Aronsson M, Dunton K, Hawe E, Wolowacz S, Quoraishi S, Durrington P, Ray KK, Bilitou A. Building a cost-effectiveness MODEL framework from a payer's perspective to assess NOVEL treatments in primary hypercholesterolaemia and MIXED dyslipidaemia: challenges in a world of LDL-C lowering. Poster to be given at the Virtual ISPOR Europe 2020; November 2020. [abstract] Value Health. 2020 Dec; 23(S2).

OBJECTIVES: To develop a robust and clinically relevant economic model to assess cost-effectiveness of a novel oral treatment in LDL-C lowering in a universe of generic statins and ezetimibe and restricted-access PCSK9 inhibitors.

METHODS: A critical review of existing economic models, prior HTA and cost-effectiveness analyses in primary hypercholesterolaemia or mixed dyslipidaemia was conducted. Clinical expertise was sought and clinical guidelines were reviewed across Europe. Efficacy data (LDL-C reduction at 12 weeks) for bempedoic acid and relevant comparators were used via a network meta-analysis based on a clinical SLR. In the absence of CV outcome data for bempedoic acid, LDL-C reduction was used to estimate CV risk reduction based on the well-recognized and established relationship (Lancet 2015; 385: 1397–405). Baseline CV risks and transition probabilities were obtained from real-world data. Cost and QOL inputs were collected by public databases and literature.

RESULTS: A Markov model with a lifetime time horizon and 1-year cycle length was developed to assess the cost-effectiveness of bempedoic acid (as single-agent pill or in fixed-dose combination with ezetimibe) versus ezetimibe, alirocumab and evolocumab. The model allowed evaluation of patients with different baseline LDL C levels, HeFH, diabetes and recurrent CV events. The model included five core CV model states: myocardial infraction, unstable angina, stable angina, ischaemic stroke, and transient ischaemic attack; prior- and post-event health states were included. Revascularisations were modelled as events which may occur in any health state. The Markov model allowed utilities and costs for multiple events to be modelled with suffcient flexibility.

CONCLUSIONS: Despite heterogeneity across studies in terms of patient characteristics and background lipid lowering therapies making indirect comparisons with other therapies challenging, external validation indicated that the model structure was conceptually appropriate and adequately captured the expected dyslipidaemia treatment pathway and outcomes for decision-making for use of bempedoic acid.

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