Heterozygous familial hypercholesterolaemia (HeFH) patients who receive standard of care (SOC) therapy yet still have significantly elevated low-density lipoprotein cholesterol (LDL-C) levels qualify for apheresis. Apheresis is an invasive, burdensome and resource intensive (RUR 2,406,252 per patient-year) LDL-C lowering procedure and should be retained for patients where other lipid-lowering therapies (LLTs) are insufficient. This analysis aims to evaluate the ability of evolocumab added to SOC to reduce apheresis use in HeFH patients through LDL-C lowering, and further to evaluate the economic impact associated with reducing apheresis use in accordance with Russian guidelines.
Patient characteristics and efficacy data were taken from the RUTHERFORD-2 phase-3 trial, due to lack of Russian data. The cohort had a baseline LDL-C level ≥160mg/dL with a mean (standard deviation) of 201 (43) mg/dL. The mean percentage reduction in LDL-C by evolocumab was 61.3%. Baseline LDL-C and LDL-C after evolocumab treatment were modeled by assuming log-normal distributions. Patients were deemed eligible for apheresis with LDL-C levels ≥300mg/dL in primary prevention (PP) or ≥200mg/dL in secondary prevention (SP) according to Russian guidelines. The use of apheresis was calculated with and without evolocumab treatment over a lifetime horizon, with costs assigned to LLTs. A previously published decision analytic model was used to track the proportion of the cohort with history of events and to account for mortality.
The model predictions indicate that 3.1% (PP) and 34.6% (SP) of patients would receive apheresis at baseline. Mean survival was increased by 5.6 years, whilst the use of apheresis was reduced by 3.8 patient-years in evolocumab treated patients. Despite increased survival, treatment with evolocumab resulted in a per-patient saving of RUR 957,016 for the overall cost of LLTs.
Evolocumab may lead to a significant reduction in apheresis use according to Russian guidelines and a subsequent savings in apheresis costs.
© 2016 Published by Elsevier Inc.