OBJECTIVES: To estimate Alirocumab´s clinical and budget impact, compared with standard lipid modifying therapy, for the private and social security health systems, covering >50% of the population. Alirocumab, a PCSK-9 inhibitor, produces a low-density lipoprotein cholesterol (LDLc) reduction up to 60% and is associated with a reduction in major adverse cardiovascular events and total mortality. Heterozygous Familial Hypercholesterolemia (HeFH) has an estimated prevalence of 1/500 and a detection rate below 1%.

METHODS: An Excel-based model was used to assess the budgetary impact of Alirocumab in Argentina for a 5-year horizon. We performed a systematic review and meta-analysis of both abstracts (from regional and national scientific sessions) and published papers reporting epidemiological, clinical and resource use data. Efficacy and effectiveness were obtained from published trials and meta-analysis. The model analysed population between 18 and 79 years old with HeFH (primary and secondary prevention). LDLc thresholds for Alirocumab’s use were LDLc ≥ 160 mg/dl and ≥ 100 mg/dl for primary and secondary prevention, according to local guidelines. Cost and prices were expressed in 2017 US Dollars. Estimated annual cost for Alirocumab is $6,651.3 USD.

RESULTS: Approximately 51,022 patients could have HeFH. All patients were over the LDLc threshold for the use of Alirocumab. Assuming all detected cases were treated, Alirocumab avoided 246 cardiovascular events. Overall budget impact of Alirocumab was $509,093 USD, $1,019,173 USD, $1,218,066 USD, $1,322,359 USD, and $1,330,442 USD in years 1, 2, 3, 4, and 5, respectively, representing a $0.03 USD per month per member covered increase over 5-year.

CONCLUSIONS: Alirocumab for the treatment of HeFH could avoid CV events in Argentina. Overall budget impact seems to be marginal as it only implies a $0.03 USD increase per each member covered.