OBJECTIVES: Homozygous familial hypercholesterolaemia (HoFH) is an ultra-rare and life-threatening disorder caused by defective low-density lipoprotein receptor (LDLR) function, resulting in extremely elevated plasma levels of LDL cholesterol (LDL-C), leading to premature atherosclerosis. Lomitapide, an oral inhibitor of the microsomal triglyceride transfer protein, is an LDLR-independent lipid-lowering therapy which was FDA and EMA approved in 2012 and 2013, respectively.

HoFH patients who do not reach their LDL-C goal on lipid lowering therapies (e.g., statins, ezetimibe, apheresis, PCSK9 inhibitors) are eligible for additional treatment with lomitapide. The objective of this study was to investigate the potential budget impact of introducing evinacumab in adult HoFH patients in England compared to current standard of care (SoC).

METHODS: An Excel-based budget impact analysis with a 5-year time horizon was conducted. The scenario assumed that adult HoFH patients who are LDLR negative, and are non-responders to PCSK9 inhibitors, or patients who do not reach their LDL-C goal, will receive lomitapide. Lomitapide treatment compliance was assumed to be 72%. A new scenario with evinacumab estimated the costs if 5% of lomitapide-eligible patients receive evinacumab each year instead. Costs (2022 £) included disease/drug monitoring, drug acquisition (list price), infusion, and apheresis. All costs were validated by a UK clinical expert with experience in managing HoFH patients.

RESULTS: Based on estimated prevalence rates reported by NHS England, 67 patients have HoFH, and 43 are eligible for lomitapide. The gross budget impact at baseline was £11,538,953 per year. Introduction of evinacumab was estimated to have an additional budget impact of £226,243 in year one, rising to £1,128,525 over 5-years.

CONCLUSIONS: Introduction of a new treatment into NHS England will affect the total costs for treating adults with HoFH compared with current SoC. This should be a consideration when appraising the value of new technologies for the management of HoFH.