers is recommended for reaching a consensus on the positioning of the biologics and the clear definitions of disease control and treatment response. Future research on resource use and utility associated with different levels of disease activity and long-term steroid use is also needed.